Tutorial- Wednesday 8:30 AM EDT
Title: Statisticians Face Challenges and Opportunities in Drug Development
Presenters: Lei Nie, Division of Biometrics II, Office of Biostatistics, OTS/CDER/FDA

Abstract:

Methodologies for drug development have evolved markedly in the past several years. Breakthroughs have been seen in the treatment of cancer and rare diseases, facilitated by medical and statistical research on biomarkers and surrogate endpoints. In addition, the goals for future drug development have become more nuanced, driven by improvements in statistical methodology and biotechnology, and trending toward patient-focused treatments and precision medicine, with formal benefit-risk analysis integrated into the review process. Newly arrived digital health technologies are expected to facilitate the implementation of decentralized clinical trials, with increasingly diverse trial participants. Statistical methodologies are advancing the use of real-world evidence to supplement substantial evidence of efficacy and to enhance drug safety. Further, novel methods, including complex and innovative designs, machine learning, and artificial intelligence, are expected to improve the efficiency of drug development, All of these changes, many of which reflect statutory mandates, demand timely responses from the FDA even while the agency is dealing with the global pandemic. In this presentation, we discuss the challenges and opportunities in drug development.


Bio:
Lei Nie is an Associate Division Director of Division of Biometrics II from the Office of Biostatistics, OTS/CDER/FDA. He is an elected fellow of the American Statistical Association. He authored/co-authored a total of 101 peer reviewed journal publications. He has contributed to dose finding and selection trials, noninferiority and biosimilarity trial design and analyses, and model informed drug development. He is interested in developing and promoting innovative statistical methods in drug development.