On April 30, National Institutes of Health (NIH) director Francis Collins made his annual appearance before the Senate Appropriations Subcommittee on Labor, Health and Human Services, Education, and Related Agencies (Labor-HHS) to discuss the President’s proposed FY 2016 budget request for the agency. The NIH director was accompanied by several institute directors: Anthony Fauci, National Institute of Allergy and Infectious Diseases (NIAID), Douglas Lowy, National Cancer Institute (NCI), Gary Gibbons, National Heart, Lung, and Blood Institute (NHLBI), Jon Lorsch, National Institute of General Medical Sciences (NIGMS), and Tom Insel, National Institute of Mental Health (NIMH).
Subcommittee Chairman Roy Blunt (R-MO) noted that throughout history the practice of medicine has been largely reactive, waiting until the onset of most diseases before being able to treat them or begin the process of curing them. He further acknowledged that science does not fully understand the genetic and environmental factors that cause major diseases and that treatment is imprecise, unpredictable, and often ineffective. Highlighting the fact that the NIH’s budget request included what he called the “revolutionary concept of precision medicine,” Blunt pledged his support for the President’s proposed Precision Medicine Initiative (PMI) (see Update, April 21, 2015) and announced his intent to prioritize funding for NIH. Despite the challenges that the Subcommittee faces in deciding how to allocate funding, the Committee, Blunt declared, “will be supportive of PMI and the ongoing work of the NIH and the promise it holds for the future.”
Subcommittee Ranking Member Patty Murray (D-WA) called NIH’s work “vitally important” to the effort to keep families and communities healthy. She emphasized that all Americans are touched by NIH-supported research. Sharing that she is deeply troubled by the steady erosion of NIH’s purchasing power over the last decade and is concerned about other areas in the budget as they relate to growing a strong middle class, Murray pointed out the budget resolution recently passed by both the House and the Senate falls short of the funding levels needed to address this need. The President’s budget calls for replacement of the automatic spending cuts, also known as sequestration, which allows for a $1 billion increase for NIH in FY 2016, said Murray.
NIH: “Focusing Intensively on Prioritization of NIH Resources”
Collins began his testimony by noting that the NIH, as a federal agency, is acutely aware that in order to achieve its mission it must serve as effective and efficient stewards of the resources it has been given by the American public. To this end, Collins testified that one of the ways that the agency is accomplishing this objective is by “focusing intensively on prioritization of NIH resources.” He explained that this effort “involves developing and applying advanced methods of portfolio analysis, identifying the most compelling opportunities within each institute and center, fostering creative trans-NIH collaborations, and enhancing use of the Common Fund.”
He stated that to support this focus on priority setting, the NIH is developing an overarching NIH strategic plan—which it will link to the individual strategic plans of the agency’s 27 institutes and centers—that reflect the rapid progress in bioscience. According to the NIH director, the plan will be completed by December 2015. In addition, he noted that the NIH is working “to optimize the peer review process to enhance diversity, fairness, and rigor, and the reproducibility of NIH-supported science.” The agency remains “100 percent committed strengthening and sustaining”the scientists it supports by incentivizing early stage young investigators, revitalizing physician-scientists training, and increasing the diversity of the NIH research workforce, he explained. Collins stressed that the NIH is confident that with those goals it will be able to support the best and brightest ideas, while maintaining “the agency’s core mission and inspiring public trust in the world’s premier biomedical research agency.”
The NIH is excited to take the lead role in the multi-agency Precision Medicine Initiative (PMI), with its near term goal of focusing on cancer, Collins noted. He explained that the longer term goal of PMI includes the launching of the “unprecedented national research cohort of one million or more volunteers who will play an active role in how their genetic, environmental, and medical information is used for the prevention of illness and management of a wide array of chronic diseases.” The objective will be to expand the benefits of precision medicine into “myriad aspects of health and health care.” Participants will share data from electronic health care records (EHRs), results of imaging and laboratory tests, lifestyle data and environmental exposure recording tracked through real-time mobile health devices, and genomic information. This will allow researchers to advance the information “derived from this cohort into new knowledge, approaches, and treatments.” The project will also lay the foundation for new prevention strategies and novel therapeutics, the director explained.
Finally, Collins pointed out that the NIH “has lost approximately 22 percent of its purchasing power for research since 2003.” Accordingly, the likelihood that a grant applicant will achieve funding after going through the peer review process “has fallen to the lowest in decades, now less than 20 percent.” The President’s budget request of $31.3 billion, $1 billion and 3.3 percent above the FY 2015 funding level, will put the NIH back on an increasingly stable trajectory. “We have never witnessed a time of greater promise for advances in medicine than right now. With your support, the future of medicine can be very bright,” Collins concluded.
Precision Medicine: One Million Person Cohort
Responding to Chairman Blunt’s inquiry of how the NIH intended to assemble the one million person cohort, Collins highlighted the recent creation of the PMI Working Group that met earlier in the week and will meet monthly between now and August. He announced that the Working Group’s next meeting will examine the question of what the ideal cohort is as it relates to demographics. The agency believes that it can use some of the cohorts that currently exist, including those put together by various health care delivery systems or the Veterans Administration (VA). There will undoubtedly be gaps in terms of representation and NIH wants to be certain that the cohort has the power to inform research on health disparities; the agency will need to figure out how to fill those gaps. The Working Group is expected to begin to make “strong recommendations” by August to allow the NIH to initiate the process of assembling this cohort.
