Patientenpartizipation in der pädiatrischen Versorgungsforschung am Universitätsklinikum Freiburg: von der Projektbeteiligung zum Patientenbeirat.

Participation of patients and relatives in research means that those affected are involved in the research process in a partnership role. Despite the growing importance of participatory approaches and the large number of available concepts, many researchers and patients are faced with the question of how participatory research can be realized and organized in concrete terms. Here we report on our experiences with two different forms of patient participation in research in the context of pediatric health care research at a university hospital: (1) In a project for the development and evaluation of a case management for patients with spinal muscular atrophy, patient representatives have an consultative role. (2) In the patient advisory board, which is to accompany the research activities of the research group at the site continuously and systematically, i.e. in all phases, the participation currently corresponds to a contributory role (involvement) which, in the future, could be moved onto the collaborative stage. In both forms of participation, the essential questions include the selection of the participating patients, the type and extent of participation, and the evaluation of the effect of participation on the research that is carried out. In our experience, both forms of participation add value to research from the perspective of all participants. At the same time, they bring different opportunities and challenges. While in project-based participation the sphere of influence is already delineated by researchers, the context of the patient advisory board provides more room and openness to develop, for example, a research agenda and thus identify new research topics. In our experience, however, sufficient resources (in terms of time and money) are required from all participants, as well as good, trusting cooperation with jointly developed processes to realize both forms of participation.

Understanding European patient expectations towards current therapeutic development in spinal muscular atrophy.

Following the 2017 approval of a first spinal muscular atrophy (SMA) treatment by the European Medicines Agency, SMA Europe launched a Europe-wide survey with the goal of understanding patients' treatment expectations, realities of daily living and access to clinical trials and therapy, and how this varied according to parameters such as age and disease severity. A response rate of 31% yielded 1474 completed surveys from 26 European countries. In line with findings from a 2015 SMA Europe-led survey, participants considered stabilization of their condition to be progress. Notably, responses indicated that the current classification of SMA at diagnosis by 'type' often does not reflect current mobility level. Large gaps in treatment access were identified that varied in particular between age and disease severity groups, yet there was high interest in clinical trial participation. In addition, alternative treatment options, including combination therapies, are now expectations. These perspectives should be central considerations through the research and development processes of new SMA therapies, through data generation and discussions on access to therapies. Results from this survey indicate that collaboration between stakeholders is essential to the foundation upon which innovative approaches for SMA treatments and access can be explored.