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In this report, each of these considerations is reviewed and their influence for trial design discussed.
These issues are described for each study and recommendations for trial design are presented.
Outcome data applying this technology is lacking, and this is instrumental for clinical decision-making and for trial design.
The Penn classification can serve as a clinical framework for trial design, laying the groundwork for future management advances.
A consensus panel was convened to review data and propose a strategy for trial design and prioritization.
Considerations also include the use of placebos, critical for trial design but an impediment to parental permission.
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Recent trials focus on cognitively-normal people, but functional decline has not been explored for trial designs in this group.
Discussions with regulatory authorities gain momentum defining the role of tau biomarkers for trial designs and how they may be further qualified for surrogate marker status.
Increased availability of biomarkers and targeted treatments has led to the need for trial designs that efficiently test new treatments in biomarker-stratified patient subgroups.
The recent growth in clinical studies by many groups within a short period of time has meant that there were no clear precedents set for trial designs.
Where possible, PTAC is looking for trial designs that test whether the therapy is working by its intended process (Workman, 2002, 2003).
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Justyna Jupowicz-Kozak
CEO of Professional Science Editing for Scientists @ prosciediting.com