FDA Perspectives on Digital Health Technologies in Clinical Trials
By: Vera Kovacevic, PhD
Interviewees: Elizabeth Kunkoski, Health Science Policy Analyst, CDER, FDA; and
Leonard Sacks, Associate Director, Office of Medical Policy, CDER, FDA
FDA Perspectives on Digital Health Technologies in Clinical Trials
By: Vera Kovacevic
Interviewees: Elizabeth Kunkoski, Health Science Policy Analyst, CDER, FDA; and
Leonard Sacks, Associate Director, Office of Medical Policy, CDER, FDA
There has been a remarkable surge in the adoption of digital health technologies (DHTs) in clinical trials over the past decade.
For instance, the relative use of DHTs in clinical trials for four chronic neurological disorders, epilepsy, multiple sclerosis, Alzheimer’s disease and Parkinson’s disease, was found to increase from 0.7% in 2010 to 11.4% in 2020. This illustrates a significant shift towards remote data collection and continuous monitoring of patient outcomes.
Wearable sensors, mobile apps and remote monitoring devices are now becoming more integral in capturing real‐time, patient-centric data that traditional in‐clinic measurements could often miss. Such digital approaches not only enhance the level of detail in the collected data but also help enable decentralized trial designs, potentially reducing logistical barriers and increasing patient participation.
The FDA has undertaken proactive measures to simplify the regulatory landscape for digital tools used in trials.
Notably, the regulatory agency has issued guidance on “Digital Health Technologies for Remote Data Acquisition in Clinical Investigations,” emphasizing rigorous verification, validation and usability evaluations to ensure that these technologies are fit for purpose.
Additional initiatives by the FDA include the establishment of the DHT Steering Committee, regular public meetings and early engagement programs under the Prescription Drug User Fee Act (PDUFA VII) to facilitate dialogue between sponsors and regulators.
These efforts aim to speed up the integration of innovative digital endpoints while promoting a more patient-centric and efficient clinical trial environment.
In this Xtalks Clinical Edge™ interview, Elizabeth Kunkoski, Health Science Policy Analyst at CDER, FDA, and Leonard Sacks, Associate Director, Office of Medical Policy at CDER, FDA, discuss the impact of DHTs in clinical trials.
Their discussion covers rigorous evaluation processes, strategies for enhancing patient engagement, challenges in endpoint development and future innovation priorities.
Ensuring Reliability Through Verification, Validation and Usability Evaluations
The two experts from CDER provide valuable insights into the evaluation process that supports the reliability of these tools.
It’s important to ensure that digital health devices function as intended while accurately capturing clinically meaningful data.
Kunkoski explains that the agency emphasizes rigorous verification and validation to ensure that a device is “fit for purpose.” She details that verification involves confirming that a sensor meets its performance specifications, while validation is divided into analytical and clinical phases.
Analytical validation ensures that raw data — such as acceleration data translated into step counts — is accurate, whereas clinical validation assesses whether the measurement is meaningful for a specific endpoint and patient population.
Moreover, Kunkoski notes that usability evaluations are essential to confirm that participants can operate the technology correctly, thereby safeguarding data integrity during clinical investigations.
Sacks further emphasizes that in addition to technical accuracy, the real-world utility of DHTs ultimately depends on user interaction; as he puts it, “if patients don’t use these things properly, they’re not going to be of any value.”
Together, their perspectives show the FDA’s stringent evaluation process. By integrating thorough verification, analytical and clinical validation and targeted usability assessments, the agency aims to ensure that DHTs deliver reliable, actionable data in clinical trials.
“The use of DHTs is one of the strategies which allows us to get data directly from patients who don’t have to come into clinic sites.”
— Leonard Sacks, Associate Director, Office of Medical Policy, CDER, FDA
“The use of DHTs is one of the strategies which allows us to get data directly from patients who don’t have to come into clinic sites.”
— Leonard Sacks, Associate Director, Office of Medical Policy, CDER, FDA
Enhancing Patient Engagement and Broadening Participation in Clinical Trials
Enhancing patient engagement and broadening participation in clinical trials is a critical aspect of modern clinical research, especially when integrating DHTs.
DHTs facilitate remote participation, which not only makes it easier for patients to contribute data from the comfort of their homes but also opens up clinical trial opportunities to groups that have historically been underrepresented.
As Kunkoski explains, “The patient doesn’t have to come into the clinic, and we really think we can reach some different patient groups that we are not otherwise able to reach, like different populations, older adults, individuals with disabilities or those that are juggling home or childcare or work responsibilities or live far from research facilities.”
