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ICER Affirms Gene Therapies for Sickle Cell Disease Cost-Effective at $2 Million

July 17, 2023

A revised report from the Institute for Clinical Evaluation and Research (ICER) has reiterated that two experimental gene therapies for sickle cell disease (SCD) would likely be cost-effective if priced at $2 million.

ICER, which assigned both therapies a placeholder acquisition cost of $2 million, concluded that BlueBird Bio’s lovotibeglogene autotemcel (lovo-cel) provides at least an incremental net benefit when compared against standard of care and may, in fact, provide a substantial net health benefit, earning a comparison rating of “incremental or better” (B+). 

Similarly, ICER’s July 13th report found that Vertex Pharmaceuticals’ and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel) may be comparable, yield an incremental net benefit or result in substantial net benefit when compared against standard of care, earning a comparison rating of “comparable or better” (C++).

BlueBird Bio and Vertex/CRISPR have submitted biologics license applications to the FDA, which were accepted in June, and regulatory decisions are slated for December. Though the companies haven’t announced U.S. prices if their products are approved, ICER estimates the therapies’ health-benefit price benchmark — the highest a manufacturer should charge based on the improvement in overall health — to range anywhere between $1.35 to $2.05 million.

Read ICER’s latest report here. — Jason Scott

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