REGENERATIVE 

MEDICINE

Federal Investment, 

Information Sharing, 

and Challenges in an 

Evolving Field

Report to Congressional Requesters

June 2015

GAO-15-553

United States Government Accountability Office
 

United States Government Accountability Office

 

Highlights of GAO-15-553, a report to 

congressional requesters

June 2015

REGENERATIVE MEDICINE

Federal Investment, Information Sharing, and 

Challenges in an Evolving Field

Why GAO Did This Study

Regenerative medicine is an 

interdisciplinary field with a focus on 

conducting research and developing 

treatments for a vast assortment of 

previously untreatable diseases and 

conditions through self-healing—a 

process by which the body uses its 

own systems to recreate cells and 

rebuild tissues and organs.

GAO was asked to review the federal 

involvement in this field. This report 

describes (1) which federal agencies 

conducted or funded regenerative 

medicine research in recent years and 

how these agencies invested their 

resources; (2) the activities these 

federal agencies undertake to share 

information across agencies; and 

(3) the challenges to advancing the 

field of regenerative medicine identified 

by federal agencies and other 

stakeholders and the steps taken, if 

any, to address them.

GAO analyzed funding data from 

seven federal agencies active in 

regenerative medicine research in 

fiscal years 2012 through 2014, the 3 

most recent years for which full funding 

data were available; reviewed agency 

documents, including reports and 

strategic plans, and an interagency 

working group’s meeting agendas and 

minutes. GAO also interviewed officials 

from the seven agencies, as well as 

nonfederal stakeholders, representing 

academic and state-funded research 

institutions, patient advocacy groups, 

and trade organizations.

In commenting on a draft of this report, 

the federal agencies that conduct, 

fund, or otherwise play a role in 

regenerative medicine research 

provided technical comments, which 

GAO incorporated where appropriate.

What GAO Found

Seven federal agencies invested—that is, conducted or funded—approximately 

$2.89 billion in regenerative medicine research in fiscal years 2012 through 2014. 

Most (88 percent) was invested by the National Institutes of Health. Agencies

funded research related to their missions, including basic research to enhance 

general scientific knowledge, clinical research to move scientific discoveries into 

practical applications, and research to develop regulatory science.

Federal Funding for Regenerative Medicine Research, Fiscal Years 2012 through 2014

These agencies have established mechanisms for sharing information on the 

wide array of research they fund. These mechanisms include participating in 

regular meetings of an interagency working group, cofunding research, and 

cosponsoring workshops. Although some nonfederal stakeholders expressed a 

desire for greater coordination of federal regenerative medicine activities, agency 

officials reported that the current approach to sharing information is appropriate 

given the diverse missions of the agencies involved, the broad range of research 

conducted in this field, and the variety of diseases and conditions that may 

benefit from new discoveries.

Agency officials and nonfederal stakeholders have identified a variety of 

challenges in advancing regenerative medicine. These include establishing 

effective collaborations between federal and nonfederal stakeholders; recruiting 

scientists versed in regenerative medicine to become federal employees; 

navigating the regulatory review and product approval process; and making 

decisions about Medicare coverage and reimbursement rates, mechanisms, and 

processes for newly approved products. Some steps have been taken to address 

these challenges, including recruiting postdoctoral fellows and providing training 

for them specifically to build the needed mix of interdisciplinary skills for research 

in regenerative medicine to address workforce challenges.

View GAO-15-553. For more information, 

contact Marcia Crosse at (202) 512-7114 or 

crossem@gao.gov.
Page i GAO-15-553 Regenerative Medicine

Letter 1

Background 3

Seven Federal Agencies Conducted or Funded a Range of 

Regenerative Medicine Research 6

Agencies Have Established Mechanisms for Sharing Information 

on Their Diverse Investments in Regenerative Medicine 

Research 14

Agency Officials and Other Stakeholders Have Identified 

Challenges in Advancing the Field of Regenerative Medicine 

and Have Taken Some Steps to Address Them 17

Agency Comments 23

Appendix I Funding for Regenerative Medicine Research by National Institutes 

of Health (NIH) by Institute, Center, or Program 26

Appendix II GAO Contact and Staff Acknowledgments 29

Tables

Table 1: Federal Agencies That Conducted or Funded 

Regenerative Medicine Research, Fiscal Years 2012 

through 2014 7

Table 2: Funding for Regenerative Medicine Research by the 

National Institutes of Health (NIH) by Individual Institute, 

Center, or Program, Fiscal Years 2012-2014 26

Figures

Figure 1: Federal Funding for Regenerative Medicine Research, 

Fiscal Years 2012 through 2014 8

Figure 2: A Colony of Human Pluripotent Stem Cells That Could 

Be Used to Create Engineered Heart Muscle Cells 10

Figure 3: A Mannequin’s Burned Forearm Being Used in DODFunded Armed Forces Institute of Regenerative Medicine 

Research to Develop Skin-Cell Printing Techniques for 

Burn Victims 12

Contents
Page ii GAO-15-553 Regenerative Medicine

Abbreviations

AFIRM Armed Forces Institute of Regenerative Medicine

CMS Centers for Medicare & Medicaid Services

DOD Department of Defense

FDA Food and Drug Administration

HHS Department of Health and Human Services

MATES Multi-Agency Tissue Engineering Sciences Interagency 

 Working Group

NIH National Institutes of Health

NIST National Institute of Standards and Technology

NNSA National Nuclear Security Administration

NSF National Science Foundation

VA Department of Veterans Affairs

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

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Page 1 GAO-15-553 Regenerative Medicine

441 G St. N.W.

Washington, DC 20548

June 23, 2015

Congressional Requesters

Regenerative medicine is an interdisciplinary field with a focus on 

conducting research and developing treatments for a wide range of 

previously untreatable diseases and conditions through self-healing—a 

process by which the body uses its own systems to reprogram cells and 

rebuild tissues and organs. Regenerative medicine has been proposed as 

an approach to treat a vast array of diseases and conditions, including 

diabetes, heart disease, renal failure, Parkinson’s disease, osteoporosis, 

limb loss, burn damage, and spinal cord injuries. Virtually any disease 

that results from malfunctioning, damaged, or failing tissues may be 

potentially cured through regenerative medicine treatments. While there 

are some regenerative medicine treatments that are currently being 

used—such as tissue-engineered products used to heal wounds and to 

induce bone and connective tissue growth and replace damaged 

cartilage—this area of science may yield many more products in the 

future.

The federal government has had a long-standing interest in regenerative 

medicine. In 2000, the White House Office of Science and Technology 

Policy established the Multi-Agency Tissue Engineering Sciences 

Interagency Working Group (MATES)—a forum for member agencies to 

exchange information and collaborate on issues related to tissue 

engineering and regenerative medicine. In 2005, the Department of 

Health and Human Services (HHS) published 2020: A New Vision—A 

Future for Regenerative Medicine, which encouraged the federal 

government to play a direct role in advancing regenerative medicine. In 

2007, in part to address the HHS report, MATES issued a multiagency 

strategic plan outlining opportunities for federal agencies to advance the 

field of regenerative medicine.1

You asked us to study current federal activities that impact the field of 

regenerative medicine. This report focuses on these activities and 

 

1

Multi-Agency Tissue Engineering Sciences Interagency Working Group, Advancing 

Tissue Science and Engineering: A Foundation for the Future and A Multi-Agency 

Strategic Plan (Washington, D.C.: June 2007).

