Early Benefit Assessments in Oncology in Germany: How Can a Clinically Relevant Endpoint Not Be Relevant to Patients?
After 4 years of early benefit assessment (EBA) in Germany, it is becoming evident that the Federal Joint Committee (FJC) frequently considers well-established clinical endpoints as not being relevant to patients. Focusing on assessments of oncology medicines, we analysed the FJC's view on primary endpoints and compared it with the approach used by regulatory authorities. Mortality data were accepted by both stakeholders. Whereas regulatory authorities accepted primary morbidity endpoints such as progression-free survival and response rates, the FJC mostly excluded these from its assessments. Health-related quality of life (HRQoL) data have been poorly reflected in the approval process; for EBAs, those data have rarely impacted on benefit ratings. We argue that agreement between regulatory authorities and the FJC is required regarding primary study endpoints that are relevant to patients, and that clarification of acceptable endpoints by the FJC, especially in the morbidity domain, has to be provided. Moreover, in order to fully acknowledge the benefit of a new medicinal product, mortality, morbidity and HRQoL should be weighted differentially, according to the condition.
Roche Pharma AG, Emil-Barrell-Str. 1, 79639, Grenzach-Wyhlen, Germany, joerg.ruof@roche.com.
Jörg
J
Ruof
Olivier
O
Flückiger
Niko
N
Andre
D000970:Antineoplastic Agents / Q000494:pharmacology*; D023381:Endpoint Determination / Q000379:methods* / Q000592:standards; D005858:Germany; D006801:Humans; D008495:Medical Oncology / Q000379:methods* / Q000592:standards; D009017:Morbidity; D009369:Neoplasms / Q000453:epidemiology* / Q000401:mortality*; D017063:Outcome Assessment, Health Care / Q000331:legislation & jurisprudence*; D011788:Quality of Life
D000970:Antineoplastic Agents