Cancer drugs form one of the most dynamic segments of pharmaceuticals, powered by rising incidence, ageing populations, deeper cancer biology, better diagnostics, and the need for more effective treatments. The complexity of cancer drug development is shifting from broad chemotherapy regimens to biomarker-driven, personalized, and combination and targeted therapies that better address drug resistance while improving outcomes. With that, cancer drugs are also among the most scrutinized compounds. Analysis of recent pipelines shows that only about one in ten oncology drugs entering development eventually secures approval from the US Food and Drug Administration. At the same time, the majority of new and/or novel oncology drugs remain in a state of limbo or in protracted clinical-phase development because further evidence is needed to support its clinical safety and efficacy, or the compound fails to meet its planned endpoint/outcome requirements, resulting in it never reaching the market.
Meanwhile, the scope of an oncology trial has expanded. Sponsors are no longer dealing with conventional chemotherapy. Access and the cost to develop an oncology drug has gone up due to an increase in access and the use of branded and novel medications. The pipeline spans everything from targeted therapy and antibody drug conjugates (ADC) to cancer vaccines, immuno-oncology, and the use of new and novel combinations across both solid tumors and hematological malignancies. Also, as the field becomes more crowded and more specialized, the demand for basic science researchers and CROs with their deep subject matter expertise in medical affairs, regulatory strategy, clinical development, and operational execution continue to grow.
The commercial stakes are high. The oncology market is forecast to grow at a compound annual growth rate of 11.5% from 2024 to 2030, reaching $412bn by the end of the decade.[1] Whether these projections come to fruition or not depends on how well the evidence generation (i.e., safety and efficacy) addresses key questions at each major inflection point of the drugs stages of development.
As of December 2025, more than three-quarters of the oncology trials recorded in GlobalData clinical trials database sit between first-inhuman (FIH)/phase I and phase II. And while only 10% of oncology drugs secure FDA approval, the majority of oncology studies that have been suspended, terminated or withdrawn over the previous two years are concentrated in these early-phases (e.g., FIH/phase I and phase II) of development. Therefore, optimizing the targeted patient population (TPP), as well as a well-defined trial strategy and design in these early and formative phases of drug development is critical.
Early-phase oncology trials must tame biological complexity, satisfy demanding regulators, stand out in a complex and competitive field and show promise for investors. Generic designs cannot survive such pressure; new oncology drugs need to have a unique selling point from their trial’s first day.
Why early trials matter
Early-phase oncology clinical trials matter because they serve as the foundation for all future drug development decisions, from identifying initial signs of clinical effectiveness, gathering valuable data on how the drug will behave in the patient (or intended treatment population), to determining new therapies where none exist, to and whether a potential new drug and the company who owns receives investor funding. Therefore, quality of early drug development choices – study design, dose, schedule, targeted patient population, endpoints – determines which programs have the best chance of progressing. What are the key levers sponsors can pull, both before a trial is underway, as it ramps up, and through to study completion?
First, are safety protocols. Early-phase trials in cancer are designed to identify a safe dose, determine how a potential new agent should be given, and to observe how the cancer agent affects the body. The patients who usually enrol in early-phase trials are generally gravely ill, heavily pre-treated and often dealing with complex physiologic and immune conditions and side effects from previous regimens. Investigators must pick apart symptoms stemming from the underlying malignancy and earlier therapies, while scrutinizing every new signal that might be linked to the investigational agent. Additionally, the oncology safety landscape is evolving rapidly. For modern targeted and biological therapies, higher doses do not always translate into better tumour control. They may however result in chronic side-effects that wear patients down over months or years, pushing them to stop treatment even when the medicine is working. So, it becomes critical to have strict protocols in place to educate and reassure patients about relevant risks, while communicating the progress of an incipient trial.
Next, trial design. Early-phase trials need to answer precise questions and generate incremental data that can be relayed to regulators, physicians, and patients to demonstrate value. These early trials offer the opportunity to test the drug’s feasibility, identify safety signals or suboptimal dosing, uncover risks, and inform go/no-go decisions. Also, effective operational design and planning at the start helps plan for or avoid delays, contributes to the generation of high-quality data, supports regulatory filings, and improves interactions with agencies for favorable decisions. For example, in studies that involve antibody-drug conjugates (ADC), dose-escalation needs to reflect both how the antibody behaving and what its toxic payload are doing to the patient’s body. Agencies such as the FDA, EMA and China’s National Medical Products Administration offer an array of accelerated pathways for these promising oncology assets. But in doing so, these agencies raise the bar on evidence quality; sponsors are expected to track drug levels and biomarkers for longer periods of time, sometimes many years. So, design choices can reduce waste as well as minimize risk. Additionally, model-based methods can cut the number of patients exposed to sub-therapeutic doses and speed completion of phase I, while still protecting safety. Such designs can increase the amount of usable data and shorten timelines compared with traditional approaches.
