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Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches

Elena Nicod, Amanda Whittal, Michael Drummond, Karen Facey
July 2020

There is growing recognition that conventional appraisal approaches may be unsuitable for assessing the value of rare disease treatments (RDTs). This study reviews whether countries have implemented separate, ‘supplemental’ appraisal/reimbursement processes for RDTs and what these look like. The appraisal processes for 32 countries were first documented through questionnaires, desk research and iterative interactions with country experts. A cross-country analysis was then conducted to identify and characterise the key features included in the supplemental processes identified for RDTs. Findings revealed that these key features have the ability to increase the likelihood of reimbursement by adjusting and/or foregoing part of the assessment process, or by accepting to pay more for the same added benefit compared to common conditions. Findings suggest that there are preferences to treat RDTs differently compared to conventional medicines, and that supplemental process features can facilitate this through more flexible and consistent decision-making.

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