• Cancer Control · Apr 2007

    Review

    Evidence-based medicine for rare diseases: implications for data interpretation and clinical trial design.

    • Madhusmita Behera, Ambuj Kumar, Heloisa P Soares, Lubomir Sokol, and Benjamin Djulbegovic.
    • Malignant Hematology Program, H. Lee Moffitt Cancer Center & Research Institute, 12902 Magnolia Drive, Tampa, FL 33612, USA.
    • Cancer Control. 2007 Apr 1;14(2):160-6.

    BackgroundThe 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.MethodsWe review the principles of evidence-based medicine to see which are particularly pertinent to the study of rare diseases.ResultsWhen 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.ConclusionsEven 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.

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