Dermatologists managing rare skin diseases without FDA-approved options must rely on rational, evidence-based off-label treatments, according to clinical presentations delivered on November 13 at the Society of Dermatology Physician Associates annual conference in Hollywood, Florida.
In dermatology, treating conditions without specific regulatory approval is a routine clinical necessity due to the thousands of identified skin diseases lacking dedicated therapies. Shannon C. Trotter of DOCS Dermatology Group explained that her daily practice relies heavily on utilizing existing drugs outside their labeled indications by targeting shared inflammatory pathways.
“In dermatology, we go off label. That is what we do,” Trotter said at the Society of Dermatology Physician Associates 24th Annual Fall Dermatology Conference.
One primary example involves spesolimab, an anti-interleukin-36 inhibitor approved for generalized pustular psoriasis. Clinicians are now turning to this biologic agent to treat pyoderma gangrenosum, a painful and deeply challenging ulcerating skin condition with known interleukin-36 upregulation. Because no FDA-approved therapies exist for pyoderma gangrenosum, case reports and mechanistic plausibility provide the foundational evidence required to justify off-label prescription and secure insurer coverage.
Establishing payer approval for unapproved indications often requires compiling published case studies and mechanistic data before submitting a claim. Trotter said that mounting clinical evidence creates a high probability of securing medication coverage even prior to formal multicenter trial conclusions. When initial denials occur, she advises contacting pharmaceutical liaisons directly to help patients obtain access.
A similar rational approach applies to Sweet syndrome, an acute febrile dermatosis also mediated by interleukin-36 pathways. By using AI-driven literature searches and studying disease pathogenesis, practitioners can identify existing targeted therapies that interrupt these specific inflammatory cascades.
Grover disease offers another clear instance where standard first-line topical steroids frequently fail to achieve adequate disease control. Medical attention has shifted toward the interleukin-4 inhibitor dupilumab, because interleukin-4 is implicated as a trigger in this disease, a condition with features overlapping those of atopic dermatitis.
“The evidence supporting dupilumab for this condition includes refractory cases,” Trotter said, citing a 2024 case study where a patient achieved stable remission lasting up to 11 months following a long list of treatment failures.
Janus kinase inhibitors also represent a versatile class of anti-inflammatory medications influencing multiple pathogenic pathways across a wide array of dermatologic diseases. Because 5000-patient trials are something "we do not see that very much in dermatology," clinicians must frequently rely on smaller case series and peer-reviewed case studies to guide safe therapeutic interventions.
Patients experiencing severe, treatment-refractory skin eruptions should consult a board-certified dermatologist to establish an evidence-based management plan. Patients should be informed of off-label prescribing and educated on the risk and benefits of off-label use.
“Patients should be informed of off-label prescribing and educated on the risk and benefits of off-label use,” Trotter said.
Andrew Baker, PA-C, MBA, the current president of the Society of Dermatology Physician Associates, emphasized that off-label use demands rigorous documentation within the medical record rather than relying on informal word-of-mouth solutions.
“In medicine, we need to support our decisions with evidence,” Baker told Medscape Medical News.
- Medscape Medical News: Rational and Systematic Use of Off-Label Therapies Critical to Managing Some Skin Diseases (November 13, 2026)
- Society of Dermatology Physician Associates (SDPA) Annual Fall Conference Proceedings
