In this webinar, Juliet Wallace (Senior Partnerships Coordinator) delves deep with our expert panel into the pivotal role of health economic evaluation in supporting market access for medical devices. Juliet puts audience questions to Hannah Palin (Director, Local Market Access), Calum Jones (Associate Director, Health Economics), and Evelyne Priestman (Consultant, Health Economics).

Hannah Palin

Director, Local Market Access

Hannah Palin specialises in the launch and commercialisation of new healthcare interventions. She is an expert in developing materials that communicate the clinical and economic value that products deliver within the care pathway. Hannah will draw on her experiences creating local launch strategies for a wide range of Medtech products.

Calum Jones

Associate Director, Health Economics

Calum Jones has deep expertise with early economics models to support strategic decision making, reimbursement and HTA. These types of models are a staple of Pharma market access, but are often overlooked by Medical Device teams. Calum will help us understand the value early modelling and evaluation can add for Medtech.

Evelyne Priestman

Consultant, Health Economics

Evelyne Priestman’s expertise lies in leveraging health economic modelling for the commercialisation and launch of medical devices. Evelyne will discuss the common obstacles that launch teams encounter when introducing new devices to local markets, and how health economics can generate the evidence needed to navigate these challenges successfully.

The post Navigating market access: Launch strategies and health economic modelling for medical devices – a live panel discussion appeared first on Mtech Access.

The conference organisers have curated an exceptional programme featuring thought-provoking presentations and dynamic panel sessions led by industry leaders driving market access strategy and practice. Following the opening keynote sessions, the conference will split into nine topic tracks and main plenary sessions each day. Here, we outline our top recommendations for sessions from each topic track.

Jump to your favourite topic to see our recommendations and some suggested pre-reading:

• Real-World Evidence & Data (in G102)
• Market Access (in the Auditorium)
• Pricing (in the Exhibition Hall)
• Pre-Launch Success (in G103)
• Reimbursement (in G104)
• Rare Diseases (in G105)
• Digital Transformation (in G106)
• Health Technology Assessment (in the Exhibition Hall)
• Health Economics & Outcomes Research (in G107)

Or consult the full agenda here.

Real World Evidence & Data

A key theme in the Real World Evidence & Data presentations is the use of artificial intelligence (AI). AI has the potential to expedite the generation, synthesis, and analysis of real-world evidence (RWE). The Wednesday afternoon panel at 14:30 will focus on this topic, featuring speakers from top Pharma companies such as Boehringer Ingelheim, Takeda, Sanofi, and Novartis, along with independent experts.

We are eager to hear what Lianne Barnieh (Associate Director, HEOR – BeiGene) will discuss in the last session on Wednesday at 17:45. Earlier in the afternoon, Trine Pilgaard (Director, Head of Market Access, Denmark and Iceland – Pfizer) will review the landscape and use of RWE across Denmark at 16:15. There is another dive into a local market on Thursday afternoon at 15:50, where Eva Susanne Dietrich (Professor, Departments for Clinical Pharmacy and Regulatory Affairs – University of Bonn) will explore RWE in Germany.

Thursday lunchtime features interesting sessions on using real-world data (RWD) for market access and value communication. Frederico Calado (Real-World Data Strategic Partnership Lead – Sanofi) will cover ‘Value based approaches to searching, screening and developing RWD strategic partnerships’ at 12:00. Elena Panitti (Global Director, Evidence Valuation and Enablement – Novartis) will present ‘Digitizing the integrated evidence plan, benefits and learnings’ at 12:40.

The afternoon panel at 14:45 continues this theme, bringing leaders from Sanofi, GSK, Johnson & Johnson Innovation, Dr Reddy’s Laboratories Ltd, Chiesi, and Takeda together to explore ‘From data to decisions: harnessing evidence for seamless product launches’.

