The COSEB (Core Outcome Sets for Epidermolysis Bullosa) workshop was held as a hybrid meeting in Amsterdam, The Netherlands on December 15th, 2023, and organised by the COSEB Steering committee and the Dystrophic Epidermolysis Bullosa Research Associations (DEBRAs) of Austria, France, Ireland, Spain, and the United Kingdom. The goal of the COSEB initiative is to establish core outcome sets for the major EB types (EB simplex, junctional EB, and dystrophic EB) by identifying the most critical outcome domains (“what” to measure) and corresponding outcome measurement instruments (“how” to measure). Such core outcome sets should be used and reported consistently across clinical trials to facilitate accurate comparison and pooling of data to expedite and improve therapy development.
Funded by the European Joint Programme for Rare Diseases (EJP-RD), the workshop aimed to foster collaboration among key stakeholders in EB research to address challenges concerning outcome measurement in EB. Specifically, its objectives included: 1) to inform all stakeholders about the methodological steps of the COSEB process, 2) to connect working group members for planning activities for the upcoming years, and 3) to assess the feasibility of the COSEB protocol and appropriateness in light of available resources to meet the needs of the working groups. A total of fifty attendants from Europe, North and South America, Australia and Asia participated in the meeting, consisting of patient advocates and EB professionals including clinical experts, industry partners, regulatory representatives and methodologists.
The workshop commenced with a presentation of the COSEB protocol by Eva Korte and Martin Laimer, both members of the COSEB founding and steering committee. They outlined the first methodological steps of getting to a short-list of candidate outcome domains, as well as the organizational structure of the research teams, including the responsibilities of the steering committee, advisory panels and working groups. The workshop continued with explanations regarding the Delphi consensus procedure and granularity of outcomes by COS methodologists Sanna Prinsen and Jan Kottner, respectively. They emphasized the value of a consensus-based standardization of trial outcomes and underscored the importance of balancing the level of granularity and specificity when defining core outcome domains. Subsequently, Gaston Sendin, research manager at DEBRA Austria, addressed the priority of getting functional and clinically meaningful outcomes and trial endpoints. He pointed out the need for collaboration among research, industry, and organizations like DEBRA to secure funding for initiatives like this. The session continued with an introduction on the consensus method of COSEB and a section on the modality to engage patients in the initiative. The moderators of the session, Peter van den Akker and Sagair Hussain, summarized the first plenary session by highlighting the importance of valid and feasible core outcomes, accurate granularity, and broad stakeholder engagement with emphasis that active involvement from the entire EB community is critical to the success of COSEB.
Subsequently, in two separate break outs, the members of the dystrophic EB and EB simplex working groups were introduced and connected. Chairs and deputy chairs were elected and a first inventory of questions and requirements for the COSEB protocol was discussed. The final plenary Q&A session together with COS methodologists, Jan Kottner and Phyllis Spuls was intended to clarify any open COS related questions. In their closing remarks, the members of steering committee summarized once more the upcoming tasks and responsibilities of the working groups and advisory panels.
In conclusion, the COSEB workshop focused on the collaborative efforts to establish core outcome domain sets for EB trials, aiming to enhance patient care and accelerate clinical translation of therapeutic innovation for patients with EB worldwide.