The story
A Phase 3 go/no-go decision that rested on evidence, not optimism.
A biotech was developing a therapy for an ultra-rare paediatric lysosomal storage disorder. Before committing to Phase 3, they needed more than a feasibility check. The real question was commercial. Even if the trial could be run, was there a viable product on the other side of it. We built the answer from the true patient population and the market's readiness for the TPP, not from published prevalence or assumed demand.
A biotech developing a therapy for an ultra-rare lysosomal storage disorder affecting young children was approaching a Phase 3 go/no-go decision. The question on the table was trial feasibility. The real question was broader: even if the trial could be run, was there a commercially viable product on the other side of it?
Unmet need was not in doubt. What was far less certain was how many children actually matched the profile the TPP described. Published prevalence figures were years old and built on incomplete diagnosis data, and paediatric-onset, ultra-rare populations are structurally undercounted, since many children never receive a confirmed diagnosis at all. Nor was it clear which PI sites combined genuine disease expertise with real patient access. Experience with the disease does not map cleanly onto patient volume.
Building the answer from the real population
We rebuilt the patient count from the ground up, working from diagnosed and likely-undiagnosed children rather than published prevalence alone. We mapped which sites could actually reach those patients, modelled what the true population meant for the enrollment window, and tested the TPP against how clinicians, patient organisations and payers actually weigh the therapy's expected benefit against existing standards of care.
The finding was stark. Under the TPP as defined, we confirmed one eligible patient worldwide.
A decision the board could defend
The company stopped the programme before further investment was committed, redirecting years of spend away from a trial that could not enroll and a product that could not sustain a market under its current profile. The work showed that unmet need alone did not translate into commercial viability. The stop decision rested on evidence the company could defend to its board, not on instinct.
"The value was not in telling us what we wanted to hear. It was in giving the board a decision we could defend with evidence."