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Abstract:
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Shortly after joining FDA as the Director of the Office of Biostatistics in CDER, I prepared a talk and later, a publication, on the role of statistics in regulatory decision-making based on my first impressions. This work included a description of the many challenges in drug regulation and made a case for the importance of statistics in facing those challenges. This past year, I completed five years on the job and spent some time revisiting my first impressions. In this JSM presentation, I will recount some experiences from those five years, focusing on areas where statistics has had a positive impact on the work of FDA. I will also outline the major challenges that I believe still exist and the steps we need to take to meet these challenges more effectively in the next five years. Specific topics will include issues in biosimilar product development, difficulties in assessing risks of rare events for marketed products, the ever-present challenges that subgroup inference poses, the importance of and difficulties in conducting multi-regional trials for a global marketplace, the heartbreak inherent in studying and regulating products for rare diseases, and an attempt to answer the question, "Is it time for Bayes methods to be seriously considered by CDER?" Training and skill sets statisticians must master to be ready for the future will also be discussed.
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