Looking back a few weeks to ISPOR Europe, Will Battershill and Hollie Wheat, Analysts from Delta Hat – Powered by Petauri™, share their key takeaways from the conference, relating to health technology assessment (HTA) decision-making. Will begins with insights on the hot topic of incorporating patient and carer perspectives, whilst Hollie dives into the use of surrogate endpoint evaluation.

Incorporating societal impacts into HTA decision-making

Will Battershill explores a key them from ISPOR Europe: the benefits of incorporating societal impacts into HTA decision-making.

Expanding HTA perspectives beyond the payer

ISPOR Europe featured some thought-provoking discussions on expanding the perspective taken in HTA beyond that of the payer, which typically focuses on costs to the health system and health-related benefits to patients. Speaker panels at the conference explored how incorporating societal impacts, such as bereavement or productivity, could provide a more holistic picture of the disease burden on individuals and their families. These broader perspectives pose practical and conceptual challenges, however, which could complicate decision-making.

Bereavement disutility debate

One hot topic was whether to include bereavement disutility in HTA models. This refers to the negative impact on the health-related quality of life of those close to a deceased patient. The idea is to address the “carer quality-adjusted life year (QALY) trap”, where extending life for a patient with informal carers is considered less valuable than for a patient without carers. Including bereavement disutility introduces a large negative effect after death, which, due to discounting, could shift cost-effectiveness outcomes.

Challenges in incorporating bereavement effects

Becky Pennington (Senior Research Fellow, University of Sheffield) presented evidence from UK household surveys. She showed that both carers and non-carers experience substantial disutility from bereavement, lasting 1–2 years. Dr Tara Lavelle (Assistant Professor of Medicine, Tufts University School of Medicine) presented research demonstrating increased carer productivity after bereavement over time.

Incorporating bereavement effects is not straightforward, however Dr Saskia Knies (Erasmus School of Health Policy & Management), representing the Dutch HTA body, highlighted challenges, such as determining the appropriate time horizon for models. She also pointed out the need to adjust willingness-to-pay thresholds to match a broader perspective. Pennington’s findings suggest that bereavement effects are similar for carers and non-carers. This raises the question of whether these effects should apply universally in survival-related HTAs, potentially diluting their impact on cost-effectiveness outcomes.

Risk of unintended consequences: Productivity gains versus survival

Lavelle’s work also raised an important issue: if post-bereavement productivity gains outweigh caregiving losses, could this unintentionally favour death over survival in societal cost-effectiveness analyses?

Incorporating broader support structures in HTA: A patient perspective

In an educational symposium on adopting a societal perspective for HTA in breast cancer, a patient representative stressed the importance of recognising broader support structures in patients’ lives. The discussions highlighted the complexity of integrating these factors into HTA frameworks, as there is no clear method to do so alongside clinical outcomes.

Complexities of expanding HTA perspectives

Expanding HTA perspectives beyond payer-centric views has prompted insightful research and facilitates a more comprehensive understanding of disease impact. However, these approaches also revealed marked methodological and ethical complexities. I felt that, without clearer frameworks for implementation, broadening HTA perspective might inadvertently introduce ambiguity rather than actionable insights.

Mtech Access presented a poster that looked at this issue as it relates to orphan disease: The Use of Patient and Carer Perspectives and Real-World Evidence in Orphan Disease Health Technology Assessment Submissions.

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Surrogate endpoint evaluation in HTA decision-making

Hollie Wheat shares her insights from the session on ‘borrowing information from diverse sources to enhance the evidence base for surrogate endpoint evaluation in HTA decision-making’.

Surrogate endpoints: Filling evidence gaps in HTA

One of the most thought-provoking and applicable sessions was on the use of surrogate endpoint evaluation in HTA decision-making, as led by Sylwia Bujkiewicz (Professor of Biostatistics, University of Leicester), with discussants Anastasios Papanikos (Principal Statistician, GSK), Janharpreet Singh (Professor of Biostatistics, University of Leicester), and Georgios Nikolaidis (Associate Director, IQVIA). Surrogate endpoint analyses help fill evidence gaps in HTA, often relevant where there is uncertainty in short-term data, for example in the context of small clinical trial data set and/or limited numbers of events observed within the period of trial follow-up. The use of surrogate endpoint analysis in HTA is ever increasing – 18 of 47 National Institute for Health and Care Excellence (NICE) technology appraisals between January 2022 and May 2023 included some type of surrogacy analysis.

That being said, existing surrogacy analysis methodology assumes that the relationship between endpoints remains the same for each arm. That is, one surrogate relationship may be applied to more than one treatment group.

Innovations in surrogate endpoint methodology

As surrogacy methodology evolves, however, new models are constantly being developed. These include Bayesian hierarchical models with treatment class as a covariate, mixture models that give more weight to studies with similar information to the study with missing data, and approaches that borrow from real-world evidence to inform surrogate relationships.

Key insights: Reducing uncertainty in surrogate endpoint predictions

One surprising insight from the session was that, with mixture models, sharing information at the progression-free survival level before predicting overall survival reduces uncertainty around the prediction.

Applying surrogate endpoint analysis to my work

I am currently working on a project involving the use of a surrogacy analysis. Since it’s my first experience with these methods, the session helped me gain a deeper understanding of how surrogate endpoints can aid HTA.

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ICER has a key role in evaluating and communicating the value of medicines to US stakeholders, to help them make informed decisions regarding budget, coverage, and resource allocation, while ensuring patients have access to innovative treatments.

In this article, we delve into ICER’s mission and its influence on drug pricing and access in the US. We also explore the importance of maximising engagement with ICER for favourable pricing and access outcomes.

Jump to:

• What is ICER?
• How does ICER conduct its evidence reviews?
• ICER’s ‘Unsupported Price Increases’ reports
• ICER’s impact on drug pricing and access
• Why should industry engage with ICER?
• 4 top tips for maximising engagement with ICER
• A suite of resources to support your conversations

What is ICER?

Founded in 2006, ICER is an independent, nonprofit research organisation, which evaluates the clinical and economic value of healthcare interventions (1).

ICER’s primary function is to conduct comprehensive evidence reviews of new pharmaceutical innovations that are likely to have an impact on the US healthcare system. ICER produces reports that evaluate the clinical efficacy, safety, and cost effectiveness of an intervention (or interventions for the same indication).

These reports are not dissimilar to those produced by European health technology assessment (HTA) agencies, such as NICE in England. Following ICER’s recommendations is not mandated within the US, however, this is the case with guidance produced by NICE and HTA bodies in other markets.

ICER’s influence on payer decision-making, price negotiations, and coverage decisions is understood to be growing. Payers, policymakers, and healthcare providers can use ICER’s reports to make informed choices regarding coverage and resource allocation for evaluated products.

Perhaps most importantly, ICER estimates a value-based price (VBP) via cost-effectiveness modelling, which serves as a reference point for Pharma companies, payers, and policymakers, with regard to pricing and reimbursement decisions.

How does ICER conduct its evidence reviews?

The first step is topic selection by surveying the drug research and development (R&D) pipeline. Priority is given to products and indications that will likely significantly impact the healthcare landscape, due to improved outcomes, financial implications, reduced health inequalities, or better alignment with healthcare reform initiatives (2).

Once a topic is selected, ICER notifies stakeholders and conducts a scoping phase to understand the disease and treatments. A draft evidence report is produced with epidemiological, clinical, and cost-effectiveness data. After public commentary, the report is updated and a public meeting is held before the final report is published. Information from the entire process is made publicly available to inform subsequent policy and decision-making.

ICER’s ‘Unsupported Price Increases’ reports

In addition to its evidence reviews, ICER publishes annual ‘Unsupported Price Increases’ reports. These highlight potential instances of excess healthcare spending, where ICER deems that product price increases are not supported by evidence of new clinical benefit. ICER’s Chief Medical Officer recently stated that price increases have slowed since they began publishing the reports, indicating its increasing influence in this area.

ICER’s impact on drug pricing and access

Although following ICER’s recommendations is not mandated, its influence on payer decision-making, price negotiations, and coverage decisions is understood to be growing.

There are notable examples of ICER reports being directly linked to:

• Negotiation of discounts
• Applications for additional requirements
• Restrictions for treatments deemed not cost effective

In 2018, CVS Caremark, one of the largest pharmacy benefit managers in the US, shifted to a value-based drug pricing strategy informed by ICER. Under this strategy, drugs surpassing the cost per quality-adjusted life year (QALY) threshold of $100,000 would be excluded from drug plans to help reduce costs for patients (3).

A recent survey found that, between 2020 and 2022, 79% of US payers were at least occasionally (59%) or often (20%) influenced by ICER’s recommendations for coverage decisions (4). The survey also found that there was a balance between using their recommendations to expand or to restrict coverage. Furthermore, their recommendations were used for contracting discussions, defining prior authorisation criteria, and validating supportive coverage decisions (4).

The passing of the Inflation Reduction Act (IRA) has put drug spending in the US firmly in the spotlight. Under the IRA, the top 10 drugs that have contributed most to government expenditure will have their prices renegotiated in 2026. Researchers are beginning to explore the potential benefits of the US adopting VBP more widely. One study found that if all drugs assessed by ICER since 2015 were priced in accordance with ICER’s estimated VBP at $100,000 per QALY, this could equate to annual savings of $11.8 billion (5).

Given ICER’s increasing influence, the benefit of proactive engagement with them as an important stakeholder in US market access should not be underestimated.

Why should industry engage with ICER?

Engaging with ICER could help companies better understand how their product’s value proposition will be received in the US healthcare market. By taking on board ICER’s evaluations and recommendations, companies can refine their value messaging, pricing strategies, and market access initiatives, to ultimately enhance their competitive positioning and market success.

In a recent interview, ICER’s President highlighted how ICER tends to reach out to companies before publishing a report, to:

• Explore their evidence further
• Request evidence that isn’t in the public domain
• Discuss limitations of the evidence
• Ask about the company’s own efforts and economic modelling

This informs ICER’s assessment approach.

Companies may also approach ICER for information on pricing before launch. ICER welcomes this open discussion and is keen to foster engagement that drives a mutually beneficial relationship with manufacturers. Pharmaceutical companies may gain an understanding of the pricing outcome earlier than if they weren’t engaged with ICER. This can enable them to prepare for negotiations given the eventuality of a price reduction compared with the list price.

So, should you engage with ICER? This will partly depend on the price that you have in mind and whether it’s likely to align with ICER’s VBP. Generally, it is probably better to engage with ICER than not. Mtech Access can support you with this process, with a focus on successful negotiation.

4 top tips for maximising engagement with ICER

There are multiple touchpoints in ICER’s review process for manufacturers to engage and influence. Research has suggested potential associations between manufacturer engagement and improved cost-effectiveness ratios. Here are some of our tips for getting the most out of your engagement with ICER:

1) Be prepared

ICER notifies manufacturers of an upcoming report topic that may affect them 5 weeks before it is publicly announced. Manufacturers can get ahead earlier than this, however, if they anticipate that their asset is likely to fulfil ICER’s topic selection criteria and will soon be undergoing Food and Drug Administration (FDA) review. Having a strategy in place and a suite of resources in development for the eventuality of topic selection will make engagement with ICER more efficient and productive.

2) Provide feedback

ICER welcomes critique and feedback from manufacturers at multiple stages throughout report and economic model development. Research has found that manufacturers can improve their chances of influencing ICER’s findings by submitting comments that are clear and actionable, and offering specific alternatives to their approach (6). Research also indicates that ICER is more likely to make changes to its base case analysis when these can be justified by comments citing alternative data or published articles.

