Read Caption
Sydney Smith

Advocacy’s Power and the Disease Olympics

One day in 2007 Ambassador Richard Sklar, who had pancreatic cancer, and Julie Fleshman, head of the nonprofit Pancreatic Cancer Action Network (PCAN), visited the D.C. office of House Representative Anna Eshoo to see if she might consider sponsoring legislation for more pancreatic cancer research. Sklar and Eshoo were friends.

“I asked him, Dick, why haven’t I heard from anyone on this?” Eshoo recalls. “And in a rapid-fire answer he said, because they’re dead.”

Eshoo learned that only 6 percent of people diagnosed with pancreatic cancer live longer than five years, and 74 percent die within a year. “The needle, so to speak, had not moved in half a century,” Eshoo says. “That, in and of itself, was an indication that whatever we were doing was clearly not enough.”

Over the next couple of years, PCAN’s advocacy efforts grew more visible because of emotional Congressional testimony by Carnegie Mellon professor Randy Pausch, who was dying of pancreatic cancer, and the news that actor Patrick Swayze was diagnosed. Eshoo introduced a bill, called The Pancreatic Cancer Research and Education Act, in September of 2008, then re-introduced it in January of 2009 and again in February 2011. As originally written, the bill would have directed the federal government to spend $888 million over five years on research and awareness campaigns focused solely on pancreatic cancer.

By August of last year, the bill was close to passing — until a bunch of scientists got wind of it and started raising hell. Their primary argument was that the bill undermined the scientific authority of the National Institutes of Health, which funds the vast majority of federal cancer research. In response to the criticism, the bill’s sponsors quickly scrapped its earmarks and even changed the name, to the Recalcitrant Cancer Research Act, to include other hard-to-treat cancers. “There were lots of behind the scenes meetings with leadership to figure out how it was going to move forward,” Fleshman says. “There were definitely many different times when we had to re-group and say, ‘OK, are we ok with this?’”

The revised bill passed the House unanimously on September 19. A week later, Harold Varmus, director of the National Cancer Institute, talked about the original bill at the National Press Club. “This is a slippery slope, and it’s a dangerous one,” Varmus said. If the bill’s original language had passed, it would have led to a disease-based competition, he added. “Very quickly, every other advocacy group would say, I want that, too. And then we have chaos.”

View Images
by Sydney Smith

In a story out yesterday in Nature Medicine, I tried to dig into this tricky issue, sometimes called the “disease olympics.” In the past couple of years, a slew of studies — conducted by economists, sociologists and political scientists — have come out showing that, over the past two decades, there’s been a dramatic rise in nonprofit disease groups and the money they spend on lobbying. Over the same period, the number of disease-specific bills shot up and NIH began classifying its grants according to disease.

Advocates say this isn’t a problem: The NIH is a publicly funded organization, after all, and should be responsive to the wishes of the public. The voices of advocates are important because they often point to clinical concerns that basic scientists don’t think about.

But basic scientists worry that advocates don’t think enough about scientific opportunity. The reason the NIH hasn’t spent more money on pancreatic cancer, for example, is because it’s inherently more difficult to study than, say, breast cancer or prostate cancer. Pancreatic cancer doesn’t give rise to noticeable symptoms until very late in the disease, so there are no screening tests that would allow researchers to track the course of those who have it. Besides, scientists say, scientific insights learned from one cancer might very well apply to another type.

In the end, I think the current system tends to create checks and balances that work pretty well. The Recalcitrant Cancer bill, for example, was eventually signed into law, and all parties involved say they’re fairly happy with it.

One thing I’m still curious about, though, is the effect of the sequester: With NIH dealing with significant budget cuts, will advocacy become more powerful, or less?

My sources had very different responses to this question. One possibility is that budget cuts won’t have much of an influence on the disease olympics. “It might be more or less across-the-board cuts, and so that it might not really effect the composition of the research that much,” says Frank Lichtenberg, a professor at Columbia Business School.

On the other hand, it’s interesting that the rise of disease-specific organizations came at the same time that the NIH budget was growing rapidly. During that period, advocacy wasn’t a zero-sum game: the government could put more money into one disease without taking from another. With the budget cuts, though, advocates could get much more competitive. “It would be a shame if that meant that diseases that had stronger advocacy would be preserved at the expense of diseases without advocates,” says Clay Johnston, director of UCSF’s Clinical and Translational Science Institute. “We would all suffer from that.”


Illustrations by Sydney Smith