Access to orphan drugs: A comprehensive review of legislations, regulations and policies in 35 countries

Todd Gammie, Christine Y. Lu, Zaheer Ud-Din Babar

Research output: Contribution to journalReview article

74 Citations (Scopus)

Abstract

Objective: To review existing regulations and policies utilised by countries to enable patient access to orphan drugs. Methods: A review of the literature (1998 to 2014) was performed to identify relevant, peer-reviewed articles. Using content analysis, we synthesised regulations and policies for access to orphan drugs by type and by country. Results: Fifty seven articles and 35 countries were included in this review. Six broad categories of regulation and policy instruments were identified: national orphan drug policies, orphan drug designation, marketing authorization, incentives, marketing exclusivity, and pricing and reimbursement. The availability of orphan drugs depends on individual country's legislation and regulations including national orphan drug policies, orphan drug designation, marketing authorization, marketing exclusivity and incentives such as tax credits to ensure research, development and marketing. The majority of countries (27/35) had in place orphan drug legislation. Access to orphan drugs depends on individual country's pricing and reimbursement policies, which varied widely between countries. High prices and insufficient evidence often limit orphan drugs from meeting the traditional health technology assessment criteria, especially cost-effectiveness, which may influence access. Conclusions: Overall many countries have implemented a combination of legislations, regulations and policies for orphan drugs in the last two decades. While these may enable the availability and access to orphan drugs, there are critical differences between countries in terms of range and types of legislations, regulations and policies implemented. Importantly, China and India, two of the largest countries by population size, both lack national legislation for orphan medicines and rare diseases, which could have substantial negative impacts on their patient populations with rare diseases.

LanguageEnglish
Article numbere0140002
Number of pages24
JournalPLoS One
Volume10
Issue number10
DOIs
Publication statusPublished - 9 Oct 2015
Externally publishedYes

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Orphan Drug Production
Legislation
laws and regulations
drugs
Marketing
marketing
Rare Diseases
Motivation
Availability
Drug Legislation
Costs and Cost Analysis
Biomedical Technology Assessment
Taxes
cost effectiveness
peers
Cost effectiveness
Population Density
research and development
Taxation
Medicine

Cite this

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abstract = "Objective: To review existing regulations and policies utilised by countries to enable patient access to orphan drugs. Methods: A review of the literature (1998 to 2014) was performed to identify relevant, peer-reviewed articles. Using content analysis, we synthesised regulations and policies for access to orphan drugs by type and by country. Results: Fifty seven articles and 35 countries were included in this review. Six broad categories of regulation and policy instruments were identified: national orphan drug policies, orphan drug designation, marketing authorization, incentives, marketing exclusivity, and pricing and reimbursement. The availability of orphan drugs depends on individual country's legislation and regulations including national orphan drug policies, orphan drug designation, marketing authorization, marketing exclusivity and incentives such as tax credits to ensure research, development and marketing. The majority of countries (27/35) had in place orphan drug legislation. Access to orphan drugs depends on individual country's pricing and reimbursement policies, which varied widely between countries. High prices and insufficient evidence often limit orphan drugs from meeting the traditional health technology assessment criteria, especially cost-effectiveness, which may influence access. Conclusions: Overall many countries have implemented a combination of legislations, regulations and policies for orphan drugs in the last two decades. While these may enable the availability and access to orphan drugs, there are critical differences between countries in terms of range and types of legislations, regulations and policies implemented. Importantly, China and India, two of the largest countries by population size, both lack national legislation for orphan medicines and rare diseases, which could have substantial negative impacts on their patient populations with rare diseases.",
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Access to orphan drugs : A comprehensive review of legislations, regulations and policies in 35 countries. / Gammie, Todd; Lu, Christine Y.; Ud-Din Babar, Zaheer.

In: PLoS One, Vol. 10, No. 10, e0140002, 09.10.2015.

Research output: Contribution to journalReview article

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