- More clinical trials succeeded between 2012 and 2014 than in prior years, reversing a steady decline in success rates, according to a new report by McKinsey researchers published in Nature last month.
- The percentage likelihood of moving from Phase 1 to market for therapeutic products hit 11.6% in the most recent three-year period studied. This was up from a low of 7.5% between 2008 and 2011.
- These findings are in the same ballpark as figures from a study by the biotech trade association BIO, which estimated the overall likelihood of approval for developmental drugs was 9.6% over the past decade.
While success rates have certainly improved since the 2008-2011 period, the 11.6% figure remains well below the high-water mark of 16.4% hit between 1996-1999.
Across all time periods, successfully navigating a drug through Phase 2 studies was consistently the most arduous. Between 2012 and 2014, 43% of biologics advanced from Phase 2 into Phase 3 testing, while only 32% of small molecules did so.
Overall, biologics were more likely to be approved than small molecules in the most recent period.
Researchers examined over 9,200 novel compounds developed between 1996 and 2014, according to Stat.
The BIO study also examined a large number of unique drugs and returned broadly similar results. The likelihood of approval, or probability of a drug moving from Phase 1 to approval, was 9.6%, although that number was weighed down by tougher odds for cancer drugs. Without oncology, likelihood of approval jumped to 11.9%.
One proven way to improve the likelihood of success is to use selection biomarkers to inform inclusion/exclusion criteria. According to Ellen Sigal, chairperson and founder at Friends of Cancer Research, “Having well-validated biomarkers in a clinical trial improves the likelihood of success four-fold."
Data from the BIO study bore this out. Although far fewer trials used selection biomarkers, overall likelihood of approval for those drugs was 25.9%, compared to 8.4% for drugs without any biomarkers.
While the studies paint a picture of a more successful clinical development environment, the rates of success in both are still low relative to investment. The pharmaceutical industry has long lamented the high cost burden and long timelines associated with bringing a drug to market, frequently invoking this as a justification for higher prices.