Unlicensed and off-label drug use: issues and recommendations.

Most adult patients would expect to receive only medicines which have been shown to be well tolerated, effective, and of a high quality. The licensing process to which most drugs are subjected gives the best assurance available that these criteria are met. However, every day, children are required to take medicines which are either not licensed, or are being used outside the terms of their license. Consequently, we cannot be sure that they meet the high standards we require for adult patients. This situation exists wherever children are treated across the world, and when they are in hospital or treated at home. The drugs involved are not obscure; they are routinely used drugs, and many are the mainstay of pediatric therapeutics. The terms 'unlicensed' and 'off-label' should not be taken to imply disapproval, nor incorrect or improper use of drugs, as such prescribing is a vital part of everyday pediatric drug therapy. It is essential because the gold-standard randomized clinical trials supporting adult medicine are often unavailable for children's treatments. Adequate information is not available to ensure that children have timely access to well tolerated and effective medicines with accurate, scientifically justified prescribing information. Many problems arise from this situation, including the lack of availability of appropriate pediatric drug formulations, poor prescribing information, and increased risk of medication errors and unanticipated adverse drug reactions. This situation is unacceptable. The US has taken the lead to change this situation by providing legislation and incentives to the pharmaceutical industry to perform pediatric clinical trials. Funding has been provided to set up research facilities where trials can be conducted by experienced pediatric investigators using imaginative, innovative, and noninvasive testing methods. The rest of the world should learn from these experiences and follow this lead to ensure that children have the same rights as adults to well tolerated and effective medicines.