Lead Investigator: Vivek Rudrapatna, University of California, San Francisco
Title of Proposal Research: Enhancing inference from real-world data using externally-derived missing data models: a pilot study of Ulcerative Colitis
Vivli Data Request: 4041
Funding Source: NIH NCATS TL1 TR001871
Potential Conflicts of Interest: None
Summary of the Proposed Research:
Real-world evidence is an emerging research area that proposes to use data from non-experimental settings, such as routine clinical care, to guide better decision making. This field has received growing interest in recent years for a variety of reasons, including the realization that randomized controlled trials are too expensive and infeasible to do for every important clinical question. Despite the promise of this field, its progress has been compromised by several major limitations, one of which is the problem of missing data. Data in routine clinical care can be missing for a variety of reasons, and traditional methods for correcting these data can fail in the presence of certain common but pathological patterns of missing data. We propose to overcome these intrinsic limitations of real-world data by exploiting the completeness of clinical trials data. In this pilot study of historical participants in Ulcerative colitis clinical trials, we aim to use the latter to answer two questions: 1) what is our best estimate of a patient’s disease activity in the presence of missing data, 2) given limited resources in real-world settings, what data are more informative and what should doctors be collecting in order to optimally understand how their patients are doing? Outcomes of this work will include 1) statistical models for missing data that may help improve our understanding of real-world treatment effectiveness, and 2) a list of highly informative features that may inform clinical practice concerning data collection. If successful, this pilot may support the utility of pursuing a similar approach for other diseases.
Requested Studies:
A Randomized, Double-Blind, Double-Dummy, Multicenter, Active-Controlled Study to Evaluate the Efficacy and Safety of Vedolizumab IV Compared to Adalimumab SC in Subjects With Ulcerative Colitis
Data Contributor: Takeda
Study ID: NCT02497469
Sponsor ID: MLN0002-3026
A Phase 3, Randomized, Placebo-Controlled, Blinded, Multicenter Study of the Induction and Maintenance of Clinical Response and Remission by MLN0002 in Patients With Moderate to Severe Ulcerative Colitis
Data Contributor: Takeda
Study ID: NCT00783718
Sponsor ID: C13006
Phase III, Multicenter, Randomized, Double-blinded, Placebo-controlled, Parallel-group Study to Examine the Efficacy, Safety, and Pharmacokinetics of Intravenous MLN0002 (300 mg) Infusion in Induction and Maintenance Therapy in Japanese Patients With Moderately or Severely Active Ulcerative Colitis
Data Contributor: Takeda
Study ID: NCT02039505
Sponsor ID: MLN0002/CCT-101
A Multi-Center, Randomized, Double-Blind, Placebo-controlled Study of Adalimumab in Japanese Subjects With Moderately to Severely Active Ulcerative Colitis.
Data Contributor: AbbVie
Study ID: NCT00853099
Sponsor ID: M10-447
A Multicenter, Randomized, Double-blind Placebo-controlled Study of the Human Anti-TNF Monoclonal Antibody Adalimumab for the Induction of Clinical Remission in Subjects With Moderately to Severely Active Ulcerative Colitis
Data Contributor: AbbVie
Study ID: NCT00385736
Sponsor ID: M06-826
A Multicenter, Randomized, Double-blind, Placebo-controlled Study of the Human Anti-TNF Monoclonal Antibody Adalimumab for the Induction and Maintenance of Clinical Remission in Subjects With Moderately to Severely Active Ulcerative Colitis
Data Contributor: AbbVie
Study ID: NCT00408629
Sponsor ID: M06-827
A Phase 3 Multicenter, Placebo-controlled, Double-blind, Randomized-withdrawal Study to Evaluate the Safety and Efficacy of Golimumab Maintenance Therapy, Administered Subcutaneously, in Japanese Subjects With Moderately to Severely Active Ulcerative Colitis
Data Contributor: Johnson & Johnson
Study ID: NCT01863771
Sponsor ID: CR100937
A Phase 3 Multicenter, Randomized, Placebo-controlled, Double-blind Study to Evaluate the Safety and Efficacy of Golimumab Maintenance Therapy, Administered Subcutaneously, in Subjects With Moderately to Severely Active Ulcerative Colitis
Data Contributor: Johnson & Johnson
Study ID: NCT00488631
Sponsor ID: CR014179
A Phase 2/3 Multicenter, Randomized, Placebo-controlled, Double-blind Study to Evaluate the Safety and Efficacy of Golimumab Induction Therapy, Administered Subcutaneously, in Subjects With Moderately to Severely Active Ulcerative Colitis
Data Contributor: Johnson & Johnson
Study ID: NCT00487539
Sponsor ID: CR014176
A Randomized, Placebo-controlled, Double-blind Trial to Evaluate the Safety and Efficacy of Infliximab in Patients With Active Ulcerative Colitis
Data Contributor: Johnson & Johnson
Study ID: NCT00036439
Sponsor ID: CR004777
A Randomized, Placebo-controlled, Double-blind Trial to Evaluate the Safety and Efficacy of Infliximab in Patients With Active Ulcerative Colitis
Data Contributor: Johnson & Johnson
Study ID: NCT00096655
Sponsor ID: CR004783
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Efficacy and Safety of Infliximab in Chinese Subjects With Active Ulcerative Colitis
Data Contributor: Johnson & Johnson
Study ID: NCT01551290
Sponsor ID: CR018769