Perspectives

If any institution can lay official claim to being the all-seeing keeper of quality and integrity in medicine and medical research it is the National Academy of Sciences’ Institute of Medicine. Since IOM attempts to give a fair shake to all significant sides of most debates in biomedicine, Science and Policy Perspectives turned to IOM’s president, cardiologist Kenneth Shine, for his slant on the implications of the doubling of NIH’s budget.

Look for no radical reformist views here: no decrying, for example, NIH’s role in the galloping commodification of scientific knowledge in the U.S., or calls for any significant transfer of NIH funds toward health services research, or for pulling back on the frequently cozy, oftentimes turbulent intellectual property arrangements between researchers and the corporations with which they are allied.

But Shine does represent what boils down to at least a quasi-independent view of the medical system. So his opinions are important for any fruitful debate on reforming the directions NIH might need to take in the era of doubling. This is the edited transcript of that interview.

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With NIH sure to be having its budget doubled between 1999 and 2003, what are the sorts of questions NIH should be asking itself and others in terms of outcomes for all the money that’s been spent and that’s going to be spent?

NIH needs to account for its spending in terms of the things they’ve done. I recently proposed to the Council of Deans of the Association of American Medical Colleges that the best response should come from the medical schools rather than from the NIH. What each medical school ought to do is identify ten or so outcomes in terms of patient care that they believe have been directly impacted by research conducted over the last few years in the NIH. I say this because there are a number of opportunities to demonstrate that expanding NIH research activity has had a real impact on how patients are cared for and I think that academic health centers are a very good source of that kind of information.

Secondly, I think that academic health centers can document the impact of such expenditures on training, particularly in clinical research. NIH has been able to substantially expand its programs that train young investigators to become clinical investigators, an activity that was a desert until the last few years. We now have a whole bunch of outstanding young people who are in the position to do clinical research, get training, and get mentored. There are also funding opportunities for mid-career scientists.

Many areas need to be broadened. But in all fairness to them, the definition of clinical research that they’ve adopted includes population-based research, including epidemiology. I think that’s an important step forward because that’s going to train people to look at the questions about what the outcomes are.
 

Do you think there’s any danger that this doubling will overbuild the system?

In terms of infrastructure, core facilities, and unexplored opportunities, there are still very substantial opportunities that need to be built upon. The real challenge is not going to be so much the effect of expenditure of money during the doubling. The real challenge will be on how you make decisions after the doubling is accomplished. The doubling clearly was never based on any precise formulation of how much additional money was required. Both the NIH and the scientific community are going to have to be more explicit in identifying what the unmet opportunities and unmet needs are in a much more specific and quantitative way.

I don’t think there’s going to be any problem identifying those opportunities. I do believe that there is a fundamental problem with regard to the imbalance in the increase of the funding for NIH versus funding for National Science Foundation, Department of Defense, and the Department of Energy. The problem is that many of the advances in the biomedical sciences and health services research depend critically on science that’s historically been going on in those other agencies.

And because those areas are underfunded, the pressure is going to be on NIH to try to decide how and in what way is it going to make investments which will have to compensate for the failure of investment in the other areas. Without that kind of a balancing act, the NIH is likely to be stressed.
 

Can NIH be integrated into the larger health care system? Spending for clinical research may have risen, but quality of care remains contentious and people seem to be getting angrier, if anything. What else needs to be better integrated at NIH through this doubling phenomenon?

The investment of the various institutes in both clinical research and particularly in health care services and overall care of patients has been very uneven. Some institutes, for example, Mental Health, are required by appropriation to spend 15% of their money on health services research. The Cancer Institute is beginning to pay a lot of attention to quality of cancer care in the country. And in collaboration with other Health and Human Services Department agencies, it is looking at projects to find out better ways to deliver cancer care. It has put out a report on palliative care in cancer and is reorganizing so that it can follow up on that.

