The call for demonstration projects aims to show the usability and capability of the innovative statistical methodologies for clinical trials in rare diseases, which have not been demonstrated on existing data for specific rare disease clinical trials yet. Necessary for a successful demonstration project is the collection of sufficient data from a rare disease clinical trial and related information to enable demonstration of the practicability, performance and opportunities of the innovative methodologies applied to these already acquired data. Trials are often performed with standard classical methodologies not specific for rare diseases resulting in a loss of power to show positive effects.
The aim of this demonstration call is not to reanalyse or question the original analysis of data from randomized controlled clinical trials, where efficacy was established, but rather to re-evaluate data that lacked efficiency because it was analysed with classical statistical methodology, which might be not feasible for trials in the rare disease context.
The call is now open for submission of 1-pager letters of interest. The deadline for submission is 15 of March 2020.
The content and procedures of the call described in this announcement may be subject to change and are not legally binding.
Please note that this call is open only for teams from institutions that are beneficiaries of the EJP RD project
Information, contact and documents following this link : https://www.ejprarediseases.org/index.php/clinical-trials-met-dem-projects-call/