FDA approves new Alzheimer's drug. Here's why Medicare refuses to pay.
The makers of Aduhelm hoped their new drug would make history as the first effective treatment to slow the progress of Alzheimer’s disease. Instead, the monoclonal antibody has headed down a more controversial path: While one government agency approved it for use, another declined to cover it, putting the future of the drug in limbo.
Drug critics say the Food and Drug Administration approved Aduhelm prematurely. Supporters counter that the Centers for Medicare and Medicaid Services unprecedented refusal to pay puts it outside the reach of most people who could potentially benefit.
At the heart of the debate lies the question of just how much benefit Aduhelm confers on those who take it — and at what cost.
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The FDA gave a greenlight to the drug last June through its accelerated approval process, a process reserved for serious diseases for which there is an unmet medical need. Contributing to the controversy, an FDA advisory committee voted nearly unanimous against approval. After the agency took the opposite tack, three members of that committee resigned.
Unlike standard approval, accelerated approval allows researchers to focus on whether a drug impacts a specific marker rather than on whether the drug improves any of disease symptoms. The agency has also applied the process to cancer drugs and a drug to treat a form of muscular dystrophy. In some instances, drugs go on to receive full approval. In others, they do not.
Trials show Aduhelm reduced 1 sign of Alzheimer's
The marker in question is amyloid plaque, clumps of protein typically seen in the brains of people with Alzheimer’s disease. While amyloid plaque is considered a defining feature of Alzheimer’s, it is also found in brains of people who do not have Alzheimer’s.
Trials showed that Aduhelm reduced amyloid plaque but skeptics say results were murkier on whether the drug lessened any symptoms.
Approval process called faulty
The accelerated approval process itself is faulty, said Dr. Jason Karlawish, co-director of the Penn Memory Center. in each cases engaging in faulty reasoning, he said.
“The FDA failed in the process with this drug,” he said. “The process that was followed to transform aducanumab (the drug’s generic name) into Aduhelm is a process that should not be repeated.”
$56,000 for a year supply
Initially Biogen, the company that makes Aduhelm, priced the drug at $56,000 for a year supply, prompting an outcry from the Alzheimer’s Association that the hefty fee would preclude the drug for many who need it.
Last December Biogen cut the drug price in half, saying that doctors were not offering the drug to those who might benefit because of financial concerns.
While the drug company said it wanted to increase access, the optics of such a dramatic price contributed to skeptics’ dim view of the drug.
Dr. Vikas Saini, chair of the Right Care Alliance, an advocacy group that focuses on appropriate patient care over the health care industry’s profit, compared the move to “a used car salesman.”
Then in April CMS weighed in on Aduhelm, saying it would only cover Aduhelm for those enrolled in clinical trials. That decision basically made the drug off limits to most people who would have been candidates for the drug.
For many, clinical trial the only option
People like Gil Raske of Miller Beach have no other option than a trial.
Diagnosed with Alzheimer’s disease about eight months ago, after experiencing a few episodes of forgetting who he was, Raske, 67, heard about the new drug from his doctor and was immediately interested.
“Aduhelm is my big push… I have heard many many positive things and they fire away the negative things,” said Raske, adding that he hoped Aduhelm would give him “a shot to extend my life further.”
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But Raske cannot afford to pay for the drug out of pocket. So earlier this spring, he applied to a few study sites in the Chicago area.
Some doctors agree that CMS misstepped in its decision not to cover Aduhelm.
Calling the move “terrible for patients,” Dr. Liana Apostolova, a distinguished professor at IU School of Medicine, who conducts research on Alzheimer’s disease, said she has discussions with patients interested in Aduhelm, covering the known risks and potential benefits.
“Restricting the use to clinical trials alone is a disservice to the patients,” she said. “Our patients have the right to be informed of any new therapy that might be available to them…. My patients need to know that we are limited by contradictory scientific results.”
Aduhelm’s future in jeopardy
These contradictory results and Aduhelm’s torturous path thus far, however, have put the drug’s future in jeopardy.
Two months ago Biogen halted plans to submit its application approval for Biogen to the European Union’s European Medicines Agency.
Then last month Biogen announced it would “substantially” eliminate its spending on Aduhelm. The company would only allocate minimal resources to its once robust program to fund its ongoing trials.
Contact IndyStar reporter Shari Rudavsky at shari.rudavsky@indystar.com. Follow her on Facebook and on Twitter: @srudavsky.