[Realist protocol for implementing integrated care pathways in France (waiver 51 LFSS 2018)]. / Évaluer autrement les parcours de soins coordonnés article 51 LFSS 2018 : une innovation, les protocoles réalistes.

The paradox of real world researches supposedly conducted in real life is that they did not succeeded in freeing themselves from the techniques of the randomised trials which they pretended to escape. In the successionist perspective of classical experimental or quasi-experimental methods, the cause always precedes the effect in a linear manner, and any interference that is likely to threaten the stability of this relationship must be neutralised by mobilising the appropriate statistical techniques. In complex systems where everything moves at the same time due to multiple interrelationships that make it impossible to construct a counterfactual, these elements are no longer considered as confounding factors that need to be controlled, but as decisive factors in the smooth running of the experiment. The protocol presented in this article proposes an alternative evaluative technique mobilising the teachings of critical realism, which seems to us to be the most appropriate for understanding what happens "in the black box" recording the events that occur between the implementation of the Article 51 experiments and the observed results. The role of the evaluator is to put the actors back at the heart of the change, since it is achieved (or not) according to their reactions and the contextual elements. This credible explanatory theory allows us to understand: how does it work? For whom does it work? Why do losers lose, winners win? and under what circumstances?

Importance of out-of-pocket costs for adult patients with atopic dermatitis in France.

BACKGROUND: Currently, few studies investigated the economic burden of atopic dermatitis (AD) in adult patients and specifically the estimation of out-of-pocket costs. Patients with skin disorders primarily use comfort care to ease dryness, itch or pain, and the costs of comfort care are not subject to any reimbursement from mandatory or complementary insurance. OBJECTIVE: The purpose of this study was to measure the medical and non-medical expenses paid by the patient. METHODS: Eczema Cohort Longitudinal Adults was a non-interventional study that aimed to assess the burden of AD in terms of quality of life and financial consequences. A self-assessment questionnaire was distributed to adult patients who were cared in four French hospitals. Patients were asked to list the resources consumed for the treatment of AD during the last 12 months and to estimate the corresponding amount of money they had to pay out of their own pockets. The severity of AD was subjected to a stratification based on the PO-SCORAD score. RESULTS: A total of 1024 patients answered the questionnaire: 31.9% with severe AD, 40.4% with moderate AD and 27.6% with mild AD. The mean annual out-of-pocket cost was €462.1 for severe AD and €247.4 for moderate AD. Emollients were the most commonly used product: 74.4% for an average out-of-pocket cost of €151.4. The out-of-pocket costs increased significantly with the severity: 27% of patients with severe AD declared having bought specially textured clothes, while 19% of patients with moderate AD reported the same. The corresponding mean out-of-pocket costs were €162 and €91, respectively. CONCLUSION: The amount of out-of-pocket costs for patients with AD for essential medical and non-medical expenses is relatively high, compared to the average out-of-pocket cost for French households. Integration of these essential resources into the list of reimbursed products and services appears necessary for a better coverage of AD.

[How to measure real-world effectiveness?] / Comment mesurer l'efficacité en vie réelle ?

The new use of medico-administrative databases is a major change in the medical assessment field. Given the limits of the randomized clinical trials when it comes to accurately represent the heterogeneity and complexity of medical care in a real-world context, an increasing demand of observational studies comes from healthcare systems as decision tools. The lack of intervention generates real-world data and assesses both the care and the outcomes of patients in routine practice. Despite this external validity, the bias and confounding factors are a challenge to the internal validity of observational studies. An appropriate study design and statistical methods are necessary to neutralize them and consider a causal relationship between a treatment and a clinical outcome.

Psychometric Validation of the 14 items ChronIc Venous Insufficiency Quality of Life Questionnaire (CIVIQ-14): Confirmatory Factor Analysis.

