Background: Presbyopia is defined as the age-related deterioration of near vision over time which is experienced in over 80% of people aged 40 years or older. Individuals with presbyopia have difficulty with tasks that rely on near vision. It is not currently possible to stop or reverse the aging process that causes presbyopia; generally, it is corrected with glasses, contact lenses, surgery, or the use of a magnifying glass. Objective: This study aimed to explore how individuals used social media to describe their experience of presbyopia with regard to the symptoms experienced and the impacts of presbyopia on their quality of life. Methods: Social media sources including Twitter, forums, blogs, and news outlets were searched using a predefined search string relating to symptoms and impacts of presbyopia. The data that were downloaded, based on the keywords, underwent manual review to identify relevant data points. Relevant posts were further manually analyzed through a process of data tagging, categorization, and clustering. Key themes relating to symptoms, impacts, treatment, and lived experiences were identified. Results: A total of 4456 social media posts related to presbyopia were identified between May 2017 and August 2017. Using a random sampling methodology, we selected 2229 (50.0%) posts for manual review, with 1470 (65.9%) of these 2229 posts identified as relevant to the study objectives. Twitter was the most commonly used channel for discussions on presbyopia compared to forums and blogs. The majority of relevant posts originated in Spain (559/1470, 38.0%) and the United States (426/1470, 29.0%). Of the relevant posts, 270/1470 (18.4%) were categorized as posts written by individuals who have presbyopia, of which 37 of the 270 posts (13.7%) discussed symptoms. On social media, individuals with presbyopia most frequently reported experiencing difficulty reading small print (24/37, 64.9%), difficulty focusing on near objects (15/37, 40.5%), eye strain (12/37, 32.4%), headaches (9/37, 24.3%), and blurred vision (8/37, 21.6%). 81 of the 270 posts (30.0%) discussed impacts of presbyopia—emotional burden (57/81, 70.4%), functional or daily living impacts (46/81, 56.8%), such as difficulty reading (46/81, 56.8%) and using electronic devices (21/81, 25.9%), and impacts on work (3/81, 3.7%). Conclusions: Findings from this social media listening study provided insight into how people with presbyopia discuss their condition online and highlight the impact of presbyopia on individuals’ quality of life. The social media listening methodology can be used to generate insights into the lived experience of a condition, but it is recommended that this research be combined with prospective qualitative research for added rigor and for confirmation of the relevance of the findings.

Background: Defining the transition from relapsing-remitting multiple sclerosis (RRMS) to secondary progressive multiple sclerosis (SPMS) can be challenging and delayed. A digital tool (MSProDiscuss) was developed to facilitate physician-patient discussion in evaluating early, subtle signs of multiple sclerosis (MS) disease progression representing this transition.Objective: This study aimed to determine cut-off values and corresponding sensitivity and specificity for predefined scoring algorithms, with or without including Expanded Disability Status Scale (EDSS) scores, to differentiate between RRMS and SPMS patients and to evaluate psychometric properties.Methods: Experienced neurologists completed the tool for patients with confirmed RRMS or SPMS and those suspected to be transitioning to SPMS. In addition to age and EDSS score, each patient’s current disease status (disease activity, symptoms, and its impacts on daily life) was collected while completing the draft tool. Receiver operating characteristic (ROC) curves determined optimal cut-off values (sensitivity and specificity) for the classification of RRMS and SPMS.Results: Twenty neurologists completed the draft tool for 198 patients. Mean scores for patients with RRMS (n=89), transitioning to SPMS (n=47), and SPMS (n=62) were 38.1 (SD 12.5), 55.2 (SD 11.1), and 69.6 (SD 12.0), respectively (P<.001, each between-groups comparison). Area under the ROC curve (AUC) including and excluding EDSS were for RRMS (including) AUC 0.91, 95% CI 0.87-0.95, RRMS (excluding) AUC 0.88, 95% CI 0.84-0.93, SPMS (including) AUC 0.91, 95% CI 0.86-0.95, and SPMS (excluding) AUC 0.86, 95% CI 0.81-0.91. In the algorithm with EDSS, the optimal cut-off values were ≤51.6 for RRMS patients (sensitivity=0.83; specificity=0.82) and ≥58.9 for SPMS patients (sensitivity=0.82; specificity=0.84). The optimal cut-offs without EDSS were ≤46.3 and ≥57.8 and resulted in similar high sensitivity and specificity (0.76-0.86). The draft tool showed excellent interrater reliability (intraclass correlation coefficient=.95).Conclusions: The MSProDiscuss tool differentiated RRMS patients from SPMS patients with high sensitivity and specificity. In clinical practice, it may be a useful tool to evaluate early, subtle signs of MS disease progression indicating the evolution of RRMS to SPMS. MSProDiscuss will help assess the current level of progression in an individual patient and facilitate a more informed physician-patient discussion.

