IDEAL: international project to explore new methods for design and analysis of clinical studies in rare diseases
A recently launched research project named IDEAL (Integrated Design and Analysis of small population group trials) will address the urgent need to develop new statistical approaches for efficient evaluation of clinical trials for rare diseases. This project aims to explore new methods for the design and analysis of clinical studies and to formulate an effective strategy for assessing clinical trials for rare diseases. It will collate all available sources of information that will help optimise the process of finding an appropriate statistical method.
An international research team under the supervision of Professor Ralf-Dieter Hilgers of the Uniklinik RWTH Aachen is supported by the EU Projektmanagement Office der RWTH Aachen. The IDEAL project consists of an advisory board of international experts with diverse professional backgrounds, that represents patient interests, pharmaceutical industry as well as regulatory delegates who will collaborate to develop new designs and sophisticated analysis methods.
The research has been funded with €3 million by the 7th Framework Programme of the European Union. The work packages focus on “the assessment of randomization, the extrapolation of dose-response information, the study of adaptive trial designs, the development of optimal experimental designs in mixed models, as well as pharmacokinetic and individualized designs, simulation of clinical studies, the involvement and identification of genetic factors, decision-theoretic considerations, as well as the evaluation of biomarkers”. Further information on the project
