Designing effectiveness studies of individualized treatments with personalized outcomes.

Abstract

Background: Patients with chronic or severe health problems or involved in rehabilitation programs often have needs that vary greatly. As a response to this, the health interventions are most often individualized and aimed at personalized outcomes.

Aim: The aim of the study is to investigate how to design clinical trials that can measure the effectiveness of individualized interventions with personalized outcomes.

Methods: With departure in a discussion of pragmatic trials designed to assess outcomes of individualisted treatments, further developments for acknowledging patients’ personal priorities in outcomes are explored, including a discussion of randomisation vs self-selection and presentation of flexible tools for the selection of personalized primary outcomes. Finally, the relevance of  a cross-disciplinary approach is suggested.

Conclusion: Trials investigating the effectiveness of individualized interventions for personalized outcomes can be based on the design of pragmatic trials, with a few additional extensions. Tools for outcome must allow for personalized preferences and may include interviews, questionnaires with open formulation of concerns, or personal prioritizing of issues covered by validated questionnaires. In order to encompass patients’ personal choice of outcomes, a cross-disciplinary methodology may be necessary.

Keywords: pragmatic trials, personalized medicine, randomization, self-select treatment, rehabilitation, patient preferences