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Advances in Small Trials dEsign for Regulatory Innovation and eXcellence

Objective

"ASTERIX will contribute towards the expected impact listed in the work program by delivering validated innovative statistical design methodologies for cost efficient clinical trials deriving reliable results from trials in small population groups, especially focusing on rare diseases. It directly addresses the rights of the estimated 30 million European patients suffering from rare diseases to the same quality or treatment as other patients. By choosing an integrated approach leveraging statistical methods, smartly combining observational and clinical data as well as improved sequential and adaptive approaches ASTERIX will improve statistical power of clinical trial design in small populations. ASTERIX will systematically include patients and the patient perspective into its research, but moreover develop the approach to involve patients in clinical trial design 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. Thus the clinical value of trial results will be enhanced considerably. Key methodological innovations include: new standards of evidence that take into account the rare prevalence of disease, leveraging prior information and the availability of multiple endpoints, enabling adaptive designs and sequential meta-analysis using multiple endpoints and providing a blue print to pro-actively share information on trials in the planning stage. The latter will ensure that each new trial in an orphan disease provides the optimum additional knowledge for patient treatment. Relevance and clinical value of the newly designed methodological approaches are validated twofold. Firstly, by cross testing with available clinical trial data of a broad range of highly relevant rare diseases and secondly, by assessing the methods against current and improved regulatory approval strategies."

Call for proposal

FP7-HEALTH-2013-INNOVATION-1
See other projects for this call

Funding Scheme

CP-FP - Small or medium-scale focused research project

Coordinator

UNIVERSITAIR MEDISCH CENTRUM UTRECHT
Address
Heidelberglaan 100
3584 CX Utrecht
Netherlands
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 750 058
Administrative Contact
Caroline Van Baal (Dr.)

Participants (6)

MEDIZINISCHE HOCHSCHULE HANNOVER
Germany
EU contribution
€ 525 200
Address
Carl-neuberg-strasse 1
30625 Hannover
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Frank Dittrich (Mr.)
MEDIZINISCHE UNIVERSITAET WIEN
Austria
EU contribution
€ 515 401
Address
Spitalgasse 23
1090 Wien
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Wolfgang Dorda (Prof.)
ACADEMISCH MEDISCH CENTRUM BIJ DE UNIVERSITEIT VAN AMSTERDAM
Netherlands
EU contribution
€ 466 640
Address
Meibergdreef 15
1105AZ Amsterdam
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Sylvia Georgiev (Ms.)
UNIVERSIDAD AUTONOMA DE BARCELONA
Spain
EU contribution
€ 512 442
Address
Calle Campus Universitario Sn Cerdanyola V
08290 Cerdanyola Del Valles
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Francisca Rabadán Sotos (Ms.)
STATISTICIANS IN THE PHARMACEUTICALINDUSTRY LIMITED LBG
United Kingdom
EU contribution
€ 68 150
Address
Durford Mill
GU31 5AZ Petersfield
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Alun Bedding (Mr.)
VERENIGING SAMENWERKENDE OUDER- EN PATIENTENORGANISATIES
Netherlands
EU contribution
€ 161 990
Address
Vredehofstraat 31
3761HA Soest
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Administrative Contact
Cor Oosterwijk (Mr.)