Don't relax FDA drug approval process, experts warn
Experts are warning that moves to deregulate America's drug approval process could see a flood of unproven and even harmful new drugs enter the market that could threaten human health.
The warning follows in which he said the US Food and Drug Administration鈥檚 drug approval process was 鈥渢oo slow and burdensome,鈥� and where he promised to 鈥渟lash the restraints, not just at FDA but across our government.鈥�
Trump鈥檚 claims reinforce comments he made in January to pharmaceutical industry executives, where he said: 鈥淲e鈥檙e going to be cutting regulations at a level that nobody鈥檚 ever seen before,鈥� 鈥� adding that up to 80 per cent of regulations could be slashed.
In a report today in the medical journal , three experts say moves to deregulate the drug testing and approvals system will harm health everywhere, not just in the US, and that such moves will also stifle innovation and waste patients鈥� and taxpayers鈥� money.
Rigorous clinical studies are still the best way to learn whether a drug works.
One of the letter鈥檚 authors,聽聽and Royal Prince Alfred Hospital, says Trump鈥檚 argument is consistent with a history of neoliberal economic thinking that claims regulatory agencies are systematically biased towards excessive caution, and that the burden of testing a drug鈥檚 efficacy before it comes to market outweighs the benefits.
鈥淭hey argue that potentially harmful drugs can be identified quickly after they go on sale,鈥� says Professor Rasko, 鈥渁nd that the FDA runs an overly stringent system that withholds or delays safe and effective drugs from patients.
These arguments are wrong, he says.
鈥淭he most extreme proponents of deregulation say the market should be the sole arbiter of utility: if a medicine sells well, then it must, therefore, be safe and effective.
鈥淎 more moderate version of this argument says reliable information on safety and efficacy can be collected after a drug is on sale, through observational studies or using biomarkers.
We鈥檙e going to be cutting regulations at a level that nobody鈥檚 ever seen before.
But Rasko and his co-authors, policy researcher, Douglas Sipp and health economist, Christopher McCabe, say relaxing the FDA鈥檚 regulatory system will subject patients to drugs that may be toxic.
They point to the iconic example of thalidomide 鈥� a 1950s drug prescribed for nausea during pregnancy 鈥� that caused more than 10,000 birth defects worldwide.
鈥淓ven 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鈥檚 disease), were found to cause harm only after they had been tested in large, mandatory trials 鈥� effects that were not seen in the smaller trials,鈥� say the authors.
Another problem with deregulatory arguments is the issue of safe but 鈥榰seless鈥� drugs.
鈥淯ntested drugs can be reasonably safe but provide no benefit,鈥� says Rasko, 鈥渁nd unregulated markets are hopeless at sifting out these 鈥榝utile drugs鈥�. We only have to consider the multibillion-dollar industries in homeopathy and other pseudo-medicines to see this.
鈥淭hese ineffective pills and potions are a massive waste of money and provide false hope to millions of people worldwide. What鈥檚 more, for progressive diseases such as cancer or multiple sclerosis, if a doctor were to prescribe a drug that didn鈥檛 work, she鈥檇 be giving a disease a free pass.鈥�
Meanwhile, the current regulatory system is working well, say Rasko and his colleagues.
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, one for lack of safety, and seven for both reasons.
Rasko says it鈥檚 important to consider how far we have come thanks to regulatory agencies like the FDA, and what鈥檚 at stake right now as President Trump聽.
鈥淭he 1938 US Food, Drug, and Cosmetic Act required only that drugs demonstrate safety,鈥� he says. 鈥淚n 1962, new legislation demanded that marketed drugs also go through well-controlled studies to test for therapeutic benefit.
鈥淢ore than 1,000 medical products were subsequently withdrawn after reviews found little or no evidence of efficacy. The free market that existed before 1962 revealed no connection between a drug鈥檚 ability to turn a profit and its clinical usefulness.鈥�
The same is likely to be true of any future deregulated market, the experts warn in聽Nature.
鈥淧atients and doctors must have access to reliable information to make educated and ethical choices.
鈥淏ut reliable information costs money and no one will invest in producing good quality evidence if they can make the same profit on a drug or technology without it.
鈥淩igorous clinical studies are still the best way to learn whether a drug works, and regulation is essential to ensure that these studies are conducted.鈥