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Application of four pricing models for orphan medicines: a case study for lumasiran

Abstract Background The combination of high prices and uncertain effectiveness is a growing challenge in the field of orphan medicines, hampering health technology assessments. Hence, new methods for establishing price benchmarks might be necessary to support reimbursement negotiations. In this stud...

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Bibliografiske detaljer
Principais autores: Noa Rosenberg, Evert Manders, Sibren van den Berg, Lisa J. Deesker, Sander F. Garrelfs, Saco J. de Visser, Jaap W. Groothoff, Carla E. M. Hollak
Format: Artigo
Sprog:Inglês
Udgivet: BMC 2024-12-01
Serier:Orphanet Journal of Rare Diseases
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Online adgang:https://doi.org/10.1186/s13023-024-03446-w
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