Neuroscience Trials of the Future Proceedings of a Workshop (2016) / Chapter Skim
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7 Improving the Evidence Base for Real-World Use
7 Improving the Evidence Base for Real-World Use
Pages 59-68
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From page 59... ...
. • Administrative claim databases with the ability to connect to clinical data elements maintained by health plans offer a rich source of mineable data for development of pragmatic trials (Cziraky)
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From page 60... ...
Historically, payers have made decisions based on medical necessity with evidence obtained from peer-reviewed articles, technology reviews, and practice guidelines from credible organizations, said Robinson Beale. She added that payers typically look not only at RCTs, but pragmatic trials as well in order to gain a real-world understanding of the efficacy and practicality of the treatment over a longer period of time than the duration of a clinical trial.
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From page 61... ...
PERSPECTIVES FROM THE PHARMACEUTICAL INDUSTRY Several participants highlighted the importance of payer concerns and how they are factored into pharmaceutical companies' drug development programs. They noted that this requires generating credible evidence of value in the real world from both observational studies, including registry studies, and randomized trials in real-world settings, also known as pragmatic trials.
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From page 62... ...
. Compared to pivotal RCTs, Stang noted that observational trials tend to follow people for a longer period of time, may include much larger and more diverse populations, and may incorporate existing data collection infrastructure such as electronic health records (EHRs)
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From page 63... ...
rathher than indiv viduals were randomized. This approaach lessenss the burden on practition ners and avoiids contaminaation across iinterventions, but inttroduces a lo oss of statistiical efficienccy (Meurer annd Lewis,, 2015)
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From page 64... ...
DATA SOURCES FOR REAL-WORLD TRIALS The databases maintained by health plans and large provider networks offer a rich source of minable data for pragmatic trials, according to several participants. For example, HealthCore maintains an integrated research database1 that captures 10 years of data from about 65 million individuals, including data from administrative claims, physician and facility claims, prescriptions, and laboratory tests, said Cziraky.
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From page 65... ...
Moreover, Cziraky stated that working through providers, these data can be connected with clinical data in the trial setting to create an integrated dataset for analysis. Burch commented that the workshop discussions suggest that distinctions between regulatory and pragmatic trials may become blurred, which raises concerns because of the potential for real-world trials to impede assay sensitivity.
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From page 66... ...
Many workshop participants highlighted the increased focus on patients and real-life outcomes in neuroscience trials of the future. Enabling technologies discussed include mobile health, remote monitoring, and wearable devices as well as other technologies that have yet to be developed, said Atul Pande.
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From page 67... ...
IMPROVING THE EVIDENCE BASE FOR REAL-WORLD USE 67 further down in the pipeline, according to some participants, include understanding how to capitalize on biomarkers as a way of teasing out subgroups within targeted populations. In addition, understanding how new technologies might be used for trialists to recruit participants more globally.
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