Following up on Blunt’s question, Ranking Member Murray noted that the million person research cohort is very intriguing and emphasized that it needs to be “done right.” She asked Collins how the NIH will ensure that “it successfully represents all elements of the U.S. population, [including] women and minorities.” He replied that the question is exactly what the PMI Working Group is examining. It may require oversampling of certain minority groups to ensure that there is enough representation to have “powerful observations made possible about health disparities.” He added that the agency thinks of the participants of the study not just as subjects or patients, but participants and NIH partners. Collins estimated that it would take at least three or four years to put the entire cohort together, but expects that NIH will begin to learn from it before it is fully amassed.
Senator Lamar Alexander (R-TN), who chairs the Senate Health, Education, Labor, and Pensions (HELP) Committee, stated his intention to make the President’s proposal one of the Committee’s top priorities within its innovation efforts (see Update, March 24, 2015). He questioned how important “a properly functioning electronic medical record systems” will be to NIH’s effort to develop the cohort. Collins responded, “Enormously,” and explained that the agency is counting on using EHRs to make it happen.
Ranking Member of the full Appropriations Committee Barbara Mikulski (D-MD) shared that she and Blunt would like the subcommittee to visit NIH to allow for a more in-depth conversation about the agency. She then turned her attention to the President’s budget request for a $1 billion increase for NIH and asked Collins if that was enough, noting that the NIH had lost 20 percent of its purchasing power due to inflation since the doubling of its budget ended in 2003. Mikulski expressed her concern that despite Collins’ increase in management capability to set priorities, he is going to have to “end up picking winners and losers.” Acknowledging her support for the Precision Medicine Initiative, Mikulski emphasized her “worry about zip code medicine,” asking, “What is it that you truly need to do the job you need to do to serve America while we are trying to do ours?” Noting the reduction in purchasing power and cuts attributed to sequestration, Collins emphasized that the President’s $1 billion increase, would go a long way in putting the NIH back “on a stable upward trajectory.” The increase would allow the NIH to give 1,200 additional grants in FY 2016, added Collins.
Decreasing Rates of Dementia?
Murray also inquired about two studies, one in the U.S. and the other in Germany, which suggest the rates of dementia are is falling. Collins cautioned that there is reason to be skeptical whether one can be completely confident in the studies’ conclusions. To that end, the National Institute on Aging within the NIH is supporting two studies to conduct a rigorous epidemiological analysis to determine whether there is evidence of decreasing incidence or whether some of this is a diagnosis issue.
Early Stage Investigators
Murray also noted that since 2009, NIH has been monitoring the disparities between application success rates for experienced investigators versus early stage investigators and asked Collins what NIH is doing to level the playing field. NIGMS director Jon Lorsch explained that the agency is looking at various ways to address the problem. In addition to targeting the first application of new investigators, he shared the NIH’s concern that a critical stage is also individuals’ renewal application, a significant vulnerability for these individuals and one that the NIH will also examine. He further highlighted the new funding mechanism pilot which provides a single grant per investigator and the plan to introduce a version targeting new investigators (see Update, October 6, 2014).
Senator Tammy Baldwin (D-WI) expressed concern regarding the next generation of innovators and researchers and the significant gap in data on the existing research workforce. She also highlighted the lack of a comprehensive way to track the success of career researchers. She noted that her bill, the Next Generation Research Act, would ensure that NIH accelerates current and new policies to address the issue and foster new researchers. She requested more information regarding why there is not a good system already in place to track information about the biomedical workforce and what steps the NIH is taking to address the gap. Baldwin also inquired about NIH’s efforts surrounding chronic pain, opioid treatment, and alternatives.
Institutional Development Award
Senator Thad Cochran (R-MS) inquired about the NIH’s Institutional Development Award (IDeA) program. Lorsch responded that the program aims to ensure that cutting-edge biomedical research is being conducted in all 50 states. The NIH is committed to the program and thinks that it is an essential part of the NIH’s portfolio, he assured the Senator. Collins explained that the President’s budget request for the program maintains the FY 2015 funding level because the program received an exceptional increase between FY 2013 and FY 2014, and the agency is attempting to normalize its funding trajectory. This effort, however, should not be seen as a lack of enthusiasm for the program, he further assured Cochran.
Prioritization of NIH Research
Senator Jerry Moran (R-KS) pointed out that Congress has deferred to NIH when it comes to the prioritization of medical research, stating, “The theory has been that scientists should make the decisions about where the most promising opportunities are in finding the cure or the treatment.” He questioned whether the NIH “is making the best decisions possible to find cures that are the most readily available and most demanded by our citizens and the population of the world.” It is going to “become incumbent upon Congress to make decisions that are better made by you,” if the “NIH doesn’t do that prioritization,” Moran asserted. Collins acknowledged the concern and referenced the development of NIH’s overarching strategic plan covering all 27 institutes and centers to guide the agency’s priority decisions.
Reflecting on his time as a medical resident, Senator Bill Cassidy (R-LA) noted that the diagnosis of AIDS used to be a death sentence. He questioned the amount of funding dedicated to HIV/AIDS research, referencing a 2011 article that suggested that the principal variable in determining funding was disability-adjusted life years (DALYs). Collins explained that the NIH looks at the public health burden, and DALYs is a very well established way to do that. The agency also looks at scientific opportunity “because it is not going to be successful to throw money at the problem if nobody has an idea about what to do about it,” Collins stated. NIH also looks at what the peer review process is telling it about the “excellence of the science,” he explained further.