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- Presentation
Questions about Pemphigus and the FDA
Description
The talk, presented by Vicki Worth, addresses the complexities surrounding pemphigus treatment and the relationship with the FDA. It highlights ongoing challenges in defining primary outcomes for clinical trials, emphasizing the need for clear measures of disease activity and patient quality of life. The discussion points to the importance of biomarkers, trial designs, steroid tapering, and the pharmacokinetics and pharmacodynamics of treatments like rituximab. Worth discusses past studies demonstrating the effectiveness of rituximab, calling for standardized outcome measures to better compare different trials and treatments. She stresses the necessity for consensus among dermatologists, patients, and the FDA regarding treatment benchmarks, particularly regarding what constitutes 'almost clear' skin. Additionally, she proposes the idea of public-private partnerships to enhance dialogue with the FDA on patient-focused drug development. Overall, the talk underscores that while progress has been made in measuring outcomes in pemphigus, significant work remains to align definitions, treatment protocols, and trial designs effectively.
View moreConclusions
- The primary outcome for pemphigus clinical trials needs to focus on meaningful disease activity measures, particularly the assessment of complete remission.
- Quality of life and biomarker evaluations are essential secondary outcomes to consider in trials.
- Current outcomes and measurement definitions for pemphigus are not fully agreed upon, necessitating further collaboration and standardization among stakeholders.
- The Pemphigus Disease Area Index (PDAI) has shown to be an effective and validated measure of disease activity and severity.
- Rituximab has proven to be superior in achieving sustained complete remission when compared to other treatments like MMF and steroid-only regimens.
- There is a need for clearer definitions of 'almost clear' in treatment assessments to facilitate approvals of new, safer medications.
- The dialogue and collaboration between the dermatology community, the FDA, and patients must continue to advance outcomes and trial designs for pemphigus treatments.
- Public-private partnerships may help streamline processes in clinical research and approval pathways, benefiting both patients and drug developers.
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- Murrell DF et al, submitted for publication
- Yale M et al, J Invest Dermatol 143:1868-1871, 2023