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Estimating the budget impact of orphan medicines in Europe: 2010 - 2020

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References

1984Official Journal of the European Communities4,027 cited
2010Rare diseases, orphan drugs and their regulation: questions and misconceptions182 cited

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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
2012Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives117 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
2015Can the EVIDEM Framework Tackle Issues Raised by Evaluating Treatments for Rare Diseases: Analysis of Issues and Policies, and Context-Specific Adaptation77 cited
2018Patient Access to Medicines for Rare Diseases in European Countries76 cited
2012Orphan Drugs for Rare Diseases74 cited
2014Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders73 cited
2016Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries67 cited

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2018

The European challenges of funding orphan medicinal products

64 cited