3. Off-label use

4. Off-label use - challenges

Off-label use of a medicine poses a range of quite different challenges:

  • ethical and legal issues for healthcare professionals (HCPs). Their choice to prescribe and dispense the medicine off-label should be based solely on therapeutic considerations in the best interest of the patient and ideally supported by evidence-based guidelines.
  • it carries an increased risk for patients. While off-label prescribing may be necessary and justified for medical reasons, an adequate level of evidence in terms of efficacy and safety is required.
  • patient information and consent are especially important. This aims to ensure that the patient is aware of the benefits and risks of off-label use and that both good and bad outcomes are duly reported. Whenever possible, patients should be initially treated following the provisions of the MA of an authorised medicine, and only change to an off-label prescription if the initial treatment shows no success or presents intolerable undesired effects.
  • medical practice not being subject to the regulation of medicines in the EU, an extensive off-label use could undercut the public expectation that there has been a proper evaluation of a product’s safety and efficacy.[1]
  • the lack of scientific knowledge associated with off-label use and the consequent likelihood that a noteworthy share of the population could receive treatments for which there are no data regarding safety and efficacy, putting their health and lives at risk.
  • off-label practices could pose a threat to public health in general because extensive uncontrolled use conflicts with any centralised approval system based on the conditioning of the marketing of a product to scientifically valid and statistically significant evidence indicating that its potential benefits are likely to outweigh its potential risks.[2]
  • economic implications need to be considered. Off-label use might arguably be associated with an increase in healthcare costs.[3] This might arise either when a more expensive off-label medicine is employed and reimbursed in favour over a cheaper on-label product or when an insurer refuses to cover the off-label treatment costs, and as a result, the out-of-pocket expenses of patients are enhanced.[4]

[1] Off-Label Use of Drugs and Medical Devices: A Review of Policy Implications, RS Stafford 2012, 91 Clinical Pharmacology and Therapeutics 920.

[2] Supra note 16

[3] RA Tabora, “Litigation Aspects of Off-Label Use: Quality of Care, Generally Accepted Medical Practices & Emergence of Cost Containment” (2009) 19(3) Albany Law Journal of Science & Technology 619; D Kwok, “Controlling Excessive Off-Label Medicare Drug Costs through the False Claims Act” (2017) 27 Health Matrix 185.

[4] D Holley, “Balancing on the Edge: The Implications and Acceptability of Off-Label Drug Use” (2009) 19 Albany Law Journal of Science & Technology 633; T Lemmens and S Gibson, “Decreasing the Data Deficit: Improving Post- Market Surveillance in Pharmaceutical Regulation” (2014) 59 McGill Law Journal 943.