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

Life Sciences, Clinical Trials, Pharmaceutical, Patient Recruitment & Retention,
  • Wednesday, February 28, 2018

The theme for Rare Disease Day, February 28, 2018 is “research.” 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.

Join us on Rare Disease Day 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.

Key topics include:

  • Bespoke trial design – like the rare disease itself, each trial demands unique customization. We’ll 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


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.

Message Presenter

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.

Message Presenter

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.

Message Presenter

Who Should Attend?

VP’s, Directors, Managers and Department Heads working within:

  • Clinical Affairs
  • Clinical Research
  • Clinical Pharmacology
  • Clinical Outsourcing
  • Project Management
  • Regulatory Affairs
  • Medical Affairs
  • Advocates & Researchers of Rare Diseases

Xtalks Partners


Medpace is a scientifically-driven, global, full-service clinical contract research organization (CRO) providing Phase I-IV clinical development services to the biotechnology, pharmaceutical and medical device industries. Medpace’s mission is to accelerate the global development of safe and effective medical therapeutics through its high-science and disciplined operating approach that leverages local regulatory and deep therapeutic expertise across all major areas including oncology, cardiology, metabolic disease, endocrinology, central nervous system and anti-viral and anti-infective. Headquartered in Cincinnati, Ohio, Medpace employs approximately 2,500 people across 35 countries.

Cincinnati Children’s Hospital Medical Center

Cincinnati Children’s is one of the nation’s leaders in pediatric healthcare, improving child health through its three integrated missions: clinical care, research, and medical education. Cincinnati Children’s was ranked as the number 3 children’s hospital in the U.S. in 2016 by U.S. News and World Report, operates the nation’s number 3 pediatric medical education program, and was the second highest recipient of NIH pediatric research grants in 2015. Cincinnati Children’s operates a 678-bed pediatric academic medical center and had more than 1.3 million patient encounters with $2.3 billion in revenue in fiscal year 2017, serving patients from all 50 states and nearly 60 different countries.

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