Report to the Advisory Committee to the Director, NIH
from the Office for Protection from Research Risks Review Panel

 
June 3, 1999

 
 

Table of Contents

Executive Summary — Charge to the Panel

Background — Context of the Panel's Review

Recommendations

Conclusion — An Opportunity for Federal Leadership

Appendices

Membership of Review Panel

Interviews Conducted

Executive Summary — Charge to the Panel

The Office for Protection from Research Risks Review Panel (Review Panel) was convened to consider two issues involving the Office for Protection from Research Risks (OPRR) and to make recommendations based on the review to the National Institutes of Health (NIH) Director's Advisory Committee. The objectives of this review were to:

1. Ensure that the organizational locus of OPRR continues to be the most appropriate for OPRR's mission and future directions of research; and

2. Advise whether there is a need for OPRR to have additional delegated authority to accomplish its mission.

While the members of the Review Panel are aware of the more broadly focused debate in the Congress, the academic literature, and in the general media about whether the federal role in regulating non-federal research should be expanded, the Review Panel was not authorized to study the issues relevant to that larger debate or to make recommendations about how those issues should be resolved. Nor was the Review Panel asked to evaluate the operation of Institutional Review Boards (IRBs). Nevertheless, in order to evaluate the appropriateness of the OPRR location, some consideration of the function of the office was necessary. The primary focus of the Review Panel efforts, however, was on the issues defined in the above-described charge. Based on the interviews conducted, the material reviewed, and discussions that occurred among the Review Panel members, the Review Panel arrived at the following findings and recommendations:

I. OPRR should be administratively relocated from its present location within the NIH.

II. OPRR should be located in the Office of the Secretary of the Department of Health and Human Services (DHHS) and report to either the Surgeon General or the Assistant Secretary for Health: Locating OPRR outside of the NIH will strengthen its ability to interact with other agencies within DHHS and with other Departments.

III. A relocated OPRR would best be able to reap the benefits of the move if the director of the office were to be a member of the Senior Executive Service.

IV. As part of the move of OPRR to a new location, the Secretary should create an independent advisory committee to provide guidance, assist in setting standards, and review the operation of the office.

V. The authority presently delegated to OPRR is adequate for it to address the tasks currently assigned to it, but resources available to OPRR may be inadequate for fulfilling its mission.

After presenting the context in which the Review Panel's deliberations occurred, this report will describe the reasoning underlying each of the recommendations and address issues relevant to the implementation of the recommendations.

BACKGROUND — Context of the Panel's Review

Nature of Research

Over recent decades major advances have been made in the prevention and treatment of disease and in improving health. These advances have been attributable in large part to the implementation of findings from research involving animals and human subjects. Improvements in the strategies for such research, as well as refinements in our understanding of ethical issues relating to protection of subjects, have developed as this research enterprise has expanded. This growing sophistication regarding research ethics and strategies for protection of subjects, as well as promulgation of federal regulations, policies, and procedures, has resulted in a research enterprise involving millions of human subjects and animals, thousands of scientists, and hundreds of institutions across the country. The benefits of this research have been substantial, leading to healthier lives, reduced disability, and prevention of suffering.

Concerns about the Protection of Human Subjects

In the last several years, however, concerns about the protection of human subjects in biomedical and behavioral research have emerged from the sub-text of scholarly discussion and have vaulted into the headlines. At the national level, for example, the National Bioethics Advisory Committee (NBAC) released a report addressing Research Involving Subjects with Mental Disorders That May Affect Decisionmaking Capacity and has indicated its plan to create an active subcommittee to consider issues involving the rights of, and protections for, human subjects involved in research. NBAC continues to focus on the interests and rights of those human subjects, including children, whose ability to provide informed consent is nascent or, in the case of those with mental disorders, is fluctuating or diminished.

There is a growing sophistication on the part of the public about the extent of the involvement of human subjects in research, the dangers that such research might present to human subjects, and the concern that the rights and welfare of human research subjects be protected. The federal government's apology to the subjects of the discredited Tuskegee syphilis study, as well as recently suspended studies involving mentally incapacitated persons, are examples of instances in which the research community has acknowledged that the use of human subjects in research was not, or may not be, appropriate. Reports of the extensive violation of the rights of human subjects involved in radiation research funded by the Federal government underscored the dangers. (Advisory Committee on Human Radiation Experiments Final Report. Washington, DC: U.S. Government Printing Office, 1995. Published as The Human Radiation Experiments (New York: Oxford University Press, 1996)).

