After a two-year bipartisan effort led by House Energy & Commerce Committee Chairman Fred Upton (R-MI), Health Subcommittee Chairman Joseph Pitts (R-PA), Ranking Minority Member Frank Pallone (D-NJ), and Congresswoman Diana DeGette (D-CO), earlier today the US House of Representatives approved the 21st Century Cures Act (H.R. 6), by an overwhelming vote of 344-77.
The bipartisan legislation proposes to bring the US health care system into the 21st Century by making changes to the way the US Food and Drug Administration (FDA) regulates many healthcare products, including pharmaceuticals, medical devices, and biologics, and by modernizing clinical trials to deliver better and faster cures to patients. The legislation also empowers the National Institutes of Health (NIH) to invest in science and medical innovation, provide greater support for outstanding scientists, promote pediatric research for rare diseases and birth defects, and take other steps to help modernize US health care research.
The legislation overcame a late snag over the past 10 days relating to concerns expressed by some fiscal conservatives that H.R. 6 establishes new “mandatory” spending totaling nearly $10 billion over five years for NIH biomedical research ($1.75 billion/year) and $550 million for additional FDA resources tied to the modernization effort. The House soundly defeated on a bipartisan basis an amendment offered by several House Republicans earlier today to convert the spending to “discretionary” appropriations (meaning potentially a lesser likelihood of ultimately being spent in the amounts envisioned). That defeat cleared the bill for its eventual final passage.
The bill now heads to the Senate, where behind the scenes work has occurred all year under the leadership of Senate HELP Committee Chairman Lamar Alexander (R-TN), Senator Richard Burr (R-NC), and Ranking Minority Member Patty Murray (D-WA), among others. Chairman Alexander has asked stakeholders for their input and staff has told us that the next step would be circulation of a discussion draft reflecting the Senate’s own policy ideas, rather than simply deliberating over the House-passed 21st Century Cures Act. The “Innovation for Healthier Americans” report issued by Mr. Alexander and Mr. Burr in late January offers some insight into the kids of issues that a Senate bill on FDA modernization would cover.
“Senators Alexander and Murray have reputations for achieving bipartisan consensus on important issues, so there is good reason to be optimistic that the Senate will have the opportunity to consider a well-crafted bill later this year. However, this area of policy is quite complex and stakeholders will need to keep a close eye on the process to protect their interests," noted Arent Fox Senior Policy Advisor and former US Senator Byron Dorgan.
Important provisions of the 21st Century Cures Act include, in part, the following:
- Reauthorize NIH for three years (FY2016 – FY2018).
- Provide increased support for innovative approaches to high risk biomedical research.
- Provide increased support for “young emerging scientists” at NIH, including tuition loan repayment and creation of a grant program to support outstanding scientists.
- Promote pediatric research by establishing a national pediatric research network to pool resources and coordinate activities related to pediatric rare diseases and birth defects.
- Create a third-party scientific research sharing system for clinical trials funded by the federal to share use and analysis of study data beyond each individual project.
- Streamline FDA’s accelerated approval process for drugs and biologics.
- Issue guidance to promote the use of adaptive clinical trial designs and use of Bayesian statistical modeling in clinical trial protocols for drugs and biologics.
- Further expedite the approval of drugs designated as breakthrough therapies as early as possible in the clinical development process.
- Issue draft guidance to clarify how drug and device manufacturers can permissibly disseminate truthful and non-misleading off-label information about the product.
- Promote development of new antibacterial and antifungal drugs.
- Accelerate the Advisory Committee review process for new vaccines.
- Extend the exclusivity period for already approved orphan drugs by six months when the drug sponsor obtains approval of a new orphan drug indication for the drug.
- Establish a priority review program for devices that represent breakthrough technologies.
- Clarify what constitutes “valid scientific evidence” in the context of the medical device review process.
- Expand the number of devices eligible to receive FDA’s Humanitarian Device Exemption by raising the eligibility threshold to diseases and conditions that affect up to 8,000 individuals in the US.
- Create a new technology ombudsman within Medicare to address problems relating to coverage of new and life-saving technologies.
- As a budgetary offset, the bill would limit federal Medicaid reimbursement to States for durable medical equipment, prosthetics, orthotics, and supplies (DMEPOS) to whatever the existing Medicare reimbursement rates are for those items.
- Establish a 21st Century Cures public-private partnership in the United States to accelerate the discovery, development and delivery of innovation cures, treatments, and preventive measures for patients.
This legislation has been a top priority of many industry associations in the health care and biotech area, and its supporters stated that H.R. 6 “has the backing of over 700 groups encompassing patient advocacy groups, rare disease groups, cancer centers, technology groups, top universities, biopharmaceutical companies, and medical device companies.” Some stakeholders have expressed concern that the bill will end up adding more complex regulations for the already highly regulated drug and devices industries to digest and implement, and will result in increased compliance costs. Therefore, the Congress can be expected to continue to deliberate on this legislation and changes are certainly possible.