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

Paracha, Noman; Hudson, Pollyanna; Mitchell, Stephen; Sutherland, C. Simone

doi:10.1007/s40273-021-01105-7

Cited by 13 publications

(11 citation statements)

References 99 publications

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“…The oral, “at-home” daily dosing of risdiplam was beneficial particularly during the COVID-19 pandemic when patients were limited or restricted from traveling to clinical sites. Taking risdiplam at home also reduced the need to travel to hospitals to receive treatment, which decreased the burden on patients and caregivers [ 31 ]. Additionally, risdiplam was a viable treatment option for individuals with SMA with advanced scoliosis who may have difficulty receiving treatment intrathecally.…”

Section: Discussionmentioning

confidence: 99%

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study

et al. 2023

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Introduction Risdiplam is a survival of motor neuron 2 ( SMN2 ) splicing modifier for the treatment of patients with spinal muscular atrophy (SMA). The JEWELFISH study (NCT03032172) was designed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of risdiplam in previously treated pediatric and adult patients with types 1–3 SMA. Here, an analysis was performed after all patients had received at least 1 year of treatment with risdiplam. Methods Patients with a confirmed diagnosis of 5q-autosomal recessive SMA between the ages of 6 months and 60 years were eligible for enrollment. Patients were previously enrolled in the MOONFISH study (NCT02240355) with splicing modifier RG7800 or treated with olesoxime, nusinersen, or onasemnogene abeparvovec. The primary objectives of the JEWELFISH study were to evaluate the safety and tolerability of risdiplam and investigate the PK after 2 years of treatment. Results A total of 174 patients enrolled: MOONFISH study ( n = 13), olesoxime ( n = 71 patients), nusinersen ( n = 76), onasemnogene abeparvovec ( n = 14). Most patients (78%) had three SMN2 copies. The median age and weight of patients at enrollment was 14.0 years (1–60 years) and 39.1 kg (9.2–108.9 kg), respectively. About 63% of patients aged 2–60 years had a baseline total score of less than 10 on the Hammersmith Functional Motor Scale–Expanded and 83% had scoliosis. The most common adverse event (AE) was upper respiratory tract infection and pyrexia (30 patients each; 17%). Pneumonia (four patients; 2%) was the most frequently reported serious AE (SAE). The rates of AEs and SAEs per 100 patient-years were lower in the second 6-month period compared with the first. An increase in SMN protein was observed in blood after risdiplam treatment and was comparable across all ages and body weight quartiles. Conclusions The safety and PD of risdiplam in patients who were previously treated were consistent with those of treatment-naïve patients. Supplementary Information The online version contains supplementary material available at 10.1007/s40120-023-00444-1.

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Section: Discussionmentioning

confidence: 99%

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study

et al. 2023

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“…In this supplement, a systematic review by Paracha et al [10] reports substantially higher direct healthcare costs among infants with SMA relative to a matched sample of infants without SMA, as well as cost differences by SMA type. Type 1 patients have the highest annual direct costs but, as a result of their relatively short life expectancy, the lowest lifetime costs.…”

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confidence: 94%

Dramatic Innovations in the Treatment of Spinal Muscular Atrophy, But Many Unknowns Remain

Skedgel

2021

PharmacoEconomics

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“…Onasemnogene Abeparvovec: Onasemnogene Abeparvovec was the second drug approved by FDA and is administered as a onetime intravenous injection. Onasemnogene Abeparvovec has been proven to have positive impacts on reducing ventilatory and dietary support needs, and improving mobility in SMA type 1 patients (4,9). Like its predecessor, Onasemnogene Abeparvovec was also covered by insurance plans in many countries.…”

Section: B) E Cacymentioning

confidence: 99%

Is it ethical to reimburse “SMA Innovative Treatments” from government resources?

Yaftian,

Mobinizadeh,

Olyaeemanesh

et al. 2023

Preprint

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Background Spinal Muscular Atrophy (SMA) is a rare genetic disorder that disturbs the quality of life. This paper aims to gather current understanding of safety, efficacy and economic aspects of common SMA treatments to help Iranian policy-makers to adopt such novel treatments for this vulnerable population. Methods This is a rapid health technology assessment (HTA) research that was conducted in four steps: database review, screening, data extraction, and thematic analysis. Inclusion criteria consisted of studies focused on assessing medical interventions’ safety, efficacy and economic aspects in SMA patients compared to those who did not receive such interventions. Results Based on current data Nusinersen was found to be most effective (increased mobility achievements in SMA type 1 and 2) with the least side effects for SMA types 1 and 2. In terms of economic evaluations, all treatments were found to be not cost effective. Conclusions The decision regarding the reimbursement for such medical drugs must not focus on their cost-effectiveness but rather on creating access to such essential care and meeting patient needs and also national budget limitations. Managed Entry Agreements (MEAs) are flexible tools that can be used by service providers or payers to negotiate and reduce the financial burden for patients and payers.

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

Cited by 13 publications

References 99 publications

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study

Dramatic Innovations in the Treatment of Spinal Muscular Atrophy, But Many Unknowns Remain

Is it ethical to reimburse “SMA Innovative Treatments” from government resources?

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