The life sciences industry is facing a period of disruption, especially in the United States, where emerging biotech and small pharma companies must navigate funding constraints, regulatory shifts, and evolving therapeutic priorities. In this environment, one strategic tool is proving indispensable: the target product profile (TPP).

What is a target product profile

A target product profile is a strategic blueprint that outlines the key objectives a drug must meet to gain regulatory approval and reach patients. It typically includes:

• Minimum viable criteria for approval.
• Base case expectations for performance.
• Aspirational goals that define true success.

This framework helps developers align scientific, regulatory, and commercial goals from the outset, ensuring that every step in the drug development process is purposeful and data-driven.

TPPs provide strategic clarity

In an environment where funding is scarce and investor scrutiny is high, TPPs serve as a company’s North Star. They provide clarity and direction, allowing teams to:

• Demonstrate a deep understanding of the disease area and unmet patient needs.
• Communicate a clear vision to potential investors and partners.
• Align internal efforts across clinical, regulatory, and commercial functions.

For emerging pharma, a well-crafted TPP signals strategic maturity and readiness to engage with stakeholders who can help bring a drug to market.

TPPs evolve across the drug lifecycle

The utility of TPPs changes as a drug progresses through development. In early-stage development, TPPs help navigate high uncertainty and establish foundational goals. In late-stage development, they become more refined, integrating robust data and commercial projections.

In large pharma, TPPs are often led by commercial teams, but the industry is shifting toward a more integrated approach. Development and commercial teams now collaborate throughout the lifecycle, ensuring that TPPs remain dynamic and relevant.

Regulatory strategy and agency engagement

Regulatory strategy is a critical component of TPP planning. While communication with agencies like the FDA may be slower during periods of administrative change, companies should:

• Leverage existing FDA resources and guidance documents.
• Engage early and strategically with regulators, especially for complex or orphan indications.
• Focus on working within established guidance rather than attempting to circumvent it.

By becoming well-versed in regulatory expectations and precedents, developers can reduce internal decision-making delays and approach agencies with well-informed proposals.

TPPs support contingency planning

Given the shifting scrutiny on certain therapeutic areas, contingency planning is essential. TPPs support this by:

• Encouraging holistic thinking about long-term regulatory and commercial goals.
• Allowing for scenario planning based on evolving standards of care and regulatory expectations.
• Facilitating strategic pivots without losing sight of core objectives.

Companies must consider not only current regulatory climates but also anticipate what approval requirements might look like five to seven years down the line.

The role of AI in TPP development

Artificial intelligence is beginning to play a role in streamlining TPP creation and refinement. Current tools can:

• Extract insights from vast quantities of scientific and regulatory literature.
• Generate first-draft TPP templates based on structured prompts.
• Assist in horizon scanning to identify changes in regulatory or therapeutic landscapes.

However, challenges remain. AI tools often rely on publicly available data, which skews toward successful trials and omits valuable insights from failed studies. Additionally, commercial data is frequently redacted or proprietary, limiting AI’s effectiveness in modeling market outcomes.

Despite these limitations, the potential for agentic AI (autonomous tools that dynamically update TPPs or flag strategic shifts) is promising. As these technologies mature, they could significantly reduce the burden on small teams juggling both strategic planning and trial execution.

Conclusion

In a time of disruption, target product profiles offer stability and strategic clarity. They empower drug developers to make informed decisions, engage effectively with regulators and investors, and adapt to changing environments. As AI tools evolve and industry practices shift toward integration, TPPs will continue to be a cornerstone of successful drug development and regulatory strategy.