Bayesian statistics and clinical trial designs for human cells and tissue products for regulatory approval

摘要

IntroductionIn order to obtain premarket approval for medical products derived from human cells or tissues in the United States (US), the European Union (EU), and Japan, data from clinical trials are typically required to evaluate product efficacy and safety. Clinical investigators or study sponsors often face challenges when designing clinical trials on human cells and tissue products with the goal of obtaining premarket approval owing to the unique characteristics of products in this category. The methods used to administer, infuse and transplant these products vary more widely than the methods used for pharmaceuticals. In addition, final product quality may vary depending on the product source, i.e., patients or donors. These products are generally intended to treat intractable and rare diseases or injuries; therefore, it may not be possible to collect a sufficient number of cases and enrollment may be a long process. Moreover, since the technology for product development in this category is relatively new, knowledge and experience from previous studies are limited.