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. 2025 Mar 21:52:101275.
doi: 10.1016/j.lanepe.2025.101275. eCollection 2025 May.

New opportunities for accessing promising non-oncological orphan drugs

Carla E M Hollak  1   2 , Carelle C Reparon-Schuijt  3 , Kathelijn Verdeyen  2   4 , Lisa J Deesker  5 , Jaap W Groothoff  5 , Josephine M I Vos  4 , Atse H Huisman  3 , Silvia van der Flier  3 , Anke A M G Pisters-van Roy  3
Affiliations

New opportunities for accessing promising non-oncological orphan drugs

Carla E M Hollak et al. Lancet Reg Health Eur. .
No abstract available

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Conflict of interest statement

The Orphan Drug Access Protocol (ODAP) is financially supported by Health Insurers Netherlands https://www.zn.nl/over-zn/about-us/ CEMH is Member of Advisory Committee to the insured healthcare package (Dutch Healthcare Institute), Chair of “Medicines for Society” and member of round table on orphan drugs (Dutch Healthcare Institute). CCRS is member of round table on orphan drugs (Dutch Healthcare Institute). KV was paid by Sanofi to participate in an educational meeting in 2024. LD received a Kidney star grant from the Amercian Society of Nephrology to participate in the ASN kidney week. JWG received consultancy fees from Alnylam. J.M.I. V recieved research grants from Beigene and AbbVie/Genmab and received comsultancy fees from Sanofi and Janssen and honoraria for lectures from BMS, Sanofi, and Amgen. A.H.H is Member of ODAP board, chair of committee for reimbursement of Health Insurers Netherlands. SvdF has nothing to disclose. A.A.M.G.P-vR is member of round table on orphan drugs (Dutch Healthcare Institute).

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References

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