Prepare for the conference with:

• On-demand webinar: Best practice RWE approaches to support economic modelling for HTA
• On-demand webinar: Engage NHS healthcare decision-makers with RWD

Market Access

The market access track offers a variety of sessions. We are particularly excited about Matt Slabbert’s (Vice President, Global Head of Public Affairs – Astellas) session: ‘Novel medicines platform: what does this mean for EU access policy’ on Wednesday at 12:25. We look forward to his insights on the long-term impact of this platform, which aims to foster collaboration and improve access to high-cost novel medicines.

The theme of multi-national collaboration continues in the Wednesday afternoon panel session. Independent experts and policy advisers will join leaders from Novartis and UCB to discuss ‘Anticipated impacts of the European Union (EU) health technology assessment (HTA) on market access’. With the roll-out of the EU Joint Clinical Assessment (JCA) this year for new cancer medicines and advanced therapy medicinal products (ATMPs), it is no surprise that this remains a hot topic.

A more surprising (and welcome) addition to the programme is Iroda Jurabekova’s (Global Director, Oncology Pricing and Market Access – Boehringer Ingelheim) session: ‘Leading an innovative culture in access teams’ on Wednesday at 15:35.

Prepare for the conference with:

• Summary and frequently asked questions (FAQs): EU Joint Clinical Assessment (JCA) – Implications for Pharma and MedTech

Pricing

The pricing agenda explores the challenges of developing fair and commercially appropriate pricing strategies from various standpoints.

For example, Malik Ait-Yahia’s (Director, Head of Market Access, Pricing and Governmental Affairs – Biogen) session approaches the topic from the perspective of emerging markets and rare disease. Join his talk, ‘Developing fair pricing to increase rare disease access in emerging countries’, at 15:35 on Wednesday in the Exhibition Hall.

On Thursday at 12:40, independent global market access expert, Neil Grubert, will cover: ‘Implications of crossborder collaborations for pricing and procurement’. Neil is an excellent speaker and very knowledgeable about the market access landscape and policy changes across key markets. His presentation is sure to be informative!

The pricing panel on Thursday at 14:45 will explore ‘Value based healthcare in theory and practice: challenges for pricing’. With panellists from Menarini, Abbott Laboratories, Novartis, BeiGene, and Dutch health insurer trade association, Zorgverzekeraars Nederland, this is set to be a thought-provoking discussion that will address real-world challenges in developing pricing strategies.

For a local, Spanish perspective on pricing, join José Luis Sánchez (Director, Market Access, Spain and Portugal – Jazz Pharmaceuticals) on Thursday afternoon at 15:50 as he explores ‘Price negotiation evolution in Spain’.

Prepare for the conference with:

• Read: What is willingness to pay? – Comparing price testing methodologies

Pre-Launch Success

Pre-launch success may seem a little ambiguous, but here, many of the presenters interpret it as “preparations and strategy for a successful launch”. For example, Olga Diachenko (Director Market Access, EMENA – Accord Healthcare) will present ‘Subnational reimbursement strategies for successful launches’ at 16:15 on Wednesday. Similarly, one of the day’s final sessions will feature Najib Slassi (Commercial Director, Gene Therapies – Novartis) presenting ‘Elevating market access to enable successful launches’. It will be interesting to see common themes between these sessions. Both sessions seem likely to discuss how global teams can best support local affiliates by providing the right evidence, resources, and propositions for local market launches and reimbursement success.

On Thursday, Tina Fischer (Head of Health Technology Assessments, Oncology – Daiichi-Sankyo) will discuss incorporating early local HTA agency feedback into strategies for wider international launches. Catch her session: ‘Incorporating early advice from HTA bodies and local levels to ensure successful international launches’ at 15:50.

Prepare for the conference with:

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

Reimbursement

Day 1 of the reimbursement sessions focuses on antimicrobials. At 14:30 on Wednesday, a panel will explore ‘The UK subscription model for antimicrobials: what’s next internationally?’. Later, at 15:35, Jennifer Quinn (Head of Global Value and Access – Debiopharm International) will discuss ‘Leveraging current reimbursement pathways to enable more equitable access for antibiotics’.

