From the Research
The correct answer is A. TRUE. The use of a lawfully marketed drug for an unapproved use (off-label use) in the course of medical practice is not considered a clinical investigation and does not require an Investigational New Drug (IND) application when the primary intent is to treat an individual patient. This practice falls under the physician's discretion to provide appropriate treatment based on their medical judgment. The FDA recognizes that off-label prescribing is a common and often necessary part of medical practice, particularly in areas like oncology, pediatrics, and rare diseases where approved options may be limited. The key distinction is that the physician is using the drug for treatment purposes rather than conducting research to investigate the drug's safety or efficacy. However, it's essential to note that if the off-label use becomes part of a systematic investigation designed to develop information about the drug's safety or efficacy, then it would be considered clinical research and might require an IND.
The provided evidence does not directly address the question of off-label drug use in medical practice. However, the principle of using the best available evidence to guide medical decisions is crucial. For instance, a study like the one on nivolumab plus ipilimumab for unresectable hepatocellular carcinoma 1 demonstrates how clinical research informs treatment options, but it does not pertain to the regulatory aspects of off-label use. Similarly, other studies such as those on gepotidacin for uncomplicated urogenital gonorrhoea 2, aspirin, steroidal and non-steroidal anti-inflammatory drugs for Alzheimer's disease 3, and mepolizumab to prevent exacerbations of COPD with an eosinophilic phenotype 4 contribute to the broader understanding of drug efficacy and safety but do not directly address the question at hand.
In real-life clinical practice, the decision to use a drug off-label is based on the physician's professional judgment, considering the patient's specific needs and the available scientific evidence, even if that evidence is not directly related to the off-label use in question. The FDA's stance on off-label use supports this practice, acknowledging its necessity in certain clinical scenarios. Therefore, the use of a lawfully marketed drug for an unapproved use, when intended to treat an individual patient, does not constitute a clinical investigation requiring an IND application.