An analysis recently published in JAMA found that clinical trials are not producing the high-quality evidence needed to guide medical decision making. According to the largest comprehensive study of ClinicalTrials.gov, the majority of clinical trials is small, and there are significant differences in methodical approaches.
“Our analysis raises questions about the best methods for generating evidence, as well as the capacity of the clinical trials enterprise to supply sufficient amounts of high-quality evidence to ensure confidence in guideline recommendations,” said Robert Califf, MD, first author of the paper, vice chancellor for clinical research at Duke University Medical Center, and director of the Duke Translational Medicine Institute.
The Clinical Trials Transformation Initiative, a public-private partnership founded by the FDA and Duke, conducted the investigation of 120,000 registered clinical trials. The project goal was to create an easily accessible database for the exploration of groups of studies by therapeutic areas, by type of sponsor, by number of participants, and by many other parameters.
According to Califf, “Ninety-six percent of clinical trials have ≤1000 participants, and 62% have ≤100. While there are many excellent small clinical trials, these studies will not be able to inform patients, doctors, and consumers about the choices they must make to prevent and treat disease.”
Among median trial sizes there was much diversity, with cardiovascular trials often twice as large as those in oncology. The study also showed major variances in critical issues often used to judge the quality of evidence for medical decisions, including the use of randomization, blinding, and data monitoring committees.
“These results reinforce the importance of exploration, analysis and inspection of our clinical trials enterprise,” said Rachel Behrman Sherman, MD, associate director for the Office of Medical Policy at the FDA’s Center for Drug Evaluation and Research. “Generation of this evidence will contribute to our understanding of the number of studies in different phases of research, the therapeutic areas, and ways we can improve data collection about clinical trials, eventually improving the quality of clinical trials.”
Source: CTTI.