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  1. Guidances | Drugs

Guidance Recap Podcast | Digital Health Technologies for Remote Data Acquisition in Clinical Investigations

Thank you for joining us for another episode of the Guidance Recap Podcast. The Guidance Recap Podcast provides highlights for FDA guidance documents straight from the authors. My name is Kylie Haskins, and I am the host for today’s podcast. In today’s episode, I am excited to be talking with Consumer Safety Officer Elizabeth (Beth) Kunkoski, from the Office Medical Policy and Associate Director Anindita (Annie) Saha from the Digital Health Center of Excellence. They will be sharing some thoughts with us on the newly published final guidance titled Digital Health Technologies for Remote Data Acquisition in Clinical Investigations. Welcome, Beth and Annie! Thank you for speaking with us today.

Podcast

Annie, for listeners less familiar with this topic, what is a digital health technology, and why is it important in medical product development?

A digital health technology (also called DHT) is a system that uses computing platforms, connectivity, software, and/or sensors, for health care and related uses. Some examples of DHTs include consumer products like fitness trackers that monitor steps, heart rate, and sleep patterns; and devices like continuous glucose monitors used in diabetes management or wearable blood pressure monitors. DHTs may also rely on or work with other technologies that support their operation, such as general-purpose computing platforms, like smartphones, as well as communication networks. Each of these DHTs collects specific data, offering insights into various aspects of an individual’s health.

DHTs are becoming important in medical product development, including during clinical investigations. Their ability to capture data remotely can allow for continuous monitoring of individuals, which is particularly beneficial in clinical trials. They can enhance the efficiency and effectiveness of investigations by providing real-time, comprehensive data and can also reduce the burden with less in-office medical visits. DHTs have the potential to provide more patient-focused data for analysis, may lead to improved patient engagement and compliance, and potentially accelerate the development and evaluation of medical products. We see that the use of DHTs can help support equity and access to trials that may not have been available to people before.

Beth, why did FDA issue guidance on Digital Health Technologies for Remote Data Acquisition in Clinical Investigations?

One of the things FDA aims to do is improve the science and efficiency of clinical investigations. Really, the question of how to use technology to improve clinical investigations has been around for decades. As a part of this effort, FDA established a public docket in 2015 to solicit input from a broad group of stakeholders on the scope and direction of new technologies and their innovative uses in clinical investigations. Public comments received in response to the docket highlighted the need for clear communication of best practices for employing these innovative technologies in clinical research, and FDA published a draft version of the guidance in 2021 to meet this need.

This guidance is piece of a larger effort by the Agency to support the use of DHTs. In 2020 FDA created the Digital Health Center of Excellence to empower stakeholders to advance health care by fostering responsible and high-quality digital health innovation. Then, the most recent Prescription Drug and the Medical Device User Fee Acts, as well as the Food and Drug Omnibus Reform Act of 2022, also known as FDORA, included DHT-related commitments. These commitments include holding public meetings, publishing guidance, developing a framework for the use of DHTs in drug and biological product development,1 and more. CDER has developed a DHT Steering Committee to facilitate consistent approaches to the review of drug submissions that contain DHT-derived data and to engage with external stakeholders. Please see CDER’s webpage “DHTs for Drug Development” for additional information.2

Annie, can you tell me more about the Digital Health Center of Excellence?

The FDA Digital Health Center of Excellence (also called DHCoE) is an office in the Center for Devices and Radiological Health (CDRH) that aims to advance the latest developments in digital health technology and provides technological and policy advice to support furthering uses of cutting-edge DHTs. The DHCoE plays a fundamental role within FDA, setting and leading strategic direction in digital health policy and to collaborate and coordinate digital health-related efforts across the Agency.

What’s exciting about the DHCoE is how we extend our services to a wide range of digital health stakeholders, not just those traditionally involved in the health care space. This comes down to translating digital innovations into practical tools directly benefiting consumers. The DHCoE connects with stakeholders and builds partnerships to support digital health advancements, shares knowledge for promoting awareness and understanding, and drives best practices in the field. The DHCoE plays a crucial role advancing regulatory approaches that aim to provide efficient and least burdensome oversight. Our work supports the Agency with crosscutting issues related to DHTs, focusing on forming policies and partnerships to nurture innovation and using DHTs to enhance health care delivery effectively.

