Chapter 40. Clinical Trials in Rare Diseases

  1. David Machin1,2,3,
  2. Simon Day4 and
  3. Sylvan Green5
  1. Simon Day

Published Online: 17 MAY 2007

DOI: 10.1002/9780470010167.ch40

Textbook of Clinical Trials, Second Edition

Textbook of Clinical Trials, Second Edition

How to Cite

Day, S. (2006) Clinical Trials in Rare Diseases, in Textbook of Clinical Trials, Second Edition (eds D. Machin, S. Day and S. Green), John Wiley & Sons, Ltd, Chichester, UK. doi: 10.1002/9780470010167.ch40

Editor Information

  1. 1

    Division of Clinical Trials and Epidemiological Sciences, National Cancer Centre Singapore, 11 Hospital Drive, Singapore 16910, Singapore

  2. 2

    UK Children's Cancer Study Group, University of Leicester, UK

  3. 3

    Medical Statistics Group, School of Health and Related Sciences, University of Sheffield, UK

  4. 4

    Licensing Division, Medicines and Healthcare products Regulatory Agency, Room 13–205 Market Towers, 1 Nine Elms Lane, London SW8 5NQ, UK

  5. 5

    Arizona Cancer Centre, Tucson, Arizona, USA

Author Information

  1. Licensing Division, Medicines and Healthcare products Regulatory Agency, Room 13–205 Market Towers, 1 Nine Elms Lane, London SW8 5NQ, UK

Publication History

  1. Published Online: 17 MAY 2007
  2. Published Print: 24 NOV 2006

ISBN Information

Print ISBN: 9780470010143

Online ISBN: 9780470010167

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Keywords:

  • clinical trials in rare diseases;
  • trial and patient registers;
  • slowing disease progression;
  • disease and patient definitions;
  • biomarkers and surrogate endpoints

Summary

This chapter contains sections titled:

  • Introduction

  • Administrative Issues

  • How Convincing is the Evidence?

  • Disease and Patient Definitions

  • Pharmacological Considerations

  • Purpose of Treatment

  • Choice of Endpoints

  • Comparator Groups

  • The Size of Treatment Effect

  • Statistical Methods

  • Summary and Conclusions

  • Acknowledgements

  • References