National Institutes of Health: Research Priority Setting, and Funding Allocations across Selected Diseases and Conditions
Released: 2014-04-30T00:00:00


Highlights: section_title: Why GAO Did This Study
paragraphs: NIH is the nation's leader in sponsoring and conducting biomedical research. In fiscal year 2012, NIH had a budget of almost $31 billion, over 80 percent of which was used to fund extramural research that supports scientists and research personnel working at universities, medical schools, and other research institutions. Twenty-four of NIH's 27 ICs that support extramural research are focused on particular diseases, conditions, or research areas, and these ICs have their own appropriations. Decisions about which projects are funded are made by these individual ICs. NIH reports funding for 235 research, condition, and disease categories in RCDC.

GAO was asked to review NIH funding related to leading diseases and health conditions. GAO examined (1) how research priorities are set at NIH, and (2) NIH allocations of research funding across selected diseases and conditions. For five ICs—National Cancer Institute; National Heart, Lung, and Blood Institute; National Institute of Allergy and Infectious Diseases; National Institute of Diabetes and Digestive and Kidney Diseases; and National Institute of General Medical Sciences—GAO reviewed documents and interviewed IC officials about priority setting. GAO reviewed NIH fiscal year 2012 funding reported by RCDC for 40 research, condition, and disease categories related to the leading causes of death in the United States and globally, and the most prevalent chronic diseases and conditions in the United States.

section_title: What GAO Found
paragraphs: Individual institutes and centers (ICs) at the National Institutes of Health (NIH) set their own research priorities, and GAO found that the five selected ICs—awarding the largest amount of research funding—that it reviewed did so considering similar factors and using various priority-setting approaches. Agency officials stated that the ICs' mission and appropriations inform priority-setting approaches. Some IC officials noted that because the costs of potential research projects generally exceed the available appropriation, the ICs must prioritize among research projects. In priority setting, IC officials reported taking into consideration scientific needs and opportunities, gaps in funded research, the burden of disease in a population, and public health need, such as an emerging public health threat like influenza that needs to be addressed. While each IC GAO examined had its own approach for setting priorities, they all considered the input of stakeholders, including the scientific community, and used some similar strategies. All five ICs developed strategic plans, though the process varied by IC. Some ICs also used annual planning activities in various forms, which then guided funding opportunity announcements. All five ICs also conducted reviews and evaluations of their research portfolios to ensure that their priorities align with scientific opportunities, research gaps, and emerging science. In addition to these efforts at the IC level, agency officials told GAO that the NIH Office of the Director provides leadership and coordinates priority setting activities, especially for those activities that involve multiple ICs.

NIH reported funding levels that varied widely for the 40 different Research, Condition, and Disease Categorization system (RCDC) categories GAO examined that correspond to the leading causes of death and the most prevalent chronic conditions. For example, NIH reported actual fiscal year 2012 funding levels ranging from $13 million for projects in the fibromyalgia category to more than $5.6 billion for projects in the cancer category. Although these categories are part of NIH's RCDC, which is used to categorize the research activities across the agency, agency officials said that the system cannot estimate a total, non-duplicated amount of funding that is specific to a given disease or condition. This is because RCDC categories are neither mutually exclusive nor exhaustive. For example, projects may be included in multiple RCDC categories, some categories are related to each other and therefore some categories may also be included within another, and funding for all diseases is not captured in the system. While RCDC is NIH's official system for reporting research funding across the ICs, two of the five ICs that GAO reviewed—the National Cancer Institute (NCI) and the National Institute of Allergy and Infectious Diseases—had their own systems for tracking their funding, which allowed them to provide more detailed information than that available from RCDC. For example, NCI has a publicly available website that specifies funding for more than 40 specific cancer types as well as almost 50 research topics that are not disease-specific. Funding for individual projects may be separated for specific studies into those cancer types. According to officials, the system enhances NCI's ability to plan and monitor its scientific investment.

