OBJECTIVES:

This study evaluates the cost-effectiveness of second line treatments of acromegaly in adults for whom surgery is no option or has not been curative and who were previously inadequately controlled with somatostatin receptor ligands (SRL).

METHODS:

The hybrid model combining a 6-months long decision-tree followed by a lifetime Markov phase was designed to evaluate the cost-effectiveness of pasireotide LAR in a cohort of patients with uncontrolled acromegaly from a Brazilian public payer perspective. Patients are assessed for response at 3 and 6 months after treatment initiation. Those who achieve full control remain on treatment, while uncontrolled patients move to subsequent treatment.

In the base case, disease control was defined as normalization of both growth hormone and insulin-like growth factor-1 (IGF-1) levels. Pasireotide was compared to maximum doses of octreotide and lanreotide (SRLs) and efficacy data was obtained from the PAOLA study. In another scenario, with IGF-1 normalization as the only biochemical control criterion, pegvisomant monotherapy and its combination with an SRL were also evaluated and their efficacy was estimated based on a network meta-analysis.

The association between acromegaly and its risk of mortality was captured through standardized mortality ratios. Quality of life was captured through health-state utilities, and disutilities related to adverse events and comorbidities.

RESULTS:

Pasireotide treatment led to greater incremental health benefits (0.29 LYs and 0.95 QALYs) than SRLs. This resulted in ICERs of R$150,051 and R$159,143 compared to maximal dose octreotide and lanreotide, respectively.

When efficacy was defined as IGF-1 normalization, pasireotide was dominant versus pegvisomant and pegvisomant + lanreotide, but less costly (-R$855,724) and less effective (-0.036 QALYs) versus pegvisomant + octreotide.

CONCLUSIONS:

The analysis showed that pasireotide LAR provides significant health benefits to acromegaly patients. With limited options for second-line medical treatment in Brazil, the reimbursement of pasireotide LAR in acromegaly can be considered cost-effective.