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General discussion2959research by sharing relevant knowledge, experience, expertise, resources, and data through the global partnership between researchers, healthcare providers, and affected individuals. Furthermore, projects such as Solve-RD, the International Rare Diseases Research Consortium (IRDiRC), European Reference Networks (ERNs), and the digital knowledgebases including Treatabolome and Treatable ID (www.treatable-id.org) are very promising in aiming to enable all individuals with a rare disorder to receive accurate diagnosis, care, and available therapy, and shorten the path to therapy by providing access to treatment information for specific disorders.68–70Clinician Experts outcomemeasuresProducer Laboratory Pharmacy Policy makersStatistician + methodologistPatient(organization) + familyClinical trial serviceOUTCOME MEASURESTRIALSResearcher Clinician AffectedindividualPOLICYFigure 3. Example of a clinical trial service for rare disorders and required collaborations. The future landscape of outcome measuresProcedures for measuring outcomes should strive for optimal patient relevance with minimally invasive methods. We should aim to engage the included patients themselves by using applicable methods, because they are the ones who truly experience their feelings with indications of discrepancies between self and proxy-raters. Simultaneously, caregivers, who have often already experienced much burden, might be slightly relieved. Furthermore, we should always question ourselves to what extent each outcome measure is relevant to the affected individual. Including personalized outcomes, such as identified by using PROMs and Goal Attainment Scaling, will contribute to the relevancy and will improve communication and shared decision-making. Annelieke Muller sHL.indd 295 14-11-2023 09:08