In trials on drugs intended for chronic diseases, the number of patients studied is not always sufficient to correctly evaluate the long-term safety and efficacy of the products. There is therefore a need for new legislation, according to a group of European researchers writing in the journal 'Plos Medicine'. The current European guidelines provide that, in order to fully evaluate the safety of medicines under development for the chronic, therefore long-term treatment of non-fatal diseases, at least 1,000 patients must take the new drug, and respectively 300 and 100 patients must use it for 6 and 12 months, before approval by the European Medicines Agency. In an analysis conducted by Ruben Duijnhoven of Utrecht University, the authors used information from the European Commission on 200 medicines approved between 2000 and 2010, to see if the number of patients included complied with the International Conference on Harmonization E1 guidelines. It emerged that the average number of patients studied before approval is 1,708 for standard drugs and 438 for orphan drugs, used to treat rare diseases. On average, products for chronic use (e.g. asthma medications) were studied in more patients (2,338) than those for 'intermediate' use, such as anti-cancers (878), or short-term , such as antibiotics (1,315). The safety and efficacy of chronic disease products have been studied in fewer than 1,000 patients for at least 6 and 12 months in 46.4% and 58.3% of cases, respectively. But the authors also found that, among 84 drugs intended for chronic use, only 69 were studied in at least 300 patients for 6 months and 67 in at least 100 patients for 12 months.
March 21, 2013 – PharmaKronos
