BRIEF—ICER draft evidence report on Hemlibra for hemophilia A

The USA’s Institute for Clinical and Economic Review (ICER) has released a Draft Evidence Report assessing the comparative clinical effectiveness and value of Hemlibra (emicizumab), developed by Roche subsidiary Genentech.

Emicizumab was approved by the Food and Drug Administration in November of 2017 for prevention of bleeding in adults and children who have hemophilia A with coagulation factor VIII inhibitors.

The draft report suggests that emicizumab could significantly reduce healthcare expenses for certain patients.

Budgetary cost savings could be as much as $1.9 million per year for patients aged 12 years or older with inhibitors to factor treatment and $700,000 for those under 12 years old, compared with patients receiving prophylaxis with bypassing agents or no prophylaxis.

The wholesale acquisition cost (WAC) of emicizumab is around $482,000 for the first year of treatment and $448,000 for subsequent years (individual dosing and thus cost is based on weight, and therapy may be used both in young children and adults.

Factor VIII is the clotting protein that is deficient in those with hemophilia A, and inhibitors interfere with the factor replacement therapy that would typically be used to treat the condition.