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But HEOR is evolving. The methodologies that underpin it are diversifying as Pharma and Medtech companies experiment with new ways to generate, synthesise, and evaluate evidence. In this changing landscape, we ask: what does HEOR really mean in 2025?

The HEOR evolution

20 years ago, ‘outcomes research’ was a catch-all phrase for studies not directly related to clinical trials. Today, systematic literature review (SLR) and network meta-analysis (NMA) have become distinct specialisms in their own right, and the term real-world evidence (RWE) has grown to encompass a broad and sometimes confusing range of activities. Likewise, where once the range of health economic model structures could be counted on one hand, now economists use a range of advanced modelling techniques from partitioned survival modelling (now commonly used to show the value of some of the most innovative cancer treatments) to regression-based modelling (where real-world data can be incorporated to better estimate relationships between variables).

Whilst these innovations have added depth to the field, they have also contributed to a dilution of the once-clear term: HEOR.

The healthcare landscape is undeniably growing in complexity, with rising life expectancies, ageing, rare, and complex populations (e.g. with genetic mutations), and increasing pressures on systems pushing decision-makers to look for solutions that address cost, value, and outcomes. HEOR is often positioned as the answer to these challenges. However, its broad scope may obscure the concrete deliverables decision-makers need.

So, what does HEOR really mean in 2025? Is it a term that's still relevant, or has it become just another buzzword?

The continued relevance of HEOR

Despite the confusion, HEOR remains a significant term in Pharmaceutical and Medtech industries. Many professionals still hold HEOR-related job titles, and the term is embedded in the vocabulary of health research.

HEOR has expanded its remit to include emerging methodologies, such as big data analytics and artificial intelligence (AI), positioning itself as a modern, data-driven discipline (we presented research on the accuracy and efficiency of automated or AI tools in SLRs at ISPOR Europe).

Even as some of its components evolve into separate disciplines, HEOR continues to capture the essence of evaluating the economic and human impact of medical treatments.

The ongoing need for HEOR outputs

The traditional outputs of HEOR, such as health economic models, NMA, and synthesised evidence, are indispensable for successful market access. These outputs directly align with the goals of Pharmaceutical and Medtech professionals: securing positive health technology assessment (HTA) recommendations, optimising pricing and reimbursement decisions, and addressing payer concerns around clinical and economic uncertainty.

For instance, in European markets, HEOR insights might focus on cost-effectiveness thresholds and health system affordability, whereas in the US, robust real-world data and patient preference studies can differentiate products for commercial payers seeking value-based care solutions. In Asia–Pacific markets, evidence often needs to address healthcare access disparities and demonstrate cost efficiency for treatments within resource-limited settings. Meanwhile, in emerging markets, HEOR may be critical in supporting priority-setting frameworks to optimise scarce healthcare budgets.

HEOR’s role in payer engagement

For Pharma and Medtech professionals, the growing importance of payer engagement makes HEOR outputs more critical than ever. Health economic models, real-world evidence, and patient-centred outcomes provide the robust evidence that payers need to make funding decisions.

Whilst HTA markets rely on systematic comparisons of clinical and economic data, payers in less formalised systems still value strong evidence that demonstrates cost effectiveness, patient impact, and affordability. Aligning HEOR deliverables to payer priorities ensures its outputs remain relevant and actionable across markets.

Our approach: Defining HEOR for today’s challenges

HEOR is often positioned as a global framework for evaluating healthcare value, even in resource-limited markets. However, such broad claims about HEOR’s global applicability may overlook the need for more specific outputs, such as context-driven analyses, priority-setting frameworks, or data synthesis tailored to local realities.

Across Petauri™, we embrace the breadth and adaptability of HEOR, recognising that its meaning can vary depending on the context. Our strength lies in the diverse range of specialisms within our team, enabling us to support clients across the entire HEOR spectrum.

Whether it’s strategically advising clients on the most relevant methods and tools, helping them cut through the noise to identify the deliverables they truly need, or producing technical outputs to the highest standard, we ensure HEOR works for today’s challenges. The challenge isn’t that HEOR is meaningless; rather, it’s that it means different things to different people – and we’re equipped to meet those varied needs.

Conclusion: HEOR as a flexible, useful framework

In 2025, HEOR may no longer be a neatly defined discipline, but its relevance is clear. For Pharma and Medtech professionals tasked with evidence generation, HEOR offers a flexible, multidisciplinary framework to tackle specific challenges, whether it’s generating economic models for HTA submissions, delivering robust real-world evidence for payers, or creating outcome frameworks that matter to patients.

Rather than focusing on what HEOR ‘is,’ the key is to align its outputs with decision-makers’ priorities. By breaking HEOR into tangible deliverables, we can demonstrate value and support impactful market access decisions.

To speak to our evidence experts about your global HEOR requirements, contact us today.

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4 FAQs about systematic literature reviews for Pharma and Medtech market access

What is the difference between a systematic literature review and a meta-analysis?

