Let's talk about it: an exploration of the comparative use of three different digital platforms to gather patient-reported outcome measures.

BACKGROUND: Patient-reported outcome (PRO) measures provide valuable evidence in clinical trials; however, poor compliance with PRO measures is a notable and long-standing problem, resulting in missing data that potentially impact the interpretation of trial results. Interactive, patient-centric platforms may increase participants' motivation to complete PRO measures over the course of a clinical trial. Thus, the aim of this study was to evaluate and optimize the usability of 3 popular consumer technologies-a traditional app-based interface, a chatbot interface, and a speech-operated interface-that may be used to improve user engagement and compliance with PRO measures. METHODS: Participants aged 18-75 years from the general United States population tested the usability of 3 ePRO platforms: a traditional app-based interface using Datacubed Health Platform (Datacubed), a web-based chatbot interface using the Orbita platform, and a speech-operated Alexa interface using an Alexa Skill called "My Daily Wellness." The usability of these platforms was tested with 2 PRO measures: the EQ-5D-5 L and the SF-12v2 Health Survey (SF-12v2), Daily recall. Using a crossover design, 3 cohorts of participants tested each ePRO platform daily for 1 week. After testing, interviews were conducted regarding the participants' experience with each platform. RESULTS: A total of 24 adults participated in the study. The mean age of participants was 45 years (range, 21-71 years), and half were female (n = 12; 50%). Overall, participants prioritized speed, ease of use, and device portability in selecting their preferred platform. The Datacubed app met these criteria and was the preferred platform among most participants (n = 20; 83%). Participants also suggested various modifications to the platforms, such as programmable notifications, adjustable speed, and additional daily reminders. CONCLUSIONS: These data demonstrate the importance of speed, ease of use, and device portability, features that are currently incorporated in the Datacubed app, in ePRO platforms used in future clinical trials. Additionally, the usability of ePRO platforms may be optimized by adding programmable notifications, adjustable speed, and increased daily reminders. The results of this study may be used to enhance the usability and patient centricity of these platforms to improve user compliance and engagement during clinical trials.

Best Practice Recommendations: User Acceptance Testing for Systems Designed to Collect Clinical Outcome Assessment Data Electronically.

Implementing clinical outcome assessments electronically in clinical studies requires the sponsor and electronic clinical outcome assessment (eCOA) provider to work closely together to implement study-specific requirements and ensure consensus-defined best practices are followed. One of the most important steps is for sponsors to conduct user acceptance testing (UAT) using an eCOA system developed by the eCOA provider. UAT provides the clinical study team including sponsor or designee an opportunity to evaluate actual software performance and ensure that the sponsor's intended requirements were communicated clearly and accurately translated into the system design, and that the system conforms to a sponsor-approved requirements document based on the study protocol. The components of an eCOA system, such as the study-specific application, customization features, study portal, and custom data transfers should be tested during UAT. While the provider will perform their own system validation, the sponsor or designee should also perform their due diligence by conducting UAT. A clear UAT plan including the necessary documentation may be requested by regulatory authorities depending on the country. This paper provides the electronic patient-reported outcome (ePRO) Consortium's and patient-reported outcome (PRO) Consortium's best practice recommendations for clinical study sponsors or their designee for conducting UAT with support from eCOA providers to ensure data quality and enhance operational efficiency of the eCOA system. Following these best practice recommendations and completing UAT in its entirety will support a high quality eCOA system and ensure more reliable and complete data are collected, which are essential to the success of the study.

Generation of evidence supporting the content validity of SF-36, FACIT-F, and LupusQoL, and novel patient-reported symptom items for use in patients with systemic lupus erythematosus (SLE) and SLE with lupus nephritis (LN).

OBJECTIVE: SLE and lupus nephritis (LN) have significant impacts on the health-related quality of life of patients living with the condition, which are important to capture from the patient's perspective using patient-reported outcomes (PROs). The objectives of this study were to evaluate the content validity of PROs commonly used in SLE and LN (36-Item Short Form Health Survey (SF-36), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) and Lupus Quality of Life (LupusQoL), as well as novel PRO symptom severity items measuring skin rash, joint pain, joint stiffness and swelling of the legs and/or feet, in both populations. METHODS: Qualitative, semi-structured, cognitive interviews were conducted with 48 participants (SLE=28, LN=20). Understanding and relevance of symptom and impact PRO concepts from existing PROs were assessed, alongside novel PRO symptom severity items with different recall periods (24 hours vs 7 days) and response scales (Numerical Rating Scale (NRS) vs Verbal Rating Scale). Interviews were conducted in multiple rounds to allow for modifications to the novel PRO items. Analysis of verbatim interview transcripts was performed. RESULTS: Symptom and impact concepts assessed by the SF-36, FACIT-F, and LupusQoL were well understood by both participants with SLE and LN (≥90.0%), with most considered relevant by over half of the participants asked (≥51.9%). All participants asked (100%) understood the novel PRO symptom severity items, and the majority (≥90.0%) considered the symptoms relevant. Minor modifications to the novel PRO items were made between rounds to improve clarity based on participant feedback. The selected 7-day recall period and NRS in the final iteration of the PRO items were understood and relevant. No differences in interview findings between the SLE and LN samples were identified. CONCLUSIONS: Findings provide evidence of content validity for concepts assessed by the SF-36, FACIT-F, LupusQoL and the novel PRO symptom severity items, supporting use of these PROs to comprehensively assess disease impact in future SLE and LN clinical trials.