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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.

The post Communicating value to US Payers: Insights for Pharma and Medtech 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.

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.

The post Global value and strategy: Sophie Streeton on her career, passions, and joining Mtech Access appeared first on Mtech Access.

The post Meet our experts at World EPA Congress 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.

There are an estimated 7,000 rare diseases worldwide for which there is no current treatment, affecting up to 30 million people in Europe and 300 million people wordwide.¹ “Rare diseases are not so rare” is indeed a suitable slogan repeated by advocates throughout the world to highlight the collective importance of this group of diseases.²

When exploring individual treatments, the rarity of rare diseases is a challenge. Rare diseases affect a small proportion of the population (<1/2,000 people in Europe), usually from birth or childhood, and are often severe.³  With such small populations, gathering disease knowledge and raising awareness can be a challenge with rare diseases.

Any medicine or device should have a pricing and market access strategy that is underpinned by a good understanding of the target patient population, including the current and anticipated future treatment pathway, epidemiology, patient subgroups and severities, and unmet needs. Consequently, gathering disease knowledge and raising awareness of patients’ needs is the first hurdle facing manufacturers looking to launch an orphan medicine or orphan device.

This series explores the challenges facing orphan medicines seeking to achieve market access in Europe. In this first article we focus on the first roadblock – disease knowledge and awareness.

Challenges in disease knowledge and awareness

Market access teams looking to launch an orphan product in Europe often find that:

• Disease background data (including treatment pathway, patient subgroups, and epidemiology) are scarce and the science is typically complex
• The unmet need is difficult to define because of the small patient population, diagnostic challenges, and patient heterogeneity
• The relatively low proportion of patients in each market typically make the disease a lower priority for regulators and payers versus more common disease areas
• Clinical research organisations may need to apply more creative recruitment strategies to enrol patients onto rare disease clinical trials
• Delays to (or lack of) diagnosis prevent early clinical trial enrolment and can lead to therapeutic windows being missed. For example, if a potentially life-changing gene therapy must be provided within the first 2 years of life, delays in time to diagnosis and ultimate access to the therapy are crucial

Solutions to improve patient access

Pharmaceutical and medical technology companies need to invest in activities around disease knowledge and education, perhaps more significantly than in other more common disease areas, including learning from best practice and worst practice analogue case studies.

Early stakeholder engagement, including policy makers, academia, payers, clinicians, and, importantly, patient advocacy organisations, is ever more critical to plan effectively for clinical and economic evidence development and operational activities.

Patient-led collaborations in rare diseases, such as the HERCULES programme for Duchenne muscular dystrophy, demonstrate how patient organisations, industry, and payer organisations can collaborate to develop disease-level knowledge and tools. This enables and accelerates access to innovative medicines for patients with rare diseases.

Pharmaceutical and medical device companies also need to identify and develop or collaborate on resources, such as patient registries and real-world data capabilities. Payers are increasingly open to models of reimbursement with data collection agreements (i.e. registries tied to relevant real-world evidence) to surpass the typical access delay whilst supporting evidence development and protecting their budget impact.

Finally, to improve patient access, manufacturers must also overcome barriers to diagnosis and increase investment in diagnostic tools to better define patient subgroups, such as research on biomarkers that have a proven relationship with clinically meaningful outcomes.

In the next article in this series, we will look at challenges in clinical evidence development, with the series then exploring economic evidence development and operational challenges.

More in this series:

Read Part 2: Clinical evidence development

Read Part 3: Economic evidence development

Read Part 4: Operational Challenges

The post Challenges for orphan medicines entering the European market – Part 1: Disease knowledge and awareness appeared first on Mtech Access.

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

Jump to:

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

What is the EU JCA?

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

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

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

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

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

When will the JCA be a requirement?

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

Will the JCA replace local HTA?

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

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

JCA methods and guidelines

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

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

JCA guidelines published by EUnetHTA21 include::

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

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

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

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

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

Who conducts and assesses the JCA?

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

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

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

The JCA scoping process

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

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

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

Four key considerations for Pharma and Medtech

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

1. Different national requirements

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

2. Risk of needing to repeat submission following CHMP opinion

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

3. Potential for an unmanageable number of PICOs

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

4. Limited contact with assessors

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

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

How can Mtech Access & Petauri™ provide support?

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

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

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

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

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