Biostatistics Weekly Seminar

Building Better Clinical Trials Through Disease Progression Modeling in GNE Myopathy

Melanie Quintana, PhD
Director and Senior Statistical Scientist
Berry Consultants

Through a case study in GNE Myopathy, a rare progressive disease, we highlight the usefulness of disease progression modeling in conjunction with natural history data to guide decision making for key trial parameters and to provide a powerful analysis model for determining if a novel therapy is effective in altering disease progression. One common challenge in designing clinical trials in slowly progressive diseases such as GNE Myopathy is the heterogeneity of progression across patient populations within different stages of the disease. A common solution is to choose a single well accepted primary outcome measure and enroll a patient population that is likely to progress the fastest and with the smallest variability on the measure. This solution is often infeasible in rare diseases such as GNE Myopathy and approaches most be developed to use patient resources more efficiently. Disease progression models provide a solution to identify and jointly model multiple outcome measures that span the entire disease spectrum. Joint modeling can then be used as a powerful analysis tool that allows patients within different stages of the disease to provide inference on the potential effect of a novel therapy.

Zoom Link to Follow
14 September 2022

Speaker Itinerary

Topic revision: r1 - 06 Sep 2022, JenaAltstatt

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