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Rory Davies – Vice President of Value and Access, Petauri Advisors, with extensive past experience in Chief Clinical roles at Cigna, Flipt, and WellDyne

Rory has over 20 years of healthcare experience, most of which has been spent overseeing formulary decisions for health plans. As the Chief Pharmacy Officer at Cigna Healthcare, he and his team were responsible for nearly $10 billion in annual medical and pharmacy spend and helped secure $750 million in savings by deploying traditional and non-traditional tools to reduce costs and improve member experience. Rory led the integration of medical and pharmacy data to demonstrate the value of medicine within the Cigna population, and he aligned the value assessment committees of Cigna and ESI post-acquisition. Rory earned his Doctor of Pharmacy at the University of Utah and resides in Chattanooga, Tennessee.

Dan Vanderpoel – Executive Vice President, Strategic Payer Marketing; Head, Client Solutions, Petauri; and former Manager, Trade Relations at Humana

With over 20 years of vast and diverse market access experience, Dan has honed his expertise through a variety of leadership roles at various payer and pharmaceutical services organizations. At Humana, Dan served on the company’s Formulary Strategy Team and P&T Committee, with sole responsibility for negotiation, implementation, and management of all Medicare rebate agreements. Dan made the transition to pharmaceutical service providers at The Hobart Group, in 2012. Most recently, Dan served as SVP, Managing Director at Precision Value and Health, where he navigated the complexities of value and access on behalf of hundreds of clients, contributing to the company’s continued growth and success.

Shana Gunderson Hua – Vice President of Value and Access, Petauri Advisors; and former Medical Affairs Executive Director at Genentech

As the Medical Affairs Executive Director at Genentech, Shana led high-impact medical and market access engagements with organized customers such as national payers, PBMs, and health systems. Prior to her tenure at Genentech, Shana was the AVP of Pharmaceutical Strategy and Contracting at Cigna and Express Scripts (now Evernorth). She spearheaded all medical rebate contract negotiations across medical benefit products, including complex treatments like cell and gene therapy, biosimilars, and medical value-based agreements. Shana also founded Innovative Pharmacy Solutions LLC, where she was at the forefront of oncology market access innovation.

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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.

What led you to pursue a career in market access?

I studied for a PhD in Developmental Neurobiology at the UCL Institute of Child Health, investigating the genetics and cellular processes involved in neural tube defects (NTDs), such as spina bifida. My research group was also interested in the mechanisms by which folate (folic acid) during pregnancy reduces the risk of NTDs, and it was the global debate around folic acid fortification of food that sparked my interest in healthcare policy and decision-making.

Outside of the laboratory, I really enjoyed the analytical and writing aspects of my PhD. As I considered a career away from academia, I discovered the world of medical writing, which led me to market access and health economics. In 2013, I joined an established market access consultancy as an Associate Medical Writer, and haven’t looked back!

How did your career progress?

As a medical writer, I had the opportunity to work on a wide variety of projects and therapy areas for both Pharma and Medtech clients. I developed global value communication materials, health technology assessment (HTA) submissions and Academy of Managed Care Pharmacy (AMCP) dossiers, local customer-facing tools, visual leave pieces, and publications. I also conducted targeted literature reviews, bespoke landscape and policy research, value proposition testing with payers and clinicians, and interactive client workshops.

As I gained experience, I enjoyed the discussions I was having with clients around content and messaging, as well as product launch strategy and positioning in different markets. Project management felt like a natural next step and my role expanded such that I was leading global projects and providing consultancy, as well as remaining actively involved in the writing side of things. In recent years, I’ve also enjoyed mentoring and training junior colleagues, and supporting business development, for example through writing proposals and developing relationships with new clients.

As part of my professional development during this time, I completed a Certificate in Health Economics and Policy at the London School of Economics and Political Science (LSE), and a City & Guilds Diploma in Leadership and Management.

What interests you most about developing global value materials?

