Although orphan drug (OD) development is challenging, incentives have enabled a
diversity to achieve EMA, typically from manufacturers of a single OD with a single
indication. Rare oncology (RO) approval is frequently supported by a single, large,
pivotal RCT often open-labelled with active control. In contrast, rare disease (RD)
have multiple, smaller studies often double-blinded with placebo. Conditional EMA
is more common for RO whereas exceptional EMA is more common for RD.
Conditional EMA often occurred for RO with ongoing phase-three pivotal studies.
In the absence of ongoing studies for RD, exceptional EMA was often granted with
the requirement to establish a registry. This review demonstrates a flexible, diverse
approach to the evidence and regulatory approval of ODs, with stark differences
between RO and RD. Subsequent assessment of the evidence for access should
adopt a bespoke approach with a framework beyond standard quality criteria.
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