Blog Post

Key Tips for Orphan Product Development

August 27, 2013

David Shoemaker

Information for this article was contributed by David Shoemaker, Senior Vice President R&D. Dr.  Shoemaker has over 25 years of experience in research and pharmaceutical development.  He has managed or contributed to dozens of INDs/CTAs and over a dozen successful NDAs, BLAs, and MAAs.  Dr. Shoemaker has authored or overseen dozens of Orphan Drug Designation applications, has developed several successful Accelerated Approval programs, and has secured several Priority Review applications.

Selecting a partner for drug development is tricky.  This is especially true when selecting a CRO to assist with orphan product development.  Finding a partner that has both the experience and expertise needed as well as being a good cultural fit for your company is critical to achieving your goals. 



  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.

Pharmaceutical and biotechnology companies can accelerate successful development of orphan products by partnering with product development service 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.

At Rho, we share a passion for discovering new treatments and have experience successfully helping companies navigate the FDA’s orphan product approval processes. But just like anything that sounds too good to be true, sound product development program decisions should stem from a keen understanding of the requirements and potential benefits of each approach. Selecting the right product development services partner can help deliver new treatments to improve and save lives as quickly as possible.