The 21st Century Cures Act, which sailed through the House and is now in the Senate, calls into question the FDA’s current regulatory process. Its supporters claim the FDA process prevents innovation in pharmaceutical products. As someone injured by thalidomide in 1960, I (Cronin) am concerned that we'll go too far in weakening the FDA approval process. My co-author (Plymate) has similar concerns. As a prescriber she may lose trust in the label “FDA approved.” Together we explored the history of the FDA as an agency, its purpose, why its regulations were instituted, and what could happen if we went back in time.
One of the greatest tragedies of the last century was the use of thalidomide during the Baby Boom. Its German developer, Grunenthal, and its U.S. distributor, Richardson-Merrell, touted thalidomide’s ability to soothe nerves, quash morning sickness, and thwart miscarriages. Richardson-Merrell executives forged a paper to market the drug as the “first safe sleeping pill” for pregnant women. Their "clinical trials" included 2.5 million tablets distributed to 20,000 U.S. patients in an uncontrolled fashion. Negative data from these trials were not reported, including infant mortality and birth defects. After pregnant women such as my mother were given this drug, many of their offspring were described as “monsters” with "flipper-like" arms and legs. I was born without lower legs and with missing fingers. Around the world, 10,000 babies were born with birth defects.
The U.S. tragedy was reduced by the FDA analyst, Dr. Frances Oldham Kelsey. Alarmed by the lack of scientific research to support thalidomide’s safety and despite pressure from the drug companies, she delayed approval, demanding more evidence. How many infants were affected in the U.S.? We will ever know, but after it was discovered that thalidomide caused major defects, the drug never received FDA approval.
Thus began a new era for the FDA. In 1962, Congress enacted laws setting up our modern FDA. The scientific and medical communities advocated that the FDA require “substantial evidence with effective data from adequate and well-controlled clinical trials” before giving its stamp of approval.
Now, some say the FDA process is too slow, keeping valuable products from patients who need them, even though the FDA has greatly sped up its process. It approves most drugs in 6-10 months, as fast as any other country. Today over 50 percent of approved drugs are fast-tracked through accelerated pathways. This year the FDA approved a record 89 percent of applications for new drugs. On top of that, the agency is in the process of streamlining its “compassionate use” pathways, allowing patients access to drugs still in investigational stages. It will soon take a physician only 45 minutes to complete such a request, and the FDA approves 97 percent of these requests. To us, this already looks more like a “rubber stamp” than a “bottleneck.”
To speed up the FDA further, the House version of the bill would mean physicians could no longer rely on “FDA approval” to ensure that antibiotics and other drugs or medical devices have been adequately tested for safety or effectiveness. The 21st Century Cures allows drugs to be approved if they are simply shown to be “non-inferior” to existing standard drugs. Medical providers need to know new drugs are “superior” to those already available, otherwise why prescribe them? But drugs could be approved based on information from test tubes, animal studies, or simply mathematical modeling. Devices might get through based on evidence from case studies or clinical experience, not aggregate data. Drafters of the bill would have us believe careful follow-up after approval would be an effective way to avoid large-scale disasters, yet the bill specifies no mechanism for the FDA to do so.
As a person injured by thalidomide, I can tell you that the FDA has never conducted a survey to locate victims. The Wikipedia estimate of 17 people injured in the U.S. is based on false data given to the FDA by Richardson-Merrell. But ask most people of my generation if they knew of someone who was missing digits or limbs, and they will reply affirmatively. We could be returning to a time in which physicians had little way of knowing if their prescriptions would help or harm their patients. We do not want the safety pendulum to swing to the extent that another thalidomide is released on the market.
Looking at the patient groups supporting 21st Century Cures, we should be able to say, "We should be leading the world in coming up with new treatments for your unmet medical needs.” We need to do that by strongly supporting innovative, scientific research through the NIH. Congress should save and strengthen the part of the bill which expands NIH funding. Let us, however, remove those sections of the bill that would allow more unsafe, ineffective drugs to be released without adequate testing.
Cronin, a practicing psychologist in Los Angeles, is the author of Mermaid: A Memoir of Resilience (W.W. Norton, 2014), which was chosen as one of The Best Memoirs of the Year by O Magazine. She won the Washington Writing Prize in Short Fiction, and her essays and stories have appeared in the Washington Post, the Huffington Post, the Daily Beast, and she's had a Notable Essay in Best American Essays.
Plymate is an internist/geriatrician in Seattle and serves as co-chair of the National Physicians Alliance FDA task force. She has no financial or lobbyist relation to this topic.