To Top

Jun. 3, 2019

TOPIC: Press Releases, Featured News, Patients & Members, Research, Patient Stories


Posted by Lisa Sencen

Real-world evidence and compelling case studies bring to life the challenges facing the estimated 30 million Americans fighting to live better with rare disorders

Washington, DC, June 3, 2019—The National Organization for Rare Disorders® (NORD), the leading independent nonprofit organization representing the estimated 30 million Americans living with rare diseases, has collaborated with Trio Health, a leading provider of real-world patient data and analytics, to publish a new book entitled The Power of Patients: Informing Our Understanding of Rare Diseases. This important collection of real-world insights is informed by scientifically validated, patient-reported, patient-experience data collected by NORD’s registry community. It was developed in partnership with six patient organizations, and features a foreword by Janet Woodcock, MD, Director of FDA’s Center for Drug Evaluation and Research.

“At NORD, our priority is to create real change and accelerate progress for the rare disease community. By working at the intersection of research, policy, and education, we demonstrate the power and possibility of patient-reported, real-world evidence,” said Peter L. Saltonstall, President and CEO of NORD. “Created in collaboration with Trio Health, The Power of Patients amplifies the experiences of individuals and families living with rare diseases, and demonstrates the importance of registries and natural history data in helping to shape the way the rare disease landscape is navigated by patients and their physicians.”

By sharing stories, celebrating community-driven teamwork and exhibiting data from NORD’s registry community, The Power of Patients aims to illustrate the promise and potential of longitudinal natural history studies.

“Our goal is to help shed light on the entire patient journey,” said Brent Clough, CEO of Trio Health, which has been providing similar real-world data on more common diseases since 2013. “We are proud to partner with NORD and fill a knowledge gap that can ultimately improve the lives of people with rare diseases.”

Data on the natural history of rare diseases can inform the design of efficient clinical trials, helping reduce the length and cost of drug development and contribute to clinical programs and therapies that meaningfully reflect the needs of the community. As Janet Woodcock, MD, notes in the book’s foreword, “Natural history registries also subserve the enormous benefit of enabling people with rare diseases to find each other and to come together to access their collective experience. Additionally, drug developers who are planning clinical trials often consult with patients with the disease to be targeted, who may best know what is feasible to do in a clinical trial, what might not work for certain patients, and may even be able to suggest patients for a trial or participate themselves.”

The Power of Patients highlights the critical importance of patient participation in registries and how essential natural history data for rare diseases is to the research and development of new treatments. With 90% of the more than 7,000 rare diseases still without an FDA-approved treatment, that is progress vital to the lives of the millions of Americans affected by rare diseases.

To obtain a copy of The Power of Patients, please contact [email protected].


## #

About the National Organization for Rare Disorders® (NORD)

The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases. NORD is committed to the identification, treatment and cure of the more than 7,000 rare diseases, of which approximately 90 percent are still without an FDA-approved treatment or therapy. Rare diseases affect 25-30 million Americans. More than half of those affected are children.

NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. For more than 35 years, NORD has led the way in voicing the needs of the rare disease community, driving supportive policies and education, advancing medical research and providing patient and family services for those who need them most. NORD is made strong together with over 280 disease-specific member organizations and their communities and collaborates with many other organizations on specific causes of importance to the rare disease patient community. Learn more at

About Trio Health

Trio Health’s mission is to improve the quality of care in patient outcomes through coordinating the efforts of all patient care stakeholders. Their first-of-its-kind Multi-Disease Platform (MDX) that tracks patients throughout the course of their treatment, giving pharmaceutical/biotechnology companies, specialty pharmacies and physicians access to information and opportunities that simply don’t exist anywhere else. Learn more at

Media Contacts


Laura Mullen
[email protected]


For Trio Health:

Brad Miles
Solebury Trout
[email protected]