OBJECTIVES

When developing a health technology that requires clinical studies, developers institute working relations with clinical investigators. Patient representatives can also create advisory boards. This was of high utility in the 1990s, in particular for the development of products to treat HIV infection. Inspired by this model, the European Organisation for Rare Diseases (EURORDIS) proposes a new programme of CABs.

EURORDIS invites developers to sign a Charter for collaboration with patients in clinical research, and provides guidelines together with a mentoring and training programme for patient networks.

METHODS

CABs help set the agenda with the developer, work on topics as diverse as study design, feasibility, informed consent and site selection, QoL and PROMs, and organize the meetings. Discussions also cover compassionate use, pricing, relative efficacy, etc. Meetings last for 2 to 4 days during which sessions with different developers can take place, along with trainings. There are confidentiality arrangements, and minutes are taken. There are also regular between-meeting teleconferences for trainings as well as action plan updates, and many CABs have instituted focused working groups focusing on ie, access and psychological support.

RESULTS

As of 2019, 5 disease-specific CABs exist of approximately 12 members each and at least six others are in discussion.

We have started to work on the metrics of markers of success. Time will be needed to see concrete results and we are hopeful from the first surveys that this form of shared decision-making is the way forward from both a utility point of view as well as an ethical point of view.

CONCLUSIONS : This patient engagement programme, with collective thinking and exchange between patients, ensures high quality dialogue with developers, and can inform both HTA decision-making along and regulatory decision-making.