Systematic Literature Review of Clinical and Economic Evidence for Spinal Muscular Atrophy.
Adv Ther
; 39(5): 1915-1958, 2022 05.
Article
in En
| MEDLINE
| ID: mdl-35307799
ABSTRACT
INTRODUCTION:
The recent advent of disease-modifying therapies (DMTs) has dramatically changed the treatment landscape of spinal muscular atrophy (SMA), and the multifaceted impact of this advancement has not been assessed thoroughly in the growing body of literature. We sought to summarize the literature on the natural history of SMA and the impact of SMA DMTs, including health-related quality of life (HRQOL) and utilities, clinical efficacy and safety, and economic impact.METHODS:
Systematic literature reviews were conducted following PRISMA guidelines with no inclusive dates. Relevant studies were identified by searching full-text databases on November 12-13, 2020, including MEDLINE, EMBASE, the Cochrane Central Register of Controlled Trials, and EconLit, conference proceedings, health technology assessment databases, and clinical trial registries. All searches used a combination of MeSH and key terms. Studies were screened according to criteria based upon population, intervention, outcomes, and study design structure.RESULTS:
Findings from 17, 23, 32, and 42 studies were included for the evaluation of natural history of SMA, HRQOL and utilities, clinical efficacy and safety, and economic impact of DMTs, respectively. Currently available data indicate that untreated SMA is associated with considerable humanistic and economic burden, with estimates of costs varying by treatment. While a variety of interventions have been evaluated in SMA clinical trials, quantitative synthesis of safety and efficacy findings was not feasible because of inconsistencies in reported outcomes. Data assessing impacts of DMTs on HRQOL were also lacking.CONCLUSIONS:
Overall, this systematic literature review highlights a clear need for up-to-date and methodologically rigorous clinical, HRQOL, and economic data to support unbiased assessments of the relative clinical and economic effectiveness of SMA treatments. More research is required to extend our understanding of the burden of SMA on HRQOL utility assessments and the impact of new DMTs on HRQOL and utilities for patients with SMA.Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Quality of Life
/
Muscular Atrophy, Spinal
Type of study:
Clinical_trials
/
Guideline
/
Health_economic_evaluation
/
Health_technology_assessment
/
Prognostic_studies
/
Systematic_reviews
Aspects:
Patient_preference
Limits:
Humans
Language:
En
Journal:
Adv Ther
Journal subject:
TERAPEUTICA
Year:
2022
Document type:
Article
Affiliation country:
Estados Unidos