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On October 30th, 2020, the Institute for Clinical and Economic Review (ICER) held a public meeting to discuss its evidence report on therapies for hemophilia A. ICER is an independent and non-partisan research organization that evaluates the clinical and economic value of prescription drugs, medical tests, and other healthcare and healthcare delivery innovations. For this report, ICER reviewed emicizumab (Hemlibra, produced by Genentech) and valoctocogene roxaparvovec (Roctavian, produced by Biomarin), a gene therapy currently in phase 3 clinical trials.

ICER鈥檚 review individually compared emicizumab and valoctocogene roxaparvovec to prophylaxis with clotting factor. ICER鈥檚 analysis found that over a lifetime compared to prophylaxis with factor replacement, emicizumab reduces cost and produces equal or substantial net health benefits for people with hemophilia A. In their assessment of valoctocogene roxaparvovec, ICER used a placeholder price of $2.5 million. At this price, when compared to prophylaxis, ICER found valoctocogene roxaparvovec would also be cost saving but given the on-going clinical trial, the evidence of health benefit was deemed promising, but inconclusive.

In the review, ICER created an economic model of costs and clinical effectiveness based on data reported from clinical trials, related studies, and evidence for patient-important outcomes. The National Hemophilia Foundation (NHF) and Hemophilia Federation of America (HFA) participated throughout the 9-month ICER review process providing evidence, expert testimony, and real-life patient testimony of the impact of hemophilia on daily living and the importance of ensuring access to potentially transformative therapies.

Throughout, NHF and HFA have stressed the importance of including real-world, patient-important outcomes when assessing the value of emicizumab or gene therapy for those living with hemophilia. Clinical trial data focuses primarily on bleeding frequency and typically undervalues outcomes important to patients.

鈥淕iven 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 related to anticipation of bleeding especially with activities, and of course, the cost of bleeding to the healthcare system must be considered to demonstrate benefit to patients, their families and society,鈥 said Leonard A. Valentino, MD, president and CEO of NHF.

These points resonated with the ICER panel. At the roundtable, the panel heard accounts from hemophilia patients and treaters about the burdens the disorder can entail: pain; long-term joint injury; burdens of administering therapies; and impact on family life, school, work, living choices, emotional health, recreation, and more.

ICER鈥檚 review is helpful for assessing the economic and clinical value of a therapy; it should not override any treatment decisions that are made between doctors and patients, said Sonji Wilkes, Senior Director of Policy and Advocacy of HFA. 鈥淭he choice of treatment for each person with hemophilia needs to be individualized, patient-centric, accessible and affordable. The full range of products 鈥 clotting factor, non-factor therapies, and eventually gene therapies 鈥 must be available for patients, working in consultation with their doctors, to develop treatment plans that best preserve their health and quality of life.鈥

鈥淭oday is the culmination of more than five years鈥 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,鈥 commented Mark W. Skinner, hemophilia community member, member of ICER鈥檚 Expert Review Panel and ICER board member. 鈥淲e appreciate ICER鈥檚 increasing efforts to engage and include patients directly in its review process and recognize that our work is not done. 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.鈥

Hemophilia experts stressed to ICER鈥檚 panel, as well as other stakeholders participating in the meeting, that all treatment options should be available to patients, without barriers due to cost, short-sighted utilization management strategies, narrow formularies or narrow provider networks. The role of the hemophilia treatment center (HTC) will also continue to be extremely important in helping patients make these complex treatment decisions, managing utilization and in providing the longitudinal follow-up required for these new treatments.

NHF, HFA and the entire bleeding disorders community share a common interest in seeking economic value for treatment, and are concerned when a medication鈥檚 price misaligns with the value it provides. We look forward to generating additional real-world data to demonstrate the value of these transformative therapies to not only better inform ICER鈥檚 economic modeling and analysis but to preserve access to these lifechanging treatments for all people with bleeding disorders.

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