• Center on Health Equity and Access
  • Clinical
  • Health Care Cost
  • Health Care Delivery
  • Insurance
  • Policy
  • Technology
  • Value-Based Care
Rare Disease : Episode 1

Episode 1 - Recognizing Rare Diseases in the United States

Video

In a peer exchange moderated by Dr. Peter Salgo, Hugh Fatodu and Drs. Patrick F. Fogarty, Maria Lopes, and Michelle Petri share their insights on the evolving dynamics of rare diseases in contemporary healthcare and managed care, and specifically address the implications for hemophilia and systemic lupus erythematosus (SLE). In the US, a rare disease is defined as a condition that affects than 200,000 persons. However, despite the 25 million American lives currently affected by more than 7,000 identified rare diseases, the patient and provider communities are still plagued by a general lack of awareness, a high risk for misdiagnosis, and a scarcity of treatment options. There is an acknowledgement among the panelists that the early recognition of diseases such as hemophilia and SLE has “very significant” implications for therapy, perhaps life-changing and even life-saving.


Related Videos
ISPOR 2024 Recap
Chris Pagnani, MD, PC
Phaedra Corso, PhD, associate vice president for research at Indiana University
Julie Patterson, PharmD, PhD
Michael Morse, MD, Duke Cancer Center
Dr Chris Pagnani
Nancy Dreyer, MPH, PhD, FISE, chief scientific advisor to Picnic Health
Seth Berkowitz, MD, MPH, associate professor of medicine, University of North Carolina at Chapel Hill
Inma Hernandez, PharmD, PhD, professor at the University of California, San Diego Skaggs School of Pharmacy and Pharmaceutical Sciences
Binod Dhakal, MD
Related Content
© 2024 MJH Life Sciences
AJMC®
All rights reserved.