COMPARATIVE 

EFFECTIVENESS 

RESEARCH

Activities Funded by 

the Patient-Centered 

Outcomes Research

Trust Fund

Report to Congressional Committees

March 2018

GAO-18-311

United States Government Accountability Office
United States Government Accountability Office

Highlights of GAO-18-311, a report to 

congressional committees

March 2018

COMPARATIVE EFFECTIVENESS RESEARCH

Activities Funded by the Patient-Centered Outcomes 

Research Trust Fund

What GAO Found

The Patient-Centered Outcomes Research Institute (PCORI) made about $2 

billion in commitments for awards in fiscal years 2010 through 2017. PCORI is a 

federally funded, nonprofit corporation established to carry out and improve 

comparative clinical effectiveness research (CER), which evaluates and 

compares the health outcomes and the clinical effectiveness, risks, and benefits 

of two or more medical treatments, services, or items. PCORI provides funding 

through award commitments from the Patient Centered Outcomes Research 

Trust Fund (Trust Fund) and may pay these awards over multiple years. Of the 

$2 billion PCORI committed as of the end of fiscal year 2017, about $1.6 billion

(or 79 percent of its commitments) is for research awards, and $325 million (or 

16 percent) is for building the capacity to use existing health data for research. 

Through fiscal year 2017, commitments for dissemination and implementation

awards—intended to share CER findings with potential users of this research—

were limited because most PCORI-funded research was still underway. PCORI 

projects to commit an additional $721 million for awards in fiscal years 2018 

through 2021. In addition to awards, PCORI spent $310 million on program and 

administrative support services in fiscal years 2010 through 2017 and projects to 

spend an additional $206 million for these services through fiscal year 2024.

PCORI’s Actual and Projected Commitments and Expenditures, Fiscal Years 2010 through 

2024 (Dollars in Millions)

Note: Amounts listed are current as of Jan. 5, 2018.

From fiscal years 2011 through 2017, the Department of Health and Human 

Services (HHS) obligated about $448 million from the Trust Fund. Of this 

amount, HHS obligated about $260 million (or 58 percent of all obligations) to the 

dissemination and implementation of CER findings. As most PCORI-funded CER 

had not yet been completed due to the time needed to conduct this research, 

HHS efforts focused instead on the dissemination and implementation of CER 

funded by other federal entities. Additionally, HHS obligated funds for efforts to 

train researchers on conducting CER, build data capacity, and on administrative 

activities. HHS projects to obligate an additional $120 million for these activities 

in fiscal years 2018 through 2020. View GAO-18-311. For more information, 

contact John E. Dicken at (202) 512-7114 or 

dickenj@gao.gov.

Why GAO Did This Study

In 2010, the Patient Protection and 

Affordable Care Act (PPACA)

authorized the establishment of PCORI 

to carry out CER and improve its

quality and relevance. PPACA also 

established new requirements for HHS 

to, among other things, disseminate 

findings from federally funded CER, 

including findings published by PCORI;

and coordinate with relevant federal 

health programs to build data capacity 

for this research. To fund CER 

activities, PPACA established the Trust 

Fund from which PCORI and HHS are 

expected to receive an estimated $4.0 

billion from fiscal years 2010 through 

2019. 

PPACA included a provision for GAO 

to review PCORI’s and HHS’s use of 

the Trust Fund. This report examines 

(1) PCORI’s use of the Trust Fund for 

CER activities, including the 

dissemination and use of research 

findings; and (2) HHS’s use of the 

Trust Fund for these activities.

GAO examined PCORI and HHS 

documents and data related to use of 

the Trust Fund, such as commitment, 

obligation, and expenditure data;

PCORI’s audited financial statements;

and descriptions of CER activities. 

GAO also interviewed PCORI and 

HHS officials responsible for planning 

and carrying out CER activities and

interviewed officials from stakeholder 

organizations representing potential 

users of CER, including public and 

private payer organizations, provider 

organizations, and patient

organizations. PCORI and HHS 

provided technical comments, which 

GAO incorporated as appropriate.
Page i GAO-18-311 Comparative Clinical Effectiveness Research Funding

Letter 1

Background 4

PCORI Committed Funds Primarily to Research and Data 

Capacity Efforts; Awards for Dissemination and Implementation 

of Findings Were Limited as Most Research Was Still 

Underway 8

HHS Obligated Funds Primarily for the Dissemination and 

Implementation of Research 19

Agency Comments 24

Appendix I Patient-Centered Outcomes Research Institute (PCORI) Award 

Commitments Made During Fiscal Years 2010 through 2017 27

Appendix II Agency for Healthcare Research and Quality’s (AHRQ) 

Dissemination and Implementation Initiatives, as of 

September 30, 2017 28

Appendix III Agency for Healthcare Research and Quality’s (AHRQ) Training 

Awards, as of September 30, 2017 31

Appendix IV Office of the Assistant Secretary for Planning and Evaluation’s 

(ASPE) Projects to Build Data Capacity, as of September 30, 2017 32

Appendix V GAO Contact and Staff Acknowledgments 36

Tables

Table 1: PCORI Award Commitments for Comparative Clinical 

Effectiveness Research Activities, Fiscal Years 2010 

through 2017 (Dollars in Millions) 8

Table 2: Heath Conditions That Received the Highest PCORI 

Research Award Commitments, Fiscal Years 2010 

through 2017 10

Contents
Page ii GAO-18-311 Comparative Clinical Effectiveness Research Funding

Table 3: HHS Obligations for Comparative Clinical Effectiveness 

Research Activities, Fiscal Years 2011 through 2017 

(Dollars in Millions) 19

Figures

Figure 1: Actual and Projected Funds Provided to the PatientCentered Outcomes Research Institute (PCORI) and the 

Department of Health and Human Services (HHS), Fiscal 

Years 2010 through 2019 7

Figure 2: PCORI Actual and Projected Commitments and 

Expenditures, Fiscal Years 2010 through 2024 (Dollars in 

Millions) 9

Figure 3: PCORI Timeline Estimates for Its Research Award 

Process 12

Figure 4: Number of PCORI-Funded Research Studies Underway 

and Completed, Fiscal Years 2017 through 2024 13

Figure 5: Process for Researcher to Receive Data Query Results 

from PCORnet Partners 15

Figure 6: HHS Actual and Projected Obligations, Fiscal Years 

2011 through 2020 (Dollars in Millions) 20

Figure 7: Overview of the AHRQ Dissemination and 

Implementation Initiative Process 22
Page iii GAO-18-311 Comparative Clinical Effectiveness Research Funding

Abbreviations

AHRQ Agency for Healthcare Research and Quality

ASPE Office of the Assistant Secretary for Planning and 

 Evaluation

CDC Centers for Disease Control and Prevention

CDS clinical decision support

CER comparative clinical effectiveness research

HHS Department of Health and Human Services

NIH National Institutes of Health

PCORI Patient-Centered Outcomes Research Institute

PCRF People-Centered Research Foundation

PPACA Patient Protection and Affordable Care Act

Treasury Department of the Treasury

Trust Fund Patient-Centered Outcomes Research Trust Fund

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

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

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necessary if you wish to reproduce this material separately.
Page 1 GAO-18-311 Comparative Clinical Effectiveness Research Funding

441 G St. N.W.

Washington, DC 20548

March 23, 2018

Congressional Committees

Clinical research is often conducted to evaluate the effectiveness of a 

specific treatment for a disease or condition; however, research is less 

often conducted to compare the relative effectiveness of two or more 

available treatment options. Comparative clinical effectiveness research 

(CER) evaluates and compares the health outcomes and the clinical 

effectiveness, risks, and benefits of two or more medical treatments, 

services, or items. For example, a recent CER study found that the life 

expectancy of women over age 50 with breast cancer in one breast, but 

who do not carry the breast cancer gene mutation, is similar whether they 

have one or both breasts removed.1 Another recent study compared the 

effectiveness and safety of treatments for childhood anxiety disorders and 

found that a combination of medications and therapy is likely more 

effective than either treatment alone.2 According to the National 

Academies of Medicine, CER can help clinicians, patients, payers, and 

others make informed decisions to improve health care.3

In 2010, the Patient Protection and Affordable Care Act (PPACA) 

authorized the establishment of the Patient-Centered Outcomes 

Research Institute (PCORI) as a federally funded, nonprofit corporation to 

improve the quality and relevance of CER.4 In addition, PPACA 

 1

Kalatu R. Davies, Abenaa M. Brewster, Isabelle Bedrosian, Patricia A. Parker, Melissa A. 

Crosby, Susan K. Peterson, Yu Shen, Robert J. Volk, and Scott B. Cantor, “Outcomes of 

Contralateral Prophylactic Mastectomy in Relation to Familial History: A Decision 

Analysis,” Breast Cancer Research, vol. 93, no. 18 (2016).

2

Z. Wang, S. Whiteside, L. Sim, W. Farah, A. Morrow, M. Alsawas, P. Barrionuevo 

Moreno, M. Tello, N. Asi, B. Beuschel, L. Daraz, J. Almasri, F. Zaiem, S. Gunjal, L. Larrea 

Mantilla, O. Ponce Ponte,A. LeBlanc, L.J. Prokop, M.H. Murad. Anxiety in Children. 

Comparative Effectiveness Review No. 192 (Rockville, Md.: Agency for Healthcare 

Research and Quality, August 2017).

3

National Academy of Medicine (formally the Institute of Medicine), Initial National 

Priorities for Comparative Effectiveness Research (Washington, D.C.: June 2009). 

4

Pub. L. No. 111-148, §§ 6301(a), 10602, 124 Stat. 119, 727, 1005 (codified at 42 U.S.C. 

§ 1320e). PCORI’s patient-centered outcomes research builds upon the definition for CER 

previously established by the Federal Coordination Council for Comparative Effectiveness 

Research. See Department of Health and Human Services, Federal Coordinating Council 

for Comparative Effectiveness Research: Report to the President and the Congress

(Washington, D.C.: June 30, 2009). For the purposes of this report, we will refer to the 

work PCORI and HHS conduct as CER.

