Most evaluations of therapeutics are performed for the purpose of FDA approval, which, according to the FDA modernization act of 1997, states that “as a general rule, two adequate and well-controlled studies are needed to prove the product's safety and effectiveness. “
Demonstrating effectiveness involves targeting improvement in a primary and possibly a secondary efficacy outcome. Demonstrating safety, while prominent in considerations, is less straightforward to quantify and evaluate. General issues relate to defining the occurrence and severity of potential adverse events. More specific issues relate to attributing an adverse event to the therapeutic being evaluated and determining whether its relative frequency is great enough to cause concern. Generally the “well-controlled studies” mentioned above are powered to detect differences in efficacy outcomes that occur with enough frequency to minimize required sample sizes. Specific adverse events are unlikely to be anticipated and, even when they are anticipated, they will generally occur with less frequency than the specified efficacy outcomes. Post-marketing surveillance is necessary to more completely describe the safety profile of any therapeutic. Even with large surveillance efforts, however, the statistical issues regarding sample sizes for detection of adverse events and also adverse drug interactions are challenging.
Learning Objectives: To be added.
Keywords: Biostatistics, Health Care Quality
Presenting author's disclosure statement:
Organization/institution whose products or services will be discussed: None
I do not have any significant financial interest/arrangement or affiliation with any organization/institution whose products or services are being discussed in this session.