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Chapter 9296Specifically, as there is an overgrowth of available outcome measurement instruments that are not always relevant to all individuals with RGNDs and ID, and development of disorder-specific outcome measures for thousands of disorders is not feasible and desirable, a core outcome set should be developed which can be extended with disorder-specific modules. Also, recent methodological innovations, such as item response theory (IRT), allow PROMs with good measurement properties to be applied across different health conditions, such as the Patient-Reported Outcomes Measurement Information System (PROMIS®).71–73 IRT can be used to create item banks, which are large sets of calibrated questions measuring a same construct. It enables efficient measurement through short forms or computerized adaptive testing (CAT) to minimize redundant items for specific affected individuals and increase relevancy and efficiency.74–77 These methods should be more frequently included in interventional research, and the use of ID user-friendly mobile apps in terms of experience sampling methods might be considered as well to track health and improve inclusivity and to know as good as possible the feelings and experiences of the concerned individual, to enable the best personalized care. Patient involvementMost importantly, it is vital that the trial and outcome measures capture as much as possible to real-life impact of a disease at an individual level, which is only feasible when the patient’s perspective is included. Therefore, affected individuals should be involved in the decision how to measure the effectiveness of an intervention, which is of pivotal importance for trial success. Trials are ultimately aimed at improving patients’ well-being. It is thus remarkable that patient involvement was mentioned in only 2% of the clinical trials included in the scoping review about outcome measures (see Chapter 5). Moreover, in a clinical trial service, different options for trial designs could be presented to affected individuals, such as a blinded or unblinded A-B design with or without placebo and the N-of-1 design with or without interim analyses (Figure 4). Based on the explained advantages and disadvantages, including the burden and level of evidence, affected individuals, parents and/or caregivers might express their preferences for a design. To foster recruitment and treatment adherence, patient involvement Annelieke Muller sHL.indd 296 14-11-2023 09:08