In the study of the therapeutic use of certain drugs, you might encounter the term “off-label”, but what does it really mean? The prescription for off-label medicines involves all therapeutic applications of a drug that differ from those for which it has been officially approved. Although this is sometimes a controversial practice, it is common in various fields, especially when drugs approved for a specific condition prove effective for other diseases. In any case, the prescription is the responsibility of a medical professional and follows precise laws.
What does “off-label” mean?
A drug is defined as “off-label” when it is prescribed for an indication, a patient population, a method, or a dosage different from that approved by regulatory authorities, such as the European Medicines Agency (EMA) or the U.S. Food and Drug Administration (FDA). This use may arise from clinical needs, emerging scientific evidence, or the lack of approved therapeutic alternatives.
What does the law say?
The regulations surrounding off-label drugs are complex and vary from country to country. At the European level, the distribution and production of medicines are governed by Directive 2001/83/EC of the European Parliament and Council. For a drug to be prescribed, it must have marketing authorization from the European Medicines Agency (EMA).
The 2001 European directive does not specify guidelines regarding off-label use but leaves the regulation of this practice to individual Member States. This provision is outlined in the Treaty on the Functioning of the European Union, which in Article 168(7) under the “public health” section states:
“Respect the responsibilities of the Member States for the definition of their health policy and for the organisation and delivery of health services and medical care”
The prescription of off-label drugs carries a series of responsibilities for the physician, who must carefully evaluate the available scientific material to assess any risks. Prescription occurs when the physician believes, based on documented data, that the patient cannot take other drugs already approved for that therapeutic indication. Furthermore, informed consent is always required: the physician must communicate to the patient why the medication is chosen, possible risks and adverse events, and the available data regarding the drug’s efficacy. Once the patient completes and signs a written consent form, the document serves as legal protection for both the physician and the patient.
Off-label drugs examples
The use of off-label drugs is widespread in several areas of medicine, such as oncology, rheumatology, neurology, psychiatry, and pediatrics. Some drugs have well-established and widespread off-label uses, including:
- Amantadine: an antidepressant used for treating headaches, anxiety, and insomnia. In recent years, its low-dose use has spread to treat certain forms of Herpes Zoster.
- Carbamazepine: generally used to treat epilepsy, it can be prescribed off-label for the treatment of neuralgia and bipolar disorder.
- Sertraline: initially developed for the treatment of depressive disorders and generalized anxiety, it can also be prescribed for post-traumatic stress disorder or, less commonly, premenstrual syndrome.
- Semaglutide: a drug used in the treatment of type 2 diabetes, which has recently gained popularity for obesity management, though this remains a controversial issue.
Flarer and the distribution of API
As a company involved in the research and distribution of Active Pharmaceutical Ingredients (APIs) for pharmaceutical, nutraceutical, and cosmetic purposes, we are aware of the precautions necessary to comply with the regulations in force in European and non-European states we work with. We provide support at every stage of API supply, including the management of documentation and requirements for the transportation and trade of the substances. We ensure the delivery of the highest-quality active ingredients on the market, supporting companies developing drugs intended for a wide range of applications, including off-label uses.
Our team of expert consultants is always available to assist our clients in selecting products and complying with regulations. Contact us for more information.