NIH Appears Before Senate Appropriations Subcommittee

On April 7, National Institutes of Health (NIH) Director Francis Collins appeared before the Senate Appropriations Subcommittee on Labor, Health and Human Services, and Education (Labor-HHS), accompanied by five of the NIH’s 27 institute and center directors and/or acting directors. Attending were: Richard Hodes, National Institute on Aging (NIA); Doug Lowy, National Cancer Institute (NCI); Nora Volkow, National Institute on Drug Abuse (NIDA); Walter Koroshetz, National Institute of Neurological Disorders and Stroke (NINDS); and Christopher Austin, National Center for Advancing Translational Sciences (NCATS).

Opening the hearing, Subcommittee Chairman Roy Blunt (R-MS) expressed his concerned with the NIH’s budget request, which he noted reduces “discretionary funding for medical research by a billion dollars,” as a result of including mandatory spending. Noting the $2 billion increase Congress provided the NIH in its FY 2016 budget—“the first significant increase in over a decade”—Blunt emphasized that at the end of the appropriations process he hoped the committee would not “look at what the administration’s asked for but look at what [the Subcommittee] would hope to be able to do.” Blunt announced that he and Senator Lamar Alexander (R-TN), chairman of the Senate Health, Education, Labor and Pensions Committee (HELP), have discussed short-term mandatory or “surge” spending for specific projects (see related story). Noting the Congressional Budget Office’s (CBO) estimate that mandatory spending is expected to increase from 13 percent of gross domestic product to 15 percent, Blunt emphasized the need to put “a structure together” that looks at priorities. He also noted that following last year’s “significant increase” in budget to NIH that it was “time to again make research funding a national priority.”

Observing that the NIH budget accounts for the largest share of the subcommittee’s resources, Ranking Member Patty Murray (D-WA) noted that science is on the “cusp of major breakthroughs” on a number of illnesses and conditions, emphasizing the importance of providing researchers and scientists the tools they need. Murray shared that in her discussions with Alexander about their legislation “to advance medical innovation in the health committee,” she has stressed the need to take “advantage of every funding opportunity including mandatory investments in the NIH.” She further shared her desire for the Subcommittee to write a bipartisan FY 2017 appropriation bill and underscored her commitment to working towards a bipartisan agreement on their innovation package.

Collins thanked the Subcommittee for the $2 billion increase in the FY 2016 Omnibus Appropriations bill, noting that the increase “energized” the scientific community and “came in an unprecedented time of scientific opportunity.” As he did before the House Subcommittee, Collins reflected “broadly on NIH’s contributions to the nation’s health.”

Blunt began the questioning portion of the hearing by asking Collins what would happen if the Subcommittee cut the NIH’s research budget by $1 billion. Collins answered that “losing a billion dollars for medical research at the present time would be devastating.” Such a cut would require the agency to cut the number of new and competing grants available by “a very substantial number.” He stressed, “Great ideas that scientists are putting forward would go unsupported.” With regard to NIH’s support of young researchers, Blunt asked Collins to describe for the Subcommittee what the agency is “seeing in the research community generally and young researchers specifically.” Collins responded that NIH has created a new program that allows “the most talented graduate students to skip the post-doc” and move directly into an independent investigator position. He also highlighted the NIH’s loan repayment program (LRP) which has allowed the agency to attract more physicians into research, noting pending legislation that would authorize an increase in the amount available for loan repayment from $35,000 to $50,000. According to Collins, NIH is ensuring that new and early stage investigators compete against each other, thereby increasing their priority scores, adding that the participants in the program are not all “young scientists.” Some of them are scientists who have worked in other fields and are now moving into biology, he explained.

