What is the Orphan Drug Designation?

The Orphan Drug Designation (ODD) is a regulatory status granted by authorities to a medicinal product that facilitates the development and approval of treatment options for a rare or “orphan” disease. The definition of an orphan disease varies across different authorities. For example, the U.S. Food and Drug Administration (FDA) defines an orphan disease as a “disease that affects less than 200,000 people in the United States” whereas for the European Medicines Agency (EMA), a disease is considered rare or orphan if it “affects fewer than 5 in 10,000 people across the European Union”. Nevertheless, a medicinal product being granted the ODD status is associated with several incentives and expedited approval. An ODD product may be exempt from regulatory fees (i..e, those for a Biologics License Application or New Drug Application) and have greater market exclusivity for a given period after obtaining approval.

What is the significance of an Orphan Drug Designation in the regulatory landscape?

A medicine being granted an orphan drug designation (ODD) is subject to an expedited approval pathway as the evaluation period is shortened to 90 days, provided the eligibility criteria and data requirements are met.

How to obtain Orphan Drug Designation

Applicants must submit a request to the relevant regulatory authority, providing comprehensive information about the drug and the disease it targets.

For example, sponsors submitting to the US FDA must complete the Form FDA 4035 and include basic elements such as administrative information, explain the disease, provide a comprehensive scientific rationale (including mechanism of action, drug description, clinical data, in vivo data, and in vitro data, demonstrate clinical superiority), and population estimate. Sponsors can submit ODD requests through the CDER NextGen portal, via email to orphan@fda.hhs.gov, or by mailing the information to the Office of Orphan Products Development.

Sponsors looking to submit designation requests to EMA can do so directly via the IRIS platform or request a pre-submission meeting with the Agency beforehand. Sponsors can use the template provided on the EMA website for sections A to E covering numerous aspects relating to the product.

Obtaining ODD confers several advantages, including 7 years of marketing exclusivity in the USA and 10 years in the EU, following marketing approval.

What are the regulatory challenges associated with obtaining an Orphan Drug Designation?

Obtaining the ODD comes with several challenges despite the advantages that it brings. Given the volume and extent of data, the application process can be relatively time-consuming as it requires detailed documents and comprehensive scientific justification. For medicines that have limited research, it can be difficult for sponsors to prove or demonstrate the medicine’s potential and its superiority. There can also be competition as many pharma companies may be looking to obtain ODD for the same orphan disease which can lead to disputes. Finally, in general, ODD developers may face challenges when navigating the complex regulatory framework for orphan/rare diseases.

DDReg’s Capabilities

DDReg specializes in navigating complex regulatory frameworks to facilitate accelerated access to life-saving treatments for rare diseases through Orphan Drug Designation (ODD). Our expert team understands the nuances of regulatory requirements, ensuring clients meet the eligibility criteria while providing comprehensive support in data preparation and submission processes. We leverage our extensive knowledge to streamline interactions with regulatory authorities, optimizing the path to designation and approval. By prioritizing innovative solutions and compliance, DDReg empowers pharmaceutical companies to bring effective therapies to market faster, ultimately improving patient outcomes and addressing critical unmet medical needs in rare disease populations.