Embracing innovation: A patient advocacy perspective on evolving trial designs

SMA Schweiz and SMA Europe, two patient advocacy organisations for the rare condition spinal muscular atrophy (SMA), work towards promoting the fastest possible access to safe and effective medicines for all individuals who can benefit from them. Their experience has shown that patient and public involvement at the design stage of clinical research directly impacts a product’s pathway to patients and leads to a better understanding of a product’s value later on. Furthermore, recent discussions about how complex clinical trial designs and decentralised clinical trials use innovative methods to conduct clinical research indicate their potential to make trials more flexible, personalised, and convenient for participants. In addition to helping shift current paradigms, innovative trial designs hold enormous potential to further advance medicines for the good of patient communities, especially in the area of rare diseases.