OP27 Engaging Patients: The EuroCAB Programme

Abstract

Introduction

When developing a health technology that requires clinical studies, developers institute working relations with clinical investigators. In certain diseases areas, patients' representatives create their own advisory boards, which proved their utility in the early 90s, in particular for the development of products to treat HIV infection. Inspired by this model, where patients with a same disease join and meet with relevant developers and discuss all aspects of the research, the European Organisation for Rare Diseases (EURORDIS) proposes a new programme of such Community Advisory Boards for Rare Diseases (CAB).

Methods

For this programme, EURORDIS invites developers to sign a Charter of principles when engaging with patients, and provides guidelines on CABs, together with a mentoring programme for patients’ networks that are less experienced with the development and the evaluation of health technologies. CABs are driven by patients who set their agenda, who sign a Memorandum of Understanding with each developer, and who organise the sessions. Sessions typically last for two to four days during which different meetings with different developers can take place, or trainings. All meetings can take place under confidentiality arrangements, and minutes are written to keep track and to follow-up with all points discussed. Participants and agendas are made public

Results

As of 2018, four CABs exist and operate (for tuberous sclerosis complex, for scleroderma, for cystic fibrosis, for Duchenne muscular dystrophy) and 18 others are in discussion with many due to start in 2019. Topics discussed cover the target population, the study feasibility, the endpoints including patient reported outcomes, the comparator choice and/or the acceptance of a placebo controlled trial, the quality of life, the practical aspects of the trials, and the identification of previously unknown or unmet patient needs/preferences. For products which are more advanced in their life-cycle, discussions can also cover compassionate use, pricing policy, relative efficacy etc.

Conclusions

This represents a well-structured programme for the engagement of patients, where collective thinking and exchange between different patients ensure high quality dialogue with developers and can inform HTA also.