One tool we have developed is the Digital Health Policy Navigator, which is accessible on the DHCoE website. It is designed to help stakeholders navigate the digital health regulatory landscape more easily by helping identify whether their product’s software functions are potentially subject to FDA’s regulatory oversight as a device, and if so, providing considerations that may assist in determining the applicable FDA-specific legal and regulatory requirements and recommendations. The Policy Navigator contributes to the broader goal of advancing high-quality digital health innovations.

Beth, can you discuss the role of DHTs in enhancing patient engagement in medical product development and trial participation diversity?

DHTs are really changing the game in patient engagement and diversifying participation in clinical trials. They allow individuals to be part of clinical trials right from their homes or workplaces, making joining clinical trials and staying engaged more convenient – this flexibility largely wasn’t possible before innovation in the field of DHTs.

Now, there’s also the concept of Decentralized Clinical Trials, for which FDA has issued draft guidance, where digital health technologies are a key component. Decentralized clinical trials let participants complete trial-related activities remotely, which means they don’t always have to go to traditional trial sites like a hospital or other clinical setting. Not only can this approach reduce the need to have multiple clinical trial sites, it can also support the inclusion of individuals who usually find it hard to participate in trials – like diverse racial and ethnic groups, older adults, individuals with disabilities, those juggling significant home or childcare responsibilities, and those living far from research facilities.

This all ties in with FDA’s Diversity Plan for Clinical Investigations, which urges sponsors to develop a Diversity Action Plan. Just to clarify, a diversity action plan is a strategic approach sponsors develop and use to ensure their trials include a broad spectrum of participants and is now mandated by law. These diversity action plans are crucial in making sure the trial outcomes are relevant to a wide array of patients. Having diverse representation is key to understanding how effective and safe medical treatments are across different demographics.

Annie, is a DHT a regulated device?

So, is a DHT a regulated device? Some DHTs are devices, and some are not. This depends on if the DHT meets the definition of a device per the Federal Food Drug & Cosmetic Act. When a DHT makes a claim (for example, through labeling) that it is intended to diagnose, treat, cure, or prevent a disease or condition, it is a medical device regulated by FDA. I’ll refer to these types of claims as medical claims. For example, if a wearable manufacturer claims that a DHT is capable of monitoring blood glucose levels for diabetes management, then that is a medical claim, which would make it a medical device under the Federal Food Drug & Cosmetic Act. On the other hand, if a DHT is marketed solely for general wellness or for non-medical purposes – say, a fitness tracker that not only counts steps but monitors sleep patterns without a connection to any specific health condition – it might not be considered a medical device.

For DHTs that are medical devices that do not have FDA marketing authorization and are used only in clinical investigations for remote data collection, the activities needed to ensure that such devices will perform as intended in the clinical investigation can vary depending on the device and its specific function, including its context of use. Where sponsors conduct the types of verification and validation activities recommended the guidance to ensure that a DHT used only in clinical investigations for remote data collection is fit-for purpose, FDA does not intend to otherwise assess sponsors’ compliance with certain regulatory requirements. This is discussed in more detail in Section III of the guidance. Overall, sponsors should engage early with the appropriate Center responsible for the medical product under investigation to discuss use of DHTs in a specific clinical trial.

In addition, there are programs to obtain formal FDA qualification for tools to assess clinical outcomes assessment or biomarkers, including those that are digitally-derived, known as Drug Development Tools or Medical Device Development Tools. These are voluntary programs that are independent of an individual marketing submission. Tools qualified under these programs have been found by FDA to be scientifically valid and to work as intended for a specified context of use, which means the tool consistently produces reliable results that can be interpreted and applied in medical product development and regulatory decision-making.

Overall, getting on the same page with FDA early regarding how you plan to use DHTs in your clinical investigations can be helpful in navigating the regulatory landscape and ensuring your use of DHTs aligns with FDA expectations.