I love the storytelling element of developing global value materials, and find it really satisfying to see these projects through from start to finish. I enjoy immersing myself in a therapy area, reviewing policy information and detailed medical data for a new product, before consolidating all of that and crafting a compelling narrative. It’s often really interesting and valuable to gather insights from external stakeholders as part of this process, to ensure the final deliverable is fit for purpose and resonates with decision-makers in key markets.

I particularly enjoy the more bespoke or challenging projects that really require us to work as part of a client’s extended team to help them navigate challenges. It could be researching a specific policy area and presenting the findings and recommendations in a concise visual slide deck, or developing a credible dossier and value narrative for a product with only limited evidence available.

Ultimately, I strive to develop high-quality, evidence-based materials that inform decision-making and support timely patient access to new treatments.

Are there any therapy areas that particularly interest you?

Yes – with my background in biology and genetics, I particularly enjoy working in rare diseases and on cell and gene therapy projects, where the need for innovative therapies is often urgent, and the pathway to reimbursement is complex. With the high number of advanced therapies currently in development, and the potential of these products to transform the lives of patients with few treatment options, it’s a really exciting area of market access to work in.

During my career, I’ve had the chance to work on rare diseases such as haemophilia, growth hormone deficiency, spinal muscular atrophy, and Duchenne muscular dystrophy, as well as rare oncology indications. As a team, we’re about to start working on an AMCP dossier for an antibody for a rare blood disorder, and I recently wrote a proposal for a suite of value communication tools for a client’s new treatment for a rare genetic condition.

What motivated you to join Mtech Access?

I was looking for the next step in my career and was keen to work across both global value communication and market access strategy projects. When the role at Mtech Access came up, it felt like a really good fit – and an exciting time to join the team, with the company recently joining Petauri™. The company’s reputation for delivering high-quality work, and its collaborative, people-first culture were also key factors in my decision to make the move.

What have you got involved with since joining Mtech Access?

It’s been an exciting few months of getting to know colleagues within Mtech Access and across the wider Petauri business. I had a comprehensive induction programme when I first started, which helped me to understand the company structure, and how the different services and supporting teams (e.g. Editorial, Marketing, and Commercial) all collaborate. I’m working mainly remotely, but it’s been great to visit our Manchester and York offices to meet and socialise with colleagues. I also attended an in-person company meeting to discuss plans for 2025.

I’ve taken a strategic lead role on a variety of projects across therapy areas such as oncology, epilepsy, chronic kidney disease, and wound care. I’ve also written proposals for a range of global value communication and market access strategy activities. It’s been really varied – every week has been different.

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

I’ve definitely learnt how welcoming and supportive everyone is! In my first week, lots of people got in touch to say hello and offer help with settling in. Everyone is approachable and always finds time to discuss things or lend a hand, regardless of seniority or how busy people are.

I’ve also been impressed with the quality of project management, and the training and best practice structures that are in place. There’s a strong work ethic and high level of professionalism; everyone I’ve met is focused on delivering excellent results for our clients.

What are you most looking forward to?

I’m looking forward to getting stuck into some exciting new projects this year, many of which involve collaboration with teams across the business. For example, I’ll be working with colleagues in our systematic literature review (SLR), Digital, and US HEOR teams. I’m also heading to ISPOR 2025 in Montreal in May, so I am really looking forward to that.

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

It would be an exciting time to work in the HTA team, particularly with the introduction of EU European Union Joint Clinical Assessments (EU JCAs) for new cancer medicines and advanced therapy medicinal products (ATMPs) from January of this year. It would also be interesting to deepen my understanding of our Digital team’s capabilities – coding is a whole different language to me!

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

I live on the edge of the Yorkshire Dales, so I try to get outside with my family for walks and days out whenever we can. When I’m not running our children around to their hobbies and parties, I try to squeeze in my weekly yoga class and the occasional swim or spinning session, or curl up with a good book.

To learn more about our expertise in global value materials and global market access strategy, please contact us here.

