Protocol for the Development of a Core Outcome Set for Inherited Ichthyosis

Area of Science:

• Dermatology
• Genetics
• Clinical Trials

Background:

• Inherited ichthyosis are rare keratinization disorders impacting epidermal barrier function.
• Current treatments focus on symptom management, lacking standardized efficacy evaluation.
• Emerging therapies like gene therapy show promise but require consistent outcome reporting.

Purpose of the Study:

• To develop a Core Outcome Set (COS) for inherited ichthyosis research.
• To define a minimum set of outcomes and baseline characteristics for effectiveness studies.
• To enhance consistency and comparability in ichthyosis clinical trials.

Main Methods:

• Following Core Outcome Measures in Effectiveness Trials (COMET) guidelines.
• Conducting a scoping literature review and patient interviews to identify potential outcomes.
• Utilizing a three-round eDelphi survey with diverse stakeholder groups (patients, clinicians, researchers, industry, regulators).
• Finalizing the COS through a virtual consensus meeting.

Main Results:

• The study protocol outlines a systematic approach to COS development.
• Stakeholder engagement ensures comprehensive and relevant outcome selection.
• Ethical approval and registration with COMET ensure rigorous methodology.

Conclusions:

• This initiative will yield the first standardized Core Outcome Set for inherited ichthyosis.
• The COS will improve consistency in reporting and reduce heterogeneity in research outcomes.
• This will facilitate more robust and comparable efficacy studies for inherited ichthyosis treatments.