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

TitleAccess and Unmet Needs of Orphan Drugs in 194 Countries and 6 Areas: A Global Policy Review With Content Analysis
Authors
Keywordsdrug regulatory
health equity
orphan drug policy
rare diseases
treatment access
Issue Date2020
PublisherElsevier Inc. The Journal's web site is located at http://www.valueinhealthjournal.com/
Citation
Value in Health, 2020, v. 23 n. 12, p. 1580-1591 How to Cite?
AbstractObjectives: 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.
DescriptionHybrid open access
Persistent Identifierhttp://hdl.handle.net/10722/293682
ISSN
2023 Impact Factor: 4.9
2023 SCImago Journal Rankings: 1.507
ISI Accession Number ID

 

DC FieldValueLanguage
dc.contributor.authorChan, AYL-
dc.contributor.authorCHAN, VKY-
dc.contributor.authorOlsson, S-
dc.contributor.authorFAN, M-
dc.contributor.authorJit, M-
dc.contributor.authorGong, M-
dc.contributor.authorZhan, S-
dc.contributor.authorGE, M-
dc.contributor.authorPathadka, S-
dc.contributor.authorCHUNG, CCY-
dc.contributor.authorChung, BHY-
dc.contributor.authorChui, CSL-
dc.contributor.authorChan, EW-
dc.contributor.authorWong, GHY-
dc.contributor.authorLum, TY-
dc.contributor.authorWong, ICK-
dc.contributor.authorIp, P-
dc.contributor.authorLi, X-
dc.date.accessioned2020-11-23T08:20:17Z-
dc.date.available2020-11-23T08:20:17Z-
dc.date.issued2020-
dc.identifier.citationValue in Health, 2020, v. 23 n. 12, p. 1580-1591-
dc.identifier.issn1098-3015-
dc.identifier.urihttp://hdl.handle.net/10722/293682-
dc.descriptionHybrid open access-
dc.description.abstractObjectives: 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.-
dc.languageeng-
dc.publisherElsevier Inc. The Journal's web site is located at http://www.valueinhealthjournal.com/-
dc.relation.ispartofValue in Health-
dc.rightsThis work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License.-
dc.subjectdrug regulatory-
dc.subjecthealth equity-
dc.subjectorphan drug policy-
dc.subjectrare diseases-
dc.subjecttreatment access-
dc.titleAccess and Unmet Needs of Orphan Drugs in 194 Countries and 6 Areas: A Global Policy Review With Content Analysis-
dc.typeArticle-
dc.identifier.emailChan, AYL: adrc@hku.hk-
dc.identifier.emailPathadka, S: swathip@hku.hk-
dc.identifier.emailChung, BHY: bhychung@hku.hk-
dc.identifier.emailChui, CSL: cslchui@hku.hk-
dc.identifier.emailChan, EW: ewchan@hku.hk-
dc.identifier.emailWong, GHY: ghywong@hku.hk-
dc.identifier.emailLum, TY: tlum@hku.hk-
dc.identifier.emailWong, ICK: wongick@hku.hk-
dc.identifier.emailIp, P: patricip@hku.hk-
dc.identifier.emailLi, X: sxueli@hku.hk-
dc.identifier.authorityChung, BHY=rp00473-
dc.identifier.authorityChui, CSL=rp02527-
dc.identifier.authorityChan, EW=rp01587-
dc.identifier.authorityWong, GHY=rp01850-
dc.identifier.authorityLum, TY=rp01513-
dc.identifier.authorityWong, ICK=rp01480-
dc.identifier.authorityIp, P=rp01337-
dc.identifier.authorityLi, X=rp02531-
dc.description.naturepublished_or_final_version-
dc.identifier.doi10.1016/j.jval.2020.06.020-
dc.identifier.pmid33248513-
dc.identifier.scopuseid_2-s2.0-85094939129-
dc.identifier.hkuros318867-
dc.identifier.volume23-
dc.identifier.issue12-
dc.identifier.spage1580-
dc.identifier.epage1591-
dc.identifier.isiWOS:000602492000007-
dc.publisher.placeUnited States-

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