title: Evidence-based medicine for rare diseases: implications for data interpretation and clinical trial design.
abstract: BACKGROUND
  The randomized, controlled trial (RCT) is the "gold standard" for establishing the effect of any intervention. This approach, however, is often not feasible with rare diseases such as cutaneous T-cell lymphoma.
  
  
  METHODS
  We review the principles of evidence-based medicine to see which are particularly pertinent to the study of rare diseases.
  
  
  RESULTS
  When an RCT is not feasible, attention is given to determining all the available prior data. Evaluation of the new data and the historic base requires attention to biases, but can allow estimation of a "true" study result.
  
  
  CONCLUSIONS
  Even when an RCT cannot be performed because of insufficient cases, utilization of evidence-based methodology can help minimize bias and maximize the truth of observed new data.
authors: Malignant Hematology Program, H. Lee Moffitt Cancer Center & Research Institute, 12902 Magnolia Drive, Tampa, FL 33612, USA.
authors: Madhusmita
authors: M
authors: Behera
authors: Ambuj
authors: A
authors: Kumar
authors: Heloisa P
authors: HP
authors: Soares
authors: Lubomir
authors: L
authors: Sokol
authors: Benjamin
authors: B
authors: Djulbegovic
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods*; D003627:Data Interpretation, Statistical; D019317:Evidence-Based Medicine / Q000379:methods*; D006801:Humans; D035583:Rare Diseases / Q000628:therapy*; D012107:Research Design