Judith Arcidiacono

US Food and Drug Administration international collaborations for cellular therapy product regulation

Cellular therapy products are an emerging medical product class undergoing rapid scientific and clinical innovation worldwide. These products pose unique regulatory challenges both for countries with existing regulatory frameworks and for countries where regulatory frameworks for cellular therapy products are under development. The United States Food and Drug Administration (US FDA) has a history of productive working relationships with international regulatory authorities, and seeks to extend this to the cellular therapy field. The US FDA and its global regulatory counterparts are engaged in collaborations focused on the convergence of scientific and regulatory approaches, and the education of scientists, clinicians, regulators, and the public at large on the development of cellular therapies.

Regulation of xenogeneic porcine pancreatic islets

Arcidiacono JA, Evdokimov E, Lee MH, Jones J, Rudenko L, Schneider B, Snoy PJ, Wei C‐H, Wensky AK, Wonnacott K. Regulation of xenogeneic porcine pancreatic islets.
Xenotransplantation 2010; 17: 329–337.

Overview of the FDA Regulatory Process

Publisher Summary This chapter provides a brief historical review of the Food and Drug Administration (FDA) and its organizational structure and discusses topics pertaining to the regulation of regenerative medicine products including possible regulatory pathways for combination products and relevant jurisdictional issues. FDA regulations are contained in the Code of Federal Regulations (CFR). Regulations for drugs, biologics, devices, and tissues, along with related regulations, may be found in various parts of Title 21 of the CFR. Guidance documents are nonbinding publications that describe the FDAs interpretation of policy pertaining to a regulatory issue or set of issues related to the design, production, labeling, promotion, manufacturing, and testing of regulated products, the processing, content, and evaluation, or approval of submissions inspection, and enforcement policies. Guidance documents, which are developed in accordance with Good Guidance Practices found at 21 CFR §10.115, are intended to clarify the FDAs current thinking related to regulatory issues and procedures. The FDA has issued “Guidance for Industry: Eligibility Determination for Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products to assist establishments making donor eligibility determinations with complying with the Donor Eligibility rule (21 CFR 1271 Subpart C).” This guidance also incorporates and finalizes the content of “Guidance for Industry, Preventive Measures to Reduce the Possible Risk of Transmission of Creutzfeldt–Jakob Disease (CJD) and Variant Creutzfeldt–Jakob Disease (vCJD) by Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps).” The US Public Health Service (PHS) agencies including the FDA, National Institutes of Health (NIH), Centers for Disease Control and Prevention (CDC), and Health Resources and Services Administration (HRSA) have worked together to address the risk of infectious disease transmission, publishing the “PHS Guideline on Infectious Disease Issues in Xenotransplantation.”

Translation of Regenerative Medicine Products Into the Clinic in the United States: FDA Perspective

Abstract The field of regenerative medicine encompasses a breathtaking array of interdisciplinary scientific approaches with the promise of delivering future therapies to meet current unmet medical needs for patients. Increasingly more of these innovative products are being translated into human clinical trials in the United States, and general familiarity of the FDA is important to efficiently navigate the process. The basics of FDA history, organization, and processes are described herein for those new to clinical translation, with more detailed content added regarding approval pathways, regulations, guidances, and select special topics of relevance to regenerative medicine. In addition to the cumulative experience of previous products, the FDA regulatory approach to medical products evaluation includes an ongoing assessment of how the science of those products informs regulatory policy. FDA engages in ongoing dialogue with the scientific community and product sponsors to continue to develop science-based regulatory review policies that are robust and predictable in order to meet the needs of the challenging array of products that are on the horizon.

Chapter 41 – Overview of the FDA Regulatory Process

Regenerative medicine encompasses a breathtaking array of interdisciplinary scientific approaches which address a broad spectrum of clinical needs. Recent advances in scientific knowledge related to cell biology, gene transfer therapy, biomaterials, immunology, and engineering principles applicable to biological systems place this community in a position to address a number of challenging and critical health needs. These include treatment of disease conditions resulting from pancreas, liver, and kidney failure; structural cardiac valve repair; skin and wound repair; and orthopedic applications. Scientific challenges include expanding the knowledge base in each discipline as well as developing an interdisciplinary approach for identifying and resolving key questions. The Food and Drug Administration’s (FDA’s) regulatory review process mirrors the scientific challenges with regard to the development of review paradigms that cross scientific disciplines. This chapter provides a brief historical review of the FDA and its organizational structure, and discusses topics pertaining to the regulation of regenerative medicine products.

FDA and NIST collaboration on standards development activities supporting innovation and translation of regenerative medicine products

The development of standards for the field of regenerative medicine has been noted as a high priority by several road-mapping activities. Additionally, the U.S. Congress recognizes the importance of standards in the 21st Century Cure Act. Standards will help to accelerate and streamline cell and gene therapy product development, ensure the quality and consistency of processes and products, and facilitate their regulatory approval. Although there is general agreement for the need of additional standards for regenerative medicine products, a shared understanding of standards is required for real progress toward the development of standards to advance regenerative medicine. Here, we describe the roles of standards in regenerative medicine as well as the process for standards development and the interactions of different entities in the standards development process. Highlighted are recent coordinated efforts between the U.S. Food and Drug Administration and the National Institute of Standards and Technology to facilitate standards development and foster science that underpins standards development.

Chapter 83 – Overview of the FDA Regulatory Process

This chapter provides a brief historical review of the Food and Drug Administration (FDA) and its organizational structure and discusses topics pertaining to the regulation of regenerative medicine products including possible regulatory pathways for combination products and relevant jurisdictional issues. FDA regulations are contained in the Code of Federal Regulations (CFR). Regulations for drugs, biologics, devices, and tissues, along with related regulations, may be found in various parts of Title 21 of the CFR. Guidance documents are nonbinding publications that describe the FDAs interpretation of policy pertaining to a regulatory issue or set of issues related to the design, production, labeling, promotion, manufacturing, and testing of regulated products, the processing, content, and evaluation, or approval of submissions inspection, and enforcement policies. Guidance documents, which are developed in accordance with Good Guidance Practices found at 21 CFR §10.115, are intended to clarify the FDAs current thinking related to regulatory issues and procedures. The FDA has issued “Guidance for Industry: Eligibility Determination for Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products to assist establishments making donor eligibility determinations with complying with the Donor Eligibility rule (21 CFR 1271 Subpart C).” This guidance also incorporates and finalizes the content of “Guidance for Industry, Preventive Measures to Reduce the Possible Risk of Transmission of Creutzfeldt–Jakob Disease (CJD) and Variant Creutzfeldt–Jakob Disease (vCJD) by Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps).” The US Public Health Service (PHS) agencies including the FDA, National Institutes of Health (NIH), Centers for Disease Control and Prevention (CDC), and Health Resources and Services Administration (HRSA) have worked together to address the risk of infectious disease transmission, publishing the “PHS Guideline on Infectious Disease Issues in Xenotransplantation.”