Letter
Page 2 GAO-18-311 Comparative Clinical Effectiveness Research Funding

established new requirements for the Department of Health and Human 

Services (HHS) to, among other things, broadly disseminate findings from 

federally funded CER, including findings published by PCORI; train 

researchers on CER methodological approaches; and coordinate relevant 

federal health programs to build data capacity for this research. To fund 

PCORI’s and HHS’s CER activities, PPACA established the PatientCentered Outcomes Research Trust Fund (Trust Fund) through which 

PCORI and HHS expect to receive an estimated total of about $4 billion 

from fiscal years 2010 through 2019.

PPACA included a provision for us to report on the use of the Trust Fund 

for CER activities.5 In this report, we examine:

1. PCORI’s use of the Trust Fund for CER activities, including the 

dissemination and use of research findings, and

2. HHS’s use of the Trust Fund for these activities.

To examine PCORI’s and HHS’s use of the Trust Fund for CER activities, 

including the dissemination and use of research findings, we reviewed 

PCORI award commitment and expenditure data and HHS data on 

obligated funds.6 We also reviewed descriptions of PCORI’s and HHS’s 

CER activities, relevant legislation, and previous GAO reports.7

Furthermore, we conducted interviews with PCORI and HHS officials 

responsible for planning and carrying out CER activities. Within HHS, we 

interviewed officials from the Agency for Healthcare Research and Quality 

(AHRQ), the Office of the Assistant Secretary for Planning and Evaluation 

 5

PPACA included provisions that we review PCORI’s research activities by 2015 and its 

use of federal funding by 2018. We completed the first of these reviews in 2015. See 

GAO, Comparative Effectiveness: Initial Assessment of the Patient-Centered Outcomes 

Research Institute, GAO-15-301 (Washington, D.C.: March 9, 2015). In addition, PPACA 

requires the Comptroller General to appoint members to serve on PCORI’s Board of 

Governors and Methodology Committee.

6

According to PCORI, “commitments” represent the amount of funding PCORI intends to 

award or has awarded to contractors. HHS obligations in a given year represent a definite 

commitment of funds that create a legal liability for the payment of goods and services 

ordered or received, although funds may be expended in subsequent years. 

7

Previous reports we reviewed included GAO-15-301 and GAO, Comparative 

Effectiveness Research: HHS Needs to Strengthen Dissemination and Dara-CapacityBuilding Efforts, GAO-15-280 (Washington, D.C.: March 3, 2015).
Page 3 GAO-18-311 Comparative Clinical Effectiveness Research Funding

(ASPE), and the National Institutes of Health (NIH).8 We also interviewed 

officials coordinating PCORI’s PCORnet initiative, the National PatientCentered Clinical Research Network, to build data capacity for research.9

We reviewed funding from fiscal year 2010, the first year funding was 

made available, through 2017, the most recent data available at the time 

of our analysis. To the extent available, we also reviewed the amount of 

funding projected beyond fiscal year 2017. Finally, to gather the views of 

potential users of CER findings, including public and private payers, 

providers, and patients, we interviewed officials from several large 

organizations that broadly represent each group of potential users.10

To assess the reliability of PCORI commitment and expenditure data and 

HHS obligation data, we collected information from PCORI and HHS 

officials regarding the reliability of the data, including the accuracy of data 

entry and the systems that contain the data. We also reviewed PCORI’s 

annual audited financial statements. On the basis of these steps, we 

determined the data were sufficiently reliable for the purposes of our 

reporting objectives.

 8

HHS conducts CER activities through AHRQ and ASPE. AHRQ’s mission is to produce 

evidence to make health care safer, higher quality, more accessible, equitable, and 

affordable, and to work within HHS and with other partners to make sure that the evidence 

is understood and used. AHRQ also coordinates with NIH on certain aspects of its work, 

such as disseminating research findings. ASPE advises the Secretary of HHS on policy 

development in health, disability, human services, data, and science, and provides advice 

and analysis on economic policy. ASPE leads special initiatives, coordinates the 

department’s evaluation, research and demonstration activities, and manages crossdepartment planning activities such as strategic planning, legislative planning, and review 

of regulations. 

9

PCORnet is a PCORI-funded initiative that supports the translation of existing clinical, 

patient, and health plan networks’ data into a common data model resulting in the 

availability of a nationally representative sample of over 128 million individuals that can be 

used in randomized clinical trials, large observational studies, and other research to make 

it faster, easier, and less costly to conduct clinical research. Its Coordinating Center leads 

data and engagement activities, connects with research partners, and supports the 

PCORnet infrastructure.

10Specifically, to gather the views of public and private payers, we interviewed officials 

from the Medicaid Medical Directors Network and America’s Health Insurance Plans (a 

trade group representing health insurers), as well as officials from two health plans. 

Likewise, to gather the views of providers, we interviewed officials from the American 

Medical Association and the American College of Physicians. Finally, to gather the views 

of patients, we interviewed officials from the National Health Council and the Partnership 

to Improve Patient Care. 
Page 4 GAO-18-311 Comparative Clinical Effectiveness Research Funding

We conducted this performance audit from May 2017 to March 2018 in 

accordance with generally accepted government auditing standards. 

Those standards require that we plan and perform our work 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.

In 2010 PPACA authorized the establishment of PCORI to improve CER 

quality and relevance. PPACA also established requirements for HHS to, 

among other things, disseminate findings from federally funded CER, 

including findings published by PCORI, and coordinate with relevant 

federal health programs to build data capacity for research. PPACA 

established a Trust Fund to fund these CER activities by PCORI and HHS 

through fiscal year 2019.

PPACA authorized the establishment of PCORI as a federally funded, 

nonprofit corporation aimed at advancing the quality and relevance of 

evidence through research to help patients, clinicians, purchasers, and 

policy-makers to make informed health care decisions.11 PCORI is 

required to identify research priorities, establish a research project 

agenda, fund research consistent with its research agenda, and 

disseminate research findings, among other responsibilities. In 2015 we 

reported that PCORI had conducted activities consistent with its 

legislative requirements.12 For example, we reported that since its 

inception in 2010, PCORI established and implemented priorities for 

funding CER and related activities, developed plans to disseminate 

funded research and track its utilization, and took steps to make its 

research more centered on outcomes prioritized by patients. Further, 

PCORI developed PCORnet as a distributed research network initiative 

 11While clinical research is often conducted to evaluate the effectiveness of a specific 

treatment for a disease or condition, research is less often conducted to compare among 

available treatment options. For example, pharmaceutical companies conduct clinical trials 

to evaluate the safety and effectiveness of a new drug to treat a specific disease or 

condition. However, prior to federal funding for CER, there was little funding available for 

research to determine whether one drug is safer or more effective than another. 

12See GAO-15-301.

Background

PCORI Activities Required by 

PPACA
Page 5 GAO-18-311 Comparative Clinical Effectiveness Research Funding

that enables electronic health-related data from multiple sources to be 

available for research.13

PPACA requires HHS to perform several requirements related to CER, 

which it has implemented through AHRQ and ASPE.14 Specifically, AHRQ 

is required to disseminate and support the incorporation of CER funded 

by PCORI and other federal entities, as well as to foster capacity for 

conducting CER by supporting training in the methods used to conduct 

such research. ASPE, in turn, is required to build data capacity for 

conducting CER. In 2015, we reported that AHRQ had taken some steps 

to disseminate research findings, but had not taken other actions to help it 

fully address its dissemination requirements. Furthermore, we reported 

that ASPE coordinated among various agencies to fund projects intended 

to build data capacity for research, but that its approach lacked key 

elements—such as defined objectives, milestones, and time frames—that 

are necessary to ensure effectiveness. In our 2015 report, we made five 

recommendations to HHS to direct AHRQ and ASPE to address these 

issues, as appropriate. HHS concurred with these recommendations and 

 13According to PCORI officials, PCORnet was developed to take existing electronic 

health-related data from multiple sources and translate certain fields of that disparate data 

into a common data model, so that the translated data can be used to conduct research. 

14PPACA enacted the new CER requirements by adding section 937 to Title IX of the 

Public Health Service Act. Pub. L. No. 111-148, § 6301(b), 124 Stat. 119, 738-740 

(codified at 42 U.S.C. § 299b-37). Section 937 requires HHS’s AHRQ to carry out 

specified functions with respect to the dissemination of CER, incorporation of research 

findings, collection of feedback, and the establishment of a grant program to train 

researchers, which AHRQ has carried out in consultation with NIH. Section 937(a)-(c), (e). 

In contrast, section 937 does not specify a particular HHS entity to carry out the functions 

specified under subsection (f) related to the coordination of federal programs to build data 

capacity for CER. The Secretary of HHS has delegated responsibility for carrying out 

section 937(f) to HHS’s ASPE.

HHS CER-related Activities 

Required by PPACA
Page 6 GAO-18-311 Comparative Clinical Effectiveness Research Funding

specified actions it would take to address them. Four of the 

recommendations have since been implemented.15

PPACA established the Trust Fund through which PCORI and HHS 

receive funds for CER activities. The law provides that for fiscal years 

2010 through 2019, the Trust Fund will receive appropriations from the 

general fund of the Treasury, transfers from the Medicare trust funds, and 

fees collected by the Department of the Treasury (Treasury) from private 

insurance and self-insured health plans. Eighty percent of the amounts in 

the Trust Fund must be made available to PCORI in fiscal years 2011 

through 2019, and Treasury must transfer the remaining 20 percent to the 

Secretary of HHS in each of those years.16 Under current law,

appropriations and transfers to the Trust Fund will end in fiscal year 2019. 

The law also provides that no amounts shall be available for expenditure 

from the Trust Fund after September 30, 2019, and specifies that any 

amounts remaining in the Trust Fund after that time will be transferred to 

the general fund of the Treasury.17 (See fig. 1 for an overview of transfers 

to the Trust Fund and distribution of funds to PCORI and HHS).

