Friday, August 28, 2015
On July 10, 2015, the US House of Representatives passed H.R. 6, the 21st Century Cures Act, with a vote of 344-77. The passage of the bill, which many have hailed as a noteworthy example of bipartisan collaboration, is the result of more than a year of congressional hearings, roundtable discussions, whitepapers, and political debate. First introduced in April 2014 by Fred Upton (R-MI), chairman of the US House of Representatives’ Energy and Commerce Committee, and Representative Diana DeGette (D-CO), the 21st Century Cures initiative seeks to “accelerate the pace of cures in America.”
The legislative phase of the 21st Century Cures initiative began in January 2015, with the release of the first discussion draft of the bill. This version proposed major changes to the drug and device development process and touched on issues related to human subjects protections, the sharing of clinical trial data, expanded access, adaptive clinical trial designs, orphan drugs, and much more. The reaction to the discussion draft was varied, with supporters hailing the collaborative and transparent nature of the bill’s development, and critics voicing concerns over provisions that they believed would negatively affect patient safety.
Over the next several months, additional discussion drafts were released and the 21st Century Cures Act was further refined, with later versions representing “a less aggressive streamlining of the drug approval process.” Nonetheless, the version of the 21st Century Cures Act that was passed by the House on July 10 proposes significant changes and has prompted debate among scientists, academics, industry representatives, patient advocacy groups, and regulators.
One element of the 21st Century Cures Act that has been met with a mixed reception are provisions that would broaden the evidence base that can be used to demonstrate the safety and efficacy of drugs and devices. Some have praised the changes for their ability to expedite the drug and device approval process, while others have expressed concern that the proposed changes will lower safety standards. In an opinion piece for The New England Journal of Medicine, Jerry Avorn, MD, and Aaron S. Kesselheim, MD, JD, MPH, raised concerns that the bill would take the United States back in time in terms of drug and device approval. They write, “Embedded in the language of the 21st Century Cures Act are some good ideas that could streamline the development and evaluation of new drugs and devices; its call for increased NIH funding may prove to be its most useful component. But political forces have also introduced other provisions that could lead to the approval of drugs and devices that are less safe or effective than existing criteria would permit.”
Tucked within the lengthy 21st Century Cures Act are a number of provisions that apply directly to the clinical trials process and human subjects protections. Early versions of the draft legislation called on the Secretary of the Department of Health and Human Services (DHHS) to make modifications to the existing regulations for the protection of human subjects so as “to ensure that human subject research that is subject to the Federal Food, Drug, and Cosmetic Act or to section 351 of this Act, and is therefore subject to parts 50, 56, 312, and 812 of title 21, Code of Federal Regulations (or any successor regulations), is not subject to subpart A of part 46 of title 45, Code of Federal Regulations (or any successor regulations).” However, in the version that was ultimately approved by the House, that language, which raised concerns among some in the research protections community, was removed.
The legislation now focuses more broadly on directing the Secretary of DHHS to make modifications to existing regulations governing the protection of human subjects in order to: harmonize FDA and DHHS requirements to the extent possible; modernize the provisions for multi-site and collaborative research projects; “reduce regulatory duplication and unnecessary delays;” and “incorporate local considerations, community values, and mechanisms to protect vulnerable populations.” The legislation also calls on the Secretary of DHHS to ensure that federally funded research can use joint or shared review and to provide further guidance on such arrangements. The use of central IRBs for the review of multi-site research is further supported later in the bill by a provision that requires Section 520 of the Federal, Food, Drug, and Cosmetic Act be amended to remove the term “local.” This change will allow for centralized review of FDA-regulated studies involving a device.
Another noteworthy provision would amend the Federal Food, Drug, and Cosmetic Act in order to allow for waivers of informed consent for drug and device research in situations where “the proposed clinical testing poses no more than minimal risk to the human subject and includes appropriate safeguards to protect the rights, safety, and welfare of the human subject.” This change would eliminate one of the significant differences between the FDA and DHHS regulations governing the protection of human subjects.
The 21st Century Cures Act also proposes a number of changes that would allow for easier access to health data for use in research. Specifically, the 21st Century Cures Act calls for amendments to the HIPAA Privacy Rule “to allow: (1) use of protected information for research purposes to be treated as use for health care operations, (2) remote access to information by researchers, and (3) individuals to authorize future use of their information for research.” In addition to the specific provisions outlined above, the bill also addresses the need to include patient perspectives in the drug development process, the promotion of pediatric research, the inclusion of under-represented communities in clinical trials, and the issue of reducing administrative burden for researchers.
With the passage of the 21st Century Cures Act by the House, attention has turned to the Senate, which is working on a parallel bill under the leadership of Lamar Alexander (R-TN), chairman of the Committee on Health, Education, Labor, and Pensions (HELP); and Patty Murray (D-WA), ranking minority member of HELP. Over the past several months, HELP has held a number of congressional hearings and is gathering information as they work to develop their own legislation aimed at fostering biomedical innovation. Most recently, Senator Alexander informed a National Academies panel charged with “develop[ing] a new framework for Federal regulation of research universities in the 21st century” that he welcomes their input and encourages the group to provide preliminary recommendations by the end of the summer, so that their work can be incorporated into the Senate’s efforts.
Earlier this year, Upton and DeGette, who moved the 21st Century Cures Act quickly through the House, shared that they hope to have a final bill signed into law by year’s end. Ultimately, however, the feasibility of that goal is dependent on the Senate, which appears to be moving at a slower pace than the House did.
Portions of this post were adapted from an article titled 21st Century Cures Looks to Modernize Clinical Trials which appeared in the May 2015 edition of the PRIM&R Member Newsletter. The PRIM&R Member Newsletter is distributed once a month to PRIM&R members. To learn more about the PRIM&R Member Newsletter, as well as other member benefits, please visit our website.