Case Study - Amyotrophic Lateral Sclerosis

Key Takeaways

This six-month phase 3 study was seeking to enroll 108 patients with amyotrophic lateral sclerosis (ALS) who had a mutation in the superoxide dismutase type 1 (SOD1) gene. BBK had successfully supported phase 1 enrollment.

Since patients may not have known whether they had the mutation, genetic testing was included as part of the initial screening.

Because of the limited patient population, efforts were needed to ensure that all study participants remained engaged with services that facilitated their participation.

The investigational drug QALSODY (tofersen) has since been approved by the FDA – its approval has been considered a major scientific advancement as the first treatment to target a genetic cause of ALS.

Solutions

BBK developed materials to keep the study top of mind at study sites and to support patient identification and consent.

Patient and referring-physician materials were educational and approachable. They helped staff to quickly identify potentially eligible patients while also providing patients and caregivers with the information necessary to facilitate consent.

Patient engagement services included reimbursement and in-country / cross-border travel for patients and their caregivers.

BBK created a unique brand that could resonate across multiple studies within the sponsor’s ALS pipeline.

1,700 Referrals generated by BBK.
77% Percentage of sites that adopted BBK’s TCN™ Card reimbursement program.
68% Percentage of sites that adopted BBK’s TCN™ Arrive travel program.

Concierge Services

Ambulance Transports

Site: Île-de-France, France
A patient in France required an ambulance to transport them to and from study visits as their condition progressed.

BBK’s concierge specialists sourced a local ambulance.
BBK coordinated payments directly to the local vendor as the site was unable to afford the expense.

Wheelchair Accessibility

Site: Quebec, Canada
A patient in Quebec required added support as their mobility declined.

BBK’s concierge specialists identified a partner in Quebec who could provide a wheelchair-accessible vehicle.
BBK coordinated accessible hotel rooms for the patient.

Hospital Bed Rental

Site: Florida, United States
Staff at a site in Florida requested assistance from BBK’s concierge specialists to help source a hospital bed that could be rented for one of their patients.