Off-Label Medicines


Off-label drugs are drugs that are used, in clinical practice, for the treatment of pathologies 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 safely and effectively).

Off-label drugs, therefore, are already registered and approved, but for different therapeutic indications than those for which they are instead prescribed.

The drugs that can be used off-label are active ingredients that have been widely used for some time, for which scientific evidence suggests their rational use even in clinical situations not expressly and officially approved.
Off-label drugs can be used on both adult and pediatric patients and are used in various branches of medicine. However, sectors such as oncology, psychiatry, neurology, hematology, transplantation and rheumatology are the areas in which they are most used.
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 at the following link :


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 matter 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 decrees that provide guidance on this. These laws and decrees are :

  • Law 648/1996;
  • Law 94/1998 on the "special use of drugs (also known as" Di Bella Law ");
  • Ministerial Decree of 18 May 2001;
  • Ministerial Decree of 8 May 2003.

Responsibility of the Doctor

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 populations of patients in conditions other than those for which the off-label drug is 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.

Improper use

As we have seen, for some drugs the so-called off-label use can be carried out, but only by following the regulations in force and only if their prescription and administration take place under the direct control of the doctor.
However, it may happen that the off-label use of drugs is carried out in an improper way (sometimes on the initiative of the patients themselves), even when the conditions for it do not exist.
This unauthorized, unregulated and unapproved use of off-label drugs is often and willingly done to save on the cost of the drugs themselves.
To clarify this concept, we can give a simple example: some patients to save on the cost of Propecia® (a drug containing the active ingredient finasteride at a concentration of 1mg and used in the treatment of androgenetic alopecia), instead of buying this medicinal product, buy finasteride tablets of 5 mg (whose indications concern the treatment of benign prostatic hypertrophy and NOT the treatment of androgenetic alopecia) and then divide them into 4 or 5 parts, taking one a day.
This incorrect procedure is done substantially to save money, both because finasteride 5 mg is now also available in the form of a generic drug - consequently it costs less in proportion to Propecia® - and because it can be prescribed by your doctor at the expense of the National Health System ( although this should not be done, since the patient in question is suffering from androgenetic alopecia and not from benign prostatic hypertrophy, a disease for which, however, the medicine can be dispensed at the expense of the NHS).
In my personal and professional opinion, this habit is strongly discouraged and should be avoided. In fact, by dividing a tablet into five parts it is not possible to know the exact amount of active ingredient that you are taking and you can thus risk, both of taking too low a dose of the drug (with the possibility of a therapeutic failure), or taking excessive dosages of the drug that can cause an increase in side effects, the consequences of which can be unpredictable and even very serious.
In fact, despite the possible savings, it is always advisable not to divide the tablets in any type of therapy, except in the case in which it is not the doctor himself to recommend it due to the lack on the market of pharmaceutical formulations with the appropriate dosage, a phenomenon that in any case nowadays it is quite rare.

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