Thursday, June 18, 2009

Political Rhetoric about Comparative Effectiveness Heats Up

There has been a lot of proposed legislation about comparative effectiveness in this congressional term, and I just ran across an interesting story that helps to understand the political forces that are opposing comparative effectiveness research. It is:

Rationing of health care is one of those issues that strikes fear in people, an I think there has been a lot of misunderstanding about it. Medicare and insurance companies make decisions every day about whether a particular medical treatment will be covered, and this is rationing (even though it is not visible). Comparative effectiveness is simply a way to help decision makers (including those who make coverage decisions) apply the best evidence to their decisions. It really will help protect us all from the bad effects of rationing.

I find it curious why rationing is trotted out as a supposed indictment of comparative effectiveness. It makes more sense to think of comparative effectiveness as a path to better and more efficient health care. It will forestall the need for uninformed rationing.

Reading About Comparative Effectiveness

Public service organizations that have an interest in health care are paying more attention to comparative effectiveness research. Some are even starting to put out publications that serve as good starting points for learning about comparative effectiveness. Fortunately, these publications generally are available online. For example, the US Chamber of Commerce published a "toolkit" on comparative effectiveness last winter. Their toolkit has a readable and concise background section and also includes links to several resources on comparative effectiveness. I recommend it, because it gives some good examples about how comparative effectiveness information can be applied to real decisions made by clinicians and patients. It is available here:

The Friends of Cancer Research (FOCR) is an advocacy organization that lobbies for increased federal funding for cancer research. It recently released a booklet on comparative effectiveness:

This publication places emphasis on an issue that frequently comes up (and is often responsible for skepticism by clinicians about comparative effectiveness). The issue is that there is substantial individual variation among people with a particular disease. By this viewpoint, clinicians need to consider carefully each patient's characteristics and individualize treatment for that person. Because comparative effectiveness measures the "average" response to comparative treatments, some may argue that it does not account for this patient variation. To its credit, the FOCR publication argues gives a few examples about research that provides insight into the sources of patient variation. The FOCR booklet argues that the spectrum of comparative effectiveness research needs to include studies that aim to discover the important patient factors that affect response to a treatment. I think this is a very reasonable point of view. In any case, though, be careful about throwing out the baby with the bathwater. It is not appropriate to dismiss the findings of comparative effectiveness research altogether, just because that research may still be limited in elucidating the sources of patient variation.

Friday, June 12, 2009

SMDM Weighs in on Comparative Effectiveness and Costs of Health Care

In a previous post I mentioned the Society for Medical Decision Making (SMDM). I need to declare a conflict of interest here. I am a member of SMDM and faithfully pay my dues every year.

SMDM is a scholarly society whose members mostly are university faculty members. It puts on a high quality scientific meeting every October and publishes a scientific journal (titled Medical Decision Making). SMDM serves as a network for scientists who are interested in how people make medical decisions. Thus, its journal and annual meeting have long been a source of information on cost effectiveness analyses. These studies use existing data to model the relationship between costs and the benefits attained from medical treatments. Cost effectiveness studies are relevant to comparative effectiveness, and cost effectiveness analyses usually compare two or more alternative treatments.

SMDM wants to influence decisions that the federal government makes about funding comparative effectiveness research, so today it put out a position paper that it is sending out to members of Congress. The position paper is available here:

SMDM calls it a Background Paper, but the paper serves to advocate for providing more federal funding for cost effectiveness analyses. The Background Paper is not too long, and it is a pretty good introductory review of comparative effectiveness and its relationship to cost analyses. When you read it, I think you'll also see that it does a pretty good job of tooting SMDM's horn.

