Off-label use is defined as the use of a pharmaceutical drug in doses, forms, patient populations, routes of administration or for indications that are not included in approved product labelling.
Off-label prescribing has great potential benefits in certain situations. It allows drugs to be used in domains of medicine in which the patient population is less likely to be included in clinical trials, such as paediatric or pregnant patients. Also, off-label prescribing is crucial in therapy of terminal and life-threatening conditions (e.g. cancer, AIDS), rare diseases and in psychiatry. By documenting off-label use of drugs, it is discovered that some medications have health benefits that go beyond what they were initially prescribed for. Off-label use of a certain drug can become a standard treatment for a given clinical condition. For example, the use of β-blockers for heart failure was considered an off-label use for many years before being formally approved.
On the other hand, absence of regulatory approval usually means lack of data regarding drug safety. If a drug is not harmful for a particular clinical situation, evidence of its safety and efficacy may not apply to others. There are multiple studies comparing drug safety of approved and unapproved uses which have concluded that ADRs occur at a higher rate among those drugs prescribed for off-label use. One of the most notable cases of an off-label use risks is the Fen‑Phen, an anti-obesity unauthorised treatment that utilised two anorectics (fenfluramine and phentermine). The combination had to be withdrawn from the market due to potentially fatal heart valve damage.
In conclusion, off-label use is a growing and necessary aspect of modern medicine. Every regulatory unapproved prescribing must be backed up by medical judgement and strong scientific evidence. Collecting data on (un)successful off-label use of drugs, as well as ADRs occurring during the process, is crucial for ensuring patients’ safety and providing maximum therapeutic effect.
Ena Bulović, Mpharm / Senior Pharmacovigilance Associate
Lašćinska cesta 40
Planinska ulica 13/2
Office: +385 1 5588 297
Pharmacovigilance: +385 1 5588 297
Clinical trials: +385 1 5614 330
Registration: +385 1 242 0873
Marketing: +385 1 2420 890
Fax: +385 1 2420 860
Tel: +385 1 5588 297
Full company name
Short company name
Marti Farm Ltd. Trade and Services
Marti Farm Ltd.
Lašćinska cesta 40, HR-10000 Zagreb
Planinska ulica 13/2, HR-10000 Zagreb
a limited liability company
Commercial Court of Zagreb
HRK 20,000.00 (paid in its entirety)
Martina Diminić Smetiško, director of the
company (Representing the company
individually and independently, Responsible
person for data protection)
HR3623600001102197724 (Zagrebačka banka)
HR4324020061100628669 (Erste banka)
Full company name: Marti Farm Ltd. Trade and Services
Short company name: Marti Farm Ltd.
Headquarters: Lašćinska cesta 40, HR-10000 Zagreb
Office: Planinska ulica 13/2, HR-10000 Zagreb
Legal form: a limited liability company
Court register: Commercial Court of Zagreb
Registration number: 080751121
Share capital: HRK 20,000.00 (paid in its entirety)
Authorized representative: Martina Diminić Smetiško, director of the company (Representing the company individually and independently, Responsible person for data protection)
Bank account: HR3623600001102197724 (Zagrebačka banka), HR4324020061100628669 (Erste banka)