What is patient-focused drug development?
Patient-focused drug development (PFDD) is a systematic approach to capture a patient’s experience, perspectives and needs, and to ensure that information can be used to inform the development of new treatments and their approval by the U.S. Food and Drug Administration (FDA).
Less than 10 percent of rare diseases have an FDA-approved treatment or therapy. Drug development can be extremely challenging for rare diseases which is why NORD is partnering to help advance PFDD with the rare disease community.
Our team works with members of the rare community to understand how they can engage in patient-focused drug development initiatives, such as Patient-Focused Drug Development (PFDD) Meetings and Listening Sessions with the FDA, and share best practices and guidance in hosting Externally-led PFDD meetings.
In 2019, NORD and the FDA entered into a collaborative Memorandum of Understanding to pilot patient listening sessions to promote early engagement for people living with rare diseases and their caregivers with FDA reviewers in their Centers for Drug Evaluation and Research, Biologics Evaluation and Research, and Devices and Radiological Health. Learn More About FDA Patient Listening Sessions
Getting the Most from the Data
Critical Path Institute (C-Path) and NORD, through a collaborative grant from the FDA, joined together to create the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®), an integrated database and analytics hub designed to be used in building novel tools to accelerate drug development across rare diseases. The platform promotes sharing patient-level data and encourage the standardization of new data collection.
If you would like to learn more or talk with the team at NORD about how to get involved, please contact us here.
If you would like to talk about contributing data or get involved in discussions around needed analytics, click here.