A systematic literature review seeks to gather and synthesise all relevant data, whether clinical or non-clinical, pertaining to a research question. This can be achieved through either narrative synthesis or a meta-analysis. A meta-analysis is a statistical method used to combine clinical outcome data from various sources, such as clinical trials, into a single analysis to compare interventions. Researchers often conduct meta-analyses after a systematic review has identified all relevant clinical data.

What is the difference between a targeted literature review and a systematic literature review?

A systematic literature review follows robust pre-specified methodology. It is good practice to pre-register protocols for a systematic review on a public database. These protocols will clearly outline the research question(s) to address, along with systematic literature review methods, search strategy, and any pre-planned analyses. This helps ensure transparency of the systematic literature review processes. Researchers should register protocols before extracting any data.

A targeted literature review focuses its scope and employs restricted methods compared with systematic literature reviews. A targeted review may only look at a single database and may have a restricted search strategy to reduce the number of results for a more focused analysis. In addition, single reviewers may be employed. As such, a targeted review will not be as robust, as there is an increased likelihood of missing studies of relevance or introducing bias.

Are systematic literature reviews qualitative or quantitative?

Systematic literature reviews can be qualitative, quantitative, or a bit of both. This will depend on your research question(s) and the evidence/data available. The best approach depends on what the reviewers discover while designing the review protocol and scoping the literature initially. Reviewers should clearly document the planned evidence synthesis in the review protocol and note any changes during the review.

How long should a systematic literature review take?

This depends on the research question(s) posed and the outputs required. As an example, take a systematic literature review that has 3,000 search results, with 200 results that requiring full-text screening. Of these, 35 are included for data extraction. We would expect a systematic literature review of this size to take between 12–18 weeks to complete from protocol development through to reporting.

11 FAQs about meta-analyses for Pharma and Medtech reimbursement

What is meta-analysis in healthcare?

A meta-analysis is a statistical method of ‘pooling’ data from multiple studies that have addressed a similar research question to provide a combined effect estimate for a clinical outcome(s) (e.g. complete response) from multiple data sources. Hence providing more power and confidence in the observed effect. Meta-analyses in healthcare combine clinical evidence from studies to ensure comprehensive assessment of treatment effectiveness across multiple interventions, most notably for health technology assessment (HTA).

Meta-analyses can include data from various types of studies (e.g. randomised controlled trials [RCTs], observational studies, single-arm studies). The study design determines the type of analysis required.

What is the difference between conventional meta-analysis and network meta-analysis?

A conventional meta-analysis compares two trial arms (e.g. drug versus placebo) from multiple studies in a pairwise manner. It aims to determine which treatment is most effective. In contrast, a network meta-analysis can compare multiple trial arms, even if they have not been directly compared in a head-to-head trial. Its goal is to determine the most effective treatment option.

When would you use a meta-analysis?

Researchers utilise meta-analysis when they plan to include multiple studies addressing the same research question. These studies typically involve similar populations, interventions, and outcomes. Homogeneity or similarity of the trial characteristics should be proven prior to including within a meta-analysis. Meta-analysis helps to synthesise and analyse data from these studies to draw robust conclusions.

Can you conduct a systematic literature review without a meta-analysis?

Yes, a meta-analysis is not always necessary. After conducting a systematic literature review, researchers synthesise all included data, whether that be statistically or narratively. The data can be used to support evidence generation requirements in various ways, such as incorporating these into HTA submissions.

If good head-to-head clinical trial data are available, it may not be necessary to conduct a network meta-analysis for the purpose of HTA. However, if other studies are available that consider interventions of interest, a network meta-analysis would be favourable to ensure all relevant data on the interventions of interest are captured. An economic model submitted to an HTA agency can then explore scenarios including all available data or just the data from the key clinical trial.

In some cases, data may not be homogenous meaning a robust meta-analysis, or network meta-analysis, is not possible.

What is the major advantage of a meta-analysis?

Meta-analyses provide greater statistical power by combining several studies into what is effectively one larger study. As a result, we can be more confident in the data. Some meta-analyses, such as indirect treatment comparisons (ITCs), enable comparisons between interventions where no direct comparison exists, for example, via an RCT.

What do you write/include in a pharmaceutical meta-analysis?

When conducting a meta-analysis of clinical trials, the aim is to identify how effective one treatment is in comparison with another. Statistical analyses are conducted on the data to estimate treatment effect. In general, the results you would expect to see include:

• Effect estimate: overall pooled estimate based on multiple studies included in the meta-analysis showing whether studies generally favour an intervention or comparator. Can be derived in many forms including, but not limited to, odds ratio, risk ratio, rate ratio mean difference, standardised mean difference
• Confidence intervals (e.g. 95%): highlights the variation associated with the effect estimate derived from multiple studies to see how much confidence should be applied to the observed effect
• p-value for effect estimate: indicates the statistical significance (i.e. p<0.05=reject null hypothesis; p≥0.05=failure to reject the null hypothesis) associated with the pooled effect estimate
• Heterogeneity (e.g. I2): provides insight on whether studies included in the pooled analyses differ in terms of the direction of effect observed (i.e. one study significantly favours the intervention, whereas another significantly favours the comparator)

What are the assumptions for meta-analyses and network meta-analyses?

