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Key Tips for Developing
By David Shoemaker, SVP, R&D
1. Work with CROs that have strong scientific, regulatory, and
A strategic approach with a focus on key milestones is critical to gain approval as quickly as possible.
Look for CROs whose strengths include the ability to conduct challenging clinical trials, knowledge of
the regulatory process, and scientific and statistical expertise to develop a plan for success at the
outset to reach approval in an expedited speedy manner. Your CRO should have successfully obtained
marketing approval for other orphan products previously. Marketing applications for orphan products
require creative regulatory and statistical strategies to leverage the data obtained on populations
much smaller than typically seen by regulators.
2. Know the “ins and outs” of the U.S. Food and Drug Administration’s
approval mechanisms to help speed orphan drug approval
Many orphan diseases represent serious or life-threatening conditions. Consequently, working with a
development partner that understands each of the accelerated development pathways
(i.e., Accelerated Approval, Priority Review, Breakthrough Therapy, and Fast Track) and the potential
benefits or lack thereof is critical. Making an informed decision on the best mechanism at the start of
the orphan drug approval process is the fastest path to approval.
3. Apply for US and European Orphan Drug Designation
There is a combined form that can be used to obtain orphan drug status simultaneously in
the US and EU. It is an option that is not being used broadly, but can result in significant
reduction of time and effort.
4. Look for a CRO partner with experience working in
small patient populations
Working with small patient populations requires building communities and developing close
connections with research foundations, advocacy groups, patients and health care providers for a
purpose-driven approach to product development. It will also be important to gain buy-in from Key
5. Validate your population
Before investing time and energy in an orphan drug application, make sure you are eligible.
Regulators are on the lookout for developers who try to “slice the salami” meaning that your orphan
population is really just a subset of a larger population from which there is no substantive difference.
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