Abstract
Healthcare decisions are more effective when the patient voice is included in clinical research, and the Food and Drug Administration encourages the patient’s voice in drug development and regulatory decision-making. Clinical trials should not only demonstrate the effect of a drug on clinical outcomes but should also demonstrate that these outcomes are important or meaningful to patients. Several qualitative and quantitative methods are available to collect patient experience data (e.g., traditional patient-reported outcome [PRO] measures, interviews with clinical trial participants). We aimed to understand if in-depth exit interviews were more effective assessments of the patient experience in recent type 1 diabetes (T1D) clinical trials than existing diabetes-specific PROs. In-depth qualitative interviews were conducted with 41 adults with T1D who had completed or withdrew from a phase 3 study of sotagliflozin, a dual inhibitor of SGLT1 and SGLT2. A targeted literature review was conducted to identify diabetes-specific PROs used in randomized controlled clinical trials of novel T1D medications reported over the past 5 years. Included trials had to investigate a pharmaceutical intervention for adults with T1D and report a diabetes-specific PRO. The concepts assessed in the PRO measures were mapped against those elicited during the 41 exit interviews. A total of 336 publications were identified in the literature search of which 26 were eligible for analysis. Eight diabetes-specific PROs were identified and reviewed from which 54 concepts related to the patient experience were identified. The patient exit interviews included 42/54 (78%) of the patient experience-related concepts identified across all 8 PROs from the literature review. Of the 8 PRO instruments, the Diabetes Quality of Life Measure (DQOL) covered the most concepts (18/54, 33%), followed closely by the Audit of the Diabetes-Dependent Quality of Life (ADDQoL; 16/54, 30%). Some of the most prominent concepts from both approaches were related to impact on life and family; fear of complications; and impact on physical activity, lifestyle and social perceptions. There were several concepts identified in the exit interviews that were not covered in any of the 8 PRO instruments (related to keeping blood sugars within a desired range, ability to manage changes in insulin use). Overall, the exit interviews appeared to provide a more comprehensive picture of patient experience domains. Although existing diabetes PRO measures cover a range of concepts and may adequately assess changes in certain outcomes, data from patient exit interviews provide more comprehensive insights into the patient experience. Exit interview data may provide a more detailed understanding of the disease burden and impact of treatment on improvements in well-being, daily functioning, and treatment satisfaction.
Customizing the Types of Technologies Used by Patients With Type 1 Diabetes Mellitus for Diabetes Treatment: Case Series on Patient Experience