Last week the House of Representatives passed what it’s calling the 21st Century Cures Act. The 362-page proposal contains regulatory changes that, according to the bill’s cover page, will “accelerate the discovery, development, and delivery of 21st century cures.” The bill’s official summary lists the essential proposals; depending on who is talking, the changes represent either great steps forward in medical research or giveaways to biomedical powerhouses.
One aspect of the bill’s passage in Congress was a political rarity: widespread bipartisan support. It passed out of committee unanimously and out of the House with a vote 344 to 77. Another unusual feature: the bill pledges budget increases to government agencies. The National Institutes of Health, the Food and Drug Administration, and the Centers of Disease Control all stand to receive additional funds over the next five fiscal years. The language in the bill – at least for now — insulates those increases from annual budget wars and future acts of sequestration.
As for the meaty content of the bill, it does propose some dramatic changes in conducting medical research. The bill’s scope is broad, nothing less than the life cycle of research from preclinical to postapproval; its four sections are titled Discovery; Development; Delivery; and Medicaid, Medicare, and Other Reforms. For this blog, I’ll stick to the proposals that are most relevant to clinical researchers, to ethical review committees (aka institutional review boards, or IRBs), and to anyone involved in the protection of participants in medical research.
Protected Health Information in Research (Sections 13442-13445, pages 64-68)
The bill wades into the question of using personal health information in medical research, and emerges with proposals to increase flexibility. The proposed changes could expand the legal definition of health care operations to include research with health data. This could allow for the use and disclosure of protected health information without prior authorization. This section of the bill also proposes to redefine FDA regulated research activities as a public health activity and to define further the elements necessary for authorizing future research.
Patient-focused Development (Section 2001, page 77)
This section reflects the growing focus on patient-centered outcomes. It calls for a formal framework to include patient perspectives – i.e., “the impact of a disease or a therapy on patients’ lives” — while developing a risk-benefits assessment of any new treatment.
Clinical Experience as Postapproval Data (Section 2062, page 108)
The bill calls for a system where information from the use of a treatment can serve two purposes after FDA approval. The House of Representatives wants real-world clinical experiences to help
- Support any new indications for an approved treatment; and
- Satisfy reporting requirements, such as collecting postapproval safety data.
Independent versus Institutional IRB Review (Section 2261)
On balance, the language of the bill seems to endorse the use of central ethical review by an IRB for multisite studies, even as it acknowledges the primary arguments for local review. The language calls for rule changes that reduce delays, updates language to accommodate multi-site studies, and acknowledge any local concerns. The section requires that all research allow the possibility for central review, whether that is by independent IRB, joint review, or another institution’s IRB. It also requires that new guidelines address concerns over local liability for decisions made elsewhere.
The bill does contain a confusing reference here. Even as it requires consideration of the models of central IRB review, it also calls for continued local review by “incorporating community values through the use of local institutional review boards.” If this language becomes law, it could complicate IRB review across multiple sites, possibly by requiring a shared review of any research. This does not seem consistent with the bill’s stated aim of reducing duplications of effort. A change in language could clarify that under centralized review an institution’s participant protection program – not necessarily its IRB – should understand and defend community values.
Central IRB Review for Devices (Section 2262, page 232)
Strictly speaking, the current regulatory language allows only for local IRB review of device research. A short section of the 21st Century Cures Act changes these regulations to match corresponding drug research guidelines and allow central IRB review of device studies.
No Informed Consent for Minimal Risk Research (Section 2263, page 233)
The new bill proposes a category of research that will not require informed consent. This is a more significant change for FDA-regulated research than it is for research covered by OHRP regulations, since OHRP has more areas where consent can be waived. If this language is approved, a range of protocols could become eligible for full waivers of consent. The bill sets two general requirements:
- The research must pose no more than minimal risk to participants; and
- Other measures to protect study volunteers must be in place.
The bill does not specify who will make the determinations about minimal risk or appropriate safeguards, but those questions seem consistent with decisions that IRBs routinely make now.
As it stands now, the 21st Century Cures Act proposes a wide selection of ideas that could change how we conduct and oversee research. The proposals I mentioned here are those that seem most relevant to researchers and to human research participant protection programs. I haven’t mentioned many other sections, such as those that call for greater use of adaptive trial designs; the regulation of drug development tools; changes in developing antibiotic treatments; or those that define “health software” and establish boundaries for what computer applications the FDA should and should not regulate. These are all interesting areas to watch as the 21st Century Cures Act continues on through the Senate.
More News About the 21st Century Cures Act
The 21st Century Cures Act has received wide coverage over the past week. Here’s a sampling of what’s available online:
New England Journal of Medicine: “The 21st Century Cures Act – Will It Take Us Back in Time?”
Wall Street Journal: “House Medical Debate Sparks Drug Safety Debate”
Wall Street Journal: “House Passes Bill to Improve Safety Monitoring of Medical Devices”
New York Times: “The FDA’s Medical Device Problem”
New York Times: “How Not to Fix the FDA”