"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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