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Activity Number: 570 - Addressing the Unique Analytic Challenges of Drug Development in Rare Diseases with the Use of Innovative Designs and Real World Evidence in Clinical Trials
Type: Topic Contributed
Date/Time: Thursday, August 6, 2020 : 3:00 PM to 4:50 PM
Sponsor: ENAR
Abstract #312374
Title: Statistical Issues in Trials for Rare Diseases Using Innovative Design and Real-World Evidence – Regulatory Experiences
Author(s): Aloka Chakravarty*
Companies: Office of Biostatistics in CDER, FDA
Keywords: Real-World Evidence; Innovative Design ; Rare Diseases

There has been considerable interest and discussion on the role of real-world evidence (RWE) and use of innovative designs in trials for rare diseases. The acceptability of them in regulatory decision making has many statistical considerations. In this presentation, we will discuss some regulatory experience in this area, along with case examples from the Complex Innovative Design (CID) Pilot program as well as the Critical Path Innovation Meetings (CPIM) submitted to the US Food and Drug Administration. In addition, some lessons learnt on best practices going forward will also be shared.

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

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