Using N-of-1 Trials to Improve Patient Management and Save Costs

Scuffham, Paul Anthony; Nikles, Jane; Mitchell, G.; Yelland, Michael; Vine, Norma; Poulos, Christopher J.; Pillans, Peter I.; Bashford, Guy; Mar, Chris Del; Schluter, Philip J.; Glasziou, Paul

doi:10.1007/s11606-010-1352-7

Cited by 92 publications

(78 citation statements)

References 23 publications

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“…N-of-one trials have been used in the past to optimise treatment for individual patients, reduce unnecessary prescribing, and increase treatment compliance (C. Nikles, Clavarino, & Del Mar, 2005;Scuffham et al, 2010). Formal "trials of therapy", such as the one described in this study, can be valuable in guiding clinical practice when there is no evidence available from group-randomised clinical trials (RCTs), when the results of such trials do not necessarily generalise to one's patient in the consultation room, or when there are other pertinent reasons to optimise treatment, for example, because of the high cost of a medicinal product (Guyatt et al, 1988).…”

Section: Discussionmentioning

confidence: 99%

Bayesian Evaluation of Informative Hypotheses

2011

Informative Hypotheses

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Section: Discussionmentioning

confidence: 99%

Bayesian Evaluation of Informative Hypotheses

2011

Informative Hypotheses

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“…Take the patient health mobile/ wearable/sensor data used in Focus Areas 2 and 3 as example: evidence is strong for the reliability of physical activity measures [85], but weaker for sleep patterns and heart rate measures [86]. Another dimension of quality and reliability in view of the patients' right to data integrity is about information security and trust management.…”

Section: Quality and Reliability Issues Of Pghdmentioning

confidence: 99%

Added Value from Secondary Use of Person Generated Health Data in Consumer Health Informatics

Hsueh¹,

Cheung²,

Dey³

et al. 2017

Yearb Med Inform

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Summary Introduction: Various health-related data, subsequently called Person Generated Health Data (PGHD), is being collected by patients or presumably healthy individuals as well as about them as much as they become available as measurable properties in their work, home, and other environments. Despite that such data was originally just collected and used for dedicated predefined purposes, more recently it is regarded as untapped resources that call for secondary use. Method: Since the secondary use of PGHD is still at its early evolving stage, we have chosen, in this paper, to produce an outline of best practices, as opposed to a systematic review. To this end, we identified key directions of secondary use and invited protagonists of each of these directions to present their takes on the primary and secondary use of PGHD in their sub-fields. We then put secondary use in a wider perspective of overarching themes such as privacy, interpretability, interoperability, utility, and ethics. Results: We present the primary and secondary use of PGHD in four focus areas: (1) making sense of PGHD in augmented Shared Care Plans for care coordination across multiple conditions; (2) making sense of PGHD from patient-held sensors to inform cancer care; (3) fitting situational use of PGHD to evaluate personal informatics tools in adaptive concurrent trials; (4) making sense of environment risk exposure data in an integrated context with clinical and omics-data for biomedical research. Discussion: Fast technological progress in all the four focus areas calls for a societal debate and decision-making process on a multitude of challenges: how emerging or foreseeable results transform privacy; how new data modalities can be interpreted in light of clinical data and vice versa; how the sheer mass and partially abstract mathematical properties of the achieved insights can be interpreted to a broad public and can consequently facilitate the development of patient-centered services; and how the remaining risks and uncertainties can be evaluated against new benefits. This paper is an initial summary of the status quo of the challenges and proposals that address these issues. The opportunities and barriers identified can serve as action items individuals can bring to their organizations when facing challenges to add value from the secondary use of patient-generated health data.

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“…The personalized or tailored nature of precision medicine to provide insight into the management of an individual patient is often alluded to as practicing medicine with an ‘n of 1' [21]. Such an approach, however, is predicated on the availability of data to discern critical variables that reflect underlying pathophysiology and likely response to certain therapies.…”

Section: Registries In Precision Medicinementioning

confidence: 99%

Innovative Interventional and Imaging Registries: Precision Medicine in Cerebrovascular Disorders

Liebeskind

2015

Intervent Neurol

427

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Background: Precision medicine in cerebrovascular disorders may be greatly advanced by the use of innovative interventional and imaging-intensive registries. Registries have remained subsidiary to randomized controlled trials, yet vast opportunities exist to leverage big data in stroke. Summary: This overview builds upon the rationale for innovative, imaging-intensive interventional registries as a pivotal step in realizing precision medicine for several cerebrovascular disorders. Such enhanced registries may serve as a model for expansion of our translational research pipeline to fully leverage the role of phase IV investigations. The scope and role of registries in precision medicine are considered, followed by a review on the history of stroke and interventional registries, data considerations, critiques or barriers to such initiatives, and the potential modernization of registry methods into efficient, searchable, imaging-intensive resources that simultaneously offer clinical, research and educational added value. Key Messages: Recent advances in technology, informatics and endovascular stroke therapies converge to provide an exceptional opportunity for registries to catapult further progress. There is now a tremendous opportunity to deploy registries in acute stroke, intracranial atherosclerotic disease and carotid disease where other clinical trials leave questions unanswered. Unlike prior registries, imaging-intensive and modernized methods may leverage current technological capabilities around the world to efficiently address key objectives and provide added clinical, research and educational value.

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Using N-of-1 Trials to Improve Patient Management and Save Costs

Cited by 92 publications

References 23 publications

Bayesian Evaluation of Informative Hypotheses

Bayesian Evaluation of Informative Hypotheses

Added Value from Secondary Use of Person Generated Health Data in Consumer Health Informatics

Innovative Interventional and Imaging Registries: Precision Medicine in Cerebrovascular Disorders

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