An Exploratory Analysis of Real-World End Points for Assessing Outcomes Among Immunotherapy-Treated Patients With Advanced Non-Small-Cell Lung Cancer

Mark Stewart, Andrew D Norden, Nancy Dreyer, Henry Joe Henk, Amy P Abernethy, Elizabeth Chrischilles, Lawrence Kushi, Aaron S Mansfield, Sean Khozin, Elad Sharon, Srikesh Arunajadai, Ryan Carnahan, Jennifer B Christian, Rebecca A Miksad, Lori C Sakoda, Aracelis Z Torres, Emily Valice, Jeff Allen, Mark Stewart, Andrew D Norden, Nancy Dreyer, Henry Joe Henk, Amy P Abernethy, Elizabeth Chrischilles, Lawrence Kushi, Aaron S Mansfield, Sean Khozin, Elad Sharon, Srikesh Arunajadai, Ryan Carnahan, Jennifer B Christian, Rebecca A Miksad, Lori C Sakoda, Aracelis Z Torres, Emily Valice, Jeff Allen

Abstract

Purpose: This pilot study examined the ability to operationalize the collection of real-world data to explore the potential use of real-world end points extracted from data from diverse health care data organizations and to assess how these relate to similar end points in clinical trials for immunotherapy-treated advanced non-small-cell lung cancer.

Patients and methods: Researchers from six organizations followed a common protocol using data from administrative claims and electronic health records to assess real-world end points, including overall survival (rwOS), time to next treatment, time to treatment discontinuation (rwTTD), time to progression, and progression-free survival, among patients with advanced non-small-cell lung cancer treated with programmed death 1/programmed death-ligand 1 inhibitors in real-world settings. Data sets included from 269 to 6,924 patients who were treated between January 2011 and October 2017. Results from contributors were anonymized.

Results: Correlations between real-world intermediate end points (rwTTD and time to next treatment) and rwOS were moderate to high (range, 0.6 to 0.9). rwTTD was the most consistent end points as treatment detail was available in all data sets. rwOS at 1 year post-programmed death-ligand 1 initiation ranged from 40% to 57%. In addition, rwOS as assessed via electronic health records and claims data fell within the range of median OS values observed in relevant clinical trials. Data sources had been used extensively for research with ongoing data curation to assure accuracy and practical completeness before the initiation of this research.

Conclusion: These findings demonstrate that real-world end points are generally consistent with each other and with outcomes observed in randomized clinical trials, which substantiates the potential validity of real-world data to support regulatory and payer decision making. Differences observed likely reflect true differences between real-world and protocol-driven practices.

Conflict of interest statement

Andrew D. Norden

Employment: Cota Healthcare, IBM

Leadership: Cota Healthcare

Henry Joe Henk

Employment: Optum

Stock and Other Ownership Interests: United Health Group

Amy P. Abernethy

Employment: Flatiron Health

Leadership: AthenaHealth, Highlander Partners, SignalPath Research, One Health Company

Stock and Other Ownership Interests: AthenaHealth, Flatiron Health, Orange Leaf Associates

Honoraria: Genentech

Patents, Royalties, Other Intellectual Property: Patent pending for a technology that facilitates the extraction of unstructured information from medical records

Other Relationship: US Food and Drug Administration

Aaron S. Mansfield

Consulting or Advisory Role: Trovagene (Inst), Genentech (Inst), Bristol-Myers Squibb (Inst), AbbVie (Inst)

Research Funding: Novartis (Inst)

Travel, Accommodations, Expenses: AbbVie, Roche

Srikesh Arunajadai

Employment: Cota Healthcare, Kantar Health

Jennifer B. Christian

Employment: IQVIA

Stock and Other Ownership Interests: GlaxoSmithKline, IQVIA

Rebecca A. Miksad

Employment: Flatiron Health

Stock and Other Ownership Interests: Flatiron Health, Roche

Consulting or Advisory Role: Advanced Medical, Grand Rounds (I), InfiniteMD

Research Funding: Bayer (Inst), Exelixis (Inst)

Other Relationship: De Luca Foundation

Lori C. Sakoda

Stock and Other Ownership Interests: Johnson & Johnson

Aracelis Z. Torres

Employment: Flatiron Health

Stock and Other Ownership Interests: Flatiron Health

No other potential conflicts of interest were reported.

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Source: PubMed

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