The value of evidence generation after receiving 510(k) regulatory clearance from the FDA

While 510(k) clearance is a key step towards market access, very little, if any, data is required to achieve this milestone. Devices granted this clearance only require substantial equivalence evidence, which often only features mechanical testing focusing on device design, durability and wear behavior. Because of this, many aspects of product clinical and safety, as well as cost-effectiveness, remain unknown following the U.S. Food and Drug Administration’s (FDA) 510(k) clearance.

Therefore, medical technology (medtech) companies must prioritize the generation of evidence to demonstrate product value. Real-world data (RWD), such as administrative claims databases, electronic medical records and patient-reported outcomes, have become an increasingly available and sought-after tool to generate real-world evidence (RWE) related to patient health outcomes and resource utilization.

Accepting RWE to support regulatory decisions

Following the FDA’s decision to accept RWE in lieu of randomized controlled trials (RCTs) to support regulatory decisions and monitor post-market safety and adverse events, the acceptance of RWE by industry leaders has continued to grow. RWE is leveraged to demonstrate product value through concrete evidence-based results, as well as to drive innovation and support competitive intelligence insights and pricing initiatives, among other market access activities. Medtech companies can best maximize the value of RWE through the development of comprehensive evidence generation strategies.

Evidence generation plans should be informed by existing research, competitors in the market, RWD dataset characteristics and unmet needs in the market.

Selecting RWD

One of the most impactful decisions in this process is the selection of RWD. There are numerous options for RWD, ranging from administrative claims datasets to patient surveys to social media, each offering their own unique clinical, safety, economic and patient-sourced insights. However, there may not be enough substantial RWE to gather at the time of 510(k) approval, even if the data has already been obtained. Therefore, it is important to acknowledge that it may take 12 months or more to accumulate and synthesize the first wave of RWE.

Developing a registry at this stage can be a powerful step towards accumulating high-value RWD that can support ongoing evidence generation. These data can be utilized in retrospective outcomes studies to generate real-world evidence that describes the value of the product and outcomes experienced by patients.

Given the numerous RWD options available, it is important to have a strategy in place to identify specific medical technologies. Having a dedicated code is the ideal option, however, this isn’t always the norm at this lifecycle stage. Leveraging indications for use, multiple procedure code logic and/or specific facility/physician information will provide more accurate identification of medical technologies. Having a strategy in place to identify products within these numerous RWD options will allow for proper outcomes analyses.

RWD should be strategically selected based on the relevance and breadth of the data in relation to the study hypotheses, as well as the study design being utilized. Additionally, outcome measures should be thoughtfully selected to address both the existing research questions; outcome measures should also highlight the product’s value versus other competing products.

Study outcomes also should be relevant and meaningful to various key stakeholders, including payer, providers, physicians and societies, who will make adoption and coverage decisions based on the presented findings. Thoughtful selection of outcomes ensures the study addresses both the needs of key stakeholders, as well as the shortcomings of previous studies that have influenced product adoption.

 [GDA1]Somehow we need to acknowledge that retro RWE may not be robust at the time of 510k approval so that it may take 12 months or more to accumulate the first wave of RW

Choosing the appropriate methodology

Lastly, the analytic approach should be carefully selected such that the methodology that is used best answers the research questions. There’s a strong need for understanding when and how certain clinical analysis are appropriate, such as safety analyses versus burden of illness analyses. There is also a need for understanding when certain economic analyses are appropriate, such as cost-effectiveness analyses versus budget impact analyses. With these insights, medtech companies can develop a comprehensive study protocol and analytic plan that not only generates new RWE to support the clinical utility value of their product, but also ultimately ensures the continued success of market access efforts. The newly generated RWE muse also demonstrate that the new technology changes patient care pathways and outcomes. Once executed, the generated evidence can have considerable impact on market access activities.

It is important to note that 510(k)-approved devices do not require data-demonstrating outcomes associated with utilization. Despite having received 510(k) approval, organizations often do not have sufficient evidence to validate their claims of similarity to existing products on the market, nor can they validate hypotheses of improved outcomes compared to existing products.

Bringing it all together

Following the generation of positive RWE, findings can be used to further market access activities in several ways. RWE findings are often used to develop and publish manuscripts for scientific journals to bolster the volume and quality of publications for a given product and/or therapeutic area. Publications not only increase industry awareness of a product’s value and evidence-backed findings, but they also demonstrate to payers that a breadth of outcomes associated with product utilization have been adequately explored and validated through the rigorous peer-review process.

RWE findings are used to support payer-facing dossiers and marketing collateral documents to demonstrate the product’s economic and clinical value. Supported by RWE findings, these documents are critical to communicating the need for and ultimately assisting in the securing of payer coverage for the medical device. RWD can also be leveraged to proactively assess market trends and competitor products and to mitigate potential impacts on market positioning.

Baker Tilly’s market access services team can help medtech companies achieve their market access needs, answering questions, providing guidance and walking organizations through the next steps in the wake of receiving 510(k) regulatory clearance. For more information or to begin a conversation, contact our team.