Page 295 - Demo

General discussion2939addition, the administrative work related to initiation of a trial may all lead to a long delay before including the first patient. How should we finance that?The trial design together with smartly chosen outcome measures might help. Despite requiring fewer participants, financial expenses are likely to remain quite similar in practice, owing to mandatory procedures for clinical trials involving medicinal products. The platform ‘Medicijn voor de Maatschappij’ (Medicine for Society) is a great initiative for sustainable and affordable availability of medicines for rare diseases. They are a knowledge hub and carry out projects to ensure availability of specific medicines. They assist in exploring the possibilities of conducting clinical trials with medicinal products (e.g., by pharmacy compounding or magistral preparation, or collaborations with pharmaceutical companies). Collaborating with pharmaceutical companies could accelerate accessibility and maintain affordability for academic research through the provision of the investigational drug. However, agreements should be carefully established in conjunction with legal research support. To our experience, arranging contracts might be a time-consuming process. Enabling ‘care for rare’ is a matter of prioritization. Opting for large trials for common diseases is an important but straightforward approach, but it may leave several patients with rare complex disorders untreated. Concurrently, the core values of an academic medical center include taking responsibility to care for all individuals. For rare complex disorders, it requires innovation and necessitates financial investment. If the academic hospitals place significance on ‘care for rare disorders’ – and I believe they should – the hospitals should further facilitate and support this kind of research, to ensure a reasonable timeframe for delivering therapy to those in need.Annelieke Muller sHL.indd 293 14-11-2023 09:08