With EU HTA, it is crucial to be clear about one thing: the Joint Clinical Assessment (JCA) is not a late market access step, but an essential element of clinical trial planning and regulatory strategy from the very beginning.

With EU HTA, it is crucial to be clear about one thing: the Joint Clinical Assessment (JCA) is not a late market access step, but an essential element of clinical trial planning and regulatory strategy from the very beginning.

In January 2025, Regulation (EU) 2021/2282 of the European Parliament came into effect. Oncology drugs and advanced therapy medicinal products (ATMPs) are now subject to joint clinical assessments (JCA). The procedure is based on the PICO framework to ascertain the research questions. The PICOs inform the data requested and hence the contents of the JCA-Dossier to be submitted by the Health Technology Developer (HTD). The PICO scoping takes place without any input from the HTD.

After the consolidated PICOs are communicated to the HTD, the HTD will be assigned 100 days to compile and submit the JCA (60 days with accelerated approval). EU HTA regulation also requires that a dossier is submitted 45 days before expected positive opinion by the CHMP at latest. All PICOs must be adressed in the JCA-Dossier by direct comparisons or indirect evidence or a thorough rationale is required why a specific PICO could not be addressed.

The European Union (EU) regulation for Health Technology Assessment (HTA) has delivered a hybrid system of national preparations, a central submission and clinical assessment, on which national supplemental sub-missions as well as Pricing & Reimbursement (P&R) negotiations and decisions will take place. The process is still far from mature. Much like the iterative process seen during the early years of the AMNOG, the JCA is a learning system, though it was launched with an incredibly ambitious timeline. The startup phase has been more resource-intensive than many anticipated—largely due to the rapid timeline for implementation and the number of stakeholders involved. Also, the practical infrastructure is still catching up to the standards set by established national procedures.

These changes also require an adjustment to internal processes and structures at company level. Regulatory and JCA processes must be aligned, and various functions at European and national level must be coordinated. Scenarios must also consider how PICOs may change should there be any adjustments to the label as part of the authorization process. The HTD will ultimately have exactly 100 days to prepare and submit the JCA dossier, which requires early preparation at company level.

EU-HTA goes hand in hand with a trend in which it is becoming more difficult to demonstrate added benefit according to traditional criteria: ATMPs and gene therapies enable increasingly specific and personalized treatment of patients. As a result, the patient groups available for clinical trials are becoming more specific and smaller. Demonstrating statistically significant superiority is therefore becoming more difficult from a methodological perspective alone and requires a shift in perspective on the part of HTA authorities.