INSTITUTE OF MEDICINE
Preserving the Public Trust
Accreditation and Human Research
Shaping the Future for Health
Participant Programs
The protection of
individuals who volunteer to participate in research is essential to the ethical
conduct of research. Such protections were not explicitly and systematically
addressed in the United States, however, until the late 1940s, when scientists
and policy makers recognized the need to respond to crimes committed by Nazi
scientists during World War II. Since then there has been a growing sensitivity
to and acceptance of the need to conduct research involving human participants
with regard for their autonomy, privacy, and safety.
Over the past 50 years, regulatory policies have evolved to create a
system of participant protections involving investigators, sponsors, research
institutions, health-care providers, federal agencies, and patient and consumer
groups. But with this enhanced system of protections comes concern about whether
its complexity and size has rendered it unresponsive to the growing pressures of
a constantly changing research environment.
The need to improve protections has become more apparent as report after
report has highlighted mounting concerns about the ability of the participant
protection system to keep up with the evolving research enterprise. Nearly all
of these reports have recommended a reexamination and modernization of the
system. In addition, in 1999 the former federal Office for Protection from
Research Risks and the Food and Drug Administration took action against several
major research universities, suspending their human research programs because of
apparent noncompliance with federal regulations. Also in 1999, Jesse Gelsinger,
an 18-year-old research volunteer, died in a gene transfer trial not because of
his underlying disease but because of the experimental intervention itself. As
the circumstances and events leading up to his death emerged, it became apparent
that the system intended to protect him from unacceptable research risks instead
failed him. Trust in the human research enterprise, embodied in an individual
consenting to participate in a study, and thereby assuming risks inherent in
that study, demands that the system responsible for protection be credible and
accountable. In policy discussions that have occurred in the wake of these The
protection of individuals who volunteer to participate in research is essential
to the ethical conduct of research. Trust in the human research enterprise…
demands that the system responsible for protection be credible and accountable.
2 events, many have suggested that one way to improve accountability in the
system is through an accreditation process. In response to a request from the
Secretary of the Department of Health and Human Services (DHHS), the Institute
of Medicine formed a committee to conduct a two-phase study to examine how to
improve the structure and function of human research participant protection
programs. This report provides the committee's response to its first task: to
review and consider proposed standards for accreditation of programs that aim to
protect research participants and to recommend an approach to monitoring and
evaluating the total system of human participants protections. The committee's
recommendations are presented in Box 1 according to the phases inherent in the
development of an accreditation process.
Human Research Participant Protection Programs
In the United States, the system of human research participants
protection traditionally has centered on the institutional review board, or IRB,
which is charged with independent review of research protocols to assess risks
and the adequacy of protections for study participants. In this report, the
committee envisions a broader human research participant protection system than
just the IRB, with multiple functional elements that in total are referred to
ashuman research participant protection programs, or HRPPPs.
The many HRPPPs in this country make up a system with four principle
functions: 1) to ensure that research design is sound and that a study's promise
for augmenting knowledge justifies the involvement of human participants; 2) to
assess the risks and benefits of a study independently of the investigators who
carry out the research; 3) to ensure that participation in research is voluntary
and informed; and 4) to ensure that participants are recruited equitably and
that risks and benefits are fairly distributed. The HRPPP, which can take many
forms in many contexts, is the functional unit that would be the subject of an
accreditation process.
Accreditation as One Approach to Improving the Systemm
In addition to improving protections, accreditation as a mark of
excellence- of achievement well beyond regulatory compliance-might offer a HRPPP
a competitive advantage over nonaccredited competitors in seeking support from
sponsors or access to participants, researchers, or students. The committee
recommends that accreditation of HRPPPs should be pursued as one promising
approach to improve the system. The first step toward this strategy is the
implementation of pilot programs to test standards, establish accreditation
processes, and build confidence in accreditation organizations. The committee
believes that the ideal accreditation body is a national independent
organization that is credible among those seeking accreditation but independent
of any particular interest group among them.
Accreditation Standards
The central focus of this report is accreditation standards, the
benchmarks by which accreditation programs measure achievement. Any set of
standards must be flexible enough to be applicable to a variety of institutions
yet rigorous enough to ensure that their enactment enhances protection of
participants in human research. In addition, they must be clearly written,
relatively straightforward to execute, consistently applicable, and measurable.
At a minimum, standards should address an organization's level of
functional performance in specific areas. Some would further argue that the
measurement should not just address what the organization is capable of doing
but what it actually does. In theory, standards should set forth maximum
achievable performance expectations for activities that affect the protection of
human research participants. Perhaps most importantly, they should be based on
widely accepted ethical principles that form the norms for research behavior.
The committee reviewed available draft standards developed independently
by Public Responsibility in Medicine and Research (PRIM&R) and by the
National Committee for Quality Assurance (NCQA) under contract to the U.S.
Department of Veterans Affairs (VA). The following criteria were used in
assessing the standards: 1) their scope and focus; 2) their relationship to the
existing regulatory standards; and 3) the extent to which the standards can be
consistently implemented, measured, and enforced; as well as their inclusion of
various key elements.
Neither set of proposed standards applies readily to the full range of
human research or to the diversity of research institutions involved. It is not
clear in all cases how the standards should be applied to nonbiomedical research
settings, contract management organizations, cooperative clinical trials groups,
independent IRBs, site management organizations, or research units within
federal agencies and private industry.
