Interventions of Interest:

  • exagamglogene autotemcel (“exa-cel”, Vertex Pharmaceuticals and CRISPR Therapeutics)
  • lovotibeglogene autotemcel (“lovo-cel”, bluebird bio)

Despite uncertainty regarding longer-term outcomes, lovo-cel and exa-cel both estimated to produce substantial gains in length and quality of life; independent appraisal committee voted that currently available evidence is adequate to demonstrate a net health benefit over standard of care.

Current evidence suggests that lovo-cel and exa-cel would achieve common thresholds for cost-effectiveness if priced between $1.35M to $2.05M; recommendations encourage drug makers to set prices toward lower end of this range to facilitate access and affordability across all insurance systems.

Final Documents

Final Evidence Report and Meeting SummaryDownload
Final Policy RecommendationsDownload
Report-at-a-GlanceDownload
The ICER Snapshot: for PatientsDownload
Draft Evidence ReportDownload
Response to Comments on Draft Evidence ReportDownload

ICER’s Chief Medical Officer David Rind, MD 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. In the US, it is a disease that heavily affects the American descendants of those who were forcibly brought here as slaves. As such the US government and US payers have special obligations to ensure access to these new transformative gene therapies for sickle cell disease, and US manufacturers have special obligations to price such therapies low enough to facilitate broad access so as to maximize benefits for this population that has suffered historic harms and ongoing discrimination.”

For questions or to request a Spanish translated Report-at-a-Glance for this assessment, please contact info@icer.org.

View the Key Stakeholder List.