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How Can Comparative Effectiveness Research Help Health Care Reform?

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In the national debate over the necessity and feasibility of significant health care reform, changes to insurance coverage, payment rules, health services delivery, and possibly malpractice laws have emerged as the basic framework of a new health care system.

How and to what extent those principles would change is the crux of the debate.

Currently, new drugs and technologies are not tested against current interventions; rather they are vetted for safety and effectiveness in a kind of vacuum.
Dr. Alvin I. Mushlin, the Nanette Laitman Distinguished Professor and chairman of the Department of Public Health at Weill Cornell Medical College, recently presented a Department of Medicine Grand Rounds lecture on the use of comparative effectiveness research as a cornerstone of health care reform.

"The major objectives behind reform are to improve health care quality, expand insurance coverage, and increase access to health care, while containing cost," Dr. Mushlin said.

Comparative effectiveness research — the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, treat, diagnose and monitor a clinical condition or to improve the delivery of care — should help assure that the goals of health care reform are met and that medical treatments are used in the best and most appropriate way. Dr. Mushlin and his co-author Dr. Hassan Ghomrawi, an instructor in public health, presented arguments for comparative effectiveness research in an article published online Jan. 6, 2010, in the New England Journal of Medicine.

Currently, new drugs and technologies are not tested against current interventions; rather they are vetted for safety and effectiveness in a kind of vacuum that is only comparing how the new treatment works compared to a placebo (or sugar pill). With comparative effectiveness research, a new cholesterol drug, for example, would be measured for effectiveness against the current standard of care to see if it really was better.

"This research would assist everyone in the health care arena — consumers, clinicians, purchasers and policy makers — in making informed decisions that would improve health care both on an individual and societal level," Dr. Mushlin said.

"[Comparative effectiveness] research would assist everyone in the health care arena in making informed decisions that would improve health care both on an individual and societal level."

– Dr. Alvin I. Mushlin
Granted, there are challenges to comparative effectiveness research. Patient populations for studies would need to be much larger than in the usual randomized control trials. It would also require longer follow-ups and it would mean basing studies in the community to ensure that the data reflected real-world medical practices. However, with the use of national databases, registries and electronic health records, many of those obstacles could be overcome.

"From the standpoint of innovation, comparative effectiveness research should be a catalyst of 'effective innovation,'" Dr. Mushlin said. "If our system evolves to depend on comparative effectiveness rather than singular efficacy compared to placebo, then there will be disincentives for 'me too' medications and interventions."

An example of comparative effectiveness research at Weill Cornell Medical College and the Hospital for Special Surgery is the Center for Research and Education in Therapeutics grant from the Agency for Healthcare Research and Quality (AHRQ), for which Dr. Mushlin is the principal investigator. Researchers on this project are studying the outcomes and costs of medical and orthopedic devices. As part of the study, the investigators have developed a registry of almost 19,000 cases from May 2007 to December 2009. Although hip, knee and shoulder joint replacement procedures are generally quite safe, therapeutically beneficial and cost effective, they are examining various models of replacement joints to see which ones function best are associated with the best short-term and long-term outcomes like the need for revision surgery.

This is the kind of long-term, real-world effectiveness study that comparative effectiveness research proponents say will lead to improved care at reduced costs.

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