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PP111 Toward Healthy Coagulation In Hemophilia
- Gerry Dolan, Erik Berntorp, Diane Nugent, Mark Skinner
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 34 / Issue S1 / 2018
- Published online by Cambridge University Press:
- 03 January 2019, p. 108
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Introduction:
Healthcare advances in hemophilia have led to near-normal life expectancies in a disorder previously associated with early death. Unlike other disorders where the therapeutic goal is to restore deficiencies to normal levels, prophylaxis in hemophilia is used to achieve a plasma level of FVIII >1%, such that severe hemophilia may be reduced to a moderate/mild phenotype. With the development of new therapies, treatment goals are evolving from on-demand treatment or prevention of bleeds to one where the risk of bleeding is minimal/absent. To accelerate this development, a new treatment paradigm is needed, with consensus from key stakeholder communities, to facilitate a shared vision for the future of hemophilia healthcare.
Methods:A panel of hemophilia providers, patient advocates, and industry representatives convened to develop a new treatment model that establishes specific treatment milestones and target outcomes in a stepwise fashion, culminating in a progressive definition of cure.
Results:To represent the collective experience of hemophilia for patients and treaters around the world, the following treatment milestones were defined based on optimized outcomes: (i) Sustain Life – prevention of premature death; (ii) Minimal Joint Impairment – improved quality of life; participation in activities of daily living; (iii) Freedom From Spontaneous Bleeds – ability to engage in low-risk activities; (iv) Attainment of ‘Normal’ Mobility – participation in work, career, and family life without restriction; (v) Able to Sustain Minor Trauma – more unrestricted lifestyle; (vi) Ability to Sustain Major Surgery or Trauma Without Additional Intervention – no dependency on specialized healthcare; (vii) Normal Hemostasis – optimal health and well-being; and (viii) Cure – health equity.
Conclusions:With milestones for disease management leading toward normalized hemostasis, this treatment model provides a vision to improve hemophilia care for all patients. And by providing achievable outcomes, the community—patients, treaters, and their industry partners—has a clear path to achieve that goal.
OP16 A Patient-centered Value Framework For Healthcare In Hemophilia
- Brian O'Mahony, Gerard Dolan, Diane Nugent, Clifford Goodman
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 33 / Issue S1 / 2017
- Published online by Cambridge University Press:
- 12 January 2018, pp. 8-9
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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.