Oncology remains the most dynamic and fastest-growing therapeutic area in clinical research. Nearly 40% of all clinical trials initiated in recent years have targeted oncology, and the global oncology drug pipeline now exceeds 5,000 compounds under investigation[1]. According to GMI, the global oncology market was estimated at US $320.3 billion in 2024 and is expected to grow to US $866.1 billion by 2034 [2], reflecting not only the explosion of new therapies but also the enormous unmet need for patients worldwide.

Yet oncology trials are among the most resource-intensive, often requiring three times the investment of trials in other therapeutic areas[3]. Sponsors face prolonged timelines, heavy regulatory oversight, and complex enrollment challenges. Advances in targeted therapies, immunotherapies, cell and gene therapies, and tumor-agnostic approaches are transforming treatment paradigms, but they also intensify operational demands.

Emerging markets are increasingly critical for patient access, yet regional disparities in healthcare infrastructure and diagnostic capabilities remain. Adaptive and platform trial designs promise efficiency, but they require sophisticated operational frameworks and regulatory navigation.

Navigating this landscape demands more than traditional CRO bandwidth. It requires therapeutic insight, global intelligence, and an ability to align science with execution. At Confidence Pharmaceutical Research, our MD-led feasibility assessments, accelerated first-patient-in strategies, and extensive oncology trial experience position us to meet the demands of this new era.

The Innovation Wave in Oncology

Targeted Therapies

Recent advances in targeted therapies in oncology over the last five years have significantly transformed cancer treatment by enabling more precise and personalized approaches [4]. Innovations include the development of small molecule inhibitors targeting previously “undruggable” mutations such as KRASG12C, antibody-drug conjugates that improve selective delivery of cytotoxic agents to cancer cells, and expanded applications of HER2-targeted therapies beyond breast cancer.

Despite these successes, significant limitations remain. [5] Targeted therapies face challenges such as intrinsic and acquired resistance often driven by tumor heterogeneity and evolutionary adaptation. Delivery issues persist, including limited tumor penetration for large molecules like monoclonal antibodies and potential systemic toxicity from extended half-lives. 

Immunotherapies and Checkpoint Inhibitors

Since the approval of the first checkpoint inhibitor in 2011, immunotherapies have reshaped oncology. Anti-PD-1 and PD-L1 agents such as pembrolizumab and nivolumab are now standard of care across multiple tumor types, while newer agents target TIGIT, LAG-3, and other immune pathways [6]. Globally, over 3,000 active immunotherapy trials are underway, many exploring novel combinations [7].

The sheer scale of the immunotherapy pipeline has created a competitive environment where differentiation depends on trial design and patient access. Trials are increasingly testing combinations of checkpoint inhibitors with chemotherapy, targeted agents, or radiation, raising complexity in both safety monitoring and enrollment strategies.

Cell and Gene Therapies

Cell and gene therapies represent the cutting edge of oncology. CAR-T therapies have demonstrated remarkable outcomes in hematologic malignancies, achieving complete remission in heavily pretreated patients[8]. Next-generation CAR-Ts are being engineered to target solid tumors, while T-cell receptor (TCR) therapies and personalized neoantigen vaccines push personalization even further.

However, these approaches create operational hurdles. Trials often require complex logistics, such as apheresis, cell modification, and reinfusion—all on tight timelines. They also demand rigorous monitoring for unique toxicities like cytokine release syndrome. For CROs, managing these studies requires specialized expertise in both trial coordination and safety oversight.

Tumor-Agnostic Approaches

One of the most disruptive trends is the approval of tumor-agnostic therapies. Pembrolizumab’s 2017 approval for MSI-H/dMMR solid tumors was the first time a cancer therapy was approved based on biomarker status rather than tumor origin [9]. TRK inhibitors such as larotrectinib and entrectinib followed, establishing biomarker-driven approvals as a regulatory reality.

Immunotherapy has also been integrated with targeted strategies, exemplified by CAR-T cell therapies that genetically engineer T cells to attack cancer, checkpoint inhibitors that release immune suppression, and emerging personalized cancer vaccines. These advances have improved outcomes in diverse cancers like lung, ovarian, prostate, and hematological malignancies, enabling more effective and less toxic treatments than conventional chemotherapy or radiation.

Future directions focus on combination therapies, novel drug delivery systems, gene editing technologies, and artificial intelligence for clinical decision support to overcome these challenges and optimize therapeutic efficacy.

While these approaches expand treatment possibilities, they complicate trial feasibility. Eligible patients are scattered across tumor types, sites, and geographies, making recruitment highly fragmented. Trials must be designed to capture diverse populations while still maintaining statistical and regulatory rigor.

Adaptive and Platform Trials

Adaptive designs are gaining traction as a way to accelerate oncology development. Platform trials such as I-SPY2 in breast cancer and Lung-MAP in NSCLC allow multiple investigational agents to be tested under a single master protocol [10]. These designs reduce redundancy and provide flexibility to add or drop treatment arms in real time.

While the benefits are clear, the operational demands are substantial. Adaptive trials require seamless data flow, advanced statistical oversight, and alignment across regulators, sponsors, and investigators. Without a strong operational framework, the efficiencies of adaptive designs risk being lost to delays and protocol amendments.

Key Operational Challenges

Enrollment in Biomarker-Defined Subgroups

Enrolling patients in biomarker-driven oncology trials can be difficult. Screen-failure rates are often high—up to 70% in PD-L1–selected studies [11]—and issues such as tissue quality or testing timelines can make it even harder to find eligible patients. In addition, biomarker prevalence can vary widely across geographies and patient populations, making country and site selection a critical determinant of success.