3) Consider analogue review

Manufacturers may be able to optimise their engagement strategies with ICER by looking at previous reviews in analogous disease areas. ICER publishes all resources from every stage of report development publicly. This provides a wealth of information on what ICER perceives to be the strengths and limitations of the product, giving manufacturers insight into the potential outcome of the review and how they can tailor engagements and strategies accordingly.

4) Get up to speed with methods of cost-effectiveness analysis

Cost-effectiveness analysis of interventions is not widely applied in the US and may be new to many market access and commercialisation teams. Given the importance of critiquing and providing feedback on ICER’s proposed economic models, it is crucial for manufacturers to understand cost-effectiveness evaluation techniques. An external agency, like Mtech Access, may be best placed to help you get up to speed. (Our expert Health Economists even offer training courses in cost-effectiveness evaluation!).

A suite of resources to support your conversations

ICER works in accordance with strict timelines, thus manufacturers may only have a few weeks to assimilate and share data. Early preparation of a suite of resources to support engagement will streamline and accelerate this process.

The following information and materials are likely to be useful to, or directly requested by, ICER during the report development process (7):

• Key publications of patient-relevant and patient-centred outcomes, especially those not adequately captured in clinical trial data
• Key research needs and contextual considerations, which can be identified through scoping and literature reviews
• Stakeholder mapping and key informant recommendations (e.g. principal investigators from clinical trials, members of internal clinical and health economics and outcomes research [HEOR] teams, and national or regional clinical experts)
• Budget impact assessments to identify services that could be cut back to release additional budget for higher cost services and innovations
• Information about manufacturing, research, and/or development costs that are important factors in justifying the prices of products, particularly for ultra-rare conditions
• Key data inputs for economic evaluation, including health state utilities, detailed safety findings, information on prior and/or subsequent treatments received, and selected tertiary outcomes (e.g. productivity)
• Estimates of product uptake and pricing

For help sourcing and developing the above information and resources, reach out to our experts today at info@mtechaccess.co.uk.

Explore ICER’s influence with us at ISPOR

ICER plays a critical role in evaluating drug value. Its impact on pricing and access is growing in the increasingly budget-focused US healthcare system.

Understanding ICER’s influence and how to maximise opportunities for engagement enables manufacturers to align product development with payer expectations, optimise pricing strategies, and enhance patient access to new therapies.

Optimal engagement with ICER can contribute to favourable outcomes. It does, however, require Pharma companies to deploy significant resource and organisation. Our experts can help you:

• Develop your pricing strategy
• Establish an understanding of cost effectiveness and VBP
• Source the necessary information and evidence
• Develop the resources you need
• Engage with ICER’s process

Email info@mtechaccess.co.uk to speak to our experts about the value of engaging with ICER as part of your US and wider global market access and commercialisation strategy.

References and further reading:

1. Institute for Clinical and Economic Review. Independent Funding. Available from: https://icer.org/who-we-are/independent-funding/. Accessed on: 26th March 2024.
2. Institute for Clinical and Economic Review. Topic Selection. Available from: https://icer.org/our-approach/methods-process/value-assessment-framework/topic-selection/. Accessed on: 26th March 2024.
3. First Report: Managed Care. CVS Shifts to ICER Value-Based Drug Pricing Strategy. 2018. Available from: https://www.hmpgloballearningnetwork.com/site/frmc/article/cvs-shifts-icer-value-based-drug-pricing-strategy. Accessed on: 26th March 2024.
4. Zheng C, et al. Impact of ICER Assessments on Payer Decision Making in the United States. Value in Health. 2023;26(4):S268.
5. Yeung K, et al. Value-Based Pricing of US Prescription Drugs: Estimated Savings Using Reports From the Institute for Clinical and Economic Review. JAMA Health Forum. 2022; 3(12):e224631.
6. Breslau RM, et al. Should Drug Companies Engage with ICER? An Empirical Analysis of How Often Manufacturers Engage with ICER and Whether Engagement May Influence ICER’s Cost‑Effectiveness Estimates. Pharmacoecon Open. 2022;6(6):893-895.
7. Institute for Clinical and Economic Review. Manufacturer Engagement. Available from: https://icer.org/our-approach/methods-process/manufacturer-engagement/. Accessed on: 26th March 2024.

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Who’s attending from Mtech Access?

David Niziol

MD & Founder

Anthony Bentley

Director – Health Economics

Clare Foy

Director – Global Market Access

Adam Brown

Director – Consulting Services

Hannah Palin

Director – Market Access

Lydia Crowe

Director – Commercial

Key reads and insights ahead of ISPOR

What role does ICER play in US market access?

We delve into ICER’s influence on drug pricing and access in the US. We also explore the importance of maximising engagement with ICER for favourable pricing and access outcomes…

Top 10 HEOR trends – A detailed look at ISPOR’s 2024–2025 topics

We bring together specialists from across Mtech Access to look at each of ISPOR’s top 10 HEOR trends for 2024-25. Jump in, as our experts give their hot-take on each trend, sharing their perspectives on what’s happening in these areas…

EThe role of economic evaluation in US healthcare

Hannah Palin (Director – Market Access) and Calum Jones (Associate Director – Health Economics) explore the landscape for economic evaluation in the US and highlight important considerations for companies seeking to infiltrate this key market….

Free guide to market access and reimbursement in key European markets

Request your copy of our popular introductory guide: ‘Market access and reimbursement in key European markets’…

AMCP dossier planning for US market access and reimbursement

What is an AMCP dossier and how are they used in US market access? Drawing on their extensive experience compiling AMCP dossiers, here, our global market access experts explain when, why and how…

EU Joint Clinical Assessment (JCA) – implications for Pharma and Medtech

What is the EU Joint Clinical Assessment (JCA) and what does it mean for market access and reimbursment in Euorpe?…

6 questions to ask when adapting global market access materials for local markets

How can you ensure that the market access materials, value proposition and evidence developed by global teams, will resonate for your local audience?…

Setting your product up for success – commercialisation strategy for the US market

How can you ensure your product is set up for success from early clinical trials right through to launch and commercialisation? In this Global Whispers webinar we explore best practice…

The post Meet us at ISPOR in Atlanta, GA, USA appeared first on Mtech Access.

Sections

1. Real-World Evidence by Clare Foy (Director – Global Market Access)
2. Drug Pricing by Louise Maddison (Senior Consultant – Global Market Access)
3. Artificial Intelligence by Richard Johnson (Senior Management Consultant – Innovation), Louise Maddison (Senior Consultant – Global Market Access), and Emily Hardy (Associate Consultant – Systematic Review)
4. Fostering Innovation by Phil Richardson (Chair & Chief Innovation Officer)
5. Health Equity by Chloe Morris (Associate Management Consultant – Innovation)
6. Accelerating Drug Approvals by Samantha Gillard (Director – HTA)
7. Value Measurement by Kaylie Metcalfe (Consultant – Health Economics)
8. Patient Centricity by Louise Maddison (Senior Consultant – Global Market Access)
9. Precision Medicine by Stephanie Swift (Senior Consultant – Systematic Review)
10. Public Health Policy by Robert Hull (Senior Consultant – NHS Insight & Interaction)

Executive Summary

Hayley Shoel and Abigail Beveridge, from our Medical Writing Team, outline what to expect from this paper in the below Executive Summary.

In January 2024, ISPOR published their ‘2024–2025 Top HEOR Trends’ report, a horizon scanning publication that highlighted key trends affecting healthcare decision-making around the world.

Since these trends resonate with our own research and activities here at Mtech Access, experts within our team have come together to share their perspectives on each of the trends. In this article, we highlight considerations for Pharma and Medtech companies looking to optimise success in market access.

We begin with a look at how Real-World Evidence (RWE) is increasingly used for regulatory and reimbursement decision-making. The authors explain how data can be collected, and spotlight the frameworks available for guidance on optimising the use of RWE.

We particularly recommend the section on Artificial Intelligence (AI), a hot topic across all sectors. Likewise, don’t miss the Fostering Innovation section, where Phil Richardson (Chair & Chief Innovation Officer) shares his insights into the importance of innovation in healthcare. He highlights Mtech Access’ initiative, the ‘Above Brand Academy’, which aims to foster collaboration between the NHS, Pharma, Medtech, and market access, to identify innovative solutions to system-, therapy area-, and disease-level problems.

Samantha Gillard (Director – HTA) explores Accelerated Drug Approval Pathways and their associated challenges. She discusses how HEOR can aid with filling data gaps and the importance of considering the ‘fourth hurdle’ of recommendation for reimbursement.

Plus much more – keep scrolling to dive in!

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Real-World Evidence

Authors: Clare Foy (Director – Global Market Access)

What is real-world evidence?

Real-world evidence (RWE) is fast becoming an innovative way to expand how we assess the value of healthcare interventions. Defined by NICE as “evidence generated from the analysis of real-world data”, RWE encompasses evidence generated from real patients using a range of study designs and analytical methods, depending on the research question (1).

RWE studies leverage real-world data (RWD), which relate to patient health status and/or the delivery of healthcare routinely collected from a variety of sources (2–4, 9). RWD helps us to understand how interventions are used in real patients, and the potential benefits and/or risks associated with their use (2–4, 9).

RWD includes electronic patient records, administrative data, claims data, registry data, patient-generated health data, and data from chart reviews and audit and service evaluations. Other examples include primary data from observational cohorts, health surveys, interviews, focus groups, social media, and patient advocacy groups (1–4).

Data can be qualitative or quantitative, and can be collected in a structured, semi-structured, or unstructured format, prospectively or retrospectively (1, 3, 11). RWE analytics can be used to extract deeper insights from rich data sets (6, 9). Artificial intelligence can also be used to analyse unstructured data and speed up the analysis process (9, 11).

Why is it important to consider RWE in decision-making?

Regulatory and reimbursement decision-making is largely informed by evidence generated in a randomised controlled trial (RCT) setting (5). RWE serves to complement this evidence base, as it enables decision makers to understand the safety and effectiveness of interventions outside of a controlled environment (3, 5, 8, 9). It also helps to provide therapeutic context for decision-making, for example, regarding the expansion of label indications (9, 10).

RWE generates real-world insight into how interventions impact real patient outcomes and experiences, and service delivery by healthcare systems (3, 6). Consideration of RWE in decision-making therefore drives timely access to innovative therapies that meet the needs of real-world patients, which may not be wholly reflected in RCT settings (4, 8, 10).

How else can we use RWE?

RWE can be used to create guidelines for clinical and social care, and public health issues by characterising health conditions, interventions, care pathways, and patient experiences (1).

RWE also informs our understanding of the economic burden of disease and how economic models are designed, populated, and validated (1). More importantly, the analysis of RWE can support with identifying and addressing health inequalities, ensuring equity of access to healthcare services for vulnerable groups in society (1).

Helpful resources

There are several RWE frameworks available that provide guidance on the use of RWE, including assessing the quality of data sources, and how RWE can be utilised for regulatory and market access purposes.

