NATIONAL 

INSTITUTES OF 

HEALTH

Better Oversight 

Needed to Help 

Ensure Continued 

Progress Including 

Women in Health 

Research

Report to Congressional Requesters

October 2015

GAO-16-13

United States Government Accountability Office
Highlights of GAO-16-13, a report to 

congressional requesters

October 2015

NATIONAL INSTITUTES OF HEALTH

Better Oversight Needed to Help Ensure Continued 

Progress Including Women in Health Research

Why GAO Did This Study

Women make up over half the U.S. 

population, but historically have been 

underrepresented in clinical research 

supported by NIH and others. As a 

result, differences in the manifestation 

of certain diseases and reactions to 

treatment in women compared with 

men were not identified. For example, 

there have been instances of women 

having adverse effects that differed 

from those of men related to 

medications and other treatments. 

NIH’s Inclusion Policy established 

requirements governing women’s 

inclusion in its clinical research.

GAO was asked to provide information 

on women’s participation in NIH 

research. Among other reporting 

objectives, GAO examined 

(1) women’s enrollment and NIH’s 

efforts to monitor this enrollment in 

NIH-funded clinical research; and 

(2) NIH’s efforts to ensure that NIHfunded clinical trials are designed and 

conducted to analyze potential sex 

differences, when applicable. To do 

this, GAO reviewed relevant laws and 

policies, including the Inclusion Policy, 

and federal standards for internal 

control; reviewed and analyzed NIH 

enrollment data from fiscal years 2005-

2014; and interviewed NIH and IC 

officials and other experts.

What GAO Recommends

GAO recommends that NIH examine 

and report more detailed data on 

women’s enrollment in NIH-funded 

studies, and collect, examine, and 

report data on the extent to which 

these studies include analyses of 

potential differences between women 

and men. NIH agreed with GAO’s 

recommendations and plans to take 

action to implement them.

What GAO Found

Data from the National Institutes of Health (NIH) show that more women than 

men were enrolled in NIH-funded clinical research for fiscal years 2005-2014, but 

NIH does not make certain enrollment data readily available to interested parties 

or examine other detailed data to identify potential challenges to enrolling women 

in specific research and disease or condition areas. In fiscal year 2014, for 

example, NIH reported that across all of the clinical research studies it funded—

including phase III clinical trials, the largest studies involving human subjects—

57 percent of enrollees (16.4 million) were women. NIH collects enrollment data 

from individual awardees through its Institutes and Centers (IC)—which generally 

fund studies in different research areas—and publicly reports data on aggregate 

enrollment as part of its implementation of the Inclusion Policy developed to 

implement provisions of the NIH Revitalization Act of 1993. However, NIH does 

not make the IC-level enrollment data from each of the 25 ICs that report data 

readily available to interested parties, so that interested parties must make an 

effort to seek out this data. In addition, NIH does not routinely examine more 

detailed enrollment data, such as enrollment data organized by the disease and 

condition being studied. As a result, NIH is limited in its ability to identify whether 

women are sufficiently represented in studies in specific areas—such as 

cardiovascular disease—or if the agency-wide data inadvertently mask 

enrollment challenges. By not examining more detailed data on enrollment below 

the aggregate level, NIH cannot know whether it is adequately including women 

in all of the research it supports, in a manner consistent with its Inclusion Policy. 

Further, NIH’s reporting and monitoring in this area is inconsistent with federal 

internal control standards, which call for agencies to have controls to help ensure 

effective information flow and effective monitoring of agency activities.

NIH requires that phase III clinical trial awardees consider whether analysis of 

potential differences in outcomes between women and men is needed in their 

studies—one of the key requirements of its Inclusion Policy; however, the agency 

does not maintain, analyze, or report summary data to oversee whether analysis 

of outcomes by sex are planned or conducted. NIH officials told GAO that they 

rely on peer review and program officer monitoring to ensure awardee 

compliance with the analysis requirement. However, NIH program officers do not 

have a required field in a reporting system or other means to record the 

information they collect to monitor awardees’ analysis plans and compliance with 

the Inclusion Policy requirement. In addition, there is no data element in NIH’s 

data system to indicate whether an awardee’s study should or does include plans 

for an analysis of potential differences in research outcomes by sex. As a result, 

NIH lacks summary data, such as the percentage of awardees in a given year 

with trials designed to identify potential differences in clinical outcomes by sex. 

Without this summary information, NIH cannot report this information in the 

agency’s biennial reports to Congress and other stakeholders. The lack of 

summary data and reporting compromises NIH’s monitoring of its implementation 

of the Inclusion Policy and conflicts with federal internal control standards, which 

call for agencies to ensure the flow of information about agency activities, provide

for internal and external communication, and conduct periodic monitoring. 

Further, it limits NIH’s assurance that it is supporting research that can be used 

to shape improved medical practice for both women and men.

View GAO-16-13. For more information, 

contact Linda Kohn at (202) 512-7114 or 

KohnL@gao.gov.

United States Government Accountability Office
Letter 1

Background 6

More Women Than Men Were Enrolled in NIH Research Overall, 

but NIH Does Not Examine Detailed Data to Identify Potential 

Challenges to Enrolling Women 11

NIH Does Not Record Whether Clinical Trials Will Analyze Sex 

Differences to Allow Summary Analysis and Reporting for 

Oversight of Its Inclusion Policy 16

Proposed NIH Policy Intended to Increase Public Reporting of 

Clinical Trial Results by Awardees and Could Increase 

Reporting of Sex-Specific Results, According to NIH Officials 20

Conclusions 23

Recommendations for Executive Action 24

Agency Comments and Our Evaluation 24

Appendix I Literature Review of Factors Affecting Women’s Participation in 

Clinical Research 28

Appendix II National Institutes of Health (NIH) Funding for Women’s Health 

Research 31

Appendix III National Institutes of Health (NIH) and Institute and Center (IC) 

Research Study Enrollment 36

Appendix IV Comments from the Department of Health and Human Services 44

Appendix V GAO Contact and Staff Acknowledgments 52

Tables

Table 1: Types of Barriers Identified for Women in Literature 

Review 29

Table 2: Types of Reasons to Participate Identified for Women in 

Literature Review 29

Contents

Page i GAO-16-13 Women in NIH Clinical Trials
Table 3: National Institutes of Health (NIH) Estimated Funding for 

Selected Diseases and Conditions with a Particular 

Relevance to Women, Fiscal Year 2014 33

Table 4: Percentage Female, Male, and Other Enrollment in All 

Clinical Research Studies, for NIH Overall and Institutes 

and Centers (IC), Fiscal Years 2011-2014 36

Table 5: Percentage Female, Male, and Other Enrollment in 

Phase III Clinical Trials, for NIH Overall and Institutes and 

Centers (IC), Fiscal Years 2011-2014 40

Figure

Figure 1: Enrollment in All National Institutes of Health (NIH) 

Funded Clinical Research by Sex, as a Percentage of 

Total Enrollment, Fiscal Years 2005-2014 13

Abbreviations

HHS Department of Health and Human Services

IC institute and center

IOM Institute of Medicine

NCI National Cancer Institute

NHLBI National Heart, Lung, and Blood Institute

NIAID National Institute of Allergy and Infectious Diseases

NIH National Institutes of Health

OER Office of Extramural Research

ORWH Office of Research on Women’s Health

This is a work of the U.S. government and is not subject to copyright protection in the 

United States. The published product may be reproduced and distributed in its entirety 

without further permission from GAO. However, because this work may contain 

copyrighted images or other material, permission from the copyright holder may be 

necessary if you wish to reproduce this material separately.

Page ii GAO-16-13 Women in NIH Clinical Trials
441 G St. N.W.

Washington, DC 20548

October 22, 2015

Congressional Requesters

The Department of Health and Human Services’ (HHS) National Institutes 

of Health (NIH) is the nation’s largest public funder of biomedical 

research, with a budget of more than $30 billion for fiscal year 2015. 

Although women make up just over half the U.S. population, their health 

needs have historically been underrepresented in the research supported 

by NIH and others.1 This underrepresentation in clinical research has led 

to failures in recognizing differences between women and men in the 

prevalence of certain diseases, in how certain diseases manifest, and in 

the reactions to treatments.2 As a result, there have been notable 

instances of women experiencing adverse effects and other poor 

outcomes that differed from those experienced by men related to health 

care treatments. For example, in 2013, the Food and Drug Administration 

lowered the recommended dose of a popular sleep drug for women after 

it was determined that women and men metabolize the drug differently. 

That difference left women with more of the drug in their bodies the next 

morning and therefore at a greater risk of driving while impaired. 

Women’s health research advocates have stated that without the routine 

consideration of the potential for these and other clinical differences 

between women and men, the nation does not receive the full value of its 

public investment in biomedical research.

It has been over 2 decades since Congress passed the NIH Revitalization 

Act of 1993 (Revitalization Act), which provided statutory requirements for 

NIH to follow regarding research on women’s health.3 Specifically, the 

1

NIH’s first policy for the inclusion of women in clinical research was established in 1986. 

Prior to that, little clinical research on women’s health was conducted, for reasons 

including concerns over fetal exposure to experimental substances, the variability in 

women’s hormonal status, and the assumption that results of research on men could be 

extrapolated to women.

2

For example, research has identified differences in how certain diseases manifest 

themselves in women compared to men, including cardiovascular disease, lung cancer, 

depression, and Alzheimer’s disease.

3

Pub. L. No. 103-43, 107 Stat. 122 (June 10, 1993). The Revitalization Act also requires 

specific actions related to enrollment of minorities in NIH clinical research. Examination of 

NIH’s activities regarding minority enrollment was outside the scope of our work.

Letter

Page 1 GAO-16-13 Women in NIH Clinical Trials

 
Revitalization Act contained provisions for the enrollment of women in 

clinical research studies—including clinical trials—and requiring that 

clinical trials be designed and carried out to be able to provide for a valid 

analysis of whether the variables being studied affect women and men 

differently.4 The Revitalization Act also required NIH to establish 

guidelines for including women in clinical research. In 2000, we examined 

NIH’s efforts to conduct research on women’s health over the previous 

decade. We reported that NIH had made significant progress in 

implementing the Revitalization Act by issuing guidelines to implement 

the law, conducting extensive training for scientists and reviewers, and 

implementing a centralized data system to monitor enrollment.5 However, 

we also recommended in 2000 that NIH continue to make improvements, 

including better implementation of the requirement that some studies be 

designed to permit analysis of results by sex, which could reveal whether 

interventions affect women and men differently. In response, NIH 

developed specific guidance on this aspect of the policy for applicants 

and for the reviewers who evaluate applicants for NIH funding.

Partly in response to our recommendation, in October 2001, NIH 

amended its Policy and Guidelines on The Inclusion of Women and 

Minorities as Subjects in Clinical Research (Inclusion Policy), which is in 

place today. The Inclusion Policy establishes two key requirements for 

NIH-funded researchers conducting clinical research studies. Specifically, 

researchers must (1) design research plans that detail the breakdown of 

their studies’ participants by sex and provide a rationale for their planned 

enrollment; and (2) for certain clinical trials, include plans for analyzing 

4

NIH defines clinical research as patient-oriented research conducted with human 

subjects (or on material of human origin such as tissues, specimens, and cognitive 

phenomena) for which a researcher directly interacts with human subjects. This includes 

all phases of clinical trials, which are research studies in which one or more human 

subjects are prospectively assigned to one or more interventions (which may include a 

placebo or other control) to evaluate the effects of those interventions—including 

experimental drugs, treatments, and devices—on health-related biomedical or behavioral 

outcomes. In addition to clinical trials, clinical research includes epidemiological, 

behavioral, and observational studies.

