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The goal of a controlled clinical trial is to compare the effects of interventions on outcomes of interest. This chapter considers the methods to limit bias and random error at each stage of a clinical trial-design, conduct, analysis and interpretation of results. Many aspects of study design relate to control of bias. The one of greatest importance is the method of assignment to treatment. Study assessments that incorporate some element of subjectivity can also be centralized. Many trials rely on a central adjudication group to make outcome assessments for all subjects in a study. In most studies, the treatments are compared with regard to multiple outcomes. From sample size considerations to central pathology review, from eligibility reviews to interim monitoring plans, all methodological considerations relate in one way or other to minimizing the potential for bias and reducing random error.
Translating laboratory discoveries into successful therapeutics can be difficult. Clinical Trials in Neurology aims to improve the efficiency of clinical trials and the development of interventions in order to enhance the development of new treatments for neurologic diseases. It introduces the reader to the key concepts underpinning trials in the neurosciences. This volume tackles the challenges of developing therapies for neurologic disorders from measurement of agents in the nervous system to the progression of clinical signs and symptoms through illustrating specific study designs and their applications to different therapeutic areas. Clinical Trials in Neurology covers key issues in Phase I, II and III clinical trials, as well as post-marketing safety surveillance. Topics addressed include regulatory and implementation issues, outcome measures and common problems in drug development. Written by a multidisciplinary team, this comprehensive guide is essential reading for neurologists, psychiatrists, neurosurgeons, neuroscientists, statisticians and clinical researchers in the pharmaceutical industry.
This chapter discusses the broad categories of clinical investigations used in post-market drug safety assessment. It presents the three main methods of clinical post-marketing safety assessment: case reports and case series; observational epidemiological studies; and clinical trials. Active surveillance systems are also being explored to identify and examine drug safety issues. Drug safety active surveillance systems, which take advantage of large repositories of automated healthcare data, are now being developed and tested by multiple organizations. The two most common observational epidemiological study designs are the case-control design and the cohort design. The majority of clinical trials are performed primarily to assess the efficacy of a product. The design of a post-marketing clinical trial testing a safety hypothesis is often an active-controlled trial that uses a non-inferiority study design. Relative to observational epidemiological studies, clinical trials designed to answer drug safety questions are usually more costly and more time-consuming.
This chapter provides an overview of outcome measures in neurology clinical trials, including developing a conceptual endpoint model, role and use of biomarkers, and considerations on how to select, use and interpret them in the context of early-stage clinical trial design. Early stage clinical trials (phase 1-2) often employ biomarker targets for proof of concept or therapeutic validation. Therapeutic development programs can be viewed as in the learn zone and confirm zone, with confirmation occurring in the phase 3 trial designed to test clinical efficacy against a standard or placebo. Structural imaging with MRI or computed tomography (CT) has been used as both an entry criteria into clinical trials and as an outcome measure. MRI has frequently been used as a measure of treatment response of multiple sclerosis (MS) patients. Researchers should define the role each endpoint is intended to play in the clinical trial.