OBJECTIVES: Recent biotherapies developed in oncology have shown bigger impact in specific populations with tumour profiles that match the drugs' mechanism. The aim of our study was to determine the impact of biomarker(BM)-based patient selection (PS) on clinical development (CD) and market-access (MA) compared to all-comers strategy.

METHODS: We built a multi-step prediction tool based on clinical trials and MA data available for biotherapies developed in hematologic an solid tumours since 2006. Trials were analysed based on whether or not patients were recruited through BM-selection. Trial success was considered when subsequent development phases were found for the treatment in a similar indication. For each CD phase and FDA application, we determined the probability of success. The probability of MA for biotherapeutic treatments with and without BM-based PS were compared by therapeutic area, depending on the clinical development phase when BM-based PS was introduced (1, 2 or 3).

RESULTS: The overall probability of success for biotherapies developed with an all-comers recruitment strategy at all CD stages was 5% (9% in haematology and 4% in solid tumours) with the biggest hurdle being phase 2 (26% success). BM-based PS was associated with a noticeable increase in development success, reaching 15% (23% in haematology, 12% in solid tumours). Introduction of BM-based PS in phase 2 had the biggest impact compared to the other CD phases (up to 42% success).

CONCLUSIONS: BM-based PS ensures a higher probability of CD and MA success, although it can lead to restrictions in eligible patients, which could lead to loss of opportunity for patients who may have benefitted of the treatment but were excluded from CD. The potential effect of BM-based PS on target population size as well as the impact of smaller population on price negotiations should be taken into consideration to determine the opportunity-cost related to this CD design choices.