Protecting Research and Research Participants
The public has a strong interest in protecting research and research participants. Federal policy must find the right balance in protecting participants while allowing critical research to continue.
BIO companies' research leads to cures and treatments for disease. Today, there are 168 biotech products on the market and they have helped a quarter billion people worldwide. Another 371 biotech medicines in late stage development are targeting more than 250 diseases.
This research cannot go forward without the human subjects who volunteer to participate. Research depends on participants' good will. Some may be just curious and interested in helping, while others may be vulnerable or scared, in turning to the unknown for help for their disease. In addition to facing their disease, in many situations, a primary motivation is to help find cures for the next generation of people with their disease. Patients deserve respect and admiration, and protection from inappropriate risks that they may be in no position to evaluate.
BIO has long advocated protection for research participants. Its Statement of Ethical Principles, drafted by its Bioethics Committee and adopted in 1997, pledges adherence to strict informed consent procedures to ensure that research participants are fully aware of the potential risks and benefits of that research.
In addition, two years ago, BIO adopted "Principles for Federal Law To Protect Research and Research Participants." BIO stated that its interest in adopting the principles was "to protect the strong public interest in the results of research and public confidence in the research process." These Principles have provided BIO with a basis for participating in what it hoped would be an active legislative and regulatory agenda strengthening and standardizing the federal laws protecting human subjects so that they apply to all research no matter what funding source.
Since then, legislators have debated the relevant issues, and BIO has provided Congressional testimony based on these Principles. Data privacy regulations have been promulgated, and BIO has provided comments and obtained important revisions based on these Principles. However, BIO companies are taking the initiative in finding ways to reassure the public that federal law establishes strong and uniform protection for research and research participants.
BIO now reconfirms its commitment to these principles and restates them in a form that researchers and research companies can look to in their conversations with research participants. Although most of these principles reflect the established practices of responsible researchers, the current system does not readily provide a basis for sponsoring companies to affirm them. The current system of ethical review that scripts communications with human subjects is, for better or worse, grounded in provider institutions' obligations as recipients of federal grants - the biotechnology sponsor's commitments are evident only in its behind-the-scenes activities in relating to the provider institution and in its submissions to the Food and Drug Administration.
By reaffirming these principles, BIO offers sponsoring companies an opportunity to communicate directly with research participants regarding: (1) the researchers' appreciation of participants' contribution to science; (2) the arrangements that researchers make to protect participants' rights and well-being; and (3) the ways that researchers protect the value of participants' contributions by safeguarding the quality and integrity of research results. BIO hopes that this will facilitate more open communication between researchers and participants that will protect research by justifying participants' trust and confidence in the shared effort to deal with the uncertainties and risk that are a necessary part of activities on the frontiers of science
Human Subject Research Is Grounded In Trust And Respect
- BIO companies' research leads to cures and treatments for disease. Today, there are 168 biotech products and they have helped a quarter billion people worldwide. Another 371 biotech medicines in late stage development are targeting more than 250 diseases.
Innovations that lead to the discovery and development of treatments for unmet clinical needs cannot proceed without three kinds of contributions, each involving its own kind of risk and uncertainty -
- The good will of individuals who volunteer to be subjects in testing new products;
- The creativity, insight, and hard work of individual scientists who pursue novel hypotheses over many years; and
- The long-term commitment of financing by investors, both public and private.
The strong public interest in the improved health and health care made possible by biotechnology research commands us to act in ways that justify the role played by all of the relevant individuals and groups:
- Research participants - individuals whose condition or disease is of interest, individuals who possess clinical or genetic attributes of interest to the researcher, and "healthy" volunteers who help in early phases of clinical research;
- Investigators -- clinicians, health care providers and scientists who assume responsibility for the care of research participants enrolled in a research protocol, and/or the analysis of research data;
- Research institutions -- hospitals and clinics (whether private or government operated) that provide the clinical setting in which investigators tend to the needs of research participants; and
- Research sponsors -- persons (whether individuals, institutions or corporations) that assume responsibility for conduct of research and data analysis, including research supporting submission of an application for approval of a new drug, device or biologic, as well as post-market testing of products.
