Admitting at the outset that measuring NIH’s value is a very difficult topic, he added, “I am not here for an academic discussion of how medical research should be valued…[that] has been said today, I am sure, by the speakers you invited. I shared, for example, in Congress that the $4 per year invested per person in the United States since the 1970s on cardiovascular research now results in $2.5 trillion of economic value every year. The problem with these kinds of statements is that you can easily make them, but you can’t easily prove them.”
Zerhouni was asked to discuss “Value of Federally Funded Biomedical Research in the Development of Medical Interventions and Treatments.” In a 20-minute address followed by questions, he argued that “the notion of value changes over time” and that narrowly defined metrics tend to be misleading. The most important criteria, he said, is whether dollars are assigned in a way that satisfies societal expectations.
Before giving his remarks, Zerhouni is greeted by NIH principal deputy director Dr. Lawrence Tabak and by NIDCR director Dr. Martha Somerman and (obscured) NIGMS acting director Dr. Judith Greenberg.
Photos: Ernie Branson
“The product of NIH should be knowledge, not products,” he said. “How do we achieve reduction in the burden of disease and reduction in the burden of health care costs? How do we transform knowledge into societal benefit?”
With the engineer’s cast of mind that distinguished his NIH leadership, Zerhouni said, “I have a simple matrix in my mind,” a series of pragmatic, measurable steps for “transforming ideas into knowledge.” He enumerated four steps in translation, T1 through T4.
“T1 is when you are truly on the edge of understanding some process or disease biology,” he explained. The criteria for valuing work at this stage “will be completely different” from how one values T4 research, which involves applying the fruits of basic research to the practice of public health. “This is getting mixed up in the public debate,” he said.
Zerhouni plumbed NIH history for examples of the interdependency of various translational steps in achieving an acknowledged public health success. It was the long-term Framingham Heart Study that picked up cholesterol as an important signal in cardiovascular morbidity and mortality, he recounted. That work led to Drs. Michael Brown and Joseph Goldstein’s Nobel Prize-winning studies that “changed the practice of medicine.
“How many times has NIH science done this?” Zerhouni asked, rhetorically. In stroke, there has been a 70 percent reduction (that’s T3, change in the practice of medicine) in recent decades. But what has been the value of NIH research when only 30 percent of patients with diabetes comply with their medications and 70 percent don’t, Zerhouni wondered? “And only 15 percent of patients with high blood pressure are compliant with their medication.”
He called implementation, or behavioral, research a worthy investment. “Look at a map of the United States,” he said. “Why are there all these pockets of disparity [in health outcomes] when the knowledge is the same everywhere?”
Zerhouni’s favorite example of NIH delivering on its promise to society is the Women’s Health Initiative’s finding, during his directorship, that hormone replacement therapy proved more harmful than helpful to women. “That’s value,” he declared, “and it’s important to quantify that value…This kind of impact is very valuable, and measurable. You can point to lives saved, quality of lives, number of years.”
Zerhouni also greets NIBIB director Dr. Roderic Pettigrew in Wilson Hall prior to his talk.
In any discussion of value, Zerhouni emphasized the need to personalize results. The goal, he said, is outcomes that relate directly to societal expectations. In a country where chronic diseases account for 80 percent of health care costs, he said, “federal investment needs to be tied to the societal needs of the day. Otherwise you risk academic isolation, or living in some theoretical realm…Patients measure, better than anybody, the value of research.”
Zerhouni said his personal view is that NIH should devote 60 percent of its budget to generate new knowledge. “I wouldn’t go below that, no matter what.”
He concluded with three lessons:
- “Don’t damage young investigators. Give ’em a chance, and give it early. Don’t kill them with rigidity—4 years of this, then 6 years of that…They end up exhausted at the end of such combat.”
- “We have moved away from studying human disease in humans,” he lamented. “We all drank the Kool-Aid on that one, me included.” With the ability to knock in or knock out any gene in a mouse—which “can’t sue us,” Zerhouni quipped—researchers have over-relied on animal data. “The problem is that it hasn’t worked, and it’s time we stopped dancing around the problem…We need to refocus and adapt new methodologies for use in humans to understand disease biology in humans.”
- “Budget pressures are going to kill any inkling of innovation.” Zerhouni warned that there are 5 diseases that, if not solved within the next 5 years, will certainly bankrupt some societies.
Zerhouni took half a dozen questions from SMRB members assembled in Wilson Hall and made a number of other observations:
- Game approaches to compliance fascinate him. “We’ve got to be able to engage the patient beyond a visit every 3 months, coupled with a series of ‘thou shalt nots.’”
- The power of social networks, or so-called “influencers,” has been underutilized in public health.
- Statins are phenomenally over-prescribed; only 10 percent of the patients who take them realize any benefit. “So 90 percent of what we do with statins is not helpful,” Zerhouni said.
Any consideration of the value of research that does not take the customer into account is doomed, he warned. “You’ve lost the debate if you lose sight of the taxpayers and the patients.”