Orphan drug status benefit

ISPOR 2021 Does Orphan Drug Status Confer Any Benefits for Products Undergoing HTA Appraisal By NICE in England?


Orphan drug status is granted for medicines proposing to target rare diseases with a high level of unmet need, offering incentives for such products. Health Technology Assessment (HTA) agencies will often consider orphan status during their assessments. The purpose of this study is to compare the assessments of orphan and non-orphan drugs by the National Institute of Health and Care Excellence (NICE) since 2018 and determine if orphan status confers any benefit.

Stay in the know, subscribe to our newsletter

Be the first to receive exclusive content on the latest from the pharmaceutical and market access sector.