Beth, why might a sponsor use a DHT in a clinical investigation?

When we talk about DHTs in clinical investigations, it’s really about tapping into their capability to collect continuous data or data at time periods outside of a clinic visit that traditional methods might miss. DHTs are quite versatile – they can capture a wide range of data points, from physiological measurements and behavioral patterns to patient-reported outcomes. Additionally, when used in a clinical investigation, they can provide essential information on the safety and efficacy or effectiveness of medical products being evaluated. Take smartwatches, for instance. They have evolved from mere fitness trackers to sophisticated health monitors with functions capable of producing electrocardiograms to detect heart rhythm irregularities. Or, inhalers, a lifeline for asthma and chronic obstructive pulmonary disease patients, which not only track medication usage but also alert users to environmental triggers in real-time.

It’s not just about collecting a variety of data. DHTs allow for data collection in real-time and in real-world settings, which can provide a more comprehensive picture of a participant’s health status than historical data only captured during periodic trial visits. In essence, DHTs offer an enhanced dimension of data collection, opening new avenues in how we conduct clinical research and evaluate medical products.

Annie, why is using a DHT in clinical investigations important to patients?

DHTs are becoming increasingly important for patients because they offer a unique advantage: they decrease the need for regular visits to the clinical trial site by participants while providing researchers with longitudinal data remotely. Traditional clinical trials are often limited by their ability to only capture a snapshot of the patient’s experience at site visits and to those patients who have the ability to get to a trial site. But with DHTs, researchers can gather data more frequently, sometimes even continuously, over a longer period while reducing the burden on patients. This provides not only a more complete picture of a patient’s health, as mentioned earlier, but also opens up possibilities for developing new endpoints that may be more relevant to patients. For example, real-time continuous glucose monitoring in patients with diabetes provides a more detailed picture of blood sugar levels throughout their daily lives compared to periodic testing. This can provide data that has the potential to make treatments more precise and responsive to individual patient needs.

DHTs also support patient-focused medical product development, which is a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into medical product development and evaluation. We’re learning from patients what is meaningful to them to measure, and oftentimes DHTs are the best tools for measuring these outcomes.

Beth, how does the sponsor define and justify an endpoint based on digitally-derived data for a clinical investigation?

Defining and justifying an endpoint based on data from DHTs in a clinical investigation depends on the same principles for developing an endpoint captured by other means. Ultimately, sponsors want to ensure that the desired clinical outcomes of the study are able to be measured by the DHT.

Sponsors should obtain input from interested parties (such as patients, caregivers, clinicians, engineers, statisticians, and regulators) to ensure that the endpoint is both clinically meaningful and the data are adequately captured by the DHT. A precise definition of an endpoint typically specifies the type of assessment(s) made (for example, activity level, average heart rate, sleep quantity and quality), the timing of the assessment(s), the tool(s) used for the assessment(s), and other details, as applicable, such as if (and if so, how) multiple assessments for a trial participant will be combined.

DHTs may serve as new ways to measure clinical characteristics or events that were previously measured in a clinical setting. Novel endpoints based on data captured by DHTs may also provide opportunities for additional insight into participant function or performance that was previously not easily measurable (for example, tremors).

If a DHT is selected, sponsors should provide evidence that the DHT’s measurements are precise and accurate such that it is fit-for-purpose for its use in the trial. Sponsors should also provide justification that the data collected by the DHT would provide meaningful insights into the safety and efficacy or effectiveness of the product under investigation.

Annie, what are the key considerations when selecting a DHT for use in a clinical investigation?

There are a few things to consider when it comes to selecting the right DHT for a clinical investigation to make sure the chosen technology lines up with the trial’s intent and the specific characteristics of the participant population. Sometimes, more than one DHT may be needed to capture the data required in the study design.

Not only should sponsors think about the disease or condition under study, but they should also consider participants themselves. Participant age, language, and ability to use technology can influence which DHT may work best for a trial.

