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Current development in clinical trials: issues old and new
1Department of Biostatistics and Medical Informatics, University of Wisconsin-Madison, WI, U.S.A. demets@biostat.wisc.edu
Abstract:
Clinical trials, especially the randomized clinical trial, have been and will remain the gold standard for the evaluation of new interventions, including pharmaceuticals, biologics, medical devices, procedures, or behavioral modifications. Despite more than five decades of experience, there are still challenges in their design, conduct, monitoring, and analyses. Some of these challenges remain and some are emerging, in part due to the progress in genomics and proteomics. These issues may be statistical, logistical, or a combination. Included are follow-up of subjects who withdraw from intervention, the proposed use of recent adaptive designs, implementing noninferiority designs, reliance on surrogate markers, and gene transfer studies. Comparative effectiveness studies are of increasing interest but present major design and analysis issues. Forces external to the trial are also becoming more common.
Insights
Clinical trials are crucial for evaluating new medical interventions but face ongoing design and analysis challenges. Emerging issues in genomics and proteomics require innovative solutions for robust clinical trial conduct.
Area of Science:
- Clinical Trials and Biostatistics
- Genomics and Proteomics
- Medical Intervention Evaluation
Background:
- Randomized clinical trials (RCTs) are the gold standard for assessing new medical interventions.
- Despite decades of experience, significant challenges persist in clinical trial design, conduct, monitoring, and analysis.
- Advancements in genomics and proteomics introduce new complexities to trial methodologies.
Purpose of the Study:
- To highlight persistent and emerging challenges in clinical trial design and conduct.
- To discuss statistical and logistical issues impacting trial evaluation.
- To address specific challenges including subject withdrawal, adaptive and noninferiority designs, surrogate markers, gene transfer studies, and comparative effectiveness research.
Main Methods:
- Review of established and evolving challenges in clinical trial methodology.
- Discussion of statistical and logistical considerations in trial design and analysis.
- Examination of issues related to subject follow-up, novel designs, and specific study types like gene transfer and comparative effectiveness studies.
Main Results:
- Clinical trials face enduring and new challenges, influenced by scientific progress.
- Issues span statistical, logistical, and ethical domains, affecting trial integrity.
- Specific concerns include managing subject withdrawal, implementing advanced designs, and analyzing complex data from gene transfer and comparative effectiveness studies.
Conclusions:
- Addressing the multifaceted challenges in clinical trials is essential for reliable evaluation of medical interventions.
- Adaptation of trial designs and analytical methods is necessary to incorporate advancements in genomics and proteomics.
- Continued research and methodological innovation are required to overcome existing and emerging obstacles in clinical trial conduct.
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