Orphan Designation Requests: What You Need to Know

Orphan Designation Requests: What You Need to Know


If you are working in the pharmaceutical or biotech industry, you may have heard about orphan designation (OD) requests. Orphan designation is a status granted by regulatory agencies. Such as the Food and Drug Administration (FDA) in the United States and the European Medicines Agency (EMA) in Europe, to drugs or biologics intended to treat rare diseases. This designation provides certain benefits to the drug developer, including tax incentives, market exclusivity, and regulatory assistance. In this essay, we will cover everything you need to know about orphan designation requests, including what they are, how to apply for them, and the benefits they offer.

What is an Orphan Designation(OD) Request?

An OD request is a formal request made to regulatory agencies to grant orphan status to a drug or biologic intended to treat a rare disease. A rare disease is a condition that affects fewer than 200,000 people in the U.S or fewer than 5 in 10,000 people in the European Union. The purpose of OD is to encourage the development of treatments for rare diseases by providing certain incentives to drug developers.

How to Apply for OD?

To apply for orphan designation, drug developers must submit a formal request to the regulatory agency in charge of drug approval in the country where they want to market their drug. In the United States, this is the FDA, while in Europe, it is the EMA. The application must provide detailed information about the drug or biologic, the disease it is intended to treat, and the evidence supporting its efficacy and safety.

Criteria for Orphan Designation

To be eligible for orphan designation, a drug or biologic must meet certain criteria. First, it must be intending to treat a rare disease as defining by the regulatory agency. Second, there must be no other available treatments for the disease or, if there are available treatments, the drug must show significant clinical improvement over existing treatments. Finally, the drug must show promise in addressing an unmet medical need. Meaning it has the potential to provide a significant benefit to patients with the rare disease.

Benefits of Orphan Designation

OD provides several benefits to drug developers. First, it grants market exclusivity for the drug for a period of time, typically 7 years in the United States and 10 years in Europe. This means that no other drug can be approved for the same rare disease during that period, providing a significant

competitive advantage. Second, it provides tax incentives to drug developers. Including a tax credit for certain clinical trial expenses and exemption from paying the FDA or EMA user fees. Third, it provides regulatory assistance to drug developers, including a streamlined drug approval process and access to scientific advice from the regulatory agency.

Challenges of OD

First, the application process can be time-consuming and costly, requiring extensive documentation and evidence. Second, drug developers must demonstrate that their drug meets the eligibility criteria for orphan designation. Which can be challenging if there are other treatments for the disease. Finally, even if a drug have OD, there is no guarantee of approval or market success.

  • Demonstrating Eligibility Criteria: Drug developers must provide evidence that their drug meets the eligibility criteria for orphan designation. Including addressing unmet medical needs and demonstrating significant clinical improvement over existing treatments.
  • Competition from Existing Treatments: If there are treatments for the rare disease. Drug developers must demonstrate that their drug provides significant clinical improvement over those treatments, which can be challenging.
  • No Guarantee of Approval: Even if a drug carry OD, it is still subject to regulatory approval. And there is no guarantee of success in the market.
  • Limited Market: The market for drugs intended to treat rare diseases is small. Which can limit the potential revenue for drug developers even with orphan designation benefits.

Future of Orphan Designation

As the pharmaceutical and biotech industry continues to grow, the importance of orphan designation is likely to increase. There are currently more than 7,000 rare diseases affecting an estimated 400 million

people worldwide, and many of these diseases have no approved treatments. OD provides a valuable incentive for drug developers to invest in the development of treatments for these diseases. It is likely that we will see an increasing number of orphan designation requests in the coming decade.


In conclusion, navigating the world of orphan designation requests can be complex. But the potential benefits for rare disease treatments are immense. Understanding the criteria, requirements, and processes involved in obtaining orphan designation is crucial for pharmaceutical companies and researchers seeking to develop therapies for rare diseases. With continued advancements in orphan drug development. We can bring hope and improved quality of life to patients affected by rare diseases, ultimately making a meaningful difference in their lives.