Conventional wisdom holds that only around 10% of drug development projects make it all the way from Phase I to approval. Two studies of clinical trial success rates now provide updated granularity to this rule of thumb, and show considerable variation by therapeutic area and drug modality.
In a first report, the industry lobby group BIO, along with analysts at BioMedTracker and Amplion, analysed 7,455 drug development programmes that moved through the clinic between 2006 and 2015. They found that the probability of success was 63% in Phase I trials, 31% in Phase II trials, 58% in Phase III trials and 85% during the regulatory review process, for an overall success rate of 9.6% (63% × 31% × 58% × 85% = 9.6%). But when they analysed the data by therapeutic area, the overall success rates ranged from 26% for haematology projects to 5% for oncology projects (see Fig. 1).
Rare disease drugs for non-cancer indications out-performed the average, with an overall success rate of 25%. Projects that included biomarkers fared similarly well, achieving an overall success rate of 26%.
The overall analysis lumped new molecular entities (NMEs; including small molecules and antibodies) together with 'non-NME' development projects such as reformulations and combinations of approved drugs (but not generics). When the analysts focused on the NMEs to assess only the most innovative therapies, the overall success rate was 6.2%. Novel biologics, including antibodies and gene therapies, performed better, with an overall success rate of 11.5%.
A second study shed further light on the clinical trial success rates and provides cause for optimism. Analysts at the consulting firm McKinsey & Company tracked the progress of 9,200 compounds that were developed between 1996 and 2014. When they calculated a rolling 3-year average, they found that success rates are on the rise. They calculated a cumulative success rate of 11.6% in 2011–2014, up from a low of 7.5% in 2008–2011 (Nat. Rev. Drug Discov. 15, 379–380; 2016).
Several of their findings mirrored those of the BIO study. McKinsey calculated an above-average overall success rate for rare disease drugs, hitting 29% over the past 3 years. Biologics had an overall success rate of 18%, twice that of the 9% success rate for small-molecule drugs.
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