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Background: We aimed to assess the effectiveness and cost of patients with first line tyrosine kinase inhibitors (TKIs) sequence of first (1G) and second generation (2G) followed by osimertinib. Materials & methods: Using the French nationwide claims and hospitalization database, we analyzed non-small-cell lung cancer patients who had been treated with osimertinib between April 2015 and December 2017, after a first line treatment with a TKI-1G/2G. Results: The median time on treatment for sequential TKI-1G/2G followed by osimertinib was 34 months (95% CI: 31–46); 13 and 12months, respectively for TKI 1G or 2G and TKI 3G, respectively. The median overall survival for sequential TKI 1G or 2G followed by osimertinib was 37 months (95% CI: 34–42). The mean monthly costs per patient was €5162. Conclusion: These results, in line with those observed during clinical trials, confirm the effectiveness of the sequence TKI-1G/2G followed by osimertinib in EGFR-mutated non-small-cell lung cancer.

Background There is a paucity of data on the epidemiology, survival estimates and healthcare resource utilisation and associated costs of patients with progressive fibrosing interstitial lung disease (PF-ILD) in France. An algorithm for extracting claims data was developed to indirectly identify and describe patients with PF-ILD in the French national administrative healthcare database. Methods The French healthcare database, the Système National des Données de Santé (SNDS), includes data related to ambulatory care, hospitalisations and death for 98.8% of the population. In this study, algorithms based on age, diagnosis and healthcare consumption were created to identify adult patients with PF-ILD other than idiopathic pulmonary fibrosis between 2010 and 2017. Incidence, prevalence, survival estimates, clinical features and healthcare resource usage and costs were described among patients with PF-ILD. Results We identified a total of 14,413 patients with PF-ILD. Almost half of them (48.1%) were female and the mean (± standard deviation) age was 68.4 (± 15.0) years. Between 2010 and 2017, the estimated incidence of PF-ILD ranged from 4.0 to 4.7/100,000 person-years and the estimated prevalence from 6.6 to 19.4/100,000 persons. The main diagnostic categories represented were exposure-related ILD other than hypersensitivity pneumonitis (n = 3486; 24.2%), idiopathic interstitial pneumonia (n = 3113; 21.6%) and rheumatoid arthritis-associated ILD (n = 2521; 17.5%). Median overall survival using Kaplan–Meier estimation was 3.7 years from the start of progression. During the study, 95.2% of patients had ≥ 1 hospitalisation for respiratory care and 34.3% were hospitalised in an intensive care unit. The median (interquartile range) total specific cost per patient during the follow-up period was €25,613 (10,622–54,287) and the median annual cost per patient was €18,362 (6856–52,026), of which €11,784 (3003–42,097) was related to hospitalisations. Limitations included the retrospective design and identification of cases through an algorithm in the absence of chest high-resolution computed tomography scans and pulmonary function tests. Conclusions This large, real-world, longitudinal study provides important insights into the characteristics, epidemiology and healthcare resource utilisation and costs associated with PF-ILD in France using a comprehensive and exhaustive database, and provides vital evidence that PF-ILD represents a high burden on both patients and healthcare services. Trial registration ClinicalTrials.gov, NCT03858842. ISRCTN, ISRCTN12345678. Registered 3 January 2019—Retrospectively registered, https://clinicaltrials.gov/ct2/show/NCT03858842

