Off-label use of drugs means the use of drugs for indications, dose, dosage forms, route, and patient population for which approval from the regulatory bodies like US Food and Drug Administration (US FDA) and Drugs Controller General of India (DCGI) has not been obtained. It does not mean that it is illegal or contraindicated to use the drug as off-label. The purpose of off-label prescribing is to provide the maximum benefit to the patients, especially the vulnerable population like pediatric, psychiatric, pregnant, and cancer patients where there is not sufficient data available for the drug use or in diseases where no treatment is yet available. Hence, valid scientific data and an expert opinion can be relied upon for off-label use. There still remains concerns for safety when prescribing for off-label purpose due to the lack of adequate safety and efficacy studies, and thus a physician must assess the risk/benefit ratio before its use. The regulatory bodies like US FDA has formulated certain guidelines for off-label use while the DCGI still does not support its use. Hence, there is no uniformity in the rules and regulations about off-label prescribing around the world, which needs to be streamlined.
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