five key tiPs foR develoPing oRPhan PRoducts

5KEY TIPS FOR DEVELOPING ORPHAN PRODUCTS

As more drug companies search for new approaches after massmarket drug revenues are lost to generic competition, orphan drug development is gaining momentum. David Shoemaker senior vice president of research and development of Rho, a CRO based in North

Carolina, is sharing fi ve tips to select the right development partner to accelerate approval for orphan drug products and get much needed medicine in the hands of patients quickly and effi ciently.

1Work with CROs that have strong scientifi c, regulatory, and statistical expertise

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 scientifi c and statistical expertise to develop a plan for success at the outset to reach approval in a 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.

2Know 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 benefi ts 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.

3Apply for US and European Orphan Drug Designation simultaneously 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 signifi cant reduction of time and effort.

4Look 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 purposedriven approach to product development. It will also be important to gain buy-in from Key Opinion Leaders.

5Validate 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. Pharmaceutical and biotechnology companies can accelerate successful development of orphan products by partnering with product development services providers with a culture of solving challenges and the scientifi c and regulatory expertise to navigate complex trials and approval processes.

To see this story online visit http://biotechnologyfocus.ca/fi ve-key-tips-fordeveloping-orphan-products/