For meta-analyses, homogeneity is a key assumption in being able to draw robust conclusions from pairwise comparisons. In the context of network meta-analysis, homogeneity is a key assumption to check there is no imbalance in the distribution of effect modifiers across the different types of direct treatment comparisons. Another important assumption to take into account for a network meta-analysis is coherence.

How many studies are needed for a meta-analysis?

Performing a meta-analysis requires a minimum of two studies. Yet, if the studies contain small sample sizes with wide confidence intervals, experts will warn against performing a meta-analysis with just two studies. This is mainly because of the imprecision observed in the effect estimate, which makes the analysis redundant for making clinical decisions about the statistically compared drugs. Many will often report two studies qualitatively and only perform a meta-analysis if they have at least three studies included.

Can a meta-analysis be qualitative?

Yes, albeit these are rare and often used in the discipline of psychology to assess key themes developing from qualitative data. These are often referred to as meta-syntheses.

What is an ITC?

An indirect treatment comparison involves conducting a meta-analysis to compare two treatments that have not been directly compared in head-to-head trials (e.g. through an RCT). Indirect treatment comparisons are conducted when a common comparator exists. Two trials may have different treatments but the same comparators, allowing that common comparator to act as an anchor. For example, where direct evidence is available for treatment A versus treatment B and for treatment A versus treatment C, the ITC allows a comparison of treatment B versus treatment C.

What are the assumptions for indirect treatment comparisons?

Homogeneity (i.e. similarity) across included trials is a key assumption for indirect treatment comparisons. In addition to homogeneity, transitivity (i.e. the ‘anchor’ comparator is similar across trials) and consistency (i.e. direct and indirect evidence are in agreement) are also important assumptions when performing indirect treatment comparisons. If these assumptions are not met, the results of the indirect treatment comparison are likely to be misleading. In the case of transitivity, if the ‘anchor’ comparator is not similar between trials, then you are introducing indirectness into the direct comparison, which is used as the platform to calculate your indirect comparison.

Do you have a different question for our experts? Send your question to info@mtechaccess.co.uk.

Our experts are here to support all your evidence synthesis, generation, and communication requirements, including all aspects of systematic literature review and network meta-analysis. We also offer education and training on key principles of evidence synthesis. To learn more, email info@mtechaccess.co.uk.

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This article was updated in November 2024, with reflections on the latest information about the EU JCA and how industry is responding.

Jump to:

• What is the EU JCA?
• When will the JCA be a requirement?
• Will the JCA replace local HTA?
• JCA methods and guidelines
• Who conducts and assesses the JCA?
• The JCA scoping process
• Four key considerations for Pharma and Medtech
• How can Mtech Access & Petauri™ provide support?

What is the EU JCA?

In January 2022, the European Parliament enacted a new EU Health Technology Assessment Regulation (HTAR), with the main aims being to:

• Standardise clinical assessment of medicines and high-risk medical devices across the EU
• Avoid duplication of work
• Improve access to innovative health technologies to patients across Europe
• Strengthen the quality of health technology assessments (HTAs)

Currently, Pharma and Medtech manufacturers have to submit evidence individually in each of the 27 EU markets; the intention of the JCA is that at least some of the assessment can be done centrally.

The JCA will provide an assessment report, which acts as a universal information source for relative treatment effects, safety, and other clinical parameters agreed in the scoping process. The report will also cover the clinical area addressed by the technology and its technical characteristics. A summary report will be developed. Both reports will be based on scientific, clinical, patient, industry, and HTA body expertise.

The introduction of the JCA should benefit smaller markets, which may not have the required capacity or expertise to conduct assessments independently.

When will the JCA be a requirement?

The JCA will be rolled out in stages. New cancer medicines and advanced therapy medicinal products (ATMPs) will require a JCA from 12th January 2025. Orphan medicines will require assessment at EU level from 13th January 2028. Finally, all other new medicines launching in the EU will need a JCA from 13th January 2030.

Will the JCA replace local HTA?

The JCA is not a full HTA and is focused only on clinical evidence. It is not an appraisal, and local agencies reserve the right to draw their own conclusions on relative efficacy and safety.

Whilst all member states will be required to give each JCA “due consideration”, each state will be able to request additional information relevant to their market. Cost-effectiveness and reimbursement decisions will be made at national level.

JCA methods and guidelines

The voluntary EU HTA cooperation, EUnetHTA21, a consortium of 12 European HTA bodies, were contracted to develop guidance documents to support the EU HTAR.

EUnetHTA21 developed HTA methodological guidelines, including guidance on scoping, evidence synthesis, endpoints, validity of studies, etc. Whilst these guidelines are written for the assessors and co-assessors, Pharmaceutical and Medtech companies can also learn from the information provided.