OBJECTIVES: The study aim was to confirm the factorial structure of the short (14 item) version of the ChronIc Venous Insufficiency quality of life Questionnaire (CIVIQ-14) using the Vein Consult Program (VCP) results. METHODS: The international VCP study sought to evaluate the impact of chronic venous disease (CVD) on health care costs and quality of life (QoL). The factorial structure of the CIVIQ-14 was evaluated using two methods: exploratory factor analysis (EFA) to calculate the probabilities of items and dimensions remaining stable and to study the dimensionality of the scale using explained variance criteria, followed by confirmatory factor analysis (CFA) to confirm the original three dimensional structure and investigate alternative models that may have arisen from the dimensionality analysis. We also used the VCP results to evaluate the psychometric properties of the questionnaire and conducted subgroup analyses on countries with validated translations. RESULTS: A total of 47,149 questionnaires from 17 countries were available in the VCP. EFA revealed both items and dimensions as 100% stable. Dimensionality analysis showed that a two factor approach could be considered. CFA revealed the CIVIQ-14 three dimensional structure to be acceptable while rejecting the two dimensional model. Psychometric analysis confirmed the construct validity, internal consistency, and known groups validity of the CIVIQ-14. The results of subgroup analyses were consistent with those of the primary analysis. CONCLUSIONS: CFA of VCP data supported the factorial structure of the CIVIQ-14. The analysis corroborates the wide use of CIVIQ-14 as a valid instrument for reporting QoL in CVD patients.

Linguistic validation of the 20 item-chronic venous disease quality-of-life questionnaire (CIVIQ-20).

Our objective was to review the linguistic validation of the 20 item-ChronIc Venous dIsease quality-of-life Questionnaire (CIVIQ-20) in the countries that have used it since its publication in 1996. Seventeen linguistic versions of CIVIQ-20 were validated using forward/backward methodology in patients presenting with chronic venous disease, stages C0s to C4 of the CEAP (clinical, aetiological, anatomical and pathological) classification (patients with venous ulcers were excluded). Most obstacles in the cross-cultural validation of CIVIQ-20 related to content and semantic equivalence. Confirmation of cultural relevance by experts with the native language as their mother tongue and the use of forward/backward translation methodology partly resolved these difficulties. CIVIQ-20 is valid for the assessment of treatment effects in multinational studies.

[Risk-sharing agreements: choice of study design and assessment criteria]. / Contrats de risk-sharing : choix des schémas d'étude et des critères de jugement.

A new taxonomy of market entry agreements (MEA), also known as risk-sharing agreements, was built. It is no longer based on the conventional distinction between outcome performance and financial contracts, proposed by Carlson. Instead, it formulates a clear distinction between monitoring studies and evaluation or impact studies. The nature of the studies implemented within these two categories is fundamentally different: monitoring studies contribute to continuous program performance tracking against expected results and evaluation studies seek to identify the specific effect associated with the treatment while controlling for potential sources of selection bias. In accordance with this framework, differential study designs, indicators and financial clauses were proposed to reduce clinical, economic and budgetary uncertainty.

Construction and international validation of CIVIQ-14 (a short form of CIVIQ-20), a new questionnaire with a stable factorial structure.

BACKGROUND: The factorial instability of the CIVIQ-20 social dimension in different populations has necessitated the development of a new stable questionnaire to interpret results from international studies. OBJECTIVE: Construction of a stable and psychometrically validated questionnaire from CIVIQ-20. METHODS AND MAJOR FINDINGS: A prospective, international study was used to construct a stable CIVIQ scale and to validate its psychometric properties. An iterative process was implemented to eliminate the more unstable items (six), and the social and physical dimensions were combined. The resulting instrument comprised 14 items, split into three dimensions (pain, physical, and psychological), and was named CIVIQ-14. The stability of the CIVIQ-14 factorial structure was confirmed in Polish, Czech, Spanish, and French populations using principal component analysis and multitrait/multimethod analysis. Psychometric assessment demonstrated that CIVIQ-14 was reliable (intra-class coefficient >0.8; weighted kappa >0.8), valid (correlation coefficients between dimension scores and clinical severity scores between 0.3 and 0.6), and sensitive (effect sizes >0.6 for psychological dimension; >0.8 for the other dimensions). CONCLUSION: CIVIQ-14 is a reliable, valid, and sensitive instrument applicable to international studies of patients with chronic venous disease.

