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What Is Wrong with Orphan Drug Policies? Suggestions for Ways Forward

Panos Kanavos, Elena Nicod
October 2012

This article reviews the key criticisms surrounding orphan medicinal product (OMP) policies and compares their weaknesses against the positive outcomes that they have had on encouraging R&D investment. It argues that while there are areas of the current policy framework which would benefit from improvement or refinement, this does not mean it should be replaced or dismissed altogether. Instead, the authors recommend changes around certain aspects, such as addressing data collection issues, more clearly defining the thresholds for excessive OMP profits, and updating assessment strategies to better accommodate OMPs and allow for more realistic, affordable payer pricing to become the norm.

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