JCA guidelines published by EUnetHTA21 include::

• JCA submission dossier guidance and template
• JCA assessment report guidance and template
• JCA procedural guideline for appointing assessors and co-assessors
• Production of JCA on medical products and medical devices

In September 2023, the Member State Coordination Group on Health Technology Assessment (HTACG) was established to oversee future joint EU HTA work. HTACG have published the following two pieces of guidance:

• Guidance on outcomes for joint clinical assessments
• Guidance on reporting requirements for multiplicity issues and subgroup, sensitivity and post hoc analyses in joint clinical assessments

The guidance on outcomes for JCAs will help member state authorities define the scope of the JCAs and help assessors report all elements that member states need for the national appraisal of the clinical added value of health technology.

The guidance on reporting requirements for multiplicity issues and subgroup, sensitivity, and post hoc analyses in joint clinical assessments describes how assessors should deal in practice with multiplicity issues and complementary analyses in JCA reports.

Who conducts and assesses the JCA?

The JCA process will be conducted by the HTACG. The HTACG comprises four subgroups, with two (Joint Clinical Assessment and Joint Scientific Consultation) directly responsible for the delivery of JCA reports. One subgroup will be responsible for the identification of emerging health technologies, and another will focus on methodology.

The JCA will be conducted by representatives from the designated subgroups who will be responsible for all JCA activities and timelines, as well as collecting input from regulatory agencies, clinicians, and patients.

For each technology requiring assessment, the JCA group will appoint two member states, one leading and one supporting, who will be responsible for assessing the manufacturer’s dossier and producing the JCA report. The appointed member states will liaise with the other member states as required.

The JCA scoping process

To inform development of the assessment scope, the manufacturer will submit elements of their European Medicines Agency (EMA) application, namely the summary of product characteristics and clinical overview. Based on this information, assessors will prepare a proposal for the assessment scope in terms of population, intervention, comparators, outcomes (PICO), which has the aim of specifying the framework for assessment and the evidence requirements the manufacturer will need to satisfy.

Each of the 27 EU member states will be required to fill out a PICO survey, to allow them to identify national needs. Following the survey, the JCA assessor and co-assessor will consolidate all PICOs, which will be validated by EU patients and clinical experts, as well as the Committee for Scientific Consistency and Quality (CSCQ).

The finalised assessment scope will be communicated to Pharmaceutical and Medtech manufacturers, from which point they have 90 days to develop and submit the JCA dossier. Submission of the dossier is required at least 45 days prior to publication of the Committee for Medicinal Products for Human Use (CHMP) opinion.

Four key considerations for Pharma and Medtech

Manufacturers can expect to face various challenges throughout the JCA process, including:

1. Different national requirements

EU member states will be able to request additional information, such as cost-effectiveness evidence, during the JCA process. Yet, additional clinical information will still be required at national level and this is where reimbursement decisions will be made. Additionally, there may be differences in accepted methods for evidence synthesis between the EU member states. Presumably, individual member states will still be able to request the same evidence as at EU level, but analysed differently.

2. Risk of needing to repeat submission following CHMP opinion

The JCA dossier submission needs to be made prior to CHMP opinion (at least 45 days prior to publication of the opinion). Since aspects of PICOs may change following CHMP opinion, there is a risk that the whole JCA process will need to be repeated.

3. Potential for an unmanageable number of PICOs

Even though PICOs at national level will be consolidated, there is no cap on the number of consolidated final PICOs. The definition of standard of care and patient populations are likely to vary across member states and so there is a risk of an unmanageable workload for manufacturers.

4. Limited contact with assessors

Manufacturers will have limited input into the JCA process. As the JCA is not an appraisal, and reimbursement decisions won’t be made, it is understood that the EU assessor and co-assessor’s contact with manufacturers will be limited to requesting clinical evidence if missing from the JCA submission dossier.

Despite the intention to avoid duplication of efforts, the four challenges above are likely to increase the workload for manufacturers.

How can Mtech Access & Petauri™ provide support?

We can help Pharma and Medtech companies with strategic considerations, evidence synthesis, and dossier development for their JCA submissions.

We are HTA experts and our team have collectively completed more than 170 HTA submissions, with a 100% success rate in oncology and rare diseases. We work closely with our clients, becoming an extension of their teams, to ensure the value of their products is appropriately communicated. We provide continuous strategic support throughout to maximise the chances of successful reimbursement, whilst project timelines are closely monitored for timely delivery.

At Mtech Access – Powered by Petauri, we have specialist teams of Systematic Reviewers and Statisticians who consistently deliver evidence synthesis according to the most robust standards, across all disease areas. Several of our Evidence Team were previously members of an Evidence Assessment Group for the National Institute for Health and Care Excellence (NICE) and use this experience to ensure that our systematic reviews adhere to HTA requirements and are of the highest quality. In addition, our large in-house team of specialist Medical Writers are experienced in writing reimbursement and HTA submission dossiers. All of our submissions undergo a thorough proofing process by our Editorial Team, who offer quality assurance.

