program can be problematic if the vendor company is not
experienced in managing such programs, and working
closely alongside sponsors to construct development programs that meet project deadlines. Contract vendors that
have integrated offerings at one site can provide significant
advantages through efficiencies of scale and experience.
Undertaking programs with shortened timelines to the
required quality and regulatory standards can prove challenging. Having worked on a number of these projects at
Catalent Pharma Solutions’ Kansas City, Missouri, a number
of critical factors have become apparent and best practice
procedures adopted. Because of the shortened timelines
associated with drugs with expedited and orphan designations, programs are established with a single, cross-func-tional team that can drive development from early-phase all
the way through to commercialization. By utilising a team
based approach, with specialists in fields ranging from formulation, pharmaceutics and analytics to packaging and
project management integrating seamlessly with the sponsors’ teams, potential root causes and optimum solutions
can be identified quicker keeping programs on-track. Of
course, development speed is worthless without regulatory
compliance and a robust quality system. Contract providers
should be able to demonstrate a successful track-record of
audits and pre-approval inspections from the FDA, EMA and
other regulatory bodies, and across multiple drug products
with expedited or orphan designations.
To summarize, several expedited program designations
and approaches are available from the FDA, of which break-
through therapy designation is the latest. A sponsor needs
to ensure that all aspects of its development programs can
support expedited designations before pursuing them. If
specialist development support is needed, sponsors need to
have close, honest relationships with the contract vendors to
enable close working relationships and rapid communication.
Risk assessment and mitigation/contingency planning is es-
sential. An integrated development program approach under
one quality system with single-point project management can
be used to leverage risk and reduce time-lines to get maxi-
mum benefit from receiving expedited drug designations.
ABOUT THE AUTHOR:
Richard J. Holl, PhD, is Director of the Pharmaceutics II
group at Catalent Pharma Solutions in Kansas City, Missouri,
where he manages a group of twenty scientists and technicians involved in contract pharmaceutical development and
manufacture. He has been at his current position for the
past 6 years, having been previously employed at several
large and small pharmaceutical companies throughout his
career including Cephalon.
His professional experience has concentrated on discovering novel drug delivery systems and in developing robust
pharmaceutical formulations and manufacturing processes.
Marketed products that he has been involved in developing
include Onsolis® and Amrix®. He is a listed inventor on 33
US and international patents or patent applications. He has
been involved in development of multiple injectable, topical,
transmucosal, and oral drug products and the processes used
in their manufacture. One particularly interesting project
involved designing an experimental module for pharmaceutical processing that flew on board the U.S. Space Shuttle.
He received his doctorate degree in chemical engineering
from Auburn University and his undergraduate chemical
engineering degree from the University of Florida. ■