The implementation of health technology assessment principles in public decisions concerning orphan drugs
Author:
Funder
OSSFOR - Osservatorio Farmaci Orfani -Roma
Publisher
Springer Science and Business Media LLC
Subject
Pharmacology (medical),Pharmacology,General Medicine
Link
http://link.springer.com/content/pdf/10.1007/s00228-020-02855-7.pdf
Reference55 articles.
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2. EMA, Annual report on the use of the special contribution for orphan medicinal products (2017)
3. Schey C, Milanova T, Hutchings A (2011) Estimating the budget impact of orphan medicines in Europe: 2010-2020. Orphanet J Rare Dis 6:62
4. Danzon PM (2018 Mar) Affordability challenges to value-based pricing: mass diseases, orphan diseases, and cures. Value Health 21(3):252–257
5. Paulden M, Stafinski T, Menon D, McCabe C (2015) Value-based reimbursement decisions for orphan drugs: a scoping review and decision framework. PharmacoEconomics 33:255–269
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1. HTA criteria adopted in different models of public healthcare systems for orphan drugs: A scoping review;Health Policy;2024-06
2. Navigating the unknown: how to best ‘reflect’ standard of care in indications without a dedicated treatment pathway in health technology assessment submissions;Journal of Comparative Effectiveness Research;2024-02
3. How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes? A Qualitative Interview Study Across European Countries;Frontiers in Pharmacology;2022-07-19
4. Key Considerations in the Health Technology Assessment of Advanced Therapy Medicinal Products in Scotland, The Netherlands, and England;Value in Health;2021-12
5. A systematic review of moral reasons on orphan drug reimbursement;Orphanet Journal of Rare Diseases;2021-06-30
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