 15See GAO-15-280. To address our recommendations, AHRQ developed time frames for 

the implementation and distribution of marketing plans and informational tools, increased 

dissemination efforts to health plans and vendors of health information technology, and 

developed plans for collaborating with NIH on dissemination efforts. ASPE implemented a 

monitoring system to track progress toward milestones and deliverables on its projects to 

build data capacity. As of January 2018, AHRQ was in the process of implementing the 

remaining recommendation, which directs AHRQ to document and complete plans to 

develop a publicly available CER database, including plans to meet the needs of various 

potential users in the general public. While AHRQ has developed a website that includes 

instructions and links for searching multiple different publicly available databases that 

have information on CER, AHRQ has not yet provided information on how it plans to meet 

the needs of various potential users in the general public, as specified in our 

recommendation.

16The law provides that amounts transferred to HHS will remain available until expended. 

For fiscal year 2010, all amounts appropriated to the Trust Fund were made available to 

PCORI.

17PCORI plans to draw down all remaining funds in the Trust Fund prior to this date to 

have funds available for anticipated expenditures after fiscal year 2019.

PPACA Funding for CER
Page 7 GAO-18-311 Comparative Clinical Effectiveness Research Funding

Figure 1: Actual and Projected Funds Provided to the Patient-Centered Outcomes Research Institute (PCORI) and the 

Department of Health and Human Services (HHS), Fiscal Years 2010 through 2019

PPACA limits the use of CER in certain ways; for example, the law 

prohibits PCORI from developing or using a dollars-per-quality adjusted 

life-year to establish what type of health care is cost effective or 

recommended, and prohibits the Secretary of HHS from using such 

measures as a threshold to determine coverage, reimbursement, or 

incentive programs under Medicare.18 HHS may use CER findings to help 

inform Medicare coverage decisions, but PPACA does not allow Medicare 

coverage to be denied solely on the basis of CER findings.19

 1842 U.S.C. § 1320e-1(e). A quality adjusted life-year is an economic evaluation that 

assesses the monetary value of both the quality and the quantity of life and the cost of 

medical interventions to determine the value of health outcomes. 

1942 U.S.C. § 1320e-1(b)(2) (c)(2). 

PPACA Limitations on Use of 

CER
Page 8 GAO-18-311 Comparative Clinical Effectiveness Research Funding

In fiscal years 2010 through 2017, PCORI committed about $1.6 billion 

(or 79 percent of its total award commitments of $2.0 billion) to awards for 

conducting CER and $325 million (or 16 percent) to awards for building 

data capacity for research. In addition, PCORI committed $93 million for 

engagement and workforce awards to involve stakeholders in the 

research process and expand the research workforce, and committed $12 

million for awards to disseminate and implement its research findings. 

Awards for the dissemination and implementation of its research findings 

were limited as of the end of fiscal year 2017, as most of this research 

was still underway.20 (See table 1 for PCORI’s award commitments for 

fiscal years 2010 through 2017.)

Table 1: PCORI Award Commitments for Comparative Clinical Effectiveness Research Activities, Fiscal Years 2010 through 

2017 (Dollars in Millions) 

Activity 2010-2013 2014 2015 2016 2017 Totala

Research awards 274 304 372 327 314 1,591

Building data capacity awards 9 95 142 44 35 325

Engagement and workforce awards 0 3 15 23 52 93

Dissemination and implementation 

awards

0 0 0 10 2 12

Totala 283 402 529 404 403 2,021

Source: GAO analysis of Patient-Centered Outcomes Research Institute (PCORI) award commitment data. Ɩ GAO-18-311

a

Totals may not add up due to rounding.

By the end of fiscal year 2024, PCORI projects to spend a total of almost 

$3.3 billion, which reflects its projected Trust Fund revenue through fiscal 

year 2019 plus interest income. This total amount encompasses the 

commitments PCORI has made for awards through fiscal year 2017, as 

well as $514 million in projected additional research award commitments 

 

20These award commitments include expenditures through fiscal year 2017 as well as 

remaining payments for these commitments to be made by 2024. Of the $2.0 billion in 

award commitments PCORI made through fiscal year 2017, PCORI spent $948 million 

through fiscal year 2017 and expects to spend the remaining $1.1 billion in fiscal years 

2018 through 2024.

PCORI Committed 

Funds Primarily to 

Research and Data 

Capacity Efforts; 

Awards for 

Dissemination and 

Implementation of 

Findings Were 

Limited as Most 

Research Was Still 

Underway
Page 9 GAO-18-311 Comparative Clinical Effectiveness Research Funding

to be made by the end of fiscal year 2019 and $207 million for other 

award commitments to be made by the end of fiscal year 2021. In 

addition to awards, the total includes PCORI’s expenditures for program 

and administrative support services in fiscal years 2010 through 2017, as 

well as projected expenditures for these services through fiscal year 

2024.21 (See fig. 2 for PCORI’s actual and projected commitments and 

expenditures and see app. I for an overview of PCORI’s awards.)

Figure 2: PCORI Actual and Projected Commitments and Expenditures, Fiscal Years 2010 through 2024 (Dollars in Millions)

Note: Amounts listed are current as of Jan. 5, 2018.

The following information provides details on PCORI’s awards related to 

research, building data capacity, engagement and workforce activities, 

and the dissemination and implementation of its research findings.

 21PCORI spent $193 million in fiscal years 2010 through 2017 for program support 

services, such as staff salaries and benefits, as well as $117 million for administrative 

support services. PCORI officials anticipate additional expenditures of $206 million for 

these services through 2024 in order to see these award commitments through to 

completion. Descriptions of the activities under each PCORI award category may include 

some program support services. PCORI projects that its administrative expenses will 

average 6 percent of its total expenditures for fiscal years 2010 through 2024.
Page 10 GAO-18-311 Comparative Clinical Effectiveness Research Funding

PCORI committed $1.6 billion, or 79 percent of its total award 

commitments, for research in fiscal years 2010 through 2017.22 In fiscal 

years 2018 and 2019, PCORI projects to commit an additional $514 

million for research awards. PCORI research awards have increasingly 

focused on conditions that impose a substantial health or financial burden 

on patients and the healthcare system. (See table 2 for information on the 

health conditions that received the highest research award funding.)

Table 2: Heath Conditions That Received the Highest PCORI Research Award Commitments, Fiscal Years 2010 through 2017 

Condition

Total 

commitments 

(dollars in 

millions) Description and examples

Mental and 

behavioral health

327 PCORI awarded funding for 93 studies on depression, substance abuse, bipolar disorder, 

schizophrenia, anxiety disorders, autism, and related projects. For example, in one study 

researchers are determining whether it is more effective to treat patients with post-traumatic 

stress and bipolar disorders through integrated care or by using telemedicine technology to 

facilitate referrals to local mental health specialists.

Cancer 286 PCORI awarded funding for 78 studies on prevention or treatments for breast, colorectal, lung, 

prostate, cervical, blood, and other cancer projects. For example, one study compared surgery, 

radiation or active surveillance outcomes for men with localized prostate cancer. 

Cardiovascular 

disease

271 PCORI awarded funding for 62 studies on congestive heart failure, hypertension, strokes, and 

other cardiovascular conditions. For example, one study demonstrated that stroke survivors 

who used the blood-thinning drug warfarin after leaving the hospital were able to stay in their 

homes an average of 46 more days than those who did not take the drug.

Neurological 

disorders

260 PCORI awarded funding for 65 studies related to Multiple Sclerosis, Alzheimer’s disease, 

dementia, and other cognitive impairment issues. For example, one study is comparing the 

effects of each of the five components of an intensive, multipronged, behavioral intervention 

that is designed to delay or prevent onset of dementia in people with mild cognitive impairment. 

Multiple chronic 

conditions

225 PCORI awarded funding for 55 studies on patients with two or more chronic conditions such as 

diabetes, hypertension, obesity, or depression, and other projects focused on multiple chronic 

conditions. For example, one study focused on comparing case management with standard 

care for older patients with multiple chronic conditions.

Source: Patient-Centered Outcomes Research Institute (PCORI) information. | GAO-18-311

Note: Studies may be counted in more than one category.

Similar to certain types of CER that may take many years, the entire 

research award process for PCORI-funded CER may span multiple years 

from the funding announcement to the dissemination of completed 

 22This amount includes $785 million PCORI has paid on research awards through fiscal 

year 2017 as well as $806 million in remaining payments for these commitments to be 

made by 2024.

Research Awards
Page 11 GAO-18-311 Comparative Clinical Effectiveness Research Funding

research.23 Specifically, the process PCORI established can take as 

many as 6 years, which includes requesting and reviewing proposals, 

awarding contracts, recruiting participants or obtaining data, conducting 

and reviewing research, and disseminating findings and typically involves 

awards that span multiple years.24 For example, PCORI estimates that 

the typical timeframe for announcing funding and selecting applications to 

receive research awards can take 8 to 11 months as PCORI brings 

scientists, patients, payers, and other stakeholders together to prioritize 

proposals based on the impact of the condition, potential to improve 

health, technical merit, patient-centeredness, and engagement. (See fig. 

3.)

 23PCORI officials told us that PCORI-funded research is considered complete only after its 

findings are posted on the PCORI website. 

24For example, in 2016 PCORI awarded $15 million for a multi-year study Comparing 

Three Medicines to Prevent Blood Clots in Patients Who Need Long Term Blood-Thinning 

Treatment. This Duke University randomized control trial will examine 3,000 patients in 60 

locations to determine whether those taking two newer medications have fewer bleeding 

complications than patients taking the older medicine and will compare the two newer 

blood thinners to each other.
Page 12 GAO-18-311 Comparative Clinical Effectiveness Research Funding

Figure 3: PCORI Timeline Estimates for Its Research Award Process

Most of PCORI’s research projects, awarded through fiscal year 2017, 

were still underway. Only 53 of its 543 research projects had been 

completed as of the end of fiscal year 2017—in part because PCORI’s 

research award process typically takes 2 to 6.5 years to complete, and 

because almost two-thirds of the funds committed for research projects 

were awarded in fiscal years 2015 through 2017.25 While most PCORIfunded research is underway, a larger number of research studies are 

projected to be completed by of the end of each year from 2018 to 2022, 

with all of the remaining studies to be completed by 2024. (See fig. 4.) 

