PCN351 COMPARATIVE STUDY OF PFS AS PRIMARY ENDPOINT IN EUROPEAN HTA (FRANCE AND GERMANY)

Abstract

Progression-Free Survival (PFS) is one of the main criteria used to assess the clinical effectiveness of oncology drugs. Its relative positioning with overall survival (OS), considered as one of the most relevant endpoints for oncology, can be questioned. Plus, its actual definition may be different from a clinical study to another, leading to different points of view within HTA agencies on how to consider this endpoint. The objective was to compare how oncology clinical studies with PFS as primary endpoint were considered in HTA opinions from two major European decision makers, the Transparency Committee (TC, France) and Gemeinsamer Bundesausschuss (GBA, Germany). Using the Prismaccess® database, we identified oncology clinical studies using PFS as primary endpoint. For each study, we identified the related marketing authorization and population and we extracted the associated HTA opinions from the TC and the GBA. Then we studied the outcomes of the opinions (especially improvement in actual benefit in France and added benefit in Germany), and how the agencies took PFS into consideration. Focusing on first applications and new indications from the agencies between 2014 and 2018, we identified 77 clinical studies. Then we obtained 42 opinions with no difference in clinical data between France and Germany. PFS was always considered in France whereas it was not taken into account in the GBA assessment. OS was present in all the studies. The assessment outcomes are different between the agencies. When France is focusing on the primary endpoint, regardless of the nature of the criterion, Germany is focusing on OS when PFS is the primary endpoint. As PFS combines morbidity and mortality, this criterion is not well considered within the GBA. The GBA is indeed more comfortable with criteria such as recurrence free survival to assess drugs, considering it as a more robust criterion.