During drug discovery, collaborations can ensure that points in the system where bottlenecks occur can be kept wide open. Programs like ‘fast track designation’, ‘translational research’ as well as ‘public/private collaborations’, all aim to serve the essential needs of manufacturers to speed up the process overall. Read the full article on “Targeted therapies: speed and collaboration” to find out what makes public/private collaborations so powerful and how the largest collaborations of its type might serve as a model for other huge projects in the future. Read the full 5-page article here: http://bit.ly/1q0wmaT

1. Targeted therapies: speed and collaboration
By Al Tuttle, Editor
Regulatory bodies get a lot of negative publicity about their treatment of the
companies and institutions they regulate. It is a natural outcome of the
regulator/regulated relationship: the governmental bodies either overstep their
authority or should not have been given such authority in the first place. However,
the regulators offer many positive aspects to drug companies and their
collaborators. For example, the FDA and EMA are vast research organizations in
their own right. They provide guidance and control, but also have support and
innovation as public objectives.
In a report about innovation and targeted patient therapies (1), the FDA delineates
some of its large array of regulatory supported programs. Aimed at expediting the
drug development pathway, the agency noted that many targeted early trials get
dramatic results because the patients are selected based on the narrow set of
criteria expected to produce the best results. The FDA will collect and collate data to
help develop wider trial results quickly so that the end result is a drug that can
withstand all regulatory scrutiny. In these cases, the agency wanted to publish ways
to get the fastest avenue for larger trials started, bring those to good conclusions
quicker and certify results more efficiently.
Using these proposals as a foundation, in June, 2013, the FDA published a guidance
for “expedited programs for serious conditions.” (2) They are aimed at answering
several important questions about expedited trials:
•
•
•
“Can FDA, drug developers, and investigators agree on a threshold to
determine when a treatment poses ‘exceptional promise’ and should thus be
treated in an expedited fashion? Can seamless drug development programs
be created to utilize natural history data or adaptive trial design concepts to
compress drug development time?
What are the ethical issues involved in identifying a promising intervention?
How should the needs of all patients with the disease be balanced against the
need for better therapy for an individual?
Can surrogate outcome measures that could be used for accelerated approval
be rapidly identified?
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2. •
Can we arrive at a consensus view of the goal of monitoring commitments
companies will make once a product is on the market after such a
development program, such as scientific expectations and timelines?”
Four to fast track
The guidance lists these four programs for expediting approvals: fast track
designation, breakthrough therapy designation, accelerated approval, and priority
review designation. All are aimed at putting the most critical medicines at the top of
the list and hastening the process through channels at all levels.
A fast track designation the designation of a drug as a fast track product “if it is
intended, whether alone or in combination with one or more other drugs, for the
treatment of a serious or life-threatening disease or condition, and it demonstrates
the potential to address unmet medical needs for such a disease or condition.” The
difference between a fast track designation and a breakthrough therapy designation
are few, but is generally used when there is a
drug therapy in place which the new therapy
would be substantially more effective.
Photo courtesy npr.org
Accelerated approval can start when a
combination of factors has been reached,
particularly advanced trials in which the therapy
has proven substantially better than an existing
drug. The manufacturer is usually required to
extend trials beyond the approval, to continue
finding favorable results.
“A priority review designation means FDA’s goal is to take action on the marketing
application 806 within 6 months (compared to 10 months under standard review).”
There are several qualifications for priority review that generally mirror those for the
other three designations, but review is done at the latest phases of trials.
Collaboration and speed
Let’s look at the effect of regulations and the accelerated discovery processes on
collaboration. Everyone agrees that one of the essential needs of manufacturers is
speeding up the process overall. However, collaborations can shorten the time by
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t: +49 (0) 30 2091 3 274 | f: +49 (0) 30 2091 3263 | e: eq@iqpc.de | w: www.iqpc.de
Visit IQPC for a portfolio of topic-related events, congresses, seminars and conferences: www.iqpc.de