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Tumor-agnostic baskets to N-of-1 platform trials and real-world data: Transforming precision oncology clinical trial
Elena Fountzilas1, Apostolia-Maria Tsimberidou2, Henry Hiep Vo2
1Department of Medical Oncology, St Luke's Clinic, Thessaloniki, Greece; European University Cyprus, German Oncology Center, Nicosia, Cyprus.
Abstract:
Choosing the right drug(s) for the right patient via advanced genomic sequencing and multi-omic interrogation is the sine qua non of precision cancer medicine. Traditional cancer clinical trial designs follow well-defined protocols to evaluate the efficacy of new therapies in patient groups, usually identified by their histology/tissue of origin of their malignancy. In contrast, precision medicine seeks to optimize benefit in individual patients, i.e., to define who benefits rather than determine whether the overall group benefits. Since cancer is a disease driven by molecular alterations, innovative trial designs, including biomarker-defined tumor-agnostic basket trials, are driving ground-breaking regulatory approvals and deployment of gene- and immune-targeted drugs. Molecular interrogation further reveals the disruptive reality that advanced cancers are extraordinarily complex and individually distinct. Therefore, optimized treatment often requires drug combinations and N-of-1 customization, addressed by a new generation of N-of-1 trials. Real-world data and structured master registry trials are also providing massive datasets that are further fueling a transformation in oncology. Finally, machine learning is facilitating rapid discovery, and it is plausible that high-throughput computing, in silico modeling, and 3-dimensional printing may be exploitable in the near future to discover and design customized drugs in real time.
Insights
Precision cancer medicine uses genomic sequencing to match patients with targeted therapies. Innovative trials and data analysis are revolutionizing cancer treatment for individual benefit.
Area of Science:
- Oncology
- Genomics
- Translational Medicine
Background:
- Precision cancer medicine relies on genomic sequencing and multi-omic interrogation to select optimal therapies.
- Traditional clinical trials evaluate therapies in patient groups based on histology, unlike precision medicine's focus on individual benefit.
- Advanced cancers are molecularly complex and heterogeneous, necessitating personalized treatment strategies.
Purpose of the Study:
- To highlight the shift from traditional clinical trials to innovative designs in precision cancer medicine.
- To discuss the role of advanced molecular profiling in identifying patient benefit.
- To explore the impact of new trial designs, real-world data, and emerging technologies on oncology.
Main Methods:
- Review of current trends in precision cancer medicine and clinical trial design.
- Discussion of biomarker-defined tumor-agnostic basket trials and N-of-1 trials.
- Exploration of the role of real-world data, master registry trials, and machine learning.
Main Results:
- Innovative trial designs like basket trials are enabling regulatory approvals for gene- and immune-targeted drugs.
- Molecular interrogation reveals cancer's complexity, driving the need for drug combinations and N-of-1 customization.
- Real-world data and machine learning are accelerating discovery and transforming oncology.
Conclusions:
- Precision cancer medicine is advancing through novel trial designs and molecular profiling.
- N-of-1 trials and real-world data are crucial for optimizing individualized cancer treatment.
- Future innovations may include real-time drug discovery and design using advanced computing and 3D printing.