The Department of Health and Human Services provided technical comments, which GAO incorporated as appropriate.

section_title: Letter
paragraphs: The National Institutes of Health (NIH) is the nation’s leader in sponsoring  and conducting biomedical research. In fiscal year 2012, NIH, an agency  of the Department of Health and Human Services (HHS), had a budget of  almost $31 billion, over 80 percent of which was used to fund extramural  research that supports scientists and research personnel working at  universities, medical schools, and other research institutions. NIH also  funds intramural research performed by NIH scientists in NIH  laboratories. To fulfill its mission to seek fundamental knowledge about  the nature and behavior of living systems and the application of that  knowledge to enhance health, lengthen life, and reduce illness and  disability, NIH funds research related to life processes and many  diseases and conditions, including those that are among the leading  causes of death both in the United States and globally.

The range of research that NIH funds is reflected in its organization,  consisting of the Office of the Director (OD) and 27 institutes and centers  (IC). The ICs that support extramural research are focused on particular  diseases, conditions, or research areas and are supported by their own  budget, mission, and staff. Each IC makes final decisions on which  projects to fund or conduct contingent on the available appropriation or  funding. Given NIH’s role in biomedical research, its broad mission, and the size of its budget, Congress and researchers have had long-standing  interest in how NIH establishes research priorities and how those  priorities guide the allocation of its resources, particularly in relation to  various diseases and conditions.

You requested that we provide information on federally funded research  related to diseases and health conditions that are the leading causes of  death or the most prevalent chronic conditions. In this report, we describe  (1) how research priorities are set at NIH, and (2) NIH allocations of  research funding across selected diseases and conditions.

In addition, appendix I of this report summarizes key findings from  published studies about the relationship between the extent of research  funded and the burden of diseases being researched, both at NIH and in  other countries. The research used measures of disease burden, such as  the prevalence of disease or conditions in a population, hospitalization  rates, and mortality associated with a particular disease.

To describe how research priorities are set at NIH, we reviewed relevant  laws and agency guidance, including documents that outline methods for  setting priorities for research funding from the five ICs that awarded the  largest amount of research funding in fiscal year 2012. The five ICs are  National Cancer Institute (NCI); National Heart, Lung, and Blood Institute  (NHLBI); National Institute of Allergy and Infectious Diseases (NIAID);  National Institute of Diabetes and Digestive and Kidney Diseases  (NIDDK); and National Institute of General Medical Sciences (NIGMS).  We also interviewed officials from OD and those five ICs about their  processes for setting priorities for research funding. We did not evaluate  the appropriateness of the IC’s processes for setting priorities for  research funding.

To describe research funding allocations across selected diseases and  conditions, we first identified the leading causes of death in the United  States, the leading causes of death globally, and the most prevalent  chronic conditions for adults in the United States. We then identified the  categories within NIH’s Research, Condition, and Disease Categorization  system (RCDC) that best matched each disease and condition we  selected, and we confirmed these matches with NIH. (See app. II for a  more detailed description of our methodology for identifying the leading  causes of death, the most prevalent chronic conditions, and their  corresponding NIH research, condition, and disease categories.) We  reviewed publically available RCDC funding data from fiscal year 2012 for  those categories. We also interviewed NIH officials responsible for RCDC  to understand the data gathered and reported on. We determined that the  data were sufficiently reliable for the purposes of this review.

We conducted our work from April 2013 to March 2014 in accordance  with generally accepted government auditing standards. Those standards  require that we plan and perform the audit to obtain sufficient, appropriate  evidence to provide a reasonable basis for our findings and conclusions  based on our audit objectives. We believe that the evidence obtained  provides a reasonable basis for our findings and conclusions based on  our audit objectives.
subsections: section_title: Background
subsections: section_title: NIH’s Organization
paragraphs: As the central office for NIH, OD establishes NIH policy and broad  themes for the agency to pursue, such as ensuring a sustainable  scientific workforce, based on national needs and scientific opportunities.  In addition, the OD is responsible for coordinating the programs and  activities that span NIH components, particularly research initiatives and  issues involving more than 1 of the 27 ICs. OD is also responsible for  ensuring that scientifically-based strategic planning is implemented in  support of research priorities, and that NIH’s resources are sufficiently  allocated for research projects identified in strategic plans. NIH conducts  and sponsors biomedical research through its ICs, each of which is  charged with a specific mission. ICs’ missions generally focus on a  specific disease, a particular organ, or a stage in life, such as childhood  or old age. The ICs support, plan, and manage their own research  programs in keeping with OD policy and priorities. Within an IC, there can  be a number of offices, centers, and divisions that focus on specific  aspects of the IC’s mission. For example, NCI has a Division of Cancer  Epidemiology and Genetics, as well as a Division of Cancer Treatment  and Diagnosis.

