Application of four pricing models for orphan medicines: a case study for lumasiran
Outcomes of the four pricing models show wide and heterogeneous price ranges. The DCF-model might be most compatible with the case of lumasiran, due to inclusion of parameters for prevalence, incidence, prescription restrictions and cost of capital. The minimum DCF price could serve as a starting point for pricing and reimbursement negotiations. Uncertainties can be solved by more transparency on input variables.
Authors
- Saco J. de Visser (ORCID: https://orcid.org/0000-0003-3649-5714)
- Jaap W. Groothoff (ORCID: https://orcid.org/0000-0002-8467-3506)
- Carla E. M. Hollak (ORCID: https://orcid.org/0000-0003-0464-1078)
- Noa Rosenberg (ORCID: https://orcid.org/0000-0002-9586-6135)
- Lisa J. Deesker
- Sander F. Garrelfs (ORCID: https://orcid.org/0000-0002-6185-2616)
- Sibren van den Berg (ORCID: https://orcid.org/0000-0001-8908-3858)
- Evert A. Manders
Institutions
- Emma Kinderziekenhuis (NL)
- Amsterdam Neuroscience (NL)
- Amsterdam University Medical Centers (NL)
- University of Amsterdam (NL)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2024-12-23
- DOI
- https://doi.org/10.1186/s13023-024-03446-w
- Citations
- 3
- Primary Topic
- Health Systems, Economic Evaluations, Quality of Life
- Type
- article
- Field-Weighted Citation Impact
- 3.39