- Where relevant to the participant's health care and medically appropriate for use in health care decision-making, sponsors should permit investigators and/or research institutions that have direct treatment relationships with a participant to communicate, at an appropriate time and in an appropriate manner, results of the research with the study participant.
Protecting The Contribution Of Research Participants Transcends National Borders
- No matter what country hosts clinical research, data collection or analysis, sponsors and investigators should protect and value the contribution of each research participant.
- Clinical trials involving the administration of a new drug to a living human being should be conducted in accord with the Good Clinical Practices developed through the International Conference on Harmonization (ICH-GCP).
- Clinical research should be conducted in accord with applicable local laws. In the U.S., the Federal Food and Drug Administration provides Good Clinical Practice Guidelines, and FDA regulations assure that ethics review is provided by Institutional Review Boards operating under the "common rule" codified at 21 CFR parts 50 and 56.
- Even where these laws do not apply, investigators and sponsors should seek review of research involving more than minimal risk to a living human being. FDA regulations provide a clear definition of "minimal risk" by referring to the risks faced by a person in every day life.
- Where a research participant will be asked to face more than minimal risk, an ethics review board operating in accord with the laws of the host country should review the potential risks to participants, and balance them with possible benefits to participants and mankind.
Disclosure And Oversight By Institutional Review Boards Provides Critical Support For Participant's Informed Consent
- No research participant should be involved in research without his or her voluntary consent to a research protocol, after an informed discussion of its risks. FDA regulations provide for rare exceptions, such as research on products used in emergency interventions, or consent by a legal representative.
- A research participant should be allowed to withdraw consent at any time for any reason.
- A research participant's authorization or specification of limitations on permissible uses of data and biological materials must be respected.
- Wherever possible, any payment to research participants, including any changes to such payments, should be reviewed by an IRB to ensure that their structure or amount does not encourage participants to inappropriately disregard research risks.
- Investigators should be forthcoming regarding potential physical, social and psychological risks to the research participant that may be associated with a research protocol, including unanticipated risks that become apparent during the conduct of the research.
Conflicts Of Interest Should Be Identified And Appropriately Managed
- Investigators and sponsors of a clinical trial should be responsive and cooperative with institutions' efforts to identify financial or other interests that may create a conflict of interest, or the appearance of a conflict in reviewing research risks.
- Investigators should disclose to a reviewing IRB or other institutional oversight committee any financial or other interest in the research protocol that could have professional, personal, or material financial implications. Sponsors of a clinical trial should be forthright in responding to requests for information about financial or other interests of investigators that may create a conflict or appearance of a conflict in conducting research.
- Investigators should disclose to participants that they are compensated for conducting clinical trials. Payments to investigators and research institutions should not be contingent on the research outcome.
- Sponsors should not reward investigators or research institutions that obtain data, recruit or enroll research participants without adhering to the IRB-approved protocol establishing acceptable risks to research participants.
Investigators and sponsors should support a reviewing IRB's determination that an interest of an investigator or research institution presents a conflict of interest, and its recommendation for dealing with the conflict, whether the interest is
- Is one of the risks that should be disclosed to research participants during the informed consent process, or
- Is an interest that can be managed by the IRB through research process and oversight.
Protecting The Contributions Of Investigators And Sponsors Requires Fairness And Foresight
- Investigators conducting clinical trials for marketing and approval cannot be compensated by sponsors with equity interests.
- Subject to arrangements for protecting participants from conflicts of interest in the conduct of a research protocol, individual scientists should be allowed to hold equity interests, directly or indirectly, in companies that sponsor or invest in research protocols. These interests should be fully disclosed to the IRB.
- Sponsors should be forthcoming in disclosing results that are of material significance for the health and health care of individuals.
- Subject to the rights of research participants to be free of unwanted publicity or interference, consents, authorizations and rights in research data shall be designed to assure investigators and sponsors the maximum value of their contributions for future research and analysis.