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Why Attend?

Rare disease clinical development is evolving rapidly, fueled by regulatory innovation, adaptive trial designs, artificial intelligence, and a more global approach to patient recruitment.

Yet success depends on more than scientific advances, it requires operational strategies that address limited patient populations, complex endpoints, and scarce natural history data.

In this webinar, industry experts from clinical operations, regulatory affairs along with a patient advocacy will discuss the trends shaping the future of rare disease research and share practical strategies for designing and executing successful global studies.

Learn how to balance innovation with execution to accelerate development while improving the experience for patients and caregivers.

Speakers

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Ella Balasa
Patient Advocate, speaker, published writer, and health engagement consultant

Ella Balasa is a patient advocate, consultant, and a person living with cystic fibrosis. She has committed her time to empowering patients and advancing healthcare strategies. She speaks publicly about the value of patient perspective and has a passion for distilling clinical information for patient communities. Through opportunities working with healthcare organizations on content strategy, writing, speaking, clinical trial development, and sharing the patient experience she aims to affect the healthcare landscape by raising awareness of rare diseases, promoting self-advocacy to patients, and valuable insights to organizations. More of her work and experiences can be found at www.ellabalasa.com.

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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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Blaine VanLeuven
Executive Director, Regulatory & Strategic Development - CMC

Blaine is a strategic leader with more than 18 years of experience working in the biopharmaceutical industry, primarily focused on Chemistry, Manufacturing, and Controls (CMC), regulatory affairs and global regulatory strategy. She provides strategic solutions and services to emerging biopharma companies from concept to commercialization. She has leveraged her regulatory expertise to support a wide variety of therapeutic treatments (small molecules, antibodies, recombinant proteins, cell and gene therapies, drug/device combination products) designed to treat a broad array of diseases and conditions (hematology, oncology, autoimmune, ophthalmic, cardiovascular, endocrine, pulmonary, CNS, anti-viral, metabolic, dermatology, rare diseases, etc.).

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