New opportunities for accessing promising non-oncological orphan drugs

被引：0

作者：

Hollak, Carla E. M. ^{[1
,2
]}

Reparon-Schuijt, Carelle C. ^{[3
]}

Verdeyen, Kathelijn ^{[2
,4
]}

Deesker, Lisa J. ^{[5
]}

Groothoff, Jaap W. ^{[5
]}

Vos, Josephine M. I. ^{[4
]}

Huisman, Atse H. ^{[3
]}

van der Flier, Silvia ^{[3
]}

Roy, Anke A. M. G. Pisters-van ^{[3
]}

机构：

[1] Univ Amsterdam, Dept Endocrinol & Metab, Amsterdam UMC Locat, Amsterdam, Netherlands

[2] Univ Amsterdam, Med Soc, Platform Amsterdam UMC Locat, Amsterdam, Netherlands

[3] Hlth Insurers Netherlands, Dept Hlth Policy, Zeist, Netherlands

[4] Univ Amsterdam, Dept Hematol, Amsterdam UMC, Amsterdam, Netherlands

[5] Univ Amsterdam, Amsterdam UMC, Emma Childrens Hosp, Dept Pediat Nephrol, Amsterdam, Netherlands

来源：

LANCET REGIONAL HEALTH-EUROPE | 2025年 / 52卷

关键词：

D O I：

10.1016/j.lanepe.2025.101275

中图分类号：

R19 [保健组织与事业（卫生事业管理）];

学科分类号：

摘要：

引用

页数：2

共 10 条

[1] Orphan drugs' clinical uncertainty and prices: Addressing allocative and technical inefficiencies in orphan drug reimbursement [J].

Eichler, Hans-Georg ;

Kossmeier, Michael ;

Zeitlinger, Markus ;

Schwarzer-Daum, Brigitte .

FRONTIERS IN PHARMACOLOGY, 2023, 14

[2]

European Medicines Agency, Orphan medicines

[3] Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1 [J].

Garrelfs, Sander F. ;

Frishberg, Yaacov ;

Hulton, Sally A. ;

Koren, Michael J. ;

O'Riordan, William D. ;

Cochat, Pierre ;

Deschenes, Georges ;

Shasha-Lavsky, Hadas ;

Saland, Jeffrey M. ;

van't Hoff, William G. ;

Fuster, Daniel G. ;

Magen, Daniella ;

Moochhala, Shabbir H. ;

Schalk, Gesa ;

Simkova, Eva ;

Groothoff, Jaap W. ;

Sas, David J. ;

Meliambro, Kristin A. ;

Lu, Jiandong ;

Sweetser, Marianne T. ;

Garg, Pushkal P. ;

Vaishnaw, Akshay K. ;

Gansner, John M. ;

McGregor, Tracy L. ;

Lieske, John C. .

NEW ENGLAND JOURNAL OF MEDICINE, 2021, 384 (13) :1216-1226

[4] Drug pricing models, no 'one-size-fits-all' approach: a systematic review and critical evaluation of pricing models in an evolving pharmaceutical landscape [J].

Manders, Evert A. ;

van den Berg, Sibren ;

de Visser, Saco J. ;

Hollak, Carla E. M. .

EUROPEAN JOURNAL OF HEALTH ECONOMICS, 2024, :683-696

[5]

medicijnvoordemaatschappij, Medicines for Society

[6] Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS [J].

Miller, Timothy M. ;

Cudkowicz, Merit E. ;

Genge, Angela ;

Shaw, Pamela J. ;

Sobue, Gen ;

Bucelli, Robert C. ;

Chio, Adriano ;

Van Damme, Philip ;

Ludolph, Albert C. ;

Glass, Jonathan D. ;

Andrews, Jinsy A. ;

Babu, Suma ;

Benatar, Michael ;

McDermott, Christopher J. ;

Cochrane, Thos ;

Chary, Sowmya ;

Chew, Sheena ;

Zhu, Han ;

Wu, Fan ;

Nestorov, Ivan ;

Graham, Danielle ;

Sun, Peng ;

McNeill, Manjit ;

Fanning, Laura ;

Ferguson, Toby A. ;

Fradette, Stephanie .

NEW ENGLAND JOURNAL OF MEDICINE, 2022, 387 (12) :1099-1110

[7] Licensing of Orphan Medicinal Products-Use of Real-World Data and Other External Data on Efficacy Aspects in Marketing Authorization Applications Concluded at the European Medicines Agency Between 2019 and 2021 [J].

Naumann-Winter, Frauke ;

Wolter, Franziska ;

Hermes, Ulrike ;

Malikova, Eva ;

Lilienthal, Nils ;

Meier, Tania ;

Kalland, Maria Elisabeth ;

Magrelli, Armando .

FRONTIERS IN PHARMACOLOGY, 2022, 13

[8] Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches [J].

Nicod, Elena ;

Whittal, Amanda ;

Drummond, Michael ;

Facey, Karen .

ORPHANET JOURNAL OF RARE DISEASES, 2020, 15 (01)

[9] Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties [J].

Pontes, Caridad ;

Manuel Fontanet, Juan ;

Vives, Roser ;

Sancho, Aranzazu ;

Gomez-Valent, Monica ;

Rios, Jose ;

Morros, Rosa ;

Martinalbo, Jorge ;

Posch, Martin ;

Koch, Armin ;

Roes, Kit ;

Rengerink, Katrien Oude ;

Torrent-Farnell, Josep ;

Torres, Ferran .

ORPHANET JOURNAL OF RARE DISEASES, 2018, 13

[10] Sutimlimab in Cold Agglutinin Disease [J].

Roeth, Alexander ;

Barcellini, Wilma ;

D'Sa, Shirley ;

Miyakawa, Yoshitaka ;

Broome, Catherine M. ;

Michel, Marc ;

Kuter, David J. ;

Jilma, Bernd ;

Tvedt, Tor H. A. ;

Fruebis, Joachim ;

Jiang, Xiaoyu ;

Lin, Stella ;

Reuter, Caroline ;

Morales-Arias, Jaime ;

Hobbs, William ;

Berentsen, Sigbjorn .

NEW ENGLAND JOURNAL OF MEDICINE, 2021, 384 (14) :1323-1334

← 1 →