Objectives: The objectives of this study were to describe the impact of systemic sclerosis associated interstitial lung disease, on quality of life, to estimate the correlation between quality of life and severity of lung disease and to assess the impact of interstitial lung disease on caregivers. Methods: Seven investigators included systemic sclerosis associated interstitial lung disease patients from December 2019 to April 2020. Sociodemographics and clinical data were collected. Patients reported outcomes and questionnaires were used with 1 generic patients reported outcome (EQ-5D-5L), 1 specific PRO (Brief Interstitial Lung Disease) and 2 self-reported questionnaires on impact of SSc complications and impact on caregivers. The correlation between forced vital capacity and EQ-5D-5L score was estimated with a multivariate linear regression model adjusted on several covariates. Results: In all, 89 patients were included. 26.4% were males, mean age was 58.2 ± 14.5 years. Mean EQ-5D-5L score = 0.79 ± 0.22 (median = 0.85). Mean EQ-5D-5L visual analog scale score = 60.8 ± 20.4 (median = 61.5). Mean King’s Brief Interstitial Lung Disease score = 58.4 ± 12.7 (median = 58.0). After adjustment on covariates, a significant correlation between forced vital capacity and EQ-5D-5L score was found with an increase of 0.003 of the EQ-5D-5L score for a 1% increase of FVC (p = 0.0096). No significant correlation between forced vital capacity and the EQ-VAS and King’s Brief Interstitial Lung Disease score were found. The impact of SSc on other organs was significantly correlated with EQ- 5D-5L score, respectively, for the impact scores on the lung system (p = 0.0003), heart system (p = 0.0182), Raynaud’s syndrome (p = 0.0015), digestive system (p = 0.0032), joints/muscles (p = 0.0003), skin (p < 0.0001), kidney (p = 0.0052) and gastro-oesophageal reflux (p = 0.0063). Significant correlations between King’s Brief Interstitial Lung Disease score and lung system (p < 0.0001), heart system (p < 0.0001), digital ulcers (p = 0.058), digestive system (p < 0.0001), kidney (p = 0.0004), skin (p = 0.0499) and gastro-oesophageal reflux (p = 0.0033) scores were found 68.5% of patients reported their need for a caregiver to help them in their daily life activities. Conclusion: Our study highlighted the strong burden of systemic sclerosis associated interstitial lung disease` for patients, especially with an impact on quality of life, on other organs manifestations and need for caregivers in their daily life.

Idiopathic pulmonary fibrosis (IPF) is a specific form of chronic, progressive, fibrosing interstitial pneumonia of unknown cause. To date, there is no specific cure for IPF, and only two treatments (pirfenidone and nintedanib) have marketing authorizations and recommendations in international and French guidelines.A cost-utility analysis (CUA) has been conducted to evaluate the efficiency of nintedanib, in comparison to all available alternatives, in a French setting using the official methodological guidelines.A previously developed lifetime Markov model was adapted to the French setting by simulating the progression of IPF patients in terms of lung function decline, incidence of acute exacerbations, and death. Considering the effect of IPF on patients' quality-of-life, a CUA integrating quality adjusted life years (QALY) was chosen as the primary outcome measure in the main analysis. One-way, probabilistic, and scenario sensitivity analyses were performed to evaluate the robustness of the model.Treatment with nintedanib resulted in an estimated total cost of €76,414 (vs €82,665 for pirfenidone). In comparison with all other available options, nintedanib was predicted to provide the most QALY gained (3.34 vs 3.29). This analysis suggests that nintedanib has a 59.0% chance of being more effective than pirfenidone and s 77.3% chance of being cheaper than pirfenidone. Sensitivity analyses showed the results of the CUA to be robust.In conclusion, this CUA has found that nintedanib appears to be a more cost-effective therapeutic option than pirfenidone in a French setting, due to fewer acute exacerbations and a better tolerability profile.

Arnaud Cavailles,1 Boris Melloni,2 Stéphane Motola,3 Florent Dayde,3 Marie Laurent,3 Katell Le Lay,4 Didier Caumette,5 Laura Luciani,4 Pierre Louis Lleu,6 Geoffrey Berthon,7 Thomas Flament8 1Service de Pneumologie, Institut du Thorax, CHU de Nantes, Nantes, France; 2Service de Pneumologie, CHU Dupuytren, Limoges, France; 3HEVA, Lyon, France; 4HEOR/RWE, Boehringer Ingelheim, Paris, France; 5Institutional and Hospital Partnership, Boehringer Ingelheim, Paris, France; 6Medical Affairs, Boehringer Ingelheim, Paris, France; 7CHRU Tours, Tours, France; 8Service de Pneumologie, CHRU Bretonneau, Tours, FranceCorrespondence: Marie Laurent 186 Avenue Thiers, Lyon 69006, FranceTel +33 4 72 74 25 60Email mlaurent@hevaweb.comPurpose: To characterise patients with chronic obstructive pulmonary disease (COPD) who are rehospitalised for an acute exacerbation, to estimate the cost of these hospitalisations, to characterise high risk patient sub groups and to identify factors potentially associated with the risk of rehospitalisation.Patients and Methods: This was a retrospective study using the French NationalHospital Discharge Database. All patients aged ≥ 40 years hospitalised for an acute exacerbation of COPD between 2015 and 2016 were identified and followed for six months. Patients with at least one rehospitalisation for acute exacerbation of COPD constituted the rehospitalisation analysis population. A machine learning model was built to study the factors associated with the risk of rehospitalisation using decisiontree analysis. A direct cost analysis was performed from the perspective of national health insurance.Results: A totalof 143,006 eligible patients were hospitalised for an acute exacerbation of COPD (AECOPD) in 2015– 2016 (mean age: 74 years; 62.1% men). 25,090 (18.8%) were rehospitalised for another exacerbation within six months. In this study, 8.5% of patients died during or immediately following the index hospitalisation and 10.5% died during or immediately after rehospitalisation (p < 0.001). The specific cost of these rehospitalisations was € 5304. The overall total cost per patient of all AECOPD-related stays was € 9623, being significantly higher in patients who were rehospitalised (€ 16,275) compared to those who were not (€ 8208). In decisiontree analysis, the most important driver of rehospitalisation was hospitalisation in the previous two years (contributing 85% of the information).Conclusion: Rehospitalisations for acute exacerbations of COPD carry a high epidemiological and economic burden. Since hospitalisation for an acute exacerbation is the most important determinant of future rehospitalisations, management of COPD needs to focus on interventions aimed at decreasing the rehospitalisation risk of in order to lower the burden of disease.Keywords: comorbidity, rehospitalisation, decisiontree analysis, cost

