EU HTA Regulation: A Brighter Future
The New EU Health Technology Assessment (HTA) Regulation introduces two key aspects: a mandatory Joint Clinical Assessment (JCA) and an optional Joint Scientific Consultation (JSC). The goal is to align HTA processes across the 27 member states. The JCA will focus on relative effectiveness and safety and the JSC will offer early guidance on evidence generation plans and development strategies. The European regulatory landscape will change significantly as drug developers will be required to adapt their strategies for evidence generation and stakeholder engagement. There will be more consistency and efficiency and will continue to allow for flexibility at the national level for economic evaluations and pricing decisions.
While marketing authorization assesses the absolute benefit-risk balance, HTA considers the relative effectiveness, safety, and often cost-effectiveness compared to existing therapies. HTA processes have varied across the 27 EU member states, prompting efforts towards greater harmonization. To date there were differing methodologies, evidence requirements, and timelines. This complexity increases development costs and can significantly delay patient access. A more unified HTA process can address these issues while reducing duplication of efforts.
The JCA will run in parallel with the marketing authorization process by the European Medicines Agency (EMA). The required assessment scope will be presented by the HTA assessors and will be defined by the four "PICO" aspects – Population, Intervention, Comparator, and Outcomes. The optional JSC will allow developers to discuss clinical study designs and evidence generation plans. It can only be granted when the clinical studies and investigations are still in the planning stage, however, so timing is key.
The JCA will initially apply to new medicines for cancer and advanced therapy medicinal products (ATMPs) from January 2025, extending to orphan drugs in 2028 and all other centrally approved products by 2030. National HTA bodies will retain responsibility for economic evaluations, country-specific considerations, and final pricing and reimbursement decisions.
Drug developers will need:
1. Earlier evidence generation planning.
2. Increased cross-functional collaboration: Closer integration between clinical development, regulatory affairs, health economics and market access teams.
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3. Targeted investment in HTA capabilities to meet JCA evidence requirements.
4. Proactive stakeholder engagement: Consulting clinicians, patient groups, and HTA bodies during evidence planning to understand the treatment landscape and unmet needs.
5. Operational readiness: Allocating sufficient resources to simultaneously manage regulatory and JCA activities under tighter timelines.
Early planning and investment in building HTA capabilities will be imperative to navigate this transition and ensure improved patient access to innovative therapies across the EU market.
This photo is in memory of a wonderful initiative I worked on in Spain to empower and educate patients and to generate awareness about access issues for novel oncology and hematology treatments. Agata Polińska and Bartosz Polinski have been advocating for improved access to treatments since 2010. I created a patient advocacy group in Spain in 2020 together with them and was President and CEO. I identified unmet needs in the Spanish patient community and created strategic alliances with medical societies and other PAGs. I designed and executed digital solutions to demonstrate the disparities in access to treatments across the EU and within Spain. I will always be grateful for the support they offered me and the passion we continue to share together. With this new regulation in Europe coming the future is looking brighter.