Work has been under way in Congress for more than a year on a sweeping new initiative designed to speed drug and device approvals and increase funding for new drug innovation.
It’s called the “21st Century Cures” initiative, and it could have a major effect on what drugs are available, including possible new treatments for Alzheimer’s and other difficult-to-treat diseases.
A draft bill to implement the Cures initiative was released in an updated 200-page document on April 30, by bipartisan leaders of the House Energy and Commerce Committee. It would steer $10 billion in mandatory spending into a National Institutes of Health (NIH) innovation fund over five years and authorize another $1.5 billion in discretionary dollars in each of the next three years. The innovation fund would be used for precision medicine, including funding the work of emerging young scientists.
The draft legislation, which included several “placeholders” for provisions yet to be ironed out, was released by Committee Chairman Fred Upton (R-MI), who said a completed bill would be ready for House floor consideration in June. He was joined by ranking minority member Frank Pallone, Jr. (D-NJ); Rep. Diana DeGette (D-CO), ranking minority member of the House Oversight and Investigations Subcommittee; Health Subcommittee Chairman Joe Pitts (R-PA) and ranking Health Subcommittee member Gene Green (D-TX).
The measure contains numerous provisions affecting the Food and Drug Administration, including reforming clinical trials and establishing an expedited approval pathway for certain medical devices, vaccines and antibiotics.
A committee statement accompanying the discussion draft said it includes provisions to:
- Incorporate the patient perspective in the discovery, development and delivery process.
- Foster development of treatments for patients facing serious or life-threatening diseases.
- Repurpose drugs for serious or life-threatening diseases and conditions.
- Modernize clinical trials.
- Break down barriers to increased collaboration and data sharing among patients, researchers, providers, and innovators.
- Help the development of personalized and precision medicines so the right patient can receive the right treatment at the right time.
- Provide for continued work in telehealth.
- Advance a truly interoperable health system.
- Provide clarity for developers of software products used in health management and medical care.
Some unresolved issues
Despite all of this, lawmakers have been unable to reach consensus on several key points, including how to encourage drug makers to take on the costly and time-consuming task of creating new drugs for rare diseases.
The original plan was to grant longer exclusivity periods for manufacturers of seldom-used therapies for unmet medical needs, and to give U.S. generic drug manufacturers a longer protection timeframe from foreign competitors. Both provisions have been dropped, however, as some advocacy groups raised concerns that a longer exclusivity period would increase costs to consumers while they waited for competitive versions to become available.
Other unresolved issues include areas related to telemedicine, the interoperability of health records and repurposing FDA-approved drugs for other uses. However, there were reports of progress on those issues.
During an Energy and Commerce Health Subcommittee hearing April 30, Pitts said an Energy and Commerce Committee Working Group on Telemedicine is working toward a “bipartisan proposal that will encourage the use of telemedicine services to improve health care quality and outcomes, increase patient access and control costs.”
On repurposing drugs, Upton said at the hearing, “We will continue to work on a policy to provide incentives to develop drugs that, while they may have failed in trials for one indication, show promise to treat patients facing other serious or life-threatening diseases. We need to ensure the scientific promise to help patients plays a more important role than patents in drug development. This policy also will include incentives for doing research on drugs that are FDA-approved but can be repurposed to help patients with different types of illnesses.”
The legislation includes a streamlined process for the FDA to approve breakthrough therapies. It would allow FDA to approve drugs with “early state clinical safety and effectiveness data,” giving manufacturers the responsibility of conducting post-approval studies.
There is agreement by many of the lawmakers involved in the development of the legislation that both FDA and NIH need more money to handle their additional responsibilities, a difficult hurdle in today’s political environment.
“We are asking the FDA to make many changes to its current operation,” DeGetto said. “We should make sure the agency has the resources to carry out these duties.”