Initial investigation of using Norwegian health data for the purpose of external comparator arms - an example for non-small cell lung cancer

Abstract

Background

Using real-world (RW) data from registries to mimic or substitute a comparator arm of clinical trials is increasingly investigated. The feasibility of using data sources for this purpose depends on the source’s completeness of the wide spectrum of a disease characteristics and relevant endpoints, which could allow for proper matching of important variables.

Materials and methods

This is a Norwegian study using data from three population-based registries, the Cancer Registry (CRN), the National Patient Registry (NPR), and the Norwegian Prescribed Drug Registry (LMR). We assessed if the registries contained the information necessary for selecting a RW cohort of patients with characteristics that mimicked the inclusion and exclusion criteria from the control arm of the phase 3 trial (KeyNote-407) on patients with stage IV, squamous NSCLC. We did this both on an aggregated - and individual data level. We also described the survival in the RW-cohorts and compared it with the survival in the control arm of the KN-407 trial.

Results

Using aggregated data from the CRN allowed us to find the patients based on some clinically relevant inclusion criteria, but only to a limited extent could we apply the exclusion criteria of KN-407. When we used individual data from the CRN, NPR and the LMR we could create a patient cohort that shared more criteria corresponding to the eligibility criteria from KN-407, including exclusion criteria. Compared to the 11.3 months (CI 95% 9.5, 14.8) median survival in the control arm of KN-407, both RW-cohorts had a shorter median survival, 7.07 months (CI 95% 6.7, 9.5) (individual) and 8.0 months (CI 95% 5.8, 10.5) (aggregated).

Conclusion

Even if we demonstrated that the registries contain clinically relevant information necessary to mimic the eligibility criteria of a selected RCT, the survival is shorter for RW patients compared to their control arm counterpart in the RCT.

Keyword:

• RWD
• Cancer Registry Norway
• NSCLC
• squamous cell carcinoma
• external control arm

Disclosure statement

SB is employed by Merck Norway. For 3 years since 2021 she holds an industrial PhD-position and during that time she is released from any mandatory work for Merck. The industrial PhD project is publicly financed by the Norwegian Research council, grant number 321291. She is enrolled as a PhD-student at the Medical Faculty at the University of Oslo, where her research focuses on the use of real-world data and how specifically Norwegian lung cancer registry data can be used as an external control arm for clinical trials. She is supervised by AH MD, PhD and ST MD, PhD.

ST MD, PhD is the external supervisor of SB. He is an external consultant to Merck Norway and a founder of the company NordicRWE.

SBB MSc, is employed by NordicRWE.

AH MD, PhD is the internal supervisor of SB. In association with research/clinical studies she has received financial support and/or study drug from AstraZeneca, Roche, Novartis, Incyte, Eli Lilly, Ultimovacs and BMS. Adv board/advise: AstraZeneca, BMS, Janssen, MSD, Pfizer, Roche, Takeda, Sanofi, Bayer, EliLilly, Abbvie. All payments to institution. The funding bodies had no role in the data collection and analysis and were not involved in the interpretation of results, writing, revision, or approval of the manuscript.

Data availability statement

The raw data supporting the conclusions of this article can be made available by the authors, without undue reservations.