This approach improves convenience and reduces the burden on participants. It also enhances the generalizability of study outcomes by capturing a broader spectrum of real-world experiences.
Sacks further emphasizes the importance of these strategies, saying, “The use of DHTs is one of the strategies which allows us to get data directly from patients who don’t have to come into clinic sites.”
By enabling remote data collection, DHTs help overcome geographical and logistical barriers that can limit trial participation.
These insights show how DHTs can transform clinical trials into more inclusive, patient-centric studies that enhance recruitment and retention. They can also ensure that trial populations more accurately represent the target population of the real-world patients who will use the medical product.
Justifying Novel Endpoints Derived from DHT Data
When it comes to establishing clinical trial endpoints based on DHT data, both experts agree that the process is similar to that for any novel endpoint.
Sponsors must justify that the endpoint is clinically meaningful and that the DHT used to collect the data has undergone rigorous verification and validation. This involves confirming that the device can reliably capture the necessary data and ensuring that the measurement truly reflects the underlying clinical event or outcome.
Kunkoski points out that sponsors should “engage a wide array of stakeholders — patients, caregivers, clinicians, engineers, statisticians and regulators — to build a robust body of evidence that supports the new endpoint.”
This collaborative approach is crucial because it ensures that all perspectives are considered in validating the endpoint’s relevance, from the patient experience to the technical accuracy of the measurement.
In addition, sponsors need to develop detailed analytical plans that address potential issues, such as handling missing data and accounting for variability in continuous measurements — challenges that are particularly pronounced with data types like step counts or daily activity levels.
As Sacks explains, “In some cases we’re measuring things which haven’t been measured before. So there’s no standard metric for comparison and one has to develop a body of evidence that supports their clinical validity.”
By addressing challenges such as establishing standard metrics, handling missing data and accounting for variability in continuous measurements, sponsors can build the necessary confidence among regulators that DHT-derived endpoints are both innovative and reliable measures of treatment efficacy.
“We recognize that sponsors may decide at many points throughout the drug development process to include a DHT in their clinical investigation. We certainly hope that the earlier they approach the FDA to discuss the topics, the more streamlined the process will be to collaborate and develop a clinical investigation that will be successful.”
— Elizabeth Kunkoski, Health Science Policy Analyst, CDER, FDA
“We recognize that sponsors may decide at many points throughout the drug development process to include a DHT in their clinical investigation. We certainly hope that the earlier they approach the FDA to discuss the topics, the more streamlined the process will be to collaborate and develop a clinical investigation that will be successful.”
— Elizabeth Kunkoski, Health Science Policy Analyst, CDER, FDA
Capturing Novel Clinical Features Beyond Traditional Methods
Obtaining novel clinical features beyond traditional methods is one of the most exciting aspects of integrating DHTs into clinical trials.
Unlike conventional assessments that provide only a snapshot of patient performance — for example, the traditional six-minute walk test that is performed at a single point in time — DHTs offer continuous monitoring. This 24/7 data collection enables the detection of transient or rare events, such as seizures, arrhythmias or falls, that might occur outside the narrow window of scheduled clinic visits.
This capability can allow for a more comprehensive view of how patients function throughout the day, whether at home, at work or even during sleep.
Moreover, DHTs can capture new types of data that were previously inaccessible.
As Sacks noted, “The advantage of these DHTs is that you can get information from patients who normally couldn’t give it, either infants or children or patients with cognitive disabilities.”
For instance, continuous monitoring can quantify behaviors such as scratching at night in children with atopic dermatitis, offering insights that traditional methods simply could not capture.
Additionally, DHTs are being leveraged to track biomarkers like glucose levels, vital signs and even more complex behavioral patterns. These data points not only enhance our understanding of a patient’s daily life but also provide a richer context for evaluating the safety and efficacy of new therapeutics.
This approach paves the way for designing more patient-centered clinical trials that can adjust to the subtle realities of living with chronic conditions.
The Importance of Early Engagement with the FDA
Early engagement with the FDA is essential for sponsors integrating DHTs into their clinical trials.
“The earlier in the programs that they can do this, the more likely we’re going to reach a common goal there,” says Sacks.
Approaching the agency at the outset of the drug development process helps to identify potential hurdles, streamline the submission process and ensure that all aspects of the DHT — from its technical performance to its clinical application — are clearly understood and aligned with regulatory expectations.
Engaging early not only facilitates a smoother review process but also fosters a collaborative environment where the FDA can provide tailored feedback on design, validation strategies and potential endpoints.