In this report, we refer to “sex” rather than “gender” because sex is a biological variable 

and gender is a psychosocial construct. 

5

GAO, Women’s Health: NIH Has Increased Its Efforts to Include Women in Research, 

GAO/HEHS-00-96 (Washington, D.C.: May 2, 2000).

Page 2 GAO-16-13 Women in NIH Clinical Trials

 
outcomes for potential sex differences, when appropriate, as determined 

by prior scientific evidence.6

Despite these efforts, reports from the Institute of Medicine (IOM) 

published in 2010 and 2012 stated that continued efforts—by NIH and 

others—are needed to improve both the overall status of women’s health 

research and, in particular, the ability of researchers to conduct analysis 

of study results by sex to determine whether outcomes differ for women 

compared to men.7 While experts acknowledge that there have been 

increases in women’s enrollment in clinical research studies since the 

passage of the Revitalization Act, the IOM and others have found that 

even when women are included in clinical trials, the results of analyses 

are often not reported by sex—even when the overall results are 

published in peer-reviewed journals. A recent study found that less than 

half of NIH-funded clinical trials overall had been published in a peerreviewed biomedical journal within 30 months of trial completion, and in 

response, NIH officials stated that other means of sharing such results—

besides publication in scientific literature—is needed.8 Experts have 

stated that insufficient reporting of clinical trial results by sex limits the 

ability of researchers to identify potentially important sex differences that 

may ultimately affect patient care.

6

NIH determined that this requirement of the Inclusion Policy is limited to phase III clinical 

trials, the largest clinical trials involving human subjects and the most likely to result in 

broad changes in public health policy and/or standards of care.

7

Institute of Medicine, Women’s Health Research: Progress, Pitfalls, and Promise 

(Washington, DC: The National Academies Press, 2010), and Sex-specific Reporting of 

Scientific Research: A Workshop Summary (Washington, DC: The National Academies 

Press, 2012). IOM is a component of the National Academy of Sciences, a private, 

nonprofit organization chartered by Congress to provide independent, objective advice on 

matters related to science, technology, and medicine.

8

J. S. Ross, et al. “Publication of NIH-funded Trials Registered in ClinicalTrials.gov: Crosssectional Analysis.” BMJ, 344:d7292. (2012).

Page 3 GAO-16-13 Women in NIH Clinical Trials

 
You asked us to provide information on women’s participation in NIHfunded clinical trials, including NIH’s policies regarding enrollment, and 

both the analysis and reporting of clinical trial results. In this report, we 

examine NIH’s implementation of the Inclusion Policy, in particular,

1. the level of women’s enrollment and NIH’s efforts to monitor this 

enrollment in NIH-funded clinical research; and

2. NIH’s efforts to ensure that NIH-funded clinical trials are designed and 

conducted to analyze potential sex differences, when applicable.

We also examine

3. NIH’s efforts to help ensure that researchers make the results of 

clinical trials public, including sex-specific results, when appropriate.

In addition, Appendix I of this report summarizes information on the 

factors affecting women’s participation in clinical research, as were most 

frequently cited in published literature.

To address these objectives, we reviewed relevant laws and policies and 

interviewed NIH officials and other relevant experts. Specifically, we 

reviewed laws and policies (including proposed policies)—in particular, 

the Revitalization Act and NIH’s Inclusion Policy. We interviewed officials 

from NIH’s Office of Research on Women’s Health (ORWH), the Office of 

Extramural Research (OER), and the National Library of Medicine. We 

also interviewed representatives from selected NIH Institutes and Centers 

(ICs) that we identified as among the largest ICs both in terms of the total 

amount of award funding provided to researchers and in terms of the total 

number of research awards made: the National Cancer Institute (NCI), 

the National Institute of Allergy and Infectious Disease (NIAID), and the 

National Heart, Lung, and Blood Institute (NHLBI).9 In addition to 

interviewing the leadership of the selected ICs, we also interviewed 

program officers from the selected ICs who work directly with NIH 

awardees. We also interviewed officials from the Society for Women’s 

Health Research, the Women’s Health Research Institute at Northwestern 

University, and the editors of one major medical journal, as well as other 

individual researchers and experts, to gain perspectives on women’s 

9

NIH is composed of 27 institutes and centers, each with its own mission; 24 of the 27 ICs 

receive their own appropriation from Congress.

Page 4 GAO-16-13 Women in NIH Clinical Trials

 
inclusion in biomedical research supported by NIH and others and on the 

analysis and reporting of research outcomes.

To describe the level of women’s enrollment and NIH’s efforts to monitor 

women’s enrollment in NIH-funded clinical research, we obtained and 

reviewed enrollment data for all NIH-funded clinical research studies 

across NIH and for each of the ICs. For the aggregate NIH data, we 

reviewed 10 years of enrollment data (fiscal years 2005-2014), and for IClevel data, we reviewed 4 years of enrollment data (fiscal years 2011-

2014) for the 25 ICs that collect and report such data.10 We also reviewed 

documentation of NIH’s data collection tools, and interviewed NIH and IC 

officials—from the offices identified above—regarding their oversight of 

enrollment data. Through our review of the data and supporting 

documentation and our interviews with NIH and IC officials, we 

determined these enrollment data to be sufficiently reliable for our 

purposes. As part of our review, we examined the extent to which NIH’s 

monitoring efforts were consistent with the standards for internal control in 

the federal government—specifically those related to information, 

communication, and monitoring.11

To describe NIH’s efforts to ensure that NIH-funded clinical trials are 

designed and conducted to analyze potential sex differences, when 

applicable, we collected and reviewed NIH guidance and tools regarding 

how to assess applicants’ and awardees’ compliance with the analysis 

requirement of the Inclusion Policy, as it applies to certain clinical trials. 

We reviewed NIH reports and documentation related to implementation of 

the Inclusion Policy. We also interviewed NIH and IC officials specifically 

regarding their efforts to monitor implementation of the analysis 

requirement of the Inclusion Policy. As part of our review, we examined 

the extent to which NIH’s efforts were consistent with the standards for 

10IC-level enrollment data were only available for these 4 years (fiscal years 2011 through 

2014), as data from fiscal year 2010 and earlier were not collected or reported in the same 

manner, making the data not comparable to data collected and reported in fiscal year 

2011 and later. In addition, we report on enrollment for only 25 of the 27 ICs because 2 

ICs, the Center for Information Technology and the Center for Scientific Review, do not 

conduct or fund any clinical research studies, and thus are not included in our discussion 

of IC enrollment.

11GAO, Internal Control: Standards for Internal Control in the Federal Government, 

GAO/AIMD-00-21.3.1 (Washington, D.C.: November 1999). Internal control is 

synonymous with management control and comprises the plans, methods, and 

procedures used to meet missions, goals, and objectives.

Page 5 GAO-16-13 Women in NIH Clinical Trials

 
internal control in the federal government—specifically those related to 

control activities, information, communication, and monitoring.12

To describe NIH’s efforts to help ensure that researchers make the 

results of clinical trials public, including sex-specific results when 

appropriate, we reviewed NIH policies and procedures regarding the 

reporting of clinical trial results (particularly through public venues), 

including proposed policies, and documentation of NIH activities related 

to sharing of clinical trial results. We examined the NIH registry and 

results website—ClinicalTrials.gov—and related materials. We also 

reviewed the publication policies of selected scientific journals to 

determine whether they addressed reporting of results by sex.13

We conducted this performance audit from September 2014 to October 

2015 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.

Headed by a presidentially appointed and Senate-confirmed director, NIH 

comprises 27 ICs and an Office of the Director. NIH’s ICs both conduct 

and support biomedical research specific to their unique missions, which 

generally focus on a specific disease, a particular organ, or a stage in life 

(e.g., childhood). Each of the ICs has its own director and staff, as well as 

its own advisory council or board, which helps to support and oversee the 

IC’s work. Within the Office of the Director are offices responsible for 

issues, programs, and activities that span NIH components, particularly 

research initiatives and issues involving multiple ICs.

NIH’s biomedical research that focuses on humans—its clinical research 

studies—includes clinical trials of biomedical or behavioral interventions 

such as new drugs, medical treatments, and surgical procedures and 

12GAO/AIMD-00-21.3.1.

13These publications included JAMA, The Lancet, Nature, Surgery, and other peerreviewed journals identified by Stanford University’s Gendered Innovations project.

Background

NIH-Supported Research 

and Clinical Trials

Page 6 GAO-16-13 Women in NIH Clinical Trials

 
devices.14 Clinical trials are divided into four phases. In phase I clinical 

trials, which typically include 20 to 80 people, researchers test a new 

biomedical or behavioral intervention on human subjects for the first time 

to evaluate safety. In phase II clinical trials, the intervention is given to a 

larger group of people, 100 to 300 participants, to further evaluate 

efficacy and safety. In phase III clinical trials, the intervention is given to 

even larger groups—from several hundred to several thousand 

participants—to compare the intervention to commonly used or 

experimental interventions.15 Finally, phase IV studies are conducted after 

the intervention has been marketed, in order to gather information on 

long-term use.

NIH’s ICs support clinical trials predominantly through “extramural 

research”—awarding funds to researchers at universities or other 

research entities (awardees) through grants, contracts, and cooperative 

agreements.16 Of NIH’s 27 ICs, almost all fund extramural research 

projects. These ICs use a standard peer review process to inform the final 

decisions on which extramural research projects to fund.17 The size and 

composition of the ICs’ clinical trial portfolios vary substantially, 

14NIH’s clinical research on human subjects is considered applied research. NIH funds 

both applied research and basic research, which does not directly involve human subjects 

or direct testing of treatments.

15In this report, when we use the term “phase III clinical trials,” we are referring to NIHdefined phase III clinical trials. An NIH-defined phase III clinical trial is a broadly based 

prospective clinical investigation, usually involving several hundred or more human 

subjects, for the purpose of evaluating an experimental intervention in comparison with a 

standard or control intervention or comparing two or more existing treatments. Often the 

aim of such investigation is to provide evidence leading to a scientific basis for considering 

a change in health policy or standards of care. The definition includes pharmacologic, 

non-pharmacologic, and behavioral interventions given for disease prevention, 

prophylaxis, diagnosis, or therapy. Community trials and other population-based 

intervention trials are also included.

16Extramural research represents about 80 percent of NIH’s budget. Intramural research, 

which represents about 10 percent of NIH’s budget, involves research performed by NIH 

scientists in NIH laboratories.

17When reviewing applications for extramural research studies, NIH follows a process of 

peer review, established by law. This peer review system has two sequential levels of 

peer review. 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 nonscience fields, including patient advocates, who, in addition to scientific merit, also 

consider the mission and strategic plan goals, public health needs, scientific opportunities, 

and portfolio balance of the IC funding the research. After NIH’s peer review process is 

concluded, IC directors make extramural funding decisions.

Page 7 GAO-16-13 Women in NIH Clinical Trials

 
depending on such factors as the IC’s budget, mission, and the scientific 

goals of any given study. For example, some ICs support few if any 

phase III clinical trials.