Thursday features sessions on innovation, sustainability, and affordability. At 12:00, Ad Antonisse (Director, Market Access & Corporate Affairs – AstraZeneca) will present ‘Innovative reimbursement schemes: uncertainties and performance indicators’. The afternoon panel at 14:45 is a must-see. Leaders from Novartis, UCB, Sanofi, Viatris, and Zorgverzekeraars Nederland will join Mary Lynne van Poelgeest-Pomfret (President of the World Federation for Incontinence and Pelvic Problems [WFIPP]) to explore ‘Sustainability of healthcare: reimbursement driven by affordability constraints’.

We’re also excited about Ankita Kaushik’s (Senior Director, HEOR, Global Value and Access – Gilead Sciences) session at 12:40 on Thursday, ‘Evidence generated from clinical trials to maximise reimbursement’.

Prepare for the conference with:

• On-demand webinar: Maximising NHS impact on a budget: assessing value in healthcare

Rare Diseases

Be sure to attend the sessions on rare diseases. Orphan and ultra-orphan medicines face unique market access and reimbursement challenges. Experts will share insights on launching innovative treatments in this field.

We’re excited about Ning Lu’s (Director, International Market Access and Pricing – Blueprint Medicines) session, ‘International access needs for orphan drugs: improving evidence and pricing strategies’, at 11:45 on Wednesday. Her global perspectives on evidence and pricing in the rare disease space will be fascinating.

On Thursday at 15:50, Sebastian Kessel (Senior Director, Head of Market Access & Public Affairs, Germany – UCB) will discuss the challenges and opportunities of launching multiple assets in one indication. An interesting strategic challenge!

Prepare for the conference with:

• On-demand webinar: Rare disease market access – Launching orphan drugs in secondary markets: the Nordic and MENA regions
• Four-part in-depth read: Challenges for orphan medicines entering the European market
• On-demand webinar: Rare disease market access: Strategic and tactical challenges facing orphan medicines entering Europe

Digital Transformation

Wednesday’s digital transformation sessions will focus on AI and RWD in market access and HEOR. Don’t miss Bill Malcolm’s (Executive Director, Global HEOR Economic & Predictive Modelling – BMS) session on ‘Generative AI applications in market access and HEOR’ at 11:45.

Thursday’s agenda, chaired by Health Innovation Network CEO, Rishi Das-Gupta, shifts the focus towards digital innovation in healthcare. It begins with a session on HTA for digital health technologies and the EDiHTA project at 11:55.

At 12:40, Aodan Tynan (Vice President, Global Market Access – Astellas) will present ‘Omni channel approaches to improve customer interactions through digital and other pathways’. It’s encouraging to see omni channel customer engagement being discussed.

Prepare for the conference with:

• On-demand webinar: Digital payer value communication tools – How to meet the needs of your internal stakeholders and customers
• Read: Digital options to enhance your local market access materials
• Read: Evolving market access pathways for AI-enabled Medtech in Europe

Health Technology Assessment

As expected, the EU JCA is a key topic in the HTA sessions. Wednesday’s panel at 14:30, titled ‘HTA Panel – Challenges for the implementation of the EU HTA’, will feature panellists from the French Authority for Health (Haute Autorité de Santé [HAS]), Consortium of Health and Social Care of Catalonia, and European Commission & National Agency for Regional Care Services, Italy. Eelko Den Breejen (Sr Director, Global Access Strategy & Pricing Oncology Team Lead, Portfolio & Market Engagement – Pfizer) will provide the industry perspective. This is a must-attend session!

On Thursday at 12:40, Helen Knight (Director of Medicines Evaluation – National Institute for Health and Care Excellence [NICE]) will present ‘Balancing cost-effectiveness and affordability: Developing methodological approaches to implement complex and high-volume products in the NHS’. Her insights on balancing these elements in healthcare systems will be intriguing.

