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.

International comparison of availability for orphan drugs: focused on approved orphan drugs in South Korea

“…In the USA, to provide simple benefits, such as tax support for orphan drug development, the FDA established and led the Rare Disease Cure Accelerator, Data Analytics Platform (RDCA-DAP) with patient groups, accumulating genetic information of patients with rare diseases. Genetic information of patients with rare diseases can be used in the development of orphan drugs (Barrett et al, 2023 ). In addition, to solve the difficulties in recruiting participants for clinical trials, active policy support is being expanded, including revision of related guidelines to use real-world evaluation (RWE) and real-world data (RWD) for the approval of orphan drugs (FDA, 2018 ).…”

Learning from conect4children: A Collaborative Approach towards Standardisation of Disease-Specific Paediatric Research Data

“…When a user requests data, the data access (if approved) is dependent upon the degree of data sharing agreed to by the data contributors. In most cases, the user operates on the data within workspaces in a trusted research environment where no data can leave the environment without a proper approval process [46,47], but users may bring in their own private data to combine with RDCA-DAP data.…”

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

“…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 …”

“…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 …”