PCORI officials told us that the institute attempts to manage its funds to 

ensure that its research awards are funded and managed through 

completion, including peer review and the distribution of research 

 25PCORI officials also told us that, after PPACA authorized the establishment of PCORI in 

2010, it took over a year to form its board of governors, hire staff, and develop policies 

and procedures for funding awards before it could begin making award commitments. 
Page 13 GAO-18-311 Comparative Clinical Effectiveness Research Funding

findings, in recognition of the time needed to conduct this research as 

well as the uncertainty regarding the total amount of funding available.26

Figure 4: Number of PCORI-Funded Research Studies Underway and Completed, Fiscal Years 2017 through 2024

a

Projects underway may be counted in multiple years.

Officials from all but one of the stakeholder organizations we 

interviewed—public and private payers, health care providers, and patient 

advocacy organizations that represented potential users of CER—

generally supported PCORI’s research award priorities. Most of the 

stakeholders we interviewed stressed the importance of research 

conducted by unbiased organizations, such as the federally funded 

research funded by PCORI and HHS. In addition, most stakeholders also 

told us that PCORI’s efforts to engage patients in the research process 

 26The fees collected by Treasury from private insurance and self-insured health plans 

depend upon the average number of covered lives in a policy in a given year and 

therefore can vary annually.
Page 14 GAO-18-311 Comparative Clinical Effectiveness Research Funding

have changed the way research is conducted for the better, such as 

prioritizing research outcomes that are most meaningful to patients. 

However, officials from an organization representing payers (and from an 

individual health plan) told us that PCORI’s priorities did not fully align 

with their needs, such as their needs for CER on certain high-cost 

conditions, medications or treatments.

PCORI committed the second largest portion of award funding—$325 

million through fiscal year 2017—for awards to build data capacity for 

research through the development of PCORnet.27 PCORI officials told us 

that the institute supported the development of the PCORnet initiative in 

order to use existing medical records and claims data and to transform 

much of that data into a common data model to be used for clinical 

research, until such time when such data will have been standardized in 

electronic health records so that they can easily be used for research. As 

of December 2017, PCORnet included 36 partner networks agreeing to 

link their electronic claims and health data.28 PCORI officials told us that 

this distributed data network already comprises a nationally 

representative sample of approximately 128 million individuals whose 

data can be used in randomized clinical trials, large observational studies, 

and other research. In fiscal years 2018 and 2019, PCORI projects to 

commit an additional $70 million for these awards to continue building this 

data capacity.

PCORnet research is managed through its Coordinating Center, which 

oversees the translation of certain categories of the partner networks’ 

data into the common data model and forges agreements with each of the 

partners to share results of queries using their data with researchers.29

 27This amount includes $115 million PCORI has paid for building data capacity awards 

through fiscal year 2017 as well as $210 million in remaining payments for these 

commitments to be made by 2024.

28These partner networks include 21 patient-powered research networks operated and 

governed by groups of patients and their partners; 13 clinical data research networks 

based in healthcare systems such as hospitals, practice-based networks, and health 

centers; and 2 health plan research networks operated by insurers.

29The Coordinating Center oversees the research infrastructure, ensures the reliability of 

the partner networks’ data, provides for data security, and develops software for the 

system. PCORnet continues to expand the categories of data that can be translated into 

the common data model, thereby expanding the data available for research. It has also 

developed Institutional Review Board systems to facilitate and speed the review of 

PCORnet research for clinical trials and is developing other resources to facilitate 

research using partners’ data.

Building Data Capacity Awards
Page 15 GAO-18-311 Comparative Clinical Effectiveness Research Funding

This research process generally starts when a researcher requests to 

query data on a specific population, after which PCORnet may approve 

the request and invite network partners to participate. Participating 

network partners then run queries on their data following established 

parameters and submit the results to a secure portal that the researcher 

can access in order to analyze the results for research. (See fig. 5.) 

PCORI officials told us that there were 32 research projects using 

PCORnet that received funding through PCORI’s research award process 

as of the end of December 2017, as well as 45 research projects funded 

by other parties, including federal agencies and private industry.

Figure 5: Process for Researcher to Receive Data Query Results from PCORnet Partners

Further, as part of its building data capacity awards, in fiscal year 2017 

PCORI committed $25 million to the People-Centered Research 

Foundation, a nonprofit foundation formed in March 2017 to support the 

network partners and other entities conducting research using PCORnet. 

This funding was provided to support this foundation’s development of a 

business plan, as well as its governance structure, to ensure the 

continuity of the PCORnet network partnership efforts after PCORI 
Page 16 GAO-18-311 Comparative Clinical Effectiveness Research Funding

funding for PCORnet ends. PCORI has indicated it may provide additional 

funding to the foundation, provided that the foundation and the networks 

make progress toward self-sustainability.

Officials from most stakeholder organizations we interviewed generally 

agreed that PCORnet offers value by improving the data available to 

conduct CER. Officials from two organizations told us that PCORnet has 

made it possible to use network partners’ aggregated data to make 

conducting research more efficient than in the past.

Through fiscal year 2017, PCORI also committed $93 million for 

engagement and workforce awards.30 For example, PCORI committed a 

total of $63 million for engagement awards, intended to involve a variety 

of stakeholders in the research process and to improve the methodology 

for carrying out CER. Engagement awards include “Eugene Washington 

Engagement Awards” that are intended to bring patients, caregivers, 

clinicians, and other healthcare stakeholders into the research process 

and to disseminate study results.31 In addition, “Pipeline to Proposal 

Awards” are intended to bring together stakeholders with strong interests 

in a specific health issue to develop research proposals to address their 

needs. Officials from the two patient advocacy organizations we 

interviewed told us that PCORI’s engagement awards have helped to 

support patient involvement in the research process. For example, one 

official noted that, while it has not been easy to find patients willing to 

participate, these awards have been important to train and support 

patients in the research process.

PCORI also committed $30 million to workforce training awards for 

clinicians and researchers. For example, one of PCORI’s career 

development programs, conducted in partnership with AHRQ, is designed 

to train clinician and research scientists to conduct patient-centered 

 30This amount includes $44 million PCORI has paid for engagement and workforce 

awards through fiscal year 2017 as well as $49 million in remaining payments for these 

commitments to be made by 2024.

31For example, the National Kidney Foundation received an award to foster collaboration 

among patients, clinicians, caregivers, researchers, health payers, and healthcare industry 

representatives to facilitate the development of research designs to measure home 

dialysis quality as well as interventions to address the barriers to maintaining dialysis 

treatment at home.

Engagement and Workforce

Awards
Page 17 GAO-18-311 Comparative Clinical Effectiveness Research Funding

outcomes research and to actively engage stakeholders in efforts to 

improve the quality and safety of care.32

Dissemination and implementation awards for PCORI-funded research 

findings thus far have been limited as most of the research was still 

underway, but, according to PCORI officials, awards for this work will 

substantially increase as research is completed. Specifically, through 

fiscal year 2017, PCORI committed a total of $12 million for awards to 

disseminate and implement PCORI-funded research by helping 

researchers and other stakeholders to publicize findings and by 

supporting patients and providers to utilize findings.33 PCORI projects to 

commit an additional $91 million for these awards in fiscal years 2018 

through 2021.

Dissemination and implementation awards are intended to encourage 

PCORI awardees that have completed research and their patient and 

stakeholder partners to pursue strategic activities to disseminate and 

implement their findings. For example, PCORI awarded about $0.4 million 

to increase awareness and promote the use of research findings on using 

technology to deliver virtual care home visits for those with Parkinson’s 

disease. According to PCORI, these funds will be used to train 

neurologists and other health professionals to provide virtual care for 

patients in their homes. In addition, as part of its efforts to summarize 

research findings, PCORI also awarded funds to the American Institutes 

for Research to establish a Translation Center that develops two 

summaries of each of PCORI’s research findings: a public abstract for 

general audiences that is also translated into Spanish and a professional 

abstract for clinicians.

In addition to awards, PCORI has fostered the dissemination and 

implementation of its research findings in other ways, including through its 

website, publications, and roundtable briefings. For example, according to 

PCORI, it posts research findings on its website within 90 days of 

receiving final peer-reviewed research results so that patients and 

providers have access to the information to make healthcare decisions. In 

addition, according to PCORI, it pays journals’ open access fees to allow 

 32We describe AHRQ’s workforce training efforts later in this report. 

33This amount includes $4 million PCORI has paid for dissemination and implementation 

awards through fiscal year 2017 as well as $8 million in remaining payments for these 

commitments to be made by 2024.

Dissemination and 

Implementation Awards
Page 18 GAO-18-311 Comparative Clinical Effectiveness Research Funding

free public access to selected research and plans to support research 

awardees to place accepted journal manuscripts in the PubMed Central 

database. PCORI also facilitates roundtable briefings that bring together 

clinicians, patients, and others with interests in recent findings in order to 

build support for immediate use of the findings. PCORI also coordinates 

its dissemination efforts with AHRQ.34

PCORI considers the implementation of its research methods and 

findings to be an integral part of its dissemination efforts and a 

culmination of its work and so has begun efforts to track implementation, 

such as the number of its findings published in peer-reviewed journals, 

and the use of its findings in clinical care. For example, PCORI officials 

told us that that there were 891 publications in peer-reviewed journals 

that resulted from studies fully or partially funded by PCORI through 

October 2017. According to PCORI, two PCORI-funded studies on 

prostate cancer, one study on oral versus intravenous antibiotics for 

certain children, and one study on self-monitoring of blood glucose were 

included in medical resource software that is used by nearly 90 percent of 

academic medical centers in the United States.

Most of the stakeholder officials we interviewed noted the importance of 

disseminating research findings quickly and in ways that are readily 

available and understandable to both experts and the general public to 

raise awareness about the findings. While officials representing two 

payers noted limitations to the usefulness of PCORI’s research findings 

because they do not take treatment costs into account, most stakeholder 

officials noted the importance of the PCORI-funded research underway 

and looked forward to utilizing the research findings once they become 

available.35 In particular, officials representing provider and patient 

advocacy organizations told us that they were interested in ensuring that 

the most important research findings would be quickly implemented by 

patients and clinicians.

 34We describe AHRQ’s dissemination efforts later in this report. 

