The growth in expenditure on orphan medicinal products (OMP) across Europe has been identified as a concern. Estimates of future expenditure in Europe have suggested that OMPs could account for a significant proportion of total pharmaceutical expenditure in some countries, but few of these forecasts have been well validated. This analysis aims to establish a robust forecast of the future budget impact of OMPs on the healthcare systems in Sweden and France.
A dynamic forecasting model was created to estimate the budget impact of OMPs in Sweden and France between 2013 and 2020. The model used historical data on OMP designation and approval rates to predict the number of new OMPs coming to the market. Average OMP sales were estimated for each year post-launch by regression analysis of historical sales data. Total forecast sales were compared with expected sales of all pharmaceuticals in each country to quantify the relative budget impact.
The model predicts that by 2020, 152 OMPs will have marketing authorization in Europe. The base case OMP budget impacts are forecast to grow from 2.7% in Sweden and 3.2% in France of total drug expenditure in 2013 to 4.1% in Sweden and 4.9% in France by 2020. The principal driver of expenditure growth is the number of new OMPs obtaining OMP designation. This is tempered by the slowing success rate for new approvals and the loss of intellectual property protection on existing orphan medicines. Given the forward-looking nature of the analysis, uncertainty exists around model parameters and sensitivity analysis found peak year budget impact varying between 2% and 11%.
The budget impact of OMPs in Sweden and France is likely to remain sustainable over time and a relatively small proportion of total pharmaceutical expenditure. This forecast could be affected by changes in the success rate for OMP approvals, average cost of OMPs, and the type of companies developing OMPs.
A child with non-ulcerating cutaneous leishmaniasis after a 3-week visit to Southern France is reported. Diagnosis was histological. The possibility of the patient having had a visceral infection was taken into account when designing the final treatment.
This paper aims to explain the results of an observational population study that was carried out between 1991 and 1995 in six regions (departments) in France. The study was to assess the relationship between temperature and mortality in a few areas of France that offer widely varying climatic conditions and lifestyles, to determine their thermal optimum, defined as a 3 degrees C temperature band with the lowest mortality rate in each area, and then to compare the mortality rates from this baseline band with temperatures above and below the baseline. The study period was selected because it did not include extreme cold or hot events such as a heatwave. Data on daily deaths from each department were first used to examine the entire population and then to examine men, women, various age groups and various causes of death (respiratory disease, stroke, ischaemic heart disease, other disease of the circulatory system, and all other causes excluding violent deaths). Mean temperatures were provided by the National Weather Service. The results depicted an asymmetrical V- or U-shaped relationship between mortality and temperature, with a thermal optimum lower for the elderly, and generally lower for women than for men except in Paris. The relationship was also different depending on the cause of death. In all cases, more evidence was collected showing that cold weather was more deadly than hot weather, and it would now be interesting to enlarge the study to include years with cold spells and heatwaves. Furthermore, the results obtained could be of great use in estimating weather-related mortality as a consequence of future climate-change scenarios.