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

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

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.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Breast Neoplasms
  • Costs and Cost Analysis
  • Female
  • Gastrointestinal Neoplasms / genetics
  • Gastrointestinal Neoplasms / mortality
  • Gastrointestinal Neoplasms / pathology
  • Gastrointestinal Neoplasms / therapy
  • Genome-Wide Association Study
  • Genomics / economics
  • Genomics / methods
  • Humans
  • Logistic Models
  • Male
  • Middle Aged
  • Neoplasms / genetics*
  • Neoplasms / mortality
  • Neoplasms / pathology
  • Neoplasms / therapy*
  • Precision Medicine / economics*
  • Precision Medicine / methods
  • Retrospective Studies
  • Sequence Analysis, DNA*
  • Treatment Outcome
  • Withholding Treatment