The FDA approved Travere's drug for the kidney disease FSGS based on the surrogate endpoint of proteinuria, despite the drug failing on the traditional eGFR endpoint. This decision, following a company-backed effort to validate proteinuria, suggests increased regulatory flexibility and creates a new pathway for kidney disease drug approvals.

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.

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.

Biotech leaders must stop viewing commercialization as a post-approval task. The critical window is Phase 2 clinical trials. By embedding patient journey and quality of life insights into secondary endpoints, companies can build a compelling value proposition for payers and physicians. Waiting until Phase 3 is too late.

Acadia's R&D process starts by considering what will ultimately matter to patients, physicians, and payers. This "end in mind" approach ensures clinical trials are designed to demonstrate meaningful, commercially relevant benefits. It forces realism about a drug's potential impact early in development, avoiding wasted resources on therapies that won't be adopted.

The CEO's team previously developed Zorampic, which failed commercially due to low efficacy and a kidney toxicity warning. This firsthand experience provided a precise roadmap for their next venture: finding a molecule that was significantly more potent and demonstrably free of the same renal safety liabilities.

The company's clinical trials go beyond standard pain scores to track improvements in function, sleep, and patient satisfaction. Demonstrating that patients can climb stairs, drive, and sleep better provides a more compelling value proposition for a faster return to normal life, resonating with patients, surgeons, and payers alike.

Ocular Therapeutix's trial prioritized a primary endpoint designed to satisfy FDA requirements for a superiority label—a key regulatory win. However, the CEO stresses that clinicians use different metrics like OCT fluid, where their drug "easily beat Eylea." This highlights a crucial strategy: separate the endpoint needed for approval from the data that drives physician adoption.