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Economic Modeling Considerations for Rare Diseases
16 references·1 citing·44 second hop
Economic Modeling Considerations for Rare Diseases (2018)Economic Modeling Considerations for Rare…Using Experts` Opinions Through Delphi Technique (2020)Using Experts` Opinions Throu…Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL) (2017)Recommendations from the Euro…Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs? (2017)Ethical imperatives of timely…Social/economic costs and health-related quality of life in patients with rare diseases in Europe (2016)Social/economic costs and hea…Meta‐analysis of few small studies in orphan diseases (2016)Meta‐analysis of few small st…Estimating Health-State Utility for Economic Models in Clinical Studies: An ISPOR Good Research Practices Task Force Report (2016)Estimating Health-State Utili…Assessing the Gold Standard — Lessons from the History of RCTs (2016)Assessing the Gold Standard —…Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework (2014)Value-Based Reimbursement Dec…Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: a comparative study of managed entry agreements across seven European countries (2013)Reconciling uncertainty of co…Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments? (2012)Paying for the Orphan Drug Sy…Modeling Good Research Practices—Overview: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force-1 (2012)Modeling Good Research Practi…Model Transparency and Validation: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force-7 (2012)Model Transparency and Valida…Model Transparency and Validation (2012)Model Transparency and Valida…Pricing and reimbursement of orphan drugs: the need for more transparency (2011)Pricing and reimbursement of …Assessing the economic challenges posed by orphan drugs (2007)Assessing the economic challe…Proxy completion of EQ-5D in patients with dementia (2005)Proxy completion of EQ-5D in …How to Value Orphan Drugs? A Review of European Value Assessment Frameworks (2021)How to Value Orphan Drugs? A …Simulation Modeling and Analysis. 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(2006)Stratified medicine: strategic and economic implications of combining drugs and clinical biomarkers (2007)Barriers for Access to New Medicines: Searching for the Balance Between Rising Costs and Limited Budgets (2018)Outrageous prices of orphan drugs: a call for collaboration (2018)A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries (2020)Cost-Effectiveness Analysis and the Consistency of Decision Making (2001)National Institute for Clinical Excellence and its value judgments (2004)QALY maximisation and people's preferences: a methodological review of the literature (2004)Orphan drug development: an economically viable strategy for biopharma R&D (2012)Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy (2023)A Guide to Simulation (1983)Decision Making in Health and Medicine: Integrating Evidence and Values (2001)Model Parameter Estimation and Uncertainty: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force-6 (2012)Does NICE have a cost‐effectiveness threshold and what other factors influence its decisions? 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References

  • 2012Model Transparency and Validation802 cited
  • 2012Modeling Good Research Practices—Overview: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force-1767 cited
  • 2016Assessing the Gold Standard — Lessons from the History of RCTs663 cited
  • 2020Using Experts` Opinions Through Delphi Technique476 cited
  • 2012Model Transparency and Validation: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force-7467 cited
  • 2007Assessing the economic challenges posed by orphan drugs279 cited
  • 2011Pricing and reimbursement of orphan drugs: the need for more transparency261 cited
  • 2016Meta‐analysis of few small studies in orphan diseases190 cited
  • 2012Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?171 cited
  • 2016Estimating Health-State Utility for Economic Models in Clinical Studies: An ISPOR Good Research Practices Task Force Report159 cited
  • 2013Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: a comparative study of managed entry agreements across seven European countries122 cited
  • 2016Social/economic costs and health-related quality of life in patients with rare diseases in Europe121 cited
  • 2017Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs?

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113 cited
  • 2005Proxy completion of EQ-5D in patients with dementia103 cited
  • 2017Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)103 cited
  • 2014Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework99 cited
  • Cited by

    • 2021How to Value Orphan Drugs? A Review of European Value Assessment Frameworks62 cited