Appropriations Subcommittee Statement on the Fiscal Year 2019 Budget

Prepared Statement of Jon R. Lorsch, Ph.D. Director, National Institute of General Medical Sciences (March 30, 2018)

Mr. Chairman and Members of the Committee: I am pleased to present the President's Fiscal Year (FY) 2019 budget request for the National Institute of General Medical Sciences (NIGMS), a component of the National Institutes of Health (NIH).

The NIGMS focuses on promoting and supporting fundamental or "basic" biomedical research that increases scientific knowledge about how living systems work, from individual molecules to cells, organs, whole organisms, and populations. The Institute's priority is to support investigator-initiated research, based on the principle that the best scientific ideas, directions, and approaches stem from scientists themselves. Thus, NIGMS aims to enable creative, innovative, and ambitious research conducted by individuals and teams of investigators to promote scientific discovery and medical advancement.

One indication of the success of the Institutes' strategy is the number of Nobel Prizes awarded to NIGMS grantees. Of the 153 Nobels given for NIH-funded research, over half—87—have been for work supported by NIGMS. This year, the Nobel Prizes in both chemistry and in physiology or medicine were awarded to multiple NIGMS grantees. The 2017 Nobel Prize in chemistry was awarded to a NIGMS grantee and two others for the development of cryo-electron microscopy (cryo-EM), a technique that simplifies and improves the imaging of biomolecules. Similarly, the 2017 Nobel Prize in physiology or medicine was awarded to three NIGMS grantees for their work on molecular mechanisms controlling circadian rhythms, more commonly known as "biological clocks." Biological clocks influence a variety of physiological responses such as alertness, hunger, metabolism, fertility, and mood; clock dysfunction is associated with various disorders, including insomnia, diabetes, and depression. These awards serve as yet another testament to how investing in the study of fundamental biological processes can yield important insights into the principles that underlie human biology, health, and disease.

Because scientific breakthroughs generally cannot be predicted in advance and often originate from unexpected or disparate strands of knowledge, a cornerstone of NIGMS' strategic plan is to support a broad and diverse portfolio of fundamental research. This strategy builds the strongest possible foundation on which breakthroughs can arise.

NIGMS Strategic Priority: New Funding Approaches to Accelerate Scientific Progress and Discovery

In order to enhance the efficiency and productivity of fundamental biomedical research, the NIGMS has developed a new mechanism to fund scientists. The Maximizing Investigators' Research Award (MIRA) seeks to transform how fundamental biomedical research is supported by providing individual investigators with a heightened level of both scientific stability and flexibility. These awards allow investigators to follow new research directions and insights in real-time, while simultaneously providing an extra year of support as part of a more coordinated scientific program (versus single project) focus. In addition, the peer review process for MIRA applicants reviews early stage investigators (ESIs) independently from established investigators (EIs), thus allowing each group of applicants to be examined relative to their own peer group. Since the creation of the program, the NIGMS has awarded 231 MIRAs to EIs and 192 MIRAs to ESIs. The MIRA program is especially beneficial for ESIs, as evidenced by the increase in the number of ESI applications from 393 in 2015 (prior to MIRA) to 649 in 2017. The number of ESIs funded by the Institute per year has nearly doubled in the same time period. The NIGMS will continue to monitor the progress and outcomes of the MIRA program as it evolves.

Because some areas of biomedical investigation require groups of investigators to synergistically work together to solve complex problems, the NIGMS recently developed and implemented a new team-science support mechanism known as the Collaborative Program Grant for Multidisciplinary Teams.2 This investigator-initiated funding opportunity supports multidisciplinary teams of researchers to work toward a shared goal that has the potential to have a major impact on one or more fields of biomedical research. Because this program only funds highly integrated research teams working toward a common objective, it will promote and enable a type of collaborative science that can't easily be supported through other kinds of research grants. Further, these new awards possess an optional component that allows for support of pilot projects by early-stage investigators to develop research in the team's area(s) of expertise with a goal of helping the junior researchers obtain independent funding.

NIGMS Strategic Priority: Develop and Sustain a Highly Skilled, Diverse, and Productive Biomedical Research Workforce

The NIGMS plays a leading role in supporting the career development and training of the next generation of scientists, including the development of institutional research capacities in regions across the country in which the levels of NIH support have been historically low.

