Resurrecting a Product from a Study That Failed to Achieve
its Primary Endpoint
When this client's lead
compound failed to achieve its primary endpoint in a pivotal
Phase 3 trial, they asked Umen & Co. to help determine
whether continued development was warranted, and if so, how
the company should proceed.
Our team and an independent
group of medical consultants reviewed all of the available
data to gain a full understanding of the drug's efficacy and
safety profile. During the review, it became apparent that
the data, including the Phase 3 trial, suggested that the
drug was intrinsically effective and devoid of any
significant safety signals. Based on these favorable trends,
the client was advised that further development should be
considered. We subsequently helped construct a new clinical
development plan aimed at gaining marketing approval.
From a strategic regulatory
standpoint, it was critical to bolster the application with
respect to efficacy as much as possible given that the
previous Phase 3 trial had not achieved its primary
endpoint. Consequently, we developed a regulatory strategy
that had the potential of providing the substantial evidence
of effectiveness necessary for approval in the shortest time
frame possible. In addition to conducting additional Phase 3
trials, this strategy positioned select Phase 2 trials and
an alternate analysis of the previous Phase 3 trial as
confirmatory evidence of effectiveness.
When the new Phase 3 trials
yielded positive results, we provided the medical writing
services to complete the clinical study reports, the ISE,
the ISS, and the Application Summary in a manner that
integrated the regulatory strategy into each of these key
NDA documents. We also worked with the client's
multidisciplinary team to draft and annotate the PI.
The NDA was ultimately approved
and the drug became the company's first marketed product.
What was once a doomed drug now benefits countless patients
and delivers considerable value to our client.