Qualitative interviews of patients with COPD and muscle weakness enrolled in a clinical trial evaluating a new anabolic treatment: patient perspectives of disease experience, trial participation and outcome assessments.

BACKGROUND: Chronic obstructive pulmonary disease (COPD) and muscle weakness can cause impaired physical function, significantly impacting patients' health-related quality of life (HRQoL). Loss of muscle strength is usually assessed through clinical and performance outcome (PerfO) assessments, which consists of tasks performed in a standardized manner, providing evidence of a patient's functional ability. However, evidence documenting the patient experience of COPD and muscle weakness is limited. METHODS: This two-stage qualitative study used semi-structured interviews in patients aged 45-80 years with COPD (post-bronchodilator forced expiratory volume in 1s [FEV1]/forced vital capacity ratio < 0.70, and FEV1% predicted of 30-80%) and muscle weakness. In Stage 1, 30-minute concept elicitation interviews were conducted with participants recruited across three US sites to explore impacts on physical functioning and activities of daily living. In Stage 2, interviews were performed with participants exiting a Phase IIa trial investigating the efficacy of a selective androgen receptor modulator (GSK2881078) on leg strength, whereby PerfOs were used to evaluate strength and physical functioning endpoints. These participants completed either 60-minute in-depth (n = 32) or 15-minute confirmatory (n = 35) interviews exploring trial experience, completion of outcome measures, disease experience and treatment satisfaction. RESULTS: In Stage 1 (n = 20), most participants described their muscles as weak (83.3%). Difficulties with walking (100%) and lifting heavy objects (90%) were reported. In Stage 2, 60-minute interviews, all participants (n = 32) reported a positive trial experience. Most participants reported that the home exercise program was easy to fit into daily life (77.8%), the PROactive daily diary was easy to complete (100%) and wearable sensors were easy to use (65.6%). However, technical issues were reported (71%), and few participants (19.4%) found physical assessments easy to complete. Improvements in muscle strength and functional limitations were reported by most participants. The shorter 15-minute confirmatory interviews (n = 35) supported the in-depth interview results. CONCLUSION: The qualitative interviews generated in-depth evidence of key concepts relevant to patients with COPD and muscle weakness and support the assessments of patient strength and physical function as outcome measures in this population in future studies. TRIAL NUMBER: GSK Stage 1: 206869; Stage 2: 200182, NCT03359473; Registered December 2, 2017, https://clinicaltrials.gov/ct2/show/NCT03359473 .

Evaluation of the Content Validity and Cross-Cultural Validity of the Study Participant Feedback Questionnaire (SPFQ).

OBJECTIVES: The Study Participant Feedback Questionnaire (SPFQ) is a patient-completed tool designed to assess patient experiences and satisfaction with aspects associated with being involved in a clinical trial. Originally developed in oncology and among English-speaking participants, the aim of the current study was to evaluate the content and cross-cultural validity of the SPFQ in other indications and non-English-speaking countries. METHODS: Semi-structured qualitative telephone interviews were conducted with 80 participants across eight non-English-speaking countries (in Europe, South America and Asia) who had received an investigational medicinal product as part of a clinical trial in the past three years. Interviews comprised concept elicitation to identify concepts of importance to participants' trial experiences, and cognitive debriefing to assess understanding and perceived importance of SPFQ instructions, items and response options. RESULTS: Concept elicitation findings supported the content validity of the SPFQ. During cognitive debriefing, SPFQ instructions and the majority of items were well understood by participants. Participants generally considered the SPFQ items important to their clinical trial experience, albeit a handful of items assessed concepts that had not been experienced by trial participants or were redundant with other SPFQ items. The instructions, response options and recall period of the SPFQ were generally well understood. No country-level differences in understanding or importance were apparent. CONCLUSION: Study findings provide evidence for the content and cross-cultural validity of the SPFQ and support implementation of the SPFQ as a means of obtaining participant feedback across global development programmes in a variety of indications.

A national survey of the oral health status of homeless veterans.

OBJECTIVES: This study reports results from a survey designed to (1) assess the oral health needs of a national sample of homeless veterans and (2) compare the dental needs of homeless veterans participating in VA-sponsored rehabilitation programs with domiciled veterans in VA substance addiction programs. METHODS: Homeless veterans enrolled in a nationwide rehabilitation program (n = 1,152) completed a survey including questions concerning patients' perceptions of their oral health, dental service needs and use, and alcohol and tobacco use. A sample of these veterans (n = 281) subsequently received dental exams. A comparison group of domiciled veterans enrolled in VA substance abuse programs (n = 339) completed a similar survey. A sample of these veterans (n = 150) also received dental exams. RESULTS: Sociodemographic variables, patient-reported oral health information and risk behaviors, and findings from dental exams described two remarkably similar populations. CONCLUSIONS: As expected, the homeless veterans exhibited poor oral health, but it was not different from domiciled veterans enrolled in substance addiction programs. Lifestyle choices, such as heavy drinking and smoking, may contribute more to poor oral health than living conditions.