Methods for the analysis of multiple endpoints in small populations: A review.

08:00 EDT 9th July 2018 | BioPortfolio

Summary of "Methods for the analysis of multiple endpoints in small populations: A review."

While current guidelines generally recommend single endpoints for primary analyses of confirmatory clinical trials, it is recognized that certain settings require inference on multiple endpoints for comprehensive conclusions on treatment effects. Furthermore, combining treatment effect estimates from several outcome measures can increase the statistical power of tests. Such an efficient use of resources is of special relevance for trials in small populations. This paper reviews approaches based on a combination of test statistics or measurements across endpoints as well as multiple testing procedures that allow for confirmatory conclusions on individual endpoints. We especially focus on feasibility in trials with small sample sizes and do not solely rely on asymptotic considerations. A systematic literature search in the Scopus database, supplemented by a manual search, was performed to identify research papers on analysis methods for multiple endpoints with relevance to small populations. The identified methods were grouped into approaches that combine endpoints into a single measure to increase the power of statistical tests and methods to investigate differential treatment effects in several individual endpoints by multiple testing.


Journal Details

This article was published in the following journal.

Name: Journal of biopharmaceutical statistics
ISSN: 1520-5711
Pages: 1-29


DeepDyve research library

PubMed Articles [43348 Associated PubMed Articles listed on BioPortfolio]

Considerations of multiple imputation approaches for handling missing data in clinical trials.

Missing data exist in all clinical trials and missing data issue is a very serious issue in terms of the interpretability of the trial results. There is no universally applicable solution for all miss...

Recent advances in methodology for clinical trials in small populations: the InSPiRe project.

Where there are a limited number of patients, such as in a rare disease, clinical trials in these small populations present several challenges, including statistical issues. This led to an EU FP7 call...

Comparison of three sample size calculation methods for non-inferiority vaccine trials with multiple continuous co-primary endpoints.

Clinical trials that study immunogenicity of combination vaccines often have less power than desirable. To make up for the reduction in statistical power at the study level, researchers have to increa...

Over three decades study populations in progressive multiple sclerosis have become older and more disabled, but have lower on-trial progression rates: A systematic review and meta-analysis of 43 randomised placebo-controlled trials.

Progression is the major driver of disability and cost in multiple sclerosis (MS). However, the search for treatments in progressive multiple sclerosis (PMS) has not mirrored the success in relapsing ...

Competition lifespan survival analysis in the Norwegian-Swedish Coldblooded Trotter racehorse.

Studies of large racing populations have established clear differences in the career profile of stallions, mares, and geldings. Multiple studies have also demonstrated positive effects on racing caree...

Clinical Trials [13515 Associated Clinical Trials listed on BioPortfolio]

Immune Response and Cytomegalovirus in Intensive Care Unit (ICU) Patients

This prospective study evaluate the immune status of patients admitted in ICU.CMV remains dormant in the body, but in people with immune deficiency, CMV could reactivate and cause life-thr...

A Review of Low-Dose CT Lung Cancer Screenings in a Community-Based Healthcare System With High Incidence

An analysis of Low-Dose Cat Scan(LDCT) Screenings for Lung Cancer completed within the St. Elizabeth system from January 2015 until February 2018. The study investigator, or designee(s), w...

Vocal Acoustic Biomarkers in Depression

This Phase II SBIR study will replicate pilot study methods establishing computer-automated methods for assessing depression severity using interactive voice response system technology and...

The Role of Sulfur Amino Acids in Risk of Kwashiorkor

This observational cross-sectional study is investigating if young children in populations with higher prevalence of kwashiorkor malnutrition have lower dietary sulfur amino acid intake th...

Collecting and Analyzing Tissue Samples From Patients Undergoing Surgery for Non-Small Cell Lung Cancer

RATIONALE: Studying samples of tumor tissue from patients with cancer in the laboratory may help doctors learn more about changes that may occur in RNA and identify biomarkers related to c...

Medical and Biotech [MESH] Definitions

Formal programs for assessing drug prescription against some standard. Drug utilization review may consider clinical appropriateness, cost effectiveness, and, in some cases, outcomes. Review is usually retrospective, but some analysis may be done before drugs are dispensed (as in computer systems which advise physicians when prescriptions are entered). Drug utilization review is mandated for Medicaid programs beginning in 1993.

Research techniques that focus on study designs and data gathering methods in human and animal populations.

Methods for performing three-dimensional measurement and motion analysis using stereoscopic radiographs.

A method of analyzing the variation in utilization of health care in small geographic or demographic areas. It often studies, for example, the usage rates for a given service or procedure in several small areas, documenting the variation among the areas. By comparing high- and low-use areas, the analysis attempts to determine whether there is a pattern to such use and to identify variables that are associated with and contribute to the variation.

Review of the medical necessity of hospital or other health facility admissions, upon or within a short time following an admission, and periodic review of services provided during the course of treatment.

Quick Search


DeepDyve research library

Relevant Topic

Clinical Approvals
Clinical trials are a set of procedures in medical research conducted to allow safety (or more specifically, information about adverse drug reactions and adverse effects of other treatments) and efficacy data to be collected for health interventions (e.g...

Searches Linking to this Article