Digital health technology-derived clinical outcome assessments: considerations for regulatory decision-making Elektra Papadopoulos, MD, MPH Acting Deputy Director Division of Clinical Outcome Assessment Office of New Drugs, CDER, FDA
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Presentation Transcript
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Digital health technology-derived clinical outcome assessments: considerations for regulatory decision-making Elektra Papadopoulos, MD, MPH
Acting Deputy Director
Division of Clinical Outcome Assessment
Office of New Drugs, CDER, FDA<br>
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Disclaimer Views expressed in this presentation are those of the speaker and do not necessarily represent an official FDA position.<br>
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What is a digital health technology (DHT)? A system that uses computing platforms, connectivity, software, and/or sensors for healthcare and related uses. These technologies span a wide range of uses, from applications in general wellness to applications as a medical device. They include technologies intended for use as a medical product, in a medical product, or as an adjunct to other medical products (devices, drugs, and biologics). They may also be used to develop or study medical products. SOURCE: BEST (Biomarkers, EndpointS and Other Tools): https://www.ncbi.nlm.nih.gov/books/NBK326791/<br>
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We are focusing on digitally collected clinical outcome assessments (COAs): Clinical outcome: An outcome that describes or reflects how an individual feels, functions or survives. Measured using COAs. Digital health technologies can be used in assessments of: Biomarker: A defined characteristic that is measured as an indicator of normal biological processes, pathogenic processes, or biological responses to an exposure or intervention, including therapeutic interventions. SOURCE: BEST (Biomarkers, EndpointS and Other Tools): https://www.ncbi.nlm.nih.gov/books/NBK326791/<br>
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An example from PRO Consortium’s Chronic Heart Failure Working Group 12th Annual PRO Consortium Workshop (April 14-15, 2021)<br>
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Potential benefits of digital health technology-derived COAs By enabling remote data capture
Endpoints that matter to patients in daily life (e.g., patients’ experiences between clinic visits)
Reduced participation burden/fewer barriers to clinical trial participation (e.g., travel)
More inclusive and generalizable trials<br>
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What does it mean for a DHT-derived COA endpoint to be fit-for-purpose*? Overall considerations same as with any COA:
Appropriate for its intended use e.g.,
Study design
Patient population
Validly and reliably measure a concept that is
Clinically relevant
Important to patients
Able to show change within the clinical trial
Can be communicated in labeling in a way that is accurate, interpretable, and not misleading (i.e., well-defined), if appropriately applied *Fit-for-purpose: A conclusion that the level of validation of a biomarker or COA is sufficient to support its proposed use.<br>
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Roadmap to patient-focused outcome measurement still applies<br>
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Limited experience in aggregating and summarizing continuously collected DHT data into a clinically meaningful endpoint
Methodological issues include (but are not limited to):
Defining activity parameters
Determining appropriate assessment periods in a day
Assessing minimal “wear time” requirements during a day
Collecting and aggregating potentially large volumes of data to derive a score
Defining the endpoint
Interpreting the data, including meaningful within-patient change
Handling missing data We’re still in the early stages<br>
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Think with the end in mind:
What is the overall COA measurement strategy? What role does the DHT-derived endpoint play and how will it complement other measures/endpoints?
What parameters are most meaningful to assess?
How will the DHT derived endpoint be defined?
How will the benefit be described to stakeholders?
Leverage recommendations and resources available
Engage with FDA early and often In closing,<br>
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Patient-Focused Drug Development Guidance Series:
https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical
Drug Development Tool Qualification process guidance:
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/qualification-process-drug-development-tools-guidance-industry-and-fda-staff
FDA COA Qualification Program Website:
https://www.fda.gov/drugs/drug-development-tool-ddt-qualification-programs/clinical-outcome-assessment-coa-qualification-program
FDA guidance on regulatory requirements for record keeping, maintenance, and access (21 CFR Part 11)
https://www.fda.gov/media/75414/download Helpful resources<br>
