Evidence-based medicine for rare diseases: implications for data interpretation and clinical trial design.
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.
Behera
Madhusmita
M
Malignant Hematology Program, H. Lee Moffitt Cancer Center & Research Institute, 12902 Magnolia Drive, Tampa, FL 33612, USA.
Kumar
Ambuj
A
Soares
Heloisa P
HP
Sokol
Lubomir
L
Djulbegovic
Benjamin
B
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