Review of cost-effectiveness analysis to study pediatric intestinal failure and transplant

TL;DR

This paper reviews how cost-effectiveness analysis can help compare treatments for pediatric intestinal failure, including transplant options.

Contribution

The paper provides a framework for applying cost-effectiveness analysis in pediatric intestinal failure treatment decisions.

Findings

Cost-effectiveness analysis can compare medical treatments versus transplant for intestinal failure.

Teduglutide and antimicrobial lock therapy are examples of costly treatments evaluated in the US.

The methodology can be adapted to different healthcare systems globally.

Abstract

Cost-effectiveness analysis is a comparative methodology used to determine how costs and benefits align for a given treatment when evaluated against one or more competing strategies. In intestinal failure, this may include direct comparisons between medical treatments vs transplant or individual care decisions. Teduglutide and antimicrobial lock therapy provide two recent examples of treatments in the United States that came under scrutiny due to cost. We demonstrate the use of cost-effectiveness analysis to critically examine these treatments in the US as an example that may be adapted to various healthcare contexts worldwide. We then discuss the required steps to employ this methodology more effectively in pediatric intestinal failure.

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