Role of OPRR in Protecting Human Subjects

OPRR has been assigned a key role in reviewing research and providing guidance on these complex issues. Some commentators and ethicists have suggested that the location of the OPRR in the governmental research structure—within the National Institutes of Health which is itself a component of DHHS—contributes to public distrust of the research enterprise with respect to the treatment of human subjects and animals. Because the NIH is a major center for the conduct of research and one of the nation's leading sources of research funding, the location of OPRR within NIH gives rise to a perception that OPRR's actions will be biased in favor of research interests and will provide insufficient protection to research subjects. (See, e.g., Moreno, Caplan, Wolpe and the Members of the Project on Informed Consent, Human Research Ethics Group, "Updating Protections for Human Subjects Involved in Research," 1998, JAMA 280: 1951-8.)

The Office for Protection from Research Risks came into existence in 1972, when Robert Q. Marston, Director of NIH, upgraded what was then known as the Institutional Relations Branch, Division of Research Grants (DRG/NIH), changed the name to the Office for Protection from Research Risks (OPRR), and directed that the office report to the Associate Director for Extramural Research. The National Research Act (P.L. 93-348), signed into law in 1974 and later amended, requires that "the Secretary shall establish a program within the Department of Health and Human Services under which requests for clarification and guidance with respect to ethical issues raised in connection with biomedical or behavioral research involving human subjects are responded to promptly and appropriately." The Act also calls for prompt actions to enforce the regulations. The OPRR direct line of reporting is to the NIH Deputy Director for Extramural Research, to the NIH Director, to the Secretary, DHHS. OPRR has approximaely 27 staff, 8 assigned to the Division of Animal Welfare, 15 assigned to the Division of Human Subjects Protection, and the remainder in administration, education, and information management. OPRR has several responsibilities including implementation of DHHS regulations for protection of human subjects (Title 45, Code of Federal Regulations, part 46). These responsibilities include negotiating formal written assurances of compliance with institutions engaged in research covered by 45 CFR Part 46, investigation and oversight of institutional compliance, and professional and public education.

Humane Care and Use of Animals in Research

Two Federal departments regulate the use of animals in research, education, and testing: the United States Department of Agriculture (USDA) and the Department of Health and Human Services (DHHS). Concerns regarding the abuse of animals in research protocols resulted in the passage of the Animal Welfare Act (AWA) in late 1966 (7 USC 2131 et seq, last amended 1985). The USDA enforces the AWA by yearly, unannounced inspections of research facilities and dealers and transporters of animals.

In response to concerns about abuse of nonhuman primates in research, NIH policy was rewritten by OPRR to provide better protection for the welfare of animals. This rewritten policy was incorporated into law by the Health Research Extension Act (P.L. 99-158). The Public Health Service Policy on Humane Care and Use of Laboratory Animals (PHS Policy) requires an assurance of compliance with the Policy and oversight by a local Institutional Animal Care and Use Committee (IACUC). The law directs the Secretary, DHHS, acting through the Director of NIH, to require assurances from all who receive funds for research involving animals administered by NIH and the national research institutes. Further the law directs the Director of NIH to suspend or revoke grants and contracts for noncompliance with PHS Policy.

The USDA and DHHS approach regulation involving animal protections differently. The USDA relies upon on-site inspections by Veterinary Medical Officers (VMO) while DHHS relies on education and training of researchers and members of IACUCs. PHS Policy relies on the Guide for the Care and Use of Laboratory Animals, a science-based, peer reviewed set of guidelines developed by the National Research Council. The Guide provides institutions flexibility in caring for animals used in a wide variety of research projects. The Division of Animal Welfare (DAW), OPRR, works with institutions in attempts to correct deficiencies and only takes action—interdiction of research funds—after failure to correct problems has been documented.

Thus, OPRR uses a collaborative approach to ensuring humane care and use of animals in research, education, and testing. The DAW, administered entirely by board certified laboratory animal veterinarians who have had experience in the field, is able to assess the general welfare and well-being of the animals involved in research. The DAW presents educational programs for veterinarians at laboratory animal science meetings and at various institutions dispersed throughout the country to help institutions achieve compliance with PHS Policy. The USDA inspections tend to focus on formal structural issues such as defects in arrangements for animal housing, engineering issues such as cage sizes, and the adequacy of records.

