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Chapter 9294FUTURE PERSPECTIVESFrom clinic to trial to careTo enable evidence-based medicine for individuals with RGNDs, an efficient framework is necessary to get therapies and trial results to the affected individual. A clinical trial service can be valuable to catalyze interventional research in rare disorders (Figure 3). A very promising initiative is the Emma Center for Personalized Medicine (www.emmacenter.nl). This dedicated personalized medicine center unites expertise and efforts regarding translational research and care activities, aimed at providing accurate diagnoses, counseling, effective interventions, accessibility, personalized care and eventually prevention. Master protocols, referring to an overarching trial design developed to evaluate multiple hypotheses, should be developed for fast access to therapies and to reduce costs as much as possible. These include basket trials (i.e., a targeted therapy is evaluated for multiple diseases that share common molecular alterations or risk factors) and umbrella trials (i.e., multiple targeted therapies are evaluated for a single disease that is stratified into multiple subgroups based on different molecular or predictive risk factors).67 These may improve efficiency in drug development by diminishing redundancy in trial implementation, enhancing recruitment, sharing control groups, and using biomarkers that are relevant to the intervention’s mechanism of action across RGNDs.66 We should utilize templates for trial development and contracts rather than constantly reinventing the wheel, not only for research but also for integration in care.Furthermore, collaboration is key. There are many steps needed for treatment success which can only be achieved by uniting expertise. A trial service is well-positioned to collaborate with METCs, hospital pharmacists or pharmaceutical companies, health insurances and regulatory authorities for accessibility. But also, international collaboration is essential for research in rare disorders, because for some disorders there are only a few affected individuals per country. Such an initiative is the ‘International Collaborative Network (ICN) for N-of-1 Trials and Single-Case Designs’ (www.nof1sced.org) which is uniquely positioned to facilitate a range of activities to promote, support and advance the use of personalized clinical studies. In this way, personalized trials will become an integral part of clinical practice and health Annelieke Muller sHL.indd 294 14-11-2023 09:08