section_title: Extramural and Intramural Research Supported by NIH
paragraphs: ICs accomplish their missions chiefly through extramural and intramural  research. Extramural research accounted for more than 80 percent of  NIH’s budget in fiscal year 2012. This research is conducted at 2,500  universities, medical schools, research organizations, and companies  who are awarded extramural research grants or extramural research and  development contracts through NIH’s competitive process. Twenty-four of  the 27 ICs fund extramural research projects, and these ICs make final  decisions on which extramural research projects to fund following a  standard peer review process.what NIH considers “unsolicited” research and research training projects  for which applications are submitted in response to broad funding  opportunity announcements that span the breadth of the NIH mission. In  addition, to encourage and stimulate research and the submission of  research applications in specific areas, many ICs will issue solicitations  that are more narrow in scope in the form of  ,  requests for applications, and requests for proposals. When reviewing  applications for extramural research projects, NIH follows a process of  peer review, established by law.sequential levels of peer review. According to NIH officials, the first level  involves panels of experts to assess the scientific merit of the proposed  science. The second level involves panels of experts and leaders of non- science fields including patient advocates that, in addition to scientific  merit, also consider the IC’s mission and strategic plan goals, public  health needs, scientific opportunities, and portfolio balance. After NIH’s   Most IC extramural funding is provided for   This peer review system has two  peer review process is concluded, IC directors make extramural funding  decisions.

Intramural research, which accounts for approximately 10 percent of  NIH’s budget, is conducted by NIH scientists in NIH laboratories. This  includes about 5,300 scientists and technical support staff who are  employees, and another 5,000 young scientists at various stages of  research training who come to NIH for a few years to work as non- employee trainees, including 3,800 postdoctoral fellows. All but 3 of the  27 ICs have an intramural research program, but the size, structure, and  activities of the programs vary greatly.

section_title: NIH’s Research, Condition, and Disease Categorization System
paragraphs: In January 2007, Congress directed NIH to establish an electronic system  to categorize the research grants and activities of OD and all the ICs. In  response, NIH created RCDC. Implemented in February 2008, RCDC  uses a computer-based text-mining tool that recognizes words and  phrases in project descriptions in order to assign NIH projects to at least  one of 235 categories of diseases, conditions, and research areas that  were developed for reporting to Congress and the public. NIH officials  said that RCDC serves as NIH’s primary computerized reporting process  to categorize its research funding. The system includes reporting tools  that can be used to generate publically-available, web-based reports on  total funding amounts for the research projects related to each RCDC  category.

section_title: Individual Institutes and Centers Set Their Own Research Priorities Using a Variety of Approaches, Including Strategic Planning, Annual Planning, and Review of Research Portfolios
paragraphs: Individual ICs at NIH set their own research priorities, and we found that  the five selected ICs we reviewed did so considering similar factors and  using various priority-setting approaches. Officials at all five of the ICs  stated that their mission and available appropriations inform priority  setting approaches. Officials at one IC noted that their IC’s mission  provides context related to why the IC was developed initially and insight  into the emerging areas of research. Officials stated that an IC’s  appropriations not only set funding parameters, but may also influence  priority setting if the appropriations include mandated spending by  Congress for a specific disease. Some IC officials noted that because  the costs of potential research projects generally exceed the available  appropriation, the ICs generally must prioritize among research projects.  In priority setting, IC officials also reported taking into consideration  scientific needs and opportunities, gaps in funded research, the burden of  disease in a population, and public health need, such as an emerging  public health threat that needs to be addressed, like influenza.

While individual ICs used different approaches to priority setting, all five  selected ICs we reviewed reported using some combination of strategic  planning, annual planning, and periodic review and evaluation of their  research portfolios as part of their approach to priority setting.