International psychometric validation of the Chronic Venous Disease quality of life Questionnaire (CIVIQ-20).

OBJECTIVE: To review the psychometric validation of the Chronic Venous dIsease quality of life Questionnaire (CIVIQ-20) in the countries that have used it since 1996. DESIGN: Prospective, clinical, international study in 18 countries. PATIENTS: Patients with venous disease of the lower limb in the clinical, aetiological, anatomical and pathophysiological (CEAP) clinical stages C0s to C4 presenting to surgical outpatient departments and general practices and receiving drug treatment for 6 months. METHODS: Quantification of symptoms on a four-point scale and pain on a visual analogue scale, and self-administration of CIVIQ-20 to patients before visit (baseline, 2, 4 and 6 months). RESULTS: In 3956 patients, CIVIQ-20 showed good internal consistency and reliability (above 0.80) through test-retest correlations. The discriminating power of items was good in known groups of patients. Factor analysis identified physical, psychological and pain factors as important, but revealed instability of the social factor. CIVIQ-20 was highly sensitive to changes in the quality of life of patients clinically improved after drug treatment. CONCLUSION: CIVIQ-20 is valuable in assessing treatment effects in longitudinal, multinational studies, but comparisons of different populations should use the global score rather than scores per dimension.

Comparative cost-minimisation of oral and intravenous chemotherapy for first-line treatment of non-small cell lung cancer in the UK NHS system.

The National Institute for Health and Clinical Excellence recommends vinorelbine (VNB), paclitaxel, docetaxel, and gemcitabine in the treatment of non-small cell lung cancer. An economic model was prepared to determine the comparative cost of these agents, including the new oral formulation of VNB from a United Kingdom National Health System perspective. Clinical effectiveness was determined from published trials. Costs of drug acquisition, administration, toxicity management, and patient transportation costs were calculated from reference publications. A Markov model was used to estimate the cost per patient over 52 weeks. Intravenous VNB, gemcitabine, paclitaxel, and docetaxel incur annual follow-up costs of 3,746 pounds, 5,332 pounds, 5,977 pounds, and 6,766 pounds, respectively, while oral VNB with outpatient administration on d1, and self-administration at home on d8 every 21 days has a cost per patient per year of 2,888 pounds. Oral VNB allows further hospital resources savings.

Estimating the cost-effectiveness of stroke units in France compared with conventional care.

BACKGROUND AND PURPOSE: The incidence of stroke in France is estimated at between 120 000 and 150 000 cases per year. This modeling study assessed the clinical and economic benefits of establishing specialized stroke units compared with conventional care. METHODS: Data from the Dijon stroke registry were used to determine healthcare trajectories according to the degree of autonomy and organization of patient care. The relative risks of death or institutionalization or death or dependence after passage through a stroke unit were compared with conventional care. These risks were then inserted with the costing data into a Markov model to estimate the cost-effectiveness of stroke units. RESULTS: Patients cared for in a stroke unit survive more trimesters without sequelae in the 5 years after hospitalization than those cared for conventionally (11.6 versus 8.28 trimesters). The mean cost per patient at 5 years was estimated at 30 983 for conventional care and 34 638 in a stroke unit. An incremental cost-effectiveness ratio for stroke units of 1359 per year of life gained without disability was estimated. CONCLUSIONS: The cost-effectiveness ratio for stroke units is much lower than the threshold (53 400 ) of acceptability recognized by the international scientific community. This finding justifies organizational changes in the management of stroke patients and the establishment of stroke units in France.

Translation and validation of a quality of life questionnaire for chronic lower limb venous insufficiency into greek.