International audience; Objectives: Understanding inhaler preferences may contribute to improving adherence in COPD patients and improving long-term outcomes. This study aims to identify and quantify preferences for convenience-related inhaler attributes in French moderate-to-severe COPD patients, with discrete choice experiment (DCE) methodology. Methods: Attributes were defined from a literature search, clinician and patient interviews: shape, dose insertion, dose preparation, dose release, dose confirmation, dose counter and reusability. An online DCE was conducted in respondents with self-reported COPD stage 2-4 recruited through a panel. The study questionnaire included twelve choice scenarios per respondent and questions on patient characteristics, treatment and disease severity. Statistical analyses used a mixed logit regression model with random effects. Utility scores were estimated for four types of inhalers: Inhaler Asoft mist inhaler; Inhaler Breusable soft mist inhaler; Inhaler Cmulti-dose dry powder inhaler; and Inhaler Dsingle dose dry powder inhaler. Results: The study was completed by 153 patients (50 females); respondents were 50.4 years old on average; 13 different inhaler devices were reported. The most preferred inhaler is L-shaped, has dose preparation with capsule insertion and a dose counter, and is reusable. Inhaler profiles A and B had the highest utilities (mean of 1.2533 and 0.9578 respectively) compared to inhaler C (0.6315) and D (0.2200). Conclusions: This study showed statistically significant results that the strongest drivers of preference in French users of inhalation devices for COPD are shape, dose counter and reusability. Conveniencerelated characteristics are important to patients and should be taken into account by clinicians prescribing these devices.

Introduction: Poor inhaler adherence is a major challenge in chronic obstructive pulmonary disease (COPD), and understanding drivers of inhaler preferences may contribute to adherence and improve long term outcomes. Aims and objective: To identify and quantify preferences for inhaler attributes in French COPD patients, by using DCE methodology. Methods: Attributes were defined from a literature search, expert and patient interviews. An online DCE with COPD patients aged 40-75, using an inhaler for COPD was conducted. The pilot study included scenarios of two fictitious inhalers described according to the attributes. The COPD Assessment Test and 18 questions on diagnosis, treatment and socio-demographic profile were included. A D-efficient design with zero priors was used. Analyses used a conditional logit model with interactions. Results: Seven inhaler attributes were defined: shape, dose insertion, dose preparation, dose release, dose confirmation, dose counter, and reusability. Twenty-one patients completed the pilot phase, and 5 did cognitive debriefing interviews. The mean CAT score was 27.14 (range 17-40). Results show the most valued characteristics to be dose insertion, dose counter and reusability, with a tendency to prefer fewer dose preparation steps, and reusable inhalers with a dose counter. Conclusion: The pilot results suggest that the selected attributes are relevant to COPD patients in France, and should therefore be taken into account by healthcare professionals considering treatment options. In the full online survey, 150 patients with COPD will be included; will confirm which attributes are most valued for French COPD patients.