Strategic planning: All five ICs we interviewed developed strategic  plans—consistent with law and NIH policy—to set priorities and goals for  research funding. According to officials at selected ICs, the  development of these plans is guided by various processes at each IC.  Although these processes vary by IC, they include an opportunity to solicit  input from stakeholders, including the scientific community, as well as  review by IC staff and leadership. Examples of IC strategic planning  activities include the following:

NIAID used a framework organized around various scientific areas  that encompassed its research to develop the strategic plans it  published in 2000, 2008, and 2013. When developing the plans,  NIAID convened groups of NIAID subject matter experts for each  scientific area and considered input offered by external organizations  and scientists to guide the process. The experts deliberated priorities  identified for each scientific area and suggested revisions to the draft  plan. For example, officials stated that NIAID has seen a shift in the  emphasis on research efforts related to human immunodeficiency  virus/acquired immunodeficiency syndrome (HIV/AIDS) from studying  single-drug therapy to the current focus on finding improved HIV  treatments and tools for preventing infection. This new focus was then  incorporated as part of the strategic plan published in 2013. Draft  strategic plans were reviewed by the NIAID Advisory Council. After  a final review by the director of NIAID, the finalized plans were  published on NIAID’s web site.

NHLBI officials said that, as of November 2013, the IC was revising  the strategic plan it published in 2007 and part of its strategic planning  process under the direction of a new director appointed in 2012. As  part of the revised process, NHLBI created the Strategic Investment  and Management Steering Committee that will help establish strategic  goals as well as advise leadership about whether proposed activities  are consistent with the IC’s strategic plan. Officials stated that the  strategic planning process will engage both NHLBI scientific staff as  well as the larger extramural community because they are  knowledgeable about research gaps within their respective fields.  Further, NHLBI officials stated that input provided by stakeholders  also helps to inform development of disease-specific planning  documents, such as one for asthma and another on sleep disorders.

Annual planning activities: In addition to the development of strategic  plans, some IC officials we interviewed told us that they conduct annual  planning activities as a part of their process to set priorities for research  funding. They told us that these annual planning activities address  ongoing and emerging needs. While the processes vary across the ICs in  terms of the level of structure and formality, officials stated that during the  annual planning process these ICs typically consider factors such as  scientific needs, research gaps, scientific opportunities, public health  needs, and the need to balance the various types of research conducted  to address all areas of the mission. Examples of annual planning activities  include the following:

NIAID uses an annual planning process to develop and select  initiatives that address special needs, gaps, and opportunities in  relevant research areas. According to NIAID officials, these initiatives  are developed through a structured process centered around two  primary events—the annual Summer Policy Retreat and the annual  Winter Program Review. NIAID staff also review their scientific areas  for the latest information on burden of illness and state of scientific  progress, which is used to inform the development of future initiatives.  According to agency officials, NIAID staff use this information, as well  as input from the scientific community, to prepare and present a  concept of an initiative to NIAID’s National Advisory Council, which  reviews, comments on, and decides whether to approve the initiative.  Once an initiative has been approved, NIAID solicits research  applications through a request for application, a program  announcement, or a request for proposal.

NIDDK uses an annual process that requires NIDDK scientific  program staff within the IC’s divisions to review the research portfolio  and to identify research opportunities. According to NIDDK, this effort  is informed by a number of factors, such as ongoing input from the  extramural research and health advocacy communities and recent  research advances. They said that each division then presents  initiative concepts built on these research opportunities at NIDDK  senior leadership meetings. The NIDDK director prioritizes the  initiative concepts and determines which will move forward based on  consideration of merit and other factors. These concepts go through a  clearance process, including review from the NIDDK National  Advisory Council. NIDDK develops funding opportunity  announcements based on these identified research opportunities,  such as requests for application that address specific scientific  questions or diseases. In addition, NIDDK conducts divisional  planning and prioritization activities during monthly meetings, retreats,  and other activities, including consideration of opportunities for funds  to support targeted emphasis areas.

Review and evaluation of research portfolio: Officials from the five  selected ICs we interviewed stated they conduct reviews of their research  portfolio to help ensure existing priorities reflect and align with current  scientific opportunities, research gaps, and emerging science. This  includes periodic formal program assessments of their research portfolio,  which the ICs used to determine if the IC is meeting its overall priorities  and goals, to maintain portfolio balance, and to make any changes to  priorities over time as science evolves. Examples of portfolio review and  evaluation activities include the following:

NCI officials stated that the IC conducts portfolio analyses, which  involve examining research areas NCI has funded to identify  opportunities for research. Officials stated that the portfolio analysis is  an iterative process that occurs continually throughout the year. One  example of this portfolio analysis has involved publishing broad  questions related to five themes for the research community on NCI’s  website and using the responses received to inform development of  requests for application and program announcements published by  NCI. Officials said they aim to generate about four new questions  each year and that the questions are generally very broad and do not  usually address specific diseases. For example, one question recently  asked how the level, type, or duration of physical activity influences  cancer risk and prognosis.

