In a rare act of bipartisanship, the House voted 344-77 last week in favor of the 21st Century Cures Act. The legislation would provide an additional $9.3 billion over the next five years to fund the National Institutes of Health and establish a Cures Innovation Fund to support breakthroughs in biomedical research. It would also provide $550 million in added funding to the Food and Drug Administration over the same period.
Supporters of the legislation, including the Pharmaceutical Research and Manufacturers of America and the Advanced Medical Technology Association, say it will remove regulatory roadblocks in the FDA review process for medications and medical devices. That, they say, would reduce the costs of bringing a product to market and ultimately lower the cost of those therapies for patients.
A key provision would change the FDA's current “breakthrough therapy designation,” a program that began as part of the 2012 FDA Safety and Innovation Act. That law allows the agency to speed up the review of drugs whose early clinical evidence shows they are better than current treatments for serious or life-threatening diseases.
The bill would also allow the FDA to grant market approval to a drug with breakthrough designation based on its early-stage testing for safety and effectiveness. After starting to market a drug, the drug's manufacturer would be required to conduct clinical trials to demonstrate safety and effectiveness. Medical-device makers also could apply for breakthrough designation for products that treat conditions where no alternative or no better alternative exists.
A Congressional Budget Office report last month estimated the program would cost $106 billion to implement beginning in fiscal 2016 through fiscal 2020 but would ultimately reduce federal spending by as much $12 billion over the next 10 years because of a series of funding offsets, including oil sales from the Strategic Petroleum Reserve.
The bill's critics say it would allow therapies to be sold before they were determined to be safe.
“What they're doing is that they're replacing the burden of proof for drug companies and device companies with a burden of uninformed decisionmaking for patients and doctors,” said Diana Zuckerman, president of the National Center for Health Research, a Washington D.C.-based not-for-profit patient-advocacy organization.
Some supporters have still criticized the bill for a provision that would extend market exclusivity to already approved drugs for as long as six months if they are approved to be repurposed for use in the treatment of rare diseases and conditions.
The Senate plans to craft a bill by the end of the year that's similar to the Cures Act, according to a staffer with the Health, Education, Labor and Pensions Committee.