What is ICER?

The Institute for Clinical and Economic Review (ICER) is a private, nonprofit organization that performs cost-effectiveness analyses of drugs. ICER focuses primarily on new-to-market or soon-to-market treatments. The goal of ICER analyses is to see if a drug’s benefit to patients aligns with its cost and its value to the health care system overall.   

What are ICER value assessments used for?  

ICER reviews are used by insurance companies to determine whether a drug should be covered, what the pricing for the drug should be and what red tape should be put in place to keep costs down. The reviews are primarily used by commercial health insurance companies. However, the Department of Veterans Affairs and Medicare Advantage plans can utilize ICER reviews. So far, ICER methodologies are not being used for Medicare but there is concern that they might be in the near future. Decisions on drug coverage for Medicaid are made on a state-by-state basis, and ICER is having an impact in some states.  

What ALS drugs will ICER review?  

ICER has informed the ALS Association that it will be performing a review of Amylyx Pharmaceutical’s drug AMX0035, and Mitsubishi Tanabe Pharma America's oral edaravone. The review process was formally announced on February 3, 2022. It will conclude September 19, 2022.

How does ICER assess the value of a treatment?  

ICER performs a review of existing clinical trial data for new treatments and other information.  From this information, they aim to answer four key questions:  

1. How well does the drug work?  
2. How much better is the new drug compared to what already exists?  
3. How does the set price of the drug compare to the effectiveness?  
4. How much would it cost to treat everyone who needs it?  

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 National Council on Disabilities and patient advocates see QALY as discriminatory because the value of life assigned to seniors, the chronically ill, or people with disabilities are viewed as lower than that of a young, healthy individual.

Another measure is known as Equal value of Life Years Gained (evLYG) emphasizes the calendar time in life gained from of a drug compared to standard care in a specific disease. However, evLYGs are not absolute calendar time, where a year of life gained is counted as 12 months. ICER has an adjustment process to create the ‘Equal Value of Life Years’ which allow evLYGs to be comparable across diseases. An evLYG is 85% of a year of life gained, plus an additional value based on ICER’s underlying quality of life estimate for that disease. Therefore, 1 equal value of Life Year Gained from a diabetes drug is numerically the same as 1 evLYG from an ALS drug, even though the actual calendar time gained might not be 12 months for either drug.

The Association has significant concerns with this approach. First, the ICER quality of life assessment process taints the evLYG measure and shifts focus away from the actual impact of a treatment to this abstract measure. More fundamentally, people living with fast acting diseases like ALS often value an additional month of life more intensely than people living with a disease with a decades long lifespan like diabetes. For more information about evLYGs see https://icer.org/wp-content/uploads/2020/12/QALY_evLYG_FINAL.pdf

Learn more about the criticisms of ICER’s methodology here.

Does ICER include patient voice in their reviews?

ICER collects written feedback from all interested parties of a review including patients and health care providers. However, concerns remain that ICER’s primary focus to assist insurance companies in making pricing and coverage determination.

How will the ICER review affect my ability to access new treatments such as AMX0035?

At this time, it is unknown how ICER’s review will impact patient access to these therapies. ICER is still in the planning phases of their review of these treatments and will release a draft report in June. 

What is the ALS Association doing to engage with ICER and protect patient access of ALS therapies?

The Association is engaging with ICER to provide insights and input from the ALS community and will provide feedback during every phase of the review process. This is part of our longstanding fight to make sure promising treatments, like AMX0035, are available for people with ALS as quickly as possible. 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.