Alternative Designs to the Traditional 3+3 Design in Phase 1 Dose Escalation Studies

Phase 1 clinical trials aim to determine the maximum tolerated dose (MTD) of a new molecule with the goal of identifying a recommended Phase 2 dose (RP2D), often the MTD itself. Ideally, the RP2D would have adequate therapeutic effect to demonstrate preliminary signs of efficacy in Phase 2, but many Phase 2 trials fail to detect a preliminary efficacy signal, prolonging the development program and increasing costs.

A common reason Phase 2 trials fail is because the RP2D selected from the Phase 1 trial was a sub-therapeutic dose. The risk of selecting a sub-therapeutic dose as the RP2D is a key limitation of the traditional 3+3 dose-escalation design in Phase 1.

In this webinar, the panelists will discuss this issue and its broader implications and highlight alternate Bayesian model-based dose escalation designs that offer a higher chance of selecting an effective therapeutic dose as the RP2D, while also providing greater efficiency than the traditional 3+3 rules-based design and increasing the speed to determining the MTD.

Speakers

Abie Ekangaki, Ph.D., Vice President, Statistical Consulting, Premier Research

Abie Ekangaki is responsible for bringing expertise in biostatistics to shape clinical trial design strategies and methodologies for regulatory submissions and otherwise and provides broad biostatistical consulting for both internal and external clients. He advises on efficient trial designs for clinical trial programs and applies his statistical expertise, drug development, and operational experience across different therapeutic areas.

With more than 25 years of experience as a biostatistician, Dr. Ekangaki has worked as a research scientist with the World Health Organization in Switzerland, as a statistics lecturer at Macquarie University in Australia, and has over 19 years in the pharmaceutical industry in both large pharma and CROs, where he has held several lead technical and senior leadership positions.

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Andreas Schreiner, Vice President Medical Affairs Neuroscience & Analgesia, Premier Research

Dr. Schreiner is Vice President, Medical Affairs Neuroscience and Analgesia at Premier Research. He has considerable experience as an investigator as well as global experience within the pharmaceutical industry and clinical research environment. In his role, he is responsible for providing strategic planning, coordination, knowledge, and expertise in neuroscience and analgesia for projects and/or standalone consultative services.

Dr. Schreiner is a certified psychiatrist and neurologist. He studied medicine at the Universities of Heidelberg and Mannheim, Germany, and completed residencies in the University Department of Neurology and in Psychiatry at the Central Institute of Mental Health, both in Mannheim, Germany.

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