CPTA: How We Do It

Critical Path to Therapeutics for the Ataxias (CPTA) creates a neutral, pre-competitive environment where stakeholders, including industry drug development professionals, subject matter experts and regulators, can collaborate and share their expertise, insights, and data to generate novel quantitative solutions to accelerate drug development. Data contributed to CPTA is hosted on C-Path’s Rare Disease Cures Accelerator – Data and Analytics Platform (RDCA-DAP), an FDA-funded initiative that provides a centralized and standardized infrastructure for data across rare and orphan diseases. Sharing data on this platform enhances the impact of ataxia data and facilitates advanced analytical approaches. These data will inform ataxia research, leading to a better understanding of these disorders, how disease progression and drug effects are measured, and will help optimize the development of novel medical products for inherited ataxias.

CPTA is focused on collaboratively driving advancement in the following areas:

  1. Analysis of existing and new endpoints to optimize measurement of disease progression and drug effects in the various ataxias.
  2. Driving towards regulatory acceptance of new endpoints and/or biomarkers for use in ataxia clinical trials.
  3. Aggregation and sharing of existing clinical data across the ataxias to inform community understanding of natural history of the different ataxias.