Real-world effectiveness of pembrolizumab among patients with TMB-H advanced solid tumors (MK-3475-G34)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Observational retrospective secondary data collection study

Study drug and medical condition

• Name of medicine:
  KEYTRUDA
• Name of medicine, other:
  Pembrolizumab
• Study drug International non-proprietary name (INN) or common name:
  PEMBROLIZUMAB
• Anatomical Therapeutic Chemical (ATC) code:
  (L01FF02) pembrolizumab

  pembrolizumab
• Medical condition to be studied:
  Neoplasm malignant

Population studied

• Short description of the study population:
  Adult patients with advanced/metastatic solid tumors, who were TMB-H (>=10 mut/mb, tested by FoundationOne CDx (F1/F1CDx) and non-microsatellite instability-high (non-MSI-H), and received pembrolizumab monotherapy in 2nd line or 2nd line+ (2L/2L+) setting.
• Age groups:
  Adult and elderly population (≥18 years)

  Adults (18 to < 65 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Elderly (≥ 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  368

Study design details

• Study design:
  It is an observational retrospective secondary data collection study using a structured licensed dataset delivered by Flatiron.
• Main study objective:
  The main objective of the study is to describe real-world effectiveness of pembrolizumab among patients with advanced solid tumors with TMB-H assayed by F1/F1CDx and to stratify by TMB cutoff points, tumor types when feasible.
• Setting:
  Adult patients with advanced/metastatic solid tumors included in the Flatiron Health-Foundation Medicine Clinical Genomic Database (CGDB).;
• Comparators:
  N/A
• Outcomes:
  The primary endpoint is real-world response rate (rwRR), which is the proportion of the patients in the analysis population who had real-world complete response (rwCR) or real-world partial response (rwPR).; Secondary endpoint is real-world duration of response (rwDOR): in the subset of patients with a rwCR or rwPR, the time from first documented evidence of rwCR or rwPR until the first documented sign of disease progression, or death due to any cause, whichever occurs first.;
• Data analysis plan:
  The analyses will be of descriptive nature in this study. Baseline characteristics (at index date) of the study population will be described including demographics, disease stage, prior treatment, Eastern Cooperative Oncology Group (ECOG), biomarker status (TMB, MSI). Duration of follow-up will also be described.; For the study endpoints, analyses will be conducted across all tumor types as well as by tumor type, by TMB cutoff points, and by TMB within each tumor type, when feasible. Point estimates and exact Clopper-Pearson Confidence Intervals (CIs) will be provided.