In fiscal year 2014, NIH’s ICs reported funding of nearly $30 billion for all 

biomedical research.18 Of that amount, NIH estimates that—based on 

reporting categories HHS developed for use by all of its agencies— 

$23.9 billion (80.3 percent) funded research related to the health of both 

women and men, $4 billion (13.2 percent) funded research related to 

women’s health, and an estimated $1.9 billion (6.4 percent) funded 

research related to men’s health. (See Appendix II for more details on 

estimated fiscal year 2014 funding for selected diseases and conditions of 

particular relevance for women.) To determine these amounts, NIH 

annually assigns its research funding to certain women’s health disease 

and condition categories—such as breast cancer or heart disease. 

Additionally, NIH classifies this funding as either related to the health of 

both sexes or as supporting research on women’s health only or men’s 

health only by using HHS’s calculation guidelines.19 NIH reports these 

funding estimates in HHS’s annual congressional budget justification and 

in ORWH’s biennial Report of the Advisory Committee on Research on 

Women’s Health.

20

The Revitalization Act required NIH to ensure the appropriate inclusion of 

women in NIH-funded clinical research, including clinical trials. The 

Revitalization Act contained provisions that required NIH to, among other 

things, ensure that women are included in all NIH-funded clinical 

research, and report on compliance with the inclusion provisions of the 

Act. Additionally, the Revitalization Act requires NIH to ensure that clinical 

trials are designed and carried out in a manner sufficient to allow for valid 

analysis of the extent to which the outcomes measured in the trial affect 

women differently than men. NIH subsequently determined that this 

18Amounts reported by NIH are based on obligations and not actual expenditures. 

19HHS’s calculation guidelines are based on enrollment for trials involving human 

subjects. For research in which men and women are included, but enrollment information 

is not known, agencies are instructed to multiply spending by 50 percent to determine how 

much was spent on women’s health and men’s health, respectively. 

20For a link to the report, visit http://orwh.od.nih.gov/about/acrwh/index.asp, which we 

accessed July 21, 2015. 

The NIH Revitalization Act 

of 1993 and NIH Inclusion 

Policy

Page 8 GAO-16-13 Women in NIH Clinical Trials
particular requirement only applied to phase III clinical trials, as they are 

the largest clinical trials involving human subjects and the closest to 

effecting broad changes in public health policy and standards of care.

The Revitalization Act also directed NIH to develop guidelines for 

including women in clinical research and report biennially to Congress on 

NIH’s compliance with this policy. The resulting Inclusion Policy, the 

current version of which has been in place since October 2001, requires 

NIH applicants conducting clinical research to

1. design research plans that detail the breakdown of their studies’ 

participants by sex and provide a rationale for their planned 

enrollment for all clinical research studies; and

2. include plans for analyzing outcomes for potential sex differences for 

NIH-defined phase III clinical trials, when appropriate, as determined 

by consideration of prior scientific evidence.21

In addition to NIH ICs, several NIH offices play a role in implementing the 

Inclusion Policy, particularly ORWH, which was established in 1990—and 

codified in the Revitalization Act—to promote women’s health research, 

and OER, which administers and manages NIH grants policies, 

operations, and data systems. Since November 2011, the implementation 

of NIH’s Inclusion Policy has been overseen by the Subcommittee on 

Inclusion Governance, which comprises senior NIH officials from ORWH, 

21Specifically, the Inclusion Policy states that researchers must consider prior studies to 

determine if (1) prior studies support the existence of significant sex differences in 

intervention effect, (2) prior studies neither support nor negate the existence of sex 

differences, or (3) prior studies support the existence of no significant differences. A

significant difference is defined as a difference of clinical or public health importance. If 

prior studies either support the existence of sex differences or neither support or negate 

the existence of sex differences, then researchers must include in their applications plans 

for collecting data and conducting valid analysis to identify potential sex differences in the 

effect of the intervention being studied. If prior studies support no significant sex 

differences in intervention effect, a specific analysis is not required; however the inclusion 

and analysis of sex is still strongly encouraged.

Page 9 GAO-16-13 Women in NIH Clinical Trials

 
OER, and several ICs.22 This committee, co-chaired by the ORWH 

director and staffed by the NIH Inclusion Policy Officer from OER, is 

charged with examining and considering current NIH policies related to 

the inclusion of women in NIH-funded clinical research. In addition, the 

implementation of the Inclusion Policy is monitored by the Advisory 

Committee on Research on Women’s Health (Women’s Health Advisory 

Committee), whose creation was mandated by the Revitalization Act, as 

well as by the individual IC’s directors and advisory councils or boards.23

During the peer review process, applicants’ plans for including women, as 

appropriate, are reviewed and assessed along with the applicants’ plans 

to meet other requirements or considerations that are outlined in the 

funding opportunity announcement or research solicitation. The outcome 

of these assessments—scores from the peer reviewers—inform the 

funding decisions made by the ICs. Prior to awards being made, program 

officers or other IC staff may advise awardees on additional information 

required before an award can be released, and the resolution of any 

concerns raised during the peer review stage—including concerns related 

to adherence to the Inclusion Policy. After awards are made, NIH’s 

awardees are responsible for managing their day-to-day activities in 

accordance with NIH requirements, and the IC making the award is 

responsible for the awarded funds and for monitoring progress and 

compliance with NIH policies, including the Inclusion Policy. IC program 

officers monitor awardees through a variety of methods—including 

reviews of reports and correspondence from the awardee, and site 

visits—to identify potential problems with scientific progress, compliance, 

and areas where technical assistance might be necessary. One such 

22Prior to 2011, NIH had other means for monitoring inclusion in clinical research. The first 

electronic data system for monitoring inclusion was deployed in 2002. In 2009, an internal 

task force was formed to consider approaches for accomplishing the goals of NIH’s 

Inclusion Policy. A specific recommendation was the restructuring of inclusion governance 

to align it more closely with the overall governance structure at NIH. The result is that this 

subcommittee is part of the overall NIH governance structure and reports to NIH’s 

Extramural Activities Working Group. Additionally, in 2011, NIH hired an Inclusion Policy 

Officer to provide a centralized point of contact to coordinate policies, procedures, and 

reporting related to the implementation of the Inclusion Policy.

23Women’s Health Advisory Committee members, who meet twice a year, are selected 

from among physicians, practitioners, scientists, and other health professionals who are 

not federal employees and who have a clinical practice, research specialization, or 

professional expertise that includes a significant focus on research relevant to women’s 

health. Also, by law, a majority of the members of the committee shall be women.

NIH’s Extramural Awards 

Management Process

Page 10 GAO-16-13 Women in NIH Clinical Trials

 
report that program officers review is the annual progress report—which 

includes data on study enrollment, among other things. Awardees submit 

information, including enrollment data, through the Electronic Research 

Administration (eRA) Commons, which is part of NIH’s electronic data 

collection and grants administration system that is used by awardees and 

program officers to access and share administrative information related to 

research awards.

NIH data show that over the last decade more women than men have 

been enrolled across all NIH-funded clinical research, including phase III 

clinical trials. NIH publicly reports aggregate enrollment numbers on a 

biennial basis; however, it does not routinely make detailed IC enrollment 

data readily available or examine more detailed enrollment data by 

disease and condition, in order to identify potential challenges to enrolling 

women in certain research and disease or condition areas.

NIH requires each awardee to report enrollment, including enrollment by 

sex, for each of their NIH-funded research awards, including phase III 

clinical trials. As of fiscal year 2015, these data are reported to NIH 

through its Inclusion Monitoring System—one part of NIH’s awardee data 

system. Program officers review these data at least annually to determine 

whether actual enrollment is consistent with the enrollment planned in the 

research design.24 The program officers use a designated checklist, 

among other tools, to document their monitoring as part of the annual 

progress report review process. According to NIH officials, awardee 

enrollment data are aggregated by each IC and presented in and 

discussed during meetings held by each IC’s advisory board or council, 

which are open to the public. The IC-level enrollment data are certified as 

24Program officers review the progress report and inclusion enrollment data and 

determine whether progress is satisfactory. If there are any concerns with progress or 

compliance, they may follow-up with the awardee in writing, by email, by phone, or at 

times, with a site visit.

More Women Than 

Men Were Enrolled in 

NIH Research 

Overall, but NIH Does 

Not Examine Detailed 

Data to Identify 

Potential Challenges 

to Enrolling Women

NIH Data Show More 

Women than Men Enrolled 

in NIH-Funded Clinical 

Research

Page 11 GAO-16-13 Women in NIH Clinical Trials

 
being compliant with the Inclusion Policy by the IC’s advisory board or 

council and by the IC Director and included in an IC-level enrollment 

report. The certified IC enrollment reports are submitted to ORWH and 

OER, where, according to NIH officials, the data are checked for 

consistency and errors as part of a quality control process.25 NIH 

aggregates the enrollment data across the agency and reports this 

aggregate data to the Women’s Health Advisory Committee, Congress, 

and the public in NIH’s biennial inclusion report.

According to the data collected by NIH, in each fiscal year from 2005 

through 2014, more women than men were enrolled in all NIH-funded 

clinical research studies, including phase III trials. (See fig. 1.) 

Specifically, in fiscal year 2014, among all NIH-funded clinical 

research studies, 57 percent of enrollees (16.4 million) were women, 

and 39 percent (11 million) were men.26 For all NIH-funded phase III 

clinical trials, in fiscal year 2014, 60 percent of enrollees were women 

(about 480,000) and 39 percent were men (about 314,000).27

25For example, NIH officials told us they look for trends in enrollment across several 

years. According to the officials, if their review of trends reveals large shifts in enrollment, 

they will conduct additional analyses to determine whether one or more large studies may 

be contributing to the observed shifts in enrollment.

26The sex of 4 percent of enrollees across all NIH-funded clinical research studies was 

unknown.

27The sex of less than 1 percent of enrollees in NIH-funded phase III clinical trials was 

unknown. 

Page 12 GAO-16-13 Women in NIH Clinical Trials

 
Figure 1: Enrollment in All National Institutes of Health (NIH) Funded Clinical Research by Sex, as a Percentage of Total 

Enrollment, Fiscal Years 2005-2014

At the individual IC level, data show that for each IC, women’s enrollment 

for all clinical research studies, including phase III clinical trials, was 

generally higher than men’s enrollment in most years from fiscal year 

2011 through fiscal year 2014. (See app. III for enrollment data at the NIH 

and IC level over this period.) Of the 25 ICs reporting enrollment in fiscal 

years 2011 through 2014, 13 ICs enrolled more women than men in the 

ICs’ clinical research studies during all four years.28 An additional 4 ICs 

28Two ICs, the Center for Information Technology and the Center for Scientific Review, do 

not conduct or fund any clinical research studies or phase III trials, and thus, are not 

included in our discussion of IC enrollment. Additionally, we do not include the National 

Center for Research Resources in our IC discussion, as it ceased operations in fiscal year 

2012, or the Office of Director and ORWH, as they are not ICs. However, each of these 

three entities funded clinical research or phase III trials in one or more years during the 

fiscal year 2013-2014 time period, and as such, their enrollment data are included in the 

totals for overall NIH clinical research studies and phase III trials.

Page 13 GAO-16-13 Women in NIH Clinical Trials

 
enrolled more women than men in 3 of the fiscal years between 2011 and 

2014. According to NIH, 10 of the 25 ICs regularly support phase III 

clinical trials, and of these, 3 (about one third) enrolled more women than 

men in each year in fiscal years 2011 through 2014, and another 2 

enrolled more women than men in 3 of the 4 fiscal years.