Please email info@mtechaccess.co.uk to discuss how we can support you with your JCA dossier.

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What led you to pursue a career in evidence synthesis and statistics?

Prior to joining Mtech Access, my career was deeply rooted in academia and healthcare research. I began by earning a bachelor’s degree in Mathematics from the University of Liverpool. This was followed by a Master’s degree in Medical Statistics from Lancaster University. Later, I completed a PhD in Medical Statistics from the University of Liverpool.

My first postdoctoral position was at the Centre for Reviews and Dissemination (CRD) at the University of York. There, I worked on mixed health technology assessment (HTA) submissions. These included single technology appraisals (STAs), highly specialised technologies (HSTs), and multiple technology appraisals (MTAs), as part of an Evidence Review Group commissioned by the National Institute for Health and Care Excellence (NICE).

6 years ago, I moved to Manchester for a second postdoctoral position, which shifted my focus towards primary care research. In this role, I evaluated interventions through network meta-analyses (NMAs) and explored causality. I also worked on workforce and exposure health data science projects using Hospital Episode Statistics (HES) data with the National Health Service (NHS), as well as rich primary care record data from the Clinical Practice Research Datalink (CPRD). My research also addressed important public health issues, including the incidence of self harm among patients and the impact of burnout on primary care staff in the UK.

With extensive experience in evidence synthesis, my research has been particularly focused on long-term conditions, digital health, and interventions aimed at improving the quality of care, and the mental health and wellbeing of healthcare workers.

What motivated you to join Mtech Access?

I spoke to many people in the market access field and consistently heard great things about Mtech Access. It is widely regarded as a leading organisation for market access and HEOR in the UK. At one point a few years ago, I had considered joining Delta Hat, which has since become a Petauri™ partner along with Mtech Access.

What drew me to Mtech Access was how well the organisation aligns with my skillset. Their collaborative approach across the specialist teams resonated with me. My strong quantitative background, particularly my statistical expertise, means that I can directly contribute to a wide range of projects to help clients navigate the complex technical requirements needed to achieve successful NICE approval.

How will joining Mtech Access help you achieve your career goals?

I’m excited to make the transition from academia to a commercial consultancy. I’m looking forward to working on a broader range of projects and eager to apply advanced statistical methodologies to tackle complex questions. The opportunity to work across various specialisms, including Health Economics, Systematic Review, and NHS Insight & Interaction, is something that I’m particularly looking forward to. Collaborating with multidisciplinary teams in these areas promises to be an enriching experience.

I also anticipate numerous opportunities to both provide training and develop new skills, learning from the different service sectors within the company. Moreover, the potential to innovate and create tools that streamline work processes across the three teams is something that I find particularly motivating.

What have you been involved with in your first few weeks with Mtech Access?

I’ve attended numerous induction meetings to get to know the team and quickly gained exposure to several exciting projects. This includes work on an oncology treatment, where I was involved in matching-adjusted indirect comparisons (MAICs) and propensity score analysis. I have also been working on NMAs evaluating interventions for Parkinson’s disease, as well as contributed to several proposals during this time.

Collaboration with our new US-based Petauri partners, The Kinetix Group, on a proposal has been another highlight.

It’s been great to get to know the team through socials too. I attended a Manchester office social featuring axe throwing and darts. I also had the chance to meet the wider team during an all-company meeting in my first week.

What have you learnt from your first interactions with the team?

The team has been incredibly positive, bringing a wealth of expertise across a wide range of skills. My new colleagues are supportive and always willing to help whenever approached, fostering a collaborative atmosphere.

In turn, I’ve received numerous statistical queries, such as requests for explanations on how vendors model longitudinal data, as well as non-statistical questions, including advice on Delphi consensus techniques.

What has most surprised you about Mtech Access?

The news about joining Petauri came as a bit of a surprise, but it’s an exciting development that promises a wider range of projects. The induction process was well-structured and organised, and the People/HR Team have been incredibly helpful throughout.

I’m particularly impressed by the diverse resources and expertise within the team, which offer great opportunities for growth. I’m especially looking forward to learning more about Connected Insights from Iain Shield – his recent showcase webinar was illuminating. Working alongside the Medical Writing and Editorial Teams has also provided further insights into technical writing and styles, which I’ve enjoyed exploring further.

If you could swap your expertise and role with a colleague from another team, who would you swap with?

Global Market Access – I’ve found learning about the team particularly fascinating. I am especially interested in how we support pricing strategies across different countries.

When you are not at work, what do you like to do?

I like to spend my free time with family, often mountain biking, likely in North Wales, or taking part in yacht or dinghy racing on a lake or reservoir.

To learn more about how our Evidence Synthesis Team can support your HEOR strategy, please email info@mtechaccess.co.uk.

If you are interested in joining our team at Mtech Access, please visit our careers page for details of our current vacancies.

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Jump to the 7 steps.

What is a systematic literature review?

