Background Rare disease communities are spread around the globe and segmented by their condition. Little research has been performed on the majority of rare diseases. Most patients who are affected by a rare disease have no research on their condition because of a lack of knowledge due to absence of common groups in the research community. Objective We aimed to develop a safe and secure community of rare disease patients, without geographic or language barriers, to promote research. Methods Cocreation design methodology was applied to build Share4Rare, with consultation and input through workshops from a variety of stakeholders (patients, caregivers, clinicians, and researchers). Results The workshops allowed us to develop a layered version of the platform based on educating patients and caregivers with publicly accessible information, a secure community for the patients and caregivers, and a research section with the purpose of collecting patient information for analysis, which was the core and final value of the platform. Conclusions Rare disease research requires global collaboration in which patients and caregivers have key roles. Collective intelligence methods implemented in digital platforms reduce geographic and language boundaries and involve patients in a unique and universal project. Their contributions are essential to increase the amount of scientific knowledge that experts have on rare diseases. Share4Rare has been designed as a global platform to facilitate the donation of clinical information to foster research that matters to patients with rare conditions. The codesign methods with patients have been essential to create a patient-centric design.
Data dictionaries for clinical trials are often created manually, with data structures and controlled vocabularies specific for a trial or family of trials within a sponsor’s portfolio. Microsoft Excel is commonly used to capture the representation of data dictionary items but has limited functionality for this purpose. The conect4children (c4c) network is piloting the Direcht clinical data modelling tool to model their Cross Cutting Paediatric Data Dictionary (CCPDD) in a more formalised way. The first pilot had the key objective of testing whether a clinical data modelling tool could be used to represent data items from the CCPDD. The key objective of the second pilot is to establish whether a small team with little or no experience of clinical data modelling can use Direcht to expand the CCPDD. Clinical modelling is the process of structuring clinical data so it can be understood by computer systems and humans. The model contains all of the elements that are needed to define the data item. Results from the pilots show that Direcht creates a structured environment to build data items into models that fit into the larger CCPDD. Models can be represented as an HTML document, mind map, or exported in various formats for import into a computer system. Challenges identified over the course of both pilots are being addressed with c4c partners and external stakeholders.
Introduction: The conect4children (c4c) consortium was setup to facilitate the development of new drugs and therapies for paediatric populations and address key challenges associated with paediatric clinical trials. Two of the major adopting principles for c4c were academia-industry partnership and data harmonisation and interoperability through common eCRF definitions. To understand the challenges arising out of these principles, the c4c team at Newcastle University conducted semi-structured interviews with four c4c industry partners.Methods: Each partner was asked 10 questions about the data standards used in their company, management and maintenance of data dictionaries, how they dealt with paediatric-specific issues, major knowledge gaps and how academia could aid in bridging these gaps. Thematic analysis was performed to identify patterns in their answers. Results: All companies use the Clinical Data InterchangeStandards Consortium (CDISC) standards but face problems when certain terminology is not included in CDISC
BACKGROUND Rare disease communities are spread around the globe and segmented by their condition. The most of the patients being affected by a rare disease have no research on their condition because of lack of knowledge due to absence of common groups from where to raise knowledge and promote research. OBJECTIVE To build a safe and secure community of rare diseases patients without geographical or language barriers to promote research. METHODS Co-creation design methodology was applied to build the first Share4Rare final version, with consultation and input from a variety of stakeholders (patients, caregivers, clinicians & researchers). RESULTS The workshops allowed us to develop a layered version of the platform, based on educating patients and caregivers with publicly accessible information, a secure community for the patients and caregivers, and a research section with the purpose of collecting patient information for analysis, which was the core and final value of the platform. CONCLUSIONS Rare diseases research requires a global collaboration in which patients and caregivers have a key role. Collective intelligence methods implemented in digital platforms allows to reduce geographical and language boundaries, and involve in a unique and universal project patients. Their contribution is essential to reduce the scientific knowledge that experts have on rare diseases. Share4Rare has been designed as a global platform to facilitate the donation of clinical information to foster research that matters to the patients with rare conditions. The co-design methods with patients have been essential to perform a patient centric design of Share4Rare.
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