FDA Orphan Drug Designation Consulting

Orphan drugs play a critical role in addressing the unmet needs of the rare disease community. For many individuals suffering from an orphan disease, their current standard of care may be insufficient or even nonexistent. Since 1983, when the Orphan Drug Act was passed by Congress, the Food and Drug Administration (FDA) has made it a priority to incentivize orphan drug research to aid the estimated 30 million Americans afflicted with a rare disease. With an FDA orphan drug designation, sponsors can receive benefits such as: 

  • Seven years of market exclusivity after marketing approval 
  • Tax credits for qualified clinical trials
  • Waiver of the Prescription Drug User Fee Act (PDUFA) charge, currently valued at $2,875,842 
  • Waiver of Pediatric Research Equity Act (PREA) requirements

Pharmaceutical companies and clinical research organizations (CROs) have a variety of intrinsic and financial reasons to pursue an orphan drug designation. However, attached to the process is a complex application and the need for relevant supporting documentation. Due to the lack of clinical studies, treatments, and in-depth knowledge surrounding these rare diseases, the FDA can be cautious with approvals. 

Therefore, when a sponsor submits an application for a potential orphan drug, they need to ensure they are delivering correct information to meet or exceed application requirements. With orphan drugs being a less common clinical study area for sponsors, the review team in the FDA Office of Orphan Products Development (OOPD) also will require educating on highly nuanced topics. 

In order to best increase the chances of a successful application and receive a designation on the fastest timeline, working with orphan drug consulting experts is a positive investment — even for veteran researchers and teams. By doing so, submitting sponsors are more likely to reduce application errors, deliver relevant and accurate data, and pass the rigorous review process conducted by the OOPD. 

Orphan Drug Programs


The OOPD has a variety of programs specific to the indications and treatments of rare diseases. Since 1983, over 600 drugs and biologic products have been successfully developed and marketed through the programs. The included programs are as follows: 

  • Orphan Drug Designation Program 
  • Rare Pediatric Disease Designation (RPD) and Voucher Programs 
  • Humanitarian Use Device (HUD) Program 
  • Orphan Products Grants Program
  • Pediatric Device Consortia Grants Program

How BioPharma Global Improves Orphan Drug Applications


The world of orphan drugs and the regulatory process attached to it remains distinct from other FDA applications and review divisions. The complex nature surrounding the topic and the detailed guidance on regulations make the application one that requires specific expertise. 

Our team at Biopharma Global has extensive experience writing, submitting, and revising orphan drug applications. With a full staff of orphan drug regulatory experts, we are able to give clients the correct guidance on how to approach the OOPD reviewers, prepare applications, and deliver to OOPD. 

While the FDA has put out information and tips on how to best create your orphan drug application, the resources fall short of being a truly useful or practical guide to a successful submission. Our team starts by reviewing in detail your current data package to determine if a path forward exists. From this audit, we use your data to write, edit, and revise your ODD application to better pass FDA review. With our help, we see sponsors regularly achieve orphan drug designation and the movement of their therapy one step closer to helping improve lives.  If you are in the stages of drug development with the end goal of applying for FDA Orphan Drug Designation, check out our white papers on Orphan Drug Designation and Overcoming Orphan Drug Exclusivity.

Got questions? Ready for a review? Let’s discuss. Schedule a free consultation today to talk with our orphan drug experts.

Biopharma Global - FDA & EMA Regulatory Affairs Consulting Services