RARE Daily

ICER Says PNH Drug Overpriced

February 2, 2024

Rare Daily Staff

The Institute for Clinical and Economic Review, the independent nonprofits that does cost-effective analysis, has released a revised Evidence Report that found two, first-in-class therapies for the rare blood disorder paroxysmal nocturnal hemoglobinuria provide important health benefits, but their long-term safety and efficacy are uncertain.

The report examines Novartis’ iptacopan and Alexion Pharmaceuticals’ danicopan.

ICER said its model for iptacopan, which is already FDA-approved and marketed as Fabhalta, using a shared-savings approach, suggests that the drug would need to be priced 70 percent lower than the current list price to meet commonly accepted thresholds.

For danicopan, which is not yet FDA-approved, ICER’s model suggests the price would need to be between $12,300 and $13,100 per year to achieve common thresholds for cost-effectiveness.

PNH is a rare, acquired blood disorder that primarily manifests in fatigue, and if severe, requires lifelong dependence on blood transfusions.

“Complement C5 inhibitors, the current standard of care, have transformed this condition. However, C5 inhibitors are extremely costly, and patients, patient advocates, and clinical experts uniformly expressed concern about the access and affordability of these lifelong PNH treatments,” said Foluso Agboola, vice president of research for ICER.

The Evidence Report will be reviewed at a virtual public meeting of the California Technology Assessment Forum (CTAF) on February 16, 2024. The CTAF is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.

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