Beware of Politicians Bearing Moonshots

In his State of the Union Address last month, President Obama said:

Last year, Vice President Biden said that with a new moonshot, America can cure cancer. Last month, he worked with this Congress to give scientists at the National Institutes of Health the strongest resources that they’ve had in over a decade. So tonight, I’m announcing a new national effort to get it done.

This week, the administration started laying out some of the details of what would be involved in this “moonshot,” and presumably more will be coming in the president’s budget proposal next week. They envision a roughly $1 billion investment, mostly through the NIH’s National Cancer Institute, and to a lesser degree through the FDA. Presidents have been promising this sort of thing for many decades, at least since Richard Nixon declared a “war on cancer” in 1971. And surely more resources would help cancer researchers, even if the analogy to a single, focused effort with one goal is misleading. What could go wrong with a moonshot to cure cancer?

Unfortunately, quite a bit.

In considering how best to help advance cancer research, the administration should review the lessons of the enormous increase in the NIH’s budget between 1998 and 2003. The agency’s budget was doubled in that period, from $15 billion to roughly $30 billion per year. The five-year doubling was the brainchild of two senators, Republican (later turned Democrat) Arlen Specter of Pennsylvania and Democrat Tom Harkin of Iowa, and it proved irresistible to members of Congress from both parties and to presidents Clinton and Bush.

It’s easy to see why. Support for spending on medical research is an unusual point of agreement in our polarized politics, and rightly so. But the extremely rapid budget increases in that five-year period turned out to create some enormous problems. I started to get a sense of these in 2005 when, as a health policy staffer in the Bush White House, I began getting lots of meeting requests from the lobbyists for the country’s largest research universities, who wanted to bring their most senior faculty members in to talk about funding for biomedical research. Again and again, the scientists would complain bitterly about flat budgets in 2004 and 2005, and how these were wrecking their research programs.

My first instinct was to think these folks were outrageously greedy. Before each of these meetings, I’d ask one of the NIH examiners at the Office of Management and Budget to put together a table for me of NIH funding over the prior decade for the particular university that was sending people to meet with me. When they raised their complaints, I’d pull the table out of a folder and show them that their public support had vastly increased over the previous few years—in many cases doubling or more. How could they possibly complain?

This was a fun ploy, and the university lobbyists certainly found it uncomfortable. But the researchers were unfazed. And after a few such conversations, I began to realize that they were right to complain, and I’d been wrong to dismiss them—or rather, that the doubling of the NIH budget over a five-year period had been disastrously misconceived.

It’s a mistake to think about medical research as a product to be purchased, so that more money means more research and more innovations. The publicly supported medical research enterprise in America is basically an academic enterprise, which means it is always engaged in the training of new researchers (who, during their training, provide the skilled labor that enables the enterprise to proceed) and always building incrementally upon various lines of inquiry. A five-year doubling of the enormous budget that supports all this was almost perfectly designed to screw up the process—drawing large numbers of graduate students into research-heavy fields and then, just as they were finishing their training, rapidly decelerating the support available for them to begin their own careers.

The steep growth in spending in that five-year period, roughly 15 percent per year, built expectations and momentum that set the NIH up for disappointment when the doubling was done. Research institutions used the added funds to start new projects, support many more students, and establish more programs and labs. But these all require continuing support, which meant that once the steep increases were done and the NIH budget returned to normal growth rates of about 3 percent per year, an enormous portion of the budget went towards continuing support for projects established in the fat years, and not enough was left to fund new ventures. The glut of graduate students had trouble finding work, had to compete (unsuccessfully) with their own mentors for funding and jobs, and faced grim prospects once their training was done.

