The Institute of Clinical and Economic Review, commonly known as ICER, has opened a review of AMX0035 to determine the cost-effectiveness of the drug. The review is expected to conclude on September 19, 2022.

ICER’s review will rely on formulas that try to combine length of life with quality of life into an objective number, but that methodology has been criticized by the rare disease and disabled communities as discriminatory.

The ALS Association is committed to making sure ICER’s review does not discriminate against people with ALS and that its analysis does not prevent people with ALS from accessing promising treatments.

ICER evaluates the cost-effectiveness of drugs

ICER is an independent organization that seeks to place a value on treatments and therapies by comparing how well they work and how much they cost.

Once its review is complete, ICER will make a recommendation to health payers such as insurance companies about how much they should pay for AMX0035, who is most likely to benefit from it and other factors for access. If ICER says a new medication does not have value, payers may decide to restrict access to it.

ICER’s methods to assign values to the quality of life can discriminate against people with disabilities

ICER uses a controversial methodology to review new treatments. The most common form is known as quality-adjusted life years, or QALY. This measurement attempts to assign a value to a year of life lived in certain states of health. These values range from 0 – representing death – to 1 – representing “optimal health.” This methodology purports to measure how much closer to 1 a treatment moves the quality of a patient’s life in order to then place a value on the drug itself.

If a drug does not help a patient achieve optimal health, it could be considered less valuable.

The rare disease and disabled communities see this measurement as discriminatory because the value of life assigned to seniors, the chronically ill or people with disabilities is viewed as lower than that of a young, healthy individual. In some instances, including advanced ALS, the QALY could rate the quality of life as worse than death.

ICER’s quality measures are determined by asking healthy people to place a value on living with a disease or disability

ICER derives its quality scores based on something called population-based surveys. By using surveys of the general public, the data is skewed toward the value healthy people say they would place on their life given hypothetical diseases or disabilities. This is controversial because surveys are not necessarily designed to capture data specifically from people with the condition about their quality of life.

By overvaluing the value a healthy person would place on their life given a hypothetical disease or disability, this methodology ignores the lived experience of people who actually have a disease or disability and who probably would not define their quality of life quite so poorly.

ICER’s decisions are used by insurance companies to set up barriers to access

ICER reviews are primarily used by private health insurance companies. However, the Department of Veterans Affairs and Medicare Advantage plans can utilize ICER reviews in pricing and coverage determinations. So far, ICER methodologies have been prohibited from being used in Medicare but that may change. Medicaid coverage determinations are made on a state-by-state basis, and ICER is having an impact in some states.

If ICER says that a medication does not have value, payers such as private health insurance companies, and government payers like Medicare, Medicaid, and the Department of Veterans Affairs, may restrict access to it. Even if access is granted, the costs may be unaffordable for most people, or the red tape involved to access it may be insurmountable.

The ALS Association is working to make sure ICER’s recommendations do not prevent people with ALS and their doctors from determining the best treatment options

The ALS Association is engaging with ICER to provide the insights and input from the ALS community and will provide feedback during every phase of the review process. The Association is also engaging with people living with ALS to ensure that access to new ALS drugs is not hindered. We will provide updates on all our ICER efforts on als.org/ICER.