ICER finds reasonable benefit for sickle cell disease gene therapies

The USA’s health technology assessor the Institute for Clinical and Economic Review (ICER) today posted its revised Evidence Report assessing the comparative clinical effectiveness and value of exagamglogene autotemcel (exa-cel), from Vertex Pharmaceuticals (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP), and lovotibeglogene autotemcel (lovo-cel), from bluebird bio (Nasdaq: BLUE) for sickle cell disease.

Although uncertainties about durability and harm remain, both lovo-cel and exa-cel are likely to substantially improve quality and length of life among patients with SCD. Ultimately, cost effectiveness will depend on the actual prices for these therapies., the ICER stated.

“Sickle cell disease can affect nearly every organ system in the body, and severe sickle cell disease affects nearly every aspect of a person’s life,” said ICER’s chief medical officer, David Rind.