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Lawmakers must ask tough questions about the 21st Century Cures Act

Most of us take for granted that the medications we use every day to prevent disease, and those we give our children when they are sick, and the medical devices used to support and improve every part of our bodies from our hearts to our hips are safe and effective. However, the history of American medicine shows that this is a relatively recent phenomenon. The 19th century was replete with a host of fraudulent, unsafe and ineffective drugs: syrups for children containing morphine, medicines for strep throat containing antifreeze, and the near-miss of thalidomide, given to pregnant women in Europe for morning sickness and which caused congenital deformities, but was kept off the market here by the wisdom of the late Frances Kelsey. These products were all associated with a series of tragedies that led to the creation and strengthening of the Food and Drug Administration (FDA), charged with protecting Americans and the public health through food and medical product regulation.

However, it is worth remembering that at each point during the last century as public health officials and Congress tried to strengthen the FDA, companies, investors and even some physicians and patient groups balked at the new requirements as a threat to clinical care, innovation and commerce. Now on Capitol Hill, members of Congress, along with lobbyists from the pharmaceutical and medical device industries and allied patient advocacy organizations, are touting miracle breakthroughs and a new era of medical innovation with the imminent vote on the 21st Century Cures Act. It’s the number one priority for the lame duck session for both House Speaker Ryan and Senate Majority Leader McConnell. Many organizations representing patients, physicians, academic medical centers, and research societies are going along for the ride, as the legislation is tied to a huge boost in funding for the National Institutes of Health, our treasured biomedical research agency in the U.S.

{mosads}To those of us who have watched and studied the FDA for years, this legislation is the culmination of years of quiet, subtle deregulation—death to the FDA by a thousand cuts – as this new law includes worrying provisions that deftly and severely weaken the evidentiary requirements for the approval of new drugs and medical devices. What couldn’t be accomplished in the 20th century, in stopping or rolling back FDA’s mandate through direct appeals, is being done by stealth in the 21st, under the rally cry of #CuresNow, while the details of the regulatory changes within the bill are rarely mentioned if at all discussed.

The problems with the 21st Century Cures Act’s FDA provisions have been covered in leading scientific publications, such as the New England Journal of Medicine, JAMA, Science and Nature.  The most egregious of these encourage the use of anecdotal clinical experience as evidence that drugs are safe and effective; allow antibiotics on the market based on pre-clinical evidence, that is, laboratory or animal studies, with little testing in humans; and weaken the already limited evidence needed to approve medical devices, even allowing companies to farm out the certification of safety of modified devices to third parties, circumventing the FDA altogether.

In the rush to pass this bill, the voices of experts on drug and device regulation have largely fallen on deaf ears in D.C. After all, who could be against legislation that promises cures for the dreaded diseases in our lifetimes? And it’s hard to resist a call for more funding for the NIH, an aspect of the bill that has no objections from any of us. But Congress, this White House and the president-elect have to take a deeper look into these proposals to see what we’ll get for all this research money: less and less information about the pills we take and devices we rely upon, which increasingly will be promoted for more and more uses, for prices that many cannot even afford.

Already there are signs that the agency, seeing the writing on the wall, is ready to capitulate to this new reality. The recent approval of Sarepta Therapeutics’s Duchenne muscular dystrophy drug eteplirsen, which was studied in an uncontrolled fashion in a handful of patients and showed no clinical benefit, shows that a drug company, harnessing the desperation of patients, could bulldoze its way to market at a price of $300,000 a year. While there was dissent within the agency over the drug’s approval, which required overruling the staff directly responsible for the review of the drug, proactively lowering the bar on approval was perhaps a last-ditch attempt to show that the FDA didn’t need new legislation to exhibit new flexibilities in its standards.

We will all rely on medicines one day, if we don’t already, so that each of us has a stake in the future of the FDA. Enacting provisions that weaken the agency ultimately won’t serve us, health care providers, or those who pay for the medications we take. The essential questions facing any user of a drug or medical device are will it prevent disease, extend life or improve health; is it better than nothing at all or existing drugs for the same condition; and does it have serious side effects in the short or long term? We must ask these questions of the currently proposed legislation: will it improve patients’ health; is it going to improve FDA regulatory quality and efficiency; and will the public be better off and safer in the short and long term? The FDA protects not only our safety but also the credibility of the medical products and therapeutics market.  Shortcuts—by changing what counts as robust clinical evidence to include anecdotal patient experiences, uncontrolled, unrandomized “real-world” data, proxy measures or surrogates for clinical benefit— risk promoting not a 21st century of cures, but a return to the 19th century of frauds.  They may serve shareholders’ needs for faster returns on investment but in the long run they will undermine the reputation of the FDA, the pharmaceutical and medical device industries, and the whole of American medicine. And if history is any indication, they may be hazardous to our health.

Gregg Gonsalves is the Co-Director of the Global Health Justice Partnership at Yale Law School-Yale School of Public Health. Daniel Carpenter is a Professor of Government at Harvard University, and the author of Reputation and Power, a history of FDA drug regulation. Joseph Ross is an Associate Professor of Medicine and Public Health at Yale.

The views expressed by authors are their own and not the views of The Hill.


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