Dose-Finding Studies Among Orphan Drugs Approved in the EU: A Retrospective Analysis.

ABSTRACT: In the development process for new drugs, dose-finding studies are of major importance. Absence of these studies may lead to failed phase 3 trials and delayed marketing authorization. In our study we investigated to what extent dose-finding studies are performed in the case of orphan drugs for metabolic and oncologic indications. We identified all orphan drugs that were authorized until August 1, 2017. European Public Assessment Reports were used to extract the final dose used in the summary of product characteristics, involvement of healthy volunteers, study type, end points used, number of patients, number of doses, studies in special populations, and dose used for phase 3 studies. Each drug was checked for major objections and dose changes postmarketing. We included 49 orphan drugs, of

SUBMITTER: Schuller Y 

PROVIDER: S-EPMC6585723 | biostudies-literature | 2019 Feb

REPOSITORIES: biostudies-literature