21st Century Cures Act will distort the meaning of ‘FDA approved’

The term “FDA approved” means a lot to those of us working in health care and the patients we treat. But if the 21st Century Cures Act becomes law — the House of Representatives approved it Wednesday and the Senate will vote on it next week — this mark of trustworthy stewardship will become a shadow of its former self.

Working in medicine and public health in Baltimore, we are witnesses to patients’ distrust of the health care system and the numerous barriers they face to quality, affordable care. We rely on the Food and Drug Administration to ensure that the treatments we provide are both safe and effective. Our patients have also placed their trust in this agency through us as their prescribers.

For example, in discussing medications to treat chronic pain with a 48-year-old single mother of five who works during the day, one of us suggested gabapentin as a treatment option. Although it is a commonly used drug, she had not heard of it. But she was reassured when told that it is FDA approved for this condition. She tried this medication, and it has provided her good pain relief.

As members of the National Physicians Alliance (NPA), we have been closely following the 21st Century Cures Act legislation and its potential impact on what “FDA approved” would mean for new drugs and devices. Our interest in the 21st Century Cures Act emerged from overarching concerns among ourselves and other alliance members that the FDA’s mission to protect public health by “assuring the safety, efficacy, and security” of new treatments was being hijacked by the pharmaceutical and medical device industries. Reviewing the latest version of the bill, released the day after Thanksgiving, we are not sanguine.

The legislation does include some redeeming features, such as increases in funding for the National institutes of Health and for addressing mental health and the opioid epidemic. All of this much-needed funding, however, comes at the expense of gutting the only agency we as providers can rely on to tell us if the treatments we are prescribing to our patients will work and won’t harm them.

The act, for instance, would pressure the FDA to approve new antibiotics, antifungals, and possibly other drugs through a new and faster pathway called the “limited population antibacterial drug” pathway. Through this expedited pathway, new medicines would be approved and labeled for use in patients with “unmet medical needs,” even though these drugs had never been tested in these specific patient populations.

The act would also allow for new high-risk medical devices to be approved based on anecdotal case studies or reports in medical journals, rather than having to be held to the gold standard of testing in clinical trials. On top of that, manufacturers would be able to make changes to these high-risk devices without any FDA oversight. Instead, the changes would be monitored by third-party contractors — paid by industry.

The most recent version of the 21st Century Cures Act has a new addition: prompting the FDA to approve new regenerative therapies based on clinical anecdotes and surrogate endpoints like shrinkage of a tumor instead of clinically relevant outcomes like longer survival or improved quality of life.

These provisions would unravel the FDA, turning it from the treatment watchdog it is today into a puppet of the pharmaceutical and medical device industry. If the 21st Century Cures Act is passed as written, clinicians could be given potentially deadly drugs and devices to prescribe to their patients, blessed by this new version of FDA approval.

The good parts of the legislation — increased funding for mental health, opioid addiction, and the NIH — are not even guaranteed. Those funds would not be mandatory under the legislation and would be subject to the whim of Congress during appropriations battles each year. This horse trade between protecting the role of the FDA and critical funding has become unequal and instead represents a lose-lose proposition for patients and providers.

Despite all of their clinical and scientific knowledge, health professionals need a way to convey to patients that treatments are safe and effective. The imprimatur of the FDA is currently a kind of Good Housekeeping Seal of approval. Weakening the FDA’s standards would undermine our trust and leave patients at a loss. Without a strong FDA, who else will let providers and patients know if a treatment will work or is even safe? Pharmaceutical companies? Medical device manufacturers?

We don’t need #CuresNow. Instead, we need legislation that will let us all continue to trust the term “FDA approved” into the 21st century.

Reshma Ramachandran, MD, is an assistant scientist in the Johns Hopkins Bloomberg School of Public Health and cochair of the National Physicians Alliance FDA Task Force. Zackary Berger, MD, is an assistant professor of medicine at the Johns Hopkins School of Medicine and a core faculty member in the Johns Hopkins Berman Institute of Bioethics.