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5 Digital Health Technologies for Pivotal Trials
Pages 61-74

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From page 61...
... (Kapur) • For regulators, DHTs can add value to clinical trials by en abling remote data collection in decentralized trial settings, broadening access for participants, and capturing novel data (e.g., continuous physiological measurements, measures of functionality)
From page 62...
... INDUSTRY PERSPECTIVE ON DIGITAL HEALTH TECHNOLOGIES IN PIVOTAL TRIALS Sean Khozin, Global Head of Data Strategy, Janssen Research & Development, LLC Khozin said that the expanding universe of big data presents complexities regarding four computational dimensions (see Figure 5-1) : • Volume: Dataset size (e.g., computing storage sizes -- megabyte, gigabyte, terabyte, and petabyte)
From page 63...
... close to the holistic edge of big data can also be used to capture patients' experiences in pivotal trials, using novel trial designs that accommodate more decentralized data collection. Due to the unique nature of data assets emerging from DHTs, he explained, it is important to consider the technical and procedural issues associated with incorporating those modalities into clinical trials.
From page 64...
... Digital Health Technologies as Diagnostic Tools Khozin spoke about how the issue of false negatives and positives could be addressed when using clinically validated DHTs for diagnostic purposes. Established methods for addressing false negatives and positives in traditional diagnostic tests will also apply to DHTs.
From page 65...
... Procedural Considerations for Using Digital Tools in Clinical Trials Khozin discussed procedural considerations related to the use of these DHTs in pivotal clinical trials, including clinical validation and the design and conduct of clinical trials (Coravos et al., 2019b)
From page 66...
... Specifically, this involves a three-step process of signal verification, analytical validation, and clinical validation. Kapur defined each term and gave examples of each: 1.
From page 67...
... Analytical validation determines whether the accelerometer is actually measuring steps and can be done by comparing the digital measure to a manual step count or using a corroborative device on people in a real-world setting to generate naturalistic datasets for comparison. Clinical validation would evaluate the association between step counts and disease severity among people with PD.
From page 68...
... Leveraging Digital Health Technologies for Recruitment, Retention, and Engagement Kapur shared some of Verily's experiences in using digital technology for recruitment, retention, and engagement. Creating a sense of human connectedness when operating at scale is a challenge encountered when DHTs are incorporated into clinical trials.
From page 69...
... Uses of Digital Health Technologies in Clinical Trials Sacks described how DHTs can fit into the design of clinical trials. For example, they could be used to improve participant screening and enrichment strategies by selecting patients based on levels of disease severity or activity levels.
From page 70...
... Another exciting opportunity DHTs provide in clinical trials is the potential to shape endpoints. To explain how DHTs can contribute value in this respect, Sacks provided an overview of endpoints used in FDA pivotal trials for 280 new drug applications between 2007 and 2015.
From page 71...
... While one of the studies was conducted in a traditional clinical trial setting and used a single dose of valbenazine, the other study tested two doses of valbenazine and incorporated DHTs by sending video recordings of participants to independent adjudicators who were blinded to the sequence of the video recordings and to the study drug allocations. In the first study, investigators blinded to treatment allocation who made successive subjective assessments on their patients reported improvement from baseline in placebo-treated patients.
From page 72...
... DISCUSSION Analytical Validation Without a Gold Standard Reference Kapur and Sacks discussed approaches that could be used to perform analytical validation on DHTs in the absence of a gold standard reference measurement. Kapur suggested a combined approach to addressing this challenge -- particularly for novel measurements -- before the widespread introduction of a tool.
From page 73...
... Sacks added that common standards are also useful because they make it possible for the digital health community to evaluate which technologies might be suitable for a proposed use and which are not. For example, technological standards for mobile phones and smart watches may allow study participants to use their own devices rather than using a study-assigned mobile phone or wearable devices.


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