About RWE

Reviewing regulations: Using RWE for regulatory decision making

RWE can be used to complement data from RCTs to gain a more complete picture of the advantages and disadvantages of medications as they are used in practice.1 Regulatory authorities are increasingly recognizing the importance of RWE,18–20 and this is important for HCPs to be aware of.

Food and Drug Administration

The United States Congress has defined RWE as data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than traditional clinical trials. The FDA uses RWD and RWE to monitor post-marketing safety and adverse events and to make regulatory decisions. The 21st Century Cures Act, passed in 2016, places additional focus on the use of RWE to support regulatory decision making, including the approval of new indications for approved drugs.18

There are several FDA guidance documents available. Please click on the links provided to find out more:

European Medicines Agency

According to the EMA, RWD holds the promise to substantially increase the effectiveness and efficiency of all processes in the development and utilization of medicines.19 These include research and development, pricing and reimbursement decisions, regulatory decision making and use in medical practice.

The EMA Executive Director and Senior Medical Officer, heads of three national EU agencies, as well as academia, payer, and Organization for Economic Co-operation and Development (OECD) representatives, stressed in a recent publication that a “learning healthcare system” will be needed to fully realize the potential of RWE.21 While there are currently no formal guidelines or frameworks published by the EMA, there are several presentations available. Please click on the links to find out more.

A learning healthcare system, based on electronic health records and other routinely collected healthcare data, would allow RWD to be continuously fed into the system, ensuring that with every new patient treated, we know more overall about the practice of medicine.21

Health Canada

Health Canada has undertaken a project aiming to improve the ability to assess and monitor the safety, efficacy and effectiveness of drugs across the drug life cycle by optimizing the use of RWE.20 It is expected that work on all parts of the project will be complete by autumn 2022, and outcomes include:

  • Increased use of RWE to enhance regulatory decision making and risk communications throughout the drug life cycle
  • Improved use and sharing of RWE with healthcare system partners
  • Increased clarity for stakeholders on where and how RWE can be used to support regulatory decision making
  • Improved access to drugs through the use of new sources of evidence to support approval of drug applications

Please visit Health Canada’s website for more information



1. Berger, M. L. et al. Pharmacoepidemiol. Drug Saf. 2017; 26: 1033–1039.2. Garrison, L. P., Neumann, P. J., Erickson, P., Marshall, D. & Mullins, C. D. Value Health J. Int. Soc. Pharmacoeconomics Outcomes Res. 2007; 10: 326–335. 3. Makady, A. et al. Value Health J. Int. Soc. Pharmacoeconomics Outcomes Res. 2017; 20: 858–865. 4. Breckenridge, A. M., Breckenridge, R. A. & Peck, C. C. R Br. J. Clin. Pharmacol. 2019; 85: 1874–1877. 5. Costello, C. et al. Future Oncol. 2019; 15: 1411–1428. 6. Richardson, P. G. et al. Blood Cancer J. 2018; 8: 109. 7. Blonde, L., Khunti, K., Harris, S. B., Meizinger, C. & Skolnik, N. S. Adv. Ther. 2018; 35: 1763–1774. 8. FDA. FRAMEWORK FOR FDA’S REAL-WORLD EVIDENCE PROGRAM. Available at: https://www.fda.gov/media/120060/download. Last accessed Sept 2021. within it. 9. Makady, A. et al. Value Health J. Int. Soc. Pharmacoeconomics Outcomes Res. 2017; 20: 520–532. 10. Shah, J. J. et al. Clin. Lymphoma Myeloma Leuk. 2017; 17: 575-583.e2. 11. Costa, L. J., Hari, P. N. & Kumar, S. K. Leuk. Lymphoma 2016; 57: 2827–2832. 12. Ganguly, S., Mailankody, S. & Ailawadhi, S. Am. Soc. Clin. Oncol. Educ. Book 2019; 519–529: doi:10.1200/EDBK_238551. 13. Pulte, E. D. et al. Blood Adv. 2018; 2: 116–119. 14. Richardson, P. G. et al. Blood 2017; 130: 3149–3149. 15. Rudrapatna, V. A. & Butte, A. J. J. Clin. Invest. 2020: 130: 565–574.16. Di Maio, M., Perrone, F. & Conte, P. The Oncologist 2020; 25: e746–e752. 17. Kim, H.-S., Lee, S. & Kim, J. H. J. Korean Med. Sci. 2018; 3. 18. Office of the Commissioner. Real-World Evidence. 2020. FDA. Available at: https://www.fda.gov/science- research/science-and-research-special-topics/real-world-evidence. Last accessed Sept 2021. 19. EMA. Harnessing the potential of real world data through a ‘learning healthcare system’. 2018. Available at: https://www.ema.europa.eu/en/news/harnessing-potential-real-world-data-through-learning-healthcare-system. 20. Health Canada. Regulatory review of drugs and devices: Strengthening the use of real world evidence for drugs. 2018. Available at: https://www.canada.ca/ en/health-canada/corporate/transparency/regulatory-transparency-and-openness/improving-review-drugs-devices/strengthening-use-real-world-evidence- drugs.html 21. Eichler, H.-G. et al. Clin. Pharmacol. Ther. 2019; 105: 912–922.


EHR/EMR, electronic health/medical records; EMA, European Medicines Association; EU, European Union; FDA, Food and Drug Administration; HCP, healthcare professional; MM, multiple myeloma; OECD, Organization for Economic Co-operation and Development; RCT, randomized controlled trial; RRMM, relapsed refractory multiple myeloma; RWD, real-world data; RWE, real-world evidence.