What does a disease deserve?
Pressure from AIDS groups such as ACT UP, protesting at the White House in 1987, propelled Congress to begin earmarking research funding for HIV/AIDS.
PHOTO: © BETTMANN/CORBIS
In the early 1990s, as the deadly HIV/AIDS epidemic marched across the United States and the world, lawmakers in Congress and top officials at the National Institutes of Health (NIH) reached an unusual understanding: Roughly 10% of the NIH budget would be dedicated to fighting the devastating disease. Since then, the steady flow of research cash—some $3 billion this year—has helped transform HIV infection from a death sentence to a manageable disease for many people, and some researchers believe they are getting closer to developing a vaccine that could halt new infections.
That special arrangement is now under fire. Health policy experts, lawmakers, and even NIH officials have wondered why, 2 decades after AIDS death rates began dropping dramatically in the United States, the disease still gets a lion's share of NIH resources. As questions have arisen about how HIV/AIDS funds are spent, NIH has also resolved to refocus AIDS money on ending the epidemic. Some voice a broader critique: that NIH's spending on a disease often doesn't align with how much suffering it causes. They note that diseases imposing a relatively small burden on U.S. society, such as AIDS, can get a larger share of NIH funding than those that cause greater harm, such as heart disease (see graph, p. 901). NIH's spending priorities can be “out of whack” says Senator Bill Cassidy (R–LA), a physician who serves on a panel that helps set NIH's budget.
Recently, while responding to pointed questions from Cassidy about the issue, NIH Director Francis Collins said the agency is ready to abandon the 10% set-aside. If Congress follows through on proposals to give NIH a hefty funding boost for 2016, its HIV/AIDS portfolio need not necessarily grow in “lock-step” with the increase as it has in the past, Collins said at a hearing. At the same time, NIH is taking a broader look at disease spending. Next month officials are expected to release an agency-wide strategic plan that they say will address how disease burden should influence the allocation of research dollars.
The ferment worries AIDS research advocates. “HIV/AIDS research could shrink as a percentage of the total [NIH budget] for the first time” in more than 2 decades, laments Kimberly Miller of the HIV Medicine Association in Arlington, Virginia, which represents health workers who treat HIV-infected patients. Long-time NIH observers also wonder whether the developments signal a re-emergence of the sometimes fierce disease funding wars of the past, when advocates for breast cancer research and other diseases battled for a bigger share of the NIH pie. Already, Cassidy has proposed shifting some AIDS spending to neurodegenerative diseases such as Alzheimer's.
Some in the biomedical research community, however, welcome these developments. “I'm glad the questioning is happening no matter how annoying it may be to some people,” says Claiborne Johnston, a stroke researcher at the University of Texas, Austin, who has studied disease funding trends. “I hope ultimately that we figure out a way to truly address, through our research and our care, some of these conditions that definitely are neglected.”
FROM ALMOST THE VERY beginning, NIH's AIDS earmark has created tensions. After activists helped persuade Congress to ramp up HIV/AIDS funding in the 1980s, lawmakers agreed to cap it at 10% of NIH's budget, but keep that proportion year after year. NIH officials argued that AIDS deserved a special allocation because it was a new disease, was still spreading, and had become the leading cause of death for U.S. adults between 25 and 44 years old. Key lawmakers were convinced, and since then AIDS research, which NIH has treated as its own distinct pool of funding, has essentially expanded in sync with the agency's overall budget.
It wasn't long before the deal inspired advocates for breast cancer and several other diseases to publicly argue that those fields, too, deserved large budget increases. Those 1990s campaigns ultimately met with mixed success in Congress. But the so-called disease wars did stir tensions within the large coalition of university, patient, and science groups that advocate for greater biomedical research spending. Those allies traditionally try to present a united front, for fear that internal divisions could fragment and endanger political support for overall NIH spending increases.
