Search for Clinically and Economically Effective Lymphomas Treatment

Abstract

trials' data were retrieved from the Haute Autorité de Santé (HAS) website and the Number Needed to Treat (NNT) to prevent one event (progression, death) was estimated. An Absolute Loss of Chance (ALOC) index was calculated over the time-lag period and per time unit combining NNT and HAS target population size. Drugs with level 5 Added Medical Value (ASMR, Amélioration du Service Médical Rendu) were excluded. Results: Twelve drugs were granted a EUMA for solid tumours in 2012 and 2013 with level 2-4 Added Medical Value: one drug of level 2-important (trastuzumab emtansine), five drugs of level 3-moderate (cabazitaxel, vemurafenib, critzotinib, pertuzumab, and enzalutamide), and six drugs of level 4-minor (eribuline, ipilimumab, abiraterone, vandetanib, axitinib, and vismodegib). The time-lags between EUMA and FPRD ranged from 7.4 months (enzatulamide) to 29.9 months (cabazitaxel). Concurrently, the overall ALOC ranged from 9 to 799 medical events which might have not been prevented unless alternative access (Compassionate, Clinical Trials), depending on drug, indication and time-lag. ConClusions: Early patient access for innovative drugs in oncology might help preventing a substantial number of progressions and deaths. ALOC might support patient access decision making process.