Orphan Disease Research Requires Uncommon Collaboration: Q&A with Dr. Murphy on Rare Disease Clinical Trials

By The Editors Talking Trials,

In advance of the World Orphan Drug Congress USA (WODC), Talking Trials sat down for a Q&A with WODC roundtable host, Michael Murphy, M.D., Ph.D., Chief Medical and Scientific Officer for Worldwide Clinical Trials. Enjoy this preview of the roundtable, titled, “When the Sum Is Greater Than the Parts: Academic, Pharmaceutical and CRO Collaboration in Orphan Disease Drug Development.”

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Adaptive Designs and Orphan Diseases: Building a Bridge of Innovation

By William L Slone, Ph.D.; deMauri Mackie Ph.D.; Michael F Murphy M.D., Ph.D.,

Adaptive study designs (ASD) increasingly are vetted for use across a wide range of therapeutic indications, particularly for orphan disease indications. The resurgence of interest in innovative clinical trial methodology is born of need for efficient and informed program development and study design suitable for investigation of unique products, in unique therapeutic indications, in which benefits associated with traditional clinical trial methodology are not applicable.

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A Methodology for Advancing the Selection of Biomarkers for Use in Psychiatric Clinical Trials – Part 2

By Natalia Drosopoulou, Ph.D., Executive Director of Global Project Management, Neuroscience, Worldwide Clinical Trials,

  In part two of this blog series on selecting biomarkers for clinical research of the central nervous system, Talking Trials asked Worldwide’s Dr. Drosopoulou, author of the recent article, “A Methodology for Advancing the Selection of Biomarkers for Use in Psychiatric Clinical Trials,” published in the Journal for Clinical Studies, to weigh in on the use of biomarkers for clinical research.

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A Methodology for Advancing the Selection of Biomarkers for Use in Psychiatric Clinical Trials – Part 1

By Natalia Drosopoulou, Ph.D., Executive Director of Global Project Management, Neuroscience, Worldwide Clinical Trials,

  For this two-part blog series, Talking Trials asked Worldwide’s Dr. Drosopoulou, author of the recent article, “A Methodology for Advancing the Selection of Biomarkers for Use in Psychiatric Clinical Trials,” published in the Journal for Clinical Studies, to weigh in on the use of biomarkers for clinical research. Clinical trials using biomarkers in patient selection have higher overall success probabilities than clinical trials without biomarkers.1 That’s one of the many compelling results from a recent study of drug development success published in the journal Biostatistics. Wong and colleagues found clinical trials using biomarkers to stratify patients exhibit almost twice the overall probability of success compared to trials without biomarkers (10.3% vs. 5.5%).

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