Last June, Biogen, a drug manufacturer based in Cambridge announced that the U.S. Food and Drug Administration (FDA) had granted “accelerated approval” for a brand name drug called Aduhelm, the “first and only Alzheimer’s disease treatment” that works by reducing amyloid beta plaques in the brain — one of the defining pathologies of Alzheimer’s.
Biogen declared their new drug had “the potential to bring hope to the future of global health … and a great step toward the advancement of holistic ecosystem solutions for this devastating disease.” Biogen cautioned that “continued approval” of Aduhelm “may be contingent upon verification of clinical benefit in confirmatory trials,” but said this “historic moment” would “spark continuous innovation in the years to come.” But what this “historic moment” sparked instead was a tangled controversy over the relationship of drug manufacturers and federal regulators.
Biogen admitted that Adulhelm could cause “serious side effects,” such as temporary swelling in areas of the brain; small spots of bleeding in or on the surface of the brain with swelling; headache, confusion, dizziness, vision changes, and nausea. Some microhemorrhaging was reported, as well as confusion/delirium and altered mental status/disorientation.
Reaction was swift. The advocacy group Public Citizen sent an “emergency response” to the FDA about what they called “the unproven medication.” “We demanded that the FDA revoke its approval because there is nowhere near enough evidence that [Aduhelm] is effective.” The group claimed that “intense pressure” from a number of groups pushed the FDA to “narrow” its prescribing recommendations. Public Citizen asked the FDA to revoke its approval altogether, because “geriatric experts and the FDA’s own independent advisory committee strongly urged the agency not to approve the medication.”
Public Citizen called for an investigation of “inappropriately close collaboration” between the FDA and Biogen. The group said 40% of people who took Aduhelm in clinical trials had swelling and bleeding in their brains, and charged that Biogen was planning to charge an average of $56,000 per patient per year for treatment. “That price tag is so exorbitant that it might single-handedly jack up insurance premiums across the board and drain tens of billions a year from Medicare,” Public Citizen said. Biogen defended the high price tag for Aduhelm treatment: “This is a disease that is already costing $355 billion per year to U.S. society.”
On July 15 the Cleveland Clinic and Mount Sinai health systems announced that “Based on the current data regarding its safety and (effectiveness), we have decided not to carry Aduhem at this time,” and called for “continued research in this area.” “The FDA’s approval of Aduhelm has raised serious concerns and questions by clinicians, patients and caregivers and a cautious approach is required,” Mount Sinai said in a statement. “Mount Sinai Health System will not administer Aduhelm until the outcome of the FDA Inspector General’s Investigation of Biogen is complete.”
The Institute of Clinical and Economic Review (ICER) voted 15-0 that there was no evidence that Aduhelm provides a net health benefit to patients. ICER has also said the drug is cost effective at no more than $8,400 per year. United Health Care, Humana, and several Blue Cross Blue Shield plans decided to wait for further information before covering Aduhelm. In response, the FDA called for an independent federal probe into its representatives’ interactions with Biogen.
Biogen estimates a target population of between 1 million to 1.2 million individuals could use this new drug. One Wall Street firm estimated that Aduhelm sales could reach $5 billion by 2025. But for now, the medication is as twisted in controversy as the disease it targets. Patients and their families who continue to hope for an effective and affordable way to treat Alzheimer’s are understandably frustrated by the delay and uncertainty swirling around this drug rollout.
In early September, two Congressional committees sent a 13-page letter to FDA commissioner Janet Woodcock, MD, saying they were “concerned by reports of unusual coordination between FDA and Biogen throughout the drug’s approval process.” The timing for this FDA investigation couldn’t be worse. An estimated 48 million adult Americans have hesitated to get a COVID shot — many skeptical of the FDA’s assurances of safety. Now 6 million Americans suffering from Alzheimer’s may have similar concerns that Big Pharma and Federal drug regulators have perhaps gotten far too cozy with one another. “Trust the science” is one thing but “Trust the Government” may be more complicated than we thought.
Al Norman worked in the elderly care field in Massachusetts for 38 years, lobbying for programs and funding to help elders and the disabled live independently at home. He is currently the Treasurer of the ‘NO on 1’ Fair Referendum Committee in Greenfield.