Related Experiment Video
Updated: Nov 2, 2025
Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 18, 2013
Food and Drug Administration approvals in phase 3 Cancer clinical trials
Joseph Abi Jaoude1, Ramez Kouzy1, Marc Ghabach1
1The University of Texas MD Anderson Cancer Center, 1515 Holcombe Blvd Unit 1422, Houston, TX, 77030, USA.
Background:
Phase 3 oncologic randomized clinical trials (RCTs) can lead to Food and Drug Administration (FDA) approvals. In this study, we aim to identify trial-related factors associated with trials leading to subsequent FDA drug approvals.
Methods:
We performed a database query through the ClinicalTrials.gov registry to search for oncologic phase 3 RCTs on February 2020. We screened all trials for therapeutic, cancer-specific, phase 3, randomized, multi-arm trials. We then identified whether a trial was used for subsequent FDA drug approval through screening of FDA approval announcements.
Results:
In total, 790 trials were included in our study, with 225 trials (28.4%) generating data that were subsequently used for FDA approvals. Of the 225 FDA approvals identified, 65 (28.9%) were based on trials assessing overall survival (OS) as a primary endpoint (PEP), two (0.9%) were based on trials with a quality of life (QoL) PEP, and 158 approvals (70.2%) were based on trials with other PEP (P = 0.01). FDA approvals were more common among industry funded-trials (219, 97.3%; P < 0.001), and less common among trials sponsored by national cooperative groups (21, 9.3%; P < 0.001). Finally, increased pre-hoc power and meeting patients' accrual target were associated with FDA approvals (P < 0.001).
Conclusions:
The majority of FDA approvals are based on data generated from trials analyzing surrogate primary endpoints and trials receiving industry funding. Additional studies are required to understand the complexity of FDA approvals.
Insights
Most Food and Drug Administration (FDA) drug approvals stem from industry-funded trials using surrogate endpoints. Meeting patient accrual targets and having high statistical power are key factors for successful oncology trial outcomes.
Area of Science:
- Oncology
- Clinical Trials
- Drug Development
Background:
- Phase 3 randomized clinical trials (RCTs) are crucial for Food and Drug Administration (FDA) drug approvals in oncology.
- Identifying factors associated with successful FDA approvals is vital for optimizing trial design and resource allocation.
Purpose of the Study:
- To identify trial-related factors associated with oncologic Phase 3 RCTs that lead to subsequent FDA drug approvals.
Main Methods:
- A systematic search of ClinicalTrials.gov for oncologic Phase 3 RCTs was conducted in February 2020.
- Trials were screened for therapeutic, cancer-specific, phase 3, randomized, and multi-arm characteristics.
- FDA approval announcements were screened to determine if trials contributed to drug approvals.
Main Results:
- Out of 790 trials, 225 (28.4%) generated data for FDA approvals.
- Most approvals (70.2%) used surrogate primary endpoints, while overall survival (OS) was primary in 28.9% and quality of life (QoL) in 0.9%.
- Industry-funded trials (97.3%) were significantly more likely to lead to approvals compared to national cooperative group-sponsored trials (9.3%). Increased statistical power and meeting patient accrual targets were also associated with approvals.
Conclusions:
- The majority of FDA approvals rely on data from trials using surrogate endpoints and industry funding.
- Further research is needed to fully understand the complex factors influencing FDA approvals in oncology.
More Related Videos
07:29Intramucosal Inoculation of Squamous Cell Carcinoma Cells in Mice for Tumor Immune Profiling and Treatment Response Assessment
Published on: April 22, 2019
09:20Preclinical Assessment of the Bioactivity of the Anticancer Coumarin OT48 by Spheroids, Colony Formation Assays, and Zebrafish Xenografts
Published on: June 26, 2018
Related Concept Videos
01:11Clinical Trials: Overview
01:16Clinical Trials
There are four phases in a clinical trial. A phase one...
01:26Drug Administration and Therapy Phases: Overview
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
01:28Preclinical Development: Overview
01:26FDA Approved Drugs: Changes to Approved Drugs
01:25Drug Regulation