These are available from the following organisations (this list is not exhaustive):

• NICE
• FDA
• EMA
• ISPOR

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References

1. NICE. Real-world evidence framework (ECD9). 2024. Available from: https://www.nice.org.uk/corporate/ecd9/resources/nice-realworld-evidence-framework-pdf-1124020816837. Accessed February 2024.
2. U.S. FDA. Real-World Evidence. 2023. Available from: https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence. Accessed February 2024.
3. U.S. FDA. Framework For FDA’s Real-World Evidence Program. 2018. Available from: https://www.fda.gov/media/120060/download?attachment. Accessed February 2024.
4. Chodankar D. Introduction to real-world evidence studies. Perspectives in clinical research. 2021;12(3):171.
5. EMA. Real-world evidence framework to support EU regulatory decision-making: Report on the experience gained with regulator-led studies from September 2021 to February 2023. 2023. Available from: https://www.ema.europa.eu/en/documents/report/real-world-evidence-framework-support-eu-regulatory-decision-making-report-experience-gained-regulator-led-studies-september-2021-february-2023_en.pdf. Accessed February 2024.
6. Champagne D, Deverson A, Pérez L, Saunders D. Creating value from next-generation real-world evidence. 2020. Available from: https://www.mckinsey.com/industries/life-sciences/our-insights/creating-value-from-next-generation-real-world-evidence#/. Accessed February 2024.
7. ISPOR. ISPOR 2024–2025 Top 10 HEOR Trends. 2024. Available from: https://www.ispor.org/heor-resources/about-heor/top-10-heor-trends. Accessed February 2024.
8. ISPOR. Unlocking The Promise of Real-World Evidence. Value & Outcomes Spotlight. 2020;6(5):26-8.
9. Deloitte Insights. Real-world evidence’s evolution into a true end-to-end capability. 2022. Available from: https://www2.deloitte.com/us/en/insights/industry/health-care/real-world-evidence-study.html. Accessed February 2024.
10. Cronenwett J. How Real-World Evidence Helps Medical Device Manufacturers Drive Value. 2023. Available from: https://medtechintelligence.com/column/how-real-world-evidence-helps-medical-device-manufacturers-drive-value/. Accessed February 2024.
11. Jayne J. Realising the Promise of Real-World Evidence in MedTech. 2022. Available from: https://medtechintelligence.com/feature_article/realizing-the-promise-of-real-world-evidence-in-medtech/. Accessed February 2024.

Drug Pricing

Author: Louise Maddison (Senior Consultant – Global Market Access).

The drug pricing landscape in the United States

The Inflation Reduction Act (IRA) was introduced in 2022 to address drug pricing and Medicare expenditure in the US. The IRA outlined several reforms relating to direct negotiation, the Part D redesign, and the inflation price cap (1).

Importantly, the IRA has empowered the Centers for Medicare & Medicaid Services to negotiate prices on behalf of Medicare for the first time (1, 2). Price negotiations for 10 drugs started in 2023, with price changes taking effect in 2026 (1, 2). The IRA drug pricing provisions are estimated to reduce the federal deficit by an estimated US$237 billion by 2031 (3).

The initial 10 drugs include medications for the treatment of blood clots, diabetes, and autoimmune diseases (1, 2). They were chosen primarily due to their high-expenditure status, although generic availability and time on the market were also considered (1, 2). By 2029, a cumulative total of 60 drugs will have been selected for negotiation (4).

At first glance, these reforms only affect a few brands in the short term; however, there will be a ripple effect both for within-class competitors and beyond the Medicare population. Prices for competitors within each class are likely to be affected, and potentially extend to other populations, as commercial payers may push for lower prices and want the same price offered to Medicare.

The inflation price cap will mean that manufacturers will be required to pay a rebate if the increase in drug prices exceeds the rate of inflation. Additionally, as part of the Part D redesign plans, manufacturers will also be required to increase their share of the costs that surpass the out-of-pocket spending cap. The Part D changes will also limit beneficiary premium increases to 6% annually; therefore, payers will look to mitigate their own increased liability by actively managing drug costs and there may be a shift towards value-based pricing in the US.

The changing landscape for drug pricing in Europe

The European Commission is proposing the largest reform in over 20 years to the European Union’s Pharmaceutical legislation. Through a patient-centred approach, the reforms will mean that patients across the European Union will have timely and equitable access to safe, effective, and affordable medicines (5).

Access to new treatments across the 27 member states of the European Union varies broadly. In 2023, patients in Germany could access 88% of medicines approved by the EMA between 2018 and 2021. Comparatively, patients in Turkey could access only 6% (6). A single market for medicines is needed to reduce these disparities.

Although pricing and reimbursement decision-making will remain with member states, the European Commission will foster transparency of price information and launch a group to steer cooperation between national pricing and reimbursement (7).

Payers are now working more closely together through the group of National Competent Authorities on Pricing and Reimbursement and Public Healthcare Payers (NCAPR), which was an ad hoc forum, but now promotes continuous voluntary cooperation (7, 8).

The European Commission further supports increased cooperation to improve affordability through joint procurement process, such as the BeNeLuxA initiative between Belgium, the Netherlands, Luxembourg, Austria, and Ireland (9), as well as the Nordic Pharmaceutical Forum (10).

As such, we may see more instances of member states banding together to increase their bargaining power through joint negotiations, and before legislation is adopted, especially with current financial pressures across healthcare systems.

The reforms also propose 2 years of data protection conditional on launch of a new medicine across all member states (7), as well as measures to encourage early market access of generic and biosimilar medicines. However, opinions are still divided on the proposed changes to the European Commission’s draft, despite time ticking down on the current parliamentary term before proceedings are suspended for the 2024 European Parliament elections (11).

European Union Joint Clinical Assessment

In other news, the European Union Joint Clinical Assessment (EU JCA) will kick off in 2025 (to read more about EU JCA, see our recent article). As EU JCA reports are non-binding, it remains unclear as to what extent individual member states will use the information, and the impact that it will have on pricing and reimbursement decision-making.

In preparation for these reforms to the landscape both in the US and Europe, manufacturers need to be alert to ongoing changes and to be agile in their pricing strategy at launch, as well as ongoing life cycle management of medicines already on the market.

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References

1. U.S. Centers for Medicare & Medicaid Services. Medicare Drug Price Negotiation Program: Selected Drugs for Initial Price Applicability Year 2026. 2023. Available from: https://www.cms.gov/files/document/fact-sheet-medicare-selected-drug-negotiation-list-ipay-2026.pdf. Accessed February 2024.
2. U.S. Department of Health and Human Services. HHS Selects the First Drugs for Medicare Drug Price Negotiation. 2023. Available from: https://www.hhs.gov/about/news/2023/08/29/hhs-selects-the-first-drugs-for-medicare-drug-price-negotiation.html. Accessed February 2024.
3. Congressional Budget Office: Nonpartisan Analysis for the U.S. Congress. How CBO Estimated the Budgetary Impact of Key Prescription Drug Provisions in the 2022 Reconciliation Act. 2023. Available from: www.cbo.gov/system/files/2023-02/58850-IRA-Drug-Provs.pdf. Accessed February 2024.
4. The White House: Statements And Releases. FACT SHEET: Biden-⁠Harris Administration Announces First Ten Drugs Selected for Medicare Price Negotiation. 2023. Available from: https://www.whitehouse.gov/briefing-room/statements-releases/2023/08/29/fact-sheet-biden-harris-administration-announces-first-ten-drugs-selected-for-medicare-price-negotiation/. Accessed February 2024.
5. European Commission. Reform of the EU Pharmaceutical legislation: Affordable, accessible, and innovative medicines. 2023. Available from: https://commission.europa.eu/strategy-and-policy/priorities-2019-2024/promoting-our-european-way-life/european-health-union/reform-eu-pharmaceutical-legislation_en. Accessed February 2024.
6. Statista. Share of medicines approved by the EMA available to patients in Europe as of 2023, by country. 2023. Available from: https://www.statista.com/statistics/1011186/rate-of-medicine-availability-europe-by-country/. Accessed February 2024.
7. European Commission. Communication From The Commission To The European Parliament, The Council, The European Economic And Social Committee And The Committee Of The Regions: Reform of the pharmaceutical legislation and measures addressing antimicrobial resistance. 2023. Available from: https://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:52023DC0190. Accessed February 2024.
8. Meeting of the National Competent Authorities on Pricing and Reimbursement and Public Healthcare Payers (NCAPR). Conference Proceedings. 2023. Conference date: 30thMarch 2023. Conference location:Stockholm/Webex. Available from: https://health.ec.europa.eu/document/download/3f428d22-966e-447b-909c-63a2f3d64301_en?filename=mp_20230330_mi_en_.pdf. Accessed February 2024.
9. Beneluxa Initiative on Pharmaceutical Policy. Beneluxa Initiative. 2024. Available from: https://beneluxa.org/collaboration. Accessed February 2024.
10. Sonne F, Juhl TN, Andreassen E, Aufrecht-Gustafsson M, Svensson M, Þórhallsdóttir Ó, et al. Nordic Pharmaceutical Forum: Strategy for 2023–2025. Available from: https://amgros.dk/media/3790/nlf-strategy-final.pdf. Accessed February 2024.
11. Science Business Publishing. As time runs out, the European Parliament remains divided over pharma reform. 2023. Available from: https://sciencebusiness.net/news/drug-development/time-runs-out-european-parliament-remains-divided-over-pharma-reform. Accessed February 2024.

Artificial Intelligence

Authors: Richard Johnson (Senior Management Consultant – Innovation), Louise Maddison (Senior Consultant – Global Market Access), and Emily Hardy (Associate Consultant – Systematic Review).

AI in HEOR – The current landscape

Artificial intelligence (AI) permeates everyday life, through technologies such as GPS navigation, digital assistants, and eCommerce. Nonetheless, the launch of ChatGPT by OpenAI in November 2022 revolutionised the world’s understanding of generative AI and the broader applications of technology automation.

Large language models (LLMs) packaged into a chatbot interface, like ChatGPT, have since been launched by organisations such as Microsoft and Google, indicating the extent of AI’s impact. To ensure that our team at Mtech Access and our clients remain ahead of the curve, we take a keen interest in advances of AI in healthcare, particularly their potential applications in HEOR and market access strategy.

AI use in the healthcare industry has accelerated globally in recent years. We have seen regulatory submissions of AI enabled devices (e.g. in radiology), plus the use of AI to support healthcare service redesign. AI has also been used to transform drug development, clinical operations, and regulatory processes (1–4).

The future of AI in healthcare

AI is likely to have a more powerful impact across the entire healthcare value chain than any other previous technology. This is because AI serves to improve healthcare outcomes (e.g. predicting disease risk, personalised treatments), whilst also introducing efficiencies for societies burdened by increasing healthcare costs (5).

Significant initiatives and investments are enabling the implementation of AI in healthcare. The World Health Organization has set up a Global Initiative on AI for Health (6), whilst the European Union has committed €50 million to funding world-class testing and experimentation facilities in healthcare (known as TEF-health) (7). The Bill and Melinda Gates Foundation has also committed US$5 million to equitable access to AI in healthcare (8).

In October 2023, the UK government committed £100 million to AI medical advancement research through the AI life sciences accelerator mission (9). Through a collaborative effort with government, industry, the NHS, and academia, the accelerator aims to drive breakthrough interventions for previously incurable diseases and conditions with high mortality and morbidity.

This effort has seven key aims:

• Accelerating the pace of studies into novel dementia treatment
• Increasing the understanding of mental health conditions, including redefining diseases and developing tools to address them
• Reducing mortality and morbidity from respiratory disease in the UK and globally
• Treatment and prevention of cardiovascular disease and its major risk factors, including obesity
• Enabling early diagnosis and treatments, including immune therapies (such as cancer vaccines)
• Sustaining the UK’s position in vaccine discovery, development, and manufacturing
• Addressing the underlying biology of ageing

These commitments will provide Pharmaceutical and Medtech companies with opportunities to overcome the key challenges in AI adoption, such as investment in data analytics, systems, process, and training (business change and transformation) (10).

