Table 2 Recommended actions to enhance site readiness for SMA clinical trials

As part of the readiness program, Cure SMA developed a list of recommended steps sites can take to support readiness for SMA clinical trials. These were based on benchmarking with other organizations, the 2017 survey of experienced research sites, and input from experienced trial practitioners. They were shared with program participants during site interviews and are also reflected in program toolkits.

Recommendations for Clinical Trial Sites

Optimize site infrastructure and logistics to accommodate patient needs

 ▪ Collocate assessments, procedures, and dosing to reduce the stress of research visits for patients and families and streamline the flow of visits for research teams.

 ▪ Offer resources to support families that travel together – such as play rooms for siblings – to reduce stress for parents.

Familiarize staff who support clinical trials with SMA and how it can affect patients and families

 ▪ Provide opportunities for team members to learn about SMA. This can promote patient-centered trial management and help them anticipate and prepare to address challenges that may arise during trials.

 ▪ Allow newer team members to shadow experienced colleagues to learn about patients and protocols.

Take proactive steps to promote strong team coordination

 ▪ Streamline operations by clearly delineating responsibilities, communicating frequently, and using checklists and scheduling tools.

 ▪ Use weekly clinic and research meetings to keep teams on the same page about matters pertinent to trial conduct.

Use checklists, templates, and SOPs to aid trial management

 ▪ Create and implement checklists, templates, and standard operating procedures (SOPs) to promote prompt and thorough completion of requisite activities.

 ▪ Use checklists and templates for important communications, to make sure patients and team members receive the information that they need when they need it.

Share information in a patient-centric way and be open to learning from patients and families

 ▪ Be attentive to how information is shared with patients and families. Communicate clearly and concisely, being mindful about word choice and understanding.

 ▪ Provide opportunities for questions and clarification of information, and be willing to listen to and learn from patients and families.