Over the last year, the U.S. House Energy and Commerce (E&C) Committee has engaged in a broad public conversation with patients, innovators, providers, regulators, consumers and researchers about what steps can be taken to bridge the gap between advances in science and the approval of new medical therapies, and how to regulate those therapies.
The result of those conversations is proposed legislation known as the 21st Century Cures Act, which seeks to overhaul federal regulation of the development of prescription drugs and devices and provide significantly more funding for National Institutes of Health research. On April 29, 2015, the E&C Committee released its most recent draft legislative proposal to implement this initiative, and the Committee is scheduled to hold a legislative markup session for the bill on Thursday, May 14. While the legislative language released is not complete, the Committee has included provisions that would, according to the stated goals of the Committee: (1) incorporate patient perspectives into the regulatory process and help address their unmet medical needs; (2) build the foundation for 21st century medicine; (3) streamline clinical trials; (4) support continued innovation at our federal public health agencies; and (5) modernize medical product regulation.
The initiative as currently written will, among other things:
- Bolster resources and increase NIH Funding
- Support young emerging scientists
- Outline FDA advancement of precision medicine
- Provide priority review for breakthrough devices
- Improve the Medical device regulatory process
- Streamline clinical trials
- Restructure the Medicare local coverage determination process
The law’s potential impact is far-reaching, affecting companies in the areas of pharmaceuticals, medical devices, biotech, genomics, lab testing, telehealth, dietary supplements and beyond. Many groups across the healthcare spectrum have provided input and comment on the basic concepts of the legislation. There has been strong support from the patient community as well; a December 2014 analysis found that 43% of comments submitted during the drafting process were from patient advocacy groups. Proponents argue that pharmaceutical and medical device research is yielding a number of pre-clinical breakthrough discoveries but few of these breakthroughs are translating into approved therapies. According to Dr. Francis Collins, Director of the National Institutes of Health (NIH), it now takes “around 14 years and $2 billion or more” to develop a new drug and “more than 95 percent of [such] drugs fail during development.” Advocates are saying that this legislation has the potential to stimulate authentic and significant advances in medical therapy innovation policy.
Critics of the legislation, however, have concerns on a number of fronts:
- They argue that the changes would result in approvals being granted to new therapies without sufficient testing for safety and efficacy. Consumer advocacy group Public Citizen described the proposals as “providing perks to the pharmaceutical and medical device industries to approve medications and devices faster based on weaker evidence.”
- Others caution that without including increased funding to the FDA, the legislation would further burden the already over-stressed agency, resulting in slower response times and having the opposite of the intended effects to streamline the approval process.
- Privacy experts are also worried that the language in the Act could weaken the HIPAA privacy protections for patient data. Most notably, the draft legislation would allow protected health information to be used for research purposes without patient consent as long as it's being used by covered entities or their business associates.
The Senate Health, Education, Labor & Pensions (HELP) Committee, under the leadership of Chairman Lamar Alexander (R- Tenn) and Ranking Member Patty Murray (D-Wash), is developing a similar blueprint. Earlier this year, the committee issued a white paper "Innovation for Healthier Americans: Identifying Opportunities for Meaningful Reform to our Nation's Medical Product Discovery and Development." Hearings have been held and the committee has formed a working group on this subject.