Workshop 2 – Clinical Trials for Advanced Therapies
Date: 24 July (Friday) 12:20 – 13:50 (GMT+8)
Venue: www.bioasiataiwan.com / Online event platform
Dr. Kazempour, President & CEO of Amarex Taiwan and its US affiliate, co-founded Amarex Clinical Research in 1998, a full-service contract research organization providing services to the pharmaceutical, biotech, and device communities. He serves on the George Washington University Regulatory Affairs Advisory Board, and teaching at George Washington University Medical Center and Montgomery College.
Dr. Kazempour has conducted research activities with the National Institutes of Health, pharmaceutical industry and various universities. While at US FDA as a reviewer, he received several awards for innovative problem solving and teamwork along with contributions to the drug approval process. During his tenure at the FDA, Dr. Kazempour also worked as a Senior Staff Fellow and as a Mathematical Statistician responsible for supervising and conducting independent statistical analyses of clinical trials and reviewing submitted protocols and Clinical Study Reports.
Dr. Kazempour’s experience in the industry and FDA has allowed him to understand biometrics, quality assurance and quality control from both a sponsor's perspective, as the provider of the data and from a regulatory perspective, as the reviewer of the data. He has worked in many therapeutic areas including cell/gene therapy, vaccines, anti-virals, CNS, and oncology as well as medical devices and digital health, as his expertise and knowledge has been a key contributor in the approval of these drugs, biologics and devices.
Dr. Kazempour has spent the past 17 years focusing on international drug and device clinical development and has significant experience working with Asian clients. He has successfully assisted Asian clients with different FDA submissions, including IND, IDE, 505(b)2, 510k, PMA, and NDA for botanical, chemical, biological, and clinical device products.
Speech title & Synopsis
The design of clinical trials for advanced therapy products often differs from other types of pharmaceutical products. FDA’s major concerns for early clinical phase trial is safety, to identify a safe does, such as maximum tolerated dose. Advanced therapy products may have little or no toxicity; therefore, using biomarkers, such as engraftment and transgene expression may be used as an alternative for primary endpoints. In this talk we focus on the challenges at the design and analysis stages of clinical development for advanced therapy products. Obviously, there are multiple regulatory opportunities that we recommend you to take advantages of them at different stages of your product development, such as Regenerative Medicine Advanced Therapy (RMAT) Designation. This designation allows you to work closely with the FDA on the program development and give you ample opportunity to have early interaction with the review Division/FDA reviewers to discuss any potential surrogate endpoints or other intermediate endpoints (i.e. Biomarkers) for your primary endpoint. One of the main challenges at the design and analysis stages is the sample size and power calculation to establish the efficacy. We have helped many biotech companies to achieve their goals in the development of marketing approval applications through the use of the adaptive trial designs that will be discussed during this presentation. Only by identifying these challenges of innovative and complicated clinical trial designs, we can offer strategies from different perspectives to overcome these challenges.