OBJECTIVES: Acromegaly is a rare, progressive disease resulting from excessive production of growth hormone (GH) and insulin-like growth factor type 1 (IGF-1).

In Europe pasireotide (PAS) long-acting release (LAR) is indicated as 2^nd line medical treatment for adults with acromegaly who failed or are not candidates for surgery and who are inadequately controlled by treatment with another somatostatin analogue (SSA).

Due to conflicting reports in the literature, the aim of this study is to systematically determine the cost-effectiveness of pasireotide (PAS) long-acting release (LAR) in the treatment of acromegaly.

METHODS: A systematic literature review was performed following Cochrane and PRISMA guidelines to find all relevant cost-effectiveness analyses including pasireotide. The search was conducted across PubMed, OVID, CRD York and Web of Science. The Joanna Briggs Institute Appraisal Tool (JBI tool) was used to assess the credibility of the studies found.

RESULTS: Out of 160 records identified, 6 unique cost-effectiveness studies were eligible for inclusion. PAS LAR was compared with pegvisomant (PeGV), SSA, and PeGV+SSA in 2^nd line medical treatment.

When comparing PAS LAR with PeGV and PeGV + SSA, analyses presented divergent results regarding incremental health benefits. Per JBI tool appraisal, the most credible study (Leonart et al. 2021) suggested that the treatment strategy with 2^nd line PAS LAR provided similar health benefits compared to a strategy of 2^nd line SSA + PeGV. All studies were consistent in indicating that PAS LAR is a more economical alternative.

Compared to 1^st generation SSA, PAS LAR presented greater QALY benefits and higher therapy costs. PAS LAR was considered to be cost-effective vs SSA in two of the studies.

CONCLUSIONS: PAS LAR offers comparable health benefits to SSA + PeGV and PeGV monotherapy at a lower therapy cost. With PAS LAR generating additional QALY, cost-effectiveness vs. SSA depends on the adopted willingness-to-pay threshold.