Wednesday, June 10, 2009

Opinion Leaders Discuss Whether Comparative Effectiveness Research Should Include the Costs of Treatments

In the 6/4/09 post on this blog I mentioned the newly created Federal Coordinating Council for Comparative Effectiveness. The coordinating council held a "listening session" in Washington, DC, today. In a listening session, officials of various organizations are invited to make 3 minute presentations to the council, and council members can follow-up with questions posed to the presenters. The listening session is available to view at the following site:

The session is fairly interesting, in that it is a broad cross-section of opinion in the medical community. Most of the presenters are representatives of large organizations, and many are from medical schools. I particularly recommend finding the presentation by Mark Roberts, MD (about half-way through the recording). You can recognize him on the video, because I think he was the only presenter with a beard. Dr. Roberts is Professor of Medicine at the University of Pittsburgh and President of the Society for Medical Decision Making (a scientific society with few ties to industry).

By way of background, the basic premise of comparative effectiveness is that alternative treatments can be compared by examining two categories of outcomes:

Effectiveness: the amount of improvement in a disease among people who use a particular treatment. This can be measured by estimates of health status (severity of symptoms or ability to perform normal activities) and/or by reduction or delay of serious events (such as length of time before death for such diseases as cancers).

Harms: the rate of unexpected events caused by a treatment, such as allergic reactions 9for drugs), new infections (for surgical procedures), or events that do not normally occur (such as bleeding).

In this approach, the cost of a treatment is not considered. A treatment would be preferred over its competitor if its effectiveness were better and its harms were less frequent. However, sometimes, it is not so simple. What if both effectiveness and harms were higher for drug A than for drug B? It is difficult to say whether either drug A or drug B would be preferred. Dr. Roberts made the argument that it is very helpful to look at costs and that this makes a lot of sense when differences in effectiveness and harms between two comparison treatments are small. This approach is known as cost effectiveness studies, and there is a well established scientific method for performing such analyses.

Nevertheless, the use of cost in comparative effectiveness studies has been a lightening rod for controversy. One of the other presenters in today's listening session was James Fasules, a pediatric cardiologist and Senior Vice President of the American College of Cardiology. Dr. Fasules argued that cost considerations would bias comparative effectiveness research and should not be included in the discussion. For yet another point of view, Consumers Union, the giant consumer advocacy organization, has sought to get involved in the debate. See their position paper that argues that comparative effectiveness can reduce the costs of health care (an argument used for including the comparative effectiveness initiative in last winter's federal economic stimulus package).

Getting back to today's listening session, I recommend that you also view the presentation by Dr. Harry Selker, which is about 3/4 through the video (in the third group of presenters). Dr. Selker is a Professor of Medicine at Tufts School of Medicine in Boston. He spoke as a representative of the Society of General Internal Medicine. Dr. Selker made some very good points about governance of federal funding for comparative effectiveness research.

Here is the complete list of speakers. Since each spoke only 3 minutes, it is failry easy to navigate through the video.

Panel 1
Larry Cohen
Executive Director
Prevention Institute

Diane Dorman
Vice President
National Organization for Rare Disorders

James Fasules
Senior Vice President
American College of Cardiology

Frederick Grover
Chair, Council on Quality, Research, and Patient Safety
The Society of Thoracic Surgeons

David Juba
Senior Research Analyst
Fundamental Clinical Consulting, LLC

Joseph Kanter
Joseph H. Kanter Family Foundation & The Health Legacy Partnership

Beth Kosiak
Associate Executive Director
American Urological Association

Diana Zuckerman
National Research Center for Women & Families

Panel 2
Ted Buckley
Director of Economic Policy
Biotechnology Industry Organization

John Cuddeback
Chief Medical Informatics Officer
Anceta - AMGA's Collaborative Data Warehouse

Bill Fox
Executive Director
The National Center for Patient Interactive Research

Martyn Howgill
Executive Director
Institute for Health Technology Studies

Polly Pittman
Executive Vice President

Mark Roberts
President and Professor of Medicine
Society for Medical Decision Making

Patricia Salber
Chief Medical Officer and Senior Vice President
Universal American Corporation

Deneen Vojta
Senior Vice President
UnitedHealth Group

Panel 3
Brendan Carr
Assistant Professor
American College of Emergency Physicians & Society for Academic Emergency Medicine

Matthew Hoffman
Director of Ob/GynResearch
The Eunice Kennedy Shriver NICHD Maternal Fetal Units Network

