FAQtual Insights: Safe Approaches to Off Label Prescribing

Off-label prescribing is more common than uncommon in dermatology, with clinicians often encountering conditions for which standard therapies are limited or even nonexistent, new evidence supports alternative approaches, or approved treatments are unavailable for certain age groups or disease severities. While off-label use can expand treatment options, it requires careful evaluation, monitoring, documentation, and patient communication.

“Off label is so on brand in dermatology,” Adam Friedman, MD, professor and chair of dermatology at the George Washington University School of Medicine and Health Sciences in Washington, DC, told Dermatology Times. “It is a perfect representation of how we so effortlessly marry pathophysiology and pharmacology with ingenuity and creativity. Not that we really have a choice given the number of diseases for which there are no FDA approved therapies. Therefore we derms must heed the call of the off-label bandits!”

The following address key questions dermatologists and other clinicians commonly face when considering off-label therapeutics.

What does “off-label” use mean in dermatology?

Off-label use refers to prescribing a medication for an indication, patient population, dosage, or route that has not been specifically approved by regulatory agencies. This practice is legal and widely accepted in dermatology when supported by evidence or clinical rationale. While off-label use is not inherently unsafe, clinicians must ensure decisions are grounded in the best available data and are appropriate for the individual patient.

How do I decide whether an off-label therapy is appropriate?

Clinicians should base decisions on a combination of clinical evidence, guideline recommendations, and patient-specific factors. This includes reviewing peer-reviewed case series or, if available studies, expert consensus statements, and clinical experience. A thorough risk-benefit assessment is essential, considering potential efficacy, adverse effects, monitoring requirements, and alternative therapies. Individualized care, factoring in patient age, comorbidities, and prior treatment responses, is critical to safe and effective off-label use.

How should I discuss off-label use with patients?

Transparent communication is essential for informed decision-making. Clinicians should explain that the therapy is not formally approved for the patient’s specific condition but may be supported by clinical evidence. Discussion should include expected benefits, potential risks, monitoring requirements, and available alternatives. Obtaining documented informed consent is strongly recommended. Clear explanations help patients and caregivers make confident decisions and enhance adherence to treatment plans.

What legal and ethical considerations apply?

Off-label prescribing is legally permissible when it follows accepted standards of care. Clinicians should document the clinical rationale, informed consent, and follow-up plan in the medical record. Staying current with professional guidelines, institutional policies, and payer requirements is important, especially if reimbursement is involved. Ethical practice depends on transparency, evidence-based decision-making, and careful monitoring of outcomes.

How should patients be monitored while on off-label therapies?

Monitoring strategies should be guided by the known safety profile of the therapy, disease characteristics, and patient-specific risks. Baseline and follow-up labs, clinical assessments, and symptom tracking may be appropriate depending on the treatment. Clinicians should remain alert for adverse events and be prepared to adjust therapy if needed. Documenting follow-up plans and outcomes is a key component of safe and responsible off-label practice.

How do I stay current with evolving evidence on off-label treatments?

Evidence in dermatology, especially for off-label therapeutics, is constantly evolving, and staying current requires more than passively reading journals. Clinicians can benefit from curated, digestible sources of information. Subscribing to alerts from professional organizations such as the American Academy of Dermatology or specialty-specific email lists can deliver timely updates directly to your inbox. Setting up literature alerts through PubMed, Google Scholar, or other databases for key topics ensures you are notified when relevant studies are published. Participating in peer discussion forums, case-based webinars, and journal clubs can also help translate emerging evidence into clinical decision-making. Combining these approaches creates a manageable, real-world strategy to stay informed without being overwhelmed by the volume of new research.

When should I seek specialist input or refer a patient?

Referral or consultation is recommended when the off-label therapy carries significant risk, when the patient’s condition is complex, or when there is limited evidence supporting its use. Collaborating with subspecialists, pharmacists, or multidisciplinary teams can enhance safety, optimize outcomes, and provide additional expertise. Early consultation may prevent complications and support more informed decision-making.

Final Thoughts

Off-label therapeutic utilization is in the dermatologist’s DNA as they can provide valuable treatment options when there are technically none, but requires careful judgment, ongoing monitoring, and clear communication with patients. By following evidence-based principles, maintaining thorough documentation, and engaging patients in shared decision-making, clinicians can safely integrate off-label treatments into practice while upholding professional and ethical standards.