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Aude Roborel de Climens,1 Amy Findley,2 Denise P Bury,3 Keri JS Brady,3 Matthew Reaney,4 Adam Gater2 1Patient-Centered Solutions, IQVIA RDS France, La Défense, Cedex, France; 2Patient-Centered Outcomes, Adelphi Values, Bollington, Cheshire, UK; 3Patient Informed Development & Health Value Translation, Sanofi, Cambridge, MA, USA; 4Patient-Centered Solutions, IQVIA, Reading, UKCorrespondence: Aude Roborel de Climens, Patient-Centered Solutions, IQVIA RDS, France, La Défense, Cedex, France, Email aude.roboreldeclimens@iqvia.comPurpose: Quantifying patient-perceived benefits and disadvantages of treatments in a real-world setting is increasingly important in healthcare decision-making. The Patient’s Qualitative Assessment of Treatment (PQAT) assesses patient-perceived benefits and disadvantages of treatment, and associated trade-offs potentially influencing patients’ willingness to continue treatment. It has then been modified to capture patients’ perceived magnitude of benefits and disadvantages of treatment quantitatively, as well as qualitatively (PQATv2). However, the PQAT and the PQATv2 were designed for use and validated in a clinical trial setting. The objective of this study was to adapt and test the content validity of a version of the PQATv2 for use in real-world settings (PQAT-RW).Patients and Methods: The PQATv2 was adapted for use in real-world settings (PQAT-RW), and its content was validated in 16 patients with varied chronic medical conditions and medication regimens via semi-structured qualitative interviews.Results: All participants reported that the PQAT-RW was “easy to understand”. The majority (n = 11/16) reported that the items covered all important aspects of their treatment experience, and that no items needed to be removed or added to the instrument. Analysis of free-text responses identified eight global concepts considered by participants when evaluating the benefits and disadvantages of treatment: treatment effectiveness, side effects and method of administration were most frequently considered (as both benefits and disadvantages), followed by frequency of administration, financial considerations, storage, packaging and drug preparation.Conclusion: The results of this study support the content validity of the PQAT-RW. They also demonstrate that using qualitative responses to contextualize quantitative responses provides unique insight into diverse and individualized patient-perceived benefits and disadvantages, and their relative importance, in real-world settings.Keywords: patient-reported outcome measure, qualitative research, real-world evidence, treatment experience

Asif H Khan,1 Adeline Abbe,1 Bruno Falissard,2 Paulo Carita,1 Claus Bachert,3,4 Joaquim Mullol,5 Matthew Reaney,6 Jingdong Chao,7 Leda P Mannent,1 Nikhil Amin,7 Puneet Mahajan,8 Gianluca Pirozzi,8 Laurent Eckert1 1Sanofi, Chilly-Mazarin, France; 2Centre de recherche en epidémiologie et santé des populations (CESP), INSERM U1018, Paris, France; 3Upper Airways Research Laboratory, Ghent University, Ghent, Belgium; 4CLINTEC, Karolinska Institutet, Stockholm, Sweden; 5Rhinology Unit & Smell Clinic, ENT Department, Hospital Clínic, Universitat de Barcelona; Clinical and Experimental Respiratory Immunoallergy, IDIBAPS; and CIBERES, Barcelona, Catalonia, Spain; 6IQVIA, Reading, UK; 7Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA; 8Sanofi, Bridgewater, NJ, USACorrespondence: Asif H KhanSanofi, 1 Avenue Pierre Brossolette, Chilly-Mazarin, 91380, FranceTel +33 1 60 49 77 77Email asif.khan@sanofi.comPurpose: Patient perspective is an important and increasingly sought-after complement to clinical assessment. The aim of this study was to transcribe individual patients’ experience of treatment in a dupilumab clinical trial through free-text responses with analysis using natural language processing (NLP) to obtain the unique perspective of patients on