CAD | Enjaymo Not Cost-Effective at Current Price, According to US Study

Enjaymo (sutimlimab), the only approved therapy for cold agglutinin disease (CAD), requires an 80% price reduction or a maximum usage duration of 1.5 years to reach standard cost-effectiveness benchmarks in the United States.
This conclusion was drawn from a cost-effectiveness study utilizing data from the Phase 3 CARDINAL clinical trial, which backed the treatment’s approval for CAD patients with a recent history of blood transfusions.
“Despite demonstrating clinical benefit in phase 3 trials, Enjaymo remains a costly biologic agent, especially if administered over an indefinite period in CAD, calling for careful consideration of its value in the care of patients living with primary CAD,” researchers stated.
The research, titled “Cost-effectiveness of sutimlimab in cold agglutinin disease,” was published in the American Journal of Hematology and carried out by a team spearheaded by scientists at Yale School of Medicine in New Haven, Connecticut.
Sanofi’s Enjaymo is the first and only approved treatment in the U.S. and the EU for CAD, specifically targeting haemolysis in adult patients, irrespective of their blood transfusion history.
This antibody-based therapy functions by inhibiting the activation of the classical complement pathway, a crucial component of the immune system involved in the autoimmune assault characteristic of CAD. The treatment is administered via infusion into the bloodstream once a week for the first two weeks, followed by biweekly infusions thereafter.
Clinical data indicates that Enjaymo reduces hemolysis, anemia, and dependency on transfusions, while enhancing quality of life for up to 2.5 years of treatment. However, it comes with a substantial cost. At a list price of $1,800 per vial, six to seven vials are necessary for each infusion of Enjaymo, depending on the patient’s weight.
Researchers recently set out to determine the cost-effectiveness of Enjaymo compared to standard care for transfusion-dependent patients with primary CAD. They utilized data from the CARDINAL study that evaluated the therapy in this specific group of CAD patients.
Health outcomes were measured in quality-adjusted life-years (QALYs), which account for both health-related quality of life and lifespan. Essentially, one QALY represents one year in perfect health.
Based on the benefits observed in the trial, researchers calculated that transfusion-dependent primary CAD patients would incur costs exceeding $2 million per QALY with Enjaymo, compared to standard care over a lifetime. This cost reduced to about $1.3 million over a period of 2.5 years.
While it is impossible to quantify exactly how much a year of perfect health is worth, economic studies in the U.S. commonly use $150,000 per QALY as a benchmark for what patients are willing to pay. Hence, the data indicate that at its current price, the cost of Enjaymo is significantly disproportionate to the anticipated benefit.
Researchers noted the low cost-effectiveness of the therapy was driven both by “high drug cost … and the small increase in QALYs associated with [Enjaymo] use in the trial.”
While the data indicate that Enjaymo isn’t cost-effective at its current price relative to standard care, “that does not mean that it has no place in the treatment of patients with CAD,” the team wrote.
Source: Cold Agglutinin Disease News