Table 1 Comparison of orphan drug regulation across the US, Europe, Japan, Korea, Taiwan and China

United States [43–46]

<200,000 (0.1% of population)

Food and Drug Administration (FDA)

7 years

50% tax credit for clinical studies

6 months

569

China [34]

Suggested prevalence 300,000–500,000

China Food and Drug Administration (CFDA)

N/A

N/A

N/A

N/A

European Union [27, 43, 47–49]

<215,000 (0.05% of population)

European Medicines Agency (EMA)

10 years

Yes, country dependent

5 months

116

Japan [16, 43]

<50,000, maximum 0.05% of population or no available treatment

Pharmaceuticals and Medical Devices Agency (PMDA)

Up to 10 years

Waived consultation fee ($20 K USD), up to 50% of development costs, 12% tax exemptions, 14$ corporate tax, ~25% reduction in review fees, portion of profits exceeding 100 M yen returned to government

10 months (vs 12 months for regular drugs)

203

Korea [16]

<20,000 or no available treatment, less than $5B won production costs/import

Korea Food and Drug Administration (KFDA)

6 years

50% subsidized application fee

6–9 months

184

Taiwan [16, 27, 43]

<2300 or 0.01% of population

Taiwan Food and Drug Administration (TFDA), Center for Drug Evaluation (CDE)

10 years

Financial subsidies not disclosed

6–10 months

77 drugs, 40 special nutrients