According to FDA guidance, Orphan Drug Designation (ODD) is a status granted by regulatory authorities to drugs or therapies that are meant to treat rare diseases or conditions. For some rare disease treatments, the low financial incentives to continue development or production led to them being “orphaned” or discontinued. To help with this, the Orphan Drug Act (a law passed by Congress in 1983) incentivizes drug development for rare diseases. [2]
What are orphan drugs?
These are drugs (including biologics) for the prevention, diagnosis, or treatment of diseases or conditions affecting fewer than 200,000 persons in the US, OR drugs that will not be profitable within 7 years following approval by the FDA.
Companies and other drug developers can request orphan drug designation, and the FDA will grant it if the drug meets the required criteria. An ODD provides financial incentives to make it easier to bring a drug or therapy to market. [14] These incentives include:
Tax credits for qualified clinical (in humans) testing
Waiver of the FDA New Drug Application or Biologics License Application (currently over $4 million for a new drug)
Eligibility for 7-year marketing exclusivity ("orphan exclusivity") upon marketing approval
In addition, the Orphan Drug Act established the Orphan Product Grants Program to provide funding for developing products for rare diseases or conditions. [10]
How do I apply for ODD?
Sponsors seeking ODD for a drug must submit a request for designation to the agency. Of note, multiple sponsors can hold a designation for the same drug and rare disease. Designations for Orphan Drug and Rare Pediatric Disease are granted by the Office of Orphan Products Development (OOPD), and this is where you send your request. It is important to note that ODD is a separate process from seeking approval or licensing. Drugs for rare diseases go through the same rigorous scientific review process as any other drug for approval or licensing. [3]
OOPD evaluates information from product sponsors to determine if drugs, biologics, or medical devices meet the criteria for certain incentives and administers grants to provide funding for research on rare diseases. The office also works on rare disease issues with medical and research communities, professional organizations, academia, government agencies, industry, and rare disease patient groups. Together with the Office of Pediatric Therapeutics and product centers, the OOPD also determines Rare pediatric disease designation for drugs or biologics that meet certain criteria.
It is therefore important for you to ensure you are delivering correct information to meet application requirements when you apply for a potential orphan drug. We recommend that you ensure your request includes the following:
ODD request statement
Administrative information
Description of the rare disease or condition, proposed indication, and need for therapy
Description of your investigational product and scientific rationale for use
Orphan drug status
Patient subset considerations and medical plausibility of the chosen subset
Regulatory status and marketing history
Documentation of patient population size
ODD application examples
The BGTC has several examples from successful ODD applications by BGTC-sponsored drug developers that you can reference for what the ODD application could look like.
Ocular
Systemic
Neurological
The FDA has a form designed to assist sponsors in providing the required content completely and succinctly for ODD requests. [8] This can be found here: Form FDA 4035. We have provided you with a template that you can use for the ODD request. This template was generated based on the National Institutes of Health (NIH) National Center for Advancing Translational Sciences (NCATS) Platform Vector Gene Therapy (PaVe-GT) team’s experience preparing an ODD request to the FDA Office of Orphan Products Development. If you need an example for reference while you prepare your request, feel free to use BGTC examples linked above. We have also listed them out below.
How to submit ODD requests [8]
You may submit ODD requests one of three ways:
Through the CDER NextGen portal
By emailing the required information to orphan@fda.hhs.gov
By mailing the required information to:
Office of Orphan Products Development
Attention: Orphan Drug Designation Program
Food and Drug Administration
WO32-5295
10903 New Hampshire Avenue
Silver Spring, MD 20993-0002
If you plan to email information to the FDA that is private, sensitive, proprietary, or commercial confidential, it is strongly encouraged to send it from an FDA-secured email address, so the transmission is encrypted. The agency will assume the addresses of emails received or email addresses provided as a point of contact are secure when responding to those email addresses.
Next, we will look at the Rare Pediatric Disease Designation.