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Retrospective observational cohort study on innovation in oncology and progress in survival: How far have we gotten in the two decades of treating patients with advanced non-small cell lung cancer as a single population?

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  • Nahila Justo
  • Jonas Nilsson
  • Beata Korytowsky
  • Johan Dalen
  • Terri Madison
  • Alistair McGuire

Abstract

We assessed the impact of new antineoplastic agents on the overall survival (OS) of advanced non-small cell lung cancer (aNSCLC) patients followed up until 2012. Multivariate regression models were run for OS (outcome) and four proxies for innovation (exposure): Index (InnovInd, for SEER-Research data 1973–2012) and three levels of aggregation of Mean Medication Vintage, i.e. Overall (MMVOverall), using data aggregated at the State Level (MMVState), and using patient-level data (MMVPatient) using data from the US captured in SEER-Medicare 1991–2012. We derived Hazard ratios (HR) from Royston-Parmar models and odds ratios (OR) from a logistic regression on 1-year OS. Including 164,704 patients (median age 72 years, 56.8% stage IV, 61.8% with no comorbidities, 37.8% with adenocarcinoma, 22.9% with squamous-cell, 6.1% were censored). One-year OS improved from 0.22 in 1973 to 0.39 in 2012, in correlation with InnovInd (r = 0.97). Ten new NSCLC drugs were approved and 28 more used off-label. Regression-models results indicate that therapeutic innovation only marginally reduced the risk of dying (HROverall = 0.98 [0.98–0.98], HRMMV-Patient = 0.98 [0.97–0.98], and HRMMV-State = 0.98 [0.98–0.98], and slightly improved 1-year survival (ORMMV-Overall = 1.05 95%CI [1.04–1.05]). These results were validated with data from the Swedish National Health Data registers. Until 2013, aNSCLC patients were treated undifferentiated and the introduction of innovative therapies had statistically significant, albeit modest, effects on survival. Most treatments used off-guidelines highlight the high unmet need; however new advancements in treatment may further improve survival.

Suggested Citation

  • Nahila Justo & Jonas Nilsson & Beata Korytowsky & Johan Dalen & Terri Madison & Alistair McGuire, 2020. "Retrospective observational cohort study on innovation in oncology and progress in survival: How far have we gotten in the two decades of treating patients with advanced non-small cell lung cancer as ," PLOS ONE, Public Library of Science, vol. 15(5), pages 1-12, May.
    • Handle: RePEc:plo:pone00:0232669
    DOI: 10.1371/journal.pone.0232669
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    References listed on IDEAS

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    1. Frank R. Lichtenberg, 2014. "Has Medical Innovation Reduced Cancer Mortality?," CESifo Economic Studies, CESifo Group, vol. 60(1), pages 135-177.
    2. Frank Lichtenberg, 2015. "The Impact of Pharmaceutical Innovation on Premature Mortality, Cancer Mortality, and Hospitalization in Slovenia, 1997–2010," Applied Health Economics and Health Policy, Springer, vol. 13(2), pages 207-222, April.
    3. Patrick Royston, 2005. "Multivariable regression models with continuous covariates, with a practical emphasis on fractional polynomials and applications in clinical epidemiology," German Stata Users' Group Meetings 2005 01, Stata Users Group.
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