The Thursday afternoon panel will revisit the topic of AI, focusing on its value in HTA. Panellists include leaders from HTA bodies, such as Dalia Dawoud (Associate Director, Research – NICE), Rosa Maria Vivanco Hidalgo (Head of HTA – AQuAS Gencat), and Oresta Piniazhko (Director, HTA Department – Ministry of Health, Ukraine). Leaders from BMS and GSK, along with Rishi Das-Gupta (CEO – Health Innovation Network) and Meindert Boysen (Independent HTA Expert & Chair – Health Technology Assessment International [HTAi] Global Policy Forum), will also participate.

Prepare for the conference with:

• Summary and FAQs: EU Joint Clinical Assessment (JCA) – Implications for Pharma and Medtech
• On-demand webinar: Artificial intelligence (AI) in clinical trials: implications for evidence synthesis and market access
• On-demand webinar: Maximising NHS impact on a budget: assessing value in healthcare

Health Economics and Outcomes Research

The HEOR agenda is varied, as expected. We’re particularly excited about Matthias Bischof’s (Senior Director, International Health Economics – Novartis) session at 16:15 on Wednesday, titled ‘Societal perspectives in cost effectiveness analyses: a comprehensive assessment’.

On Thursday at 12:40, Christina Vandorou (Director, Macroeconomics and Health Policy, EMEA –Johnson & Johnson Innovative Medicine) will explore ‘The value of investing in healthcare systems for the future’. This broad but enticing topic promises to offer valuable insights.

Prepare for the conference with:

• Quick read: What does HEOR really mean in 2025?
• On-demand webinar: De-mystifying health economic model classifications and structures

Learn more about our plans for World EPA Congress and connect with our team here.

The post Your essential guide to the World EPA Congress 2025: Top sessions and pre-reading appeared first on Mtech Access.

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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.

The post What does HEOR really mean in 2025? appeared first on Mtech Access.

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

Incorporating societal impacts into HTA decision-making

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

Expanding HTA perspectives beyond the payer

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

Bereavement disutility debate

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

Challenges in incorporating bereavement effects

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

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

Risk of unintended consequences: Productivity gains versus survival

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

Incorporating broader support structures in HTA: A patient perspective

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

Complexities of expanding HTA perspectives

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

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

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Surrogate endpoint evaluation in HTA decision-making

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

Surrogate endpoints: Filling evidence gaps in HTA

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

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

Innovations in surrogate endpoint methodology

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

Key insights: Reducing uncertainty in surrogate endpoint predictions

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

Applying surrogate endpoint analysis to my work

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

The post Insights into HTA decision-making from ISPOR Europe appeared first on Mtech Access.

Here, we sit down with Hannah Gillies (Senior Consultant – Health Economics) and Hannah Baker (Consultant – Health Economics) who, in addition to working on some of the most challenging health economic modelling projects we deliver, have recently been sharing their expertise through training.

How does your health economics training work, and who is it for?

At Mtech Access – Powered by Petauri™, we provide bespoke health economics training tailored specifically to the needs of market access teams in the pharmaceutical and medical device industries. Our approach is highly flexible, starting with a scoping session to understand each client’s unique challenges and objectives. This allows us to customise the content to ensure it's relevant and applicable to their work.

We offer both in-person and virtual training sessions, or even a combination of both, depending on the team size and location. We generally recommend holding the technical training in person, to allow for sufficient support to be given to all attendees during the practical exercises and promote full engagement.

Drawing from our real-world project experience, we aim to equip teams with the tools and skills needed to navigate the complex world of health economics. This training is particularly beneficial for market access professionals responsible for understanding economic models and utilising them in their decision-making processes.

What key topics does your health economics training cover?

