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August 10, 2015
Quarterly Article
Diana M. Zuckerman
December 2024
Dec 19, 2024
Back to The Milbank Quarterly
Within days of the Supreme Court’s decision to save rather than eviscerate the Affordable Care Act, the House of Representatives did something surprising: They passed a groundbreaking health care law with strong bipartisan support. That sounds like good news. However, this groundbreaking health care law is almost as controversial as the previous one.
How is it that the Democrats and Republicans in the US House of Representatives, who agree on so little, managed to agree on a bill called the 21st Century Cures Act? The spokespersons for the bill, Rep. Fred Upton (R-MI) and Rep. Diana DeGette (D-CO), claim that the support reflects a thoughtful, bipartisan working relationship.1 Critics of the bill offer other explanations: 1) most Congressional staff never read the 362-page bill (which in addition to being lengthy was filled with medical jargon that members and staff did not understand), 2) Republican supporters had a powerful lobbying and negotiating strategy that overwhelmed Democrats, and 3) corporations used patient groups to do their lobbying.2
Here are some of the details that will help you judge for yourself.
The 21st Century Cures Act passed the US House of Representatives in July amid praise from hundreds of patient organizations, well-known charities, and universities. These organizations focused on one page of the bill, which provides almost $9 billion of additional funding for the National Institutes of Health (NIH) over 5 years, and on 61 pages that provide research prizes and grants for new scientists. They were optimistic that the bill would provide “cures”—or at least treatments to prolong life—for desperate patients.
Although greatly outnumbered, well-respected researchers and public health and consumer advocates raised alarms that the bill would also drastically lower Food and Drug Administration (FDA) approval standards. More than 150 pages specify drastic changes in how the FDA decides which drugs and devices can be sold in the United States. For example, the proposed law criticizes the gold standard of randomized double-blind clinical trials as old fashioned and urges the FDA to rely on other types of evidence, such as preclinical studies (eg, rat studies and test tube research), case histories (detailed anecdotes about individual patients), and clinical experience (defined in the bill to include anecdotal information from small numbers of patients).3
Those changes would make it easier for drug and medical device companies to sell more of their products more quickly, but unfortunately many of those products will not cure patients, or even help them feel better. The history of the FDA is a history of strengthening legislative safeguards in response to tragedies like thalidomide, diethylstilbestrol (DES), and the Dalkon Shield intrauterine device. Those tragedies resulted in stronger standards for proving safety and efficacy that the proposed law would weaken or eliminate. Critics charge that instead of moving us into the 21st century, the bill takes us backwards.4
The 21st Century Cures Act would also take away the FDA’s authority to ensure that innovative medical software is accurate (Section 2241). Doctors are becoming more dependent on electronic medical records that remind them about a patient’s medical history and warn them about drug interactions and other dangers. People’s lives can depend on that software accurately telling patients and doctors what tests a patient needs, when they need an insulin injection, and other potentially life-saving alerts. If software isn’t regulated, the company is the only one checking its accuracy and reliability. The justification for eliminating the FDA’s authority is that the agency’s review process might delay availability of new software by a few months. But deregulation wouldn’t just eliminate independent review of the software before it is sold—it would also eliminate mandatory public reporting of life-threatening and fixable malfunctions of software already on the market. Under the proposed law, if a product fails and a patient dies, there would be no one to ensure that patients and doctors are warned of those risks. Without that monitoring and oversight mechanism, it would take years before many product flaws became obvious and were therefore corrected.
The proposed law would allow companies to give doctors expensive gifts in the form of tuition and speaking fees for continuing medical education courses at exotic locations, without making those gifts public (Section 3041). This creates an enormous loophole in the Physician Sunshine Act that is current law, passed 3 years ago by Congress to make gifts public in hopes of discouraging physicians from accepting gifts that could influence treatment decisions.
Under current law, new medical devices such as hips, spinal implants, and even many life-saving cardiac devices are almost never required to be proven safe or effective in clinical trials.5 The 21st Century Cures bill would lower the standards even more: Instead of having to prove to the FDA that changes to a previously approved high-risk device do not compromise safety or effectiveness, device companies could pay a third party to review their quality control mechanisms to determine if the company could make changes in the design or materials without applying for FDA approval (Section 2221). Some point out that this creates a strong conflict of interest. Others compare it to the fox paying a friend to guard the henhouse.
The proposed law would also worsen the epidemic of antibiotic resistance. Although it “encourages the development and use of new antimicrobial drugs,” it does so by relying on preliminary studies of small numbers of patients for FDA approval of new drugs (Section 2121). It then encourages hospitals to prescribe those poorly tested new drugs for Medicare patients. Lower standards and high use of unproven drugs by the elderly puts lives at risk. And, encouraging the overuse of new antibiotics instead of saving them as a last resort will spur more superbugs in our hospitals.
In their support for the bill, members of Congress expressed compassion for patients who have nowhere to turn for effective treatments. Many of those same members oppose Medicaid expansion and previously cut NIH research funding by billions of dollars. Unfortunately, the bill’s increase in research funding is still far below what it would have been had Congress not previously cut NIH’s annual budget.
Congress envisions NIH research as the way to save patients’ lives, through scientific discoveries that result in better medical treatments. They don’t realize that the FDA is a public health agency with regulatory safeguards to ensure those treatments are as safe and effective as the companies claim they are. The current FDA leadership is relieved that the proposed bill does not force them to allow anecdotal evidence to trump science, and Democratic staff share that view1—but those assurances hold no guarantee regarding future FDA officials reflecting very different priorities of future administrations.
The proposed law would enable medical products to be sold on the basis of preliminary studies, turning patients into unwitting guinea pigs while making them pay for the privilege. In the past, patients have suffered or died unnecessarily before better research provided clear evidence that a new medical product, such as Vioxx for pain and Avastin for breast cancer, was less safe or less effective than expected. Since new drugs in the United States are always more expensive than older ones (whether they work as well or not), lowering FDA standards would add to the fiscal insecurity of Medicare, Medicaid, and Veterans’ health care. Those higher prices would also stimulate an increase in the costs of health insurance. That is not the “cure” that public health experts are looking for.
References
Diana M. Zuckerman is president of the National Center for Health Research and founder of its Cancer Prevention and Treatment Fund. She obtained her PhD at Ohio State University and did her post-doctoral training in epidemiology at Yale University. She was on the faculty at Vassar College and Yale University and conducted research at Harvard University before moving to Washington, DC, to work on health policy issues in the US Congress and the Clinton administration. She frequently testifies before Congress, the FDA, and state legislators. She is the first nonphysician inducted into the Women in Medicine International Hall of Fame, and serves on the Board of Directors of the Reagan-Udall Foundation and the Alliance for a Stronger FDA. She is the author of five books, several book chapters, and dozens of articles in academic journals.
