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As data is captured through electronic health records, registries and unique device identifiers, the generation of evidence based on this data is expected to play a larger role in informing orthopaedic manufacturers’ decisions before and after regulatory approval. While regulators, payors, hospitals and manufacturers back this approach, they acknowledge that gaps remain in capturing the data and using it to build evidence that satisfies regulatory requirements.
Priority considerations include how to generate evidence to support regulatory market decisions, device indication expansion, postmarket studies, postmarket surveillance and reimbursement decisions.
The National Evaluation System for health Technology Coordinating Center (NESTcc), an initiative of the Medical Device Innovation Consortium (MDIC), is one entity working to support the sustainable generation of Real-World Evidence (RWE) using Real-World Data (RWD).
FDA awarded MDIC an initial grant to develop NESTcc in 2016, and the agency continues to provide funding through MDUFA and participate in its governance. NESTcc has garnered support from industry associations like AdvaMed and the Medical Device Manufacturers Association, and 12 Data Network Collaborators including DukeHealth, HealthCore, Mayo Clinic and Mercy. Further, orthopaedic manufacturers are among NESTcc’s early partners.
In November 2018, NESTcc announced its first round of RWE Test-Cases to explore the feasibility of industry using RWD. These feasibility studies are the first funded projects to leverage the NESTcc Data Network. Multiple test-cases are focused on postmarket surveillance in orthopaedics.
- Two test-cases will use electronic health record data to assess the feasibility of using RWD to conduct proactive postmarket surveillance. Lumbar interbody systems and pediatric craniomaxillofacial distractors are the technologies of interest. The European Union’s Medical Device Regulation will require proactive surveillance to maintain a CE Mark. These test-cases will potentially develop methodology and approaches for proactive surveillance that can be adopted by manufacturers.
- One test-case will examine the feasibility of registry and claims data linkages, taking information from the American Joint Replacement Registry (AJRR), Mayo Clinic and HealthCore to evaluate the clinical outcomes of total knee arthroplasty implant survivorship, mortality, revisions, readmissions and more.
- Additionally, one test-case will bring together DJO Global, DePuy Synthes, Smith & Nephew, Stryker, Zimmer Biomet and AJRR, with the American Academy of Orthopaedic Surgeons serving as the neutral convener.
The robust list of stakeholders working with NESTcc demonstrates that regulators, payors, industry and hospitals support a role for RWD and RWE. We asked Rachel R. Rath, Deputy Director for the NESTcc, what is important for orthopaedic device manufacturers to know about RWD and RWE today.
How does NESTcc define RWE and RWD?
Rath: The FDA’s definition of RWE is clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD. And RWD are the data relating to patient health status and/or the delivery of healthcare routinely collected from a variety of sources, including, in the case of NESTcc’s Data Network, electronic health records, pharmacy data, public and private claims, registries, patient-generated data and unique device identifiers.
I expect most people would say that orthopaedics is in the infancy stage of meaningfully using this data. What is important to know about RWE and RWD in the context of healthcare today? What will be the role of RWE and RWD five years from now?
Rath: We are in the process of working with medical device ecosystem stakeholders to identify the most critical areas and the best approaches for using RWE to answer important questions to various stakeholders including manufacturers, patient groups, health systems, payors and regulators.
One mechanism we have in place for understanding the capabilities of the RWD partners that form the NESTcc Data Network are the test-cases. The first test-case projects have been approved and the second round is currently in proposal development. We will be announcing the second round of approved projects this spring.
The Round 1 Test-Cases provide a learning opportunity as they help test the ability of the Data Network to answer questions for devices at various stages of the Total Product Life Cycle and multiple regulatory pathways (510(k) and PMA) and to address changes within the regulatory environment, including the new EU MDR. In particular, two test-cases look at the ability to use the RWD available through the NESTcc Data Network to develop methodologies and approaches to support the new EU MDR requirements for “proactive surveillance.”
In addition to the broad test-cases, we have targeted activities underway to address specific areas of high-priority. One area is understanding the role of patient-generated health data, for which we had a targeted call for test-cases along with Round 2.
NESTcc is also in the process of launching an Active Surveillance Task Force to develop a blueprint for NESTcc’s advancement of active surveillance activities, to help address the timely and robust identification of safety signals for medical devices.
RWD can be collected through a variety of sources, offering new opportunities to generate new types of information, including the patient experience. RWD opens the possibilities to understand data from the course of practice. This can provide insights on ways to use medical products and provide valuable information for clinicians, patients, regulators and manufacturers.
In the next few years, we hope that RWE will enable researchers to better understand the real‐world patient experience and will support decisions from the healthcare community for improved clinical practice and overall outcomes.
Within five years, we hope that NESTcc is operational and sustainable, bringing together robust sources of high-quality RWD with the ability to answer questions of high importance to the medical device ecosystem.
What best practices can you offer industry?
Rath: Over the next eight to 12 months, the Round 1 Test-Cases will complete preliminary and final deliverables that will include lessons learned and best practices for using RWE to answer the aforementioned important questions. Insights generated through the test-cases over the next year will be disseminated to advance the knowledge within the ecosystem. While the test-cases answer specific questions of interest to specific industry partners, the learnings from these test-cases will be broadly applicable, exploring the research process and methodologies, and administration of research conducted across multiple Network Collaborators within the NESTcc Data Network.
In addition to the learnings that will be generated from the test-cases, lessons will be generated and shared through the development and implementation of the first version of the Data Quality Framework.