“We recognize that sponsors may decide at many points throughout the drug development process to include a DHT in their clinical investigation. We certainly hope that the earlier they approach the FDA to discuss the topics, the more streamlined the process will be to collaborate and develop a clinical investigation that will be successful,” explains Kunkoski.
Some of the key benefits of early FDA engagement include:
- Alignment on Technical Specifications: Early discussions ensure that the DHT meets rigorous verification and validation requirements.
- Risk Mitigation: Addressing challenges such as data variability, missing data and usability concerns early on helps in developing robust risk management strategies.
- Regulatory Clarity: Early engagement provides clarity on documentation requirements and the agency’s expectations regarding novel endpoints, ensuring that sponsors can prepare comprehensive and compliant submissions.
- Enhanced Collaboration: By participating in initiatives like the FDA’s DHT Steering Committee, sponsors benefit from ongoing dialogue and collective problem-solving, which ultimately helps align technical and clinical goals.
- Streamlined Clinical Trial Design: Input from the FDA during the IND phase or before the end-of-Phase 2 meeting (EOP2) can inform key aspects of trial design, including endpoint justification and patient engagement strategies, leading to a more efficient investigation.
By initiating conversations at the earliest stages, sponsors set the stage for a more predictable and aligned regulatory pathway.
This proactive approach increases the likelihood of developing a clinical study using a DHT that meets regulatory standards and addresses patient needs effectively.
Priorities for DHT Innovation
Looking ahead, Sacks says the FDA sees tremendous potential for DHT innovation to reshape clinical trials and ultimately improve patient outcomes.
He highlights two priority areas that have not yet been fully tapped.
First, pediatric diseases represent an opportunity where continuous, remote data collection can transform how trials are conducted in a population that traditionally faces significant logistical challenges.
Digital tools could enable direct measurement of physical function in children, improve recruitment in rare pediatric disorders and ultimately facilitate the development of more effective treatments for this vulnerable group.
The second key area is interactive DHTs — tools that require active patient engagement rather than passive data collection. Sacks suggests that these technologies can capture more nuanced data through tasks like strength tests, cognitive assessments or balance exercises.
Interactive DHTs can not only provide clinically meaningful endpoints by measuring dynamic aspects of patient performance, but they can also foster a more engaging and personalized experience for patients.
These technologies could enable real-time feedback and adjustment, allowing patients to actively participate in the measurement process rather than simply having data passively recorded.
This focus on interactive tools opens the door for novel approaches that capture subtle changes in physical or cognitive functions — data that might otherwise go unnoticed with traditional passive monitoring. In turn, this could enhance the overall quality and relevance of clinical trial outcomes.
The FDA’s evolving approach to DHTs in clinical trials marks progress in modernizing clinical research. By emphasizing robust verification and validation, facilitating broader patient participation and encouraging early dialogue with sponsors, the agency is laying a strong foundation for the future of clinical investigations.
The insights shared by Kunkoski and Sacks highlight the potential of DHTs to improve data collection and endpoint evaluation and ultimately enhance patient outcomes across diverse therapeutic areas.
ABOUT Elizabeth Kunkoski
Beth Kunkoski currently works in FDA’s Center for Drug Evaluation and Research (CDER), Office of Medical Policy (OMP) Clinical Methodologies Team. She oversees several digital health technology projects fulfilling PDUFA commitments and other efforts related to electronic records and signatures in clinical investigations (Part 11). She previously worked in the Center for Devices and Radiological Health (CDRH) overseeing the review of orthopedic devices. She earned a Master’s Degree in Biomedical Engineering and a Bachelor’s Degree in Chemical Engineering from the University of Michigan.
ABOUT Leonard Sacks
Leonard Sacks received his medical education in South Africa, moving to the USA in 1987, where he completed fellowships in Immunopathology and Infectious Diseases. He worked as an attending physician at the Veteran’s Affairs Medical Center in Washington DC and in South Africa at Sizwe hospital; a referral center specializing in tropical infectious diseases including drug resistant tuberculosis. He joined the FDA in 1998 as medical reviewer in the Office of New Drugs with a special interest in drugs for tuberculosis and malaria. His subsequent positions included acting director of the Office of Critical Path Programs and Associate Director for clinical methodology in the Office of Medical Policy in the Center for Drug Evaluation and Research. In this capacity he has led efforts to support innovative approaches to clinical trials including the use of digital health technology, the decentralization of trial activities and the integration of clinical care and clinical research.