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Failure is an option: learning from unsuccessful proof-of-concept trials

Stefan Schäfer1, Peter Kolkhof

  • 1Cardiology Research, Bayer Schering Pharma, Wuppertal, Germany. Stefan.schaefer@bayerhealthcare.com

Drug Discovery Today
|October 29, 2008
PubMed

Insights

Drug development faces high attrition rates, particularly in Phase II clinical trials, due to a lack of efficacy. This highlights a critical gap in translating preclinical findings into successful human treatments.

Area of Science:

  • Pharmacology and Drug Development
  • Clinical Trials
  • Translational Medicine

Background:

  • High attrition rates persist in drug development, with clinical efficacy failures being the primary reason for program discontinuation.
  • Phase II clinical trials represent a critical juncture, often serving as the first assessment of a drug's pharmacodynamic action and proof of concept.
  • Attrition rates approximate 60-70% across diverse therapeutic areas, irrespective of the predictive accuracy of animal models.

Purpose of the Study:

  • To analyze the high attrition rates in drug development, focusing on the causes of discontinuation.
  • To investigate the disproportionately high failure rates observed in Phase II clinical trials.
  • To examine the challenges in translating preclinical data into demonstrated clinical benefit.

Main Methods:

  • Statistical analysis of recent drug development attrition data.
  • Review of common reasons for drug development program discontinuation.
  • Comparative analysis of attrition rates across different therapeutic areas.

Main Results:

  • Lack of clinical efficacy is the leading cause for drug development program discontinuation.
  • Attrition rates are highest during Phase II clinical trials, where proof of concept is typically established.
  • Consistent high attrition rates (60-70%) are observed across various therapeutic areas, including CNS and cardiovascular medicine.

Conclusions:

  • The translation of preclinical data into clinical efficacy remains a significant challenge in drug development.
  • Improving the predictive accuracy of preclinical models and trial designs is crucial to reduce Phase II attrition.
  • Addressing the efficacy gap is essential for increasing the success rate of new drug development programs.

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