Maud Pignata,1 Christos Chouaid,2 Katell Le Lay,3 Laura Luciani,3 Ceilidh McConnachie,4 James Gordon,5 Stéphane Roze1 1HEVA-HEOR, Lyon, 2Centre Hospitalier, Intercommunal de Créteil, Créteil, 3Boehringer Ingelheim, Paris, France; 4Ossian Health Economics and Communications, Basel, Switzerland; 5Boehringer Ingelheim, GmbH, Germany Background and aims: Lung cancer has the highest mortality rate of all cancers worldwide. Non-small-cell lung cancer (NSCLC) accounts for 85% of all lung cancers and has an extremely poor prognosis. Afatinib is an irreversible ErbB family blocker designed to suppress cellular signaling and inhibit cellular growth and is approved in Europe after platinum-based therapy for squamous NSCLC. The objective of the present analysis was to evaluate the cost-effectiveness of afatinib after platinum-based therapy for squamous NSCLC in France.Methods: The study population was based on the LUX-Lung 8 trial that compared afatinib with erlotinib in patients with squamous NSCLC. The analysis was performed from the perspective of all health care funders and affected patients. A partitioned survival model was developed to evaluate cost-effectiveness based on progression-free survival and overall survival in the trial. Life expectancy, quality-adjusted life expectancy and direct costs were evaluated over a 10-year time horizon. Future costs and clinical benefits were discounted at 4% annually. Deterministic and probabilistic sensitivity analyses were performed.Results: Model projections indicated that afatinib was associated with greater life expectancy (0.16years) and quality-adjusted life expectancy (0.094 quality-adjusted life years [QALYs]) than that projected for erlotinib. The total cost of treatment over a 10-year time horizon was higher for afatinib than erlotinib, EUR12,364 versus EUR9,510, leading to an incremental cost-effectiveness ratio of EUR30,277 per QALY gained for afatinib versus erlotinib. Sensitivity analyses showed that the base case findings were stable under variation of a range of model inputs.Conclusion: Based on data from the LUX-Lung 8 trial, afatinib was projected to improve clinical outcomes versus erlotinib, with a 97% probability of being cost-effective assuming a willingness to pay of EUR70,000 per QALY gained, after platinum-based therapy in patients with squamous NSCLC in France. Keywords: cost, cost-effectiveness, afatinib, lung cancer

Objective A survey was performed in 5 European countries (France, Germany, Italy, Portugal, and Spain) to estimate the prevalence of fibromyalgia (FM) in the general population. Methods In each country, the London Fibromyalgia Epidemiological Study Screening Questionnaire (LFESSQ) was administered by telephone to a representative sample of the community over 15 years of age. A positive screen was defined as the following: (1) meeting the 4-pain criteria alone (LFESSQ-4), or (2) meeting both the 4-pain and the 2-fatigue criteria (LFESSQ-6). The questionnaire was also submitted to all outpatients referred to the 8 participating rheumatology clinics for 1 month. These patients were examined by a rheumatologist to confirm or exclude the FM diagnosis according to the 1990 American College of Rheumatology classification criteria. The prevalence of FM in the general population was estimated by applying the positive-predictive values to eligible community subjects (ie, positive screens). Results Among rheumatology outpatients, 46% screened positive for chronic widespread pain (LFESSQ-4), 32% for pain and fatigue (LFESSQ-6), and 14% were confirmed FM cases. In the whole general population, 13 and 6.7% screened positive for LFESSQ-4 and LFESSQ-6, respectively. 3The estimated overall prevalence of FM was 4.7% (95% CI: 4.0 to 5.3) and 2.9% (95% CI: 2.4 to 3.4), respectively, in the general population. The prevalence of FM was age- and sex-related and varied among countries. Conclusion FM appears to be a common condition in these 5 European countries, even if data derived from the most specific criteria set (LFESSQ-6) are considered.

