Diana Zuckerman, PhD, American Journal of Public Health
December 16, 2016
The public health community welcomed the Obama Administration with enthusiasm. The George W. Bush Administration had been criticized for having “made decisions about important public health issues based solely on political considerations, not scientific ones.”1 Even President Bush’s former surgeon general, Richard Carmona, told a Congressional Committee that he was muzzled on a wide range of topics, including stem cells, sex education, and secondhand smoke.1 The Obama Administration reversed that bias on a wide range of health issues, but one federal agency has been a clear disappointment to many of us who have watched it closely: the Food and Drug Administration (FDA).
The Obama Administration has been a strong voice on many public health issues, such as tobacco control, healthful eating, childhood obesity, campus sexual assaults, and reproductive health. The Affordable Care Act (ACA), despite some disappointments, is still viewed as a major accomplishment.
But while other federal agencies strengthened their scientific credibility, the FDA caved in to political pressure by approving new medical products based on lower standards of scientific evidence. Given the antiregulatory policies of previous Republican Administration, will the Trump Administration also align with industry against public health? Or will it take a more populist position, protecting patients against a system that seems rigged to protect corporate profits?
Food and Drug Administration Promises
Shortly after being named FDA Commissioner, Margaret Hamburg and her deputy Joshua Sharfstein wrote an unprecedented editorial, “The FDA as a Public Health Agency.”2 They addressed the dilemma that the agency is accused of having only two speeds for approval decisions: “too fast and too slow.” They admitted that because new products are approved on the basis of relatively small studies, safety problems are more likely to emerge later, when the products are widely used. But, they pointed out the other side of the debate: “people with life-threatening diseases have no time to wait.” They promised a “public health approach [that] recognizes that the potential good of a new medical product or policy must be balanced against the potential harm.”2(p2494)
The editorial also promised the FDA would be transparent, providing the data on which it bases its regulatory decisions and explaining its decision-making process to the public. They agreed to attack public health problems by collaborating with other federal agencies.
Despite their well-thought-out analysis, several major public health problems have worsened in the last eight years that are linked to the FDA. Pundits predict these trends will worsen under the Trump Administration, but since the Trump campaign expressed concern about the costs of medical treatment without specifically supporting the views of Big Pharma, it may be possible to reverse rather than worsen the lowering of standards at the FDA.
Faster and More Expensive Cures
Congress has repeatedly criticized FDA approval as too slow, holding hearings and considering legislation focused on that message. Despite that pressure, FDA has the authority and responsibility to enforce the law requiring medical products be scientifically proven to be safe and effective. Instead, they increasingly ignored the importance of replication as a key principle in science, frequently approving treatments based on one clinical trial rather than two. Contrary to the Obama Administration’s commitment to cure cancer, the FDA approved many cancer drugs based on shorter-term studies of smaller numbers of patients, usually allowing companies to study outcomes measured by biomarkers rather than meaningful clinical outcomes such as survival or fewer days in the hospital.3 The FDA required postmarket studies to shore up that weaker scientific evidence, but only a small minority reported a significant clinical benefit.3 The problem of unproven treatments is not unique to cancer drugs; once a drug or medical device is on the market, it stays there for many years even when its safety and benefits are not confirmed.4,5 This contributes to skyrocketing health care and insurance costs, as patients pay for expensive new treatments that are ineffective or inferior to older, less expensive treatments.
Unproven Medical Devices
The Obama Administration made a promising start in 2008 when they selected a new director to oversee the FDA Center for Medical Devices and Radiologic Health. The Center had never received the resources or attention it deserved, and in 2008, fewer than five percent of medical devices, including the minority of implants, were required to be tested in clinical trials. The FDA sought advice from the Institute of Medicine (IOM), which issued a report in 2011 referring to the major FDA pathway for getting devices on the market, called the 510(k) process, as “fatally flawed” because it did not require evidence of safety or effectiveness.
Paradoxically, the scathing IOM report dramatically halted FDA reforms of device standards rather than inspiring a public health–oriented change of standards. The IOM report concluded that the defective 510(k) pathway could not be fixed. One possible solution could have been to move more devices to the more stringent approval process, requiring clinical trial evidence of safety and effectiveness for all life-saving and life-sustaining implanted devices, such as cardiac implants, spinal implants, and joint replacements.
Instead, the FDA took the opposite approach, changing the definitions to justify not requiring evidence of safety or effectiveness for life-saving devices. For example, the FDA had previously claimed that many spinal implants were eligible for 510(k) reviews because they were “moderate-risk” rather than “high-risk.” The FDA now categorizes both moderate-risk and high-risk devices as appropriate for the no-clinical-trials-required standards of a 510(k) review. The more stringent review that requires one clinical trial, which FDA used for fewer than five percent of medical devices in 2008, is used for even fewer devices today. And despite promises of transparency, no scientific evidence for most devices is available to the public.6
The FDA has been strongly criticized for approving opioid drugs that have been widely abused, resulting in an epidemic of abuse and fatalities. The FDA is clearly not the major culprit, but has the epidemic been exacerbated by FDA decisions? The FDA has been criticized for approving opioids that claimed to have abuse-deterrent properties, even when patients could easily find ways around those deterrents. And, although the FDA is not supposed to interfere with the practice of medicine, the law encourages the FDA to use risk-mitigation strategies (called REMS) to reduce misuse of drugs that could be harmful. The FDA spent years developing REMS that require physicians to be trained on how to reduce inappropriate opioid prescribing, but those REMS were never proven effective.7 It has become increasingly obvious that those REMS aren’t working well. It wasn’t until 2016, after several US senators cited the opioid epidemic as a reason to reject the nomination of Robert Califf as FDA Commissioner, that the FDA announced changes in its opioid policies. None involve working with other federal agencies that are also involved in trying to reduce opioid abuse.
Under the Obama Administration, health disparities have been a major public health concern. But, like the administrations before them, the strategy has been to improve access to medical care, with no attention to the FDA’s failure to require diversity in clinical trials or statistical analyses to determine if new drugs or medical devices are safe and effective for major demographic subgroups. A recent analysis by the National Center for Health Research found that most drugs or devices are not adequately studied on people of color or patients older than 65 years; this means that treatment safety and efficacy is usually unknown for most Medicare patients and many Americans (http://bit.ly/2fsbEnw). Although women are almost always included in clinical trials, almost half the studies did not analyze data to determine if the drugs or devices were safe and effective for women (http://bit.ly/2fsbEnw).
Issues Have Gotten Worse
Health disparities, the opioid epidemic, and the skyrocketing costs of prescription medical treatments that are undermining the ACA, Medicare, and Medicaid, are three major public health issues. Those issues have gotten worse, not better, in the last years of the Obama Administration’s FDA. Harmonization with other federal programs has not improved.
The FDA will need to dramatically change course in the next administration to address those public health problems. And while strengthening patient safeguards may not seem a likely priority for a Republican Administration, candidate Donald Trump promised to do things differently. Unfortunately, his only words about FDA focused on the need for “new and innovative” treatments, rather than safe, effective, or affordable ones. Since he apparently isn’t beholden to pharmaceutical and medtech company contributions, can he be persuaded to follow through on his populist promises by helping patients get what they really want—treatments that work and don’t destroy their quality of life? Those are the criteria that previously made FDA approval the gold standard for the world.
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