NIH collects and reviews aggregated enrollment data from the ICs; 

however, NIH officials do not make these IC-level enrollment data readily 

available to the public or other interested parties. Specifically, the IC-level 

enrollment data are not published as part of the overall NIH biennial 

report on enrollment, are not shared with the Women’s Health Advisory 

Committee, and are not available for download from the ORWH website. 

Individual official IC enrollment reports were also not available through 

the websites of the three ICs in our review. Beginning in the fiscal year 

2013-2014 reporting period, NIH required all ICs to submit their IC 

enrollment report numbers in a standard format, which may allow for 

easier public sharing of these data going forward.

Additionally, NIH officials do not routinely examine detailed enrollment 

data by sex beyond the IC level—such as by a specific research area or 

disease or condition being studied—to identify potential challenges to 

enrolling women in these areas, because enrollment data are currently 

not available in this format. The guidance NIH provided to the ICs for 

submitting their fiscal year 2013-2014 enrollment data to ORWH and 

OER explains that ICs may choose to further break out their reported 

enrollment data by disease area, portfolio area, or in some other manner. 

According to NIH officials, certain ICs analyze enrollment data by various

disease categories when necessary. However, the IC officials we spoke 

with told us that their data systems are not capable of systematically 

aggregating enrollment data in this manner for routine reporting. When 

asked if it would be possible to aggregate enrollment data by disease and 

condition, IC officials said that they would be able to do so, but it would be 

a time intensive, manual process given current data system limitations. 

Further, NIH officials told us they expect NIH’s new enrollment data 

system deployed in October 2014 to increase functionality in examining 

enrollment data in different ways, but as of July 2015 the officials did not 

have specific plans or details available. In addition, NIH officials stated 

that because an individual IC’s research generally focuses on a specific 

disease, a particular organ, or a stage in life, the enrollment data that are 

aggregated at the IC level would roughly correspond with major disease 

and condition categories and could be used, to some extent, as a proxy 

for disease and condition enrollment data. However, this proxy method 

NIH Does Not Make ICLevel Enrollment Data 

Readily Available or 

Examine Detailed 

Enrollment Data to Identify 

Potential Challenges to 

Enrolling Women in 

Specific Research and 

Disease Areas

Page 14 GAO-16-13 Women in NIH Clinical Trials
does not take into account the fact that many ICs are responsible for 

research that includes multiple diseases, organs, or stages in life; for 

example, NHLBI’s research portfolio includes studies of heart, lung, and 

blood related diseases and conditions. In addition, research on certain 

diseases and conditions—such as obesity—falls under the purview of 

multiple ICs.

NIH’s practices of not sharing the IC-level enrollment data and not 

examining detailed data on enrollment by sex—by specific research area 

or the disease or condition being studied—are inconsistent with several 

federal standards for internal control.29 Specifically, the internal control 

standards for information and communications state that for an entity to 

run and control its operations, it must have relevant, reliable, and timely 

communications relating to internal as well as external events. 

Information is needed throughout the agency to achieve all of its 

objectives, and effective communication should occur in a broad sense, 

with information flowing down, across, and up the organization. In 

addition to internal communications, management should ensure there 

are adequate means of communicating with, and obtaining information 

from, external stakeholders who may have a significant impact on the 

agency achieving its goals (e.g., the Women’s Health Advisory 

Committee). Federal internal control standards for monitoring call for 

management to assess the quality of agency performance over time and 

ensure that the findings of audits and other reviews are promptly 

resolved.

Because NIH does not readily share IC-level enrollment data with the 

public and other interested parties, such as the Women’s Health Advisory 

Committee—through means such as the ORWH website or the biennial 

NIH enrollment report—those interested in reviewing IC-level enrollment 

information would have to attend—or watch online if webcast—each 

individual IC’s advisory board or council meeting or specifically seek out 

or request any public record resulting from these meetings to have 

access to these data. In addition, by not routinely examining more 

detailed enrollment data that is aggregated by sex—such as data at the 

disease and condition level—NIH is limited in its ability to identify whether 

women are sufficiently represented in studies in specific areas that cross 

ICs—such as obesity. Further, NIH does not have information of sufficient 

29GAO/AIMD-00-21.3.1. 

Page 15 GAO-16-13 Women in NIH Clinical Trials

 
detail to monitor and determine if the aggregate enrollment data from 

across NIH inadvertently mask low enrollment for particular research 

areas or diseases or conditions. At an April 2015 Women’s Health 

Advisory Committee meeting, some committee members raised such 

concerns, noting that published studies on clinical trials of specific 

diseases and conditions, such as cardiovascular disease, appeared to 

show that women’s enrollment was lower than the enrollment that NIH 

had reported in the aggregate. The committee members acknowledged 

that there could be many reasons for such discrepancies, but noted that 

they would like to see more detailed enrollment data to improve their 

understanding of the data and ensure that women are being appropriately 

included in NIH-funded clinical trials.

NIH’s Inclusion Policy requires that individual awardees conducting phase 

III clinical trials consider whether analysis of potential differences in study 

outcomes between women and men is needed in their studies, consistent 

with the Revitalization Act’s provisions regarding the design of certain 

clinical trials. However, the agency does not maintain, analyze, or report 

summary data to oversee whether analysis of outcomes by sex are 

planned or conducted, when applicable, across all NIH-funded clinical 

trials.

Under the Inclusion Policy, applicants seeking funds for phase III clinical 

trials must consider prior scientific evidence and assess whether an 

analysis of potential sex differences is merited, and if so, develop a plan 

to analyze study results accordingly.30 Both this consideration and the 

plan for analysis, if appropriate, are to be included in the awardee’s 

application for funding.31 To ensure awardees’ compliance with this 

30NIH’s policy states that awardees need to plan on conducting an analysis of potential 

sex differences if the findings of previous studies (1) support or (2) neither support nor 

negate the existence of sex differences. If prior studies support the existence of no 

significant differences, no further analysis is required.

31In June 2015, NIH announced plans for a revised policy requiring the consideration of 

sex as a biological variable in applications for studies of vertebrate animals and humans, 

stating that failing to account for sex as a biological variable in all biomedical research 

may undermine the rigor, transparency and generalizability of research findings. This 

revised policy will result in changes to fiscal year 2016 research grant applications, to be 

in effect for fiscal year 2017 funding. According to NIH, this change will not alter the 

existing Inclusion Policy, but over time, the change will build a better knowledge base 

about the influence of sex as a biological variable and better inform the design of clinical 

research going forward.

NIH Does Not Record 

Whether Clinical 

Trials Will Analyze 

Sex Differences to 

Allow Summary 

Analysis and 

Reporting for 

Oversight of Its 

Inclusion Policy

Page 16 GAO-16-13 Women in NIH Clinical Trials

 
requirement, NIH officials told us they rely on the agency’s peer review 

process for reviewing applications, and after awards are made, on IC 

program officers’ monitoring of individual awardees. NIH has guidelines 

for peer reviewers to use when assessing applications and rating 

applicants’ inclusion plans during peer review. This review typically 

includes an evaluation of the proposed study design and assessment of 

whether any plans for conducting an analysis of potential sex differences 

are “acceptable” or “unacceptable.” The assessment and rating are based 

on consideration of prior scientific evidence that either supports or 

negates the existence of differences in outcomes by sex. Peer reviewers 

document this assessment in a summary statement provided to the ICs 

for final award determinations. According to NIH officials, if reviewers 

determine that an applicant’s plans are not acceptable, the applicant is 

barred from funding until the plans are addressed and deemed 

acceptable by IC officials. After awards are made, program officers from 

the ICs that fund the studies are responsible for monitoring awardees’ 

overall progress on an ongoing basis. Specifically, program officers are to 

review awardees’ annual progress reports, which the Inclusion Policy 

states should address analysis of potential sex differences, as 

appropriate.32 Officials from one IC told us that, through their regular 

interactions with awardees, program officers are very familiar with the 

design of their awardees’ trials and whether any such analysis is planned 

or underway.

NIH program officers monitor individual awardees’ compliance with the 

analysis requirement of the Inclusion Policy; however, the agency lacks 

summary data on awardees’ analysis plans, including the percentage of 

awardees in a given year with trials designed to identify potential sex 

differences, when applicable. Currently, program officers review 

awardees’ progress reports—including any information reported regarding 

the analysis of potential sex differences—but do not have means, such as 

a written checklist or a required field in an electronic reporting system, for 

recording the information obtained through this monitoring, as they do for 

monitoring enrollment. NIH’s awardee data system includes information 

on whether individual awards include phase III trials. However, the data 

system does not have a data element that denotes whether an awardee’s 

study should include or has plans for an analysis of potential sex 

32The NIH guidance for awardees completing the annual progress report states “if 

analysis has begun or data has been published, report any progress made in evaluating 

potential differences by sex.”

Page 17 GAO-16-13 Women in NIH Clinical Trials

 
differences. Although such information is included in the narrative 

included in awardees’ funding applications, this information cannot be 

easily aggregated for summary reporting, NIH officials explained, and 

therefore they do not estimate the proportion of trials being conducted at 

any one time that are designed and explicitly intended to identify 

differences in study outcomes by sex. NIH officials also told us that they 

plan to add a question for this type of monitoring to the existing electronic 

checklist used by program officers in the fall of this year, to be 

implemented for awards funded in fiscal year 2016.

Because NIH does not have summary data regarding the analysis 

requirement of the Inclusion Policy, it has not reported summary 

information on this aspect of the Inclusion Policy to key stakeholders—

including the Women’s Health Advisory Committee and the Congress. 

Notably, NIH’s agency-wide biennial reports on the status of the Inclusion 

Policy do not include information on the extent to which NIH-funded 

phase III trials included plans to conduct analyses of potential sex 

differences, or on the overall status of this aspect of the Inclusion Policy. 

Instead, the report focuses primarily on NIH-wide aggregate enrollment.33

NIH officials told us they rely on program officers’ monitoring of individual 

awardees to ensure that the analysis requirement of the Inclusion Policy 

is being implemented appropriately after awards are made, because part 

of the program officer’s role is to ensure satisfactory scientific progress as 

well as compliance with NIH policies. The officials added that they were 

not sure of the utility of summary reporting in this case.

NIH’s lack of summary data and reporting regarding the analysis 

requirement of the Inclusion Policy conflicts with federal internal control 

standards.34 First, federal internal control standards require that federal 

agencies have control activities in place to ensure that management’s 

directives are carried out and that these controls are monitored. These 

standards also state that information should be recorded and 

communicated to management and other responsible officials in a form 

and within a time frame that enables them to carry out their internal 

control and other responsibilities.

33The report also includes information on the number of applications with inclusion plans 

considered acceptable and unacceptable at peer review, which NIH officials stated is an 

important part of ensuring compliance with this requirement.

34GAO/AIMD-00-21.3.1. 

Page 18 GAO-16-13 Women in NIH Clinical Trials

 
Without summary data on the funded phase III clinical trials that are 

intended to provide information on potential sex differences—including 

the number of such trials funded in a given year—senior NIH officials are 

limited in their ability to effectively oversee the implementation of the 

Inclusion Policy to assess whether changes are needed to their 

procedures. Further, NIH cannot provide this information to stakeholders 

such as the Women’s Health Advisory Committee and Congress in its 

regular reporting on other aspects of the Inclusion Policy. As a result, 

these stakeholders lack assurance that the agency is implementing the 

Inclusion Policy as intended and in a manner consistent with the 

Revitalization Act’s provisions regarding the design of certain clinical 

trials. In its fiscal year 2011-2012 Report of the Advisory Committee on 

Research on Women’s Health, NIH previously acknowledged that 

inclusion is not just a matter of having women and men included in clinical 

studies; rather, the scientific value of research studies is greatly 

enhanced by providing knowledge about differences and/or similarities 

between different populations affected by the diseases under study. 

