Alzheimer’s Association says accelerated drug approvals necessary in search for treatment
Medicare's decision to limit coverage for a controversial new Alzheimer's drug has prompted an outcry among Alzheimer's advocates.
The drug is Aduhelm, and despite widespread enthusiasm for it among Alzheimer's patients and advocates, it hasn't been proven to slow the disease. The drug was given accelerated approval by the Food and Drug Administration because it reduces the plaque that develops in the brains of most Alzheimer’s patients.
However, nearly 4 in 10 patients in clinical trials developed side effects, such as brain swelling or brain bleeds. FDA's approval of the drug is being investigated by a number of federal agencies and two congressional committees. And Medicare has decided to only provide coverage for people participating in clinical trials.
That decision has come under fire by Alzheimer's advocates such as Katherine Lambert, who heads the Alzheimer’s Association in the Carolinas and Georgia. She said that Medicare should pay for Aduhelm if doctors want to prescribe it and that limiting the coverage to those in clinical trials is unfair.
“It has created some very drastic barriers to an FDA-approved treatment,” Lambert said.
Although Lambert acknowledges that Aduhelm is not a cure, she said it is a step toward finding one.
“I don’t know of any drug that the first drug out in that class of medications was the be-all and end-all solution,” she said.
And once a drug has FDA approval, the Alzheimer’s Association’s job is to make sure everyone gets access, Lambert said. With at least three similar plaque-reducing Alzheimer’s drugs now in the pipeline, the association wants to make sure they can get accelerated approval — like Aduhelm did.
'We definitely want the accelerated pathway to continue'
Typically, the law requires drug companies to show drugs have a clear clinical benefit before they can be sold. For example, they should slow the disease or reduce symptoms. Accelerated approval gets around that. It allows the FDA to OK promising drugs — like cancer medications shown to shrink tumors — before they’ve been through the lengthy clinical trials necessary to demonstrate they prolong lives. That’s been important for people with terminal diseases who don’t have other options.
“Other FDA-approved therapies for cancer, heart disease, HIV/AIDS, there was access to those,” Lambert said.
Aduhelm got accelerated approval because it eliminates the amyloid plaque in the brain. But unlike the evidence linking tumor size to cancer survivability, there’s a question about whether plaque elimination reduces symptoms or disease progression.
The Alzheimer’s Association paid for a live-streamed event advocating for accelerated approvals with the publisher of The Hill, a daily newspaper for Congress. The association’s chief science officer, Maria Carrillo, told The Hill’s audience, “We want to make sure Congress maintains and strengthens the accelerated approval pathway because we are one of those diseases with a critical unmet need.”
Ohio U.S. Rep. Brad Wenstrup was another speaker at the event. He’s a member of the Republicans’ Healthy Future Task Force, which is responsible for developing the party’s health care agenda. It supports expanding the FDA’s use of accelerated approvals and preventing “bureaucrats” from overruling doctors. That’s a reference to the Centers for Medicare and Medicaid Services, or CMS, limiting payment for Aduhelm.
“We definitely want the accelerated pathway to continue,” Wentrup said. “ I do get concerned when I see where the FDA approves a drug for Alzheimer's and then CMS says we’re not going to pay for it.”
Pharmaceutical trade associations and at least 22 drug manufacturers have been lobbying about accelerated approval this year. That’s according to research by Open Secrets, which tracks money in politics.
Drug manufacturers also gave the Alzheimer’s Association $2.4 million last year, according to the association’s filing with the IRS. More than half of that, $1.8 million, came from companies developing antiplaque drugs like Aduhelm. That includes nearly $500,000 from Aduhelm’s manufacturer, Biogen.
Drug companies are also among the top contributors to Wenstrup. They gave him $89,000 last year, according to Open Secrets.
'A purple siege'
But the association has another asset: patients and their family members. The association's Alzheimer's Ambassadors program has close to 535 volunteers — one for each member of Congress. They talk to their members all year round. And this week, hundreds of ambassadors and other volunteers will head to Washington wearing their purple association sashes.
“It’s a purple siege almost to Washington,” Lambert said.
Laurie Waters is part of that purple siege. She was diagnosed with early onset Alzheimer's five years ago, and she’s the ambassador to U.S. Rep. Ralph Norman, of South Carolina. She said she’ll tell him about the dashed hopes of many Alzheimer’s patients because of Medicare’s decision.
Waters has been Norman’s ambassador for years, explaining the Association’s priorities, such as more funding for research.
“I keep him up to date on everything that he needs to know, what’s going on, what needs to be passed, what the Alzheimer's Association needs,” She said. “He’s amazing.”
The admiration is mutual.
“She’s a true patriot,” Norman said.
Norman is one of 78 House Republicans who wrote to Medicare, expressing concern about its decision to limit coverage of Aduhelm. But Norman said he doesn’t fault Medicare.
“They’ve got every right to get all the data,” Norman said. “And when you have this many prestigious hospitals, the Cleveland Clinic and others that are just not going to prescribe it, you have to take note of that.“
He said also hopes Medicare will do something about Aduhelm’s high price.
“Hopefully they would negotiate or at least suggest lower pricing if the clinical trials come in in a positive light,” Norman said.
'We don't want to chill innovation'
But Medicare can't negotiate prices without Congress changing the law, and so far that’s nowhere on the list of priorities set out by those pushing for more accelerated approvals.
The high launch prices of accelerated-approval drugs have got a lot of people worried, like Congress’ nonpartisan advisers on Medicare. With Medicare spending on physician-administered drugs like Aduhelm now topping $40 billion a year, they plan to send legislators some options on how they can contain costs. One of those options would allow Medicare to cap how much it pays for accelerated approval drugs until there’s clear evidence they actually slow disease.
But those are fighting words for the drug industry. Jocelyn Ulrich of the pharmaceutical trade association PhRma said companies need the incentive of higher prices.
“We don’t want to chill innovation in this space,” Ulrich said.
So if the FDA approves a medicine, Medicare should pay whatever drug companies want to charge even if they haven’t demonstrated their drugs slow the disease, Ulrich said. And she said Medicare’s decision to limit payment for Aduhelm hurt innovation, too.
“This is really hurting the entire signal to other companies who are trying to pursue what looks like the most promising scientific theory we have right now for patients who have no other options,” she said.
But with 6.5 million Alzheimer’s patients and no limits on what it can charge, any drug that’s shown to slow Alzheimer's disease is set to earn billions. So although it announced that it is cutting its sales workforce for Aduhelm, Biogen is working on another plaque-reducing drug. And at least two other companies, Lilly and Roche, have plaque-reducing drugs in development. So expect the battle over accelerated approval and Medicare’s reimbursement to continue.