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Why Attend?

Understanding the patient pathway – what it means for a person to have that actual rare disease – before study initiation is critical to running effective rare disease studies.

Many trials continue to overlook key barriers to participation, from accessibility and trust to communication and support. This webinar will explore how applying insights from an understanding of the patient pathway in both study design and execution can improve trial enrollment, retention, and outcomes in rare disease studies.

Key Discussion Points:

  • How to gather insights into the patient pathway
  • Understanding the root causes of low patient participation and why traditional trial models may be falling short in rare disease studies
  • Practical strategies to build patient-centric trials – applying patient pathway insights to protocol and study design
  • Practical strategies for executing patient-centric trials - applying patient pathway insights to study execution and operations
  • The role of patient advocacy groups, digital engagement, and transparency in building trust and long-term participation.

Speakers

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Gemma O’Connor
Director, Project Management

Gemma is a Project Director with over 20 years of clinical research experience, including 14 years in project management roles with a specialist focus on patient education, recruitment and retention. She has a proven record of supporting Caidya’s biopharma clients with timely and high-quality delivery of their studies. She has experience across a number of therapy areas, including expertise in Rare Disease (Lipoprotein Lipase Deficiency, Glioblastoma, Lupus, Familial Hypercholesterolemia, TGCT) and Pediatrics (Vaccination, Pancreatitis, Bacterial Infection)

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Jonathan Kornstein
Vice President of Rare Disease and Pediatrics, Caidya

Jonathan Kornstein is Vice President of Rare Disease and Pediatrics at Caidya, where he leads teams delivering clinical trial services tailored to the unique challenges of rare disease and pediatric development. In his role, he oversees operational, clinical, and medical specialists to design and execute patient-centric studies, and provides strategic guidance to clinical management, commercial development, and business development functions.

He brings approximately three decades of experience in the pharmaceutical and clinical research organization industry. He has guided clinical strategies that balance operational rigor with patient needs, and he leads discussions and thought leadership on rare disease trial execution and patient-centric research approaches.

He holds a bachelor’s degree in biology from Hamilton College in Clinton, New York, and is based in Chapel Hill, North Carolina.

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