The 21st Century Cures Act passed the House and Senate with overwhelming bipartisan support and has now been signed into law by President Barack Obama. On its surface, there seems to be little controversy in supporting more funding to the National Institutes of Health (NIH) for innovative research, more expeditious approval of promising drugs by the Federal Drug Administration (FDA), and better approaches to combat the opioid abuse epidemic.
However, there are a number of troubling provisions in this potential law of which the public should be aware.
To outline the advantages and concerns, I created a series of winners and losers from which readers can draw their own conclusions:
- Drug companies: It shouldn’t surprise anyone that the pharmaceutical industry is ecstatic over the law, as the bar for approval of drugs under development is made significantly lower by reducing the rigor required for approval of new drugs by the FDA. Traditionally, rigorous double-blinded peer review studies are required prior to consideration and approval.
Under the new legislation, pharmaceutical companies will only be required to submit a “data summary” for new pharmacological agents or new indications to existing products, which will make the introduction of new products far easier. Also, drug companies will be able to promote off-label usage of their existing products, which means that it will no longer be necessary to conform to previously approved indications, age groups, dosages or routes of administration of drugs currently on the market.
That will greatly expand pharmaceutical markets and distribution channels.
- The NIH and other healthcare research agencies and individuals: The Public Health Service Act will be amended as a part of this law to provide additional funding to the NIH through 2018 ($4.8 billion) to establish the NIH Innovation Fund, which will support promising healthcare research and innovation. The emphasis of this program will be the support of emerging researchers (particularly in the area of the treatment of pediatric diseases) through grants and loan repayment programs to stimulate accelerated advancements in this area.
Three key Obama administration programs that will receive further funding include: The Cancer Moonshot, the precision medicine initiative and the BRAIN initiative to deepen our understanding regarding central nervous system function and disease prevention/treatment.
- Medical device manufacturers: Similar to the benefit to the pharmaceutical industry, the law allows for swifter approval of new medical devices that are considered “breakthrough” with off-label use, which will greatly expand markets and channels of distribution.
- The FDA: Already stymied with deteriorating labs, understaffing and somewhat compromised approval processes for new drugs and medical devices, the FDA is not expected to fare better under the new law.
The primary issue is that the FDA is often seen to work for the pharmaceutical industry (which provides a majority of its funding support) rather than serving to protect the interests of consumers and the public as the independent watchdog and approval agency it is purported to be. Lowering the bar for the rapid and expeditious approval of pharmaceutical and medical device products with expansion of off label uses will only further undermine the FDA’s ability to protect the public from potential harm.
- Evidence-based medicine: The law allows for the use of “real-world evidence”—which may include anecdotal studies or observations, patient or professional opinion, as well as non-validated or scientifically measured outcomes—to justify the approval of a new treatment product. This undermines the movement of the healthcare industry to be more reliant on scientifically applied research methodology through randomized clinical trials and less reliant on empirical or even observational data.
- Regenerative medicine: Regenerative medicine such as stem cell research, which focuses on working to regenerate healthy tissue from diseased tissue in stroke, Parkinson’s disorder, diabetes and Alzheimer’s disease patients, receives no additional funding or support from this law.
- Consumer advocacy groups and patients: Organizations such as the National Center for Health Research lobbied hard against this law due to the obvious potential dangers that will be created with the more liberal approval and utilization of pharmaceutical agents and medical devices. This situation accentuates the “buyer beware” attitude that the general public will be forced to adopt with the release of potentially harmful products.
Jonathan H. Burroughs, M.D., MBA, FACHE, FAAPL, is a certified physician executive and a fellow of the American College of Healthcare Executives and the American Association for Physician Leadership. He is president and CEO of The Burroughs Healthcare Consulting Network and works with some of the nation's top healthcare consulting organizations to provide "best practice" solutions and training to healthcare organizations.