A bill to speed the discovery and development of new medical treatments was approved by a strong majority in the U.S. House of Representatives today. The legislation, known as the 21st Century Cures Act, has evolved slightly from the version that won unanimous support in the House’s energy and commerce committee in May. And although it continues to enjoy the backing of hundreds of industry, research, and patient organizations, some scientists and advocates are asking whether the effort to speed cures to patients might unintentionally compromise their safety.
The bill calls for new funding for the National Institutes of Health (NIH) through a temporary “innovation fund,” which would give the agency an extra $8.75 billion over 5 years; $500 million per year would fund specific projects within NIH’s 27 institutes, including research on biomarkers and precision medicine. The remaining money would support young scientists: high-risk, high-reward research; intramural research; and other areas. The fund lost more than $1 billion from its previous $10 billion authorization as sponsors struggled to pay for it amid tight budget restrictions. But Republican lawmakers failed to pass an amendment that would have made the fund discretionary—subject to the annual budget appropriations process and thus budget caps—rather than mandatory.
The bill would also authorize $550 million for the Food and Drug Administration (FDA) and give the agency a host of new responsibilities. These include a new priority review process for some antibiotics, an expedited approval pathway for medical devices, and the creation of new guidance documents to advise drug developers.
Several provisions aim to smooth the approval pathway for high-priority treatments. One would allow FDA to approve certain medical devices based on case studies and published medical reports rather than data from larger clinical trials. Under another, the agency could approve drugs to treat rare, life-threatening infections based on smaller clinical trials. This could make the process easier for drug-makers that have trouble recruiting enough patients. “I think there’s pretty broad understanding that this is necessary,” says Amanda Jezek, vice president of public policy at the Infectious Diseases Society of America in Washington, D.C. “This would pave the way for studies that simply were be not able to be done before.”
But some are worried about the practical consequences of speedier approval. “For us, there are certainly concerns about … the misperception that you might be able to speed innovation by lowering standards for safety and efficacy, and we think that would be a terrible mistake,” said former FDA commissioner Margaret Hamburg at a National Press Club event, just before stepping down from her post in April.
In a perspective published in The New England Journal of Medicine last month, drug epidemiologist Jerry Avorn and public health policy expert Aaron Kesselheim of Harvard University laid out more detailed concerns. They say that new provisions for devices could lower safety standards, and they suggest that fast-tracked antibiotics might pose a risk to patients who do not fit the profile of the limited pool of patients on whom they were tested. They also criticize a provision that would increase Medicare reimbursements to hospitals that use newer and more expensive antibiotics for serious infections. That move is intended to create a greater profit incentive for developers, but some argue it could increase hospital use of the drugs and encourage more bacterial resistance.
Some policymakers apparently share that concern: An amendment passed with the bill directs the Centers for Disease Control and Prevention to study whether this incentive could actually contribute to the resistance problem. The updated version of the bill will now head to the Senate, which has yet to release its own biomedical innovation bill.