DIA BSWG KOL Lecture Series, L27

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Bayesian Shared Parameter Analysis of Mortality and Function within an Adaptive Platform Trial for ALS

Melanie Quintana PhD
Director and Senior Statistical Scientist Berry Consultants

Abstract: The HEALEY ALS Platform Trial is simultaneously testing multiple novel treatment regimens in persons with ALS with the goal of rapidly finding novel treatments. Within ALS, regulatory guidance recommends an integrated analysis of survival and function to assess efficacy of novel therapies. The traditional approach is a joint rank analysis of survival and function. This approach is non-parametric but lacks a clinically meaningful summary of treatment benefit. Additionally, within the platform trial setting there are complexities, such as modeling potential differences in shared placebo participants over time and across regimens, that require a more parametric modeling framework. Here, we describe the primary analysis model for the HEALEY ALS Platform trial, a novel approach for integrating mortality and function into a single primary analysis model that provides clinically meaningful and interpretable summaries of treatment benefit and allows for dynamic sharing of placebos across treatment regimens and over time.

Bio: Melanie Quintana is a Director and Senior Statistical Scientist at Berry Consultants, where she is an expert in utilizing Bayesian statistics to design innovative clinical trials and answer complex clinical questions across a wide range of therapeutic areas. Her work includes numerous examples in designing platform trials and clinical trials in rare and progressive disease with a focus on developing models of disease progression to design better and more powerful clinical trials. Before joining Berry Consultants, she earned her Ph.D. in Statistics from Duke University and went on to pursue a Postdoc in Biostatistics at The University of Southern California. While at Duke and USC, she worked closely with collaborators within multiple large studies and consortiums around the nation to develop and implement analytical strategies to assess genetic risk factors for various complex diseases. This work led to a passion for consulting and collaborating with clinical experts to find analytical solutions to hard medical problems, which has only expanded since joining Berry Consultants in 2013.

Slides will be provided before the presentation at: http://www.bayesianscientific.org/kol-lecture-series/

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