Posted by Nanhua Zhang on March 27, 2023
Study Authors
Nanhua Zhang, Steven J Steiner, Robert Baldassano, Shiran Chen, Stanley Cohen, Michael D Kappelman, Shehzad Saeed, Laurie S Conklin, Richard Strauss, Sheri Volger, Eileen King, and Kim Hung Lo
Study Background
With the rich real-world data (RWD) in the ImproveCareNow (ICN) registry, we are working with a pharmaceutical company to establish how well and how safe some medicines are for pediatric inflammatory bowel disease (IBD) patients. Also, this study will be part of the evidence that supports how research studies are submitted to the United States Food and Drug Administration (FDA). We have developed a detailed research design and statistical analysis plan for the real world study to look at some challenges using RWD. One of the challenges is that there is missing data in the ICN registry dataset. To be able to figure out how best to handle this, we conducted a study to investigate which statistical models, using a technique called multiple imputation (MI), worked best to get the most accurate results.
Multiple imputation is a way to fill in the missing values multiple times based on statistical models to create several complete datasets, and analysts can then combine the results from each imputed dataset. An important outcome of this study will be that it has potential to speed up the FDA decision-making process which will make effective treatment available to more pediatric IBD patients sooner. It will also save millions of dollars on confirmatory clinical trials that would have had to take place.
Study Overview
We are working with a pharmaceutical company to leverage the ICN registry data to study the effectiveness of a medicine in real world setting. The primary outcome we are looking at is called the “short Pediatric Crohn’s Disease Activity Index” or sPCDAI. The sPCDAI is used to measure how active Crohn’s disease is in a pediatric patient. However, this measure is missing in some cases in the ICN registry dataset. To address this, the study published in the Journal of Comparative Effectiveness Research compared how well different statistical multiple imputation (MI) methods worked to get the most accurate results. The participants in this study included moderate-to-severe pediatric Crohn’s disease patients in ICN that had never used the biologic medicine called ustekinumab.
Study Results—What does it all mean?
Our study shows that multiple imputation can minimize the impact of missing data in real world studies using the ICN registry data. This is an important step to leverage RWD to support FDA decision-making. The future of research involving registry data is bright and our study may serve as a model for other RWD analyses.
An important outcome of this study will be that it has potential to speed up the FDA decision-making process which will make effective treatment available to more pediatric IBD patients sooner.
Study status
Published in February 2023. You can locate it:
- In the Journal of Comparative Effectiveness Research
- In the 2023 Publications listings on our website
About ICN Research Explained
We believe that in order to truly outsmart IBD, the questions we ask and the answers we find must be generated by and be useful to the people whose lives they will impact – patients and their families. ImproveCareNow (ICN) research is prioritized based on what matters most to patients and parents, and we are committed to sharing our results so everyone can understand and take advantage of what’s been learned. The ICN Research Committee has teamed up with patients and parents/caregivers to create and share accessible summaries of completed research called ICN Research Explained
Prepared by: Nanhua Zhang
Reviewed by: Lisa Pitch, Parent Reviewer, ICN Research Committee
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