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New consortium forms to address the issue of harmonization of outcome measurements in EB research

Harmonization of outcome measurements in EB research

Advancements in understanding epidermolysis bullosa (EB) have led to the development of new therapies and use of repurposed drugs, which are currently being tested in clinical trials with a diverse range of outcome domains and measurement instruments. Heterogeneity in outcome measurement could hinder comparability, data consolidation, and cross-study interpretability.

A core outcome set (COS) is a methodology to work towards harmonization in measuring the crucial outcomes of a certain disease that should be at least measured in every intervention study in that disease. The COS methodology is solid and will give direction to using uniform definitions and, eventually instrument choice and optimization. This will be done in a global consensus project by hearing and appreciating the voices of all stakeholder groups involved in EB research and care, including patients, their representatives, clinicians, researchers, industry partners and regulators.

The development of a COS for EB will be done in four steps: setting a clear scope, defining core outcome domains, recommending outcome measurement instruments for these core outcome domains, and disseminating the findings by consensus papers. Throughout the course of the initiative, patient-centricity, feasibility, and clinical meaningfulness of outcome measurement are of the greatest importance.

The kick-off meeting

The virtual kick-off meeting of the initiative on 12 April 2023 went tremendous, with over 100 people taking part from all over the world. EB experts Dimitra Kiritsi and Martin Laimer presented their perceived challenges in outcome measurement in EB research. They highlighted aspects including the rarity and heterogeneity of the disease, lack of repository of validated outcomes and outcome measurement instruments, inconsistent outcome definitions, trial design complexities, and trial burden for patients. Regulators Thorsten Olski and Caroline Auriche (EMA) outlined that this heterogeneity of the disease and therapeutic approaches affects specific trial outcomes and needs to be considered in the development plan for a therapy and choice of primary endpoint. In the presentation of scoping review authors Eva Korte and Tobias Welponer (EB House Austria), the heterogenous reporting of outcomes in the research landscape of EB was shown. Rainer Riedl (DEBRA Austria) and Elizabeth Vroom (Duchenne World Organization) stressed the importance of patient centricity, feasibility and clinical meaningfulness in outcome measurement. In addition, many other studies, meetings and conversations with different stakeholder groups in the field of EB point towards the need for more harmonization of outcome measurement in EB.

Meeting synopsis are available as download below.


What is a core outcome set (COS)?

The suggested methodology to use in this harmonization process is that of a core outcome set (COS). This COS development is not something that needs to be invented for EB, but it has been done for multiple other diseases, ranging from very rare to very frequent and for different settings, like clinical follow-up and trials.

A core outcome set is ‘a (multi-stakeholder) consensus-based agreed minimum set of outcome domains and measurement instruments that should be measured and reported in all clinical trials of a specific disease or trial population’. In other words: what are the crucial outcomes of a certain disease that should be at least measured in every intervention study in that disease. Important to note: the core outcomes are the minimum, if it is necessary for the quality of the study that more outcomes are measured, this is of course always possible.

Thus, in aiming at defining a COS for EB, agreements will be reached using a voting system, which will be done online with an eDelphi procedure program (Delphi plain language summary for COMET website.pdf ( Once the list for voting has been prepared, all stakeholders involved in EB research and care are invited to vote.


If you want to be involved, or would like to receive additional information, please contact project coordinators Marieke Bolling and Eva Korte.


Relevant links related to this initiative

Links to COS group page

C3: Epidermolysis Bullosa (

COMET: COMET Initiative | Core Outcome Set for Epidermolysis Bullosa (COSEB) (


Publications related to the initiative

Heterogeneity of Reported Outcomes in Epidermolysis Bullosa Clinical Research: A Scoping Review as a First Step Towards Outcome Harmonisation | British Journal of Dermatology | Oxford Academic (

Epidermolysis bullosa: time to come together for better outcomes | British Journal of Dermatology | Oxford Academic (

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