The majority of postapproval clinical trials conducted by pharmaceutical companies explored novel or expanded uses as opposed to monitoring original indications, according to results from a cross-sectional study.
“The U.S. Food and Drug Administration can use postmarketing requirements to mandate pharmaceutical companies to conduct clinical trials after the approval of novel therapeutics,” wrote Joshua J. Skydel of Tufts University, Boston, and colleagues in JAMA Network Open.
“From 2009 through 2012, the FDA approved 110 novel therapeutics for 120 indications,” the researchers reported.
Of those approved, 37 novel agents were identified that lacked requirements for postmarket monitoring. Data was obtained from the Drugs@FDA database and consisted of industry-sponsored postapproval clinical studies conducted in the United States.
Postapproval study information included 600 trials registered on ClinicalTrials.gov that were primarily aimed at producing additional efficacy and safety data in approved or unapproved uses. The majority of these trials were for novel therapeutics being evaluated in oncology- and hematology-related indications.
After analysis, the researchers found that most of these trials examined agents for novel or expanded indications (60.5%) or widened populations of the initially indicated condition (20.3%).
In addition, several of these studies were nonrandomized (59.8%), had small sample sizes (median, 44 participants), and did not include comparators (63.5%).
“Among 600 trials, only 12.0% exclusively evaluated the originally approved indication,” the authors wrote.
The researchers acknowledged that a key limitation of the study was the inclusion of only postapproval clinical studies affiliated with pharmaceutical companies.
“The FDA may need to impose additional postmarketing requirements to generate further evidence for the original indications,” they concluded.
The study was funded by the Tufts University School of Medicine and the Laura and John Arnold Foundation. The authors reported financial affiliations with Johnson & Johnson, Medtronic, the Blue Cross Blue Shield Association, the National Institutes of Health, the Laura and John Arnold Foundation, and the Food and Drug Administration.
SOURCE: Skydel JJ et al. JAMA Netw Open. 2019 May 9. doi: 10.1001/jamanetworkopen.2019.3410.