disease impact and unmet needs with existing treatment to inform future trial design.Patients and Methods: Patients with chronic rhinosinusitis with nasal polyps (CRSwNP) who were enrolled in a Phase IIa randomized controlled trial comparing dupilumab with placebo (NCT01920893) were invited to complete a self-assessment of treatment (SAT) tool at the end of treatment, asking, “What is your opinion on the treatment you had during the trial? What did you like or dislike about the treatment?” Free-text responses were analyzed for the overall cohort and according to treatment assignment using natural language processing including sentiment scoring. In a mixed-methods approach, quantitative patient-reported outcome (PRO) results were utilized to complement the qualitative analysis of free-text responses.Results: Of 60 patients enrolled in the study, 43 (71.6%) completed the SAT and responses from 37 patients were analyzed (placebo, n = 16; dupilumab, n = 21). Word analyses showed that the most common words were “smell,” “improve,” “staff,” “great,” “time,” and “good.” Across the whole cohort, “smell” was the most common symptom-related word. The words “smell” and “experience” were more likely to occur in patients treated with dupilumab. Patients treated with dupilumab also had more positive sentiment in their SAT responses than those who received placebo. The results from this qualitative analysis were reflected in quantitative PRO results.Conclusion: “Smell” was important to patients with CRSwNP, highlighting its importance as a patient-centric efficacy outcome measure in the context of clinical trials in CRSwNP.Trial Registration: ClinicalTrials.gov, NCT01920893. Registered 12 August 2013, https://www.clinicaltrials.gov/ct2/show/NCT01920893.Keywords: CRSwNP, free-text data mining, patient perspective, self-assessment, sense of smell

Urpo Kiiskinen,1 Stephan Matthaei,2 Matthew Reaney,3 Chantal Mathieu,4 Claes-Göran Östenson,5 Thure Krarup,6 Michael Theodorakis,7,* Jacek Kiljanski,8 Carole Salaun-Martin,9 Hélène Sapin,9 Bruno Guerci10 1Eli Lilly, Helsinki, Finland; 2Quakenbrück Diabetes Center, Quakenbrück, Germany; 3Eli Lilly, Windlesham, Surrey, UK; 4Department of Endocrinology, UZ Gasthuisberg, Leuven, Belgium; 5Department of Molecular Medicine and Surgery, Karolinska Institutet, Stockholm, Sweden; 6Department of Endocrinology, Bispebjerg Hospital, Copenhagen, Denmark; 7Department of Clinical Therapeutics, University of Athens School of Medicine, Athens, Greece; 8Eli Lilly, Warsaw, Poland; 9Eli Lilly, Neuilly Cedex, France; 10Department of Diabetes, Metabolic Diseases, and Nutrition, Hôpital Brabois, Vandoeuvre-Lès-Nancy, France *Michael Theodorakis was affiliated with the institution shown above at the time of the study, but has since left this institution Purpose: CHOICE (CHanges to treatment and Outcomes in patients with type 2 diabetes initiating InjeCtablE therapy) assessed patterns of exenatide bid and initial insulin therapy usage in clinical practice in six European countries and evaluated outcomes during the study. Methods: CHOICE was a 24-month, prospective, noninterventional observational study. Clinical and resource use data were collected at initiation of first injectable therapy (exenatide bid or insulin) and at regular intervals for 24 months. Costs were evaluated from the national health care system perspective at 2009 prices. Results: A total of 2515 patients were recruited. At the 24-month analysis, significant treatment change had occurred during the study in 42.2% of 1114 eligible patients in the exenatide bid cohort and 36.0% of 1274 eligible patients in the insulin cohort. Improvements in glycemic control were observed over the course of the study in both cohorts (P < 0.001 for both), but mean weight was reduced in the exenatide bid cohort (P < 0.001) and increased in the insulin cohort (P < 0.001) by 24 months. Across all countries, total per patient health care