Navigating the FDA Digital Health Technology Guidance: Insights for Respiratory Drug Developers

In December 2023, the FDA published its final release of “Digital Health Technologies for Remote Data Acquisition in Clinical Investigations” a pivotal step in formalizing regulatory standards and guidance for remote data collection in clinical trials.

As the FDA continues to signal its endorsement of collecting objective data in daily life (e.g. cough and wheeze levels) that “provide a broader picture of how participants feel or function in their daily lives”, respiratory drug developers must consider using digital health technologies (DHTs) in their trials and understand the latest guidance for implementation.

For sponsors’ convenience, we have summarized five key sections from the FDA’s guidance:

1. Selection of a Digital Health Technology and Rationale for Use in a Clinical Investigation
2. Digital Health Technology Description in a Submission
3. Verification, Validation, and Usability Evaluations of Digital Health Technologies
4. Evaluation of Endpoints Involving Data Collected Using Digital Health Technologies
5. Risk Considerations When Using Digital Health Technologies

The foundation of a successful clinical research study lies in the careful selection of a DHT. Strados Labs summarizes the five key principles for sponsors:

1. Consider Clinical Events and Technical Specifications: Thoroughly assess factors such as the clinical event or disease characteristics to be measured, technical specifications, trial population, and trial design. Examine user interface design, data accuracy, reliability, security, and the impact on trial participants.
2. Tailor to Clinical Trial Population: Consider diverse factors like education, language, age, health, and technical aptitude. Paramount to this is conducting usability evaluations to gather feedback and ensure effective use.
3. Identify Technical and Performance Specifications: Determine minimum technical and performance specifications. Specify products meeting these criteria, with the flexibility to consider new models or versions during the trial.
4. Analyze Design and Operation Specifications: Assess design, ease of use, feedback mechanisms, power requirements, operational specifications, alerts, environmental considerations, network capacity, and cybersecurity safeguards. These factors impact trial participants’ compliance, data integrity, and overall success of the clinical trial.
5. Evaluate Participants’ Own DHT and/or Other Technologies: Weigh the pros and cons of allowing participants to use their DHTs. While familiar DHTs may reduce burden, sponsor-provided options should be available to prevent exclusion. Consider specialized uses and the availability of sponsor-provided telecommunication services.

When incorporating DHTs in clinical investigations, sponsors must provide a comprehensive description in submissions. Key components to include are:

1. DHT Suitability and Design: Detail suitability, design, technological features, data output, and measurement methods of the DHT. Describe cybersecurity and usability aspects, access control, and data integrity maintenance.
2. Usability Aspects and Data Flow: Explain the data flow to electronic repositories to ensure consistent data collection that upholds privacy, security, and data integrity throughout the clinical investigation.

Verification and validation steps are necessary to ensure the suitability of Digital Health Technologies (DHTs) for remote data collection, irrespective of device classification. Verification confirms accurate parameter measurement, while validation confirms appropriate assessment of clinical events. This involves comprehensive testing, including benchtop studies, testing in various populations, and contextual considerations.

Interoperability and usability evaluations are essential for DHT verification and validation. Sponsors must ensure seamless information exchange among connected systems by adopting public data exchange standards. Usability evaluations should be used to assess efficient remote use, ensuring accurate measurements and sustained participant engagement. Insights gained can contribute to enhancing design, user-friendliness, and training for trial participants and personnel.

Sponsors are encouraged to engage manufacturers, patients, caregivers, and experts in the process. Key considerations involve comparing measurements with references, evaluating influencing factors, validating calibration, ensuring consistency across DHTs, and addressing potential disparities between remote and clinic measurements.

It is essential to establish clear and reliable methods for assessing participants’ responses to medical products using DHT data. Seeking input from diverse stakeholders ensures clinical meaningfulness and sufficient data capture. Sponsors should also engage in discussions with the relevant review division regarding their endpoint development plans to align with regulatory expectations.

Defining Endpoints

When defining endpoints, specify assessment type (e.g., cough frequency and severity, lung sounds, activity level, sleep/wake time), timing, and tools used, considering the combination of multiple assessments for participants. Consulting disease-specific FDA guidance and relevant review divisions is recommended for specific endpoint considerations.

Novel Endpoints

Novel clinical endpoints often emerge through the use of sensor-based DHTs. Sponsors should justify the use of novel endpoints by assessing whether they reflect participant well-being, predict clinical benefit, relate to existing endpoints, and serve as reliable measures of disease severity. We emphasize early engagement with regulators in the development process.

