Off-label use — prescribing a medication for an indication, patient population, dose, or route not specifically approved by regulatory agencies — is a legal and widely accepted practice in dermatology when supported by evidence or clinical rationale. Clinicians often encounter conditions for which standard therapies are limited or even nonexistent, where emerging evidence supports alternative approaches, or where approved treatments are unavailable for certain age groups or disease severities. In these settings, off-label use can meaningfully expand treatment options.
However, it also requires careful evaluation, monitoring, documentation, and patient communication. Clinical decisions must remain grounded in the best available data.
Cross Pollination
For clinicians who treat pediatric patients, off-label prescribing is the norm rather than the exception. Peter A. Lio, MD addressed the subject at the 2026 Masters of Pediatric Dermatology meeting in a pragmatic and thought-provoking session, noting that “we, as dermatologists, and particularly those who see kids, are experts in off-label therapy.”
For decades, many inflammatory and autoimmune skin diseases had no FDA-approved options at all, Dr. Lio added, using lichen planus as an example: “How many FDA-approved treatments are there for lichen planus?” he asked. “There are exactly zero.” Therefore, if clinicians were to limit themselves strictly to labeled indications, many patients would simply go untreated.
According to Dr. Lio, the physician’s role is to thoughtfully explore alternatives grounded in pathophysiology and clinical experience, a process he described as “cross-pollinating” ideas across diseases that share immune pathways or mechanistic overlap. This kind of translational thinking has historically driven innovation in dermatology.
One recent example involves dupilumab, initially approved for atopic dermatitis but used off-label in bullous pemphigoid after clinicians recognized its mechanistic relevance. As clinical experience accumulated, dupilumab ultimately became the first and only approved treatment for bullous pemphigoid, illustrating how exploratory use can inform formal regulatory approval.
Transparency and Trust
For Dr. Lio, the concept of off-label care includes over-the-counter regimens. He notes that while these recommendations are rarely label-specific, they can meaningfully influence outcomes. He also emphasizes that off-label prescribing is not improvisation, but rather a deliberate, evidence-informed process that must be accompanied by candid discussion with patients.
Because transparent communication is essential for informed decision-making, clinicians should explain when a 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. Documented informed consent is strongly recommended. Clear explanations help patients and caregivers make confident decisions and enhance adherence to treatment plans.
Key Takeaways
- Off-label prescribing in dermatology is common, driven by limited standard therapies and emerging evidence, requiring careful evaluation and patient communication.
- Off-label use involves prescribing for unapproved indications, necessitating decisions based on clinical evidence, guidelines, and patient-specific factors.
- Off-label therapy, when grounded in sound science and shared decision-making, is not a departure from good medicine — it is often its engine.
Safe and Effective
When considering off-label treatments, clinicians should evaluate clinical evidence, guideline recommendations, and patient-specific factors. Supporting data may include peer-reviewed case series, clinical studies when available, expert consensus statements, and clinical experience. A thorough risk-benefit assessment, considering potential efficacy, adverse effects, monitoring requirements, and alternative therapies, is essential. Furthermore, individualized care, factoring in patient age, comorbidities, and prior treatment responses, remains critical to safe and effective off-label use.
“Off-label is so on-brand in dermatology,” observed Adam Friedman, MD, professor and chair of dermatology at the George Washington University School of Medicine and Health Sciences in Washington, DC. “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.”
Off-label prescribing is legally permissible when it follows accepted standards of care. Clinicians should document the clinical rationale, informed consent discussion, and follow-up plan in the medical record. Ethical practice depends on transparency, evidence-based decision-making, and careful monitoring of outcomes.
Final Thoughts
In the constantly evolving landscape of dermatology practice, off-label therapeutic use can provide valuable treatment options when approved therapies are limited or unavailable. Its use 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.