For smaller cohorts in early-phase oncology, every data point counts. Patient recruitment and retention are vital considerations for success. These smaller cohorts are essential because they offer the ability to maximize patient safety while allowing for rapid, flexible adaptation of drug dosage based on near real-time data, all in an effort to minimize the number of patients exposed to a toxic or subtherapeutic dose. Also, thoughtful informed-consent documents, supplemented with brochures, videos or trial websites, help inform patients and families of what is being asked of them. Finally, smaller cohorts can improve the operational success of a trial by having a manageable number of patients and their visit schedules as closely as possible with standard care reduces, to improve patient compliance and reduce extra trips to hospital.
Linked to this is site and investigator selection. For the first dose-escalation cohorts it may make sense to limit the number of centers to those that can open quickly, recruit consistently and handle complex safety monitoring required of early-phase oncology trials. For example, in early-phase trials, patient interaction with the investigator (physician) is frequent to manage safety risk and provide immediate monitoring of the patient. If there is a safety signal, the investigator can move quickly, make incremental adjustments to pinpoint the exact dosage where the balance between safety and efficacy is ideal.
Behind the scenes, robust data management and analytics tie these elements together. Early phase oncology studies generate complex datasets on the behavior of therapeutic agent, alongside reams of diagnostic, laboratory and safety information. Early-phase drug development programs benefit when high quality data provides near real time views of emerging dose response patterns and safety trends. In doing high quality data can inform decision-making for timely adjustments, while keeping trials consistent with regulatory expectations even when adversity strikes.
Many levers, little time – what should sponsors do?
Translating this blueprint into an actionable and executable plan is demanding. Many biopharma innovators often lack the internal bandwidth and subject matter expertise (SME) to combine cutting edge trial design, global site networks, regulatory knowhow and continuous safety oversight to adequately reduce risk and meet development timelines in this demanding environment. Specialist CROs can help address these problems.
These highly capable CROs boast deep therapeutic knowledge across the oncology world, including a deep understanding of the complexities navigating these newer modalities such as immuno-oncology. They also offer credible early phase capabilities. For example Caidya, one such CRO, supports sponsors from pre-NDA planning and clinical development plan (CDP) design and planning, to FIH/Phase 1 and 2 studies, through to late-Phase 3 programs and commercial stage clinical pharmacology. Its early-phase services include preparing IND documentation, supporting dose escalation and expansion study design, performing pharmacokinetic analysis and writing the final reports that underpin submissions. These are the basic clinical phase trial checkboxes that all leading CROs must be able to tick.
Also, the operational strength and expertise of top CROs should also shine through. Again, Caidya is the exemplar; 97% of the company’s project managers have experience in oncology clinical research. Caidya also reports site activation-to-study close times in oncology that are more than 60% faster than industry averages, and it operates in more than 50 countries, including extensive coverage in North America, Europe, and the Asia-Pacific region, including deep coverage in China.
What can set world class CROs apart is their investment in less obvious levers of early development. These can make or break a trial. Feasibility specialists help sponsors identify high-performing sites and understand local patient pathways. Clinical pharmacology teams explore exposure-risk balance and support dose selection for biologics licence applications. Medical monitoring groups provide 24/7 coverage. Patient-support services – travel assistance, local laboratory options and flexible visit schedules – help retain participants however long and demanding a study proves to be.
Crucially, it is this mix of design expertise, operational discipline and therapeutic depth, at the absolute outset of a trial, that sets the leading CROs apart. Can you afford to miss out on their expert support? To learn more about the full range of what CROs like Caidya can offer, and why rolling this out right at the beginning of an oncology trial is essential, download the whitepaper on this page.
Caidya is the trade name of dMedClinical Co. Ltd. and its holdings. Clinipace, Inc. is one company in the Caidya group of companies. Clinipace, Inc. is owned, operated, managed, and controlled by dMedClinical Co. Ltd., a privately held company, whose investors include entities located in the People’s Republic of China (PRC), and which may be subject to PRC laws and regulations that differ from those of the United States.
[1] GlobalData: Analyst Briefing: The Oncology Casino: High Stakes, Few
Winners, January 2025