Purpose: This study explored perceptions of meaningful weight-loss and the level of change on two patient-reported outcome (PRO) measures, the 36-item Short Form Health Survey® [SF-36v2®] and Impact of Weight on Quality of Life Lite-Clinical Trials^© [IWQOL-Lite-CT^©], that individuals living with overweight or obesity consider to be meaningful and indicative of treatment success. Methods: Thirty-three qualitative interviews were conducted in the US with adults living with overweight or obesity. Concept elicitation explored perceptions of minimally important/meaningful weight-loss using open-ended questions. Cognitive debriefing was used to understand thresholds for meaningful change on both measures. Results: Most participants (n = 23/33) expected a 5% total body weight-loss to yield some benefit in physical functioning, while all participants expected a 10% weight-loss to provide a meaningful and noticeable improvement in their physical functioning. Participants indicated that an item-level 1-point score change on each measure would represent a noticeable improvement in physical functioning and indicate treatment success. Conclusions: Participants expected moderate weight-losses to be noticeable, with ≥ 10% weight-loss yielding the most consistent results. The findings suggested that both measures provide strong opportunity to demonstrate treatment benefit in relation to physical functioning as a small change on the response scale would represent a noticeable improvement in participants’ daily lives.

BACKGROUND Presbyopia is defined as the age-related deterioration of near vision over time which is experienced in over 80% of people aged 40 years or older. Individuals with presbyopia have difficulty with tasks that rely on near vision. It is not currently possible to stop or reverse the aging process that causes presbyopia; generally, it is corrected with glasses, contact lenses, surgery, or the use of a magnifying glass. OBJECTIVE This study aimed to explore how individuals used social media to describe their experience of presbyopia with regard to the symptoms experienced and the impacts of presbyopia on their quality of life. METHODS Social media sources including Twitter, forums, blogs, and news outlets were searched using a predefined search string relating to symptoms and impacts of presbyopia. The data that were downloaded, based on the keywords, underwent manual review to identify relevant data points. Relevant posts were further manually analyzed through a process of data tagging, categorization, and clustering. Key themes relating to symptoms, impacts, treatment, and lived experiences were identified. RESULTS A total of 4456 social media posts related to presbyopia were identified between May 2017 and August 2017. Using a random sampling methodology, we selected 2229 (50.0%) posts for manual review, with 1470 (65.9%) of these 2229 posts identified as relevant to the study objectives. Twitter was the most commonly used channel for discussions on presbyopia compared to forums and blogs. The majority of relevant posts originated in Spain (559/1470, 38.0%) and the United States (426/1470, 29.0%). Of the relevant posts, 270/1470 (18.4%) were categorized as posts written by individuals who have presbyopia, of which 37 of the 270 posts (13.7%) discussed symptoms. On social media, individuals with presbyopia most frequently reported experiencing difficulty reading small print (24/37, 64.9%), difficulty focusing on near objects (15/37, 40.5%), eye strain (12/37, 32.4%), headaches (9/37, 24.3%), and blurred vision (8/37, 21.6%). 81 of the 270 posts (30.0%) discussed impacts of presbyopia—emotional burden (57/81, 70.4%), functional or daily living impacts (46/81, 56.8%), such as difficulty reading (46/81, 56.8%) and using electronic devices (21/81, 25.9%), and impacts on work (3/81, 3.7%). CONCLUSIONS Findings from this social media listening study provided insight into how people with presbyopia discuss their condition online and highlight the impact of presbyopia on individuals’ quality of life. The social media listening methodology can be used to generate insights into the lived experience of a condition, but it is recommended that this research be combined with prospective qualitative research for added rigor and for confirmation of the relevance of the findings.

