ISPOR Europe Diaries 2024

EU HTA-R – The countdown to January 2025 was ever present at ISPOR Europe 2024

Ensuring organizational readiness for the EU Health Technology Assessment Regulation – in particular Joint Clinical Assessment – was the topic that drew crowds and dominated conversation at ISPOR Europe 2024 in Barcelona, Spain. This focus on improving health technology assessment across EU member states with landmark regulation seemed appropriate given that “generating evidence toward health and well-being” was the conference’s theme.

New cancer medicines and advanced therapy products will require a JCA from January 12, 2025. Orphan medicines will require EU level assessment from January 13, 2028, and all other medicines being launched in the EU will need JCA from January 13, 2030. As soon as we return from the Christmas Break JCA will become a reality.

Dr Roisin Adams, Head of Health Technology Assessment (HTA) Strategy and External Engagement at the National Centre for Pharmacoeconomics, Ireland, said approximately 30 assessments are planned for 2025, with outputs expected by late 2025 or early 2026. 

Prepared to deliver?

With so many joint clinical assessments planned it is clear that some companies are prepared for the JCA process and some good examples were featured at the conference. Dr Vanessa Schaub, Head of Global & EU HTA Strategy, F. Hoffmann-La Roche Ltd, Switzerland, a speaker at the Day 2 Plenary Session, said early efforts to prepare for JCA have been key to Roche’s readiness for JCA. Roche has been preparing since 2021 when the EU HTA-R was adopted. With two oncology products due for JCA in 2025, Roche has dedicated the past 3 years to overhauling its internal processes, created a cross-functional Access Squad for external and internal EU HTA readiness, and invested in training. Schaub described the changes as “a massive change management process,” through which Roche has treated HTA as equal to regulatory functions. 

Schaub touched on Population, Intervention, Comparator, Outcome Study (PICOs) design for JCA noting that although estimating PICOs is challenging – especially in oncology due to numerous subpopulations and comparators – early preparation and pre-specified plans can help companies to manage the risks. Schaub said AI and digital tools are likely to be leveraged in the future to streamline processes, but she also said the related legal and ethical considerations will need to remain uppermost in mind.

As the JCAs commence don’t forget about the Joint Scientific Consultations

In other key EU HTA-R developments we learned that 10 Joint Scientific Consultations (JSCs) are planned for 2025 alongside two JSC request periods from February 3 to March 3, and June 2 to June 30 2025. The JSC request periods are designed to give pharmaceutical companies and research organizations access to scientific advice from the regulatory authority or scientific committees. Conversation swirled around the fact that the industry wants to see more request periods built in and spread throughout the year. This would give more opportunities for companies to submit their requests in the specified time frames to benefit from expert advice to guide them through the new EU HTA-R process.

Updates at ISPOR on the key operational challenges

Manufacturers are still facing multiple challenges in preparing for the EU HTA-R. For many the latest issues concerned timelines. Among key learnings from ISPOR Europe 2024:

• Submission expectations: For planning purposes, manufacturers are urged to be realistic about when they expect to submit their documents. It is essential to provide as much detail as possible about the indication
• Handling longer clock stops or changes in indication: As the JCA timeline is mandated by the regulation, it remains unchanged regardless of delays or changes during the regulatory process on the European Medicines Agency (EMA) side, such as clock stops. The assessment phase is also limited to 100 days
• Late changes to indication: Late changes can be challenging. Most changes are minor (e.g. restricting the patient population) and are likely reflected among  the multiple PICOs, therefore it was mentioned that major adjustments may not be necessary. However, substantial changes in the indication are a different matter and will require careful consideration on how to proceed to comply with the mandated timeline in the regulation
• Early scope sharing: As the Coordination Group is aware of the concerns of manufacturers regarding the timeline, the panel mentioned that they will try to share the JCA scope as soon as possible, not necessarily by the maximum date specified in the implementing act
• Subgroup considerations: Again for planning purposes, it was requested that manufacturers share their Letter of Intent (LOI) as soon as possible. This would allow for a timely identification of  member states that have the expertise to act as assessors and co-assessors, and to identify relevant experts
• Coordination with regulatory milestones: It is essential to remember that the dossiers  for the EMA and JCA are separate

Conclusion

While manufacturers are challenged by the EU HTA-R, member states who were initially opposed to mandatory joint HTA now see it as a bridge to improving access to new technologies with high quality assessment. Germany, France and Spain, for example, are all in the final stages of aligning their national HTA processes with the EU HTA-R and JCA. With a couple of weeks to go, we were happy to hear the announcements that Europe is generally ready for the EU HTA-R.