Participant-funded clinical trials on rare diseases
نویسندگان
چکیده
منابع مشابه
How should individual participant data (IPD) from publicly funded clinical trials be shared?
BACKGROUND Individual participant data (IPD) from completed clinical trials should be responsibly shared to support efficient clinical research, generate new knowledge and bring benefit to patients. The Medical Research Council (MRC) Hubs for Trials Methodology Research (HTMR) has developed guidance to facilitate the sharing of IPD from publicly funded clinical trials. METHODS Development of ...
متن کاملGood practice principles for sharing individual participant data from publicly funded clinical trials
Sharing Individual Participant Data (IPD) from completed clinical trials offers numerous well recognised advantages that can advance clinical research and benefit patients. The clinical trial community, including publicly funded trials, has a duty to facilitate this process. In our recent survey, publicly funded clinical trials units (CTUs) from the UKCRC registered CTUs network were supportive...
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Background The evaluation of treatments for rare diseases presents a number of challenges for trial practitioners, regulators and policy makers. Small sample sizes mean that ‘standard’ approaches to trial design and analysis may not be appropriate and alternatives such as Bayesian trial designs have been recommended [Lilford et al 1995]. However, little is known about the design and analysis ap...
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Development of treatments for rare diseases is challenging due to the limited number of patients available for participation. Learning about treatment effectiveness with a view to treat patients in the larger outside population, as in the traditional fixed randomised design, may not be a plausible goal. An alternative goal is to treat the patients within the trial as effectively as possible. Us...
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Preparation of this manuscript was supported in part by grants from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (AR/AI44724, AR47298), the National Institute of Child Health and Human Development/National Center for Medical Rehabilitation Research (HD33989), and the Fetzer Institute. Portions of this paper were summarized at the second meeting of the Initiative on ...
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ژورنال
عنوان ژورنال: Anales de Pediatría (English Edition)
سال: 2020
ISSN: 2341-2879
DOI: 10.1016/j.anpede.2020.03.005