DHTs may be unfamiliar to both study personnel and participants. The following considerations aim to operationalize DHTs in clinical trials, reducing risks, and enhancing the efficiency of data collection.

Clinical Risks

1. Evaluate physical features for potential discomfort and risk of injury.
2. Conduct safety testing and usability evaluations to minimize risks associated with DHT use.
3. Provide instructions for re-use and cleaning to prevent infection or adverse events.
4. Evaluate the risk of erroneous measurements impacting treatment.
5. Consider cybersecurity threats that may affect DHT functionality and participant safety.

Privacy-Related Risks

1. Be aware of unique privacy risks, including potential disclosure of personally identifiable information or participant locations.
2. Consider end-user licensing agreements that allow data sharing and modifying them to protect participant privacy.
3. Ensure appropriate security safeguards to prevent unauthorized access to data.

Informed Consent

1. Use DHTs for electronic informed consent, following FDA regulations.
2. Address privacy concerns and security measures in the informed consent process.

As clinical investigations continue to evolve, the introduction of guidance on Digital Health Technologies represents a noteworthy advancement. This development paves the way for trials that are not only more efficient but also participant friendly. At Strados Labs, we are committed to being a trusted partner, helping sponsors understand and navigate the FDA’s latest recommendations.

When incorporating DHTs in clinical investigations, sponsors must provide a comprehensive description in submissions. Key components to include are:

1. DHT Suitability and Design: Detail suitability, design, technological features, data output, and measurement methods of the DHT. Describe cybersecurity and usability aspects, access control, and data integrity maintenance.
2. Usability Aspects and Data Flow: Explain the data flow to electronic repositories to ensure consistent data collection that upholds privacy, security, and data integrity throughout the clinical investigation.

Interoperability and usability evaluations are essential for DHT verification and validation. Sponsors must ensure seamless information exchange among connected systems by adopting public data exchange standards. Usability evaluations should be used to assess efficient remote use, ensuring accurate measurements and sustained participant engagement. Insights gained can contribute to enhancing design, user-friendliness, and training for trial participants and personnel.

Sponsors are encouraged to engage manufacturers, patients, caregivers, and experts in the process. Key considerations involve comparing measurements with references, evaluating influencing factors, validating calibration, ensuring consistency across DHTs, and addressing potential disparities between remote and clinic measurements.

It is essential to establish clear and reliable methods for assessing participants’ responses to medical products using DHT data. Seeking input from diverse stakeholders ensures clinical meaningfulness and sufficient data capture. Sponsors should also engage in discussions with the relevant review division regarding their endpoint development plans to align with regulatory expectations.

Defining Endpoints

When defining endpoints, specify assessment type (e.g., cough frequency and severity, lung sounds, activity level, sleep/wake time), timing, and tools used, considering the combination of multiple assessments for participants. Consulting disease-specific FDA guidance and relevant review divisions is recommended for specific endpoint considerations.

Novel Endpoints

Novel clinical endpoints often emerge through the use of sensor-based DHTs. Sponsors should justify the use of novel endpoints by assessing whether they reflect participant well-being, predict clinical benefit, relate to existing endpoints, and serve as reliable measures of disease severity. We emphasize early engagement with regulators in the development process.

DHTs may be unfamiliar to both study personnel and participants. The following considerations aim to operationalize DHTs in clinical trials, reducing risks, and enhancing the efficiency of data collection.

Clinical Risks

1. Evaluate physical features for potential discomfort and risk of injury.
2. Conduct safety testing and usability evaluations to minimize risks associated with DHT use.
3. Provide instructions for re-use and cleaning to prevent infection or adverse events.
4. Evaluate the risk of erroneous measurements impacting treatment.
5. Consider cybersecurity threats that may affect DHT functionality and participant safety.

Privacy-Related Risks

1. Be aware of unique privacy risks, including potential disclosure of personally identifiable information or participant locations.
2. Consider end-user licensing agreements that allow data sharing and modifying them to protect participant privacy.
3. Ensure appropriate security safeguards to prevent unauthorized access to data.

Informed Consent

1. Use DHTs for electronic informed consent, following FDA regulations.
2. Address privacy concerns and security measures in the informed consent process.

As clinical investigations continue to evolve, the introduction of guidance on Digital Health Technologies represents a noteworthy advancement. This development paves the way for trials that are not only more efficient but also participant friendly. At Strados Labs, we are committed to being a trusted partner, helping sponsors understand and navigate the FDA’s latest recommendations.