Off-label use

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 The combination had to be withdrawn from the market due to potentially fatal heart valve damage.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.