NIGMS conducts formal program evaluations to determine what  benefits the program has produced overall, including scientific  advances, new technologies, and cutting-edge paradigm shifts, such  as changes in the understanding of human biology. In addition,  officials told us that NIGMS divisions conduct an annual review, which  is generally prepared for NIGMS’s director, and provides information  about the scientific advances that can be attributed to the work  conducted in each division. This includes items such as the number of  grants funded, the number of new principal investigators, the types of  research conducted, and an overview of any new science performed.

While the individual ICs set their own priorities, according to NIH officials,  the OD provides leadership to the ICs and helps coordinate priority  setting activities, especially for efforts that involve multiple ICs. For  example, NIH officials reported that the director of NIH meets with all IC  directors weekly to discuss research priorities, investments, and concerns  that may affect an IC or NIH overall. In addition, the Advisory Committee  to the Director assists the director of NIH with making major planning and  policy decisions, including those related to research priorities and the  allocation of funds for major, NIH-wide projects and identifies areas that  have the potential for significant improvements both within NIH and within  the scientific community at large. For example, the NIH Director charged  the committee with examining NIH’s data needs to determine whether the  agency is positioned to handle significant amounts of data and how to  make these data accessible to researchers. In addition, NIH established a  special division within the Office of the Director—the Division of Program  Coordination, Planning, and Strategic Initiatives—to help manage large  and complex research portfolios across NIH. The offices within this  division organize formal discussions to obtain input from all of the IC  directors as well as members of the scientific community to identify areas  of significant scientific interest or need that typically span the interests of  multiple ICs. NIH officials also stated that although each IC has at least  one strategic plan in place, in some instances NIH has developed  strategic plans for particular disease or topic areas that cross multiple  ICs, such as one related to women’s health research.

section_title: NIH Funding Levels Varied Widely For Selected Categories of Research, Conditions, and Diseases Corresponding to the Leading Causes of Death and Chronic Conditions
paragraphs: In fiscal year 2012, NIH reported funding levels that ranged widely—from  $13 million for projects in one RCDC category to more than $5.6 billion for  another—for the 40 different RCDC categories we examined. (See   table 1.) We determined that these RCDC categories were the best match  for the most frequent causes of death in the United States, the most  frequent causes of death globally, and the most prevalent chronic  conditions in the United States. (See app. II for an explanation of how  we identified these diseases and conditions and matched them with  RCDC categories.)

NIH officials said that RCDC is the agency’s official system for reporting  research funding across the agency’s ICs and it provides a method for  reporting consistent information about NIH funding. As noted by the  agency, RCDC produces standard reports across all ICs using a process  that is reproducible. To facilitate this, all ICs are to use the same  definitions of research, diseases, and conditions for the RCDC  categories. Further, the system allows for detailed reports, including the  total funding for a category as well as specific information about the  projects under each category, such as the title of the project, the IC  supporting the research, and the project number.

Agency officials said that the system was not designed to be able to  estimate a total, non-duplicated amount of funding specific to a given  disease or condition because RCDC categories are neither mutually  exclusive nor exhaustive. Specifically:

Projects may be reported in multiple categories. NIH officials said that  the categories within the system are not mutually exclusive and  therefore a project may be included in multiple categories. Officials  told us that, on average, a single project may fall into five or six RCDC  categories.

Categories may be contained in another category. Some categories  are inherently related and therefore an entire RCDC category can also  be contained in another category. For example, the category of  cancer includes other cancer categories, such as breast cancer, lung  cancer, or prostate cancer, and the research funding reported in each  of these categories will also be reported in the cancer research total.

Categories do not exist for all diseases. RCDC also does not capture  the funding for all diseases that NIH studies. As of November 2013,  RCDC publicly reported on 235 different categories, but agency  officials told us that there are an additional 30 to 40 categories used  for congressional reporting only, and about 50 categories on a waiting  list to be developed for inclusion in the system.

