Articlehybrid2014

Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework

Mike Paulden · Tania Stafinski · Devidas Menon · Christopher J. McCabe

99 citations0/0 files storedPharmacoEconomics

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Pharmaceutical Economics and Policy Health Systems, Economic Evaluations, Quality of Life Economic and Financial Impacts of Cancer

Abstract

BACKGROUND: The rate of development of new orphan drugs continues to grow. As a result, reimbursing orphan drugs on an exceptional basis is increasingly difficult to sustain from a health system perspective. An understanding of the value that societies attach to providing orphan drugs at the expense of other health technologies is now recognised as an important input to policy debates. OBJECTIVES: The aim of this work was to scope the social value arguments that have been advanced relating to the reimbursement of orphan drugs, and to locate these within a coherent decision-making framework to aid reimbursement decisions in the presence of limited healthcare resources. METHODS: A scoping review of the peer reviewed and grey literature was undertaken, consisting of seven phases: (1) identifying the research question; (2) searching for relevant studies; (3) selecting studies; (4) charting, extracting and tabulating data; (5) analyzing data; (6) consulting relevant experts; and (7) presenting results. The points within decision processes where the identified value arguments would be incorporated were then located. This mapping was used to construct a framework characterising the distinct role of each value in informing decision making. RESULTS: The scoping review identified 19 candidate decision factors, most of which can be characterised as either value-bearing or 'opportunity cost'-determining, and also a number of value propositions and pertinent sources of preference information. We were able to synthesize these into a coherent decision-making framework. CONCLUSION: Our framework may be used to structure policy discussions and to aid transparency about the values underlying reimbursement decisions for orphan drugs. These values ought to be consistently applied to all technologies and populations affected by the decision.

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2009Preferred reporting items for systematic reviews and meta-analyses: the PRISMA statement 2009Preferred reporting items for systematic reviews and meta-analyses: the PRISMA Statement.2005Scoping studies: towards a methodological framework 2002Qualitative data analysis for applied policy research 2010Qualitative data analysis for applied policy research 2002Analyzing Qualitative Data 2008The NICE Cost-Effectiveness Threshold 2001Synthesising research evidence.

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