A systematic literature review (SLR) (often shortened to systematic review [SR]) is essentially a thorough analysis of all relevant research on a specific topic. True systematic literature reviews must be comprehensive, transparent, and reproducible.

A systematic literature review identifies, appraises, and summarises literature to answer a prespecified question through a clearly defined protocol. Reviewers systematically screen and analyse published evidence from multiple sources to identify relevant studies and extract data.

How are systematic reviews used in healthcare?

The health and life sciences industry use systematic reviews to evaluate the efficacy and safety of new medicines, diagnostics, and medical devices. Systematic reviews provide a comprehensive synthesis of existing research, helping stakeholders to make informed decisions about market access and regulatory approval.

Systematic reviews offer reliable evidence-based information to support informed choices and practices. By aggregating data from multiple studies, systematic reviews offer a robust foundation for assessing the effectiveness and cost effectiveness of treatments.

With systematic literature reviews seen as the gold standard for evidence-based medicine, decision-makers, from regulatory agencies to payers and health insurers, expect to see a systematic review in your evidence dossier.

Systematic reviews are often published in peer-reviewed academic journals. These publications serve as authoritative references for payers, policy-makers, and other stakeholders involved in healthcare decision-making. Publishing evidence is often a key step in the market access journey.

The 7 steps of a systematic literature review

There are several published guidelines, such as those issued by the Cochrane Collaboration®[1] and Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA statements) [2], which provide guidance on conducting and reporting a robust systematic literature review.

The process of conducing a gold-standard systematic literature review typically involves 7 stages:

1. Protocol and registration
2. Electronic database search
3. Title and abstract screening
4. Full publication review
5. Hand searching
6. Data extraction and quality assessment
7. Report

Step 1: Protocol and registration

The first step in a systematic literature review is to write the protocol. The protocol must clearly define the study question and describe the methodology to be used.

The reviewer should begin the protocol by describing the background, aim, and purpose of the systematic literature review. They should also clarify the study question.

The protocol should then define the eligibility criteria used to assess each publication. This is a list of inclusion and exclusion criteria that indicates whether a publication is eligible for inclusion in the systematic literature review. The eligibility criteria specify the Population, Intervention, Comparator, Outcomes, and Study design (PICOS). These criteria can also specify certain factors, such as the publication date, language, and location of the included studies.

The protocol should also state the methodology intended for each stage of the review, as this ensures that the systematic literature review is robust and reproducible, and conforms to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses Protocols (PRISMA-P) checklist [3].

After writing the protocol, reviewers should register the systematic literature review with the International Prospective Register of Systematic Reviews (PROSPERO) to prevent duplicate reviews and facilitate potential future publication.

Step 2: Electronic database search

The reviewer conducts an electronic database search to identify all potentially relevant publications that should be included in the review. Each search should be specifically designed to reflect the PICOS. The search parameters are specifically designed to capture all relevant literature, with a balance of sensitivity and recall, so that large numbers of irrelevant records are not retrieved.

The search should interrogate all relevant databases, such as PubMed®, Embase®, MEDLINE®, and the Cochrane Library.

All search sources, methods, and strategies should be included in the report to ensure that the search is transparent and replicable.

Step 3: Title and abstract screening

Step 2 should provide the reviewer with a list of published research that may be relevant to their study question. As this can be quite a long list, the first step is to screen the results using the title and abstract.

Reviewers screen the title and abstract of identified citations to determine if they meet the eligibility criteria outlined in the protocol. Publications may be excluded based on various factors, including the following:

• Disease is not relevant to the study question
• Intervention is not relevant to the study question
• Duplicate publication to another in the search
• Study design is not relevant in this case
• Animal/in vitro study, if study question is on human health (or vice versa)
• The publication reports the protocol only, with no results
• Linked to another publication in the list (such as a conference abstract that has been superseded by a full journal article)

At this stage, citations should not be excluded solely because they do not report a relevant outcome in the abstract, as the full publication may contain additional outcomes not mentioned in the abstract. If it is at all unclear as to whether a publication meets any of these criteria, then it is typically included. For example, it may not be easy to identify all relevant outcomes or subgroups from an abstract, so the publication will be included at this stage.

To prevent reviewers from overlooking any relevant citations, two separate reviewers should independently screen the title and abstract.

Step 4: Full publication review

In Stage 4, reviewers proceed to assess any studies identified as potentially relevant during the title and abstract screening through a full publication review. This involves a complete review of each publication to determine if it meets each of the eligibility criterion, with a particular focus applied to the Methods and Results of each publication. At this stage, reviewers can exclude studies that do not have relevant outcomes. Similarly to title and abstract screening, this should be conducted by two independent reviewers.

During this stage, it maybe be helpful for reviewers to capture certain study outputs, such as study design, number of participants, trial location, and reported outcomes. Mapping these outputs helps to identify publications of the same study and prioritise publications for data extraction. It is particularly useful for systematic literature reviews that are focused on real-world evidence (RWE), as this can identify a large number of relevant publications.

