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Key objectives of the initiative include:
Developing a step-by-step framework (PE Digital Roadmap) for implementing meaningful patient engagement in digital health
Clarifying the role of patients in designing and developing digital health solutions
Addressing challenges in digital health stakeholder alignment through the Stakeholder Expectations Matrix
Promoting transparency in patient involvement processes for digital health solutions

Findings
Patient experience data provides critical context for regulatory review by illuminating disease burden, unmet medical needs, and the aspects of a condition that matter most to patients.
A systematic approach is necessary to ensure patient experience data is robust enough for regulatory consideration, moving beyond anecdotal evidence to scientifically rigorous data collection.
Early engagement between sponsors and the FDA is a key factor for successfully incorporating patient perspectives into a drug development program.
The value of patient-reported outcomes (PROs) and other clinical outcome assessments (COAs) is highly context-dependent, varying significantly across different diseases and patient populations.

Recommendations
Drug sponsors should leverage the FDA's meeting process to discuss their strategies for collecting and submitting patient experience data early in the development lifecycle.
Sponsors should utilize the repository of meeting reports as a learning resource to understand best practices and common challenges in patient-focused drug development for specific conditions.
Patient advocacy groups should actively participate in these discussions to ensure the full spectrum of patient experiences is captured and communicated to both regulators and developers.
Researchers should develop and validate novel tools and methodologies for capturing and analyzing patient experience data that are meaningful for both clinical and regulatory purposes.

Regulatory Considerations
Patient experience data is a key component of the benefit-risk assessment, providing evidence that can inform regulatory decisions regarding a drug's approval and labeling.
The FDA's review of patient experience data is guided by a commitment to patient-focused drug development, as mandated by the 21st Century Cures Act and supported by user fee agreements like PDUFA.
The scientific rigor of data collection and analysis is paramount; for patient experience data to be influential, it must meet high standards of validity and reliability.
Transparency is a core principle, and the publication of these meeting reports is intended to provide clear examples of how patient input can be effectively integrated into regulatory submissions.

Findings
Traditional, site-based clinical trials often create significant burdens for participants, which can hinder recruitment, retention, and the enrollment of diverse populations.
A lack of early and sustained patient engagement in trial design can lead to research protocols that are misaligned with patient needs and endpoints that are not meaningful to them.
The underrepresentation of diverse racial, ethnic, and other demographic groups in clinical trials limits the generalizability of study results and can perpetuate health disparities.
Emerging digital health technologies (DHTs) and real-world data (RWD) present significant opportunities to make clinical trials more efficient, patient-centric, and inclusive, but their adoption has been inconsistent.

Recommendations
Sponsors and research teams should engage patients and patient advocacy groups as active partners throughout the entire clinical trial lifecycle, from design to dissemination.
Decentralized clinical trial (DCT) elements should be incorporated to reduce patient burden, improve access for diverse populations, and enhance the quality of data collection.
Trial sponsors must develop and implement proactive strategies to enhance the diversity and inclusion of trial participants to ensure results are applicable to all patient populations.
Novel endpoints derived from DHTs should be developed and validated to capture more objective, real-world measures of how patients feel, function, and survive.
Multi-stakeholder collaboration between industry, academia, patient groups, and regulators is essential to address systemic challenges and improve the clinical trial enterprise.

Regulatory Considerations
Early and frequent communication with regulators, such as the FDA, is critical when implementing novel approaches like DCTs or developing new digital endpoints for pivotal trials.
Regulatory frameworks must support the use of innovative technologies and trial models while ensuring data integrity, reliability, and patient safety.
The use of a single Institutional Review Board (IRB) for multi-site trials is a key regulatory-supported mechanism for streamlining ethics review and increasing trial efficiency.
When using DHTs and decentralized methods, robust plans for data quality, privacy, and security are necessary to meet regulatory standards for trial data submission.

Findings
A comprehensive catalogue of over 340 symptoms and impacts was identified across ten symptom domains and two functional impact domains. Strongest evidence for relevance in early disease was found for tremor, fine motor dexterity, gait, stiffness, and slowed movements. Common non-motor symptoms include cognitive alterations, mood changes such as anxiety or depression, sleep disturbances, fatigue, and urinary dysfunction. Significant variability exists in how these concepts are currently measured and classified in literature, often confounding symptoms with functional impacts. There is a notable lack of diversity in existing research, with over 93% of qualitative data originating from white populations in the US, UK, and Canada.

