Typically, the starting dose in a Phase 1 trial is too low to show efficacy. For CDR Life, observing immunological activity and biomarker improvement in their very first patient was a rare and remarkable event that provided the first tangible sign their scientific platform could become a real therapeutic.

To demonstrate a long-term survival benefit without a new trial, Neuvivo hired a research firm to track down patients from the original study. By collecting "last date alive" information in a blinded fashion, they generated statistically significant survival data years after the trial concluded.

Despite rigid protocols, investigators must use their clinical judgment, informed by prior data, to enroll patients they believe will genuinely benefit. This patient-centric approach is viewed as not only ethical but also crucial for achieving a positive trial outcome, blending the art of medicine with the science of research.

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.

Running an unusually long, two-year Phase 2 trial allowed Vera to demonstrate stabilization of GFR, a hard kidney function endpoint. This robust, long-term data was crucial for de-risking their Phase 3 program and ultimately securing a coveted Breakthrough Therapy Designation from the FDA, accelerating their path to market.

Corvus Pharmaceuticals' ITK inhibitor received FDA encouragement to proceed directly from Phase 1 to a Phase 3 registrational trial for T-cell lymphoma. This was due to the disease's high mortality, lack of effective treatments, and the drug's exceptionally strong early survival data.

Cereno Scientific chose a Phase 2b trial over a combined 2b/3 to maintain flexibility. A combined trial locks in the design for both phases upfront, whereas a standalone 2b allows for optimization before Phase 3 and creates a cleaner, more attractive asset for a potential acquisition deal.

Despite FDA readiness for a final Phase 3 trial, Connect Biopharma chose to run more Phase 2 studies. They discovered their long-term asthma drug worked in hours, not weeks, and are now pivoting to prove its value in acute, emergency situations, which informs a stronger, more targeted Phase 3 design.

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.