HTA challenges for appraising rare disease interventions viewed through the lens of an institutional multidimensional value framework-Reference-Cited by-同舟云学术

HTA challenges for appraising rare disease interventions viewed through the lens of an institutional multidimensional value framework

Published:2022-12-26 Issue:2 Volume:23 Page:143-152
ISSN:1473-7167
Container-title:Expert Review of Pharmacoeconomics & Outcomes Research
language:en
Short-container-title:Expert Review of Pharmacoeconomics & Outcomes Research

Author:

Wagner Monika¹,Goetghebeur Mireille M¹²,Ganache Isabelle¹³,Demers-Payette Olivier¹²,Auclair Yannick¹,Olivier Catherine¹³,Arbour Sylvie¹,de Guise Michèle¹⁴

Affiliation:

1. Bureau - Méthodologies et éthique, Institut national d’excellence en santé et en services sociaux (INESSS), 2021 Avenue Union, bureau 1200; Montréal H3A 2S9, Quebec, Canada

2. School of Public Health, Department of Management, Evaluation and Health Policy, Université de Montréal; 7101, avenue du Parc, 3e étage H3N 1X9 Montréal, Quebec, Canada

3. School of Public Health, Department of Social and Preventive Medicine, Université de Montréal, 7101avenue du Parc, 3e étage H3N 1X9 Montréal, Quebec, Canada

4. Institut national d'excellence en santé et en services sociaux (INESSS), 2021 Avenue Union, bureau 1200; Montréal H3A 2S9, Québec, Canada

Funder

funded

Publisher

Informa UK Limited

Subject

Pharmacology (medical),Health Policy,General Medicine

Link

https://www.tandfonline.com/doi/pdf/10.1080/14737167.2023.2161513

Reference62 articles.

1. Ministère de la Santé et des Services Sociaux. Pour une meilleure reconnaissance et prise en charge des personnes atteintes de maladies rares - Politique québécoise pour les maladies rares, 22-916-01W, Gouvernement du Québec. 2022 [cited 2022 Aug 31]. https://publications.msss.gouv.qc.ca/msss/document-003397/.

2. Spotlight on rare diseases

3. Regroupement québécois des maladies orphelines. Informations sur les maladies rares et orphelines [updated 2015; cited 2015; cited 2021 Nov 30]. Available from: https://rqmo.org/information-sur-les-maladies-rares-et-orphelines/

4. FDA Approval and Regulation of Pharmaceuticals, 1983-2018

5. Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases

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