Sponsors should be sure that the DHT can accurately measure what the study needs it to measure. This means diving into the technical details, like the type of sensors a DHT has, how much data it can store, and how that data will be transmitted. These are critical factors to ensure the DHT is fit-for-purpose, which means checking if the DHT is suitable for the specific context of the clinical trial.

The physical design of the DHT (factors like size, weight, how easy it is to use, and battery life) are also crucial. Considering these elements is key to making sure the device is user-friendly and that sponsors don’t run into issues such as data loss or participant drop out. Sponsors should also consider whether participants can use their own DHTs or other technology in a clinical trial or solely use a sponsor-provided one. It may be beneficial for participants to use familiar technology to boost usability, adherence to study protocols, and improve trial accessibility; but it is also important that data quality is consistent across all technology used, which can be more challenging when participants use their own technology.

Selecting a DHT for a clinical investigation to meet the trial’s needs comes down to a balance of technical suitability, patient compatibility, and operational practicality.

Annie, how should a sponsor approach verification and validation of a DHT for a clinical trial?

The sponsor should start with an initial assessment of the DHT – looking at how it fits into the trial and what impact measurement variation may have on the trial outcomes. Diving into the technical considerations, verification is the next step when determining if a DHT is fit-for-purpose. It is a rigorous check to confirm the DHT is measuring exactly what it’s supposed to, with the accuracy and precision necessary for the trial. For example, a less precise step counter may be acceptable for a trial evaluating general changes in mobility but might not be acceptable for a trial evaluating small mobility differences.

Validation is next, which is a bit different. Here, you’re confirming that the DHT is suitable to measure the clinical event or characteristic you are interested in for your target participant population. Sticking with our step counter example, validation would ensure measuring steps provides a meaningful evaluation of changes in patient mobility resulting from the treatment under evaluation. If the treatment was aimed at improving the patients’ ability to stand up from a chair, then a basic step counter might not necessarily be validated for this use.

This process of verification and validation (V&V) is really a balance of two perspectives together: analytical and clinical. On the analytical side, it is about the accuracy of the DHT’s measurements. Clinically, it’s establishing that measurements, in this case steps, are relevant and meaningful for the investigation.

When determining if a DHT is fit-for-purpose in a clinical trial, it’s not just about the technical side of things, usability evaluations matter too. Usability evaluations look at the DHT in real-world settings with actual users and can help identify any potential issues early on when planning your trial. The information gathered from usability testing should be analyzed and documented with a keen eye on consistency of measurements, reliability, and meeting regulatory standards.

In summary, the sponsor takes the V&V and usability feedback and refinements to fine-tune the use of the DHT, so it is tailored for the investigation.

Beth, could you explain the importance of data protection and cybersecurity in the context of DHTs used in clinical investigations?

When a DHT is used to collect data in a clinical trial, there is a lot of data collected for each trial participant. Sponsors must have policies and procedures in place to ensure the integrity of the data for the trial and to protect the privacy of trial participants. Often a DHT manufacturer has end-user license agreements in place to specify who has access to the data collected by the DHT. The sponsor of the trial is responsible for ensuring participants are informed of who has access to the data in the trial, which may include the DHT manufacturer. The informed consent process should clearly communicate this information to trial participants.

FDA issued the draft guidance titled Electronic Systems, Electronic Records, and Electronic Signatures in Clinical Investigations: Questions and Answers in March 2023 with recommendations on ensuring the integrity of data collected in a clinical trial with a specific section discussing data collected by DHTs.

In addition, FDA has a dedicated cybersecurity webpage and it is a treasure trove of guidelines and recommendations to help mitigate cybersecurity risks with DHTs. It’s a key resource for sponsors in understanding how to guard against potential threats and protect the sensitive data involved in clinical trials.

How do DHTs impact the design and data analysis of clinical investigations?

With the use of DHTs, the design and analysis of the clinical investigation have the potential to be different from a traditional trial, particularly in terms of data collection and statistical analysis. You could be looking at a scenario where data is coming in continuously throughout the duration of the trial. This means you’re dealing with a lot more data which has the potential to increase complexity of the analysis.