However, without assurance that its clinical trials are being designed and 

conducted as directed under the law and its implementing policy, NIH’s 

insight regarding the interpretation, validation, and generalizability of 

findings resulting from the research it supports—as these findings apply 

to both women and men—is diminished, potentially limiting the value of 

NIH-funded research.

Page 19 GAO-16-13 Women in NIH Clinical Trials
NIH has developed and proposed a policy intended to increase public 

reporting of clinical trial results by requiring all NIH-funded clinical trials to 

be registered and have results submitted to its registry and results 

database, ClinicalTrials.gov.35 ClinicalTrials.gov contains accessible and 

searchable information on publicly and privately supported clinical trials 

and observational studies that is provided and updated by NIH awardees 

and other researchers. Currently, all NIH awardees are encouraged to 

register their trials with ClinicalTrials.gov at the beginning of the study, but 

only certain trials—those of certain drugs and devices regulated by the 

Food and Drug Administration—must register and provide summary 

results as required by law.36 According to NIH officials, as of July 2015, 

there were approximately 195,000 studies registered in ClinicalTrials.gov, 

and almost 18,000 of these have summary results information posted. 

Under the proposed NIH policy, all NIH-funded clinical trials, regardless of 

trial phase or type of intervention being studied, would have to be 

registered and submit summary results—including participant flow, 

baseline demographics such as the sex and age of participants, primary 

35ClinicalTrials.gov was created in response to the Food and Drug Administration 

Modernization Act of 1997, which required HHS, through NIH, to establish a registry of 

clinical trials information for both federally and privately funded trials of experimental drugs 

for serious or life-threatening diseases or conditions. Pub. L. No. 105-115, § 113, 111 

Stat. 2296, 2310-12 (Nov. 21, 1997) (codified as amended at 42 U.S.C. § 282(i)). NIH’s 

National Library of Medicine manages ClinicalTrials.gov.

At the same time that NIH issued its proposed policy—in November 2014—HHS 

published for public comment a proposed rule to clarify and expand (as permitted) the 

Food and Drug Administration Amendments Act of 2007 requirements for certain clinical 

trials of Food and Drug Administration-regulated products to register and submit summary 

results to ClinicalTrials.gov. Pub. L. No. 110-85, § 801, 121 Stat. 823, 904-22 (Sept. 27, 

2007) (codified as amended at 42 U.S.C. § 282(j)). NIH officials anticipate the final policy 

will be issued in the first quarter of fiscal year 2016.

36Applicable clinical trials subject to the ClinicalTrials.gov reporting requirements of the 

Food and Drug Administration Amendments Act of 2007 generally include: (1) trials of 

drugs: controlled clinical investigations—other than phase I investigations—of a drug 

subject to FDA regulation authorized by section 505 of the Federal Food Drug, and 

Cosmetic Act or section 351 of the Public Health Service Act, and (2) trials of devices 

subject to sections 510(k), 515, and 520(m) of the Federal Food, Drug, and Cosmetic Act: 

controlled trials with health outcomes, other than small feasibility studies, and pediatric 

postmarket surveillance. Pub. L. No. 110-85, § 801, 121 Stat. 823, 904-22 (Sept. 27, 

2007) (codified as amended at 42 U.S.C. § 282(j)).

Proposed NIH Policy 

Intended to Increase 

Public Reporting of 

Clinical Trial Results 

by Awardees and 

Could Increase 

Reporting of SexSpecific Results, 

According to NIH 

Officials

Page 20 GAO-16-13 Women in NIH Clinical Trials

 
and secondary outcomes, and adverse events—to ClinicalTrials.gov.37

According to NIH, if the proposed policy goes into effect, compliance may 

be enforced through possible suspension or termination of funding and 

noncompliance could impact future funding decisions. NIH sought public 

comments on the proposed policy from November 2014 through March 

2015 and, as of August 2015, was analyzing the comments it received. 

NIH anticipates that the final policy will be issued in the first quarter of 

fiscal year 2016.

NIH officials told us that the proposed policy for clinical trial registration 

and results submission was not intended to increase reporting of sexspecific results to ClinicalTrials.gov; however, the officials also said that 

there is the potential for more reporting of sex-specific results, given the 

overall increase expected in the number of reported studies. Since the 

proposed policy would require awardees to report results for their 

prespecified primary and secondary outcome measures as part of the 

summary results submission, the reporting of sex-specific results would 

depend on the design of the trial, according to NIH officials. Specifically, if 

sex differences were among the prespecified primary and secondary 

outcomes studied in a specific trial, officials said, then that would be 

reflected in the results submitted to ClinicalTrials.gov.

In issuing the proposed policy, NIH stated that its awardees are expected 

to make their trial results available to the research community and to the 

public at large in order to contribute to scientific knowledge and, 

ultimately, public health. NIH proposed the policy partly in response to a 

recent study, which found that within 30 months of trial completion, the 

results of less than half of NIH-funded clinical trials had been published in 

a peer-reviewed biomedical journal, the traditional method for sharing 

results.38 NIH stated that because journal publication of clinical trials 

results is not always possible, it is important to provide other ways for 

clinical trial results to be disseminated and publicly available to 

researchers, health care providers, and others.

37Participant flow is a description of the number of research participants starting and 

completing the study. HHS defines as “primary” the outcome measure that is considered 

the most important, although more than one may be appropriate under specific 

circumstances. The proposed definition of “secondary outcome measure” would include all 

pre-specified outcome measures that are not designated as “primary” and for which a 

specific analysis plan exists, either in the protocol or the statistical analysis plan.

38J. S. Ross, et al. “Publication of NIH funded trials.”

Page 21 GAO-16-13 Women in NIH Clinical Trials

 
NIH has made efforts to encourage sex-specific reporting of clinical trial 

results. In NIH’s fiscal years 2011-2012 Report on the Advisory 

Committee for Research on Women’s Health, ORWH stated that it is only 

through sex-specific reporting that full information becomes available to 

the public and to scientists who can then use such data to inform future 

studies, thereby building the knowledge base in a manner that takes into 

consideration the influences of sex on health and disease. Specifically, 

NIH has worked with journal editors and others to encourage reporting of 

results by sex. Specifically, in 2011, NIH asked IOM to convene a 

workshop of researchers, journal editors, and others on the topic to 

discuss the importance of reporting results by sex and the implications of 

this issue for journals’ reporting policies. According to Stanford 

University’s Gendered Innovations project, 32 peer-reviewed journals 

worldwide have editorial policies requiring that clinical trial researchers 

include information on results by sex when they submit articles for 

publication.39 However, editors from one medical journal that we spoke 

with stated that when evaluating whether results should be reported by 

sex, it is important to consider whether examining sex differences is a 

primary outcome of the study, and whether the trial was big enough for a 

valid subgroup analysis—i.e., analysis of the effect of the intervention on 

two or more different groups of participants, such as women and men. 

They emphasized that if a study was not designed for a subgroup 

analysis by sex and one was performed, the results could be erroneous.

NIH has also made efforts to facilitate the sharing of clinical trials results, 

including sex-specific information, through venues other than journals and 

ClinicalTrials.gov. NIH has a number of policies that promote the 

dissemination of research results—and the underlying data—and guide 

awardees in disseminating their results, including the NIH Data Sharing 

Policy, among others.40 Additionally, related to the sharing of sex-specific 

information, NIH hosts a Women’s Health Resources portal and a 

Women’s Health topic page on the Medline Plus webpage, which includes 

links to other information about women’s health from journal articles and 

ClinicalTrials.gov. The agency also reports summaries of research related 

39The Gendered Innovations project was initiated at Stanford University in 2009 to provide 

scientists and engineers with practical methods for sex and gender analysis. Gendered 

Innovations involves experts from across the U.S. and the European Union.

40Other NIH data sharing policies include the NIH Public Access Policy, the NIH Model 

Organism and Related Resources Sharing Policy, and the NIH Genomic Data Sharing 

Policy.

Page 22 GAO-16-13 Women in NIH Clinical Trials

 
to women’s health in the biennial Report of the Advisory Committee on 

Research on Women’s Health.

Although NIH has made progress in the 2 decades since the 1993 

Revitalization Act regarding the inclusion of women in NIH-funded clinical 

research, opportunities remain for NIH to further extend the value of its 

investment in medical research. NIH is responsible for ensuring that the 

nation receives the greatest benefit of the large federal investment in 

clinical research by fully implementing its Inclusion Policy, such that 

women are adequately included in NIH-funded clinical trials when 

appropriate, and that potential sex differences may be identified. By not 

readily sharing IC-level enrollment data, NIH limits the public’s and other 

interested parties’ ability to gain insight into enrollment issues at each of 

the ICs, putting the onus of obtaining these data on the interested parties 

themselves to attend or view online up to 25 individual IC board or council 

meetings or request any public record of these meetings. By not 

examining more detailed enrollment data—such as data aggregated by 

research area or specific to various diseases and conditions—NIH cannot 

know whether it is adequately including women across all of the research 

it supports. Without this greater insight into enrollment for specific to 

diseases and conditions, NIH is limited in its ability to assess whether its 

programs that support cross-cutting research spanning multiple ICs are 

successfully including women in clinical research or facing challenges 

that the agency should address. Further, the lack of summary data and 

reporting about the extent to which awardees plan to conduct or perform 

analyses of potential sex differences in phase III clinical trials 

compromises NIH’s oversight and jeopardizes the agency’s ability to 

provide assurances over the Act’s provisions regarding the design of 

certain clinical trials and meet the purposes of its Inclusion Policy. Without 

summary data, such as the proportion of trials being conducted that 

intend to analyze differences in outcomes for men and women, and 

reporting on that data, NIH and Congress cannot know whether or to what 

extent current efforts are helping to ensure that differences in clinical 

outcomes by sex are identified and that NIH is supporting research that 

can be used to shape improved medical practices for both women and 

men.

The overall increase in the enrollment of women in NIH-funded clinical 

research studies, such that women have been a significant proportion of 

research subjects for nearly 2 decades, is a noteworthy achievement for 

NIH. To continue to build on this achievement—and consistent with 

federal internal control standards—NIH should turn its focus to assessing 

Conclusions

Page 23 GAO-16-13 Women in NIH Clinical Trials
whether the agency is meeting the purposes of the Inclusion Policy, and if 

it is not, take the needed corrective actions.

To ensure effective implementation of the Inclusion Policy in a manner 

consistent with the Revitalization Act’s provisions regarding the design of 

certain clinical trials, the NIH Director should take the following five 

actions:

• make IC-level enrollment data readily available through public means, 

such as NIH’s regular biennial report to Congress on the inclusion of 

women in research, or through NIH’s website;

• examine approaches for aggregating more detailed enrollment data at 

the disease and condition level, and report on the status of this 

examination to key stakeholders and through its regular biennial 

report to Congress on the inclusion of women in research;

• ensure that program officers have a means for recording information 

obtained from monitoring awardees’ plans for and progress in 

conducting analyses of potential differences in outcomes by sex;

• on a regular basis, systematically collect and analyze summary data 

regarding awardees’ plans to conduct analyses of potential sex 

differences, such as the proportion of trials being conducted that 

intend to analyze differences in outcomes for men and women; and

• report on this summary data and the results of this analysis in NIH’s 

regular biennial report to Congress on the inclusion of women in 

research.

