The Wide Variety of Clinical Assessments Conducted

A multitude of studies are conducted to examine the safety and efficacy of a new investigational drug in humans. Among the goals of clinical development are the following  

Additional safety data and effectiveness data (see Chapter 13) are collected in therapeutic use studies. The following statement by Piantadosi (2005) is particularly salient here  

Human Pharmacology Assess tolerance. Describe or define pharmacokinetics (PK) and pharmacodynamics (PD). Explore drug metabolism and drug interactions. Estimate (biological) activity. Dose-tolerance studies. Single- and multiple-dose PK and/or PD studies. Drug interaction studies. 

Therapeutic Exploratory Explore use for the targeted indication. Estimate dosage for subsequent studies. Provide basis for confirmatory study design, endpoints, methodologies. Earliest trials of relatively short duration in well-defined narrow patient populations, using surrogate of pharmacological endpoints or clinical measures. Dose-response exploration studies. 

Therapeutic Confirmatory Demonstrate/confirm efficacy. Establish safety profile. Provide an adequate basis for assessing benefit/risk relationship to support licensing. Establish dose-response relationship. Adequate and well-ontrolled studies to establish efficacy. Randomized parallel dose-response studies. Clinical safety studies. Studies of mortality/morbidity outcomes. Large simple trials. Comparative studies. 

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