Letter
Page 2 GAO-15-553 Regenerative Medicine

describes (1) which federal agencies conducted or funded regenerative 

medicine research in recent years and how these agencies invested their 

resources; (2) the activities these federal agencies undertake to share 

information across agencies; and (3) the challenges to advancing the field 

of regenerative medicine identified by federal agencies and other 

stakeholders, and the steps taken, if any, to address them.

To describe which federal agencies conducted or funded regenerative 

medicine research and how these agencies invested their resources, we 

reviewed federal documentation on regenerative medicine, such as the 

MATES website and strategic plan and the HHS report. We contacted all 

federal agencies that were listed on the MATES website to determine if 

they had conducted or funded regenerative medicine research from fiscal 

years 2012 through 2014, the 3 most recent years for which full funding 

data were available. For the agencies that had conducted or funded 

regenerative medicine research during this time frame, we interviewed 

agency officials and reviewed relevant agency reports to determine their 

activities in regenerative medicine. We also analyzed funding data for 

regenerative medicine research during this period, which agencies 

provided to us or we obtained from publicly available databases. We used 

a standard definition of regenerative medicine—to include research on 

tissue engineering, stem cells, and cell and gene therapies—which we 

discussed with each federal agency. We assessed the reliability of data 

from each agency by reviewing related documentation, performing data 

reliability checks (such as examining the data for missing values), and 

interviewing agency officials. After taking these steps, we determined that 

the data we used were sufficiently reliable for our purposes.

To describe the activities federal agencies that have conducted or funded 

regenerative medicine research undertake to share information across 

agencies, we reviewed reports and other relevant documentation from 

these agencies. We also reviewed the strategic plan, agendas, and 

meeting minutes for MATES from fiscal years 2012 through 2014 and 

interviewed agency officials about their information sharing activities. 

Evaluating the effectiveness of these information sharing activities was 

not within the scope of our review.

To describe the challenges that federal agencies and other stakeholders 

in regenerative medicine research have identified and what steps, if any, 

have been taken to address them, we interviewed agency officials from 

those agencies that conducted or funded regenerative medicine research 

in fiscal years 2012 through 2014. We also sought to obtain input from a 

variety of knowledgeable nonfederal stakeholders, including those 
Page 3 GAO-15-553 Regenerative Medicine

representing academic and state funded or supported research 

institutions, such as the California Institute of Regenerative Medicine, 

University of Washington Institute for Stem Cells and Regenerative 

Medicine, and Wake Forest Institute for Regenerative Medicine. We also 

interviewed representatives from patient advocacy groups, such as the 

Genetics Policy Institute and the Parkinson’s Action Network, and trade 

organizations—the Alliance for Regenerative Medicine and the 

Regenerative Medicine Foundation. We identified these nonfederal 

stakeholders from a literature review and by asking the nonfederal 

stakeholders we interviewed to provide referrals to others knowledgeable 

about regenerative medicine.

We conducted this performance audit from July 2014 to June 2015 in 

accordance with generally accepted government auditing standards. 

Those standards require that we plan and perform the audit to obtain 

sufficient, appropriate evidence to provide a reasonable basis for our 

findings and conclusions based on our audit objectives. We believe that 

the evidence obtained provides a reasonable basis for our findings and 

conclusions based on our audit objectives.

Regenerative medicine offers the possibility of new and highly 

individualized treatments for a variety of diseases and conditions; 

scientists believe that the promise of regenerative medicine is to provide 

cures for virtually any disease that results from malfunctioning, damaged, 

or failing tissues. Regenerative medicine incorporates self-healing—

where the body uses its own systems, sometimes with help from added 

biological material from outside the body—to reprogram cells or rebuild 

organs. Scientists are working in the field of regenerative medicine to 

develop products that could help manage many pressing problems in 

health care. For example, a shortage of organ donors means many 

people die while waiting for an organ transplant; however, scientists are 

pursuing ways for organs to be restored by using therapies to regenerate 

the cells of the organ itself or applying tissue engineering strategies to 

grow a replacement organ outside of the body for transplantation. 

Similarly, the steady increase of diabetes in the population could result in 

more patients seeking treatment for complications from that disease. 

Scientists are exploring techniques to foster regeneration of pancreatic 

cells, which could help address the cause of diabetes—inadequate insulin 

secretion. Discoveries from regenerative medicine research might be 

used to treat patients of all ages. For example, work done with 

engineered heart cells could lead to discoveries for pediatric patients who 

suffer from congenital heart defects, as well as adult patients with 

Background
Page 4 GAO-15-553 Regenerative Medicine

damage caused by heart disease. Regenerative medicine might also lead 

to treatments for people with limb loss or damage and severe burns. 

Scientists are working to find ways to improve bone restoration to reduce 

the pain, bleeding, and scars created through current bone harvesting 

techniques, as well as developing a system to decrease the amount of

skin that would need to be harvested to treat a burn patient.

In order to develop products for a broad range of conditions and 

diseases, regenerative medicine brings together a wide variety of 

scientific disciplines—including biology, chemistry, engineering, and 

physics—to advance knowledge and conceive new clinical applications. 

Regenerative medicine research encompasses the use of the following:

• Tissue engineering. This includes the practice of combining cells, 

biologically active molecules, and scaffolds—which support the tissue 

growth—into functional tissues. The goal of tissue engineering is to 

restore, maintain, or improve damaged tissues or whole organs. 

Scientists may develop tissues using a patient’s own cells, which can 

reduce the possibility of infection or tissue rejection. According to the 

National Institutes of Health (NIH), an agency within HHS, tissue 

engineering currently plays a relatively small role in patient treatment, 

although bladders, small arteries, skin grafts, cartilage, and even a full 

trachea have been implanted in patients. Work in the field of tissue 

engineering has resulted in skin and cartilage products, which contain 

cells grown in tissue cultures outside of the patient, that have been 

approved by the Food and Drug Administration (FDA), another 

agency within HHS. 

• Stem cells. Cells are the building blocks of regenerative medicine 

research. Scientists must first understand and control the cells that 

make up tissues in the body in order to build tissues. Stem cell 

research can contribute to the understanding of how cells form 

different tissues. Stem cells are unique in that they are pluripotent—

they can self-renew or be developed into many different cell types in 

the body, such as a muscle cell, a red blood cell, or a brain cell. 

According to NIH, there are many ways to categorize stem cells. One 

system, which takes into account the source of the original cell, 

divides stem cells into two kinds: embryonic stem cells— which have 

the ability to form potentially any cell type that makes up the body—

and nonembryonic “somatic” or “adult” stem cells—which are found 

within existing tissues and organs and can form some or all of the cell 
Page 5 GAO-15-553 Regenerative Medicine

types to maintain and repair the tissue or organ in which they are 

found.2

• Gene or cell therapies. These therapies are considered regenerative 

medicine products and can result from stem cell research. Gene 

therapy and cell therapy aim to repair the direct cause of genetic 

diseases in the DNA or cells by modifying individual genes or cell 

populations for the treatment of a disease. FDA has approved some 

cell therapies for use, such as one to repair cartilage damage, through 

cell biopsy and reimplantation.

Scientists also work with induced pluripotent stem cells—adult 

cells that have been genetically reprogrammed to an embryonic stem 

cell–like state.

3

Given the diverse scientific disciplines involved in this research and the 

array of diseases and conditions it may impact, stakeholders must employ 

multiple approaches for developing regenerative medicine products. For 

example, these approaches include using 3D printers to create 

experimental muscle tissue for reconstructive surgery or to create a skin

equivalent that can be used for burn wounds; infusing cells from donated 

human cord blood to treat patients with blood disorders; and developing 

composite bone scaffolds—structures to support bone regeneration. 

