DOI: 10.25934/pr00009870
title: Integration of Biogen Choroideremia Data into the Rare Diseases Cures Accelerator Data and Analytics Platform (RDCA-DAP)

description: The Rare Disease Cures Accelerator Data and Anaytics Platform (RDCA-DAP) is a neutral platform that houses patient data from diverse sources, including clinical trials, patient registries and hospital medical records across a multitude of rare diseases. RDCA-DAP’s objective is to promote the sharing of already collected patient data, and to help data contributors improve their future data collection. RDCA-DAP hosts such data in a format ready for regulatory submissions but also suitable for scientific and analytical work. By doing so, the RDCA-DAP aims to accelerate the understanding of disease progression, the development of better clinical and biological measures to assess patients’ diseases, and to facilitate the design of innovative clinical trials. RDCA-DAP is positioned to generate solutions that will facilitate the development of new drugs for rare diseases patients. As such, the utility of patient data is maximized, and the data may be leveraged to develop tools accessible by clinicians (from academia and industry) that will optimize and accelerate drug development across rare diseases.
RDCA-DAP key objectives include:
 Development of a database of patient data across all rare diseases comprised of data from a wide range of sources that can be searched in multiple ways and used for analysis. The database is secured and protects patients’ identity from being revealed.
 Sharing of data with the global research community through a secure interface with appropriate privacy controls. Data may be used within the platform and/or some data may be available for download in formats suitable for submissions to regulatory authorities (FDA, EMA)
 Aiding in the analysis of the data by providing analytical functionalities and tool in the platform, as well as opportunities to collaborate with C-Path data science team.
 Connecting RDCA-DAP with other platforms hosting patient data, including Vivli, to ensure the platform does not work in isolation. In turn, this will increase the amount of data available to scientists and maximize opportunities for innovation and science.

RDCA-DAP aims to be a diseases agnostic effort to help as many rare diseases as possible. In the spirit of expanding our database and diversifying the diseases we work on, we selected two new datasets for choroideremia (NCT03359551 ; NTCNCT03496012), a rare eye disorder. This request was informed by extensive discussions between RDCA-DAP and the Choroideremia Research Foundation, identifying the critical need to build a comprehensive natural history of the disease and make that information available through our platform to international scientists. The data will be fully incorporated into RDCA-DAP for future analyses, and external sharing to RDCA-DAP users, as defined by Vivli, Biogen and RDCA-DAP.

We selected two choroideremia datasets to expand the pilot of mutual sharing of rare diseases data between RDCA-DAP and Vivli. RDCA-DAP currently host data over 35 different rare disease, but the sharing of these data will be the first rare eye disease data shared to the platform. RDCA-DAP have been engaging with multiple rare eye disorders communities and will continue to work to gather additional data in that space with critical unmet needs. The data will be fully incorporated into RDCA-DAP for future analyses, and external sharing to RDCA-DAP users, as defined by Vivli, Biogen and RDCA-DAP.