Demo Medicare Emerges As Coverage Option For Alzheimer’s Disease Drug | New
WASHINGTON – The decision to cover an expensive and controversial new drug for Alzheimer’s disease could be settled by letting Medicare conduct its own trial to study the implications of payment and treatment.
Aduhelm, from drugmakers Biogen and Eisai, got fast-track approval from the Food and Drug Administration earlier this month over objections from the agency’s advisory committee. While clinical trials have shown that the drug significantly reduces beta-amyloid plaques in the brain – which are associated with the disease – the drug has failed to show that it slows the progression of the disease itself.
The companies now have nine years to complete an additional clinical trial demonstrating the drug can reduce the onset of Alzheimer’s disease, although revocation of drug approval is rare.
Three members of the FDA advisory committee resigned following Aduhelm’s approval, pointing to the lack of data on effectiveness and the high risk of dangerous side effects such as bleeding and swelling in the brain. Biogen’s list price of $ 56,000 also sparked an immediate reaction from patient advocates, including the Alzheimer’s Association, who pushed the drug to cross the finish line.
Attention has now shifted to the Centers for Medicare & Medicaid Services, which must determine if and how to cover an unproven drug at a cost capable of bankrupting the Medicare program. The drug also has major implications for the Medicaid program, which has less power to exclude coverage.
A few influential experts are now backing an idea to test both the cost and treatment implications through a payment pilot project under the Center for Medicare and Medicaid Innovation. The office has extensive power to force providers to participate in experiments that violate federal law.
Peter Bach, director of Memorial Sloan Kettering’s Center for Health Policy & Outcomes, and Craig Garthwaite, director of healthcare at Northwestern University’s Kellogg School of Management, wrote an editorial last week urging CMS to undertake a demonstration that would pay full price for the drug in some parts of the country, while it pays nothing in others. This move would largely limit the use of the drug to the areas covered, creating a control arm that would give CMS more data on the actual effectiveness of the drug.
“Assessing whether the payments are worth it is exactly the sort of thing the Medicare Research Center was created for,” they wrote.
Bach chairs the Medicare Evidence Development & Coverage Advisory Committee, or MEDCAC, which is sometimes responsible for assisting CMS when the agency establishes national coverage standards for a particular product. Regional contractors make independent coverage decisions in the absence of a determination of national coverage.
CMS has not said whether it plans to release a national decision or whether it was requested by an outside group, but industry experts are confident that a national decision will be the end result. If so, the agency could bring in a handful of experts from the advisory committee to help with the deliberations.
The idea for the demonstration is also endorsed by Joseph Ross, vice chair of the advisory committee and professor at the Yale School of Medicine.
“My point is that if this product is available in the market, then anyone who gets it should take part in a clinical trial or some type of study so that we can better understand the results associated with it,” did he declare. Call CQ. “The challenge of this is just a study of patients receiving this therapy is that in order to be able to observe changes in the progression of Alzheimer’s disease, any slowing of the decline in cognitive status would be really difficult to disentangle without a control arm. “
Melissa Garrido, associate research professor at the Boston University School of Public Health and a member of the advisory committee, said she was not sure CMS would determine the drug is medically necessary. Some type of decision taking into account a requirement for additional evidence and data is possible, she said, although a demonstration would have to take into account differentiators such as income and geographic disparities. Patients will receive the drug by monthly infusions and will also need access to MRIs and PET scans to monitor progress and potential side effects.
“If a difference in the results is observed in the treatment and comparison group, it is possible that this is due to socio-economic factors and not necessarily to the drug itself,” she said.
Bach, Ross or Garrido are not guaranteed to be part of the advisory board if CMS uses the panel for assistance. The agency chooses a handful of MEDCAC experts to analyze a product based on members’ expertise in a particular area. Detailed test data for the drug is not yet public, and CMS could also reverse the committee’s recommendations, as the FDA canceled its own advisory group.
CMS administrator Chiquita Brooks-LaSure also did not share her thoughts on how the agency should approach Aduhelm’s coverage, despite acknowledging the severity of the disease. Alzheimer’s disease affects more than 6 million Americans and is the sixth leading cause of death in the United States.
“We are looking at our options and will be looking very closely at the science going forward,” she said on June 14.
A demonstration could bridge the gap between patients demanding access to potential treatment and the need for a cost-conscious approach to collect more data, but it could also delay coverage further. The national determination process could take around nine to 12 months, and developing and implementing rules for a mandatory payment demonstration could take up to two years.
Mai Pham, founder of the Institute for Exceptional Care and former director of innovation at CMMI, said CMS should consider a “tiered” experiment if it decides to undertake a demonstration. CMS could make coverage conditional on clinical improvements made during treatment, she said, or cap the duration of treatment altogether.
Aduhelm has only been tested in patients with early-stage Alzheimer’s disease, but the FDA has approved the drug for patients at all stages of the disease. The drug could also be limited to early stage patients or those in clinical trials.
Either way, CMS will need to factor the drug’s ripple effects into all of its programs, including outcome-based payment experiences like responsible care organizations. ACOs are groups of physicians and other providers whose payments are based on their ability to keep patients healthy and spend little.
“They are ranked against a target set for them by CMS,” Pham said. “This goal is based on historical spending. Therefore, this target does not know anything about the new drug.
The political considerations are high. Pham said there was “no way” the lobbying forces behind the drug would allow the agency to block half the country from accessing the drug if they think they can stop it.
“I don’t know where the politics of this will end up,” Pham said. “Obviously, they didn’t end up in the right place with FDA approval. But it’s not obvious to me, after winning this battle, if this wing of the lobby is now weakened from all the pushbacks or if they feel emboldened.