National Organization for Rare Disorders Launches Education Series to Advance Patient Involvement in Rare Disease Drug Development

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

WASHINGTON, Oct. 16, 2023 /PRNewswire/ -- 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 "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 today by NORD President and CEO Peter L. Saltonstall at the organization's 2023 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.

"C-Path is proud to support this education series and it's great to see the roll out begin," said Collin Hovinga, Pharm.D., M.S., FCCP, Vice President of Rare and Orphan Disease Programs at C-Path. "We're committed to listening to people with lived experience as their insight, experiences, and perspectives play a vital role in drug development for rare diseases. C-Path's rare and orphan programs, including RDCA-DAP, rely on patient data for progress and their contributions are not just data points to us; they are the heartbeat of the mission to create efficient treatments for all."

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. 

To access "Rare Disease Drug Development Series: What Patients and Advocates Need to Know," visit learn.rarediseases.org. For more information, please contact [email protected].

About the National Organization for Rare Disorders (NORD)

With a 40-year history of advancing care, treatments and policy, the National Organization for Rare Disorders (NORD) is the leading and longest-standing patient advocacy group for the 30 million Americans living with a rare disease. An independent 501(c)(3) nonprofit, NORD is dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 330 patient organization members, is committed to improving the health and well-being of people with rare diseases by driving advances in care, research and policy. For more information, visit rarediseases.org.

About the Critical Path Institute (C-Path)

Critical Path Institute (C-Path) is an independent, nonprofit established in 2005 as a public-private partnership, in response to the FDA's Critical Path Initiative. C-Path's mission is to lead collaborations that advance better treatments for people worldwide. Globally recognized as a pioneer in accelerating drug development, C-Path has established numerous international consortia, programs and initiatives that currently include more than 1,600 scientists and representatives from government and regulatory agencies, academia, patient organizations, disease foundations and pharmaceutical and biotech companies. With dedicated team members located throughout the world, C-Path's global headquarters is located in Tucson, Arizona and C-Path's Europe subsidiary is headquartered in Amsterdam, Netherlands. For more information, visit c-path.org or email [email protected].

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SOURCE National Organization for Rare Disorders (NORD)