Design and analysis of clinical trials for small rare disease populations: J Rare Dis Res Treat

R-D Hilgers, Franz König, Geert Molenberghs, Stephen Senn

Research output: Contribution to journalArticleResearchpeer-review

Abstract

The mostly unmet need as well as the pressure to show efficacy of new therapies to treat rare diseases contrasts with the limited possibility to use traditional statistical methods to design and analyse clinical trials in this setting. Within this paper, we will refer to the current state of design and analysis methods, as well as practical conditions to be considered when conducting a clinical trial for rare diseases. We will embed the research of the IDeAl project within this setting and give some first recommendations to improve the methodology for clinical trials in rare diseases.
Original languageEnglish
Pages (from-to)53-60
Number of pages8
JournalJournal of Rare Diseases Research & Treatment
Volume1
Issue number3
DOIs
Publication statusPublished - 23 Nov 2016

Keywords

  • Clinical trials Rare disease populations IDeAl consortium

Fingerprint

Dive into the research topics of 'Design and analysis of clinical trials for small rare disease populations: J Rare Dis Res Treat'. Together they form a unique fingerprint.

Cite this