Observational Study
Copyright ©The Author(s) 2025. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Gastrointest Oncol. Aug 15, 2025; 17(8): 109544
Published online Aug 15, 2025. doi: 10.4251/wjgo.v17.i8.109544
Cost-effectiveness analysis of pancreatic enzyme replacement therapy in patients with pancreatic exocrine insufficiency in China
Hansoo Kim, Joshua Byrnes, Kui-Rong Jiang, Zhuan Liao, Arun Jones, Kyoo Kim, Dafni Fragkogianni, Keith J Roberts
Hansoo Kim, Joshua Byrnes, Arun Jones, Health Economics Group, Griffith University, Gold Coast QLD4215, Queensland, Australia
Kui-Rong Jiang, Department of General Surgery, The First Affiliated Hospital of Nanjing Medical University, Nanjing 210009, Jiangsu Province, China
Zhuan Liao, Shanghai Institute of Pancreatic Diseases, Shanghai 201613, China
Kyoo Kim, Dafni Fragkogianni, Abbott Products Operations AG, Hegenheimermattweg 127, 4123 Allschwil, Switzerland
Keith J Roberts, Department of HBP and Liver Transplantation Surgery, Queen Elizabeth Hospital, University Hospitals Birmingham, Birmingham B15 2GW, United Kingdom
Author contributions: Kim H, Byrnes J, Jiang KR, Kim K, Liao Z, Roberts KJ contributed to conceptualization; Kim H, Byrnes J, Roberts KJ contributed to data curation, formal analysis, writing original draft; Fragkogianni D, Kim H, Byrnes J, Kim K, Roberts KJ contributed to funding acquisition; Jones A, Fragkogianni D, Kim H, Byrnes J, Jiang KR, Kim K, Liao Z, Roberts KJ contributed to investigation, writing review and editing; Kim H, Byrnes J, Jiang KR, Liao Z, Roberts KJ contributed to methodology; Jones A, Fragkogianni D, Kim H, Byrnes J, Kim K, Roberts KJ contributed to project administration; Jones A, Kim H, Byrnes J, Roberts KJ contributed to software, visualization; Fragkogianni D, Kim H, Byrnes J, Jiang KR, Kim K, Liao Z, Roberts KJ contributed to resources, supervision; Kim H, Byrnes J, Jiang KR, Liao Z, Roberts KJ contributed to validation.
Institutional review board statement: This study did not involve human and/or animal subjects and is therefore not subject to approval from an Institutional Review Board.
Informed consent statement: This study did not involve human subjects and therefore no informed consent was required or sought.
Conflict-of-interest statement: Kim H has received consulting fees from Abbott, CSL Behring, Bristol Myers-Squibb, Novartis and Sanofi in the past 5 years. Kim K and Fragkogianni D are employed by Abbott; Byrnes J discloses investigator-initiated research and educational grants paid to his employer (Griffith University) from 3M, NeoNav, BD-Bard, as well as consultancy payments paid to these employers from 3M, BD-Bard, Edwards Life Sciences, and Abbott. No conflicts are declared by the remaining authors.
STROBE statement: The authors have read the STROBE Statement—a checklist of items, and the manuscript was prepared and revised according to the STROBE Statement-a checklist of items.
Data sharing statement: Technical data and data on file will be made available on contact of the corresponding author of this article at arun.jones@griffith.edu.au.
Open Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Arun Jones, MSc, Health Economics Group, Griffith University, 1 Parklands Dr, Gold Coast QLD4215, Queensland, Australia. arun.jones@griffith.edu.au
Received: May 15, 2025
Revised: May 29, 2025
Accepted: July 7, 2025
Published online: August 15, 2025
Processing time: 91 Days and 16.7 Hours
Abstract
BACKGROUND

Pancreatic exocrine insufficiency (PEI) leads to fat malabsorption and maldigestion and is most commonly treated with pancreatic enzyme replacement therapy (PERT). Patients suffering from PEI in China are more likely not to receive adequate treatment as this drug is not part of the Chinese national essential medicine list.

AIM

To examine the cost-effectiveness of PERT for patients suffering from PEI in China.

METHODS

A decision analytical Markov model was constructed to simulate the progress of patients with PEI in China. The population included in the analyses were patients suffering from PEI with advanced (non-resectable) pancreatic cancer, who have undergone surgery due to pancreatic cancer and who have undergone endoscopic treatment due to chronic pancreatitis. The cost-effectiveness analyses were undertaken from a Chinese societal perspective comparing PERT with no PERT. The incremental cost-effectiveness ratio in United States dollars per quality adjusted life year (QALY) gained is the main outcome. Input was informed by publicly available data supplemented with expert clinical advice.

RESULTS

The cost-effectiveness analyses estimated that PERT resulted in additional 0.45 to 2.93 QALYs at discounted costs of between 4315 dollars to 15193 dollars. This resulted in an incremental cost-effectiveness ratio of 5178 dollars to 9533 dollars per QALY. The one-way sensitivity analyses showed that the main drivers of the model were the cost of PERT and overall survival.

CONCLUSION

This study demonstrates that PERT is a cost-effective treatment for patients suffering from PEI in China.

Keywords: Pancreatic exocrine insufficiency; Pancreatic enzyme replacement therapy; Cost-effectiveness analysis; National essential medicine list; China

Core Tip: Pancreatic enzyme replacement therapy (PERT) is a first-line treatment for pancreatic exocrine insufficiency (PEI). In China, PERT is not yet listed on the national essential medicine list, which is often a pre-requisite for patients in China to gain access to life-saving treatments. Chinese health policy has an increased emphasis on cost-effectiveness analysis as a prerequisite for drug reimbursement. This analysis undertakes a cost-effectiveness analysis on PERT for the treatment of PEI from a Chinese societal perspective, finding that PERT is cost-effective for the treatment of PEI in China.