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All Times EDT

Wednesday, September 23
Wed, Sep 23, 1:30 PM - 2:45 PM
Virtual
Real-World Data in the Lifecycle of Drug Development for Rare Disease

Using Historical Control for Regulatory Approvals in US: A Practical Review Focusing on Diseases in Small Population (301162)

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*Ziliang Li, Vertex Pharmaceuticals 
Chenkun Wang, Vertex 

Keywords: Historical Control, single-arm, regulatory submission

In life-threatening/rare diseases, randomized controlled trial often runs into feasibility and/or ethical issue. Single-arm interventional trial with historical control (HC) as the comparator, highlighted in the recent FDA Real World Evidence framework, provides an alternative approach to assess the effectiveness of the investigative therapy in these challenging scenarios. In recent years, quite a few novel therapies for rare diseases and in oncology for small population were developed utilizing historical data and several gained approvals. In this presentation, we will review a selected set of drug development programs/filings which used HC in the pivotal studies and discuss the challenges/disadvantages of using HC and the mitigation strategies.