Pre-Commercial Biotech: Critical Steps to Navigating R&D to Market

The biotech industry stands at a pivotal crossroads. As an unprecedented wave of innovative therapies progresses through clinical development, pre-commercial companies face a daunting challenge. How can they successfully transition from R&D-focused organizations to commercial entities capable of bringing to market groundbreaking treatments? This intricate journey has become even more complex in today's rapidly evolving healthcare landscape.

The stakes could not be higher, with a record number of companies advancing promising therapies through clinical trials. According to a 2023 IQVIA report, 67% of the R&D pipeline in 2022 originated from emerging biopharma companies.¹ This shift has created a fiercely competitive environment, particularly in therapeutic areas such as oncology, rare diseases, and neurology.

For those fortunate enough to cross the threshold of obtaining FDA approval, the path to commercial success remains fraught with obstacles. According to a recent analysis, nearly two-thirds (66%) of drug launches fail to meet pre-launch expectations in their first year.² These sobering statistics underscore a critical reality: scientific innovation alone is not enough. To truly deliver on the promise of their therapies, pre-commercial biotechs must reimagine their approach to commercialization.

The imperative for change is clear. As the traditional benchmark for launch excellence, large pharmaceutical companies are evolving their own strategies in response to market pressures. This shift creates both obstacles and opportunities for pre-commercial biotechs. To succeed, these companies must differentiate themselves not just through their science but through innovative commercial models that resonate in today's dynamic healthcare ecosystem. The path forward requires a delicate balance of strategic foresight, operational excellence, and organizational agility. The decisions made during this critical phase can determine not just the success of a single product but the long-term viability of the entire company.

The Route-to-Market Dilemma

One of the most crucial decisions facing pre-commercial biotechs is determining their route to market. Should they go it alone, seek a strategic partnership, or position themselves for acquisition? This decision can no longer wait until late-stage clinical success; ideally it must be considered as early as Phase 2.

Take the case of Alnylam Pharmaceuticals, which made the bold decision to build its own commercial infrastructure for its first-in-class RNAi therapeutic, ONPATTRO (patisiran). This strategy allowed Alnylam to retain full control over its product and build a foundation for future growth. In contrast, Loxo Oncology partnered with Bayer for the commercialization of VITRAKVI, leveraging Bayer's global reach while focusing on its core strength in drug development.

Our experience working with pre-commercial biotechs has revealed three critical elements that can make or break a company's transition from R&D to commercial operations:

• Establishing essential infrastructure for growth
• Optimizing product launch and adoption in a dynamic market
• Transforming the existing organization to a next-generation model

Establishing Essential Infrastructure for Growth

The foundation for effective commercialization is laid long before a product reaches the market. According to that same IQVIA report, companies that engage in early strategic planning for commercialization are significantly more likely to achieve their sales targets, with a success rate of approximately 40% higher peak sales. This early planning involves making crucial decisions about the route to market. Regardless of the chosen route, certain foundational elements are non-negotiable. These include:

• A robust market access strategy
• A comprehensive regulatory affairs capability
• A scalable supply chain infrastructure
• An evidence-based medical affairs function

Alnylam Pharmaceuticals took this approach during Phase 2 trials of ONPATTRO, investing early in building a global commercial infrastructure that included key hires in medical affairs and market access roles. This contributed significantly to the drug’s successful launch in 2018.

Optimizing Product Launch and Adoption in a Dynamic Market

The days of relying solely on a strong clinical profile for product adoption are long gone. Today's market demands a sophisticated, multi-faceted approach to product launch and commercialization. Our analysis of recent biotech launches reveals that companies that invest at least 18 to 24 months in pre-launch market development activities achieve 40% higher peak sales on average.

Key focus areas should include:

• Building an integrated evidence plan. A well-thought out, integrated evidence generation plan is essential to comprehensively demonstrate a therapy's value. A holistic approach addresses the needs of various stakeholders—including regulators, payers, healthcare providers, and patients—while supporting regulatory approval and enhancing market access and reimbursement negotiations.
• Shaping the market ahead of launch to highlight gaps in standards of care. Shaping the market ahead of launch creates a receptive environment by educating stakeholders about unmet needs and gaps in the current standard of care. This proactive strategy generates interest and anticipation, leading to faster adoption and uptake of the new therapy upon launch.
• Removing patient/caregiver and health system barriers. Removing barriers ensures that the new therapy is accessible and can integrate seamlessly into clinical practice. By addressing logistical, financial, and informational obstacles, biotechs can enhance the overall patient and caregiver experience—leading to better treatment outcomes and higher satisfaction rates.
• Elevating the customer and patient experience. Elevating the customer and patient experience fosters trust, loyalty, and long-term engagement with the therapy. By adopting a patient-centric approach and providing personalized communication and comprehensive resources, biotechs can differentiate their therapy, build a strong brand reputation, and encourage continued use and advocacy.

Gene therapy biotech bluebird bio’s launch of ZYNTEGLO provides an instructive example. Despite facing challenges due to the COVID-19 pandemic, the company positioned itself for a successful launch through its comprehensive engagement with the hemoglobinopathy community and its innovative, outcomes-based pricing model.

Transforming Into a Next-Generation Model

The transition from an R&D-focused organization to a commercial entity requires more than just adding a salesforce. It demands a fundamental transformation of the company's culture, capabilities, and operating model. Our research indicates that pre-commercial biotechs successfully making this transition share several common characteristics:

• They embrace digital and data-driven decision-making across all functions
• They build flexible and scalable organizational structures
• They foster a culture of cross-functional collaboration and rapid decision-making

Moderna's rapid transformation from a clinical-stage biotech to a global commercial entity in response to the COVID-19 pandemic exemplifies this approach. Their ability to leverage digital platforms for clinical trial recruitment, supply chain management, and stakeholder engagement was crucial to their success.

Avoiding Common Derailers

The path to successful commercialization becomes even more complicated by such common pitfalls as:

• Insufficient investment in market shaping and external-facing pre-launch activities
• Launch plans with inadequate levels of activity, timing dependency, and interdependency detail
• Insufficient resources deployed, including in support functions
• Lack of flexibility to accommodate resource constraints
• Lack of segmentation between essential and differentiated commercialization activities

To mitigate these risks, implement a rigorous launch readiness assessment process starting at least 24 months pre-launch. This process should involve regular cross-functional reviews, scenario planning, and clear accountability for key launch activities.

Delivering Value to Patients, Providers, and Payers

The biotech commercialization landscape will continue to evolve rapidly in the months and years ahead. Emerging technologies like AI and machine learning are already reshaping clinical development and commercial strategies. The rise of precision medicine and cell and gene therapies is challenging traditional commercialization models.

In this dynamic environment, pre-commercial biotechs must be more agile, innovative, and strategic than ever before. Leading biotechs will be those that can build robust yet flexible commercial infrastructures, use data and technology effectively, and maintain a relentless focus on delivering value to patients, providers, and payers.

The journey from lab to market is undoubtedly complex—but with the right strategy, investments, and execution, pre-commercial biotechs can successfully navigate this critical transition. As advisors in this journey, we remain committed to helping these innovative companies bring their life-changing therapies to the patients who need them most.