Date: Oct. 3, 2002
Contacts: Christine Stencel, Media Relations Officer
Chris Dobbins, Media Relations Assistant
(202) 334-2138; e-mail <>

Broader Federal Protections, Other Reforms Needed
To Ensure Safety of Human Research Participants

WASHINGTON -- Broader federal oversight is needed to ensure that the health and well-being of people who are enrolled in research studies, whether publicly or privately funded, are better-protected, says a new report from the Institute of Medicine (IOM) of the National Academies.

Congress should require every organization conducting research with human subjects to do so under the authority of a research participant protection program, which would be subject to federal oversight. However, ultimate responsibility for ensuring that the essential protections are in place and followed must rest with the highest levels of the research organization's leadership, said the committee that wrote the report.

"It is understandable that the public has come to perceive that research institutions put more emphasis on insulating themselves from liability than on protecting people from harm," said committee chair Daniel Federman, senior dean for alumni relations and clinical teaching and professor of medicine and medical education, Harvard Medical School, Boston. "There is no single cause for the errors and mishaps that unfortunately have resulted in the deaths of some research participants in recent years. Rather, a combination of stresses, weaknesses, and lack of accountability have strained the current hodgepodge of protections to the point that fundamental changes are needed to protect all participants and keep public trust from being irrevocably eroded."

The IOM report was commissioned following the death of 18-year-old Jesse Gelsinger during a 1999 clinical study at the University of Pennsylvania. This case, along with incidents at other research centers, highlighted growing problems, such as conflicts of interest, inadequate monitoring and oversight, and insufficient communication with participants.

Federal agencies that conduct studies on humans follow a set of principles for protecting research participants known as the "Common Rule." But jurisdiction of this rule does not extend to non-federally funded research, although a bill has been proposed to make it law. Private-sector research to develop drugs, biologics, or medical devices is held to similar protection standards overseen by the Food and Drug Administration (FDA). However, other privately sponsored research that is outside FDA's authority is not required to follow specific protection standards, although many private organizations have developed their own guidelines. Without universal standards, though, participants may not be consistently afforded basic protections, such as adequate information about risks or assurance that researchers do not have conflicts of interest, the committee said. Moreover, improvements must be made in the organization, funding, and oversight of these programs in both the public and private sectors.

The participant protection program proposed by the committee is a system of interdependent elements -- the investigators, the institution, the staff that monitors safety and data collection, the boards that review the scientific and ethical integrity of proposed research, and the research sponsor -- linked through explicit responsibilities for participant protection. Many of the functions recommended for the protection program currently are carried out by institutional review boards (IRBs), the groups originally created by research institutions to review the ethics of proposed research involving human subjects. However, as the number of studies performed each year has increased and demands on the research oversight system have intensified, IRBs have been called on to handle an ever-wider array of tasks, including institutional risk management, regulatory compliance, evaluation of increasingly complex scientific issues, and assessments of conflicts of interest.

IRBs must return to the focused role they were originally intended to serve -- reviewing the ethical issues of proposed protocols -- because the boards do not necessarily have the expertise, authority, or resources to carry out all of these additional tasks on their own, the committee said. Issues pertaining to institutional interests, such as compliance with rules and regulations, should be managed by other entities within the protection program. In most cases, existing offices or departments, such as an organization's compliance office and risk counsel staff, should be able to assume these responsibilities. Assessments of potential conflicts of interest should be the responsibility of research organizations' conflict-of-interest oversight bodies. Review of the scientific merits of proposed research should be carried out separately from the ethical review, either by a subcommittee of the IRB or by a different group of experts.

To ensure that the entire protection system receives credible, expert advice, Congress should establish an independent, multidisciplinary, nonpartisan advisory body. Its membership should include individuals who can provide the perspective of the research participant. Since 2000, the National Human Research Protections Advisory Committee, created by the U.S. Department of Health and Human Services, has provided expert advice to federal agencies on issues of participant protections. However, it was recently disbanded, although the administration has signaled that a new committee likely will be formed.

In addition, reasonable compensation should be provided to people who are harmed as a result of their participation in studies, the committee said. Currently, the only recourse for such participants is to file lawsuits. While suits may be the appropriate avenue in cases where injuries result from negligence, misconduct, or product defect, research is never a risk-free enterprise and injuries may occur through no fault of the researchers or institutions. No-fault cases add to the judicial system's burden and compound the injured parties' aggravation.

Acknowledging that more data are needed on the extent to which illness and injury happen in studies, the committee recommended the immediate creation of a no-fault compensation system to provide injured participants or their survivors quicker resolution of claims and relieve some of the burden on the courts. Compensation should include at least the costs of medical care and rehabilitation and could be paid for either by the research organizations or potentially through a federal compensation program. In addition, an examination of the burden of lost wages, and whether these should be compensated, should be undertaken. In cases where fault can be proved, participants could still seek redress through lawsuits.

The report calls for a number of changes in the way institutions inform and solicit input from research participants. The process of informed consent should focus on informing volunteers, not on protecting institutions, the committee said. Informed consent should entail an ongoing series of conversations between the investigators and the participants, rather than a single conversation or signing of a document at the beginning of the process. These conversations should not only clearly communicate any changes in the nature of the study, but also reiterate the risks, benefits, and other details necessary for individuals to make informed decisions about their ongoing participation.

The study was sponsored by the U.S. Department of Health and Human Services and the Greenwall Foundation. The Institute of Medicine is a private, nonprofit institution that provides health policy advice under a congressional charter granted to the National Academy of Sciences. A committee roster follows.

Read the full text of Responsible Research: A Systems Approach to Protecting Research Participants for free from the National Academies at Printed copies are available for purchase from the National Academies Press website or by calling (202) 334-3313 or 1-800-624-6242. Reporters may obtain a pre-publication copy from the Office of News and Public Information (contacts listed above).

Board on Health Sciences Policy

Committee on Assessing the System for Protecting Human Research Participants

Daniel D. Federman, M.D.* (chair)
Senior Dean for Alumni Relations and Clinical Teaching, and
Carl W. Walter Distinguished Professor of Medicine and Medical Education
Harvard Medical School

Daniel L. Azarnoff, M.D.*
D.L. Azarnoff Associates, and
Senior Vice President of Clinical and Regulatory Affairs
Cellegy Pharmaceuticals
San Francisco

Tom Beauchamp, Ph.D.
Kennedy Institute of Ethics
Georgetown University
Washington, D.C.

Timothy Stoltzfus Jost, J.D.
Robert L. Willett Family Professor of Law
Washington and Lee University School of Law
Lexington, Va.

Patricia A. King, J.D.*
Carmack Waterhouse Professor of Law, Medicine, Ethics, and Public Policy
Georgetown University Law Center
Washington, D.C.

Roderick J.A. Little, Ph.D.
Department of Biostatistics
School of Public Health
University of Michigan
Ann Arbor

James McNulty
National Alliance for the Mentally Ill, and
President, Depressive/Manic Depressive Association of Rhode Island

Anne Petersen, Ph.D.*
Senior Vice President for Programs
Kellogg Foundation
Battle Creek, Mich.

Bonnie W. Ramsey, M.D.
Department of Pediatrics
School of Medicine
University of Washington

Lydia Villa-Komaroff, Ph.D.
Vice President for Research
Northwestern University
Evanston, Ill.

Fran Visco, J.D.
The National Breast Cancer Coalition
Washington, D.C.


Laura Lyman Rodriquez, Ph.D.
Study Director

* Member, Institute of Medicine