Cost-effectiveness of personalized medical treatment in acromegaly: a post hoc analysis of the ACROFAST study

Abstract Context Medical treatment of acromegaly is currently performed through a trial-and-error approach using first-generation somatostatin receptor ligands (fgSRLs) as first-line drugs, with an efficacy of 50%. Some biomarkers can predict patient response, potentially benefiting nonresponders by using personalized treatment approaches. The ACROFAST study revealed that the probability of disease control was 2.53 times higher (CI 1.30-4.80) with personalized treatment, and insulin-like growth factor 1 (IGF1) normalization occurred in 78% vs 53% of patients, compared with standard treatment (P < .05). Objective To evaluate the cost-effectiveness of personalized medicine based on predictive biomarkers, from the results of the ACROFAST study. Methods Post hoc analysis from the ACROFAST study comparing cost-effectiveness of a protocol of medical treatment based on predictive biomarkers to fgSRLs (n = 32) vs standard treatment (n = 36) for 12 months. Costs analyzed were visits, examinations, study of biomarkers (121€/patient), and pharmacological treatment according to the prices established by the Spanish National Health Care System. Medium-term costs were evaluated, considering a projection of up to 2 years in uncontrolled cases, assuming positive and negative scenarios with additional monitoring costs and a 25% likelihood of disease progression per follow-up visit. Results The personalized protocol reduced the average cost per patient required to achieve disease control by 22% (15 127€ vs 19 420€). These results represent an annual saving of 15 263€ per patient achieving hormonal control compared to the standard treatment. Conclusion Personalized medicine, using a relatively straightforward biomarker-based protocol, enables a greater proportion of patients to attain hormonal control and proves to be a cost-effective strategy for managing acromegaly.