In January 2015, the House Energy and Commerce Committee released a discussion draft of the 21st Century Cures Act, a bipartisan bill aimed at overhauling how FDA regulates drugs and devices, which builds on an effort known as the 21st Century Cures Initiative. The initiative launched in April 2014 and has been the subject of many congressional hearings and roundtables, as well as several white papers.
Corresponding to the release of the 21st Century Cures discussion draft, two top Republicans from the Senate Health, Education, Labor and Pensions ("HELP") Committee released "Innovation for Healthier Americans," a report aimed at "identifying and advancing specific steps to better align public policy to support medical innovation and patient access to new medicines and technologies," focusing primarily on ways to improve FDA and the National Institutes of Health ("NIH"). The report, which calls for stakeholder feedback by February 23, 2015, was a precursor to a February 3, 2015 announcement from Senators Lamar Alexander (R-TN) and Patty Murray (D-WA), the Chairman and Ranking Member of the Senate HELP Committee, that they are launching a bipartisan initiative to examine the process for providing safe treatments, devices, and cures to patients and the roles of the FDA and NIH in that process. Similar to the work done on the 21st Century Cures Initiative, the senators are creating a bipartisan working group and plan to hold a series of hearings. Any resulting legislation will likely complement the House's intended 21st Century Cures Act.
The lengthy and long-anticipated 21st Century Cures Act discussion draft addresses regulation of drugs and biologics with an eye toward speeding up approvals (Title I); enhancing discovery and development of new treatments (Title II); changes to the clinical trial process (Title III); reforms for NIH and FDA related to research, hiring, and succession planning (Title IV); and revamping certain aspects of medical device regulation (Title V). A summary of each title is provided below.
Title I. Title I would require FDA to include patient experience data when assessing the risks and benefits of certain therapies and to issue guidance describing the use of surrogate endpoints in the drug-approval process. In addition, it would permit FDA to give early approval to breakthrough therapies and provide a new pathway for approving antibiotics based on the limited and specific patient populations who need them. Another provision of this title seeks to ease patient access to drugs outside of approved clinical trials, commonly referred to as "compassionate use." In order to accelerate new cures for high-need populations, the bill would provide funds to repurpose already-approved drugs for new indications and "streamline" data review for such applications. Title I would also expand exclusivity for several categories of drugs, including new antibiotics, orphan drugs, and certain new drug products if they have an added patient or public health benefit over a previously approved version of the same drug. Perhaps most controversial, one discussion draft proposal would provide up to 15 years of marketing exclusivity for drugs that petition for and receive designation from the Department of Health and Human Services ("HHS") as a "dormant therapy."
Title II. Title II of the draft bill would create an "Innovative Cures Consortium," broader than, but based on, the EU's Innovative Medicines Initiative, to pool the resources of industry, government, and academia to accelerate cure development and delivery by nongovernment entities. Under this title, FDA would be required to issue guidance on accelerating certain therapies, including regenerative medicine and genetic technologies for rare diseases. Title II also proposes definitions for "health software" and related terms that would be incorporated into the Federal Food, Drug, and Cosmetic Act ("FDCA"), which does not currently define these terms. The discussion draft also contains a notation indicating that the drafters are struggling with how to ensure that the definition does not inadvertently exempt products that should be regulated. Data sharing is another key aspect of the draft bill. Title II would require increased data sharing from entities receiving federal funding (through federally funded health insurance programs or NIH grants, for example) and would establish a data-sharing framework to enhance patient treatment and care. The proposed draft legislation would also call for FDA to issue guidance on the coordination of regulatory activities for combination products. The discussion draft contains placeholders for subtitles that would address regulation of diagnostic devices and health care data interoperability, but no further details are available yet.
Title III. Title III focuses on clinical trials and the challenges of balancing the protection of human subjects and resource efficiency for those conducting the trials. One way the bill addresses these challenges is by encouraging the use of adaptive clinical trials, which use prospectively designed benchmarks as decision points to make study design changes. The draft bill would require HHS to provide assistance to any federal agency trying to improve oversight of human subject research, particularly with regard to vulnerable populations. Provisions in this title would also provide a mechanism for FDA to periodically evaluate post-approval studies, taking into account any recent changes in medical practice. Finally, Title III would establish a global pediatric clinical trial network to engage with foreign regulatory entities and share knowledge on pediatric research.
Title IV. Title IV puts a greater focus on the various agencies involved in the discovery, development, and delivery of therapeutic products. Title IV contains proposals to increase research funding for and decrease the administrative burdens on NIH. The discussion draft seeks to accelerate vaccine approval by proposing a 180-day standard timeline and an expedited review process for the Centers for Disease Control and Prevention ("CDC") to evaluate vaccines. Title IV also contains significant proposals related to FDA. It would create a process for FDA to designate eligible infectious diseases for priority review vouchers at least every five years and calls on FDA to issue final guidance on changes to approved biologics. The discussion draft also includes a succession-planning provision addressing how FDA should handle retirements of critical personnel and would promote professional development throughout FDA. The discussion draft also proposes revisions to the Sunshine Act, namely, exempting certain industry-sponsored educational activities from reporting requirements. A provision addressing telemedicine would improve the delivery of health care to Medicare beneficiaries. Title IV contains several other provisions relating to Medicare, which is not the focus of this publication. Finally, this title proposes to ease restrictions on experimental medical devices, subject to listing and informed consent requirements, among others.
Title V. Title V concentrates primarily on medical devices but also contains provisions related to drugs. Certain drugs and biosimilars designated as "American-Manufactured" would be granted an exclusivity extension of undetermined length. Title V would also require FDA to update its Good Manufacturing Practices guidance for drug products. Concerning medical devices, the discussion draft would extend FDA's use of third-party device reviews to two new areas: facility inspections and the assessment and certification of "a facility's capability to evaluate and implement" modifications to approved or cleared devices. Title V also creates premarket notification requirements for Class I devices, relaxes requirements for predicate device indications and humanitarian device exemptions, proposes changes to the classification review process, and sets out supply chain security measures for medical devices similar to those enacted for drugs in 2013. Finally, the draft bill would define "valid scientific evidence" in the medical device context, would require FDA to decide whether to recognize medical device standards developed by outside organizations within 60 days of publication, and would direct FDA to train medical device regulators to use the "least burdensome approach," including publishing updated guidance on the subject.
Although the discussion draft from the 21st Century Cures Initiative is not a formal legislative proposal, the 350-page document has the appearance of a bill in most respects, save a few placeholders and notes. Going forward, the committee will collect comments on the draft and will likely take up the legislation later this year.
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