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Session 7 Track B: Exploring Novel Ways of Evaluating New Compounds and Designing Clinical Trials
Session Chair(s)
Yatika Kohli, PhD, MBA
Chief Regulatory and Strategy Officer
NoNO Inc, Canada
Trials for rare conditions are challenging often due to a lack of reliable study endpoints, outcome measures, and biomarkers as well as few natural history studies that often can inform clinical studies. Traditional approaches to conducting randomized controlled trials (RCT) and development is becoming less viable as we understand more about certain diseases. To overcome this, researchers are exploring novel ways of evaluating new compounds and designing clinical trials. Innovative trials are not just aimed at being smaller and cheaper, often more importantly innovations are aimed at asking broader, better questions, and getting more robust answers at the end of the trial. For example, use of umbrella study or basket study designs in Cancer research allow researchers to assess either the impact of different drugs on different mutations in a single type of cancer or the impact of the drug or drug combinations on different types of cancer. Similarly, while enrichment designs allow one to consider multiple patient populations, an adaptive trial design allows the researcher to alter aspects of the study based on seeing how patients are responding to treatments whilst the study is running. This session aims to bring together industry experts from small-medium enterprises to discuss the challenges of and the novel approaches to drug development for rare diseases.
Learning Objective : At the conclusion of this session, the participant should be able to:- Identify some of the innovative non-traditional approaches to new and future drug development
- Understand the challenges in orphan drug development and rare diseases
Speaker(s)
Non-Traditional Approaches to Clinical Development: Lessons Learned from Orphan Drugs
Oxana Iliach, PhD
Certara, Canada
Senior Director Regulatory Strategy
Approaches to Conducting Emergency Research in the Pre-Hospital Setting
Michelle Leroux
Astrazeneca, Canada
Associate Director, Evidence Project Management
A Role of Technology and Virtual Trials in the Evolution of Clinical Trial Productivity: Case for Diabetes Mellitus
Jovana Kapor, MD, MSc
Endocrinology Center of Excellence, IQVIA, Canada
Senior Medical Director, Medical Strategy Lead, Endocrinology
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