Controlled access to lumasiran in primary hyperoxaluria type 1: evaluation of a new access route for orphan drugs in the Netherlands
This national ODAP protocol enabled access to lumasiran therapy for severely ill patients, prevented unnecessary treatment in others, and provided new insights into the real-world effectiveness of lumasiran in PH1 patients through systematic monitoring. It may serve as a template for future access routes to new expensive therapeutics in orphan diseases.
Authors
- Jaap W. Groothoff (ORCID: https://orcid.org/0000-0002-8467-3506)
- Eiske M. Dorresteijn (ORCID: https://orcid.org/0000-0002-1290-4395)
- Carla E. M. Hollak (ORCID: https://orcid.org/0000-0003-0464-1078)
- Azam Nurmohamed (ORCID: https://orcid.org/0000-0001-7666-643X)
- Nicole C. A. J. van de Kar (ORCID: https://orcid.org/0000-0002-1990-1189)
- Lisa J. Deesker
- David Severs (ORCID: https://orcid.org/0000-0002-9993-9995)
- Casper F. M. Franssen (ORCID: https://orcid.org/0000-0003-1004-9994)
- Sander F. Garrelfs (ORCID: https://orcid.org/0000-0002-6185-2616)
- Ankoor Roy
Institutions
- University Medical Center Groningen (NL)
- Radboud University Nijmegen (NL)
- University of Groningen (NL)
- Erasmus MC (NL)
- Radboud University Medical Center (NL)
- Emma Kinderziekenhuis (NL)
- Amsterdam Neuroscience (NL)
- Radboud Institute for Molecular Life Sciences (NL)
- National Health Insurance Fund (RS)
- Erasmus MC - Sophia Children’s Hospital (NL)
- Amsterdam University Medical Centers (NL)
- University of Amsterdam (NL)
- Erasmus University Rotterdam (NL)
Publication Details
- Journal
- Nephrology Dialysis Transplantation
- Published
- 2025-03-22
- DOI
- https://doi.org/10.1093/ndt/gfaf060
- Citations
- 6
- Primary Topic
- Kidney Stones and Urolithiasis Treatments
- Type
- article
- Field-Weighted Citation Impact
- 9.11
Funders
- Amsterdam University Medical Centers
- Zorginstituut Nederland