On July 10, 2015, the U.S. House of Representatives passed with an overwhelming majority (344-77), the 21st Century Cures bill (H.R. 6), a high-profile bipartisan bill intended to speed up and improve the process for approving innovative drugs and medical devices, and to address other issues, including those regarding clinical trial design, research funding, and interoperability of health technology (i.e., a health care system’s ability to connect with other systems).
If enacted into law in its current form, the bill will establish a Cures Innovation Fund to support biomedical research, providing $8.75 billion for the National Institutes of Health (NIH) and $550 million for the federal Food and Drug Administration (FDA). It also will reauthorize funding for the NIH through fiscal year 2018.
The legislation includes a variety of offsets to pay for new costs, including provisions related to Medicare drug and durable medical equipment payment policy, Medicare imaging reimbursement, and civil monetary penalties in cases of Department of Health and Human Services (HHS) grant or contract fraud, among others (see our related posts on Health Industry Washington Watch).
Before coming to the House floor, the 352-page bill was subject to several drafts, spending more than a year in development at the Energy and Commerce Committee, where it unanimously passed last month 51-0 after series of hearings and roundtable discussions by lawmakers.
The bill as approved consists of four titles (Title I “Discovery”; Title II “Development”; Title III “Delivery”; Title IV “Medicaid, Medicare, and Other Reforms”). Of particular interest to drug developers, health care IT professionals, and those involved in clinical trials, are the following sections of Title II “Development”:
- Subtitle A, Patient-Focused Drug Development – Would require FDA to implement a “structured risk-benefit assessment framework” that incorporates “patient experience data” in the new drug development process. Patient experience data means data collected by “patients, parents, caregivers, patient advocacy organizations, disease research foundations, medical researchers, research sponsors,” or other parties determined appropriate by FDA, that is intended to facilitate or enhance FDA’s risk-benefit assessments, “including information about the impact of a disease or a therapy on patients’ lives.” Including patient experience data is intended to integrate patient experience with the development of potential treatments.
- Subtitle B, Qualification and Use of Drug Development Tools – Would require FDA to establish a process for the qualification of “drug development tools” that can help “translate scientific discoveries into clinical applications.” For the purpose of streamlining clinical trials, Subtitle B also would allow trial sponsors to request that the Secretary of HHS agree to an “accelerated approval development plan,” which must include agreement on surrogate endpoints, study design, and the magnitude of the effect of the drug on the surrogate endpoint that would be sufficient to demonstrate the safety and efficacy of the product.
- Subtitle D, Modern Trial Design and Evidence Development – Would require FDA to investigate and issue final guidance to assist sponsors in incorporating adaptive trial design and Bayesian methods into proposed clinical protocols and new drug and biologic applications. This subtitle also would require FDA to establish a program to evaluate the potential use of evidence from clinical experience to help support the approval of a new indication for a drug.
- Subtitle G, Antibiotic Drug Development – Would create a process under which FDA and a sponsor could expedite the development and availability of treatments for serious or life-threatening bacterial or fungal infections in patients with unmet needs.
- Subtitle I, Orphan Product Extensions Now; Incentives for Certain Products for Limited Populations – Would extend the exclusivity period for six months for an approved drug already on the market when FDA approves a new indication for the drug for a rare disease or condition. This subtitle also would reauthorize the priority review voucher incentive program for rare pediatric diseases.
- Subtitle L, Priority Review for Breakthrough Devices – Would require FDA to establish a program to provide priority review for medical devices: (1) “representing breakthrough technologies”; (2) without any other approved alternatives; (3) offering significant advantages over existing approved or cleared alternatives; or (4) “the availability of which are in the best interest of patients.”
- Subtitle N, Sensible Oversight for Technology Which Advances Regulatory Efficiency – Would restrict FDA’s ability to regulate software that currently falls within the definition of a medical device under the Food, Drug, and Cosmetic Act. Under this provision, FDA could regulate only “health software” that: (1) is “intended for use to analyze information to provide patient-specific recommended options to consider in the prevention, diagnosis, treatment, cure, or mitigation of a particular disease or condition”; or (2) FDA “determines poses a significant risk to patient safety.” All other “health software” would be exempt from FDA regulation.
- Subtitle O, Streamlining Clinical Trials – Would require the HHS Secretary to harmonize differences between HHS Human Subject Regulations and the FDA Human Subject Regulations. This subtitle also would permit the use of non-local institutional review boards for review of investigational device exemptions and human device exemptions. In addition, it would waive the informed consent requirement for individuals participating in device and drug trials that pose no more than minimal risk to the participants.
H.R. 6 is now on its way to the Senate, which has held a number of hearings related to issues in the 21st Century Cures, but has yet to advance any legislation. Stay tuned.
Summer Associate Yetundi Oni also contributed to this blog post.