Kevin F. Erickson, MD, MS; Glenn M. Chertow, MD, MPH; Jeremy D. Goldhaber-Fiebert, PhD
Grant Support: By the Agency for Healthcare Research and Quality (F32 HS019178) and National Institutes of Health (DK085446 and AG037593).
Potential Conflicts of Interest: Disclosures can be viewed at www.acponline.org/authors/icmje/ConflictOfInterestForms.do?msNum=M13-0381.
Reproducible Research Statement: Study protocol and data set: Available from Dr. Erickson (e-mail, email@example.com). Statistical code: Not available.
Requests for Single Reprints: Kevin Erickson, MD, MS, Stanford University School of Medicine, 117 Encina Commons, Stanford, CA 94305-6019; e-mail, firstname.lastname@example.org.
Current Author Addresses: Drs. Erickson and Goldhaber-Fiebert: Stanford University School of Medicine, 117 Encina Commons, Stanford, CA 94305-6019.
Dr. Chertow: Division of Nephrology, Stanford University School of Medicine, 777 Welch Road, Suite DE, Palo Alto, CA 94304.
Author Contributions: Conception and design: K.F. Erickson, G.M. Chertow, J.D. Goldhaber-Fiebert.
Analysis and interpretation of the data: K.F. Erickson, G.M. Chertow, J.D. Goldhaber-Fiebert.
Drafting of the article: K.F. Erickson.
Critical revision of the article for important intellectual content: K.F. Erickson, G.M. Chertow, J.D. Goldhaber-Fiebert.
Final approval of the article: K.F. Erickson, G.M. Chertow, J.D. Goldhaber-Fiebert.
Statistical expertise: K.F. Erickson, J.D. Goldhaber-Fiebert.
Administrative, technical, or logistic support: K.F. Erickson, G.M. Chertow.
Collection and assembly of data: K.F. Erickson.
Erickson KF, Chertow GM, Goldhaber-Fiebert JD. Cost-Effectiveness of Tolvaptan in Autosomal Dominant Polycystic Kidney Disease. Ann Intern Med. 2013;159:382-389. doi: 10.7326/0003-4819-159-6-201309170-00004
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Published: Ann Intern Med. 2013;159(6):382-389.
In the TEMPO (Tolvaptan Efficacy and Safety in Management of Autosomal Dominant Polycystic Kidney Disease and Its Outcomes) trial, tolvaptan significantly reduced expansion of kidney volume and loss of kidney function.
To determine how the benefits of tolvaptan seen in TEMPO may relate to longer-term health outcomes, such as progression to end-stage renal disease (ESRD) and death, and cost-effectiveness.
A decision-analytic model.
Published literature from 1993 to 2012.
Persons with early autosomal dominant polycystic kidney disease.
Patients received tolvaptan therapy until death, development of ESRD, or liver complications or no tolvaptan therapy.
Median age at ESRD onset, life expectancy, discounted quality-adjusted life-years and lifetime costs (in 2010 U.S. dollars), and incremental cost-effectiveness ratios.
Tolvaptan prolonged the median age at ESRD onset by 6.5 years and increased life expectancy by 2.6 years. At $5760 per month, tolvaptan cost $744 100 per quality-adjusted life-year gained compared with standard care.
For patients with autosomal dominant polycystic kidney disease that progressed more slowly, the cost per quality-adjusted life-year gained was even greater for tolvaptan.
Although TEMPO followed patients for 3 years, the main analysis assumed that clinical benefits persisted over patients' lifetimes.
Assuming that the benefits of tolvaptan persist in the longer term, the drug may slow progression to ESRD and reduce mortality rates. However, barring an approximately 95% reduction in price, cost-effectiveness does not compare favorably with many other commonly accepted medical interventions.
National Institutes of Health and Agency for Healthcare Research and Quality.
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