The ALS Association has formally objected to the use of controversial measures to evaluate ALS drugs that can make it harder to find effective new ALS treatments and get them to the ALS community as quickly as possible. These measures have been identified by the National Council on Disability as being inherently discriminatory against people with disabilities.
Two new therapies, AMX0035 and an oral form of edaravone, are scheduled for FDA review this year. This is great news, but we are concerned that a review by The Institute for Clinical and Economic Review (ICER) may interfere with access to these drugs for anyone who might benefit from them.
The Association filed its objections as part of ICER’s recently closed comment period for a draft scoping document, which outlines ICER’s plans to conduct their assessment of these two ALS therapies. The Association is specifically objecting to the use of quality adjusted life years, often called QALYs, and equal value life years gained (evLYG) as the measures used to determine cost effectiveness, pointing out that neither measure adequately captures the value of life from the perspective of a person living with ALS.
“ICER’s model is built on assumptions that don’t make sense for the ALS community, that fail to capture the engagement and value of life for people living with ALS,” said Dr. Neil Thakur, chief mission officer at The ALS Association.
QALYs attempt to assign a value to a year of life lived in certain states and, as previously stated, have been identified by the National Council on Disability as being inherently discriminatory against people with disabilities, as their lives are assigned a lower quality score and are therefore implicitly deemed less worthy of being extended. Equal value life years, which represent an attempt to address objections to QALYs, do not address the quality of life that is highly valued by ALS patients by virtue of being quality-neutral. They fail to capture the actual benefit to the ALS community of a drug that could extend life by six months.
“Their approach to placing a value on additional months of life gained fails to fully capture the value of additional time with your family, of being able to see a child get married or a grandchild graduate from college – the things that truly give life meaning,” Thakur said.
The Association is also objecting to ICER’s failure to include the cost of medical and supportive caregiving in framework for their primary analysis.
The Association’s engagement with ICER is part of our longstanding fight to make sure promising treatments, like AMX0035 and Oral Edaravone, are available and accessible for people with ALS as quickly as possible.
We will keep fighting to make sure:
- All people with ALS are provided immediate, full coverage and affordable access to new therapies;
- Payors use methodologies that value the lives of all people with ALS;
- Health care utilization techniques and/or other administrative barriers that delay or decrease access to drugs for people with ALS and other neurodegenerative diseases are prohibited; and
- The use of arbitrary, discriminatory value assessments that limit access to ALS drugs, such as the use of metrics like Quality Adjusted Life Year (QALY) or the Equal Value Life Years Gained (evLYG) are prohibited.
You can read our full response to ICER’s draft scoping document here.
Comments
I've recently been diagnosed with ALS and desperate to find some type of help
So sorry to hear that you are living with this disease, Terry. Local ALS Association chapters provide free information, resources and support. You can find your chapter's contact information at als.org/chapters.
Thank you for bringing up this issue and for objecting to the metrics used by ICER. For someone living with ALS, getting six months more can greatly enhance their quality of life, as you wrote, and six months is a greater gain than any of the other treatments currently available offer.
i have been diagnosed with ALS on 12/21/22 and am devastated that there is no cure and AMX0035 is not approved by FDA. I had never heard of ALS
Surinder. I am so sorry to hear your diagnosis. Please know you are not alone. ALS association has great support groups to share information and help each other know how to navigate the maze. The ALSA support group zooms are very helpful. And ALS news forums are very helpful. Only those of us who have had this devastating diagnosis truly understand. My husband of 50 years and the love of my life received his diagnosis 09/24/21. We are all here to help each other. You are not alone.
Your article really resonated with me tonight. I spent most of today dealing with incompetence and lack of awareness. Actually, it is more accurate to say lack of basic human kindness. I finally said to the young man who was trying to justify that my husband’s power wheelchair had still not been ordered five months after his ALS clinic put in the order, “ if this was your wife struggling to breath….if you were watching her dying before your eyes…..losing more and more independence each day…maybe then you would care enough to realize ALS families don’t count our lives in months or years but hours and minutes”….Thank you for fighting the war against the discrimination. While you fight the war, we the families, will continue to fight our own day to day battles to give our loved ones with ALS as much comfort and independence as possible.
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