TY - JOUR
T1 - Comparing Survival Outcomes for Advanced Cancer Patients Who Received Complex Genomic Profiling Using a Synthetic Control Arm
AU - O’Haire, Sophie
AU - Degeling, Koen
AU - Franchini, Fanny
AU - Tran, Ben
AU - Luen, Stephen J.
AU - Gaff, Clara
AU - Smith, Kortnye
AU - Fox, Stephen
AU - Desai, Jayesh
AU - IJzerman, Maarten
N1 - Funding Information:
The authors acknowledge all contributing members of the Melbourne Genomics Health Alliance Solid Tumour Flagship team and the Molecular Pathology department of the Peter MacCallum Cancer Centre. We thank all patients and their families for their involvement in the study.
Funding Information:
SOH, KD, FF, CG, SJL, SF and JD declare no potential conflicts of interest. MIJ receives unrestricted research funding to the University of Melbourne from Illumina. BT reports grants and personal fees from Amgen, grants and personal fees from Astra Zeneca, grants from Astellas, grants and personal fees from BMS, grants and personal fees from Janssen, grants and personal fees from Pfizer, grants and personal fees from MSD, grants and personal fees from Ipsen, personal fees from IQVIA, personal fees from Sanofi, personal fees from Tolmar, personal fees from Novartis, grants and personal fees from Bayer, personal fees from Roche, outside the submitted work.
Funding Information:
The CGP study was funded the Melbourne Genomics Health Alliance. No funding was received for performing this analysis. Sophie O’Haire was supported by an Australian Government Research Training Program (RTP) Fee-Offset Scholarship through The University of Melbourne.
Publisher Copyright:
© 2022, The Author(s).
PY - 2022/9
Y1 - 2022/9
N2 - Background: Complex genomic profiling (CGP) has transformed cancer treatment decision making, yet there is a lack of robust and quantifiable evidence for how utilisation of CGP improves patient outcomes. Objective: This study evaluated cohort level clinical effectiveness of CGP to improve overall survival (OS) in real-world advanced cancer patients using a registry-based matched control population. Patients and methods: Two cohorts of advanced and refractory cancer patients were seen in consecutive series for early phase trial enrolment consideration. The first cohort (CGP group) accessed tumour profiling via a research study; while the second cohort that followed was not profiled. Overall survival between cohorts was compared using Kaplan-Meier curves and Cox proportional hazard models. Potential confounding was analysed and adjusted for using stabilised weights based on propensity scores. Results: Within the CGP group, 25 (17.6%) patients received treatment informed by CGP results and this subgroup had significantly improved survival compared with CGP patients in whom results did not impact their treatment (unadjusted HR = 0.44, (0.22–0.88), p = 0.02). However, when comparing the entire CGP cohort with the No CGP cohort, no significant survival benefit was evident with adjusted median OS for CGP of 13.5 months (9.2–17.0) compared with 11.0 (9.2–17.4) for No CGP (adjusted HR = 0.92, (0.65–1.30), p = 0.63). Conclusions: This study utilised real-world data to simulate a control arm and quantify the clinical effectiveness of genomic testing. The magnitude of survival benefit for patients who had CGP result-led treatments was insufficient to drive an overall survival gain for the entire tested population. Translation of CGP into clinics requires strategies to ensure higher rates of tested patients obtain clinical benefit to deliver on the value proposition of CGP in an advanced cancer population.
AB - Background: Complex genomic profiling (CGP) has transformed cancer treatment decision making, yet there is a lack of robust and quantifiable evidence for how utilisation of CGP improves patient outcomes. Objective: This study evaluated cohort level clinical effectiveness of CGP to improve overall survival (OS) in real-world advanced cancer patients using a registry-based matched control population. Patients and methods: Two cohorts of advanced and refractory cancer patients were seen in consecutive series for early phase trial enrolment consideration. The first cohort (CGP group) accessed tumour profiling via a research study; while the second cohort that followed was not profiled. Overall survival between cohorts was compared using Kaplan-Meier curves and Cox proportional hazard models. Potential confounding was analysed and adjusted for using stabilised weights based on propensity scores. Results: Within the CGP group, 25 (17.6%) patients received treatment informed by CGP results and this subgroup had significantly improved survival compared with CGP patients in whom results did not impact their treatment (unadjusted HR = 0.44, (0.22–0.88), p = 0.02). However, when comparing the entire CGP cohort with the No CGP cohort, no significant survival benefit was evident with adjusted median OS for CGP of 13.5 months (9.2–17.0) compared with 11.0 (9.2–17.4) for No CGP (adjusted HR = 0.92, (0.65–1.30), p = 0.63). Conclusions: This study utilised real-world data to simulate a control arm and quantify the clinical effectiveness of genomic testing. The magnitude of survival benefit for patients who had CGP result-led treatments was insufficient to drive an overall survival gain for the entire tested population. Translation of CGP into clinics requires strategies to ensure higher rates of tested patients obtain clinical benefit to deliver on the value proposition of CGP in an advanced cancer population.
UR - http://www.scopus.com/inward/record.url?scp=85137473260&partnerID=8YFLogxK
U2 - 10.1007/s11523-022-00910-0
DO - 10.1007/s11523-022-00910-0
M3 - Article
C2 - 36063280
AN - SCOPUS:85137473260
SN - 1776-2596
VL - 17
SP - 539
EP - 548
JO - Targeted Oncology
JF - Targeted Oncology
IS - 5
ER -
