Exploring the Potential for Collaborative Use of an App-Based Platform for n-of-1 Trials Among Healthcare Professionals That Treat Patients With Insomnia

Bobe, Jason; Freitas, Jessica K De; Glicksberg, Benjamin S.

doi:10.3389/fpsyt.2020.530995

Cited by 4 publications

(5 citation statements)

References 40 publications

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“…Key hurdles, expressed by experts in our survey, included time and other efforts needed to plan and conduct the trials, as well as a lack of training in ITTs [ 22 ]. This is in line with other work that has shown that the know-how and efforts associated with profound RBAs needed to justify and plan ITTs are also key barriers in other fields [ 69 ].…”

Section: Discussionsupporting

confidence: 90%

RETRACTED: An Innovative Tool for Evidence-Based, Personalized Treatment Trials in Mucopolysaccharidosis

et al. 2023

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Mucopolysaccharidosis (MPS) is a group of rare metabolic diseases associated with reduced life expectancy and a substantial unmet medical need. Immunomodulatory drugs could be a relevant treatment approach for MPS patients, although they are not licensed for this population. Therefore, we aim to provide evidence justifying fast access to innovative individual treatment trials (ITTs) with immunomodulators and a high-quality evaluation of drug effects by implementing a risk–benefit model for MPS. The iterative methodology of our developed decision analysis framework (DAF) consists of the following steps: (i) a comprehensive literature analysis on promising treatment targets and immunomodulators for MPS; (ii) a quantitative risk–benefit assessment (RBA) of selected molecules; and (iii) allocation phenotypic profiles and a quantitative assessment. These steps allow for the personalized use of the model and are in accordance with expert and patient representatives. The following four promising immunomodulators were identified: adalimumab, abatacept, anakinra, and cladribine. An improvement in mobility is most likely with adalimumab, while anakinra might be the treatment of choice for patients with neurocognitive involvement. Nevertheless, an RBA should always be completed on an individual basis. Our evidence-based DAF model for ITTs directly addresses the substantial unmet medical need in MPS and characterizes a first approach toward precision medicine with immunomodulatory drugs.

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Section: Discussionsupporting

confidence: 90%

RETRACTED: An Innovative Tool for Evidence-Based, Personalized Treatment Trials in Mucopolysaccharidosis

et al. 2023

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“…The response options of the items ran in a positive direction and were confirmed with high values. The questionnaire of the ESITT was adopted for MPS clinicians according to a validated tool [ 29 ] and is included as supplemental information .…”

Section: Methodsmentioning

confidence: 99%

“…Nevertheless, success is not achieved and guaranteed in all uses, because cumulative and curative treatments are unsuitable for ITTs. More progress can be expected with drugs that have a quick onset of action and a quick wash out [ 25 , 29 ]. These ITTs, which are often crossover trials, double-blinded, and randomized [ 26 , 28 , 30 ], can be conducted in three different ways.…”

Section: Introductionmentioning

confidence: 99%

Individual Treatment Trials—Do Experts Know and Use This Option to Improve the Treatability of Mucopolysaccharidosis?

2023

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Mucopolysaccharidoses (MPS) are a group of rare, heterogeneous, lysosomal storage disorders. Patients show a broad spectrum of clinical features with a substantial unmet medical need. Individual treatment trials (ITTs) might be a valid, time- and cost-efficient way to facilitate personalized medicine in the sense of drug repurposing in MPS. However, this treatment option has so far hardly been used—at least hardly been reported or published. Therefore, we aimed to investigate the awareness and utilization of ITTs among MPS clinicians, as well as the potential challenges and innovative approaches to overcome key hurdles, by using an international expert survey on ITTs, namely, ESITT. Although 74% (20/27) were familiar with the concept of ITTs, only 37% (10/27) ever used it, and subsequently only 15% (2/16) published their results. The indicated hurdles of ITTs in MPS were mainly the lack of time and know-how. An evidence-based tool, which provides resources and expertise needed for high-quality ITTs, was highly appreciated by the vast majority (89%; 23/26). The ESITT highlights a serious deficiency of ITT implementation in MPS—a promising option to improve its treatability. Furthermore, we discuss the challenges and innovative approaches to overcome key barriers to ITTs in MPS.

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“…Another way to reduce costs and barriers is to make it easier for individuals to find relevant trials (or to design their own), complete enrollment procedures, and provide meaningful informed consent. A number of electronic platforms, many created for use on mobile devices, have been used or are in development ( Barr et al, 2015 )( Bobe et al, 2020 )( Konigorski et al, 2020 )( Kravitz et al, 2020 ). As (n.d.-q) point out, however,…”

Section: Emerging Technologies and Approachesmentioning

confidence: 99%

Conduct and Implementation of Personalized Trials in Research and Practice

Kravitz

Duan

2022

Harvard Data Science Review

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The mainstay of evidence development in medicine is the parallel-group randomized controlled trial (RCT), which generates estimates of treatment efficacy or effectiveness for the average person in the trial. In contrast, personalized trials (sometimes referred to as 'single-person trials' or 'N-of-1 trials') assess the comparative effectiveness of two or more treatments in a single individual. These single-subject, randomized crossover trials have been used in a scattershot fashion in medicine for over 40 years but have not been widely adopted.An important barrier is the paucity of strong evidence that personalized trials improve outcomes. However, the principal impediment may have less to do with proof of efficacy than with practical aspects of design and implementation. These include decisions about treatment regimen flexibility, blinding, and washout periods as well as organizational, clinician, and patient-level challenges. After reviewing the essential elements of personalized trials, this article addresses these speed bumps and fundamentally asks, 'Why have personalized trials not been more widely adopted, and how can they be made more readily deployable and useful?' The article concludes by suggesting ways in which emerging technologies and approaches promise to overcome existing barriers and open promising vistas for the next generation of personalized-trial researchers and practitioners.

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Exploring the Potential for Collaborative Use of an App-Based Platform for n-of-1 Trials Among Healthcare Professionals That Treat Patients With Insomnia

Cited by 4 publications

References 40 publications

RETRACTED: An Innovative Tool for Evidence-Based, Personalized Treatment Trials in Mucopolysaccharidosis

RETRACTED: An Innovative Tool for Evidence-Based, Personalized Treatment Trials in Mucopolysaccharidosis

Individual Treatment Trials—Do Experts Know and Use This Option to Improve the Treatability of Mucopolysaccharidosis?

Conduct and Implementation of Personalized Trials in Research and Practice

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