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Shining a light in the black box of orphan drug pricing

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References

2011Pricing and reimbursement of orphan drugs: the need for more transparency261 cited
2012SOCIETAL VIEWS ON NICE, CANCER DRUGS FUND AND VALUE‐BASED PRICING CRITERIA FOR PRIORITISING MEDICINES: A CROSS‐SECTIONAL SURVEY OF 4118 ADULTS IN GREAT BRITAIN208 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
2009The US Orphan Drug Act: Rare disease research stimulator or commercial opportunity?166 cited
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2011Estimating the budget impact of orphan medicines in Europe: 2010 - 2020133 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
2010A comparative study of European rare disease and orphan drug markets121 cited
2013A Pilot Study of Multicriteria Decision Analysis for Valuing Orphan Medicines111 cited
2012What Is Wrong with Orphan Drug Policies?110 cited

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2016Gene Therapies: The Challenge of sUper-High-Cost Treatments and How to Pay for them58 cited
2016Potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement process of orphan drugs54 cited

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