Number of patients studied before EU drug approval “insufficient”

The number of patients studied before regulatory drug approval in the European Union (EU) is insufficient, according to research published in PLoS.

Before any new drug can receive marketing approval for use in humans, it must undergo extensive laboratory and clinical testing to determine its safety and efficacy. The researchers detailed that, to fully evaluate safety and efficacy of medicines intended for long-term treatment of non-life-threatening diseases, too few patients were required to be included in studies.

Current guidelines dictate that, before a drug can be approved by the European Medicines Agency (EMA), at least 1,000 – 1,500 patients in total should be exposed to the new drug, with at least 300 patients using the drug for 6 months and 100 for 12 months.

In the study, researchers identified 200 unique new drugs, including 39 orphan drugs, that were approved in the EU between 2000 and 2012. Of the 84 medicines intended for chronic use, they noted that the median total number of patients studied before approval was 2,338. However, not all studies met the requirements for 6 and 12 months exposure – 82% of chronic use drugs were tested in the required number of patients for 6 months, and 80% for 12 months. Fewer than 1,000 patients were exposed to the drug for 6 months and 12 months in 46% and 58% of new medicines, respectively.

The researchers wrote that, for chronic medications, the clinical safety guidelines required too few patients to be studied long-term. The figures of 300 and 100 for 6-mo and 12-mo respectively were deemed too low to have a reasonable likelihood of detecting long-term adverse events. The researchers suggested that a long-term study size of at least 1,000 would be more appropriate, and called for a re-evaluation of the requirements.

Do you think a re-evaluation of long-term study size requirements for chronic medications is needed? Do you agree that too few patients are required to be tested for long-term use?

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