Not all projects that NIH funds are included. The protocols that NIH  develops for tracking funding include certain thresholds for inclusion.  Therefore, a project that is only minimally related to a particular  category may not be included in the funding for that category because  the description of the project does not adequately match the terms  defining that category. NIH officials told us that 3 to 5 percent of NIH  funded research projects do not appear in any RCDC category.

Some ICs have their own systems that track and report funding within  their research portfolios. Of the five selected ICs we reviewed, two had  their own systems for tracking their funding.

NCI maintains a publicly available website that communicates funding  decisions across the research supported by NCI. The system was  developed in 1998 and categorizes research into more than 40  specific cancer types, as well as almost 50 research topics that are  not disease-specific. Funding for individual projects may be separated  and reported in the NCI system by the specific types of cancers being  studied. According to NIH officials, the system is limited to those  research projects funded by NCI and therefore does not include  information about research studies addressing cancer that are funded  solely by other ICs. NCI officials told us that they use the system to  report data to stakeholders, including Congress, and that the analyses  they conduct based on these data enhance NCI’s ability to plan and  monitor its scientific investment.

NIAID also has an internal coding system which, according to agency  officials, was instituted in 1979 and codes each individual research  project it funds by areas of study. Staff members manually assign  codes. NIAID officials noted that projects may have multiple codes  depending on the specific goals of the project, and these codes reflect  the percentage of relevance to that specific goal, such that the total  values add to 100 percent. Officials noted that the system helps NIAID  respond to requests for funding information that is not included in  RCDC. For example, when addressing a question about how much  NIAID has invested in influenza research, officials said they examine  research project funding for the various subtypes of influenza such as  H1N1 and avian influenzas (including H5N1 and H7N9). These  subcategories are included in the internal coding system, but are not  covered by RCDC. Officials noted that NIAID uses RCDC for the  official budget numbers that NIH reports to Congress.

section_title: Agency Comments
paragraphs: We provided a draft of this report to HHS. The Department provided  technical comments, which we incorporated as appropriate.

As agreed with your office, unless you publicly announce the contents of  this report earlier, we plan no further distribution until 30 days from the  report date. At that time, we will send copies to the Secretary of the  Department of Health and Human Services, the Director of the National  Institutes of Health, and other interested parties. In addition, the report will  be available at no charge on the GAO website at http://www.gao.gov.

If you or your staff have any questions about this report, please contact  me at (202) 512-7114 or kohnl@gao.gov. Contact points for our Offices of  Congressional Relations and Public Affairs may be found on the last page  of this report. Major contributors to this report are listed in appendix III.

section_title: Appendix I: Studies about the Relationship between Research Funding and Burden of Disease
paragraphs: To provide information on studies about the relationship between  research funding and burden of disease at the National Institutes of  Health (NIH) and in other countries, we identified such studies relevant to  NIH through on-line searches and we provide information on the two most  recent of the studies. Additionally, for other countries, we identified  published studies through a search of various databases, and we provide  information from two of the more recent such studies. We did not identify  research funding levels in other countries.

Studies have been conducted that examine the relationship between  research funding and burden of disease in the United States and other  countries. Some researchers have reported that no single measure of  disease burden captures the impact of various diseases and that different  measures of burden may result in different conclusions about funding.

Studies on the relationship between research funding by NIH and burden  of disease found varied relationships depending upon the burden of  disease measures used. For example,

A 2013 study assessed the allocation of research funds across 107  diseases using NIH’s Research, Condition, and Disease  Categorization system (RCDC) funding data and found a strong and  statistically significant relationship between NIH funding and deaths  and hospitalizations. As a result, according to the study, the data  suggest that NIH funding is responsive to these two measures of  disease burden. Further, the study noted that the data were consistent  with the argument that it is more feasible for NIH to respond to  disease burden considerations through directed or applied research  funding, such as funding for clinical trials, than through investigator- initiated basic research.

A 2011 study to assess the correlation between NIH research funding  and burden of disease using data from 2004 and 2006 found that  current levels of NIH disease-specific research funding correlated  modestly with U.S. disease burden.

The study noted that there could  be a number of reasons that alignment of funding and burden of  disease were not better correlated, including that basic science  research has consistently accounted for 55 percent of NIH spending  and it is difficult to attribute to a specific disease, contributing  uncertainty to the analysis and reducing correlations. Measures of  burden of disease for this study were incidence, prevalence, mortality,  years of life lost to premature death (YLL), and disability-adjusted life  years (DALY).

