For whom is the EJP RD Virtual Platform intended?
Onboarding resources to the VP benefits data resource managers by making their data reusable for rare disease research as well as data users like rare diseases researchers, clinicians, etc, who will have more data available for their analyses. The VP is designed to meet the needs of diverse stakeholders, each with specific objectives related to accessing and processing (meta)data.
Common users of the VP include:
Clinicians conducting research projects (e.g., European Research Network (ERN) researchers) whose main objectives include accessing the rare disease (meta)data available through the VP to conduct large-scale transversal studies, and building cohorts (e.g., for clinical trials). They can use the user-friendly interface with safeguards to prevent illegal or unethical data use. ERN patient registry developers can also create specialized interfaces for clinical researchers by incorporating VP functions in its user interface.
Application developers whose main objectives are to develop new functionalities and applications for their organizations, e.g., adding functions to a clinical data platform in a hospital or creating user-friendly interfaces for given target users or for given tasks. These new functionalities could consist of (but are not limited to) various forms of analysis over multiple resources, such as queries for basic information, performing various forms of statistics, or running computational workflows. To fulfil these objectives, application developers will need a standardized API, like that of the VP, which in turn accesses standardized information from the resources.
Data scientists whose objectives are to develop analysis methods across any number of resources with relevant data, e.g., by computational workflows, artificial intelligence or statistical methods, knowledge discovery, etc. To fulfil these objectives, the data scientists require that data in resources are standardized and machine actionable. In other words, spending months to make data compatible for each analysis will be outdated in the VP.
Patient representatives whose main objectives are to foster optimal use of healthcare data derived from rare disease patients. Their primary objective is to ensure that relevant resources pertaining to rare diseases are easily accessible and usable by all those working towards patient priorities, such as improving quality of life, facilitating earlier access to care, and advancing the understanding of rare diseases, new drugs, and therapies. In addition, patients would benefit from a simple way to access their own data, see how their data is used in the VP, and be provided with statistics on their disease.