There is a consensus that regenerative medicine may be defined as the 

development of products to repair or replace human cells, tissue, or organ 

function lost due to age, disease, damage, or congenital defects.

 

2

There have been limitations on the use of federal funds for research on human 

embryonic stem cells since 1996. Over time, some of these limitations have changed. 

Most recently, in 2009, in response to an Executive Order, NIH issued new guidelines for 

conducting embryonic stem cell research and subsequently created a registry of human 

embryonic stem cell lines that are eligible for use in federally funded research. Exec. 

Order No. 13,505. 74 Fed. Reg. 10667 (Mar. 9, 2009).

3

Drugs, medical devices, and biologics are subject to a risk-based regulatory approach 

before they can be marketed in the United States. The level of FDA regulatory oversight is 

dependent on the characteristics (chemical and physical makeup) of the product and the 

intended use of the product. For many regenerative medicine products, the level of 

oversight includes premarket review to support clearance, approval, or licensure prior to 

marketing.
Page 6 GAO-15-553 Regenerative Medicine

We identified seven federal agencies that conducted or funded 

regenerative medicine research in fiscal years 2012 through 2014. In 

addition to FDA and NIH, the Department of Commerce’s National 

Institute of Standards and Technology (NIST), the Department of Defense 

(DOD), the Department of Energy’s National Nuclear Security 

Administration (NNSA), the Department of Veterans Affairs (VA), and the 

National Science Foundation (NSF) conducted research on regenerative 

medicine during this time period.

Regenerative medicine research conducted or funded by federal 

agencies falls into several categories. Some agencies conduct basic 

research, which is designed to provide general scientific knowledge that 

may provide a foundation for other research related to their unique 

missions. One such example is developing new techniques to preserve 

organs and large tissues. Some conduct translational research to move 

laboratory-generated research into practical applications in humans and 

the development of best practices. Some agencies also fund clinical 

research, such as testing a new treatment of amyotropic lateral sclerosis, 

also known as Lou Gehrig’s disease. In addition, certain agencies 

perform research focused on a specific population, such as research to 

develop treatments for wounded military personnel. Finally, some 

agencies conduct research to advance regulatory science and to develop 

consensus and measurement standards, for example improving 

measurements of certain cell characteristics.4

It is important to note that while agencies focus on research related to 

their individual missions, they may conduct research in more than one 

category. For example, NIH funds some research that could be 

considered basic research in addition to its translational and clinical 

research. Table 1 summarizes information regarding the efforts of the 

seven federal agencies that conducted or funded regenerative medicine 

research in fiscal years 2012 through 2014.

 

4

Consensus standards are voluntary documentary standards developed by interested 

stakeholders, such as industry, government, and academia, using a consensus-based 

process; measurement standards ensure reliability and repeatability of measurements. 

The Office of Management and Budget establishes policies on the federal use and 

development of voluntary consensus standards. See Office of Management and Budget, 

Federal Participation in the Development and Use of Voluntary Consensus Standards and 

in Conformity Assessment Activities, Circular A-119 Revised (Washington, D.C.: Feb. 10, 

1998).

Seven Federal 

Agencies Conducted 

or Funded a Range of 

Regenerative 

Medicine Research
Page 7 GAO-15-553 Regenerative Medicine

Table 1: Federal Agencies That Conducted or Funded Regenerative Medicine Research, Fiscal Years 2012 through 2014

Federal agency Agency mission Regenerative medicine research conducted includes

Department of Defense Provide the military forces needed to deter 

war and to protect the security of the 

country

Research and applications for active-duty personnel 

including limb repair, traumatic brain injury, and 

battlefield injuries

Department of Veterans 

Affairs 

Provide veterans and eligible beneficiaries 

with benefits and services

Research and applications for the veteran population, 

including limb repair, traumatic brain injury, and care for 

wounded warriors, as well as for stroke, glaucoma, and 

other conditions for an aging veteran population

Food and Drug Administration 

within the Department of 

Health and Human Services

Protect the public health by ensuring the 

safety, efficacy, and security of human 

drugs, biological products, medical devices, 

food supply, cosmetics, and products that 

emit radiation

Safety and effectiveness research related to regulation of 

regenerative medicine products and standards 

development

National Institute of 

Standards and Technology 

within the Department of 

Commerce

Promote innovation and industrial 

competitiveness by advancing 

measurement science, standards, and 

technology

Measurement science and development of consensus 

documentary standards and standard reference 

materials to facilitate commercialization of regenerative 

medicine productsa

National Institutes of Health 

within the Department of 

Health and Human Services

Seek knowledge about the nature and 

behavior of living systems and the 

application of that knowledge to enhance 

health, lengthen life, and reduce illness and 

disability

Biomedical applications and basic, clinical, and 

translational research, including stem cells and tissue 

engineering

National Nuclear Security 

Administration within the 

Department of Energy

Enhance national security through the 

military application of nuclear science; 

responsible for issues of nuclear defense, 

nonproliferation, and naval research

Research and development, including basic research 

and experiments designed to determine the utility of new 

scientific ideas, technical concepts, or devices

National Science Foundation Promote and advance fundamental 

scientific progress

Basic research with a focus on expanding current 

scientific knowledge

Source: GAO analysis of agency interviews and information. | GAO-15-553

a

Consensus standards are voluntary documentary standards developed by interested stakeholders, 

such as industry, government, and academia, using a consensus-based process; measurement 

standards ensure reliability and repeatability of measurements.

Federal funding for regenerative medicine varies considerably among 

these seven federal agencies. They have invested a total of 

approximately $2.89 billion in regenerative medicine research from fiscal 

years 2012 through 2014, 88 percent of which was invested by NIH, as 
Page 8 GAO-15-553 Regenerative Medicine

shown in figure 1. The agencies’ funding ranged from $2.39 million 

funded by NIST to $2.54 billion funded by NIH during this period.5

Figure 1: Federal Funding for Regenerative Medicine Research, Fiscal Years 2012 through 2014

Note: For purposes of this report, we use the term “funding” to refer to obligations. An obligation is a 

definite commitment that creates a legal liability of the federal government for payment of goods and 

services ordered or received. A federal agency incurs an obligation, for example, when it awards a 

grant.

 

5

NSF collects and makes publicly available its research funding data; however, the 

agency does not routinely categorize its awards and projects as regenerative medicine 

research. This information can be obtained through a search of award abstracts on its 

publicly-available database of research projects. To generate a list of NSF regenerative 

medicine awards, we searched this database and asked NSF to review our findings. In 

some instances, we identified research efforts that included applications relevant to 

regenerative medicine, even though this may not have been the purpose of the study. 

NSF officials did not always agree that a project should be categorized as related to 

regenerative medicine. They emphasized that the agency’s mission is to conduct basic 

research, not research for a particular disease, condition, or treatment type. However, 

based on our definition of regenerative medicine research, we included these research 

efforts in the total reflected for NSF.
Page 9 GAO-15-553 Regenerative Medicine

NIH, which funds basic, translational, and clinical research, made the 

largest investment in regenerative medicine during this period. Its 

$2.54 billion resulted in about 2,400 awards per year from fiscal years 

2012 through 2014. Over 80 percent of NIH’s overall research funding 

goes to extramural research, which supports scientists and research 

personnel working at universities, medical schools, and other research 

institutions; however, it also funds intramural research—that is, research 

performed by NIH scientists in NIH laboratories. Within NIH, 22 of its 27 

institutes and centers, which are focused on particular diseases, 

conditions, or research approaches, funded some regenerative medicine 

research, demonstrating the breadth of the field. In addition, funding for 

regenerative medicine research came from the Office of the Director and 

the NIH Common Fund.