Now, that uneasy alliance has again come under pressure as the threat of AIDS has faded domestically. As new drugs made the disease more manageable, the number of deaths directly attributed to HIV plummeted in the United States from 45,000 per year in 1995 to 7000 in 2013. At the same time, some patient groups have recently helped persuade some lawmakers that they should be concerned about the much higher toll of other diseases, such as stroke and diabetes, and the staggering projected costs of caring for Alzheimer's patients. Representative Andy Harris (R–MD), another physician who has taken an interest in NIH policy, noted at a hearing last March that NIH spends 100 times less per U.S. heart disease death than it does per AIDS death. The “stunning” discrepancy “needs to be justified,” he said.
A matter of proportion CREDITS: (GRAPHIC) A. CUADRA/SCIENCE; (DATA) NIH
Many AIDS research advocates and NIH officials continue to defend the earmark, arguing that the global AIDS death rate remains high, and that researchers are making strides toward a vaccine. “I'm looking forward to a time when … we have ended the AIDS epidemic and there won't be any argument about what you want to do with the money, because you won't need the money,” said Anthony Fauci, the head of the National Institute of Allergy and Infectious Diseases (NIAID), which oversees about half of NIH's AIDS spending, during a hearing this past April. Robert Eisinger, acting director of the NIH Office of AIDS Research, also notes that the billions spent on AIDS research have produced “numerous crossover benefits” for other diseases—new drugs that treat hepatitis B infection came out of AIDS research, for example.
Yet congressional support for the AIDS set-aside has begun to crack. In a report accompanying the 2015 spending bill for NIH, House of Representatives and Senate appropriators omitted some oft-repeated instructions telling NIH to keep AIDS at 10% of its budget. The omission signaled their desire for NIH to abandon the earmark, says Chris Meekins, a staffer for Harris.
WITHIN NIH, top officials are also scrutinizing AIDS spending, about half of which is doled out by institutes and centers that don't focus primarily on infectious disease. Last year, Collins ordered a sweeping review of the AIDS portfolio. One pilot analysis by NIH staff found a surprise: 15% of grants did not have HIV/AIDS in the title or abstract, suggesting they were only remotely connected to AIDS. That finding added to existing concerns, both within and outside of NIH, that requiring institute directors to spend their dedicated AIDS money can encourage them to look far afield for projects to fund, or force them to lower their standards. At NIH's National Heart, Lung, and Blood Institute, for example, 42% of AIDS grant proposals received funding in 2012, compared with just 18% for non-AIDS projects, Cassidy noted at the October Senate hearing. “They're getting too much money for HIV/AIDS,” Cassidy said. “Can we move money out of that area?”
NIH's new AIDS research priorities
Vaccines, new therapies, cure strategies, HIV-associated diseases, related basic research
Basic research and projects on health and social issues that “meaningfully” include HIV/AIDS and “will advance HIV treatment or prevention”
Epidemiology of diseases that occur with HIV/AIDS and basic studies on copathogens that are not in the context of HIV infection, behavioral studies where HIV/AIDS is only one outcome
This past August, in a bid to tighten controls on AIDS spending, Collins announced a revised set of research priorities (see box, right). Vaccines and potential cures are at the top; at the bottom are studies of diseases that sometimes co-occur with AIDS, such as malaria, and basic virology and immunology work that doesn't necessarily include HIV. Also at risk of losing new AIDS funding, the memo suggests, is behavioral research that doesn't focus specifically on AIDS, such as studies of risky sexual behaviors and drug abuse. Although exactly how the new priorities will influence NIH's funding decisions is uncertain, many behavioral researchers are “very nervous,” says epidemiologist David Celentano of Johns Hopkins University in Baltimore, Maryland.
At some NIH institutes, directors have found that disbursing the AIDS money is a distraction from their main mission. Thomas Insel, who stepped down last month as director of the National Institute of Mental Health (NIMH), says he was surprised to realize 3 years ago that the $180 million his institute was spending on AIDS research (12% of NIMH's budget) nearly matched its spending on disorders such as anxiety and schizophrenia. Insel shifted some of the AIDS grants and his HIV/AIDS program staff to NIAID, to ensure better coordination with that institute's bigger AIDS program.
