PCN25 - ROLE OF PROGRESSION FREE SURVIVAL IN THE APPROVAL PROCESS AND EUROPEAN HTA ASSESSMENTS

Abstract

Most cancers are characterized by a progressive course of disease, ultimately leading to death of the patient. Thus, in the medical world, prevention of disease progress, measured by progression free survival (PFS), is considered a vital parameter with a clear patient relevant benefit. This is reflected in a large fraction of clinical studies observing cancer treatment, that placed PFS as the primary endpoint, meaning that PFS results determine the success of the treatment. While the European Medicines Agency (EMA) considers PFS to be an important factor in assessing the efficacy of a new drug, HTA agencies such as G-BA, NICE and HAS are divided. The aim of this research is to analyze the previous and current assessments of PFS by the European HTA agencies and compare them to the assessment by EMA. Published HTA documents of G-BA, HAS and NICE have been reviewed for the role of PFS. The results were then compared with the CHMP decisions and EPARs. The analysis of the EMA approvals showed that over the past years, PFS results have been a major decisive factor for granting marketing approval to new drugs. In contrast European HTAs indicate a contradictory approach. In no case of all drugs assessed by G-BA with PFS as endpoint, PFS was considered as patient-relevant endpoint and thus did not contribute to the additional benefit of the drug. The other HTA agencies showed similar results, albeit in a weaker form. There is a lack of alignment between the EMA and European HTA agencies, especially the German G-BA with respect to PFS. This alignment is also missing within the G-BA. While the medical side of the G-BA favors PFS as patient relevant, payers do not regard PFS patient relevant, and do not give PFS credit in determining an additional benefit.