NORD Launches Education Series to Advance Patient Involvement in Rare Disease Drug Development

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Series available in English and Spanish – Created in partnership with the FDA and Critical Path Institute 

Today, the National Organization for Rare Disorders (NORD) launched a new education series in English and Spanish titled, “Rare Disease Drug Development: What Patients and Advocates Need to Know,” designed to help patients and patient advocacy groups understand the drug development process.  

The goal of this educational series is to engage the rare disease community on their essential role in the drug development process and ultimately help produce more and better treatments for rare disease,” said Rebecca Aune, Director of Education Programs at the National Organization for Rare Disorders. “It was created to address key educational needs and gaps with input from patient organizations, patient advocates, and FDA reviewers, all of whom have a variety of experience with the drug development process for rare diseases. Moreover, this series is presented in the two most spoken languages in the United States.” 

Each module is available in a mix of formats to accommodate multiple learning styles, including animated videos, expert interviews, infographics, fact sheets, checklists, and interactive quizzes. The first modules, on “The Drug Development Process,” “Stakeholder Roles in Drug Development,” and “Natural History Studies,” are now available for free at learn.rarediseases.org 

Future modules, to be rolled out in 2024, will cover “Patient Experience Data,” “Designing Trials for Small Populations,” “Clinical Trial Endpoints and Clinical Outcome Assessments,” and more. Each module is designed to be valuable on its own, and together they provide a comprehensive understanding of the drug development process, the role of the patient, and the evidence needed to demonstrate that a new treatment for a rare disease works. 

The curriculum is a collaboration between NORD, the U.S. Food and Drug Administration (FDA), and the Critical Path Institute (C-Path) funded through the Rare Disease Cures Accelerator—Data and Analytics Platform (RDCA-DAP). It was announced by NORD President and CEO Peter L. Saltonstall at the 2023 NORD Rare Diseases and Orphan Products Breakthrough Summit to an audience of patient advocates, researchers, drug developers, and regulators, including FDA Commissioner Robert M. Califf, M.D., who will address Summit attendees tomorrow morning. 

There are more than 7,000 different rare diseases, of which 95% lack any FDA-approved treatment.  Rare diseases impact small patient populations of fewer than 200,000 people in the United States—sometimes far fewer. These smaller patient populations make it difficult to diagnosis and identify patients to participate in research, which is necessary for developing treatments. For treatments and research to occur for any given rare disease, patients and patient advocacy groups must be active participants in the drug development process. 

Watch the trailer for this education series below:

To access “Rare Disease Drug Development Series: What Patients and Advocates Need to Know,” visit learn.rarediseases.org. For more information, please contact media@rarediseases.org