Conducting early phase oncology clinical trials: key considerations
Early phase oncology trials are the first stage in testing new cancer treatments. This usually encompasses phase I and phase II of the three main phases of clinical trials, where patients are amongst the first to test a new treatment.
Early phase oncology trials generally test gradually increased dosage levels of an experimental drug. The toxicity and overall safety profile, pharmacokinetics, and preliminary evidence of antitumor efficacy, including biomarkers, are then correlated to the different dose administered. The ultimate purpose of the studies is to establish the dosage level that will be administered in Phase 3 registration trials to support marketing authorization (‘approval’) by regulatory authorities.
As our understanding of cancer has progressed over the last decades, the methodology of early-phase oncology trials has also become much more sophisticated in recent years.
As a result, modern approaches to early phase oncology trials take into account , in the new fields of targeted therapy, immunotherapy and cell therapy, areas that were less considered in the past, such as proof-of-concept definition, optimization of timelines, early KOL involvement, and competitive asset positioning.
Conducting early phase oncology studies: webinar
This one-hour webinar focuses on conducting early-phase Oncology studies. It looks at strategic considerations for the molecule, including a brief review of the dynamic research landscape of oncology drug development, patient recruitment and product development aspects. View more information about our full range of oncology CRO services.
The Conducting Early Phase Oncology Studies webinar includes:
- What is on the horizon in oncology drug development
- Clinical development strategy and tactics
- Clinical design and consideration:
- Early vs. late phase
- Regulatory considerations
- Clinical aspects – cure or control
- Patient centricity – practical considerations
- Trial design – early phase 3+3, Rolling 6 and other designs
- Protocol development – clear unambiguous protocols, sense checking with KOLs and subject matter experts. The importance of understanding how to ‘operationalize’ the protocol with thorough feasibility
- Proof of concept – how do you confirm for MAA?
Explore our related webinar: State of the Art in Early Phase Oncology Trial Design.
The purpose of early-phase oncology trials
Early-phase oncology trials are conducted to find out how safe and effective a treatment is. Early phases of oncology studies help researchers identify:
- Optimal dosages of the treatment
- How often it should be administered
- Side effects and toxicity
- Effects on the cancer
- Pharmacokinetics – how the body absorbs, transports and eliminates the treatment
- Pharmacodynamics – the effect of the treatment on key biological processes (modulation of disease-relevant signalling pathways, receptor occupancy, etc).
Data from early-phase oncology research can be used to inform how the rest of the trial should progress, particularly in adaptive oncology trials, where real-time data is used to modify the study as it progresses.
The evolution of early-phase oncology trials
Cancer is now commonly understood as many types and molecular subtypes of disease. It is an increasingly segmented therapeutic area, requiring complex, agile study designs to meet persistent unmet medical needs.
Operating beyond the traditional scope of general toxicity, safety and efficacy, early-phase cancer trials require more advanced and tailored methods of research, namely investigating molecular, genomic and biomarker-driven principles. Agile approaches to such translational medicine can make oncology clinical trials different from other therapeutic areas.
Early phases of clinical research are a challenging step in the clinical development of any drug. In rare disease and oncology, there can be additional challenges in patient recruitment due to the small size of molecularly-defined population subsets.
The sample size can become even smaller when the study requires patients with matching specific genomic types of cancer, which is often one of the key issues surrounding the development of new chemotherapy drugs.
As a result, it is crucial to consider the role of the patient in early phase oncology trials, integrating effective patient engagement strategies into the study design. Doing so can help avoid the risk of dropout and reduced sample sizes during later stages.
Regulatory and ethical considerations
Studies that evaluate patient understanding of their involvement in clinical research find that 30% of patients do not understand that they may not directly benefit from taking part in a given clinical trial. This highlights the ethical need to ensure that patients are fully informed about the potential risks and benefits of taking part in clinical research.
Not only does this require regulatory procedures and documentation, there is also a responsibility for researchers to ensure that the patient is fully informed, and given the opportunity to ask questions about the trial.
This is particularly important in oncology research as many patients with advanced cancer may consider a clinical trial as their only remaining treatment option.
At Simbec-Orion, we take an agile approach to complex clinical studies, with full CRO services to support a range of clinical development needs. Our expertise includes clinical pharmacology and trial management services in oncology and rare disease therapeutic areas.
Get in touch to find out how we can help.