PHS Policy, in theory, extends to other agencies which fund research that uses animals. For example, NSF requires institutions to have an assurance on file with OPRR. There is no instance, however, of DAW interdicting funds from other agencies. DAW, as OPRR in general, is in a position of potential conflict when dealing with the NIH intramural programs. OPRR's current location is perceived as compromising its ability to oversee research funded by other agencies and by NIH intramural programs. On the other hand, OPRR's current location most likely facilitates efforts of laboratory animal veterinarians in DAW to remain connected to the research community.

Justice and Fairness in Research

The sources of funding for scientific research are also relevant to the context in which OPRR functions. Federal dollars used to support research create an obligation on the part of the federal government to ensure that research involving human subjects is conducted with appropriate consideration for the integrity of science and for the rights, interests, and safety of research subjects. In addition, the federal government has an obligation to ensure equitable access to potentially beneficial research protocols—an obligation that has been highlighted in the search for effective treatments, for example, for HIV infection and for the control of Alzheimer's disease and other dementing disorders. The scientific community has become increasingly sensitive to the problems raised by the exclusion or nonparticipation of women and people of color from some research protocols and, under the leadership of the NIH, researchers have actively attempted to address these issues. It is also clear that excluding the mentally ill and children from research will, in some cases, bar them from the benefits of such research. If broad political and social support for federal investment in scientific research is to be maintained, access must be open to all groups regardless of gender, race, ethnicity, religion, disability, or socioeconomic status.

Concerns about Non-Federally Funded Research

Federal dollars, however, do not represent the total national investment in research. Private capital increasingly supports new research, especially in biotechnology. Institutional Review Boards attached to private companies that are not associated with academic institutions receiving federal funds, or that are not regulated by the Food and Drug Administration (FDA), are not bound, and may not feel constrained, by the regulations governing research with human subjects. These private arrangements generally lack the oversight of OPRR and are not covered by the Multiple Project Assurances that regulate organizations with federal support and multiple assurance agreements. If human subjects involved in these outside studies are to be protected adequately, new arrangements will need to be forged.

Dangers of Concurrent State and Local Regulation

These developments in research structure and funding, and the increasingly public nature of the debate, make it imperative for OPRR to assume a leadership position in discussions about the intellectual architecture and the concrete realities of research protocols and in discussions about those statutes, regulations, and ethical principles designed to protect human subjects and the welfare of animals. Absent OPRR's exercise of such leadership, the prospect is increased that states and local communities will extend efforts to regulate research conducted within their borders by layering specific state and local dictates that require alternative protocol designs on top of federal regulations. Such state and local regulation would pose a substantial burden to a national research enterprise.

For example, in early 1999, the Maryland Attorney General's Research Working Group presented legislation to the Maryland General Assembly proposing requirements for research involving decisionally incapacitated persons. In addition to specifying certain obligations on the part of researchers, the legislation would require certain IRBs to submit annual reports to the Maryland Secretary of Health and Mental Hygiene and the Maryland Attorney General. (Senate Bill 306 and Senate Bill 307 were introduced in February 1999 to the Maryland General Assembly but not enacted.) Further action is not expected on this issue during this legislative session. In New York State, the case of T.D. v. New York State Office of Mental Health, 91 N.Y. 2d. 860 (1997), challenged the rules of the Office of Mental Health governing the inclusion of institutionalized persons without decisional capacity in research protocols. As a result of that case and the conclusion of the New York Court of Appeals that the existing rules violated the rights of patients, a committee appointed by the state Commissioner of Health has recommended additional regulations for the protection of adult subjects in non-Federally funded research, who cannot provide ethically and legally adequate informed consent. New York State also convened working groups to address the protections for "normal healthy volunteers" in any research, however funded, and to examine issues involving the participation of children in research. In the area of animal welfare, in 1989, the city of Cambridge, Massachusetts, enacted an ordinance creating the position of commissioner of laboratory animals with the authority to inspect animal laboratories and review research protocols to ensure that all work meets federal standards for pain control.

Increasingly, research involving human subjects is conducted within complex multistate and multinational collaborative protocols and arrangements. The activities undertaken in Maryland and New York indicate how complex and perhaps unworkable multistate trials could become in the absence of federal regulation in which the public has confidence. State variations in the requirements for informed consent and research conditions could be difficult to manage. Similar issues arise in multinational research. The forthcoming multinational malaria vaccine trials will challenge researchers and regulators to design protocols that provide appropriate protections for the research subjects. Fragmentation of research regulation will substantially interfere with multistate and multinational research.

