Experts call for culture change, standards to fulfill the promise of RWE

BOSTON – There needs to be a cultural shift to foster trust between regulators and the medtech industry when developing real-world evidence (RWE) to support the marketing of medical devices, according to experts who spoke at The MedTech Conference on Monday. They also noted that developing standards is key to expanding the use of RWE.

Heather Colvin, senior director for international harmonization at Johnson & Johnson MedTech, emphasized that for RWE to be used more broadly, there needs to be a culture change. Traditionally, evidence has been gleaned from controlled clinical studies that examine a small patient cohort and then extrapolate the data to a broader, more diverse population out of necessity, she said. However, due to advancements in digital technology within the healthcare system, researchers can now gather far more data from observational studies.

Colvin said that stakeholders need to change the paradigm and see RWE as another form of clinical evidence in the total evidentiary package that, until now, has been difficult to study. She said that real-world data is especially useful for hard-to-study populations, such as pediatric patients.

"We as a community need to change the discussion around from what is real-world evidence to start talking about what is clinical evidence writ large and how is real-world evidence a component of your total package," said Colvin. "A key part is it's really about establishing what good research looks like specific to these new data sources. […] We need to build trust by building a more standardized approach to good real-world evidence research,” she added.

Juan Espinoza, executive director at the Consortium for Technology & Innovation in Pediatrics (CTIP), said that FDA has authorized only about 250 devices that have used RWE in some form in their premarket submissions since 2016, compared with the thousands of devices the agency has authorized in the same timeframe. However, he said there is a culture shift happening at the agency and among other stakeholders toward greater trust in RWE-generated data.

Daniel Caños, director of the Office of Clinical Evidence and Analysis at the Office of Product Evaluation and Quality at the Center for Devices and Radiological Health, echoed those sentiments, noting an evolution in the sources used to obtain RWE. He said that in the past, much of it was based heavily on registries that placed a considerable burden on providers, but now researchers have access to large, aggregated datasets.

Caños discussed FDA's 2025 guidance on RWE to support medical device regulatory decision-making. He said the guidance is about ensuring the relevance and reliability of RWE information used for regulatory decisions and noted that the principles in the guidance are also well established in the International Organization for Standardization's good clinical practice standard, ISO 14155: Clinical investigation of medical devices.

"We've been working within our center as we had the rollout of the guidance to talk about these principles," said Caños. "We provide support in the review."

"The guidance comes at an important time for us as we think about the landscape of real-world evidence and our work with the [Medical Device Innovation Consortium (MDIC) National Evaluation System for health Technology (NEST)] specifically," he added.

Simon Mason, chief enterprise officer at MDIC’s NEST, also said that there needs to be a change in the culture, and that his organization has been at the forefront of bringing stakeholders together to discuss what that culture should look like and develop standards to support it.

"There's an interesting elephant in the room when it comes to RWE: culture," said Mason. "I think it is probably the biggest issue here."

Mason said that over the past five years, NEST has been working to develop consistent standards that are acceptable to regulators and the industry. He said that from the beginning FDA has had concerns about how RWE information is presented, while on the industry side, companies are reluctant to invest in collecting RWE information because of the challenges they've faced from reviewers.

Mason noted that while there are thousands of companies interested in using RWE, there is only one FDA, and it is important that they can all agree on the right standard when submitting RWE information. He said that also entails training FDA staff in the standards so they can apply them consistently. He also cautioned industry that RWE evidence is about creating more certainty about their products and isn’t meant to be a shortcut.

"I think a lot of the industry saw this as a way to save time, which it is, it's a way to save money, which it is, but it's not a way to cut corners," said Mason.

Colvin noted that European regulators and industry stakeholders across the pond have been working to develop the Integration of Heterogeneous Data and Evidence towards Regulatory and HTA Acceptance (IDHERA) public-private partnership to help integrate and analyze RWE health data for clinical care, research, regulatory approval, and Health Technology Assessment (HTA). She said they used feedback from the FDA, NEST, and other stakeholders to develop a roadmap for presenting RWE in a more standardized way, intended to present good data practices, data integrity, and research transparency. She also said an updated version of the roadmap is expected to be released in March 2027.

"We mapped out a very simple process that's going to look very similar to good clinical practice intentionally, but it's unique to real-world data sources, and it really focuses on trust and transparency," said Colvin. "What do you need to do and document in your research process, but then also try to delve into the data integrity and governance issues, because regulators are concerned, and they are very justified in their concern that people are going to monkey with the data."

While NEST is funded through medical device user fees and is US-focused, Mason said that collaborating with international partners and regulators is important to achieve harmonization. He said that all organizations developing standards and roadmaps for using RWE need to work together.

"Our standards that we create in the US are presumably reproducible in other parts of the world, and so we encourage them to take our standards," said Mason. "There is nothing unique about what we do here, and that is something that as each of these organizations get together, and I think everyone in the room is involved in these organizations.