FDA clinical trials draft guidanceThe US FDA released draft guidance this week on the “problems posed by multiple endpoints in the analysis and interpretation of study results and how these problems can be managed in drug and biologic clinical trials,” Zachary Brennan of RAPS reports. The FDA justifies the guidance due to the greater likelihood of making false conclusions about the effects of a given drug as the number of endpoints in a single clinical trial increases.

The FDA states that the guidance serves to “describe various strategies for grouping and ordering endpoints for analysis and applying some well-organized statistical methods for managing multiplicity within a study in order to control the chance of making erroneous conclusions about a drug’s effects.” (FDA Draft Guidance). Efficacy endpoints measure a drug’s effects. They may include assessments of clinical events (e.g. mortality, pulmonary exacerbation), patient symptoms (e.g. pain, depression), measures of function (e.g. ability to walk or exercise), or surrogates of these events or symptoms. The draft guidance lists the three families for classifying endpoints within a clinical trial: primary, secondary, and exploratory. To demonstrate the study objective of effectiveness, a statistical analysis must be performed to rule out chance as the explanation for a trial’s results. The guidance goes on to discuss the test of hypothesis and several other statistical methods used within clinical research to test against the probability of chance.

The FDA’s explanation of various statistical models and their usage cases dominates the rest of the draft guidance. However, this in-depth analysis drives home the message of this guidance. The FDA states that “the chance of making a false positive conclusion, concluding that a drug has a beneficial effect when it does not, is of primary concern” (FDA Draft Guidance). Our team of experts at Pearl IRB strive to stay up to date with FDA’s guidance to ensure that our clients remain informed about the necessary regulations. This allows clinical trials to run smoothly as our clients march towards product commercialization. If you have a study that needs reviewed or are in the beginning phases of research for a new treatment, please contact us today.