Our training can cover both foundational and advanced topics in health economic modelling. We work with client teams to select the topics of most relevance to their role. We often start by introducing the basics of economic modelling, ensuring that participants are familiar with core concepts and how to work with models in Excel®. Key topics we address, all supported by practical sessions in Excel, include:

• An introduction to health economic modelling
• Techniques for dealing with uncertainty and interpreting model outputs
• Validating and interacting with economic models

For teams looking to dive deeper, we explore more advanced subjects, such as survival analysis and regression-based modelling, and specific requirements for health technology assessment (HTA) submissions around the world. These topics are particularly for market access teams involved in global projects and those preparing for specific HTA submissions, such as NICE in England.

Is the training market specific?

Whilst our training can be adapted to different markets, we ensure it is closely aligned with the specific needs of the client team. We’ve successfully delivered training in both the US and Europe, tailoring our sessions to fit the team’s responsibilities within their market.

For example, a session delivered to a US-based team might emphasise modelling requirements for Academy of Managed Care Pharmacy (AMCP), whereas for a Canadian team, we might shift the focus to Canada’s Drug Agency (CDA-AMC) guidelines. This ensures that no matter the market, the training is relevant and directly applicable to the team’s strategy.

How is the training brought to life? Can you share some examples of interactive or practical activities you use in your training sessions?

We know the best training is interactive and engaging, rather than hours of passive lecturing. Although some theoretical foundations need to be covered, we always mix up the delivery with practical exercises, particularly in Excel. We also incorporate group activities, discussions, and question and answer (Q&A) sessions to keep the content dynamic. A key aspect of our training is that we involve multiple trainers, allowing for different perspectives to keep things lively and engaging.

One of our most popular practical exercises is around calculating economically justifiable prices, a critical skill in health economics. We provide structured Excel sheets that participants can work through, giving them hands-on experience with the modelling techniques. This practical approach ensures that trainees leave the session with the skills to apply their learnings immediately.

The example below shows an excerpt from one of our popular practical exercises.

What do you think has been most valuable to the market access teams you’ve trained? What have they found most interesting and useful?

We’ve successfully delivered health economics training to market access professionals with a wide range of experience in economic modelling. Regardless of their background, participants have found our practical exercises particularly valuable. These exercises help market access managers gain the confidence to really “get under the hood” of the models they commission.

In feedback, trainees have highlighted the excellent presentation quality, engaging hands-on activities, and the expertise of our trainers, who simplify complex technical concepts with clear examples.

What do you most enjoy about delivering the training?

Hannah Gillies: Delivering health economics training is an incredibly rewarding experience. For me, one of the highlights is the practical Excel sessions. It’s great when participants have that lightbulb moment, as they start to understand how everything fits together in a real-world example. Being in the room to support participants as they work through exercises and seeing them gain confidence is always fulfilling.

Hannah Baker: I particularly enjoy the discussions that emerge during the sessions. These conversations often lead to debates or philosophical questions about the foundations of health economics, which makes the training even more enriching for everyone involved. Whilst these discussions flow more naturally in face-to-face sessions, we also make space for them in virtual training through breakout discussions.

To learn more about our health economics training sessions or commission a workshop for your team, email info@mtechaccess.co.uk.

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“It has been accepted that orphan medicinal products are not normal goods, but neither are they luxury goods that you can choose not to have. As a society, we do have an inclination to fund those most in need. Why this happens is still a matter of debate, but it has been shown that there is willingness to care for those more in need”, says Oriol de Sola-Morales, a former Director of a regional HTA Agency in Spain and founding member of the international body EUnetHTA² in The European Journal of Health Economics (2019).

In this age of rampant innovation coupled with economic recession and increasing budget pressure, healthcare system affordability is more critical than ever, especially in an area with high price-per-treatment tags. The list of the most expensive drugs is now headed by advanced, potentially curative therapies that are administered once, and which often target rare diseases. Thus, there is a significant need to model the economic benefits of such high-cost medicines and demonstrate the overall economic value to payers and healthcare systems.

Despite a greater willingness to fund and award higher prices to orphan medicines, as shown in the quote above, pharmaceutical manufacturers still need to balance affordability by healthcare systems with the capacity to recoup investments made with a smaller target population and typically highly innovative technologies.

