Patient-Level Trial Data Sharing: Issues

New England Journal of Medicine
October 24, 2013  Vol. 369 No. 17

Access to Patient-Level Trial Data — A Boon to Drug Developers
Hans-Georg Eichler, M.D., Frank Pétavy, M.Sc., Francesco Pignatti, M.D., and Guido Rasi, M.D.
N Engl J Med 2013; 369:1577-1579October 24, 2013DOI: 10.1056/NEJMp1310771

The provision of access to clinical trial results that include patient-level data is generating much debate. A growing chorus of transparency advocates is pushing for open access to these data, making a case on the basis of respect for patients’ altruism, the need to safeguard public health, and distrust in the integrity and completeness of published trial information.1 We at the European Medicines Agency (EMA) have been actively engaged in this debate, and the EMA has recently published a draft of a policy that would make patient-level data in its possession publicly accessible. The principle of privacy protection will inform the EMA’s policy and activities; robust and proportionate measures will be adopted to safeguard patients’ privacy, in compliance with applicable data-protection legislation.2

Pharmaceutical-industry organizations, however, have expressed concern that “one of the risks to innovation is disclosure to competitors of companies’ trade secrets and proprietary information that could allow others to `free ride’ off of the substantial investments of innovators”; they fear “degradation of incentives for companies to invest in biomedical research.”3

Industry leaders have rightly complained about the unsustainability of the current drug development and business model. The timelines and costs of clinical drug development are increasing relentlessly, and the attrition rate of assets in development remains high. At the same time, growing cost pressures in all health care environments are forcing restrictions on drug use, aiming to limit coverage only to patients who can be expected to benefit from a given intervention and for whom that intervention is clearly cost-effective.

Contrary to industry fears, we argue that access to full — though appropriately deidentified — data sets from clinical trials will benefit the research-based biopharmaceutical industry. We predict that it will help to increase the efficiency of drug development, improve cost-effectiveness, improve comparative-effectiveness analysis, and reduce duplication of effort among trial sponsors…

Health Law, Ethics, and Human Rights
Preparing for Responsible Sharing of Clinical Trial Data
Michelle M. Mello, J.D., Ph.D., Jeffrey K. Francer, J.D., M.P.P., Marc Wilenzick, J.D., Patricia Teden, M.B.A., Barbara E. Bierer, M.D., and Mark Barnes, J.D., LL.M.
N Engl J Med 2013; 369:1651-1658October 24, 2013DOI: 10.1056/NEJMhle1309073

Data from clinical trials, including participant-level data, are being shared by sponsors and investigators more widely than ever before. Some sponsors have voluntarily offered data to researchers,1,2 some journals now require authors to agree to share the data underlying the studies they publish,3 the Office of Science and Technology Policy has directed federal agencies to expand public access to data from federally funded projects,4 and the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) have proposed the expansion of access to data submitted in regulatory applications.5,6 Sharing participant-level data may bring exciting benefits for scientific research and public health but may also have unintended consequences. Thus, expanded data sharing must be pursued thoughtfully.

We provide a suggested framework for broad sharing of participant-level data from clinical trials and related technical documents. After reviewing current data-sharing initiatives, potential benefits and risks, and legal and regulatory implications, we propose potential governing principles and key features for a system of expanded access to participant-level data and evaluate several governance structure…