$2M Price Tag for Sickle Cell Gene Therapies Could Be Cost-Effective Under Certain Scenarios, Finds ICER Analysis

Prices for incoming sickle cell disease gene therapies from Vertex Pharmaceuticals Inc VRTX/CRISPR Therapeutics AG CRSP, dubbed exagamglogene autotemcel (exa-cel), and Bluebird bio Inc’s BLUE lovotibeglogene autotemcel (lovo-cel) at $2 million could be cost-effective under some scenarios.
Although “these gene therapies have an incremental cost-effectiveness that is above commonly cited thresholds from the health care system perspective.”
The analysis comes from a U.S. drug pricing group, Institute for Clinical and Economic Review (ICER).
Results showed that at the placeholder price of $2 million per treatment course for lovo-cel or exacel (to be paid upfront), 15.4% of people (N=385 people per year) could be treated over five years without crossing the ICER budget impact threshold of $777 million per year.
Also Read: Newly Approved Drugs In US Command Over $200,000 On An Average.
Similarly, 33.9%, 24.7%, and 19.4% could be treated with either lovo-cel or exa-cel without reaching the potential budget impact threshold at the three threshold prices (approximately $0.90 million, $1.24 million, and $1.58 million per treatment).
“The draft cost-effectiveness findings were driven by the lifetime opportunity to improve health and reduce VOC-related and other SCD-related costs, and to improve productivity and reduce caregiver costs in the modified societal perspective,” ICER said.
Bluebird Bio signaled a delay in its sickle cell disease drug application to the FDA in its Q4 earnings release.
Vertex and CRISPR completed the rolling Biologics License Applications to the FDA for (exa-cel) for sickle cell disease and transfusion-dependent beta-thalassemia.

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