6

 

6

The Office of the Director is responsible for coordinating some programs and activities 

that span NIH components, particularly research initiatives and issues involving more than 

one of the institutes or centers. The NIH Common Fund, formerly the NIH Roadmap for 

Medical Research, is a series of initiatives designed to speed the movement of scientific 

discoveries. According to NIH, it provides a framework of the priorities the NIH must 

address in order to optimize its entire research portfolio and lays out a vision for a more 

efficient and productive system of medical research.

The single biggest investment in NIH’s 

regenerative medicine research was from its National Heart, Lung, and 

Blood Institute, which is responsible for about 24 percent of NIH’s 

regenerative medicine funding in this time period. One example of 

research funded by this institute is a project that tests the effectiveness of 

a new technique for developing engineered heart cells for cardiac 

regeneration and restoration of vascular function. Figure 2 shows a 

colony of pluripotent stem cells that could be used for the development of 

engineered heart cells. Following the National Heart, Lung, and Blood 

Institute, the next largest investments in regenerative medicine research 

from NIH came from the National Institute of Diabetes and Digestive and 

Kidney Diseases, the National Cancer Institute, and the National Institute 

of Neurological Disorders and Stroke, having each funded over 

$200 million in research for fiscal years 2012 through 2014. NIH’s 

investments by these institutes further illustrate the broad range of 

conditions and diseases being studied within the field of regenerative 

medicine. Examples of the research conducted by these institutes include 

work to develop transplantable liver grafts for curing or treating liver 

dysfunction and failure, develop novel strategies to treat a variety of 

cancers through stem cell transplantation, and determine the extent to 

which cell grafts can improve recovery in stroke patients. (See app. I for 
Page 10 GAO-15-553 Regenerative Medicine

more details on NIH’s funding for regenerative medicine by institute or 

center from fiscal year 2012 through fiscal year 2014.)

Figure 2: A Colony of Human Pluripotent Stem Cells That Could Be Used to Create 

Engineered Heart Muscle Cells

Note: The colony of cells was produced through the National Institutes of Health-funded research at 

the University of Washington Institute for Stem Cell and Regenerative Medicine.

DOD is the second largest funder of regenerative medicine research, 

and its investment in regenerative medicine research totaled almost 

$253 million, or about 9 percent of the total federal investment in 

regenerative medicine, in fiscal years 2012 through 2014.7

 

7

Although DOD’s research efforts are led by individual components within the department, 

all of its research is focused on active-duty personnel. As a result, we have chosen to 

report DOD activities at the department level.

DOD funded 

approximately 178 projects focused on the health needs of active-duty 

military personnel. Within DOD, the Defense Health Program is the 

largest funder of regenerative medicine research, having invested more 
Page 11 GAO-15-553 Regenerative Medicine

than $120 million in fiscal years 2012 through 2014.8 The Defense Health 

Program funds a variety of projects, including research on regeneration of 

cochlear hair cells—damaged through loud noises and blasts—to aid in 

hearing restoration. After the Defense Health Program, the Office of 

Naval Research and the U.S. Army are the next largest funders within 

DOD, each having invested over $42 million. During fiscal years 2012 

through 2014, the U.S. Army, the Defense Health Program, and the Office 

of Naval Research invested in the Armed Forces Institute of Regenerative 

Medicine (AFIRM).9 AFIRM in turn awarded $42.66 million in funding for 

regenerative medicine research at U.S. academic institutions and private 

companies during this same time period. AFIRM currently supports 

research on topics related to developing advanced treatment options for 

severely wounded warriors.10

 

8

The Research, Development, and Acquisition Directorate in the Defense Health Agency 

provides oversight of Defense Health Program funding and focuses on, among other 

things, advancing medical research and development for wounded warriors, expediting 

the delivery of products and solutions to servicemembers and their families, and 

advancing the state of medical science in areas of the most pressing need.

An example of research funded by AFIRM 

is work focused on developing approaches to improve burn treatment 

(see fig. 3).

9

AFIRM is a multi-institutional, interdisciplinary network—including U.S. Army; U.S. Navy, 

Office of Naval Research; U.S. Air Force; NIH; VA; and DOD—working to develop 

advanced treatment options for wounded servicemembers.

10AFIRM’s research currently focuses on five specific areas: (1) extremity injury treatment 

to decrease the need for amputation; (2) craniomaxillofacial reconstructions to help 

servicemembers who have experienced massive bone and tissue loss to the face and 

head; (3) skin injury treatment to improve burn and wound healing; (4) vascular composite 

tissue allotransplantation and immunomodulation to improve and preserve the functionality 

and sustainability of hand, arm, and face transplants; and (5) repair and reconstruction 

needed to treat certain genitourinary and lower abdominal injuries caused by explosions.
Page 12 GAO-15-553 Regenerative Medicine

Figure 3: A Mannequin’s Burned Forearm Being Used in DOD-Funded Armed 

Forces Institute of Regenerative Medicine Research to Develop Skin-Cell Printing 

Techniques for Burn Victims

VA, the third largest funder of federal regenerative medicine research, 

funded 94 regenerative medicine projects in fiscal years 2012 through 

2014. According to VA, all of the research is awarded through a 

competitive process and takes place within the VA health care system, 

funding practicing clinicians who are aware of the research needs that will 

improve veterans’ health. VA’s average annual award amount was 

around $225,000 and was as much as $1.66 million during this time 

period. Among the projects VA funded was a study to develop a minimally 

invasive therapy that could normalize disc function in the lower back while 

promoting tissue regeneration using stem cells. The results of this study 

could impact both wounded warriors and aging veterans suffering from 

chronic lower back pain. Another VA research project is evaluating 

whether replacement of damaged cells in the eyes with induced 

pluripotent stem cells could help preserve vision for patients suffering 

from glaucoma. VA has also conducted research to reprogram blood cells 

into stem cells and other types of cells to rebuild injured bones and 

regenerate organs.

The remaining federal agencies—NSF, NNSA, FDA, and NIST—

combined make up less than 2 percent of the federal investment in 

regenerative medicine research in fiscal years 2012 through 2014. NSF 
Page 13 GAO-15-553 Regenerative Medicine

and NNSA focus on basic research, while FDA focuses on both 

regulatory research and consensus standards development and NIST on 

standards development. 

• NSF, which supports extramural research, funded 107 projects, 

totaling approximately $40.23 million, which includes basic scientific 

research that may have applications beyond regenerative medicine. 

One example of NSF-funded research includes a project designed to 

improve efficacy of stem cell transplantation into the brain for tissue 

regeneration.

• NNSA, which also conducts basic scientific research, undertook one 

project, totaling about $4.81 million, and examined increasing the 

biocompatibility of polymers, which could be used as implantable 

sensors in some regenerative medicine applications, to decrease 

rejection of these polymers from the body.11

• FDA scientists conduct the majority of the regenerative medicine 

research the agency funds, and it has invested about $8.63 million to 

help advance regulatory science for regenerative medicine products. 

For example, one such project is to develop improved methods for 

evaluating experimental cell-based products to reliably predict product 

performance.

• NIST conducts research to advance measurement science and 

standards, and it invested about $2.39 million for 8 regenerative 

medicine projects to advance the field. One example of NIST’s work is 

developing statistical tests that will allow determination of the 

accuracy of cell counts for the development of cell therapies.

