Case Study — Pediatric Spinal Muscular Atrophy — Neurology & Rare Disease

Key Takeaways

This study evaluated an investigational therapy for spinal muscular atrophy (SMA). To be eligible, participants had to be genetically diagnosed with SMA, presymptomatic and ≤ 6 weeks of age.

As time was of the essence, the sponsor wanted to implement a comprehensive travel and reimbursement program that would eliminate the logistical and financial concerns that could prevent a family from participating in the study.

Challenges

Randomization needed to occur within six weeks of birth and before the newborn became symptomatic; therefore, educating the physician community about the study was critical.

Logistical and financial barriers had to be eliminated in order to ensure all potentially eligible patients could be considered for participation and, if enrolled, could participate for the duration of the study.

Complex travel support had to be managed (e.g., obtaining travel visas, facilitating cross-border travel and accelerated booking) during an emotionally difficult time for parents.

Solution and Impact

The study leveraged BBK Travel^TM as an adaptive solution that could fit the needs of all travelers.

Over the course of the study, there were several instances where the program evolved to meet the needs of this unique pediatric population. Some instances called for a patient ambassador to assist with translation, medical care and transportation. Other instances called for special overnight accommodations outside of a traditional hotel stay.

In reducing the time it might take sites to do this coordination, site staff were freed up to focus on patient care.

19 ENROLLED PATIENTS
22 MONTHS OF SUPPORT
17 SITES
11 COUNTRIES

772 Reimbursed Visits Over the course of the study, the site staff reimbursed families for 772 visits — including initial screening visits — contributing to overall study satisfaction. All families supported with a TCN Card payment and reimbursement program remained in the study for the duration.
418 Travel Itineraries BBK’s Concierge specialists removed major barriers to participation by arranging full-service transportation (e.g., air, ground), hotel accommodations, visa assistance and translation.