Patient advocates have been involved in DARE-NL from the early discussions and setup into the organization and implementation of plans. A patient advisory board (PAB) with representatives from four patient organisations and a patient relations liaison has been established to represent the patient interests and meets at least three times a year. The project leader will also involve the PAB on an ad hoc basis to obtain advise on specific technical issues as well as strategic questions that arise during the project.

The goals of the patient advocate are:

• Optimal patient care
• Timely access to new treatments
• Influence on clinical trials and guidelines for treatment
• Information on scientific research and involvement in discussions about future developments

Based on these goals, the PAB contributes to DARE-NL in several key areas:

• Registries for Hospital Exemption and ATMP trials

Currently, a substantial number of ATMPs without market authorization are clinically used through a hospital exemption (HE). An HE is only possible for ATMP treatments on a non-routine basis, for an individual patient, and when no centrally authorized treatment or clinical trial is available. However, it is currently unclear which, and where, ATMPs are used by means of HE. Therefore, setting up an HE registry with all ongoing HE, would be a highly effective step for bringing innovative ATMPs closer to patients. Similarly, a registry specifically for (academical or commercial) oncological ATMPs that are currently undergoing clinical evaluation is not yet available. Such Dutch ATMP-trial registry, which should include a non-technical description, would be beneficial for patients that would be looking to join a trial that is recruiting (facilitating recruitment) and/or interested in state-of-the-art developments in cancer research. Both registries would be very useful tools for authorities and other stakeholders (such as KWF) as well, to evaluate the status of ATMP development in the Netherlands. The PAB will actively contribute to setting up these registries and they will design a dissemination strategy for informing their members. Whenever possible these registries will align with or use already existing registries or initiatives.

• Facilitate discussions with regulatory authorities

The PAB will be actively involved in the discussions with regulatory authorities with regard to market access (WP6.1), representing the interest of cancer patients and aiming to make promising ATMP drug candidates available for patients as soon as possible.

• Setup of HTA and clinical studies

While the setup of clinical studies is not part of the DARE-NL infrastructure, it is important to start with the design of such studies in an early stage, and involve all stakeholders as early as possible. The PAB will help ATMP researchers with the design of clinical studies, such as proper patient selection, in-and exclusion criteria, endpoints, and management of side effects. In parallel, the PAB will support with assessment and design of appropriate patient reported outcomes (PROs) for HTAs of ATMPs under development.

• Patient perspective on Dutch ATMP R&D strategy.

Finally, patient representatives will provide their perspective on the role of ATMPs in future anticancer therapy, outlining their vision on the research and development priorities for ATMPs for the coming 5 years. The goal of this vision is to ensure the generation of a range of new advanced therapeutics that are developed towards the needs of all cancer patients as much as possible.