Session 7 – Cell & Gene Therapies
Date: 26 July (Friday) 9:00 – 12:00 Venue: 3F, Bldg. A, CTBC Financial Park, Ballroom A
China Biotech Services Holdings Limited
Dr. Michael Yao is former senior Medical officer in USFDA. He has been licensed as Federal physician. He is senior clinical faculty in national naval hospital and national institute of health. He received USFDA Outstanding Career Service Award in 2017. He is members of American Medical Association, American Academy of EMG, American Society of Neuroscience, American Academy of Microbiology, American College of Rheumatology and American Pain Society.
During the USFDA, Dr. Yao was responsible for the evaluation of clinical trials of a wide variety of investigational cellular and gene therapy biologic products including device/biologic combination products used for the treatment of a wide range of clinical indications. He had provided comprehensive reviews and comments of products designed to treat these indications as a senior clinical reviewer and expert for all cellular and gene therapies for a variety of indications including rare disease, pediatric and oncology indications. He was a major member of the team to draft cellular and gene therapy guidance for multiple indications. He also provided guidance to sponsors before and during the clinical studies, focusing on clinical trial design, protection of human subjects and efficient product development in communications through pre-IND, pre- IDE, pre-510(k) and pre-BLA meetings, and in the review of INDs and IDEs, 510(k) s and BLAs.
In the FDA regulatory prospective, Dr. Yao was a major advisories and chairpersons of FDA internal working groups and committees, such as FDA Rheumatology Working Group (RA/OA/SLE-guidelines), FDA Cartilage Repair Working Group (Co-Chair for CBER), FDA/OWH Science Review Committee and FDA Multi-Center Regenerative Medicine Working Group, etc. Dr. Yao had been as advisories of federal and congress committees for national health care, national security and Alzheimer disease. Dr. Yao had been involved in finalizing“the 21 st Century Cures Act” in 1996. Dr. Yao also have been involved in a variety of academic activities in teaching and training programs of regulatory science, tumor immunology therapy, stem cell therapy and gene therapy, as a senior faculty and/or advisor in the NMPA and the Chinese Academy of Engineering. Currently, he is visiting professors in Beijing University and Tsinghua University. He has appointed as CEO and CMO in Bioceltech Therapeutics, Ltd. He, also, is Co-Chairman of Gem Flower Pharma Tech (Beijing) Limited. Session Speech Title & Synopsis: USFDA Regulatory Perspectives for Cancer Immunotherapy
This presentation majorly proposes to discuss: 1) basis for US regulatory approvals; 2) overview of US FDA cancer immunotherapy approvals; 3) regulatory perspectives for clinical considerations on cancer immunotherapy. For product approvability, generally clinical data should demonstrate substantial evidence to support the claims of effectiveness that should base upon 1) adequate and well-controlled clinical studies; 2) acceptable safety (ratio of benefit-risk). Accelerated approval pathway usually apply for oncological indications, which may use a surrogate endpoint that is reasonably likely, based on epidemiologic, therapeutic, pathophysiologic, or other evidence, to predict clinical benefit, or on the basis of an effect on a clinical endpoint other than survival or irreversible morbidity. However, post-marketing study will be required to verify its clinical benefit. Important CAR-T Clinical Considerations include: 1) challenges in enrolling patients with different tumor histology;2) prior treatment requirement; 3) Patient performance and organ function; 4) companion diagnostic for identifying targeted antigen;4) because of toxicity concern, unclear role of CAR-T in earlier-stage disease. In summary, 1) cancer Immunotherapy has evolved tremendously and many life-saving immunotherapies have been approved within the last decade; 2) novel and better science has been the driving force for this evolution; 3) many regulatory challenges exist, especially in the era of novel therapies (e.g., CAR-T, OVT and neoantigens), their combinations and biomarkers; 4) interaction and collaboration of stakeholders are key for future success.