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Activity Number: 439 - Role of External Evidence in Drug Development: Current Practices and Future Perspectives
Type: Topic Contributed
Date/Time: Thursday, August 6, 2020 : 10:00 AM to 11:50 AM
Sponsor: Biopharmaceutical Section
Abstract #312309
Title: Statistical Innovation in Rare Disease Clinical Development
Author(s): Kannan Natarajan*
Companies: Pfizer Pharmaceuticals Inc.
Keywords: Innovative design; Bayesian; small population

Traditional statistical principles are often not ideal to establish the benefit risk of a new drug in rare disease. With a significant unmet medical need in some of the diseases, it is unethical and often not feasible to conduct large trials with either placebo or active controls. Different strategies of using a synthetic control in clinical trial design will be highlighted with possible pros and cons. In addition, the talk will elaborate an example on the need for replacing traditional study endpoints and testing procedures with innovative methods that could be significantly efficient in assessing the treatment benefit.

Authors who are presenting talks have a * after their name.

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