OBJECTIVES: To understand potential evidence requirements for the EU JCA and how these may impact stakeholders by simulating the PICO scoping process for two anticancer IMPs.

METHODS: PICO requests by 14 representative member states (MS; Belgium, Czech Republic, Denmark, Finland, France, Germany, Hungary, Ireland, Netherlands, Poland, Portugal, Slovakia, Spain, Sweden) were simulated based on proposed European Medicines Agency labels for the IMPs, European/local treatment guidelines, expected new treatments, and health technology assessment reports of analogue products. After development of local PICOs by affiliates of the health technology developer (HTD), PICOs were consolidated according to JCA scoping guidelines, and the type and number of analyses predicted to be required was summarized.

RESULTS: The simulation for IMP1 was finalized in November 2023. It predicted 8-29 PICOs, of which 5-20 would require indirect treatment comparisons (ITCs), across 54 outcomes, leading to 240-1,566 analyses requests. For IMP2, the simulation was finalized in April 2024. It predicted 12-31 PICOs, 11-26 of which would require ITCs, across 19-32 outcomes, leading to 228-992 analyses requested.

CONCLUSIONS: In indications where treatment patterns vary across MS, numerous PICOs may be requested which would require a substantial number of analyses to be presented, thus placing a significant burden on HTDs to provide extensive evidence within challenging timelines. As EU JCA guidance continues to evolve, further work is needed to ensure that evidence requirements for HTDs are feasible, relevant and proportionate. Allowing HTDs to participate in the PICO scoping process would facilitate this. Moreover, it remains unclear how MS will integrate the EU JCA into existing national processes and the potential impact on patient access timelines. Addressing these challenges will require close collaboration amongst the Coordination Group, HTDs, MS, and other key stakeholders, so that the EU JCA can meet its core objective of providing faster patient access to innovative medicines.