Davies, E.H., Matthews, C., Merlet, A. et al. Time to See the Difference: Video Capture for Patient-Centered Clinical Trials. Patient (2022) in conjunction with Alexion, AstraZeneca Rare Disease explores the role of technology in rare disease drug development and how video recording and analysis can enable more patient-centric clinical trials, which will be a major drive to support at-home assessments, especially in rare and neurodegenerative diseases.


Developing therapeutics for the treatment of rare diseases usually requires a strong understanding of the natural history of the disease. Often, it also requires the creation of novel assessment tools and clinical trial endpoints. In diseases where mobility is impacted, the use of video to capture the impact of the disease and the assessment of specific parameters, such as gait and stride length, can help design sensitive endpoints. Video as an assessment tool also allows the use of historical videos or videos filmed by non-experts outside of clinical settings. Given the increased use of telemedicine, the use of video may be a useful addition to clinical trial assessments.

Two cases are presented: (1) the use of video in the development of asfotase alfa (Strensiq®) in hypophosphatasia is detailed as an example of the utility of this type of assessment in rare diseases; and (2) a home-setting video tool that was developed and validated (SARAhome) from a commonly used clinical scale (Scale for the Assessment and Rating of Ataxia [SARA]), allowing patients to record their own severity of ataxia. While there are certain limitations associated with video assessment, advancing technologies such as automated analysis and machine learning provide a tremendous opportunity for automated analysis of video recordings, reducing the bias associated with human assessment.

In this paper we explore key points for decision-makers, namely:

Novel assessment tools such as video technology can capture how a patient functions and also provide meaningful endpoints in rare disease development.
Video technology allows for clinical outcome assessments to be offered at home as well as in hospitals, reducing the burden of travel to sites.
Early planning to validate the approach with regulators is essential to allow for standardized data collection and alignment with real-world evidence.

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Authors: Elin Haf DaviesClare MatthewsAdeline Merlet & Martine Zimmermann

Davies, E.H., Matthews, C., Merlet, A. et al. Time to See the Difference: Video Capture for Patient-Centered Clinical Trials. Patient (2022).

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