Emulation of the KEYNOTE-189 Trial Using Electronic Health Records

NCT05908799 | Completed FDA Drug

• 1 other identifier: 1
• Study Type: observational
• Target: 1,854
• Locations: 1 country
• Sites: 1

Brief Summary

Investigators are building an empirical evidence base supporting the utility of real-world data through the emulation of randomized controlled trials in the oncology setting. The purpose of this work is to demonstrate whether real-world evidence studies can provide reliable conclusions on treatment effectiveness to inform further applications of real-world data in pharmaceutical product label expansion, post-marketing safety, and other purposes that are complementary to RCTs.

Conditions

Non-small Cell Lung Cancer; Metastatic Lung Cancer

Keywords

real-world evidence; real-world data; oncology; pharmacoepidemiology; comparative effectiveness research

Outcome Measures

Primary Outcomes (2)

• Overall Survival - Mortality Hazard Ratio

  Relative hazard of mortality due to any cause

  Through study completion (earliest of 640 days or censoring)

• Overall Survival - Mortality Risk

  12-month survival probability

  1 year, year 1

Study Arms (2)

Pembrolizumab + Chemotherapy

Exposure Group

Drug: Pembrolizumab + Pemetrexed-Platinum

Chemotherapy

Reference Group

Drug: Pemetrexed-Platinum

Interventions

Pembrolizumab + Pemetrexed-Platinum DRUG

Medication records in EHR database used to define exposure group

Pembrolizumab + Chemotherapy

Pemetrexed-Platinum DRUG

Medication records in EHR database used to define comparator group

Chemotherapy

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Non-small cell lung cancer patients with evidence of metastatic disease and a subsequent record indicating a first-line treatment.

You may qualify if:

• Has evidence in electronic health record indicating a 'lung' tumor site, non-squamous morphology (e.g., adenocarcinoma, large cell, etc), and a stage IV or metastatic disease in the form of diagnosis codes or derived variables from physician notes or linked data from a tumor registry. There is no evidence of other lung cancer types (i.e., mesothelioma, small cell, etc.).
• Does not have evidence of HGVS codes in health record indicating EGFR or ALK mutations.
• Has no evidence of use of guideline-recommended systemic cancer therapy\* for NSCLC on or after first date associated with stage IV (or metastatic) disease and prior to index treatment.

You may not qualify if:

• Has evidence of squamous cell lung cancer morphology in the form of diagnosis codes or derived variables from physician notes or linked information from a tumor registry.
• Has evidence of systemic cancer therapy utilization on or after first date associated with stage IV (or metastatic) disease and prior to index treatment. Patient has no evidence of biologic therapy at any time prior to the first record indicating an exposure of interest.
• Has evidence in medical record or tumor registry of primary malignancies beyond the lung tissue, except basal cell carcinoma of the skin, superficial bladder cancer, squamous cell carcinoma of the skin, in situ cervical cancer, or other in situ cancers.
• Has evidence of central nervous system (CNS) metastases and/or carcinomatous meningitis in the 2 weeks prior to initiation.
• Has a record of use of one of the following agents in the 2 years prior to the index date: methotrexate, sulfasalazine, hydroxychloroquine, leflunomide, azathioprine, etanercept, adalimumab, infliximab, certolizumab, golimumab, anakinra, tocilizumab, sarilumumab, abatacept, rituximab, tofacitinib, barivitinib, upadacitinib, mycophenolate.
• Has at least two records 60 days apart indicating use of one of the following agents in the 3 months prior to the index date: prednisone, prednisolone, methylprednisolone, dexamethasone, hydrocortisone.
• Has evidence of use of one of the following agents prior to the index date: pembrolizumab, nivolumab, cemiplimab, atezolizumab, durvalumab.

Sponsors & Collaborators

• Aetion, Inc.: lead

Study Sites (1)

Aetion

Boston, Massachusetts, 02109, United States

Location

Related Publications (1)

• Merola D, Campbell U, Gautam N, Rubens A, Schneeweiss S, Wang SV, Carrigan G, Chia V, Ovbiosa OE, Pinheiro S, Bruno A, Jiao X, Stewart M, Hendricks-Sturrup R, Rodriguez-Watson C, Khosla S, Zhang Y, Rimawi M, Huang J, Taylor A, Becnel L, McRoy L, Eckert J, Taylor B. The Aetion Coalition to Advance Real-World Evidence through Randomized Controlled Trial Emulation Initiative: Oncology. Clin Pharmacol Ther. 2023 Jun;113(6):1217-1222. doi: 10.1002/cpt.2800. Epub 2022 Dec 7.

  PMID: 36408668 BACKGROUND

MeSH Terms

Conditions

Carcinoma, Non-Small-Cell Lung; Lung Neoplasms; Neoplasms

Interventions

pembrolizumab

Condition Hierarchy (Ancestors)

Carcinoma, Bronchogenic; Bronchial Neoplasms; Respiratory Tract Neoplasms; Thoracic Neoplasms; Neoplasms by Site; Lung Diseases; Respiratory Tract Diseases

Study Officials

• David Merola, PharmD, PhD

  Aetion, Inc.

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Scientist

Study Record Dates

• First Submitted: June 7, 2023
• First Posted: June 18, 2023
• Study Start: June 5, 2023
• Primary Completion: August 31, 2023
• Study Completion: August 31, 2023
• Last Updated: September 13, 2023

Record last verified: 2023-09

Locations

US (1)