Minority groups could have a harder time accessing
The draft coverage determination aims to allow for additional studies on the effectiveness of Biogen’s Aduhelm, which won FDA approval in June 2021 despite pushback from scientific advisers. But limiting Medicare coverage could be detrimental to Black people, Hispanics, and other groups that are typically underrepresented in drug studies, in part because they may not have easy access to them.
“If we’re going to require or make provisions that certain kinds of technology or innovations are available to those that participate in clinical trials,” then “we need to expand the potential for diverse patient populations to participate in those clinical trials,” Esther Krofah, executive director of FasterCures and the Center for Public Health at the Milken Institute, said.
The Centers for Medicare & Medicaid Services should use this new drug as an opportunity to promote diversity in enrollment and ensure that clinical trial populations are reflective of the communities most impacted by the memory-destroying disease, she said.
Roughly 8% of participants in clinical trials for novel drugs approved by the Food and Drug Administration in 2020 identified as Black or African American, according to the agency’s Drug Trials Snapshots Report—lower than the approximately 13% of Black Americans within the U.S. population. Hispanic Americans accounted for 11% of 2020 clinical trial participants, despite making up about 18.5% of the U.S. population.
Meanwhile, approximately 18.6% of Black Americans and 14% of Hispanic citizens age 65 and older have Alzheimer’s disease, compared with 10% of white adults in this age group, according to advocacy group UsAgainstAlzheimer’s.
The CMS said Jan. 11 that while Aduhelm could help patients with Alzheimer’s, there is also the potential for side effects like headaches, dizziness, falls and brain bleeds.
The proposal “is the result of robust evidence analysis conducted through a thorough review process that found while there may be the potential for promise with this treatment, there is also the potential for harm to patients,” Lee Fleisher, CMS chief medical officer and director of the Center for Clinical Standards and Quality, said in a statement.
The public has 30 days to comment on the proposed National Coverage Determination before the CMS makes a final decision. If the proposal is finalized, the CMS said it will determine which clinical trials and patients meet the agency’s coverage eligibility requirements on a case-by-case basis.
Stars Must Align
The CMS’s coverage determination would apply to all antibody-based drugs that target amyloid plaques—the sheets of abnormal protein that accumulate in patients’ brains—in qualified clinical trials. Those trials must satisfy certain coverage criteria, including that they enroll “a representative patient population.”
The CMS cited the “disappointing lack of inclusion of underserved populations in past trials” in its decision memo.
But policy analysts say longstanding barriers, including a person’s physical access to academic sites carrying out trials, can limit research on potentially groundbreaking treatments from reaching those most impacted by Alzheimer’s.
“Basically, the stars have to align in all sorts of ways that are easier for the privileged and harder for the disadvantaged,” Holly Fernandez Lynch, a medical ethics professor at the University of Pennsylvania, said.
“There are long-standing equity problems in trial enrollment, unfortunately,” she said. “You typically have to live near the trial site or have the resources to travel. You have to be able to take time away from work and your caregivers need to do the same. You have to have reliable transportation.”
Some patients covered by Medicaid also may not have easy access to the “highly sophisticated academic medical sites” conducting clinical trials on the Alzheimer’s drug, Krofah said. She added that diverse patient populations are oftentimes “not asked even to participate” in clinical trials “even if they meet all the inclusion-exclusion criteria.”
There are “a variety of biases,” Krofah said, “whether they are potential non-adherence or not likely to remain engaged throughout the duration of the trial.”
The FDA granted accelerated approval to Aduhelm even though two clinical trials offered mixed signals about the drug’s efficacy. A committee of outside scientific advisers to the agency said the drug shouldn’t have been approved. Three panelists have since resigned.
Medical research and insurance companies, citing continued uncertainty over the treatment’s efficacy and cost, welcomed the CMS decision on Aduhelm.
Compiling and analyzing additional data on Aduhelm “will provide much-needed insights into the drug’s safety and effectiveness,” Adam Myers, senior vice president and chief clinical transformation officer (CCTO) for the Blue Cross Blue Shield Association, said in a statement. Private insurers in the U.S. have mostly declined to cover the treatment due to questions on its efficacy and whether its benefits outweigh risks.
“We will continue working with the administration and the health care industry to advance affordable, clinically-appropriate treatments for incurable conditions, including Alzheimer’s disease,” added Myers, whose association represents dozens of health insurance companies providing coverage to millions of Americans.
Limiting coverage to clinical trial participants will “help ensure that patients and family members are aware that taking Aduhelm is part of an experiment, despite Aduhelm’s FDA approval,” said Diana Zuckerman, founder and president of the National Center for Health Research.
Broad coverage and access to a medication is more likely when “clinicians are convinced of an accelerated approval drug’s promise, such that they are willing to prescribe it,” and “payers are convinced the drug works and is a good value, such that they are willing to pay for it,” Fernandez Lynch said. “Neither of those are true for Aduhelm.”
But Alzheimer’s Association CEO Harry Johns said if the CMS decision is finalized, access to Aduhelm would “now only be available to a privileged few—those with access to research institutions—exacerbating and creating further health inequities.”
The announcement is “shocking discrimination against everyone with Alzheimer’s disease, especially those who are already disproportionately impacted by this fatal disease, including women, Blacks and Hispanics,” Johns said in a statement.
Policy analysts say clinical researchers can promote greater diversity in trials for potential Alzheimer’s treatments by encouraging “lots of sites to participate,” using “telehealth visits as much as possible,” and making “appointments at convenient times for participants and caregivers,” Fernandez Lynch said.
The CMS could also work with the FDA and National Institutes of Health to develop a “national community-based clinical trial infrastructure” that prioritizes engagement with racial and ethnic minorities and other groups typically underrepresented in trials, Krofah said.
While equitable patient access should be a priority, the federal government should balance this with the safety and effectiveness of a treatment, both of which need further investigation in the case of Aduhelm, Fernandez Lynch said.
“What we’re talking about is equitable access to a chance of benefit, not necessarily to benefit itself,” she said. “But that’s still an important equity challenge.”