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

Orphanet Journal of Rare Diseases
Elena NicodKaren Facey

Abstract

There is increasing recognition that conventional appraisal approaches may be unsuitable for assessing the value rare disease treatments (RDTs). This research examines what supplemental appraisal/reimbursement processes for RDTs are used internationally and how they can be characterised. A qualitative research design was used that included (1) documentation of country appraisal/reimbursement processes for RDTs via questionnaires, desk research and iterative interactions with country experts to produce country vignettes, and (2) a cross-country analysis of these processes to identify and characterise features in supplemental processes for RDTs, and compare them to countries without supplemental processes. Thirty-two of the 37 invited countries participated in this research. Forty-one percent (13/32) use supplemental processes for RDTs. Their level of integration within standard processes ranged from low to high, characterised by whether they are separate or partially separate from the standard process, adapted or accelerated standard processes, or standard processes that may be applied to RDTs. They are characterised by features implemented throughout the appraisal process. These features are mechanisms that allow application of...Continue Reading

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Citations

May 29, 2021·International Journal of Technology Assessment in Health Care·Amanda WhittalKaren Facey
Jun 1, 2021·Frontiers in Pharmacology·Alessandra BlondaSteven Simoens

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