Chapter 4 – Section 5
Leveraging data and evidence is essential in evaluating the long-term effectiveness and safety of pediatric DHTs. Use this section to learn how to bridge clinical trial insights with everyday healthcare practices to enhance regulatory approvals and inform more tailored healthcare interventions.
- Broader patient inclusion and greater trial diversity: RWD allows for diverse demographic representation, enhancing the generalizability of clinical trial results in pediatric populations.
- Natural history insights to understand disease progression: RWD provides crucial information about disease trajectories in children, which can inform study design and endpoints for clinical investigations.
- Capturing long-term outcomes and longitudinal data: RWD supports longitudinal studies, enabling researchers to track health outcomes and treatment effectiveness over time.
- Continuous improvement: RWE enables developers to refine DHTs based on insights gained from real-world usage.
- Impact: Enhanced product features lead to better user experiences and increased efficacy in real-world applications.
- Facilitating regulatory approvals: Regulatory agencies, like the FDA, are integrating RWE into their frameworks, allowing for expedited pathways to market for effective DHTs tested in clinical settings.
- Informing clinical practice: RWE provides the empirical evidence needed to shape clinical guidelines, ensuring DHTs are implemented based on robust real-world data.
Real-World Evidence of Omnipod® 5 Automated Insulin Delivery System Use in 69,902 People with Type 1 Diabetes
This study examines an automated insulin delivery system using real-world evidence (RWE) to assess the system’s effectiveness for managing type 1 diabetes (T1D) across a large cohort of 69,902 users. Through RWE, the study demonstrates that pediatric and adult users consistently achieve target glucose levels in everyday settings, confirming clinical trial results. The users maintained an average time-in-range (TIR) of 70%, which supports the system’s capacity for sustained blood glucose management outside controlled environments.
Understanding Use of Real-World Data and Real-World Evidence to Support Regulatory Decisions of Medical Product Effectiveness
The increasing role of RWE in the regulatory approval process for pediatric medical products can present both challenges and opportunities. The study highlights how RWE can supplement traditional clinical trial data, providing insights into the safety and effectiveness of products used in pediatric populations, which are often underrepresented in clinical studies. The use of RWE allows regulators to better understand the real-life impacts of these products on children’s health outcomes, thereby facilitating more informed decision-making.
Translating research to routine care: A business case
Bridging the gap between clinical research and practice is vital for ensuring that insights from clinical trials are effectively translated into everyday healthcare. Engaging stakeholders, including patients and caregivers, fosters collaboration and enhances the practical applicability of research findings. Digital measures and tools developed for clinical investigation should include a strategy for adoption into routine care settings to maximize patient and healthcare system benefits. Enabling continued use beyond trials can…
Increase your market size. Expand market appeal for developers, especially in pediatrics, where clinical trial markets are limited.
Drive patient access. Support post-market assessments, label expansions, and identification of eligible patient populations.
- Partnerships between academia, industry, and clinical settings facilitate the translation into effective healthcare practices.
- Understanding barriers to the uptake of DHTs in clinical trials aids in developing strategies that enhance integration into healthcare systems.
- Involving patients and caregivers in DHT development and trial design ensures tools address their needs, boosting participation and adherence and meaningful impacts on health.
- Collaborate with regulatory agencies to ensure compliance and accelerate the pathway from clinical investigation to routine care.
- Integrate real-world data alongside clinical trial data to validate DHT effectiveness in routine care. Real-world evidence strengthens the case for adoption by showing sustained, practical benefits, and can support regulatory submissions for broader use.
Centering patient needs delivers value to your clinical trial
This study explores the impact of patient-centric initiatives (PCIs) and evaluates return on engagement (ROE) in clinical research.
- ROE from PCIs showed improved protocol feasibility and trial efficiency.
- Cost-benefit analysis revealed higher patient satisfaction with minimal financial burden.
- Increased collaboration with patients led to better data relevance and trial retention.
