Predicting PICOs to Prepare for Joint Clinical Assessment: Learnings From 19 Case Studies

OBJECTIVES: As of January 2025, all new oncology medicines and advanced therapy medicinal products (ATMPs) will be subject to joint clinical assessment (JCA). As the final scope will only be shared 60-100 days ahead of the JCA dossier deadline, health technology developers (HTDs) will need to anticipate the likely JCA scope.

METHODS: The JCA scoping process was simulated for 19 products (3 hypothetical products and 16 investigational products expected to undergo JCA). European treatment guidelines and the competitive landscape were used to anticipate the PICO (population, intervention, comparator, outcomes) proposal. Local treatment guidelines, analogues and local stakeholder input was used to anticipate member state (MS) responses for a selection of EU MS. The latest EUnetHTA 21 scoping guideline was followed to consolidate the PICO and predict the JCA scope.

RESULTS: PICOs were simulated for 18 oncology products and one genetic disorder; 2 were advanced therapy medicinal products (ATMPs), and 3 had orphan drug status. The median number of PICOs was 8 (range 5 to 18) and 65% of PICOs would require an ITC, while worst-case scenarios showed that up to 88 PICOs could be expected. The minimum number of analyses requested ranged from 175 to 720.

CONCLUSIONS: The scoping approach results in a significant number of PICOs, with many requiring ITCs. Based on a representative selection of 8-10 MS, HTDs can predict these PICO requests. PICO prediction should be done early in the development process to inform clinical trial design and evidence generation planning (e.g., subgroup analyses, ITCs) and to develop a justification for PICOs that cannot be addressed. The PICO simulation should be rerun after trial completion to identify any changes in the treatment landscape.