Donna Mongiello, RN, BSN, Vice President Strategic Solutions

Last week, YPrime was among nearly 2000 participants who descended upon sunny Orlando for the 10th Annual SCOPE meeting. Expert perspectives, insights gained from lessons learned and forward-looking predictions across 10 parallel tracks focused on functional areas and emerging disciplines within clinical development.

The Sensors, Wearables and Digital Biomarkers forum demonstrated how new technology and digital approaches are transforming the development of emerging therapies. Large pharma dominated this discussion, with representatives from Abbvie, GSK, Pfizer, Janssen, Roche and Novartis. Common themes and food for thought across an array of presentations included:

Therapeutic Area Focus. Scores of examples involving sensors, wearables and digital biomarkers focused on CNS-related indications, reinforcing how neuroscience represents the biggest opportunity for sensor-based measures, due to well-known challenges and potential benefits:

  • A lack of classical biomarkers in CNS diseases
  • Complementary objective data and qualitative evidence from patients and caregivers
  • The ability to generate a richer data set, while reducing the bias of traditional clinical scales

Use cases highlighted pilot study results of digital biomarkers across Parkinson’s disease, multiple sclerosis, Huntington’s disease, amyotrophic lateral sclerosis and schizophrenia, featuring algorithm testing, technology validation and assessment of exploratory endpoints.

Recognition of Progress. In her opening remarks, chairperson Michelle Cruothamel of GSK tracked the digital progress of the last 10 years. She also described “sensorification,” referring to the disconnect between hype about digital health expectations and actual delivery within clinical development. Many of the case studies that followed reflected efforts that began around 2013. The takeaway? While progress is slow, biopharma commitment is real, and the focus is on learning. Timothy Kilchermann presented a tripartite analysis schema that resulted from Roche’s digital biomarker efforts in Huntingdon’s disease:

  • Agreement and correlation between sensor-based measures and clinical scales is key.
  • Patient Compliance – Remote patient monitoring can translate patient behavior into digital information, providing clear insights into daily compliance and task completion. Data presented on patient compliance by Michelle Marlborough of AiCure revealed that approximately 40% of clinical trial participants don’t take the study drug. What’s more is that over time, even the most engaged participants eventually stray from dosing regimens.
  • Augmentation refers to detection of patterns of importance via greater sensitivity analysis that maybe otherwise easily overlooked. Biomarker data can measure disease pathology and progression, not only through more feature analysis, but also through assessment of signs not reported by patients that may be important indicators in disease progression. Granular sensitivity analysis also offers the potential to predict events that ultimately lead to earlier disease detection, as well as the use of smaller patient sample sizes and shorter trial durations. Michelle Marlborough took this concept one step further when discussing the capture, measurement and modification of patient behavior through computer vision technology. Patient-specific models capture deviations in “normal” behavior across facial or audio expressivity, movement, concentration and cognition. What’s promising for current drug development efforts is the applicability of these models across all disease areas, she concluded.

Planning that’s grounded in product strategy. Technology selection, advised Luis Garcia-Gancedo of GSK, must be based on thorough measurement methodology, with suitability for the patient population and disease characteristics. Next, the clinical hypothesis needs to map to the value generation plan. A typical plan to generate value spans patient centricity, efficacy and efficiency. Citing CTTI’s novel endpoints project, short-term benefits for patient centricity may include development of high quality, mobile technology-derived endpoints that are meaningful to patients. Medium- and long-term benefits may include fewer barriers to trial participation and more trials that yield better information to inform labelling claims and reimbursement decisions.

Open Collaboration. Nearly every presenter spoke to the importance of sharing data to advance the science and promote adoption of digital health. This concept was explicitly articulated by Timothy Kilchermann of Roche, using floodlightopen as an example. As an open app research trial for multiple sclerosis, researchers can access the complete dataset and integrate the digital assessments into their own research. Panel discussion participants also emphasized the value of working with industry consortia for endpoint development.

Regulatory considerations. Regulatory uncertainty is a perennial theme with novel technology. Kelley Erb discussed Pfizer’s engagement with FDA involving novel assessments of motor functions for people with Parkinson’s disease. Dual objectives consisted of informing the agency and obtaining feedback on development and validation for use supporting clinical endpoints. In summarizing FDA responses, he explained the agency’s desire to see exploration of new clinically relevant information that cannot be obtained through existing outcome measures. Erb also urged the importance of identifying the right measurement first, followed by finding by the appropriate fit-for-purpose technology. The panel discussion that followed reinforced how it’s often not clear how a new digital biomarker will expand information already provided by a current clinical assessment. This challenge persists, even with the potential to see way beyond a typical PRO, with daily fluctuations, progress over time, and better longitudinal data over snapshot assessments.

External partnerships are critical for accelerating success and overcoming challenges of how to collect and analyze data, especially the task of managing and contextualizing vast quantities of data. Collaborations mentioned included GSK and McLaren Applied Technologies, Propeller Health, among others.

Overall, the presenters gave the audience reason to notice the progress and celebrate industry achievements. The most convincing reminders of how far we have come came from Stephen Amato and Greg Silvesti through these observations:

  • When explaining the five development stages of endpoint assessments through mobile technology collection created by the Pfizer Innovation Research Lab, Stephen Amato concluded that many biopharma leaders are beyond the pilot, working towards validation. His conclusion suggested more progress in clinical development is underway than often acknowledged.
  • There was clear consensus on the idea that while iteration is fundamental to success, it’s also the area where most people give up, explained Greg Silvesti of AbbVie. The process of testing, learning, refining and implementing requires the long view. Small wins vs. big milestones are critical for pushing the future of clinical research.