Design and conduct of clinical trials for breast cancer

Vicki Klimberg, Yu Shyr, Thomas Wells

Research output: Chapter in Book/Report/Conference proceedingChapter

Abstract

The National Institutes of Health (NIH) defines a clinical trial as "a research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes." There are generally two types of clinical trials: controlled and uncontrolled. Uncontrolled trials lack a comparison group. Controlled trials involve two or more study treatments, at least one of which is a control treatment (i.e., a standard against which new treatments are measured). The main focus of this chapter is interventional trials including cohort (cross-sectional, case-control) studies and interventional trials, that is, intervention(s) applied to the study population synonymous with clinical trials in most texts. Dr. William Thomas Beaver was the clinical pharmacologist at Georgetown University who is credited with drafting the initial regulations defining "adequate and controlled" clinical studies. "Clinical trials are systematic experiments performed on human beings for the purpose of assessing the safety and/or efficacy of treatments or care procedures. The function of the controlled clinical trial is not the 'discovery' of a new drug or therapy. Discoveries are made in the animal laboratory, by chance observation, or at the bedside by an astute clinician. The function of the formal controlled clinical trial is to separate the relative handful of discoveries which prove to be true advances in therapy from a legion of false leads and unverifiable clinical impressions, and to delineate in a scientific way the extent of and the limitations which attend the effectiveness of drugs".

Original languageEnglish (US)
Title of host publicationThe Breast
Subtitle of host publicationComprehensive Management of Benign and Malignant Diseases
PublisherElsevier Inc.
Pages362-376.e1
ISBN (Print)9780323359559
DOIs
StatePublished - Aug 24 2017

Fingerprint

Controlled Clinical Trials
Clinical Trials
Breast Neoplasms
National Institutes of Health (U.S.)
Laboratory Animals
Therapeutics
Case-Control Studies
Rodentia
Placebos
Observation
Safety
Drug Therapy
Health
Research
Pharmaceutical Preparations
Population

Keywords

  • And IV
  • Case-control
  • Cohort
  • Cross-sectional
  • Crossover clinical trials
  • II
  • III
  • Interventional
  • Phase I
  • Power analysis
  • Sample size determination

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Klimberg, V., Shyr, Y., & Wells, T. (2017). Design and conduct of clinical trials for breast cancer. In The Breast: Comprehensive Management of Benign and Malignant Diseases (pp. 362-376.e1). Elsevier Inc.. https://doi.org/10.1016/B978-0-323-35955-9.00027-1

Design and conduct of clinical trials for breast cancer. / Klimberg, Vicki; Shyr, Yu; Wells, Thomas.

The Breast: Comprehensive Management of Benign and Malignant Diseases. Elsevier Inc., 2017. p. 362-376.e1.

Research output: Chapter in Book/Report/Conference proceedingChapter

Klimberg, V, Shyr, Y & Wells, T 2017, Design and conduct of clinical trials for breast cancer. in The Breast: Comprehensive Management of Benign and Malignant Diseases. Elsevier Inc., pp. 362-376.e1. https://doi.org/10.1016/B978-0-323-35955-9.00027-1
Klimberg V, Shyr Y, Wells T. Design and conduct of clinical trials for breast cancer. In The Breast: Comprehensive Management of Benign and Malignant Diseases. Elsevier Inc. 2017. p. 362-376.e1 https://doi.org/10.1016/B978-0-323-35955-9.00027-1
Klimberg, Vicki ; Shyr, Yu ; Wells, Thomas. / Design and conduct of clinical trials for breast cancer. The Breast: Comprehensive Management of Benign and Malignant Diseases. Elsevier Inc., 2017. pp. 362-376.e1
@inbook{67dcd1f6e5ff401d99995a9c0b0c78f5,
title = "Design and conduct of clinical trials for breast cancer",
abstract = "The National Institutes of Health (NIH) defines a clinical trial as {"}a research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes.{"} There are generally two types of clinical trials: controlled and uncontrolled. Uncontrolled trials lack a comparison group. Controlled trials involve two or more study treatments, at least one of which is a control treatment (i.e., a standard against which new treatments are measured). The main focus of this chapter is interventional trials including cohort (cross-sectional, case-control) studies and interventional trials, that is, intervention(s) applied to the study population synonymous with clinical trials in most texts. Dr. William Thomas Beaver was the clinical pharmacologist at Georgetown University who is credited with drafting the initial regulations defining {"}adequate and controlled{"} clinical studies. {"}Clinical trials are systematic experiments performed on human beings for the purpose of assessing the safety and/or efficacy of treatments or care procedures. The function of the controlled clinical trial is not the 'discovery' of a new drug or therapy. Discoveries are made in the animal laboratory, by chance observation, or at the bedside by an astute clinician. The function of the formal controlled clinical trial is to separate the relative handful of discoveries which prove to be true advances in therapy from a legion of false leads and unverifiable clinical impressions, and to delineate in a scientific way the extent of and the limitations which attend the effectiveness of drugs{"}.",
keywords = "And IV, Case-control, Cohort, Cross-sectional, Crossover clinical trials, II, III, Interventional, Phase I, Power analysis, Sample size determination",
author = "Vicki Klimberg and Yu Shyr and Thomas Wells",
year = "2017",
month = "8",
day = "24",
doi = "10.1016/B978-0-323-35955-9.00027-1",
language = "English (US)",
isbn = "9780323359559",
pages = "362--376.e1",
booktitle = "The Breast",
publisher = "Elsevier Inc.",

