Access and Unmet Needs of Orphan Drugs in 194 Countries and 6 Areas: A Global Policy Review With Content Analysis.

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Abstract

Three hundred million people living with rare diseases worldwide are disproportionately deprived of in-time diagnosis and treatment compared with other patients. This review provides an overview of global policies that optimize development, licensing, pricing, and reimbursement of orphan drugs.

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Journal

Journal ID (iso-abbrev): Value Health

Title: Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research

Publisher: Elsevier BV

ISSN (Electronic): 1524-4733

ISSN (Print): 1098-3015

Publication date (Electronic): December 2020

Volume: 23

Issue: 12

Affiliations

[1 ] Centre for Safe Medication Practice and Research, Department of Pharmacology and Pharmacy, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong.

[2 ] International Society of Pharmacovigilance, London, United Kingdom.

[3 ] Department of Infectious Disease Epidemiology, London School of Hygiene & Tropical Medicine, London, United Kingdom; Modelling and Economics Unit, National Infections Service, Public Health England, London, United Kingdom; School of Public Health, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong.

[4 ] National Rare Diseases Registry System of China, Beijing, China; Rare Diseases Research Center, Chinese Academy of Medical Sciences, Beijing, China.

[5 ] Rare Diseases Research Center, Chinese Academy of Medical Sciences, Beijing, China; Peking Union Medical College Hospital, Beijing, China.

[6 ] Department of Paediatrics and Adolescent Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong.

[7 ] Centre for Safe Medication Practice and Research, Department of Pharmacology and Pharmacy, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong; Department of Paediatrics and Adolescent Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong; Department of Social Work and Social Administration, Faculty of Social Sciences, The University of Hong Kong, Hong Kong.

[8 ] Department of Social Work and Social Administration, Faculty of Social Sciences, The University of Hong Kong, Hong Kong; Sau Po Centre on Ageing, The University of Hong Kong, Hong Kong.

[9 ] Centre for Safe Medication Practice and Research, Department of Pharmacology and Pharmacy, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong; Centre for Medicines Optimisation Research and Education, Research Department of Policy and Practice, University College London School of Pharmacy and University College London Hospital, London, United Kingdom.

[10 ] Department of Paediatrics and Adolescent Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong. Electronic address: patricip@hku.hk.

[11 ] Centre for Safe Medication Practice and Research, Department of Pharmacology and Pharmacy, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong; Department of Paediatrics and Adolescent Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong; Department of Social Work and Social Administration, Faculty of Social Sciences, The University of Hong Kong, Hong Kong. Electronic address: sxueli@hku.hk.

Article

Publisher Item ID: S1098-3015(20)34413-2

DOI: 10.1016/j.jval.2020.06.020

PubMed ID: 33248513

SO-VID: 3dd4a78a-85f0-4f62-9a02-7e41c3cbf0f0

History

Keywords: drug regulatory,health equity,orphan drug policy,rare diseases,treatment access

Data availability:

Keywords: drug regulatory, health equity, orphan drug policy, rare diseases, treatment access

Access and Unmet Needs of Orphan Drugs in 194 Countries and 6 Areas: A Global Policy Review With Content Analysis.

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