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.
An analysis of the effect of drug pricing provisions in the Build Back Better Act on pharmaceutical innovation
Charles River Associates has previously assessed the implications of proposals to implement international reference pricing in the US and found they would...