CQ WEEKLY – COVER STORY
June 2, 2012 – 2:57 p.m.
Rebranding the NIH
By John Reichard, CQ Staff
Who better than Francis Collins to convince appropriators of the power of medical research to alleviate suffering and save lives? Every Monday from his post atop the National Institutes of Health, Collins surveys cutting-edge developments across all of biomedical research, much of it funded out of his agency’s $30.7 billion budget and even coming out of the labs of his own in-house scientists.
Collins has plenty of expertise based on his own work as a scientist as well. As head of the National Human Genome Research Institute at NIH, Collins led the international effort that in 2003 successfully “sequenced” all the genes in the human body. The feat has been compared with obtaining all the pages of a manual needed to make a human being.
In short, Collins possesses an understanding shared by few others of how the human organism can go awry — and of how to try to redirect in more benign fashion the biological processes leading to thousands of diseases. Like his predecessors, he is well-equipped to explain the potential of medical science to lessen suffering and save lives.
In years past, that was enough to secure appropriations abundant enough to help make NIH the world’s premier biomedical research agency. Now, in today’s climate of fiscal austerity, Collins is worried that the message needs a boost.
NIH is facing the prospect of deep cuts now and for years to come. Like other federal managers, Collins is reworking his budget pitch to emphasize how much is at stake in funding his agency at desirable levels, and of the dire consequences of failing to do so.
Increasingly, he’s describing NIH not just as a powerful force in the war against disease thanks to its focus on basic research, but also as an incubator of new jobs and new products.
As the nation struggles to spur growth and cut debt, he is presenting medical research as key to economic renewal.
And just like boosters of other agencies, NIH supporters are playing the Asia card, citing the vast increases in spending on medical research by China and India as U.S. spending flattens out.
“In this climate it almost seems like it’s not enough to say what we’re about is to improve human health,” Collins told medical research organizations in May. “We also have to justify expenditures coming from the taxpayers in terms of how those will boost our sagging economy.”
Other agencies, such as NASA and the Pentagon, figured out years ago that it wasn’t enough to do their jobs — they had to create jobs, too — in order to get consistent congressional support for their programs, particularly during hard times.
But scientists have tended to shy away from predictions about how and when their work will pay off. And to those accustomed to such reticence, the message Collins is now delivering on Capitol Hill may seem odd.
While it isn’t quite the pitch a CEO would make to institutional investors during a Wall Street road show — he isn’t specifying the new products he’ll develop, when they will come on the market and how much they’ll generate in sales — his bid to woo a bigger taxpayer investment is similar. Like a CEO, he’s trying to attract money by promising concrete results for investments made in his organization.
Rebranding the NIH
Along with major universities and other organizations that benefit from the agency’s funding, Collins is emphasizing that NIH is an economic engine.
The greatest and most immediate threat to NIH comes from the 2011 Budget Control Act that mandates automatic cuts to discretionary spending starting in 2013, slashing an estimated 8 percent from NIH’s current annual appropriation. Stopping those across-the-board cuts is a challenge only Congress can meet.
In the event that Congress overrides the law, it will probably pick and choose favorites throughout the government. That’s where the efforts of agency heads would be most likely to pay off, if only around the margins, as well as in years to come when the law gives Congress greater flexibility in allocating cuts.
The NIH, already with a large bipartisan fan base on the Hill backed by lobbying from many disease groups, starts with an advantage. But Collins still has plenty of work to do.
He has made his case on and off Capitol Hill throughout the spring. He’s done it in testimony before House and Senate Appropriations subcommittees in late March, in forums in April and May sponsored by private groups that lobby for medical research dollars and in an appearance with pharmaceutical executives at the National Press Club. He also appeared at events sponsored by United for Medical Research, a coalition of universities and patient advocacy groups, and the Information Technology and Innovation Foundation, a nonpartisan think tank funded by various businesses.
Collins has sounded two themes in his appearances: He has sought to inspire appropriators about the bounty to be reaped from a bigger investment in NIH. At the same time, he has voiced alarm about the fate of the American medical research enterprise even as he jingles the keys to a vast treasure chest of promising research leads.
Jobs and Jobs
And, who better for Collins to quote than the late Steve Jobs to make lawmakers believe in the great economic potential of medical research? The Apple co-founder described his stunningly successful products as laying at “the intersection of technology and the liberal arts.” Collins reminded lawmakers in the House and the Senate of how Jobs updated that quote shortly before his death in 2011.
Jobs told biographer Walter Isaacson that after his cancer diagnosis, his son Reed began spending summers in the Stanford University oncology lab. The younger Jobs worked on sequencing DNA to find genetic markers for colon cancer.
