Nearly 14 percent of all drugs in clinical trials eventually win approval from the FDA — a much higher percentage than previously thought, according to a new study from the MIT Sloan School of Management.
Approval rates ranged from a high of 33.4 percent in vaccines for infectious diseases to 3.4 percent for investigational cancer treatments. Previous estimates placed overall success rates, from Phase I through to FDA approval, between 9 and 11 percent.
In addition, the study found that trials that used biomarkers to stratify patients tended to be more successful than those that did not, pointing to the growing role of companion diagnostics in improving clinical trial success rates and future designs.
The MIT study, published in the journal Biostatistics, has broad implications for investors, regulators, policymakers and clinicians in calculating the probability of the success of investments in drug development programs.
“Without accurate and timely estimates, resources may be misallocated and financial returns may be misjudged, which leads to higher development costs, higher-priced drugs, and lost opportunities for investors and, more importantly, patients,” said study author Andrew Lo, director of MIT’s Laboratory for Financial Engineering.
While approval rates fell between 2005 and 2013, they have trended upward since. Oncology, for example, had the lowest overall approval rate, but estimates increased to 8.3 percent in 2015, partly due to progress in the development of immunotherapies, the university said.
The research was based on Informa’s Citeline dataset, which contains more than 400,000 entries corresponding to 185,994 unique clinical trials of over 21,000 compounds. Automated algorithms traced each drug’s development path, and determined the probability of success within a matter of hours, MIT said. By comparison, ClinicalTrials.gov contains over 217,000 clinical trial entries.
“We hope to provide this information on a regular basis — it’s not just a one-shot deal,” Lo said.
“As clinical trial success rates improve for certain diseases, it’s likely that more investment capital will flow into those areas. For diseases where success rates stall, public policy can play an important role by increasing research funding or providing more incentives to risk-tolerant investors and philanthropic organizations.”