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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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.

Orphan medicines reimbursement cost the health systems of EU member states about €20–25 billion between 2000 and 2017. This was in addition to the EU and national public funding invested in research.¹ Such high costs, downstream of regulatory incentives, create national and local access challenges which are bottlenecks to fulfilling the promise of addressing the needs of rare disease patients across EU countries.

The market access journey for orphan medicines is complex. Nevertheless, more companies, both large and small, are entering the rare diseases market. Worldwide orphan drug sales are forecast to grow at a compound annual growth rate of 12.3% from 2019 to 2024, which is approximately double the rate forecast for the non-orphan drug market.²

Interestingly, smaller biotech/pharma organisations are overrepresented on the list of the top 20 orphan R&D products (Phase 3) in the pipeline (forecasted sales), whereas larger pharmaceutical companies tend to develop these assets at a later stage, after company acquisition or in-licensing strategies.

At Mtech Access, we are excited to be in a position to support companies looking to launch their orphan medicines in Europe, some of whom may be embarking on this journey for the first time. In this final article, I look at the operational challenges such companies may face when launching into the European market.

Operational challenges

Launch planning and preparation is especially challenging for orphan medicines, whose manufacturers can face the following hurdles:

• Given the low number of patients, a wide geographic dispersal often means there are fewer medical staff and resources than for other disease areas. For example, patients from several regions or even neighbouring countries may be treated at one or two specialist treatment centres. On the other hand, the prevalence of certain rare diseases, especially genetic diseases, may be significantly greater in particular regions or countries than in others, which may justify prioritising staff and resources to those areas. Manufacturers may even plan to launch in those countries first, even if they are typically not their priority markets.
• Engagement with payers, clinicians, and patient organisations is crucial to ensure optimal patient access at launch, particularly with rare diseases due to the disease awareness  and clinical challenges discussed in the previous articles of this series. However, manufacturers need to navigate legal and ethical hurdles in planning for these interactions. Failure to comply with best practices may result in significant damage to a company’s reputation, as has happened with some past orphan medicine launches.
• The high price/value often associated with these medicines further exposes manufacturers to increased public scrutiny.
• Finally, manufacturers need to map and navigate different national and subnational HTA processes and requirements.

This series explores the challenges facing orphan medicines seeking to achieve market access in Europe. In this article we explore the fourth roadblock – operational challenges.

Solutions to improve patient access

International collaboration and HTA is becoming more relevant and can support manufacturers looking to launch a new orphan medicine. For example, EUnetHTA, which has now published several assessments in high-cost disease areas, provides an assessment shortly after regulatory approval, thus pooling resources, setting evidence standards, and accelerating assessments and access to patients in several geographies. With the associated geographical dispersal, legal, and ethical hurdles and reputation risk, development of a thoughtful and coordinated engagement plan is perhaps more crucial than in other disease areas, as it is key to align regional and affiliate strategy and incentives with broader global strategy coordination.

Furthermore, manufacturers need to anticipate and address potential funding gaps, predicted through engagement with national, regional, and local funding bodies. For example, manufacturers can participate in horizon scanning consultations, improve forecasting, and look to create innovative payment mechanisms to address budget impact concerns. Early access programmes, conditional licensing, and managed entry agreements can all support progressive patient access. Registry-linked real-world data collection can in turn address payer concerns around clinical and economic uncertainty. For example, recent orphan medicine launches have offered retroactive rebates, deferred payments and instalment options, and outcomes-based/pay-for-performance contracts.

Finally, manufacturers can proactively engage with public bodies looking to address challenges related with orphan medicine access through public-private partnerships. For example, companies or their partners have collaborated with public health bodies in the prevention, detection, and diagnosis of rare diseases.³

More in this series:

Read Part 1: Disease knowledge and awareness

Read Part 2: Clinical evidence development

Read Part 3: Economic evidence development

The post Challenges for orphan medicines entering the European market – Part 4: Operational challenges appeared first on Mtech Access.

