Use of real-world data for the development and the follow-up of drugs in rare diseases. The example of immune thrombocytopenia and autoimmune hemolytic anemia

2025/05/01 by Guillaume Moulis, Yoann Zadro, Agnès Sommet +2 · 1 voice
Medicine · #Platelet Disorders and Treatments #Blood groups and transfusion #Chronic Lymphocytic Leukemia Research

paper · doi:10.1016/j.revmed.2025.03.424

openalex publication_date 2025/05/01 · openalex created_date 2025/10/10 · openalex updated_date 2026/07/16

Abstract

This review describes the role of real-world data (RWD) at each step of drug development for rare diseases like immune thrombocytopenia (ITP) and autoimmune hemolytic anemia (AIHA). We also describe the main sources of RWD in rare diseases and how the generation of real-world evidence (RWE) is crucial for decisions of regulatory health agencies regarding drugs for rare disease. Cette mise au point décrit l’intérêt des « données de vie réelle » tout au long du développement des médicaments pour les maladies rares comme le purpura thrombopénique immunologique (PTI) et l’anémie hémolytique auto-immune (AHAI). Nous décrivons aussi les principales sources de données de vie réelle dans les maladies rares et comment la génération de « preuves de vraie vie » est désormais nécessaire aux agences de régulation sanitaires pour évaluer l’efficacité et la sécurité des médicaments ainsi que leur place dans la stratégie thérapeutique des maladies rares.

Citations

• New insights into the epidemiology of immune thrombocytopenia in adult patients: Impact for clinical practice
• Target Trial Emulation
• Performing pharmacoepidemiological studies using the French health insurance data warehouse (SNDS): How to translate guidelines into practice
• Use of the French National Health Data System (SNDS) in pharmacoepidemiology: A systematic review in its maturation phase

Discussions

• Use of real-world data for the development and the follow-up of drugs in rare diseases. The example of immune thrombocytopenia and autoimmune hemolytic anemia 💉🩸💊 doi.org/10.1016/j.re... [bsky, 1 points, 0 comments]

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