Every Patient Matters: Bespoke Digital Endpoint Strategies for Rare Disease Drug Development
Session introduction
Register for this webinar to gain insights on how to select, operationalize and protect endpoints that stand up to regulatory scrutiny and support confident decision-making in rare disease drug development.
Session information:
Time:
- 11:00 a.m. – 12:00 p.m. EDT (NA-East)
- 04:00 p.m. – 05:00 p.m. BST (UK)
- 05:00 p.m. – 06:00 p.m. CEST (EU-Central)
Rare disease drug development presents unique challenges: patient populations are often minimal, clinical endpoints may not be well-established and traditional approaches to evidence generation cannot always be applied. To succeed, sponsors need flexible, carefully tailored digital endpoint strategies that can adapt to the realities of rare disease trials.
In this webinar, the featured speakers will illustrate how integrated solutions can help sponsors design endpoints that are both meaningful and reliable when every datapoint counts, by:
- Highlighting how eCOA strengthens the validity of clinical scales, reduces variability and safeguards endpoint quality through structured site engagement and rater training
- Exploring the complexities of implementing imaging in rare disease populations, including special considerations for paediatric and other unique populations
- Examining the role of continuous ECG monitoring and cardiac imaging in detecting disease progression, as well as the regulatory expectations for QT assessments
Register today for this webinar to gain insights on how to select, operationalize and protect endpoints that stand up to regulatory scrutiny and support confident decision-making in rare disease drug development.
Speakers
Mark Opler, Ph.D.
Chief Research Officer, eCOA Neuroscience at Clario
Dr. Mark Opler is the Chief Research Officer of eCOA Neuroscience at Clario. In this role, he directs scientific research and leads ongoing studies in psychiatry, measurement development, psychometric validation and other areas of neuroscience. He is also the Executive Director of the PANSS Institute. He has served as a Faculty Member in the Department of Psychiatry at New York University School of Medicine and in the Department of Neuroscience at Columbia University, College of Physicians and Surgeons. His academic research focuses on the etiology, phenomenology and treatment of serious and persistent mental disorders. He is a co-author and developer of several clinical assessment tools, including the SNAPSI, CGI-DS and NY-AACENT. He is also a contributor to the latest edition of the PANSS Manual©.
Dr. Opler has received research support from the US NIMH, the Brain & Behavior Foundation (formerly NARSAD), the Stanley Medical Research Institute and the Qatar National Research Fund. He has co-authored more than 50 peer-reviewed publications and has contributed to multiple book chapters and review articles on clinical assessment, research methodology and mental health.
He received his Ph.D. and MPH from Columbia University, as well as his BSc from SUNY at Stony Brook. He is a graduate of the Psychiatric Epidemiology Training Program at Columbia University and completed his postdoctoral fellowship at the New York State Psychiatric Institute.
Scott J. Hunter, Ph.D.
Senior Scientific Expert, Neurodevelopmental and Rare Disease, and Epilepsy at Clario
Dr. Scott Hunter is a Senior Scientific Expert specializing in neurodevelopmental and rare diseases, as well as epilepsy. With a distinguished background as a former Professor of Psychiatry and Behavioral Neuroscience, and Pediatrics at the University of Chicago, Dr. Hunter is a recognized authority in the field.
His expertise spans the design and validation of clinical outcome assessments (eCOA, PRO, ClinRO and ObsRO), clinical trial endpoint identification and the application of best practices in data capture for neurodevelopmental, behavioral and cognitive measures. Dr. Hunber’s work has focused on a wide range of therapeutic areas, including neurogenetic disorders, paediatric and adult epilepsies and paediatric cancers. He has also held key roles such as Director of Neuropsychology at UChicago Medicine and has served as a former NIH-funded PI and Co-PI.
Thomas Fuerst, Ph.D.
Chief Science Officer at Clario
Dr. Thomas Fuerst is the Chief Science Officer at Clario, where he leads scientific strategy and innovation across imaging and endpoint technologies for clinical trials. He has extensive expertise in advanced imaging modalities, including XR, CT and HRCT, MRI, ultrasound, DXA, QCT and photography. Dr. Fuerst has been at the forefront of developing and applying novel methods to generate high-quality, reliable data for global studies.
His therapeutic area expertise spans musculoskeletal and metabolic diseases, as well as inflammatory and autoimmune conditions. He has contributed to advancements in assessing bone, muscle and fat composition, as well as imaging-driven insights into arthritis (osteoarthritis, rheumatoid, psoriatic and spondylarthritis), gout, systemic sclerosis and thyroid eye disease. Dr. Fuerst earned his Ph.D. from the University of Wisconsin-Madison and has authored numerous scientific publications advancing the use of imaging biomarkers in clinical research.
In his role at Clario, he continues to collaborate with sponsors, regulators and the scientific community to ensure imaging endpoints support meaningful outcomes in clinical development.
Vic Patel, M.D., Ph.D.
Vice President & Chief Medical Officer, Cardiology at Clario
Dr. Vic Patel is board-certified in cardiology and holds a Ph.D. in Biophysics, having conducted extensive research on the mechanisms of cardiac arrhythmias. He was a Faculty Member at the University of Pennsylvania School of Medicine for 11 years, where he directed molecular arrhythmia research and practiced clinical electrophysiology. Dr. Patel then transitioned into drug development and cardiac safety as a clinical development leader and therapy area head, overseeing all phases of development.
At Clario, Dr. Patel oversees cardiology consulting and ECG/Holter core lab services while leading the biostatistics and medical writing groups. He works with our global customers to ensure their needs are met with robust, cost-effective solutions.