Ranking Member Murray noted the Vice President’s Cancer Moonshot Initiative has increased awareness of some of the barriers to sharing data among cancer researchers and asked Collins to describe how NIH has fostered better collaboration and the ensuing effect of such collaboration. Collins responded that the agency is “very intensely focused on the idea of data sharing.” Using Alzheimer’s disease research as an example, NIA Director Richard Hodes noted that efforts in this area reflect understanding from the public and private sectors regarding common purpose and the advantage of working together “to ultimately accomplish a common goal of finding diagnostic and therapeutic interventions.” Replying to Murray’s follow-up question on the big data challenges facing researchers, Collins stated that the “NIH has major investments in the space of Big Data.” Recognizing that this would be “a major need and a potential threat,” he explained that the agency has put together a plan resulting in $100 million of spending on its Big Data to Knowledge (BD2K) initiative, which aims to take large data sets and develop appropriate standards. He added that the initiative is also a major source of training for the next generation of data scientists.

Research Priorities

Ranking Member of the full Appropriations Committee, retiring Senator Barbara Mikulski (D-MD), said that representing the NIH, which is headquartered in her state, has been one of the greatest joys of her time in the Senate. She echoed the other Subcommittee members’ concerns regarding mandatory funding for NIH. She asked Collins for his professional judgement of what NIH needs. Collins replied, “a stable trajectory of inflation plus five percent for multiple years in a row would be a wonderful way to support medical research in the way that it needs to be.” He acknowledged that the agency’s budget would double in seven or eight years at such a rate. Continuing the line of questions, Mikulski asked, “What would we get for the money?” She noted that while she supports the “new ideas” included in the FY 2017 budget request, she wanted to know the most promising areas in existing programs. Collins responded that “great promise” resides in “diabetes, heart disease, cancer, autism, vaccines, and Alzheimer’s disease.”

Noting that he and Collins have discussed how to prioritize spending within NIH, Senator Jerry Moran (R-KS) stated his belief that spending decisions about what research is funded are “best made by science and medicine.” But he asked Collins to comment on NIH’s process for determining where to increase spending when it means needing to reduce spending elsewhere. Collins answered that NIH faces difficult choices every day, “especially over the course of the last decade or more where success rates for grants have been the lowest in history, below 20 percent.” He lamented having to turn away science that is “exciting” and “well-designed.”

Regarding priority setting, NINDS Director Walter Koroshetz cautioned the committee to think about the importance “of the foundation of basic research,” noting that it is hard to predict where the next discovery will come from.

NIH Innovation Bill

HELP Committee Chairman Alexander expressed his appreciation to Blunt and Murray for their leadership last year in providing the “significant increase” in funding to NIH. Noting that he “was glad to support” their efforts, he shared that he did not think there was one person on the Subcommittee “who does not hope that they can find ways to make it a pattern this year and in the future.” Pointing out that the HELP Committee completed its work on its innovation companion bill to the House’s 21^st Century Cures bill the previous day, Alexander announced that the Subcommittee’s remaining task is to determine how to pay for the bill. He asked Collins if the five priorities included in the innovation bills—precision medicine, Cancer Moonshot, BRAIN Initiative, the Young Investigators Corps, and the Big Biothink Award—were still NIH priorities and if the NIH could support the initiatives without creating so-called funding cliffs. He also wanted to know what the agency would do about oversight “in terms of a strategic plan for each of those five areas.” Collins responded that for those five areas the agency could within a few years “identify components that could be nicely supported through this mechanism and would not result in a cliff.” NIH would “expect to have appropriate oversight about how those dollars would be spent.” The agency would be happy to submit a work plan—and be held accountable for the plan—over the course of the coming years.

Precision Medicine Initiative

Senator Bill Cassidy (R-LA) asked Collins about NIH’s “business plan” for the Precision Medicine Initiative (PMI). He specifically asked Collins how NIH plans to recruit a million people from all socioeconomic groups, noting the substantial expense of such a longitudinal study. He also wanted to know if private entities would have to pay to access the data and if those who contribute their data would benefit as well. Collins answered that the NIH has deliberated as to how it is going to enroll a million people and have the data be generalizable for the U.S. population, noting that participants will include individuals who are currently involved in health provider organizations and have applied to be a part of the initiative. That, however, will not be sufficient to ensure that the initiative is “fully diverse in terms of socioeconomics and ethnicity of the country,” Collins acknowledged. Consequently, NIH will have to do outreach to community health centers that are supported by the Health Resources and Services Administration (HRSA). Moreover, Collins explained, anybody can volunteer to take part in PMI beginning in August or September. The data will be made available to any qualified researcher. The pharmaceutical industry will have the opportunity to learn from the initiative, said Collins, noting that he does not see “a great alternative.” It would be hard to erect a barrier without “slowing down the progress of the research overall,” he explained. Regarding whether participants would receive financial benefit as a result of participation, Collins noted that the general position by bioethicists is that providing a financial incentive “might actually be a disservice” and may be seen by potential participants as an “inducement” to take part.

