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Advances in Small Trials dEsign for Regulatory Innovation and eXcellence

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."
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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)

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MEDIZINISCHE HOCHSCHULE HANNOVER

Germany

EU Contribution

€ 525 200

MEDIZINISCHE UNIVERSITAET WIEN

Austria

EU Contribution

€ 515 401

Academisch Medisch Centrum bij de Universiteit van Amsterdam

Netherlands

EU Contribution

€ 466 640

UNIVERSITAT AUTONOMA DE BARCELONA

Spain

EU Contribution

€ 512 442

STATISTICIANS IN THE PHARMACEUTICALINDUSTRY LIMITED LBG

United Kingdom

EU Contribution

€ 68 150

VERENIGING SAMENWERKENDE OUDER- EN PATIENTENORGANISATIES

Netherlands

EU Contribution

€ 161 990

Project information

Grant agreement ID: 603160

Status

Closed project

  • Start date

    1 October 2013

  • End date

    30 September 2017

Funded under:

FP7-HEALTH

  • Overall budget:

    € 4 141 786

  • EU contribution

    € 2 999 881

Coordinated by:

UNIVERSITAIR MEDISCH CENTRUM UTRECHT

Netherlands

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