The public health benefit of medicines: how it has been assessed in France? The principles and results of five years' experience.

Health technology assessment seeks to inform health policy- and decision-makers by promoting use of current best evidence and by addressing country specific factors, such as local context and values. In France, public health benefit (PHB) is one of the criteria used to inform decisions on the reimbursement of medicines. This article describes the methodological framework and the results after five years of assessment of PHB, by the French National Authority for Health. The semi-quantitative method used includes three dimensions that are: (1) the ability of a drug to improve the population's health status, (2) the drug's adequacy to cover public health needs, and; (3) the impact of the drug on the healthcare system. From 2005 to 2010, the PHB of 530 drugs was estimated, and 72% were assessed as having no PHB. The PHB was "low" for 88% of drugs expected to have a PHB, "medium" for 10%, and was considered to be "high" in only one case. The results of this experience show that it is feasible to assess the public health impact of drugs. But the high level of uncertainties at the time of a drug's first appraisal limits the assessment, which obviously has to be completed by reappraisal with post-marketing studies.

[French Health Authority (HAS) Guidance: the post-registration studies on health technologies (drugs, medical devices and instruments): principles and methods]. / Guide de la Haute autorité de santé (HAS) : les études post-inscription sur les technologies de santé (médicaments, dispositifs médicaux et actes) : principes et méthodes.

The assessment of a health technology is frequently accompanied by uncertainty about its impact, at short or long terms, on the health of the population. The Health Authorities may request additional « post-registration ¼ data that will allow a relevant reassessment of these technologies. The responsibility to collect this information lies with the industry and the HAS evaluates the methodology. This guideline provides practical benchmarks on methodological aspects of these studies. It describes the different types of studies to consider depending on the objectives, including the use of databases and cohorts and European studies. It emphasizes the importance of establishing a scientific committee, clearly defining the objectives of the study, justifying the methodological choices, documenting the representativeness or completeness of centers, investigators and patients, limiting the number of lost of follow-up patients and missing data, describing the statistical analysis methods, the bias and their possible impact on results. The publication of the results of these studies is strongly encouraged.

[Not Available]. / Guide de la Haute autorité de santé (HAS) : les études post-inscription sur les technologies de santé (médicaments, dispositifs médicaux et actes) : principes et méthodes.

The assessment of a health technology is frequently accompanied by uncertainty about its impact, at short or long terms, on the health of the population. The Health Authorities may request additional « post-registration ¼ data that will allow a relevant reassessment of these technologies. The responsibility to collect this information lies with the industry and the HAS evaluates the methodology. This guideline provides practical benchmarks on methodological aspects of these studies. It describes the different types of studies to consider depending on the objectives, including the use of databases and cohorts and European studies. It emphasizes the importance of establishing a scientific committee, clearly defining the objectives of the study, justifying the methodological choices, documenting the representativeness or completeness of centers, investigators and patients, limiting the number of lost of follow-up patients and missing data, describing the statistical analysis methods, the bias and their possible impact on results. The publication of the results of these studies is strongly encouraged.

The French Observational Cohort of Usual Smokers (FOCUS) cohort: French smokers perceptions and attitudes towards smoking cessation.

