Skip to main content
  • Textbook
  • © 2019

Efficacy Analysis in Clinical Trials an Update

Efficacy Analysis in an Era of Machine Learning

  • It shows, for the first time, that machine learning methodologies can be used for assessing efficacy data of controlled clinical trials
  • It confirms, that machine learning methodologies provide better sensitivity of testing
  • It confirms, that machine learning methodologies are more informative

Buy it now

Buying options

eBook USD 59.99
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book USD 79.99
Price excludes VAT (USA)
  • Compact, lightweight edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info
Hardcover Book USD 109.99
Price excludes VAT (USA)
  • Durable hardcover edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Other ways to access

This is a preview of subscription content, log in via an institution to check for access.

Table of contents (20 chapters)

  1. Front Matter

    Pages i-xi
  2. Traditional and Machine-Learning Methods for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 1-35
  3. Optimal-Scaling for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 37-53
  4. Ratio-Statistic for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 55-61
  5. Complex-Samples for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 63-73
  6. Bayesian-Network for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 75-85
  7. Evolutionary-Operations for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 87-94
  8. Automatic-Newton-Modeling for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 95-105
  9. High-Risk-Bins for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 107-118
  10. Balanced-Iterative-Reducing-Hierarchy for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 119-135
  11. Cluster-Analysis for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 137-146
  12. Multidimensional-Scaling for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 147-171
  13. Binary Decision-Trees for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 173-184
  14. Continuous Decision-Trees for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 185-194
  15. Automatic-Data-Mining for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 195-210
  16. Support-Vector-Machines for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 211-221
  17. Neural-Networks for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 223-236
  18. Ensembled-Accuracies for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 237-251
  19. Ensembled-Correlations for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 253-267
  20. Gamma-Distributions for Efficacy Analysis

    • Ton J. Cleophas, Aeilko H. Zwinderman
    Pages 269-278

About this book

Machine learning and big data is hot. It is, however, virtually unused in clinical trials. This is so, because randomization is applied to even out multiple variables

Modern medical computer files often involve hundreds of variables like genes and other laboratory values, and computationally intensive methods are required

This is the first publication of clinical trials that have been systematically analyzed with machine learning. In addition, all of the machine learning analyses were tested against traditional analyses. Step by step statistics for self-assessments are included

The authors conclude, that machine learning is often more informative, and provides better sensitivities of testing than traditional analytic methods do


Authors and Affiliations

  • Albert Schweitzer Hospital, Department Medicine, Sliedrecht, The Netherlands

    Ton J. Cleophas

  • Dept. Biostatistics and Epidemiology, Academic Medical Center, Amsterdam, The Netherlands

    Aeilko H. Zwinderman

About the authors

The authors are well-qualified in their field. Professor Zwinderman is past-president of the International Society of Biostatistics (2012-2015), and Professor Cleophas is past-president of the American College of Angiology (2000-2002). 

Professor Zwinderman is one of the Principle Investigators of the Academic Medical Center Amsterdam, and his research is concerned with developing statistical methods for new research designs in biomedical science, particularly integrating omics data, like genomics, proteomics, metabolomics, and analysis tools based on parallel computing and the use of cluster computers and grid computing.   


Professor Cleophas is a member of the Academic Committee of the European College of Pharmaceutical Medicine, that provides, on behalf of 22 European Universities, the Master-ship trainings  "Pharmaceutical Medicine" and "Medicines Development".  


From their expertise theyshould be able to make adequate selections of modern methods for clinical data analysis for the benefit of physicians, students, and investigators. The authors have been working and publishing together for 18 years, and their research can be characterized as a continued effort to demonstrate that clinical data analysis is not mathematics but rather a discipline at the interface of biology and mathematics.


The authors as professors and teachers in statistics at universities in The Netherlands and France for the most part of their lives, are concerned, that their students find regression-analyses harder than any other methodology in statistics. This is serious, because almost all of the novel methodologies in current data mining and data analysis include elements of regression-analysis, and they do hope that the current production "Regression Analysis for Starters and 2nd Levelers" will be a helpful companion for the purpose.
 
Five textbookscomplementary to the current production and written by the same authors are 

Statistics applied to clinical studies 5th edition, 2012, Machine learning in medicine a complete overview, 2015, 
SPSS for starters and 2nd levelers 2nd edition, 2015, 
Clinical data analysis on a pocket calculator 2nd edition, 2016, 
Modern Meta-analysis, 2017
Regression Analysis in Medical Research, 2018 
all of them published by Springer 

Bibliographic Information

  • Book Title: Efficacy Analysis in Clinical Trials an Update

  • Book Subtitle: Efficacy Analysis in an Era of Machine Learning

  • Authors: Ton J. Cleophas, Aeilko H. Zwinderman

  • DOI: https://doi.org/10.1007/978-3-030-19918-0

  • Publisher: Springer Cham

  • eBook Packages: Biomedical and Life Sciences, Biomedical and Life Sciences (R0)

  • Copyright Information: Springer Nature Switzerland AG 2019

  • Hardcover ISBN: 978-3-030-19917-3Published: 25 September 2019

  • Softcover ISBN: 978-3-030-19920-3Published: 25 September 2020

  • eBook ISBN: 978-3-030-19918-0Published: 03 September 2019

  • Edition Number: 1

  • Number of Pages: XI, 304

  • Number of Illustrations: 251 b/w illustrations, 44 illustrations in colour

  • Topics: Biomedicine general, Statistics for Life Sciences, Medicine, Health Sciences, Biostatistics

Buy it now

Buying options

eBook USD 59.99
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book USD 79.99
Price excludes VAT (USA)
  • Compact, lightweight edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info
Hardcover Book USD 109.99
Price excludes VAT (USA)
  • Durable hardcover edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Other ways to access