Adrienne Y L Chan1, Vivien K Y Chan1, Sten Olsson2, Min Fan1, Mark Jit3, Mengchun Gong4, Shuyang Zhang5, Mengqin Ge1, Swathi Pathadka1, Claudia C Y Chung6, Brian H Y Chung6, Celine S L Chui7, Esther W Chan1, Gloria H Y Wong8, Terry Y Lum8, Ian C K Wong9, Patrick Ip10, Xue Li11. 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.
Abstract
OBJECTIVES: 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. METHODS: Pharmaceutical legislation and policies related to access and regulation of orphan drugs were examined from 194 World Health Organization member countries and 6 areas. Orphan drug policies (ODPs) were identified through internet search, emails to national pharmacovigilance centers, and systematic academic literature search. Texts from selected publications were extracted for content analysis. RESULTS: One hundred seventy-two drug regulation documents and 77 academic publications from 162 countries/areas were included. Ninety-two of 200 countries/areas (46.0%) had documentation on ODPs. Thirty-four subthemes from content analysis were categorized into 6 policy themes, namely, orphan drug designation, marketing authorization, safety and efficacy requirements, price regulation, incentives that encourage market availability, and incentives that encourage research and development. Countries/areas with ODPs were statistically wealthier (gross national income per capita = $10 875 vs $3950, P < .001). Country/area income was also positively correlated with the scope of the respective ODP (correlation coefficient = 0.57, P < .001). CONCLUSIONS: Globally, the number of countries with an ODP has grown rapidly since 2013. Nevertheless, disparities in geographical distribution and income levels affect the establishment of ODPs. Furthermore, identified policy gaps in price regulation, incentives that encourage market availability, and incentives that encourage research and development should be addressed to improve access to available and affordable orphan drugs.
OBJECTIVES: 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. METHODS: Pharmaceutical legislation and policies related to access and regulation of orphan drugs were examined from 194 World Health Organization member countries and 6 areas. Orphan drug policies (ODPs) were identified through internet search, emails to national pharmacovigilance centers, and systematic academic literature search. Texts from selected publications were extracted for content analysis. RESULTS: One hundred seventy-two drug regulation documents and 77 academic publications from 162 countries/areas were included. Ninety-two of 200 countries/areas (46.0%) had documentation on ODPs. Thirty-four subthemes from content analysis were categorized into 6 policy themes, namely, orphan drug designation, marketing authorization, safety and efficacy requirements, price regulation, incentives that encourage market availability, and incentives that encourage research and development. Countries/areas with ODPs were statistically wealthier (gross national income per capita = $10 875 vs $3950, P < .001). Country/area income was also positively correlated with the scope of the respective ODP (correlation coefficient = 0.57, P < .001). CONCLUSIONS: Globally, the number of countries with an ODP has grown rapidly since 2013. Nevertheless, disparities in geographical distribution and income levels affect the establishment of ODPs. Furthermore, identified policy gaps in price regulation, incentives that encourage market availability, and incentives that encourage research and development should be addressed to improve access to available and affordable orphan drugs.
Authors: Maarten J Postma; Declan Noone; Mark H Rozenbaum; John A Carter; Marc F Botteman; Elisabeth Fenwick; Louis P Garrison Journal: Orphanet J Rare Dis Date: 2022-04-05 Impact factor: 4.123
Authors: Monica Mazzucato; Cinzia Minichiello; Andrea Vianello; Laura Visonà Dalla Pozza; Ema Toto; Paola Facchin Journal: Front Pharmacol Date: 2022-09-30 Impact factor: 5.988