* * * * *

This brief overview of current issues and future challenges created by the participation of human subjects and the use of animals in scientific research indicates that adequate and appropriate regulation is a sophisticated undertaking. Effective federal oversight will require the energies of an agency with the independence, resources, and institutional support to have credibility with the public as well as with the scientific community. The Review Panel concluded that this opportunity for federal leadership can be realized if the following recommendations are adopted, and implemented vigorously.

RECOMMENDATIONS

I. OPRR should be administratively relocated from its present location within the NIH.

Interviews with knowledgeable persons, both within and outside of government (see Appendix 2 for a list of persons interviewed), confirmed the position, previously expressed in contract papers prepared for NBAC, that in its present location, as a part of the NIH and reporting to supervisors within the NIH, OPRR is not perceived as an independent office. (See "Report to NBAC, Location of OPRR within the NIH: Problems of Status and Independent Authority" by John C. Fletcher; "Report to NBAC, Reflections on the Organizational Locus of the Office for Protection from Research Risks" by Charles R. McCarthy; "Report to NBAC, An Examination of Issues Presented by Proposals to Unify and Expand Federal Oversight of Human Subject Research" by C.K. Gunsalus.) This perception of dependence and the concerns about conflicts of interest that arise therefrom compromise the ability of OPRR to function most effectively in providing ethical and regulatory leadership in the arenas of research with human subjects and with animals. After considering the advantages and disadvantages of OPRR's current location, the Review Panel concluded that relocating OPRR was the only way to address these perceptions and concerns and to ensure OPRR's independence and maximize its effectiveness.

A. Conflicts of Interest Arising from OPRR Location Within NIH

OPRR is perceived to be affected by conflicts of interest because of its position in the hierarchy of the NIH and its concomitant obligation to review research conducted by NIH researchers. OPRR's connection to NIH confers status by virtue of association with one of the premier research institutions in the world and provides OPRR, in theory, with immediate access to scientific expertise. While this aspect of the NIH connection enhances the status and reputation of OPRR in the scientific community, the relationship to NIH increases the public perception that OPRR will be biased in the direction of protecting research interests at the expense of protections for human subjects and animals. All of the intramural research at the NIH that OPRR is required to review arises within divisions at the NIH that are placed equal to, or above, OPRR in the table of organization. This positioning leads inevitably to an appearance of conflict that likely, in specific cases, reflects reality. Even the perception of conflict interferes with a vigorous pursuit of the mission of the office. Both the appearance and the actuality of a conflict of interest are of concern to the Review Panel.

These concerns are neither abstract nor hypothetical. The staff of the OPRR reported that at times their suggestions for policy had been modified before reaching the Office of the Secretary, thus subsuming their intentions to the agendas of other divisions within the NIH. This past experience causes concerns but raises opportunities. One of OPRR's obligations is to create a robust and productive dialogue with all of the divisions within the NIH that might be affected by new guidelines or regulations. Once the discussions can be shared and disagreements and disputes recorded as part of the process of deliberation, OPRR should be more willing, rather than less, to engage in consultation. Once OPRR is not in the line of authority within the NIH, it should be more comfortable in engaging NIH in discussions on difficult issues knowing that if consensus eludes the process, the differences of opinion will be presented to the Secretary for discussion.

B. Concern About Conflicts of Interest Limit OPRR's Influence Within NIH

Quite paradoxically, because of its placement and the resulting appearance of conflict, there is substantial concern within the NIH that OPRR has been excessively remote from, and unresponsive to, certain issues and concerns of programs at the NIH. There is some shared feeling among researchers at the NIH that, in compensating for the appearance of conflict, OPRR has fostered a perception of harsher review for intramural research and has held intramural researchers "at arms' length" without adequate consultation and support from OPRR.

C. OPRR's Location Within NIH Compromises Its Effectiveness with Entities Outside NIH

A third concern is that the placement of OPRR within the NIH compromises its effectiveness with other components within DHHS and with other agencies within the federal government. The stature, authority, and effectiveness of the OPRR are diminished because it is perceived by other departments and agencies as a subordinate division within the NIH. For example, the NIH, the Centers for Disease Control and Prevention (CDC), and the Food and Drug Administration (FDA) have parallel and equal status on the DHHS organizational chart. (Among the other DHHS agencies of parallel and equal status that fund research are the Agency for Health Care Policy and Research (AHCPR) and the Health Resources and Services Administration (HRSA)). To the extent that OPRR exercises leadership or oversight over FDA and CDC in OPRR's administration of the Common Rule and animal welfare policies, this oversight appears to be coming from "below" the FDA and CDC. Similarly, OPRR's interactions with agencies such as the Departments of Agriculture and Defense are viewed as compromised because of its location in NIH. OPRR's location does not signal to other Cabinet departments that OPRR speaks for the Secretary and DHHS. As long as OPRR is located within the NIH, its ability to exercise authority in interactions over other components of DHHS of equal or higher stature and in interactions with Cabinet departments or significant components of other governmental agencies will be compromised.

