Integration of Biogen Rare Disease Data into the Rare Diseases Cures Accelerator Data and Analytics Platform (RDCA-DAP)

Lead Investigator: Alexandre Betourne, Critical Path Institute
Title of Proposal Research: Integration of Biogen Rare Disease Data into the Rare Diseases Cures Accelerator Data and Analytics Platform (RDCA-DAP)
Vivli Data Request: 7592
Funding Source: RDCA-DAP is funded by the FDA.
Potential Conflicts of Interest: None.

Summary of the Proposed Research:

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.

We selected a clinical trial in Progressive Supranuclear Palsy (PSP) to pilot the mutual sharing of rare diseases data between RDCA-DAP and Vivli. RDCA-DAP currently host the control arm of the PSP PASSPORT clinical trial, as well and additional datasets for the disease. With ongoing scientific work to model disease progression in PSP patients and to build a clinical trial simulator that will help clinician design future trials, this additional data will feed in our ongoing work and help accelerate the effort. 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.

Requested Studies:

Study of BIIB092 in Participants With Progressive Supranuclear Palsy (PASSPORT)
Data Contributor: Biogen
Study ID: NCT03068468
Sponsor ID: NCT03068468