Context The multidimensional nature of the fibromyalgia syndrome means that different instruments need to be used to assess the patients’ perception of this constellation of physical and psychological symptoms and their impact on their daily lives. Six questionnaires (Multidimensional Fatigue Inventory MFI, Fibromyalgia Impact Questionnaire FIQ, Multiple Ability Self-report Questionnaire MASQ, State-Trait Anxiety Inventory STAI, Beck Depression Inventory-II BDI-II and Patient Global Impression of Change PGIC) were linguistically validated into 12 languages. Methods The standardized cross-cultural adaptation process includes the following steps: forward translation, backward translation, and review of the version by a clinician and comprehension tests on subjects in the target country. Results Regardless of the instruments and dimensions studied, the same translation and cultural adaptation issues arose: (1) an issue that is strictly related to translation, for example, the word “things” was translated as “something”; (2) literal translation is possible but culturally irrelevant, for example the expression “to walk several blocks”, which is a completely abstract concept in Europe, was translated as “to walk for more than one kilometre”; (3) the translation needed to be reformulated or the tense needed to be changed for idiomatic reasons. For example, the present perfect does not exist in German and so the present simple was used in the first version. The imperfect was eventually used with adverbs such as “lately”. Conclusions Linguistic adaptation was completed according to a recognized and rigorous method allowing for the wide-scale use of these patient-reported outcomes instruments in international studies.

Resume Objectifs La symptomatologie complexe de la fibromyalgie necessite l’utilisation de plusieurs instruments pour evaluer la perception par le patient de cette constellation de symptomes physiques et psychologiques et identifier leur impact sur la vie quotidienne. Six questionnaires ( Multidimensional Fatigue Inventory, Multiple Ability Self-Report Questionnaire, State-Trait Anxiety Inventory, Fibromyalgia Impact Questionnaire, Beck Depression Inventory-II, Patient Global Impression Of Change ) ont ete adaptes linguistiquement en 12 langues. Methodes Le processus d’adaptation culturelle comprenait deux traductions forward , une traduction backward , une revue de la version par un expert clinicien et des tests de comprehension. Resultats Independamment du questionnaire et des dimensions etudiees, les memes types de difficultes ont ete rencontres : (1) difficulte a traduire un terme par un equivalent direct. Par exemple, le mot things a ete traduit par something ; (2) une traduction litterale etait possible mais culturellement non pertinente. Par exemple, to walk several blocks a ete traduit par to walk more than one kilometer ; (3) les temps des verbes ont du etre reformules ou modifies. Par exemple, le present perfect n’existant pas en allemand, le present simple a ete utilise initialement. Finalement, l’imparfait a ete utilise en raison de l’utilisation d’adverbes tels que lately (recemment). Conclusion Une methode d’adaptation linguistique rigoureuse et reconnue assure une equivalence des concepts evalues dans les langues et permet l’utilisation dans des etudes internationales de questionnaires remplis par des patients fibromyalgiques.

Objectives To estimate the impact of drotrecogin alfa (DA) on intensive care workload in an observational study while illustrating the use of propensity score (PS) matching to control for recruitment bias. Methods PREMISS is a prospective, multicenter pre–post study. Its goal was to evaluate DA in the treatment of severe sepsis with multiple organ failure. Inclusions took place before (control patients) and after (DA-treated patients) the drug's market authorization. Workload was measured in euros using the French classification of medical procedures. It was compared between the groups via random effects gamma regression using two techniques: 1) regression adjusting for the patients' initial characteristics on the whole population; and 2) PS matching. A structural equation model was used to explore the pathways leading to a workload increase. Results Drotrecogin alfa is estimated to increase intensive care unit (ICU) workload by 20% ( P = 0.045) according to the multivariate model and 34% ( P = 0.002) according to the PS-matched one. In the structural equation model fitted, only DA's direct effect on the occurrence of bleeding events reaches significance ( P = 0.024). Conclusions We found a significant effect of DA on ICU workload with both standard methods of adjustment and PS matching. This effect appears to be mainly due to DA's effect on bleeding events. The analysis illustrated the usefulness of PS methods in the analysis of observational data, as it leads to conclusions similar to the traditional multivariate regression approaches while avoiding making too many adjustments, allowing focusing on the treatment effect.

