All Times EDT
Keywords: estimands, ICE, missing data, sensitivity analysis, treatment discontinuation, neuroscience
The ICH E9 (R1) Addendum on Estimands and Sensitivity Analysis in Clinical Trials was finalized in Nov 2019 and subsequently implemented by many regulatory agencies, including the FDA. This roundtable discussion focuses on the implementation of estimands, choice of intercurrent event (ICE) strategies, and corresponding missing data approaches and sensitivity analyses in neuroscience (NS) clinical trials. Although NS is a highly heterogeneous therapeutic area, there are many common characteristics across NS indications. For example, many NS indications (especially neuropathic pain, posttraumatic stress disorder, autism spectrum disorder, depression, Alzheimer’s disease, and others) are non-life threatening and post ICE data collection is usually feasible, allowing for the use of treatment policy to address ICEs. Another example of these common characteristics may be that the extent and pattern of data missingness are usually difficult to estimate for many NS clinical trials. Therefore, continuous and detailed documentation of missingness is not only critical for the success of the current trial but also necessary to inform future studies.
This roundtable facilitates a casual chat among clinical and regulatory biostatisticians on both technical (such as methods to account for ICE strategy choices in sample size estimation; and methods to account for the uncertainty of post ICE data collection) and practical topics (such as ways to boost patient retention after early treatment discontinuation; and ways to maximize data collection post treatment discontinuation in a longitudinal trial). We have come up with questions that apply to a wide audience in neuroscience: biostatisticians, clinicians, operations, and regulators. Come join us and bring your own questions too!