Building smarter early phase oncology programs for biotechs

From first-in-human to “go/no go,” explore strategies for navigating the complexities of early phase oncology development with confidence.

Early phase oncology trials, including Phase I and Phase I/II studies, represent the first opportunity to evaluate a new cancer therapy in patients. At this stage, the focus of these first-in-human studies is on establishing safety and tolerability, defining an appropriate dose, characterizing side effects, and beginning to assess early signs of clinical activity. For biotech developers entering this space, balancing the scientific, operational and regulatory demands of a trial — often with limited resources and experience — can be daunting. And when potentially life-saving treatments are on the line, every moment matters. The ability to make fast, accurate decisions is essential.

This is where choosing the right contract research organization (CRO) becomes consequential. In early phase oncology, timelines are tight, complexity is high and missteps are costly. A CRO with deep early phase expertise enables biotech sponsors to navigate risk, control costs and move forward with confidence — without sacrificing quality or scientific rigor. Engaging a CRO early in development, even amid competing priorities and funding constraints, ensures not only that the next milestone is met, but that each decision that follows rests on a strong foundation.

The PPD™ clinical research business of Thermo Fisher Scientific provides a comprehensive suite of services tailored for small to mid-sized biotech companies. We bring expertise and efficiency to every phase of oncology development — from pre-clinical research to global commercialization — so you can reach your next decision point with more confidence.

The interconnected challenges of early phase oncology development

Early phase oncology development brings a unique set of challenges, including dose escalation, patient recruitment, complex protocol designs, substantial data requirements, and supply chain and manufacturing considerations, which rarely exist in isolation. Instead, they compound one another — meaning a delay or misstep in one area can quickly cause a ripple effect across the entire program. Successfully navigating early phase oncology clinical trials requires coordination, foresight and an integrated approach to development.

The foundation of decision-making and value

Dose escalation strategies must balance patient safety with the need to reach biologically meaningful doses quickly. For biotech developers, dose escalation is also a primary driver of value creation and risk reduction. The data generated in Phase I inform “go/no-go” decisions, shape regulatory strategy, and underpin discussions with investors and potential partners.

As regulators increasingly expect model-informed dose selection supported by pharmacokinetics (PK), pharmacodynamics (PD), and biomarker data, dose escalation is no longer based on trial-and-error alone. Getting it right determines whether a program advances with clarity and momentum — or stalls due to uncertainty, late-emerging toxicity or inconclusive signals.

Avoiding the patient recruitment bottleneck

Patient recruitment often becomes a critical bottleneck, especially as eligibility criteria narrow and competition for patients intensifies. Biotech sponsors must identify the right patients at the right time — typically within small, advanced-disease populations — while overcoming hesitancy around first-in-human studies and the logistical demands of complex trial protocols.

Delays in recruitment can postpone dose escalation, downstream milestones, and funding opportunities, making enrollment challenges not just operational obstacles but considerable risks that can determine a program’s success or failure.

Determining necessary risk

Complex protocol designs increase operational, regulatory and data management demands at a time when resources are limited. While these designs are necessary to answer critical questions around safety, dose optimization, and biomarkers, features such as multiple cohorts, adaptive elements, intensive sampling, and biomarker-driven eligibility can slow site activation and complicate execution.

Protocol complexity also increases the risk of amendments, which can add cost, lengthen timelines, and disrupt site and patient engagement. Without careful planning and experienced oversight, complexity can hinder advancement rather than accelerate it.

Deriving insights from data

Early phase oncology trials generate large volumes of data across safety, PK/PD, imaging and biomarkers. While this density of data is necessary, it can strain lean biotech teams and burden sites and patients when collection requirements are not clearly prioritized.

The real risk is not collecting too little data, but collecting too much without a clear strategy for integration and interpretation. Without timely access to decision-ready insights, biotech sponsors can become overwhelmed by information while still struggling to make confident dose escalation or “go/no-go” decisions.

Taking supply chain and manufacturing into consideration

Supply chain and manufacturing are critical enablers of early phase oncology trials, directly influencing study start-up, dose escalation, and the ability to adapt as data emerge. Biotech developers must ensure reliable drug supply, appropriate batch sizing, and sufficient release and stability data while managing risks associated with long lead times, limited flexibility, and reliance on single-source vendors or early-stage contract development and manufacturing organizations.

Manufacturing constraints can force conservative dose decisions, pauses in escalation or delays in expansion — regardless of clinical readiness. Decisions made early can also have downstream regulatory and scalability implications, making proactive planning essential to preserving program momentum.

Move forward with confidence

With so much on the line, the PPD clinical research business is built to enable biotechs to meet these challenges head-on. 

Deep expertise where it matters most

Our teams bring deep experience in safety management and dose escalation, optimizing trial duration and maintaining momentum during critical early decisions. We have extensive experience supporting innovative therapies and novel mechanisms — and understand how to translate cutting-edge science into executable clinical strategies.

Our experience with innovative trial designs is especially critical in early phase oncology, where efficiency and adaptability can significantly influence timelines and outcomes. From dose-optimization strategies to master protocol designs, our approach enables more efficient decision-making, supports earlier identification of clinical signals, and accelerates progression through key development milestones without compromising safety or quality.

Anchored by a trusted global site network and early phase oncology expertise, our patient recruitment strategies enable efficient site activation, enrollment and retention. Our precision medicine capabilities — including next-generation sequencing and biomarker-driven enrollment — empower sponsors to identify early clinical activity while minimizing unnecessary patient exposure.

With in-depth knowledge of local regulatory environments, strong relationships with investigators and key opinion leaders, and embedded proximity teams that efficiently activate sites, we are positioned to support smooth trial operations across regions. And our rigorous quality management systems ensure the delivery of high-quality, reliable data that stands up to regulatory and investor scrutiny.

Designed with emerging biotechs in mind, our flexible, right-sized pricing models combine efficient project management with operational excellence to help you stay on budget, reduce risk, and keep your program moving forward.

Integrated solutions for faster, smarter decisions

Biotech sponsors benefit from our experience with innovative trial designs and a robust technology stack — including AI-enabled tools — which enable them to manage data volume and complexity and support faster, better-informed decisions. Our Accelerator™ Drug Development solutions integrate clinical operations, data, biomarker, regulatory, and manufacturing considerations from day one, creating alignment across the development lifecycle.

When additional perspective is needed, our consulting services connect biotech sponsors with top investigators and experienced oncology experts who bring hands-on, real-world insight.

Our approach brings precision to navigating early phase complexity, helping to accelerate timelines while maintaining control over both quality and budgets. By entrusting our experienced teams to manage the operational details, biotech leaders gain more time to focus on strategy and day-to-day business priorities.

Transforming early uncertainty into confident next steps

Early phase oncology development is where uncertainty is highest — but it’s also where the right partnership matters most. With our comprehensive capabilities, unmatched expertise, and shared biotech mindset, we enable biotech developers to turn early phase clinical data into confident next steps — faster, smarter and with greater certainty.