Rare Disease | Genetic Condition
Hereditary Inclusion Body Myopathy
Date of Event
Name of Event
Overcoming Challenges in Orphan Drug Development
Event Details
Educational Seminar in Orphan Drug Development, followed by Reception and Silent Auction to benefit HIBM Research Group https://www.agilityrarediseasefund.org/
Anticipated Number of Attendees
Is Event Open to the Public?

Overcoming Challenges in Orphan Product Development

Benefiting: Biopharmaceutical companies involved with rare/orphan drug development, and HIBM Research Group, Inc. (HRG)

Each year, February 28, is designated as Rare Disease Day. Agility Clinical and BIOCOM are proud to join many other organizations around the world in support of raising awareness of the challenges, hopes and needs of those living with rare diseases.

Rare diseases affect one in 10 Americans, 30 million people in the United States, and 300 million people globally. Of the approximately 7,000 identified rare diseases, less than 500 have an FDA approved treatment available. Many have genetic origins and affect young adults and children. It estimated that more than 50 percent of those affected by rare diseases are children, making rare diseases the most deadly and debilitating diseases for children worldwide.

For patients, even getting a diagnosis can become a quest. Worse yet, patients are too often confronted with a lack of treatment for their condition. Biopharmaceutical companies working on bringing orphan treatments to patients face many challenges as well, from regulatory strategy to planning and conducting successful clinical studies. Understanding how to overcome some of the unique challenges of orphan clinical studies is critical to developing a well-designed drug development program.

Please join us for this informative presentation, followed by a Q&A panel discussion, and wine and hors d’ oeuvres reception and silent auction fundraiser in support of finding a treatment for Hereditary Inclusion Body Myopathy (HIBM). For detailed information, please visit www.agilityrarediseasefund.org. HIBM are a group of rare/orphan genetic disorders. HIBM causes progressive muscle wasting and weakness that begins in young adulthood and can lead to severe disability within 10-20 years.


Tim Cote, MD, MPH,
Principal and CEO, Cote Orphan Consulting
Former Director of the FDA Office of Orphan Products Development
Former CMO of the National Organization for Rare Disorders
“News from the Orphanage”

Babak Darvish, MD & Daniel Darvish, MD
HIBM Patients, Advocates and Researchers
Founders of Advancement of Research for Myopathies (ARM) and HIBM Research Group (HRG)
“The ARM and HRG Story”

Bruno Gagnon 
VP, Clinical Operations, BioMarin
“Challenges of Conducting Trials in Orphan Diseases”

Christa van Kan
Director Clinical Operations, PSR
“Collaborative Drug Development for Orphan Drugs”

The speakers will be joined by Elizabeth Ludington, PhD (VP, Biometrics, Agility Clinical) in a Q&A panel discussion immediately following the presentations.