Watch the Webinar

Watch the Webinar

Getting Treatment Options to Rare Disease Patients Faster: Putting Patients First in Clinical Trials

Getting Treatment Options to Rare Disease Patients Faster: Putting Patients First in Clinical Trials

In clinical research, the relationship between patient and researcher is symbiotic – the patient community needs researchers for cures and treatments and researchers need patients to participate to ensure meaningful outcomes. With rare disease, this relationship is intensified on both sides: patients and their caregivers often have few treatment options while researchers have a smaller pool of potential participants.

Watch as experts from Medpace, a global clinical research organization, and Cincinnati Children’s Hospital Medical Center discuss how patient-focused clinical trial design and collaborative approaches to site enrollment can accelerate clinical research. Attendees of this webinar will also hear from a parent of a child with Duchenne’s Muscular Dystrophy (DMD) to understand the impact of research on her family, what it means to be an advocate, as well as the personal challenges.

Points of discussion include:

  • Bespoke trial design – like the rare disease itself, each trial demands unique customization. We discuss the considerations, challenges, and strategies that work
  • Putting the patient first – Patient-centricity is more than a buzzword. How early planning, open communication, and well-executed operations can drive success for patients and researchers
  • Optimizing patient recruitment to accelerate research today – and trends for tomorrow
  • CRO and site collaboration – best practices
  • A Family’s Story – A parent’s personal experience navigating clinical research for her son with DMD—What she’s learned as well as advice for both researchers and parents

Learn More About the Speakers

Michelle Petersen, MS, Sr. Associate Director, Clinical Trial Management, Medpace

Michelle Petersen has 10 years’ experience in preclinical and clinical research which includes managing global studies in Phases I through III of development. She has managed multiple global rare/ultra-rare disease trials with a focus on neuromuscular and pediatric disorders. Ms. Petersen is a member of Medpace’s specialized Rare Disease Consortium and Patient Recruitment Team, and she is adept in developing strategies necessary to address the potential challenges of recruitment and logistical requirements for studies of this nature. Ms. Petersen holds a Master of Science degree in Physiology from the University of Cincinnati College of Medicine.


John Lynn Jefferies, MD, MPH, FACC, FAHA, FHFSA, Director, Advanced Heart Failure and Cardiomyopathy Programs, Cincinnati Children’s Hospital Medical Center, University of Cincinnati

John Lynn Jefferies, MD, MPH, is Director of the Advanced Heart Failure and Cardiomyopathy Services at Cincinnati Children’s Hospital Medical Center. He is also an attending Heart Failure Cardiologist at the University of Cincinnati Hospital and the Christ Hospital in Cincinnati, OH. He specializes in advanced heart failure and cardiomyopathy care for both children and adults. He is certified as a Diplomate in Internal Medicine and Adult Cardiovascular Diseases as well as Advanced Heart Failure and Transplant Cardiology. Dr. Jefferies is also a Pediatrician and Pediatric Cardiologist. He is a Professor of Pediatric Cardiology and Adult Cardiovascular Diseases.


Beth Woelfel Harvey, Executive Director, JB’s Keys to DMD, Mother of child with Duchenne Muscular Dystrophy

Beth is the mother of two children, Caroline (12) and JB (10). Beth is raising her children outside of Boston. JB was diagnosed with Duchenne Muscular Dystrophy (DMD) in 2009 at 16 months old. When JB turned 2, Beth and her family founded an organization, JB’s Keys to DMD, to raise awareness of DMD while appropriating funds for quality care and experiences, ensuring affected children will have the opportunities of a lifetime. JB has participated in two clinical trials and two research studies. Beth has firsthand experience navigating the clinical trial landscape to decide on the best clinical trial for her son. In addition, she has knowledge about the stress of dealing with the logistics and emotional challenges of participating in trials. Beth holds a Bachelor of Arts in History from the College of The Holy Cross in Worcester, MA.

Contact us to learn more about rare disease clinical research