Other components within DHHS and other federal agencies who are signatory to the Common Rule may also perceive that OPRR's position compromises independent review of NIH intramural research and affects the evenhanded review of research conducted outside of the NIH. Any such perceptions impede the ability of OPRR to interpret and enforce the Common Rule effectively in relation to agencies that may be unaccustomed to measuring their overall plans for research and specific protocols against the rules governing human subjects protection and animal welfare that have become increasingly incorporated into the culture of the NIH. (See "Report to NBAC, An Examination of Issues Presented by Proposals to Unify and Expand Federal Oversight of Human Subject Research" by C.K. Gunsalus.) If it is to be a strong and effective leader, OPRR's actions and advice must be perceived as unaffected by institutional bias, informed by unassailable expertise in scientific methods and ethics, and characterized by fairness and impartiality. OPRR's subordinate position within the NIH does not foster or enhance its eminence with other departments or its connections with other agencies.

D. Oversight of OPRR Compromised by Location Within NIH

Because the officials to whom OPRR reports are concerned about its place in the table of organization and about possible charges of unfairness by OPRR and others, some supervisors report difficulty in providing adequate critique of the office's performance and sufficient oversight for plans, policies, and process. Some persons who stand in supervisory positions to OPRR are reluctant to risk the charge that their suggestions for change and their criticisms of actions are motivated by parochial interests rather than by an honest review of performance that, in their judgment, could be enhanced or improved. Some commentators suggest that OPRR actually has too much authority without equally effective oversight, supervision, and review. For example, they argue that many statements offered by OPRR are interpreted by researchers and institutions as equal to the federal regulations in applicability to research design and the practice of IRBs. Given this position of power, those pronouncements, some argue, should be reviewed by knowledgeable persons outside of the OPRR to ensure that OPRR's interpretations reflect best ethical precepts, developing practices, and scholarship in the ethics and research community before attaining the virtual status as new regulations.

E. Ensuring OPRR's Efficacy and Independence After Relocation

1. Maintaining OPRR's Understanding of the Research Enterprise

As a counterbalance to some of the arguments stated above, moving OPRR from within the NIH raises the prospect that it will lose touch with the "culture" of research and be less acute in its perception of newly developing issues. Some persons discussing these issues with this Review Panel fear that moving the office outside of the NIH will diminish its sensitivity to the apprehensions and the operations of the research community. OPRR, if relocated, would need to be aware of, and alert to, these reasonable concerns in order to guard against irrelevance and inappropriate action. OPRR would need to commit time and attention of its staff to participate in educational seminars, colloquia, and discussions that would keep it educated about and in the midst of those discussions that provide the grounding for new research. In terms of involvement in research, the physical and organizational location may be less important than the intellectual commitment to being part of the research enterprise. OPRR should always consider itself part of the research community, although an independent, critical, and questioning colleague.

2. Securing Resources to Support OPRR's Mission

The Secretary, in conjunction with the Congress and the components of the government that rely on OPRR to regulate research risks, must take steps to ensure that OPRR, wherever located, is able to obtain and maintain the budgetary and personnel resources it requires to carry out its mission effectively. OPRR's current location within the funding stream of the NIH places OPRR among the privileged. The Review Panel recognizes that any new organizational arrangement may disrupt what is now a reliable funding situation and exposes OPRR to the risk that it will be more vulnerable to legislative efforts to impose fiscal constraint or achieve other political goals.

The Review Panel urges that the Secretary establish a clear approach to funding OPRR's activities before any move. One possible approach would be to use NIH funds. This approach could be justified as part of NIH's lead role in maintaining the infrastructure that supports the national scientific research enterprise. Potential concerns created by the use of NIH funds would be counterbalanced by OPRR's organizational independence from the NIH. Alternatively, the Secretary could request a system whereby all federal agencies that fund scientific research would be required to set aside a fraction of a percentage of research budgets to support the operation of OPRR. The Review Panel notes approvingly the manner in which a portion of funds supporting the Human Genome Project were allocated to provide for research on ethical and legal issues created by the mapping of the human genome. These funds have been expended concurrently with the expenditures for the scientific aspects of the project. Were OPRR to be funded out of research budgets, the necessary percentage of the total budget of any one entity would be very modest.

