The Institute for Clinical and Economic Review on Monday released a Final Evidence Report and Report-at-a-Glance on emicizumab (Hemlibra, Genentech) for the prevention of bleeds (prophylaxis) in a subset of individuals with hemophilia A who have inhibitors to the clotting protein Factor VIII.
ICER’s report was reviewed at a public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), where a majority of the Council voted that evidence suggests a net health benefit of emicizumab compared to either no prophylaxis or prophylaxis with bypassing agents, therapies commonly used in patients with inhibitors. The council also emphasized the drug’s benefits beyond those captured in clinical trials, including simpler therapy administration, reduced caregiver burden, and patients’ increased ability to participate in work, school, or other activities.
In addition to the benefits that emicizumab offers patients, ICER’s report also found that the drug lowers overall costs of treatment for patients with hemophilia A and inhibitors as compared to current therapies.
“Emicizumab is an exciting innovation. It addresses unmet clinical needs in a group of patients with a very high burden of illness, while producing overall cost savings in the health system,” ICER Chief Medical Officer David Rind said. “However, emicizumab can be cost-saving at a very high price only because it is being compared to the huge costs currently associated with treating hemophilia. High drug prices, paired with an insurance structure that often requires significant patient cost sharing, results in overwhelming financial burdens for families year after year – so extreme that even a cost-saving treatment like emicizumab likely won’t prevent these families from reaching their out-of-pocket maximum payment. Payers, manufacturers, and policy makers must seek new approaches to address financial toxicity across the hemophilia landscape.”
To read the full press release on ICER’s website, click here.