Hemophilia Treatments Should Be Open to All, Regardless of Cost, ICER Told

The full range of hemophilia treatments should be made available to patients without consideration of cost or other barriers, leaders of patient associations and others argued at a public meeting hosted by the Institute for Clinical and Economic Review (ICER) to consider its recent analysis of hemophilia A therapies.

The virtual meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC) — part of ICER — was a public review of that analysis, a report focused on the economic and clinical value of Roche‘s Hemlibra (emicizumab) and BioMarin‘s experimental gene therapy Roctavian

Patients, caregivers, clinicians, and leaders with the National Hemophilia Foundation (NHF) and the Hemophilia Federation of America (HFA) participated in the Oct. 30 roundtable discussions, the NHF reported in a press release, detailing the many “burdens” of this disorder.

“Given the transformative nature of the therapies under consideration, patient important outcomes including joint pain, limitations on activities, lost time from school or work, emotional well-being, psychological stress …  and of course, the cost of bleeding to the healthcare system must be considered to demonstrate benefit to patients, their families and society,” Leonard A. Valentino, MD, president and CEO of NHF, said the release.

Such points “resonated” with the ICER panel, the NHF stated. It and others worked with ICER for months, stressing the need to include patient experiences and perspectives, rather than clinical trial data alone, in determinations of a treatment’s value.

“Today is the culmination of more than five years’ worth of work by the national and international hemophilia communities to define patient-important outcomes and collect real-world evidence to assess the true reflection of value for a new therapy: the difference it makes in the lives of those living with the disease,” said Mark W. Skinner, a member of the hemophilia community, and ICER’s expert review panel and its board.

ICER’s report, made public Oct. 16, found that Hemlibra, co-developed by Chugai Pharmaceutical and Genentech — both part of the Roche group — had comparable or even superior clinical benefits than prophylactic, or preventive, factor VIII (FVIII) replacement therapy and was cost-saving as a treatment for people with hemophilia A.

Roctavian, even if priced of $2.5 million, could also be cost-effective compared with prophylactic treatment, but further evidence from clinical trials is needed.

“We appreciate ICER’s increasing efforts to engage and include patients directly in its review process and recognize that our work is not done,” Skinner said at the roundtable. “Enhancing the patient voice is vital if patient advocacy groups, providers, manufacturers, payers and policymakers are to find common ground and ensure high-value therapies remain within reach of patients.”

While ICER’s review assessed the economic impact of the therapies and their effectiveness, deciding which therapy to take should be a decision between a doctor and patient, said Sonji Wilkes, HFA’s senior director of policy and advocacy.

“The choice of treatment for each person with hemophilia needs to be individualized, patient-centric, accessible and affordable,” Wilkes said. “The full range of products — clotting factor, non-factor therapies, and eventually gene therapies — must be available for patients, working in consultation with their doctors, to develop treatment plans that best preserve their health and quality of life.”

Hemophilia experts also highlighted that costs, disease management strategies, and network provider services should not be obstacles to access to the full range of available therapies.

“NHF, HFA and the entire bleeding disorders community share a common interest in seeking economic value for treatment,” the NHF release concluded.

“We all look forward to generating additional real-world data to demonstrate the value of these transformative therapies to not only better inform ICER’s economic modeling and analysis but to preserve access to these life-changing treatments for all.”