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New Sickle Cell Treatments & Therapies: How much will it cost and who will pay?

Value Assessments, Quality-Adjusted Life Years (QALY), and the ICER Conundrum

Data, Resources, and Information compiled as part of a partnership with the Sickle Cell Community Consortium, the Partnership to Improve Patient Care, Sickle Cell Disease Association of America  , Sick Cells, the National Minority Quality Forum  and Sickle Cycle.


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:


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