In this blog, we talk with Heiko Götte, Senior Expert Biostatistician at Merck about his upcoming presentation at Cytel’s East User Group Meeting on 14th and 15th November at Merck Darmstadt, in Germany. The topic Heiko will address is Decision Making in Development Programs with Targeted Therapies and he explains to us why this is a key topic for pharmaceutical companies today as they strive to improve their decision-making, and what delegates can expect to take away from the presentation.
Cytel: How did you develop your interest in the topic of decision-making in development programs with targeted therapies?
Heiko Götte (HG): This is a topic that is regularly discussed in early phase trials. One or several biomarkers are analyzed retrospectively. They were not included in the trial design because they became important after the trial started (external information) or there was no (validated) assay available/ no cut-off defined to stratify subjects at trial entry. It is natural to focus on the best performing biomarker subgroups. However, the observed treatment effects are on average overestimated and expectations for further development are overoptimistic. Although the general problem is known and acknowledged, the magnitude of over optimism is often not investigated and statistical methods for adequate decision making were needed.
Cytel: Why is this an important topic?
HG: This is a high profile topic in many pharmaceutical companies today. High phase III failure rates force the pharmaceutical industry to improve their decision making. Realistic assumptions about the potential for a successful phase III trial are an important basis for decision making. This requires considering the whole development program (not just separate trials) as well as considering statistical measures which take uncertainty and selection into account.
Cytel: What can those who hear the talk expect to take away?
HG: I hope attendees of my talk become aware/ are reminded of subgroup selection bias and take the proposed methods into account when they face questions like: Should we continue developing this drug? In which population? Do we trust the retrospective biomarker results and include only biomarker positive patients in a future trial? How do we use the observed data to adequately plan a confirmatory follow-up trial?
About Heiko Götte
Heiko Götte holds a PhD from Johannes Gutenberg-University Mainz, Germany and his professional experience includes research assistant at the Institute of Medical Biostatistics, Epidemiology and Informatics, Johannes Gutenberg University. Since 2009, he's been a biostatistician at Merck KGaA in Darmstadt.
In his talk Decision Making in Development Programs with Targeted Therapies, Heiko will address these key points:
• Development programmes for targeted therapies usually consist of early phase trial for identifying a target subgroup among several biomarker candidates followed by a confirmatory trial
• Results from early phase trial influence go/no go decision, choice of confirmatory trial design as well as interim decision rules of a potential adaptive design
• Early phase results need to be adjusted for data-driven selection to allow adequate decision making
About the East User Group Meeting: Innovations in Clinical Trials Symposium & Hands-on East Training
Since 2011, the Innovations in Clinical Trials Symposium & Hands-on East Training has brought together industry experts, thought leaders and applied statisticians to discuss the future of clinical trials. This annual meeting guides the development of the industry's leading clinical trial design software. Join your peers at Merck in Darmstadt, Germany to see the newest developments in East.
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