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General discussion2899the patient perspective, and have become important for shared-decision making and value-based healthcare.50 PROMs can be used for monitoring and informing care. It may improve communication between patient and clinician and about treatment outcomes, resulting in increased patient satisfaction with care. Noticeable, it is not feasible and desirable to develop and validate disorderspecific outcome measures for all thousands of rare disorders, as there is already an overgrowth of outcome measurement instruments, complicating generalizability. However, the tendency towards development of disorderspecific outcome measures and questionnaires designed ad hoc for a trial demonstrates the struggle researchers may experience: lack of an appropriate way to measure what is relevant to affected individuals.One way to measure what matters to affected individuals is the use of personalized outcome measures like the personal questionnaire and Goal Attainment Scaling (GAS),51 which is also used in the clinical trials explained in Chapter 3 and 4. These instruments are generally intended for standardized evaluation of an intervention’s effectiveness based on individualized goals. Goals could be different for each patient, but it has to do something with the construct that the treatment will target. In this way, outcomes can be measured that are specifically relevant to the affected individual, which eventually enhances treatment adherences as well.52 Treatment goals often differ which might unintentionally exclude affected individuals from clinical trials as frequently used outcome measures might not be relevant. This type of instrument is especially meaningful for heterogeneous and small populations, although it has not yet been validated for these populations.PROMs, GAS or experience sampling methods may thus be considered, enabling quantitative expression of meaningful subjective patient experiences while translating these into evidence. In care setting, each additional outcome measure could be considered a burden, whereas in research setting we should gain knowledge and explore, since there is a lot unknown yet . However, we should still pursue to minimize burden as much as possible. This could be discussed with patient (representatives) as well. The patient’s perspective should be included as much as possible by Annelieke Muller sHL.indd 289 14-11-2023 09:08