Deciphering Early Signals That Shape Future Therapies

In today’s high-stakes, fast-paced environment for drug development, actions taken in the early stages of research and development (R&D) are shaping more than just clinical pathways. These decisions are helping define the future of drug access, affordability, and market alignment. The planning signals emerging from biopharmaceutical companies carry meaningful implications for a wide range of health care stakeholders, including payers, providers, and policymakers.

Results from a recent industry survey indicate a significant transformation in how companies are approaching early development now and what needs to be prioritized for future success. From the widespread adoption of target product profiles (TPPs) to the use of advanced technology and external partners, developers are taking a more structured and strategic approach to decision-making in phase I and earlier.

Bridging the Gap Between Ambition and Execution

As pipelines expand and therapies become more specialized, early-stage planning has become a crucial step in clinical trials. According to McKinsey, the number of distinct drugs in development nearly doubled between 2012 and 2022, driven by advances in novel modalities and deeper understanding of disease biology. This added complexity makes strategic planning not just important, but essential.

Nearly 83% of drug developers surveyed currently use TPPs to guide development efforts and align internal teams around a shared vision for clinical, regulatory, and commercial success. However, only 17% of respondents reported exploring multiple TTPs per asset.

While this seems low, there is a direct impact further down the line for payers, because one fixed trajectory rarely meets the diverse and evolving expectations of the health care system. A drug may gain regulatory approval but fall short commercially if developers fail to consider variables such as clinical differentiation, population targeting, or real-world applicability. Exploring multiple TPPs—including both minimally viable and aspirational profiles—and accounting for changes in standards of care enable teams to model a broader range of scenarios, adapt to new data, and generate the type of evidence that supports strategic positioning, investment decisions, and long-term commercial success.

Evidence Planning is Access Planning

Data from the survey suggests that most TPPs are tested primarily through regulatory reviews and feasibility studies. While this approach helps establish basic approvability and operational viability, it falls short of addressing market access potential. Developers increasingly recognize the need to evaluate expected clinical and commercial value, not just whether a product meets regulatory thresholds.

In a 2025 report, McKinsey cited the need for end-to-end development strategies that begin at first-in-human trials. The research emphasizes that early integration of cross-functional insights—spanning clinical, regulatory, and commercial teams—can streamline development and increase the likelihood of commercial success. This report also shows how proactive scenario planning and internal alignment reduce trial amendments and associated delays.

Smaller Companies, Bigger Stakes

While large pharmaceutical companies often focus on risk mitigation and cost management, smaller biotechnology and emerging pharmaceutical organizations prioritize commercial viability and technical feasibility from the outset. These companies rarely bring products to market themselves, but they must still demonstrate value to secure licensing deals, investment, or acquisition.

This emphasis is especially important as smaller companies are increasingly the originators of high-cost, high-impact therapies, particularly in oncology, neurology, and rare diseases. Their early planning decisions can determine whether these therapies launch with sufficient real-world data, health economic modeling, or indications that align with the unmet health needs of certain populations.

By understanding the unique challenges these firms face in planning and generating evidence, stakeholders across the health care system can better anticipate how emerging therapies may fit into future treatment landscapes.

External Collaboration: A New Pillar of Early Development Strategy

Strategic planning is no longer confined within internal R&D teams. Stemming from the same survey, above 64% of biopharmaceutical organizations are actively seeking external expertise, particularly in regulatory strategy, clinical subject-matter guidance, and TPP development. This shift reflects a broader recognition that early-stage decisions are more robust when informed by cross-disciplinary and cross-organizational perspectives.

These collaborations go well beyond operational outsourcing. Developers are engaging academic researchers, health economists, modeling experts, and real-world evidence specialists to define and refine assumptions early. Their input helps teams explore alternative development scenarios, pressure-test go/no-go decisions. and better anticipate the clinical and commercial evidence that may be required down the line, including what stakeholders like regulators, clinicians, and payers may expect.

Advanced Technology: A Tool for Strategic Precision

Among all the changes underway in early development, the rise of artificial intelligence (AI) and advanced analytics may be the most transformative. Across both executive and operational roles, survey respondents identified AI as the top accelerator of early-stage R&D, with applications ranging from protocol optimization to biomarker discovery and scenario modeling.

However, technology alone is not a differentiator. The true impact lies in how it is deployed. When data scientists collaborate with clinical, regulatory, and commercial experts, AI can be used to simulate development pathways, optimize evidence plans, and even predict real-world impact, all before the first patient is enrolled.

Deloitte’s 2025 R&D reinforces this shift. Their findings show a rebound in expected returns across top biopharmaceutical portfolios, attributed in part to earlier integration of analytics and more disciplined strategic planning. Organizations that apply advanced tools to frame development decisions, not just to accelerate them, are better positioned to deliver both clinical value and market readiness.

As demand for differentiated data and outcomes-based evidence continues to grow, AI-supported scenario modeling will help bridge the gap, generating insights that support both internal investment decisions and eventual validation by regulators and access decision-makers.

Building Smarter Foundations for Development and Access

Early-stage planning is evolving from a tactical process to a strategic imperative. As pipelines grow more complex and stakeholder expectations rise, success will depend on how well companies align clinical goals, regulatory pathways, and value narratives from the outset.

Whether through external collaboration or intelligent use of emerging technology, forward-thinking organizations are laying the foundation for development that is not only efficient, but also evidence-rich, access-aware, and ultimately patient-relevant. This alignment doesn’t happen at launch, it starts at discovery.

About the Author

Ian Fisher, Head of Development Analytics, Regulatory Affairs and Drug Development Solutions at IQVIA leads a team of experienced analysts to provide drug developers with insights and direction for indication prioritization, target product profiles, clinical development planning and asset valuation. Fisher and his team leverage structured and instructed data and information within structured decision-making frameworks to increase the efficiency, transparency and success of strategic program development. Fisher has over 22 years' experience across drug development and holds master’s degrees in Chemistry and Health Economics.