Recommendations
Researchers and clinicians should utilize the proposed Domain-Category-Concept-Experience schema to ensure consistency and parsimoniousness in outcome selection. Selection of concepts for clinical trials should be evidence-based, focusing on those demonstrated to be both prevalent and bothersome to patients. Future research must prioritize the inclusion of culturally, racially, and geographically diverse populations to ensure the model's universal applicability. Stakeholders should adopt lay-friendly terminology, such as using ""slow movements"" instead of ""bradykinesia,"" to better reflect the patient perspective. Continuous re-evaluation of the model is necessary to maintain alignment with emerging biological staging systems for neuronal synuclein disease.

Regulatory Considerations
The consensus model was developed to align specifically with FDA guidance on patient-focused drug development (PFDD) to support regulatory-ready endpoints. Meaningful aspects of health should be identified through direct patient report to satisfy evidentiary requirements for ""fit-for-purpose"" clinical outcome assessments. Evidence-based SOFT report cards provide a transparent method for justifying the selection of concepts of interest in regulatory submissions. Early engagement with agencies is encouraged to ensure selected endpoints are sensitive to treatment effects and reflect what matters most to patients. Harmonization of concept definitions is a critical prerequisite for the successful qualification of new drug development tools.

Findings
While verification, analytical validation, and clinical validation have been well-established, usability validation has not been systematically incorporated into digital health technology evaluation.
Variability in device designs, patient populations, and regulatory environments creates barriers to widespread adoption of sensor-based digital health technologies.
Usability problems, such as poor user interfaces and technical errors, can lead to significant data loss in clinical trials and real-world applications.
While some guidance exists for usability in medical devices, there is no unified global standard for assessing usability in digital health products, leading to inconsistencies in implementation.
Stakeholders, including regulators, industry leaders, and researchers, recognize the need for usability validation to ensure the real-world effectiveness of digital health technologies.

Recommendations
Adopt the V3+ framework as a standardized method to ensure that usability is rigorously tested alongside verification, analytical validation, and clinical validation.
Establish clear protocols for usability testing, including use specification development, risk analysis, iterative formative evaluations, and summative evaluations.
Bring together regulators, technology developers, clinicians, and patients to create guidelines that ensure fit-for-purpose digital health solutions.
Work with regulatory agencies such as FDA, EMA, and MHRA to establish harmonized global standards for usability validation.
Encourage the publication of usability study results, including negative findings, to facilitate transparency and continuous improvement in digital health technologies.

Regulatory Considerations
Agencies like FDA and EMA increasingly require usability data for digital health technologies, but standardized methodologies are still evolving.
Usability validation should align with regulatory requirements for medical devices and digital biomarkers, ensuring clinical relevance and data integrity.
Digital health technologies must adhere to HIPAA, GDPR, and other data protection regulations while ensuring seamless usability.
Poor usability can lead to missing or unreliable data, which affects regulatory submissions and real-world evidence generation.
A consistent approach to usability evaluation is needed to support regulatory decision-making and digital health product approvals globally.

Findings
Existing guidelines lack pragmatic application and systematic approach for device selection.
Device choice is dependent on measurement objectives, user population, and available resources.
Current frameworks do not systematically consider verification, validation, feasibility, and protocol design.
Rapid obsolescence of digital devices due to technological advancements.
Need to incorporate social/psychological factors into device selection.

Recommendations
Develop a practical guide with a systematic approach for selecting wearable devices.
Use five core criteria: continuous monitoring capability, device suitability and availability, technical performance, feasibility of use, and cost evaluation.
Prioritize feasibility of use to ensure user needs are incorporated into the selection process.
Adapt guide criteria to accommodate novel innovations.
Foster clarity and transparency in decision-making among researchers, HCPs, and device users.

Regulatory Considerations
Follow FDA guidance for digital health technology usage in clinical investigations.
Consider CTTI recommendations for improving clinical trial quality and efficiency.
Use ePRO Consortium's factors for device suitability in regulatory trials.
Apply international guidelines for specific measurements when available.

