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The European challenges of funding orphan medicinal products
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The European challenges of funding orphan medicinal products (2018)The European challenges of funding orphan…Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group (2018)Challenges in Research and He…Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL) (2017)Recommendations from the Euro…Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries (2016)Systematic review on the eval…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…What Is Wrong with Orphan Drug Policies? (2012)What Is Wrong with Orphan Dru…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…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 …Assessing the economic challenges posed by orphan drugs (2007)Assessing the economic challe…Official Journal of the European Communities (1984)Official Journal of the Europ…How to Value Orphan Drugs? A Review of European Value Assessment Frameworks (2021)How to Value Orphan Drugs? A …Priority setting of health interventions: the need for multi-criteria decision analysis. (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)Turning a Blind Eye (2013)Orphan drugs policies: a suitable case for treatment (2014)Patient Access to Medicines for Rare Diseases in European Countries (2018)Establishing a reasonable price for an orphan drug (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)Linking payment to health outcomes: A taxonomy and examination of performance-based reimbursement schemes between healthcare payers and manufacturers (2010)Performance-Based Risk-Sharing Arrangements—Good Practices for Design, Implementation, and Evaluation: Report of the ISPOR Good Practices for Performance-Based Risk-Sharing Arrangements Task Force (2013)Managed Entry Agreements for Pharmaceuticals in the Context of Adaptive Pathways in Europe (2018)Performance-based managed entry agreements for new medicines in OECD countries and EU member states (2019)Rare diseases, orphan drugs and their regulation: questions and misconceptions (2010)Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives (2012)Does NICE have a cost‐effectiveness threshold and what other factors influence its decisions? A binary choice analysis (2004)Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries (2015)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 (2009)Preferred reporting items for systematic reviews and meta-analyses: the PRISMA Statement. (2009)Why rare diseases are an important medical and social issue (2008)A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) (2015)American Society of Clinical Oncology Statement: A Conceptual Framework to Assess the Value of Cancer Treatment Options (2015)Multi-Criteria Decision Analysis (MCDA) Models in Health Technology Assessment of Orphan Drugs—a Systematic Literature Review. Next Steps in Methodology Development? (2018)Comparing access to orphan medicinal products in Europe (2019)Implementing reflective multicriteria decision analysis (MCDA) to assess orphan drugs value in the Catalan Health Service (CatSalut) (2019)The Emperor of All Maladies: A Biography of Cancer (2010)Multiple Criteria Decision Analysis for Health Care Decision Making—An Introduction: Report 1 of the ISPOR MCDA Emerging Good Practices Task Force (2016)Multiple Criteria Decision Analysis for Health Care Decision Making—Emerging Good Practices: Report 2 of the ISPOR MCDA Emerging Good Practices Task Force (2016)Environmental management of a tourist destination (2000)The Government of Risk (2001)Online Behavioral Advertising: A Literature Review and Research Agenda (2017)
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References

  • 1984Official Journal of the European Communities4,027 cited
  • 2007Assessing the economic challenges posed by orphan drugs279 cited
  • 2011Pricing and reimbursement of orphan drugs: the need for more transparency261 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
  • 2011Estimating the budget impact of orphan medicines in Europe: 2010 - 2020133 cited
  • 2018Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group131 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
  • 2012What Is Wrong with Orphan Drug Policies?110 cited
  • 2017Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)103 cited
  • 2016Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries67 cited

Cited by

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

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