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Leveraging real-world data (RWD) and real-world evidence (RWE) to drive innovation seems like an obvious step for orthopedic manufacturers during the development of new products.
Vast amounts of relevant RWD are available from sources like electronic medical records (EMRs) and medical product registries to identify unmet clinical needs, improve the engineering of clinically effective devices and accelerate the introduction of new products to the market.
Tapping into that information to develop RWE is no easy task, however, according to Richard Smith, Interim Head of the National Evaluation System for Health Technologies (NEST). In fact, the complexity of accessing RWD was the catalyst for the formation of the organization he now runs.
Smith pointed to a complex array of circumstances that makes leveraging RWD and RWE more difficult for orthopedic implant manufacturers than it is for, say, pharmaceutical companies. The main issue is how outcomes data are collected.
“RWD and RWE have been used in the pharmaceutical arena for quite some time, but the medical device space is unique,” Smith said.
“Pharmaceutical companies benefit from having established national drug codes in every element of health records,” he added. “Identifying specific drugs that have been used and linking them to outcomes is far easier than doing the same for medical devices. Unique identifiers that must be adhered to by medical device companies are just finding their way into the orthopedic ecosystem.”
Smith pointed to the lack of a codified process for gathering the information for orthopedic companies as a shortcoming that needs to be addressed before access to the robust collection of data required to develop RWE will improve.
“Legislation requires electronic health records to include a field for recording unique device identifiers, but there is currently no standard of how that data is inputted,” he said. “The needed information can be found in unobstructed, unstructured data, making collection difficult for orthopedic manufacturers. That’s why they’re a bit behind the adoption curve.”
However, advanced new technologies will help fill those gaps and address persistent issues related to accessing RWD, Smith said.
This should come as welcome news to orthopedic device companies, who are likely to derive significant benefits from an enhanced usage of RWD and RWE. Randomized clinical trials (RCTs) are expensive and sometimes fail to reveal critical patient care and product performance information that can be shown through RWD.
“There’s hope that as orthopedic care continues to evolve, increasing amounts of RWD data will be collected,” Smith said.
Diving Into the Data
FDA credits the exponential rise in the use of computers, mobile devices, wearable technology and other biosensors to gather and store huge amounts of health-related data for the increased usage and awareness of RWD and RWE.
The agency is working with NEST, part of the Medical Device Innovation Consortium, to integrate data from clinical registries, EMRs and medical billing claims to generate and leverage RWE at each stage of the orthopedic device lifecycle.
According to Smith, FDA wants assurances that an appropriate level of attention is spent on understanding the unique designs of medical devices. He said the agency also wants to ensure that RWD and RWE are properly assessed from a Fit-for-Purpose perspective.
The randomized control trials (RCTs) that higher class orthopedic devices must undergo before gaining FDA approval are expensive to conduct and difficult to design in ways that truly assess how they are used in practice.
Those inherent limitations have increased interest among orthopedic device manufacturers in using RWE to complement or possibly replace RCTs in the product development process.
RWD and RWE are playing increasing roles in decisions that impact the lifecycles of orthopedic implants. FDA uses the information to conduct postmarket safety surveillance, track adverse events and make regulatory decisions. During postmarket reporting, RWD can generate large amounts of useful information on the performance of products after they’ve entered the market.
RWD provided by postmarket surveillance on how orthopedic products are used and how well they perform in clinical settings can lead to an expansion of approved indications, identify unmet clinical needs and inform the design of next-generation devices.
Orthopedic companies are leveraging the data to bolster clinical trial designs and observational studies designed to engineer new implants.
Product engineers use RWD and RWE in numerous ways to support clinical trial designs and develop new technologies, according to Smith. The control groups in RCTs are very rigid, but RWD can identify optimal patient populations to include in trials or be used to design studies that mimic actual patient care pathways.
“There are novel ways to develop RWE early on in the lifecycle of products to ensure there’s a better chance they will achieve the same outcome of RCTs,” Smith said.
Long-Term Benefits
NEST has explored the feasibility of using RWD sources to develop objective performance criteria (OPC) that could be used during premarket regulations of new devices, assessment of investigational device exemptions and decisions regarding label changes of existing devices.
Adoption of premarket OPC could improve timely access to innovative devices, according to Paul Voorhorst, Vice President of Worldwide Clinical Research at DePuy Synthes. He made the comments during a NEST Forum Test-Case presentation about developing OPC for hip and knee replacement outcomes.
Voorhorst said numerous high-quality national joint replacement registries hold decades of data accessible to researchers during premarket investigations. “This is an area ripe for OPCs because conducting RCTs in surgical treatments can prove difficult for numerous reasons published in the literature,” he said.
Transparency of OPC and its development and use from multiple stakeholders would aid in its adoption, according to Voorhorst. “I’m excited about having a multistakeholder panel and group working on this issue,” he said.
OPC shouldn’t be a snapshot of data, pointed out Voorhorst. “It would ideally be updated through an iterative process over time,” he added. “We need to improve patient care, and OPC is one way of doing that.”
If RWD is accessed appropriately and analyzed effectively the information will provide insights into device performance and design that could only have been known through RCTs.
“Hopefully, RWD collection will eventually be a more cost-effective option,” Smith said. “That’s why there’s an increased awareness of how RWD can generate quality RWE.”
Orthopedic companies that understand the benefits and limitations of RWD can use it effectively throughout the complete product lifecycle, according to Smith. “If it’s used correctly and FDA has confidence in the way that it’s applied,” he said, “it’s going to help get products through the application process that much quicker.”
