Economics of Individualization in Comparative Effectiveness Research and a Basis for a Patient-Centered Health Care

The United States aspires to use information from comparative effectiveness research (CER) to reduce waste and contain costs without instituting a formal rationing mechanism or compromising patient or physician autonomy with regard to treatment choices. With such ambitious goals, traditional combinations of research designs and analytical methods used in CER may lead to disappointing results. In this paper, I study how alternate regimes of comparative effectiveness information help shape the marginal benefits (demand) curve in the population and how such perceived demand curves impact decision-making at the individual patient level and welfare at the societal level. I highlight the need to individualize comparative effectiveness research in order to generate the true (normative) demand curve for treatments. I discuss methodological principles that guide research designs for such studies. Using an example of the comparative effect of substance abuse treatments on crime, I use novel econometric methods to salvage individualized information from an existing dataset.

[1]  Anirban Basu,et al.  Value of Information on Preference Heterogeneity and Individualized Care , 2007, Medical decision making : an international journal of the Society for Medical Decision Making.

[2]  J. Stoner,et al.  Cross-Over Trials in Clinical Research (2nd ed.) (Book) , 2004 .

[3]  T. Philipson,et al.  Endogenous Cost-Effectiveness Analysis in Health Care Technology Adoption , 2009 .

[4]  Costas Meghir,et al.  Identification of Treatment Effects Using Control Functions in Models with Continuous, Endogenous Treatment and Heterogeneous Effects , 2008 .

[5]  Fahad Khalil,et al.  Third party purchasing of health services: patient choice and agency. , 2005, Journal of health economics.

[6]  Pravin K. Trivedi,et al.  Bayesian analysis of a self-selection model with multiple outcomes using simulation-based estimation: an application to the demand for healthcare , 2003 .

[7]  S. Murphy,et al.  An experimental design for the development of adaptive treatment strategies , 2005, Statistics in medicine.

[8]  K. Arrow,et al.  Uncertainty and the Welfare Economics of Medical Care (American Economic Review, 1963) , 2001, Uncertain Times.

[9]  P. Holland Statistics and Causal Inference , 1985 .

[10]  A. Fendrick,et al.  A benefit-based copay for prescription drugs: patient contribution based on total benefits, not drug acquisition cost. , 2001, The American journal of managed care.

[11]  M. Pauly Adverse Selection and Moral Hazard: Implications for Health Insurance Markets , 2008 .

[12]  James J. Heckman,et al.  Longitudinal Analysis of Labor Market Data , 1985 .

[13]  D. Rubin Estimating causal effects of treatments in randomized and nonrandomized studies. , 1974 .

[14]  S. Tunis,et al.  Does comparative-effectiveness research threaten personalized medicine? , 2009, The New England journal of medicine.

[15]  H. James VARIETIES OF SELECTION BIAS , 1990 .

[16]  D. Rubin,et al.  Inference from Iterative Simulation Using Multiple Sequences , 1992 .

[17]  D. Simpson,et al.  A national 5-year follow-up of treatment outcomes for cocaine dependence. , 2002, Archives of general psychiatry.

[18]  H. Pollack,et al.  Social costs of robbery and the cost-effectiveness of substance abuse treatment. , 2008, Health economics.

[19]  R. Lalonde Evaluating the Econometric Evaluations of Training Programs with Experimental Data , 1984 .

[20]  D. Stryer,et al.  Practical clinical trials: increasing the value of clinical research for decision making in clinical and health policy. , 2003, JAMA.

[21]  A A Stinnett,et al.  Mathematical programming for the efficient allocation of health care resources. , 1996, Journal of health economics.

[22]  Lola Butcher,et al.  Value-based insurance design. , 2009, Biotechnology healthcare.

[23]  IN REPLY TO PATRICK M. FLYNN AND COLLEAGUES , 2001 .

[24]  Nestor E Terleckyj,et al.  Household Production and Consumption , 1976 .

[25]  David J. Vanness,et al.  Perspectives on Mean-based Evaluation of Health Care , 2006 .

[26]  Stephen Senn,et al.  Cross-over trials in clinical research , 1993 .

[27]  J. Heckman Dummy Endogenous Variables in a Simultaneous Equation System , 1977 .

[28]  R. Hubbard,et al.  COMMENT ON “NONRESPONSE AND SELECTION BIAS IN TREATMENT FOLLOW-UP STUDIES” , 2001, Substance use & misuse.

[29]  Charles F. Manski,et al.  Evaluating Welfare and Training Programs. , 1994 .

[30]  W. Manning,et al.  Health insurance: the tradeoff between risk pooling and moral hazard. , 1996, Journal of health economics.

[31]  Donald B. Rubin,et al.  Bayesian Inference for Causal Effects: The Role of Randomization , 1978 .

[32]  M. Pauly,et al.  Benign moral hazard and the cost-effectiveness analysis of insurance coverage. , 1990, Journal of health economics.

