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For many years, psychologists and other social scientists have been pushing for the individual patient's perspective – priorities, needs, feelings, and functioning – to be incorporated into drug development. This is usually achieved through the use of patient-reported outcome measures (PROMs) in clinical trials. This paper discusses some key issues in the use of PROM data as the sole method of generating information about the patient's perspective and outlines the relevance of narrative evidence to enhance understanding and interpretation of PROM data. The development and use of PROMs situates them at the vertex of two very different trends in medicine: patient-centered care and standardization. Indeed, the application of PROMs – which pull in the direction of standardization – results in a narrow conception of evidence by overriding the subjectivity of individual experiences, beliefs, and judgments. Without additional context, PROM data cannot easily support individual patient-level care. When collected systematically and with an interpretive phenomenological approach, narrative data can contain valuable information about the patient experience that numerical ratings from PRO measures do not capture. [ABSTRACT FROM AUTHOR]

Background: Approximately 7–10% of Parkinson's disease (PD) patients carry a GBA (Glucocerebrosidase) mutation (GBA-PD patients), which may influence the disease's clinical course. Objectives: This study aimed to explore the patient experience of GBA-PD and identify the most important symptoms and impacts to inform clinical trial measurement strategies. Methods: Twenty PD patients (n = 15 GBA-PD; n = 5 idiopathic-PD) participated in qualitative interviews which explored concepts spontaneously reported or identified through a literature review. Telephone interviews with five expert clinicians included discussion of a preliminary conceptual model derived from literature. Verbatim transcripts were thematically analysed. Results: Thirty symptoms reported by patients were categorized as motor, non-motor, and cognitive/psychiatric. Tremor (n = 13), memory loss (n = 13), rigidity/stiffness (n = 11), and speech problems (n = 11) were considered the most important and impactful symptoms by GBA-PD patients, although other symptoms were also relevant to the majority of patients. Key impacts included: sleep disturbances (n = 13), handwriting changes (n = 13), reduced social interaction (n = 12), dyskinesia (n = 10), depressed mood (n = 9), and fear of falling (n = 8). Key symptoms and impacts reported by GBA-PD patients were consistent with those reported by idiopathic-PD patients. Clinician interview results supported the patient findings, although some clinicians indicated that cognitive/psychiatric symptoms may present earlier in GBA-PD patients. The concepts emerging from the research informed updates to a conceptual model of GBA-PD patients' disease experience. Conclusions: The findings provide in-depth understanding of the patient experience of GBA-PD. The findings confirm that the concepts relevant to assess in GBA-PD are consistent with those relevant to assess in idiopathic-PD; however, greater consideration of cognitive/psychiatric symptoms may be warranted in GBA-PD populations. [ABSTRACT FROM AUTHOR]

Aims To compare treatment satisfaction among people with type 2 diabetes receiving dulaglutide 1.5 mg and dulaglutide 0.75 mg (a once-weekly, long-acting, glucagon-like peptide-1 receptor agonist) with those receiving either exenatide or placebo ( AWARD-1 study) or metformin ( AWARD-3 study) over 52 weeks. Methods The Diabetes Treatment Satisfaction Questionnaire status version ( DTSQs) and change version ( DTSQc) were used to evaluate total treatment satisfaction and perceived frequency of hyperglycaemia and hypoglycaemia. Results In the AWARD-1 study, significant improvements from baseline were observed in total DTSQs score for both dulaglutide doses (26 and 52 weeks) and exenatide (26 weeks). The improvement was significantly greater with both dulaglutide doses compared with placebo (26 weeks) and exenatide (26 and 52 weeks). The perceived frequency of hyperglycaemia was lower for all groups at 26 and 52 weeks compared with baseline. The improvement was greater with both dulaglutide doses and exenatide compared with placebo at 26 weeks, and was also greater with both dulaglutide doses compared with exenatide at 26 and 52 weeks. The exenatide group had an increase in perceived frequency of hypoglycaemia at 26 and 52 weeks. In the AWARD-3 study, significant improvements from baseline were observed for total DTSQs scores in all groups at 26 and 52 weeks. Perceived frequency of hyperglycaemia was lower for all groups at 26 and 52 weeks compared with baseline, and this improvement was greater with both dulaglutide doses compared with metformin at 52 weeks. Conclusions Dulaglutide was associated with improvements in treatment satisfaction and a decrease in perceived frequency of hyperglycaemia. [ABSTRACT FROM AUTHOR]

