Ethical and Human Subject Protection
- Institutional Review Board (IRB) review of protocols
- Informed Consent
Under FDA regulations, a protocol under which an individualized ASO drug product is administered to a human subject must be reviewed by an IRB (21 CFR Part 56), which must fully evaluate the protocol and ensure that risks to the subject are reasonable in relation to the anticipated benefits.
The IRB should be provided with the results of all relevant non-clinical safety studies in animals that have been conducted. If the ASO drug product will be administered to a child, the IRB must ensure that the protocol complies with the requirements under FDA’s regulations on human subject protection (21 CFR part 50), subpart D.
Under FDA regulations, informed consent must be obtained under circumstances that provide prospective participants, or their legally authorized representatives, sufficient opportunity to consider whether to participate and that minimize the possibility of coercion or undue influence (21 CFR 50.20). The sponsor should include a copy of the informed consent document in the original IND submission.
The informed consent document and the consent discussion should appropriately emphasize in a clear manner that the ASO drug product is experimental, the reality that the benefit is uncertain, and the potential risks are unknown, and that additional costs to the participant may be associated with the administration of the drug product. When appropriate, the consent document and consent discussion should include information that the administration of the ASO drug product will be the first use in humans of the investigational drug and relevant information from nonclinical safety studies in animals that have been conducted that could potentially inform the safety of the participant.