The goal in this work package is to shift from focusing on mechanistic, disease-centered outcomes to patient-centered treatment models.
The WP emphasizes measurable goals like integrating patient-reported outcomes (PROs) and complex data (e.g., psychological and social factors) to enhance treatment personalization while also fostering critical reflection on the specific limitations and challenges of (re-)classifying medical conditions.
Current trials mainly focus on disease mechanisms and objective outcomes, but patient perceptions and expectations of treatments—especially biological interventions—are crucial for evaluating effectiveness. TRANSCEND aims to elevate the patient perspective, moving it from a secondary endpoint to a core focus, guiding future research and policy.
This part of TRANSCEND focuses on identifying patient-oriented outcomes, acknowledging the diversity in disease and symptom presentation. It involves collecting PROs and input from caregivers, especially in autism clinical trials. Associated partners (e.g., Orikami, Ink, LAVA and the German Federal Ministry of Health) play roles in helping us to revise and enrich existing outcome measurements.
The DCs will also develop qualitative evaluations of "disease perception" and "expectations" related to biological treatments and contribute to methodological and conceptual innovations regarding disease (re-)classification.
This work package includes work performed by doctoral candidates at KU Leuven under the primary supervision of Kaat Alaerts, at the University of Bielefeld with Lara Keuck, at the Bordeaux Montaigne with Steeves Demazeux, and at the UMC in Utrecht with Edwin van Dellen, with secondments and interactions throughout the network and associated partners.
The work package involves collaboration between neuroscientists and philosophers of medicine to promote critical discussion on experimental design and translation, as well as dissemination of insights from these practices to wider academic and non-academic audiences.