DHTs may also introduce challenges when it comes to participant adherence and compliance, something that cannot be overlooked because it directly affects the data’s completeness and integrity. You have to consider how comfortable participants are with the technology, their ability to use the technology correctly, and the absence of a healthcare professional monitoring. How easily can participants use the DHT, how consistent they are with using it, and any technical glitches they encounter – can all impact the data quality and the potential for missing data. To counter this, sponsors must consider this as they develop their statistical analysis plan. Features like automatic reminders to participants, having a run-in period for participants to get used to the DHT, and proactive sponsor outreach may be part of a sponsor’s strategy to combat data quality issues. While DHTs may introduce the risk of missing data, they allow for the collection of a broader range of data, offering much deeper insights into how a medical product impacts a participant. It's all about planning and preparing a robust statistical analysis plan that addresses the considerations discussed today.

Annie, what future developments do you foresee in the integration of DHTs into clinical research?

The integration of DHTs into clinical research is paving the way for some exciting developments in clinical trials. Take, for instance, how DHTs with sensors are revolutionizing how we can collect new types of data to measure what is important to patients. In diseases that affect children, where the traditional trial’s focus was on what a parent reports on their child’s functioning, DHTs can enable tracking of the child’s physical function directly. Analyzing this information might shed light on a more meaningful improvement for patients and could support potential advancements in treatments tailored to improve patient quality of life.

What’s more, DHTs are transforming patient engagement in research. They can collect extensive real-world data as patients go about their daily lives, moving away from the limited snapshots usually captured during periodic clinical visits. It’s a shift toward gathering holistic data, crucial in designing trials that directly impact patients’ quality of life. DHTs are also expanding the footprint of where clinical trials can take place, meeting a participant wherever they may be. Individuals can now participate from their homes or other locations outside of the clinic, offering insights into how diseases influence daily living and overall well-being, which aligns with the trend toward pursuing decentralized trials. FDA’s draft guidance on decentralized clinical trials is a testament to this shift, outlining how technologies like DHTs can be used for remote data collection.

Beth, for our final question, what are a few of key items that you especially want listeners to remember?

As we close our discussion on digital health technologies for remote data acquisition in clinical investigations, there are a few key takeaways we’d love for our listeners to remember. First and foremost, DHTs are truly reshaping the world of clinical trials. It is an exciting time, and we look forward to seeing how these technologies will continue to evolve.

One of the most significant impacts of DHTs is how they are increasing the flexibility of clinical trials. Trials become more accessible and convenient by enabling data collection right where participants live and work. This isn’t just a win for participant convenience; it’s a big step toward diversifying trial populations. Trials become more representative and inclusive, paving the way for research findings that truly reflect the broader population.

Then there’s the role of DHTs in patient-focused medical product development. These technologies can measure the health outcomes that matter most to patients. This means we can better evaluate whether treatments are making a meaningful difference to the patient.

Lastly, advancing the use of DHTs in clinical investigations is a priority for FDA. Engaging early with the Agency when planning to integrate DHTs into clinical investigations can make all the difference in a trial’s success. To support this, FDA launched the DHCoE and the DHT Steering Committee. Both efforts coordinate digital health work across the Agency and support for the inclusion of DHTs in clinical investigations. Listeners should also keep an eye out for more tools and resources from the Agency, aimed at supporting the growth and integration of DHTs in clinical research.

Beth, Annie, thank you for taking the time to share your thoughts on the guidance on digital health technologies. We all have learned so much from your insights on this document. We would also like to thank the guidance workgroup for writing and publishing this guidance.

To the listeners, we hope you found this podcast useful. We encourage you to look at the snapshot and to read the guidance.


1 Framework for the Use of Digital Health Technologies in Drug and Biological Product Development is available at: https://www.fda.gov/media/166396/download
2 Digital Health Technologies (DHTs) for Drug Development webpage available at: https://www.fda.gov/science-research/science-and-research-special-topics/digital-health-technologies-dhts-drug-development#dht

 

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