Step 5: Hand searching (and reference checking)

In addition to the database search, reviewers should conduct ad hoc searching of supplementary sources. To do this, the reviewer should search conference abstracts from relevant conference proceedings from the past 3 years using key terms.

It is also important to search through references of the eligible studies already identified. References may contain other relevant studies that should be included in the new systematic literature review.

When using the systematic literature review to support a reimbursement submission, it's beneficial to explore various global health technology assessment (HTA) websites for relevant submissions. This can yield valuable insights and potentially unique outcomes data.

Step 6: Data extraction and quality assessment

The reviewer then extracts data from the relevant identified publications into a data extraction table (DET). The DET captures details of each clinical trial, most commonly:

• The study design of each clinical trial, such as the number of participants enrolled, the eligibility criteria of the study, and information about treatment
• Baseline characteristics of enrolled participants, such as age, sex, disease duration, and disease-specific characteristics
• Outcomes of interest
• Risk of bias

If the DET will be used in a subsequent meta-analysis, it is crucial to design it in a way that supports this.

In some systematic literature reviews, it may be appropriate to extract data from the publication directly into the final report, rather than into a DET. This may be appropriate in a smaller systematic literature review, or for ones with limited outcomes of interest.

Simultaneously, reviewers should conduct a quality assessment of each study to evaluate potential sources of bias. Various validated tools and checklists are available for appraising and assessing the risk of bias in studies. The most appropriate assessment will depend on your study design.

Step 7: Report

The final stage of a systematic literature review is writing the report. The aim of the report is to answer the study question defined in the protocol. A systematic literature review report will typically include the following sections:

• Executive Summary
• Introduction and background
• Aims and objectives
• Methodology
• Result tables and summary of findings
• Discussion
• Conclusion

The report should follow the guidance provided by PRISMA, including the PRISMA diagram. This diagram details the number of citations identified during the database searches and handsearching, and the number of citations included and excluded at each stage of the review, as well as the reasons for exclusion.

The methodology outlined in the report should align with the methodology described in the protocol.

The discussion should describe how the results have answered the study question. It should also address the robustness of the evidence base, outline any data gaps, and recommend areas for future research.

The final report will then be ready for inclusion in your evidence dossier. It can be used in global value dossiers and reimbursement dossiers for HTA submissions. Most reports are also submitted to reputable journals for publication.

Conducting a systematic literature review

Do you have a question about the systematic literature review process? Are you looking for an expert to develop an systematic literature review to support your market access and reimbursement strategy? To speak to our Evidence Synthesis Team, email info@mtechaccess.co.uk.

References:

The post 7 steps of a systematic literature review appeared first on Mtech Access.

What led you to pursue a career in market access?

I come from a science background, having studied Biochemistry at the University of Oxford. My course was a 4-year combined undergraduate Master’s, with a year in the lab. I quickly found that lab work wasn’t for me, but I still wanted a job based in science, just outside of the lab.

I found a job working for a boutique consultancy, where the bulk of the work involved primary and secondary (desk-based) research. Our research informed market assessments and product evaluations for assets that were in the early stages of development.

From there, I moved to a broader market access consultancy, working on a wide variety of projects across the product lifecycle. I particularly enjoyed the medical writing and value communication side of things, which led me to where I am now!

As a Medical Writer, what do you most enjoy working on?

I would say that manuscripts are probably my favourite. I love the challenge of writing a condensed piece that fully communicates the study and implications for the therapy area or healthcare system.

Most of my experience is in writing manuscripts, global value dossiers (GVDs), and value propositions. I’m excited to get involved in more health technology assessment (HTA) submissions at Mtech Access, as there’s such a wealth of knowledge and experience in the team.

What motivated you to join Mtech Access?

I was keen to specialise in medical writing and progress in a technical role, rather than taking on a broader project management consulting role as my career advanced. Mtech Access have a specialist Medical Writing Team, with a more technical career pathway. This means that I can stay involved in the writing and data.

Through the interview process, it was clear that Mtech Access had a really friendly atmosphere within the team. They also offer flexible working and great benefits. It was a no-brainer really!

What have you been involved with in your first few weeks at Mtech Access?

There have obviously been a handful of induction sessions to introduce me to the company and ways of working. I’ve had a mix of meetings with different members of the team, along with some hands-on tasks to immerse myself in Mtech Access’ house writing style.

I was able to get involved in some project work early on too. I’m currently working on a manuscript and conference poster. These report the results of a systematic review and meta-analysis conducted by the Mtech Access Evidence Team.

In the next few weeks, we’ll also be kicking off a project working on a formulary pack and value proposition for a client’s diabetes treatment.

Outside of the project work, I’ve been involved in a marketing piece looking at Mtech Access’ view on ISPOR’s top 10 HEOR trends for 2024/25. It has been interesting to work on something a little different, and to read the perspectives of some of our experts in the team.

I’m home-based most of the time (it’s great to have that flexibility!), but my closest office is Manchester. I’ve managed to make a few trips in to see the team there. We’ve had a team lunch and there’s also another social planned soon. Everyone has been super welcoming!

