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1 December 2022

R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 10

Authors: Alex Simpson and Sreeram V Ramagopalan [email protected]Author Info & Affiliations

Publication: Journal of Comparative Effectiveness Research

Volume 12, Number 1

https://doi.org/10.2217/cer-2022-0194

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Abstract

In this latest update we discuss the transportability of comparative effectiveness evidence across countries. We highlight results of a survey indicating that European HTA agencies are reluctant to accept real-world data from other countries, review recent benefit assessments indicating a potential softening of a requirement for the use of local real-world data in Germany, and outline a recent review presenting approaches that can correct for a lack of transportability.

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References

1.

Hernán MA, Hernández-Díaz S. Beyond the intention-to-treat in comparative effectiveness research. Clin. Trials 9(1), 48–55 (2012).

2.

Westreich D, Edwards JK, Lesko CR, Cole SR, Stuart EA. Target validity and the hierarchy of study designs. Am. J. Epidemiol. 188(2), 438–443 (2019).

3.

Bullement A, Podkonjak T, Robinson MJ et al. Real-world evidence use in assessments of cancer drugs by NICE. Int. J. Technol. Assess. Health Care (2020). https://pubmed.ncbi.nlm.nih.gov/32646531/

4.

Hatswell AJ, Baio G, Berlin JA, Irs A, Freemantle N. Regulatory approval of pharmaceuticals without a randomised controlled study: analysis of EMA and FDA approvals 1999–2014. BMJ Open 6(6), e011666 (2016).

5.

Patel D, Grimson F, Mihaylova E et al. Use of external comparators for health technology assessment submissions based on single-arm trials. Value Health 24(8), 1118–1125 (2021).

6.

Westreich D, Edwards JK, Lesko CR, Stuart E, Cole SR. Transportability of trial results using inverse odds of sampling weights. Am. J. Epidemiol. 186(8), 1010–1014 (2017).

7.

Hogervorst MA, Pontén J, Vreman RA, Mantel-Teeuwisse AK, Goettsch WG. Real world data in health technology assessment of complex health technologies. Front. Pharmacol. 13, 837302 (2022).

8.

Institute for Quality and Efficiency in Health Care. Development of scientific concepts for the generation of routine practice data and their analysis for the benefit assessment of drugs according to §35a Social Code Book V (SGB V). (2020). www.iqwig.de/en/projects/a19-43.html

9.

National Institute for Health and Care Excellence. Real-world evidence framework update. (2022). www.nice.org.uk/corporate/ecd9/chapter/overview

10.

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).

11.

Institute for Quality and Efficiency in Health Care. Supply-related data in manufacturer dossiers: things are not yet running smoothly. (2022). www.iqwig.de/en/presse/press-releases/press-releases-detailpage_67103.html

12.

Institute for Quality and Efficiency in Health Care. Sotorasib (NSCLC) - Nutzenbewertung gemäß § 35a SGB V. (2022). www.iqwig.de/download/a22-28_sotorasib_nutzenbewertung-35a-sgb-v_v1-0.pdf

13.

Institute for Quality and Efficiency in Health Care. Amivantamab (NSCLC) - Nutzenbewertung gemäß § 35a SGB V. (2022). www.iqwig.de/download/a22-05_amivantamab_nutzenbewertung-35a-sgb-v_v1-0.pdf

14.

Degtiar I, Rose S. A review of generalizability and transportability. (2021). https://arxiv.org/abs/2102.11904

15.

Phillippo DM, Ades AE, Dias S, Palmer S, Abrams KR, Welton NJ. Methods for population-adjusted indirect comparisons in health technology appraisal. Med. Decis. Making 38(2), 200–211 (2018).

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