Abstract 15848: Hardly a Windfall: Prescribing Practices in Pediatric Heart Failure Before and After Food and Drug Administration Approval

Abstract

Background: Prior to the 2019 Food and Drug Administration (FDA) approval of ivabradine and sacubitril/valsartan, there were no medications approved for the treatment of pediatric heart failure (PHF). There are minimal data regarding the use of on- or off-label medications in PHF. Aims: We aim to describe medication use in PHF, factors associated with PHF medication use, and test the hypothesis that FDA approval is associated with changes in prescriber practice. Methods: Utilizing the Truven Health Marketscan Claims Encounter Database (2013-2021), we determined the prevalence of medication [diuretics, angiotensin-converting enzyme inhibitors (ACEi)/angiotensin receptor blockers (ARBs), beta-blockers, pulmonary vasodilators, ivabradine, and sacubitril/valsartan] use in children ≤19yo with PHF and cardiomyopathy (CM), with a focus on use before and after FDA approval. Results: Of 2,218 children with PHF and CM, 76% were prescribed ≥ 1 HF medication. Diuretics (57%) and ACEi/ARBs (57%) were used most frequently ( Fig 1A) . Only 0.4% of patients were prescribed ivabradine and 3% sacubitril/valsartan. Patients enrolled in Medicaid were more likely to be prescribed HF medications than those with commercial insurance (OR 1.83, 95% CI 1.50-2.22). After FDA approval in 2019, sacubitril/valsartan prescription rose significantly from 0.4% in 2013-2018, to 3.9% in 2020-2021 (p <0.001), and ivabradine use increased from 0.1% in 2018 to 0.5% (p = 0.043) in 2020-2021, but both remained rare (Fig 1B) . Conclusion: Diuretics and ACEi/ARBs are the most commonly used PHF medications, despite not being approved for this indication. The two FDA approved medications are used in ≤5% of patients. While FDA approval is important, it did not substantially influence physician prescribing behavior for PHF. Further study is needed to understand the value of FDA approval and the potential socioeconomic implications of the association between insurance type and medication use.