 

11NNSA officials told us the agency does not plan to conduct additional regenerative 

medicine research in the foreseeable future.
Page 14 GAO-15-553 Regenerative Medicine

Agencies that have conducted or funded regenerative medicine research 

have established mechanisms for sharing information across agencies on 

the wide range of research they undertake. These information-sharing 

mechanisms include regular meetings of MATES and more collaborative 

ad hoc activities such as cofunding research and cosponsoring 

workshops related to regenerative medicine.12 Given the broad range of 

disciplines and research missions represented by the agencies that are 

members in MATES, monthly meetings provide a forum for participants to 

stay informed about regenerative medicine research and other activities 

undertaken by member agencies. Six of the seven federal agencies with

investments in regenerative medicine research from fiscal years 2012 

through 2014 are current or former members of MATES: NIH, FDA, DOD, 

VA, NSF, and NIST.13 According to meeting minutes from fiscal years 

2012 through 2014, discussion topics included presentations on research 

conducted by MATES member agencies, updates from scientific 

conferences and meetings attended by MATES members, ideas for 

updating the 2007 MATES strategic plan,14

One recent activity undertaken by MATES was a joint workshop its 

members coordinated as part of an international tissue engineering and 

regenerative medicine conference held in December 2014 in Washington, 

D.C. Officials noted that the goal of the event was to show how the 

various federal agencies supported a range of regenerative medicine 

activities consistent with their individual missions. Officials from MATES 

member agencies NSF, NIH, DOD, NIST, and FDA presented information 

on the regenerative medicine research conducted or funded by their 

agencies, demonstrating the diversity of approaches taken by each. An 

and updates from attendees 

on current and planned regenerative medicine research.

 

12MATES is currently chaired by a representative from FDA. According to officials, the 

position of chair rotates among member agencies.

13Officials from NNSA told us that they do not consider their agency to be a member of 

MATES, although the Department of Energy remains on the membership roster on the 

MATES website. In addition, the Environmental Protection Agency, National Aeronautics 

and Space Administration, Department of Agriculture, and Centers for Medicare & 

Medicaid Services remain on the membership roster on the MATES website. However, 

these agencies are not currently members of MATES nor did they conduct or fund 

regenerative medicine research during fiscal years 2012 through 2014.

14Advancing Tissue Science and Engineering. As of April 2015, MATES officials told us 

the effort to update the plan was underway, but no draft or interim documentation on this 

effort was available for our analysis.

Agencies Have 

Established 

Mechanisms for 

Sharing Information 

on Their Diverse 

Investments in

Regenerative 

Medicine Research
Page 15 GAO-15-553 Regenerative Medicine

official from NSF emphasized the agency’s basic research—funding novel 

ideas for eventual application in regenerative medicine such as 

fundamental studies on cells and disease modeling. An NIH official 

presented work the agency has funded to develop clinical trials for stem 

cell therapies, emphasizing its role in translational research. An official 

from DOD’s AFIRM discussed the primary goals for the agency’s 

investments in research focused on applications for its specific 

population—military personnel—such as tissue salvage, restoration, and 

regeneration. The NIST presentation at the workshop focused on that 

agency’s role in performing research in measurement science for 

advancing technology by bringing partners together to develop standards, 

including measurement standards related to regenerative medicine. The 

presentation communicated results from NIST’s partnership with a private 

company to develop methods for improving the measurement confidence 

of cell counting—a key cell attribute for research and development and 

quality control in bioprocessing—to facilitate product development.

Participation in MATES is voluntary and agency representatives attend as 

their interest and availability allows. A review of available MATES meeting 

minutes from fiscal years 2012 through 2014 shows regular attendance 

by officials from some member agencies, with less regular attendance by 

other participating agencies. Specifically, of the 29 meetings for which 

minutes were archived over the 3-year period, officials from NIH and FDA 

attended all 29. In addition, officials from NIST, DOD, NSF, and VA 

attended 21, 11, 7, and 3 meetings, respectively. Officials from agencies 

who attend the meetings regularly told us they believe this venue is a 

highly effective way to share information with colleagues across agencies.

In addition to participation in MATES, agencies engage in a range of ad 

hoc activities for sharing information and collaborating with one another in 

the area of regenerative medicine such as cofunding research, serving as 

advisors to each other on certain projects, and cosponsoring workshops 

related to regenerative medicine. For example, in 2006, three agencies—

NIH, NSF, and NIST—issued the joint funding announcement, “Enabling 

Technologies for Tissue Engineering and Regenerative Medicine,” which 

resulted in a diverse set of funded applications, including one for the 

development of an artificial liver in mice and technology to extend liver 

preservation. NIH and VA have cofunded research projects with DOD’s 

AFIRM and also serve on advisory committees for its programs including 

on traumatic brain injury and spinal cord injury. Another example of 

interagency advising for regenerative medicine projects is DOD’s and 

VA’s participation as ex-officio members of NIH’s National Institute of 

Neurological Disorders and Stroke Advisory Council, which oversees all 
Page 16 GAO-15-553 Regenerative Medicine

of that institute’s activities, including regenerative medicine research. In 

addition, there are numerous examples of agencies collaborating on 

jointly held regenerative medicine workshops such as the 2012 Functional 

Imaging for Regenerative Medicine workshop held by NIST, in 

conjunction with MATES, to identify imaging needs and challenges in 

tissue engineering and regenerative medicine and FDA and NIH jointly 

held workshops in 2011 and 2012 on stem cell research, product 

development, and regulatory issues.

Officials from the agencies that conduct or fund regenerative medicine 

research reported that the current methods in place for sharing 

information across agencies are appropriate given the diverse missions of 

the agencies involved, the broad range and type of research conducted 

and funded in this field, and the variety of diseases and conditions that 

may benefit from new discoveries. Some officials reported that there are 

benefits to the federal government’s decentralized approach to not only 

sharing information, but also to conducting and funding this research. For 

example, some officials we spoke with said the decentralized nature of 

regenerative medicine activities generates diversity of thought in 

regenerative medicine studies and allows agencies the flexibility to make 

their own plans and priorities for advancing the field according to each 

agency’s mission. In addition, it allows the research and science to 

progress on multiple fronts simultaneously, according to each agency’s 

expertise. Further, some officials told us that a more formalized approach 

to information sharing and collaboration across agencies that added 

bureaucracy—such as a requirement to have certain information cleared 

through formal channels which may slow communication—could inhibit 

the flow of information rather than enhance it.

In contrast to the current approach to information sharing and 

coordination across federal agencies, some nonfederal stakeholders we 

spoke with expressed a desire for greater coordination of federal 

regenerative medicine activities with the hope that more focused attention 

in this area could attract more resources and faster advancement in the 

field. For example, an official from one nonfederal stakeholder group 

suggested that a statement of commitment from the federal government 

to a given set of regenerative medicine priorities could help to invigorate 

the field and encourage greater support for the research. An official from 

another nonfederal stakeholder group told us that the organization 

encourages a national strategy for regenerative medicine research as a 

means to enhance information sharing across agencies, ensure the 

research funding allocated to this field is used wisely, speed the 

development of new regenerative medicine products, and keep the United 
Page 17 GAO-15-553 Regenerative Medicine

States at the forefront of innovations in this field. However, these 

organizations did not provide any specific examples of how the current 

level of coordination among federal agencies has prevented a specific 

regenerative medicine therapy from reaching patients or otherwise had a 

negative effect on the field.

