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U.S. National Institutes of Health

Federal Regulation of Tissue Research

Research on human tissue has led to significant improvements in medical treatment and in understanding the etiology of disease.1 Breakthroughs in molecular science now permit the study of the causes and pathways of disease, reinforcing hopes of earlier and more accurate diagnoses and allowing the possibility of developing individually tailored treatments and therapies. In parallel with scientific advances, extensive public discussion is under way among scientists, privacy advocates, patient groups, and ethicists over the collection, storage, and use of human tissue samples for research.

Tissue research involves a range of activities including the collection, storage, analysis, or transfer of tissue samples and data. A research study might collect samples and data in one state, transfer those samples and data to a central repository in another state, and then disclose the data to researchers in a third state. Molecular analysis of a tissue sample might be defined as a “use” of a sample or a “genetic test,” while sending the sample or research data to a collaborator in another state might constitute a disclosure of medical or genetic information. A routine research activity undertaken on a daily basis across the states could potentially involve laws governing informed consent, confidentiality of medical information, restrictions on the retention of genetic information, and human subject protections, using divergent standards in multiple states.

The questions surrounding the control and use of excised tissue and associated data focus on ways to facilitate important scientific research while upholding the key legal and ethical principles of individual autonomy and privacy. Specific issues include the conditions necessary for the disclosure of private medical information; whether each new use of human tissue specimens requires individual consent; the scope and specificity of that consent; and when deidentified or anonymous specimens and associated data2 may be used for research.

Research that is conducted or supported by the federal government, including research using human tissue specimens, residual diagnostic specimens, or medical information that is not otherwise exempt, is governed by the “Federal Policy for the Protection of Human Subjects”3 set forth in the Code of Federal Regulations. The Department of Health and Human Services (HHS) has codified the federal policy at 45 C.F.R. part 46, Subpart A (known as the “Common Rule”), that applies to HHS-funded research4 that is not otherwise exempt, and in 21 C.F.R. parts 50 and 56, which similarly govern research on products regulated by the Food and Drug Administration (FDA). According to HHS regulations at 45 C.F.R. 46.101(b)(4), the study or collection of “existing data” (including stored samples, records, pathological specimens, or diagnostic specimens) is deemed exempt from the requirements of the Common Rule, if these sources are publicly available or if the information is recorded by the investigator in such a manner that subjects cannot be identified, either directly or through identifiers linked to subjects.

Federal regulations covering protection of human subjects offer little overt direction regarding the use of human tissue for research. According to HHS regulations, tissue research is deemed human subjects research subject to federal regulations when identifiable private information is obtained about a living individual or when an investigator obtains data through interaction or intervention with an individual.5 The FDA regulations apply when research is conducted for an application that will be submitted to the FDA.

The substantive requirements under both sets of regulations are similar:6 The proposed research activity must be submitted to an institutional review board for scientific and ethical review (unless the activity is classified as exempt), and informed consent of the subject must be obtained or waived. FDA regulations do not permit waiver of informed consent for human subjects research, except under certain emergency circumstances. Certain types of research are eligible for expedited review according to the Office for Human Research Protections (OHRP) and FDA guidance,7 including the prospective collection of biological specimens by noninvasive means when taken for research purposes.8

Federal policy directs Institutional Review Boards (IRBs) to weigh the potential risks of the research, which may include risks to the privacy of the individual if personal information is disclosed. Federal policy for protection of human subjects explicitly does not preempt applicable state laws or local laws or regulations that provide additional protections for human research subjects.9

A new federal regulation, the “Privacy Rule,” established under the authority of the Health Insurance Portability and Accountability Act of 1996 (HIPAA) and implemented in April 2003,10 imposes additional limitations on the research uses and disclosures of identifiable patient information. While the Privacy Rule does not cover tissue specimens per se, the data associated with tissue specimens may be considered protected health information and therefore may be subject to restrictions on use and disclosure. In general, HIPAA requires patient authorization for specific uses of protected health information, although certain exceptions apply.11 HIPAA does not preempt state laws that impose more stringent protection on individually identifiable health information, but rather defers to the higher standard of privacy protection.12

The federal regulations for protection of human research subjects and the federal Privacy Rule explicitly state that they do not affect state or local laws or regulations which otherwise apply and which provide additional protections.13 As a result, when conducting research activities in certain states, researchers need to be aware of state laws that impose additional requirements beyond those set forth in the federal regulations. This survey will review the laws that impose additional requirements in each state and will provide an explanation of the purpose of these laws and their protections.