Unique characteristics of regulatory approval and pivotal studies of orphan anticancer drugs in Japan

In conclusion, we demonstrated t hat orphan anticancer drugs in Japan have unique characteristics compared to non-orphan drugs: shorter regulatory review and pivotal studies frequently using phase II, non-randomized, or non-controlled designs and response rate as a primary endpoint, with fewer patients.
Source: Investigational New Drugs - Category: Drugs & Pharmacology Source Type: research