“One of the very few silver linings about me getting sick is that Reed’s gotten to spend a lot of time studying with some very good doctors,” Jobs said. “His enthusiasm for it is exactly how I felt about computers when I was his age. I think the biggest innovations of the 21st century will be the intersection of biology and technology. A new era is beginning, just like the digital one when I was his age.”
Jobs “was spot on,” Collins told senators.
Collins also gave examples of some of the amazing products that may soon emerge from the work at NIH, a standard part of the agency’s annual show-and-tell before the people who hold the purse strings.
Rebranding the NIH
“One striking example is the cost of sequencing a human genome,” Collins said, adding that it won’t be long before individuals will be able to have their own genomes sequenced. Doctors then will be better able to tell which diseases an individual has or is likely to develop. They’ll be able to urge lifestyle changes to prevent a disease, or, if treatment is needed, to prescribe the most effective drugs given a person’s genetic makeup.
“Eleven years ago, it cost $100 million; five years ago, $10 million; today, less than $8,000 and heading down,” he said. Within the next year or two, “a couple of U.S. companies plan to sell machines that sequence a genome in a single day for a thousand dollars or less using devices . . . the size of a postage stamp.”
Another example cited by Collins: taking an individual’s skin cells and tweaking them to become “stem” cells capable of growing into various types of tissues in the human body. These cells can be induced to become cells affected by a particular medical condition, such as Lou Gehrig’s disease.
Creating such cells in a petri dish makes it much easier to understand how a disease damages the body and to screen various compounds to see which ones might affect the ailment. “This technology is now being used to develop exciting new models of disease, the so-called diseases in a dish that are expanding our understanding of human biology as well as opening the door to new treatment possibilities,” Collins said.
He also recently has described potentially major breakthroughs in the treatment of Alzheimer’s disease. Bexarotene, a drug originally developed to treat cancer, dramatically reduces levels of amyloid plaque in mice, he said. Some scientists think that the plaque is responsible for destroying brain function.
Scientists also have learned that an abnormal protein called “tau” advances neuron by neuron in the brain. “The neurons are not just committing suicide, they’re also committing homicide on their neighbors,” Collins observed. If that process can be stopped, then Alzheimer’s could be slowed or prevented, he says.
But a prediction by a celebrated innovator and examples of fascinating new technology aren’t enough to sway the minds of budgeteers in Congress and the White House. Collins warns that anecdotes and what he calls hand-waving arguments — those that lack rigor and substance despite a showy presentation — aren’t going to work in today’s budget climate.
Mindful of the challenge, the nation’s leading research centers and patient groups have commissioned a series of studies to build the case for a steady increase in the NIH budget that avoids the kind of feast-or-famine grant funding that makes medical science an uninviting career choice despite its dazzling promise. It’s research that Collins has cited repeatedly in his public appearances in recent weeks.
According to one such study underwritten by United for Medical Research, NIH funding in 2011 directly or indirectly supported 432,000 American jobs. It fueled job growth of 10,000 or more jobs in 15 states and generated $62 billion in economic activity.
The basic medical research performed by NIH undergirds innovation in the private sector, the study added. Citing an analysis in 2000 by Congress’ Joint Economic Committee, the study said, “The NIH research portfolio expands the life sciences knowledge base, which in turn creates opportunities for private research to build on this base — akin to passing a relay race baton from NIH-funded research findings to the commercial research agenda of pharmaceutical companies and other private entities.”
The JEC study cited earlier findings that of 32 innovative drugs introduced before 1990, 60 percent would not have been discovered or would have been markedly delayed without NIH research support.
“NIH serves as the foundation for the entire U.S. medical innovation sector that employs 1 million U.S. citizens, generates $84 billion in wages and salaries, and exports $90 billion in goods and services,” Collins told the Senate Labor-HHS-Education Appropriations Subcommittee on March 28.
Partnering With Big Pharma
Rebranding the NIH
Lest there be any doubt about NIH’s importance to the private sector, Collins also can talk about a new deal with the pharmaceutical industry.
In early May, Collins joined with executives from three of the world’s biggest drugmakers to announce that NIH is offering funds to academic researchers to further develop experimental drugs shelved by the companies despite passing muster in human safety trials.
Drugs that don’t pan out for one use sometimes work well for another. However, companies often aren’t willing to invest in exploring other uses.
Under the initiative, NIH is soliciting grant applications from university researchers and other scientists outside the original company who have a hunch that a drug might work for another use and are looking to test it. The company donating the drug retains ownership rights, but grant winners have intellectual property rights to their studies and can publish the results of their work.
