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Clinical Trial Adaptation by Matching Evidence in Complementary Patient Sub-groups of Auxiliary Blinding Questionnaire Responses.

Arandjelović O - PLoS ONE (2015)

Bottom Line: The main goal is to make the process of introducing new medical interventions to patients more efficient, either by reducing the cost or the time associated with evaluating their safety and efficacy.The principal challenge, which is an outstanding research problem, is to be found in the question of how adaptation should be performed so as to minimize the chance of distorting the outcome of the trial.In this paper we propose a novel method for achieving this.

View Article: PubMed Central - PubMed

Affiliation: Centre for Pattern Recognition and Data Analytics, School of Information Technology, Deakin University, Geelong, Victoria, Australia.

ABSTRACT
Clinical trial adaptation refers to any adjustment of the trial protocol after the onset of the trial. Such adjustment may take on various forms, including the change in the dose of administered medicines, the frequency of administering an intervention, the number of trial participants, or the duration of the trial, to name just some possibilities. The main goal is to make the process of introducing new medical interventions to patients more efficient, either by reducing the cost or the time associated with evaluating their safety and efficacy. The principal challenge, which is an outstanding research problem, is to be found in the question of how adaptation should be performed so as to minimize the chance of distorting the outcome of the trial. In this paper we propose a novel method for achieving this. Unlike most of the previously published work, our approach focuses on trial adaptation by sample size adjustment i.e. by reducing the number of trial participants in a statistically informed manner. We adopt a stratification framework recently proposed for the analysis of trial outcomes in the presence of imperfect blinding and based on the administration of a generic auxiliary questionnaire that allows the participants to express their belief concerning the assigned intervention (treatment or control). We show that this data, together with the primary measured variables, can be used to make the probabilistically optimal choice of the particular sub-group a participant should be removed from if trial size reduction is desired. Extensive experiments on a series of simulated trials are used to illustrate the effectiveness of our method.

No MeSH data available.


Related in: MedlinePlus

Adopted statistical model for a three-tier feedback questionnaire (conceptual illustration)—the probability densities of the measured trial outcome across the three control (solid lines) and treatment sub-groups (dotted lines).Diagram reproduced with permission.
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pone.0131524.g001: Adopted statistical model for a three-tier feedback questionnaire (conceptual illustration)—the probability densities of the measured trial outcome across the three control (solid lines) and treatment sub-groups (dotted lines).Diagram reproduced with permission.

Mentions: Sub-group 6: participants of the treatment group who believe they were assigned to the treatment group (sub-group GT+).


Clinical Trial Adaptation by Matching Evidence in Complementary Patient Sub-groups of Auxiliary Blinding Questionnaire Responses.

Arandjelović O - PLoS ONE (2015)

Adopted statistical model for a three-tier feedback questionnaire (conceptual illustration)—the probability densities of the measured trial outcome across the three control (solid lines) and treatment sub-groups (dotted lines).Diagram reproduced with permission.
© Copyright Policy
Related In: Results  -  Collection

License
Show All Figures
getmorefigures.php?uid=PMC4498692&req=5

pone.0131524.g001: Adopted statistical model for a three-tier feedback questionnaire (conceptual illustration)—the probability densities of the measured trial outcome across the three control (solid lines) and treatment sub-groups (dotted lines).Diagram reproduced with permission.
Mentions: Sub-group 6: participants of the treatment group who believe they were assigned to the treatment group (sub-group GT+).

Bottom Line: The main goal is to make the process of introducing new medical interventions to patients more efficient, either by reducing the cost or the time associated with evaluating their safety and efficacy.The principal challenge, which is an outstanding research problem, is to be found in the question of how adaptation should be performed so as to minimize the chance of distorting the outcome of the trial.In this paper we propose a novel method for achieving this.

View Article: PubMed Central - PubMed

Affiliation: Centre for Pattern Recognition and Data Analytics, School of Information Technology, Deakin University, Geelong, Victoria, Australia.

ABSTRACT
Clinical trial adaptation refers to any adjustment of the trial protocol after the onset of the trial. Such adjustment may take on various forms, including the change in the dose of administered medicines, the frequency of administering an intervention, the number of trial participants, or the duration of the trial, to name just some possibilities. The main goal is to make the process of introducing new medical interventions to patients more efficient, either by reducing the cost or the time associated with evaluating their safety and efficacy. The principal challenge, which is an outstanding research problem, is to be found in the question of how adaptation should be performed so as to minimize the chance of distorting the outcome of the trial. In this paper we propose a novel method for achieving this. Unlike most of the previously published work, our approach focuses on trial adaptation by sample size adjustment i.e. by reducing the number of trial participants in a statistically informed manner. We adopt a stratification framework recently proposed for the analysis of trial outcomes in the presence of imperfect blinding and based on the administration of a generic auxiliary questionnaire that allows the participants to express their belief concerning the assigned intervention (treatment or control). We show that this data, together with the primary measured variables, can be used to make the probabilistically optimal choice of the particular sub-group a participant should be removed from if trial size reduction is desired. Extensive experiments on a series of simulated trials are used to illustrate the effectiveness of our method.

No MeSH data available.


Related in: MedlinePlus