Access to medicines for rare diseases: A European regulatory roadmap for academia.

ABSTRACT: Background: Novel or repurposed medicines for rare diseases often emerge from fundamental research or empirical findings in academia. However, researchers may be insufficiently aware of the possibilities and requirements to bring novel medicinal treatment options to the patient. This paper aims to provide an easily applicable, comprehensive roadmap designed for academic researchers to make medicines for rare diseases available for patients by addressing the relevant regulatory frameworks, including marketing authorization and alternative routes. Methods: Key points of the regulatory chapters "Placing on the Market" and "Scope" of Directive 2001/83/EC relating to medicinal products for human use were summarized. Provisions in EU directives regarding blood products, radiopharma

SUBMITTER: Rosenberg N 

PROVIDER: S-EPMC10012277 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature