Qualitative Interviews to Better Understand the Patient Experience and Evaluate Patient-Reported Outcomes (PRO) in RLBP1 Retinitis Pigmentosa (RLBP1 RP).

INTRODUCTION: RLBP1 RP is an autosomal recessive form of retinitis pigmentosa (RP), characterized by night blindness, prolonged dark adaptation, constricted visual fields and impaired macular function. This study aimed to better understand the patient experience of RLBP1 RP and evaluate the content validity of existing patient reported outcome (PRO) instruments in this condition. METHODS: Semi-structured concept elicitation and cognitive debriefing interviews were conducted with RLBP1 RP patients in Canada and Sweden. Interviews started with open-ended concept elicitation questioning, and then patients were cognitively debriefed on The National Eye Institute Visual Functioning Questionnaire (NEI VFQ-25), the Low Luminance Questionnaire (LLQ) and four light/dark adaptation items of the Visual Activities Questionnaire (VAQ). Qualitative interviews were also conducted with three expert clinicians. Anonymized, verbatim transcripts were analyzed using thematic analysis. RESULTS: Twenty-one patients were interviewed (Canada n = 10; Sweden n = 11). Symptoms reported included night blindness (n = 21), difficulty adapting to changes in lighting (n = 21) and difficulties seeing in bright lighting (n = 18). Patients experienced substantial impacts on daily activities (n = 21) and physical functioning (n = 17). Patients had difficulty interpreting and selecting a response for some items in the NEI VFQ-25 and LLQ. Some items were not relevant to patients' disease experience. There were both gaps and overlaps in the conceptual coverage of the instruments. CONCLUSIONS: Visual impairment due to RLBP1 RP has a substantial impact on physical functioning and daily activities. To adequately assess all important symptoms and associated functional impacts in RLBP1 RP, it is recommended to either modify one or more existing instruments or to develop a new non-syndromic RP specific instrument.

Evaluating Patient Experiences in Dry Eye Disease Through Social Media Listening Research.

INTRODUCTION: Social media listening (SML) is an approach to assess patient experience in different indications. This is the first study to report the results of using SML to understand patients' experiences of living with dry eye disease (DED). METHODS: Publicly available, English-language social media content between December 2016 and August 2017 was searched employing pre-defined criteria using Social Studio®, an online aggregator-tool for posts from social media channels. Using natural language processing (NLP), posts were indexed using patient lexicon and disease-related keywords to derive a set of patient posts. NLP was used to identify relevance, followed by further manual evaluation and analysis to generate patient insights. RESULTS: In all, 2279 possible patient records were identified following NLP, which were filtered for relevance to disease area by analysts, resulting in a total of 1192 posts which formed the basis of this study. Of these, 77% (n = 915) were from the USA. Symptoms, causes, diagnosis and treatments were the most commonly discussed themes. Most common symptoms mentioned were eye dryness (138/901), pain (114/901) and blurry vision (110/901). Pharmaceutical drugs (prescription and over-the-counter; 55%; 764/1393), followed by medical devices (20%; 280/1393), were mentioned as major options for managing symptoms. Of the pharmaceutical drugs, eye drops (33%; 158/476) and artificial tears (10%; 49/476) were the most common over-the-counter options reported, and Restasis® (22%; 103/476) and Xiidra® (6%; 27/476) were the most common prescription drugs. Patients voiced a significant impact of DED on their daily activities (4%; 9/224), work (23%; 51/224) and driving (12%; 26/224). Lack of DED specialists, standard diagnostic procedures, effective treatment options and need to increase awareness of DED among patients were identified as the key unmet needs. CONCLUSIONS: Insights revealed using SML strengthen our understanding about patient experiences and their unmet needs in DED. This study illustrates that an SML approach contributed effectively in generating patient insights, which can be utilised to inform early drug development process, market access strategies and stakeholder discussions. FUNDING: Novartis Pharma AG, Basel, Switzerland. Plain language summary available for this article.

Resource Utilization Among Glaucoma Patients in the UK Treated with Beta-Blocker and Non-Beta-Blocker Adjunctive Therapy: A Retrospective Cohort Analysis.

INTRODUCTION: Few studies have examined outcomes and potential complications among glaucoma patients who are prescribed topical beta-blockers. This study examined resource usage (number of GP visits and hospitalizations) and diagnoses of respiratory or cardiovascular conditions among glaucoma patients prescribed beta-blockers compared to patients not prescribed beta-blockers. METHODS: A retrospective cohort analysis was conducted using data from the UK Clinical Practice Research Datalink (CPRD) database over the period January 1, 2006 to March 31, 2014. Adult patients with at least one diagnosis of glaucoma were categorized into beta-blocker users and non-beta-blocker users. Beta-blocker users were further separated into patients that maintained beta-blocker therapy and patients that discontinued beta-blocker treatment in year 2 of the post-index period. The CPRD data was queried directly to obtain the number of GP visits, and hospitalizations were extracted by linking the CPRD and Hospital Episode Statistics (HES) patient-level data. RESULTS: In the 12 months after being prescribed beta-blockers, patients that later discontinued beta-blocker treatment had a significantly higher average number of hospitalizations than patients that maintained beta-blocker therapy and the non-beta-blocker users (p < 0.05). In the year after beta-blocker initiation, there was a statistically significant within-group difference pre- and post-beta-blocker initiation for all groups, but the greatest number of GP visits occurred in the patients that subsequently discontinued beta-blocker treatment (mean 19.27). Patients that discontinued beta-blocker treatment were significantly more likely to have cardiovascular events than non-beta-blocker users in the post-index period (p < 0.05). CONCLUSION: This study suggests that the introduction of beta-blockers in a certain group of patients who later discontinue their use is associated with increased use of medical resources (higher number of GP visits and hospitalizations) in glaucoma patients in the UK, which may be indicative of a potential relationship between use of topical beta-blockers in glaucoma therapy and adverse outcomes.

Treatment costs of cystoid macular edema among patients following cataract surgery.

PURPOSE: The current costs of treating cystoid macular edema (CME), a complication that can follow cataract surgery, are largely unknown. This analysis estimates the treatment costs for CME based on the recently released US Medicare data. SETTING: Nationally representative database. DESIGN: Retrospective analysis of the 2011 through 2013 Medicare 5% Beneficiary Encrypted Files. METHODS: Beneficiaries who underwent cataract surgery were identified and stratified by diagnosis of CME (cases) or no diagnosis of CME (controls) within 6 months following surgery. Claims and reimbursements for ophthalmic care were identified. Subgroup analyses explored the rates of CME in beneficiaries based on the presence of selected comorbidities and by the type of procedure (standard vs complex). Total Medicare and ophthalmic costs for cases and controls are presented. The analysis explored the effect of considering diabetic macular edema (DME) and macular edema (ME) as exclusion criteria. RESULTS: Of 78,949 beneficiaries with cataract surgery, 2.54% (n=2,003) were diagnosed with CME. One-third of beneficiaries had one or more conditions affecting retinal health (including diabetes), 4.5% of whom developed CME. The rate of CME, at 22.5%, was much higher for those patients with preoperative DME or ME. Ophthalmic charges were almost twice as high for cases compared with controls (US$10,410 vs $5,950); payments averaged 85% higher ($2,720 vs $1,470) (both P<0.0001). CONCLUSION: Substantial costs can be associated with CME; beneficiaries whose retinas are already compromised before cataract surgery face higher risk. Cost savings could be realized with the use of therapies that reduce the risk of developing CME. Future analyses could identify whether and to what extent comorbidities influence costs.