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For instance, identifying Mild Cognitive Impaired who are most likely to decline during a trial, and thus most likely to benefit from the treatment, will improve trial efficiency and power to detect treatment effects.
This may be particularly relevant to efforts to improve trial efficiency in order to test multiple targeted new agents in combination.
Selecting high-risk patients based on predictions from a nomogram can also help to improve trial efficiency; for example, trials evaluating a treatment strategy could target patients with poor prognoses.
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Extensive simulations demonstrate that the proposed new design not only provides better therapeutic effect by reducing the probability of treating patients at under-dose levels while protecting patients from being overdosed, but also improves trial efficiency and increases the accuracy of dose recommendation for subsequent clinical trials.
Other examples of the reuse of clinical data to support clinical research include the feasibility platform for stroke studies (FePASS), an open access online web-system that allows users to obtain eligible patient counts for stroke trials based on user-defined eligibility criteria (EC) [ 7] and the Feasibility Assessment and Recruitment System for Improving Trial Efficiency (FARSITE) tool [ 8].
Simulation results demonstrate that EWOC-NETS-TITE can substantially improve the trial efficiency and accuracy of MTD determination as well as allow patients to be entered in a staggered fashion to significantly shorten trial duration.
Such a panel might improve clinical trial efficiency by guiding subject enrollment and monitoring disease progression.
Phase I trials usually involve a small number of patients so that fully utilizing all toxicity information including time to event toxicity data is key to improving the trial efficiency and the accuracy of MTD estimation.
Simulation results show that this design has good operating characteristics and improves the accuracy of MTD, trial efficiency, therapeutic effect, and overdose control relative to EWOC which is used as a representative of designs treating toxicity response as a binary indicator of DLT.
In later phases, alternative clinical endpoints and enriching sensitive study populations through innovative trial designs could improve the efficiency of clinical trials for oligonucleotide therapeutics.
Also, inclusion of baseline HRQoL as a stratification factor could increase trial efficiency, create more homogeneous treatment groups and improve understanding of trial results.
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