Estimating cost-effectiveness from claims and registry data with measured and unmeasured confounders

Statistical Methods in Medical Research
Elizabeth A HandorfNandita Mitra

Abstract

The analysis of observational data to determine the cost-effectiveness of medical treatments is complicated by the need to account for skewness, censoring, and the effects of measured and unmeasured confounders. We quantify cost-effectiveness as the Net Monetary Benefit (NMB), a linear combination of the treatment effects on cost and effectiveness that denominates utility in monetary terms. We propose a parametric estimation approach that describes cost with a Gamma generalized linear model and survival time (the canonical effectiveness variable) with a Weibull accelerated failure time model. To account for correlation between cost and survival, we propose a bootstrap procedure to compute confidence intervals for NMB. To examine sensitivity to unmeasured confounders, we derive simple approximate relationships between naïve parameters, assuming only measured confounders, and the values those parameters would take if there was further adjustment for a single unmeasured confounder with a specified distribution. A simulation study shows that the method returns accurate estimates for treatment effects on cost, survival, and NMB under the assumed model. We apply our method to compare two treatments for Stage II/III bladder cancer, co...Continue Reading

References

Aug 1, 1991·Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology·D F CellaL H Maurer
Jan 1, 1988·American Journal of Industrial Medicine·O Axelson, K Steenland
Jan 1, 1988·American Journal of Industrial Medicine·M H GailJ H Lubin
Aug 7, 1996·Journal of the National Cancer Institute·J S GoodwinW C Hunt
Oct 9, 1997·The New England Journal of Medicine·J J BarendregtP J van der Maas
May 5, 1998·Medical Decision Making : an International Journal of the Society for Medical Decision Making·A A Stinnett, J Mullahy
Jan 23, 1999·Medical Decision Making : an International Journal of the Society for Medical Decision Making·D F HeitjanW Whang
Jun 22, 2000·Health Economics·D F Heitjan
Sep 17, 2002·Biometrics·Heejung Bang, Anastasios A Tsiatis
Jan 9, 2003·Statistical Methods in Medical Research·A R WillanE B Chen
Mar 26, 2003·Statistics in Medicine·D Y Lin
Aug 23, 2003·Biostatistics·D Y Lin
Jan 31, 2004·PharmacoEconomics·Marc F BottemanRobert Hauser
Apr 15, 2004·Statistics in Medicine·Daniel F HeitjanHuiling Li
Oct 19, 2004·Statistics in Medicine·Hongyu Jiang, Xiao-Hua Zhou
Oct 30, 2004·Statistics in Medicine·Andrew R WillanAndrea Manca
Dec 21, 2004·The European Journal of Health Economics : HEPAC : Health Economics in Prevention and Care·L Di Matteo
Feb 25, 2005·Health Economics·Jan B Oostenbrink, Maiwenn J Al
Sep 15, 2005·Statistics in Medicine·Alka IndurkhyaDeborah Schrag
Jan 21, 2006·Journal of Evaluation in Clinical Practice·Susanna DoddPaula Williamson
Jun 17, 2006·Statistics in Medicine·Nandita Mitra, Daniel F Heitjan
Sep 26, 2006·Statistics in Medicine·Lawrence C McCandlessAdrian Levy
Mar 31, 2007·Neurotherapeutics : the Journal of the American Society for Experimental NeuroTherapeutics·James C Cleland, Eric L Logigian
Sep 6, 2007·Pediatric Neurosurgery·Jill C Glick, Kelley Staley
May 1, 2008·Journal of the National Cancer Institute·K Robin YabroffMartin L Brown
Oct 1, 2001·Expert Review of Pharmacoeconomics & Outcomes Research·H A GlickD Polsky
Jul 29, 2010·Health Economics·Anirban Basu, Willard G Manning
Jan 14, 2011·Journal of the National Cancer Institute·Angela B MariottoMartin L Brown
Jun 21, 2011·CA: a Cancer Journal for Clinicians·Rebecca SiegelAhmedin Jemal
Mar 28, 2012·Journal of Pain & Palliative Care Pharmacotherapy·UNKNOWN Centers for Disease Control and Prevention, Public Health Service, U.S. Department Health
Jan 1, 2013·The Annals of Applied Statistics·Elizabeth A HandorfNandita Mitra
Jan 5, 2018·Statistical Methods in Medical Research·Jiaqi LiNandita Mitra

❮ Previous
Next ❯

Citations

Jan 22, 2021·CJC Open·Jean-Eric TarrideGord Blackhouse

❮ Previous
Next ❯

Software Mentioned

R

Related Concepts

Trending Feeds

COVID-19

Coronaviruses encompass a large family of viruses that cause the common cold as well as more serious diseases, such as the ongoing outbreak of coronavirus disease 2019 (COVID-19; formally known as 2019-nCoV). Coronaviruses can spread from animals to humans; symptoms include fever, cough, shortness of breath, and breathing difficulties; in more severe cases, infection can lead to death. This feed covers recent research on COVID-19.

Blastomycosis

Blastomycosis fungal infections spread through inhaling Blastomyces dermatitidis spores. Discover the latest research on blastomycosis fungal infections here.

Nuclear Pore Complex in ALS/FTD

Alterations in nucleocytoplasmic transport, controlled by the nuclear pore complex, may be involved in the pathomechanism underlying multiple neurodegenerative diseases including Amyotrophic Lateral Sclerosis and Frontotemporal Dementia. Here is the latest research on the nuclear pore complex in ALS and FTD.

Applications of Molecular Barcoding

The concept of molecular barcoding is that each original DNA or RNA molecule is attached to a unique sequence barcode. Sequence reads having different barcodes represent different original molecules, while sequence reads having the same barcode are results of PCR duplication from one original molecule. Discover the latest research on molecular barcoding here.

Chronic Fatigue Syndrome

Chronic fatigue syndrome is a disease characterized by unexplained disabling fatigue; the pathology of which is incompletely understood. Discover the latest research on chronic fatigue syndrome here.

Evolution of Pluripotency

Pluripotency refers to the ability of a cell to develop into three primary germ cell layers of the embryo. This feed focuses on the mechanisms that underlie the evolution of pluripotency. Here is the latest research.

Position Effect Variegation

Position Effect Variagation occurs when a gene is inactivated due to its positioning near heterochromatic regions within a chromosome. Discover the latest research on Position Effect Variagation here.

STING Receptor Agonists

Stimulator of IFN genes (STING) are a group of transmembrane proteins that are involved in the induction of type I interferon that is important in the innate immune response. The stimulation of STING has been an active area of research in the treatment of cancer and infectious diseases. Here is the latest research on STING receptor agonists.

Microbicide

Microbicides are products that can be applied to vaginal or rectal mucosal surfaces with the goal of preventing, or at least significantly reducing, the transmission of sexually transmitted infections. Here is the latest research on microbicides.