At Confidence, feasibility is not a paper exercise. Our assessments incorporate a competitive environment, standards of care, diagnostic capacity, referral patterns, and patient flows to ensure selected sites can realistically deliver eligible patients. By aligning enrollment planning with biomarker realities, sponsors avoid delays that stem from overestimating site potential.

Regional Disparities in Patient Access

The global burden of cancer is shifting, with over 60% of new cases now occurring in low- and middle-income countries [12]. Yet most oncology trials remain concentrated in North America and Western Europe. This imbalance not only slows enrollment but also limits the generalizability of trial results.

Emerging regions like Eastern Europe, Latin America, and parts of Asia-Pacific are becoming increasingly vital for recruitment. However, infrastructure for advanced diagnostics may be inconsistent, regulatory timelines vary widely, and investigator experience with complex protocols is uneven.

Confidence integrates regional intelligence into feasibility, mapping healthcare infrastructure, laboratory capacity, and patient referral networks before finalizing site selections. This ensures sponsors expand into geographies that can deliver patients without compromising trial quality. Our MDs often identify overlooked regions where patient populations are robust but underutilized, unlocking recruitment potential that traditional feasibility metrics miss.

Complexity of Adaptive and Platform Trial Designs

Adaptive and platform trials offer the promise of efficiency but demand a higher level of operational maturity. Mid-course adaptations—such as adding arms, re-estimating sample size, or stratifying by biomarker—require pre-specified rules, regulatory engagement, and robust statistical frameworks [13].

For CROs, this means managing real-time data integration, coordinating across multiple stakeholders, and ensuring investigators are trained in adaptive protocols. Without these capabilities, adaptive trials risk generating more complexity than clarity.

Confidence has operational models tailored to adaptive and platform designs. We run periodic operational reviews to track enrollment velocity, screen-fail patterns, data timeliness, and site productivity; we stand up cross-functional site-engagement task forces to clear bottlenecks; and we maintain amendment-readiness and rapid-response monitoring so that, once the DMC/SDC issues an adaptation decision (e.g., arm add/drop, allocation change), sites, documents, and systems are updated quickly and compliantly—without slipping timelines.

The Confidence Perspective

At Confidence Pharmaceutical Research, oncology is not just a therapeutic area—it is a proving ground for our philosophy of executional clarity. The intersection of biomarkers, global patient access, and adaptive designs demands a CRO partner who can think strategically while acting decisively.

• Feasibility first
  Our MD-led feasibility assessments go beyond counting sites. We evaluate diagnostic pathways, molecular testing turnaround, and referral networks. This ensures sponsors select sites that can actually deliver eligible patients, not just those with historical enrollment volume.
• Accelerated FPI
  Delays at first-patient-in cascade into missed milestones downstream. By aligning activation timelines with actual patient availability, we help sponsors achieve FPI faster without sacrificing quality. Our approach reduces wasted time at sites unlikely to enroll and concentrates resources where patients are most likely to be found.
• Global oncology expertise
  From early-phase studies in specialized oncology units to large-scale registrational trials, we bring the operational depth needed to manage biomarker-driven and adaptive designs. Our experience spans North America, Europe, Asia, and Latin America, allowing sponsors to tap into diverse patient pools with confidence.

Conclusion

The oncology pipeline is expanding at unprecedented speed, propelled by immunotherapies, cell and gene therapies, and tumor-agnostic approaches. Yet innovation alone is not enough—operational realities determine whether new therapies reach patients on time.

Enrollment challenges, global disparities, and the complexity of adaptive designs create risks that can derail even the most promising programs. To overcome these barriers, sponsors need CRO partners who combine therapeutic insight with operational agility.

At Confidence, we bring MD-led feasibility, accelerated start-up strategies, and deep oncology trial experience to every program. Beyond biomarkers, we see oncology as the place where science and strategy converge—and where execution defines success.

References

1. ClinicalTrials.gov. Trends in Oncology Clinical Trials. Accessed 2025.
2. Global Market Insights. Oncology Market Size, Share, and Forecast, 2024–2034 [Internet]. Delaware (US): Global Market Insights; 2024
3. Tufts Center for the Study of Drug Development (CSDD). Cost of Clinical Trials Analysis. 2022.
4. Desai A, et al. Top advances of the year: Precision oncology. Cancer. 2023
5. Lui B, et al. Exploring treatment options in cancer: tumor treatment strategies. Bomedicines. 2025
6. Tang J, et al. The landscape of immune checkpoint inhibitor trials. Nature Reviews Drug Discovery. 2021.
7. Haslam A, Prasad V. Estimation of the number of checkpoint inhibitor trials. JAMA Oncology. 2019.
8. June CH, Sadelain M. Chimeric antigen receptor therapy. New England Journal of Medicine. 2018.
9. U.S. FDA. FDA approves first cancer treatment for any solid tumor with a specific genetic feature. 2017.
10. Barker AD, et al. I-SPY 2: an adaptive breast cancer trial. Clinical Pharmacology & Therapeutics. 2009.
11. Hirsch FR, et al. PD-L1 immunohistochemistry assays for lung cancer. Lancet Oncology. 2017.
12. Sung H, et al. Global cancer statistics 2020. CA: A Cancer Journal for Clinicians. 2021.
13. Ventz S, et al. Statistical considerations for adaptive oncology trials. Journal of Clinical Oncology. 2019.