Cure SMA Advocates for SMA Community at ICER Hearing

On Thursday, March 7, Cure SMA testified on behalf of the SMA community at a meeting of the Institute for Clinical and Economic Review (ICER). ICER is an independent health policy group that evaluates the effectiveness of drug treatments, and the costs associated with those treatments.

The purpose of the meeting was to review both Spinraza and Zolgensma. Testimony was given by Mary Schroth, MD, chief medical officer for Cure SMA. Jaimie Vickery, vice president of policy and advocacy, also attended.

One of Cure SMA’s longstanding priorities is to advocate for individuals and families affected by SMA. We believe that decisions about care should be made by patients, their families, and their medical care team, based on what works best for that individual. We work to ensure full access to all therapies, treatments, devices, and family services. We believe that no one impacted by SMA should be denied access to potentially life-saving therapy, intervention or expert care provider.

How an ICER Review Works

Reviews like the one ICER is working on can be used to evaluate new drug treatments, especially groundbreaking treatments like Spinraza and Zolgensma. It is critical that these reviews are accurate and fair, and that there are no errors or gaps that could skew the results.

Most importantly, the voices of affected individuals and their families and caregivers must be heard throughout the process. Just as we advocated for the patient voice to be incorporated into the drug development and FDA review process, so the patient voice must be front and center in discussions regarding drug access.

In fact, one focus of our feedback to ICER was the importance of including the SMA patient voice. We were proud to stand with several members of our community, who also testified at this hearing and brought their voices to the process. We thank Dany Sun and Brandi Akins for traveling to Boston and for their important testimony.

Our Message to ICER

Individuals and families affected by SMA have consistently stated that small changes in response to treatment can make a big difference. Individuals and families have also consistently stated that stopping progression of SMA is a meaningful outcome for therapy. These patient concerns have been documented in peer-reviewed scientific literature, based on research conducted and funded by Cure SMA. In our interactions with ICER, we encouraged them to give these outcomes the fullest consideration.

It is also important that these reviews reflect the unique challenges and opportunities of drug development for rare conditions like SMA. ICER’s report uses benchmarks that are more suited to drug development for non-rare conditions, even though they themselves acknowledge that drug development for rare diseases is unique. In our testimony, we also encouraged ICER to make sure their benchmarks reflect the fact that SMA is a rare disease.

ICER’s report also fails to accurately reflect how Spinraza and Zolgensma, along with other therapies in development, are transforming the experience of SMA. What was once the leading genetic cause of death is now treatable, and individuals affected by SMA are gaining decades of life from these therapies. Any evaluation must take into account this significant change.

Finally, ICER’s report contains a number of scientific errors, both in the biology of SMA, and in the clinical trial data. We communicated to ICER that these errors, if not corrected, will raise serious questions about the credibility of their analysis. 

What Happens Next

ICER will take all the feedback received in this meeting into account, and will issue a final report at the end of this month. ICER fulfills an advisory role only. While we do not expect that their final report will have any direct impact on insurance coverage or access to SMA therapies, we will review it and determine if further action is needed.

With Spinraza approved by the FDA, and Zolgensma expected to be approved later this spring, access to therapy is one of the most important issues for our community. Our role has been to work directly with federal and state government and commercial payors to advocate for favorable coverage decisions for all those living with SMA to ensure full access to all therapies, treatments, and devices. We will continue to do so alongside our families. Additionally, we are working to expand access to treatments by establishing a series of SMA care centers across the United States and making multiple treatment options available.

Please reach out to [email protected] with any questions on ICER’s report or our efforts around treatment access.

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