FDA ‘Grievously and Horribly Broken’: Professor
The FDA regulatory system is “grievously and horribly broken” and there is no signal that it is getting better any time soon. That’s the perspective of University of California San Francisco associate professor of medicine Vinay Prasad. Writing in a hard-hitting online Medpage Today essay, Prasad says the agency’s recent accelerated approval of Biogen’s aducanumab to treat Alzheimer’s disease “shows just how broken the agency is, and reminds us that we all have to pay for it.”
Before turning to the aducanumab controversy, Prasad notes that in late April, the FDA Oncologic Drugs Advisory Committee reviewed six accelerated approvals involving a group of cancer immunotherapies where clinical trials had failed to confirm that the drugs extended survival or improved quality of life. “Yet, in four of the six cases, the advisory committee voted to keep the accelerated approvals intact,” he writes. “The lesson was painfully clear: once the toothpaste is out of the tube it is hard to get it back in. Once doctors get used to using a therapy, pulling a drug from the market or, in this case, revoking indications, is hard, even after the drugs fail to confirm the benefit.”
For the aducanumab accelerated approval, Prasad says it is uncertain whether the reduction in the rate of amyloid plaque on brain scans that the drug achieves means that Alzheimer’s patients live longer or better lives. “Moreover, the drug has various side effects and a whopping price tag: $56,000 a year,” he adds.
“In the last three months,” Prasad writes, “we have seen that FDA does not have the ability to revoke accelerated approvals, even when the drugs and their sponsors fail to meet the promises made. And to wash that down, FDA has now approved another uncertain drug for people suffering from Alzheimer’s, against the wishes of a different advisory panel. With Alzheimer’s disease affecting six million Americans, the financial and human implications of the approval are staggering.”
He cites an estimate that if aducanumab is prescribed to just one-third of eligible patients, it would cost Medicare $112 billion a year, considerably more than any other medication.
According to the essay, many observers don’t fully appreciate how FDA has taken an indefensible position. He says FDA does not guarantee that drugs coming to the U.S. market actually help Americans live longer or better lives beyond what could be achieved without the drugs. “At the same time,” Prasad writes, “FDA insists on interfering in the market and sets arbitrary standards for approval. The combined effect is the worst of all possible worlds: we don’t know if drugs work and the companies can charge massive prices for them.”
Prasad offers two visions of what FDA could be. In one, it fulfills the purpose of shielding desperate, sick, and vulnerable patients from making choices that may not be in their best interests. “By that metric,” he declares, “our system has failed. FDA often makes products available with no robust proof that they are effective, such as in the case of aducanumab and remains reluctant to withdraw the products or specific indications even when trials fail to confirm the benefit of living longer or better lives.”
A second vision, he says, would be to abolish the agency completely, believing that without FDA the market would work things out and the cost of prescription drugs would drop significantly.
“Instead of either of these visions, we have an agency that lives between the two,” Prasad writes. “FDA poses a series of hurdles to get a drug to market, but none of those hurdles are an assurance a drug works or has net benefit to people. Then the agency stamps drugs with their imprimatur…. Small firms are kept off the market and larger firms specialize in jumping through the hoops. Once approved, costs can be astronomical and the FDA seal of approval justifies the high price. Along the way, no one seems to ask whether patients are better off. From this vantage, FDA looks like an agency whose goal is to preserve pharmaceutical profits. Coincidentally, among medical reviewers at the agency, the prime destination after working at FDA is either as a consultant for or an employee of a biopharmaceutical company.”
Prasad closes by pointing out that former FDA commissioner Stephen Hahn has joined a healthcare venture capital firm.