Rare Disease Day, set for February 29, is intended to raise awareness around the world about rare diseases and their impact on patient lives. Worldwide Clinical Trials will mark the day with a free webcast that demonstrates new perspectives on the study of rare diseases, highlighting the need for the industry to rethink its approach to clinical trial design and execution within this complex area of medicine.
The presentation, "Methods in Medicine: Conducting Rare Disease Studies with Efficiency and Innovation," will take place on Feb. 29 at 10 a.m. ET. To register, please visit www.worldwide.com/events.
Attendees of the webinar will hear from Worldwide’s Chief Medical & Scientific Officer Dr. Michael Murphy, along with other experts, who will provide insight into the strategic regulatory and operational challenges of orphan, or rare, disease drug trials. This includes the impact of patient and physician interaction and cultural rapport and the importance of family engagement. The models of participatory research will also be discussed, along with guidance on the need to demonstrate product value, which can enable patient access following market authorization.
"Worldwide is pleased to join efforts around the globe on Rare Disease Day to shine a spotlight on the patient voice and the work being done in R&D programs in this important area of medicine," said Murphy. "Our webcast will highlight the unique requirements of clinical trials in rare disease, with particular emphasis on the reciprocal relationships across stakeholders that lead to successful development and commercialization."
A disease is defined as rare in the U.S. when it affects fewer than 200,000 Americans at any time; in Europe, it is defined as rare when it affects fewer than one in 2,000. Eighty percent of rare diseases have identified genetic origins, while others are the result of infections, allergies and environmental causes. Fifty percent of those touched by rare diseases are children. For more information on Rare Disease Day, visit www.RareDiseaseDay.org.