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Drug Discovery Today|September 1, 2025
Addressing global regulatory challenges in rare disease drug developmentEnrico Costa, Vijayalekshmi Ajith, Ahmed F Al Khaldi, et al.
Gene Therapy|March 14, 2024
Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designationMaria Elisabeth Kalland, Tomas Pose-Boirazian, Gloria Maria Palomo, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 3, 2024
Navigating the orphan medicinal product designation: Evidence requirements for gene therapies in EuropeGloria M Palomo, Tomas Pose-Boirazian, Frauke Naumann-Winter, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 5, 2023
The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal ProductsGloria M Palomo, Tomas Pose-Boirazian, Frauke Naumann-Winter, et al.
Frontiers in Medicine|September 13, 2021
Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit-The EU Regulator's PerspectiveMaria E Sheean, Frauke Naumann-Winter, Giuseppe Capovilla, et al.
Drug Discovery Today|October 9, 2017
Nonclinical data supporting orphan medicinal product designations: lessons from rare neurological conditionsMaria E Sheean, Violeta Stoyanova-Beninska, Giuseppe Capovilla, et al.
Drug Discovery Today|April 29, 2020
Assessment of significant benefit for orphan medicinal products by European regulators may support subsequent relative effectiveness assessments by health technology assessment organizationsRick A Vreman, Angela S de Ruijter, Anna Zawada, et al.
Drug Discovery Today|November 10, 2019
Nonclinical data supporting orphan medicinal product designations in the area of rare infectious diseasesMaria E Sheean, Eva Malikova, Dinah Duarte, et al.
Cancer Management and Research|March 6, 2025
Drug Repurposing in Pancreatic Cancer: A Multi-Stakeholder Perspective to Improve Treatment Options for Pancreatic Cancer PatientsEvelyn Hewitt, Gauthier Bouche, Alexandre Costa Alencar, et al.
Clinical Pharmacology and Therapeutics|July 4, 2024
Real-World Evidence to Support EU Regulatory Decision Making-Results From a Pilot of Regulatory Use CasesStefanie Prilla, Sophie Groeneveld, Alexandra Pacurariu, et al.
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