Findings
Sites must establish effective data privacy and security plans, especially considering regional and global regulations like GDPR.
Risk mitigation is critical, including plans to address unanticipated issues and potential patient disengagement due to technology challenges.
Budgeting and contracting often involve additional considerations, such as storage, training, and technical support requirements for connected devices.
Sites require adequate training to ensure staff and patients are prepared to use connected devices efficiently.
Companion applications or services often play an essential role in device functionality and data transmission.

Recommendations
Develop a clear plan for data pathways, including storage, security, and regulatory compliance.
Establish detailed risk mitigation and management strategies to handle unexpected challenges.
Ensure comprehensive training programs for site staff and patients to enhance device usability.
Incorporate device storage and resource allocation into budgeting and contracting processes.
Facilitate effective communication with sponsors and vendors to resolve operational and technical issues promptly.

Regulatory Considerations
Ensure connected devices comply with CFR 21, Part 11, and other relevant data collection and transmission regulations.
Understand and adhere to local and regional data privacy laws, such as GDPR, when managing patient data.
Verify that appropriate licenses and regulatory approvals are in place for device data transmission and storage.
Assess and address shipping and handling regulations for devices, ensuring safe and compliant transportation.

Findings
Challenges in patient adherence and acceptability of digital endpoints.
Issues with algorithm validation and use in diverse populations.
Barriers due to proprietary software and lack of transparency.
Vast heterogeneity in digital endpoints and lack of standards.
Need for ongoing ethical support and consideration of environmental impact.

Recommendations
Foster multi-stakeholder cooperation and open-forum discussions.
Integrate patient needs into the design of digital health technologies.
Include implementation science expertise in research proposals.
Develop standards for selecting and reporting digital endpoints.
Provide ongoing ethical support throughout the research lifecycle.

Regulatory Considerations
Early engagement with regulators is crucial.
Understanding regulatory requirements for clinical trials versus clinical care.
Need for harmonised terminology and guidelines for digital endpoints.

Findings
Product development firms are hesitant to increase DHT use despite regulatory support.
Conventional hardware-based technologies are preferred over newer digital tools.
Operational barriers contribute to the low adoption of DHTs in product development trials.

Recommendations
Reduce operational barriers to facilitate DHT adoption.
Provide additional regulatory clarity to encourage DHT use.
Encourage the incorporation of more DHTs and patient-centric endpoints in clinical trials.

Regulatory Considerations
The FDA's guidance on DHT use is evolving and not yet fully formalized.
There is a need for harmonization between US and non-US regulatory agencies.
The impact of recent regulatory support may take years to be fully realized.

Findings
No uniform definition exists for nocturnal scratching, leading to inconsistencies in data interpretation and measurement across studies.
There are significant differences in how scratching behaviors are defined, recorded, and analyzed, making cross-study comparisons difficult.
The term “nocturnal” is often used imprecisely, as sleep periods vary among individuals (e.g., shift workers, patients with disrupted sleep patterns).
Traditional methods such as videography and clinician observations are expensive, labor-intensive, and impractical for widespread use.
Advances in sensor-based wearables and machine learning present opportunities to create objective, scalable, and patient-centric digital measurement tools.

Recommendations
Define nocturnal scratching as a rhythmic and repetitive skin-contact movement occurring within a delimited sleep period, rather than restricting it to nighttime.
Implement standardized ontologies to guide measurement definitions, ensuring consistency across studies and clinical applications.
Encourage the creation and validation of wearables and machine learning algorithms for objective, scalable measurement of scratching.
Engage researchers, clinicians, patients, and regulatory bodies to drive consensus on measurement definitions and methodologies.
Establish digital measures as key endpoints in clinical trials, supporting their validation and regulatory acceptance.

Findings
Standard assessments lack sensitivity in early stages of neurodegenerative diseases.
Challenges with the validity and quality of RMT measurements.
Issues related to equity and inclusion in deploying digital tools.
Importance of considering feasibility, acceptance, usability, and ecological validity of digital endpoints.

Recommendations
Develop regulatory strategies early on.
Ensure equity and inclusion in deploying digital tools.
Address challenges related to the validity and usability of digital endpoints.
Promote public-private partnerships to address privacy and security concerns.
Involve patients and stakeholders in the design and implementation of digital tools.

Regulatory Considerations
Acceptance of digital endpoints by regulatory authorities is crucial.
Validation with current gold standards and clinically meaningful legacy endpoints.
Ensure data security and privacy.