Diane Nugent1, Brian O'Mahony2, Gerry Dolan3. 1. Children's Hospital of Orange County, University of California at Irvine, Irvine, California and Center for Inherited Blood Disorders, Orange, California. 2. Irish Haemophilia Society, Trinity College, Dublin, Ireland. 3. Guy's and St Thomas' NHS Trust, London, UK.
Abstract
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.
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.
Authors: Erna C van Balen; Brian O'Mahony; Marjon H Cnossen; Gerard Dolan; Victor S Blanchette; Kathelijn Fischer; Deborah Gue; Jamie O'Hara; Alfonso Iorio; Shannon Jackson; Barbara A Konkle; Diane J Nugent; Donna Coffin; Mark W Skinner; Cees Smit; Alok Srivastava; Fred van Eenennaam; Johanna G van der Bom; Samantha C Gouw Journal: Res Pract Thromb Haemost Date: 2021-03-06