35Officials representing two payers told us that cost is a critical component of the 

information they need to make decisions about coverage policies. However, according to 

PCORI, it does not fund research that analyzes cost effectiveness because of restrictions 

in PPACA prohibiting it from developing or using a dollars-per-quality adjusted life-year to 

establish what type of health care is cost effective or recommended. See 42 U.S.C. § 

1320e-1(e).
Page 19 GAO-18-311 Comparative Clinical Effectiveness Research Funding

Between fiscal years 2011 and 2017, HHS’s AHRQ obligated about $260 

million (or 58 percent of HHS’s $448 million in total obligations) for the 

dissemination and implementation of CER findings. According to AHRQ 

officials, because most PCORI-funded research had not been completed 

by the end of fiscal year 2017, these efforts were primarily focused on the 

dissemination and implementation of research funded by other entities, 

including NIH and the Centers for Disease Control and Prevention (CDC). 

Additionally, AHRQ obligated $94 million for efforts to train researchers 

on conducting CER, and ASPE obligated $85 million for efforts to build 

data capacity. AHRQ and ASPE have obligated a total of $9 million for 

administrative activities during those years. Table 3 provides an overview 

of HHS’s obligations in each fiscal year.

Table 3: HHS Obligations for Comparative Clinical Effectiveness Research Activities, Fiscal Years 2011 through 2017 (Dollars 

in Millions) 

Activitya 2011 2012 2013 2014 2015 2016 2017 Totalb

Dissemination and implementation 4.9 31.1 28.3 82.6 73.0 40.2 260.1

Training 2.4 11.4 19.9 22.2 22.9 14.9 93.8

Building data capacity 3.0 7.1 11.3 20.7 17.3 25.8 85.2

Administrative 0.0 0.4 0.9 1.8 2.1 2.1 2.0 9.3

Totala 0.0 10.7 50.5 61.3 127.6 115.3 82.9 448.4

Source: GAO analysis of Department of Health and Human Services (HHS) obligation data. Ɩ GAO-18-311

a

HHS’s dissemination and implementation as well as its training activities are managed by the Agency 

for Healthcare Research and Quality, while HHS’s data capacity initiatives are managed by the Office 

of the Assistant Secretary for Planning and Evaluation.

b

Totals may not add up due to rounding.

AHRQ and ASPE plan to obligate an additional $120 million for 

dissemination and implementation, training, building data capacity, and 

administrative activities during fiscal years 2018 through 2020. They 

expect to have $245 million available to fund ongoing and future CER 

activities, based on expected transfers from the Trust Fund in fiscal years 

2018 and 2019.36 (See fig. 6.)

 36As stated previously, for each of fiscal years 2011 through 2019, Treasury must transfer 

20 percent of Trust Fund revenue to HHS. Amounts transferred to HHS remain available 

until expended. The fees collected by Treasury from private insurance and self-insured 

health plans depend upon the average number of covered lives in a policy in a given year 

and therefore can vary annually.

HHS Obligated Funds 

Primarily for the 

Dissemination and 

Implementation of 

Research
Page 20 GAO-18-311 Comparative Clinical Effectiveness Research Funding

Figure 6: HHS Actual and Projected Obligations, Fiscal Years 2011 through 2020 (Dollars in Millions)

Note: Amounts listed are current as of Sept. 30, 2017. HHS’s dissemination and implementation as 

well as its training activities are managed by the Agency for Healthcare Research and Quality, while 

HHS’s data capacity initiatives are managed by the Office of the Assistant Secretary for Planning and 

Evaluation.

The following information provides details on HHS-funded projects related 

to dissemination and implementation, training on conducting CER, and 

building data capacity.

During fiscal years 2011 through 2017, AHRQ obligated a total of $260 

million for CER dissemination and implementation initiatives and plans to 

obligate an additional $93 million for these initiatives in fiscal years 2018 

through 2020. According to officials, AHRQ plans to fund additional 

dissemination and implementation initiatives in fiscal years 2018 and 

2019 but had not finalized those plans as of January 2018. (See app. II 

for an overview of all of AHRQ’s dissemination and implementation 

initiatives.) AHRQ’s dissemination and implementation initiatives 

comprise efforts to synthesize CER findings, translate and communicate 

research findings to potential users, and implement them:

• Synthesis of CER findings: According to AHRQ officials, AHRQ’s 

Evidence-Based Practice Centers developed 48 systematic reviews of 

CER findings based on completed research. As of the end of fiscal 

year 2017, 40 of these reviews had been published, while 8 were still 

in progress. Officials told us that these systematic reviews have likely 

Dissemination and 

Implementation
Page 21 GAO-18-311 Comparative Clinical Effectiveness Research Funding

not included PCORI-funded research, as most of that research had 

not been completed by the end of fiscal year 2017.

• Translation and communication of CER findings: AHRQ funded 

initiatives, which—according to the agency—are aimed at making 

CER findings accessible and understandable to health care 

professionals, patients, and others. For example, AHRQ developed a 

“Library of Patient-Centered Outcomes Research Resources” website 

with links to CER databases maintained by other entities including 

NIH and PCORI.37 Another example is AHRQ’s “John M. Eisenberg 

Center for Clinical Decisions and Communications Science,” which 

translates research findings into information that can be used by 

consumers, health care providers, and policymakers.38

• Implementation of CER findings: AHRQ funded four key initiatives to 

implement CER findings. According to AHRQ officials, one of the four 

initiatives includes PCORI-funded research, while the other three 

have thus far focused on implementing existing CER funded by other 

entities:

• The “Dissemination and Implementation Initiative” was designed 

to disseminate and implement government-funded CER findings—

including PCORI-funded findings—relevant to physicians, 

healthcare providers, patients, and others. This initiative consists 

of a multi-step approach for identifying several areas of CER each 

year that—according to AHRQ officials—have the greatest 

potential for impact and are feasible to implement. (See figure 7 

for an overview of this process.) According to AHRQ officials, as 

of December 2017, 37 findings have been nominated for 

consideration under AHRQ’s Dissemination and Implementation 

Initiative, including 5 findings nominated by PCORI. According to 

these officials, 1 of the findings PCORI has nominated is under 

consideration for implementation. Two were rejected—1 because 

 37The website is intended to assist researchers who may be conducting new studies, as 

well as clinicians, policymakers, consumers, and others who are seeking access to 

evidence-based health information. AHRQ did not use any resources from the Trust Fund 

to support this effort during the time of our review but plans to in the future. 

38According to AHRQ, the Eisenberg Center develops products to support informed, 

shared, and evidence-based health care decisions regarding tests or treatments for 

different types of health conditions. The products, including research summaries and 

patient decision aids, are presented in short, easy-to-use formats. In addition, the 

Eisenberg Center works with local and national patient and consumer organizations to 

identify opportunities to disseminate research findings. 
Page 22 GAO-18-311 Comparative Clinical Effectiveness Research Funding

of insufficient impact and the other because of challenges in 

implementation feasibility. (Two are still under review.)

Figure 7: Overview of the AHRQ Dissemination and Implementation Initiative Process

• The “Evidence Now” initiative disseminates CER evidence directly 

to primary care practices and supports them in implementing 

clinical and organizational evidence in practice through regional 

cooperatives.

• The “Comparative Health System Performance Initiative” 

established three centers of excellence and a coordinating center 

to identify, classify, track, and compare health systems. AHRQ’s 

goal is to understand the factors that affect health systems’ use of 

CER and to identify best practices in disseminating and using 

CER.
Page 23 GAO-18-311 Comparative Clinical Effectiveness Research Funding

• The “Clinical Decision Support (CDS) Initiative” is designed to use 

CDS to promote the timely incorporation of CER findings into 

clinical practice.39

Some of AHRQ’s dissemination and implementation initiatives—such as 

“Evidence Now” and “CDS Initiative”—include an evaluation component, 

as described in app. II. According to AHRQ officials, as of January 2018 

results from these evaluations were not yet available.

Between fiscal years 2011 and 2017, AHRQ obligated a total of $94 

million for awards supporting training in the methods used to conduct 

CER. AHRQ plans to obligate an additional $14 million for training on 

conducting CER by fiscal year 2020. AHRQ has funded eight categories 

of awards for individual researchers or research institutions. For example, 

AHRQ’s “Infrastructure Development Program in Patient-Centered 

Outcomes Research” award supports institutions in the development of 

their capacity to conduct and implement CER. Its “Institutional Mentored 

Career Development Award Program in Patient-Centered Outcomes 

Research” award supports the development of researchers in academic 

and applied settings. (See app. III for an overview of these awards.) 

Starting in fiscal year 2018, AHRQ plans to fund an additional training 

award category in conjunction with PCORI. AHRQ developed a plan to 

evaluate its training activities and, according to AHRQ officials, the 

evaluation is expected to be funded in fiscal year 2018.

Between fiscal years 2012 and 2017, ASPE obligated a total of $85 

million for 30 projects designed to build data capacity for conducting CER 

and plans to obligate an additional $6 million to existing projects and 1 

new project through fiscal year 2019. Officials told us that ASPE plans to 

fund additional projects to build data capacity in fiscal years 2018 and 

2019, based on HHS leaders’ priorities, but had not finalized those plans 

as of January 2018. ASPE manages these projects, which are largely 

carried out by other HHS agencies through interagency agreements and 

are intended to develop and maintain a comprehensive, interoperable 

data network to collect, link, and analyze data on outcomes and 

 39CDS provides health care providers and other individuals with knowledge and personspecific information, intelligently filtered or presented at appropriate times, to enhance 

health and health care. CDS encompasses a variety of tools, such as computerized alerts 

and clinical guidelines, to enhance decision-making in the clinical workflow. 

Training on Conducting CER

Building Data Capacity
Page 24 GAO-18-311 Comparative Clinical Effectiveness Research Funding

effectiveness from multiple sources for CER.40 (See app. IV for an 

overview of these activities.)41

In response to a recommendation in our 2015 report on HHS’s CER 

activities, ASPE implemented a monitoring system to track progress 

toward its milestones and deliverables for these projects.42 ASPE also 

contracted to evaluate its projects to build data capacity for CER. The 

evaluation, completed in December 2017, found that ASPE made 

progress managing these projects towards the core functionalities 

outlined in its strategic framework. However, among other things, the 

evaluation found that additional efforts are needed to explore how to 

enhance data privacy and security, ensure data quality, and 

operationalize related standards. According to ASPE officials, the 

evaluation will inform the development and implementation of future 

ASPE projects to build data capacity for conducting CER.

