Increasing uptake through collaboration in the development of core outcome sets: Lessons learned at OMERACT 2023.

OBJECTIVE: This manuscript highlights the importance of enhancing the uptake of Core Outcome Sets (COS) by building partnerships with Collaborators and addressing their needs in COS development. METHODS AND SETTING: This session was structured as a simulation, resembling a format akin to a classic television game show. The moderator posed a series of questions to eight different Collaborator groups who briefly described the importance of COS within their areas of interest. Previous studies examining the uptake of individual core outcomes revealed disparities in uptake rates. The Identified barriers to the uptake of COS include the lack of recommendations for validated instruments for each domain, insufficient involvement of patients and key Collaborator groups in COS development, and a lack of awareness regarding the existence of COS. CONCLUSIONS: This analysis underscores the need for COS development approaches that prioritize the inclusion of patients and diverse Collaborator groups at every stage. While current studies on COS uptake are limited, future research should explore the broader implementation of COS across diverse disease categories and delve into the factors that hinder or facilitate their uptake such as, the importance of COS developers extending their work to recommending domains with well validated instruments. Embracing patient leadership and multifaceted engagement is essential for advancing the relevance and impact of COS in clinical research.

Extrapolation in the development of paediatric medicines: examples from approvals for biological treatments for paediatric chronic immune-mediated inflammatory diseases.

The European Union (EU) Paediatric Regulation requires that all new medicinal products applying for a marketing authorisation (MA) in the EU provide a paediatric investigation plan (PIP) covering a clinical and non-clinical trial programme relating to the use in the paediatric population, unless a waiver applies. Conducting trials in children is challenging on many levels, including ethical and practical issues, which may affect the availability of the clinical evidence. In scientifically justified cases, extrapolation of data from other populations can be an option to gather evidence supporting the benefit-risk assessment of the medicinal product for paediatric use. The European Medicines Agency (EMA) is working on providing a framework for extrapolation that is scientifically valid, reliable and adequate to support MA of medicines for children. It is expected that the extrapolation framework together with therapeutic area guidelines and individual case studies will support future PIPs. Extrapolation has already been employed in several paediatric development programmes including biological treatment for immune-mediated diseases. This article reviews extrapolation strategies from MA applications for products for the treatment of juvenile idiopathic arthritis, paediatric psoriasis and paediatric inflammatory bowel disease. It also provides a summary of extrapolation advice expressed in relevant EMA guidelines and initiatives supporting the use of alternative approaches in paediatric medicine development.