I think many of NIH’s institutes need to make larger investments in health services research. After all, that’s where you get the evidence as to whether things are working or not. They need to be focused on the full range of research that relates to care. The Agency for Health Care Research and Quality is a grossly underfunded agency. So even if it receives substantial increases in its appropriation, the NIH still has a major responsibility there.
 

Do you think the genomic work will fulfill all the exciting prophecies?

The reality is that we are still a long way from the successful clinical application of gene therapy. It will happen, but I don’t know when. It’s clearly been presented in a way that suggests it’s right around the corner. And in fact there’s going to be a very significant period of time before that kind of therapy is proven. I think diagnostics will have a big impact in the next few years and drug development will have a big impact. We’ve already seen some of that.
 

How do you respond to the common observation that this high-tech era of medical research can only lead to higher and higher health care costs?

There’s no question that the more technology you have, the more you want to use. And the safer and simpler it is, the more you do use it. Increasingly, one’s going to have to be much more careful about understanding the marginal value of new technologies, what in fact is producing substantial additional value as opposed to very tiny incremental value. I happen to be a very, very strong proponent of the concept of well-informed joint physician-patient decisionmaking. And in that process, given recent work, we know that if patients are fully informed, they often select less radical, less expensive treatments than their physicians do. Everything IOM has done on quality of care suggest that patients do have to be participants. If they’re fully informed they often don’t use all of the advances to the same extent that physicians think they are required to do.

Academic institutions have an increasing responsibility to assess new technologies, to demonstrate whether they are substantially cost effective. There’s a whole shtick here. We have a tendency to bring on new technologies but not to replace the old ones. So you have people getting umpty-ump scans until they get their helical MRI when in fact all they needed was the helical MRI.

It’s also clear that the fastest growing area in health care costs is drugs. Expenditures are increasing at 14% to 17% a year. And part of the reason for that is that we’ve got much newer and much more effective drugs and therefore there are more options available. But it’s also clear that advertising is driving the increased price of drugs, that pharmaceutical houses are spending huge amounts of money in marketing these drugs, and that physicians have to become much better informed about the choices of medications rather than relying on the drug company salespeople to help them understand what to do. There is a tremendous tendency, again, for physicians to use the newest product, and they ought to be much more conscientious in carefully assessing what the incremental value is in determining whether they want to go to the newest, most expensive drug.
 

Where do you think the popularity of alternative and complementary therapies is taking the system?

While some of these alternative therapies may be harmful, and may be the result of charlatanism, some of them may have real benefit. That’s why I strongly support their study and evaluation.

I say this because I’m a cardiologist and the history of digitalis is a case in point to those of us in the field. Digitalis was discovered by William Withering after going up to see the Widow of Shropshire in 1770’s. She would make a tea she gave to patients who had dropsy, fluid accumulation, and they would often pee away all the fluid. Withering heard about it and went up and talked to her and he looked at the herbs and roots she put in her tea and recognized that one of them was digitalis purpura which was an agent that, for example, deer wouldn’t eat. He was a botanist and he inferred from this that there must be an active principle in this. So he ground it up and he fed it to patients and sure enough he could cure dropsy when it was related to heart failure. And that’s how digitalis was discovered.

There will always be individuals who are not comfortable with allopathic approaches to care. But I think the fact that physicians have the feeling that they are much more hassled, that both physicians and patients are concerned about the quality of the time they have to spend with each other, is one of the factors which make alternative therapies much more attractive. One of the most important elements of alternative therapy is that it moves the source of control to the patient. The patient decides who he or she is going to see, what he or she does. Under those circumstances, the sense of control over one’s environment, the control of one’s body, is extremely important. And until or unless allopathic medicine can give patients the sense that they have a real say in what happens to them, I think you’ll see increasing pressure for people to use these alternative therapies.

 
So much of one’s health status and prospect has to do with behavior and attitudes toward one’s body. You go to the beach in the summer and can’t help noticing how out of shape many people are. And yet, people are more than ever aware of the importance of physical fitness, especially as they move toward their middle years. So we have this national contradiction. Where do you think we are headed here?

