The RUDY study platform - a novel approach to patient driven research in rare musculoskeletal diseases.
Research into rare diseases is becoming more common, with recognition of the significant diagnostic and therapeutic care gaps. Registries are considered a key research methodology to address rare diseases. This report describes the structure of the Rare UK Diseases Study (RUDY) platform that aims to improve research processes and address many of the challenges of carrying out rare musculoskeletal disease research. RUDY is an internet-based platform with online registration, initial verbal consent, online capture of patient reported outcome measures and events within a dynamic consent framework. The database structure, security and governance framework are described.



There have been 380 participants recruited into RUDY with completed questionnaire rates in excess of 50 %. There has been one withdrawal and two participants have amended their consent options.



The strengths of RUDY include low burden for the clinical team, low research administration costs with high participant recruitment and ease of data collection and access. This platform has the potential to be used as the model for other rare diseases globally.
Oxford NIHR Musculoskeletal Biomedcial Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK. kassim.javaid@ndorms.ox.ac.uk.|The Botnar Research Centre, NIHR Oxford Musculoskeletal BRU, NDORMS, University of Oxford, Oxford, OX3 7HE, UK. kassim.javaid@ndorms.ox.ac.uk.
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Javaid
Oxford NIHR Musculoskeletal Biomedcial Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
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Forestier-Zhang
Oxford NIHR Musculoskeletal Biomedcial Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
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Watts
Oxford NIHR Musculoskeletal Biomedcial Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
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Turner
Oxford NIHR Musculoskeletal Biomedcial Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
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Ponte
Nuffield Department of Population Health, University of Oxford, Oxford, UK.
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Teare
Oxford NIHR Musculoskeletal Biomedcial Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
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Gray
Oxford NIHR Musculoskeletal Biomedcial Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
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Gray
Oxford NIHR Musculoskeletal Biomedcial Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
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Popert
Oxford NIHR Musculoskeletal Biomedcial Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
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Hogg
Oxford NIHR Musculoskeletal Biomedcial Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
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Barrett
Oxford NIHR Musculoskeletal Biomedcial Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
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Pinedo-Villanueva
Oxford NIHR Musculoskeletal Biomedcial Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK.|MRC Lifecourse Epidemiology Unit, University of Southampton, Southampton, UK.
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Cooper
Academic Unit of Bone Metabolism, Metabolic Bone Centre, Northern General Hospital, Sheffield, UK.
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Eastell
Academic Unit of Child Health, University of Sheffield, Sheffield, UK.
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Bishop
Oxford NIHR Musculoskeletal Biomedcial Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
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Luqmani
Oxford NIHR Musculoskeletal Biomedcial Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
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Wordsworth
Nuffield Department of Population Health, University of Oxford, Oxford, UK.
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Kaye
D016208:Databases, Factual; D006801:Humans; D009140:Musculoskeletal Diseases; D018579:Patient Selection; D035583:Rare Diseases; D012042:Registries; D006113:United Kingdom
Database management systems; Dynamic consent; Patient reported outcome measures; Rare diseases