Revolutionizing Drug Development? First Primary Qualification by a Regulatory Agency Ushers in New Era for Digital Endpoint
By damien eggenspieler, Sysnav Healthcare Program Director
Approval of novel drugs is at a record high in the last years: CDER approved 46 new drugs per year from 2020 to 2022 vs. 30 per year from 2010 to 2012, a 50% increase (link to FDA website). Among them, the rate of expedited development and review methods grew from 60% (in 2015) to 65% (in 2022). So, while we are all watching and praising AI as an amazing technology to generate new drug candidates, one thing is certain: we will need to equip the industry with new Drug Development Tools. Tools that can help drug developers to better prioritize their portfolio based on early efficacy data, tools that provide regulators with robust data for accelerated decision making and tools that can comfort patients and patient communities of the efficacy of the newly available treatments.
Digital endpoints are the cornerstone of those new tools. They offer the potential to provide improved validation and sensitivity in detecting the efficacy of drugs — advancements that could, in turn, lead to early decision making, leaner and shorter clinical trials (Colloud et al., 2023). These efficiencies are particularly beneficial for clinical trials in rare diseases in which participant recruitment can be a challenge. What’s more, for healthcare providers, the real-time data collection enabled by wearable technologies can lead to improved assessment of patient outcomes to guide treatment decisions and allow for more personalized care.
A dire need for improved Drug Development Tools
Regulatory agencies around the world involved in drug development have recently upgraded their program to qualify endpoints used to determine success in a clinical trial. The European Medicines Agency (EMA) was the first to qualify a digital outcome measure: it recently qualified stride velocity 95th centile (SV95C) as a primary digital endpoint in superiority studies as an alternative to the 6-Minute Walking Test for ambulant Duchenne Muscular Dystrophy (DMD) patients aged 4-years and older. DMD is a rare genetic disease that affects the muscles, leading to weakening and muscle wasting that can impair mobility and eventually become fatal (NCATS GARD). This qualification makes SV95C the first digital endpoint to receive primary endpoint qualification by a regulatory agency, marking a major milestone in the integration of digital healthtech to evolve drug development, patient participation in clinical trials, and more precise measurement of treatment effectiveness.
SV95C: The first digital endpoint qualified by a regulator as a primary endpoint
Conventionally used endpoints are measured in clinical settings by healthcare professionals. These assessments have limited reliability and sensitivity and place an immense burden on patients suffering from debilitating diseases that affect mobility, which makes it difficult to travel to the clinic. With recent advances in technology, digital endpoints have emerged as compelling alternatives. SV95C is an objective digital ambulation measure of peak performance, derived from Sysnav’s Syde® wearable device, which relies on state-of-the-art motion capture technology to provide objective, real-world measurements of user movement. By employing sensors across diverse device types and real-life contexts, it becomes possible to generate meaningful data beyond clinical settings, such as during patients' everyday activities. This approach contributes to the enhancement of convenience and efficiency for patients, caregivers, clinicians, drug developers, and healthcare providers alike. (Landers et al., 2021)
Expanding digital endpoints to new conditions and beyond clinical development
The expansive potential of digital health technologies and their application in digital outcome measures for clinical trials and healthcare practices extends beyond DMD to other neuromuscular and neurological diseases.
Digital health technologies and the digital endpoints they enable unlock a path to more efficient, more convenient clinical research and improved patient outcomes. Now is the time to evolve the way mobility outcomes are measured. The qualification of SV95C as a primary digital endpoint in DMD is a significant advancement but is only the first step to build the right set of tools for drug developers and to exploit the full potential of technology to drive meaningful improvements in patient care.
A collective endeavor
The ultimate goal is to improve the lives of patients who live with severe conditions and deserve more responsible and precise data collection from the scientific community to inform treatment decisions, patient outcomes, and drug discovery. Over the years, we have developed a cutting-edge digital endpoint platform, making us a partner of choice for those seeking to transform the way clinical trials are conducted. But the SV95C success was only possible with the involvement of the entire Duchenne community, from patients’ organizations across the globe (from Parent Project Muscular Dystrophy to MD UK, World Duchenne and many others) to many clinicians, boards and drug developers.
To venture into drug development and clinical research arenas beyond DMD, there is a pressing need for continued refinement and validation of endpoints specifically tailored to these indications. This crucial task calls for continued collaborative efforts between pioneers in digital health technology, academic experts, pharmaceutical developers and regulators.
Regulatory bodies are prioritizing this topic. We are noticing this every day, from the quality of exchanges, the engagement of the officials involved in the review processes, and the accelerated answers to requests. We are witnessing the growing endorsement from patients, advocacy organizations, and caregivers, enabling widespread support for digital endpoint development and the implementation of digital healthcare technology approaches in research and practice. The industry is gaining in maturity and structure with groups like the DiMe (Digital Medicine) Society, C-PATH Institute, and others who share policy and development guidelines.
Conclusion
The future success of drug development requires tools that detect meaningful changes in disease progression earlier and faster. Robust digital endpoints have the potential to revolutionize the tracking of disease progression, lessen the burden on patients and benefit drug development efforts. The first-ever regulatory qualification of SV95C as a primary efficacy endpoint is a notable milestone, but only the tip of a powerful and promising iceberg that can transform outcomes for patients who are still waiting for effective treatments.