We provided a draft of this report to PCORI and HHS for review and 

comment. PCORI and HHS provided technical comments, which we 

incorporated as appropriate.

We are sending copies of this report to the appropriate congressional 

committees, the Executive Director of PCORI, the Secretary of Health 

and Human Services, the Director of AHRQ, the Assistant Secretary for 

 40The activities are carried out by the following HHS agencies: AHRQ, ASPE, CDC, 

Centers for Medicare and Medicaid Services, Food and Drug Administration, Health 

Resources and Services Administration, NIH, National Libraries on Medicine, and Office of 

the National Coordinator for Health Information Technology. In fiscal year 2012, ASPE 

obligated about $0.2 million for the establishment of intra-departmental partnerships 

between ASPE and other agencies within HHS. 

41ASPE’s projects are designed to align with the five core functionalities identified in 

HHS’s Patient-Centered Outcomes Research Trust Fund strategic plan: 1) use of clinical 

data for research; 2) standardized collection of standardized clinical data; 3) linking clinical 

and other data; 4) collection of participant-provided information via new data collection 

technologies that provide a means for collecting patient generated information critical to 

patient-centered research; and 5) use of enhanced publicly-funded data systems for 

research.

42See GAO-15-280. In response to GAO’s recommendation, in each quarter the agencies 

responsible for implementing each project are required to report on performance towards 

goals and objectives, among other requirements. ASPE developed “individual dashboard” 

reports for the projects that include objectives, milestones, and timeframes, as specified in 

GAO’s recommendation.

Agency Comments
Page 25 GAO-18-311 Comparative Clinical Effectiveness Research Funding

ASPE, and other interested parties. In addition, the report will be available 

at no charge on GAO’s website at http://www.gao.gov.

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

me at (202) 512-7114 or dickenj@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 major contributions to this report 

are listed in app. V.

John E. Dicken

Director, Health Care
Page 26 GAO-18-311 Comparative Clinical Effectiveness Research Funding

List of Committees

The Honorable Orrin G. Hatch

Chairman

The Honorable Ron Wyden

Ranking Member

Committee on Finance

United States Senate

The Honorable Lamar Alexander

Chairman

The Honorable Patty Murray

Ranking Member

Committee on Health, Education, Labor, and Pensions

United States Senate

The Honorable Greg Walden

Chairman

The Honorable Frank Pallone Jr.

Ranking Member

Committee on Energy and Commerce

House of Representatives

The Honorable Kevin Brady

Chairman

The Honorable Richard Neal

Ranking Member

Committee on Ways and Means

House of Representatives
Appendix I: Patient-Centered Outcomes 

Research Institute (PCORI) Award 

Commitments Made During Fiscal Years 2010 

through 2017

Page 27 GAO-18-311 Comparative Clinical Effectiveness Research Funding

Award 

category Award category descriptiona

Total 

commitments 

(dollars in 

millions)

Research 

awards

These awards generally fund research studies in priority areas for conditions that impose a 

substantial burden on patients and the healthcare system.

Information about individual research awards can be found at: 

https://www.pcori.org/research-results?f%255B0%255D=field_project_type%3A298&f%5B0%5D=fiel

d_project_type%3A298#search-results.

1,591

Building data 

capacity 

awards

These awards fund infrastructure projects to build data capacity through the development of 

PCORnet and support for clinical and patient-powered data research networks.

Information about individual building data capacity awards can be found at: 

https://www.pcori.org/research-results?f%5B0%5D=field_project_type%3A441#search-results.

325

Engagement 

and workforce 

awards

Engagement awards fund projects to improve the methodology for carrying out research by involving 

patients, caregivers, clinicians, and other healthcare stakeholders into the research process. 

Workforce training awards provide accredited continuing education opportunities, in coordination with 

the Agency for Healthcare Research and Quality, for researchers and clinicians.

Information about individual engagement awards and workforce awards can be found at: 

https://www.pcori.org/research-results?f%5B0%5D=field_project_type%3A299#search-results and

https://www.pcori.org/research-results/2017/k12-institutional-mentored-career-development-program.

93

Dissemination 

and 

implementation 

awards

Dissemination and implementation awards are intended to help researchers and other stakeholders 

to publicize findings, and support the utilization of findings for patients and providers.

Information about individual dissemination and implementation awards can be found at: 

https://www.pcori.org/research-results?f%5B0%5D=field_project_type%3A308#search-results.

12

Source: GAO analysis of PCORI documentation. | GAO-18-311

a

All websites accessed on January 23, 2018.

Appendix I: Patient-Centered Outcomes 

Research Institute (PCORI) Award 

Commitments Made During Fiscal Years 

2010 through 2017
Appendix II: Agency for Healthcare Research 

and Quality’s (AHRQ) Dissemination and 

Implementation Initiatives, as of September 30, 

2017

Page 28 GAO-18-311 Comparative Clinical Effectiveness Research Funding

Initiative title Description

Total 

obligations 

(dollars in 

millions)

Focusing on the ABCs: 

Accelerating the 

Dissemination and 

Implementation of 

Patient-Centered 

Outcomes Research 

(PCOR) Findings into 

Primary Care Practice 

(Evidence Now)a

Disseminates evidence to primary care practices and supports them in implementing clinical 

and organizational evidence in practice through regional cooperatives. AHRQ awarded a 

separate grant to establish an independent, external evaluation to study improvements in 

the delivery of the ABCs. 

96.1b

Comparative Health 

System Performance in 

Accelerating PCOR

Develops three Centers of Excellence on “Comparative Health System Performance in 

Accelerating PCOR Dissemination.” According to AHRQ, the Centers of Excellence will 

identify and classify characteristics of health care systems over 5 years. They will also 

identify ways to assess the quality and cost of such systems, including their use of PCOR, 

understand the characteristics of high performing systems, and identify what system 

characteristics are associated with more rapid adoption and diffusion of PCORrecommended practices throughout a system. 

34.2c

The Evidence-Based 

Practice Centers –

Evidence Synthesis and 

Translation

Evaluates and synthesizes research findings to aid decision-making for patients, providers, 

and payers, among others. 

27.9d

John M. Eisenberg 

Clinical Decisions and 

Communications Science 

Center

Translates PCOR findings into tools, such as research summaries and decision aids, 

designed to help patients and consumers, clinicians, and policymakers make informed and 

evidence-based health care decisions.

15.2

Disseminating PCOR to 

Improve Healthcare 

Delivery Systems

Expands an existing initiative or creates a new initiative that supports multi-site, multi-region, 

multi-stakeholder dissemination and implementation of evidence. 

13.2

Methods for 

Dissemination and 

Translation

Develops and tests methods for translating and disseminating PCOR findings to hard-toreach audiences, including patients with low health literacy, disadvantaged populations, 

isolated clinicians and policy makers, and other decision makers who may not have had the 

benefit of more traditional translation and dissemination efforts.

11.4

Dissemination and 

Implementation of PCOR 

Findings to Clinicians

Integrates PCOR into clinical practice using various methods shown to improve the uptake 

of scientific evidence in clinical decision making. Grantees were asked to consider both 

educational theory and the relevance of “new media” as they designed their programs.

6.9

Horizon Scanning 

System: Dissemination of 

Information on Emerging 

Interventions

Searches for emerging interventions, prioritizes those most likely to have a large impact in 

the near future, and disseminates the information to the public. According to AHRQ, the 

Horizon Scanning System screened more than 22,000 potential intervention leads and 

tracked over 2,300 intervention topics.

5.5

National Guideline 

Clearinghouse

Provides targeted audiences—such as providers and payers—with an accessible tool for 

obtaining objective, detailed information on evidence-based clinical practice guidelines to 

further their dissemination, implementation, and use. 

5.3

Registry of Patient 

Registries 

Promotes collaboration, reduces redundancy, and improves transparency in patient 

registries. 

5.1

Appendix II: Agency for Healthcare Research 

and Quality’s (AHRQ) Dissemination and 

Implementation Initiatives, as of September 

30, 2017
Appendix II: Agency for Healthcare Research 

and Quality’s (AHRQ) Dissemination and 

Implementation Initiatives, as of September 30, 

2017

Page 29 GAO-18-311 Comparative Clinical Effectiveness Research Funding

Initiative title Description

Total 

obligations 

(dollars in 

millions)

Electronic Data Methods 

Forum

Created informational tools to support the dissemination and implementation of PCOR 

findings, including best practices and new knowledge about the use of electronic health 

record data for research and quality improvement. 

5.1

Supporting Decisions 

With PCOR: PCOR 

Clinical Decision Support 

Initiative

Promotes the timely incorporation of PCOR findings into clinical practice—which 

encompasses a variety of tools to enhance clinical decision-making. 

5.0e

National Initiative 

(Publicity Center)

Collaborated with 176 national organizations to disseminate materials for the Effective 

Health Care Program.

4.2

“Value of Using Medical 

Evidence” – A Paid MultiMedia Campaign

Multi-media campaign to educate health care consumers about the value of reviewing 

medical evidence when weighing treatment options. 

3.4

Educating the Educators Conducted and disseminated research to develop a process for shared decision making that 

includes exploring and comparing the benefits, harms, and risks of each option through 

meaningful dialogue about what matters most to the patient. 

3.1

Patient-Generated 

Health Outcomes Data 

and Clinical Decision 

Support Using Smart 

Device Technology

Collects patient-generated health data, integrates patient-generated health data with PCOR 

evidence, and disseminates PCOR findings using mobile health technology. 

3.0

Closing the Gap in 

Disparities with PCOR

Identifies ways to reduce health care differences across diverse populations with a particular 

focus on minority populations in under-resourced healthcare settings. 

