ALS Association opposes use of ‘failed’ profitability measures – ALS News Today

The ALS Association has strongly objected to the use of two controversial measures of cost-effectiveness in upcoming reviews of AMX0035 and oral edaravone, which are under review as treatments amyotrophic lateral sclerosis (ALS) in the United States.

These cost-effectiveness measures fail to value the lives of ALS patients, argue the objections.

The written objections were filed with the Institute for Clinical and Economic Review, known as ICER, during a comment period for a draft guidance document. The paper outlines how the institute plans to conduct cost-effectiveness analyzes of these potential therapies for ALS, which are currently undergoing regulatory review in the United States.

ICER is a nonprofit organization whose cost-effectiveness assessments are increasingly influencing drug pricing and reimbursement negotiations in the United States.

“The ICER model is built on assumptions that don’t make sense to the ALS community, that fail to capture the commitment and value of life for people living with ALS,” said Neil Thakur, PhD, ALS Association Mission Director. blog post.

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The targeted measures – quality-adjusted life years, called QALYs and years of equal value life gained, or evLYG – were previously found to be inherently discriminatory against people with disabilities by the National Council on Disability. A 2019 report from the council focused on how these measures contribute to “the historic and continuing devaluation of the lives of people with disabilities”, including those with ALS.

QALYs is a measure that assigns a score of quality, or utility of health, to a year of life lived in certain health states, with zero representing a year in the worst state of health and a score of 1 representing a year in perfect health. health.

These health services are based on surveys that mainly concern non-disabled people. Thus, according to critics, the lives of people with disabilities are given a lower quality score and are therefore deemed less worthy of prolonging.

Moreover, even small quality of life improvements are extremely valuable for people with disabilities, and this is simply not captured by QALYs, advocates say.

The second measure, evLYG, was created to address objections to the use of QALYs. It is an unweighted measure of years of life extended with a particular treatment and is used by ICER in addition to QALYs to assess whether there are significant differences between the two measures.

However, evLYG continues to rely on health services that advocates say fail to take into account the complexity of the preferences and experiences of people with disabilities, including those with ALS.

As such, the two measures fail to capture the value of life from the perspective of someone living with ALS – and the real benefit of a therapy that could extend life by six months – the association said in the blog post.

“Their approach of valuing the extra months of life gained fails to fully capture the value of the extra time spent with your family, of being able to see a child get married or a grandchild graduate from college – the things that give really a meaning to life,” Thakur said in his blog.

In its objection comment, the ALS Association asked ICER to detail how it will measure quality of life and assign patients to health states, as well as how this aligns with ALS patients’ perspectives on the value of their quality of life and health.

The association also asked the institute to include the cost of medical and supportive care in its main analysis.

“Successful treatment of ALS will prolong life, improve quality of life and reduce acute and supportive care costs,” the association said in its objection comment.

“All cost savings should be included in a cost-benefit analysis of treatment – ​​whether costs are arbitrarily classified as medical or non-medical,” the ALS Association wrote.

The advocacy group said in the blog post that its mission is clear: “The Association’s engagement with ICER is part of our long-standing fight to ensure that promising treatments, like AMX0035 and Oral Edaravone, are available and accessible to people with ALS as quickly as possible. .”

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Amylyx Pharmaceuticals’ AMX0035, administered twice daily orally or via feeding tube, is a fixed-dose combination of tauroursodeoxycholic acid and sodium phenylbutyrate. Each compound, already in clinical use, is known to be safe and well tolerated, and to protect nerve cells from cellular stress-induced damage.

Data from the Phase 2/3 CENTAUR clinical trial (NCT03127514) and its open-label extension study (NCT03488524) showed that AMX0035 was generally safe and significantly slowed disease progression, while reducing the risk 44% death rate in ALS patients.

The benefits of AMX0035 are now confirmed in the largest Phase 3 PHOENIX trial (NCT05021536), enrolling up to 600 adults with ALS; more contact information and the current 33 study sites can be found here.

The therapy is now undergoing priority review in the United States – with a decision expected no later than June 29 – and is being reviewed in Canada and Europe.

The oral formulation of edaravone (MT-1186), from Mitsubishi Tanabe Pharma America (MTPA), is designed to have a similar clinical profile to the company’s Radicava, an intravenous (into the vein) formulation of edaravone that has been approved in the United States for ALS since 2017.

Edaravone works by reducing oxidative stress, a form of cell damage thought to be one of the causes of nerve cell death in ALS.

Radicava has already been shown to safely and effectively slow functional decline in ALS clinical trials. Interim results from the open-label Phase 3 trial (NCT04165824) demonstrated that a six-month treatment with oral edaravone was generally well tolerated.

An international Phase 3b trial (NCT04569084) testing the safety and efficacy of two oral edaravone regimens against a placebo is currently enrolling up to 380 adults with ALS.

Oral edaravone is undergoing priority review in the United States, with a decision expected by May 12.

Sara H. Byrd