The clinical assessment of studies on orphan drugs in relation to the EMA’s authorization marketing decisions in Europe
The types of clinical trials and their methodological characteristics are correlated with the EMA's decisions. Randomization, double blinding, and active-controlled trials reduce the odds of ODs receiving EMA special statuses. In contrast, phase II/III trials, specific durations of treatment, and specific safety and efficacy follow-ups increased these odds.
Authors
- S Jakubowski (ORCID: https://orcid.org/0000-0001-6419-9686)
- Krzysztof Piotr Malinowski (ORCID: https://orcid.org/0000-0003-2189-0498)
- Paweł Kawalec
Institutions
- Jagiellonian University (PL)
Publication Details
- Journal
- Frontiers in Pharmacology
- Published
- 2025-03-31
- DOI
- https://doi.org/10.3389/fphar.2025.1558987
- Primary Topic
- Pharmaceutical Economics and Policy
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Uniwersytet Jagielloński Collegium Medicum