Commentary Orphan drugs revisited
نویسندگان
چکیده
C. MCCABE, A. TSUCHIYA, K. CLAXTON and J. RAFTERY From the Section of Health Economics and Decision Science, School of Health and Related Research, University of Sheffield, Department of Economics, University of Sheffield, Sheffield, Department of Economics and Related Studies, University of York, York, and Wessex Institute for Health Research and Development, University of Southampton, Southampton, UK
منابع مشابه
The correlation between HTA recommendations and reimbursement status of orphan drugs in Europe
BACKGROUND The aim of this study was to review and compare types of reimbursement recommendations for orphan drugs issued by eight European health technology assessment (HTA) agencies and the reimbursement status of these drugs in the corresponding countries. Separate calculations were also performed for three sub-groups: ultra-orphan drugs, oncology orphan drugs and other (non-ultra, non-oncol...
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BACKGROUND Health Canada has defined rare diseases as life-threatening, seriously debilitating, or serious chronic conditions affecting a very small number of patients (~1 in 2,000 persons). An estimated 9 % of Canadians suffer from a rare disease. Drugs treating rare diseases (DRDs) are also known as orphan drugs. While Canada is currently developing an orphan drug framework, in the United Sta...
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BACKGROUND The relatively low budget impact of orphan drugs is often used as an argument in reimbursement decisions. However, overall, the budget impact of orphan drugs can still be substantial. In this study, we assess the uptake and budget impact of orphan drugs in the Netherlands. METHODS We examined the number of orphan drugs, the number of patients and budget impact of orphan drugs in th...
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BACKGROUND The pricing mechanism of orphan drugs appears arbitrary and has been referred to as a "black box". Therefore, the aim of this study is to investigate how drug- and disease-specific variables relate to orphan drug prices. Additionally, we aim to explore if certain country-specific pricing and reimbursement policies affect the price level of orphan drugs. METHODS Annual treatment cos...
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