Using Historical Data in Developing Novel Therapies: A Roadmap and Case Studies

• Activity Number: 45 - Statistical Methods Utilizing Historical and Real-World Data for Clinical Studies
• Type: Topic-Contributed
• Date/Time: Sunday, August 8, 2021 : 3:30 PM to 5:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Rui (Sammi) Tang* and chenkun wang
• Companies: Servier Pharmaceuticals and vertex pharmarcetical
• Keywords: historical control; RWE; rare disease ; real world data; oncology

Abstract

In life-threatening/rare diseases, randomized controlled trial often runs into feasibility and even ethical issue. Single-arm interventional trial with historical control (HC) as the comparator, highlighted in the recent FDA Real World Evidence (RWE) 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 in oncology and rare diseases were developed with the support of historical data and several gained approvals based on single-arm pivotal trial. A roadmap to using HC in clinical trials by DIA NEED team will be introduced and then followed by discussions on selected NDA/BLA filing cases where HC was used as the comparator in the pivotal trials, providing some practical guidance on incorporating historical data from the stage of trial design to the stage of data analysis. We published our main findings and recommendation at Orphanet Journal of Rare Diseases in 2020.