Cytel is excited to invite you to join us in our next presentation in the "New Horizons Webinar Series", which will introduce you to the latest innovations in statistical trial design. Cytel's Ursula Garczarek will be presenting a case study on the value of detailed clinical trial simulations for rare diseases. 

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In this case study, Ursuala will present on the journey of increased understanding of the features that matter the most from simulations for a pivotal trial in some rare genetic eye disease.

The primary endpoint is visual acuity in terms of logMAR (logarithm of the Minimum Angle of Resolution). The MAR is determined from the smallest optotype a patient can correctly identify on a chart. From previous studies in the field, it is expected that there are at least two clusters for the strength of the response: high responders and low responders. For hypothesis testing this combines the difficulty of small sample sizes with ties and a variance heterogeneity between control group and treated group.

The size of the impact from these features on the operational characteristics of different statistical approaches was higher than expected. It is an inconvenient insight though, as it means that one should run clinical trial simulations on patient level rather than statistical simulations on group level for clinical trials in rare diseases. As with groupwise assumptions, one uses standard asymptotics and approximations all to easily.

Speaker Bio

Ursula Garczarek, Ph.D. is Principal at Strategic Consulting in Cytel. She has extensive experience in providing statistical support for clinical and non-clinical aspects of product development within both pharmaceutical and consumer companies.
As a member of Cytel’s Strategic Consulting team, Ursula provides guidance to trial sponsors on optimizing their development strategy, and successfully implementing trial design innovations. She applies new and pragmatic methodologies to address the needs and requirements of the sponsor within the regulatory environment. She is experienced with interactions with the FDA and the EMA for general drug development, medical devices and in rare diseases.
Prior to Cytel, Ursula was the Program Leader Data Science at Unilever R&D (NL), and Biostatistician at Roche Diagnostics GmBH (DE) developing multi-marker diagnostics based on proteomics and metabonomics approaches. She received her Ph.D. from the University of Dortmund (DE), in context of a collaborative research center on complexity reduction in high-dimensional data spaces on Machine Learning and Statistics.