In his first speech to Congress last month, President Trump chastised the U.S. Food and Drug Administration (FDA) for having a “slow and burdensome” approval process for new drugs. That process, he charged, was holding up the delivery of much-needed treatments to desperately ill individuals.
He then promised to “slash the restraints, not just at the FDA but across the government.”
Trump’s apparent choice for head of the FDA, Dr. Scott Gottlieb, a physician who has strong ties to the drug and medical device industries and who currently serves as a resident fellow at the conservative American Enterprise Institute, has also advocated for speeding up the agency’s approval process.
Congress, too, seems on board with that approach. Late last year they passed — and President Obama signed into law — the 20th Century Cures Act. It makes it much, much easier for drug and medical device companies to push their products through the government approval process, in large part by lowering the bar for the type of evidence the companies must provide to demonstrate the safety and effectiveness of their products.
This loosening of the approval process — particularly the prospect that large clinical trials may no longer be necessary — has raised huge concerns. Critics have warned that it could lead to “frightening” and “disastrous” consequences, for it will expose more Americans to drugs and medical devices that not only don’t work, but that may cause great harm.
The potential to harm
Three more experts — Douglas Sipp of the RIKEN Center for Developmental Biology in Japan, health economist Christopher McCabe of the University of Alberta in Canada and clinical hematologist Dr. John Rasko of the University of Sydney in Australia — added their voices to that criticism late last week in a commentary in the science journal Nature. Interestingly, they point out that not only does drug deregulation put more people’s health at risk (not just in the U.S., but around the world), it also serves as a drain on the economy.
Deregulation, they argue, stifles innovation and wastes patients’ and taxpayers’ money.
And this harm — both to the economy and to people’s health — is not just the result of toxic drugs slipping through a lax FDA approval process, they add. Deregulation will also allow many safe but ineffective drugs onto the market — drugs that also have the potential of damaging people’s lives.
The danger of ‘futile’ drugs
“Unregulated markets are hopeless at sifting out these ‘futile’ drugs (witness the multibillion-dollar industries in homeopathy and other pseudo-medicines), unlike the current system,” explain Sipp, McCabe and Rasko. “In January 2017, the FDA released a report identifying 22 products that were initially promising but disappointed in later-stage clinical trials: 14 for lack of efficacy, 1 for lack of safety, and 7 for both reasons. “
“Futile drugs, even the non-toxic ones, cause real harms,” they add. “They waste money for both patients and taxpayers. Marketing useless drugs wastes industry resources that could be used in developing effective therapies, squanders opportunities for patients to receive beneficial medical care, engenders false hope in miracle cures, and leads to cynicism about the value of research.”
Still, deregulation’s greatest potential for harm, particularly in human terms, lies in the risk that dangerous, toxic drugs will get on the market before they are fully tested. Sipp, McCabe and Rasko cite the tragic example of thalidomide, a treatment marketed in the late 1950s and early 1960s for nausea during pregnancy. Before it was withdrawn, thalidomide caused more than 10,000 babies to be born with birth defects — although not in the United States, because the FDA refused to approve the drug for that use.
“Even in the past dozen years, initially promising drugs, such as torcetrapib (for reducing cholesterol and heart-disease risk) and semagacestat (for improving cognition in people with Alzheimer’s disease), were found to cause harm only after they had been tested in large, mandatory trials — effects that were not seen in the smaller trials,” Sipp, McCabe and Rasko point out.
Needed: reliable information
Trump administration officials and others who argue for “slashing the restraints” around the FDA’s drug-approval process fail to recognize that such actions come with a huge hidden — and very human — cost. Sipp, McCabe and Rasko explain:
Drug companies cannot afford to generate reliable evidence for efficacy unless their competitors are all held to the same high standards. Efficacy requirements level the playing field and ensure that the health sector receives the data needed to inform good therapeutic and economic decisions. The government, insurers, patients and others need to know whether medicines are likely to provide benefits. Patients and physicians must have access to reliable information to make educated and ethical choices.
Rigorous clinical trials remain the best way to generate that evidence — and regulation is needed to make sure that companies do that type of research.
“The FDA’s gatekeeper role makes the medical marketplace function,” Sipp, McCabe and Rasko conclude. “The economic benefits of good research and a healthier population will be lost without incentives to find truly effective drugs.”
Rasko was a bit more blunt in a comment to an Australian reporter: “If these sorts of changes go through,” he said, “I can see a world in 10 years where the snake-oil salesman is back. It will all come down to marketing.”
FMI: You can read the commentary on Nature’s website.