Strategic Insights From 5 Years of the EU HTA Regulation: Unleashing the Ambition for Orphan Products and Vaccines

Building on a decade of collaboration with EUnetHTA, the EU health technology assessment (HTA) regulation established a formal framework for member states to collaborate on the clinical assessment of new health technologies, particularly medicines and high-risk medical devices. The regulation aims to prevent duplication of effort for all stakeholders, pool expertise and resources, and accelerate patient access to innovative and life-saving treatments. Currently, only oncology products and advanced therapy medicinal products (ATMP) are subject to the Joint Clinical Assessment (JCA), with orphan medicinal products and all medicinal products, including vaccines, to follow in 2028 and 2030, respectively. Leveraging over 30 years of HTA experience and expertise, we have supported a wide range of companies to prepare internally for new ways of working and to integrate development of JCA submissions within existing activities and country submissions. We are also preparing integrated evidence plans and JCA submissions for a range of therapies that will be subject to the JCA by the end of the decade. Our experts will share our key learnings and insights and apply them to practical solutions for orphan products and vaccines - two areas that present challenges, but also general learnings that can be applied to all technologies. We will share case studies from the oncology and ATMP space, to bring to life the most pressing challenges and apply our learnings to unlock the potential of the regulation for orphan medicinal products, vaccines, and patients. Many countries have modified their approach to the appraisal of orphan medicines and vaccines to account for their unique challenges. Whilst it remains unclear how countries will incorporate such a JCA report into decision making we will explore what is currently known and how this might apply to these critical technologies.

Sponsored by Corporate Partner, Thermo Fisher Scientific