Value lies in the eyes of the beholder, and in a healthcare reimbursement setting, there are several different stakeholders and perspectives on these issues. At the World EPA Congress 2022, in Amsterdam, there were various panel sessions, roundtables, and discussions of key themes around navigating the market access landscape for biopharmaceuticals by generating evidence that can communicate value and mitigate uncertainty in the post-pandemic world. The separate tracks at the conference were built around pricing, market access, evidence, and rare/advanced therapies.
The Congress started with a plenary discussing a post-pandemic market access environment and the lessons that can be learned from the challenges presented by the pandemic. Key solutions centered around increasing public-private partnerships as well as the importance and acceptability of real-world evidence generation. In particular, registries not just for pharmacovigilance but also for measuring patient outcomes, along with using digital tools to facilitate patient monitoring and management. Shifting to telemedicine, increasing patient engagement and home health with self-administered therapies were also seen as the likely changes that could significantly impact healthcare delivery moving forward.
Several presenter discussed the importance of real-world evidence data generation, patient registries, and digital therapeutics in facilitating conditional pricing / provisional funding of therapies with limited clinical data in order to reduce access delays. For example, the latest initiatives in Spain with the Valtermed system, and in the Netherlands with the DRUG Access Protocol (DAP) offer mechanisms for stakeholders to collaborate on gathering evidence while continuing to provide patient access. Although, price re-negotiations and funding decisions based on real-world outcomes seem an appropriate way forward, panelists also voiced reservations given the administrative burden they place on physicians and healthcare systems.
Many sessions at the EPA focused on the fact that only about 5% of rare diseases have any indicated treatments. Inequitable access to these therapies is a huge issue, not only across Europe but also in the emerging markets. Although efforts are being made in the individual countries to address these issues, a harmonized approach is needed to ensure sustainability in the development of orphan therapies. Jack Mycka, Vice President- PRMA & Emerging Biotech Commercialization at Indegene, presented at the conference, on the balance between investing in the development of treatments for orphan/rare diseases and ensuring patients’ access to them has evolved.
From a Health Technology Assessment (HTA) perspective, there is little clarity on the clinical trial requirements and the PICO (PICO – Population, Intervention, Comparator(s), Outcomes) framework, considering the European joint clinical assessment (JCA) is supposed to apply to advanced therapy medicinal products (ATMPs) and oncology therapies from 2025 and all orphan drugs from 2027. The effect of these changes at the individual country level also remains to be seen.
The evolution of the gene therapy market in Europe was a significant topic at the conference, especially with the recent European withdrawal of Zynteglo, a one-time treatment for beta thalassemia. Presenters debated how the value of a one-time gene therapy needs a fresh approach for payment and funding with some discussions around joint procurement and funding. Cross-border health care solutions were discussed given the complexity of manufacturing and the solidarity principle of the EU.
Manufacturers trying to disrupt the market and change how pharma currently thinks about Pricing, Reimbursement, and Market Access (PRMA). Although some interesting approaches were discussed to make drugs affordable, recent FDA decisions on Lilly’s therapy with clinical trials conducted only in China give pause for thought.
With the shift in focus on how to approach market access in emerging markets, several presenters discussed the huge potential, given the size of the population, as well as and the challenges with infrastructure, assessment and pricing processes, and lack of transparency.
Two other key areas that received considerable attention were the importance of the patient voice before initiation of clinical trials, throughout the clinical development process and the rise of digital therapeutics as well as the increasing recognition of their importance in terms of improving patient outcomes.
Overall, the EPA Congress underscored the fact that this is an exciting and dynamic time to be involved in the advancement of new therapies. The rapid development and approval of the COVID-19 vaccines has given a boost to the biopharmaceutical industry and has underscored the advances that can be made when stakeholders work together to achieve a common goal. While budgets have been constrained and affordability has become an even bigger issue, understanding the market access environment and the challenges and opportunities it presents has never been more vital. Contact us to learn more!