What is the relevance of progression free survival in the approval process and at European HTA assessments?

The Progression Free Survival (PFS) rate is one of the most important criterion for measuring effectiveness of drugs in oncology. It determines, how long patients with a certain disease survive without the disease progressing. SKC has investigated how the PFS influences the approval or reimbursement assessments of HTA agencies. This was managed by looking at four different drugs for cancer treatment (Ribociclib/Kisquali®, Palbociclib/Ibrance®, Olaparib/Lynparza® and Niraparib/Zejula®) and reviewing documents from different HTA agencies regarding said drugs.

In Europe as well as in the USA, PFS was relevant for approval decisions. While in Europe PFS was considered relevant measures of patients benefit in most cases by the EMA, in the US-American FDA the relevancy depends on the magnitude of the effect in comparison to already available therapies. For reimbursement decisions, PFS was significantly less valued than it was for general approval decisions. In the UK, the PFS was used as only a minor factor for reimbursement discussion, while in France and Germany PFS was not taken into consideration at all. The analysis shows, that the EMA used PFS in four out of four cases, whereas the French HAS and the British NICE only used it in one out of four cases. The German G-BA did not use it a single time. This results out of the other used factors in HTA agencies: They see PFS only as a combination of mortality and morbidity, who they already use in the shape of OS (Overall Survival rate) and quality of life data.

As experts in the field of drug approval, SKC is particularly interested in the mutually influencing price regulation of German, European and American drugs.

BY Prof. Matthias P. Schönermark, M.D., Ph.D., managing director and Dr. Angelika Deichmann