Aims Quality of life (QoL) is recognized widely as an important health outcome in diabetes, where the burden of self-management places great demands on the individual. However, the concept of QoL remains ambiguous and poorly defined. The aim of our review is to clarify the measurement of QoL in terms of conceptualization, terminology and psychometric properties, to review the instruments that have been used most frequently to assess QoL in diabetes research and make recommendations for how to select measures appropriately. Methods A systematic literature search was conducted to identify the ten measures most frequently used to assess QoL in diabetes research (including clinical trials) from 1995 to March 2008. Results Six thousand and eight-five abstracts were identified and screened for instrument names. Of the ten instruments most frequently used to assess ‘QoL’, only three actually do so [i.e. the generic World Health Organization Quality of Life (WHOQOL) and the diabetes-specific Diabetes Quality of Life (DQOL) and Audit of Diabetes-Dependent Quality of Life (ADDQoL)]. Seven instruments more accurately measure health status [Short-Form 36 (SF-36), EuroQoL 5-Dimension (EQ-5D)], treatment satisfaction [Diabetes Treatment Satisfaction Questionnaire (DTSQ)] and psychological well-being [Beck Depression Inventory (BDI), Hospital Anxiety and Depression Scale (HADS), Well-Being Questionnaire (W-BQ), Problem Areas in Diabetes (PAID)]. Conclusions No single measure can suit every purpose or application but, when measures are selected inappropriately and data misinterpreted, any conclusions drawn are fundamentally flawed. If we value QoL as a therapeutic goal, we must ensure that the instruments we use are both valid and reliable. QoL assessment has the proven potential to identify ways in which treatments can be tailored to reduce the burden of diabetes. With careful consideration, appropriate measures can be selected and truly robust assessments undertaken successfully. [ABSTRACT FROM AUTHOR]

Summary: Atopic dermatitis (AD) is a chronic skin disease occurring in about 5 to 10% of adults worldwide. It can cause intense and persistent itch. For almost two‐thirds of patients with moderate‐to‐severe AD, the itching lasts at least 12 hours a day, and is severe to unbearable. In clinical trials that are looking at how effective a treatment for AD is, itch intensity must be measured. Given itch is subjective, its intensity is most accurately reported by patients themselves. In this study, investigators from the U.S.A. and Europe developed a scale of itch called the Peak Pruritus Numerical Rating Scale (NRS). The scale measures the worst itch (peak pruritus) during the past 24 hours by asking patients "On a scale of 0 to 10, with 0 being 'no itch' and 10 being 'worst itch imaginable', how would you rate your itch at the worst moment during the previous 24 hours?". To ensure the scale is reliable and valid (i.e., appropriate for assessing itch in AD patients), patients were interviewed about their opinions of the scale. The scale was then implemented in three large clinical trials of adults with moderate‐to‐severe AD. Interviewed patients said that the scale provided a relevant, clear, and comprehensive assessment of itch severity. As hypothesized, scores from the Peak Pruritus NRS showed moderate‐to‐strong correlation with other measures of itch, and weak‐to‐moderate correlation with measures of AD signs (clinical symptoms). The investigators concluded that the Peak Pruritus NRS is a well‐defined, reliable, sensitive, and valid scale for evaluating worst itch intensity in adults with moderate‐to‐severe AD. Linked Article: Yosipovitch et al. Br J Dermatol 2019; 181:761–769 [ABSTRACT FROM AUTHOR]