Skip to main content
Open access
Industry Update
4 January 2024

R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 14

Abstract

In this latest update we highlight: a publication from the US FDA regarding the definitions of real-world data (RWD) and real-world evidence (RWE); a publication from academic researchers on a demonstration project for target trial emulation; a publication from the National Institute of Health and Care Excellence (NICE) on the 1 year anniversary of their RWE framework; and a publication from NICE and Flatiron Health on the utility of US RWD for initial UK health technology assessment decision making.

Formats available

You can view the full content in the following formats:

References

1.
Rahman M, Dal Pan G, Stein P et al. When can real-world data generate real-world evidence? Pharmacoepidemiol. Drug Saf. (2023) (Online ahead of print).
2.
Yuan L, Rahman M, Concato J. Comparison of two assessments of real-world data and real-world evidence for regulatory decision-making. Clin Transl Sci. (2023) (Online ahead of print).
3.
Wieseler B, Neyt M, Kaiser T et al. Replacing RCTs with real world data for regulatory decision making: a self-fulfilling prophecy? BMJ. 380, e073100 (2023).
4.
Hernán MA, Robins JM. Using big data to emulate a target trial when a randomized trial is not available. Am. J. Epidemiol. 183(8), 758–764 (2016).
5.
National Institute for Health and Care Excellence. NICE real-world evidence framework (2022). https://www.nice.org.uk/corporate/ecd9/resources/nice-realworld-evidence-framework-pdf-1124020816837
6.
Canada's Drug and Health Technology Agency. Guidance for Reporting Real-World Evidence (2023). https://www.cadth.ca/sites/default/files/RWE/MG0020/MG0020-RWE-Guidance-Report-Secured.pdf
7.
Vanier A, Fernandez J, Kelley S et al. Rapid access to innovative medicinal products while ensuring relevant health technology assessment. Position of the French National Authority for Health. BMJ Evid. Based Med. (2023). (Online ahead of print).
8.
Simpson A, Ramagopalan SV. R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 7. J. Comp. Eff. Res. 11(10), 699–701 (2022).
9.
Bray B, Ramagopalan SV. R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 12. J. Comp. Eff. Res. 12(7), e230092 (2023).
10.
Moler-Zapata S, Hutchings A, O'Neill S et al. Emulating target trials with real-world data to inform health technology assessment: findings and lessons from an application to emergency surgery. Value Health. 26(8), 1164–1174 (2023).
11.
Simpson A, Ramagopalan SV. R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 8. J. Comp. Eff. Res. 11(13), 915–917 (2022).
12.
Duffield S, Jónsson P. The real-world impact of National Institute for Health and Care Excellence's real-world evidence framework. J. Comp. Eff. Res. 12(11), e230135 (2023).
13.
Wang SV, Pottegård A, Crown W et al. HARmonized Protocol Template to Enhance Reproducibility of hypothesis evaluating real-world evidence studies on treatment effects: a good practices report of a joint ISPE/ISPOR task force. Pharmacoepidemiol. Drug Saf. 32(1), 44–55 (2023).
14.
Mpofu P, Kent S, Jónsson P et al. Evaluation of US oncology electronic health record real-world data to reduce uncertainty in health technology appraisals: a retrospective cohort study. BMJ Open. 13(10), e074559 (2023).
15.
Regier DA, Pollard S, McPhail M et al. A perspective on life-cycle health technology assessment and real-world evidence for precision oncology in Canada. NPJ Precis. Oncol. 6(1), 76 (2022).