Drug safety
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The use of data mining has increased among regulators and pharmaceutical companies. The incremental value of data mining as an adjunct to traditional pharmacovigilance methods has yet to be demonstrated. Specifically, the utility in identifying new safety signals and the resources required to do so have not been elucidated. ⋯ The number of DRPECs identified for each drug approximately correlated with the number of unique PTs in the database. Over one-half of DRPECs were either labelled as per the company's reference safety information (RSI) or were under review after identification by traditional pharmacovigilance activities, suggesting that for marketed products these methods do identify adverse events detected by traditional pharmacovigilance methods. Approximately three-quarters of the 861 DRPECs identified were closed without case review after triage. Of the approximately one-quarter of DRPECs that required formal case review, seven resulted in an addition to the RSI for the relevant products. While this pilot does not allow us to comment on the utility of routine data mining for all products, it is significant that several new safety concepts were identified through this prospective exercise.
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Drugs for rare diseases, so-called orphan drugs, are often intended for serious or chronically debilitating diseases. Safety information is more limited at the time of approval for orphan drugs as a result of various factors, such as the limited number of patients in clinical trials, quality of the clinical trials and special approval procedures. Several studies have been conducted on safety-related regulatory actions for drugs, but none of these have specifically focused on orphan drugs. ⋯ The probability of a first safety-related regulatory action for an orphan drug was slightly lower than that reported in the literature for biologicals in one study and new molecular entities in another study. However, detection of safety issues may be complicated by the limited experience with orphan drugs in practical use due to the low prevalences of the diseases they are used for. Doctors and pharmacists should therefore be vigilant with regard to the occurrence of a safety-related issue for orphan drugs.