2023, European Medicines Agency (EMA)
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Drug development requires reliable & sensitive endpoints to assess how patients feel, function, and survive. Validated digital technologies can provide precise and robust clinical assessments of motor function through continuous real-life patient monitoring.
The first-ever qualification by regulators of a digital endpoint, stride velocity 95th centile (SV95C) as a primary endpoint for Duchenne Muscular Dystrophy opens new possibilities for clinical trials. It shows the full potential of digital endpoints when they are robust, meaningful for patients, sensitive to change, validated by regulators, and adopted by the industry for drug development.
In the future, digital endpoints will play a key role in tracking treatment effectiveness and providing evidence for medical recommendations, enabling faster development & more personalized healthcare for patients.
Traditional functional assessment of mobility involves in-clinic tests such as the 6-minute walk test (6MWT), the time 25-foot walk test (25FWT), the 10-meter test, 4 stair climbing test, or Time up-and-go (TUG).
These assessments of motor function have limitations:
Traditional assessments do not accurately represent what patients can do because they don’t consider short-term fluctuations like fatigue, or motivation. They also add a logistical burden to patients.
Traditional assessments sometimes fail to show enough proof of a treatment's effectiveness. Regulators are confronted with ambiguity in assessing the drug's effectiveness because of inadequate data.
Traditional assessments of motor function fail to measure treatment effectiveness accurately. There have been recent examples of drug development being discontinued and reimbursement issues due to the inability of traditional assessments to highlight treatment efficacy.
Currently, The potential of Digital Health Technologies (DHTs] in clinical trials remains untapped. It is because commercial wearables lack the analytical validity to be useful in clinical settings.
New technology is needed to bridge this gap and bring true value to patients, clinicians & decision-makers. Regulatory-grade wearables have the analytical ability to deliver precise digital health measurements and improve the traditional ‘gold standards’ all while ensuring the utmost privacy of the patient.
Why use digital endpoints in clinical trials :
Digital endpoints and technologies enable convenient, non-intrusive monitoring of patients in their daily lives. This leads to higher compliance and retention rates in clinical trials:
- 4.53/5 score on the usability satisfaction scale.
- 93% compliance rate during clinical trials
Digital endpoints offer more reliable and sensitive assessments than traditional methods. Continuous passive monitoring reduces bias and accurately reflects the patient's real-world function, providing a detailed picture of health status and treatment response.
Digital endpoints can detect treatment effects more quickly and with fewer patients; For instance, to demonstrate the same evolution in a statistically significant way, a DMD study would have to include 112 patients over 1 year using the 6MWT as opposed to 23 patients over 6 months using SV95C.
Digital endpoints could significantly increase the expected net present value (eNPV) of clinical trials, especially in phase 3 trials, where the increase in eNPV ranged from $27 million to $48 million. Thus, the community needs endpoints that can help to conclude faster and with more confidence on treatment benefits.
The Syde platform includes two wearable sensors for precise movement capture, a back-end IT platform with algorithms for digital endpoint calculation, and services for clinical operations.
This validation offers a valuable alternative to the 6-Minute Walking Test for evaluating ambulant DMD patients.
SV95C is more sensitive to change compared to traditional gold standards. It shows the effect of treatment with statistical differences.
This qualification and the promising results in other pathologies open up new horizons, paving the way for the widespread adoption of digital endpoints as key evaluation criteria in clinical trials.
By personalizing the treatment for every patient, the clinical treatment paradigm is shifting from a trial-and-error approach to "the right drug, at the right time, for the right patient."
Sysnav Healthcare invests in R&D and technologies that produce, manage, and analyze real-world data.
By offering individualized and cost-effective personalized medical solutions, we aim to transform healthcare.