Clinical Trial Readiness for Spinal Muscular Atrophy: Experience of an International Educational-Training Initiative.

Several successful clinical trials have been conducted in spinal muscular atrophy (SMA) over recent years which have led to the approval of splicing modifiers and gene transfer therapies. With an increasing number of other agents progressing through pre-clinical and clinical development, increasing worldwide clinical trial readiness is becoming essential.SMA Europe initiated a clinical trial readiness project, which included the development of a pilot face-to-face educational-training initiative for clinical specialists and physiotherapists involved in SMA, with an emphasis on the patient perspective. Participants were selected through two surveys and, ahead of the meeting, a mock protocol with specific questions was provided. The initiative involved a series of presentations, role-play and interactive exercises. We describe here our experience and evaluation of this educational-training initiative, emphasising scientific aspects, psychosocial implications and level of satisfaction.From a participant, patient and industry perspective, such training was considered successful and met the objective, which was to improve clinical trial readiness in emerging sites. Resource planning, ethical considerations and communication with patients were identified as three important topics for future training. This initiative highlights the need to develop a training programme to achieve clinical trial readiness across Europe and showcases a collaborative effort with different stakeholders, clinicians, patient advocacy groups and sponsors to address an important issue.

Mitigating the effects of COVID-19 on the work of a children's clinical research facility.

The coronavirus disease 2019 (COVID-19) pandemic has affected almost every aspect of healthcare, including research. Many ongoing clinical trials were paused or adjusted to the new circumstances, the safety of all involved and patient outcomes being central concerns. Upcoming trials were put on hold and new trials were quickly designed and conducted to investigate COVID-19 treatments and vaccines. This article is a reflection on the effects of the pandemic on the work of a children's clinical research facility in the UK and how some of these effects were mitigated. It describes the adaptations made to the delivery of study interventions and to staff's ways of working, showing that some of the changes prompted by the pandemic had positive effects that will extend beyond. While this article relates to a single children's research facility, many of the lessons learned can be applied more widely in research and clinical care settings.