Comparative Effectiveness Research Evidence, Medicine, and Policy

For all its costs, flaws, and inequities, American health care is fundamentally rooted in a belief that treatment should be based on solid scientific research. To this end, between 2003 and 2010, three different federal laws were enacted, the most recent being the Affordable Care Act of 2010, that mandated new federal investments in a type of clinical research called comparative effectiveness research (CER) -- research into what works best in medical care.

Comparative Effectiveness Research: Evidence, Medicine, and Policy provides the first complete account of how -- and why -- the federal government decided to make CER an important feature of health reform. Despite earlier legislative uptake of policy proposals on CER, support for federal mandates took dramatic twists and turns, with eventual compromises forged amid failing bipartisan alliances, special interests, and mobilized public opinion.

Based on exhaustive research and first-hand interviews, the authors examine where CER fits in the production of scientific evidence about the benefits and harms of treatments for human diseases and conditions. Their work offers sobering confirmation that contemporary American medical care falls, not surprisingly, well short of the evidence-based ideal.

Comparative Effectiveness Research demonstrates that dealing constructively with the vast uncertainties inherent to medical care requires policies to make the generation of high-quality evidence an inseparable part of routine health care.

Carol M. Ashton, MD, MPH, is John F., Jr. and Carolyn Bookout Professor of Surgical Quality and Outcomes Science at The Methodist Hospital, and Co-Director of the Center for Outcomes Research at The Methodist Hospital Research Institute in Houston, Texas.

Nelda P. Wray, MD, MPH, is Research Scientist, Methodist Institute for Technology, Innovation and Education at The Methodist Hospital, and Co-Director of the Center for Outcomes Research at The Methodist Hospital Research Institute in Houston, Texas.

Introduction

Part I: Evidence

1. The Generation of Evidence in Medicine: Evaluating the Benefits and Harms of Treatment
2. The Use of Evidence: Federal Regulation of Prescription Drugs and Medical Devices
3. The Subversion of Evidence: The Rosiglitazone Story
4. Operate First, Evidence Later: The Special Case of Surgery
5. Doctors and Evidence: Evidence-based Medicine

Part II: Politics and Policy
Introduction to Part II

6. Comparative Effective Research: A New "Silver Bullet" for U.S. Health Care Costs
7. The Legislative Odyssey of Comparative Effectiveness Research: Birth in 2002 to the First Federal Mandate in 2003
8. 2005 to 2007: The Health Services Research Community Stirs, the "B" Word Awakens Policymakers, and the House CHAMP Act Creates Divisions
9. 2008 to 2010: The Baucus-Conrad Bills, the American Recovery and Reinvestment Act, and the Patient Protection and Affordable Care Act
10. The Scope, Financing, and Processes of Federally-mandated Comparative Effectiveness Research and Why They Matter

Part III: Interests
Introduction to Part III

11. Aligning Clinical Practice with Best Evidence of Comparative Effectiveness: Payment Policy for Evidence-based Medical Care
12. Dealing with Uncertainty: Payment Policy for the Generation of Evidence as Part of Clinical Practice

Epilogue