Meanwhile, the growth was also too fast to develop new major research institutions, so that almost all the money went to the few large giants that had been best funded before the doubling began. While the size of the average NIH grant grew a lot during that period (the average size of an individual investigator grant grew by nearly a quarter) the pool of institutions receiving funding hardly changed at all. As the Chronicle of Higher Education found in reviewing lists of NIH grantees in the wake of the doubled budget, of the 515 institutions receiving direct grants to investigators in 2003, only five had not been receiving such grants prior to 1998. The portion of grants going to the top 100 research institutions in 2003 was 92 percent, exactly the same as it was in 1998. In other words, more NIH funding meant more money for the same relatively small group of elite institutions. And even at those institutions, senior researchers were frustrated when the doubling was completed, and made their way to Capitol Hill and the White House to complain.

Today, more than a decade after the doubling ended, I think it’s fair to say that the larger budgets have been better digested, and the American biomedical research enterprise is in stronger shape for the greater support. But for much of the last decade, between 2000 and 2010, this was often not the case.

And even where the budget increases funded important new work that led to real medical progress (it’s hard to say, of course, what would or would not have happened without the added spending), the effect of the design of the doubling on the morale of American medical researchers was certainly negative. It left this highly valued and amply funded field feeling like it was being mistreated by Washington. This contributed to an intense politicization of medical research, even though support for the NIH has always been bipartisan. And I think it also contributed to the tenor and character of the stem-cell debate that was so prominent a decade ago—into which American biologists poured their frustration over funding and in which, in retrospect, it should be perfectly clear that the American biomedical research community utterly disgraced itself.

Even if you agree that greater support for medical research is a good idea, in other words, how that support is provided matters, and large and sudden bursts of money can actually do real harm to exactly the work they aim to help. The portion of the “moonshot” money intended for the NIH runs a real risk on this front.

The FDA element, meanwhile, is a little less clear. Here, too, it’s easy to appreciate the intentions behind the proposal, but it’s not entirely obvious what the problem to be solved is exactly. Ten years ago, maybe even five years ago, you could have plausibly argued that the FDA was moving much too slowly on cancer drugs. I don’t think you can really argue that today. The agency’s Office of Oncology and Hematology Products has pretty dramatically changed its attitude toward new drug applications.

To be sure, the requirements for precision and certainty in drug trials are still excessive, as they are throughout the FDA’s work, and this shows up in more than just approval times. We shouldn’t judge the FDA’s influence on drug development by just looking at how long it takes drugs to be reviewed: The relationship between the agency and the drug companies is a very complex and adaptive one. The drug makers adjust their work at every stage of drug development to improve the chances that they’ll satisfy the FDA’s demands at the end, which means they now spend much more time developing extended, detailed data in advance of the final FDA review than they once did. That does need to change. And some other reforms (especially approval reciprocity with drug-safety agencies in other developed countries) could well improve things too.

But I would argue that the FDA’s treatment of cancer drugs should actually be a model for the agency to follow in approving drugs for other diseases. This probably has to do especially with the fact that the agency knows that drugs it approves for cancer treatment will be prescribed and monitored by specialists, and the FDA trusts specialists not to behave irresponsibility—while it has absolutely no confidence in the general practitioners that prescribe most drugs. This lack of confidence in most physicians is actually at the heart of a lot of the problems with today’s FDA approval process, and its importance is often under-appreciated. But it means that cancer drugs are among those treated most rationally by the agency. I’m as down on red tape as anyone, but I don’t think the FDA process is the reason we haven’t cured cancer, and I’m actually pretty certain that a highly effective drug for a major cancer would be approved very quickly.

That such drugs remain very rare is a function of the difficulty involved in understanding and treating the more than 100 different diseases we call cancer. And pretending that getting there is just a matter of spending more money does no one any service. It is more likely that, as with the nation’s intense investment in genomics in the 90s and cell biology in the 2000s, what the research will teach us is that human biology is even more complex than we imagined. This would be a step forward, and both the human genome project and the emphasis on cell science have borne some fruit and were worthwhile. But neither has been quite as transformational as its (political) advocates claimed because medical research just doesn’t work that way.

Hard as it is to say, the best thing politicians can do for cancer research may be more like more of the same than a moonshot. And at the very least, they should strive to do no harm.