}

TY - CHAP

T1 - Design and conduct of clinical trials for breast cancer

AU - Klimberg, Vicki

AU - Shyr, Yu

AU - Wells, Thomas

PY - 2017/8/24

Y1 - 2017/8/24

N2 - The National Institutes of Health (NIH) defines a clinical trial as "a research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes." There are generally two types of clinical trials: controlled and uncontrolled. Uncontrolled trials lack a comparison group. Controlled trials involve two or more study treatments, at least one of which is a control treatment (i.e., a standard against which new treatments are measured). The main focus of this chapter is interventional trials including cohort (cross-sectional, case-control) studies and interventional trials, that is, intervention(s) applied to the study population synonymous with clinical trials in most texts. Dr. William Thomas Beaver was the clinical pharmacologist at Georgetown University who is credited with drafting the initial regulations defining "adequate and controlled" clinical studies. "Clinical trials are systematic experiments performed on human beings for the purpose of assessing the safety and/or efficacy of treatments or care procedures. The function of the controlled clinical trial is not the 'discovery' of a new drug or therapy. Discoveries are made in the animal laboratory, by chance observation, or at the bedside by an astute clinician. The function of the formal controlled clinical trial is to separate the relative handful of discoveries which prove to be true advances in therapy from a legion of false leads and unverifiable clinical impressions, and to delineate in a scientific way the extent of and the limitations which attend the effectiveness of drugs".

AB - The National Institutes of Health (NIH) defines a clinical trial as "a research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes." There are generally two types of clinical trials: controlled and uncontrolled. Uncontrolled trials lack a comparison group. Controlled trials involve two or more study treatments, at least one of which is a control treatment (i.e., a standard against which new treatments are measured). The main focus of this chapter is interventional trials including cohort (cross-sectional, case-control) studies and interventional trials, that is, intervention(s) applied to the study population synonymous with clinical trials in most texts. Dr. William Thomas Beaver was the clinical pharmacologist at Georgetown University who is credited with drafting the initial regulations defining "adequate and controlled" clinical studies. "Clinical trials are systematic experiments performed on human beings for the purpose of assessing the safety and/or efficacy of treatments or care procedures. The function of the controlled clinical trial is not the 'discovery' of a new drug or therapy. Discoveries are made in the animal laboratory, by chance observation, or at the bedside by an astute clinician. The function of the formal controlled clinical trial is to separate the relative handful of discoveries which prove to be true advances in therapy from a legion of false leads and unverifiable clinical impressions, and to delineate in a scientific way the extent of and the limitations which attend the effectiveness of drugs".

KW - And IV

KW - Case-control

KW - Cohort

KW - Cross-sectional

KW - Crossover clinical trials

KW - II

KW - III

KW - Interventional

KW - Phase I

KW - Power analysis

KW - Sample size determination

UR - http://www.scopus.com/inward/record.url?scp=85054317071&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85054317071&partnerID=8YFLogxK

U2 - 10.1016/B978-0-323-35955-9.00027-1

DO - 10.1016/B978-0-323-35955-9.00027-1

M3 - Chapter

SN - 9780323359559

SP - 362-376.e1

BT - The Breast

PB - Elsevier Inc.

ER -