This series explores the challenges facing orphan medicines seeking to achieve market access in Europe. In this article we discuss the third roadblock – economic evidence development.

Where separate HTA processes exist and are already adapted to the orphan medicine reality, manufacturers should aim to meet the criteria and requirements for selection via these routes. Where this is not possible, manufacturers must navigate the regular frameworks and processes, and anticipate and overcome hurdles posed by payers. The disease knowledge and awareness and clinical evidence development challenges described in Parts 1 and 2 of this series combine to make economic modelling in rare diseases particularly difficult.

Challenges in generating economic evidence

Below, Calum Jones (Associate Director – Health Economics) explains the challenges faced by health economists when looking to demonstrate the value of orphan medicines:

• If the natural history of the disease is poorly understood such that few, if any, previously published model structures can be leveraged, structural conceptualisation of the patient pathway will likely be intellectually demanding and require multiple assumptions. This may necessitate the development of a de novo Payers may struggle to accept this heightened uncertainty in the economic impact of the new orphan therapy.
• The core underlying clinical trial(s) for the new treatment may have several submission-undermining issues, including a limited duration (for what may be a chronic debilitating disease), the use of surrogate endpoints rather than true clinical outcomes, and small patient numbers leading to weakly powered trials with wide confidence intervals around estimated effect sizes.
• A heterogenous patient population can complicate the use of assumptions that apply to the whole patient cohort, e.g. identification of the minimum clinically meaningful time interval to detect differences between the new treatment and comparator (i.e. the model cycle length).
• Existing literature may provide little or no data on effective comparators for the new intervention nor on management costs for the rare disease.
• High prices for new rare disease treatments, a relative lack of standardisation in disease management, and substantial uncertainties in underlying effectiveness and cost data can often cause incremental cost effectiveness ratios to exceed commonly accepted thresholds.

Solutions to improve economic evidence generation for rare diseases

The benefits and costs associated with treating a rare disease often extend indirectly beyond the patient. Existing HTA processes infrequently consider indirect effects by default; however, manufacturers can aim to communicate significant socioeconomic benefits and costs linked to patients’ economic participation, and associated alleviation of the caregiver burden.²

Metrics for capturing humanistic and societal costs and benefits include patient quality of life, caregiver quality of life, loss of productivity (patient and caregiver), hours of formal and informal care provided, and loss of employment.

Patient registries and real-world data linked with country-relevant healthcare resource utilisation measures can also support the economic argument for a new orphan medicine where long-term data from clinical trials are often scant.

The disease pathway structure and/or accompanying assumptions for a rare disease can often be informed by previously published economic models in analogue or proxy disease areas. For example, while the evidence base for neurodegenerative disorders as a whole is growing, this is not true for distinct subsets of this disease area. Consequently, modelers exploring the cost-effectiveness of a new motor neurone disease treatment (compared to standard of care) may benefit from pragmatically leveraging previously published model structures and assumptions relating to Alzheimer’s or Huntington’s disease, while diligently outlining the inherent limitations of doing so.

Learnings taken from proxy diseases should generally be applied conservatively, with the conceptualised patient pathway and chosen model type always validated by key clinical experts experienced in the management of the rare disease in question. Rationales should be clearly stated and, if possible, referenced.

More in this series:

Read Part 1: Disease knowledge and awareness

Read Part 2: Clinical evidence development

Read Part 4: Operational Challenges

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About our panel

Dan Howard (Associate Director – Health Economics, Mtech Access)

Dan has 10 years’ experience as a technical, hands-on health economist in both consulting and industry. He has specialised in oncology and rare disease modelling across a breadth of indications, and has led or contributed to over 20 HTA submissions. Dan is also experienced in the development of indirect treatment comparisons in the context of single-armed oncology trials, which are becoming all the more common with accelerated regulatory pathways and corresponding earlier reimbursement timelines. Dan has an MSc in Health Economics and Decision Modelling from the University of Sheffield and an MPhil in Neuroscience from the University of Manchester.

