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 scientific and regulatory expertise to navigate complex trials and approval processes.
The National Organization for Rare Disorders reports nearly 7,000 orphan diseases affecting nearly 30 million Americans. As more drug companies search for new approaches after mass-market drug revenues are lost to generic competition, orphan drug development is gaining momentum.
1. Work with CROs that have strong scientific, 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 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 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 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 Opinion Leaders.
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.
Please click here to view a slideshow on “Key Tips for Developing Orphan Products” by David Shoemaker for more information.