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Innovative methodology for small populations research

Objective

The HEALTH.2013.4.2-3 call identifies a need for new or improved statistical methodology for clinical trials for the efficient assessment of safety and/or efficacy of treatment for small population groups. This project brings together international experts in innovative clinical trial design methodology in these specific areas along with key stakeholders including regulatory authorities, industry, clinicians and patient groups to address this need. Our aim is the development novel methodology for the design and analysis of clinical trials in small populations. We will focus on four specific areas where we believe there are particular challenges: (i) early phase dose-finding studies in small populations, (ii) decision-theoretic methods for clinical trials in small populations, (iii) confirmatory trials in small populations and personalised medicines, (iv) use of evidence synthesis in the planning and interpretation of clinical trials in small populations and rare diseases.

We will build on recent research advances, of our own and of others in this area. In the rare disease setting, we will focus on Bayesian and decision-theoretic methods that formally enable comparison of the gain in information with the cost, both in economic and opportunity terms, of clinical experimentation, and assess how information from outside the trial can formally be incorporated in the design and decision-making processes. In the personalised medicine setting, we will develop methods that allow evaluation of efficacy in a number of sub-populations simultaneously in a confirmatory clinical trial without any reduction in scientific or statistical rigour.

Call for proposal

FP7-HEALTH-2013-INNOVATION-1
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Coordinator

THE UNIVERSITY OF WARWICK
Address
Kirby Corner Road - University House
CV4 8UW Coventry
United Kingdom

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Nigel Stallard (Prof.)
EU contribution
€ 814 478,86

Participants (7)

UNIVERSITAETSMEDIZIN GOETTINGEN - GEORG-AUGUST-UNIVERSITAET GOETTINGEN - STIFTUNG OEFFENTLICHEN RECHTS
Germany
EU contribution
€ 385 732,80
Address
Robert-koch-strasse 40
37075 Goettingen

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Christiane Hennecke (Mrs.)
MEDIZINISCHE UNIVERSITAET WIEN
Austria
EU contribution
€ 423 809,20
Address
Spitalgasse 23
1090 Wien

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Wolfgang Dorda (Prof.)
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
EU contribution
€ 466 608,80
Address
Rue De Tolbiac 101
75654 Paris

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Activity type
Research Organisations
Administrative Contact
Isabelle Verdier (Mrs.)
BUNDESINSTITUT FUR ARZNEIMITTEL UND MEDIZINPRODUKTE
Germany
EU contribution
€ 48 647
Address
Kurt Georg Kiesinger Allee 3
53175 Bonn

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Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Administrative Contact
Sandra Ludwig-Merseburg
STOCKHOLMS UNIVERSITET
Sweden
EU contribution
€ 50 680
Address
Universitetsvagen 10
10691 Stockholm

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Ulla Jungmarker (Mrs.)
IQVIA LTD.
United Kingdom
EU contribution
€ 24 026
Address
Brook Drive, Green Park
RG2 6UU Reading, Berkshire

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Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Alex Dmitrienko (Dr.)
Clinical Trials Consulting & Training Limited
United Kingdom
EU contribution
€ 54 521,95
Address
Portway 53
MK18 3PL North Marston

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Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Simon Day (Dr.)