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Patients, payers and developers of Orphan Medicinal Products: lessons learned from 10 years' multi-stakeholder dialogue on improving access in Europe via MoCA.
Cavaller-Bellaubi M, Hughes-Wilson W, Kubinová Š, Van de Casteele M, Van Lente EJ, Degortes E, Pontén J, Eichler HG, Le Cam Y, Boselli S, Bucsics A. Cavaller-Bellaubi M, et al. Among authors: le cam y. Orphanet J Rare Dis. 2023 Jun 12;18(1):144. doi: 10.1186/s13023-023-02774-7. Orphanet J Rare Dis. 2023. PMID: 37308991 Free PMC article.
Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL).
Annemans L, Aymé S, Le Cam Y, Facey K, Gunther P, Nicod E, Reni M, Roux JL, Schlander M, Taylor D, Tomino C, Torrent-Farnell J, Upadhyaya S, Hutchings A, Le Dez L. Annemans L, et al. Among authors: le cam y, le dez l. Orphanet J Rare Dis. 2017 Mar 10;12(1):50. doi: 10.1186/s13023-017-0601-9. Orphanet J Rare Dis. 2017. PMID: 28283046 Free PMC article.
Medicines Adaptive Pathways to Patients: Why, When, and How to Engage?
Eichler HG, Bedlington N, Boudes M, Bouvy JC, Broekmans AW, Cerreta F, Faulkner SD, Forda SR, Joos A, Le Cam Y, Mayer MH, Pirard V, Corriol-Rohou S; ADAPT SMART Consortium. Eichler HG, et al. Among authors: le cam y. Clin Pharmacol Ther. 2019 May;105(5):1148-1155. doi: 10.1002/cpt.1121. Epub 2018 Sep 11. Clin Pharmacol Ther. 2019. PMID: 29901216 Free PMC article. Review.
The evolution of adaptiveness: balancing speed and evidence.
Eichler HG, Barker R, Bedlington N, Bouvy JC, Broekmans AW, Bucsics A, Cerreta F, Corriol-Rohou S, Granados A, Le Cam Y, Schuurman A. Eichler HG, et al. Among authors: le cam y. Nat Rev Drug Discov. 2018 Dec;17(12):845-846. doi: 10.1038/nrd.2018.90. Epub 2018 Jul 6. Nat Rev Drug Discov. 2018. PMID: 29977052 No abstract available.
EUROPLAN: a project to support the development of national plans on rare diseases in Europe.
Taruscio D, Gentile AE, De Santis M, Ferrelli RM, Posada de la Paz M, Hens M, Huizer J, Fregonese L, Stefanov R, Bottarelli V, Weinman A, Le Cam Y, Gavhed D, Mincarone P, Bushby K, Frazzica RG, Donati C, Vittozzi L, Jessop E. Taruscio D, et al. Among authors: le cam y. Public Health Genomics. 2013;16(6):278-87. doi: 10.1159/000355932. Epub 2014 Feb 3. Public Health Genomics. 2013. PMID: 24503588
27 results