Studies of research funding in other countries have also found variations  in relationships between funding and burden of disease. For example,

A 2012 study found that research expenditures by governmental  agencies and charities for four of the foremost chronic diseases in the  United Kingdom—cancer, coronary heart disease, stroke and  dementia—was not aligned with burden of disease. Specifically,  research funding for dementia and stroke was disproportionately small  in comparison with funding for cancer and coronary heart disease. In  this study, burden of disease was measured by prevalence and  DALYs.

A 2004 study found a significant relationship between research  funding by the National Health and Medical Research Council in  Australia and several measures of burden of disease, variations over the 6-year span reviewed in the study using different  measures. Measures of burden of disease for this study were  incidence, mortality, YLLs, years of life lost to disability, and DALYs.

The National Health and Medical Research Council is the main Australian body charged  with the responsibility of supporting medical and public health research and training in  Australia.

section_title: Appendix II: Leading Causes of Death and Chronic Conditions and their Corresponding National Institutes of Health Categories
paragraphs: To determine the leading causes of death in the United States, we  reviewed data on causes of death in 2011 that were reported by the  Centers for Disease Control (CDC). First, we identified the 15 leading  causes of death reported by CDC. Then, we identified subcategories of  those leading causes of death. To do so, we used as the cut off the  number of deaths for the 15th leading cause of death—which was 18,090  deaths from pneumonitis due to solids and liquids. We included those  sub-categories of causes of death, for example pneumonia, where the  number of deaths reported was greater than 18,090. See table 2 for the  leading causes of deaths in the United States in 2011, including the sub- categories with at least 18,090 related deaths in 2011, and the number of  deaths attributed to these causes.

To determine the leading causes of death globally, we reviewed the  analysis in the Global Burden of Disease Study. We identified the 15  leading causes of death globally in 2010 that were reported in this study.  See table 3 for the leading causes of deaths globally.

To determine the most prevalent chronic diseases and conditions in the  United States, CDC provided us with a list of the 13 most prevalent  chronic diseases and conditions for adults, and four subcategories. See  table 4 for a list of the most prevalent chronic diseases and conditions for  adults identified by CDC.

To determine the corresponding categories from the National Institutes of  Health’s (NIH) Research, Condition, and Disease Categorization system  (RCDC) for the diseases and conditions that are the leading causes of  death in the United States, the leading causes of death globally, and the  most prevalent chronic diseases and conditions in the United States, we  identified the International Classification of Diseases (ICD-10) codes  associated with each of these diseases and conditions and, using their  related descriptions, compared them with the RCDC category  descriptions. We then provided our list of diseases, conditions, and ICD- 10 codes with the corresponding RCDC categories to NIH for the  agency’s review and concurrence. NIH officials confirmed most of our  matches. For some of the diseases and conditions, officials noted the  RCDC category that we identified was the closest match, but the category  was substantially broader than the RCDC category it was selected to  represent or it was substantially narrower than the RCDC category it was  selected to represent. In other cases, NIH officials noted that there was  not an RCDC category that was a close enough fit to the disease  category we were trying to represent. See table 5 for the leading causes  of death in the United States and globally, as well as the most prevalent  chronic diseases and conditions, and their corresponding RCDC category  or categories.

section_title: Appendix III: GAO Contact and Staff Acknowledgments
subsections: section_title: GAO Contact

section_title: Staff Acknowledgments
paragraphs: In addition to the contact name above, Karen Doran (Assistant Director),  George Bogart, Adrienne Daniels, Carolyn Feis Korman, Cathy Hamann,  Natalie Herzog, Amy Leone, and Andrea Richardson made key  contributions to this report.


