Measure for Measure

The Journal Of Life Science
By Peter J. Pitts
March 17, 2008

Health technology assessment must use a patient-centric model if it is to be more than a political tool to cut cost and reduce care.

Like other cost-based practices, health technology assessment—an effort to manage the use of medical technologies by evaluating their cost effectiveness—is designed to reduce costs and restrict patient care. But, properly designed in a way to provide and pay for the best care for patients, it can also be patient-centric. Just as we need new and better tools for drug development, we need them for effective measurement as well.

Today, health technology assessment is a short-term, short-sighted, politically-driven policy that results in one-size-fits-all medicine. While it may provide transitory savings in the short-term, current strategies result in a lower quality of care that result in higher healthcare costs over time.

Restrictive formularies and health care systems that deny patients access to the right medicine in the right dose at the right time but pay for more invasive and expensive procedures later on have their priorities upside down.

So why is the current health technology assessment model enjoying such wide support? It is because it drapes a veil of pseudo-science around the blunt instrument of one-size-fits-all price controls.

I’ve just returned from a European road trip with Frank Lichtenberg, an economist and professor of business at Columbia University, where we spoke in Brussels, Rome, and Berlin on health technology assessment. I represented The Center for Medicine in the Public Interest, which is part of a transatlantic public policy institute consortium on the future of health technology assessment.

Conflicting Interests
On the other side of the pond, Sir Michael Rawlings, chairman of United Kingdom’s National Institute for Health and Clinical Excellence or NICE, told the British House of Commons that comparative effectiveness, a means of health technology assessment, is not based on empirical research. “There is no empirical research anywhere in the world, it is really based on the collective judgment of the health economists we have approached across the country,” he said. “It is elusive.”

The problem is that health technology assessment, as it is currently designed, places into conflict the short-term budgeting dilemmas of governments elected for relatively short periods of time with the ever-lengthening life spans of their electorates.

According to my traveling companion Lichtenberg, for health technology assessment to yield valid decisions in practice, it is necessary to have reliable estimates of not only cost, but the other inputs as well, such as QALY, a measure of the quality of life and VSLY, the value of a statistical life year. He believes that incorrect estimates of some or all of these key inputs are often used. Due to these estimation biases, health technologies that are truly cost-effective may often be rejected as cost-ineffective.

Consider the recent debate over the utility of new cancer treatments. Lichtenberg makes the point that even though, over the past 30 years, the U.S. Mortality Age-Adjusted Rates for cancer have remained relatively constant—leading to articles in both the lay and scientific press about that lack of progress on the war on cancer—the often ignored reality is that five-year relative survival rates for all cancer sites have increased from to 65.9 percent in 2000 from 50.1 percent in 1975. (For more specifics on both the economic impact of new treatments and their impact on cancer survival, please see the paper that Dr. Lichtenberg wrote for the Center for Medicine in the Public Interest in 2007 www.cmpi.org).

On our Grand Tour of health technology assessment in Europe, Lichtenberg cited two crucial studies, pointing out how healthcare economists must seriously reconsider the outdated estimates of a QALY. In one the value of a statistical life for prime-aged workers has a median value of about $7 million in the United States. In the other, the value of a life year is calculated at $373,000.

In Berlin we were joined by Christian Behles, director and professor of drug regulatory affairs at the University of Bonn and an advisor to the German government. Professor Behles pointed out that while Germany’s Institute for Quality and Efficiency in Healthcare or IQWiG casts a suspicious eye on industry-designed pharmaco-economic studies, they use industry-sponsored randomized control trials as the basis of their comparative effectiveness findings.

He also noted that these randomized controlled trials were not designed to be used for head-to-head comparisons. In other words, IQWiG embraces industry-sponsored randomized controlled trials data that was not designed to be used comparatively, while rejecting industry-sponsored data that was specifically designed to show the value of a new innovative medicine. When is an industry study not an industry study? It seems that, for IQWiG, the answer is “when it’s convenient.”

Attention must be paid, not only in Congress, but at institutions overseas. If the devil is in the details (and it is), it’s time for a deep dive beyond simplistic and self-serving “comparative effectiveness.”

New Model Needed
A health technology assessment model for the 21st Century should reflect and measure individual response to treatment based on the combination of genetic, clinical, and demographic factors that indicate what keep people healthy, improve their health, and prevent disease. A rapidly aging society demands a new healthcare paradigm capable of providing for its needs in the 21st century. Equality of care must be matched with quality of care.

In an era of personalized medicine, one-size-fits-all treatments and reimbursement strategies are dangerously outdated. We are early in this debate, but at least we can all agree that this is not, and must not be exclusively, a debate about saving money. It must be about patient care.

Peter J. Pitts is president of the Center for Medicine in the Public Interest and a former Associate Commissioner of the FDA.