PRM187 - Patient Reported Outcomes in the Development of new Medications for Type 2 Diabetes: a Review of Recently Approved Products

Abstract

Patient-Reported Outcome (PRO) instruments are increasingly being utilized in drug development to provide a holistic understanding of the drugs impact. Such data can be beneficial in clinical decision making. This review sought to identify and discuss the PRO instruments used in Phase 3 trials of the newer classes of drugs for the treatment of Type 2 diabetes (T2DM). A search was conducted in medline, psychinfo, cinahl, and clinicaltrials.gov using 17 drug names contained within the following classes: GLP-1 receptor agonists, novel insulins, SGLT-2 inhibitors, and DPP-4 inhibitors. This search was supplemented with EASD and ADA abstract database searches from 2012-2014. PRO instruments used in Phase 3 trials were identified and categorized by measurement concept(s). The items and domains contained within the PRO instruments were further explored and summarized. Twenty PRO instruments have been used, although none in DPP-4 inhibitor trials. PRO instruments were used to measure six separate concepts: treatment satisfaction (10/17 drugs), health-related quality of life (HRQoL) (8/17), impact of weight/eating-related outcomes (6/17), symptoms of diabetes (3/17), psychological well-being (3/17), and cognitive functioning (1/17). Although several PRO instruments were used to measure the same concept, the items and domains that comprised that concept differed across instruments. For example, only two of the seven treatment satisfaction questionnaires contain items pertaining to the injectable therapy device. In addition, some PRO instruments did not comprehensively evaluate the concept that they claimed. For example, an item-level analysis of the EQ-5D, identified as a measurement of HRQoL, suggests that it more accurately measures functional health status, thereby representing only one dimension of HRQoL. PRO data are available for many recently approved drugs in T2DM. More work is needed to highlight the relevance and importance of PRO data from pivotal trials as an important consideration for physicians when making treatment decisions.