Marilyn Long, left, helps her brother, Jeff Malanoski, who has Down syndrome and Alzheimer’s disease, at their home in Elk Grove Village, Illinois in 2014. (Chris Walker/Chicago Tribune/TNS)
After complaints of discrimination, federal authorities are scrapping a plan that would have prevented people with Down syndrome from accessing a new treatment for Alzheimer’s disease, even if they are predisposed to the disease.
The lifetime risk of developing Alzheimer’s disease is more than 90% for people with Down syndrome, advocates say. So when the Centers for Medicare and Medicaid Services proposed a coverage plan for Aduhelm, a controversial new drug for Alzheimer’s disease, proponents were dismayed that the proposal excluded people with Down syndrome.
Aduhelm was approved by the Food and Drug Administration last year despite questions from many experts about its actual effectiveness. However, CMS makes its own coverage decisions, and since most people with Down syndrome are covered by Medicare or Medicaid, the agency’s decision matters.
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Under a proposal released in January, CMS sought to make Aduhelm available to people on Medicare only if they participated in clinical trials, citing concerns about the treatment’s potential harm to patients. People with Down syndrome and any other conditions that “could significantly contribute to cognitive decline” would be excluded from these trials, according to the CMS proposal.
“This would mean that anyone with Down’s syndrome and Alzheimer’s who, in consultation with their medical and support teams, wanted to try this very expensive drug as a treatment option, would not have had their costs covered by Medicare because it was not allowed to be part of clinical trials,” said Heather Sachs, director of policy and advocacy at the National Down Syndrome Congress.
The Down syndrome community flooded CMS with more than 1,800 comments lambasting the proposal to explicitly exclude people with intellectual and developmental disabilities. Not only would the policy prevent people with Down syndrome from accessing Aduhelm, but without clinical trial data on this population, advocates feared doctors could miss important information to guide treatment decisions for people with Down syndrome. of chromosomal disease.
Today, the agency is changing course. In its final decision released earlier this month, CMS removed the exclusion for people with Down syndrome.
“The proposal (national coverage determination) included criteria that would have excluded certain key patient subpopulations (e.g. patients with Down syndrome). Based on public feedback, we are not finalizing patient exclusion criteria to allow appropriate access to patient subpopulations who may need treatment based on ongoing research,” it reads. in a CMS fact sheet that was published alongside the determination.
This decision does not guarantee access to Aduhelm, but allows people with Down syndrome to participate in clinical trials. Proponents note, however, that drug companies have been reluctant to include people with Down syndrome in trials.
“We are one step closer to our goal of ensuring adults with Down syndrome have equitable access to Alzheimer’s disease treatment options,” reads a joint statement from Down Syndrome Affiliates in Action, GiGi’s Playhouse Down Syndrome Achievement Centers. , the Global Down Syndrome Foundation , the LuMind IDSC Foundation, the National Down Syndrome Congress and the National Down Syndrome Society. “The next phase of our collective advocacy must focus on supporting the inclusion of people with Down syndrome in clinical trials for treatments for Alzheimer’s disease.”