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Activity Number:
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570
- Addressing the Unique Analytic Challenges of Drug Development in Rare Diseases with the Use of Innovative Designs and Real World Evidence in Clinical Trials
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Type:
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Topic Contributed
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Date/Time:
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Thursday, August 6, 2020 : 3:00 PM to 4:50 PM
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Sponsor:
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ENAR
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Abstract #313428
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Title:
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On Randomized Delayed-Start Design with Integrated Analysis of Efficacy for Drug Development in Rare Diseases
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Author(s):
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Qing Liu* and Fred Holdbrook
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Companies:
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Quantitative and Regulatory Medical Science, LLC and Amicus Therapeutics, Inc.
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Keywords:
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Intra-patient comparison;
Integrated analysis of efficacy;
Randomized delayed-start design;
Gene therapy;
Rare disease;
Complex innovative design
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Abstract:
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A randomized delayed-start design consists of two-stages where patients in the first stage are randomized to receive a new treatment or a control and in the second stage patients randomized to receive the control in the first stage switch to receive the new treatment. We develop an integrated analysis of efficacy (IAE) where efficacy from stage one from a parallel group comparison is combined with efficacy from second stage from intra-patient comparison. As a result of IAE, the trial is 55% to 80% more efficient than a traditional parallel group design, which is often not feasible for rare disease drug development.
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Authors who are presenting talks have a * after their name.
