OBJECTIVES: Beginning in 2025, oncology medicines submitted for European regulatory approval will also be subject to European Joint Clinical Assessment (JCA) process. The scope for the JCA will be developed based on Member State requests for inclusion of data relating to specific patient groups (P), interventions (I), controls (C), and outcomes (O). In this study, we aimed to simulate a hypothetical JCA scoping process in the relapsed / refractory multiple myeloma (RRMM) space to understand potential number of PICOs that might be requested and identify avenues for limiting the overall number within the final scope.

METHODS: Available RRMM treatment and reimbursement guidelines were assessed in Member States to identify key patient population and comparator dynamics for a hypothetical intervention for patients with 2 or more lines of prior therapy. Potential PICOs that can be requested by each Member State were assessed. PICOs were then consolidated based on previously provided examples to identify minimum number of PICOs in the final scope.

RESULTS: Our research identified definition of a relevant patient population and comparator selection to be the most impactful drivers of the size of the PICO universe. The number of potential populations were strongly driven by the diversity of treatments available in 1L and 2L settings across Member States. Similarly, comparator component also demonstrated significant variation according to reimbursement outcomes within each country.

CONCLUSIONS: While allowing all Member States request clinical data necessary for assessment of therapies within their treatment landscapes, JCA process should also consider practicality of performing such assessments in complex landscapes, such as MM. Close collaboration with medical community to identify patient populations or comparators that might be considered “equivalent” can be leveraged as a tool to streamline assessment scope, lowering process burden both for assessors and technology developers.