Interview: Emmes Mindy Leffler - are video based assessments the way forward to evaluate patients at home?

By Liza Laws

- Last updated on GMT

© Getty Images
© Getty Images

Related tags Duchenne muscular dystrophy Clinical trial Contract research organization Emmes Video assessment tool

Individuals diagnosed with Duchenne muscular dystrophy (DMD) often develop compensatory movement patterns due to muscle weakening.

The Duchenne Video Assessment (DVA) evaluates the ease of movement in patients by identifying these compensatory patterns. To conduct the assessment, caregivers are instructed to record videos of patients performing designated movement tasks at home using a secure mobile application. Certified physical therapists (PTs) trained in DVA evaluation then analyze these videos using scorecards containing predefined compensatory movement criteria. This study aimed to create and enhance the DVA scorecards. OSP spoke to Mindy Leffler, managing director of Qualitative Research and Psychometrics from Emmes, a full-service CRO to find out more.  

OSP: Can you explain how the Duchenne Video Assessment (DVA) differs from traditional clinical trial assessments for Duchenne muscular dystrophy (DMD)? 

ML:​ The DVA tool differs significantly from traditional clinical trial assessments in that it brings the assessment directly into the home environment of patients. This approach captures the daily function and experiences of patients in their real-world settings, providing a more authentic representation of their abilities. Unlike clinic-based assessments, which may not fully reflect the challenges and nuances of daily life with DMD, the DVA offers a more holistic view. Additionally, the DVA utilizes video recordings to objectively measure functional abilities, enabling a more consistent and reliable assessment process. 

OSP: How does the DVA incorporate patient and caregiver feedback into its assessment process? 

ML:​ Patient and caregiver feedback are integral to the development and refinement of the DVA tool. The team behind the DVA has personal experiences and involvement with DMD trials, which has led to a deep understanding of the challenges faced by patients and families. This firsthand knowledge, combined with ongoing communication with patients, caregivers, and clinicians, ensures that the DVA addresses the most relevant and meaningful aspects of daily function for individuals with DMD. The input from these stakeholders helps shape the tool's design, functionality, and relevance to the DMD community. 

What potential benefits do you foresee in using the DVA as a primary endpoint in clinical trials for DMD? 

ML:​ Using the DVA as a primary endpoint in DMD clinical trials offers several significant benefits. Firstly, it provides a more sensitive and meaningful measure of patients' functional abilities, capturing subtle changes that may not be detected by traditional assessments. This enhanced sensitivity allows for a more accurate evaluation of the effectiveness of potential treatments, leading to more informed clinical decisions. Additionally, the DVA's home-based approach reduces the burden on patients and families, making participation in trials more accessible and feasible. By incorporating real-world experiences into the assessment process, the DVA offers a comprehensive view of patients' daily lives, guiding the development of therapies that improve quality of life. 

OSP: How do you anticipate the DVA's qualification by the FDA could impact the broader landscape of rare disease research and clinical trials? 

ML: The qualification of the DVA by the FDA represents a significant milestone in the field of rare disease research and clinical trials. It sets a precedent for the use of video-based assessments as primary or secondary endpoints, paving the way for more innovative and patient-centric approaches to trial design. By acknowledging the unique challenges faced by rare disease populations and offering a solution that addresses these challenges, the FDA's qualification of the DVA opens doors for similar tools in other disease areas. This shift towards more meaningful and patient-relevant endpoints has the potential to revolutionize how rare diseases are studied and treated, leading to improved outcomes for patients worldwide. 

OSP: Could you elaborate on the challenges faced in measuring treatment effects in rare disease populations and how the DVA aims to address them? 

