The future of Rare Disease Drug development: the Rare Disease Cures Accelerator Data Analytics Platform (RDCA-DAP)

Abstract

Rare disease drug development is wrought with challenges not the least of which is access to the limited data currently available throughout the rare disease ecosystem where sharing of the available data is not guaranteed. Most pharmaceutical sponsors seeking to develop agents to treat rare diseases will initiate data landscaping efforts to identify various data sources that might be informative with respect to disease prevalence, patient selection and identification, disease progression and any data projecting likelihood of patient response to therapy including any genetic data. Such data are often difficult to come by for highly prevalent, mainstream disease populations let alone for the 8000 rare disease that make up the pooled patient population of rare disease patients. The future of rare disease drug development will hopefully rely on increased data sharing and collaboration among the entire rare disease ecosystem. One path to achieving this outcome has been the development of the rare disease cures accelerator, data analytics platform (RDCA-DAP) funded by the US FDA and operationalized by the Critical Path Institute. FDA intentions were clearly focused on improving the quality of rare disease regulatory applications by sponsors seeking to develop treatment options for various rare disease populations. As this initiative moves into its second year of operations it is envisioned that the increased connectivity to new and diverse data streams and tools will result in solutions that benefit the entire rare disease ecosystem and that the platform becomes a Collaboratory for engagement of this ecosystem that also includes patients and caregivers.

Advancing the utilization of real‐world data and real‐world evidence in clinical pharmacology and translational research—Proceedings from the ASCPT 2023 preconference workshop

“…Improved data quality would also ensure that integrated data potentially used for regulatory submission can be trusted along with the code, models, and solutions derived from the data. 28…”

“…Collaborative “honest broker” arrangements and modern digital research environments (DREs) are urgently needed to ensure data sharing and improve the quality of the underlying source data. Improved data quality would also ensure that integrated data potentially used for regulatory submission can be trusted along with the code, models, and solutions derived from the data 28 …”