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.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
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Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Topic(s)
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
FP7-HEALTH-2013-INNOVATION-1
See other projects for this call
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Coordinator
52074 Aachen
Germany
The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.