ML:​ Measuring treatment effects in rare disease populations presents several challenges, including the limited availability of validated outcome measures and the heterogeneity of patient experiences. Traditionally the endpoints were measured in a clinical setting where a patient is expected to perform a certain task to show treatment effect. These tasks are often standardized across disabilities and are not specific to neuromuscular disorders. Traditional endpoints may also not capture the full range of functional abilities and disease progression seen in rare diseases like DMD. The DVA addresses these challenges by providing a standardized, objective, and home-based assessment tool that quantifies daily function. It measures tasks which are more meaningful to the patient for example wearing their own T shirt or eating with a spoon. Through the utilization of video recordings, the DVA provides a more comprehensive perspective on patients' abilities over time. This allows clinicians and researchers to accurately track disease progression and treatment effects. Importantly, it all occurs in a familiar setting for the patient, where they feel most comfortable, without the added stress of travel and clinic visits.

OSP: How has the reception been from patients, caregivers, and clinicians regarding the implementation of the DVA in clinical trials? 

ML:​ The reception to the DVA from patients, caregivers, and clinicians has been overwhelmingly positive. Patients and families appreciate the tool's ability to capture their daily experiences and functional abilities in a more meaningful way. For caregivers, the DVA offers a less burdensome and more accessible option for participating in clinical trials. Clinicians recognize the value of the DVA in providing objective and reliable data on patients' progress, enabling more informed treatment decisions. Overall, the implementation of the DVA has been met with enthusiasm and support from all stakeholders involved in DMD research and care. 

OSP: Are there any plans to expand the use of the DVA beyond DMD trials to other rare diseases or conditions? 

ML:​ If we take a step back here, the significance for the industry could be enormous, what we are doing will effectively create a precedent and roadmap for other ‘in home’ video assessment endpoints. So we are already looking at modeling a similar system in other trial and therapeutic areas – for example, Angelman Syndrome, Primary Myocardial Myopathies, and Limb Girdle Muscular Dystrophies. It’s difficult to put an exact number of trials but with the rise of remote studies this really could be a big number in the future that have small or larger parts of the patient experience monitored at home. For example, in areas like neuromuscular, and neurodevelopmental disorders, and even trials exploring areas like ALS, it’s easier to see how positive an impact it could have.

OSP: Can you provide insights into the timeline for the FDA's review process of the DVA qualification plan and any subsequent steps expected? 

ML:​ This a great question, but not one we can answer on behalf of the FDA. However, we do expect feedback by July on the data submitted and, of course, the process from here is that we are collecting and analyzing further data over the next two years. The biggest significance of this is that our submission opens a discourse with the regulators about the potential for video-based outcomes to serve as a primary or secondary outcome in clinical trials. This is completely different from how video in trials is currently used, where until now it’s been used as just an anecdotal tool for applying pressure to regulators. The discourse will enable us to fully differentiate the robustness of what we’ve developed from these anecdotal methods. So it’s very much the first step, and a big one, toward the validation of video endpoints. We are building a tool with an end-to-end audit trail, video quality control, ‘rater’ training, and certification program, it’s a quantifiable methodology.

OSP: What measures are in place to ensure the reliability and consistency of the DVA assessments conducted remotely? 

ML:​ The DVA incorporates several measures to ensure the reliability and consistency of assessments conducted remotely. These include rigorous training programs for raters who evaluate the video recordings, standardized protocols for conducting assessments, and quality control measures to monitor video recording and data collection. Additionally, the DVA's quantification methodology has been validated to ensure accurate and consistent results across different raters and settings. By adhering to strict guidelines and protocols, the DVA maintains the integrity of its assessments and provides reliable data for clinical trials. 

OSP: In your opinion, how might the incorporation of home-based assessments like the DVA influence the future design and conduct of clinical trials? 

ML:​ The incorporation of home-based assessments like the DVA has the potential to revolutionize the design and conduct of clinical trials, particularly in rare diseases. By bringing assessments directly into patients' everyday environments, these tools offer a more authentic and comprehensive view of their daily functions and experiences. This shift towards patient-centricity not only reduces the burden on patients and families but also improves the accuracy and relevance of trial outcomes. Additionally, home-based assessments enable more diverse and inclusive trial participation, as patients from different geographic locations can participate without the need for frequent travel. Overall, the use of home-based assessments like the DVA enhances the patient experience, increases the efficiency of trials, and leads to more meaningful and impactful results. 

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