Patient-Driven eData

Event driven clinical trial design

Most errors in clinical trials are a result of poor planning. Fancy statistical methods cannot rescue design flaws. Thus careful planning with clear foresight is crucial. Issues in trial conduct and analyses should be anticipated during trial design and thoughtfully addressed. Fundamental clinical trial design issues are discussed.

Keywords: p-value, confidence intervals, intent-to-treat, missing data, multiplicity, subgroup analyses, causation

1. Introduction

The objective of clinical trials is to establish the effect of an intervention. Treatment effects are efficiently isolated by controlling for bias and confounding and by minimizing variation. Key features of clinical trials that are used to meet this objective are randomization (possibly with stratification), adherence to intent-to-treat (ITT) principles, blinding, prospective evaluation, and use of a control group. Compared to other types of study designs (e.g., case-control studies, cohort studies, case reports), randomized trials have high validity but are more difficult and expensive to conduct.

2. Design Issues

There are many issues that must be considered when designing clinical trials. Fundamental issues including clearly defining the research question, minimizing variation, randomization and stratification, blinding, placebos/shams, selection of a control group, selection of the target population, the selection of endpoints, sample size, and planning for interim analyses will be discussed and common terms are defined.

Terms in clinical trial design

2.1 What is the question?

The design of every clinical trial starts with a primary clinical research question. Clarity and understanding of the research question can require much deliberation often entailing a transition from a vague concept (e.g., “to see if the drug works” or “to look at the neuro-biology of the drug”) to a particular hypothesis that can be tested or a quantity that can be estimated using specific data collection instruments with a particular duration of therapy. Secondary research questions may also be of interest but the trial design usually is constructed to address the primary research question.

BMC Medical Research Methodology at the 35th Annual Conference of the ..  — BMC Pediatrics
The conference will focus on issues such as design and analysis of clinical trials, methods in biostatistics and development of clinical prediction models.

Wiley-Interscience Bayesian Methods and Ethics in a Clinical Trial Design
Book (Wiley-Interscience)
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Popular Q&A

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When Is IRB Review of Clinical Trial Websites Required?

When information posted on a clinical trial website goes beyond directory listings with basic descriptive information, such information is considered part of the informed consent process and therefore requires IRB review and approval. Basic descriptive information includes:

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