Agency officials and nonfederal stakeholders have identified a range of 

challenges impacting the field of regenerative medicine. Specifically, they 

noted challenges with establishing effective collaboration mechanisms 

between federal and nonfederal stakeholders; finding qualified scientists 

versed in regenerative medicine and recruiting them to become federal 

employees; navigating the regulatory review process when seeking FDA 

approval of a new product; and setting payment policies and Medicare 

reimbursement mechanisms, processes, and rates for FDA-approved 

regenerative medicine products. In some cases, federal and nonfederal 

stakeholders have found strategies for addressing these challenges.

Federal collaboration with nonfederal stakeholders. Officials from 

three of the seven agencies we spoke with that conducted or funded 

regenerative medicine research in fiscal years 2012 through 2014, as well 

as representatives from some nonfederal stakeholder groups we 

interviewed, noted it is challenging for federal agencies to collaborate with 

nonfederal entities. Although some agencies described mechanisms they 

can use—such as memoranda of understanding or other formal 

agreements—to collaborate with individual nonfederal entities,15

 

15For example, NIH officials noted that they can and do form public-private partnerships 

and establish memoranda of understanding with nonfederal research organizations to 

accomplish specific research goals on an as-needed basis. In addition, officials said they 

can use public workshops and conference calls to discuss items of mutual interest with a 

foundation or industry partner.

some 

officials and representatives from nonfederal stakeholder groups pointed 

out that there is no systematic mechanism for federal agencies to 

collaborate with nonfederal entities that may also be conducting or 

funding regenerative medicine research, such as state-funded research 

institutions and private foundations. Some officials noted that stronger 

relationships and communication among federal and nonfederal 

stakeholders could help federal agencies stay informed about nonfederal 

research and help ensure the best use of those agencies’ investments in 

research in the field. Officials from one agency noted that interacting with 

Agency Officials and 

Other Stakeholders 

Have Identified 

Challenges in 

Advancing the Field 

of Regenerative 

Medicine and Have 

Taken Some Steps to 

Address Them
Page 18 GAO-15-553 Regenerative Medicine

officials from state-funded research institutions and foundations at 

national meetings and conferences helps to build relationships that allow 

federal officials to stay connected to the work happening outside of the 

federal sector. Officials from multiple agencies cited recent restrictions on 

travel to, and therefore, reduced attendance at, scientific meetings and 

conferences as a barrier to their work in regenerative medicine. They told 

us that their previous conference participation allowed them to maintain 

connections to the wider scientific community.16

To address this challenge, agency officials said they try to make the most 

of the very limited travel opportunities they have, while largely relying on 

their personal and professional relationships and networks to stay 

informed about relevant nonfederal funding and program updates. To 

increase their opportunities for maintaining connections to nonfederal 

stakeholders, FDA officials said they try to persuade conference 

organizers to hold meetings in the Washington, D.C., area where many 

federal scientists are employed, so that limited travel funds can be 

preserved. For example, FDA officials noted that MATES was able to 

present its December 2014 joint workshop as part of an international 

tissue engineering and regenerative medicine conference when 

conference organizers agreed to hold the event in Washington, D.C. 

Officials from FDA also suggested that convening a group of regenerative 

medicine stakeholders from across the federal government, industry, and 

academia to discuss progress in the field and opportunities for 

collaboration across stakeholder groups could be a valuable endeavor. 

To that end, FDA is discussing the possibility of holding a forum for 

stakeholders with the Institute of Medicine to discuss topics ranging from 

the state of the science to commercialization of regenerative medicine 

products. Officials noted that other federal agencies would be welcome in 

 

16We recently reported that for those working in defense science and technology, 

conference participation can promote communication with peers in other U.S. agencies 

and academia, as well as those from other countries. In addition, this communication 

helps to provide leadership across an array of individual technical fields, ensure the 

technical quality of research, and recruit new scientists and engineers to work for the 

federal government. DOD and Department of Energy officials have identified risks 

associated with reduced conference participation including a potential decline in the 

quality of scientific research, difficulty in recruiting and retaining qualified scientists and 

engineers, and a diminished leadership role for both agencies within the global science 

and technology community. See GAO, Defense Science and Technology: Further DOD 

and DOE Actions Needed to Provide Timely Conference Decisions and Analyze Risks 

from Changes in Participation, GAO-15-278 (Washington, D.C.: Mar. 4, 2015).
Page 19 GAO-15-553 Regenerative Medicine

such a forum that would also include leaders in the field from academia 

and industry.

Federal regenerative medicine workforce. According to federal officials 

we interviewed from two agencies, attracting and hiring researchers with 

the appropriate expertise is challenging, given that the field changes very 

rapidly and the federal hiring system can move slowly. In addition, some 

officials noted that finding researchers with suitable interdisciplinary 

expertise is difficult. Also, an official at one agency told us that there may 

be a belief among some scientists that the federal government is not 

supportive of stem cell-related research. To address workforce 

challenges, officials from two agencies have recruited postdoctoral 

fellows and provided training for them specifically to build the needed mix 

of interdisciplinary skills. Officials from one agency noted that high profile 

fellowships can also serve to highlight interagency collaborations that 

could enhance staff members’ knowledge and expertise. They suggested 

that postdoctoral fellowships such as one that existed in the past between 

NIH and NIST that allow scientists to work across agencies could be 

focused on regenerative medicine to build expertise in this area of 

research. Officials from one agency said they have recruited postdoctoral 

fellows to work in regenerative medicine through the National Research 

Council Postdoctoral Fellowship Program. In 2008, FDA initiated a

multicenter fellowship in regenerative medicine to bring in scientists with 

advanced degrees to enhance multidisciplinary training within the agency. 

This fellowship provides the opportunity for collaboration between FDA’s 

Center for Biologics Evaluation and Research and its Center for Devices 

and Radiological Health. Though the exact number of fellows varies 

based on budget and availability of projects, officials noted there are 

typically two to six fellows at a time who actively participate in the 

regulation of devices and biologics. According to FDA, the agency often 

hires these fellows into permanent positions after their fellowships end, 

which enables FDA to enhance its staff with additional individuals who are 

cross-trained in devices and biologics—a helpful skill set for reviewing 

regenerative medicine products. FDA officials also noted that fellows who 

have gone on to scientific careers outside of the FDA add value to the 

field of regenerative medicine because of their familiarity with FDA 

regulatory processes and the interdisciplinary training their fellowships 

provided. One official noted he was encouraged by a growing recognition 

among universities and industry of the importance of interdisciplinary 

approaches and training for this field.
Page 20 GAO-15-553 Regenerative Medicine

Regulatory review process. Some nonfederal stakeholders and officials 

from one agency we spoke with said that, because regenerative medicine 

products may be highly individualized treatments and do not conform to a 

single type of therapy, obtaining FDA approval for them may be 

challenging. FDA maintains discrete pathways for each product type—

drugs, devices, biological products, and combination products—that 

sponsors of new medical products, including regenerative medicine 

products, must follow to obtain FDA approval.17 According to FDA, some 

stakeholders advocate for a separate FDA approval pathway for 

regenerative medicine products, noting that, for one thing, they are 

different from drugs because they typically are not distributed throughout 

the entire body and are also different from devices. One stakeholder we 

interviewed noted that the combination product pathway is difficult to 

navigate for regenerative medicine products because it requires meeting 

the criteria and requirements for multiple review pathways, for example, 

for both drugs and devices, instead of just one. In addition, nonfederal 

stakeholders identified as a challenge the time and expense of 

successfully shepherding a product through the review process and 

gaining approval for it. They raised concerns that these characteristics of 

the current regulatory system may create a disincentive for product 

sponsors to bring regenerative medicine products to market in the United 

States. Stakeholders noted that this particular challenge is significant; 

however, it is not unique to regenerative medicine. For example, we 

previously reported that, on average, drug sponsors can spend over 

13 years studying the benefits and risks of a new compound and several 

hundred million dollars completing these studies before seeking FDA’s 

approval of a new drug application.18

 

17Among other things, a combination product is one that comprises two or more regulated 

components, i.e., drug/device, biologic/device, drug/biologic, or drug/device/biologic, that 

are physically, chemically, or otherwise combined or mixed and produced as a single 

entity.

