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Review
. 2021 Jun;30(6):685-693.
doi: 10.1002/pds.5222. Epub 2021 Mar 18.

Real-world evidence to support regulatory decision making: New or expanded medical product indications

Jessica M Franklin  1 , Kai-Li Liaw  2 , Solomon Iyasu  2 , Cathy W Critchlow  3 , Nancy A Dreyer  4
Affiliations
Review

Real-world evidence to support regulatory decision making: New or expanded medical product indications

Jessica M Franklin et al. Pharmacoepidemiol Drug Saf. 2021 Jun.

Abstract

There is increasing interest in utilizing real-world data (RWD) to produce real-world evidence (RWE) on the benefits and risks of medical products that could support regulatory approval decisions. The field of pharmacoepidemiology has a long history of focusing on data and evidence that would now be termed "real-world," including evidence from healthcare claims, registries, and electronic health records. However, several emerging trends over the past decade are converging to support the use of these and other RWD sources for approval decisions, and there are several recent examples and ongoing research that demonstrate how RWE may be used to support regulatory approval of new or expanded indications. The goal of this article is to review the current landscape and future directions of the use of RWE in this context. This manuscript is endorsed by the International Society for Pharmacoepidemiology (ISPE).

Keywords: external control arms; healthcare databases; nonrandomized studies; pharmacoepidemiology; pragmatic clinical trials; real-world evidence.

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References

REFERENCES

    1. U. S. Food and Drug Administration. Prescription Drug User Fee Act (PDUFA)-PDUFA VI: Fiscal Years 2018-2022.
    1. Bonamici S. H.R.34-114th Congress (2015-2016): 21st Century Cures Act. 2016. https://www.congress.gov/bill/114th-congress/house-bill/34. Accessed June 15, 2017.
    1. US FDA. Framework for FDA's real-world evidence program. 2018. https://www.fda.gov/media/120060/download
    1. Eichler H, Bloechl-Daum B, Broich K, et al. Data rich, information poor: can we use electronic health records to create a learning healthcare system for pharmaceuticals? Clin Pharmacol Ther. 2019;105(4):912-922. https://doi.org/10.1002/cpt.1226.
    1. Tanaka S, Yamamoto T, Oda E, Nakamura M, Fujiwara S. Real-world evidence of raloxifene versus alendronate in preventing non-vertebral fractures in Japanese women with osteoporosis: retrospective analysis of a hospital claims database. J Bone Miner Metab. 2018;36(1):87-94. https://doi.org/10.1007/s00774-016-0809-0.

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