The vast majority of existing data that assesses the effectiveness and safety of drugs, biologics, and medical devices has never been shared with the public in any fashion. The results of clinical trials testing the effects of these products in humans are traditionally shared through medical journals and presentations, but the companies who fund these trials (trial sponsors) and own the data control what data the public sees and how it is presented. Historically, trial sponsors have accordingly only published or acknowledged some of these results, considering the information valuable and propriety. The Food and Drug Administration (FDA) must treat this data as confidential. Thus, the public never benefits from the hidden wealth of data, both for therapies that have failed to attain approval the FDA and those used by millions of patients every year.
The public health benefits of clinical trial data transparency are numerous. The disclosure of detailed data allows independent researchers to review and verify a product’s safety and efficacy, both good and bad. Adverse reactions to drugs can be discovered and better understood before they harm patients. Researchers and companies can look at the data in different ways to learn about new ways to help patients. A more comprehensive picture of an expensive therapy’s effects can allow a better assessment justifying its use over a cheaper alternative. Overall, patients can be more confident in products if they stand up to independent scrutiny and the product’s maker shows it has nothing to hide.
Legislative efforts towards greater transparency have traditionally focused on requiring trial sponsors to register studies in the national database ClinicalTrials.gov, a requirement that informs the government and the public of a study’s existence. As registration has become the expected practice, however, efforts to increase clinical trial transparency have focused on encouraging results reporting by trial sponsors. Health advocates and medical researchers have repeatedly called on industry and all researchers for greater data sharing, culminating in policies like the British Medical Journal’s requirement that its authors commit to providing the detailed data supporting their studies’ conclusions to researchers who request it for re-analysis. Some companies have promised to make clinical trial data for their products available to outside researchers. In 2014, the European Medicines Agency approved a policy to proactively publish clinical study reports for future drugs that receive market approval in the European Union.
Today, ClinicalTrials.gov houses the registration information of more than 178,000 clinical trials, but the summary results of only about 15,000 trials. In November 2014, the Department of Health and Human Services and the National Institutes of Health issued a proposed rule that would formally implement requirements for clinical trial sponsors to submit summary trial results, regardless of whether the tested product was ultimately approved by the FDA. The rule in its current form only seeks to provide summaries of certain aspects of the study, including any adverse events reported in taking the drug. Detailed information regarding the results and methods of a clinical trial would not be submitted to the database.
The proposed rule represents yet another incremental step on the path to greater clinical trial data disclosure and sets the transparency bar a little higher by making at least some reporting of clinical trial results the norm. But the rule’s reach and scope of disclosure is limited. Significant barriers remain to achieving the full potential of detailed data disclosure, mostly fueled by fears that companies owning the data will be subject to unfair competition. Third parties could also misconstrue the data, resulting in exaggerated or inaccurate negative reports that scare doctors and patients away from effective and valuable therapies and subject companies to more lawsuits. These fears can be addressed by controlling access to the data and requiring fairness and transparency from those who use it. Such measures can ensure that those who are granted access to the data aim to contribute to the overall body of scientific knowledge and public health while protecting the data from those who seek to use it for other purposes.
Both companies and patients alike stand to benefit from increased transparency because it can bolster confidence in the drugs, devices, and products the public uses. Further, disclosure allows the scientific community to re-look at clinical trial data and glean valuable new knowledge that companies may not have discovered on their own. All stakeholders—regulators, companies, researchers, and patients alike—must continue working towards solutions that promote greater data transparency.
This post was developed by Caroline Lee, J.D. candidate at the University of Maryland Francis King Carey School of Law, under the supervision of Kathleen Hoke, Director at the Network for Public Health Law–Eastern Region at the University of Maryland Francis King Carey School of Law.
The Network for Public Health Law provides information and technical assistance on issues related to public health. The legal information and assistance provided in this post does not constitute legal advice or legal representation. For legal advice, readers should consult a lawyer in their state.
Support for the Network is provided by the Robert Wood Johnson Foundation (RWJF). The views expressed in this post do not necessarily represent the views of, and should not be attributed to, RWJF.