Rather than waiting for late-stage development, biotech startups should integrate commercial planning into early trials. This means building in data collection for payers, pricing, and patient access from the start. This "think with the end in mind" approach ensures the company has the right data for pivotal trials and market access.
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While the CEO of Transgene aspires to launch their product independently in Europe, he acknowledges the immense cost of a Phase 3 trial. A partnership with a large pharmaceutical company is viewed as the most realistic "best case scenario" to accelerate development and de-risk the final, most expensive stage.
The company's long-term plan is to handle drug development through to a successful New Drug Application (NDA) filing, then partner with a larger pharmaceutical company for marketing and sales. By deliberately avoiding the need to build a commercial sales force, they maintain focus on their core competency: drug development and clinical execution.
Don't wait until Phase 3 to think about commercialization. Biotech firms must embed secondary endpoints in Phase 2 trials that capture quality of life and patient journey insights. This data is critical for building a compelling value proposition that resonates with payers and secures market access.
The traditional drug-centric trial model is failing. The next evolution is trials designed to validate the *decision-making process* itself, using platforms to assign the best therapy to heterogeneous patient groups, rather than testing one drug on a narrow population.
Ainsworth believes a responsible biotech entrepreneur envisions the end goal—acquisition or IPO—from day one. At RetroSense, this meant constantly engaging with potential acquirers like Allergan to understand their needs and generate the specific data required to become an attractive M&A target.
The process of testing drugs in humans—clinical development—is a massive, under-studied bottleneck, accounting for 70% of drug development costs. Despite its importance, there is surprisingly little public knowledge, academic research, or even basic documentation on how to improve this crucial stage.
A crucial piece of advice for biotech founders is to interact with patients as early as possible. This 'patient first' approach helps uncover unmet needs in their treatment journey, providing a more powerful and differentiated perspective than focusing solely on the scientific or commercial landscape.
Resolution Therapeutics' CEO warns that manufacturing process changes cannot wait for pivotal trials in cell therapy. The drug product used in a Phase 1/2 study must be highly comparable to the final commercial version to avoid extremely costly delays and extensive comparability studies later in development.
Airway Therapeutics' CEO founded a CRO to resolve the disconnect between academic research's discovery focus and industry's market-driven goals. This "translator" model aligned incentives and regulatory understanding, fostering more efficient drug development by merging clinical feasibility with commercial targets.
In a challenging market, founders must demonstrate a clear trajectory from idea to meaningful clinical activity data. Lengauer provides a concrete financial map: $7-15 million to a development candidate, then an additional $30-50 million to reach the key clinical value inflection point that attracts later-stage investors.