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Current legislation is insufficient to evaluate the long-term safety and efficacy of chronic drugs for patients before regulatory approval.

By Anonymous on Mar 20, 2013

Recently, European researchers led by Robert Duijnhoven from Utrecht University gathered information from the European Commission relating to 200 medicines that were approved between 2000 and 2010. The authors of this study, as published in PLOS Medicine, used this information to investigate whether the amount of patients included before approval met the International Conference on Harmonisation E1 guidelines.

These guidelines currently state that, a full evaluation of the safety of medicines created for long-term chronic treatment of non-threatening diseases, requires about 1000 patients to take the new drug. Out of this 1000, 300 patients must use the drug for 6 months and 100 patients must use it for 12 months, in that order, before it is approved by the European Medicines Agency.

This research was funded through a grant from the Medicines Evaluation Board (MEB) as a result of the Regulatory Science partnership between the MEB and Utrecht University.

As a result of their research, the authors discovered that the investigation into the safety and efficacy of chronic drug treatment was conducted with less than the required quota of patients for the 6 and 12 month timescale in 46.4% and 58.3% of new drugs, correspondingly. Among the 84 medicines for chronic use, only 69  were studied with 300 patients for 6 months, whilst 67 medicines were studied with the required 100 patients for 3 months.

Somewhat surprisingly, medicines for chronic use were studied in 2338 patients although drugs for intermediate use were only tested with 878 and short term use drugs were tested by 1315 individuals.

When commenting on these findings, the researchers said, “In light of new scientific and legislative tools to monitor benefits and risks in clinical use, discussion of the long-term exposure requirements for approval of medicines, particularly for medicines intended for chronic use, seems warranted. Such a discussion should involve healthcare providers, patients, and academia, as well as industry and regulators, and should include debate on the level of acceptable uncertainty, especially for adverse events and the long-term outcomes for chronic medication.”