5. Off-label use across specific patient groups

Off-label use happens in all patient groups, but it is more common among certain patient populations. According to the ´Study on off-label use of medicinal products in the European Union´, such uses are particularly widespread in paediatrics—where children are often excluded from clinical trials—and in the area of rare diseases. Most studies reported in literature on off-label use in adults focus on specific therapeutic areas, such as oncology including haematology, psychiatry, or on rheumatology. The most frequently mentioned therapeutic area was oncology, including haematology. Pregnant women and the elderly are also groups of concern.

Rare diseases as a group are unique insofar as for many rare diseases there is no approved medicine, leaving off-label use as the only treatment. A 2012 survey in France among rare disease centres (92 out of 131 centres participated) identified 480 off-label practises corresponding to 82 rare diseases. In a survey, ‘EURORDIS Rare Diseases Europe’ estimated that 23% of the participating patients with rare diseases benefit from an off-label use product (120 out of 524 responses). In Hungary, 2% of the authorised off-label cases concern rare diseases.

The HMA states that particularly in paediatrics, the term “off-label use” mainly implies the absence of safety information, age-appropriate formulations, strengths and dosing recommendations for the physician and in consequence for the patient.

In the following two examples for off-label use in specific patient groups, paediatrics and oncology, are given.