Evaluation of orphan maintained biological medicinal products in the European Union between 2018 to 2023: a regulatory perspective
The Orphan Regulation is essential in advancing treatments for rare diseases, fostering innovation while addressing unmet medical needs. Nonetheless, the insufficient return on investment criterion remains underused, whereas refining major contribution to patient care guidelines and incorporating real-world evidence may enhance regulatory evaluations.
Authors
- Laura Marchetti (ORCID: https://orcid.org/0000-0002-2110-9481)
- Maria Letizia Trincavelli (ORCID: https://orcid.org/0000-0001-8124-977X)
- Anna Maria Piras (ORCID: https://orcid.org/0000-0002-4082-6400)
- T. Fraisse (ORCID: https://orcid.org/0000-0002-2388-1838)
- Paolo Caferra (ORCID: https://orcid.org/0009-0008-4379-5524)
Institutions
- University of Pisa (IT)
- Université de Montpellier (FR)
- Centre Hospitalier Universitaire de Montpellier (FR)
- Sanofi (Netherlands) (NL)
Publication Details
- Journal
- Expert Opinion on Biological Therapy
- Published
- 2024-10-26
- DOI
- https://doi.org/10.1080/14712598.2024.2422360
- Citations
- 1
- Primary Topic
- CRISPR and Genetic Engineering
- Type
- article
- Field-Weighted Citation Impact
- 0.23