Comparative Effectiveness: Are We Ready?

The cardiology community, under the leadership of the American College of Cardiology and American Heart Association, has struggled for the last 2 decades with the task of creating appropriateness guidelines for the care of cardiac patients.

The rigorous, open process has been a struggle to provide a scientific foundation for the confirmation of guideline recommendations.

It has taken on a new dimension with the forthcoming health care reform under consideration by Congress, which has given comparative effectiveness research (CER) a major role in establishing the payment parameters for appropriate use of drugs and devices within Medicare. The final construct of the CER process will have an immense impact on how we practice cardiology and will extend well beyond the use of guidelines in our clinical decision making.

It has been estimated that 30% of all medical spending has no discernible benefit. The bill for this useless care totals approximately $700 billion.

To deal with this presumed “waste,” the federal government plans to use CER to gain more data to establish guidelines and recommendations about the efficacy of current therapy primarily in the Medicare population. Because of the size of Medicare, these changes will likely impact the entire insurance industry.

Some political conservatives would suggest that this will result in rationing of care. In fact, this is precisely what is intended. But of course we have had economically imposed rationing for some time.

Ensuring the most judicious use of resources measured by effectiveness and cost is certainly a worthwhile goal. Whether the medical community is now or will ever be ready to fill this role is open to considerable question.

Cardiology guidelines that have been developed have had limited success. At best, they have provided marginal improvement in clinical care (Am. Heart J. 2009;158:546–53). Only 19% of current guidelines are supported by

Even when a clinical trial shows a positive benefit, its effect on clinical care is slow. Rarely is a single trial's demonstration of a drug's efficacy sufficient to change medical care substantially. The development of convincing data that will gain the approval of the Food and Drug Administration costs money and time.

Changes are even more difficult when carrying out comparative trials as CER advocates propose. By their nature, comparative trials in which one therapy is compared with another take large patient numbers.

One of the few comparative trials sponsored by the National Heart, Lung, and Blood Institute—the Antihypertensive and Lipid-Lowering Treatment to Prevent Heart Attack Trial—compared antihypertensive drugs and was planned to provide the final answer to the question of what is the most effective therapy. ALLHAT's outcome had little effect on clinical care and cost more than $100 million. Few comparative trials have been carried out since then because of the expense. Randomized trials sponsored by pharmaceutical companies are planned to test new drugs in comparison to older, accepted medication or a placebo.

The current Congressional plan includes more than $1 billion for studies comparing drugs and devices to “save money and lives.”

It is proposed that a federal health board or a comparative effectiveness agency will be created to institute a process of compliance and implementation of its decisions. CER decisions could become de facto administrative decisions and could determine what care can and should be provided within Medicare. It is anticipated that in some instances, the agency will seek input from specialty societies rather than leaving the decision process to a group of experts in Washington.

How comparative effective research will be carried out is now under review and its ultimate impact on care remains uncertain.

It is certainly unrealistic to presume that we are close to providing the scientific answer to the appropriateness conundrum. Effectiveness is not easily defined, but we can usually define ineffectiveness when we see it. Bridging the gap between these two extremes is easier said than done.

The cardiology community, under the leadership of the American College of Cardiology and American Heart Association, has struggled for the last 2 decades with the task of creating appropriateness guidelines for the care of cardiac patients.

The rigorous, open process has been a struggle to provide a scientific foundation for the confirmation of guideline recommendations.

It has taken on a new dimension with the forthcoming health care reform under consideration by Congress, which has given comparative effectiveness research (CER) a major role in establishing the payment parameters for appropriate use of drugs and devices within Medicare. The final construct of the CER process will have an immense impact on how we practice cardiology and will extend well beyond the use of guidelines in our clinical decision making.

It has been estimated that 30% of all medical spending has no discernible benefit. The bill for this useless care totals approximately $700 billion.

To deal with this presumed “waste,” the federal government plans to use CER to gain more data to establish guidelines and recommendations about the efficacy of current therapy primarily in the Medicare population. Because of the size of Medicare, these changes will likely impact the entire insurance industry.

Some political conservatives would suggest that this will result in rationing of care. In fact, this is precisely what is intended. But of course we have had economically imposed rationing for some time.

Ensuring the most judicious use of resources measured by effectiveness and cost is certainly a worthwhile goal. Whether the medical community is now or will ever be ready to fill this role is open to considerable question.

Cardiology guidelines that have been developed have had limited success. At best, they have provided marginal improvement in clinical care (Am. Heart J. 2009;158:546–53). Only 19% of current guidelines are supported by

Even when a clinical trial shows a positive benefit, its effect on clinical care is slow. Rarely is a single trial's demonstration of a drug's efficacy sufficient to change medical care substantially. The development of convincing data that will gain the approval of the Food and Drug Administration costs money and time.

