Rare Daily Staff

The Institute for Clinical and Economic Review, an independent, nonprofit that analyzes the cost-effectiveness of therapeutics, issued a report that found Roche’s Hemlibra delivered health benefits for patients with hemophilia A with inhibitors and that drug lowers overall costs of treatment compared to current therapies.

ICER also noted Hemlibra carried other benefits not captured in clinical studies including simpler therapy administration, reduced caregiver burden, and patients’ increased ability to participate in work, school, or other activities.

Separately, the U.S. Food and Drug Administration granted Hemlibra Breakthrough Therapy designation for people with hemophilia A without factor VIII inhibitors. If Roche is successful at winning approval for this expanded use of the product it could significantly grow its market as only about 25 percent of patients develop inhibitors. Breakthrough Therapy Designation is designed to accelerate the development and review of medicines intended to treat a serious condition with preliminary evidence that indicates they may demonstrate a substantial improvement over existing therapies.

Hemlibra, generically known as emicizumab, is a bispecific factor IXa- and factor X-directed antibody. It is designed to bring together factor IXa and factor X, proteins required to activate the natural coagulation cascade and restore the blood clotting process for people with hemophilia A. Hemlibra is a prophylactic treatment that can be administered by a once-weekly subcutaneous injection.

Despite the overall cost savings and benefits, ICER noted that the high costs of therapies for the indication skews the analysis and that the high cost of these treatments need to be addressed.

“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,” said David Rind, chief medical officer of ICER.  “Payers, manufacturers, and policy makers must seek new approaches to address financial toxicity across the hemophilia landscape.”

In situations where emerging therapies appear cost-saving at a high price only because the existing standard of care is so expensive, reasonable value-based pricing for new treatments requires consideration of a new paradigm for “shared savings” between innovators and society—preserving the profit incentive for developing more cost-efficient therapies while slowly decreasing the overall cost of care, ICER said.

It also warned that given that Hemlibra may gain indications for broader use, indication-specific pricing will likely be essential to tailor the price to reflect the clinical and economic value of the drug in different patient populations.

April 17, 2018
Photo: David Rind, chief medical officer of ICER