Mass General Brigham, the state’s largest health care provider, said Tuesday that it will not administer Biogen’s controversial new Alzheimer’s drug to patients, dealing another setback to the Cambridge company and its expensive treatment.
The network, which includes the flagships Massachusetts General Hospital and Brigham and Women’s Hospital, is the latest major US health care system to opt against offering monthly infusions of the drug, called Aduhelm, over concerns about its safety and effectiveness. The Cleveland Clinic, Mount Sinai Health System in New York, and Providence in Renton, Wash., made similar moves in July after weighing the risks and benefits of the medicine. But Mass General Brigham’s decision holds special significance because of its proximity to Biogen.
“We understand that this may be difficult news for some of our patients,” Mass General Brigham said in a statement. “However, we fully support continued evaluation of this medication, and should new data become available, we will re-evaluate this decision.”
The announcement followed a decision on Sept. 20 by the hospital system’s Pharmacy and Therapeutics Committee, a panel of medication experts that reviewed the $56,000-a-year treatment, according to Bridget Perry, a Mass General Brigham spokeswoman.
The evaluation included a “thorough review of clinical, economic and operational considerations,” according to the Mass General Brigham statement, which did not provide details.
Biogen was quick to criticize the decision.
“Patients and their families deserve to have choice and access to FDA-approved treatments, including the only Alzheimer’s disease treatment that addresses a defining pathology of the disease,” Biogen said in a statement. “It is disappointing that Massachusetts General Hospital has chosen to deny patients that choice.
Aduhelm is a monoclonal antibody made from the immune cells of older people with no or uncommonly slow cognitive decline. It reduces a sticky protein called amyloid that clumps into plaque in the brains of people with Alzheimer’s. Biogen and some doctors contend that amyloid buildup causes the cognitive impairment that marks the deadly neurological disease, but that remains unproven and is fiercely contested.
Biogen was unaware of any other hospital systems in Massachusetts that have opted not to administer Aduhelm, according to a company spokeswoman, Ashleigh Koss.
Joanne Pike, chief strategy officer for the Alzheimer’s Association, said the nonprofit group was “appalled” by Mass General Brigham’s decision. The Food and Drug Administration approved the drug on June 7 to treat mild cognitive impairment, she said, “because there is an unmet need, and the treatment is likely to provide meaningful clinical benefit.”
But Dr. Sam Gandy, who runs a center for cognitive health at New York’s Mount Sinai, said the latest announcement is further evidence that experts believe the drug doesn’t work.
“This emperor has no clothes,” he said in an e-mail. He added that no patients or caregivers have asked him about getting the medicine in the past four to six weeks, possibly because they have “become educated by the media.”
Aduhelm was approved by the FDA on June 7 as the first new treatment for Alzheimer’s disease since 2003, but it immediately faced a backlash. The Mass General Brigham announcement adds to Biogen’s woes, but the system’s decision was not entirely surprising.
Two doctors at Mass General Brigham who were helping to develop a policy on the medicine told the Globe in July that the hospital system was unlikely to offer Aduhelm to patients taking blood thinners because of concerns about the risk of bleeding in the brain.
Dr. Teresa Gomez-Isla and Dr. Kirk Daffner, Alzheimer’s specialists at Massachusetts General and Brigham and Women’s, respectively, noted that patients on anticoagulant medications were excluded from clinical trials of the drug. Biogen’s medicine was associated with “microhemorrhages,” or small amounts of bleeding, in the brains of some patients in the studies, but usually didn’t cause serious problems.
Although the FDA on July 8 narrowed its previous recommendations for using Aduhelm, the revised guidelines didn’t mention that it wasn’t tested on patients taking blood thinners. That omission alarmed some prominent US doctors who say such patients could be at risk for more serious bleeding.
At least 8 million Americans take blood thinners, according to the nonprofit National Blood Clot Alliance, making it one of the most commonly prescribed classes of drugs. Anticoagulants keep blood flowing smoothly in veins and arteries, and physicians often recommend them to patients at risk for heart attacks and strokes.
The FDA approved Aduhelm over the objections of an advisory committee of scientific experts who cited weak evidence that the drug worked, including conflicting results in two late-stage clinical trials. Three members of the advisory panel resigned in protest.
The approval also triggered an extraordinary backlash from some doctors and insurers. At the time, the Alzheimer’s Association skewered the $56,000-a-year list price, calling it “simply unacceptable.”
Point32Health, the state’s second-biggest private health insurer ― a combined Harvard Pilgrim Health Care and Tufts Health Plan ― said in July that it will not cover the drug because of fundamental concerns about its effectiveness and safety. A number of affiliates of Blue Cross and Blue Shield in other states have declined to cover it.
The vast majority of the roughly 6 million Americans with the disease — 96 percent by one estimate — are over age 65 and would rely on Medicare as their primary insurer. The Centers for Medicare & Medicaid Services, or CMS, began a review of Medicare coverage of Aduhelm in July, a process expected to take about nine months.
Earlier this month, Biogen executives admitted publicly that the launch of Aduhelm was going slower than expected. A recent story published by STAT reported that just over 100 patients with Alzheimer’s had been infused with Aduhelm as of Sept. 11 — a number that is rising slowly, but is drastically below Biogen’s internal projections and Wall Street’s expectation that thousands of patients would be using the drug by now. STAT obtained the information from a person with knowledge of the company’s deliberations.
Two House committees are investigating the FDA’s approval of Aduhelm and have sought a raft of documents on how the agency made the decision, who was involved, and whether the staff had inappropriate interactions with Biogen executives. Members of Congress have cited STAT’s reporting that Biogen had an off-the-books meeting with a top agency official. The Department of Health and Human Services’ Office of Inspector General has a separate investigation into the FDA’s use of a fast-track pathway to approve Aduhelm.
Jonathan Saltzman can be reached at firstname.lastname@example.org.