Our insights on AI

Calum Jones recently spoke with Dr George Magrath (CEO, Lexitas) about the use of AI in clinical trials (11).

They discussed how AI technologies can help to understand how patients are likely to respond to an intervention, which can help refine clinical trial endpoints. This means that clinical trials can be kept to a minimum required size, whilst retaining sufficient statistical power to demonstrate intervention safety and efficacy (11).

They also discussed how AI can be used to identify the most appropriate patients for enrolment, which increases the speed to clinical trial. This AI-enabled approach is particularly suitable for rare diseases, but can help patients gain faster access to medications regardless of indication (11).

Considerations for the use of AI in healthcare

Amongst the excitement, the use of AI raises concerns around data protection, data quality, and ethics. As technology advances, the latest guidance, frameworks, and evidence requirements should remain at the forefront of discussions related to AI solutions.

No industry-wide framework has been adopted yet, but several resources are available (12–18). In addition, NICE has developed the evidence standards framework for digital health technologies to support digital health market access in England (17). The framework outlines how new digital health technologies should be assessed, to ensure that they are clinically effective and offer value to health care systems (19).

The NICE META tool can be used to assess digital technologies like AI. The tool serves to address gaps in evidence and development plans, to understand how AI technologies can be brought to market. Samantha Gillard, along with colleagues Calum Jones and Hannah Palin recently discussed the role of the NICE META tool in evidence strategy (20).

The use of AI automation in HEOR and market access strategy

Moving from clinical development onto HEOR and market access strategy, AI automation can be applied at various stages. It can be used to support predictive analytics for drug pricing, improving efficiencies in systematic literature reviews (SLRs) and regulatory submissions, and to pre-empt the outcome of HTA agency negotiations.

AI automation tools optimise time and provide budget savings, which can help a business stay competitive in an evolving market. That said, the decision to use AI automation tools should not be taken lightly. Considerations regarding output quality, methodological transparency, data security, and ethical responsibility are vital before selecting a tool in this increasingly saturated field.

Relevant published guidance on the use of automated tools to support HEOR should also be considered. For example, the use of AI automated tools for conducting SLRs is not currently part of guidance issued by Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) (18) or NICE for health technology assessment submissions (21), but is highlighted in the Cochrane Handbook (19). As a result, there remains ambiguity in the HEOR community about the acceptance of using AI automation tools to support SLR methodology.

The future of AI in market access

It is important to work with agencies who have a strong technical understanding of the market access landscape and the capabilities needed to develop and deliver market access strategies in key global markets.

As AI continues to permeate the healthcare industry, we must keep up with the changing landscape of reimbursement pathways and evidence requirements. Only then can Pharmaceutical and Medtech companies stay ahead of the AI adoption curve and reap the benefits that this technology can bring.

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References

1. U.S. FDA. Artificial Intelligence and Machine Learning (AI/ML) – Enabled Medical Devices. 2023. Available from: https://www.fda.gov/medical-devices/software-medical-device-samd/artificial-intelligence-and-machine-learning-aiml-enabled-medical-devices. Accessed February 2024.
2. NHS England. The NHS AI Lab: Accelerating the safe adoption of artificial intelligence in health and care. Available from: https://transform.england.nhs.uk/ai-lab/. Accessed February 2024.
3. U.S. FDA. Using Artificial Intelligence & Machine Learning in the Development of Drug & Biological Products. 2023. Available from: www.fda.gov/media/167973/download. Accessed February 2024.
4. U.S. Department of Health and Human Services. Department of Health and Human Services: Artificial Intelligence Use Cases Inventory. 2024. Available from: https://www.hhs.gov/about/agencies/asa/ocio/ai/use-cases/index.html. Accessed February 2024.
5. World Economic Forum: Health and Healthcare. 5 steps to put healthcare on the AI fast-track. 2024. Available from: https://www.weforum.org/agenda/2024/01/healthcare-ai/. Accessed February 2024.
6. World Health Organization. Global Initiative on AI for Health. 2024. Available from: https://www.who.int/initiatives/global-initiative-on-ai-for-health. Accessed February 2024.
7. European Commission. Shaping Europe’s digital future: Sectorial AI Testing and Experimentation Facilities under the Digital Europe Programme. 2024. Available from: https://digital-strategy.ec.europa.eu/en/activities/testing-and-experimentation-facilities. Accessed February 2024.
8. Bill & Melinda Gates Foundation. Gates Foundation Selects Nearly 50 Global Health and Development Projects That Will Contribute to Shaping Equitable Access to AI. 2023. Available from: https://www.gatesfoundation.org/ideas/media-center/press-releases/2023/08/grand-challenges-rfp-recipients-ai-large-language-models. Accessed February 2024.
9. GOV.UK. Technology in health and social care. Press release: New £100 million fund to capitalise on AI’s game-changing potential in life sciences and healthcare. 2023. Available from: https://www.gov.uk/government/news/new-100-million-fund-to-capitalise-on-ais-game-changing-potential-in-life-sciences-and-healthcare. Accessed February 2024.
10. GOV.UK. Research and innovation in health and social care. News story: UK life sciences vision set to deliver life-changing innovations. 2021. Available from: https://www.gov.uk/government/news/uk-life-sciences-vision-set-to-deliver-life-changing-innovations. Accessed February 2024.
11. Mtech Access. Artificial intelligence (AI) in clinical trials: implications for evidence synthesis and market access. 2023. Available from: https://mtechaccess.co.uk/ai-clinical-trials-evidence/. Accessed February 2024.
12. U.S. FDA. Marketing Submission Recommendations for a Predetermined Change Control Plan for Artificial Intelligence/Machine Learning (AI/ML)-Enabled Device Software Functions: Draft Guidance for Industry and Food and Drug Administration Staff. 2023. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/marketing-submission-recommendations-predetermined-change-control-plan-artificial. Accessed February 2024.
13. GOV.UK. Guidance: Software and Artificial Intelligence (AI) as a Medical Device. 2023. Available from: https://www.gov.uk/government/publications/software-and-artificial-intelligence-ai-as-a-medical-device/software-and-artificial-intelligence-ai-as-a-medical-device. Accessed February 2024.
14. WHO. Regulatory considerations on artificial intelligence for health. 2023. Available from: https://www.who.int/publications/i/item/9789240078871. Accessed February 2024.
15. WHO. Ethics and governance of artificial intelligence for health: Guidance on large multi-modal models. 2024. Available from: https://www.who.int/publications/i/item/9789240084759. Accessed February 2024.
16. National Academy of Medicine. Health Care Artificial Intelligence Code of Conduct. 2024. Available from: https://nam.edu/programs/value-science-driven-health-care/health-care-artificial-intelligence-code-of-conduct/. Accessed February 2024.
17. NICE. Evidence standards framework for digital health technologies (ECD7). 2022. Available from: https://www.nice.org.uk/corporate/ecd7. Accessed February 2024.
18. Page MJ, Moher D, Bossuyt PM, Boutron I, Hoffmann TC, Mulrow CD, et al. PRISMA 2020 explanation and elaboration: updated guidance and exemplars for reporting systematic reviews. British Medical Journal. 2021;372.
19. Cochrane Training. Cochrane Handbook for Systematic Reviews of Interventions: Version 6.4. 2023. Available from: https://training.cochrane.org/handbook/current. Accessed February 2024.
20. Mtech Access. Evidence strategy for Medical Technologies and Diagnostics. 2023. Available from: https://mtechaccess.co.uk/evidence-strategy-for-medical-technologies-and-diagnostics/. Accessed February 2024.
21. NICE. Single technology appraisal (STA): Specification for manufacturer/sponsor submission of evidence. 2012. Available from: https://www.nice.org.uk/media/default/about/what-we-do/nice-guidance/nice-technology-appraisals/specification-for-manufacturer-sponsor-submission-of-evidence-june-2012.doc. Accessed February 2024.

Fostering Innovation

Author: Phil Richardson (Chair & Chief Innovation Officer).

Looking beyond product

In the commercialisation of Pharmaceutical and medical technology interventions, innovation spans the whole value chain, from drug discovery to patient use, and remains the biggest source of sustainable competitive advantage. However, it will struggle to deliver meaningful changes in healthcare if the focus remains on just developing products.

Innovation includes case finding, which can seem counterintuitive, particularly for organisations that market products and services for the end of complex disease pathways. There is a tension between some traditional approaches of pharmaceutical treatment and the drive by healthcare systems to identify cases earlier.

Patients who are pre-diabetic or pre-cancer, where there may not be an obvious marker, need innovative thinking and brave decisions about resource allocation. Identifying disease early in patients, as a mantra, could change paradigms for research and development priorities, as well as the go-to-market strategies for traditional products.

Being the first organisation to bring a product to market does not translate to being the most successful; focus on business models, culture, service delivery, and patient quality of care and safety may be more valuable (1).

The holistic view of a product indeed includes the product itself, but also its delivery and impact on the patient experience. This is known as product surround. In other industries, the innovation of product surround versus the product alone is advocated for (1), yet pharmaceutical innovations mostly focus on the product alone.

More investment is needed to innovate how healthcare service and delivery, and the products themselves, interact with one another. Focusing too heavily on the product alone means that the transformative opportunities and synergies that exist could be overlooked. Patient centricity is at the core of innovative solutions.

The transformative power of innovation in healthcare

NHS England acknowledge that innovation “not only transforms patients’ lives, but also brings significant benefits to the UK economy and society” (2). The NHS Accelerated Access Collaborative has outlined the ingredients for innovative success (2). These include a focus on defined stages for innovation, how clinical skills and leadership can be best utilised, redesign of the treatment pathway to foster innovation, and the need to focus on local versus national scale implementation (2).

Innovation comes from diversity of thinking, experience, motivation, empowerment, engagement, co-design, co-production and, observation. Data collected ethnographically through active observation is more insightful than analysis of historical data. It is critical that we sense-and-respond to the current landscape, rather than catalogue historical transactions based on traditional understanding.

Funding innovation in healthcare

Funding and incentives are also critical for fostering innovation (2). Limited funding for future therapies stifles what is possible. Equally, healthcare systems with budget and resource constraints are unable to innovate beyond symptom-led problem solving. Game-changing approaches, which seem obvious with hindsight, are needed.

One example of this is our plan for an Above Brand Academy, which is an Mtech Access initiative focused on establishing partnerships between the NHS, Pharma, Medtech, and market access. It will create a collaborative space for organisations to work above-brand and above-symptom to innovate aetiology-based solutions.

By fostering innovation and pushing the boundaries for industry, the Above Brand Academy will offer opportunities to work alongside the health service to develop sustainable changes using a combination of applied resources. It is an opportunity for global strategists and in-market expertise to connect with healthcare professionals, payers, policy makers, and operational leads.

The approach is theme based, with a recent focus on cancer and early identification generating meaningful follow-up conversations. Over time the intent is to bring in a strong patient voice that is representative of the theme being examined.

More help is at hand. In the UK, the refocusing of Academic Health Science Networks into Health Innovation Networks signals the ambition to drive innovation adoption at scale. The NHS Clinical Entrepreneur Programme brings clinical entrepreneurs right into the heart of the NHS (3). The NHS Digital Academy has also been set up to drive excellent digital leaders in NHS England (4).

Get a jump start by talking to the Innovation Team at Mtech Access.

