Abstract
AbstractBackgroundDrug development programmes in rare diseases have many challenges, some of which differ from those facing researchers working on common diseases, like the scarcity of patients.Over the past years, research and regulatory initiatives, as well as resources have been implemented to expedite drug development for rare diseases. Nevertheless, these tools have been developed in the context of different projects and with diverse aims. Therefore, they have not yet been structured to encompass the conduct of clinical trials as a whole. To address this issue, the EJP RD (European Joint Program for Rare Diseases) has developed the Rare Diseases Clinical Trial Toolbox.PurposeThis toolbox collates the accumulated knowledge, experience, and resources (collectively termed ‘tools’) generated by projects, research infrastructures and/or other organizations into a structured, practical and guided instrument to help clinical trialists and trial managers understand the regulations and requirements for conducting trials, with a special focus on investigator-initiated trials for rare diseases.MethodsThe toolbox is organized into five domains: research question, plan, execution, analysis, and end of trial. Each domain describes one or several activities to be considered in this step and indicate at what stage of the trial pathway these activities should take place, regardless of the therapeutic area. Each activity is further linked to specific resources (the tools) that are relevant for those activities. Associated resources are in the public domain developed in the context of research projects or by relevant clinical research stakeholders. Selected tools must be of fundamental importance to clinical trials and be applicable torare diseases clinical research. Rare-diseases specific resources are highlighted as such and include those specially relevant to paediatric clinical research, considering than one half of rare disorders affect children and some 60% of designated orphan medicines are intended for paediatric use.ResultsThe current version of the Toolbox includes 111 resources tagged as relevant for any of the 18 activities within the clinical trial outline. Overall, 75 % of all resources are relevant to any clinical trial while 25 % are tagged as “rare disease specific”.ConclusionAccess to public resources relevant to the development of clinical trials for rare diseases is sometimes challenged by limited awareness and/or the absence of an adequate framework that enables their findability. This Toolbox aims at building a framework supporting the optimal use of existing tools.
Publisher
Cold Spring Harbor Laboratory
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