3. Insulating OPRR from Inappropriate Political Influence

Finally, some who question the need to relocate the office point to its greater political vulnerability in a location outside of the NIH. Given that OPRR has the ability effectively to suspend a research institution's activities for failure to comply with animal welfare or human protection requirements, its power can sometimes generate strong political reaction. In addition to budgetary vulnerability, OPRR's location within the Office of the Secretary could expose it to greater political influence from a variety of sources within and outside the Executive Branch. The Review Panel believes that the risk of political vulnerability does not outweigh the benefits in credibility and operational integrity to be gained by relocation.

II. OPRR should be located in the Office of the Secretary of DHHS and report to either the Surgeon General or the Assistant Secretary for Health: Locating OPRR outside of the NIH will strengthen its ability to interact with other agencies within DHHS and with other Departments.

The previous section reviewed the ways in which the location of OPRR within NIH is perceived to limit its ability to regulate the activities of NIH researchers in an unbiased manner, and the constraints this location creates on NIH officials exercising appropriate supervision. The Review Panel concluded that merely elevating OPRR on the NIH organizational chart would not enhance OPRR's ability to exercise a leadership role in interactions with other components of DHHS and with other departments and agencies in the government. At issue here is not a superficial concern about location on an organizational chart, but a substantive concern that the interests of the human subjects and animals involved in research will not be adequately protected if the entity charged with their protection does not have the actual and perceived authority to achieve cooperation or compliance from other entities in the government.

Several commentators with whom the Review Panel consulted indicated, and common sense suggests, that the organizational placement of an entity such as OPRR signals the importance attached to its work. Given the increasing public concern about the protections afforded the human subjects and animals and the important role research involving human subjects and animals continues to play in science, protecting human subjects and ensuring the humane treatment of animals must be a central value in the scientific community. These protections must be accorded a central value in order to maintain the credibility of science in the eyes of the public. Public financial and intellectual support for research require a credible office protecting human subjects of research and requiring the humane care and use of animals in research. Thus, ensuring the welfare of these subjects and animals is an essential component of an effective and stable scientific research enterprise.

The Review Panel considered several possible administrative locations including within the Executive Office of the President, other locations within DHHS, or an independent agency, but concluded that it should be in the Office of the Secretary. Relocating OPRR to the Office of the Secretary will make OPRR more effective in exercising a leadership role in the research community on issues relating to the protection of human subjects and the welfare of animals. Some commentators have recommended a shift in the focus of OPRR away from activities associated with the assurance process to education, leadership, and training. If these were to be new areas of concentration for OPRR staff, a location outside of NIH and near the Secretary of DHHS would permit new alliances with departments within DHHS and with other federal agencies. If the emphasis within OPRR shifts from supervision of assurances to educational and training interventions, as discussed elsewhere in this report, OPRR also could become a powerful forum for intellectual exploration of challenging human subjects protection issues. Located prominently in the Office of the Secretary, and staffed with persons of the requisite stature and expertise, OPRR's persuasive, as well as its legal authority, would be recognized by those under its supervision or participating in its programs.

III. A relocated OPRR would best be able to reap the benefits of the move if the director of the office were to be a member of the Senior Executive Service.

The best insurance against the exertion of inappropriate influences on OPRR's activities is the appointment of a director and other staff for OPRR who have national stature in the scientific, ethics, and legal communities, who have demonstrated integrity, and who are committed to the protection of human research subjects and the welfare of animals used in research. OPRR's regulatory responsibilities and position of moral leadership dictate that the office be directed by a person who has substantial expertise in, or experience with, the design and conduct of scientific research, substantial familiarity with the complex ethical, legal, and regulatory issues that are involved in research, and substantial interpersonal skills of the type essential to communicate ideas effectively and to persuade others to act based on advice given. This person should be respected by the community of scientists governed by OPRR and by the scholarly ethics community that participates in discussions of protection of human subjects and animals. Reclassifying the position of director as a member of the Senior Executive Service (SES) should permit a director of OPRR to be recruited who will be able to fulfill all of these roles, will provide creative and strong leadership in discussions of complex and difficult issues, and will assist in designing action plans to improve the protection of human subjects and the welfare of animals. The Review Panel recognizes that given the specialization inherent in all fields, any director will necessarily possess stronger credentials in some of the desired areas of experience than in others. For this reason, OPRR must build a staff that can collectively bring to bear an appropriate range of expertise in the areas of science and ethics relevant to its work.

