Catalyzing International Harmonization of Real World Evidence Standards

Background

International harmonization of real-world data and evidence (RWD/E) standards is a goal among real-world data/real-world evidence (RWD/E) policy stakeholders. To support this goal, the Duke-Robert J. Margolis Institute for Health Policy (“Duke-Margolis”) developed the online International Harmonization of RWE Standards Dashboard (“Dashboard”) to provide timely updates.

Methods

We sought guidance for industry (draft and final) and related literature available online and published in English language by medical product regulators globally. Consultations were held with Duke-Margolis RWE Collaborative Members to identify, collate, and interpret documents. We developed an online Tableau tool summarizing the number of guidance documents and definitions of fit-for-purpose RWD/E, quality, reliability, and relevance published to date.

Results

As of June 2024, the United States Food and Drug Administration (FDA) has released eleven guidance documents (n= 11), followed by the European Medicine Agency (EMA) and the Pharmaceuticals and Medical Devices Agency (PMDA – Japan) which have released seven (n = 7) guidance documents. Taiwan FDA and the National Medical Products Administration (NMPA)/China Center for Drug Evaluation, and have released six (n= 6) documents. The Korea Ministry of Food and Drug Safety (MFDS) has released four documents (n=4) and Health Canada/Canada’s Drug and Health Technology Agency (CADTH) has released three (n= 3) documents. The Saudi Food and Drug Administration (SFDA) the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) and Australia Therapeutic Goods Administration have each released two (n= 2) documents. Lastly, the Swiss Medic, Danish Medicines Authority, the Brazilian Health Regulatory Agency (ANVISA) and the Singapore Health Sciences Authority, have each released one (n= 1) document.

To date, ‘RWD/E’ has been defined by twelve (n= 12) regulators (ANVISA, EMA, FDA, CADTH, MFDS, MHRA, NMPA/China Center for Drug Evaluation, PMDA, SFDA, Swiss Medic, TFDA and TGA). ‘Quality’ has been formally defined by six (n= 6) regulators (ANVISA, EMA, FDA, TFDA, and MHRA), while two (n= 2) have informally defined this term (CADTH and MFDS). ‘Relevance’ has been defined by six (n= 6) regulators (ANVISA, EMA, FDA, MFDS, NMPA/China Center for Drug Evaluation and TFDA). ‘Reliability’ has been defined by five (n= 5) regulators (EMA, FDA, MFDS, TFDA, NMPA/China Center for Drug Evaluation). Lastly, ‘fit-for-purpose’ has been defined by three (n= 3) regulators (EMA, FDA, TFDA). Of these four terms, we observe inconsistencies across definitions that warrant further consideration to support harmonization efforts.

Conclusions

Global regulatory agency efforts are underway to support the international harmonization of RWE standards across regulatory agencies. Duke-Margolis will continue to update the Dashboard to provide updates.