Presentation (CTI)

OBJECTIVES: EUHTA regulation has applied from the 1^st January 2025 for oncology products and ATMPs. The first ones reportedly undertaking JCA are tovorafenib and lifileucel (search as of 03/06/2025). The aim of this study is to predict the scope (PICOs) of those first JCAs.
METHODS: A TLR including the following, was conducted to align with the resources that assessors are likely to use: 1. Phase 2/3 clinical trials for the products under assessment, giving their likely positioning 2. Two most recent HTAs, in relevant indications, performed by lead assessors, co-assessors and 2 others 3. Relevant clinical practice guidelines in those countries where HTAs are gathered Consolidation used a 4-step process to ensure that member state needs are translated in the lowest possible number of PICOs.
RESULTS: For tovorafenib, indicated in the treatment of paediatric low-grade glioma (LGG), Ireland, Germany, France and Sweden were the markets of interest. Where 2 clinical practice guidelines were identified from the European society for Paediatric Oncology and German Society for Paediatric Oncology and Haematology, along with 3 HTAs resulting in 5 potential sets of PICOs. There were broadly consistent Populations, Comparators (standard of care chemotherapy) and relevant Outcomes. For lifileucel, indicated for the treatment of melanoma, France, Poland, Spain and Sweden were markets of interest. Where 4 clinical practice guidelines have been identified, along with 6 HTAs, primarily for Keytruda, Opdivo and nivolumab-relatlimab combination therapy, resulting in 9 potential sets of PICOs.
CONCLUSIONS: Based on the clinical trials of tovorafenib and lifileucel, predicting the PICOs in tovorafenib will be easier for the manufacturer than for lifileucel, primarily due to the lack of treatment options in LGG. The PICOs used in the tovorafenib trial are therefore closer to our predictions, however this study did not look at all member states, which is a limitation.