Patient and Stakeholder Engagement in the National Strategy for Gene- and Cell-Based Therapies (PSE@GCT)

Patient and Stakeholder Engagement (PSE) refers to the active involvement of patients and other relevant stakeholders from the very beginning and throughout the entire process of the National Strategy for Gene- and Cell-Based Therapies (National Strategy GCT). This includes integrating their perspectives and needs as individuals with lived experience. This approach ensures that projects and measures are designed in a patient-centered way.

The German Federal Ministry of Research, Technology and Space (BMFTR) has commissioned the Berlin Institute of Health at Charité (BIH) to bring relevant stakeholders together to develop and implement a National Strategy GCT for Germany. A particular focus is on integrating the patients’ perspective. The PSE@GCT project is a joint initiative of the National Network Office GCT Germany and the PSE team at the BIH QUEST Center.

In the current implementation phase of the National Strategy GCT, patient representatives are involved in multiple action areas, including “Networking and Support for Stakeholders”, “Training and development of skills”, “Research and Development”, “Marketing Authorization and transition to patient care”, and “Interaction with the Society”. An example is the collaboration in a subgroup of the action field “Interaction with the Society”. Within this subgroup, curated information on GCT is being developed to provide patients, family members, and patient and self-help organizations with reliable and accessible information. This process is carried out in a participatory manner with patient representatives, researchers, and other stakeholders.

Another key component is the display of patient and self-help organizations in the GCT-Atlas. As a central information platform for GCT, the Atlas presents various actors of the GCT ecosystem on interactive maps. It facilitates orientation and supports networking among patients, researchers, clinics, and other partners.

In the field of GCT research, measures have been also developed to raise awareness of Patient & Stakeholder Engagement (PSE) among researchers and other stakeholders and to support the planning and implementation of PSE in research projects. At the same time, the exchange between the research community and the patient community will be strengthened.

Furthermore, measures are being developed to institutionalize PSE at the BIH and to strengthen collaboration and exchange with existing PSE networks and working groups, thereby advancing and implementing patient engagement at the national level.