Please use this identifier to cite or link to this item: http://bura.brunel.ac.uk/handle/2438/15093
Title: Achieving the "triple aim" for inborn errors of metabolism: a review of challenges to outcomes research and presentation of a new practice-based evidence framework
Authors: Potter, BK
Chakraborty, P
Kronick, JB
Wilson, K
Coyle, D
Feigenbaum, A
Geraghty, MT
Karaceper, MD
Little, J
Mhanni, A
Mitchell, JJ
Siriwardena, K
Wilson, BJ
Syrowatka, A
Keywords: Science & Technology;Life Sciences & Biomedicine;Genetics & Heredity;GENETICS & HEREDITY;inborn errors of metabolism;practice-based evidence;rare diseases;research network;STEM-CELL TRANSPLANTATION;UREA CYCLE DISORDERS;PHENYLALANINE-HYDROXYLASE DEFICIENCY;COA DEHYDROGENASE-DEFICIENCY;ACID OXIDATION DISORDERS;EVIDENCE-BASED MEDICINE;SYRUP-URINE-DISEASE;SAPROPTERIN DIHYDROCHLORIDE;CLINICAL-TRIALS;RARE DISEASES
Issue Date: 2012
Publisher: NATURE PUBLISHING GROUP
Citation: GENETICS IN MEDICINE, 2013, 15 (6), pp. 415 - 422 (8)
Abstract: Across all areas of health care, decision makers are in pursuit of what Berwick and colleagues have called the “triple aim”: improving patient experiences with care, improving health outcomes, and managing health system impacts. This is challenging in a rare disease context, as exemplified by inborn errors of metabolism. There is a need for evaluative outcomes research to support effective and appropriate care for inborn errors of metabolism. We suggest that such research should consider interventions at both the level of the health system (e.g., early detection through newborn screening, programs to provide access to treatments) and the level of individual patient care (e.g., orphan drugs, medical foods). We have developed a practice- based evidence framework to guide outcomes research for inborn errors of metabolism. Focusing on outcomes across the triple aim, this framework integrates three priority themes: tailoring care in the context of clinical heterogeneity; a shift from “urgent care” to “opportunity for improvement”; and the need to evaluate the comparative effectiveness of emerging and established therapies. Guided by the framework, a new Canadian research network has been established to generate knowledge that will inform the design and delivery of health services for patients with inborn errors of metabolism and other rare diseases.
URI: http://bura.brunel.ac.uk/handle/2438/15093
DOI: http://dx.doi.org/10.1038/gim.2012.153
ISSN: http://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000319979800001&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=f12c8c83318cf2733e615e54d9ed7ad5
http://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000319979800001&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=f12c8c83318cf2733e615e54d9ed7ad5
1098-3600
Appears in Collections:Dept of Clinical Sciences Research Papers



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