Related Experiment Video
Updated: Dec 9, 2025
Dynamic Digital Biomarkers of Motor and Cognitive Function in Parkinson's Disease
Published on: July 24, 2019
Clinical trial design: Past, present, and future in the context of big data and precision medicine
1Division of Hematology/Oncology, Knight Cancer Institute, Oregon Health and Science University, Portland, Oregon.
Abstract:
Clinical trials are fundamental for advances in cancer treatment. The traditional framework of phase 1 to 3 trials is designed for incremental advances between regimens. However, our ability to understand and treat cancer has evolved with the increase in drugs targeting an expanding array of therapeutic targets, the development of progressively comprehensive data sets, and emerging computational analytics, all of which are reshaping our treatment strategies. A more robust linkage between drugs and underlying cancer biology is blurring historical lines that define trials on the basis of cancer type. The complexity of the molecular basis of cancer, coupled with manifold variations in clinical status, is driving the individually tailored use of combinations of precision targeted drugs. This approach is spawning a new era of clinical trial types. Although most care is delivered in a community setting, large centers support real-time multi-omic analytics and their integrated interpretation by using machine learning in the context of real-world data sets. Coupling the analytic capabilities of large centers to the tailored delivery of therapy in the community is forging a paradigm that is optimizing service for patients. Understanding the importance of these evolving trends across the health care spectrum will affect our treatment of cancer in the future and is the focus of this review.
Insights
Cancer treatment advances are shifting from traditional trials to precision medicine. New clinical trial designs integrate multi-omic data and machine learning for tailored cancer therapies.
Area of Science:
- Oncology
- Translational Medicine
- Bioinformatics
Background:
- Traditional clinical trials (Phase 1-3) support incremental advances in cancer treatment regimens.
- Evolving cancer research capabilities include targeted therapies, comprehensive datasets, and computational analytics, reshaping treatment strategies.
- The molecular complexity of cancer and patient variability necessitate individually tailored combination therapies.
Purpose of the Study:
- To review the evolving landscape of cancer clinical trials.
- To highlight the paradigm shift towards precision medicine and novel trial designs.
- To discuss the integration of multi-omic data and machine learning in cancer research.
Main Methods:
- Review of current trends in cancer clinical trial design.
- Analysis of the impact of molecular profiling and big data in oncology.
- Exploration of machine learning applications in real-world data analysis for cancer treatment.
Main Results:
- The traditional cancer trial framework is being redefined by advances in targeted therapies and molecular biology.
- Precision medicine, utilizing tailored drug combinations, is emerging as a new standard.
- Integration of large-scale data analytics, including machine learning, enhances treatment optimization.
Conclusions:
- The future of cancer treatment lies in adaptive clinical trial designs that leverage molecular insights and real-world data.
- A collaborative model between specialized centers and community settings is crucial for delivering optimized, personalized cancer care.
- Understanding these evolving trends is essential for advancing cancer therapy.
Related Concept Videos
01:16Clinical Trials
There are four phases in a clinical trial. A phase one...
01:11Clinical Trials: Overview
01:23Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
01:15Study Design in Statistics
Does aspirin reduce the risk of heart attacks? Is one brand of fertilizer more effective at growing roses than another? Is fatigue as dangerous to a driver as the influence of alcohol? Questions like these are answered using randomized experiments with proper...
02:50Combination Therapies and Personalized Medicine
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
01:20Study Designs in Epidemiology
Observational studies are those where the researcher does not intervene but rather observes natural variations. They include cross-sectional, cohort, and...