Les médicaments orphelins : rétrospective et perspectives du marché européen
Orphan drugs : retrospective and prospective analysis of the european market
Résumé
The constantly evolving pharmaceutical industry is seeking growth levers. Orphan medicinal products, so-called orphan drugs, are designed to treat rare diseases, with a statistical prevalence of less than 1 in 2,000 people. The thousands of rare diseases that exist affect millions of Europeans. Often fatal, they represent a serious public health problem. European policy encourages manufacturers to develop orphan drugs within a harmonised framework but each member state retains control of prices and reimbursement levels, which can lead to disparities within the EU. In addition, European legislation grants orphan drug sponsors commercial exclusivity, something that is suspected of creating a monopolistic situation and excessive prices. Orphan drugs are becoming increasingly popular with big pharmaceutical companies, leading to a fast-growing turnover. This trend appears to simultaneously address a societal issue and manufacturers' quest for growth levers. However, the increase in health spending allocated to orphan drugs is prompting concern on the part of the authorities. The latest economic forecasts published in July 2012 are pessimistic. In the light of this economic context, what is the retrospective and prospective view of the orphan drugs market in Europe (history, regulations, epidemiology, economic model, monopoly, pricing, size / cover / limits of the market) ?