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A Method for Utilizing Bivariate Efficacy Outcome Measures to Screen Agents for Activity in 2 Stage Phase II Clinical Tr

OutlineIntro to the Classic Method of Evaluating Drug Efficacy (Response)The Development of

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A Method for Utilizing Bivariate Efficacy Outcome Measures to Screen Agents for Activity in 2 Stage Phase II Clinical Tr

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    1. A Method for Utilizing Bivariate Efficacy Outcome Measures to Screen Agents for Activity in 2 Stage Phase II Clinical Trials

    3. Classic Method for Evaluating Drug Efficacy: Tumor Response

    4. At Baseline

    7. So, the progression of the disease before cytotoxic treatment is characterized by growing tumors and spread of distant metastases. Whereas effective cytotoxic treatment reduces the tumor burden over time. So, the progression of the disease before cytotoxic treatment is characterized by growing tumors and spread of distant metastases. Whereas effective cytotoxic treatment reduces the tumor burden over time.

    13. So we have our patient with a worsening disease condition until he is treated with an effective cytostatic agent. Treatment results in stabilization of his disease.So we have our patient with a worsening disease condition until he is treated with an effective cytostatic agent. Treatment results in stabilization of his disease.

    19. Here is a graph of the parameter space. The vertical axis defines the probability of response and the horizontal axis defines the probability of PFS at 6 months. The null hypothesis defines the null space according to this rectangle. Clinically significant improvements are defined beyond this rectangle. A good design has small power here and high power here.Here is a graph of the parameter space. The vertical axis defines the probability of response and the horizontal axis defines the probability of PFS at 6 months. The null hypothesis defines the null space according to this rectangle. Clinically significant improvements are defined beyond this rectangle. A good design has small power here and high power here.

    20. How To Begin?

    21. The Data in General Look Like:

    22. The PMF The probability mass function can be written as a function of the cell counts.The probability mass function can be written as a function of the cell counts.

    24. The CDF

    27. Two-Stage Designs Two stage designs have 2 sets of decision criteria.Two stage designs have 2 sets of decision criteria.

    35. The Stage 1 Sample Space leading to Acceptance of Ho in Stage 2 is provided graphically below.The Stage 1 Sample Space leading to Acceptance of Ho in Stage 2 is provided graphically below.

    39. Finalization

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