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Comparative Study
. 2021 Aug;10(15):5131-5140.
doi: 10.1002/cam4.4076. Epub 2021 Jun 21.

Clinical and cost outcomes following genomics-informed treatment for advanced cancers

Deirdre Weymann  1 , Samantha Pollard  1   2 , Brandon Chan  1 , Emma Titmuss  3 , Alexandra Bohm  3 , Janessa Laskin  4   5 , Steven J M Jones  3   6 , Erin Pleasance  3 , Jessica Nelson  3 , Alexandra Fok  3 , Howard Lim  4   5 , Aly Karsan  3   4   7 , Daniel J Renouf  4   5 , Kasmintan A Schrader  6   8   9 , Sophie Sun  4   5 , Stephen Yip  7   10 , David F Schaeffer  11 , Marco A Marra  3   6 , Dean A Regier  1   2
Affiliations
Comparative Study

Clinical and cost outcomes following genomics-informed treatment for advanced cancers

Deirdre Weymann et al. Cancer Med. 2021 Aug.

Abstract

Background: Single-arm trials are common in precision oncology. Owing to the lack of randomized counterfactual, resultant data are not amenable to comparative outcomes analyses. Difference-in-difference (DID) methods present an opportunity to generate causal estimates of time-varying treatment outcomes. Using DID, our study estimates within-cohort effects of genomics-informed treatment versus standard care on clinical and cost outcomes.

Methods: We focus on adults with advanced cancers enrolled in the single-arm BC Cancer Personalized OncoGenomics program between 2012 and 2017. All individuals had a minimum of 1-year follow up. Logistic regression explored baseline differences across patients who received a genomics-informed treatment versus a standard care treatment after genomic sequencing. DID estimated the incremental effects of genomics-informed treatment on time to treatment discontinuation (TTD), time to next treatment (TTNT), and costs. TTD and TTNT correlate with improved response and survival.

Results: Our study cohort included 346 patients, of whom 140 (40%) received genomics-informed treatment after sequencing and 206 (60%) received standard care treatment. No significant differences in baseline characteristics were detected across treatment groups. DID estimated that the incremental effect of genomics-informed versus standard care treatment was 102 days (95% CI: 35, 167) on TTD, 91 days (95% CI: -9, 175) on TTNT, and CAD$91,098 (95% CI: $46,848, $176,598) on costs. Effects were most pronounced in gastrointestinal cancer patients.

Conclusions: Genomics-informed treatment had a statistically significant effect on TTD compared to standard care treatment, but at increased treatment costs. Within-cohort evidence generated through this single-arm study informs the early-stage comparative effectiveness of precision oncology.

Keywords: biostatistics; genomic sequencing; healthcare costs; precision oncology; quasi-experimental methods; treatment outcomes.

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Conflict of interest statement

Brandon Chan, Steven J.M. Jones, and Marco A. Marra report no conflicts of interest. Deirdre Weymann and Samantha Pollard codirect IMPRINT Research Consulting and have consulted for Roche Canada. Janessa Laskin has received honoraria for academic talks from: Roche Canada, Pfizer Canada, Astra‐Zeneca Canada, and BI Canada; her institution has received research funding for her projects from: Roche Canada, Asta‐Zeneca Canada, and BI Canada. Daniel J. Renouf disclosures include research funding and honoraria from Bayer and Roche, and travel funding and honoraria from Servier, Celgene, Taiho, Ipsen, and Astra Zenec. Howard Lim has received honoraria from Eisai, Taiho, Roche, Lilly, BMS, Amgen, and Leo for consultant work and is an investigator on trials with Bayer, BMS, Lilly, Roche, Astra‐Zeneca, and Amgen. Sophie Sun has received research grant and honoraria funding from Astra‐Zeneca. Stephen Yip is an advisory board member for and has received travel allowance from Amgen, AstraZeneca, Bayer, Norvatis, and Roche. Dean A. Regier has received speaking honoraria from Roche Canada. DF Schaeffer has received honoraria from Alimentiv, Pfizer, Merck, and Diaceutics. Kasmintan A. Schrader has received speaking honoraria from AstraZeneca and Pfizer.

Figures

FIGURE 1
FIGURE 1
Study flow diagram
FIGURE 2
FIGURE 2
Pre‐ and post‐sequencing trends in average (A) time to treatment discontinuation; (B) time to next treatment; and (C) treatment cost
See this image and copyright information in PMC

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