2.9

Regional Partnership 

Development Offices

Established five regional offices, responsible for developing and cultivating dissemination 

partnerships within each region. 

2.5

Implementation of a 

Systematic Review Data 

Repository Collaborative

This repository houses study data extracted from primary research publications during the 

course of conducting systematic reviews. It is designed to increase the transparency of 

comparative effectiveness reviews, improve the ability to update systematic reviews, 

improve the quality of abstracted data, and enhance the efficiency and reduce the costs of 

conducting reviews.

1.9

Deliberative Approaches 

for Patient Involvement 

in Implementing 

Evidence-Based Health 

Care

Gathers input from patients on a complex topic related to the implementation of evidencebased health-care decision making.1.9

Methods Guidance to 

Improve the 

Dissemination and 

Implementation of 

Systematic Reviews

Increases the relevance of AHRQ systematic reviews for patients, clinicians, and 

policymakers by examining and addressing challenging topic areas that may affect the 

credibility and utility of the review for end users and that are areas of inconsistency or 

variation among AHRQ systematic reviews.

1.3

Developing and 

Evaluating Methods for 

Record Linkage and 

Reducing Bias in Clinical 

Patient Registries

Conducted three projects to improve the development of registries, a major activity of 

AHRQ’s Effective Health Care Program.

1.1
Appendix II: Agency for Healthcare Research 

and Quality’s (AHRQ) Dissemination and 

Implementation Initiatives, as of September 30, 

2017

Page 30 GAO-18-311 Comparative Clinical Effectiveness Research Funding

Initiative title Description

Total 

obligations 

(dollars in 

millions)

Continuing Education of 

Comparative Clinical 

Effectiveness Research 

(CER)

Provides online continuing education materials that inform physicians and other health care 

providers about PCOR from the Effective Health Care Program.

0.9

Evaluating the 

Knowledge and 

Education Needs to 

Students of Health 

Professions on PCOR

Worked with health professional student associations to evaluate students’ understanding of 

the importance and clinical applicability of PCOR and shared decision-making to their 

practice and evaluated students’ educational needs and preferences related to integrating 

PCOR findings into their training curricula. 

0.7

Methods Center in 

Decision and Simulation 

Modeling

Created a decision-modeling methods center that reviewed the existing research and 

guidance published on modeling methods with input from a multidisciplinary group of 

experts. 

0.7

Enhancing CER Data 

Resources

Provides for maintenance and updating of existing data resources to conduct future CER 

through a grant competition. The grants fund three to four 1-year pilot projects aimed at 

enabling a future, larger competition to enhance the data infrastructure and move the 

resources to self-sustaining models. 

0.7

Dissemination & 

Implementation of PCOR

Disseminates CER findings published by the Patient-Centered Outcomes Research Institute 

(PCORI) and other government entities to providers, patients, payers, and others. This 

initiative consists of a seven-step approach for identifying research findings that have the 

greatest potential for implementation.

0.4f

Integrating PCOR into 

Clinical Decision Support 

Systems: An 

Environmental Scan

Provided an understanding of how AHRQ could effectively disseminate and promote PCOR 

findings and tools in the development and maintenance of clinical decision support systems. 

The project included a market analysis and an assessment of potential stakeholders and 

audiences, including vendors of health information technology focused on clinical decision 

support. Information gathered from this project directly informed the concept for the PCOR 

clinical decision support initiative that was launched in 2016.

0.3

PCOR Dissemination: InStore PromotionPromoted PCOR through public service announcements nationwide. 0.2

Increasing Access to 

PCOR/CER Findings

Created a new page on AHRQ’s website that highlights the agency’s own resources, as well 

as directs researchers, health professionals, patients, caregivers, and families to additional 

databases that collect information on CER. These databases provide summaries of findings 

from a wide range of CER findings and research that is in progress.

0.0g

Source: GAO analysis of AHRQ documentation. | GAO-18-311

a

PCOR is a form of CER.

b

AHRQ plans to obligate an additional $21.3 million for this initiative in fiscal year 2018.

c

AHRQ plans to obligate an additional $24.7 million for this initiative in fiscal years 2018 through 

2020.

d

AHRQ plans to obligate an additional $16.0 million for this initiative in fiscal years 2018 and 2019.

e

AHRQ plans to obligate an additional $30.0 million for this initiative in fiscal years 2018 through 

2020.

f

AHRQ plans to obligate an additional $0.4 million for this initiative in fiscal year 2018.

g

AHRQ plans to obligate $0.2 million for this initiative in fiscal year 2018.
Appendix III: Agency for Healthcare Research 

and Quality’s (AHRQ) Training Awards, as of 

September 30, 2017

Page 31 GAO-18-311 Comparative Clinical Effectiveness Research Funding

Award title Description

Total 

obligations 

(dollars in 

millions)

Infrastructure 

Development Program in 

Patient-Centered 

Outcomes Research 

(PCOR)a

Funds a 5-year, renewable effort to support the development of PCOR capacity among 

institutions that have basic health services research capacity but need to develop capacity to 

conduct and implement PCOR. The program would potentially include institutions located in 

geographic areas that lack capacity, and institutions that serve predominantly minority 

populations. 

33.3

Institutional Mentored 

Career Development 

Award Program in PCOR

Supports the development of researchers in academic and applied settings. The program 

combines didactic and experiential opportunities, focusing on the generation, adoption, and 

spread of new scientific evidence. The goal is to improve population-specific health 

outcomes by developing and disseminating evidence-based information to patients, 

clinicians, and other decision-makers, responding to their expressed needs, about which 

interventions are most effective for which patients under specific circumstances. 

26.3b

Researcher Training and 

Workforce Development 

in Methods and 

Standards for 

Conducting PCOR 

Studies

Provides basic, advanced, and experiential training on the methods to conduct PCOR, 

particularly prospective observational research, registries, and clinical trials. The program 

was open to researchers employed in both the public and private sectors, particularly those 

who serve minorities, economically or medically disadvantaged populations. 

8.9c

Pathway to 

Independence in PCOR

Facilitates the transition of postdoctoral candidates from mentored to independent research 

positions, accelerating research independence for PCOR researchers. 

7.9

Individual Mentored 

Career Development 

Award Program in PCOR

Provides support for intensive, research career development for individual investigators in 

academic or applied settings, leading to research independence in the field of PCOR and 

the generation and translation of new scientific evidence and analytic tools. 

7.6d

Research Career 

Enhancement Awards for 

Established Investigators 

in PCOR

Provides career development awards for established investigators to further develop their 

research expertise in PCOR methodologies. This concept seeks to accelerate the 

development of the research workforce capable of conducting PCOR.

5.5

PCOR Training Provides 2-year fellowships for training in PCOR. A focus for these fellowships is recruitment 

of trainees from diverse disciplines, including social and behavioral sciences, business, and 

engineering. The expected output of these fellowships is trained PCOR researchers. 

4.2

Training the Next 

Generation of PCOR 

Investigators to Lead 

Research on the 

Learning Healthcare 

System

Establishes an expert panel, comprised of 7 to 10 leaders in the fields of learning healthcare 

system, health services research, and PCOR, to assess the current state of health services 

research and PCOR training and recommend ways to improve core 

competencies/curriculum to meet the needs of the health system. Develops a report 

summarizing the panel’s recommendations concerning current deficiencies and 

recommendations regarding skills and competencies needed to meet the challenges. 

0.2

Source: GAO analysis of AHRQ documentation. | GAO-18-311

a

PCOR is a form of CER.

b

AHRQ plans to obligate an additional $7.3 million for this award in fiscal year 2018.

c

AHRQ plans to obligate an additional $2.4 million for this award in fiscal year 2018.

d

AHRQ plans to obligate an additional $4.1 million for this award in fiscal years 2018-2020.

Appendix III: Agency for Healthcare 

Research and Quality’s (AHRQ) Training 

Awards, as of September 30, 2017
Appendix IV: Office of the Assistant Secretary 

for Planning and Evaluation’s (ASPE) Projects 

to Build Data Capacity, as of September 30, 

2017

Page 32 GAO-18-311 Comparative Clinical Effectiveness Research Funding

Project title Description

Total 

obligations 

(dollars in 

millions)a

Complete

Creating the Foundational Blocks 

for the Learning Health Care 

System: Data Access Standards 

for Electronic Health Records

Developed technical standards for how health care providers, researchers, and the 

public health community access and extract data from electronic health records to 

conduct Patient-Centered Outcomes Research (PCOR).b

5.2

Creating the Foundational Blocks 

for the Learning Health Care 

System: Structured Data Capture

Identified and developed the functional and technical specifications necessary to 

enable electronic health record systems to retrieve, display, and fill a structured 

form or template and store and submit the completed form to an external repository. 

4.2

Maintenance and Support of 

Chronic Conditions Warehouse 

for Comparative Clinical 

Effectiveness Research (CER)

Provided researchers with access to the Centers for Medicare & Medicaid Services’ 

Chronic Conditions Warehouse, which contains Medicare and Medicaid beneficiary, 

claims, and assessment data, and supported infrastructure enhancements to 

conduct CER.

3.0

Expanding Data Collection for the 

National Program of Cancer 

Registries for CER

Longitudinal follow-up of certain cancer patients to assess vital statistics, disease 

recurrence, disease progression, and additional treatment types. Treatment data 

submitted each year to the Centers for Disease Control and Prevention and 

provided to researchers through the National Center for Health Statistics Research 

Data Center.

2.6

Strengthening and Expanding 

Community Health Applied 

Research Network Registry to 

PCOR

• Included clinical encounters for all patients and all conditions seen at the 

community health centers from 2006 to 2013 in the Community Health Applied 

Research Network Registry data warehouse, a research network comprising 

18 community health centers. A de-identified analytic file and associated data 

codebook were developed to support the use of analytic files by researchers 

outside of the network.

• Established a process for investigators to access the data warehouse through 

the development of a data access plan.

• Maintained the infrastructure for PCOR and for quality improvement in the 

safety net.

2.0

Development of Data 

Infrastructure for Use of Electronic 

Health Records in CER

Developed common data elements and standards for CER .The results were the 

initial entries into the National Institutes of Health’s National Library of Medicine 

common data element repository.

