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Estimating Decision-Relevant Comparative Effects Using Instrumental Variables

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Abstract

Instrumental variables methods (IV) are widely used in the health economics literature to adjust for hidden selection biases in observational studies when estimating treatment effects. Less attention has been paid in the applied literature to the proper use of IVs if treatment effects are heterogeneous across subjects. Such a heterogeneity in effects becomes an issue for IV estimators when individuals’ self-selected choices of treatments are correlated with expected idiosyncratic gains or losses from treatments. We present an overview of the challenges that arise with IV estimators in the presence of effect heterogeneity and self-selection and compare conventional IV analysis with alternative approaches that use IVs to directly address these challenges. Using a Medicare sample of clinically localized breast cancer patients, we study the impact of breast-conserving surgery and radiation with mastectomy on 3-year survival rates. Our results reveal the traditional IV results may have masked important heterogeneity in treatment effects. In the context of these results, we discuss the advantages and limitations of conventional and alternative IV methods in estimating mean treatment-effect parameters, the role of heterogeneity in comparative effectiveness research and the implications for diffusion of technology.

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References

  1. Amemiya T (1974) The non-linear two-stage least squares estimator. J Econom 105–110

  2. Angrist J, Imbens G, Rubin D (1996) Identification of causal effects using instrumental variables. J Am Stat Assoc 91:444–455

    Article  MATH  Google Scholar 

  3. Bang H, Robins JM (2005) Doubly robust estimation in missing data and causal inference models. Biometrics 61:962–972

    Article  MathSciNet  MATH  Google Scholar 

  4. Basu A (2009) Individualization at the heart of comparative effectiveness research: the time for i-CER has come. Med Decis Mak 29(6):N9–N11

    Article  Google Scholar 

  5. Basu A, Heckman J, Navarro-Lozano S, Urzua S (2007) Use of instrumental variables in the presence of heterogeneity and self-selection: an application to treatments of breast cancer patients. Health Econ 16(11):1133–1157

    Article  Google Scholar 

  6. Basu A, Philipson T (2010) Impact of comparative effectiveness research on health and healthcare spending. NBER working paper No. w15633

  7. Björklund A, Moffitt R (1987) The estimation of wage gains and welfare gains in self-selection. Rev Econ Stat 69(1):42–49

    Article  Google Scholar 

  8. Blundell R, Powell J (2003) Endogeneity in nonparametric and semiparametric regression models. In: Hansen L, Dewatripont M, Turnovsky SJ (eds) Advances in economics and econometrics. Cambridge University Press, Cambridge, pp 312–357

    Google Scholar 

  9. Brooks JM, Chrischilles E, Scott S, Chen-Hardee S (2003) Was lumpectomy underutilized for early stage breast cancer? Instrumental variables evidence for stage II patients from Iowa. Health Serv Res 38(6):1385–1402. Part I

    Article  Google Scholar 

  10. Earle CE, Tsai JS, Gelber RD, Weinstein MC, Neumann PJ, Weeks JC (2001) Effectiveness of chemotherapy for advanced lung cancer in the elderly: instrument variable and propensity analysis. J Clin Oncol 19(4):1064–1070

    Google Scholar 

  11. Fisher B, Bauer M, Margolese R et al. (1985) Five-year results of a randomized clinical trial comparing total mastectomy and segmental mastectomy with or without radiation in the treatment of breast cancer. N Engl J Med 312:665–673

    Article  Google Scholar 

  12. Franklin BA (2008) Lessons learned from the COURAGE trial: generalizability, limitations, and implications. Prev Cardiol 11(1):5–7

    Article  Google Scholar 

  13. Hadley J, Mitchell JM, Mandelblatt J (1992) Medicare fees and small area variations in the treatment of localized breast cancer. N Engl J Med 52:334–360

    Google Scholar 

  14. Hadley J, Polsky D, Mandelblatt JS, Mitchell JM, Weeks JC, Wang Q, Hwang Y-T, OPTIONS Research Team (2003) An exploratory instrumental variable analysis of the outcomes of localized breast cancer treatments in a Medicare population. Health Econ 12:171–186

    Article  Google Scholar 

  15. Heckman JJ (1996) Comments on angrist, imbens, and rubin: identification of causal effects using instrumental variables. J Am Stat Assoc 91:434

    Article  Google Scholar 

  16. Heckman J (1997) Instrumental variables: a study of implicit behavioral assumptions used in making program evaluations. J Hum Resour 32(3):441–462

    Article  MathSciNet  Google Scholar 

  17. Heckman J (2001) Micro data, heterogeneity, and the evaluation of public policy: Nobel Lecture. J Polit Econ 109(4):673–748

    Article  MathSciNet  Google Scholar 

  18. Heckman J, Honore B (1990) The empirical content of the Roy model. Econometrica 58:1121–1149

    Article  Google Scholar 

  19. Heckman JJ, Vytlacil EJ (1999) Local instrumental variables and latent variable models for identifying and bounding treatment effects. Proc Natl Acad Sci USA 96(8):4730–4734

    Article  MathSciNet  Google Scholar 

  20. Heckman J, Vytlacil E (2005) Econometric evaluation of social programs. In: Heckman J, Leamer E (eds) Handbook of econometrics, vol 6. Elsevier, Amsterdam

    Google Scholar 

  21. Heckman J, Vytlacil E (2005) Structural equations, treatment effects and econometric policy evaluation. Econometrica 73(3):669–738

    Article  MathSciNet  MATH  Google Scholar 

  22. Heckman J, Vytlacil EJ (2007) Econometric evaluation of social programs, part II: using the marginal treatment effect to organize alternative econometric estimators to evaluate social programs, and to forecast their effects in new environments. In: Heckman J, Leamer E (eds) Handbook of econometrics, vol 6B. Elsevier, Amsterdam