Given the rapidly evolving landscape of science and medicine, the Institute is working to catalyze the modernization of graduate education. Recently, the NIGMS issued a new notice of funding opportunity (NOFO) for its pre-doctoral T32 training grants3 that focuses on addressing several key issues such as: shifting the emphasis from simply teaching scientific "facts" to teaching both scientific and professional skills; developing the acumen needed to become rigorous and responsible scientists; supporting a safe, inclusive and diverse training environment; promoting the use of evidence-based teaching and mentoring practices; and enhancing student career development. Over the next several years, the NIGMS will work to implement this revised T32 program and will carefully evaluate its outcomes. In addition, the NIGMS is also working to ensure that emerging issues can be quickly incorporated and addressed in the training process, as appropriate. For example, the Institute has recently supported the development of open-access curricular training modules in areas related to improving the rigor and reproducibility of biomedical research. These modules are available on the NIGMS website, 4 with more modules due to be added this year. The goal is to generate useful resources for graduate training programs.

Through its Institutional Development Award (IDeA) program, the NIGMS provides targeted support to help broaden the geographic distribution of biomedical research funding by enhancing the competitiveness of investigators located at academic institutions in states having a historically low level of NIH support. The IDeA Centers of Biomedical Research Excellence (COBRE), for instance, support thematic, multidisciplinary centers that expand and develop faculty research capabilities and research infrastructure, in part through the development of core technology facilities needed to carry out modern multidisciplinary collaborative research. Another important aspect of the IDeA program is its support of medical research for rural and underserved communities. The IDeA Clinical and Translational Research Network (CTR), for instance, provides support for clinical and translational research that addresses conditions that have been traditionally higher among certain regions, populations or communities, including (but not restricted to) cancer, cardiovascular disease, and substance abuse disorders.

Because developing a well-trained research workforce begins with early outreach and education, the NIGMS was proud to welcome the NIH Science Education Partnership Awards (SEPA) to the Institute in 2017.5 SEPA supports diversity in the workforce by providing opportunities for students (specifically at the pre-kindergarten to grade 12 levels) from underserved communities to learn about careers in basic or clinical research. Ten of the fourteen SEPAs in IDeA states are currently in partnerships with IDeA COBREs or IDeA Networks of Biomedical Research Excellence (INBREs). An Institute goal is to fund at least one SEPA in every state.

NIGMS Strategic Priority: Create and Share Cutting-Edge Tools and Resources

Given that technology plays a critical role in biomedical research, the NIGMS continues to place a high level of significance on supporting the development and dissemination of innovative, new technologies that have the potential to transform research. To this end, the NIGMS supports a multi-phased funding strategy that consists of support for an initial proof-of-concept phase for a given technology followed by potential support for a second prototype refinement phase. These phases can then lead to the adoption and expansion of the technology via commercialization through small business innovation research or small business technology transfer (SBIR/STTR) grants. Technologies can also be applied toward answering specific scientific questions through their incorporation into regular research project grants or, once sufficiently mature, through their incorporation into resources supported by the Institute's Biomedical Technology Research Resources (BTRR) program.6 Programs such as the BTRR form an important part of the NIGMS' portfolio as the Institute seeks to ensure broad access across the research community to cutting-edge technological resources. For example, synchrotron-based technologies, an extremely powerful source of X-rays, are critical for structural biology research; more than 90% of all three-dimensional structures of biological molecules in the Protein Data Bank7 were determined using data from synchrotrons. The NIGMS recently shifted its support for these important resources from research grants to a mechanism focused on user-access, utility and efficiency of operations. A similar model will be used to support the new national cryo-electron microscopy centers.

Conclusion

Mr. Chairman, in this statement, I have tried to highlight just a few examples of how NIGMS' programs maximize the scientific returns on the taxpayers' investments in fundamental biomedical research. As the scientific enterprise and national clinical landscape continue to evolve, the NIGMS looks forward to continuing to meet and address both the challenges and opportunities associated with this dynamic environment, and in so doing, to the many more advances that will emerge from institutions, laboratories, research teams, and individual investigators across the nation. We thank you for your continued support and for this opportunity to describe some of the new initiatives at NIGMS.