America’s science policies are changing. America’s scientists need to change with them. The recent controversy over the rapid approval of Alzheimer’s treatments by the Food and Drug Administration is a lesson in the costs of failing to do so.
A year ago, the FDA fast-tracked Biogen’s EduHelm (edukanumab) — the first new Alzheimer’s drug in nearly 20 years — even as an expert panel almost unanimously recommended against giving it the green light. The decision has been so opposed that lawmakers are now trying to replace the FDA’s accelerated approval process.
In addition to fueling controversy, the approval is a rare reveal in the hidden workings of America’s scientists – and how flawed those workings can be.
The accelerated approval is intended to allow people with serious, life-threatening illnesses to take experimental treatments that the agency has not yet approved as “safe and effective.” The FDA endorsed aducanumab based on the drug’s ability to reduce beta-amyloid, one of the defining pathologies of Alzheimer’s. There is no data yet that shows whether Biogen’s drug helps patients by slowing their progressive disabling cognitive impairment.
Rapid approval and policies such as “right to try” and extended access allow patients to seek unproven treatment. These policies share a common logic: Scientists need to listen to patients – and what they are hearing often is frustration. In explaining the approval of aducanumab, FDA scientists said that patients and their families have made it clear that they are willing to accept the drug’s uncertainties and risks in order to access its potential benefits.
There is nothing wrong with policies that respond to the wishes of patients. The problem is that policy-makers and the public too often overlook how even scientists can be motivated by personal interests, including frustration.
Following the aducanumab decision, the press exposed heated debate within the FDA during the drug evaluation process. Some employees were so committed to scientific approval that they had close contact with Biogen employees who were reportedly desperate to approve the drug. Some agency officials collaborated with Biogen to prepare a road map for approval, flouting the normal process of expecting the company to make its case independently. The Office of the Inspector General of the Department of Health and Human Services is now investigating these relationships. Three FDA advisers resigned over the approval.
Educanumab wasn’t the only potential Alzheimer’s treatment that popped up over the past year. Cassava Sciences’ drug Simufilm, which was promoted as the first to significantly improve cognition in Alzheimer’s patients, was awarded a National Institutes of Health grant of more than $20 million, which will be used to fund drug development. There was massive investment in support. But in August 2021, a whistleblower claimed that there were data manipulation and flawed methods. The company now faces scrutiny by the NIH and the Securities and Exchange Commission.
Scientists’ professional decisions can also be shaped by money, as a key measure of the importance of research and the reputation of a scientist. In the world of biopharma, potential revenue is often used to denote how “innovative” a finding is: the more money a product is likely to generate, the more it is seen as innovative.
Scientists’ failure to consider their own uncertainties and personal motivations has real consequences for patients. Medicare announced in April that it would not cover aducanumab for patients except in clinical trials; Biogen will “substantially end” commercial production of the drug. Instead of meeting patients’ hope, aducanumab’s quick acceptance betrayed it — and further undermined the credibility of public procedures around science.
Jason Karlavish 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.