    Google Scholar 

  23. Heckman JJ, Urzua S, Vytlacil E (2006) Understanding instrumental variables in models with essential heterogeneity. Rev Econ Stat 88(3):389–432

    Article  Google Scholar 

  24. Hernan MA, Alonso A, Logan R et al. (2008) Observational studies analyzed like randomized experiments: an application to postmenopausal hormone therapy and coronary heart disease. Epidemiology 19:766–779

    Article  Google Scholar 

  25. Humphrey LL, Chan BK, Sox HC (2002) Postmenopausal hormone replacement therapy and the primary prevention of cardiovascular disease. Ann Intern Med 137:273–84

    Google Scholar 

  26. Imbens G, Angrist J (1994) Identification and estimation of local average treatment effects. Econometrica 62(2):467–475

    Article  MATH  Google Scholar 

  27. McClellan M, McNeil B, Newhouse J (1994) Does more intensive treatment of acute myocardial infarction reduce mortality? JAMA 272(11):859–866

    Article  Google Scholar 

  28. McFadden D (1973) Conditional logit analysis of qualitative choice behavior. In: Zarembka P (ed) Frontiers in econometrics. Academic Press, New York

    Google Scholar 

  29. McFadden D (1981) Econometric models of probabilistic choice. In: Manski CF, McFadden D (eds) Structural analysis of discrete data with econometric applications. MIT Press, Cambridge

    Google Scholar 

  30. Mullahy J (1997) Instrumental variable estimation of count data models: applications to models of cigarette smoking behavior. Rev Econ Stat 79:586–593

    Article  Google Scholar 

  31. Quandt RE (1972) A new approach to estimating switching regression. J Am Stat Assoc 67(338):306–310

    Article  MATH  Google Scholar 

  32. Quandt RE (1958) The estimation of parameters of a linear regression system obeying two separate regimes. J Am Stat Assoc 53(284):873–880

    Article  MathSciNet  MATH  Google Scholar 

  33. Polsky D, Mandelblatt JS, Weeks J, Venditti L, Hwang YT, Glick HA, Hadley J, Schulman KA (2003) Economic evaluation of breast cancer treatment: considering the value of patient choice. J Clin Oncol 21:1139–1146

    Article  Google Scholar 

  34. Robins JM, Rotnitzky A, Zhao LP (1995) Analysis of semiparametric regression models for repeated outcomes in the presence of missing data. J Am Stat Assoc 90:106–121

    Article  MathSciNet  MATH  Google Scholar 

  35. Rossouw JE, Anderson GL, Prentice RL et al. (2002) Risks and benefits of estrogen plus progestin in healthy postmenopausal women: principal results from the women’s health initiative randomized controlled trial. JAMA 288:321–33

    Article  Google Scholar 

  36. Rosenbaum PR, Rubin D (1983) The central role of the propensity score in observational studies for causal effects. Biometrika 70:41–55

    Article  MathSciNet  MATH  Google Scholar 

  37. Roy AD (1951) Some thoughts on the distribution of earnings. Oxf Econ Pap 3:135–146

    Google Scholar 

  38. Rubin D (1973) The use of matched sampling and regression adjustment to remove bias in observational studies. Biometrics 29:185–203

    Article  Google Scholar 

  39. Rubin DB (1997) Estimating causal effects from large data sets using propensity scores. Ann Intern Med 127:757–763

    Google Scholar 

  40. Scharfstein DO, Rotnitzky A, Robins JM (1994) Adjusting for nonignorable drop-out using semiparametric nonresponse models. J Am Stat Assoc 94:1096–1120 (with rejoinder, 1135–1146)

    Article  MathSciNet  Google Scholar 

  41. Steering Committee on Clinical Practice Guidelines for the Care and Treatment of Breast Cancer (1998) Mastectomy or lumpectomy? The choice of operation for clinical stages I and II breast cancer. Can Med Assoc J 158(Suppl. 3):S15–S21

    Google Scholar 

  42. Stock JH, Trebbi F (2003) Who invented instrumental variable regression? J Econ Perspect 17(3):177–194

    Article  Google Scholar 

  43. Stukel TA, Fisher ES, Wennberg DE, Alter DA, Gottlieb DJ, Vermeulen MJ (2007) Analysis of observational studies in the presence of treatment selection bias: effects of invasive cardiac management on AMI survival using propensity score and instrumental variable methods. JAMA 297:278–285

    Article  Google Scholar 

  44. Sturmer T, Schneeweiss S, Rothman KJ, Avorn J, Glynn RJ (2007) Performance of propensity score calibration—a simulation study. Am J Epidemiol 165:1110–1118

    Article  Google Scholar 

  45. Terza JV, Basu A, Rathouz PJ (2008) Two-stage residual inclusion estimation: addressing endogeneity in health econometric modeling. J Health Econ 27(3):531–543

    Article  Google Scholar 

  46. Vanness DJ, Mullahy J (2006) Perspectives on mean-based evaluation of health care. In: Jones A (ed) The Elgar companion to health economics. Edward Elgar Publishing, Cheltenham

    Google Scholar 

  47. Virnig BA et al (2007) Increased use of breast-conserving surgery: preferred treatment or failure to provide adequate local therapy? Breast Cancer Res Treat 106 (Supp 1): Abstract 4065

    Google Scholar 

  48. Yitzhaki S (1989) On using linear regression in welfare economics. Working paper 217, Department of Economics, Hebrew University

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Basu, A. Estimating Decision-Relevant Comparative Effects Using Instrumental Variables. Stat Biosci 3, 6–27 (2011). https://doi.org/10.1007/s12561-011-9033-6

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