Background: Heart failure with preserved ejection fraction (HFpEF) is a heterogenous clinical syndrome. Individuals living with HFpEF and obesity display a distinct phenotype that places considerable burden on their health-related quality of life. This study explored signs, symptoms, and impacts most relevant to individuals living with HFpEF and obesity. Clinician interviews provided a clinical perspective on the patient experience. Methodology: Qualitative interviews were conducted virtually with adults in the US diagnosed with HFpEF and obesity (body mass index ≥ 30.0 kg/m2). Clinicians in the US, China, and Germany with ≥ 3 years of experience treating HFpEF were also interviewed. Patient interviews were conducted until conceptual saturation was achieved. Content analysis was conducted using Atlas.ti v9.0 software. A conceptual model (CM) of the lived experience of HFpEF and obesity based on patient and clinician feedback was developed. Results: Twenty-two patients and 6 clinicians participated. The CM demonstrated that living with HFpEF impacts many facets of life, with patient and clinician participants reporting impacts on their (or their patients’) physical functioning (e.g., walking), activities of daily living (e.g., household chores) and work. Impacts on emotional well-being (e.g., feeling anxious or worried), social lives, relationships, and sleep were also reported. Shortness of breath and fatigue were spontaneously noted as salient and bothersome symptoms by most patient participants with edema noted by most on probing; this was further corroborated by clinicians who also considered these symptoms as the most bothersome symptoms of HFpEF. Conclusions: This study highlighted the substantial burden of living with HFpEF and obesity. The resultant conceptual model highlights the key concepts that should be considered in patient-focused drug development.

Background: Clinician-reported outcome measures (ClinROs) are completed by trained clinical experts, yet the signs and behaviors they capture must reflect meaningful aspects of health to patients. FDA Patient Focused Drug Development (PFDD) Guidance indicates the importance of establishing content validity for ClinROs. Patient and caregiver input can support the conceptual relevance of ClinROs and inform score interpretation such as meaningful change thresholds. Objective: To synthesize lessons learned from four qualitative case studies in which patient or caregiver input informed (1) ClinRO content validity and (2) within-patient clinically meaningful change thresholds, and to develop methodological recommendations for future studies. Methods: We reviewed four, purposively selected, qualitative studies designed to inform the development or validation of ClinROs. Contexts were varied across neurodegenerative and autoimmune conditions. Across studies, concept elicitation and cognitive debriefing interviews were conducted with patients or caregivers alongside clinical expert interviews. Visual aids and task adaptations were used to support participants’ comprehension of clinical terminology and inform scoring discussions. Results: We propose six methodological recommendations for incorporating patient/caregiver input during ClinRO development and validation. When gathering patient/caregiver input to inform content validity, researchers should: (1) frame the purpose; (2) use multimodal supports; (3) simplify scoring for the task; (4) map language. When developing an endpoint hierarchy researchers should align endpoints with patient priorities. When informing meaningful change thresholds, researchers should elicit discussion of patient perspective. The reviewed studies confirmed that patients and caregivers could generally understand and participate in interview tasks using visual aids provided. Conclusions: Incorporating patient/caregiver input during ClinRO development/validation strengthens content validity and provides qualitative insights into meaningful change thresholds to inform score interpretation. Practical techniques (advance framing, visual aids, simplified scoring) support patient/caregiver engagement with complex ClinROs. Methodological recommendations are provided for incorporating patient/caregiver input into ClinRO evidence generation to inform patient-focused clinical trial measurement strategies.