title: State of rare disease management in Southeast Asia.
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.
authors.affiliation: Social and Administrative Pharmacy, School of Pharmaceutical Sciences, Universiti Sains Malaysia, 11800, Minden, Penang, Malaysia. aakmal@usm.my.
authors.forename: Asrul Akmal
authors.identifier: 0000-0002-5629-9270
authors.initials: AA
authors.lastname: Shafie
authors.affiliation: School of Pharmacy, Monash University Malaysia, Jalan Lagoon Selatan, 47500, Bandar Sunway, Selangor, Malaysia.|Center of Pharmaceutical Outcomes Research, Faculty of Pharmaceutical Sciences, Naresuan University, Phitsanulok, Thailand.|School of Pharmacy, University of Wisconsin, Madison, WI, USA.|School of Population Health, University of Queensland, Brisbane, QLD, Australia.
authors.forename: Nathorn
authors.initials: N
authors.lastname: Chaiyakunapruk
authors.affiliation: Social and Administrative Pharmacy, School of Pharmaceutical Sciences, Universiti Sains Malaysia, 11800, Minden, Penang, Malaysia.
authors.forename: Azuwana
authors.initials: A
authors.lastname: Supian
authors.affiliation: Health & Life Sciences, Asia-Pacific, Oliver Wyman, Singapore, Singapore.
authors.forename: Jeremy
authors.initials: J
authors.lastname: Lim
authors.affiliation: Health & Life Sciences, Asia-Pacific, Oliver Wyman, Singapore, Singapore.
authors.forename: Matt
authors.initials: M
authors.lastname: Zafra
authors.affiliation: Social and Administrative Pharmacy, School of Pharmaceutical Sciences, Universiti Sains Malaysia, 11800, Minden, Penang, Malaysia.
authors.forename: Mohamed Azmi Ahmad
authors.initials: MA
authors.lastname: Hassali
mesh_terms: D001208:Asia; D001210:Asia, Southeastern / Q000453:epidemiology; D002170:Canada; D019468:Disease Management; D005602:France; D006801:Humans; D007214:Indonesia; D008296:Malaysia; D009965:Orphan Drug Production; D010679:Philippines; D035583:Rare Diseases / Q000188:drug therapy / Q000453:epidemiology*; D056910:Republic of Korea; D012846:Singapore; D013624:Taiwan; D013785:Thailand; D014744:Vietnam
keywords: Orphan drug; Rare disease; Southeast Asia