If the outside tests show promising results for another use, the company that owns the drug would be expected to step in and fund final testing and negotiate an agreement with the grant winner on royalties from subsequent sales. The deal, described by one NIH official as unprecedented, is, in effect, a recognition that private sector drug development could be much more productive with some federal research dollars thrown in.
Collins said researchers now know the molecular basis of 4,500 diseases but have effective treatments for only about 250. “Clearly, we need to speed the pace at which we are turning discoveries into better health outcomes,” he said.
Collins seemed exhilarated by the partnership, in which Pfizer, Eli Lilly and AstraZeneca are putting up about 20 compounds for outside study. “Especially now when resources are quite tightly constrained, for the public sector, for the private sector, coming up with more creative ideas about how we can do this more efficiently and more cost effectively, is something we think about every day. I’m just delighted that we’re here today.”
Currently, the partnership is limited to testing drugs that haven’t gone beyond phase one safety testing. But companies also discontinue research on drugs that have shown signs of being effective for a particular use but are set aside because of a change in markets. Those products as well could eventually be donated by drugmakers.
NIH officials voiced optimism that the number of participating companies and products will grow. Collins says it’s a boon to the agency because a number of medical breakthroughs have come from testing new uses of drugs that haven’t at first panned out. One example is AZT, the first drug approved by the Food and Drug Administration to lengthen the lives of those infected by the AIDS virus. It originally was a bust as a cancer treatment.
The venture is just a small part of the activities of a $500 million center recently launched at NIH to get more products coming out of drug and device industry pipelines.
The new National Center for Advancing Translational Sciences has a jargony name, but what it means is that too many promising discoveries in the lab never lead to new drugs and medical devices — and that NIH thinks it can find ways to remove those bottlenecks in development.
One such impediment is the time it takes to figure out whether a particular drug compound is safe and effective. To shorten that process, scientists funded by NIH and the Defense Department aim to develop a chip that mimics how humans respond to a drug. They’ll spend the next five years working with each other to place 10 different types of human tissue on a chip so they’ll interact with drugs in the lab the same way they do in living patients.
Rebranding the NIH
“By providing a better model to predict drug safety and efficacy, the most promising drug candidates can be identified more quickly and moved forward into development,” Collins testified March 28.
But Collins also warned in his recent appearances that an agency brimming with potential to generate jobs and products is at severe risk if its budget is cut 8 percent next year under the budget control law.
The impact on research would be “devastating,” he told a March 14 conference sponsored by Research America. NIH officials estimate that if their budget stays flat next year they will be able to fund 9,415 research grants. An 8 percent cut would mean funding 2,300 fewer.
Since Congress doubled the NIH budget over a five-year period that ended in 2003, “we’ve effectively lost 20 percent of our purchasing power for medical research as inflation has eroded a flat budget,” he said.
“That means that an investigator who comes to NIH with their best ideas, seeking to get supported for the work they’re doing at one of our great universities ... has now only one chance out of seven [of] getting supported. And that is the lowest it’s been in history.” If the cut occurs, the odds will get worse. Collins said “the temptation therefore is to do something else. And if we lose this generation of young researchers because they get discouraged, they’re not going to come back.”
Dan Perry, executive director of the Alliance for Aging Research, confirmed the trend, saying that with flat budgets, “It’s almost like getting struck by lightning that you’re going to get funded to do research.”
A predictable upward trajectory in funding would be “enormously supportive of the enterprise,” Collins told research groups in May.
There are reasons to question the pitch Collins is making. The members of United for Medical Research all have a financial stake in increased funding so it’s not a disinterested source for job estimates.
Also, it can take a very long time for breakthroughs to emerge from advances in basic research such as gene sequencing and the development of embryonic stem cells — two recent advances that patient advocacy groups point to as potentially major new sources of medical breakthroughs.
Progress from sequencing the genome to applying gene science to health is slower than many would like, Anindya Dutta of the University of Virginia told CQ Researcher in a 2011 interview. “Ten years from now we’ll have a better understanding of our bodies, in 20 years we’ll better understand disease and in 30 years, we’ll begin to have therapies,” Dutta said.
Also, lawmakers must choose among painful alternatives as they try to reduce deficit spending. They must weigh whether cuts to programs ranging from Medicare to Medicaid to food stamps to defense would be even more damaging than those to NIH. But the early signs are that the Collins pitch is working and that NIH is one agency lawmakers want to shield from cuts. “I believe that NIH funding should be a priority and that its benefits extend well beyond its research discoveries,” said Sen.
