The 21st Century Cures Act 1 is focused on improving the discovery, development and delivery of promising new treatments and cures. It offers solutions to improve how clinical trials are conducted and the FDA’s evaluation of drugs and devices. It has a broad base of support, including “health care organizations, patients, advocates, researchers, and innovators”. 2
- “…the 21st Century Cures Act would create a more streamlined drug approval system by revolutionizing procedures for clinical trials. The new rules would make bringing a medication to market less expensive, less time consuming, and more predictable. The result will be that medications will cost less and patients will have access to them sooner.”– Peter Pitts, a former associate commissioner of the Food and Drug Administration 3
- “PhRMA applauds the House of Representatives for the overwhelming passage of the 21st Century Cures Act and looks forward to continuing our work with the Senate to ensure biomedical advances continue and are available to the patients who need them to live longer, healthier lives.” 4
However there are voices raising questions regarding the possible unintended consequences of the act.
- Steve Avorn, MD, professor of medicine at Harvard Medical School, wrote an article in the New England Journal of Medicine that provides a thoughtful contrarian view on the current chorus of support for the Act. 5
- “Embedded in the language of the 21st Century Cures Act are some good ideas that could streamline the development and evaluation of new drugs and devices; its call for increased NIH funding may prove to be its most useful component. But political forces have also introduced other provisions that could lead to the approval of drugs and devices that are less safe or effective than existing criteria would permit.”
- “Over the past 80 years, this country’s regulatory approach has embraced steadily improving criteria for accurately assessing therapeutic efficacy and risk. Patients and physicians would not benefit from legislation that instead of catapulting us into the future, could actually bring back some of the problems we thought we had left behind in the 20th century.”
- Rita Redberg, MD, a cardiologist at the University of California, San Francisco, and Sanket Dhruva, MD, a cardiologist at Yale University wrote an op-ed piece in the New York Times regarding the 21st Century Cures act. 6
- “The legislation, disingenuously titled the 21st Century Cures Act, would make it possible for companies that produce high-risk medical devices to submit evidence of safety and efficacy based on sources other than clinical trials, including case histories (i.e., the experiences of individual patients). In other words, anecdotal evidence, rather than the scientific studies, could be used to approve devices.”
- “The 21st Century Cures Act would subject millions of Americans to unsafe or untested medical devices. We urge the Senate, as it takes up the bill, to avoid these dangerous provisions. Unlike medical drugs, which can readily be discontinued if problems are found, many medical devices are permanently implanted and cannot easily be removed if found to be defective. Stricter evidence standards and increased federal funding of the F.D.A. are needed to ensure that innovative medical devices lead to better health.”
Will the anticipated decreased development costs lower the cost to patients?
- Not likely.
- In the United States, historically pharmaceutical pricing has been based on what the market will bear, not development costs or value.
Will patients benefit from changing the evidence hurdle for FDA registration?
- Not likely.
- Changing from efficacy to effectiveness moves the decision criteria from causal to correlational.
So, while there has been a robust social media campaign and ground swell of support for the 21st Century Cures Act, the discovery, development and delivery of healthcare innovation is complex. Full speed ahead? Perhaps we should pump the brakes.
References
1 – http://www.gpo.gov/fdsys/pkg/BILLS-114hr6rfs/pdf/BILLS-114hr6rfs.pdf
2 – www.energycommerce.house.gov/icymi/21st-century-cures-interoperability-improves-lives
4 – www.phrma.org/media-releases/phrma-statement-on-house-passage-of-21st-century-cures-act
5 – www.nejm.org/doi/full/10.1056/NEJMp1506964
6 – www.nytimes.com/2015/07/17/opinion/the-fdas-medical-device-problem.html?_r=1
