Table 1.

Rare diseases regulatory environment 2010–2020

	USA	European Union	Canada	Japan	Australia
Orphan drug legislation	Yes	Yes	No	Yes	Yes
Other rare disease legislation	•Creating Hope Act (RPD PRV)-2012a •Twenty-First Century Cures Act (Cancer Moonshot)-2016 •RACE Act-2017
Rare disease guidances/guidelines	Yes	Yes	No	No	Use EMA
Economic incentives	•Research grants •Tax credits •Reduced fees •Market exclusivity •Vouchers	•Market exclusivity •Reduced fees •Incentives at member state level	•Tax credits •Waived fees	•Market exclusivity •Drug price premium	•Reduced fees
Treatment access programs	Yes	Yes	Yes	Yes	Yes
National rare disease strategy	No	No	Under discussion	No	Plan published 2018

^aRPD PRV = rare pediatric disease priority review program. The RPD PRV program was reauthorized in 2016 and 2020