De-Risking Early Clinical Development

IQVIA is a global provider of clinical research services, healthcare data, advanced analytics, and technology solutions that help life sciences companies accelerate the development and delivery of innovative medicines. By combining deep scientific expertise with extensive healthcare data and AI-powered insights, IQVIA supports pharmaceutical and biotechnology companies across the drug development lifecycle—from discovery and clinical trials to regulatory approval and commercialization.

In this conversation with Contract Pharma, Elizabeth Allen, PhD., Vice President, Early Phase Center of Excellence, Raymond Cook, Global Head, Early Phase Center of Development, and Janet Rosasco, Senior Therapeutic Strategy Lead Director, highlight IQVIA’s capabilities in early clinical development. Specifically, they explore how IQVIA helps biotechs navigate uncertainty, what it means to be a successful strategic development partner, how the company ensures a seamless transition from early- to late-phase trials and more.

Contract Pharma: With increasing scientific complexity—such as precision medicine, rare diseases, and cell/gene therapies—what shifts is IQVIA seeing in early-phase design and execution requirements?

Elizabeth Allen: As scientific complexity increases, early-phase study designs have shifted from traditional safety- and PK-focused Phase I studies toward integrated, translational studies designed to generate accelerated go/no-go evidence. Early-phase studies increasingly incorporate adaptive and hybrid designs, enabling the use of emerging clinical data, seamless transitions from healthy volunteers to patients, biomarker-driven patient selection, quantitative PK/PD modelling, and early proof-of-concept objectives within a single protocol. This change in approach facilitates safer and better decision-making based on real-time data, avoids redundancy in scientific design, and can significantly accelerate early-phase programs where appropriate.

Early-phase rare disease studies require innovative approaches to overcome limited populations, while cell and gene therapies demand enhanced safety oversight, specialized operational infrastructure, manufacturing integration, and closer regulatory engagement. Successful early-phase execution now requires a coordinated multidisciplinary approach spanning clinical pharmacology, translational sciences and biomarker expertise, PK/PD modelling, regulatory strategy, and targeted early-phase patient recruitment strategies, with the overall goal of accelerating decision-making while maintaining scientific rigor and patient safety.

CP: Early-phase biotech sponsors face intense uncertainty and capital constraints, with success rates from Phase I to approval remaining low. How is IQVIA helping emerging biotechs de‑risk early clinical development and secure investor confidence in this environment?

Raymond Cook: Biotechnology funding has become increasingly selective, with investors requiring not only compelling science but also clear evidence of development feasibility, capital efficiency, and value creation. IQVIA helps emerging biotechs reduce early-stage development risk by combining strategic planning, innovative clinical trial methodologies, global operational capabilities, and data-driven decision support.

IQVIA works with sponsors to align Target Product Profiles, clinical development strategies, regulatory pathways, and commercial objectives early in development, helping ensure that limited capital is directed toward milestones that maximize asset value and investor appeal. Through scenario modeling, probability-of-success assessments, and development pathway optimization, sponsors can make more informed portfolio decisions and clearly communicate their strategy to investors.

To accelerate progression from First-in-Human studies to Proof-of-Concept, IQVIA leverages adaptive and hybrid study designs, model-informed drug development, biomarker strategies, and advanced analytics. These approaches enable earlier detection of safety, pharmacodynamic, and efficacy signals, supporting faster go/no-go decisions while reducing development risk and unnecessary expenditure.

IQVIA also helps sponsors establish meaningful value inflection points throughout development which include biomarker readouts, interim analyses, and operational milestones all of which provide investors with ongoing evidence of progress and execution. This is particularly important in today’s capital-constrained environment, where maintaining investor confidence between major clinical readouts is critical.

Supported by a global network of clinical pharmacology units, sites, laboratories, data sciences, and regulatory experts, IQVIA offers an integrated development model that can streamline execution, reduce operational complexity, and accelerate timelines. The result is a development strategy focused on generating high-quality evidence faster, enabling emerging biotechs to de-risk programs, preserve capital, and strengthen confidence among investors, boards, and potential partners.

CP: Sponsors are increasingly seeking CRO partners who serve as strategic co-developers rather than transactional vendors. How does IQVIA position itself as a long-term, integrated development partner rather than merely a service provider?

Cook: Sponsors want CRO partners who can offer integrated capabilities, scientific insight, and shared accountability across the full development lifecycle. IQVIA is committed to be a long-term development partner rather than a transactional service provider. At IQVIA we differentiate through an end-to-end, globally integrated model that spans early phase through commercialization, minimizing fragmentation and ensuring continuity of knowledge, teams, and strategy. By engaging early in clinical development planning, protocol design, and regulatory strategy, IQVIA operates as an extension of sponsor teams, helping shape asset strategy and not just execute studies.

A key differentiator for IQVIA is our unmatched data and analytics ecosystem, enabling more informed decisions across feasibility, patient recruitment, endpoint strategy, and real-world evidence generation. This is complemented by a technology-enabled delivery platform, providing real-time insights, increased efficiency, and scalable execution. In addition, IQVIA has continued to invest in bringing on previous regulators to help sponsors navigate complex regulatory decisions, ensuring that development programs are strategically designed to support successful submissions, approvals, and long-term asset value creation.

We also bring global scale with deep local expertise, allowing sponsors to optimize development strategies across geographies while balancing speed, cost, and regulatory acceptability. This is particularly critical in accelerating early phase to proof-of-concept transitions, where IQVIA integrates clinical pharmacology capabilities, site networks, and translational expertise to reduce timelines and development risk.

