Off-label drugs in otolaryngological practice against the background of legal conditions of Polish legislation

Introduction: ‘Off-label drug use’ refers to the administration of drugs for unapproved indications or age groups, a different dosage or other form of administration. Considering the legal issues, there clearly exists a need to implement rules that would regulate the use of pharmaceuticals outside the scope of a marketing authorisation. The brevity and diversity of Polish laws in the field of health care leads to many interpretative doubts associated with particular legal acts. Aim: We aimed to present clinical examples from everyday practice of off-label drug use from the medical and legal perspectives, and to support it with relevant legal acts. Material and method: Off-label drug use in various otolaryngology subspecialties – otology (mesna), laryngology (bevacizumab, cidofovir and botulinum toxin) and head and neck surgery (botulinum toxin) – are presented and discussed in detail. Results: Fourteen Polish legal acts regarding off-label drug use and 4 from EU legislation are commented on. The algorithm of cascade of decision-making processes in off-label drug use is shown. Conclusions: Off-label use of medicinal products is not prohibited in Poland or the EU; nevertheless, it is undeniable that the unclear legal situation regarding the use of medicinal products for nonregistered indications creates difficulties. To minimise a doctor’s liability risk, obtaining the informed consent from the patient for such treatment is advisable.