IV. As part of the move of OPRR to a new location, the Secretary should create an independent advisory committee to provide guidance, assist in setting standards, and review the operation of the office.

As discussed in Section V of this report, the Review Panel concluded that OPRR has sufficient statutory and delegated authority to pursue its goals. It does not, at present, however, have the intellectual and moral stature to provide the creative and directive leadership in the research community nationally and internationally that will be increasingly critical in multi-institutional and multinational research.

One aspect of the solution to this problem is the creation of an advisory committee, composed of scientists, ethicists, physicians, former research subjects, animal experts, and members of the public, which could provide broader scientific and ethical resonance to discussions at OPRR. Such a group could assist OPRR in remaining current with emerging ethical challenges and developing scientific trends in research involving human subjects and animals. This advisory committee should be composed of leaders in research design and in the ethics of research who are capable of raising questions about new arenas for research and new approaches to research designs and to the protection of human subjects and animals. An advisory committee of research practitioners and scholars could ensure that the most current scientific debates and issues are brought to the attention of OPRR and could offer suggestions, comments, and criticisms about the operation of the office. This committee could review draft policies and pronouncements with OPRR and help it to anticipate the effect of new guidelines on the research community and on the agenda for the effective regulation of research with human subjects and with animals.

The advisory committee could also transmit to OPRR concerns of the research community about its positions or actions. It might assist OPRR to assess whether it is using its authority appropriately and creatively in support of ethical research designs and the proper conduct of research or whether it is creating increasing numbers of paper requirements that add to the burden of research administration without really enhancing protections for human subjects and animals. An advisory committee composed of recognized experts widely respected in their respective fields might also provide some barrier to the inappropriate incursions of the political system and might help to buffer inappropriate criticism of OPRR.

V. The authority presently delegated to OPRR is adequate for it to address the tasks currently assigned to it, but resources available to OPRR may be inadequate for fulfilling its mission.

The conduct of research involving human subjects and animals is currently regulated through the supervision of institutional review boards. OPRR promulgates guidelines and initiates new regulations relating to the activities of IRBs, the operation of institutional assurances, and the establishment and management of a process for responding to violations of the rights of human subjects and to violations of animal welfare. To carry out these responsibilities, OPRR has been delegated the authority to: initiate the process for promulgating regulations—subject to the departmental clearance process; negotiate and approve assurances of compliance; make determinations concerning noncompliance with previously negotiated assurances, including entering any restrictions or suspensions on such assurances; render authoritative interpretations of the Common Rule and the Policy for the Humane Care and Use of Laboratory Animals; issue advisory interpretations to other federal departments and agencies; identify locations and agendas for site visits and inspections; and develop and conduct educational workshops. OPRR has available one enormously powerful intervention, used infrequently, which is to suspend all federally funded research involving human subjects, or in other circumstances, involving animals, in an institution that OPRR finds is out of compliance with the regulations. This authority is well known and respected by the research community, and is thought to be a substantial factor motivating institutional compliance with the relevant legal requirements.

Based on its discussions with OPRR, the NIH legal advisor, and other experts from within and outside the government, the Review Panel concluded that OPRR has sufficient legal authority to carry out an active and effective agenda to ensure the protection of the human subjects and animals participating in federally funded research. As discussed in Section I of this report, OPRR's current location deprives it of the stature necessary to be as effective as its legal authority would seem to allow. The structural recommendations contained in this report are aimed at resolving that deficiency. Notwithstanding the availability of adequate legal authority, the Review Panel has become aware of thoughtful critiques of the manner in which OPRR exercises its authority. These critiques question the effort and resources devoted to routine negotiation and approval of assurances at the expense of educating institutions and IRB members about the legal and ethical requirements governing the involvement of humans and animals in research, and reviewing the operation of IRBs for compliance and efficacy. (See, e.g., Department of Health and Human Services, Office of Inspector General, Institutional Review Boards: A Time for Reform, 19-21 and App. D-5 (June 1998)). It appears to the members of the Review Panel that critiques of this nature merit serious consideration by the Secretary and by OPRR, whatever decision is made about the organizational location of OPRR. If a decision were to be made to change OPRR operations in the manner these critiques suggest, additional budgetary and personnel resources would be required to render OPRR effective in this more active mode. In any event, critiques of OPRR make clear that OPRR needs to exert more leadership in education and training, and in providing technical assistance to, and evaluation of, IRBs. Wherever OPRR is located, more resources will need to be made available to enable OPRR to fulfill the promise of even the current statutory and regulatory scheme.

