Nd2 Cost-Utility Analysis of Single Dose Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1 Compared to Chronic Nusinersen Treatment

Malone, Daniel C.; Dean, Rebecca; Miller, Beckley; Arjunji, R.; Feltner, Douglas E.; Sproule, D.M.; Jensen, I.; Maru, B.; Dabbous, O.

doi:10.1016/j.jval.2019.04.060

Cited by 3 publications

(9 citation statements)

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“…The nine publications included in this SLR comprised four conference abstracts [53][54][55][56], two peer-reviewed, original research articles [57,58] and three distinct HTA submissions: one submission to the Canadian Agency for Drugs and Technologies in Health (CADTH) published in 2018 [59], one submission (TA588) to the UK NICE published in 2018 [60] and one submission to the US ICER Group published in 2019 [61]. The CADTH [59], NICE [60] and US ICER Group [61] HTA submissions comprised economic models from studies conducted in Canada, the UK and the US, respectively.…”

Section: Cost-utility Analysis Publication Typesmentioning

confidence: 99%

Section: Conference Abstracts and Journal Articlesmentioning

confidence: 99%

“…Six economic evaluations, published as conference abstracts or journal articles, represented five studies conducted in the US [53][54][55][56][57] and one published economic evaluation reported a study conducted in Sweden [58]. Two publications based on a study conducted by the same research group in the US were published as an abstract [53] and a full-text publication [57]. The evaluations considered patients with early infantile-onset SMA (most likely to develop Type 1 or Type 2 SMA) (n = 4) [53][54][55]57], later-onset SMA (n = 1) [56] and both infantile-and later-onset SMA (n = 1) [58].…”

Section: Conference Abstracts and Journal Articlesmentioning

confidence: 99%

“…Two publications based on a study conducted by the same research group in the US were published as an abstract [53] and a full-text publication [57]. The evaluations considered patients with early infantile-onset SMA (most likely to develop Type 1 or Type 2 SMA) (n = 4) [53][54][55]57], later-onset SMA (n = 1) [56] and both infantile-and later-onset SMA (n = 1) [58]. Treatment comparisons considered across the six published economic evaluations included nusinersen versus SOC [54][55][56], nusinersen + SOC versus SOC [58], onasemnogene abeparvovec versus SOC [54], onasemnogene abeparvovec compared with nusinersen + SOC [57] and onasemnogene abeparvovec compared with nusinersen as the SOC [53].…”

Section: Conference Abstracts and Journal Articlesmentioning

confidence: 99%

“…The evaluations considered patients with early infantile-onset SMA (most likely to develop Type 1 or Type 2 SMA) (n = 4) [53][54][55]57], later-onset SMA (n = 1) [56] and both infantile-and later-onset SMA (n = 1) [58]. Treatment comparisons considered across the six published economic evaluations included nusinersen versus SOC [54][55][56], nusinersen + SOC versus SOC [58], onasemnogene abeparvovec versus SOC [54], onasemnogene abeparvovec compared with nusinersen + SOC [57] and onasemnogene abeparvovec compared with nusinersen as the SOC [53]. An incremental cost per quality-adjusted life-year (QALY) gained was reported in all six publications [53][54][55][56][57][58].…”

Section: Conference Abstracts and Journal Articlesmentioning

confidence: 99%

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Systematic Literature Review to Assess Economic Evaluations in Spinal Muscular Atrophy (SMA)

et al. 2021

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Background Spinal muscular atrophy (SMA) is a rare, progressive neuromuscular disease that affects individuals with a broad age range. SMA is typically characterised by symmetrical muscle weakness but is also associated with cardiac defects, life-limiting impairments in respiratory function and bulbar function defects that affect swallowing and speech. Despite the advent of three innovative disease-modifying therapies (DMTs) for SMA, the cost of DMTs in addition to the costs of standard of care can be a barrier to treatment access for patients. Health Technology Assessment (HTA) decision makers evaluate the cost effectiveness of a new treatment before making a reimbursement decision. Objective The primary objective was to conduct a systematic literature review (SLR) to identify the modelling approaches used in economic evaluations that assess current approved treatments in SMA, with a secondary objective to widen the scope and identify economic evaluations assessing other (non-SMA) neuromuscular disorders. Methods An SLR was performed to identify available economic evaluations associated with any type of SMA (Type 1, 2, 3 and/or 4). Economic evaluations associated with other (non-SMA) neuromuscular disorders were identified but not further analysed. Electronic searches were conducted in Embase, MEDLINE, Evidence-Based Medicine Reviews and EconLit via the Ovid platform in August 2019, and were supplemented by searches of the grey literature (reference lists, conference proceedings, global HTA body websites and other relevant sources). Eligibility criteria were based on the population, interventions, comparators and outcomes (PICO) framework. Quality assessment of full publications was conducted with reference to a published checklist. Results Nine publications covering eight unique studies met all eligibility criteria for inclusion in the SLR, including four conference abstracts, two peer-reviewed original research articles and three HTA submissions (conducted in Canada, the US and the UK). Evaluations considered patients with early infantile-onset (most likely to develop Type 1 or Type 2 SMA), lateronset SMA and both infantile-and later-onset SMA. Data for the identified economic models were collected from literature reviews and relatively short-term clinical trials. Several intent-to-treat clinical trial populations were used in the studies, which resulted in variation in cycle length and different outcome measures to determine clinical efficacy. The results of the quality assessment on the five full-text, peer-reviewed publications found that they generally provided clear descriptions of objectives, modelling methods and results. However, key decisions, such as choice of economic evaluation, model type and choice of variables for sensitivity analysis, were often not adequately justified. Conclusions This SLR highlights the need for economic evaluations in SMA to better align in modelling approaches with respect to (i) consistency in model structure and use of motor function milestones as health states; (ii) con...

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