David Madigan - Towards systematic evidence generation from real-world healthcare data

Towards systematic evidence generation from real-world healthcare data
By David Madigan

Abstract:
In practice, our learning healthcare system relies primarily on observational studies generating one effect estimate at a time using customized study designs with unknown operating characteristics and publishing – or not – one estimate at a time. When we investigate the distribution of estimates that this process has produced, we see clear evidence of its shortcomings, including an apparent over-abundance of estimates where the confidence interval does not include one (i.e. statistically significant effects). We propose a standardized process for performing observational research that can be evaluated, calibrated and applied at scale to generate a more reliable and complete evidence base than previously possible, fostering a truly learning healthcare system. We demonstrate this new paradigm by generating evidence about all pairwise comparisons of treatments for hypertension for a relevant set of health outcomes using nine large electronic healthcare record databases from 3 continents. In total, we estimate more than 1M hazard ratios, each using a comparative effectiveness study design and propensity score stratification on par with current state-of-the-art, albeit one-off, observational studies. Moreover, the process enables us to employ negative and positive controls to evaluate and calibrate estimates ensuring, for example, that the 95% confidence interval includes the true effect size approximately 95% of time. The result set consistently reflects current established knowledge where known, and its distribution shows no evidence of the faults of the current process.

Bio:
David Madigan is Professor of Statistics at Columbia University in New York City and Dean Emeritus of Arts and Sciences. He received a bachelor’s degree in Mathematical Sciences and a Ph.D. in Statistics, both from Trinity College Dublin. He has previously worked for AT&T Inc., Soliloquy Inc., the University of Washington, Rutgers University, and SkillSoft, Inc. He has over 200 publications in such areas as Bayesian statistics, text mining, Monte Carlo methods, pharmacovigilance and probabilistic graphical models. He is an elected Fellow of the American Statistical Association, the Institute of Mathematical Statistics, and the American Association for the Advancement of Science. He has served terms as Editor-in-Chief of Statistical Science and of Statistical Analysis and Data Mining – the ASA Data Science Journal.

Twitter: @davidbmadigan

Presented at the 2019 New York R Conference (May 10th, 2019)