Held annually on the last day of February, Rare Disease Day is celebrated globally to raise awareness about the impact rare disease has on patient’s lives. Since inception by EURORDIS, Rare Disease Day has generated thousands of events worldwide reaching millions of people.
As a global CRO specialized in rare disease, Medpace understands how rare diseases affect patients globally. Notably, 350 million people suffer from a rare disease around the world – that is greater than the entire population of the United States. With so many rare disease patients globally, support and awareness are critical.
2019 Rare Disease Day Initiatives
In support of this important cause, Medpace is participating in 2019 Rare Disease Day through a number of initiatives.
Webinar: How Advanced Therapies are Changing the Landscape of Rare Diseases
The webinar will discuss how advanced therapies are dramatically changing the way patients are treated for a wide variety of diseases – particularly for patients with rare diseases and orphan indications. Learn more and register here.
The webinar will touch on topics including:
- Scientific Advancements: The new and exciting landscape for advanced therapies in rare disease for a variety of indications, with a deeper dive into hematology and ophthalmology
- Regulatory Landscape: Key characteristics and considerations for obtaining orphan drug development (ODD) designation in the US as well as an overview of the comparative differences among current global orphan product laws and regulations
- Operational Considerations: Lessons-learned and best practices for operationalizing rare disease clinical trials including identifying, enrolling and retaining patients, as well as special considerations for ATMPs
Showing Our Brightest Support
A Look Back at Last Year
For 2018 Rare Disease Day, employees at Medpace headquarters in Cincinnati Ohio were invited to sign the Medpace Rare Disease Day banner. Medpace presented the banner to a local partner, Cincinnati Children’s Hospital Medical Center, as a thank you for being a part of our mission to advance rare disease research.
Medpace also celebrated 2018 Rare Disease Day with a webinar highlighting how to get treatment options to rare disease patients faster. Experts from Medpace and Cincinnati Children’s Hospital Medical Center discussed how patient-focused clinical trial design and collaborative approaches to site enrollment could accelerate clinical research. The webinar also featured a parent of a child with Duchenne’s Muscular Dystrophy to help people better understand the impact of research on her family, personal challenges, and what it means to be an advocate.