In 2017 L-Glutamine became commercially available as a new treatment for sickle cell disease, doubling the number of FDA-approved treatments from 1 to 2.  At the end of 2019 two new FDA-approved treatments for sickle cell disease were made commercially available, once again doubling the number of treatments available to 4.  We are deeply concerned about the upcoming value assessment of 3 of these 4 treatments for sickle cell disease by the Institute for Clinical and Economic Review (ICER).  

The methodology implemented to determine the “cost-effectiveness” of treatments by ICER relies on a metric called the quality-adjusted life-year (QALY).  Congress has historically opposed this approach, having banned their use to determine coverage in Medicare, barred by the Patient-Centered Outcomes Research Institute (PCORI), and a HHS-determined violation of the ADA.  This metric has long been called out by the disability community for its inherent discrimination against people with disabilities due to placing a lower value on disabled lives. Similarly, the metric exacerbates disparities in health care by relying on population surveys to: determine the quality of life weights, utilities for its calculations, and bargain on randomized clinical trials which, as you know, are not representative of minority populations.  

Presentation: An Introduction to ICER and its implication on new SCD Treatments

 

Review The ICER Sickle Cell Disease Draft Evidence Report :

   Crizanlizumab, Voxelotor, and L-Glutamine for Sickle Cell Disease: Effectiveness and Value

 

Webinar Series to inform and educate:

• Webinar #1: An Introduction to ICER and potential implication for new sickle cell disease treatment (video: https://www.youtube.com/watch?v=SNjSD1HSAR0&t=2s)
• Webinar #2: The ICER Draft Evidence Report on Sickle Cell Disease: An Overview, Next Steps and Expectations  (RSVP: dgleason@pipcpatients.org)

 

Take the “My Life with Sickle Cell Disease” survey.  The results of the survey will impact the ICER review in several ways:

• It will allow ICER to more accurately measure the impact SCD has on day-to-day activities. 
• It will give caregivers a voice, which is often missing from clinical trial data. 
• It will help ICER put a price tag on some of the expenses that result from SCD. Specifically, the survey will capture information about out-of-pocket costs and how much is spent out of cash or bank accounts to help manage SCD. 

 

Resources and References

• Don’t legalize discrimination against people with disabilities 
• ICER discriminates against people with rare diseases
• Five Ways the Institute for Clinical Economic Review Fails Patients & People with Disabilities
• Reducing Prescription Drug Costs in Colorado