“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

The post Challenges for orphan medicines entering the European market – Part 3: Economic evidence development appeared first on Mtech Access.

Clinical evidence generation is a crucial stage in the market access journey for all new treatments, but can be a more significant hurdle for orphan medicines.

The characteristics of rare diseases make meeting payer evidence requirements difficult. When a non-rare disease treatment enters the market, a payer is typically presented with high quality evidence from the manufacturer, including extensive Phase 3 clinical trial data for the new treatment and comparisons with other currently available interventions. This depth of evidence is often unavailable for rare diseases.

Challenges of generating clinical evidence

Orphan drug manufacturers frequently face the following challenges when generating clinical evidence:

• Due to the small target population and the severity of most rare diseases, trial design and enrolment is challenging and far from the ideal double-blinded, comparative, parallel design of gold-standard randomised clinical trials. My colleague, Shona Lang (Director – Systematic Review), previously explored this and other related challenges in her article ‘How clinical judgement can strengthen evidence-based medicine’
• Due to the rarity and chronic nature of many of these diseases, it is often difficult to demonstrate a statistically significant impact on a mortality outcome within a randomised trial duration; thus, trials may rely on biomarkers and surrogate endpoints. For payers, this adds uncertainty around the clinical evidence, as impact on hard endpoints is key
• Rare disease indications are often heterogeneous and patients enrolled in trials may have associated co-morbidities, which, coupled with small patient numbers, further contribute to evidence generation challenges. Whilst this suboptimal level of clinical evidence is acknowledged and addressed at the regulatory level, guidance is scarce or non-existent at national and subnational payer level¹
• Manufacturers looking to develop an orphan medicine or orphan device often need to address the requirements for paediatric medicines at an early stage for regulatory approval, given that many rare diseases affect children²
• Furthermore, the regulatory environment for orphan medicines is well-established, but is less mature for orphan devices. In July 2024, the European Commission published new guidance to define orphan devices and address some of the challenges faced by manufacturers of medical devices indicated for use in rare diseases³

It should be noted that some countries have adapted their reimbursement processes to recognise these differences. A recent study found that 13/32 European countries use supplemental appraisal/reimbursement processes for orphan medicines.⁴ These allow application of different evidence requirements and thresholds (e.g. higher incremental cost-effectiveness ratio thresholds may be accepted by the National Institute for Health and Care Excellence (NICE) when products are assessed via the highly specialised technologies route) or may lead to exemption from the health technology assessment (HTA) process in some markets (e.g. Germany) if certain criteria are met. On the other hand, where specific processes do not exist for orphan medicines, manufacturers need to understand how the general processes and requirements may challenge successful reimbursement in those countries.

From January 2028, orphan medicines will be eligible for assessment under the European Union Joint Clinical Assessment (EU JCA) process. Each member state will be required to fill out a population, intervention, comparator(s), outcome(s) (PICO) survey to allow the EU JCA committee to identify national needs. The finalised PICOs will be communicated to the manufacturer prior to the dossier submission.⁵ It will be extremely challenging for manufacturers to generate evidence that satisfies all the PICOs, and national HTA bodies will continue to be responsible for reimbursement decisions. My colleague, Samantha Gillard (Director – HTA), discusses this in her article ‘EU Joint clinical assessment – implications for Pharma and Medtech’.

For orphan devices, the national HTA and reimbursement environment is even less mature. The Haute Autorité de Santé (HAS) in France, NICE in England, and Gemeinsamer Bundesausschuss (G-BA) in Germany do not have specific appraisal or reimbursement processes for orphan devices, suggesting that they will be assessed under the same pathways as non-orphan medical devices

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

More in this series:

Read Part 1: Disease knowledge and awareness

Read Part 3: Economic evidence development

Read Part 4: Operational challenges

The post Challenges for orphan medicines entering the European market – Part 2: Clinical evidence development appeared first on Mtech Access.