AIM: In order to provide a practical instrument for quality of life measures, for researchers and primary care physicians in Greece, we decided to translate and validate the published postal questionnaire CIVIQ (Chronic Venous Insufficiency Questionnaire) into the Greek language. METHODS: The study took place in 2 vascular surgical clinics. Fifty patients with known chronic venous insufficiency (CVI) were asked to complete the questionnaire. A debriefing method was used for the cultural adaptation. To measure test-retest reliability, 50 patients with known CVI disease, were asked to complete the questionnaire. RESULTS: The adaptation of CIVIQ questionnaire in the Greek language demonstrated its good validity (Cronbach's alpha=0.92) and reproducibility (coefficient = 0.98). CONCLUSIONS: CIVIQ seems to be a reliable tool in helping to assess the health status of the Greek general population and could be used to contribute to the identification of the magnitude of the problem of CVI in the Greek setting.

Quality of life (Spain and France): validation of the chronic venous insufficiency questionnaire (CIVIQ).

A health-related quality of life (HRQOL) assessment is particularly necessary for patients with chronic venous insufficiency (CVI), as is an assessment of the objective signs (edema, dilated veins, ulceration and other lesions). A comprehensive 3-year research program was undertaken in France to construct and validate the Chronic Venous Insufficiency Questionnaire (CIVIQ), a questionnaire designed specifically to evaluate the quality of life for CVI sufferers. The next step was to implement a program of cross-cultural validation in several countries. Translation into Spanish was undertaken following international guidelines. Face validity was verified with a pilot test among 12 Spanish patients. Psychometric validation was performed in Spain (n = 476 patients with CVI). The Spanish CIVIQ demonstrated very good internal consistency, high reproducibility and responsiveness, as well as longitudinal clinical validity. It appears to be a valuable instrument for assessing improvement in patient quality of life in response to both therapy in clinical practice and clinical trials.

[Lymphedema and quality of life]. / Lymphoedème et qualité de vie.

Quality of life measurement shows interesting parameters in chronic lymphedema, related to the illness consequences and therapeutic strains. The ULL27 specific scale is an evaluation instrument which adds to the information about the patient and enables adaptation of the therapeutic strategy and also measurement of the treatment's impact of lymphedema.

[The European Evaluation of Vertigo (EEV) scale: a clinical validation study]. / Validation clinique d'une échelle du vertige: EEV (European Evaluation of Vertigo).

Up until now, the questionnaires used to evaluate vertigo have been self-administered questionnaires that rate either symptoms and/or their consequences. In contrast, the European Evaluation of Vertigo scale (EEV) is a physician-administered questionnaire that only assesses symptoms of the vestibular syndrome: illusion of movement, duration of illusion, motion intolerance, neurovegetative signs, and instability. Twenty-six ear, nose, and throat (ENT) specialists participated in this validation study conducted among 123 patients suffering from different types of vertigo. The reliability, responsiveness and construct, convergent and discriminant validity of the EEV scale were assessed. Construct, convergent and discriminant validity was determined by comparing the scores on the EEV scale with those obtained using the following validated scales: patient diary and conventional scale designed by the DPHM, functional scale of the AAO-HNS, and SF-36 scale, a generic quality of life scale. With the exception of the "neurovegetative signs" item, the reliability of the EEV scale is good, particularly in terms of interrater reliability (r = 0.93 for the global score), and responsiveness is high (p < 0.01). EEV scale has good correlations with the items of patient diary, DPHM scale and "physical" dimensions of SF-36, from the first evaluation, whereas this scale has poor correlation at Day 0, with the items of AAO scale and the "psychological" dimensions of SF-36. These results confirm that EEV scale is exclusively a symptomatic scale and does not interfere with the psychism. The EEV scale is therefore a validated physician-administered questionnaire capable of monitoring the course of vertigo and of assessing the efficacy of anti-vertigo treatments.

[Quality is expensive]. / La qualité coûte cher.

Over the past decade, as pressure to control costs has accelerated, the term cost effective has come increasingly into common usage. The central purpose of cost-effectiveness analysis is to compare the relative value of different interventions in creating better health. This article presents the rationale for using such an approach to prioritize public and private healthcare programmes.

