Cost-Effectiveness of Tolvaptan Vs. Angiotensin-Converting Enzyme Inhibitors for US Patients with Autosomal Dominant Polycystic Kidney Disease
Author(s)
Aaron S. Samson, PharmD, MS-HOPE Candidate, La'ron Browne, MD, Shlok Rohatagi, PharmD Candidate, Maria Mansour, PharmD Candidate, Anushyaa Vasudevan, MS Candidate, Laura A. Clark, BS, MS,PhD;
Rutgers University, Piscataway, NJ, USA
Rutgers University, Piscataway, NJ, USA
Presentation Documents
OBJECTIVES: To determine if tolvaptan (+/- angiotensin-converting enzyme inhibitor (ACE-I)) is more cost-effective than ACE-I monotherapy in treating autosomal dominant polycystic kidney disease (ADPKD).
METHODS: A predictive Markov-state transition model was developed to simulate two ADPKD patient cohorts in the U.S. Costs and quality-adjusted life years (QALYs) were estimated in hypertensive ADPKD patients using a societal perspective that included direct and indirect costs related to ADPKD. Annual direct and indirect costs for specific chronic kidney disease stages (CKD) were derived from an ADPKD societal economic burden article (costs adjusted to 2023 medical-care inflation) and were added to price of using tolvaptan or ACE-I in that stage. Either cohort was treated with an ACE-I and/or tolvaptan (+/- ACE-I). Transition probabilities for ACE-I were sourced from a cost-effective analysis (CEA) of ACE-Is and angiotensin II receptor blockers (ARBs) in ADPKD. In the absence of tolvaptan transition from literature, a kidney function decline ratio was derived and multiplied by the transition probabilities for ACE-I. The time horizon was set to 30 years, with 1-year duration to cycle shift. A CEA was conducted to estimate the incremental cost-effectiveness ratio (ICER) of tolvaptan vs ACE-I per one additional year of prevented death. A one-way probabilistic sensitivity analysis was conducted, with +/-10% variation in probabilities and costs. A willingness to pay (WTP) of $150,000 was used for comparison.
RESULTS: Total annual healthcare costs accrued after 30 years for ADPKD patients using tolvaptan was estimated to be $7,939,288, compared to ACE-I at $2,048,018. Life expectancy was increased by 2.97 years among patients taking tolvaptan. ICER of $1,875,909/QALY implies tolvaptan is not cost-effective.
CONCLUSIONS: Tolvaptan is not more cost effective than ACE-I under the current WTP threshold. Tolvaptan offers higher effectiveness, but the substantial incremental cost ($5.8M) outweighs the benefit of additional QALYs.
METHODS: A predictive Markov-state transition model was developed to simulate two ADPKD patient cohorts in the U.S. Costs and quality-adjusted life years (QALYs) were estimated in hypertensive ADPKD patients using a societal perspective that included direct and indirect costs related to ADPKD. Annual direct and indirect costs for specific chronic kidney disease stages (CKD) were derived from an ADPKD societal economic burden article (costs adjusted to 2023 medical-care inflation) and were added to price of using tolvaptan or ACE-I in that stage. Either cohort was treated with an ACE-I and/or tolvaptan (+/- ACE-I). Transition probabilities for ACE-I were sourced from a cost-effective analysis (CEA) of ACE-Is and angiotensin II receptor blockers (ARBs) in ADPKD. In the absence of tolvaptan transition from literature, a kidney function decline ratio was derived and multiplied by the transition probabilities for ACE-I. The time horizon was set to 30 years, with 1-year duration to cycle shift. A CEA was conducted to estimate the incremental cost-effectiveness ratio (ICER) of tolvaptan vs ACE-I per one additional year of prevented death. A one-way probabilistic sensitivity analysis was conducted, with +/-10% variation in probabilities and costs. A willingness to pay (WTP) of $150,000 was used for comparison.
RESULTS: Total annual healthcare costs accrued after 30 years for ADPKD patients using tolvaptan was estimated to be $7,939,288, compared to ACE-I at $2,048,018. Life expectancy was increased by 2.97 years among patients taking tolvaptan. ICER of $1,875,909/QALY implies tolvaptan is not cost-effective.
CONCLUSIONS: Tolvaptan is not more cost effective than ACE-I under the current WTP threshold. Tolvaptan offers higher effectiveness, but the substantial incremental cost ($5.8M) outweighs the benefit of additional QALYs.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
SA32
Topic
Study Approaches
Topic Subcategory
Decision Modeling & Simulation
Disease
SDC: Urinary/Kidney Disorders