In Congress, some lawmakers are taking direct aim at the earmark. This past summer, during a Senate spending panel debate on NIH's 2016 appropriation, Cassidy proposed taking $235 million out of NIAID's AIDS budget and giving it to other institutes studying neurodegenerative diseases.
The proposal dovetailed with a major push by patient groups to increase NIH funding for Alzheimer's disease, which has helped produce a 30% funding leap for Alzheimer's over the past 4 years. But Robert Egge, chief public policy officer for the Alzheimer's Association, and George Vradenburg, chair of the influential group UsAgainstAlzheimer's, say their organizations did not ask Cassidy to offer his Alzheimer's proposal.
In the end, Cassidy's measure failed. Still, the move shook the AIDS research community. “To find more money for Alzheimer's you have to cut HIV/AIDS, [that] makes no sense,” says Miller, who is part of a coalition of AIDS groups now lobbying to preserve AIDS funding.
THE DEBATE over how much NIH's spending priorities should reflect disease burden goes back to at least the late 1990s, when The New England Journal of Medicine published an analysis suggesting that NIH wasn't spending enough on certain high-burden conditions, such as peptic ulcers and pneumonia. In a 2011 study revisiting the issue in PLOS ONE, Johnston and co-authors found that diseases with strong advocates (breast cancer, for example) enjoy relatively robust funding, whereas spending lags on those that carry a stigma, such as depression, alcoholism, and lung cancer, despite the higher burden they impose on society. Although “you can't expect a perfect correlation” with funding, says Johnston, “the clearest take-home message is: Some conditions below the line are those where we blame the victim.”
This past summer, for the first time, NIH published its own comparison of health burden and spending. It confirmed that AIDS and cancer receive relatively generous funding, whereas others, such as migraine and chronic obstructive pulmonary disease, receive disproportionately little. An agency official commented on a blog that “we're looking forward to using these analyses as a jumping off point for a larger conversation about priority setting.” And Collins has said in hearings that a new NIH-wide strategic plan due out in December will address disease burden.
Some observers are skeptical that the new plan will have much influence, given the many factors that influence spending, from tight budgets to the power of certain disease groups. And NIH officials caution that, in the end, disease burden data, which have limitations, can't alone be the basis for funding allocations. Institutes must consider the scientific quality of proposals, as well as the need to balance basic and clinical research. They note that it makes sense to spend relatively more on rare diseases that otherwise would be neglected, because they can shed light on common diseases. And infectious diseases can't be funded strictly based on the number of U.S. cases, because it's not possible to predict when viruses such as Ebola and severe acute respiratory syndrome—which have had little impact in the United States—might flare up and cross borders. At the same time, pouring new money into a disease just because it imposes a high social cost can be misguided, NIH insiders say. If the field lacks a critical mass of researchers, or promising research avenues, the money could be wasted.
Johnston and others who call for more attention to disease burden don't buy that. “It's a circular argument,” he says. “Scientific promise happens when you have investments in an area. … If you fund the science, there will be more promise in that area.”
Insel agrees, to a point. He says that when he did a disease burden analysis of NIMH and NIH spending on mental illnesses compared with other diseases, he was struck by how every mental illness was “under the line,” or appeared to get short shrift. This year he shored up funding for the two areas that lagged the most—suicide prevention and eating disorders—in order to stimulate fresh approaches. A decision NIMH made a few years ago to ramp up autism research—amid a push from activists—had convinced him that doing so wouldn't be a waste of dollars where there is “scientific traction.” After the infusion of new funding into autism, he says, “we saw better proposals.”
Whether that kind of redistribution catches on across NIH remains to be seen. But some AIDS researchers are already resigned to losing their special status, whether the initiative comes from Congress or NIH itself. “For a long time we were the golden child. … We had a 30-year run,” Celentano says. But now, “I think we need to justify better why we should be at the table.”