Nonetheless, the committee concluded that an accreditation program(s)
should be pilot tested and that the NCQA standards are more suitable than those
prepared by PRIM&R, not only in VA facilities, but, with modification, for
the accreditation of other research institutions. The NCQA standards are the
strongest basis for use in the accreditation of other research institutions
because they include specific attention to quality improvement, provide
flexibility in achieving performance goals (e.g., increased protection of
research participants), and are explicit in their grounding in current
regulations.
In expanding the draft NCQA standards for use beyond VA facilities, the
committee recommends that the standards be strengthened in several specific
ways. These include: how investigators will be reviewed, beyond the review of
protocols by IRBs; how sponsors will be assessed; how participants will be
involved in setting performance standards; and how oversight mechanisms can
ensure participants' safety.
The committee makes two additional recommendations regarding standards.
First, the formulation of accreditation standards, the accreditation process,
and HRPPP operations should directly involve research participants. Second, the
accreditation process should accommodate organizations involved in research
beyond the traditional research organization models provided by academic health
centers and VA facilities. The accreditation process also should be appropriate
for research methods other than clinical research.
Evaluating HRPPP Pilot Accreditation Programs
Launching the HRPPP accreditation programs will take some time.
Experience will best guide judgments about the costs and benefits of an
accreditation strategy. Even as the pilot projects are being planned and
implemented, however, forethought about how to evaluate them is in order. The
committee recommends that DHHS commission studies to gather baseline data on the
current system of protections for human participants in research, that Congress
request an evaluation from the DHHS Office of the Inspector General.
For More Information…
Copies of Preserving the Public Trust: Accreditation and Human
Research Participant Programs are available for sale from the National
Academy Press; call (800) 624-6242 or (202) 334-3313 (in the Washington
metropolitan area), or visit the NAP home
page.
This study was funded by the Department of Health and Human Services and
the Greenwall Foundation. The Institute of Medicine is a private, nonprofit
organization that provides health policy advice under a congressional charter
granted to the National Academy of Sciences. For more information about the
Institute of Medicine, visit the IOM Home Page
© 2001 by the National Academy of Sciences.
All rights reserved. Permission is granted to reproduce this document in
its entirety, with no additions or alterations.
COMMITTEE ON ASSESSING THE SYSTEM FOR PROTECTING HUMAN RESEARCH
SUBJECTS
DANIEL D. FEDERMAN (Chair), Dean for Clinical Teaching, Harvard
University, Boston, MA
DANIEL AZARNOFF, President, D.L. Azarnoff Associates,
San Francisco, CA
TOM BEAUCHAMP, Professor, Kennedy Institute of Ethics,
Georgetown University, Washington, DC
TIMOTHY STOLTZFUS JOST, Newton D.
Baker, Baker and Hostetler Professor of Law and Health Services Management and
Policy, Columbus, OH
PATRICIA A. KING, Carmack Waterhouse Professor of Law,
Medicine, Ethics, and Public Policy, Georgetown University Law Center,
Washington, DC
RODERICK J.A. LITTLE, Chair, Department of Biostatistics,
School of Public Health, University of Michigan, Ann Arbor, MI
JAMES McNULTY,
President, Depressive/Manic Depressive Association of Rhode Island, Bristol,
RI
ANNE PETERSEN, Senior Vice President-Programs, Kellogg Foundation, Battle
Creek, MI BONNIE W. RAMSEY, Professor, Department of Pediatrics, University of
Washington School of Medicine, Seattle, WA
LYDIA VILLA-KOMAROFF, Vice
President for Research, Northwestern University, Evanston, IL
FRAN VISCO,
President, The National Breast Cancer Coalition, Washington, DC
Expert Advisers
KAY DICKERSIN, Associate Professor, Department of Community Health,
Brown University, Providence, RI
ALBERTO GRIGNOLO, Senior Vice President and
General Manager for Worldwide Regulatory Affairs, PAREXEL Internat6 Del,
Waltham, MA
MARY FAITH MARSHALL, Professor of Medicine, School of Medicine,
Kansas University Medical Center, Kansas City, KS
CAROL SAUNDERS, President,
Center for Clinical Research Practice, Wellesley, MA
DENNIS TOLSMA,
Director, Clinical Quality Improvement, Kaiser Permanente, Atlanta, GA
Liaisons
RICHARD J. BONNIE, John S. Battle Professor of Law and Director,
Institute of Law, Psychiatry, and Public Policy, Charlottesville, VA
NANCY
NEVELOFF DUBLER, Director, Division of Bioethics, Montefiore Medical Center;
Co-director, Certificate Program in Bioethics and Medical Humanities, Professor
of Bioethics, Albert Einstein Medical College, Bronx, NY
ELENA OTTOLENGHI
NIGHTINGALE, Scholar-in-Residence, Institute of Medicine and National Research
Council, Washington, DC
PILAR OSSORIO, Assistant Professor of Law and
Medical Ethics, Associate Director of the Center for the Study of Race and
Ethnicity in Medicine, University of Wisconsin, Madison Law School, Madison, WI
Study Staff
LAURA LYMAN RODRIGUEZ, Study Director ROBERT COOK-DEEGAN, Senior Program
Officer
JESSICA AUNGST, Research Assistant
NATASHA DICKSON, Project
Assistant
IOM Board on Health Sciences Policy
Staff
ANDREW POPE, Board Director ALDEN CHANG, Administrative Assistant CARLOS
GABRIEL, Financial Associate
Consultant
KATHI HANNA