Changes are even more difficult when carrying out comparative trials as CER advocates propose. By their nature, comparative trials in which one therapy is compared with another take large patient numbers.

One of the few comparative trials sponsored by the National Heart, Lung, and Blood Institute—the Antihypertensive and Lipid-Lowering Treatment to Prevent Heart Attack Trial—compared antihypertensive drugs and was planned to provide the final answer to the question of what is the most effective therapy. ALLHAT's outcome had little effect on clinical care and cost more than $100 million. Few comparative trials have been carried out since then because of the expense. Randomized trials sponsored by pharmaceutical companies are planned to test new drugs in comparison to older, accepted medication or a placebo.

The current Congressional plan includes more than $1 billion for studies comparing drugs and devices to “save money and lives.”

It is proposed that a federal health board or a comparative effectiveness agency will be created to institute a process of compliance and implementation of its decisions. CER decisions could become de facto administrative decisions and could determine what care can and should be provided within Medicare. It is anticipated that in some instances, the agency will seek input from specialty societies rather than leaving the decision process to a group of experts in Washington.

How comparative effective research will be carried out is now under review and its ultimate impact on care remains uncertain.

It is certainly unrealistic to presume that we are close to providing the scientific answer to the appropriateness conundrum. Effectiveness is not easily defined, but we can usually define ineffectiveness when we see it. Bridging the gap between these two extremes is easier said than done.

The cardiology community, under the leadership of the American College of Cardiology and American Heart Association, has struggled for the last 2 decades with the task of creating appropriateness guidelines for the care of cardiac patients.

The rigorous, open process has been a struggle to provide a scientific foundation for the confirmation of guideline recommendations.

It has taken on a new dimension with the forthcoming health care reform under consideration by Congress, which has given comparative effectiveness research (CER) a major role in establishing the payment parameters for appropriate use of drugs and devices within Medicare. The final construct of the CER process will have an immense impact on how we practice cardiology and will extend well beyond the use of guidelines in our clinical decision making.

It has been estimated that 30% of all medical spending has no discernible benefit. The bill for this useless care totals approximately $700 billion.

To deal with this presumed “waste,” the federal government plans to use CER to gain more data to establish guidelines and recommendations about the efficacy of current therapy primarily in the Medicare population. Because of the size of Medicare, these changes will likely impact the entire insurance industry.

Some political conservatives would suggest that this will result in rationing of care. In fact, this is precisely what is intended. But of course we have had economically imposed rationing for some time.

Ensuring the most judicious use of resources measured by effectiveness and cost is certainly a worthwhile goal. Whether the medical community is now or will ever be ready to fill this role is open to considerable question.

Cardiology guidelines that have been developed have had limited success. At best, they have provided marginal improvement in clinical care (Am. Heart J. 2009;158:546–53). Only 19% of current guidelines are supported by

Even when a clinical trial shows a positive benefit, its effect on clinical care is slow. Rarely is a single trial's demonstration of a drug's efficacy sufficient to change medical care substantially. The development of convincing data that will gain the approval of the Food and Drug Administration costs money and time.

Changes are even more difficult when carrying out comparative trials as CER advocates propose. By their nature, comparative trials in which one therapy is compared with another take large patient numbers.

One of the few comparative trials sponsored by the National Heart, Lung, and Blood Institute—the Antihypertensive and Lipid-Lowering Treatment to Prevent Heart Attack Trial—compared antihypertensive drugs and was planned to provide the final answer to the question of what is the most effective therapy. ALLHAT's outcome had little effect on clinical care and cost more than $100 million. Few comparative trials have been carried out since then because of the expense. Randomized trials sponsored by pharmaceutical companies are planned to test new drugs in comparison to older, accepted medication or a placebo.

The current Congressional plan includes more than $1 billion for studies comparing drugs and devices to “save money and lives.”

It is proposed that a federal health board or a comparative effectiveness agency will be created to institute a process of compliance and implementation of its decisions. CER decisions could become de facto administrative decisions and could determine what care can and should be provided within Medicare. It is anticipated that in some instances, the agency will seek input from specialty societies rather than leaving the decision process to a group of experts in Washington.

How comparative effective research will be carried out is now under review and its ultimate impact on care remains uncertain.

It is certainly unrealistic to presume that we are close to providing the scientific answer to the appropriateness conundrum. Effectiveness is not easily defined, but we can usually define ineffectiveness when we see it. Bridging the gap between these two extremes is easier said than done.