Jeffrey Lerner
President and Chief
ECRI Institute

Mark Pilley
Medical Director
RS Medical

Harry Selker
Dean, Clinical and Translational Science Institute, Tufts University, Chair Society of General Internal Medicine Health Policy Committee on Research, Society of General Internal Medicine

Eduardo Siguel

Ronald Stewart
Board Chairman
National Trauma Institute

Grace-Marie Turner
Galen Institute

Friday, June 5, 2009

What Other People are Saying About Comparative Effectiveness

As mentioned in yesterday's post, comparative effectiveness (CE) has become a an important issue in the healthcare reform discussions. This is because it provides a method to evaluate the value of treatments and procedures. As clinicians evaluate CE information, they can make better choices and possibly make care more efficient. CE does not tell doctors what to do, it just gives them better information.

This is a theme that it is is important to remember. CE is a process that has value if it leads to better decisions. Better decisions (by both doctors and patients) lead to better health. So, the question is: Do we have a system of CE research that flows through to better decisions. The federal government does have such a program, in the Agency for Healthcare Research and Quality (AHRQ). Take a look at the following statement by Dr. Carolyn Clancy, who is the Director of AHRQ. She describes the approach AHRQ has taken to pursue a CE research agenda.

Some people have questioned whether the federal government, including AHRQ and the NIH, is doing a good job creating and managing a CE research program. Both last year and again this year, members of Congress have proposed new laws that would change how federal funding for CE research would be managed. Take a look at this blog entry for a discussion of these proposals.

If you would like a more detailed discussion of the research infrastructure for CE, the American Colleg of Physicians put out a position paper last year.

Thursday, June 4, 2009

Welcome to the Comparative Effectiveness Blog

About this time last year (2008), most people probably had not heard the term comparative effectiveness, but a lot has happened since then. In the summer of 2008, bills promoting comparative effectiveness were introduced in both the U.S. Senate and House. Even though these bills never came to a floor vote, the publicity surrounding the bills expanded the discussion. With the election of a new president and large Democratic majorities in both houses last November, the movement for national reform of the healthcare delivery system gained momentum. As part of the stimulus package passed by Congress this winter, $1.1 billion of new federal spending was committed to comparative effectiveness research. A new Federal Coordinating Council for Comparative Effectiveness Research also was created. People began to realize that comparative effectiveness is something to pay attention to.

Comparative effectiveness is a process for evaluating medical evidence as it applies to the comparison of treatments or other medical interventions. The above-mentioned Federal Coordinating Council just published the "official definition" which is:

"Comparative effectiveness research is the conduct and synthesis of systematic research comparing different interventions and strategies to prevent, diagnose, treat and monitor health conditions. The purpose of this research is to inform patients, providers, and decision-makers, responding to their expressed needs, about which interventions are most effective for which patients under specific circumstances. To provide this information, comparative effectiveness research must assess a comprehensive array of health-related outcomes for diverse patient populations. Defined interventions compared may include medications, procedures, medical and assistive devices and technologies, behavioral change strategies, and delivery system interventions. This research necessitates the development, expansion, and use of a variety of data sources and methods to assess comparative effectiveness. "

This definition is broad and complicated, but it has some important features. First, it emphasizes that the purpose of comparative effectiveness research is to help people make decisions. Second, it draws attention to the fact that not all people with a particular disease are the same. A particular intervention may be best for certain people, while a different intervention may be best for others. One way the definition could be improved is to expand on what is meant by outcomes. Most medical treatments provide benefits by reducing the severity of (and sometimes curing) a particular disease. However, treatments sometimes also can harm people. For example, most people are aware that antibiotics made from penicillins can cause severe (even fatal) allergic reactions. These harms, while often rare, are also important outcomes. In evaluating the effectiveness of medical treatments and interventions, both benefits and harms must be considered.

Where can people get more information about comparative effectiveness? One of the best places to start is a Web site by the federal Agency for Healthcare Research and Quality. The site's address is:

It has a variety of reports that summarize comparative effectiveness for different diseases. It also has summaries of the scientific issues that underlie how to conduct comparative effectiveness research.