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References

1. Forbes. Leadership: Don’t Waste Innovation on Products. 2013. Available from: https://www.forbes.com/sites/christinecrandell/2013/02/03/best-innovation-doesnt-come-in-products/. Accessed February 2024.
2. NHS England. Blog: The ingredients for success: how to bake innovation into the NHS. 2023. Available from: https://www.england.nhs.uk/aac/2023/12/11/blog-the-ingredients-for-success-how-to-bake-innovation-into-the-nhs/. Accessed February 2024.
3. NHS Clinical Entrepreneur Programme. Supporting innovation in the NHS. 2022 [AB4] . Available from: https://nhscep.com/. Accessed February 2024.
4. NHS England. NHS Digital Academy. 2024[AB5] . Available from: https://www.england.nhs.uk/digitaltechnology/nhs-digital-academy/. Accessed February 2024.

Health Equity

Author: Chloe Morris (Associate Management Consultant – Innovation).

Health equity is a fundamental human right that ensures every individual has access to the highest attainable standard of health without discrimination or bias. It also aims to achieve the highest level of health for all people (1).

Health equity versus health equality

Health equity and health equality are two distinct concepts. They refer to differences in health outcomes between distinct groups of people.

Health equity aims to achieve fairness in health outcomes by addressing systemic barriers and providing resources based on individual needs. It recognises that different populations have varying health needs and aims to eliminate unjust differences (1).

On the other hand, health equality provides the same level of care to everyone, regardless of their specific needs. It assumes that equal treatment will lead to equal outcomes, but this approach may not account for existing disparities (2).

It is important to focus on these differences to be able to address health disparities and promote health equity for all individuals and communities. The goal to achieve health equity is shared by various organisations and initiatives, including the All Our Health initiative(3), Department of Health and Human Services (HHS), and the World Health Organisation (4). Health equity has re-emerged as a prominent trend for 2024/2025 in the realm of HEOR, having previously featured in the 2022/2023 HEOR report (5).

The relationship between funding and health equity is complex. In the UK, the NHS has been criticised for differential quality and funding in health equity. A national survey found that equity is not addressed systematically below strategic levels, and equity does not shape funding decisions, programme development, implementation, and monitoring.

However, there are several initiatives that address social determinants of health, such as the Thrive LDN Partnership (6), the NHS Health check programme (7), and the Healthy Start programme (8), which involve identifying and preventing cardiovascular disease, and improving access to healthy food for families on low incomes.

Addressing health equity

Addressing health equity and inequality requires a focus on neighbourhoods, communities, and populations, and putting the person at the centre of decision-making. Agencies cannot do this on their own; they need to be intricately connected to health systems, primary care, and secure health data environments.

At Mtech Access, we understand the importance of health equity, and are on focusing initiatives such as population health management. This is a data-driven, proactive care approach to reduce inequalities and optimise health outcomes across different populations, sub-populations, and individuals. As we carve out a path towards a future that is healthier and equitable, it is crucial that we position individuals at the heart of decision-making.

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References

1. WHO. Health Equity. Available from: https://www.who.int/health-topics/health-equity#tab=tab_1. Accessed February 2024.
2. Office for Health Improvement & Disparities. Health disparities and health inequalities: applying All Our Health. 2022. Available from: https://www.gov.uk/government/publications/health-disparities-and-health-inequalities-applying-all-our-health/health-disparities-and-health-inequalities-applying-all-our-health. Accessed February 2024.
3. GOV.UK. Health disparities and health inequalities: applying All Our Health. 2022. Available from https://www.gov.uk/government/publications/health-disparities-and-health-inequalities-applying-all-our-health/health-disparities-and-health-inequalities-applying-all-our-health. Accessed February 2024.
4. WHO. Special initiative for action on the social determinants of health for advancing health equity. Available from:https://www.who.int/initiatives/action-on-the-social-determinants-of-health-for-advancing-equity. Accessed February 2024.
5. ISPOR. ISPOR 2024–2025 Top 10 HEOR Trends. 2024. Available from: https://www.ispor.org/heor-resources/about-heor/top-10-heor-trends. Accessed February 2024.
6. Transformation Partners in Health and Care. Thrive LDN. Available from: https://www.transformationpartners.nhs.uk/programmes/thrive/. Accessed February 2024.
7. NHS. NHS Health Check. Available from: https://www.nhs.uk/conditions/nhs-health-check/. Accessed February 2024.
8. GOV.UK. Healthy Start. Available from: https://www.gov.uk/healthy-start. Accessed February 2024.

Accelerating Drug Approvals

Author: Samantha Gillard (Director – HTA).

The goal for both drug manufacturers and healthcare professionals is to make medicines available to patients as soon as possible. This is particularly true in rare diseases and cancer where no treatments are currently available and/or patient survival rates are exceptionally low. However, this presents a challenge, with new drugs taking an average of 10 to 15 years to get from the bench to a patient, and less than 10% of drug candidates making it into Phase 2 trials (1).

Overcoming challenges associated with accelerated assessment

Changes in regulatory processes in recent years have sought to facilitate faster drug approvals. In the USA, these include the Fast Track, Breakthrough Therapy, Priority Review, or Accelerated Approval pathways (2). In Europe, drugs may undergo accelerated assessment, conditional marketing authorisation, or approval under extraordinary circumstances (3).

While these are positive initiatives, they may pose challenges for drug manufacturers for several reasons. Firstly, data may be limited to Phase 2 trials, which can be short in duration. Furthermore, these trials may use surrogate endpoints and may not include a comparator arm. The result being that there is potential for significant uncertainty in the efficacy of a drug.

HEOR plays a vital role in filling data gaps that may exist. In the case of a product where only surrogate endpoints are available, additional data are needed to prove that the these translate into clinically relevant patient outcomes. A systematic literature review may be used to identify evidence that surrogate markers do indeed translate into clinical endpoints.

Similarly, if only short-term data are available, additional evidence is required to estimate the longer-term clinical impact that the therapy may have. Some of the potential uncertainty with immature datasets could be addressed by health economic modelling, informed by clinical experts.

HEOR can also step in to help when no comparator data were collected in the trial. In such cases, techniques such as matching-adjusted indirect comparison (MAIC) or using RWE may be used to generate a synthetic control arm.

The “fourth hurdle”: Health technology assessment

It is also important to remember that, in most countries, regulatory approval does not automatically translate into market access and reimbursement. Drugs also need to pass the “fourth hurdle” of a positive recommendation by an HTA body to get reimbursement.

Unlike regulatory approval, which is limited to efficacy and safety of a drug, HTA also considers the cost-effectiveness of a product. This is essentially whether it is ‘good value for money’ to the healthcare system, relative to current standard of care. Drug manufacturers are therefore challenged with not only considering the efficacy and safety of their treatment, but also the overall cost of their treatment relative to current standard of care.

In the UK it takes around 18 months from the beginning of the NICE process until a recommendation for the drug is published, and a further 90 days for the funding mandate to be granted. These timelines assume that the drug is recommended at the first NICE committee meeting, which is not always the case. If additional committee meetings are needed, the timelines will be extended further. While NICE are looking to expedite their timelines with the introduction of new assessments, such as the proportionate approach (4), this is not suitable for all drugs.

Accelerated approvals can be a win-win situation for everyone, provided drug manufacturers are fully aware of the limitations of their evidence package and make every attempt to fill data gaps ahead of submission to both regulatory authorities and HTA bodies.

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References

1. Sun D, Gao W, Hu H, Zhou S. Why 90% of clinical drug development fails and how to improve it? Acta pharmaceutica Sinica B. 2022;12(7):3049-62.
2. U.S. FDA CDER. Advancing Health Through Innovation: New Drug Therapy Approvals. 2022. Available from: https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/new-drug-therapy-approvals-2022#innovation. Accessed February 2024.
3. EMA. Human Medicines Highlights. 2022. Available from: https://www.ema.europa.eu/en/documents/report/human-medicines-highlights-2022_en.pdf. Accessed February 2024.
4. NICE. Proportionate approach to technology appraisals: final report 2022–23. 2023. Available from: https://www.nice.org.uk/Media/Default/About/what-we-do/PATT/PATT-final-report-2022-23.pdf. Accessed February 2024.

Value Measurement

Author: Kaylie Metcalfe (Consultant– Health Economics).

QALYs as the value measure of choice

For a long time, health technology assessment (HTA) agencies have considered quality-adjusted life years (QALYs) the measure of choice for valuing health outcomes. QALYs allow assessment of both the quality and quantity of life and are required for cost-utility analyses where the cost per QALY of two or more treatments is compared.

To determine the cost-effectiveness of a new treatment versus the current standard of care, the incremental costs are divided by the incremental QALYs. This gives us the incremental cost-effectiveness ratio, which can be compared against a willingness-to-pay threshold.

In the context of HTAs, the amount a health system is willing to pay for an additional health outcome (i.e. QALY) is different for the different agencies. For NICE, this is typically between £20,000 and £30,000 per QALY (1), whereas for the Institute for Clinical and Economic Review (ICER) in the USA, this can be between US$100,000 and US$150,000 per QALY (2).

Criticism and alternatives for QALY

Although use of the QALY measurement is standard practice for many economic evaluations used for HTA, it is not without criticism. A key concern is that a life extension for a person with poor baseline health-related quality of life (HRQoL) generates fewer QALYs than an equivalent extension of life for a person with good HRQoL. This measure may therefore be considered discriminative of people with chronic conditions, disabilities, and the elderly (3).

Increasingly, HTA agencies are considering alternatives to the QALY, and different ways of addressing such perceived discrimination. For example, ICER use the equal value of life years gained (evLYG) measure. With evLYG, equal weighting is given to a year of life added for a vulnerable patient population, and a year of life for healthier members of the community (4).

In the UK, since the publication of the new methods guide in early 2022 (1), NICE now consider the use of decision modifiers in the form of QALY weights. QALY weightings for disease severity are used – the more severe the condition, the higher the weighting. The aim of this again is to address the perception of patient outcomes being undervalued in cases where baseline HRQoL is poor. Other HTA agencies, such as Sweden’s Dental and Pharmaceutical Benefits Agency and the Scottish Medicines Consortium (5), also use decision modifiers. It is possible that such an approach may be used more widely as exposure to the use of modifiers and understanding grows.

It is clear that there are limitations with the QALY, and the conversation is ever evolving. However, we are constrained by the preferences of HTA agencies and how this plays a part in decision making. It is not a simple ‘switch’, and there are interdependencies to be considered.

For example, the use of different value measures will require reassessment of cost-effectiveness criteria, as decision makers often rely on the consideration of a willingness-to-pay threshold that is based on the cost per QALY metric.

However, the recent introduction of new ways to overcome the challenges associated with the use of QALYs has been a positive step, and it is important to be cognisant of these advances.

In the context of HTA and outside of it, it is important to challenge how patient outcomes are measured and be aware of the complete picture. Aside from what is possible to capture within a QALY calculation, what is the value of a new treatment to patients? Considering alternative value measurement methodologies and ensuring patients are engaged in these types of questions may help to build this picture.

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References

1. NICE. NICE health technology evaluations: the manual. 2022. Available from: https://www.nice.org.uk/process/pmg36/resources/nice-health-technology-evaluations-the-manual-pdf-72286779244741. Accessed February 2024.
2. ICER[HS1] . ICER’s Reference Case for Economic Evaluations: Elements and Rationale. 2023. Available from: https://icer.org/wp-content/uploads/2023/10/ICER_Reference-Case_For-Publication_Sept2023.pdf. Accessed February 2024.
3. Paulden M, Sampson C, O’Mahony JF, Spackman E, McCabe C, Round J, et al. Logical Inconsistencies in the Health Years in Total and Equal Value of Life-Years Gained. Value in Health. 2023[KM2] . S1098-3015(23)06201-0. Online ahead of print.
4. ICER. Value Assessment Framework. 2023. Available from: https://icer.org/wp-content/uploads/2023/10/ICER_2023_VAF_For-Publication_101723.pdf. Accessed February 2024.

