Albane Degrassat-Théas

Reimbursement status, access delays and prices of orphan drugs in France: a population-stratified analysis of indications approved between 2021 and 2024

Abstract Background More and more treatments are available for patients with rare diseases. However, a marketing authorization (MA) for a new treatment does not mean access in France for all patients. Moreover, while rare diseases affect children mostly, paradoxically it seems th...