Session (CTI)

Purpose: In Europe, Regulatory and HTA guidelines are misaligned regarding requirements of Patient-Reported Outcomes and Health-Related Quality of Life (HRQoL) data. This has led to uncertainty for Sponsors in determining what data is best submitted. The purpose of this workshop is to review potential approaches for alignment and submission. Description: EMA CHMP Guideline (2005) uses ‘optional’ terminology in the advice regarding HRQoL data and requires the majority of domains to be significantly improved and adjusted multiplicity to grant an HRQoL label. On the contrary, Theidel and Van Schulenburg (2016) described that trials including HRQoL instruments had percentage-wise higher chances of receiving added benefit ratings in Germany during HTA review, even if based on 1-2 symptoms or symptom impacts, e.g. in oncology. Other national EU agencies may also be receptive towards other PRO instruments to obtain a favourable approval and preferential reimbursement. Most recently, new Joint Clinical Assessment webinars from May 2025 were missing some standard evidence guardrails, suggesting that anything Patient-Centred may go — creating opportunities as well as confusion for PRO experts. Dr Katja Rudell will introduce relevant guidelines and outline potential risks of growing patient centred evidence in the EU space (10 minutes) - and conduct a pulse check which guidelines are favoured by the ISPOR audience members (5 minutes), Dr Bryan Bennett will provide an industry perspective on the myriad of guidelines on product PRO/COA strategy in his discussions (10 minutes). Prof. Olivier Chassany will close the workshop presentation with an elaborated checklist approach that aims to reduce confusion on simplified processes of presenting patient-centred evidence for HTA from his experience as a reviewer of such data (15 minutes). Audience participation for discussions during each presentation and Q&A session at the end (15 minutes) will foster engaging talks on the problems and solutions of European submissions to elevate the importance and value of PRO, and HRQL data.