Rebranding the NIH
Both Shelby and the panel’s Democratic chairman,
House Republicans also show signs of wanting to protect the NIH budget, although how many favor doing so is not yet clear.
A March 20 letter to top House appropriators from 154 House members — a dozen of them Republicans — urged that the NIH budget be bumped up from $30.7 billion this year to “at least $32 billion” in fiscal 2013.
Several GOP lawmakers on the House Appropriations Committee have cited NIH as an important booster of their state economies or as an important agency to fund. Rep.
“If we’re going to spend federal dollars, we ought to spend those federal dollars in a way that has the greatest impact on the economy and that has the greatest impact on the future prosperity of the country,” he said. “I can think of really no better way to do that than investment in research.”
GOP lawmakers at the March 20 House Labor-HHS-Education Appropriations Subcommittee hearing defended the NIH’s Institutional Developmental Award (IDeA) program, which ships medical research dollars to 23 states with poor track records of competing for NIH grants. They objected to an Obama administration proposal to shift to other parts of NIH $50 million of the $275 million program.
Fred Taylor, who heads the program at NIH, says it begins to change the workforce in states such as West Virginia. IDeA grants fund the research of veteran scientists who do have a track record of winning competitive NIH grants. These scientists mentor other young scientists so they, too, can compete successfully for NIH money.
“Then the scientists need technical people,” Taylor explains. “So then they start training technical people in order to take the jobs that are coming open in the labs. And then we’ve seen in a lot of places the spinoffs of medical industrial parks, where companies begin.”
Challenge From Abroad
At best, it appears that NIH is on track for flat funding next year or an increase of about $1 billion dollars.
That’s not the way U.S. competitors are treating their medical research budgets, even as they struggle with their own debt woes. Collins said that he meets regularly with the heads of government medical research agencies of other industrialized countries, all of which are increasing their budgets.
The budgets of China and India next year will see double-digit percentage increases — in the case of China, 25 percent. “I’m the only guy sitting at the table going, ‘Well, we’ll be lucky if we’re flat,’” Collins said.
Rebranding the NIH
A report issued by the two sponsors of the meetings (United for Medical Research and the Information Technology and Innovation Foundation) said that the Chinese government plans to invest $309 billion in biotechnology over the next five years, dwarfing the $160 billion projected for NIH over that period under current trends.
“A growing number of countries, including China, Germany, India, Singapore, Sweden, the United Kingdom and others have recognized that life sciences represent a high-wage, high-growth industry and have taken measures seeking to wrest life sciences leadership from the United States,” the report says.
Other countries invest more than the United States in biomedical research relative to the sizes of their economies, it adds, urging that the United States boost its share to 0.25 percent of GDP, which would be about $40 billion, compared to $30.7 billion now.
Collins told research groups that since the genome-mapping project was completed in 2003, “We have basically ceded leadership in genomics... to other places, and particularly to China.” He said that “our dominance of biomedical research cannot be taken anymore as a given. It is under serious threat.”
Visiting Shenzhen, a city north of Hong Kong, Collins said, “You will see there a remarkable facility with 4,000 of the smartest Chinese scientists that you will ever hope to meet all working together in a renovated shoe factory that’s been turned into an absolute marvel of technological achievement and is populated not only with people but with instrumentation which exceeds anything that we have in the United States. And that happened so quickly that probably people hardly even noticed it was happening. And that’s just one example.”
One apparent flaw of the report is that it does not factor in the large U.S. private investment in the life sciences industry and only compares amounts spent by governments. Research and development expenditures by the U.S. life sciences industry — which includes biotech, medical device, medical instrument and pharmaceutical companies — will total $73 billion in 2012, according to a December 2011 study by the Batelle research group. Adding in the $30 billion or so spent on NIH, that pushes the public-private investment in the United States to around $100 billion and makes the Chinese investment seem less overwhelming by comparison.
But given the successes of the Chinese government in targeting other areas of the economy for growth, it’s fair to say that U.S. leadership in biomedical research cannot be taken for granted unless Congress undertakes a new round of aggressive NIH funding.
So far, neither Republican nor Democratic leaders of Congress are showing any indications of taking up that challenge, with some GOP and industry leaders boosting the notion that the private sector can be counted on to do the most fruitful research and development.
It may take drug company executives coming forward in a much more public way to say how important NIH is to their own innovation efforts to begin changing minds on Capitol Hill.
FOR FURTHER READING Labor-HHS-Education appropriations, CQ Weekly, p. 869; health budget, p. 354; health research, 2010 CQ Weekly, p. 1972; health care overhaul, 2010 Almanac, p. 9-3.