What has surprised you most about Mtech Access?

Thankfully there haven’t been any huge surprises, as they were very transparent throughout the interview process! The culture communicated on the company website is very ‘people first’, and indicates a company aiming for an ‘egoless’ organisation. I’ve been pleasantly surprised at how much this is actually reflected in reality. Everyone is so open to helping with any queries and sharing knowledge.

If you could swap your expertise and role with a colleague from another team, who would you swap with?

I’m really spoilt for choice on this one, as there are so many different areas of expertise in the company. I think I’d have to choose the Health Economics Team. They work with such complicated technical models, and the work they do is very impressive.

I’d also swap with the NHS Insight & Interaction Team. A lot of my previous experience has been with Global and US-based clients, so I’d love to have their knowledge on the UK market.

When you are not at work, what do you like to do?

I have a 1-year-old daughter and a 4-year-old toy labradoodle, both of whom keep me very busy outside of work! Hobby-wise (when I have the time) I love anything creative – painting, calligraphy, photography, and also baking.

Would you like to learn more about our Medical Writing expertise and how this is utilised across global value dossiers, reimbursement and health technology assessment dossiers, and local communication materials? If so, please email info@mtechaccess.co.uk.

Are you interested in joining our team at Mtech Access? Visit our careers page for details of our current vacancies.

The post Joining a specialist Medical Writing Team – an interview with Hayley Shoel appeared first on Mtech Access.

What led you to pursue a career in HEOR?

My undergraduate degree, Economics and Mathematics at the University of Liverpool, introduced me to Health Economics. The content of this module was so fascinating that I was inspired to pursue a career in this area.

This led me to work for the NHS as a Data Analyst at a Mental Health Trust. Then, after taking a career break to travel, I decided to develop my knowledge of HEOR by completing an MSc in Health Economics and Decision Modelling at the University of Sheffield. It was at this point that I moved into consultancy, where I have been for 8 years.

I worked as a Health Economist, whilst also having a focus on evidence projects. This involved building and reviewing health economic models, conducting systematic literature reviews, facilitating structured expert elicitation, sourcing country-specific data, and reviewing submissions for NICE, SMC, NCPE, and AWMSG. I was also an assessor for the SMC.

What motivated you to join Mtech Access?

After 8 years of working for a small consultancy, I felt it was time for a change. I was ready to explore the next step in my career and wanted to experience what it was like working for a larger company. However, I appreciated the values of the smaller, family-run company that I was used to. Luckily, I found this middle ground between the two at Mtech Access, where the ethos is very much about putting people first.

I was also conscious of wanting to join a company where there was emphasis on ‘quality over quantity’. I felt Mtech Access had this positive reputation.

Whilst I was sure that I wanted to join Mtech Access, I wasn’t quite sure where I’d best fit in, as my background covers health economics, evidence, and market access. Chris Costello (Talent Acquisition Manager, Mtech Access) helped me find the right role that would suit me.

What have you got involved with in your first few weeks with Mtech Access?

I’ve had lots of training across all aspects of the company. It has been great to be introduced to the different people and teams in Mtech Access. This has helped me gain an understanding of how the company fits together as a whole, and become familiar with all the different faces. It has been a useful process, and it was nice to do the onboarding with some other new starters, so we weren’t on our own during the process.

Before I’d even started at Mtech Access, I felt very welcomed. I attended both the whole company Christmas party and the Manchester office Christmas social. It was there that I met my team and lots of others, before joining in the new year.

Following the initial 2-week onboarding, and around ongoing training, I’ve also started to get stuck in with projects. I’ve been working on systematic literature review updates in oncology and blood disorders.

What has most surprised you about Mtech Access?

I have found it really reassuring to have all the training. It feels like there is a really supportive culture. The values on the website are all accurate. Everyone is really responsive, happy to help, and on the ball!

It’s a nice atmosphere, everyone seems happy and positive, and overall it’s exceeded my expectations.

If you could swap your expertise and role with a colleague from another team, who would you swap with?

A role in the Commercial/Marketing Team would be interesting. Their work is so varied and covers an array of different outputs and topics. They’re also able to collaborate with colleagues across all aspects of the business.

When you are not at work, what do you like to do?

My 2-year-old boy keeps me busy and well-entertained. I also enjoy anything active: netball, CrossFit/Metcon classes, cycling, swimming, and walks with our dog; as well as watching sports and socialising with friends and family.

As a family, we also love to travel. Last year we completed a ‘bucket list’ trip to Sri Lanka and the Maldives with friends, which was a dream come true. This year, we’re going to Bali and Australia. We also enjoy exploring the UK and Europe. The dream would be to have a campervan, so we can go on more trips like this!

To learn more about our Evidence Synthesis and HEOR services, please email info@mtechaccess.co.uk.

If you are interested in joining our team at Mtech Access, please visit our careers page for details of our current vacancies.

The post A people-first consultancy – joining the Evidence Team 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.

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