The first study to estimate the costs of the pivotal clinical trials the FDA relies on to approve new drugs was published on September 24, 2018 as an “Online First” in JAMA Internal Medicine. ISMP senior scientist Thomas J. Moore is the lead author for the groundbreaking paper, in a collaboration with three coauthors at the Johns Hopkins Center for Drug Safety and Effectiveness.
“For years, there have been claims that it is becoming prohibitively expensive to adequately test a new drug,” said lead author Moore. “The purpose of this study was to focus on actual pivotal studies that were required, with the goal of placing a realistic price tag on this essential form of scientific evidence.”
Key findings include:
- The study provides realistic cost estimates of pivotal clinical trials that established drug benefits to support FDA approval of 59 new drugs that were approved in 2015-2016.
- The median estimated cost of a clinical trial was just $19 million, with half of the 138 trials studied clustered between $12 million and $33 million.
- The highest cost trials–with estimates up to $345 million–were for new drugs that were similar to drugs already available and already proven in treating serious illnesses.
Study limitations are discussed at length in the paper. The most important to note are that these are estimated costs based on 52 variables, and do not reflect all clinical studies or overall drug development costs.
The senior author of the study was Caleb Alexander, co-director of the Johns Hopkins Center for Drug Safety and Effectiveness, and the study was supported in part by a grant from the Laura and John Arnold Foundation.
An abstract and copy of paper are available through the JAMA Internal Medicine website.
More information on the safety of prescription drugs after FDA marketing approval is available through ISMP’s QuarterWatchTM Reports publication, which is published four times a year as a public service to provide an independent perspective on emerging drug risks.