ALS Association Urges CMS to Improve Patient Coverage and Services


The organization called attention to unintended harms of the current Medicare benefit structure and the need for the Center for Medicare & Medicaid Services to be more “patient-focused” in its assessments.

Neil Thakur, PhD, chief mission officer, ALS Association

Neil Thakur, PhD

In late September 2021, the ALS Association was invited by the Center for Medicare & Medicaid Services (CMS) to present its case for patients with amyotrophic lateral sclerosis (ALS) in front of the center’s head, Administrator Chiquita Brooks-LaSure. The organization offered its insight into the current structure of Medicare benefits for this patient population, calling for particular focus on issues with home health care and access to durable medical equipment, and pointing out unintended harms brought on by this established structure.1

“People living with ALS are often avoided or rejected by Medicare-certified home health agencies because they are high-cost patients,” Neil Thakur, PhD, chief mission officer, ALS Association, said on the call. “In the cases when people with ALS are able to access Medicare home health, they often are shortchanged in terms of the number of hours and kinds of services provided, or even told they are being discharged from care because they have shown no improvement.”

“In short, the current structure of the benefit disincentivizes Medicare home health agencies from serving people with ALS,” he added. Thakur noted that even in the instances when these individuals get access to equipment to help with mobility and speech issues, the limitations on Medicare-approved technology inadvertently result in losses of potential benefits and valuable time for patients with ALS.

One such example he pointed to was the speech generation software-equipped tablets that they often receive—tablets that are locked to prevent their use for anything aside from the software. This, Thakur explained, limits their ability to communicate and engage with the outside world in the way the majority of Americans do, via platforms such as social media and email. As Medicaid does not currently fund those functions for the equipment, the manufacturers are not incentivized to innovate and improve device function or adjust their prices because of policy-driven market distortion, he said.

Another example Thakur provided was the limitations in access to complex wheelchairs, often prescribed by clinicians due to movement issues, which can be accompanied by “fail first” processes that are particularly challenging for those with progressing neuromuscular disease. Or, as Thakur said, “Medicare says that the seat elevation that they need to go to the bathroom by themselves and the ramp they need to get outside are not ‘medically necessary.’”

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Currently, the therapeutic landscape for patients with ALS is relatively bare, with riluzole (Rilutek; Sanofi) and edaravone (Radicava; MT Pharma) the only options currently approved for use. Additionally, in August, the community was dealt a blow with the Alexion Pharmaceuticals announcement of the discontinuation of its phase 3 trial of ravulizumab (Ultomiris) in the CHAMPION-ALS trial (NCT04248465). The decision was reached based on a recommendation from the Independent Data Monitoring Committee’s prespecified interim analysis which suggested that the trial be discontinued because of a lack of efficacy.2

Although, there have been some promising advances in recent months as well, with the FDA granting orphan drug designation to ALS001, an investigational agent from Coya Therapeutics in July,3 and Cytokinetics announcing open enrollment for a new phase 3 trial, COURAGE-ALS (NCT04944784), for reldesemtiv in patients with ALS in August, following promising results from a phase 2 clinical trial.4 As well, in September, Clene Nanomedicine announced the launch of its second FDA expanded access program (CNMAu8.EAP02) for its lead drug candidate, CNM-Au8, for ALS, in conjunction with the Healey ALS Platform Trial.5

Despite this progress, though, the disease remains fatal, with no cure. “The average survival after diagnosis is about 2 to 5 years, and the majority of people with ALS are Medicare recipients,” Thakur said in the CMS call.1 “Unlike most Medicare recipients who need extensive homecare, and Rehabilitative Services, people with ALS do not improve, and the intensity of their service needs increase over time. That means standard Medicare cost control approaches don't work very well.”

This is not the first time that the ALS Association has pled with a federal institution this year, calling in April for the FDA to “move with the urgency of its Canadian and European Union counterparts” to approve one of the existing investigational agents with promise, AMX0035 (Amylyx Pharmaceuticals).6 Then, in September, when Amylyx announced its intention to bring the combination agent to the agency for review, the association urged the FDA to approve the treatment for all people with ALS as soon as possible.7,8

The organization was an early funder of the development for AMX0035, and in March this year invested in another investigational intervention, a cell therapy being developed by investigators at the Houston Methodist Neurological Institute and Massachusetts General Hospital. Along with the Muscular Dystrophy Association and ALS Finding a Cure, it provided an additional $1.1 million in supplementary funding after originally providing $2.4 million for this research.9

Thakur closed his call with CMS by urging the center to “become more patient-focused,” calling it “essential” for it to leverage patient feedback and experience data in payment decisions. This engagement with the community, he explained, could bring about critical input on patient-focused outcomes, more effective structure, and better cost control. “Ultimately, we need CMS to help us turn ALS from a fatal disease into a livable one. And I think we can do that,” Thakur concluded.

1. ALS Association to CMS Administrator: We Urge You to Listen to People Living with ALS. ALS Association. September 22, 2021. Accessed October 6, 2021.
2. Update on CHAMPION-ALS Phase III trial of Ultomiris in amyotrophic lateral sclerosis. News release. AstraZeneca; Alexion. August 20, 2021. Accessed October 6, 2021.
3. Coya Therapeutics receives orphan drug designation from the FDA for ALS001, an autologous treg cell therapy, for the treatment of ALS. News Release. Coya Therapeutics. July 7, 2021. Accessed October 6, 2021.
4. Cytokinetics announces start of COURAGE-ALS, a phase 3 clinical trial of reldesemtiv in patients with amyotrophic lateral sclerosis. News release. Cytokinetics. August 2, 2021. Accessed October 6, 2021.
5. Clene announces initiation of a second FDA expanded access program with CNM-Au8 for people living with amyotrophic lateral sclerosis. News release. Clene Nanomedicine. September 23, 2021. Accessed October 6, 2021.
6. ALS Association Calls on FDA To Follow the Lead Of Canadian and European Counterparts in Expediting Access To Promising New Treatment. News release. ALS Association. April 14, 2021. Accessed October 6, 2021.
7. Amylyx Pharmaceuticals Announces Plan to Submit New Drug Application (NDA) for AMX0035 for the Treatment of ALS in the Coming Months. News release. Amylyx Pharmaceuticals. September 15, 2021. Accessed October 6, 2021.
8. ALS Association Applauds Amylyx's AMX0035 Announcement, Urges Swift FDA Approval. News release. ALS Association. September 15, 2021. Accessed October 6, 2021.
9. The ALS Association, Muscular Dystrophy Association, and ALS Finding a Cure Provide Additional Funding for Novel ALS Research. News release. March 19, 2021. Accessed October 6, 2021.
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