Patient Reported Outcomes (PROs) used in recent Phase 3 trials for Type 2 Diabetes: A review of concepts assessed by these PROs and factors to consider when choosing a PRO for future trials

Abstract

Aims: Many treatment options are available for people with Type 2 Diabetes (T2D). While the goal of treatment is to reach target HbA1c levels, the psychological experience may be more tangible and meaningful for the patient. Together with biomarkers, Patient-Reported Outcome (PRO) data provide a holistic understanding of the clinical and psychosocial impact of T2D and its treatment.

Methods: A literature review was conducted in multiple databases to identify PRO endpoints used in Phase 3 trials of newer classes of drugs for the treatment of T2D.

Results: This review identified five PRO concepts which were evaluated as endpoints in Phase 3 trials of GLP-1 receptor agonists, novel insulins, SGLT-2 inhibitors, and DPP-4 inhibitors; symptoms, health-related quality of life, psychological well-being, satisfaction with treatment/health and impact of weight. Twenty PRO questionnaires were used to measure these concepts/endpoints directly from patients. The relevance of, and scientific basis for, these concepts as endpoints are discussed, the similarities and differences between questionnaires targeting the same concepts/endpoints are explored, and the sensitivity of the questionnaires to the experimental intervention is summarized. In addition, factors that should be considered when choosing PROs for future T2D trials are discussed.

Conclusions: The information gained from PROs in clinical trial research is important in defining treatment benefit within the context of the trial, and the potential benefit (i.e. better adherence) in clinical practice. However, variable results have been observed in recent trials and careful and systematic consideration should be given to PRO selection for future studies of T2D.

Keywords: Literature review; Patient-Reported Outcomes.