Efficacy Assessment

  • J. Rick Turner


In addition to having an acceptable safety profile, an investigational drug needs to display beneficial therapeutic effects for it to receive marketing approval, that is, it needs to have a favorable benefit–risk profile. Therapeutic benefit, operationalized as efficacy, is evaluated in therapeutic exploratory and therapeutic confirmatory trials, trials in which the subjects have the disease or condition of biological concern.


Interim Analysis Adaptive Design Statistical Analysis Plan Equivalence Trial Superiority Trial 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

Further Readings

  1. Cook TD, DeMets DL (Eds), 2008, Introduction to Statistical Methods for Clinical Trials. Boca Raton, FL: Chapman & Hall/CRC.Google Scholar
  2. Julious SA, Tan S-B, Machin D, 2010, An Introduction to Statistics in Early Phase Trials. Chichester, UK: Wiley-Blackwell.CrossRefGoogle Scholar
  3. Kay R, 2007, Statistical Thinking for Non-statisticians in Drug Regulation. Chichester, UK: Wiley.CrossRefGoogle Scholar
  4. Rosenberg MJ, 2010, The Agile Approach to Adaptive Research: Optimizing Efficiency in Clinical Development. Hoboken, NJ: Wiley.CrossRefGoogle Scholar
  5. Chow S-C, Chang M, 2007, Adaptive Design Methods in Clinical Trials. Boca Raton: Chapman & Hall/CRC.Google Scholar
  6. Turner JR, Durham TA, 2009, Integrated Cardiac Safety: Assessment Methodologies for Noncardiac Drugs in Discovery, Development, and Postmarketing Surveillance. Hoboken, NJ: Wiley.Google Scholar
  7. Ellenberg SS, Fleming, DeMets DL, 2003, Data Monitoring Committees in Clinical Trials: A Practical Perspective. Hoboken, NJ: Wiley.Google Scholar
  8. Friedman LK, Furberg CD, DeMets DL, 1998, Fundamentals of Clinical Trials, 3rd Edition. New York, NY: SpringerGoogle Scholar
  9. Hwang IK, Lan KKG, 2006, Interim analysis and adaptive design in clinical trials. In Buncher CR, Tsay J-Y (Eds), Statistics in the Pharmaceutical Industry, 3rd Edition. Boca Raton: Chapman & Hall/CRC, 245–284.Google Scholar
  10. Machin D, Campbell MJ, 2005, Design of Studies for Medical Research. Chichester, UK: Wiley.CrossRefGoogle Scholar
  11. Piantadosi S, 2005, Clinical Trials: A Methodologic Perspective, 2nd Edition. Hoboken, NJ: Wiley.CrossRefGoogle Scholar
  12. Faich GA, Stemhagen A, 2005, Cardiac arrhythmia assessments in Phase IV clinical studies. In Morganroth JM, Gussak I (Eds), Cardiac Safety of Noncardiac Drugs: Practical Guidelines for Clinical Research and Drug Development. Totowa, NJ: Humana Press, 229–237.CrossRefGoogle Scholar
  13. Matthews JNS, 2006, Introduction to Randomized Controlled Clinical Trials, 2nd Edition. Boca Raton: Chapman & Hall/CRC.CrossRefGoogle Scholar
  14. Spiegelhalter DJ, Abrams KR, Myles JP, 2004, Bayesian Approaches to Clinical Trials and Health-care Evaluation. Hoboken, NJ: Wiley.Google Scholar

Copyright information

© Springer Science+Business Media, LLC 2011

Authors and Affiliations

  1. 1.Cardiac Safety Services QuintilesDurhamUSA

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