OP16 A Patient-centered Value Framework For Healthcare In Hemophilia. (2017)

• Record Type: Journal Article
• Authors: O'Mahony, Brian; Dolan, Gerard; Nugent, Diane; Goodman, Clifford
• Is Part Of: International journal of technology assessment in health care. Volume 33:Supplement 1(2017)
• Page Start: 8
• Page End: 9
• Publication Date: 2017
• Subjects: Medical technology -- Periodicals; Technology assessment -- Periodicals; 610.28
• DOI: 10.1017/S0266462317001210
• Languages: English
• ISSNs: 0266-4623

Abstract

Abstract : INTRODUCTION: Hemophilia is a rare, inherited bleeding disorder affecting an estimated 400, 000 people worldwide (1). Characterized by spontaneous bleeding and long-term, irreversible joint damage, persons with hemophilia are often limited in normal day-to-day activities, including work/school, and require comprehensive care at specialized treatment centers. With replacement therapies extending survival by decades and vastly improving quality of life (QoL), routine prophylaxis is considered the standard-of-care in developed countries. However, due to the cost of replacement factor, access to treatment remains a challenge, and increased scrutiny over funding has been augmented by growing demands on healthcare budgets (2). Thus, the hemophilia community shares a unified goal of objectively defining patient-centered value in hemophilia care. METHODS: Using a three-tiered outcomes hierarchy model initially described by Porter (3), an international, multidisciplinary panel of health economics outcomes researchers and hemophilia experts developed a value framework for decision makers to assess value of various healthcare interventions in hemophilia. RESULTS: The three tiers for assessing value are: (i) Health status achieved/retained; (ii) Process of recovery; and (iii) Sustainability of health. Tier one measures survival, quality of life (QoL), and hemophilia-specific outcomes of bleeding frequency, musculoskeletal complications, and severe bleeds, as well as function/activity (that is, lifestyle impairment). Tier two measures time to initial treatment or recovery and time missed at education/work, as well as disutility of care (that is, inhibitor development, pathogen transmission/infections, orthopedic intervention, and venous access). Tier three measures avoidance of bleeds, maintenance of productive lives, and long-term health, while capturing long-term consequences of insufficient therapy or age-related complications. Applicability of the framework can be demonstrated in areas of healthcare delivery, treatment regimen, and innovation for new therapies. CONCLUSIONS: This value framework represents an initial collaboration with stakeholders to define and organize an array of patient-centric outcomes of importance in hemophilia into a practical tool that can influence treatment and funding decisions in hemophilia care.