IOM has taken this problem of behavior very seriously. Half of all health care costs are related to unhealthy behaviors and we, in collaboration with the National Research Council, have recently published six reports on the social and behavioral sciences for health. We need substantial investment in research in how to motivate people toward healthy behaviors. Obesity is a good example of an epidemic that we don’t know very much about in terms of really altering how people handle their behavior. Some of the preliminary research in that area is absolutely fascinating. So there needs to be a major investment in the social and behavioral sciences.
 

With all these newer factors in the thinking about health and disease, do you think the system is headed toward some kind of quantum jump in how we approach medicine and research?

There are certain clear trends. There’s no question that genomics will be a major activity over the next ten or fifteen years. But I think one of the most important developments over the next decade of two will be the move from reductionism to increasing integration. What I mean by that is that if you start with the genome, you have to move from DNA to genes. That means you need to look at tertiary structure of proteins, you need to look at how cells are organized, how the components of cells interact with each other, how cells interact within organs, how organs function. I think we’re going to see a rebirth of interest in physiology as opposed to molecular biology.

Ultimately you’re looking at systems of organ behavior. You can’t talk about brain function, for example, at the level of a gene solely. You now have to integrate your knowledge. That ultimately gets you into the whole question of population. We’re not going to do away with reductionism, but we’re going to see many more attempts to integrate knowledge during the first half of the 21^st century.

We’re going to see more research at the interfaces of the sciences, between mathematics and physiology, for example, and between chemistry and biology, engineering and medicine, and nanotechnology and health. These connections are already accelerating. On the health care side, I think the potential for systems engineering to enter into the health care system is an important notion. Physicians have historically eschewed things that came from industry. We never thought of ourselves as an industry even though we’re 14% of the Gross Domestic Product. I think we’re going to see the applications of human factors research in the way hospitals operate. So the interface issue is a profound one. We’re going to see an increasing interaction among all of the life sciences together. That is, biotechnology is not only critical in health. It’s critical in agriculture, and environmental remediation in the chemical industry. You can go on and on with these sorts of connections.

 
How do you respond to the critics who say that with all the money spent by NIH on research, so little progress has been made against many common diseases?

Well, my field is cardiology. We’ve seen extraordinary improvements in age-adjusted mortality. The American Heart Association’s programs in education also had a lot do with that. But certainly a lot of the research that went into understanding cholesterol and the development of drugs to control hypertension came out of NIH. And if you think about it, in the history of the world, there was not much increase in the life expectancy of adults until about 1960. Until that time, most of the increase in life expectancy was because of decreased infant-child mortality. But in the last 40 years, we’ve seen an increase in the life expectancy of people at age 65.

Now the cancer story is much slower. But the reality is, for example, that my brother-in-law survived 25 years after having grade four Hodgkin’s disease and was cured of it. When I was a medical student that was absolutely incurable. We can cure testicular cancer. Eighty percent of childhood leukemias are now curable. None of them were curable when I was in training. And we’re beginning to see some improvement in the mortality of some of the more resistant tumors. So while progress has been difficult in cancer, there have been islands of extraordinary success.
 

What sorts of studies are in the works at IOM?

Quality of health care in America was an overarching activity which we identified in 1996. That’s why we produced “To Err Is Human,” “The Quality Chasm,” and “Quality Cancer Care,” and the report on the state of quality in health care in America. The government is going to start in 2003 reporting on the state of quality of health care in America. We did a report on how to do that, and we’re going to do additional reports on identifying priority conditions. We have started the first of six studies on the uninsured in America, we are just about to start a study on the future of the academic health center, and we anticipate a series of studies on information technology, not only for the health care system but also for patients, for providers, and for the public. Those are overarching themes. In addition, we’re continuing to work on human participant safety, integrity in research and about 100 other things.

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