2.0

Strategic Opportunities for 

Building Data Infrastructure for 

CER

Developed a conceptual framework and environmental scan; produced policy 

documents ranging from patient-initiated data, through research data on care 

processes, transitions and coordination, to researcher access to claims data; and 

developed the ‘HHS Strategic Roadmap for Building Data Capacity for Clinical 

Comparative Effectiveness Research.’ 

1.5

CER Inventory The overall CER Inventory project was to design and implement a system for the 

categorization and cataloguing of CER activities through a web-based tool. Due to 

the rapidly evolving technologies supporting web-based search engines, and the 

improved methods for identification of more recent CER, the development of the 

CER Inventory (as a web-based search engine using a retrospective algorithm) was 

determined to have been superseded by existing search engine tools available. 

0.8

Appendix IV: Office of the Assistant 

Secretary for Planning and Evaluation’s 

(ASPE) Projects to Build Data Capacity, as 

of September 30, 2017
Appendix IV: Office of the Assistant Secretary 

for Planning and Evaluation’s (ASPE) Projects 

to Build Data Capacity, as of September 30, 

2017

Page 33 GAO-18-311 Comparative Clinical Effectiveness Research Funding

Project title Description

Total 

obligations 

(dollars in 

millions)a

Office of the Secretary PatientCentered Outcomes Research 

Trust Fund evaluation

Designed and conducted an independent evaluation of the ASPE portfolio to 

systematically assess progress related to the strategic framework functionalities. 

0.7

Multi-Payer Claims Database

Beta Test

Conducted CER analyses on the beta release of the Multi-Payer Claims Database 

and evaluated beta testers’ experiences requesting and using data from the MPCD 

for research. Results of the beta test found that the project was successful in 

achieving the key objectives of building a pilot database. 

0.2

Improving Beneficiary Access to 

Health Information “Blue Button” 

to Enable a ‘Data-as Service’ 

Platform

Planned for development and implementation of the Centers for Medicare &

Medicaid Services’ Blue Button—a service that allows patients to access their own 

health information in electronic form. 

0.2

In Progress

Enhancing Data Resources for 

Studying Patterns and Correlates 

of Mortality in PCOR

Linkage of data on fact, cause, and manner of death from the National Death Index 

to several federal population-based health data platforms in order to demonstrate 

the feasibility of such linkage, enable PCOR on patterns and correlates of mortality 

via the resulting linked data; and to facilitate collaboration between federal partners 

regarding strengthening the infrastructure and methods for linking healthcare data 

to mortality outcomes and using such linked data for PCOR.

6.6

Improving the Mortality Data 

Infrastructure for Patient-Centered 

Outcomes

Improve the infrastructure to support timely and complete mortality data collection 

through more timely delivery of state death records to the National Death Index 

database and by linking National Death Index database records with nationally 

collected hospital datasets to obtain a more complete picture of patient care.

6.5

Security and Privacy Standards 

for Patient Matching, Linking and 

Aggregation

Identify the best patient attributes to address the challenge of linking patients’ data 

across research, clinical, and claims data sets in order to support the PCOR data 

infrastructure that enables standardization and sharing of patient data across 

organizations. 

5.5

Developing a Strategically 

Coordinated Registry Network for 

Women’s Health Technologies

Create a coordinated registry network for women’s health technologies that will 

collect patient reported outcomes and employ structured data capture from 

electronic health records for data collection and exchange. 

5.1c

Harmonization of Various 

Common Data Models and Open 

Standards for Evidence 

Generation

Build data infrastructure for conducting PCOR using data from routine clinical 

settings. The sources of these data may include, but are not limited to, insurance 

billing claims, electronic health records, and patient registries. This project intends 

to harmonize several existing common data models, potentially including PCORnet 

and other networks.

4.1d

Advancing the Collection and Use 

of Patient-Reported Outcomes 

through Health Information 

Technology

Develop technical tools for collecting and integrating patient-reported outcome 

assessments into electronic health records or other health information technology 

products.

3.9e

Improving Beneficiary Access to 

Their Health Information through 

an Enhanced Blue Button Service

Create an interface that enables CMS beneficiaries to connect their 

MyMedicare.gov data to applications and services they trust, including research 

platforms related to research studies in which the beneficiary may be interested in 

participating.

3.8

Resource Center Provide technical assistance to the Trust Fund awardees in informatics and assist 

ASPE in setting up additional oversight processes and procedures to monitor 

progress.

3.5f
Appendix IV: Office of the Assistant Secretary 

for Planning and Evaluation’s (ASPE) Projects 

to Build Data Capacity, as of September 30, 

2017

Page 34 GAO-18-311 Comparative Clinical Effectiveness Research Funding

Project title Description

Total 

obligations 

(dollars in 

millions)a

PCOR: Privacy and Security 

Blueprint, Legal Analysis and 

Ethics Framework for Data Use, 

and Use of Technology for 

Privacy

Develop a privacy and security data infrastructure blueprint, legal analysis, and 

ethical framework to address legal and privacy and security related policy issues 

that affect the use of data for various types of PCOR.

3.4

Harmonization of Clinical Data 

Element Definitions for Outcome 

Measures in Registries

Convene clinical topic-specific working groups to discuss the data definitions 

currently in use and how these definitions can be harmonized to promote common 

definitions for outcome measures across systems. These common definitions are to 

be made publicly available to PCOR researchers and analysts.

3.1

Development of a Natural 

Language Processing Web 

Service for Public Health Use

Develop a natural language processing service that will be accessible and publicly 

available to researchers on the Public Health Community Platform – a cooperative 

platform for sharing interoperable technologies to address public health priority 

areas aimed at improving population health outcomes and health equity (e.g., 

tobacco use). 

3.0

Technologies for Donating 

Medicare Beneficiary Claims Data 

to Research Studies

Leverage the Sync for Science and Blue Button application programming interface 

programs to enable Medicare beneficiaries to donate their medical claims data for 

scientific research studies.

2.5g

Utilizing Data from Various Data 

Partners in a Distributed Manner

Develop and test the capability to conduct timely and secure distributed regression 

analysis in distributed data networks. Additionally, explore the feasibility of creating 

virtual linkage capabilities to utilize data from multiple data sources and data for one 

specific patient with information at different institutions.

2.5

Collection of Patient-Provided 

Information through a Mobile 

Device Application for Use in 

Comparative Effectiveness and 

Drug Safety Research

Create the infrastructure for collecting data from patients through a mobile device 

application, allowing patient-generated data to be linked with a single data partner 

that participates in the Food and Drug Administration’s Sentinel distributed network. 

The project will develop and pilot a mobile application to capture data from pregnant 

women who volunteer to participate. 

2.0

Conceptualizing a Data 

Infrastructure for the Capture and 

Use of Patient-Generated Health 

Data 

Develop a policy framework for the use of patient-generated data in research and 

care delivery that addresses data collection tools, data donation policies, regulatory 

gaps, combining data with medical record data, and interoperability of data across 

health information systems and devices.

2.1

Standardization and Querying of 

Data Quality Metrics and 

Characteristics for Electronic 

Health Data

Create and implement a metadata standard data capture and querying system for 

data quality and characteristics, data source and institutional characteristics, and 

“fitness for use.”

1.8

Cross-Network Directory Service Create an interoperable service that allows data partners to participate in multiple 

data research networks, query across the networks, and share analytic capabilities 

and knowledge across networks. The project will be piloted across two existing 

networks: Food and Drug Administration’s Sentinel and PCORnet.

1.5

Use of ADAPTABLE Trial to 

Strengthen Methods to Collect 

and Integrate Patient-Reported 

Information with Other Data Sets 

and Assess Its Validity

Generate tools and data standards that could be deployed in other CER studies by 

leveraging the infrastructure of an existing research study called the ADAPTABLE 

trial (Aspirin Dosing: A Patient-Centric Trial Assessing Benefits and Long Term 

Effectiveness). This trial is the first major randomized comparative effectiveness 

trial to be conducted by PCORnet.

1.1
Appendix IV: Office of the Assistant Secretary 

for Planning and Evaluation’s (ASPE) Projects 

to Build Data Capacity, as of September 30, 

2017

Page 35 GAO-18-311 Comparative Clinical Effectiveness Research Funding

Project title Description

Total 

obligations 

(dollars in 

millions)a

Creation of LOINC Equivalence 

Classes

Create a flexible, extensible, and computable mechanism for rolling data into 

clinically relevant equivalence groups that enable more efficient processing 

aggregation of laboratory data and other data from diverse health information 

technology systems. The primary focus of this work will be on laboratory tests. 

1.0

Source Data Capture from 

Electronic Health Records: Using 

Standardized Clinical Research 

Data

Create a single point data capture approach from the electronic health record to 

electronic data capture systems using the Retrieve Form for Data Capture 

standard. Stakeholders will be provided with a tool to seamlessly integrate 

electronic health record and electronic data capture systems.

0.7

Source: GAO analysis of ASPE documentation. | GAO-18-311

a

In addition to the projects listed, ASPE plans to obligate $2.0 million for one new project starting in 

fiscal year 2018.

b

PCOR is a form of CER.

c

ASPE plans to obligate an additional $0.4 million for this project in fiscal year 2018.

d

ASPE plans to obligate an additional $0.4 million for this project in fiscal year 2018.

e

ASPE plans to obligate an additional $0.1 million for this project in fiscal year 2018.

f

ASPE plans to obligate an additional $2.0 million for this project in fiscal year 2019.

g

ASPE plans to obligate an additional $1.4 million for this project in fiscal year 2018.
Appendix V: GAO Contact and Staff 

Acknowledgments

Page 36 GAO-18-311 Comparative Clinical Effectiveness Research Funding

John E. Dicken, (202) 512-7114 or dickenj@gao.gov

In addition to the contact named above, Karin Wallestad, Assistant 

Director; Michael Zose, Analyst-in-Charge; Kye Briesath; Laurie Pachter; 

Vikki Porter, and Jennifer Whitworth made key contributions to this report.

Appendix V: GAO Contact and Staff 

Acknowledgments

GAO Contact

Staff 

Acknowledgments

(102064)
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