CONCLUSION — An Opportunity for Federal Leadership

Biomedical research has expanded our knowledge of health and disease, and has reduced the burden of illness and disability, and improved quality of life of persons throughout the world. The United States is the unqualified leader in biomedical research, and has played an important role in defining ethical standards for the conduct of research involving human subjects and animals. OPRR plays a unique role in the planning for, and in the conduct and supervision of, research in this nation. It is the location in the federal government that is charged exclusively with the obligations to oversee the ethical conduct of research, the rights and welfare of human subjects, and the welfare of animals. OPRR is the repository of the nation's moral concerns about ethical conduct of research with human subjects and animals. It is charged to recognize and respond to the ethical dilemmas created by clinical research, including a central concern which is the use of people, who may or may not receive individual benefit, for the development of knowledge and benefit of society and other individuals. OPRR plays a critical role in the regulatory scheme and the ethical consciousness of the national research community. A relocated and more clearly independent OPRR could serve as a forum for discussion and debate on new ideas and creative ways to protect human subjects and the welfare of animals.

Research with human subjects will only become more ethically complex in the future. For example, genetics research will involve politically challenging protocols from the perspective of the protection of human subjects. Strong leadership of OPRR, whose expertise and fairness are acknowledged and whose guidance is sought by the research community and by groups of discussants or dissidents in new debates, can assist in developing approaches to these issues that appropriately balance the interests of human subjects with the individual and social benefits of research.

The leadership of an independent and strong OPRR is uniquely positioned to help overcome the barriers of distrust so that the data derived from human subjects research have the broadest applicability and relevance. A strong OPRR which is also recognized as a leader in discussions about the welfare of animals can be more effective with other agencies in forging rules that promote research and support a vibrant notion of animal welfare. Those advocates interested in the welfare of animals would appreciate a forum to discuss new possibilities for shielding animal subjects from unnecessary pain and suffering. Leadership in these sensitive arenas can be exercised only by persons of intellectual stature and moral standing. The recommendations developed by the Review Panel should enable OPRR to assume a leadership position in these critically important deliberations and debates.


OPRR Location Review Panel

Nancy Neveloff Dubler, LL.B. (Co-Chair)
Director
Division of Bioethics
Department of Epidemiology and Social Medicine
Montefiore Medical Center
Bronx, New York

Renee M. Landers, Esq. (Co-Chair)
Ropes & Gray
Boston, Massachusetts

Baruch A. Brody, Ph.D.
Director
Center for Medical Ethics and Health Policy
Baylor School of Medicine
Houston, Texas

Ralph B. Dell, M.D.
Director
Institute for Laboratory Animal Research
National Academy of Sciences
Washington, D.C.

Ruth Macklin, Ph.D.
Professor of Bioethics
Albert Einstein College of Medicine of Yeshiva University
Bronx, New York

June E. Osborn, M.D.
President
Josiah Macy, Jr. Foundation
New York, New York

Staff Director: Terrie Wetle, Ph.D.
Deputy Director
National Institute on Aging


OPRR Location Review Panel Interviews

 
Dr. Harold Varmus
Director, NIH
Full Committee

Dr. Gary Ellis
Director, OPRR
Full Committee

Dr. Tom Puglisi
Division of Human Subjects Protections, OPRR
Full Committee

Dr. Gary Chadwick
President, ARENA
Full Committee

Mr. Robert Lanman
NIH Legal Advisor, OGC/DHHS
Full Committee

Dr. Mark Yessian
Regional Inspector General for Evaluation and Inspections, DHHS
Region I Boston Office
Co-Chair

Dr. David Satcher
Assistant Secretary for Health and Surgeon General, DHHS
Co-Chairs

Dr. John Mahoney
Management Consultant
Full Committee

Dr. Michael Gottesman
Deputy Director for Intramural Research, NIH
Full Committee

Dr. Wendy Baldwin
Deputy Director for Extramural Research, NIH
Full Committee

Dr. Dixie Snider
Associate Director for Science Policy, CDC
Co-Chairs

Dr. William Raub
Deputy Assistant Secretary for Science Policy, DHHS
Co-Chairs

Dr. Lily Engstrom
Senior Policy Analyst, DHHS
Co-Chairs

Dr. Stuart Nightingale
Associate Commissioner for Health Affairs, FDA
Co-Chairs

Dr. Sharon Holston
Deputy Commissioner for External Affairs, FDA
Co-Chairs