The Pragmatist&Apos;S Guide to Comparative Effectiveness Research

Following an acrimonious health care reform debate involving charges of "death panels," in 2010, Congress explicitly forbade the use of cost-effectiveness analysis in government programs of the Patient Protection and Affordable Care Act. In this context, comparative effectiveness research emerged as an alternative strategy to understand better what works in health care. Put simply, comparative effectiveness research compares the efficacy of two or more diagnostic tests, treatments, or health care delivery methods without any explicit consideration of costs. To economists, the omission of costs from an assessment might seem nonsensical, but we argue that comparative effectiveness research still holds promise. First, it sidesteps one problem facing cost-effectiveness analysis--the widespread political resistance to the idea of using prices in health care. Second, there is little or no evidence on comparative effectiveness for a vast array of treatments: for example, we don't know whether proton-beam therapy, a very expensive treatment for prostate cancer (which requires building a cyclotron and a facility the size of a football field) offers any advantage over conventional approaches. Most drug studies compare new drugs to placebos, rather than "head-to-head" with other drugs on the market, leaving a vacuum as to which drug works best. Finally, the comparative effectiveness research can prove a useful first step even in the absence of cost information if it provides key estimates of treatment effects. After all, such effects are typically expensive to determine and require years or even decades of data. Costs are much easier to measure, and can be appended at a later date as financial Armageddon draws closer.

[1]  J. Skinner,et al.  Is American Health Care Uniquely Inefficient? , 2008, The journal of economic perspectives : a journal of the American Economic Association.

[2]  Katherine Baicker,et al.  Health Care Spending Growth and the Future of U.S. Tax Rates , 2011, Tax Policy and the Economy.

[3]  Harlan M Krumholz,et al.  Responding to an FDA warning--geographic variation in the use of rosiglitazone. , 2010, The New England journal of medicine.

[4]  D. Berman,et al.  Optimal medical therapy with or without PCI for stable coronary disease. , 2007, The New England journal of medicine.

[5]  Milton C Weinstein,et al.  Comparative effectiveness and health care spending--implications for reform. , 2010, The New England journal of medicine.

[6]  P. Bach,et al.  How Medicare could use comparative effectiveness research in deciding on new coverage and reimbursement. , 2010, Health affairs.

[7]  J. Richardson,et al.  Who cares about cost? Does economic analysis impose or reflect social values? , 1995, Health policy.

[8]  Heidi L. Williams,et al.  Estimating Marginal Returns to Medical Care: Evidence from At-Risk Newborns , 2008, The quarterly journal of economics.

[9]  Alona Muzikansky,et al.  Early palliative care for patients with metastatic non-small-cell lung cancer. , 2010, The New England journal of medicine.

[10]  A. Garber,et al.  Economic foundations of cost-effectiveness analysis. , 1997, Journal of health economics.

[11]  Ara Darzi,et al.  Better: A surgeon’s notes on performance , 2007 .

[12]  A. Gouveia Your Money or Your Life: Strong Medicine for America's Health Care System , 2006 .

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

[14]  J. Skinner,et al.  The Efficiency of Medicare , 2001 .

[15]  Y. Schenker,et al.  Patients' and cardiologists' perceptions of the benefits of percutaneous coronary intervention for stable coronary disease. , 2011, Annals of internal medicine.

[16]  L. Baker,et al.  Physician practice size and variations in treatments and outcomes: evidence from Medicare patients with AMI. , 2007, Health affairs.

[17]  Joseph P. Newhouse,et al.  Does More Intensive Treatment of Acute Myocardial Infarction in the Elderly Reduce Mortality? Analysis Using Instrumental Variables , 1995 .

[18]  D C Hadorn,et al.  Setting health care priorities in Oregon. Cost-effectiveness meets the rule of rescue. , 1991, JAMA.

[19]  A. Garber,et al.  The potential impact of comparative effectiveness research on U.S. Health Care expenditures , 2010, Demography.

[20]  A. Chandra,et al.  Uncomfortable arithmetic--whom to cover versus what to cover. , 2010, The New England journal of medicine.

[21]  Fedde Scheele,et al.  Better, a surgeon’s notes on performance , 2012, Perspectives on Medical Education.

[22]  David O. Meltzer,et al.  Accounting for future costs in medical cost-effectiveness analysis. , 1997 .

[23]  J. Ferguson,et al.  Court-ordered reimbursement for unproven medical technology. Circumventing technology assessment. , 1993, JAMA.

[24]  J. Skinner,et al.  Technology Growth and Expenditure Growth in Health Care , 2011, National Bureau of Economic Research bulletin on aging and health.

[25]  Edward C. Norton,et al.  Health Care Expenditures , 2009 .

[26]  C Fraser,et al.  Laparoscopic surgery for inguinal hernia repair: systematic review of effectiveness and economic evaluation. , 2005, Health technology assessment.