[33]  J. Heckman,et al.  Longitudinal Analysis of Labor Market Data: Alternative methods for evaluating the impact of interventions , 1985 .

[34]  Donald A. Berry,et al.  Optimal adaptive randomized designs for clinical trials , 2007 .

[35]  Barack Obama,et al.  PLAN TO LOWER HEALTH CARE COSTS AND ENSURE AFFORDABLE, ACCESSIBLE HEALTH COVERAGE FOR ALL , 2008 .

[36]  A Thomas McLellan,et al.  Cost-effectiveness analysis of addiction treatment: paradoxes of multiple outcomes. , 2004, Drug and alcohol dependence.

[37]  The Economics of Comparative Effectiveness Studies , 2012, PharmacoEconomics.

[38]  Richard H. Thaler,et al.  The Value of Saving a Life: Evidence from the Labor Market , 1976 .

[39]  Christy K. Scott,et al.  Out of touch or on the money: Do the clinical objectives of addiction treatment coincide with economic evaluation results? , 2004, Journal of substance abuse treatment.

[40]  C. Manski MAXIMUM SCORE ESTIMATION OF THE STOCHASTIC UTILITY MODEL OF CHOICE , 1975 .

[41]  M. Pauly,et al.  Moral hazard in insurance, value-based cost sharing, and the benefits of blissful ignorance. , 2008, Journal of health economics.

[42]  Jonathan Cylus,et al.  Health spending projections through 2018: recession effects add uncertainty to the outlook. , 2009, Health affairs.

[43]  Mark V. Pauly,et al.  Readings in the economics of contract law: The economics of moral hazard: comment , 1982 .

[44]  Andrew M. Jones The Elgar Companion to Health Economics , 2007 .

[45]  Rajeev Dehejia,et al.  Program Evaluation as a Decision Problem , 1999 .

[46]  J J Heckman,et al.  Local instrumental variables and latent variable models for identifying and bounding treatment effects. , 1999, Proceedings of the National Academy of Sciences of the United States of America.

[47]  A. Roy Some thoughts on the distribution of earnings , 1951 .

[48]  D. Gerstein,et al.  Nonresponse and Selection Bias in Treatment Follow-up Studies , 2000, Substance use & misuse.

[49]  Clarence Balden Randall,et al.  Report to the President and the Congress , 1954 .

[50]  D. McFadden Conditional logit analysis of qualitative choice behavior , 1972 .

[51]  M. French,et al.  The relative contribution of outcome domains in the total economic benefit of addiction interventions: a review of first findings. , 2003, Addiction.

[52]  A A Stinnett,et al.  Net Health Benefits , 1998, Medical decision making : an international journal of the Society for Medical Decision Making.

[53]  E. Duflo,et al.  How Much Should We Trust Differences-in-Differences Estimates? , 2001 .

[54]  Andreas P. Gambrellac Technological change and the growth of health care spending , 2010 .

[55]  J. Lieberman,et al.  The National Institute of Mental Health Clinical Antipsychotic Trials of Intervention Effectiveness (CATIE) project: schizophrenia trial design and protocol development. , 2003, Schizophrenia bulletin.

[56]  Jeffrey A. Smith,et al.  Evaluating the Welfare State , 1998 .

[57]  K. Arrow,et al.  EXISTENCE OF AN EQUILIBRIUM FOR A COMPETITIVE ECONOMY , 1954 .

[58]  A. Basu Individualization at the Heart of Comparative Effectiveness Research: The Time for i-CER Has Come , 2009, Medical decision making : an international journal of the Society for Medical Decision Making.

[59]  R. Quandt A New Approach to Estimating Switching Regressions , 1972 .

[60]  G. Zarkin,et al.  The effect of treatment completion and length of stay on employment and crime in outpatient drug-free treatment. , 2002, Journal of substance abuse treatment.

[61]  R. Quandt The Estimation of the Parameters of a Linear Regression System Obeying Two Separate Regimes , 1958 .

[62]  A T McLellan,et al.  Evaluating the effectiveness of addiction treatments: reasonable expectations, appropriate comparisons. , 1996, The Milbank quarterly.

[63]  John C. Hershey,et al.  Carrier Screening for Cystic Fibrosis , 1998, Medical decision making : an international journal of the Society for Medical Decision Making.

[64]  J. I The Design of Experiments , 1936, Nature.

[65]  Mark Sculpher,et al.  Subgroups and Heterogeneity in Cost-Effectiveness Analysis , 2012, PharmacoEconomics.

[66]  M. French,et al.  Benefit-cost analysis of addiction treatment: methodological guidelines and empirical application using the DATCAP and ASI. , 2002, Health services research.

[67]  James J. Heckman,et al.  Alternative methods for evaluating the impact of interventions: An overview , 1985 .