By James E. Valentine* & Anne Marie Murphy –
The first month of 2015 has seen two policy proposals that would expand clinical trials data sharing. On January 14, the Institute of Medicine (“IOM”) released its report on “Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risks,” recommending an approach to clinical trial data sharing that parallels the clinical trial life cycle. While IOM does not have authority to enforce these recommendations, its report may set expectations for stakeholders for the responsible sharing of clinical trial data. Meanwhile, the House Energy & Commerce Committee released a discussion draft of 21st Century Cures Act (see our previous post here), which includes provisions on “Improving Clinical Trial Data Opportunities for Patients.” A report from the Senate Health, Education, Labor & Pensions Committee, titled “Innovation for Healthier Americans,” also includes provisions on clinical trial reform. These proposals come just two months after NIH issued its proposed rule for clinical trials registration and results reporting for ClinicalTrials.gov (see our previous posts here and here).
IOM’s Recommendations for Clinical Trial Data Sharing
The IOM report sets out a plan that would allow for the sharing of clinical trial data. Doing so presents risks, burdens, and challenges, particularly on regulated industry. The report takes into consideration the benefits, risks, and challenges of data sharing for key stakeholders, including study participants, sponsors, regulators, investigators, research institutions, journals, and professional societies. IOM sets out four broad recommendations for clinical trial data sharing:
- Data sharing should be the expected norm with commitments from stakeholders to move toward this goal;
- Various types of clinical trial data should be shared no later than the times specified in the report (see timeframes in the figure below);
- Holders of clinical trial data should operationally mitigate the risk and enhance the benefits of sharing sensitive clinical trial data; and
- A multistakeholder body with global reach and broad representation should be convened to address challenges with data sharing.
A key concern addressed by IOM is the risks associated with sharing clinical trial data, particularly participant data and clinical study reports. The report recommends addressing this through controls on data access in ways that would not compromise the usefulness of the data sharing for the generation of additional scientific knowledge. Appreciating that de-identification and data security alone do not always provide adequate protection, IOM determined that data use agreements could be a vehicle for reducing these risks and disincentives for sharing clinical trial data.
Additionally, IOM developed an approach to clinical trial data sharing for various key points along the clinical trial life cycle (see figure below), endorsing specific timeframes and providing recommended formats for sharing clinical trial data.
21st Century Cures’ Proposed Improvements
The draft 21st Century Cures Act includes a subtitle devoted to “Building a 21st Century Data Sharing Framework.” Two of the provisions offered by Representatives Morgan Griffith (R-VA), Leonard Lance (R-NJ), and Larry Bucshon (R-IN) directly relate to FDA-regulated clinical trials. These provisions, Sections 2081 and 2082, seek to (a) establish a data sharing framework to enable patients and physicians to better identify ongoing clinical trials and (b) facilitate further research using clinical trial data.
Section 2081 would amend Section 402(j) of the Public Health Service Act (42 U.S.C. § 282(j)) to have NIH standardize data on ClinicalTrials.gov to allow for easier use of the data by the public and make registration and results information easily comparable. It would also require a data format that employs comprehensive health care terminology, so that it is compatible with electronic health records and can match to diagnosis or procedure coding systems (e.g., the International Classification of Diseases or the Current Procedural Terminology). The focus of this section is recruitment information, such as eligibility criteria. The provision would also require NIH to convene a public meeting to seek advice on these enhancements, including on usability, functionality, and search capability. The deadline for implementation of these requirements would be one year after the date of enactment.
Section 2082 would lead to the development of a clinical trial data system for purposes of allowing registered users to conduct further research using clinical trial data that would serve as a guide for future medical product development. FDA and NIH would enter into a collaborative agreement (to be known as the “Clinical Trial Data System Agreement”) to implement systems that would make standardized, de-identified clinical trial data available. The systems would consist of solely government-sponsored trials of medical products that are approved or have discontinued development. Certain highly qualified individuals at educational institutions or nonprofits could apply to enter into a cooperative agreement with the agencies so long as they are not involved in the sponsoring or conduct of clinical trials and agree to disseminate results of their research. These individuals would serve as neutral third parties, working with industry, academic institutions, and FDA, and would protect confidential information contained in the system.
Three Frameworks Moving Forward
Although they have no direct link to NIH’s rulemaking, it is unclear how either the IOM recommendations or provisions in the 21st Century Cures Act may influence NIH’s proposed framework. For example, the IOM report recommends making lay summaries of results available to trial participants and summary level results be made available publicly. Whether such non-technical and technical summaries should be required is being explicitly considered under the NIH rulemaking.
Both IOM and Congress’s policy proposals have generated additional attention on the benefits and risks of sharing clinical trial information; meanwhile, NIH is continuing to collect comments on its proposed rule through February 19.