Only about 1 out of every 10,000 

chemical compounds initially tested for their potential as new medicines is 

found safe and effective, and eventually approved by FDA, making the 

drug discovery and development process complex, time consuming, and 

costly. 

18See GAO, New Drug Development: Science, Business, Regulatory, and Intellectual 

Property Issues Cited as Hampering Drug Development Efforts, GAO-07-49 (Washington, 

D.C.: Nov. 17, 2006).
Page 21 GAO-15-553 Regenerative Medicine

FDA officials counter that developing a new regulatory review pathway for 

regenerative medicine products would require that a legal definition for 

regenerative medicine be established. However, these officials added that 

development of such a pathway is unnecessary because the agency has 

an adequate understanding of the science behind regenerative medicine 

and has instituted a sound process for reviewing product applications and 

regulating these products through existing pathways. FDA officials said 

that, as of April 2015, the agency had approved 14 regenerative medicine 

products, for uses including the repair or regeneration of skin and 

cartilage.

19

Related to FDA’s review process, officials from two agencies cited as a 

challenge FDA’s restrictions on sharing certain information gained from its 

product reviews about the fruitfulness of particular avenues of research. 

Officials from these two agencies said that finding mechanisms by which 

FDA can share its knowledge of sponsors’ challenges without disclosing 

proprietary information would be helpful. Further, they noted that this 

information may be valuable for other agencies for prioritizing and funding 

research. For example, officials at NIST said they would like to help 

develop measurement standards for regenerative medicine products, and 

FDA is in a unique position to identify trends seen across similar types of 

They explained that these products were approved through 

the existing pathways for biological products or devices, with some 

approved by both pathways as combination products. In addition, they 

said there are many more regenerative medicine products in earlier 

phases of FDA’s review process. FDA officials said that one way they 

plan for the review of new regenerative medicine products is by meeting 

with potential product sponsors before an application is submitted. Such 

sponsor-initiated meetings can be as informal as a sponsor calling FDA to 

discuss ideas and seek advice for future applications, or through more 

formal, documented meetings, sometimes years before they intend to 

submit an application. FDA officials said they archive general information 

from these meetings, such as the type of product and a sponsor’s 

anticipated time frame for submitting an application for review, which can 

be helpful for the agency when determining its needs for staff and 

scientific expertise.

 

19FDA officials noted that there are perhaps hundreds more that could be considered 

regenerative medicine products if one expanded the definition to include products 

regulated as tissues and certain types of devices. In terms of its regulation of biologics 

products, FDA makes a distinction between cellular therapy products, such as cellular 

immunotherapies, and tissue products, such as bone and ligaments.
Page 22 GAO-15-553 Regenerative Medicine

products that may help suggest what standards should be priorities for 

development. However, FDA officials stated that FDA cannot disclose 

measurement information to NIST if it constitutes a trade secret or 

confidential commercial information—for example, a measurement 

protocol that may offer a competitive advantage.20

Setting payment policies for regenerative medicine products. 

Officials from one agency and other stakeholders raised concerns that the 

Centers for Medicare & Medicaid Services (CMS) is not planning 

sufficiently for the availability of future regenerative medicine products 

following FDA approval, when CMS will need to consider setting Medicare 

payment policies.

FDA generally does 

not disclose any information about the status or contents of an application 

submitted to the agency until the product has been approved. FDA may 

share some limited information with other federal agencies when an 

established memorandum of understanding specifies the information to 

be shared. FDA officials said that limitations on sharing information with 

other agencies and stakeholders are always an issue for FDA and that 

this is not a challenge unique to regenerative medicine. Officials from 

FDA said that, although they limit the information they share on specific 

product applications, sponsors of products in review at FDA are free to 

invite others to the meetings and discussions they hold with FDA.

21

CMS officials countered this criticism by pointing out that although the 

agency does not have any unique policies or procedures for making 

coverage determinations for regenerative medicine products overall, it 

has made national coverage determinations on regenerative medicine 

products, such as stem cell transplants.

Officials from one agency cited CMS’s lack of 

involvement in MATES and engagement in the field of regenerative 

medicine in general as a reason for concern that CMS may not have a 

good sense of the types of regenerative medicine products being 

developed and the time frames for when those products might arrive on 

the market.

22

 

20See 18 U.S.C. § 1905, 21 U.S.C. § 331(j).

CMS officials explained that for 

21The proceedings from GAO’s 2007 forum on the future of the U.S. health care system 

note that federal payment models have had a strong influence on private payers. See 

GAO, Highlights of a Forum: Health Care 20 Years From Now—Taking Steps Today to 

Meet Tomorrow’s Challenges, GAO-07-1155SP (Washington, D.C.: September 2007).

22National coverage determinations stipulate coverage rules for the items and services 

that are reasonable and necessary for the diagnosis or treatment of an illness or injury.
Page 23 GAO-15-553 Regenerative Medicine

all coverage decisions, CMS follows the same process: a company 

requests a coverage decision for an FDA-approved device or product; 

CMS reviews documentation, including materials associated with FDA’s 

review and decision making; and CMS determines if the product or device 

is “reasonable and necessary” and, therefore, whether it should be 

approved for coverage by Medicare. CMS officials said that because the 

agency’s role is also to decide how much to pay for a new product, 

officials will not make such decisions unless CMS has determined, in fact, 

that Medicare will cover the device or product. In response to concerns 

about a perceived lack of planning by CMS, officials from CMS told us 

they stay informed about new products—including those related to 

regenerative medicine—through communications directly with drug 

sponsors and manufacturers who may engage with CMS while a product 

is moving through the regulatory review process at FDA. CMS officials 

said that although they do not take steps to proactively reach out to the 

regenerative medicine research community, they welcome product 

sponsors to contact them about the status of potential new products. 

CMS officials said if they encounter an area where they need additional 

scientific expertise to help establish payment policy, they may consult 

with their colleagues at NIH or obtain advice from the Institute of 

Medicine. Officials from DOD—which has its own system for determining 

payment policies for the products that are covered by its TRICARE 

program—emphasized the importance of planning for coverage decisions 

particularly because these types of treatments are new and do not have 

precedents for how to structure payments.23 These DOD officials noted 

that they have been working with the Military Health System and 

TRICARE administrators to plan for payment issues as new regenerative 

medicine products become available.

We provided a draft of this report to the Department of Commerce, DOD, 

the Department of Energy, HHS, NSF, and VA for review and comment. 

We received technical comments from the Department of Commerce, 

DOD, HHS, NSF, and VA which we incorporated, as appropriate. In its 

technical comments, VA also indicated its agreement with our 

conclusions. The Department of Energy did not provide any comments.

 

23TRICARE is the health care program for military servicemembers (active duty, 

Guard/Reserve, retired) and their families and is a component of the Military Health 

System.

Agency Comments
Page 24 GAO-15-553 Regenerative Medicine

As agreed with your offices, unless you publicly announce the contents of 

this report earlier, we plan no further distribution until 30 days from the 

report date. At that time, we will send copies of this report to the 

Secretaries of Commerce, Defense, Energy, Health and Human Services, 

and Veterans Affairs, as well as to the Director of the National Science 

Foundation. In addition, the report will be available on the GAO website at 

http://www.gao.gov.