Patient Centricity

Author: Louise Maddison (Senior Consultant – Global Market Access).

Hearing the patient voice

Patient centricity is becoming a core element of product and service development as more patients and their caregivers are now proactively seeking inclusion and transparency in the treatment decision-making process (1). A cultural shift is evolving whereby manufacturers and service leads are actively listening to what the patient needs, rather than purely focusing on the impact of the intervention on symptoms. Understanding the broader impact on patients’ lives is vital in contributing to the service or product’s value.

There is a clear need for industry to partner with patients across the value chain from research, development, distribution, and access, to co-create meaningful solutions and bring about better improved outcomes. Engaging the patient with plain language summaries, patient-focused endpoint strategy, and patient-centric clinical trials is a useful starting point.

Some pharmaceutical manufacturers are going one step further as they realise the importance of the patient voice throughout their organisation. These companies are shifting their operating models to embed patient-centric processes across product development and commercialisation (2,3).

One area of the healthcare industry that inputs the user voice upfront to co-create solutions is Medtech, with its reach into consumer and digital health. There are learnings from the development of wearables, patient apps, and healthcare devices via ‘design thinking approach’ and other methodologies (e.g. Agile), which could be transferred to pharmaceutical and service development.

Financial risks of not embracing patient-centricity

With rapidly rising healthcare costs and constrained budgets, payers are increasingly looking to reduce costs and improve both quality of care and health equity through value-based healthcare management and outcome-based payments (4). Additionally, regulators are increasingly demanding patient experience data, and health technology assessment (HTA) agencies are seeking evidence that demonstrates value to patients (5-11).

Failure to listen to patients and to collaborate on solutions they desire, rather than what you think they need, will result in products and services with limited value and uptake which in turn leads to poor outcomes. There could be financial consequences not only for the manufacturer (e.g. outcome-based agreements), but also more widely for the healthcare system, with increased healthcare resource utilisation and added pressure to already stressed systems.

Working with patients early on through compliant engagement, to hear their lived experiences, can offer valuable insights to inform disease area strategies, product development, and patient services. Coupled with broader artificial intelligence-driven insights from real-world patient data (e.g. social media, electronic healthcare records), further innovation of patient-centric solutions can be realised.

The integration of patient-centricity across all stages of the product and service development lifecycle is essential to provide treatment interventions for patients that meet their needs. Consider the impact of patient centricity in the wider healthcare ecosystem, and work with patients and partners to co-create best in class products and services. The result will be enhanced patient experience and improved health outcomes, so that patients can live better lives.

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References

1. Vanstone M, Canfield C, Evans C, Leslie M, Levasseur MA, MacNeil M, et al. conceptualizing patients as partners in health systems: a systematic review and descriptive synthesis. Health Research Policy and Systems. 2023;21(12):1-14.
2. Sharma R, Ahmed S, Campagnari J, Huff W, Lloyd L. Embedding Patient‑Centricity by Collaborating with Patients to Transform the Rare Disease Ecosystem. Pharmaceutical Medicine.2023;37:265-273.
3. Patients as Partners in Clinical Research. Novartis’ Bold Vision to Change How the Entire Sector Engages Patients. 2023. https://theconferenceforum.org/editorial/novartis-global-head-of-patient-engagements-bold-vision-to-change-how-the-entire-sector-engages-patients. Accessed January 2024.
4. Lewis C, Horstman C, Blumenthal D, Abrams MK. Commonwealth Fund: Value-Based Care: What It Is, and Why It’s Needed. 2023. Available from: https://www.commonwealthfund.org/publications/explainer/2023/feb/value-based-care-what-it-is-why-its-needed. Accessed January 2024.
5. U.S. FDA. FDA Patient Engagement Overview. 2020. Available from: https://www.fda.gov/patients/learn-about-fda-patient-engagement/fda-patient-engagement-overview. Accessed January 2024.
6. U.S. FDA. FDA patient-focused drug development guidance series for enhancing the incorporation of the patient’s voice in medical product development and regulatory decision making. 2024. https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical. Accessed January 2024.
7. EMA. Engagement Framework: EMA and patients, consumers and their organisations. 2022. Available from: https://www.ema.europa.eu/en/documents/other/engagement-framework-european-medicines-agency-and-patients-consumers-and-their-organisations_en.pdf. Accessed January 2024.
8. EMA. Patient experience data in EU medicines development and regulatory decision-making. 2022. Available from: https://www.ema.europa.eu/en/documents/other/executive-summary-patient-experience-data-eu-medicines-development-and-regulatory-decision-making-workshop_en.pdf. Accessed January 2024.
9. MHRA. Patient involvement strategy: one year on. 2023. Available from: https://www.gov.uk/government/publications/patient-involvement-strategy-one-year-on/patient-involvement-strategy-one-year-on#progress-made. Accessed January 2024.
10. European Network for Health Technology Assessment. Guidance on patient and healthcare professional involvement. 2023. Available from: EUnetHTA-21-D7.2-Guidance-for-involvement-of-patient-and-clinical-expert-in-JSC-and-JCA-v1.0.pdf. Accessed January 2024.
11. Single ANV, Facey KM, Livingstone H, Silva AS. Stories of Patient Involvement Impact in Health Technology Assessments: A Discussion Paper. International Journal of Technology Assessment in Health Care. 2019;35(4):266-272.

Precision Medicine

Author: Stephanie Swift (Senior Consultant – Systematic Review).

What is precision medicine?

Precision medicine is a healthcare approach that treats patients based on biological characteristics, such as their genetic profile. It has broad applications across a wide range of indications, including oncology, inherited genetic diseases, orphan diseases, infectious diseases, and more.

Yet achieving precisely targeted treatment outcomes often comes with a hefty price tag that restricts their cost-effective use to a small, very defined patient population.

Precision medicine for rare diseases

For rare diseases caused by specific genetic mutations, precision medicine can offer a potentially curative solution for patients who otherwise would continue to experience severe and debilitating symptoms. For example, CRISPR therapies can edit faulty genes, and gene therapies can supply functional copies of mutated genes to offer precisely targeted treatment solutions. While several recent HTA submissions for these kinds of therapies in the rare disease space have come with a list price of over £3 million, these can still be approved as cost effective due to the small number of affected patients who are eligible for treatment.

Precision medicine for oncology

In the oncology space, precision medicine can be used to target tumours based on their component genetic mutations. Many tumours share key mutations, particularly when they arise in similar tissues, like the lung or pancreas. Precision medicine can target these common mutations across patients to provide an off-the-shelf therapy based on a shared genetic profile.

Alternatively, the unique genetic ecosystem inherent to each individual patient’s tumour can be leveraged in customised precision treatments, such as adoptive T cell therapy and CAR-T cell therapy. These approaches harvest patient immune cells, manipulate them ex vivo in the lab, and reinfuse them back into the patient.

But cell therapies are also expensive – with some HTA submissions reaching a list price of more than £300,000 per infusion.

Further applications

Beyond rare diseases and oncology, patient unique genetic signatures can be targeted to provide a wide range of health improvements, from microbiome transplantation, to enhanced gut health, to predicting and preventing the development of new diseases, such as diabetes or Alzheimer’s disease. Prediction modelling, point of care diagnostics, digital health, and machine learning are all being expanded within the precision medicine space to provide clinically meaningful insights that can have a substantial impact on human health.

Future perspectives

Several global ‘grand challenge’ initiatives continue to work on bringing down the cost of precision medicines to enable broader patient access, including the establishment of genomic networks of excellence in England that will embed artificial intelligence into the personalised medicine pathway of care (1).

As the cost of genome sequencing also continues to fall, this creates a future where understanding a patient’s genetics can become a cost-effective way to drive their choice of treatment and enhance their prognosis. As the precision treatment pathway becomes cheaper, more precisely targeted treatments are likely to be recommended that improve patient outcomes on a large scale and drive a revolution in conventional treatment paradigms.

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References

1. NHS England. Genomic Networks of Excellence. Available from: https://www.england.nhs.uk/genomics/nhs-genomic-networks-of-excellence/. Accessed February 2024.

Public Health Policy

Author: Robert Hull (Senior Consultant – NHS Insight & Interaction).

Since the COVID-19 pandemic, public health policy (PHP) has been in sharper focus than ever before. Health and care systems across the globe struggle with the challenges of ageing populations, high burdens of chronic diseases, and rising costs.

These challenges are compounded by global factors such as climate change and economic recessions alongside more local issues, such as recruitment and retention of the workforce required to meet the needs of these health and care systems. Significant transformation is needed to meet these challenges, including the adoption of innovative technologies and redesigning of clinical pathways.

Public health policy in England

The NHS long term plan (1) lays out the key aims and focuses of PHP in England. Companies who can align their medicines and technologies to these key areas and other national priorities are likely to receive greater traction in the NHS and could be eligible for additional funding opportunities.

A significant area of focus for the government is the ‘Core20PLUS5’. The Core20PLUS5 lays out the key clinical areas that affect the most vulnerable adults (2), children, and young people (3) in England. There is considerable scope for innovative approaches that support these populations and clinical challenges, for companies who are able to demonstrate value and impact.

The NHS is also looking for greater use of virtual wards and artificial intelligence (AI) to enable more efficient delivery of healthcare. Virtual wards can reduce the need for people to attend comparatively expensive hospitals and instead allow them to be treated in the community (4). However, these pathways must be well evidenced and evaluated in order to be seen as suitable for adoption.

The adoption of AI technologies can allow NHS staff to be more efficient. Key initiatives include AI software to support clinicians in identifying cancers more rapidly, and clinical pathway redesign to remove bottle necks and automate administrative tasks.

Beyond immediate clinical transformation, the NHS looks to reduce its climate impact through its Net Zero agenda, recognising that climate change and carbon emissions have a significant impact on our health (5). From April 2024, all companies looking to supply to the NHS will need to publish their carbon reduction plan (previously this was only for contracts greater than £5 million) (6).

Implications for industry

It is insufficient for companies to simply align to these goals. The NHS remains under considerable financial pressure. Any new intervention must be supported by robust health economic data that are able to demonstrate a thorough understanding of the systems and pathways to be disrupted, the stakeholders who will be involved, and the costs that can be released through implementation.

Companies looking to understand these areas in greater depth should look to our NHS Insight & Interaction team and Deep Insights reports. These will help companies to understand the top priorities of NHS organisations, their measures of success and overall strategy, how to influence them, and the key language to use to ensure that communications are as impactful as possible.

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References

1. NHS. The NHS Long Term Plan. 2019. Available from: https://www.longtermplan.nhs.uk/publication/nhs-long-term-plan/. Accessed February 2024.
2. NHS England. Core20PLUS5 (adults) – an approach to reducing healthcare inequalities. Available from: https://www.england.nhs.uk/about/equality/equality-hub/national-healthcare-inequalities-improvement-programme/core20plus5/. Accessed February 2024.
3. NHS England. Core20PLUS5 infographic – Children and young people. 2022. Available from: https://www.england.nhs.uk/long-read/core20plus5-infographic-children-and-young-people/. Accessed February 2024.
4. NHS England. Virtual wards. Available from: https://transform.england.nhs.uk/information-governance/guidance/virtual-wards/. Accessed February 2024.
5. NHS England. Greener NHS. Available from: https://www.england.nhs.uk/greenernhs/. Accessed February 2024.
6. NHS England. Greener NHS Suppliers. Available from: https://www.england.nhs.uk/greenernhs/get-involved/suppliers/. Accessed February 2024.

