Off-label drugs are drugs that are used, in clinical practice, for the treatment of diseases and disorders not foreseen in the summary of product characteristics (document authorized by the Ministry of Health that provides information for health professionals on how to use a medicine in safe and effective way).
Off-label drugs, therefore, are already registered and approved, but for different therapeutic indications than those for which they are instead prescribed.
Off Label Medicine
Off-label drugs can be used on both adult and pediatric patients and used in different branches of medicine. However, sectors such as oncology, psychiatry, neurology, hematology, transplantology and rheumatology are the areas in which they are used most often.
The AIFA (Italian Medicines Agency) draws up and constantly updates a series of lists containing all those drugs for which an off-label use is also envisaged. These lists can be consulted directly on the AIFA website and below
Off-label use of drugs can only be carried out in certain cases and only following very specific regulations.
The laws enacted so far on the subject and currently in force allow doctors to prescribe certain drugs for a use other than that for which the medicine was approved, but only on the basis of documented scientific evidence and only in the event that therapeutic alternatives are not available best.
In truth, there is no real law that clearly and completely regulates the prescription and use of off-label drugs. However, there are some laws and some decrees that provide guidance on this.
As mentioned, the law allows the doctor to prescribe the administration of off-label drugs under his direct responsibility.
However, since the use of drugs for clinical conditions not officially approved, the doctor has the duty to inform the patient (from whom, by law, he must then obtain consent), providing information on the reasons that lead him to use off-the-shelf drugs. label and the potential risks associated with them.
Even if supported by scientific evidence, the use of off-label drugs is connected to potential risks for the patient who takes them. In fact, the efficacy and safety of use of these active ingredients have been studied and tested in patient populations in different conditions than those for which the off-label drug is instead prescribed.
Therefore, patients may respond to treatment with off-label drugs unexpectedly and new undocumented side effects may also arise.
Unfortunately, however, in some cases the doctor cannot behave differently and the use of off-label drugs is the only therapeutic strategy available.