Question-summary annotations: questions: question: What the GAO find about the five ICs at the NIH that received the most amount of funding?
answer: Individual institutes and centers (ICs) at the National Institutes of Health (NIH) set their own research priorities, and GAO found that the five selected ICs—awarding the largest amount of research funding—that it reviewed did so considering similar factors and using various priority-setting approaches.

question: Why do ICs need to set priorities for funding?
answer: Agency officials stated that the ICs' mission and appropriations inform priority-setting approaches. Some IC officials noted that because the costs of potential research projects generally exceed the available appropriation, the ICs must prioritize among research projects.
child_questions: question: What might the ICs need to consider as priorities in research projects?
answer: In priority setting, IC officials reported taking into consideration scientific needs and opportunities, gaps in funded research, the burden of disease in a population, and public health need, such as an emerging public health threat like influenza that needs to be addressed.
child_questions: question: What did all ICs consider for setting priorities?
answer: While each IC GAO examined had its own approach for setting priorities, they all considered the input of stakeholders, including the scientific community, and used some similar strategies.

question: What strategies in planning did all ICs have?
answer: All five ICs developed strategic plans, though the process varied by IC. Some ICs also used annual planning activities in various forms, which then guided funding opportunity announcements. All five ICs also conducted reviews and evaluations of their research portfolios to ensure that their priorities align with scientific opportunities, research gaps, and emerging science.

question: Outside of the ICs, what did the NIH provide?
answer: In addition to these efforts at the IC level, agency officials told GAO that the NIH Office of the Director provides leadership and coordinates priority setting activities, especially for those activities that involve multiple ICs.

questions: question: What did GAO examine about the NIH reports?
answer: NIH reported funding levels that varied widely for the 40 different Research, Condition, and Disease Categorization system (RCDC) categories GAO examined that correspond to the leading causes of death and the most prevalent chronic conditions. For example, NIH reported actual fiscal year 2012 funding levels ranging from $13 million for projects in the fibromyalgia category to more than $5.6 billion for projects in the cancer category.
child_questions: question: Why can the system not estimate the funding specific to a given disease or condition?
answer: Although these categories are part of NIH's RCDC, which is used to categorize the research activities across the agency, agency officials said that the system cannot estimate a total, non-duplicated amount of funding that is specific to a given disease or condition. This is because RCDC categories are neither mutually exclusive nor exhaustive. For example, projects may be included in multiple RCDC categories, some categories are related to each other and therefore some categories may also be included within another, and funding for all diseases is not captured in the system.

question: What is a major example of the issue with NIH's RCDC funding categorization?
answer: While RCDC is NIH's official system for reporting research funding across the ICs, two of the five ICs that GAO reviewed—the National Cancer Institute (NCI) and the National Institute of Allergy and Infectious Diseases—had their own systems for tracking their funding, which allowed them to provide more detailed information than that available from RCDC. For example, NCI has a publicly available website that specifies funding for more than 40 specific cancer types as well as almost 50 research topics that are not disease-specific.
child_questions: question: What is an example of NCI's funding process?
answer: Funding for individual projects may be separated for specific studies into those cancer types. According to officials, the system enhances NCI's ability to plan and monitor its scientific investment.

questions: question: What is NIHs relationship to biomedical research?
answer: NIH is the nation's leader in sponsoring and conducting biomedical research.

question: What was the status of NIH's budget and use in 2012?
answer: In fiscal year 2012, NIH had a budget of almost $31 billion, over 80 percent of which was used to fund extramural research that supports scientists and research personnel working at universities, medical schools, and other research institutions.
child_questions: question: What are the similarities of the ICs that support extramural research?
answer: Twenty-four of NIH's 27 ICs that support extramural research are focused on particular diseases, conditions, or research areas, and these ICs have their own appropriations.

question: What influence do ICs have on funding projects?
answer: Decisions about which projects are funded are made by these individual ICs. NIH reports funding for 235 research, condition, and disease categories in RCDC.

questions: question: What did GAO research regarding the NIH funding?
answer: GAO was asked to review NIH funding related to leading diseases and health conditions. GAO examined (1) how research priorities are set at NIH, and (2) NIH allocations of research funding across selected diseases and conditions.
child_questions: question: How did the GAO look further into specific ICs?
answer: For five ICs—National Cancer Institute; National Heart, Lung, and Blood Institute; National Institute of Allergy and Infectious Diseases; National Institute of Diabetes and Digestive and Kidney Diseases; and National Institute of General Medical Sciences—GAO reviewed documents and interviewed IC officials about priority setting.

question: What did the GAO review for the NIH's 2012 fiscal year?
answer: GAO reviewed NIH fiscal year 2012 funding reported by RCDC for 40 research, condition, and disease categories related to the leading causes of death in the United States and globally, and the most prevalent chronic diseases and conditions in the United States.