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Regulatory Focus — Mark McClellan: More alignment needed on RWD terminology

Regulatory Focus — Mark McClellan: More alignment needed on RWD terminology

A lack of harmonized terminologies could be impeding the global uptake of real-world data (RWD) and real-world evidence (RWE), Mark McClellan, founding director of the Duke-Margolis Center for Health Policy at Duke University, said at a 20 February meeting on the use of RWD/RWE to support oncology applications. The meeting was sponsored by the Duke-Margolis Center for Health Policy, the Friends of Cancer Research, and Aetion.

He said that regulators around the world are taking steps to address some of the challenges identified in June 2022 by the International Coalition of Medicines Regulatory Authorities (ICMRA). Key challenges identified by the group include harmonization of terminologies for RWD and RWE; convergence on best practices; addressing public health challenges and emerging health threats; and enabling greater transparency for RWD.

While progress is being made toward harmonizing terminology, more work is needed, said McClellan, who served as FDA commissioner from 2002 to 2024. Last month, the Duke-Margolis Center published a dashboard that displays the current state of RWD and RWE uptake globally. It lists the 13 countries that have RWD regulations and defines key terminologies such as RWD, data quality, data reliability, data relevance and fit for purpose. It also lists the number of guidance documents that each regulatory authority has issued on RWD.

The dashboard includes data on Health Canada, the European Medicines Agency (EMA) Singapore’s Health Sciences Authority, FDA, the Danish Medicines Agency, Australia’s Therapeutic Goods Administration, Swissmedic, the Saudi Food and Drug Authority, Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), China’s National Medical Product Agency (NMPA), the UK’s Medicines & Healthcare products Regulatory Agency (MHRA), the Korean Ministry of Health and Welfare (MOHW), and the Taiwan FDA.

The dashboard shows that the US FDA has published 12 documents on RWD and RWE, the most of any of the regulators, followed by EMA, the Taiwan FDA, the NMPA and PMDA.

The dashboard also shows nine regulators define RWD, while data quality is defined by three, reliability is defined by three, relevance is defined by four and fit for purpose defined by two.

While there is some alignment on the terminology, more harmonization is needed, McClelland said. For example, while the US FDA and the China NMPA define data reliability in similar ways, the terms data relevance and data reliability are defined differently by EMA and US FDA.

“Some harmonization is emerging for some key terms in this space, but there are some differences too,” he noted.

McClellan also cited a commentary by officials from the US FDA Oncology Center of Excellence and published in the journal Pharmacoepidemiology and Drug Safety that found a similar divergence in how these terms are defined by industry. The authors noted that “given varying operational definitions of RWD and RWE in the stakeholder community, these terms have often been used inconsistently and sometimes interchangeably. As a result, confusion can arise when similar data sources and study designs are characterized differently in different settings.”