The National Center for Advancing Translational Sciences (NCATS) released its new Toolkit for Patient-Focused Therapy Development at a public meeting on the NIH campus on September 8. The toolkit is a centralized portal of the “how-to” resources and tools that patient support organizations can use to advance translation for their diseases, from discovery through clinical trials, to regulatory and industry interactions, to post-approval access issues. It guides patients and patient organizations by giving them the means to engage in research, drug development and post-approval issues themselves. While the resources are primarily focused on rare diseases, the tools are useful for all diseases.
This toolkit presents several advantages:
• Collaborative creation of a well-designed source for online educational and informational research resources and tools.
• Provision of a single online portal with resources that patient groups can readily access along with context.
• Improved coordination rather than re-creation of existing resources.
• Identification of gaps in online resources, and information dissemination to patient groups.
• Promotion of continuity across the lifecycle of the drug development process.
This toolkit was developed in close collaboration between the rare diseases patient advocacy community and the NCATS’ Office of Rare Diseases Research. In this months’ NCATS Director’s message, Dr Austin, Director of NCATS and Chair of the IRDiRC Consortium Assembly, explained that it was one of his first directives “to involve patients in every project we do from the beginning, and to develop the science of patient and community engagement, with best practices that can be disseminated to all doing translational research.” This toolkit is a step towards that goal.