BRIEF—ICER Draft Evidence Report on therapies for sickle cell disease

The USA’s Institute for Clinical and Economic Review (ICER) has released a  Draft Evidence Report assessing the comparative clinical effectiveness and value of treatments for sickle cell disease, including crizanlizumab (Adakveo, from Novartis), voxelotor (Oxbryta, from Global Blood Therapeutics), and L-glutamine (Endari, from Emmaus Life Sciences).

Health technology assessor ICER’s draft report notes that sickle cell disease has been an historically underserved area for research, with no new treatments for decades; as such, patients and clinicians have struggled with a system plagued by racism and underfunding. While assessing the value of these treatments, policymakers will need to consider the substantial lifelong burden associated with sickle cell disease, as well as the potential for these treatments to reduce the disparity in life expectancy between black and white Americans.

This draft report will be open to  public comment until February 20, 2020. Based on stakeholder feedback, ICER may revise key assumptions and findings for its Evidence Report, which will be published on March 12, 2020.