Emmes is seeking US FDA approval for its Duchenne Video Assessment (DVA) qualification plan, which is designed to help Duchenne muscular dystrophy (DMD) sufferers take part in trials.
The plan – which was developed by Emmes Endpoints Solutions, the contract research organization's (CRO’s) unit that creates outcome measures for decentralized and hybrid trials – will address problems that have stymied DMD research, according to a spokesperson.
“The advantage of DVA is that it is done in the home, in a real-world environment – so we see the difference a therapy is potentially making to the patient’s actual experience, not their experiences as monitored in a clinical setting. The challenge, until now, has been quantifying that process in a meaningful, impartial, and measurable way.”
The spokesperson added that “The development team has a personal involvement with DMD trials and we were left frustrated with the poor quality of existing traditional endpoints – that were both more taxing on patients but also did not show meaningful improvements in real world settings. And this is really the key to how and why we came to do this, we wanted to be able to more accurately measure the real-world improvements seen in patients undertaking everyday tasks.”
The drug industry’s track record on DMD is not good. Over the last ten years more than 40 clinical trials in Duchenne have failed to achieve statistically significant positive results on their primary outcomes.
Emmes expects the Food and Drug Administration (FDA) to provide initial feedback on the DVA plan by July 2024, with further data to be collected and analysed over next two years.
The spokesperson said “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 to how video in trials is currently used, where until now it’s been used mainly as an anecdotal tool. The discourse with the regulators will enable us to fully differentiate the robustness of what we’ve developed from these anecdotal methods.”
Expansion
Development of the DVA was initiated by Casimir, a patient-focused contractor Emmes acquired in March 2022. According to research published after the takeover, the DVA measures patient ease of movement through identification of compensatory patterns.
It also directs caregivers to record patients performing specific movement tasks at home using a secure mobile application. DVA-certified physical therapists (PTs) then score the videos using scorecards with pre specified compensatory movement criteria.
And the approach has the potential for wider application according to the spokesperson, who said, “We are already looking at modelling a similar system in other trial and therapeutic areas – for example, Angelman syndrome, primary myocardial myopathies and limb girdle muscular dystrophies.”
The CRO may also seek input from regulators outside the US. The spokesperson told us “We started this in the US, but now with the wider Emmes Group and our European colleagues, there is the possibility of trying to work with EMA as well.”
DepositPhotos/DmitriyDemidovich