costs for the 24 months post baseline were €3997.9 in the exenatide bid cohort and €3265.5 in the insulin cohort (€1791.9 versus €2465.5 due to costs other than those of injectable therapy). When baseline direct cost and patients' and disease characteristics were controlled for, mean direct costs differed by country (P < 0.0001), irrespective of treatment initiated, and the mean cost difference between treatments varied by country (P < 0.0001). Conclusion: Much of the higher mean cost of exenatide bid, compared with insulin, therapy was compensated for by lower mean costs of other health service utilization. Costs associated with exenatide bid or insulin initiation varied across countries, highlighting the need to avoid generalization of resource use and cost implications of a particular therapy when estimated in specific country settings. Keywords: exenatide, health care costs, injectable therapy, insulin, resource use, type 2 diabetes mellitus

Claes-Göran Östenson,1 Stephan Matthaei,2 Matthew Reaney,3 Thure Krarup,4 Bruno Guerci,5 Jacek Kiljanski,6 Carole Salaun-Martin,7 Hélène Sapin,7 David Bruhn,8 Chantal Mathieu,9 Michael Theodorakis10 1Department of Molecular Medicine and Surgery, Karolinska Institute, Stockholm, Sweden; 2Diabetes-Center Quakenbrück, Quakenbrück, Germany; 3Eli Lilly, Windlesham, Surrey, UK; 4Department of Endocrinology I, Bispebjerg Hospital, Copenhagen, Denmark; 5Diabetology, Metabolic Diseases and Nutrition, Brabois Hospital, CHU Nancy, and INSERM CIC, ILCV, Vandoeuvre Lès Nancy, France; 6Eli Lilly, Warsaw, Poland; 7Eli Lilly, Neuilly Cedex, France; 8Eli Lilly, San Diego, California, USA; 9Department of Endocrinology, UZ Gasthuisberg, Leuven, Belgium; 10Department of Clinical Therapeutics, University of Athens School of Medicine, Athens, Greece* *Michael Theodorakis was affiliated with the institution shown at the time of the study, but has since left this institution Objective: The CHanges to treatment and Outcomes in patients with type 2 diabetes initiating InjeCtablE therapy (CHOICE) study assessed time to, and reasons for, significant treatment change after patients with type 2 diabetes (T2DM) initiated their first injectable glucose-lowering therapy (exenatide twice daily [BID] or insulin) in routine clinical practice, and these patients’ clinical outcomes, in six European countries. This paper reports interim data from the first 12 months of the study. Research design and methods: CHOICE (NCT00635492) is a prospective, noninterventional, observational study. Clinical data were collected at initiation of first injectable therapy and after approximately 3, 6, and 12 months. Results: Of 2497 patients enrolled in CHOICE, 1096 in the exenatide BID and 1239 in the insulin cohorts had ≥1 post-baseline assessment and were included in this analysis. Overall, 32.2% of the exenatide BID cohort and 29.1% of the insulin cohort (Kaplan–Meier estimates) had significant treatment change during the first 12 months, most commonly discontinuing injectable therapy or adding new T2DM therapy, respectively. Glycemic control improved in both cohorts, but weight loss occurred only in the exenatide BID cohort (mean change –3.3 kg). Hypoglycemia occurred in 13.2% of the exenatide BID cohort and 28.6% of the insulin cohort (82.8% and 55.6% of these patients, respectively, received sulfonylureas). The post hoc endpoint of glycated hemoglobin < 7%, no weight gain, and no hypoglycemia was attained at 12 months by 24.3% and 10.3% of patients who had data at 12 months and who were receiving exenatide BID and insulin, respectively. Conclusion: About 30% of patients in CHOICE changed treatment in the first 12 months after initiation of first injectable therapy (exenatide BID or insulin). Overall, both cohorts achieved improved glycemic control, which was accompanied by a mean weight loss in the exenatide BID cohort. Keywords: type 2 diabetes mellitus, exenatide, insulin, injectable therapy