LegislationOn July 10, 2014, the U.S. House of Representatives overwhelmingly passed legislation intended to speed the approval of drugs and medical devices and increase funding for medical research. The central aim of H.R. 6, the “21st Century Cures Act” (Cures bill), is to accelerate the drug development process by reducing or removing regulatory obstacles. Specifically, the Cures bill seeks to:

  • Modernize the Food and Drug Administration (FDA) drug and medical device approval process;
  • Place greater regulatory attention on personalized medicine and rare diseases; and
  • Provide for significant funding increases for the FDA and National Institutes of Health (NIH).

The vote margin of 344–77 in the House shows the strong bipartisan support that the Cures bill has been receiving.

A prior version of the Cures bill would have revised the current approval process in a way that many critics, including the FDA, believed would present risk to patient safety, and would have provided brand-name drugs additional market time without competition from generic versions. The legislation that was passed, however, incorporated less extensive modifications to the drug approval process.

A key section of the Cures bill would revise the FDA’s “breakthrough therapy designation,” a program that allows the FDA to accelerate review of drugs that, based on early clinical evidence, present a significant improvement over current treatments for serious or life-threatening diseases or conditions. The Cures bill would authorize the FDA to grant market approval to a drug with breakthrough therapy designation based on early-stage safety and effectiveness test results; however, the drug’s manufacturer would be required to conduct clinical trials establishing safety and effectiveness shortly after marketing the drug. Additionally, medical device makers would be able to apply for breakthrough therapy designation for devices that either treat diseases or conditions for which there are no alternative treatments, or considerably improve upon currently approved therapies.

The Cures bill also would instruct the FDA to study how readily available data, such as information from provider practices or disease registries, could be used to approve new drug indications. Further, it would direct the FDA to consider suggestions regarding methods that could measure a treatment’s effectiveness without having to wait to determine whether a patient’s health was improved, such as use of biomarkers. Additionally, the Cures bill would alter the current method of approval for medical devices by authorizing the use of evidence such as peer-reviewed journal articles and patient medical histories.

With regard to funding, the Cures bill would provide for $8.75 billion over the next five years to the NIH, as well as $550 million in additional funds for the FDA. Funding would come from both the sale of crude oil from the Strategic Petroleum Reserve and a reduction in the amount of Medicaid reimbursement states receive for the purchase of certain durable medical equipment to equal the Medicare reimbursement rate.

Various groups praise the legislation for, among other things, fostering medical research, speeding marketing approval for new products, increasing transparency of the approval process, and focusing efforts on personalized medicine. Critics argue that the legislation would reduce efficacy and safety testing for antibiotics and high-risk medical devices, increase risk from drug-resistant bacteria by encouraging use of antibiotics (and new antibiotics rather than older ones), and increase healthcare costs by adding exclusivity for orphan drugs, among other effects.

The White House has issued a statement of administrative policy (SAP) on the legislation. The SAP mentions various benefits of the Cures bill, such as encouraging biomarker development to improve studies of new therapies, increasing access to and “interoperability” of electronic health records, and advancing personalized medicine. Yet, the SAP mentions various challenges, such as funding levels and mechanisms, the effect on drug costs of extending marketing exclusivity for drugs intended to treat rare diseases, and the “undermin[ing of] regulatory standards by allowing unproven uses of therapies to be marketed to health care payors as though such uses had been proven safe and effective.”

These and many other issues likely will be taken up as the Senate considers parallel legislation this fall. The original sponsors of the Cures bill, Chairman Fred Upton (R-MI) and Rep. Diana DeGette (D-CO), hope to have legislation to the President before the end of this year.

The full text of the legislation and other materials are available from the House Energy and Commerce Committee website. For additional background, see our postings in the Health Law Update blog.