Advances in Small Trials dEsign for Regulatory Innovation and eXcellence (ASTERIX)
The ASTERIX project develops statistical methods for cost efficient clinical trials for small populations. These methods have a particular relevance in the development of new therapies for rare diseases.
The possibilities to investigate new therapeutic options is very limited because of small patient groups and often do not lead to a clear result on the efficacy of new therapies. More than 30 million people in the EU are affected by one of the more than 5000 known, rare diseases and the developed methods shall support the evidence based development of new therapies.
Key methodological approaches include: leveraging prior information and the availability of multiple endpoints, enabling adaptive designs and sequential meta-analysis. The relevance of these approaches will be investigated in the context of the assessment of medicines for rare diseases by regulators in the course of marketing authorisations.
ASTERIX systematically includes patients and the patient perspective into its research in a methodological sound manner. Pre-clinical data, historical data on disease characteristics, and benefit and risk as perceived by patients will systematically inform and weigh relevant endpoint measures.
MedUni Vienna Researchers
The Medical University of Vienna is project partner under the leadership of Martin Posch
Scientific staff at the Medical University of Vienna:
Sonja Zehetmayer (Univ. Ass., maternity leave)
Robin Ristl (PostDoc)
Susanne Urach (PhD Student)
Johann Hainfellner (Ao.-Univ. Prof.)
Harald Heinzl (Univ. Prof.)
Bernd Jilma (Ao. Univ.-Prof.)
Project coordinator: Kit Roes, Director Quality and Patient Safety (UMC Utrecht) and Professor of Biostatistics (Julius Center UMC Utrecht)
University Medical Center Utrecht (NL)
Medizinische Hochschule Hannover (D)
MedUni Vienna (AUT)
The Academic Medical Center (NL)
Universitat Autònoma de Barcelona (ES)
Statisticians in the Pharmaceutical Industry/ European Federation of Statisticians in the Pharmaceutical Industry (UK)
VSOP (NL) / EGAN (BE)
7 project partners
Seventh Framework programme,
New methodologies for clinical trials for small population groups
|Project duration||01/10/2013 – 01/10/2017|
|Funding volume, Medical University of Vienna||672,259.20 €|
|Funding volume, total||4,141,786.00 €|