TY - JOUR
T1 - Microsimulation Model to Determine the Cost-Effectiveness of Treat-to-Target Strategies for Ulcerative Colitis
AU - Dulai, Parambir S.
AU - Sandborn, William J.
AU - Murphy, James
N1 - Funding Information:
Funding Parambir S. Dulai is supported by an American Gastroenterology Association Research Scholar Award.
Funding Information:
Conflicts of interest These authors disclose the following: Parambir S. Dulai reports consulting and research grants from Takeda, Janssen, Pfizer, AbbVie, Buhlmann, and Polymedco. William J. Sandborn reports personal fees from Kyowa Hakko Kirin, Millennium Pharmaceuticals, Celgene Cellular Therapeutics, Santarus, Salix Pharmaceuticals, Catabasis Pharmaceuticals, Vertex Pharmaceuticals, Warner Chilcott, Cosmo Pharmaceuticals, Ferring Pharmaceuticals, Sigmoid Biotechnologies, Tillotts Pharma, Am Pharma BV, Dr. August Wolff, Avaxia Biologics, Zyngenia, Ironwood Pharmaceuticals, Index Pharmaceuticals, Nestlé, Lexicon Pharmaceuticals, UCB Pharma, Orexigen, Luitpold Pharmaceuticals, Baxter Healthcare, Ferring Research Institute, Novo Nordisk, Mesoblast, Shire, Ardelyx, Actavis, Seattle Genetics, MedImmune (AstraZeneca), ActoGeniX NV, Lipid Therapeutics Gmbh, Eisai, Qu Biologics, Toray Industries, Teva Pharmaceuticals, Eli Lilly, Chiasma, TiGenix, Adheron Therapeutics, Immune Pharmaceuticals, Celgene, Arena Pharmaceuticals; personal fees from Ambrx, Akros Pharma, Vascular Biogenics, Theradiag, Forward Pharma, Regeneron, Galapagos, Seres Health, Ritter Pharmaceuticals, Theravance, Palatin, Biogen, University of Western Ontario (owner of Robarts Clinical Trials); and grants and personal fees from Prometheus Laboratories, AbbVie, Gilead Sciences, Boehringer Ingelheim, Amgen, Takeda, Atlantic Pharmaceuticals, Bristol-Myers Squibb, Genentech, GlaxoSmithKline, Pfizer, Nutrition Science Partners, Receptos, and Amgen; grants, personal fees, and nonfinancial support from Janssen; and grants from the Broad Foundation, American College of Gastroenterology, and Exact Sciences. James Murphy reports compensation from consultation with Boston Consulting Group.
Publisher Copyright:
© 2021 AGA Institute
PY - 2021/6
Y1 - 2021/6
N2 - Background & Aims: Little is known about the cost effectiveness of endoscopy or biomarker-based treat to target monitoring of patients with ulcerative colitis (UC). Methods: We used a microsimulation model to identify the most cost effective treat to target monitoring strategy for patients with UC staring therapy with biologics or small molecule inhibitors. We assessed symptoms (rectal bleeding) alone, a combination of symptoms and a biomarker (fecal calprotectin), and endoscopy. Transition probabilities, costs, and quality-adjusted life year (QALY) estimates were derived from published estimates. The microsimulation model tracked an individual patient's disease course and treatment exposures to modify downstream treatment effectiveness, probabilities, and disease outcomes. The primary analysis included 100,000 individuals over 5 years with a willingness to pay threshold of $100,000/QALY. Probabilistic sensitivity analyses were performed with 500 samples and 250 trials, in addition to multiple 1-, 2-, and 3-way microsimulation sensitivity analyses. Results: A total of 32 treatment sequencing algorithms were modeled alongside 3 disease monitoring strategies within a treat to target approach for UC. Combination symptom and biomarker-based monitoring resulted in the highest QALY estimate among all the treatment sequencing algorithms. However, monitoring disease activity with symptoms alone was the most cost-effective strategy in 86% of scenarios, followed by combination symptom and biomarker monitoring in 9%, and endoscopy monitoring in 5%. Results were sensitive to treatment costs, patient willingness to consider colectomy as a treatment option, and endoscopy costs. Endoscopy-based monitoring was favored when treatment costs were high and patients were unwilling to undergo colectomy. Conclusions: The combination symptom and biomarker-based monitoring resulted in the highest QALY estimate. However, symptom-based monitoring is the most cost-effective approach to implementing treat to target monitoring for patients with UC receiving biologics and small molecule inhibitors.
AB - Background & Aims: Little is known about the cost effectiveness of endoscopy or biomarker-based treat to target monitoring of patients with ulcerative colitis (UC). Methods: We used a microsimulation model to identify the most cost effective treat to target monitoring strategy for patients with UC staring therapy with biologics or small molecule inhibitors. We assessed symptoms (rectal bleeding) alone, a combination of symptoms and a biomarker (fecal calprotectin), and endoscopy. Transition probabilities, costs, and quality-adjusted life year (QALY) estimates were derived from published estimates. The microsimulation model tracked an individual patient's disease course and treatment exposures to modify downstream treatment effectiveness, probabilities, and disease outcomes. The primary analysis included 100,000 individuals over 5 years with a willingness to pay threshold of $100,000/QALY. Probabilistic sensitivity analyses were performed with 500 samples and 250 trials, in addition to multiple 1-, 2-, and 3-way microsimulation sensitivity analyses. Results: A total of 32 treatment sequencing algorithms were modeled alongside 3 disease monitoring strategies within a treat to target approach for UC. Combination symptom and biomarker-based monitoring resulted in the highest QALY estimate among all the treatment sequencing algorithms. However, monitoring disease activity with symptoms alone was the most cost-effective strategy in 86% of scenarios, followed by combination symptom and biomarker monitoring in 9%, and endoscopy monitoring in 5%. Results were sensitive to treatment costs, patient willingness to consider colectomy as a treatment option, and endoscopy costs. Endoscopy-based monitoring was favored when treatment costs were high and patients were unwilling to undergo colectomy. Conclusions: The combination symptom and biomarker-based monitoring resulted in the highest QALY estimate. However, symptom-based monitoring is the most cost-effective approach to implementing treat to target monitoring for patients with UC receiving biologics and small molecule inhibitors.
KW - Disease Progression
KW - IBD
KW - Management
KW - Outcome
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U2 - 10.1016/j.cgh.2020.05.010
DO - 10.1016/j.cgh.2020.05.010
M3 - Article
C2 - 32437872
AN - SCOPUS:85103975841
VL - 19
SP - 1170-1179.e10
JO - Clinical Gastroenterology and Hepatology
JF - Clinical Gastroenterology and Hepatology
SN - 1542-3565
IS - 6
ER -