e20548 Background: The irreversible ErbB family blocker afatinib and the reversible EGFR tyrosine kinase inhibitor gefitinib were compared in the multicenter, international, randomized, head-to-head phase 2b LUX-Lung 7 trial (LL7) for first-line treatment of advanced EGFR mutation-positive non-small-cell lung cancer (EGFRm+ NSCLC). We aimed to assess the cost and health outcomes of afatinib and gefitinib in this setting. Methods: A partitioned survival model was designed to assess the cost-effectiveness of afatinib versus geftinib in the French context for EGFRm+ NSCLC.Outcomes and safety are taken primarily from the head-to-head LL7 trial. Only direct medical costs were considered. Resources use and utilities were derived from the trial, expert panel and published literature. Incremental cost-effectiveness ratios (ICER) were calculated in the common EGFR population and also, in the sub-groups with EGFR Exon 19 deletion (del 19) and EGFR Exon 21 L858R (L858R) mutation over a 10 year-time horizon. Deterministic and probabilistic sensitivity analyses were performed. Results: For common EGFR+ NSCLC, the ICER of afatinib versus gefitinib was €45,211 per QALY (with a gain of 0.170 QALYs, and an incremental cost of €7,697). The ICERs for del 19 and L858R populations were €38,970 and €52,518 respectively. Afatinib had a probability of 100% to be cost-effective at a willingness-to-pay threshold of €70,000 for patients with common EGFR mutation, and also in the del 19 and L858R subgroups. Conclusions: Afatinib is a cost-effective treatment compared to geftinib in patients with EGFRm+ NSCLC with an ICER varying between €38,970/QALYs and €52,518/QALYs.

Background: Stroke is the second leading cause of death and a first leading cause of acquired disability in adults worldwide. This study aims to evaluate the current management and associated costs of acute ischemic stroke (AIS) for patients admitted in stroke units in France and over a one-year follow-up period as well as to assess the impact of improved thrombolytic management in terms of increasing the proportion of patients receiving thrombolysis and/or treated within 3 h from the onset of symptoms. Methods: A decision model was developed, which comprises two components: the first corresponding to the acute hospital management phase of patients with AIS up until hospital discharge, extracted from the national hospital discharge database (PMSI 2011), and the second corresponding to the post-acute (post-discharge) phase, based on national treatment guidelines and stroke experts' advice. Five post-acute clinical care pathways were defined. In-hospital mortality and mortality at 3 months post-discharge was taken into account into the model. Patient journeys and costs were determined for both phases. Improved thrombolytic management was modeled by increasing the proportion of patients receiving thrombolysis from the current estimated level of 16.7 to 25% as well as subsequently increasing the proportion of patients treated within 3 h of the onset of symptoms post-stroke from 50 to 100%. The impact on care pathways was derived from clinical data. Results: Among 202,078 hospitalizations for a stroke or a transient ischemic attack (TIA), 90,528 were for confirmed AIS, and 33% (29,999) of them managed within a stroke unit. After hospitalization, 60% of discharges were to home, 25% to rehabilitative care and then home, 2% to rehabilitative care and then a nursing home, 7% to long-term care, and 6% of stays ended with patient death. Of a total cost over 1 year of €610 million (mean cost per patient of €20,326), 70% concern the post-acute phase. By increasing the proportion of patients being thrombolyzed, costs are reduced primarily by a decrease in rehabilitative care, with savings per additional treated patient of €1,462. By adding improved timing, savings are more than doubled (€3,183 per additional treated patient). Conclusions: This study confirms that the burden of AIS in France is heavy. By improving thrombolytic management in stroke units, patient journeys through care pathways can be modified, with increased discharges home, a change in post-acute resource consumption and net savings.

Fibromyalgia is a chronic, non inflammatory, pain syndrome, characterized by widespread musculoskeletal pain and excessive fatigue. Although it is considered like being the second pathology met in ambulatory rheumatology practice, it is again, poorly understood by medical and scientific community.value the level of knowledge, attitudes and practices of Tunisian physicians, general practitioners concerning fibromyalgia.It was about a cross-sectional survey using questionnaire, achieved by a representative sample of Tunisian physicians, general practitioners. These last, answered a questionnaire previously used in France, in Portugal and in Russia, valuing their knowledge on fibromyalgia, their attitudes and practice in the face of patients suffering from fibromyalgia.Fibromyalgia was recognized as a clinical entity by 26.7% of our physicians. Each general practitioner followed an average of 5.7 patients. The diagnosis of fibromyalgia was made based on widespread pain that persists more than 3 months, sensitive to NSAIDs, for 48% of general practitioners, or on tenderness that occurs in precise, localized areas of the body (trigger points) according to 54% among them. Main symptoms of Fibromyalgia were known with various degrees: pains (87.9%), excessive fatigue (85.7%), muscular weakness (78%), sleeping troubles (65.9%) and psychological disturbance (68.1%). Knowledge of physicians about attitudes and practices in front of cases of fibromyalgia were limited.Knowledge of this disease is still mysterious and not well know. General practitioner are aware of fibromyalgia which not included in the program during that training in the medical school. In consequence, our institution, our medical schools in Tunisia should teach this disease to our student and during post graduate courses.

