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Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose?
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Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose? (2022)Assessing the value of orphan drugs using…Defining Elements of Value in Health Care—A Health Economics Approach: An ISPOR Special Task Force Report [3] (2018)Defining Elements of Value in…Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare (2018)Advanced therapy medicinal pr…A Systematic Review of Studies Comparing the Measurement Properties of the Three-Level and Five-Level Versions of the EQ-5D (2018)A Systematic Review of Studie…A Health Economics Approach to US Value Assessment Frameworks—Summary and Recommendations of the ISPOR Special Task Force Report [7] (2018)A Health Economics Approach t…Using Cost-Effectiveness Analysis to Address Health Equity Concerns (2017)Using Cost-Effectiveness Anal…An Introduction to the Principles of Morals and Legislation (2017)An Introduction to the Princi…Extended Cost-Effectiveness Analysis for Health Policy Assessment: A Tutorial (2016)Extended Cost-Effectiveness A…Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries (2015)Access to Orphan Drugs: A Com…Orphan drugs: the regulatory environment (2012)Orphan drugs: the regulatory …Pricing and reimbursement of orphan drugs: the need for more transparency (2011)Pricing and reimbursement of …Estimating the budget impact of orphan medicines in Europe: 2010 - 2020 (2011)Estimating the budget impact …QALYs: Some Challenges (2009)QALYs: Some ChallengesWhy rare diseases are an important medical and social issue (2008)Why rare diseases are an impo…Drugs for exceptionally rare diseases: do they deserve special status for funding? 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(2012)Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy (2023)The Quality of Life (1993)Patterns and problems of deliberate self-poisoning in the developing world (2000)Capability and Well-Being (2007)Optimal Cost-Effectiveness Decisions: The Role of the Cost-Effectiveness Acceptability Curve (CEAC), the Cost-Effectiveness Acceptability Frontier (CEAF), and the Expected Value of Perfection Information (EVPI) (2008)Welfarism, extra-welfarism and capability: The spread of ideas in health economics (2008)Scoring the Icecap‐a Capability Instrument. Estimation of a UK General Population Tariff (2013)Preferred reporting items for systematic reviews and meta-analyses: the PRISMA statement (2009)Preferred reporting items for systematic reviews and meta-analyses: the PRISMA Statement. 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(1979)Quality criteria were proposed for measurement properties of health status questionnaires (2006)Development and preliminary testing of the new five-level version of EQ-5D (EQ-5D-5L) (2011)A Comparison of the EQ-5D-3L and EQ-5D-5L (2020)Health-Related Quality of Life and its Associated Factors in COVID-19 Patients (2020)The Danish EQ-5D-5L Value Set: A Hybrid Model Using cTTO and DCE Data (2021)A theory of justice original edition (1971)Judgment under Uncertainty: Heuristics and Biases (1974)Prospect Theory: An Analysis of Decision under Risk (1979)Ambiguity and Self-Evaluation: The Role of Idiosyncratic Trait Definitions in Self-Serving Assessments of Ability (2002)Norm Theory: Comparing Reality to Its Alternatives (2002)Equity, environmental justice and sustainability: incomplete approaches in climate change politics (2003)Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years (2010)The supportive care needs of parents with a child with a rare disease: results of an online survey (2016)Alone in a Crowd? Parents of Children with Rare Diseases’ Experiences of Navigating the Healthcare System (2018)Foundations of Social Theory (1991)Universals in the Content and Structure of Values: Theoretical Advances and Empirical Tests in 20 Countries (1992)Back to Bentham? Explorations of Experienced Utility (1997)
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References

  • 2017An Introduction to the Principles of Morals and Legislation6,717 cited
  • 2008Why rare diseases are an important medical and social issue621 cited
  • 2018Defining Elements of Value in Health Care—A Health Economics Approach: An ISPOR Special Task Force Report [3]544 cited
  • 2018A Systematic Review of Studies Comparing the Measurement Properties of the Three-Level and Five-Level Versions of the EQ-5D355 cited
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  • 2004Is economic evaluation in touch with society's health values?297 cited
  • 2015Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries278 cited
  • 1994Nonconsequentialist decisions269 cited
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  • 2016Extended Cost-Effectiveness Analysis for Health Policy Assessment: A Tutorial251 cited
  • 2005Drugs for exceptionally rare diseases: do they deserve special status for funding?220 cited
  • 2009QALYs: Some Challenges187 cited
  • 2018A Health Economics Approach to US Value Assessment Frameworks—Summary and Recommendations of the ISPOR Special Task Force Report [7]152 cited
  • 2012Orphan drugs: the regulatory environment144 cited

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  • 2011Estimating the budget impact of orphan medicines in Europe: 2010 - 2020133 cited
  • 2018Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare124 cited
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