Institute for Clinical and Economic Review’s Final Report on Emicizumab Shows New Treatment Improves Health While Lowering Costs for Certain People with Hemophilia A

-Report also highlights need for future pricing of hemophilia treatments that allows for "shared savings" between innovators and society-

BOSTON, April 16, 2018 - The Institute for Clinical and Economic Review today 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," noted David Rind, MD, MSc, ICER's Chief Medical Officer. "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."

Policy Implications

Following the voting session during the New England CEPAC meeting, a policy roundtable of experts including patient representatives, clinical experts, public and private payers, and drug manufacturer representatives convened to discuss the implications of the evidence for policy and practice. Key recommendations stemming from the roundtable discussion include:

  • 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.
  • In assessing the value of treatments for hemophilia, payers should be aware of important benefits and contextual considerations that are not typically captured in cost-effectiveness analyses.
  • As leaders in working with manufacturers and other stakeholders to develop core sets of patient-important outcomes for clinical trials, hemophilia patient organizations should continue to advance their work in this area and serve as mentors for other groups seeking to introduce more patient-centric outcomes in clinical research targeting other diseases.
  • Given that emicizumab 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.

Additional policy implications, as well as more detailed discussion of the implications, are available in ICER's Final Report.

About ICER

The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER's reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

ICER's reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER's reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER's website.

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