It is estimated that up to half of all clinical trials conducted and completed have never had their results published.
What’s the Issue?
Reporting biases in the published literature—whereby the results of research are inconsistently or selectively reported—is a well-known phenomenon and has been a source of concern in the health care field since at least the 1980s, particularly in the realm of therapeutic drug trials. Despite several efforts over the ensuing decades to address this issue, it is estimated that up to half of all clinical trials conducted and completed have never had their results published.
This lack of transparency in clinical research has serious implications for patients, providers, and health systems more generally. Although previous efforts have failed to fully address the problem, recent policy initiatives in both the United States and abroad offer new opportunities to address outstanding issues.
In the near term, legislative efforts by the US government are under way to improve clinical trial data sharing. The 21st Century Cures Act, which was passed by the House in July 2015, contains provisions that would direct the NIH to improve the standardization of data housed in ClinicalTrials.gov. These changes are intended to facilitate use of the database by the public as well as communication between the database and other health information technologies (such as electronic health records).
The legislation also directs the FDA and the NIH to establish a repository of all data from certain clinical trials that have been sponsored by either HHS or other developers that have granted the HHS permission to share their data. These data would then be made available for further study by scientific and medical researchers. This effort would run as a pilot program for seven years, after which it could be adopted as a permanent piece of the research infrastructure.
The focus is now on the Senate, which has been developing its own bill aimed at reforming the biomedical research infrastructure. It remains to be seen how the two versions will be reconciled and whether these provisions will be preserved. Regardless, the existing proposals would not substantially affect the FDA’s ability to share data beyond its current initiatives. Congressional action may be required to move this issue forward.
Over the longer term, it is unclear how the existing and emerging data-sharing initiatives will evolve, and their ultimate usefulness will depend on a range of issues that are still under discussion. There are ongoing questions, for example, over some of the details of the European Medicines Agency’s data-sharing policies, including how anonymization and redacting of sensitive information will be handled and how disputes over data sharing will be resolved. The agency’s final policy on this issue is forthcoming.
The major industry data-sharing programs are also largely untested, and it remains to be seen whether those platforms and systems are structured in a way that can allow for meaningful research. Further incentives or enforcement mechanisms may also be necessary to encourage academic researchers to share data more broadly. Careers in academia depend on an individual researcher’s ability to publish based on data collected over months or years, which does not generally encourage broad sharing.
As experience with these data-sharing programs grows, it will likely be necessary to identify and disseminate best practices. The recent IOM report called for the establishment of forums of diverse stakeholders, led by trusted, impartial entities, that could facilitate this exchange and help drive consensus on solutions to outstanding challenges. However, it is unclear which entity would lead such efforts or how the process would be funded.