Lead Investigator: Jiajun Yan, McMaster University
Title of Proposal Research: Statistical Methods for Analyzing EuroQol 5 Dimension 5 (EQ-5D) in Randomized Clinical Trials
Vivli Data Request: 9253
Funding Source: None
Potential Conflicts of Interest: None
Summary of the Proposed Research:
The EQ-5D (EuroQol five-dimensions) questionnaire is a patient-reported outcome that measures individual’s health-related quality of life (HRQoL) from 5 dimensions, including mobility (MO), self-care (SC), usual activities (UA), pain/discomfort (PD), and anxiety/depression (AD). The original version EQ-5D has three response options indicating no, some, and extreme problems in each of the five dimensions (the EQ-5D-3L (EuroQol five-dimension three-level)), which were later expanded into 5 levels: no, slight, moderate, severe, and extreme problems (the EQ-5D-5L (EuroQol five-dimension five-level)). The EQ-5D questionnaire has been widely collected in randomized clinical trials (RCTs) and extensively used in economic evaluation for reimbursement decision across various disease areas. However, using EQ-5D to evaluate treatment efficacy on HRQoL is rather limited, partially due to the lack of methodological guideline.
There are guidelines on analyzing and reporting EQ-5D data, but these mostly focus on non-controlled studies where EQ-5D data is only collected once. However, in RCTs, with the aim of testing the effect of the treatment , EQ-5D data are usually collected at multiple timepoints during the trial. Our recent research shows that there are significant variations in the statistical methods that have been used to analyze EQ-5D data in the RCTs.
Through access to published RCTs where the EQ-5D was collected, we plan to apply and compare commonly used methods and new methods to analyze the EQ-5D data across a wide range of disease areas and trial designs. We will examine model assumptions, evaluate model performance/goodness of fit, and compare model results in each trial analysis.
This project will produce empirical (observed) evidence in comparing statistical models for estimating treatment effect on the EQ-5D. Furthermore, the output from this research program can be used to develop practical guidance on analyzing the EQ-5D in the RCT setting. Such guidance can potentially increase awareness and usage of EQ-5D to assess treatment effect on patients’ HRQoL.
Requested Studies:
PHIRST-1: Randomized, Double-Blind, Placebo-Controlled Phase 3 Study of the Phosphodiesterase Type 5 (PDE5) Inhibitor Tadalafil in the Treatment in Patients With Pulmonary Arterial Hypertension
Data Contributor: Lilly
Study ID: NCT00125918
Sponsor ID: 10303
A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study, With a Vedolizumab IV Reference Arm, to Evaluate the Efficacy and Safety of Vedolizumab Subcutaneous as Maintenance Therapy in Subjects With Moderately to Severely Active Ulcerative Colitis Who Achieved Clinical Response Following Open-Label Vedolizumab Intravenous Therapy
Data Contributor: Takeda
Study ID: NCT02611830
Sponsor ID: MLN0002SC-3027
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
A Randomized, Double-Blind, Active-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Baricitinib (LY3009104) in Patients With Moderately to Severely Active Rheumatoid Arthritis Who Have Had Limited or No Treatment With Disease-Modifying Antirheumatic Drugs
Data Contributor: Lilly
Study ID: NCT01711359
Sponsor ID: 14062
A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study Evaluating the Efficacy and Safety of Baricitinib (LY3009104) in Patients With Moderately to Severely Active Rheumatoid Arthritis Who Have Had an Inadequate Response to Tumor Necrosis Factor Inhibitors
Data Contributor: Lilly
Study ID: NCT01721044
Sponsor ID: 14058
A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Baricitinib (LY3009104) in Patients With Inadequate Response to Conventional Disease-Modifying Antirheumatic Drugs With Moderately to Severely Active Rheumatoid Arthritis
Data Contributor: Lilly
Study ID: NCT01721057
Sponsor ID: 14059
A Randomized, Controlled, Multicenter, Open Label Study With Blinded Assessment of the Efficacy of the Humanized Anti-IL-23p19 Risankizumab Compared to FUMADERM® in Subjects With Moderate to Severe Plaque Psoriasis Who Are Naïve to and Candidates for Systemic Therapy
Data Contributor: AbbVie
Study ID: NCT03255382
Sponsor ID: M16-178
A 52-Week Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Ixekizumab (LY2439821) in bDMARD Naive Patients With Nonradiographic Axial Spondyloarthritis
Data Contributor: Lilly
Study ID: NCT02757352
Sponsor ID: 16180
Multicenter, Double-blind, Randomized, Parallel-group, Monotherapy, Active-control Study to Determine the Efficacy and Safety of Daclizumab High Yield Process (DAC HYP) Versus Avonex® (Interferon β 1a) in Patients With Relapsing-Remitting Multiple Sclerosis
Data Contributor: Biogen
Study ID: NCT01064401
Sponsor ID: 205MS301
A Phase 3 Randomized, Rater-Blinded Study Comparing Two Annual Cycles of Intravenous Alemtuzumab to Three-Times Weekly Subcutaneous Interferon Beta-1a (Rebif®) in Treatment-Naïve Patients With Relapsing-Remitting Multiple Sclerosis
Data Contributor: Sanofi
Study ID: NCT00530348
Sponsor ID: CAMMS323
A Phase 3, Randomized, Rater- and Dose-Blinded Study Comparing Two Annual Cycles of Intravenous Low- and High-Dose Alemtuzumab to Three-Times Weekly Subcutaneous Interferon Beta 1a (Rebif®) in Patients With Relapsing Remitting Multiple Sclerosis Who Have Relapsed On Therapy
Data Contributor: Sanofi
Study ID: NCT00548405
Sponsor ID: CAMMS32400507
A Randomized, Open Label, Multicenter Study of Cabazitaxel Versus an Androgen Receptor (AR)-Targeted Agent (Abiraterone or Enzalutamide) in mCRPC Patients Previously Treated With Docetaxel and Who Rapidly Failed a Prior AR-targeted Agent (CARD)
Data Contributor: Sanofi
Study ID: NCT02485691
Sponsor ID: LPS14201
A Phase 3, Randomized Study Of SU011248 Versus Interferon-Alfa As First-Line Systemic Therapy For Patients With Metastatic Renal Cell Carcinoma
Data Contributor: Pfizer Inc.
Study ID: NCT00083889
Sponsor ID: A6181034