Human Tissue as a Biologic Drug: The Pathway to BLA (Individual)

This session will discuss the unique developmental and regulatory considerations of tissue products regulated as biologics and, therefore subject to Section 351 of the Public Health Service Act (PHS). The speakers will discuss the many clinical, regulatory, and legal requirements demanded of these products that distinguish them from other products.

In addition to complying with 21 CFR 1271 as an HCT/P, tissue products regulated as biologics also meet the Food, Drug, and Cosmetic Act’s definition of a drug. Therefore, not only must these products comply with the donor eligibility requirements and current Good Tissue Practices (cGTPs) outlined in 1271, but they must also comply with current Good Manufacturing Practices (cGMPs) in 21 CFR 210 and 211 and the Quality System Regulations in 21 CFR 820. These products must also demonstrate identity, purity, potency, and safety as per 21 CFR 600. Safety and efficacy must be proven in the clinical setting. Before being introduced into the clinical setting, a successful Investigational New Drug (IND) application must be in effect. The many requirements of 21 CFR 312 must be followed as the product moves from Phase I through Phase 3 clinical trials. If a sponsor believes their product has demonstrated both safety and efficacy in the clinical trials, they will submit a Biological License Application (BLA) to the FDA. As part of the BLA application, a pre-market approval inspection is conducted. If a BLA is granted, a sponsor may begin to lawfully market their product. The sponsor may be required to launch a Phase 4 post-marketing study to gather additional efficacy data in specific populations, examine side effects associated with long-term use, and/or additional safety information.

Melissa O'Connor, MS, CTBS, RAC, F-RAPS

Chief Quality & Regulatory Officer

StimLabs, LLC

Melissa has 20+ years of experience in medical device, combination product, and human tissue new product development research, engineering, and commercialization. She has experience in Quality System design, implementation, maintenance, and compliance. Melissa has held Quality leadership positions serving as the Management Representative, Head of Quality and official FDA correspondent for prescription eye-glass lens manufacturer QSpex Technologies, Inc., and Chemence Medical Products, Inc, a global manufacturer of adhesive-based Class II medical devices for would closure. During an 11-year tenure with Kimberly-Clark Health Care, Melissa held positions in Regulatory Affairs, Product Development, and Process Engineering. In addition to Quality Systems expertise, Melissa is skilled at developing comprehensive regulatory strategies to support new product development, including pre-market approval requirements for U.S. and E.U. She is a member of the Regulatory Affairs Professional Society (RAPS) and holds an active RAC certification. Melissa holds a B.S. degree in Biological Sciences from the University of Georgia and an MS Degree from the University of Georgia School of Pharmacy in Biomedical and Pharmaceutical Regulatory Affairs. Melissa is a founding member and currently serves as the Chief Quality and Regulatory Officer for StimLabs, LLC based in Roswell, Georgia. In her current role, Melissa is responsible for all aspects of Quality and Regulatory Affairs for StimLabs including Quality Assurance, Quality Control, Quality Engineering, Quality Management Systems, Donor Services, and global regulatory affairs. Melissa is a member of the AATB Quality Counsel and has been serving as a member of the AATB program committee for the last three years and is the chair of the committee this year. In collaboration with AATB council membership and AATB Liaisons, Melissa was responsible for development of the agenda and speaker content for this year’s meeting.

Barbara Bardaro, Ph.D.

With almost 20 years of experience in biomedical translational research and a Ph.D. in Cell Biology and Biotechnology, Barbara has extensive expertise in biologic drug development. After obtaining her Ph.D. at the University of Messina in Italy, Barbara went on to hold numerous positions within the University of Illinois at Chicago’s Department of Surgery. While there, her roles within the Islet Transplant Program included Director of Regulatory Affairs, Chief of Operations, and Director of Quality Assurance. Through these roles, she used her vast knowledge of biologic drug development to successfully coordinate regulatory submissions for a human islet cell therapy as it moved from the pre-clinical stage to IND submission through Phases 1, 2, and 3 clinical trials, and ultimately a BLA.

After her work at the University of Illinois at Chicago, Barbara took on various leadership roles in Quality, Manufacturing, and Operations. Barbara is a Current Good Manufacturing Practices (CGMP) Certified Professional and a Chemistry, Manufacturing, & Controls (CMC) Certified Professional, which, along with her education and experience in the industry, make her the consummate expert in the complicated and challenging field of cell-based biologics development.