Expediting regulatory guidance for RWE in medical devices: Why it matters for patients
Real-world evidence (RWE) can enhance our understanding of the safety, performance, and effectiveness of medical technologies, and health authorities are increasingly recognizing its benefits in informing regulatory decisions across the product lifecycle. Registries and electronic health record (EHR) data are used as primary sources of clinical evidence to support labeling indications and indication expansions in the pre-marketing setting.1 Real-world data (RWD) also are addressing important questions related to the post-marketing safety of medical devices.2,3Emerging RWE-focused regulatory policies and guidance are supporting this momentum, helping to codify best practices and provide stakeholders with guidance on expectations for "regulatory-grade" RWE generation. To maximize the value of innovative medical technologies for patients, it is critical to identify current barriers and solutions that will facilitate the adoption of regulatory guidance for RWE generation for medical devices and support broader harmonization efforts globally.
Trust in RWE remains a barrier
IDERHA (Integration of Heterogeneous Data and Evidence towards Regulatory and HTA Acceptance), a public-private partnership funded by EU’s Innovative Health Initiative (IHI), published a landscape analysis in June 2024, which found that trust in RWE continues to be a barrier to the acceptance of RWE for regulatory decisions for medical technologies.4,5As part of their goal to advance the use of RWE to support healthcare decision-making, IDERHA analyzed 50 RWD/RWE policies for medicines and medical devices published between 2017 and January 2024 from regulatory and health technology assessment (HTA) agencies around the world. The findings showed a shift towards integrating RWE into the clinical evidence portfolio, yet also demonstrated important opportunities to advance RWE acceptance through greater demonstration of its robustness. For example, concerns continue to exist around the need for more standardized RWE-specific research practices, how to assess individual study designs and determine whether RWD is “fit-for-purpose,” and whether analytical approaches adequately address information bias, confounding, and generalizability of findings.5
Building from experience
Building trust and providing tangible examples that enable health authorities to verify the reliability of RWE require diverse inputs and pragmatic mechanisms to integrate feedback into practice. Multi-stakeholder public-private partnerships have shown to be an effective mechanism for co-developing solutions that can help inform best practices, build trust, and ultimately deliver value to patients.An example of how public-private partnerships can serve as a test bed for new approaches and solutions is the July 2023 US Food and Drug Administration (FDA) approval of a label expansion for a Johnson & Johnson medical device (cardiac ablation catheter) based solely on a comparative RWE study using EHR databases.6 The study was one of 21 test cases funded by the FDA through the National Evaluation System for health Technologies Coordinating Center (NESTcc), an initiative of the Medical Device Innovation Consortium (MDIC). It was conducted in collaboration with Mercy Health, Mayo Clinic, and Yale New Haven Health.
Throughout the process of designing, implementing, and interpreting the results of the RWE study, Johnson & Johnson and its collaborators worked closely with the US FDA to verify the appropriateness of the study design along with the relevance and reliability of the RWD to address the agency’s questions about the accuracy, validity, and generalizability of the study results.7 Insights from this NESTcc test case later supported a second label expansion for several other catheter-related products and have informed the development of a NEST data quality framework to help standardize RWE generation for medical devices. Such tools enable health authorities to better assess and understand the data’s fitness for purpose. By demonstrating the feasibility of using RWE to assess the safety and effectiveness of devices, the NESTcc test cases underscore the value of PPPs in advancing the use of RWE in regulatory decisions.
In Europe, RWE can play an important role in meeting EU-MDR (European Union Medical Device Regulation) Post-Market Clinical Follow-Up (PMCF) requirements introduced in 2021. Yet, there is currently no regulatory framework for using RWD and/or RWE under the EU-MDR. Public-private partnerships such as IDERHA are helping to bring key challenges like this to the table and formulate collaborative solutions between device makers, regulators, notified bodies, and health technology assessment agencies. For example, using learnings from the previously mentioned policy landscape assessment and building on best practices from NEST and other similar initiatives, IDERHA is working to develop policy recommendations to advance the development of regulatory frameworks for RWE for medical devices in the EU.5
In China, the National Medical Products Administration (NMPA) and the Hainan Medical Product Administration (HNMPA) launched an RWE pilot deployment program at the BoAo Lecheng International Medical Tourism Zone in 2019. Its goal was to encourage industries to utilize the hospital-based RWE collected in the Boao Lecheng Pilot Zone to support product registration in China, helping to reduce the requirement of conducting a clinical trial in the country when the product is already licensed abroad.8 Johnson & Johnson was one of the participating companies in the RWE pilot. In combination with extensive global data and the BoAo RWE study, an innovation in precision laser cataract surgery obtained product registration in China in January 2021.
While we acknowledge there are several complexities involved with creating and harmonizing standards for RWE guidance in medical technologies given differences from product to product and study to study, it is important that public-private partnerships continue to identify best practices and inform the development of tools like the NEST data quality framework to enable health authorities to not only trust, but also independently verify the quality of the RWE in their assessments. These resources will be critical as stakeholders explore emerging areas for RWE application in medical technologies, such as active safety surveillance.
Conclusion
Clear, consistent regulatory guidance on RWE can have a significant impact on patient care. It facilitates systematic and transparent data collection and analysis, providing a clearer picture of a medical devices’ performance in real-world settings. This can help manufacturers identify potential safety issues more quickly and validate effectiveness across diverse patient populations, such as medically underserved patients. It also can help accelerate the approval process for new devices, which enables faster access to innovations for patients. The availability of robust RWE benefits healthcare providers as well by informing treatment decisions that lead to improved medical outcomes for their patients. Additionally, regulatory guidance on RWE builds trust in the safety and effectiveness of medical devices by promoting data transparency, while supporting the adoption of cost-effective medical technologies for more efficient healthcare delivery.As new regulatory policies are being developed, key stakeholders must be engaged to ensure that the needs of end-users (e.g., patients and clinicians), as well as researchers, regulators, and industry, are considered. As demonstrated in the examples shared, collaboration is essential to expedite the adoption of regulatory guidance, promote the use of RWE in regulatory decision-making for medical technologies, facilitate harmonization efforts, and ultimately maximize RWE's potential to improve patient outcomes.
Josh Gagne is Vice President and Head of Global Epidemiology, Office of the Chief Medical Officer, Johnson & Johnson; Paul Coplan is Vice President and Head of MedTech Epidemiology & Real-World Data Sciences, Office of the Chief Medical Officer, Johnson & Johnson; Rita Peeters is Senior Director, EMEA Regulatory Policy, Johnson & Johnson MedTech; Heather Colvin is Director, Regulatory Affairs, Evidence and Outcomes, Johnson & Johnson MedTech. Shumin Zhang, Senior Director, Regulatory Real-World Evidence, MedTech Epidemiology & Real-World Data Sciences, Office of the Chief Medical Officer, contributed to this article.
References
All reference verified 26 August 2024
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