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In the largest-ever report of its kind, drugs entering clinical development in Phase I were found to have only a 1 in 10 chance of FDA approval.
In collaboration with Amplion, a biomarker business intelligence company, and BioMedtracker, a service that tracks a drug’s likelihood of approval (LOA) by FDA, the Biotechnology Innovation Organization (BIO) released a report on May 25, 2016 that examined clinical trial success rates from 2006 to 2015 (1).
According to the report, across all developmental categories, the overall likelihood that a candidate is approved and makes it to market (beginning from Phase I) is approximately 10%. For biologics specifically, this LOA was found to be approximately 12%. Vaccines, specifically, had an approximately 16% LOA from Phase I.
If a product fails in clinical trial development, it typically occurs when a product is to advance from Phase II to Phase III; according to the report, approximately 70% of developmental candidates do not advance to Phase III. The authors wrote this was likely due to the fact that Phase II is when proof-of-concept is first tested in human models.
Of each of the individual phases, Phase I had the highest success rate (approximately 63%), which the report authors said was because this phase is “typically conducted for safety testing and is not dependent on efficacy results for candidates to advance.” They also may have the highest rate because some companies do not publicly disclose failures at this early a stage, contributing to reporting bias.
Across 14 specific disease categories, oncology medications had the lowest LOA value (approximately 5%), while hematology had the highest LOA (approximately 26%). The failures in the oncology category typically occurred during Phase III transition. Interestingly, although oncology candidates had the lowest chance of approval, those that were approved were pushed through the regulatory process in approximately one year, nearly twice as fast as candidates that were approved in the neurology category. Oncology drugs, the authors noted, were the most likely to benefit from FDA’s expedited approval pathways, contributing to their fast approval rates.
Authors of the report confirmed that compared with Phase III success rates of drugs that did not incorporate selection biomarkers, success rates for drugs with selection biomarkers had a higher LOA (55% and approximately 77%, respectively). “The large differences in Phase II and III transition success rates are quite convincing, quantitatively, of what many drug developers have long argued anecdotally-enrichment of patient enrollment at the molecular level is a more successful strategy then heterogenous enrollment,” the authors wrote. They did not elaborate, however, on how this biomarker-based strategy would specifically help drive market value or uptake of a medication over one that was approved with the same indication that did not include an accompanying diagnostic test.