Table 1 National initiatives to support rare diseases and access to orphan drugs
| Â | Indicator | Philippines | Singapore | Malaysia | Indonesia | Vietnam | Thailand |
|---|---|---|---|---|---|---|---|
Health system | GDP per capita (USD) | 2,588 | 54,007 | 10,440 | 3,551 | 1,755 | 5,480 |
Health expenditure per capita (USD) | 119 | 2,426 | 410 | 108 | 103 | 215 | |
Total health expenditure (% of GDP) | 4.6 | 4.7 | 3.9 | 3.0 | 6.6 | 3.9 | |
Health coverage (%) [37] | 76 | 100 | 100 | 48 | 55 | 98 | |
Out of pocket health expenditure (% of total expenditure on health) | 52.0 | 58.6 | 35.6 | 45.3 | 48.8 | 13.1 | |
Life expectancy at birth (years) [40] | 69 | 82 | 75 | 70 | 73 | 75 | |
Mortality rate, infant (per 1,000 live births) [40] | 22 | 1.8 | 7 | 32 | 16 | 11 | |
Governance | Presence of national strategy | Yes | No | In progress | No | No | No |
Definition of rare disease | Yes | Yes | Yes | No | No | No | |
Comprehensive rare disease legislation | Yes (2015) | No | No | No | No | No | |
Orphan drug act/ legislation | Pending approval | Yes (1991) | No | No | No | No | |
Awareness | Patient support/advocacy groups | PSOD | RDSS | MLDS, MMA, MRDS | No | NPH RD club | Genetic LSD Foundation |
Patient support group activity | Yes (since 2010) | Yes (since 2013) | Yes (since 2010) | No | Yes (since 2013) | Yes (since 2011) | |
Media attention (Based on Factiva search from 2009–2015) | 72 articles | 48 articles | 33 articles | 13 articles | 26 articles | 12 articles | |
Clinical expertise | Presence of patient registries | Largely institution specific | No | No | Planned, LSD diseases | ||
Presence of centre of expertise | Yes (1) | Yes (2) | Yes (3) | No | Yes (1) | Yes (2) | |
Presence of national guidelines for treatment | No | No | No | No | No | No | |
Professional societies to support specialist | No | No | Limited | No | No | No | |
Funding | Government funding for rare disease treatments | Limited | Limited | Limited | No | No | Limited |
Government funding for rare disease research | No | Yes | No | No | No | No | |
Non-government access to rare disease treatment | Some charitable and industry funds | Some charitable and industry funds | Some charitable and industry funds, employer benefits | No | No | Some charitable and industry funds | |
New born screening | National neonatal screening programs | Yes | Yes | Yes | No | Yes | Yes |
Percentage of new born screened | 28Â % | 100Â % | >95Â % | <1Â % | 31Â % | >97Â % | |
Source of payment for the new born screening | OOP/insurance | OOP (40Â %) | Gov./ OOP | OOP | Government | ||