Fibromyalgia syndrome (FMS) is an under-diagnosed disorder of unknown aetiology, characterized by chronic widespread muscular pain, often accompanied by somatic and psychological symptoms. Several studies have described the impact of FMS on patients’ functionality, disability and quality of life. Other studies have reported on the burden to patients, healthcare payers and society. This review brings the existing evidence together and concludes that the patient burden of fibromyalgia is very high in comparison with many other conditions. The burden to healthcare payers and society is important as well, and can be mostly explained by factors not directly related to the treatment of FMS. Data suggest that the cost before diagnosis may even be higher than the cost after diagnosis. It is very likely that the combination of symptoms not only complicates the recognition and treatment of FMS, but also magnifies the burden of FMS. Despite the complex and controversial construct of this syndrome, the results in terms of patient, healthcare payer and societal burden are quite consistent.

Objectives:The aim of this study was to estimate the expected cost and clinical benefits associated with the use of drotrecogin alfa (activated) (Xigris; Eli Lilly and Company; Indianapolis, IN) in the French hospital setting.Methods:The recombinant human activated PROtein C Worldwide Evaluation in Severe Sepsis (PROWESS) study results (1,271 patients with multiple organ failure) were adjusted to 9,948 hospital stays from a database of Parisian area intensive-care units (ICUs)—the CubRea (Intensive Care Database User Group) database. The analysis features a decision tree with a probabilistic sensitivity analysis.Results:The cost per life year gained (LYG) of drotrecogin treatment for severe sepsis with multiple organ failure (European indication) was estimated to be $11,812. At the hospital level, the drug is expected to induce an additional cost of $7,545 per treated patient. The incremental cost-effectiveness ratio ranges from $7,873 per LYG for patients receiving three organ supports during ICU stay to $17,704 per LYG for patients receiving less than two organ supports.Conclusions:Drotrecogin alfa (activated) is cost-effective in the treatment of severe sepsis with multiple organ failure when added to best standard care. The cost-effectiveness of the drug increases with baseline disease severity, but it remains cost-effective for all patients when used in compliance with the European approved indication.

Background Recombinant human activated protein C (rhAPC) has been reported to be cost-effective in severely ill septic patients in studies using data from a pivotal randomized trial. We evaluated the cost-effectiveness of rhAPC in patients with severe sepsis and multiple organ failure in real-life intensive care practice. Methods We conducted a prospective observational study involving adult patients recruited before and after licensure of rhAPC in France. Inclusion criteria were applied according to the label approved in Europe. The expected recruitment bias was controlled by building a sample of patients matched for propensity score. Complete hospitalization costs were quantified using a regression equation involving intensive care units variables. rhAPC acquisition costs were added, assuming that all costs associated with rhAPC were already included in the equation. Cost comparisons were conducted using the nonparametric bootstrap method. Cost-effectiveness quadrants and acceptability curves were used to assess uncertainty of the cost-effectiveness ratio. Results In the initial cohort (n = 1096), post-license patients were younger, had less co-morbid conditions and had failure of more organs than did pre-license patients (for all: P < 0.0001). In the matched sample (n = 840) the mean age was 62.4 ± 14.9 years, Simplified Acute Physiology Score II was 56.7 ± 18.5, and the number of organ failures was 3.20 ± 0.83. When rhAPC was used, 28-day mortality tended to be reduced (34.1% post-license versus 37.4% pre-license, P = 0.34), bleeding events were more frequent (21.7% versus 13.6%, P = 0.002) and hospital costs were higher (€47,870 versus €36,717, P < 0.05). The incremental cost-effectiveness ratios gained were as follows: €20,278 per life-year gained and €33,797 per quality-adjusted life-year gained. There was a 74.5% probability that rhAPC would be cost-effective if there were willingness to pay €50,000 per life-year gained. The probability was 64.3% if there were willingness to pay €50,000 per quality-adjusted life-year gained. Conclusion This study, conducted in matched patient populations, demonstrated that in real-life clinical practice the probability that rhAPC will be cost-effective if one is willing to pay €50,000 per life-year gained is 74.5%.