We would love to hear your thoughts on the trends and the research initiatives mentioned above. To share your views please email info@mtechaccess.co.uk.

The post Top 10 HEOR trends – A detailed look at ISPOR’s 2024–2025 topics appeared first on Mtech Access.

We recommend that our readers across the life sciences and healthcare sectors bear in mind the following questions when considering each of the identified trends:

• What can I do now in light of these insights?
• What impact might they have on my HEOR and market access strategies?
• How can I stay ahead of the curve?
• What support do I need from agency partners?

In this article, Prof. Phil Richardson (Chief Innovation Officer & Chair) prompts some discussion points for Pharma and Medtech to explore internally. He also indicates where expert consultancy support could add value, and what to look for for in an agency partner.

Jump to:

1. Real-world evidence
2. Drug pricing
3. Artificial intelligence
4. Fostering innovation
5. Health equity
6. Accelerated drug approvals
7. Value measurement
8. Patient centricity
9. Precision medicine
10. Public health

Real-world evidence is a team sport

As real-world evidence (RWE) begins to have a more significant role in healthcare, specialists and HEOR generalists need to work together. You need access to an HEOR strategist who can help plan and organise relevant RWE. It’s also important to work with someone who understands trial design and can help put an evidence strategy together.

Drug pricing – a science or an art?

Drug pricing and working through reimbursement strategy for Pharma interventions needs a team that can bring together the art of decision making with the science of data. A close relationship with the regulators is as important here as, for instance, the ability to model QALYs. You need to be talking to someone who not only knows the HEOR space, but has taught it. In turn, this HEOR expert will need a colleague who has senior experience in the local health environment (e.g. as a payer or commissioner).

Are you ready for AI in product launches?

AI in product commercialisation is a tricky topic. There is a great deal of excitement surrounding the art of the possible, some around the art of the probable, and little in the way of what is currently practical. Faced with navigating the rhetoric and technical uncertainty, you need to think about those who have provided high-quality digital products and services to date.

Not only are they likely to be connected to the right technological solutions, but are also more likely to be in the right frame of mind to automate and build in machine learning and AI. Therefore, you need access to innovators with a digital background in HEOR delivery.

Innovation surrounds us in healthcare

We are surrounded by innovation, from continual improvement to market disruption. Innovation can work well in HEOR, but you need the right innovation partner to know when risk aversion, appearing as decision avoidance, is actually a high-risk move.

Start asking key questions of your Innovation partner, such as what proportion of revenue is committed to innovation? Is there a dedicated team? Does the Innovation Lead sit on the board? Is there a track record of innovation adoption? Are they connected to health systems as well as industry networks? Can you have direct access to the Innovation Lead and understand their product and service roadmap?

Addressing the challenge of health equity

Health equity has always been a goal. However, focus on products and pathways is not a route to addressing health equity challenges. Instead, we should focus on neighbourhoods, communities, and populations. Placing the person at the centre of decision making has finally come of age.

Agencies cannot do this on their own. They need to be closely connected to health systems, primary care, and secure health data environments. Having a greater understanding of related data, such as deprivation, is also key. If you’re working with a UK agency, ask the question – how many people from the NHS, public health, and local authorities are you directly working with?

Accelerated approvals – a win-win for all

Accelerating drug approvals and the adoption of Medtech innovations for patient benefit is the drum beat of every organisation in our industry. Speed to market and sustainable competitive advantage are the main tenets of market access strategies. Yet for some small (and larger) enterprises, it can take 7+ years for an initial tested design to reach patient hands.

As a piece in this puzzle, health technology assessment (HTA) timelines must be challenged. You need to question why decision making cannot be quicker, asking – who is focused on your access speed and the technical content of your submission?

Are you viewing value from every angle?

When considering value, you need to explore all contributing factors. Are you experiencing enough of a challenge when you mention value? Are you seeing your team exhibit divergent thinking to explore all the factors that could contribute to better patient outcomes and a better healthcare experience?

Is the patient at the centre of your thinking?

One way to drive patient centricity is to surround yourself with colleagues and consultancy partners who have this focus, who ask why the patient is not at the centre. Are you focusing on the patient, as well as the spreadsheet? Does your team have a designated Chief Patient Officer? How many patients are engaged under a patient leadership programme?

Shape the landscape for precision medicine

Precision medicine will be at its peak when we precisely understand the patient. Chasing the solution without the bedrock of personalised information is a ‘rush to standstill’ strategy. In many health systems, general practice is not connected to the nearby tertiary treatment centre. This creates a lack of cohesion, which can lead to, for example, blood tests being repeated in both settings.

It’s time to shift part of the process and digital muscle to the Market Access and Sales teams in the field, to build partnerships and help navigate the journey. You need to ask – who is already doing this? Can I volunteer my organisation to help shape the landscape, rather than just mapping it?

Public health or population health

We need to stop talking about public health and start championing population health management and the granularity of community and neighbourhood.

Strategic planning must embrace the wider determinants of care, understanding socioeconomic impact, psychosocial analysis, macroeconomics, and all the factors that determine good health outcomes for people. There is excellent information available on the wider determinants of health. The ability to understand related information and the impact on outcomes is essential. Specialised help is available in a limited number of HEOR agencies, but it is worth asking – who can help?

Turning trends into actions that deliver results

The above discussion should give you a handy checklist for the next meeting your team has with your consultancy.

Who is propelling your success and who is completing the tasks? What matters most to you in the context of the opportunities and threats that emerge within these trends? How can you best leverage the combined efforts of your in-house team and your strategic HEOR and Market Access partner?

We believe that the trends put forward by ISPOR can be more than points of interest. With this insight, leadership teams can develop responses that mitigate or take advantage of the opportunities or threats, to drive better performance and improve patient outcomes.

Finding the headroom to do this is a challenge. HEOR leaders are facing the overlapping pressures of budgeting, sales performance, workforce turnover, competing priorities, and the changing dynamic of healthcare delivery, funding, and regulation. Value and Market Access field teams are battling the need for operational performance with that of transformation for the future. Something has to give.

Receiving timely support has become ever more critical in this dynamically changing environment. Being able to quickly engage with a trusted adviser who has access to an expert team across the whole HEOR pathway is an essential component of any strategy. The creation of an adjunct team, knowledgeable about your business and capable of adapting and responding to your needs, is the innovative way to adapt and respond to change.

We believe we can be the perfect partners for these discussions. If you’d like the opportunity to explore this with our Innovation Team and our HEOR, Market Access, and Value experts, drop us an email at info@mtechaccess.co.uk.

The post ISPOR’s top 10 HEOR trends – time to take action appeared first on Mtech Access.

In all, we presented 7 independent research posters at ISPOR Europe, from 7th–9th November 2022. In this series of interviews we sit down with poster authors to learn a bit more about their research.

What topic does your poster explore?

On 1st February 2022, the National Institute for Health and Care Excellence (NICE) published updated manuals for methods, processes and topic selection for health technology assessments (HTAs). One of the changes made during this update was the replacement of the end-of-life decision modifier with a decision modifier that placed additional value on treatments that produced health gains for severe diseases.

Our poster explored whether treatments appraised by NICE prior to the introduction of the disease severity modifier might have received different recommendations had they been assessed under the updated methods.

What inspired you to pursue this topic?

I joined Mtech Access in November 2021 and, having not previously worked on a HTA, I’d had no interaction with NICE’s methods of evaluation. When the topic was proposed to me, it seemed like a good way to familiarise myself with HTAs at NICE and how the updated methods would affect my work in HTA at Mtech Access going forward.

How did you and your co-writers work together to conduct the research and develop the poster?

Dan ran searches to identify single technology appraisals (STAs) published between June 2021 and 2022 that had been developed using NICE methods from before February 2022.

Together, Dan and I reviewed the included appraisals and extracted the data needed to estimate a decision modifier for severity of disease. This included standard of care quality-adjusted life years (QALYs) and baseline demographic data. Using an online QALY shortfall calculator, I inputted the extracted data and calculated the absolute and proportional QALY shortfall, and the potential QALY weighting for disease severity.

Elizabeth provided strategic support throughout the research and development of our poster. She reviewed our research methods and provided advice on how to interpret our findings.

What was the most interesting aspect of your research?

I was interested to see the implied recommendations under NICE’s new methods. While two of the appraisals’ original most plausible ICERs were below NICE’s usual willingness-to-pay threshold of £30,000 per QALY; replacing the end-of-life criteria with the disease severity modifier would not have affected their positive recommendation.

However, three of the appraisals’ most plausible ICERs were between £30,000 and £50,000 per QALY, exceeding NICE’s usual willingness-to-pay threshold. The end-of-life criteria is likely to have been key in their recommendation. Yet, two of these appraisals’ most plausible ICER would have exceeded the willingness-to-pay threshold implied by the disease severity modifier. As such, their original positive recommendation may have been reversed under the new methods.

Were the results as you anticipated when you began your research? Were there any surprises?

I was somewhat surprised that all five appraisals that would have met the criteria for a disease severity modifier were for cancer treatments. Other severe diseases did not appear to meet the criteria for a disease severity modifier in our review.

What’s the next step for your research?

Going forward, I would like to explore appraisals further into the past to increase the number of qualifying appraisals that we could draw conclusions from. The appraisals that we reviewed were taken from a 12-month period. This only gave us 89 technology appraisal documents to review.

In addition, many of the appraisals we reviewed did not publish baseline patient characteristics or standard of care QALYs. So, we could not establish if they would have qualified for the modifier.

This led to a very limited data set. We could only evaluate recommendation decisions for five qualifying appraisals. The others in our time period either did not report these data, or did not qualify for a severity modifier.

As a result, we were unable to draw robust conclusions from our work as we had very few appraisals available to us to fully evaluate. The true impact of NICE’s new method could be explored in more depth if we could evaluate more appraisals.

What have you learnt that you can apply to future research?

I learnt the importance of thorough and systematic research through the process of developing the poster. It involved a careful evaluation of the 89 technology appraisal documents from NICE’s website to establish the applicability of the disease severity modifier. We had to pay very close attention to the details of those documents to pick up on the relevant information for our poster!

I also became much better acquainted with the process that manufacturers navigate to gain NICE recommendations for their products. STAs contain an abundance of documents detailing this process and I learnt how to navigate through these to identify the information I needed for this poster.

I think this new understanding will be of great help when working on future HTA submission projects, as I am now a lot more familiar with the process.

View the poster here.

If you would like to discuss this research with the poster authors or to learn more about how we can support you with health economic evaluation, health technology assessments and NICE reimbursement, email info@mtechaccess.co.uk.

The post ISPOR Research Poster – A Retrospective Analysis of the NICE Disease Severity Modifier appeared first on Mtech Access.

In all, we presented 7 independent research posters at ISPOR Europe, from 7th-9th November 2022. In this series of interviews we sit down with poster authors to learn a bit more about their research.

What topic does your poster explore?

The poster focuses on people that are the closest to patients and are likely to be affected by a patient’s disease or condition, i.e. informal carers.

Carers are often family members, children or parents of children, or patients’ friends. The poster evaluates carers’ health-related quality of life (HRQoL), which is likely to be affected as a consequence of living or caring for an affected patient.

