Estimation of clinical trial success rates and related parameters
Menée à partir d'un échantillon de 406 038 données d'essais cliniques ayant évalué au total plus de 21 143 médicaments entre 2000 et 2015, cette étude estime les taux de réussite des essais cliniques, notamment en cancérologie, analyse leur évolution puis identifie les facteurs associés
Previous estimates of drug development success rates rely on relatively small samples from databases curated by the pharmaceutical industry and are subject to potential selection biases. Using a sample of 406 038 entries of clinical trial data for over 21 143 compounds from January 1, 2000 to October 31, 2015, we estimate aggregate clinical trial success rates and durations. We also compute disaggregated estimates across several trial features including disease type, clinical phase, industry or academic sponsor, biomarker presence, lead indication status, and time. In several cases, our results differ significantly in detail from widely cited statistics. For example, oncology has a 3.4% success rate in our sample vs. 5.1% in prior studies. However, after declining to 1.7% in 2012, this rate has improved to 2.5% and 8.3% in 2014 and 2015, respectively. In addition, trials that use biomarkers in patient-selection have higher overall success probabilities than trials without biomarkers.
Biostatistics , résumé, 2017