Finally, IQVIA supports flexible partnership and commercial models, aligning incentives with sponsor outcomes and enabling long-term program-level collaboration. Combined with deep therapeutic and scientific expertise, this positions IQVIA as a partner focused on maximizing asset value and probability of success, rather than delivering individual studies in isolation.

CP: How does IQVIA ensure seamless transition from early‑ to late‑phase trials—maintaining continuity, preserving institutional knowledge, and reducing handoff friction for sponsors?

Janet Rosasco: At IQVIA, we are committed to maintaining continuity and minimizing friction during handoffs between early and late-phase trials. Both our early development and late phase teams operate under the same SOPs and systems, ensuring consistency in trial management and preserving valuable institutional knowledge throughout the process. While there are specific considerations necessary for early development studies—such as retaining the flexibility required for early phase clinical pharmacology trials and ensuring rigorous subject safety and dose escalation practices—these are carefully balanced with our standardized approach.

As a compound transitions from Phase I to Phase II and beyond, our early development Project Leads play a pivotal role in sharing key information. This includes details about the compound, insights into study conduct, the client’s preferred communication methods, and important lessons learned during the trial. This proactive knowledge transfer is a cornerstone of our process and helps maintain continuity for sponsors.

Additionally, our early and late phase teams draw upon the same resources for critical functions such as Regulatory, Data Management, Biostatistics, Medical Monitoring, Pharmacovigilance, Records and Information Management, Supportive Services, and Finance. Whenever possible, we aim to assign the same experienced team members from Phase I studies through to Phase II and III, allowing sponsors to benefit from established relationships and team experience.

CP: How does IQVIA help sponsors navigate diverse regulatory environments while still accelerating early development? 

Rosasco: IQVIA combines global regulatory expertise, local market knowledge, integrated development teams, and data-driven decision making. We conduct operations in more than 100 countries providing sponsors with access to local regulatory specialists who understand regional requirements, agency expectations, and evolving guidance.

IQVIA’s Drug Development & Regulatory Services (DDRS) team supports submissions across the globe, using regional experts to optimize clinical development timelines. In addition, our global early development site network can leverage Central ECs/IRBs, and many sites can also support EC submissions directly. IQVIA’s healthcare data assets include information derived from over one billion non-identified patient records globally, enabling sponsors to make evidence-based decisions regarding.

All combined this enables sponsors to select the right countries, engage regulators proactively, execute complex early phase studies efficiently, and accelerate the path from First-in-Human to proof-of-concept while maintaining compliance, quality, and patient safety.

CP: With innovative trial designs becoming essential, how is IQVIA using adaptive and hybrid designs to improve safety, predictability, and speed in early-phase trials?

Allen: IQVIA increasingly uses adaptive and hybrid trial designs to replace traditional sequential Phase I studies with more integrated, data-driven approaches, which improves safety, accelerates learning, and supports better and faster development decisions. Rather than conducting separate studies for SAD, MAD, and patient cohorts, hybrid protocols can combine healthy volunteers and patients within a single study, creating a seamless transition from Phase I to Phase II while reducing operational delays and duplicate start-up activities.

Adaptive designs enable protocol elements to be modified in response to emerging data without compromising scientific validity. IQVIA routinely incorporates real-time safety, PK/PD, biomarker, and early efficacy data into dose-escalation decisions, allowing cohort sizes to be re-estimated, ineffective arms to be stopped, promising cohorts to be expanded, and sampling schedules to be refined. This approach improves participant safety, reduces uncertainty, and supports more informed go/no-go decisions earlier in development.

By utilizing model-informed techniques, e.g., PK/PD modelling, simulation, receptor occupancy assessments, biomarkers, and early QT analyses, we are able to optimize dose selection and forecast clinical outcomes. These quantitative tools help identify the right dose, exposure, and therapeutic response while minimizing unnecessary development risk.

For complex programs, including rare diseases and precision medicine therapies, IQVIA designs customized hybrid studies that integrate dose-finding, biomarker-enrichment strategies, patient onboarding, and seamless phase transitions within a single protocol. Basket and adaptive cohort-expansion approaches allow resources to be focused on the most promising populations and indications based on interim data.

Overall, IQVIA’s adaptive and hybrid design strategy is intended to generate earlier proof-of-mechanism and proof-of-concept signals, improve patient safety through continuous data review, reduce development uncertainty, and accelerate advancement from first-in-human studies to Phase IIa and beyond.

CP: IQVIA recently expanded its drug discovery platform through the acquisition of Charles River’s early discovery assets. How will this integration enhance early clinical development for pharmaceutical partners?

Cook: At IQVIA, we’ve built an early phase model designed to remove those disconnects bringing together regulatory strategy, translational insight, and high-performing Phase 1 delivery into a single, integrated pathway from IND to first-in-human readout.

With the recent IQVIA acquisition of certain discovery services assets from Charles River Laboratories IQVIA is able to provide a true molecule to human offering. These assets include five sites specializing in a range of in vitro drug discovery services, including established laboratory based New Approach Methodologies (NAMs) and a small molecule AI platform, designed to accelerate discovery programs and support growing demand for non-animal research methods. Since their inception, these assets have enabled over 100 molecules to enter clinical trials and include several commercially approved drugs.

The acquisition complements IQVIA Laboratories’ existing drug discovery capabilities to create a fully integrated, end-to-end drug discovery platform spanning target identification, hit-to-lead, lead optimization and early safety assessment. Pharmaceutical and Biotech sponsors will benefit from the integration of IQVIA’s combined scientific, regulatory and clinical expertise from early-stage discovery research through to clinical development.