Digital endpoints in neuroscience: From breakthrough potential to breakthrough results
The landscape of neuroscience trials is transforming before our eyes. What was once considered futuristic - digital biomarkers, wearable sensors, AI-powered analytics - is now delivering measurable results in clinical development. The question isn't whether to integrate digital endpoints anymore. It's how to do it strategically.
Listen to our two-part podcast series where our experts share real-world insights, regulatory strategies, and practical guidance for moving from pilot to scale.
The momentum is building fast
Digital endpoints are gaining serious regulatory traction. The FDA has already approved stride velocity measurements for muscular dystrophy trials1, and we're seeing breakthrough approvals for Parkinson's disease monitoring using patient-generated wearable data2.
The regulatory appetite is there too. With December 2023's FDA guidance on Digital Health Technologies for Remote Data Acquisition3, plus evolving EU frameworks, agencies are actively embracing these tools while maintaining rigorous standards for validation and clinical meaningfulness.
“Our industry is experiencing a strong push to move from exploratory biomarkers to clinically validated endpoints,” said Alicia Baker McDowell, DRSc, MS, EMBA, Vice President and Head of Global Regulatory Strategy. “This shift is crucial for building robust validation datasets before pivotal trials. Early dialogue with health authorities can help provide clarity on the right DHI as well as proactively identify and address potential regulatory concerns.”
Where the real value lies
The power of digital endpoints isn't just in replacing traditional measures - it's in what happens when you combine them. Think about it: instead of relying solely on subjective patient reports about sleep quality in a pain study, you can now objectively measure actual sleep patterns, movement, and even brain activity through digital biomarkers. This hybrid approach is transforming how we understand treatment effects and overcome persistent challenges like placebo response.
But perhaps most importantly, digital endpoints are empowering patients like never before. Wearables and digital monitoring tools give participants unprecedented insight into their condition, allowing them to take ownership and control of their disease journey4. As our population ages and becomes increasingly digitally enabled - with older patients embracing technology as part of everyday life - we need to challenge outdated perceptions about digital adoption and think bigger about who can benefit from these innovations.
For precision medicine in neuroscience, digital endpoints offer something invaluable: the ability to detect individual variability and build confidence that your drug is working as intended in each patient, not just at the population level.
From theory to practice
In our conversation with AiCure® in our recent podcast, we revealed what separates successful digital health implementations from those that struggle to deliver value. Timing matters – whether you're in early development or well into your program, digital endpoints can add value, but starting earlier gives you strategic advantage. Choosing the right partner goes beyond technology specs; look for vendors who understand the science, navigate regulatory requirements, and fit with your team culture and patient population needs. Most importantly, think beyond measurement – the most successful implementations evolve dynamically throughout trials, providing insights that compound across your entire development strategy.
Success demands the right operational approach too. You need to plan for device variability, patient compliance challenges and the complexities of real-world data collection. But with AI analytics now processing data in seconds rather than weeks, these operational challenges are becoming increasingly manageable. The winning formula? Early regulatory dialogue, smart vendor collaboration, and a willingness to start with exploratory endpoints that build toward primary endpoint validation
The future is hybrid
What excites us most is where this is heading. Imagine Alzheimer's trials where traditional cognitive assessments are enhanced by continuous monitoring of sleep, movement and brain function. Or pain studies where objective biomarkers help overcome the subjectivity around pain assessment that has long challenged this therapeutic area.
Your next move
The key to success? Start thinking beyond traditional endpoints now. Begin with exploratory measures to gain experience, engage with regulators early and often, and choose technology partners who understand both your indication and your patients.
“We often recommend incorporating DHI in Phase I to capture valuable data as these studies often enroll patients who can cope with longer study durations,” said Leone
“Oftentimes in Phase II, DHI can capture sufficient preliminary positive data with high clarity. If a sponsor doesn’t need to repeat their Phase II study or can expedite their program as a result of DHI, the investment more than demonstrates its value.”
Ready to explore how digital endpoints can transform your neuroscience trials?. Contact us today
References
- 1. Mullard A. FDA approves first gene therapy for Duchenne muscular dystrophy, despite internal objections. Nat Rev Drug Discov. 2023;22:610–611. Available from: https://www.nature.com/articles/d41573-023-00103-y
- 2. Schalkamp AK, Peall KJ, Harrison NA, Sandor C. Wearable movement-tracking data identify Parkinson’s disease years before clinical diagnosis. Nat Med. 2023;29:1745–1755. Available from: https://www.nature.com/articles/s41591-023-02440-2
- 3. U.S. Food and Drug Administration. Digital Health Technologies for Remote Data Acquisition in Clinical Investigations. Guidance for Industry, Investigators, and Other Stakeholders. December 2023. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/digital-health-technologies-remote-data-acquisition-clinical-investigations . Last accessed 20th August 2025.
- 4. Smuck M, Odonkor CA, Wilt JK, Schmidt N, Swiernik MA. The emerging clinical role of wearables: factors for successful implementation in healthcare. npj Digit Med. 2021;4:45. Available from: https://www.nature.com/articles/s41746-021-00418-3 . Last accessed 20th August 2025.