PURPOSE: To discuss strategies by which trial endpoints might be selected and, ultimately, translated into meaningful and patient-focused treatment claims.

DESCRIPTION: Trial endpoints should reflect treatment benefits and harms to patients based on how they feel, function, and/or survive. Selecting endpoints raises challenges in that they summarize concepts, how those concepts are measured (e.g., tools used, and how tools are administered), and data analytic strategies (e.g., group vs. within person comparisons, time to event vs fixed time point). There is a lack of clarity about the methods by which endpoints are developed and defined, and we make the case that several stages in the process are not incorporating patient perspectives. To frame the workshop, Dr. Shields (10 minutes) will articulate a model emphasizing how patient and other stakeholder input could be used to guide endpoint selection decisions. This will be followed by gauging audience experience in selecting trial endpoints. Next, Dr. Powers (15 minutes) will engage the audience to reflect these participant experiences, compare them to his own experiences defining endpoints as an FDA reviewer, physician, and outcomes scientist, and provide a summary of “things to think about” for endpoints. He will highlight particular facets of how and where patient perspectives could have informed endpoint development. Next, Dr. Stone (15 minutes) will present several new pain indices derived from momentary, real-time research; how stakeholders evaluated these indices; how the indices performed in trials; and how the indices relate to performance outcomes. These findings highlight quandaries in the selection of trial endpoints, especially how patient perspectives may not align with other data. The final 15 minutes of the workshop will be spent eliciting and integrating audience perspectives and suggestions to reflect how principles, guidelines, and data from multiple sources may inform clinical trial endpoints in a more systematic and transparent process.