We provided a draft of this product to HHS for comment, and HHS 

responded with comments provided by NIH. In its written comments, 

reproduced in appendix IV, NIH generally concurred with our findings and 

recommendations. NIH also provided technical comments that were 

incorporated into the final report, as appropriate.

In commenting on our first recommendation to make IC-level enrollment 

data readily available to the public, NIH agreed and indicated that there 

are opportunities for the agency to increase the accessibility of IC-level 

enrollment data. NIH also stated that the agency has begun to 

standardize IC enrollment reporting and will continue this effort by 

standardizing data tables and graphics for ICs to provide for the NIH-wide 

Recommendations for 

Executive Action

Agency Comments 

and Our Evaluation

Page 24 GAO-16-13 Women in NIH Clinical Trials
biennial reports. NIH did not provide a timeline for making this information 

readily available to the public.

NIH agreed with our second recommendation to examine approaches for 

aggregating more detailed enrollment information at the disease and 

condition level, and to report on the status of this examination to key 

stakeholders. In its comments, NIH also reiterated what we describe in 

our report: some ICs conduct analysis of enrollment by disease or 

condition on an as-needed basis. NIH noted that the agency is working on 

ways to analyze enrollment at the disease and condition level across the 

ICs. NIH did not provide information on when the agency would be able to 

analyze these enrollment data, but it did state that when the agency is

able to perform the analysis, NIH would make the results readily available 

through NIH’s biennial inclusion reports or other means.

NIH agreed with our third recommendation to ensure that program 

officers have a means for recording their monitoring of awardees’ plans 

for and progress in conducting analysis of potential sex differences, and 

confirmed that the agency plans to add questions that would facilitate this 

type of monitoring into the existing checklist program officers use to 

document other types of monitoring beginning in fiscal year 2016.

In commenting on our fourth and fifth recommendations regarding 

collecting and reporting summary data on awardees’ plans for sexdifferences analysis, NIH agreed that it is critical to obtain more 

information on which clinical trials involve analyses of sex differences, 

and described some alternative data collection approaches for improving 

oversight of this issue. We maintain that thoughtful, useful analysis and 

summary reporting would improve NIH’s oversight of this aspect of the 

Inclusion Policy. Our recommendation was not intended to prescribe or 

limit the type of analysis performed or the data collected by NIH; instead

we provided an example that NIH could adapt as needed, and we 

encourage the agency to explore the best alternatives for their analyses. 

In other general comments, NIH also noted other opportunities that 

support oversight, such as the importance of peer reviewers in examining 

applicants’ plans for including women prior to funding decisions, and 

expanded reporting in ClinicalTrials.gov. In addition, the agency noted 

that ClinicalTrials.gov could engender greater transparency of clinical trial 

results and help assure that the analyses required under the Inclusion 

Policy are being completed.

Page 25 GAO-16-13 Women in NIH Clinical Trials
As agreed with your offices, 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 of this report 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 Web site at 

http://www.gao.gov.

If you or your staff have any questions about this report, please contact 

me at (202) 512-7114 or at kohnl@gao.gov. Contact points for our Offices 

of Congressional Relations and Public Affairs may be found on the last 

page of this report. GAO staff who made key contributions to this report 

are listed in appendix V.

Linda T. Kohn

Director, Health Care

Page 26 GAO-16-13 Women in NIH Clinical Trials
List of Requesters

The Honorable Barbara Mikulski

Ranking Member

Committee on Appropriations

United States Senate

The Honorable Patty Murray

Ranking Member

Committee on Health, Education, Labor, and Pensions

United States Senate

The Honorable Frank Pallone, Jr.

Ranking Member

Committee on Energy and Commerce

House of Representatives

The Honorable Kirsten Gillibrand

United States Senate

The Honorable Debbie Stabenow

United States Senate

The Honorable Elizabeth Warren

United States Senate

The Honorable Lois Capps

House of Representatives

The Honorable Rosa L. De Lauro

House of Representatives

The Honorable Nita Lowey

House of Representatives

Page 27 GAO-16-13 Women in NIH Clinical Trials
Appendix I: Literature Review of Factors 

Affecting Women’s Participation in Clinical 

Research

We reviewed 34 published journal articles that specifically identified 

barriers to or reasons for women’s participation in clinical research. We 

conducted an initial literature search that identified 168 studies published 

from January 2004 through October 2014 on the topic of women in 

clinical research.1 After reviewing abstracts and the full text of some 

articles, we narrowed this group down to the 34 articles included in this 

report that focus on the factors specifically affecting women’s participation 

in clinical research.2 We also spoke with or received written responses 

from officials and program officers from 3 institutes and centers (ICs) of 

the National Institutes of Health (NIH) about the factors affecting women’s 

participation in clinical research reported by NIH awardees.

In our review of the literature, the three most commonly cited barriers 

women face when considering whether to participate in clinical research 

were fear of experimentation/trust issues, health related concerns, and 

transportation/convenience issues. (See table 1.) Fear of 

experimentation/trust issues included distrust of physicians and the 

medical community and fear that the experimental treatment may be 

inferior to the conventional treatment(s). Health related concerns cited in 

the articles included concerns about the potential side effects or other 

adverse events associated with the experimental treatment. Some of the 

transportation/convenience concerns cited in the articles included 

difficulty traveling to a clinic (no transportation or a long distance to the 

facility) and general inconvenience associated with participating in the 

research.

1

We conducted a search of relevant databases—including MEDLINE, BIOSIS Previews, 

Embase, International Pharmaceutical Abstracts, NTIS: National Technical Information 

Service, PAIS International, PsycINFO, Sociological Abstracts, PapersFirst, WorldCat, 

and CINAHL—using search terms related to women and clinical trials. The search also 

focused on articles on U.S.-based populations.

2

We reviewed abstracts for all 168 articles, as available, and selected 71 articles for 

review of the full text.

Appendix I: Literature Review of Factors 

Affecting Women’s Participation in Clinical 

Research

Page 28 GAO-16-13 Women in NIH Clinical Trials

 
Appendix I: Literature Review of Factors 

Affecting Women’s Participation in Clinical 

Research

Table 1: Types of Barriers Identified for Women in Literature Review

Barrier Type Number of Articles 

Fear of experimentation/trust concerns 19

Health related concerns 19

Transportation/convenience issues 16

Other 15

Trial/study related concerns 13

Lack of or competing interests 12

Work/financial reasons 9

Home and family responsibilities 8

Cultural concerns/language issues 6

Lack of knowledge about research 5

Stigma 4

Source: GAO literature review. | GAO-16-13

The three most common reasons for women’s participation in clinical 

research identified in our literature review were personal/health benefits, 

altruism, and a general category of “other” reasons. (See table 2.) 

Personal/health benefits included access to new treatment and drugs, 

while “other” reasons included the fact that the research was being done 

at a clinic where the participant had already received care or was being 

conducted by clinical staff with whom the participants were familiar. 

Participation due to altruism included the desire to help science.

Table 2: Types of Reasons to Participate Identified for Women in Literature Review

Types of Reasons to Participate Number of Articles

Personal/health benefits 18

Other 15

Altruism 14

Financial benefits 11

Family/medical support and encouragement 10

Free care/services/treatment 6

Interest in research 5

Source: GAO literature review. | GAO-16-13

Page 29 GAO-16-13 Women in NIH Clinical Trials
Appendix I: Literature Review of Factors 

Affecting Women’s Participation in Clinical 

Research

The NIH IC officials and program officers we spoke with generally agreed 

that the barriers and reasons for participation that we identified through 

our literature review were consistent with those they have encountered 

when working with their awardees. In addition, officials from one IC we 

spoke with identified other reasons women participate in clinical research, 

as cited by awardees. These reasons included the opportunity to learn 

about the disease being studied and how to manage it, and a sense of 

pride associated with participation. In contrast, these officials stated that 

for some diseases, the effect or perceived effect on future fertility could 

be a barrier to women’s participation.

Page 30 GAO-16-13 Women in NIH Clinical Trials
Appendix II: National Institutes of Health (NIH)

Funding for Women’s Health Research

To determine the amount of NIH funding for research on women’s health, 

we collected and reviewed NIH women’s health budget information for 

fiscal years 2009-2014. We also interviewed officials from NIH and the 

Department of Health and Human Services (HHS) regarding the budget 

categories that NIH uses in its women’s health budget, which were 

originally developed for use in reporting by all HHS agencies.

NIH, like all HHS agencies, annually compiles a summary table that 

estimates funding for women’s health research. Per HHS’s guidance, NIH 

classifies its funding into 16 categories and about 120 sub-categories 

representing specific diseases and conditions; and three categories by 

sex—funding for women’s health research, funding for men’s health 

research, and funding for research related to both women’s and men’s 

health. Since the sex categorization of funding is based on enrollment, a 

portion of funding for research studies for certain diseases that primarily 

affect women—such as cervical cancer—but are reported as including 

male participants may be categorized as related to men’s health or both 

sexes. NIH reports these funding estimates in HHS’s annual 

congressional budget justification and in the biennial Report of the 

Advisory Committee on Research on Women’s Health.

1

To more closely examine the amount NIH funded for research on 

selected diseases and conditions with a particular relevance to women, 

we developed a list of diseases and conditions using the top 10 diseases 

and conditions in each of the Centers for Disease Control and 

Prevention’s 2012 lists of leading causes of death for women and leading 

chronic diseases for women. We supplemented this information with the 

diseases and conditions with a particular relevance to women that were 

identified in a 2010 Institute of Medicine report, Women’s Health 

Research: Progress, Pitfalls, and Promise.

2

We worked with NIH and several of its institutes and centers (IC) to 

assign each of the diseases and conditions in our list to the NIH budget 

categories that were included in the women’s health budget. These 

1For a link to the report, visit http://orwh.od.nih.gov/about/acrwh/index.asp, which 

we accessed July 21, 2015. 

2

Institute of Medicine, Women’s Health Research: Progress, Pitfalls, and Promise

(Washington, DC: The National Academies Press, 2010).

Appendix II: National Institutes of Health 

(NIH) Funding for Women’s Health Research

Page 31 GAO-16-13 Women in NIH Clinical Trials
Appendix II: National Institutes of Health (NIH) 

Funding for Women’s Health Research

matches allowed us to provide an estimate of funding for these selected 

diseases and conditions, which are shown in Table 3.

NIH officials told us that determining the amount NIH funded for women’s 

health research overall—as well as for specific diseases and conditions—

is difficult, and the resulting information are estimates, rather than actual 

amounts, due to challenges compiling the funding data. These challenges 

include: (1) methodological issues in assigning research funding to a sex 

category, especially for basic research, which does not include human 

subjects, (2) difficulties assigning NIH research funding to broad HHSdetermined disease categories, (3) research projects that overlap disease 

categories, but must be assigned to a single disease category, and 

(4) variation in data collection processes at the IC level.

