Alzheimer's patients are desperate for treatment. But the FDA can't throw out its standards.

·5 min read

Like many government agencies, the Food and Drug Administration is at its best when no one notices it, quietly helping us avoid unsafe and ineffective products. Unfortunately, the agency keeps making decisions that garner front-page news.

In June, the FDA approved Aduhelm (generic name aducanumab), the first new treatment for Alzheimer’s disease in two decades. As bioethicists who work in the areas of Alzheimer’s disease and pharmaceutical policy, we can attest that this decision has generated more controversy than enthusiasm – and for good reasons.

The drug has questionable benefits, substantial risks, a prescription label that remains overbroad and a hefty price tag.

The FDA’s cozy interactions with pharmaceutical company Biogen, and its use of the accelerated approval pathway without consulting its committee of independent advisers, also raises concerns.

But there’s one especially challenging issue in the Aduhelm saga that hasn’t received adequate attention: the central role that patient desperation played in the FDA’s decision-making. In justifying Aduhelm’s approval, FDA leadership asserted that patients and families “made it clear that they are willing to accept the trade-off of some uncertainty about clinical benefit in exchange for earlier access to a potentially effective drug.”

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Is patient willingness to try Aduhelm really sufficient to justify approval? Congress has pushed the FDA to give greater weight to patient perspectives, and the FDA has increasingly prioritized patient engagement. But what role should patient perspectives play in drug approval decisions?

The FDA sidelines itself

Alzheimer’s disease is feared because it robs patients of their minds and memories. Patients are justifiably desperate for disease-modifying therapies. Unfortunately, people with a range of other devastating diseases also lack treatment options. FDA leaders have suggested that in the face of such desperation, refusal to quickly approve potentially promising drugs would somehow be anti-patient. But what’s really anti-patient is trading on patients’ desperation by offering up short-term “wins” that will likely only benefit drug companies in the long run.

In approving Aduhelm, the FDA seemingly embraced the view that, for serious and life-limiting diseases without good treatments, their role should be to just get out of the way. This was the foundational premise of the Trump administration’s 2018 “Right to Try” law, falsely billed as a way to facilitate access to investigational drugs for dying patients. But for FDA leadership to adopt this mindset is a shocking abdication of the agency’s responsibilities, both to patients suffering today and to those who will need safe and effective treatments in the future.

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If the standard for drug approval moving forward is simply going to be that “patients are willing to try,” then we don’t need the FDA at all. In fact, sidelining the FDA is precisely the agenda of some conservative organizations. But they’re wrong. As our students quickly learn when they hear the history of ineffective and unsafe treatments that proliferated before the modern FDA, we do need the agency, badly.

Tasked with ensuring that drugs are safe and effective, FDA offers a critical defense against companies profiteering on desperation. Yet in recent years, FDA has repeatedly and frustratingly missed critical opportunities to help politicians and the public appreciate its value.

Up until the 1960s, when the FDA was granted authority to require that drugs were effective before they could be sold on the market, patients and their doctors had little way of knowing which drugs actually worked. Even HIV patients, who were instrumental in pushing the FDA to allow earlier access to promising drugs – staging die-ins on the agency’s doorstep – ultimately came to recognize the importance of rigorous approval standards.

Desperation can't justify bad decisions

To be clear, the FDA absolutely should consider patient views when deciding the fate of new drugs. Science can offer data, but the FDA must then make value judgments. Are the benefits of a new drug worth the risks? How much certainty should be sacrificed for speed of approval? Patients have the greatest stake in those judgments, and FDA needs to hear their voices.

But patients’ willingness to take risks does not mean that FDA should approve drugs without a strong reason to believe they work. A “something is better than nothing” mindset is understandable for individual patients – and that’s why FDA permits some pre-approval uses through a pathway called Expanded Access. But when that mindset is adopted as a regulatory approval standard, it is harmful to patients, the scientific enterprise and trust in FDA.

Holly Fernandez Lynch in Philadelphia in January 2020.
Holly Fernandez Lynch in Philadelphia in January 2020.

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Drug development takes time, and it is undoubtedly tragic that FDA approval of a safe and effective drug can come too late for some sick patients. But the alternative is also tragic. If FDA short-circuits the review process – granting approval earlier and earlier on weaker and weaker evidence – we’ll end up trading good drugs in the future for weak drugs now.

Andrew Peterson in London, Canada, in 2016.
Andrew Peterson in London, Canada, in 2016.

The Aduhelm debacle makes this clear. The drug’s accelerated approval will likely derail critical scientific progress. Patients may be reluctant to participate in future research with the potential to find effective drugs if they can take Aduhelm instead. Drug companies will be satisfied with the pursuit of unproven endpoints in clinical trials if it helps them maximize profit. All told, this will negatively affect future patients, without giving patients today what they so desperately need: effective treatments.

Emily A. Largent in Cambridge, Massachusetts, in March 2016.
Emily A. Largent in Cambridge, Massachusetts, in March 2016.

When our students learn about the FDA’s history, they recognize its importance and value. We don’t have to choose between an FDA that ignores patients and one that uses their desperation as a fig leaf for bad decisions. Nor do we have to choose between approval standards that demand absolute certainty about a drug’s safety and efficacy and a “patient desperation” standard. Patients have a critical role to play in the approval process, but balance is essential.

In close cases, patients’ voices may be determinative. But the approval of Aduhelm was by no means a close case. The FDA needs to find its own voice and remind America what it’s here for.

Holly Fernandez Lynch (@HollyLynchez) and Emily A. Largent (@emily_a_largent) are medical ethics and health policy professors at the University of Pennsylvania Perelman School of Medicine. Andrew Peterson (@_APeterson_) is a philosophy professor at George Mason University and a researcher at University of Pennsylvania Memory Center.

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This article originally appeared on USA TODAY: Alzheimer's treatment: FDA sullies reputation in drug approval

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