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Biostatistics Seminar Series: 11/16, Monday 2pm, “Causal Inference in Rare Diseases, In Practice” by Dr. Rima Izem of Children’s National Research Institute and the George Washington University.


Dear Colleagues,

The Division of Biostatistics at the Department of Preventive Medicine invites you to attend the following seminar.

Date: Monday, 11/16/2020

Time: 2 P.M., Central Time (US and Canada)

ZOOM Virtual Room Connection:  

Join from PC, Mac, Linux, iOS or Android: https://tennessee.zoom.us/j/96950930905

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Or Telephone:


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    Meeting ID: 969 5093 0905

    International numbers available: https://tennessee.zoom.us/u/agKiFakW9

Or an H.323/SIP room system:

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    Meeting ID: 969 5093 0905

    SIP: 96950930905@zoomcrc.com

Speaker: Dr. Rima Izem (Children’s National Research Institute and the George Washington University)

Title: “Causal Inference in Rare Diseases, In Practice”

Abstract: This presentation will give an overview of how researchers can measure the impact of medical interventions on patients with rare diseases using longitudinal data. In the United States, rare diseases affect 30 million patients in approximately 7000 disease areas. Causal inference to inform therapy decisions in this area is challenged by several factors: the patient population is small, the genotype or phenotype is variable, and the disease pathways can be uncharacterized or lengthy. These challenges are not specific to the rare disease setting as they apply more largely to answering causal inference questions in the era of precision medicine where public health decisions rely on subgroups of sample sizes. In 2019, the Food and Drug Administration published documents guiding best practices on data collection, study design and use of registry data in the rare diseases setting. Over the past few years, a few algorithms were published to guide researchers to appropriate study design. This presentation will give an overview of best design practices and innovative analysis methods and illustrate their use with case studies in rare diseases.


About the speaker:
Dr. Rima Izem is an Associate Professor of Pediatrics at Children’s National Research Institute and George Washington University. At Children’s National, she supports different aspects of clinical research as they relate to study design and statistical analyses of clinical and epidemiological studies in the pediatric population. Her research interests include study design in rare diseases, causal inference, and signal detection in large electronic healthcare databases. She received her PhD in Statistics from the University of North Carolina at Chapel Hill.

(Speaker’s webpage: https://childrensnational.org/research-and-education/about-cri/faculty-and-leadership-directory/izem-rima)

We look forward to seeing you all among us. 

Chi-Yang Chiu, Ph.D.

Assistant Professor of Biostatistics

Department of Preventive Medicine, UTHSC