Our research focused on the technology and highly specialised technology appraisals that are included in the remit of work carried out by the National Institute for Health and Care Excellence (NICE) in the United Kingdom. Our research prioritised adult populations, as previous research highlighted a low number of appraisals in which carers’ HRQoL was considered.

What inspired you to pursue this topic?

The research was inspired by a journal publication that I presented during our internal Journal Club. The journal club takes place every month and is an opportunity for Health Economics team members to share and discuss research. At each meeting, one of the team takes the lead and chooses a journal publication of interest to present. They summarise the research and present it to the team for wider discussion.

Earlier this year, I introduced a review by Pennington (2020), which was focused on carers’ HRQoL in NICE appraisals published up to 2019.

During my preparation, I was surprised by the low number of appraisals that considered carers’ well-being in conditions that affected adults only (10 out of 27 appraisals). In addition, Pennington (2020) highlighted only a limited number of sources of data for carers’ HRQoL that were used in these appraisals (two literature sources).

Therefore, I decided to pursue this topic further to report on the inclusion of carers’ HRQoL in NICE appraisals focused on adult populations published after 2019. I aimed to evaluate the number of appraisals that considered carers’ HRQoL and whether new literature sources of carer HRQoL estimates were utilised.

How did you and your co-authors work together to conduct the research and develop the poster?

I shared the research idea with Elizabeth and Catrin following my presentation at Journal Club.

I was responsible for the initial research. This involved searching for NICE appraisals, checking all company submissions and other NICE committee papers. The process required good organisation of work, and my systematic review skills were highly utilised!

Following the screening of the papers, I performed a simple data extraction of information from the appraisals that was later used for the development of the poster.

Elizabeth and Catrin were both present during all stages of the research and development of the poster. We spent many afternoons discussing the progress and my findings as well as planning the next steps for the research. Their expertise was invaluable!

For you, what was the most interesting aspect of your research?

Going through the company submissions and the NICE committee papers; it was interesting to follow the appraisal process from the beginning and notice all the discussions had along the way. This gave me a better understanding of the complexity of the NICE process, more so than simply attending NICE committee meetings.

However, there is a lack of methodological guidance for the manufacturers in situations when inclusion of carer HRQoL is appropriate. That was noticeable when going through the process of screening the appraisals, especially since the inclusion of carer HRQoL estimates can have a substantial impact on the results of the health economic model. However, it is important to note the consequences of routinely including carer HRQoL. For example, there can be difficulties accurately comparing appraisals that considered carer HRQoL estimates with those that did not.

Were the results as you anticipated when you began your research? Were there any surprises?

I did not expect any particular results; I started the project with an open mind and curiosity. However, considering the results of the previously mentioned paper by Pennington (2020), finding a greater number of appraisals focused on adult populations and any new publications reporting new estimates for carer HRQoL would be an indication of the progress in this area of health economics.

However, a couple of research findings are worth highlighting.

Firstly, company submissions that focused on diseases other than those affecting the patient’s nervous system did not often consider carers’ HRQoL. This is likely to be driven by the availability of estimates for carer HRQoL. Secondly, most papers relied on the estimates derived from a single value accepted by NICE over 15 years ago in 2006. It is unclear whether more robust evidence was published in recent years and the estimate is the best possible guess to value carers’ HRQoL.

What was your most important conclusion?

Informal care is an important aspect of the support system for patients with chronic diseases or those recovering from a disease.

Therefore, it is important to consider whether the inclusion of carer HRQoL in the health economic modelling is appropriate at the early stages of the model conceptualisation. Early assessment of the feasibility of including carers HRQoL, considering the available evidence, will allow implementation of the best methodology.

In addition, as the health technology assessment is a collaborative process, the voice of patients should be included as part of the appraisal process.

What would you like to explore next?

I believe there is a lot of potential for further research.

It would be interesting to explore whether any new sources of carer HRQoL estimates were recently published, but not yet utilised in the NICE appraisals. Pennington (2020) highlighted only two sources of estimates that were used in the NICE appraisals focused on adults, whereas our findings highlighted no new literature sources. Therefore, a systematic review looking for newly published carer HRQoL estimates, focusing on the population of the United Kingdom, would be an important next step.

In addition, it would be interesting to see whether any new literature sources have been utilised in the NICE appraisals for other populations such as paediatric or mixed populations.

What have you learnt that you can apply to future client projects?

This research has seen us develop a good understanding of the disease areas and carer HRQoL estimates that were utilised in the NICE appraisals, particularly those focused on adult populations. This knowledge will help to facilitate further discussions with clients regarding the inclusion of carer HRQoL in their health economic models.

In particular, our research has highlighted the importance of utilising a robust methodology to incorporate HRQoL estimates.

View the poster here.

If you would like to discuss this research with the poster authors or to learn more about how we can support you with health economic modelling, systematic review and/or health technology assessment, email info@mtechaccess.co.uk.

The post ISPOR Research Poster – Carer HRQoL in NICE appraisals appeared first on Mtech Access.

In all, we presented 7 independent research posters at ISPOR Europe, from 7th–9th November 2022. In this series of interviews we sit down with poster authors to learn a bit more about their research.

What topic does your poster explore?

In February 2022, NICE published updated manuals for methods, processes and topic selection for health technology assessments. As part of this, the fast-track appraisal (FTA) process, first introduced in 2017, was replaced by a simplified cost comparison process.

Prior to February 2022, a product could be assessed by FTA via two routes:

1. With an incremental cost per quality-adjusted life year gained of <£10,000 (low incremental cost-effectiveness ratio [ICER] route)
2. With efficacy and safety equivalent to existing approved treatments at lower cost (cost comparison route)

This poster reports a review of completed FTAs since 2017 and reflects on what can be learned from these FTAs for future cost comparisons.

What inspired you to pursue this topic?

Though I have been lucky enough to work on an FTA with a client, I was aware that only a limited number of medicines had successfully navigated an FTA. Indeed, I was unaware of any that had been assessed via the low ICER FTA route. As FTAs were originally expected to provide faster access to medicines via a streamlined process, I was interested to understand how many medicines had been assessed via FTA. For those assessed via the cost comparison route, I was interested to understand what we could learn from the methodologies used.

What aspects of your findings would you highlight for our readers?

I identified 16 FTAs, of which only one was assessed via the low ICER route. There was documentation available on the NICE website for 14 of the remaining 15 cost comparisons. Of these, 10 involved an economic model, with the remaining four providing a simple annual summary of expected costs.

Were the results as you anticipated when you began your research?

My hypothesis was that there were only a handful of FTAs and that the majority, if not all of these, would have been undertaken via a cost comparison route.

I was surprised to find that there was in fact one FTA assessed via the low ICER route. And, although there are a limited number of FTAs, interestingly, there seemed to be an increase in FTA appraisals carried out in the last year. Six of the 16 FTAs were published in 2022, with the remaining 10 published over the preceding 5 years.

What was your most important conclusion?

The focus of NICE committee discussions around cost comparison FTAs centred on the appropriateness of comparator selection and the available evidence for short- and long-term equivalence of efficacy and safety.

Manufacturers should therefore focus on understanding their clinical data and the most appropriate comparator for the evaluation. The cost comparison analyses themselves varied in complexity but, in general, were largely well received.

What’s the next step for your research?

It will be interesting to see how cost comparison evolves at NICE under the new methods and whether there is an increase in the number of cost comparison assessments.

What was it like presenting the poster live at ISPOR Europe?

I thoroughly enjoyed being at ISPOR this year and seeing so many familiar faces as well as lots of new ones. The poster sessions were really well attended and I met and chatted to so many different people.

View the poster here.

If you would like to discuss this research with the Elizabeth or to learn more about how we can support you with health economic modelling and/or health technology assessment, email info@mtechaccess.co.uk.

The post ISPOR Research Poster – The Evolution of Cost Comparison at NICE appeared first on Mtech Access.

In all, we presented seven independent research posters at ISPOR Europe, from 7th–9th November 2022. In this series of interviews we sit down with poster authors to learn a bit more about their research.

What research question does your poster explore?

Our poster is titled ‘The Assessment of Surrogate Endpoints and their Impact on HTA Recommendations’. In other words, we were looking to understand how HTA agencies make decisions about products that have surrogate endpoint data. Surrogate endpoints can be a laboratory measurement or a physical sign that can be used as a substitute for a clinically meaningful endpoint that measures directly how a patient feels, functions or survives. Surrogate endpoints are used in clinical trials because results can often be measured more quickly than waiting for hard clinical endpoints, resulting in earlier HTA assessment and approval of drugs.

What inspired you to pursue this topic?

We decided to pursue this topic after researching HTA methodological guidelines to determine their acceptance of surrogate endpoints as a primary endpoint.

We found it interesting that HTA markets varied in their documented acceptance of data from surrogate endpoints as part of the evidence package. Therefore, we wanted to identify the correlation between surrogate endpoint acceptability by HTA bodies and the recommendations awarded by those HTA bodies.

How did you and your co-writers work together to conduct the research and develop the poster?

As part of our work for a client company, we had previously investigated HTA methodological guidelines to determine the acceptance of surrogate endpoints as a primary endpoint. This proved a really interesting topic and piqued our interest to take the research further. From there, we decided which markets we were most interested in to identify a correlation between surrogate endpoints and HTA recommendations. This resulted in us looking at CADTH, NICE, HAS, and G-BA HTA decisions.

The NICE website and all Technology Assessments published by NICE between 2021–2022 were screened. Therapies with trial data for a surrogate endpoint as the only primary outcome and their corresponding HTA outcomes were extracted. We then searched for reviews of these therapies by CADTH, G-BA, and HAS, and extracted their HTA outcomes. All results were then compared to identify differences between HTA bodies and their acceptance of the use of surrogate endpoints.

For you, what was the most interesting aspect of your research?

I found it interesting that the acceptance of surrogate endpoints and the detail of methodological guidelines differed between HTA bodies. More definitive guidance is needed for sponsors, along with clearer documentation in HTA decision making. Currently it is difficult to determine whether surrogate endpoints will be accepted by specific HTA bodies and whether alternative options need to be explored.

Were the results as you anticipated when you began your research?

I expected to find more detailed documentation around the acceptance or non-acceptance of surrogate endpoints in HTA decisions. These weren’t always provided by HTA bodies.

What was your most important conclusion?

The use of surrogate endpoints as primary endpoints is becoming more common in clinical trials, and it’s important to continue to keep up to date with any methodological guideline updates that HTA bodies may develop in the future.

In addition, our interpretation is only based on what was documented. Acceptance or non-acceptance of surrogate endpoints may have been taken into further consideration than those documented, and other factors such as clinical benefit uncertainty, drug safety, and other aspects of study design could also have impacted HTA recommendations.

What’s the next step for your research?

It would be interesting to investigate surrogate endpoint acceptance and methodological guidance in other markets such as Australia, another mature HTA market, and the US, a as a diverse multi-stakeholder market.

What have you learnt that you can apply to future research?

We hope our findings highlight the need for clearer guidance from regulators about how companies can use surrogate endpoint data effectively so that patient-centric reimbursement decisions can be made. We also acknowledge that there is a need to validate surrogate endpoints, and we have experience in that from other client project work. We hope to conduct further work in this area to add to the body of research in the field.

View the poster here.

If you would like to discuss this research with the poster authors or to learn more about how we can support you with market access strategy and/or health technology assessment, email info@mtechaccess.co.uk.

The post ISPOR Research Poster – Assessment of Surrogate Endpoints in HTAs appeared first on Mtech Access.