Multidisciplinary Approach Improves Spinraza Dosing Process in SMA Patients, US Study Shows

Adopting a multidisciplinary approach can optimize pretreatment evaluations and improve the dosing process for Spinraza (nusinersen) in people with spinal muscular atrophy (SMA), a U.S. study shows.

This approach, which also used computer simulations to optimize treatment schedules and reduce missed treatments, improved anesthetic methods and minimized complications associated with treatment administration.

The researchers pointed to insurance authorization as the main reason for substantial delays in treatment, calling for a clearer and faster authorization process.

The study, “A multidisciplinary approach to dosing nusinersen for spinal muscular atrophy,” was published in the journal Neurology Clinical Practice.

Biogen’s Spinraza, which is administered directly into the spinal canal, a process called intrathecal delivery, was the first therapy approved for children and adults with SMA types 1–3. Spinraza is given at days 1, 15, 29, and 59 in the first two months of treatment, followed by maintenance therapy every four months.

Due to the clinical features of SMA, Spinraza administration usually involves anesthesia and respiratory support. In patients with scoliosis — the most common symptom of SMA — additional medical procedures may be required to allow access to the spinal canal.

Spinraza’s intrathecal administration, its high cost of $125,000 per dose, and its scheduling logistics — the therapy requires extensive coordination among multiple services — challenge its implementation in health care centers.

“This complexity, in addition to the high cost of treatment, may lead to overburdening of an institution’s dosing resources, causing delays in treatment initiation and limiting patients’ access to therapy and may result in barriers to coverage,” the researchers said.

Despite Spinraza’s broad approval by the U.S. Food and Drug Administration (FDA), insurance authorization must be obtained prior to starting therapy. In addition, proof of the treatment’s response may be required by some insurers to allow continued coverage.

The importance of an experienced multidisciplinary team for the success of Spinraza programs was previously highlighted in an Italian study.

Now, clinicians in the U.S. are reporting the benefits of a multidisciplinary approach based on the first 62 SMA patients — 33 males and 29 females — treated with Spinraza at Children’s Hospital of Philadelphia from January 2017 to June 2018.

Most patients (94%) were 18 or younger at the time of treatment initiation. Among them, 25 (40%) were diagnosed as having SMA type 3, 21 (34%) as type 2, and 16 (26%) as type 1.

“A collaborative interdepartmental team composed of health care providers from neurology, orthopedics, pulmonary, palliative care, ethics, and teams familiar with triaging based on limited resources such as transplant and oncology, as well as patient advocacy and a parent of a child with SMA, devised a comprehensive stepwise protocol to maximize patient inclusion and safety,” the researchers said.

Before treatment, each patient was evaluated by a team that included a neurologist, a pulmonologist, an anesthesiologist, and a physical therapist. All were seen in a multidisciplinary Spinraza screening clinic specially created to expedite this process and determine patient-specific care during Spinraza administration. Patient visits were scheduled to occur every week for four months.

Computer simulations were used to “optimize treatment start dates, minimize delayed doses because of limited resources like weekend coverage and holidays, and keep patient volume manageable on any given day,” the researchers said.

While more than half of the patients (62%) were treated within two months from the date of their Spinraza screening visit, 25% had to wait more than three months to receive treatment. This lag was mostly due to delays in insurance approval, the investigators said.

Patients with SMA type 1 had the shortest waiting time — a median of 2.5 weeks — to insurance approval and subsequent treatment initiation. Those with type 2 had to wait the longest, a median of 12.5 weeks.

“Perhaps the greatest unforeseen challenge [in enrollment] … was the need for insurance prior authorization and lack of consensus for patient eligibility from insurance companies, despite FDA approval,” the researchers said. They noted that “the multiple insurances and varying ways of addressing the new medication staggered the patient enrollment more so than initial visits in screening clinic.”

The results showed that the multidisciplinary treatment approach helped prevent major dosing delays. Moreover, it was found to reduce the level of sedation used on following doses in nearly 40% of the 49 patients initially needing it. Recovery time also was reduced from four to two hours after the first dose, and from two hours to one in subsequent doses.

Both anesthesia and Spinraza were well-tolerated. Adverse events associated with treatment included pain at injection site (10 patients), headache (nine patients), and vomiting (two patients).

According to the researchers, these findings highlight how a comprehensive program using a multidisciplinary approach before, during, and after treatment “can optimize the screening process and dosing experience for [Spinraza] therapy and allocate resources both ethically and efficiently.”