Reforming EU and national orphan drug regulations to improve outcomes for patients with rare diseases

BMJ. 2023 May 9:381:e072796. doi: 10.1136/bmj-2022-072796.

Authors

Philip Kranz¹, Natalie McGauran², Rita Banzi³, Can Ünal², Fabian Lotz², Thomas Kaiser²

Affiliations

¹ Institute for Quality and Efficiency in Health Care, Cologne, Germany philip.kranz@iqwig.de.
² Institute for Quality and Efficiency in Health Care, Cologne, Germany.
³ Mario Negri Institute for Pharmacological Research, IRCCS, Milan, Italy.

PMID: 37160303
DOI: 10.1136/bmj-2022-072796

No abstract available

MeSH terms

Drug and Narcotic Control*
Humans
Orphan Drug Production
Patients
Rare Diseases* / drug therapy