Skip to main content

Integrated DEsign and AnaLysis of small population group trials

Objective

There exist more than 7000 rare diseases worldwide and the European Society of Paediatric Oncology stated that 75% of rare diseases affect children and 30% of rare disease patients die before the age of five. Usual statistical methods for proving efficacy and safety of therapies fail to provide cost-efficient and reliable results in small populations. There is a pressing need to integrate a broad range of innovative methodologies improving clinical trials in the setting of small sample population groups (SPG). The objective of this research is to produce methods of general applicability irrespective of indication by Integrated DEsign and AnaLysis of clinical trials in SPG (IDEAL) through a multidisciplinary closely collaborating consortium of researchers from European universities, research institutes and industry. The consortium will work in 10 WPs, focussed on assessment of randomization procedures, extrapolating dose-response information, investigation of adaptive designs, optimal designs in mixed models, pharmacogenetic designs, simulation of clinical trials, genetic factors influencing the response, decision analysis and biomarker surrogate endpoints as well as WPs on project management and dissemination of results. Relevant stakeholder concerns (patient needs, regulatory issues, reimbursement, clinical feasibility) will be monitored by a Clinical Scientific Advisory Board. Because of its integrative structure, this research program extends previous approaches, which focus on a certain methodology only. In its totality, the WPs constitute a logically coherent set of methodologies that is of sufficient breadth to tackle these important, multidisciplinary challenges. By combining, enhancing and developing different statistical methodologies and assessment methods, this research program will impact the scientific discussion in promoting efficient statistical methodology for clinical trials in SPG, also in view of existing regulatory guidance in the EU.

Field of science

  • /medical and health sciences/clinical medicine/oncology

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

UNIVERSITAETSKLINIKUM AACHEN
Address
Pauwelsstrasse 30
52074 Aachen
Germany
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 595 400
Administrative Contact
Volker Legewie (Mr.)

Participants (9)

RHEINISCH-WESTFAELISCHE TECHNISCHE HOCHSCHULE AACHEN
Germany
EU contribution
€ 111 040
Address
Templergraben 55
52062 Aachen
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Ernst Schmachtenberg (Prof.)
RUHR-UNIVERSITAET BOCHUM
Germany
EU contribution
€ 294 400
Address
Universitaetsstrasse 150
44801 Bochum
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Martina Stein (Mrs.)
MEDIZINISCHE UNIVERSITAET WIEN
Austria
EU contribution
€ 466 160
Address
Spitalgasse 23
1090 Wien
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
€ 262 720
Address
Rue De Tolbiac 101
75654 Paris
Activity type
Research Organisations
Administrative Contact
Hamid Boumedine (Mr.)
LUXEMBOURG INSTITUTE OF HEALTH
Luxembourg
EU contribution
€ 199 969
Address
Val Fleuri 84
1526 Luxembourg
Activity type
Research Organisations
Administrative Contact
Thomas Lentz (Mr.)
UPPSALA UNIVERSITET
Sweden
EU contribution
€ 323 424
Address
Von Kraemers Alle 4
751 05 Uppsala
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Elisabeth Jonsson (Ms.)
POLITECHNIKA WROCLAWSKA
Poland
EU contribution
€ 197 370
Address
Wybrzeze Wyspianskiego 27
50-370 Wroclaw
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Malgorzata Bogdan (Dr.)
CHALMERS TEKNISKA HOEGSKOLA AB
Sweden
EU contribution
€ 324 416
Address
-
41296 Goeteborg
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Helena Rafstedt (Ms.)
UNIVERSITEIT HASSELT
Belgium
EU contribution
€ 183 550
Address
Martelarenlaan 42
3500 Hasselt
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Hilde Pirard (Mrs.)