Essential list of medicinal products for rare diseases: recommendations from the IRDiRC Rare Disease Treatment Access Working Group

Abstract Background Treatments are often unavailable for rare disease patients, especially in low-and-middle-income countries. Reasons for this include lack of financial support for therapies and onerous regulatory requirements for approval of drugs. Other barriers include lack of reimbursement, adm...

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Bibliographic Details

Published in:	Orphanet Journal of Rare Diseases
Main Authors:	William A. Gahl, Durhane Wong-Rieger, Virginie Hivert, Rachel Yang, Galliano Zanello, Stephen Groft
Format:	Article
Language:	English
Published:	BMC 2021-07-01
Online Access:	https://doi.org/10.1186/s13023-021-01923-0