FDA’s Oncology Center of Excellence staff have conducted several pooled analyses of oncology clinical trial data in recent years. Pooled analyses are a type of meta-analysis that involve investigation of patient-level data from multiple studies. Despite methodological challenges, pooled analyses have provided valuable information to support oncology drug development and can play an important role in building evidence for surrogate endpoints.
This page includes articles from OCE Publications that involve pooled analyses of clinical trial data submitted to FDA. These analyses considered different research questions analyzing specified groups of clinical trials. Common themes include studies evaluating underrepresented groups and special populations, clinical, pathologic and/or genetic subgroups, relationships among study endpoints, safety and/or efficacy of regulated products, and use of external controls.
2024
Efficacy of Poly (ADP-ribose) Polymerase Inhibitors by Individual Genes in Homologous Recombination Repair Gene-Mutated Metastatic Castration-Resistant Prostate Cancer: A US Food and Drug Administration Pooled Analysis.
Cytoreductive nephrectomy in the era of immune checkpoint inhibitors: a U.S. FDA pooled analysis.
A Pooled Analysis of Treatment-Free Survival in Advanced Renal Cell Carcinoma.
Correlations of response rate and progression-free survival with overall survival in immunotherapy trials for metastatic non-small-cell lung cancer: an FDA pooled analysis.
2023
FDA Analysis of Ineligibility for Acute Myeloid Leukemia Clinical Trials by Race and Ethnicity.
Association between antibiotics and adverse oncological outcomes in patients receiving targeted or immune-based therapy for hepatocellular carcinoma.
Eligibility criteria and Enrollment of a Diverse Racial and Ethnic population in Multiple Myeloma Clinical Trials.
The Association Between Baseline Hepatic or Renal Function and Clinical Outcomes for Patients with Non-Small Cell Lung Cancer Treated With a PD-1/PD-L1 Blocking Antibody Using Real-World and Trial Data.
Enrollment representation of age, race, and ethnicity in ovarian cancer registrational clinical trials (2010-2020): An evaluation by the U.S. Food and Drug Administration.
A U.S. Food and Drug Administration-pooled Analysis of Frontline Combination Treatment Survival Benefits by Risk Groups in Metastatic Renal Cell Carcinoma.
Use of patient-reported outcomes (PRO) data to complement exposure-response analysis in early clinical cancer drug development.
2022
Association of Radiation Therapy with Risk of Adverse Events in Patients Receiving Immunotherapy: A Pooled Analysis of Trials in the US Food and Drug Administration Database.
Floor and ceiling effects in the EORTC QLQ-C30 Physical Functioning Subscale among patients with advanced or metastatic breast cancer.
Response Rate, Event-Free Survival, and Overall Survival in Newly Diagnosed Acute Myeloid Leukemia: US Food and Drug Administration Trial-Level and Patient-Level Analyses.
Analysis of racial and ethnic disparities in multiple myeloma US FDA drug approval trials.
Updated FDA pooled analysis of pain medication use in trial participants with HR+, HER2-negative metastatic breast cancer treated with endocrine therapy and a CDK 4/6 inhibitor.
2021
Model Development of CDK4/6 Predicted Efficacy in Patients with Hormone Receptor-Positive, Human Epidermal Growth Factor Receptor 2-Negative Advanced or Metastatic Breast Cancer.
Review of patient-reported outcomes in multiple myeloma registrational trials: highlighting areas for improvement.
Survival outcomes in older men with non-metastatic castration-resistant prostate cancer treated with androgen receptor inhibitors: a US Food and Drug Administration pooled analysis of patient-level data from three randomised trials.
Patient-Reported Outcomes After Treatment Discontinuation: Commercial Clinical Trial Data from Four Cancer Types.
Treatment-related toxicity and improved outcome from immunotherapy in hepatocellular cancer: Evidence from an FDA pooled analysis of landmark clinical trials with validation from routine practice.
Overall survival in patients with hormone receptor-positive, HER2-negative, advanced or metastatic breast cancer treated with a cyclin-dependent kinase 4/6 inhibitor plus fulvestrant: a US Food and Drug Administration pooled analysis.
Floor and ceiling effects in the EORTC QLQ-C30 Physical Functioning Subscale among patients with advanced or metastatic breast cancer.
2020
Idelalisib for Treatment of Relapsed Follicular Lymphoma and Chronic Lymphocytic Leukemia: A Comparison of Treatment Outcomes in Clinical Trial Participants vs Medicare Beneficiaries.
CDK4/6 inhibitor treatment for patients with hormone receptor-positive, HER2-negative, advanced or metastatic breast cancer: a US Food and Drug Administration pooled analysis.
An Empirical Investigation of Bayesian Clinical Trial Design in Metastatic Breast Cancer.
An FDA Analysis of Survival Outcomes Comparing an Adjuvant Paclitaxel and Trastuzumab Trial to an External Control from Historical Clinical Trials.
US Food and Drug Administration review of statistical analysis of patient-reported outcomes in lung cancer clinical trials approved between January 2008 and December 2017.
2019
Comparison of iRECIST versus RECIST V.1.1 in patients treated with an anti-PD-1 or PD-L1 antibody: pooled FDA analysis.
Patient reported outcomes in anti-PD-1/PD-L1 inhibitor immunotherapy registration trials: FDA analysis of data submitted and future directions.
Analysis of time-to-treatment discontinuation of targeted therapy, immunotherapy, and chemotherapy in clinical trials of patients with non-small-cell lung cancer.
Analysis of the Association Between Adverse Events and Outcome in Patients Receiving a Programmed Death Protein 1 or Programmed Death Ligand 1 Antibody.
Prognostic Value of the Lung Immune Prognostic Index for Patients Treated for Metastatic Non-Small Cell Lung Cancer.
Analyzing patient-reported outcome data when completion differs between arms in open-label trials: an application of principal stratification.
Machine-Learning and Stochastic Tumor Growth Models for Predicting Outcomes in Patients with Advanced Non-Small-Cell Lung Cancer.
Global Variation in Opioid Use in Prostate Cancer Trials.
Outcomes of Older Women with Hormone Receptor-Positive, Human Epidermal Growth Factor Receptor-Negative Metastatic Breast Cancer Treated with a CDK4/6 Inhibitor and an Aromatase Inhibitor: An FDA Pooled Analysis.
CDK4/6 inhibitor treatment for patients with hormone receptor-positive, HER2-negative, advanced or metastatic breast cancer: a US Food and Drug Administration pooled analysis.
2018
Survival of ethnic and racial minority patients with multiple myeloma treated with newer medications.
Patients with melanoma treated with an anti-PD-1 antibody beyond RECIST progression: a US Food and Drug Administration pooled analysis.
US Food and Drug Administration Pooled Analysis to Assess the Impact of Bone-Only Metastatic Breast Cancer on Clinical Trial Outcomes and Radiographic Assessments.
FDA analyses of survival in older adults with metastatic non-small cell lung cancer in controlled trials of PD-1/PD-L1 blocking antibodies.
Blinding and Patient-Reported Outcome Completion Rates in US Food and Drug Administration Cancer Trial Submissions, 2007-2017.
Evaluation of Overall Response Rate and Progression-Free Survival as Potential Surrogate Endpoints for Overall Survival in Immunotherapy Trials.
Exploration of a Novel Intermediate Response Endpoint in Immunotherapy Clinical Studies.
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