Evidence-Based Medicine and Rare Diseases.

  title={Evidence-Based Medicine and Rare Diseases.},
  author={Simon J Day},
  journal={Advances in experimental medicine and biology},
This chapter discusses the meaning of evidence-based medicine and where it relates to randomised controlled trials, but also where it does not. The need for good quality evidence is stressed through a discussion of high failure rates in drug development and arguments against access to unlicensed (and largely untested) treatments are set out (despite the… CONTINUE READING