A new toolkit for conducting clinical trials in rare disorders

J Popul Ther Clin Pharmacol. 2014;21(1):e66-78. Epub 2014 Feb 23.


Evidence based medicine requires strong scientific evidence upon which to base treatment. Because the available study populations for rare diseases are small, this evidence is difficult to accrue. Investigators need to consider a flexible toolkit of methods to deal with the problems inherent in the study of rare disease. We present some potential solutions in this paper.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Clinical Trials as Topic / methods*
  • Evidence-Based Medicine
  • Humans
  • Orphan Drug Production / methods*
  • Rare Diseases / drug therapy*
  • Research Design