FDA takes new fire for approving drugs too fast, with too little evidence – JD Supra

Patrick Malone & Associates P.C. | DC Injury Lawyers
Taxpayers and lawmakers may be grasping just how far in the wrong direction the federal Food and Drug Administration has gone in approving prescription drugs for sale on U.S. markets — in too much haste and with too little facts about whether the new drugs really work and are safe.
The issue, of course, may have stormed into public awareness when drug maker Biogen got the FDA to give fast-track approval for Aduhelm. It’s a medication targeted at Alzheimer’s but with light evidence of its benefits to patients. Biogen set such a sky-high price for Aduhelm that Medicare announced one of its biggest, recent premium increases and an expert furor exploded over the med and its approval.
That, in turn, has put the FDA processes under new, intense scrutiny, particularly as critics noted that 14 of 50 new drugs approved last year alone by the agency, including Aduhelm, received expedited review, Axios, the news and information site reported.
The New York Times found that the agency gave its speediest consideration to 278 drugs since Congress allowed this approval process, prodded by pro-business advocates who argued that regulators should not stall innovative treatments and markets could help decide drugs’ benefits. But as the newspaper also reported of the hastened regulatory scrutiny:
“The [FDA expedited] approvals do not prove that a drug extends survival or improves quality of life. Instead, drugs can be put on the market based on a single study with a positive finding — like tumor shrinkage — and kept on the market if a follow-up study proves a benefit. This pathway, meant for serious conditions and unmet medical needs, has given patients earlier access to lifesaving drugs, a point of pride for industry groups like BIO, the Biotechnology Innovation Organization. A BIO representative told lawmakers last week that he supported a pending plan for drug makers to use real-world evidence to more quickly prove that an accelerated approval drug works. PhRMA, [a lobbying group] which also represents drug makers, said it supported the program in its current form.
“Yet critics and watchdog groups contend that Medicare has spent billions on accelerated approval drugs, even as drug makers drag their feet to complete the required follow-up studies which, if unfavorable, can lead to withdrawal of the drug. In some cases, fast-tracked drugs that showed little benefit stayed on the market anyway. Speeding up science has long been fraught: The FDA was heavily criticized for its actions on Vioxx, a pain drug that had been approved under expedited review that was later withdrawn in 2004 over findings that it increased heart attacks and strokes. Even more avenues for expedited reviews were granted under the 21st Century Cures Act in 2016.”
ALS drug advances with spare study
The newspaper and the Associated Press both report that the FDA is coming under new fire as it reviews a new drug, Amylyx, for amyotrophic lateral sclerosis, a fatal neurological disorder. ALS patients and their families have campaigned aggressively for the FDA to review the drug, which its maker itself had planned to conduct more and longer studies on.
But the FDA shocked watchdogs when it reversed itself and told the maker to submit it for agency consideration based on the limited studies it has — and over which experts disagree as to what the research means.
ALS patients and their advocates say that the small information available is enough to persuade them that the drug is better than nothing in dealing with a condition in which medicine typically offers little hope.
But that argument — also offered by patients advocating for Aduhelm in treating Alzheimer’s — puts the FDA out of dealing in science and may cast the agency in a role of offering terrible false hopes, or worse, to people already struggling with awful conditions, critics say.
The Los Angeles Times, now joined by the New York Times, has reported that speedy FDA approval of drugs, combined with poor follow-up on promises that further studies will be conducted, has produced dismal, dismaying results with Makena. It is a medication touted, as the New York Times said, “to forestall preterm birth and improve the health of a baby.” It has become an important but ineffective way, critics say, for doctors to deal with the shame of maternal mortality, especially for black women.
Makena has been heavily promoted and widely prescribed in this country, creating challenges to determine whether the slim, initial evidence for its use supported claims for it that caused the FDA to give it speedy approval years ago. But now, longer, more rigorous trials have raised huge doubts, the New York Times reported:
“By 2019 … results of a large study conducted mostly in Europe were in. They suggested that the drug had no effect: The percentage of women who gave birth preterm while on the drug was about the same as those given a placebo. The FDA examined the data to see if there was a subgroup of patients in the United States, including 113 black women, who benefited. It couldn’t find one. In October 2020, the FDA announced it wanted to discontinue use of the drug. The drug’s maker, then AMAG Pharmaceuticals, asked for a hearing, arguing in part that the studies left open the question of whether their drug benefited high-risk black women. ‘Our view is that given the results of both of these trials, additional research is merited,’ said Francesco Tallarico, general counsel for Covis Pharma.”
Longer view isn’t convincing for speedy approvals
Hmm, isn’t the point of the FDA review to decide, before a drug gets to market, whether it is safe and effective, especially in use in situations of extreme consequence? The New York Times cited research about the overall track record of the FDA speedy reviews:
“Of the 253 drugs authorized under accelerated approval since 1992, nearly half — 112 — have not been proven to extend survival or improve quality of life, according to an investigation in the BMJ published last year. Two dozen of the drugs had been on the market for five years or more. Another study showed that 20% of 93 cancer drug treatments cleared since 1992 were proven to extend overall survival, while others remained on the market after follow-up studies showed more modest gains, like delaying tumor growth.
“The FDA said overall survival improvement can be hard to assess, as it takes years to achieve. That study reported that one drug, Avastin, got accelerated approval to treat glioblastoma, a brain cancer. Even though a follow-up study did not show extended survival or improved quality of life, Avastin still received full approval for that use in 2017. The same drug was used to treat breast cancer and is the only example of the FDA revoking accelerated approval for one use of a drug — despite emotional pleas to allow it for cancer patients in 2011. In 2010, the FDA backed off its decision to withdraw Midodrine for patients with dangerously low blood pressure, just a month after telling the drug maker it had ‘not been able to provide evidence of the drug’s benefit.’ Researchers called the agency’s retreat a ‘dangerous and unwise precedent.’”
In my practice, I see not only the harms that patients suffer while seeking medical services, but also the damage that can be inflicted on them by dangerous drugs. It has become daunting for patients to access and afford safe, efficient, and excellent health care due to the skyrocketing cost, complexity, and uncertainty of treatments and prescription medications, too many of which turn out to be risky drugs.
In our current system, a lot of the important parties in the U.S. health care system — patients, especially, but also doctors and hospitals and insurers — rely on the FDA to uphold its crucial mission of safeguarding us all from drugs that imperil patients, do not work or do so only marginally, and can hit them with bankrupting costs.
Yes, the agency must not be a monolith and must be responsive to rapid advances, as well as the compassionate needs of really sick patients. The HIV-AIDS crisis opened many eyes to how the FDA can be a stubborn blockade to therapeutic progress with lifesaving result.
But there is a safe, appropriate, and sensible middle ground that lawmakers, especially, need to find, so Big Pharma, in its stampede to maximize its profits, does not shove around the guardians of the public’s safety and wellbeing. The FDA, while being responsive to people’s real lives, also cannot offer false hope and allow desperately ill patients to become medical experiments of a kind, testing nostrums on them based on sparse information.
We have much work to do to make the FDA a reliable, evidence-based, cost-conscious protector of the public and the validator of drugs that are safe, effective, accessible, and affordable.
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DISCLAIMER: Because of the generality of this update, the information provided herein may not be applicable in all situations and should not be acted upon without specific legal advice based on particular situations.
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