BACKGROUND: Despite increasing governmental anti-smoking measures, smoking prevalence remains at a high level in France. METHODS: The objectives of this panel study were (1) to estimate smoking prevalence in France, (2) to identify smokers' profiles according to their perceptions, attitudes and behaviour in relation to smoking cessation, (3) to determine predictive factors of quit attempts, and (4) to assess tobacco-related behaviours and their evolutions according to the changes in the smokers' environments. A representative sample of French population was defined using the quota method. The identified cohort of smokers was assessed, in terms of smoking behaviour, previous quit attempts, and intention to quit smoking. RESULTS: A response rate of 66% for the screening enabled to identify a representative sample of the French population (N = 3 889) comprising 809 current smokers (21%). A majority of current smokers (63%) had made an attempt to quit smoking. Main reasons for having made the last attempt were cost (44%), social pressure (39%), wish to improve physical fitness (36%), fear of a future smoking-related disease (24%), and weariness of smoking (21%). Few attempts (16%) were encouraged by a physician. In those who used some kind of support (38%), NRT was the mostly used. Relapse was triggered by craving (45%), anxiety/stress (34%), a significant life event (21), weight gain (18%), and irritability (16%). Depression was rarely quoted (5%). Forty percent of smokers declared they intended to quit smoking permanently. Main reasons were cost (65%), physical fitness improvement (53%), fear of a future smoking-related disease (43%), weariness of tobacco (34%), and social pressure (30%). Using a smoking cessation treatment was considered by 43% of smokers that intended to quit. Barriers to smoking cessation were mainly fear of increased stress (62%), irritability (51%), and anxiety (42%), enjoying smoking (41%), and weight concerns (33%). CONCLUSION: Smoking prevalence and smoking cessation attempts rate were lower in this survey than in previous reports. Cost and social pressure were the main reasons for quitting smoking, maybe an effect of dramatic tax increases and smoking ban.

Cost-effectiveness analysis of aldosterone blockade with eplerenone in patients with heart failure after acute myocardial infarction in the French context: the EPHESUS study.

BACKGROUND: The Eplerenone Post-Acute Myocardial Infarction Heart Failure Efficacy and Survival Study (EPHESUS) randomized clinical trial demonstrated the efficacy of eplerenone, a new aldosterone antagonist diuretic, with standard treatment versus standard treatment alone in the reduction of cardiovascular mortality and cardiovascular-related hospital readmissions for patients with heart failure after an acute myocardial infarction. AIM: We assessed the incremental cost per life-year saved of eplerenone in the French context versus standard treatment. METHODS: A within-trial study was designed. A piecewise regression model yielded death rates and survival gains adjusted for patients' characteristics, based on the extraction of comparable patients from the Saskatchewan Health database. Resource use was collected alongside the clinical trial data. Only direct medical costs were considered. All costs were in 2003 euros. Costs and outcomes were discounted at 5%. RESULTS: The overall mortality rate was 14.4% in the treatment group versus 16.7% in the placebo group (p=0.008). Combined cardiovascular deaths and hospitalization rates were 26.7% in the treatment group versus 30.3% in the placebo group (p=0.002). The discounted survival gain was 3.2 weeks. The incremental cost per life-year saved was euro15,382 (95% confidence interval 8274-42,723). Seventy-four per cent of the values of the incremental cost-effectiveness ratio fell under a euro15,000 per life-year saved threshold. CONCLUSION: The cost of eplerenone leads to an acceptable level of incremental cost per life-year saved when compared with existing treatments in the cardiovascular domain for the prevention of cardiovascular death and morbidity in patients with heart failure after an acute myocardial infarction.

Cost-effectiveness of atorvastatin in the prevention of cardiovascular events in diabetic patients: a French adaptation of CARDS.

INTRODUCTION: We estimated the cost-effectiveness of atorvastatin in the primary prevention of cardiovascular events in patients with type 2 diabetes using data from the Collaborative AtoRvastatin Diabetes Study (CARDS). METHODS: A total of 2838 patients aged 40-75 years with type 2 diabetes and no documented history of cardiovascular disease and without elevated low-density-lipoprotein cholesterol were recruited in the UK and in Ireland. Patients were randomly allocated to atorvastatin 10mg daily (n=1428) or placebo (n=1410) and were followed up for a median of 3.9 years. Direct treatment costs and effectiveness were analysed to provide estimates of cost per event avoided and cost per life-year gained over the trial period and over a patient's lifetime. RESULTS: The incremental cost-effectiveness ratio over the trial period was estimated to be Euro 3862 per clinical event avoided. Over the patient's lifetime, the incremental cost per life-year gained was Euro 2506 when considering cardiovascular deaths, and Euro 1418 per year when considering all-cause death. CONCLUSIONS: Primary prevention of cardiovascular disease with atorvastatin is cost-effective in patients with type 2 diabetes, with the incremental cost-effectiveness ratio for this intervention falling within the current acceptance threshold.