WASHINGTON—A new draft of House legislation that creates significant new regulatory leeway for drug and device companies now includes more funding for the National Institutes of Health. The revised draft represents a compromise with Democrats who withdrew support for the initial bill.
Leaders of the House Energy & Commerce Committee are circulating a new “discussion draft” of the 21st Century Cures bill that would raise NIH funding to $31.8 billion in fiscal 2016 and to $34.85 billion in fiscal 2018. Congress appropriated $30.3 billion for the NIH in fiscal 2015, and President Barack Obama requested (PDF) $31.3 billion for fiscal 2016.
The bill would also create an NIH Innovation Fund of roughly $2 billion per year between fiscal years 2016 and 2020. The fund would support precision medicine, young emerging scientists and other unidentified sources of medical innovation.
Research!America, an advocacy group promoting healthcare research, said it was “thrilled” by the new version of the bill but warned that the final legislation must allow the NIH to decide how to allocate the dollars.
But consumer watchdog Public Citizen called the NIH funding boost a horse trade “providing perks to the pharmaceutical and medical device industries to approve medications and devices faster based on weaker evidence.”
The bill would significantly revise regulations governing the Food and Drug Administration's oversight of the drug, device and software sectors.
Pharmaceutical Research and Manufacturers of America, the industry's trade group, issued a statement applauding the House committee for its work.
The drug industry would benefit from the bill in several ways. Perhaps most significantly would be the proposed legislation's enhanced consideration of “real-world evidence.” Manufacturers would be able to expand a drug's indications based on observational data or registries indicating how it performs in the field. Critics say the provision would lead the government to endorse drugs for new uses without adequate testing for safety and efficacy.
The FDA would also be required to develop more surrogate endpoints to allow shorter clinical trials. For example, the FDA might accept evidence that a new cancer drug effectively shrinks tumors rather than requiring a longer and more expensive clinical trial showing reduced mortality.
The device industry would also benefit. A proposed “breakthrough device” program would provide a faster path to market for devices that might substantially raise the standard of care.
An additional provisional section would expand the FDA's “humanitarian device exemption” for products that treat rare diseases. Under current law, a device treating a disease affecting fewer than 4,000 patients per year receives less regulatory scrutiny. The provisional section would raise that cap to 8,000.
Dr. Margaret Hamburg, who stepped down as the FDA commissioner last month, has said the bill's approach is misguided. "Shortening review times is not going to create the scientific understanding and the research and development that needs to be done to translate exciting opportunities in science into new products," Hamburg told Modern Healthcare in a recent interview. “That is the wrong mechanism to achieve that goal.”
FDA officials told lawmakers during a Senate committee hearing Tuesday that the agency is already overburdened and underfunded, compromising its ability to efficiently evaluate new and modified drugs and devices.
The draft bill, however, calls for the FDA to issue new guidance in areas ranging from the use of Bayesian statistics in clinical trials to precision medicine, and would require the agency to convene seven public meetings or workshops. The word “guidance” occurs 98 times in the 199-page bill. But, at the moment, there are no specific additional appropriations for the agency.
The legislation would also revise the regulatory categories for healthcare software. Any technology falling under the category of “health software,” which includes administrative and other software deemed low-risk, would be exempt from FDA scrutiny.
Some clinical decision-support software, which guides clinicians with alerts or alarms, would fall outside that exemption.
It's unclear, however, how much clarity the bill would offer developers of such software, said Bradley Thompson, a health information technology expert with the law firm Epstein Becker Green.
The bill “gives FDA quite a bit of latitude to decide what (clinical decision-support software) to regulate,” Thompson said. “But the original purpose of this legislation was to more clearly define the dividing line between regulated and unregulated software.”
The health IT community also expected the legislation to tackle the lackluster ability of health IT systems to exchange data. The draft bill includes a placeholder for interoperability guidance but no details.
“We have no doubt it will be part of the package before it is formally introduced,” said Dan Haley, vice president of government and regulatory affairs at electronic health-record vendor Athenahealth.
The bill includes a similar placeholder for telehealth. That industry has lobbied aggressively for legislation to create a distinct Medicare billing code for remote-patient monitoring.