The U.S. Food and Drug Administration (FDA) will soon hold a public hearing on how interim data from cardiovascular outcomes trials (CVOTs) can be released without compromising the integrity of the research. Cardiovascular clinical trials are used to assess the safety and efficacy of a drug, but the agency is considering whether the results of these trials should be kept a secret until the research is complete.
According to the Regulatory Affairs Professionals Society, clinical trials are required to determine the risks of drug before it is approved. Tens or hundreds of millions of dollars can be spent on this type of testing. Beginning in the mid-2000s, the FDA started to shift more towards postmarket trials, meaning that they allow companies to sell their product before the trials as long as they are completed by a certain date.
Some trials “are not large enough to assess the risk of rare serious events such as heart attacks, strokes or death.” the FDA states. Large CVOTs are often utilized in these cases to analyze the drug’s safety before it is approved.
The Federal Food, Drug and Cosmetic Act (FD&C Act) requires interim data for postmarketing studies “be made available immediately for public disclosure,” The FDA, however, is worried that releasing data before the study is done will bias the trials. The interim data may indicate the drug is safe or has risks, and these findings might influence people who are directly involved in the research.
In its Federal Register notice, the FDA quoted the International Conference on Harmonization’s (ICH) E9 Statistical Principles for Clinical Trials in stating that “all staff involved in the conduct of the trial should remain blind to the results of such [interim] analyses because of the possibility that their attitudes to the trial will be modified and cause changes in the characteristics of patients to be recruited or biases in treatment comparisons,”
As a potential solution, the agency is advising that the interim results be reviewed by an Independent Data Monitoring Committee (DMC) rather than investigators. However, there are times when this data is made widely available. “Sponsors and other interested parties with access to interim data may have difficulty managing the remainder of the trial in an objective manner, particularly if changes to the trial protocol are needed for other reasons,” the FDA wrote. In the FDA’s upcoming meeting, the agency wants public input on the transparency of interim data for these trials.