Introduction: Therapeutic advances over the past 30 years have led to longer life expectancy and improved quality of life (QOL) for persons with hemophilia. Access to innovative therapy may be compromised if treatment decisions are driven solely by cost. New strategies are needed to assess true therapeutic values, along with financial cost, as physicians, policymakers, payers and manufacturers work together to improve patient care.
Aim: To provide an evidence-based assessment of the value of prophylaxis vs on-demand therapy for hemophilia, based on a widely recognized three-tiered value framework approach for assessing a range of therapeutic interventions.
Methods: Data from six randomized clinical trials (ESPRIT, Joint Outcomes Study, SPINART, LEOPOLD II, ADVATE and POTTER) and four observational studies comparing primary and secondary prophylaxis vs on-demand therapy were applied to a hemophilia value framework.
Results: Both primary and secondary prophylaxis showed advantages in Tier 1 "Degree of health/recovery" outcomes, including measures of bleeding, musculoskeletal complications, pain, function/activity and QOL. Tier 2 "Process of Recovery" outcomes, also favoured prophylaxis, including measures of recovery time, return to normal activities, orthopaedic intervention and venous access. In Tier 3 "Sustainability of Health Recovery," measures of breakthrough bleeds, joint preservation, sustained productivity and QOL showed significant improvement with prophylaxis.
Conclusion: The hemophilia value framework affirmed value of primary and secondary prophylaxis vs on-demand therapy, with clinical benefit demonstrated in all three tiers. This analysis also demonstrates clinical utility of the value framework process in the determination of optimal and cost-effective hemophilia care for all stakeholders.
Keywords: factor VIII; hemophilia; prophylaxis; value framework.
© 2018 The Authors. Haemophilia Published by John Wiley & Sons Ltd.