State of rare disease management in Southeast Asia

Shafie, Asrul Akmal, Chaiyakunapruk, Nathorn, Supian, Azuwana, Lim, Jeremy, Zafra, Matt and Hassali, Mohamed Azmi Ahmad (2016) State of rare disease management in Southeast Asia. Orphanet Journal of Rare Diseases, 11 1: 107. doi:10.1186/s13023-016-0460-9


Author Shafie, Asrul Akmal
Chaiyakunapruk, Nathorn
Supian, Azuwana
Lim, Jeremy
Zafra, Matt
Hassali, Mohamed Azmi Ahmad
Title State of rare disease management in Southeast Asia
Journal name Orphanet Journal of Rare Diseases   Check publisher's open access policy
ISSN 1750-1172
Publication date 2016-08-02
Sub-type Article (original research)
DOI 10.1186/s13023-016-0460-9
Open Access Status DOI
Volume 11
Issue 1
Start page 107
Total pages 11
Place of publication London, United Kingdom
Publisher BioMed Central
Language eng
Abstract Rare diseases, also referred to as orphan diseases, are characterised by their low prevalence with majority of them are chronically debilitating and life threatening. Given the low prevalence and the widely dispersed but very small patient base for each disease, there may often be a disproportion in the availability of treatments and resources to manage patients, spur research and train experts. This is especially true in Southeast Asian countries that are currently in the process of implementing or revising their universal health coverage schemes. This paper aims to examine the status of rare disease management in Southeast Asian countries. It will serve as the basis for a more active discussion on how countries in the region can address an under-recognised rare disease burden and enhance national and regional capacities.

The study consists of literature reviews and key stakeholders interviews in six focus countries, including the Philippines, Singapore, Malaysia, Indonesia, Vietnam, and Thailand and five countries as best practice, comprising of France, Canada, Australia, Taiwan, and South Korea. Rare disease management initiatives across each country were examined based on the World Health Organization's framework for action in strengthening health systems.

The results suggest rare disease management remains challenging across Southeast Asia, as many of the focus countries face fundamental issues from basic healthcare systems to funding. Nonetheless, there are substantial improvement opportunities, including leveraging best practices from around the world and organising a multi-stakeholder and regional approach and strategy.

Southeast Asian countries have made significant progress in the management of rare disease, but there remain key areas for substantial development opportunities.
Formatted abstract
Background: Rare diseases, also referred to as orphan diseases, are characterised by their low prevalence with majority of them are chronically debilitating and life threatening. Given the low prevalence and the widely dispersed but very small patient base for each disease, there may often be a disproportion in the availability of treatments and resources to manage patients, spur research and train experts. This is especially true in Southeast Asian countries that are currently in the process of implementing or revising their universal health coverage schemes. This paper aims to examine the status of rare disease management in Southeast Asian countries. It will serve as the basis for a more active discussion on how countries in the region can address an under-recognised rare disease burden and enhance national and regional capacities.

Methods: The study consists of literature reviews and key stakeholders interviews in six focus countries, including the Philippines, Singapore, Malaysia, Indonesia, Vietnam, and Thailand and five countries as best practice, comprising of France, Canada, Australia, Taiwan, and South Korea. Rare disease management initiatives across each country were examined based on the World Health Organization's framework for action in strengthening health systems.

Results: The results suggest rare disease management remains challenging across Southeast Asia, as many of the focus countries face fundamental issues from basic healthcare systems to funding. Nonetheless, there are substantial improvement opportunities, including leveraging best practices from around the world and organising a multi-stakeholder and regional approach and strategy.

Conclusions: Southeast Asian countries have made significant progress in the management of rare disease, but there remain key areas for substantial development opportunities.
Keyword Orphan drug
Rare disease
Southeast Asia
Q-Index Code C1
Q-Index Status Provisional Code
Institutional Status UQ

Document type: Journal Article
Sub-type: Article (original research)
Collections: HERDC Pre-Audit
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