Presentation (CTI)

OBJECTIVES: The EU-HTA framework represents the central gateway to European pricing and reimbursement. Exhaustive PICO (Population, Intervention, Comparator, Outcome) prediction remains a major challenge for pharmaceutical companies in the preparation of the Joint Clinical Assessment (JCA). This analysis compares the P- and C-component from the PICO scope in the first JCA procedure for Tovorafenib in pediatric low-grade glioma with European guideline recommendations. The findings aim to inform clinical development strategies and facilitate optimal preparation for future EU-HTA assessment.
METHODS: Guidelines were identified using a dual search approach. First, relevant guidelines referenced in the JCA dossier and report were extracted. Second, a structured free-text search was conducted to identify additional guidelines in EU-HTA member countries. Duplicates were removed, and only the most recent guideline versions were included. It was determined whether and to what extent P and C can be derived from guideline recommendations.
RESULTS: The subpopulations defined in the PICO scope are consistent with the patient population from the pivotal trial of Tovorafenib (FIREFLY-1) as well as with the approved indication of C Dabrafenib + Trametinib. There is a considerable overlap between the C therapies in the PICO scope and those recommended by guidelines. However, the PICO scope also includes C therapies not supported by guideline recommendations, while some guideline-recommended C are not included in the PICO scope.
CONCLUSIONS: This analysis reveals that the definition of the P can be derived from the pivotal trial and the approved indication of relevant medicinal products. Guideline recommendations for treatments do not fully cover the C therapies specified in the PICO scope. Overall, a comprehensive understanding requires further consideration of local insights and in-depth knowledge of HTA agency requirements. Since this analysis is limited to the first JCA procedure, additional insights into PICO development are expected as more JCA reports will become available.