Risdiplam reduced health care resource utilization (HCRU) and disease-related complications in a cohort of patients with spinal muscular atrophy (SMA), including those who were previously treated with nusinersen. Researchers presented their findings at AMCP 2023.

“Prior claims analyses found substantial HCRU and disease-related complications for patients with SMA prior to approval of disease-modifying therapies (DMTs); however, limited data exists for patients treated with DMTs,” explained researchers. 

The retrospective analysis was conducted among 63 patients treated with risdiplam to reveal patient characteristics, HCRU, and trends in SMA-related complications.
The patients were identified by data from the PharMetrics Plus database and were included in the study if they: 

• had 1 or more inpatient or 2 or more outpatient diagnosis codes for SMA;
• had 1 or more claims for risdiplam between August 1, 2020, and December 31, 2021; and
• had continuous enrollment for at least 1 year prior to and at least 30 days after the index date, defined as the date of the first risdiplam claim. 

Patients initiated risdiplam at a mean age of 20.0 (SD 14.2) years, and 70% of the participants previously received treatment with nusinersen.

Mean follow-up was 9.9 (SD 4.4) months. Overall, risdiplam was associated with decreases in disease-related complications, including acute respiratory failure (-8%), chronic respiratory failure (-3%), pneumonia/dyspnea (-19%), orthopedic complications (-11%), and voice and speech impediment/disorders (-11%). Similar reductions were seen in patients previously taking nusinersen, at rates of -5%, -11%, -25%, -11%, and -14%, respectively. 

Researchers also found 86% of patients had no hospitalizations pre- or post-index. 

Among those hospitalized, the number of hospitalizations increased from 1.44 (SD 0.73) pre-index to 1.99 (SD 0.91) post-index; however, the mean length of stay decreased from 16.2 (SD 14) days to 7.8 (SD 13.3) days, and the number of inpatient services utilized decreased from 9.3 (SD 6.5) to 6.3 (SD 6.9). 

Additionally, the mean number of outpatient visits decreased from 84.1 (SD 88.9) pre-index to 76.8 (SD 88) post-index.

“These findings suggest that risdiplam may be effective in reducing HCRU and disease-related complications among patients with SMA,” said researchers. “Future analyses are needed to assess risdiplam’s impact on health care costs and other SMA-relevant outcomes, as well as a comparative analysis among patients on other SMA treatments and those who are untreated.” 

Reference:
Pineda E, Boudreau D, Liu Y, Dickendesher T, Shapouri S, Shieh P. A retrospective analysis of healthcare resource utilization and disease-related complications among risdiplam-treated patients with spinal muscular atrophy. J Manag Care Spec Pharm. 2023;29(10-a suppl):S1-S137. https://www.jmcp.org/pb-assets/Poster%20Abstract%20Supplements/AMCP2023_PosterAbstractSupplement_0317-1679318682267.pdf