Joseph O’Reilly (Principal Medical Statistician, Arcturis)

As a member of the RWE team at Arcturis, Joe is responsible for the development and implementation of statistical solutions for clients and the advancement of internal research programmes to devise novel methods for observational clinical research. He has led projects developing external control arms from RWD, which have been used to support NICE HTA submissions. Prior to joining Arcturis, Joe was a post-doctoral Biostatistician and Epidemiologist in The Institute of Genetics and Cancer at the University of Edinburgh. Joe obtained his PhD in Bayesian method development from the University of Bristol.

Nick Latimer (Analyst, Delta Hat; Professor of Health Economics, University of Sheffield; former NICE Appraisal Committee member)

Nick is a former National Institute for Health and Care Excellence (NICE) Appraisal Committee member, who has authored five NICE Decision Support Unit (DSU) technical support documents related to survival analysis, treatment switching, and partitioned survival models.

In addition to his role at Delta Hat, Nick is a Professor of Health Economics at the University of Sheffield. His current work involves investigating the use of cancer registry datasets to estimate the comparative effectiveness of treatments.

Samantha Gillard (Director – HTA, Mtech Access)

Sam has 21 years’ experience within market access and health economics, and leads the Mtech Access HTA Team. Having worked with the majority of the top 25 pharma companies and numerous medical device companies, Sam understands the varied challenges in getting a product to market and can offer strategic advice across the product life cycle. Sam has worked across a wide range of disease areas, with particular expertise in oncology, diabetes, cardiology, wound care, hip replacement, transplantation, and psoriasis. Sam has a PhD in neuropharmacology from the University of London.

Arcturis use RWD and specialise in applying innovative RWE techniques to support the discovery and development of new medicines.

Delta Hat specialise in the technical aspects of evidence-based medicine and modelling, as well as acting as external reviewers for NICE and the Scottish Medicines Consortium (SMC).

Mtech Access are experts in preparing all aspects of HTA submission dossiers and health economic models.

The post Best practice RWE approaches to support economic modelling for HTA appeared first on Mtech Access.

What led you to pursue a career in health economics?

My first degree was in Chemistry, which I studied at the University of Bristol. After that, I worked as a commercial strategy consultant in the healthcare sector for many years, nurturing a long-standing interest in pursuing another degree. During my time advising clients on critical commercial issues, I realised the importance of understanding the economic principles that drive decision-making within healthcare systems. This curiosity and desire to deepen my knowledge led me to explore the field of Health Economics, which I studied at the University of York while working for a medical technology developer.

What motivated you to join Mtech Access?

I have known Mtech Access for a while and have always admired the people there, as well as their ethos and work culture. The combination of my practical experience, academic knowledge, and appreciation for Mtech Access ultimately led me to join the team.

Here, I can leverage my expertise to help clients navigate the complex landscape of healthcare economics and strategy in an environment that aligns with my values and professional aspirations.

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

After an eventful first week, I found myself immersed in a variety of projects. My tasks ranged from developing a commercial cost calculator tool to contributing to a NICE HTA submission.

Additionally, I engaged in business development activities, facilitating several introductory calls and submitting a proposal for an evidence generation project for a major Medtech client.

The dynamic environment and diverse responsibilities have already provided me with valuable experiences and insights into the impactful work that we do here.

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

The team at Mtech Access represent an incredible wealth of expertise. The collaborative working culture here allows me to access and benefit from this extensive knowledge at any time. Beyond just what happens on individual projects, the team and Mtech Access seamlessly help each other; rather than waiting to be told what to do, people are proactive about complementing each other’s know-how and skills on every project.

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

I love what I do, and while I greatly admire the work of my colleagues, I wouldn't want to trade roles with anyone.

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

When I’m not at work, I like to spend time with my family, have dinner parties with friends, go to the theatre, and read.

To learn more about how our Health Economics Team can support your HEOR strategy, 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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