Pro45 Patient-Reported Real-World Treatment Patterns for Charcot-Marie-Tooth Disease in the Uk and Us Assessed Using a Digital 'Bring Your Own Device Platform'

Abstract

Conclusions: Most medicines receiving an initial Orphan Designation in Europe maintain this at market authorization. For the ones that do not, this is due to withdrawal of the orphan designation by the sponsor, caused by the disease prevalence and the significant benefit criteria no longer being met. Although orphan drug legislation has incentivized drug development for rare diseases in Europe, most of these drugs received an earlier FDA approval and overcoming the frequently greater reimbursement/payer hurdle in Europe can further add to this access discrepancy.