Senator Thad Cochran (R-MS) inquired about the Jackson Heart Study, which involves a research cohort of 5,300 in Jackson, Mississippi, and whether the study will be part of the national research cohort. Collins answered that the Jackson Heart Study has “already taught us a great deal about what those environmental, genetic, and social and behavioral risk factors are turning out to be,” and the 5,300 individuals who have volunteered to be a part of that study. The NIH is continuing to look for ways to connect PMI efforts with other kinds of studies, Collins explained, noting his hope that those who have already taken part in research studies would be particularly interested in the precision medicine initiative.

Murray expressed concern about the diversity of the million person cohort and asked Koroshetz how the NIH would ensure diversity, including “working with experts” to avoid the “risk of bias” that could affect the cohort’s usefulness. Koroshetz explained that it means NIH needs to “have overrepresentation of groups that are traditionally underrepresented,” which means minorities and individuals from lower socioeconomic status.

Opioid Abuse/ Federal Pain Research Strategy

Senator Shelly Moore Capito (R-WV) noted that West Virginia has been hit particularly hard by the opioid abuse epidemic. She asked NIDA Director Nora Volkow to elaborate on NIDA’s partnership with the Appalachian Regional Commission to improve opioid intervention services. Volkow replied that the partnership is to create the infrastructure that would allow NIDA to launch a research project and provide information about the nature of problems in the state related to injection drug use, the associated consequences, along with the type of medical infrastructure that could be deployed. Volkow explained that, unfortunately, this information is not currently available and is not integrated as to allow NIDA to deploy the type of implementation research that the institute is interested in conducting to contain the injection drug use epidemic in that area. Currently, the information collected related to opioid overdoses varies by state, she explained. Further, there is consensus that we are underestimating the number of deaths associated with the epidemic.

Senator Jean Shaheen (D-NH) followed up on Capito’s question regarding the heroin and opioid epidemic. Noting that New Hampshire is currently experiencing the “highest percentage of overdose deaths of any state in the country,” Shaheen asked Volkow about NIH resources to assist those on the front line of the epidemic. Volkow explained that as an institute and the NIH, research is the way that the agency has responded and by providing products to address and prevent overdoses from opioids. She cited the drug Intranasal Narcan as an example of a product approved by the Food and Drug Administration that can overcome an overdose. Volkow emphasized, however, that it is not just the issue of deployment but a lack of structure available for patients to take advantage of the treatment, a problem especially in rural communities. Accordingly, NIDA is working with pharmaceutical companies to develop medications that would be easier to adhere and require less infrastructure to sustain. Additionally, the institute is supporting implementation research to discern how to take advantage of the health care system so that “they are involved in the deployment of medical assisted therapy for individuals with the opioid use disorder because it has been shown to prevent overdoses.”

Capito also inquired about efforts to find a non-addicting pain medication. Koroshetz explained that one of the major challenges within the NIH’s Neuroscience Blueprint is to develop products that would prevent the acute to chronic pain transition that they believe occurs in brain circuits after someone has been exposed to acute pain.

Noting that the NIH was working on the Federal Pain Research Strategy, Senator Brian Schatz (D-HI) wanted to know when the strategy would be completed and launched and who would be appointed to the committee. Koroshetz responded that the issue was complicated as there are several documents out there. There is the National Pain Strategy, which directs the U.S. medical care system on how to improve pain care in both a safe and effective manner. There is also the National Pain Research Strategy, which is ongoing and scheduled to be completed in January 2017. Scientists, individuals who provide care for pain, and the pain patient will convene to work out the highest priorities in the different areas.

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