P.062 Burden of illness of spinal muscular atrophy (SMA): an update

Abstract

Background: In this retrospective claims analysis, real-world healthcare resource use (HRU) and costs among SMA type 1 (SMA1) patients were assessed. Methods: SMA1 patients were identified from Symphony Health’s Integrated Dataverse® (09/01/2016–08/31/2018). The study period spanned from the index date (date of first SMA1 diagnosis after nusinersen approval [12/23/2016]) until death/end of available data. HRU and costs per-patient-per-year (PPPY; 2018USD) were described during the study period for all patients and after treatment initiation for nusinersen-treated patients. Results: A total of 349 SMA1 patients (median age=1 year; 55.6% female) with median follow-up of 7.9 months were included. The proportion of patients receiving mechanical ventilation, nutritional support, and physical therapy/rehabilitation was 46.4%, 46.1%, and 22.6%. Patients had, on average, 59.4 days with medical visits/year (14.1 inpatient, 13.4 respiratory failure-related). The 45 nusinersen-treated patients had, on average, 56.6 days with medical visits/year (4.6 inpatient, 11.4 respiratory failure-related). Excluding nusinersen-related costs, mean healthcare costs PPPY were $137,627 (median: $43,167) for all patients and $92,618 ($29,425) for nusinersen-treated patients. Mean nusinersen-related costs were $191,909 ($144,487) per month for the first 3 months post-initiation and $36,882 ($16,132) per month thereafter. Conclusions: HRU and costs associated with SMA1 are substantial, even among patients treated with nusinersen.

Systematic Literature Review to Assess the Cost and Resource Use Associated with Spinal Muscular Atrophy Management

“…A total of 34 publications, comprising 32 unique studies of patients with SMA that met all eligibility criteria were identified in the original search. Two conference abstracts were linked [48,49], and one conference abstract and one manuscript were linked [50,51]. However, these abstracts and manuscripts contained unique data; therefore, all were included as discrete publications.…”

“…The final list of 51 publications comprised 27 full-text publications [50,, 23 conference abstracts [48,49,51,52,[79][80][81][82][83][84][85][86][87][88][89][90][91][92][93][94][95][96][97] and one slide deck [98]. Cost and resource use data were reported in 23 publications [48, 50, 53-57, 60, 61, 68, 70-77, 79, 84, 85, 92, 98].…”

“…Of the 51 publications included in the SLR, 12 included data that examined the use of DMTs nusinersen and onasemnogene abeparvovec; four were full-text publications [50,61,63,68], whereas eight were abstracts [48,49,51,81,88,90,91,93]. Because of earlier approval in the USA and Europe for nusinersen, most of the 12 publications examined nusinersen only [50,51,61,68,81,88,90,93], whereas three publications compared treatment with onasemnogene abeparvovec vs nusinersen [48,49,63], and one publication examined onasemnogene abeparvovec [91]. No data are currently available for the effect of treatment with risdiplam on cost and resource use as a result of the recent approval of risdiplam in the USA and Europe.…”

“…Short-term follow-up of patients treated with nusinersen showed that invasive respiratory support may still be required despite treatment with a DMT [81]. Another study found that although cost and resource use in nusinersentreated patients were substantial, a reduction in associated costs occurred 3 months after initiation of treatment [51]. Two abstracts, published by the same research group, suggested that onasemnogene abeparvovec, a single-dose treatment, may lead to lower estimated direct medical costs for patients with Type 1 SMA compared with treatment with nusinersen [48,49].…”