Abstract
Challenges to the adequacy of the blood supply necessitate innovation in the development of safe and effective alternatives to traditional transfusion blood products. In the United States, the development of such products occurs in the context of regulatory oversight by FDA. The statutory standard for approval of a drug or biological drug is “substantial evidence of effectiveness” which is determined through the conduct of “adequate and well controlled clinical trials.” FDA has provided extensive guidance on the design of clinical trials that meet these statutory and regulatory requirements. FDA has recognized the need to expedite the development and review of drugs/biological drugs when the disease or condition being treated is serious or life-threatening, and has provisions and programs for developing products for rare diseases. In addition to these programs, FDA has also developed programs for making drugs/biological drugs available earlier under a number of programs that include Expanded Access, Emergency Use Authorization, and “Right to Try.” This chapter reviews the current landscape of programs potentially available for the development of alternatives to red blood cells when red blood cell transfusion is indicated but is not possible.
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Silverman, T.A. (2022). Regulatory Perspectives on Clinical Trials for Oxygen Therapeutics When Transfusion of Red Blood Cells is Not an Option. In: Liu, H., Kaye, A.D., Jahr, J.S. (eds) Blood Substitutes and Oxygen Biotherapeutics. Springer, Cham. https://doi.org/10.1007/978-3-030-95975-3_43
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