Controversies in off‐label prescriptions in dermatology: the perspective of the patient, the physician, and the pharmaceutical companies

Abstract

The term "off-label drug use" involves the prescription of medications for unapproved indications or in unapproved dosage, dosage form, or route of administration. Off-label medications are common in the management of dermatologic diseases. In the recent years, new indications for botulinum toxin, biologics, spironolactone, topical calcineurin, and topical vitamin D analogues emerged, and these drugs are being used off-label to treat a variety of conditions. The high cost and long time required for the approval discourage pharmaceutical companies from developing studies and pursuing the approval by regulatory agencies. Patients and physicians may have a different perspective regarding the off-label use of medications. The ethical discussion is based on the different opinions concerning the need for informed consent by the patient and how this could be applicable in the clinical practice.

Current regulatory approaches for accessing potential COVID-19 therapies

“…Moreover, the dosing regimen in the protocols even for off-label use is not the same between countries, not to mention other programs. Under ideal conditions, the off-label program would constitute in the creation of a target patient population, informed consent and track and follow-up reports [8,9]. Still, prescribing an already approved medicine either for an indication, a dose or a way that is not approved for COVID-19 seems to be very challenging for clinicians.…”

“…Still, prescribing an already approved medicine either for an indication, a dose or a way that is not approved for COVID-19 seems to be very challenging for clinicians. Therefore, under the COVID-19 emergency conditions, it is hard to believe that the off-label use would result to be the best approach for accessing potential medicines, considering the ongoing regulatory debates and the difficulties in assessing riskbenefit for each patient due to the pressurized and stressful situation [8,9].…”

Role of botulinum neurotoxin–A in cerebral revascularization graft vasospasm prevention: current state of knowledge

“…3 Similar to numerous currently practiced unlabeled BTX-A and other off-label medication uses, 12,13 BTX-A use in graft vasospasm is unlabeled and will likely remain so given the costly and time-consuming nature of obtaining FDA approval for new indications of currently approved drugs. 21,73 Unlabeled drug use, defined as prescribing currently available medications for an indication that does not have FDA approval (including unapproved patient populations, doses, or administration forms), 73 is nonetheless at the treating physician's discretion for indications that they believe are in the patient's best interests based on credible evidence. 21,73…”

“…21,73 Unlabeled drug use, defined as prescribing currently available medications for an indication that does not have FDA approval (including unapproved patient populations, doses, or administration forms), 73 is nonetheless at the treating physician's discretion for indications that they believe are in the patient's best interests based on credible evidence. 21,73…”

Reply to “Innovation and off‐label use, the French case and more” by Braillon and Lexchin

“…5 Second, major barriers to research into new indications of drugs include very high costs of clinical trials and time required to conduct them rather than off-label use. 6 Third, in some specialties, especially in paediatrics, off-label prescribing is particularly prevalent due to a lack of on-label information about use of many drugs in children, which in turn is a consequence of inadequate number of clinical trials performed on children. 7 Thus, prevalent off-label use in paediatrics is not a cause but rather a result of a lack of approved treatments meeting the contemporary standards of evidence-based medicine.…”