Vertex/CRISPR, bluebird sickle cell therapies could be cost-effective - ICER
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Two new gene therapies for sickle cell disease from Vertex Pharmaceuticals (NASDAQ:VRTX)/CRISPR Therapeutics (NASDAQ:CRSP) and bluebird bio (NASDAQ:BLUE) could be cost-effective if their price tags are no more than $2.05M.
A new assessment released by the Institute for Clinical and Economic Review (ICER) found that Vertex (VRTX)/CRISPR's (CRSP) exagamglogene autotemcel (exa-cel) and bluebird's (BLUE) lovotibeglogene autotemcel (lovo-cel) would be cost-effective if priced between $1.35M and $2.05M.
Exa-cel has an FDA action date of Dec. 8, while lovo-cel's in Dec. 20.
Regarding evidence of efficacy, ICER gave exa-cel a C++ rating, saying it would result in incremental net benefit, or result in substantial net benefit when evaluated against standard of care. For lovo-cell, the grade was B+, noting the treatment would provide at least an incremental benefit.
An independent appraisal committee overwhelmingly voted (13-1 for each therapy) that current evidence is adequate that both therapies provide a net benefit compared to the standard of care.
ICER is also recommending that manufacturers and payers work together on alternative payment models for gene therapies. This is to address both the significant short-term budget impact of the treatments and the uncertainty surrounding their longer-term safety and benefits.

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Compare metrics for BLUE to CRSP, VRTX