Page 32 GAO-16-13 Women in NIH Clinical Trials
Appendix II: National Institutes of Health (NIH) 

Funding for Women’s Health Research

Table 3: National Institutes of Health (NIH) Estimated Funding for Selected Diseases and Conditions with a Particular 

Relevance to Women, Fiscal Year 2014

NIH women’s health research category(s) 

funding in thousands

Disease or condition

NIH women’s health 

research category(s)

Whether 

category(s) is a 

direct matcha Women Men Both Total

Accidents 

(unintentional injuries)

Unintentional injury Yes $230 636 24,257 25,123

Alcohol and drug addiction Alcohol — $20,096 22,693 113,472 156,261

Illegal drugs — $125,522 129,360 221,170 476,052

Subtotal Nob $145,618 152,053 334,642 632,313

Alzheimer’s disease Alzheimer’s disease Yes $106,729 88,825 304,141 499,695

Arthritis diagnosis No corresponding category N/A N/A N/A N/A N/A

Asthma Asthma Yes $49,078 48,546 128,323 225,947

Autoimmune diseases Lupus erythematosus — $44,836 4,669 27,151 76,656

Multiple sclerosis — $5,672 11,072 66,929 83,673

Scleroderma — $12,336 102 2,441 14,879

Sjogren’s syndrome — $17,458 0 375 17,833

Diabetes — $106,607 76,701 88,563 271,871

Immune disorders: other — $517 396 184,372 185,285

Subtotal Nob $187,426 92,940 369,831 650,197

Breast cancer Breast cancer Yes $705,090 68 1,430 706,588

Cervical cancer Reproductive cancers: 

cervical

Yes $84,966 1,108 6,700 92,774

Chronic fatigue syndrome Chronic fatigue syndrome Yes $1,668 790 632 3,090

Chronic joint symptoms Chronic pain conditions Noc $3,948 10,500 120,601 135,049

Chronic lower respiratory 

diseases

Asthma Nod $49,078 48,546 128,323 225,947

Pulmonary diseases $82,792 83,493 333,214 499,499

Chronic pain conditions Chronic pain conditions Yes $3,948 10,500 120,601 135,049

Colorectal cancer Colorectal cancer Yes $131,348 231 129,825 261,404

Depression Depression/mood 

disorders

Noc $20,219 2,708 118,513 141,440

Diabetes mellitus Diabetes Yes $106,607 76,701 88,563 271,871

Eating Disorders Eating disorders Yes $4,924 0 6,315 11,239

Fibromyalgia Fibromyalgia and 

eosinophilic myalgia

Noc $3,652 0 0 3,652

Page 33 GAO-16-13 Women in NIH Clinical Trials
Appendix II: National Institutes of Health (NIH) 

Funding for Women’s Health Research

NIH women’s health research category(s) 

funding in thousands

Disease or condition

NIH women’s health 

research category(s)

Whether 

category(s) is a 

direct matcha Women Men Both Total

Gynecologic cancers other 

than cervical cancer

Reproductive cancers: 

ovarian

— $111,573 0 0 111,573

Reproductive cancers: 

vaginal, uterine and other

— $25,489 0 0 25,489

Subtotal Nob $137,062 0 0 137,062

Hearing trouble Ear diseases and 

disorders

Noc $12,514 7 215,658 228,179

HIV/AIDS AIDS/HIV Yes $167,978 71,539 2,228,561 2,248,078

Hypertension Heart disease Nod $125,873 121,968 790,991 1,038,832

Other cardiovascular 

diseases/disorders

$132,560 102,727 791,654 1,026,941

Incontinence Urinary incontinence — $7,772 0 0 7,772

Fecal incontinence — $1,179 131 0 1,310

Subtotal Nob $8,951 131 0 9,082

Influenza and pneumonia Global health Noc $11,509 67,485 1,788,623 1,867,617

Irritable bowel syndrome Irritable bowel syndrome Yes $7,962 815 749 9,526

Lower back pain Chronic pain conditions Noc $3,948 10,500 120,601 135,049

Lung cancer Lung cancer Yes $150,678 230 119,998 270,906

Major cardiovascular 

diseases

Heart disease Noc $125,873 121,968 790,991 1,038,832

Malignant neoplasms Cancer (ALL) Yes $2,246,960 79,466 4,010,650 5,337,076

Maternal mortality and 

morbidity

Pregnancy/pregnancy 

prevention/maternal health

Noc $225,407 741 14,663 240,811

Melanoma Other neoplasms Noc $37,816 77,829 3,752,697 3,868,342

Memory and cognitive 

changes associated with 

perimenopause

Menopause Noc $24,945 0 200 25,145

Mental illness other than 

depression

Mental health (ALL minus 

depression/mood 

disorders)

Noe $55,169 43,437 1,108,536 1,207,142

Migraines Migraine Yes $50 50 856 956

Neck Pain Chronic pain conditions Noc $3,948 10,500 120,601 135,049

Nephritis, nephrotic 

syndrome and nephrosis 

Kidney and urologic: Other Noc $1,438 5,845 407,939 415,222

Page 34 GAO-16-13 Women in NIH Clinical Trials
Appendix II: National Institutes of Health (NIH) 

Funding for Women’s Health Research

NIH women’s health research category(s) 

funding in thousands

Disease or condition

NIH women’s health 

research category(s)

Whether 

category(s) is a 

direct matcha Women Men Both Total

Non-malignant 

gynecological disorders

Endometriosis/ 

leiomyomas

— $8,238 0 417 8,655

Pelvic floor disorders — $1,223 0 0 1,223

Female reproductive 

physiology

— $64,968 0 0 64,968

Subtotal Nob $74,429 0 417 74,846

Osteoporosis Osteoporosis (including 

fractures)

Yes $88,553 7,214 10,956 106,723

Other diseases of the 

respiratory system

Pulmonary diseases Noc $82,792 83,493 333,214 499,499

Pregnancy-related issues Pregnancy/pregnancy 

prevention/maternal health

Noc $225,407 741 14,663 240,811

Septicemia Blood diseases Noc $37,646 47,807 415,524 500,977

Sexual dysfunction No corresponding category N/A N/A N/A N/A N/A

Sinusitis Infectious diseases: other Noc $1,523 822 650,482 652,827

Stress-related disorders Psychosocial stress Noc $6,129 865 20,795 27,789

Thyroid disease Thyroid 

diseases/conditions

Noc $11,184 2,796 0 13,980

Type 2 diabetes Diabetes Noc $106,607 76,701 88,563 271,871

Unintended pregnancy Pregnancy/pregnancy 

prevention/maternal health

Noc $225,407 741 14,663 240,811

Sources: GAO analysis of NIH data. | GAO-16-13

Notes: According to NIH, funding figures are categorized as either inseparably combined—in the 

“Both” category—or as related to research on women’s health or men’s health. In cases in which NIH 

did not believe that a GAO-identified disease or condition could be linked to an NIH budget category, 

the NIH Research Category is labeled as “N/A” for not applicable.

a

A “yes” in this column indicates that the GAO-identified category and the HHS-defined category are 

identical or nearly identical.

b

NIH officials stated that this GAO-identified disease or condition represented multiple NIH budget 

categories, so funding figures for all categories are listed. The categories were summed and are also 

represented as a subtotal.

c

The funding figure listed for research on this GAO-identified disease or condition is an estimate 

based on the closest category match possible. Actual funding may be less or more than the dollar 

amount listed.

d

NIH officials stated that this GAO-identified disease or condition could be assigned to one of two 

categories, so funding figures for both categories are listed.

e

NIH officials stated that this GAO-identified disease or condition represented the larger “Mental 

Health” category, minus the subcategory of “Depression/Mood disorders,” so we calculated this figure 

accordingly to present a single amount.

Page 35 GAO-16-13 Women in NIH Clinical Trials
Appendix III: National Institutes of Health (NIH) 

and Institute and Center (IC) Research Study 

Enrollment

Tables 4 and 5 below present NIH and IC-level enrollment data, by sex, 

for fiscal years 2011 through 2014—for all clinical research studies and 

for phase III clinical trials, respectively.

Table 4: Percentage Female, Male, and Other Enrollment in All Clinical Research 

Studies, for NIH Overall and Institutes and Centers (IC), Fiscal Years 2011-2014

All NIH clinical research

NIH or IC 2011 2012 2013 2014

NIH, overall

Female 59.4 57.0 56.7 57.2

Male 39.3 41.8 42.1 38.6

Other 1.3 1.1 1.3 4.1

Fogarty International Center

Female 49.9 47.1 60.3 60.7

Male 48.5 51.8 38.3 38.5

Other 1.7 1.1 1.5 0.8

National Cancer Institute

Female 61.7 55.7 57.1 60.9

Male 37.8 44.0 42.5 35.1

Other 0.5 0.3 0.4 4.1

National Center for Advancing Translational Sciencesa

Female 0.0 59.0 36.7 0.0

Male 0.0 41.0 63.3 0.0

Other 0.0 0.0 0.0 0.0

National Center for Complementary and Integrative Health

Female 47.2 63 62.5 64

Male 51.8 34.1 28.4 27.7

Other 0.9 2.9 9.1 8.2

National Eye Institute

Female 51.6 52.3 55.9 56.2

Male 47.8 46.7 43.7 43.7

Other 0.6 1.0 0.4 0.1

National Heart, Lung, and Blood Institute

Female 47.9 57.7 57.5 49.9

Male 50.2 41.1 41.5 48.6

Other 1.9 1.3 1.0 1.5

Appendix III: National Institutes of Health 

(NIH) and Institute and Center (IC) Research 

Study Enrollment 

Page 36 GAO-16-13 Women in NIH Clinical Trials
Appendix III: National Institutes of Health (NIH) 

and Institute and Center (IC) Research Study 

Enrollment

All NIH clinical research

NIH or IC 2011 2012 2013 2014

National Human Genome Research Institute

Female 54.4 45.4 46.3 48.5

Male 36.6 43.9 50.5 51

Other 9.0 10.7 3.2 0.5

National Institute on Aging

Female 54.1 54.4 51.7 53.2

Male 45.4 44.7 44.4 43.5

Other 0.5 0.9 3.9 3.3

National Institute on Alcohol Abuse and Alcoholism

Female 46.8 46.6 45.3 44.3

Male 52.9 52.9 53.0 55.2

Other 0.3 0.5 1.7 0.6

National Institute of Allergy and Infectious Diseases

Female 48.0 50.6 49.8 50.9

Male 50.6 47.4 48.5 48.8

Other 1.3 2.0 1.7 0.3

National Institute of Arthritis and Musculoskeletal and Skin Diseases

Female 58.9 57.0 57.8 56.6

Male 38.8 41.8 41.3 43.0

Other 2.3 1.2 0.8 0.4

National Institute of Biomedical Imaging and Bioengineering

Female 63.6 52.9 64 64.1

Male 35.1 44.8 34.8 35.6

Other 1.3 2.3 1.1 0.3

Eunice Kennedy Shriver National Institute of Child Health and Human Development

Female 66.7 74.1 71.6 58.3

Male 31.8 24.5 27.1 26.9

Other 1.5 1.4 1.3 14.8

National Institute on Deafness and Other Communication Disorders

Female 50.0 52.1 48.4 47.4

Male 49.9 47.0 50.7 51.9

Other 0.2 0.9 1.0 0.6

National Institute of Dental and Craniofacial Research

Female 56.6 57.3 58.9 52.3

Male 40.4 39.0 37.0 38.9

Other 3.0 3.7 4.1 8.9

Page 37 GAO-16-13 Women in NIH Clinical Trials
Appendix III: National Institutes of Health (NIH) 