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

me at (202) 512-7114 or crossem@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 appendix II.

Marcia Crosse 

Director, Health Care
Page 25 GAO-15-553 Regenerative Medicine

List of Requesters

The Honorable Lamar Alexander

Chairman

The Honorable Patty Murray

Ranking Member

Committee on Health, Education, Labor, and Pensions

United States Senate

The Honorable Orrin G. Hatch

Chairman

Committee on Finance

United States Senate

The Honorable Johnny Isakson

Chairman

Committee on Veterans’ Affairs

United States Senate

The Honorable Barbara Boxer

United States Senate
Appendix I: Funding for Regenerative Medicine 

Research by National Institutes of Health (NIH) 

by Institute, Center, or Program

Page 26 GAO-15-553 Regenerative Medicine

NIH was the largest federal funder of regenerative medicine research in 

fiscal years 2012 through 2014, having invested more than $2.54 billion 

into research in this field. Of NIH’s 27 institutes and centers, 22 funded 

some regenerative medicine research during the time period of our review. 

Two other entities within NIH—the Office of the Director and the NIH 

Common Fund—also funded regenerative medicine research during this 

time period. Table 2 shows the level of investment by each funding entity 

at NIH.

Table 2: Funding for Regenerative Medicine Research by the National Institutes of Health (NIH) by Individual Institute, Center, 

or Program, Fiscal Years 2012-2014

2012 2013 2014

Institute, center, 

or program

Funded 

amount ($)

Number 

of awards

Funded 

amount ($)

Number 

of awards

Funded 

amount ($)

Number 

of awards

Total amount 

fundeda ($)

Percentage 

of total 

funding

National Heart, 

Lung and Blood 

Institute 212,869,528 532 208,251,404 500 196,051,892 459 617,172,824 24.26

National Institute of 

Diabetes and 

Digestive and 

Kidney Diseasesb 90,073,024 274 81,142,224 274 93,233,038 258 264,448,286 10.39

National Cancer 

Institute 81,631,471 227 80,376,050 226 77,256,469 209 239,263,990 9.40

National Institute of 

Neurological 

Disorders and 

Stroke 73,812,668 253 72,285,570 249 72,913,912 248 219,012,150 8.61

National Institute of 

General Medical 

Sciences 69,145,159 161 64,132,487 173 59,256,816 174 192,534,462 7.57

National Institute of 

Arthritis and 

Musculoskeletal 

and Skin Diseases 62,726,547 215 62,533,204 233 65,809,026 239 191,068,777 7.51

National Institute of 

Dental & 

Craniofacial 

Research 41,946,674 123 40,335,711 121 36,798,976 116 119,081,361 4.68

National Institute 

on Aging 30,543,698 118 29,411,199 124 30,090,503 116 90,045,400 3.54

National Eye 

Institute 26,149,555 69 27,408,283 73 33,930,305 95 87,488,143 3.44

Appendix I: Funding for Regenerative 

Medicine Research by National Institutes of 

Health (NIH) by Institute, Center, or Program
Appendix I: Funding for Regenerative Medicine 

Research by National Institutes of Health (NIH) 

by Institute, Center, or Program

Page 27 GAO-15-553 Regenerative Medicine

2012 2013 2014

Institute, center, 

or program

Funded 

amount ($)

Number 

of awards

Funded 

amount ($)

Number 

of awards

Funded 

amount ($)

Number 

of awards

Total amount 

fundeda ($)

Percentage 

of total 

funding

National Institute of 

Allergy and 

Infectious 

Diseases 31,038,642 79 26,926,178 79 28,919,209 80 86,884,029 3.41

NIH Common 

Fundc 32,645,867 33 24,987,560 35 27,403,675 35 85,037,102 3.34

National Institute of 

Child Health and 

Human

Development 27,766,968 104 24,987,273 100 24,604,887 96 77,359,128 3.04

National Institute of 

Biomedical 

Imaging and 

Bioengineering 25,266,650 83 21,688,945 70 23,735,698 73 70,691,293 2.78

National Institute 

on Deafness and 

Other 

Communication 

Disorders 19,198,575 63 15,653,688 61 18,656,713 63 53,508,976 2.10

Office of the 

Directord 14,907,260 36 15,530,283 45 12,559,782 33 42,997,325 1.69

National Institute of 

Environmental 

Health Sciences 12,400,682 26 14,897,373 31 11,611,414 24 38,909,469 1.53

National Institute 

on Alcohol Abuse 

and Alcoholism 5,532,702 21 6,708,626 21 5,691,276 19 17,932,604 0.70

National Institute of 

Mental Health 7,059,618 20 4,024,101 13 5,137,875 17 16,221,594 0.64

National Human 

Genome Research 

Institute 4,709,383 8 4,627,132 8 4,144,462 8 13,480,977 0.53

National Center for 

Advancing 

translational 

Sciences 2,442,338 5 2,650,712 7 5,044,348 14 10,137,398 0.40

National Institute 

on Drug Abuse 4,504,775 11 2,251,541 6 3,327,704 10 10,084,020 0.40

National Institute of 

Nursing Research 192,032 2 217,566 1 409,598 0.02

National Institute 

on Minority Health 

and Health 

Disparities 170,541 1 129,281 1 299,822 0.01
Appendix I: Funding for Regenerative Medicine 

Research by National Institutes of Health (NIH) 

by Institute, Center, or Program

Page 28 GAO-15-553 Regenerative Medicine

2012 2013 2014

Institute, center, 

or program

Funded 

amount ($)

Number 

of awards

Funded 

amount ($)

Number 

of awards

Funded 

amount ($)

Number 

of awards

Total amount 

fundeda ($)

Percentage 

of total 

funding

Fogarty International 

Centere 107,411 2 102,040 2 209,451 0.01

Total 876,841,768 2,466 831,258,431 2,453 836,177,980 2,386 2,544,278,179 100.00

Source: GAO analysis of NIH funding data. | GAO-15-553

a

For purposes of this report, we use the term “funding” to refer to obligations. An obligation is a definite 

commitment that creates a legal liability of the federal government for payment of goods and services 

ordered or received. A federal agency incurs an obligation, for example, when it awards a grant.

b

Data for the National Institute of Diabetes and Digestive and Kidney Diseases includes regenerative 

medicine projects funded through the Special Statutory Funding Program for Type 1 Diabetes 

Research, which is administered by the National Institute of Diabetes and Digestive and Kidney 

Diseases.

c

NIH Common Fund, formerly the NIH Roadmap for Medical Research, is a series of initiatives 

designed to speed the movement of scientific discoveries. It provides a framework of the priorities the 

NIH must address in order to optimize its entire research portfolio and lays out a vision for a more 

efficient and productive system of medical research.

d

The Office of the Director is responsible for coordinating some programs and activities that span NIH 

components, particularly research initiatives and issues involving more than one of the institutes or 

centers.

e

The Fogarty International Center supports and facilitates global health research conducted by U.S. 

and international investigators.
Appendix II: GAO Contact and Staff 

Acknowledgments

Page 29 GAO-15-553 Regenerative Medicine

Marcia Crosse, (202) 512-7114 or crossem@gao.gov

In addition to the contact named above, Geri Redican-Bigott, Assistant 

Director; Jill K. Center; Cathleen J. Hamann; Erin C. Henderson; and 

Jennifer Whitworth made key contributions to this report.

Appendix II: GAO Contact and Staff 

Acknowledgments

GAO Contact

Staff 

Acknowledgments

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