America’s science policies are changing. America’s scientists need to change with them. The recent controversies over the Food and Drug Administration’s accelerated approval of an Alzheimer’s treatment are a lesson in the costs of failing to do so.
A year ago, the FDA fast-tracked Biogen’s Aduhelm (aducanumab) — the first new Alzheimer’s drug in almost 20 years — even after an expert panel nearly unanimously recommended against giving it the green light. The decision has been so contested that lawmakers are now trying to change the FDA’s accelerated approval process.
Besides fueling controversy, the approval is a rare reveal into the hidden workings of America’s scientists — and how those workings can be flawed.
The purpose of accelerated approval is to allow people with serious, life-threatening diseases lacking effective treatments to take experimental treatments the agency hasn’t yet approved as “safe and effective.” The FDA backed aducanumab based on the drug’s ability to reduce beta-amyloid, one of Alzheimer’s defining pathologies. There isn’t yet data that show whether Biogen’s drug helps patients by slowing their progressive disabling cognitive impairments.
Accelerated approval and policies such as “right to try” and expanded access allow patients to take unproven treatments. These policies share a common rationale: Scientists need to listen to patients—and what they’re often hearing is desperation. In explaining aducanumab’s approval, FDA scientists said that patients and their families made it clear they’re willing to accept the drug’s uncertainties and risks to access its potential benefits.
There’s nothing wrong with policies that respond to patients’ desires. The problem is that policymakers and the public too often ignore how scientists can also be driven by personal interests, including desperation.
Following the aducanumab decision, the press uncovered sharp debates within the FDA during the drug evaluation process. Some staff scientists were so committed to approval they had close contact with Biogen employees who were reportedly desperate for the drug to get approved. Some agency officials collaborated with Biogen early on to chart a road map to approval, flouting the usual process of expecting the company to make its case independently. The Department of Health and Human Services’ Office of Inspector General is now investigating these relationships. Three FDA advisers resigned over the approval.
Aducanumab wasn’t the only potential Alzheimer’s treatment overhyped this past year. Cassava Sciences’ drug simufilam, promoted as the first to significantly improve cognition of Alzheimer’s patients, was rewarded more than $20 million in National Institutes of Health grants which it leveraged into massive investments to support the drug’s development. But in August 2021, a whistleblower claimed there was data manipulation and flawed methods. The company now faces investigations by the NIH and the Securities and Exchange Commission.
Scientists’ professional judgments can also be shaped by money as a key measurement of the importance of research and a scientist’s reputation. In the biopharma world, potential revenue is often used to denote how “innovative” a discovery is: The more money a product is likely to generate, the more it’s seen as innovative.
Scientists’ failure to reckon with their uncertainties and personal motivations has real consequences for patients. Medicare announced in April that it won’t cover aducanumab for patients except in clinical trials; Biogen will “substantially eliminate” commercial production of the drug. Instead of fulfilling patients’ hope, the accelerated approval of aducanumab betrayed it — and further undermined the credibility of public processes around science.
Jason Karlawish is a professor at the University of Pennsylvania’s Perelman School of Medicine and author of “The Problem of Alzheimer’s: How Science, Culture and Politics Turned a Rare Disease into a Crisis and What We Can Do About It.” ©2022 Los Angeles Times. Distributed by Tribune Content Agency.