and Institute and Center (IC) Research Study 

Enrollment

All NIH clinical research

NIH or IC 2011 2012 2013 2014

National Institute of Diabetes and Digestive and Kidney Diseases

Female 57.1 54.6 53.5 54.8

Male 41.9 44.3 45.7 43.9

Other 1.1 1.1 0.9 1.3

National Institute on Drug Abuse

Female 45.8 46.3 46.8 41.0

Male 49.5 48.8 50.3 57.0

Other 4.7 4.9 2.9 2.0

National Institute of Environmental Health Sciences

Female 73.5 71.8 69.9 73.0

Male 26.2 28.1 29.9 26.8

Other 0.3 0.2 0.2 0.2

National Institute of General Medical Sciences

Female 54.8 62.8 54.0 80.2

Male 44.8 35.6 43.8 18.6

Other 0.3 1.6 2.2 1.2

National Institute of Mental Health

Female 52.7 47.0 42.6 49.6

Male 45.8 51.4 56.0 49.5

Other 1.5 1.7 1.4 0.9

National Institute of Minority Health and Health Disparities

Female 60.2 63.4 70.4 59.1

Male 38.8 35.1 29.0 38.9

Other 1.0 1.5 0.6 2.1

National Institute of Neurological Disorders and Stroke

Female 51.3 50.3 53.6 53.8

Male 45.2 44.4 43.7 44.3

Other 3.5 5.3 2.6 1.9

National Institute of Nursing Research

Female 60.4 59.9 59.2 65.8

Male 38.3 38.2 40.5 33.5

Other 1.4 2.0 0.4 0.7

National Library of Medicine

Female 59.6 48.2 54.7 44.9

Male 40.4 51.8 45.2 52.2

Other 0.0 0.0 0.1 3.0

Page 38 GAO-16-13 Women in NIH Clinical Trials
Appendix III: National Institutes of Health (NIH) 

and Institute and Center (IC) Research Study 

Enrollment

All NIH clinical research

NIH or IC 2011 2012 2013 2014

NIH Clinical Center

Female 38.5 38.1 38.0 36.6

Male 42.5 42.1 44.0 44.4

Other 19.1 19.8 18.0 19.0

Source: NIH. | GAO-16-13

Notes: Two ICs, the Center for Information Technology and the Center for Scientific Review, do not 

conduct or fund any clinical research studies, and thus, are not included in this table. Additionally, we 

do not include the National Center for Research Resources in this table, as it ceased operations in 

fiscal year 2012, or the Office of Director and Office of Research on Women’s Health, as they are not 

ICs. However, each of these three entities funded clinical research in one or more years during the 

fiscal year 2011-2014 time period, and as such, their enrollment data are included in the totals for 

overall NIH clinical research studies. “Other” enrollment includes participants whose sex was not 

reported or is otherwise unknown.

a

IC did not begin operations until fiscal year 2012 and maintains a very small portfolio of clinical 

research.

Page 39 GAO-16-13 Women in NIH Clinical Trials
Appendix III: National Institutes of Health (NIH) 

and Institute and Center (IC) Research Study 

Enrollment

Table 5: Percentage Female, Male, and Other Enrollment in Phase III Clinical Trials, 

for NIH Overall and Institutes and Centers (IC), Fiscal Years 2011-2014

NIH or IC 2011 2012 2013 2014

NIH, overall

Female 57.0 62.1 73.3 60.0

Male 38.0 32.7 25.9 39.4

Other 5.0 5.2 0.7 0.6

Fogarty International Centera

Female 0.0 0.0 0.0 0.0

Male 0.0 0.0 0.0 0.0

Other 0.0 0.0 0.0 0.0

National Cancer Institute

Female 61.4 62.1 81.1 61.7

Male 38.5 37.8 18.8 38.2

Other 0.0 0.1 0.0 0.1

National Center for Advancing Translational Sciencesa,b

Female 0.0 0.0 0.0 0.0

Male 0.0 0.0 0.0 0.0

Other 0.0 0.0 0.0 0.0

National Center for Complementary and Integrative Healthc

Female 0.0 0.0 0.0 54.1

Male 0.0 0.0 0.0 45.9

Other 0.0 0.0 0.0 0.0

National Eye Institute

Female 47.8 48.6 62.3 56.8

Male 52.2 51.4 37.7 43.2

Other 0.0 0.0 0.0 0.0

National Heart, Lung, and Blood Institute

Female 49.8 41 42.7 44.9

Male 50.2 58.9 57.3 55.1

Other 0.0 0.1 0.0 0.0

National Human Genome Research Institutea,d

Female 0.0 0.0 0.0 0.0

Male 0.0 0.0 100.0 100.0

Other 0.0 0.0 0.0 0.0

Page 40 GAO-16-13 Women in NIH Clinical Trials
Appendix III: National Institutes of Health (NIH) 

and Institute and Center (IC) Research Study 

Enrollment

NIH or IC 2011 2012 2013 2014

National Institute on Agingc

Female 54.7 0.0 50.5 75.1

Male 45.3 0.0 49.5 24.9

Other 0.0 0.0 0.0 0.0

National Institute on Alcohol Abuse and Alcoholismc

Female 0.0 0.0 81.1 79.3

Male 0.0 0.0 18.9 19.2

Other 0.0 0.0 0.0 1.5

National Institute of Allergy and Infectious Diseases

Female 37.8 42.6 51.4 45.8

Male 62.2 57.4 48.6 54.2

Other 0.0 0.0 0.0 0.0

National Institute of Arthritis and Musculoskeletal and Skin Diseasesc

Female 50.0 50.0 50.0 50.0

Male 50.0 50.0 50.0 50.0

Other 0.0 0.0 0.0 0.0

National Institute of Biomedical Imaging and Bioengineeringa

Female 0.0 0.0 0.0 0.0

Male 0.0 0.0 0.0 0.0

Other 0.0 0.0 0.0 0.0

Eunice Kennedy Shriver National Institute of Child Health and Human Development

Female 64.3 75.0 75.2 73.1

Male 34.9 23.6 23.3 25.4

Other 0.8 1.4 1.4 1.5

National Institute on Deafness and Other Communication Disordersc

Female 0.0 0.0 26.5 0.0

Male 0.0 0.0 73.5 0.0

Other 0.0 0.0 0.0 0.0

National Institute of Dental and Craniofacial Research

Female 49.2 62.6 51.2 60.7

Male 50.8 35.0 48.3 39.1

Other 0.0 2.4 0.5 0.2

National Institute of Diabetes and Digestive and Kidney Diseases

Female 75.2 77.0 65.8 67.5

Male 24.8 23.0 32.8 32.1

Other 0.0 0.0 1.4 0.4

Page 41 GAO-16-13 Women in NIH Clinical Trials
Appendix III: National Institutes of Health (NIH) 

and Institute and Center (IC) Research Study 

Enrollment

NIH or IC 2011 2012 2013 2014

National Institute on Drug Abuse

Female 37.4 38.3 41.1 25.5

Male 39.8 40.1 56.5 73.8

Other 22.8 21.6 2.5 0.8

National Institute of Environmental Health Sciencese

Female 67.6 50.9 0.0 0.0

Male 32.4 49.1 0.0 0.0

Other 0.0 0.0 0.0 0.0

National Institute of General Medical Sciencesa

Female 0.0 0.0 0.0 0.0

Male 0.0 0.0 0.0 0.0

Other 0.0 0.0 0.0 0.0

National Institute of Mental Health

Female 64.5 49.4 51.1 52.5

Male 35.5 50.6 48.9 47.0

Other 0.0 0.0 0.0 0.5

National Institute of Minority Health and Health Disparitiesa

Female 0.0 0.0 0.0 0.0

Male 0.0 0.0 0.0 0.0

Other 0.0 0.0 0.0 0.0

National Institute of Neurological Disorders and Stroke

Female 38.6 40.0 39.4 43.5

Male 61.4 60.0 60.6 56.4

Other 0.0 0.0 0.1 0.0

National Institute of Nursing Researchc

Female 0.0 47.7 62.8 83.7

Male 0.0 52.3 37.2 16.3

Other 0.0 0.0 0.0 0.0

National Library of Medicinea

Female 0.0 0.0 0.0 0.0

Male 0.0 0.0 0.0 0.0

Other 0.0 0.0 0.0 0.0

NIH Clinical Centerc

Female 46.1 46.3 44.9 45.5

Male 53.9 53.7 55.1 54.5

Other 0.0 0.0 0.0 0.0

Source: NIH. | GAO-16-13

Page 42 GAO-16-13 Women in NIH Clinical Trials
Appendix III: National Institutes of Health (NIH) 

and Institute and Center (IC) Research Study 

Enrollment

Notes: Two ICs, the Center for Information Technology and the Center for Scientific Review, do not 

conduct or fund any clinical research studies or phase III trials, and thus, are not included in this 

table. Additionally, we do not include the National Center for Research Resources in this table, as it 

ceased operations in fiscal year 2012, or the Office of Director and Office of Research on Women’s 

Health, as they are not ICs and did not fund phase III clinical trials during the fiscal year 2011-2014 

time period. “Other” enrollment includes participants whose sex was not reported or is otherwise 

unknown.

a

This IC did not report any enrollees in IC-funded phase III clinical trials in fiscal years 2011-2014.

b

This IC did not begin operations until fiscal year 2012.

c

These ICs enrolled fewer than 1,200 participants in each year for fiscal years 2011-2014. According 

to NIH, these ICs typically support very few phase III clinical trials.

d

There was only one individual participant enrolled in the years for which this IC reported enrollment.

e

According to NIH, the National Institute of Environmental Health Sciences portfolio supports very few 

NIH-defined phase III clinical trials. The enrollment data reported in fiscal years 2011and 2012 

primarily reflects the initial recruitment of mothers and young children into a study in fiscal year 2011 

and fathers and older siblings in fiscal year 2012.

Page 43 GAO-16-13 Women in NIH Clinical Trials
Appendix IV: Comments from the Department Appendix IV: Comments from the of Health and Human Services

Department of Health and Human Services

Page 44 GAO-16-13 Women in NIH Clinical Trials
Appendix IV: Comments from the Department 

of Health and Human Services

Page 45 GAO-16-13 Women in NIH Clinical Trials
Appendix IV: Comments from the Department 

of Health and Human Services

Page 46 GAO-16-13 Women in NIH Clinical Trials
Appendix IV: Comments from the Department 

of Health and Human Services

Page 47 GAO-16-13 Women in NIH Clinical Trials
Appendix IV: Comments from the Department 

of Health and Human Services

Page 48 GAO-16-13 Women in NIH Clinical Trials
Appendix IV: Comments from the Department 

of Health and Human Services

Page 49 GAO-16-13 Women in NIH Clinical Trials
Appendix IV: Comments from the Department 

of Health and Human Services

Page 50 GAO-16-13 Women in NIH Clinical Trials
Appendix IV: Comments from the Department 

of Health and Human Services

Page 51 GAO-16-13 Women in NIH Clinical Trials
Appendix V: GAO Contact and Staff 

Acknowledgments

Linda T. Kohn, (202) 512-7114 or kohnl@gao.gov

In addition to the contact named above, Karen Doran, Assistant 

Director; Amanda Cherrin; Emily Loriso; and Julie T. Stewart made 

key contributions to this report. Jennie F. Apter, Leia Dickerson, 

Krister Friday, and Jacquelyn Hamilton also contributed to the 

development of this report.

Appendix V: GAO Contact and Staff 

Acknowledgments

GAO Contact

Acknowledgments

(291240) Page 52 GAO-16-13 Women in NIH Clinical Trials
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