Development and First Use of the Patient's Qualitative Assessment of Treatment (PQAT) Questionnaire in Type 2 Diabetes Mellitus to Explore Individualised Benefit-Harm of Drugs Received During Clinical Studies.

INTRODUCTION: Individualised benefit-harm assessments can help identify patient-perceived benefits and harms of a treatment, and associated trade-offs that may influence patients' willingness to use a treatment. This research presents the first use of a patient-reported outcome measure designed to assess patient-perceived benefits and disadvantages of drugs received during clinical studies. METHODS: The Patient's Qualitative Assessment of Treatment (PQAT) was developed in English and cognitively tested with US (n = 4) and Canadian (n = 3) patients with type 1 and type 2 diabetes mellitus (T2DM). The revised version of the PQAT comprises three qualitative open-ended questions focused on the benefits and disadvantages of treatment and reasons why patients would choose to continue/discontinue treatment. A final quantitative question asks patients to evaluate the balance between benefits and disadvantages using a 7-point scale. The revised version of the questionnaire was administered as an exploratory endpoint in a phase II clinical trial for a new injectable treatment for T2DM. Qualitative data were analysed using thematic analysis, and relationships between qualitative and quantitative data were identified. RESULTS: Patient-reported benefits of treatment administered during the clinical trial included clinical markers of efficacy and subjective markers. Disadvantages reported by patients were mainly related to drug adverse effects or to the mode of administration. Of the 57 patients completing the PQAT, 70.2% reported being willing to continue treatment, with 59.6% reporting that the benefits outweighed the disadvantages. The reported benefits of feeling better and improved energy levels were more likely to be associated with a more positive ratio (70% and 71.4%, respectively), while the disadvantages of fatigue, headaches, and stomach pain were associated with a negative ratio and patients not being willing to continue the treatment. CONCLUSIONS: The PQAT is a unique patient-reported outcome tool designed to aid understanding patients' real experience of benefits and disadvantages of a treatment. It combines the richness of qualitative data with quantitative data-information valuable for various stakeholders to make well-informed treatment decisions. TRIAL REGISTRATION: ClinicalTrials.gov identifier: NCT02973321.

Irritable bowel syndrome in France: quality of life, medical management, and costs: the Encoli study.

AIM: To assess the proportion of physician-diagnosed irritable bowel syndrome (IBS) patients who conform to Rome II criteria, and evaluate the impact of IBS on quality of life (QoL) and costs in France. METHODS: This cross-sectional study collected information retrospectively from the preceding year. Physicians (randomly selected) enrolled their first four IBS patients during the study period. Patients were classified (secondarily) according to Rome II criteria. IBS-related costs during the previous year were estimated. RESULTS: Of the 452 physician-diagnosed IBS patients (mean age 53.9+/-14.9 years; 75.4% women), 23% did not meet the diagnostic criteria; 36.5% met all diagnostic criteria, and 32% experienced severe or very severe abdominal pain/discomfort (Subjects' Global Assessment of relief). IBS patients had lower QoL (SF-36 scale) than the general French population, and Rome II patients had the lowest QoL. Most prescriptions treated abdominal pain (72%) and bloating (58%). Approximately 8% of patients had been hospitalized due to IBS during the preceding year. The average total annual direct cost/patient was 756 euro. CONCLUSIONS: These data confirm that IBS involves large direct medical costs in France, and that IBS patients experience lower QoL than the general population. A small and not significant economic difference between the Rome II and non-Rome II IBS patient groups was observed.

[Perspectives of general practitioners on the role of patients' caregivers in the process of consultation and treatment]. / Représentations par les médecins généralistes du rôle de l'entourage accompagnant le patient.

The aim of this study is to describe, from the perspective of general practitioners, the role of relatives and caregivers who accompany a patient in their consultation and treatment processes. 435 general practitioners filled out two questionnaires: the first was self administered and the second described specific clinical situations and the possible role of the family caregiver in the case of a patient with Alzheimer's diseases, Parkinson's disease, depression, epilepsy, asthma, cardiovascular disease, or type II diabetes. General practitioners think that overall they offer satisfactory responses to relatives' requests and concerns regarding the disease itself and its treatment. However, they do not feel skilled or qualified enough to answer them with regard to administrative or social questions. The relative's role is for the most part, perceived as positive, and seems to contribute to the efficacy of the care provided. This description corresponds to a new trend in modern medical practice, dominated by the burden of chronic and disabling diseases which implies, and in some cases requires, the need to involve a relative's presence and on-going support.

Epidemiology, impact, management, and cost of Paget's disease of bone in France.

OBJECTIVES: To evaluate the epidemiology, quality-of-life (QoL) impact, management, and cost of Paget's disease of bone. METHODS: Retrospective and prospective data were collected in a representative cross-section of office-based and hospital-based rheumatologists in France, in early 2005. Each rheumatologist included consecutive outpatients with Paget's disease seen over a 2-month period. For each patient, a medical questionnaire and the 36-item Short-Form self-questionnaire (SF-36) evaluating health-related quality of life were completed. A descriptive analysis of the patient population was conducted. Medical costs over the last 12 months were estimated. RESULTS: Four hundred and forty-six medical questionnaires and 387 SF-36 questionnaires were available. Mean age was 74 years, and 58.3% of patients were male. The diagnosis was fortuitous (usually radiological) in 39.2% of cases. Complications occurred in 40% of cases; 97.8% of complications involved the joints. Bisphosphonate therapy was used in nearly 84% of patients. The SF-36 scores were significantly decreased compared to the general population, in both males and females. Total cost of management was less than one-third the cost of managing type 2 diabetes mellitus. CONCLUSION: The results confirm the well-established characteristics of Paget's disease. Bisphosphonate therapy was widely used. Quality of life was significantly altered. The cost of management was moderate.

Management of chronic myeloid leukemia in France: a multicentered cross-sectional study on 538 patients.

PURPOSE: Little is known about the actual management and treatment of chronic myeloid leukemia (CML) in clinical practice, although there have been many recent changes, such as the introduction of imatinib. PATIENTS AND METHODS: A two-phase cross-sectional observational study with retrospective data collection was conducted in France. In the first phase information regarding health services treating patients with CML was collected. In the second phase, centers caring for 10 or more patients were asked to provide data regarding patients diagnosed with CML that had had a consultation or been hospitalized in the last 3 months. RESULTS: All French departments of hematology (n=218) were contacted by phone. The median number of patients followed per center is 6 (range 0--200). The median number of new patients seen during the last 12 months was 2 (range 0--60). In the second phase 538 patients were included, the sex ratio being 1.14 and median age 55. At the time of diagnosis, 96.8% (n=519) were in chronic phase, 2.2% (n=12) in accelerated phase and 0.9% (n=5) in blastic phase. Eighty-two percent (n=443) of the patients have been treated by interferon (IFN). Sixteen point 3% (n=87) of the patients received a bone marrow transplantation (BMT). Forty-six percent (n=236) of the patients were treated with imatinib. CONCLUSIONS: This is the first study providing detailed descriptive data concerning the use of medications and procedures in a large population of patients from the medical centers involved in treating CML patients in France. Further observational studies are needed to assess the impact of different treatment strategies and economic impact of CML care in France.