Internet pharmaco-economic studies in metastatic colorectal cancer.

An interactive Web site has been developed: http://smbh7.smbh.univ-paris13.fr, which uses a Markov model to calculate the management costs for metastatic colorectal cancer. This site allows drug usage costs, daily tariff costs per site, local ISA point values and the cost to the society of the chemotherapies prescribed to be recorded by cycle in a de-centralised manner. The overall cost of treatment may be calculated by one of these four units from the time when the first chemotherapy was administered until the patient has escaped from first or second line treatment. The median time to progression and the median survival time are key parameters used to calculate costs as they determine the number of patients who remain on treatment, course by course. Effectiveness results have been measured in terms of progression free survival or of global survival. Eight treatment strategies have been examined. It is possible to add new treatment regimens or new compounds into the existing pre-formatted tables. This software enables budgets to be planned depending on the regimen used and the number of patients treated. It also allows the different treatment options to be classified with respect to their incremental cost effectiveness ratio, which is defined by the additional cost of one treatment option compared to another divided by the corresponding increase in effectiveness.

A cost-effectiveness analysis of stays in intensive care units.

OBJECTIVE: To evaluate patient outcome and the efficiency of stays in intensive care units (ICUs). DESIGN: Prospective study. SETTING: Seven ICUs of teaching hospitals in the Paris area. PATIENTS: Two hundred eleven stays including one in three consecutive patients admitted from September to November 1996. MEASUREMENTS AND MAIN RESULTS: For each patient, the following information was collected during the ICU stay: diagnosis, severity scores, organ failures, workload, cost and mortality. A cost-effectiveness ratio was computed for 176 stays with at least one organ failure, at hospital discharge and 6 months later. Quality of life was measured with EuroQol questionnaires 6 months after discharge in 64 patients representing 62 % of the patients contacted. The mean total ICU cost per stay was US$ 14,130 (+/- 6,550) (higher for non-survivors--US$ 19,060, median 10,590--than for survivors US$ 12,370, median 5,780). The incremental cost-effectiveness ratio was US$ 1,150 per life-year saved and the incremental cost-utility ratio was US$ 4,100 per quality-adjusted life-year (QALY) saved, without discounting. These results compare favourably with other health-care options. However substantial variations were observed according to age, severity, diagnosis, number of organ failures and discount rate. Intoxication had the lowest ratio (US$ 620/QALY) and acute renal insufficiency the highest (US$ 30,625/QALY). CONCLUSIONS: This work provides medical and economic information on ICU stays in teaching hospitals and enables comparisons with other health-care options.

[Evaluation of the quality of life of schizophrenic patients: validation of the brief version of the Quality of Life Interview]. / Evaluation de la qualité de vie des patients schizophrènes: validation de la version courte de la QoLI.

UNLABELLED: The main objective of the study was to determine the psychometric properties (mainly, validity and reliability) of the French language version of the brief Quality of Life Interview (QoLI). That instrument evaluates both the subjective and objective aspects of quality of life. METHODS: 128 patients fulfilling the DSM IV criteria for schizophrenia were included. Quality of life was evaluated using the brief QoLI. Schizophrenic symptoms were evaluated using the Positive and Negative Syndrome Scale (PANSS). The validity of the internal structure of the QoLI was investigated by means of item analysis, study of the correlations between the items and between the item-dimensions and principal component analysis addressing the subjective fields. The validity of the external structure was mainly investigated through the nomological validity study. The reliability of the scale was evaluated by studying the internal consistency. In addition, the acceptability of the scale was documented. RESULTS: The results of the study of the validity of the internal structure confirmed the pertinence of the pre-defined fields, particularly the subjective fields. Only some of the subjective fields of the QoLI showed significant correlations with the PANSS sub-scales. The acceptability of the scale was satisfactory. CONCLUSIONS: The psychometric properties of the French language version of the brief QoLI appeared satisfactory. The brief QoLi was shown to be an easily used instrument for assessment of the various objective and subjective aspects of the quality of life of schizophrenic patients.