Advanced therapies (ATs), including gene and stem cell therapy, hold great potential for preventing and ameliorating many rare neurological disorders (RNDs) in children. These technologies are set to expand across modalities, potentially disrupting and augmenting conventional therapeutic pipelines, with the rapid pace of development highlighting data gaps and implementational challenges. We conducted a two-round modified Delphi study to co-develop a practice framework supporting the safe and effective application of advanced and/or experimental neurotherapeutics for children with rare neurological disorders within a public health ecosystem. The study generated 101 consensus recommendations encompassing criteria to 1) facilitate equitable and timely therapeutic access, 2) optimise transparent communication and shared decision making with families, 3) incorporate disease and patient level considerations for minimising risk and optimising safety within advanced therapeutic research, 4) strengthen resourcing of health systems to enable longitudinal evaluation of treatment effects and safety. Embedding this framework into practice will depend on enhancement of workforce training, establishment of digital infrastructure, fit-for-purpose clinical environments and education and engagement of patients, families and the broader community.
Lim et al. (Wed,) studied this question.