[27]  E S Fisher,et al.  Variation in carotid endarterectomy mortality in the Medicare population: trial hospitals, volume, and patient characteristics. , 1998, JAMA.

[28]  Shoshannah A. Pearlman The Patient Protection and Affordable Care Act , 2013, Journal of the American Psychiatric Nurses Association.

[29]  G. Hawker,et al.  Knee arthroscopy in England and Ontario: patterns of use, changes over time, and relationship to total knee replacement. , 2008, The Journal of bone and joint surgery. American volume.

[30]  George Tomlinson,et al.  Randomized, controlled trial of an interactive videodisc decision aid for patients with ischemic heart disease , 2000, Journal of General Internal Medicine.

[31]  Philip S. Wang,et al.  Medicaid's prior authorization program and access to atypical antipsychotic medications. , 2007, Health affairs.

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

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

[34]  M. Mcclellan,et al.  Is technological change in medicine worth it? , 2001, Health affairs.

[35]  R. W. Hansen,et al.  The price of innovation: new estimates of drug development costs. , 2003, Journal of health economics.

[36]  J. Newhouse Assessing health reform's impact on four key groups of Americans. , 2010, Health affairs.

[37]  B. Kisloff Implications of regional differences in spending. , 2004, Annals of internal medicine.

[38]  Amitabh Chandra,et al.  Productivity Spillovers in Health Care: Evidence from the Treatment of Heart Attacks , 2007, Journal of Political Economy.

[39]  D. Meltzer,et al.  The economics of comparative effectiveness research , 2010, Neurology.

[40]  Rajesh K. Gupta,et al.  Limited good and limited vision: multidrug-resistant tuberculosis and global health policy. , 2005, Social science & medicine.

[41]  J. Skinner,et al.  Is technological change in medicine always worth it? The case of acute myocardial infarction. , 2006, Health affairs.

[42]  N Engl,et al.  A controlled trial of arthroscopic surgery for osteoarthritis of the knee , 2005 .

[43]  K. O'malley,et al.  A controlled trial of arthroscopic surgery for osteoarthritis of the knee. , 2002, The New England journal of medicine.

[44]  R. Thaler,et al.  Nudge: Improving Decisions About Health, Wealth, and Happiness , 2008 .

[45]  C. O. Kennedy A controlled trial , 1971, British Homeopathic Journal.

[46]  B. Robberstad QALYs vs DALYs vs LYs gained: What are the differences, and what difference do they make for health care priority setting? , 2005 .

[47]  H. Sox,et al.  The role of costs in comparative effectiveness research. , 2010, Health affairs.

[48]  Stanley E. Kaufman,et al.  Effect of PCI on quality of life in patients with stable coronary disease. , 2008, The New England journal of medicine.

[49]  T. Schacker,et al.  Clinical and Epidemiologic Features of Primary HIV Infection , 1996, Annals of Internal Medicine.

[50]  Kevin M. Murphy,et al.  The Value of Health and Longevity , 2005, Journal of Political Economy.

[51]  D. Nathan Time for Clinically Relevant Comparative Effectiveness Studies in Type 2 Diabetes , 2011, Annals of Internal Medicine.

[52]  D. Blumenthal,et al.  Patient Protection and Affordable Care Act , 2010 .

[53]  M. Pincus 'Health care: Who knows best?' , 2010 .

[54]  P. Royle,et al.  Clinical effectiveness and cost-effectiveness of immediate angioplasty for acute myocardial infarction: systematic review and economic evaluation. , 2005, Health technology assessment.

[55]  D. Brenner,et al.  Computed tomography--an increasing source of radiation exposure. , 2007, The New England journal of medicine.

[56]  T. Ryan,et al.  Relation between operator and hospital volume and outcomes following percutaneous coronary interventions in the era of the coronary stent. , 2000, JAMA.

[57]  Van V. Brantner,et al.  Estimating the cost of new drug development: is it really 802 million dollars? , 2006, Health affairs.

[58]  M. Maguire,et al.  Identifying and eliminating the roadblocks to comparative-effectiveness research. , 2010, The New England journal of medicine.

[59]  S. Zenios,et al.  An empiric estimate of the value of life: updating the renal dialysis cost-effectiveness standard. , 2009, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[60]  Elliott S Fisher,et al.  Discretionary decision making by primary care physicians and the cost of U.S. Health care. , 2008, Health affairs.

[61]  J. Concato,et al.  Randomized, controlled trials, observational studies, and the hierarchy of research designs. , 2000, The New England journal of medicine.

[62]  Joseph S. Pliskin,et al.  Health Economic Evaluations: The Special Case of End-Stage Renal Disease Treatment , 2002, Medical decision making : an international journal of the Society for Medical Decision Making.