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Do the Government's Biomedical Initiatives Ever Lead to Medical Progress?

Over time, sure, but in the immediate future, social science will play a bigger role in any large-scale improvements to human health.


Writing in the December issue of Wired, Bill Gates explained that when he first started considering philanthropy, he thought he could make the biggest impact by funding basic scientific research. "If we don't give scientists the room to deepen our fundamental understanding of the world,” he wrote, “we won't provide a basis for the next generation of innovations." But then Gates changed his mind. He decided to focus his efforts on the urgent needs of the world's poor, who have major problems that can be solved with the science and technology that are already on hand—like a 25-cent vaccine for measles, a disease that was killing more than a half-million children each year at the time.

The National Institutes of Health has clearly embraced the first option, becoming one of the largest sponsors of basic research in the world. Tasked with improving the nation's health, the NIH is committed to the idea that pure research devoted to a fundamental understanding of living things is one of the most effective ways to improve our ability to prevent, diagnose, and treat disease. Because the NIH, with it's larger budget, funds more biology than other U.S. government science agencies, the vast majority of U.S. biologists who study life at the molecular scale are labeled as "biomedical" researchers, despite the fact that their intellectual interests, mine included, are no more disease-oriented than those of the physicists who work with the Large Hadron Collider. These biologists' labs are often housed in medical schools and their research proposals are required to include a nod toward disease relevance. But their research into the molecular workings of bacteria, yeast, algae, worms, flies, fish, mice, and cultured cells in a dish is selected almost entirely for its fundamental intellectual merit, and not its specific medical promise.

Don't expect the discoveries coming from the Human Genome Project or the Cancer Genome Atlas to dramatically revolutionize medicine anytime soon.

Not surprisingly, the arrangement of supporting almost all basic biological science with funds for biomedical research tends to skew public expectations about potential medical benefits. The NIH's big projects, the ones that garner major headlines, like the National Cancer Act of 1971, the Human Genome Project, the Cancer Genome Atlas, and Obama's BRAIN Initiative, come with big promises about the payoff to human health, but they almost invariably funnel a large fraction of the money into basic research. As the years pass and people realize that we still get cancer or that the Human Genome Project hasn't changed what happens when you visit your physician, these projects are dismissed as failures.

So, has the NIH mixed up its priorities? Is basic research genuinely contributing to medical progress? How much medical progress is there, anyway?

WHEN YOU LOOK CLOSELY at the data, it's clear that we're continually making surprisingly good medical progress—even on the relatively short scale of a decade. In a 2010 review of how the U.S. was meeting its health goals, the assistant secretary for health at the Department of Health and Human Services noted that between 2000 and 2006 alone, U.S. life expectancy increased by 1.2 percent when measured from birth and by nearly six percent when measured from age 65. Looking at major causes of death, the news is generally good. Between 1999 and 2007, death rates from heart disease dropped by 33 percent. In the Annual Report to the Nation on the Status of Cancer, a collaboration between the American Cancer Society, the Centers for Disease Control, and the National Cancer Institute, researchers reported that death rates from cancer have decreased steadily by an average of 1.5 percent per year among adults, and two percent per year among children.

However, much of this progress is due to the fact that people have changed some unhealthy behaviors—smoking, in particular. Forty percent of the drop in U.S. cancer death rates come from reductions in smoking alone. It's clear that the biggest medical gains in the foreseeable future will be achieved by getting people to smoke less, eat better, and exercise more. A 2009 study found that a large fraction of premature deaths in the U.S. can be attributed to smoking, high blood pressure, obesity, and alcohol abuse. If we're looking for rapid, dramatic improvements in our collective health, understanding how to change the behavior of humans is more important than understanding how to change the behavior of a diseased cell.

Although the role of behavior in our health is a very big deal, we shouldn't dismiss the medical benefits of basic research. Unfortunately, those benefits are less direct and therefore harder to quantify. With a few exceptions—antibiotics, diuretics, vaccines, anti-retroviral therapy for HIV—the fruits of basic science discoveries rarely make an immediate, large impact on health. When we fund basic science to achieve better health, we're playing a very long game.

But this is rarely acknowledged. News reports on basic research will suggest that scientists are about to do things like cure Down Syndrome, despite the fact that the results are often clinically useless. It's hard to blame the news media for getting this wrong, because researchers and the NIH play the same game with the public that they play with each other—calling this science “biomedical” when it's really about understanding basic biology.

Because the medical value of basic research develops over a long time scale, perhaps the best way to take stock of its impact is to look back at the basic discoveries made in the last century and ask, how would our practice of medicine today be different if they hadn't been made? Major discoveries in biochemistry, genetics, and microbiology made decades ago thoroughly pervade medicine today. As these discoveries accumulated, they coalesced to create a context of understanding, without which modern medical practice would make little sense. The routine blood tests your physician orders are useful because of basic research on metabolism and the immune system conducted between 1930 and 1970. Many cancer treatments today would be inconceivable without key discoveries made in the 1970s of how cells control their reproduction. The discovery of the genetic code in the mid-20th century led to the production of synthetic insulin in 1978 and many other drugs since then.

So don't expect the discoveries coming from the Human Genome Project or the Cancer Genome Atlas to dramatically revolutionize medicine any time soon. Despite the bold claims made about the medical benefits of genetic screening or the potential to fight obesity by controlling our gut microbes, the real payoff from today's discoveries in genetics and microbiology will develop incrementally over the decades to come. The best way to improve human health in the immediate future is to follow Bill Gates' lead, and put our effort into small changes that make a large impact. But if we want to keep improving human health over the next century, we need to follow Gates' first inclination and "deepen our fundamental understanding of the world."