Cost-effectiveness of pasireotide long-acting release in acromegaly: a systematic literature review and methodology assessment.
The cost-effectiveness of pasireotide LAR compared to pegvisomant remains inconclusive, with some studies favoring pegvisomant while others support pasireotide LAR as a cost-effective alternative.
Where it sits
this study against the rest of the pasireotide corpusSummary and findings
This study assessed the cost-effectiveness of pasireotide long-acting release (LAR) in adults with acromegaly compared to other second-line therapies, particularly pegvisomant. Six publications were included in the analysis, revealing variability in findings. The study aimed to clarify the economic implications of pasireotide LAR as a treatment option.
Abstract
<h4>Background</h4>Acromegaly is a rare and progressive condition caused by excessive secretion of growth hormone and insulin-like growth factor type 1. Pasireotide long-acting release (LAR) is indicated as a second-line therapy for adults with acromegaly who are unsuitable for or unresponsive to surgery and inadequately controlled with first-generation somatostatin receptor ligands (FGSRLs). Although its efficacy and safety have already been established, its cost-effectiveness remains unclear. The primary objective of this study was to systematically assess the cost-effectiveness of pasireotide LAR compared to other second-line medical options, particularly pegvisomant.<h4>Methods</h4>A systematic literature review was conducted in May 2024 in Medline, Medline In Process, Web of Science, and the Centre for Reviews and Dissemination (CRD), York. Studies were eligible if they were full or partial economic analyses of pasireotide LAR as a second-line pharmacological treatment for adult patients with acromegaly. Studies not available in English and publications prior to 2009 were excluded. Included articles were assessed for transparency using the appropriate checklists. Data extraction focused on costs, incremental cost-effectiveness ratios and quality-adjusted life years. Formal data synthesis of outcomes was not undertaken due to the small number of studies and notable heterogeneity between them. Methodological assessment involving the evaluation of model inputs, data sources, and their alignment with the evidence on the course of disease and clinical practice was performed to examine the main factors contributing to discrepancies in cost-effectiveness outcomes and assess the credibility of the results.<h4>Results</h4>Of 160 records identified, six publications met the inclusion criteria. Their findings demonstrated variability. The critical assessment highlighted considerable variability in methodological rigor among the included studies.<h4>Conclusions</h4>Evidence on pasireotide LAR's cost-effectiveness versus pegvisomant regimens remains inconclusive. Although two studies indicated that pegvisomant is more cost-effective, the study with the highest methodological credibility found that pasireotide LAR is a cost-effective alternative.
Background
Acromegaly is a rare condition linked to excessive growth hormone and insulin-like growth factor type 1 secretion. While pasireotide LAR is established as a second-line therapy for patients unresponsive to surgery or first-generation somatostatin receptor ligands, its cost-effectiveness compared to alternatives like pegvisomant is not well defined. This study aims to clarify the economic viability of pasireotide LAR in treating acromegaly.
Methods
A systematic literature review was conducted in May 2024, searching Medline, Medline In Process, Web of Science, and the Centre for Reviews and Dissemination. Eligible studies included full or partial economic analyses of pasireotide LAR as a second-line treatment for adult acromegaly patients. Studies not available in English and those published prior to 2009 were excluded. Data extraction focused on costs, incremental cost-effectiveness ratios, and quality-adjusted life years.
Results
Six publications met the inclusion criteria from an initial 160 records. The findings from these studies showed variability, and the critical assessment revealed considerable differences in methodological rigor. No formal data synthesis was performed due to the small number of studies and their notable heterogeneity.
Interpretation
The inconclusive evidence on pasireotide LAR's cost-effectiveness compared to pegvisomant indicates that while some studies suggest pegvisomant is more cost-effective, others with higher methodological credibility support pasireotide LAR as a viable alternative. The variability in findings and methodological rigor limits the ability to draw definitive conclusions, which is crucial for clinical decision-making.
Key findings
- Of 160 records identified, six publications met the inclusion criteria.
- Two studies indicated that pegvisomant is more cost-effective.
- The study with the highest methodological credibility found that pasireotide LAR is a cost-effective alternative.
Limitations
- small n=6 studies included
- not all studies had high methodological rigor
- no formal data synthesis due to study heterogeneity
- inconclusive evidence on cost-effectiveness