Saturday, October 24, 2020

Guidelines To Write and Submit an Orphan Drug Application For A Rare Disease

March 17, 2010 by  
Filed under Featured Stories

While working on our orphan drug application for Cyclodextrin for the treatment of Niemann Pick Type C disease, I searched the Internet looking for examples of an actual orphan drug application that was filed with the U.S. Food and Drug Administration.

Not surprisingly, I could not find any ‘real applications’ that could be used as a reference or starting point. Since most applications are filed by Pharma or BioTech companies, the applications are typically kept confidential.

Here is a PDF document of the actual orphan drug application we filed with the FDA a few weeks ago (it takes a minute to download as the PDF is large). I hope that the posting of our completed application will benefit someone else who is going through the application process and is not sure where to start.

Dr. Timothy Cote stressed at the FDA Orphan Drug Workshop at Keck Graduate Institute that too many companies over complicate the process and file volumes of data when it’s not really necessary. Making a request for orphan drug designation is a  simple process and the application can be 10 or so pages with backup material.

Things To Consider Before Filing Orphan Drug Application

Here are some key things I learned at the FDA Workshop on how to file for a request for orphan designation (RFD):

  • In 2009, 250 requests for orphan drug designation were filed with the FDA, and 160 received it.  According to the FDA, roughly 60-70% of applications result in granting of orphan status
  • Denials were generally as a result of not meeting rare disease prevalence requirements (ie. trying to submit for something that is not a rare disease or a subset of a larger disease)
  • Here is what you will need to be prepared to answer in your filing:
    • What is the disease and is the disease rare (less than 200,000 prevalence)?
    • Will your drug treat this rare disease?
    • Can you demonstrate that there is “promise” that the drug will be effective in treating the rare disease. According to the FDA, “promise” is liberally interpreted to include:
      • Data from clinical trials OR
      • Data from case studies/reports OR
      • Data from animal models OR (rarely)
      • Data from in vitro experiments
      • Theories are NOT accepted
  • Expect roughly 60 days to get a decision once you make a filing
  • A negative decision can always be overturned.  The record remains forever open according to Dr. Cote
  • If you receive a designation, you need to file an annual report each year to give an update on your progress. You can now file a single annual report with the FDA and EMA.  If we receive a designation for Cyclodextrin, I will write a separate blog on other requirements we will need to fulfill

Suggested Reference Materials

Here is some suggested reference materials that the FDA uses to guide people who call the FDA with questions on creating requests for orphan designations (RFD).

Dr. Cote told the group that the FDA’s Office of Orphan Products Development is there to help people with the process. I have had many dealings now with the staff at the FDA and they have been extremely helpful.

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12 Responses to “Guidelines To Write and Submit an Orphan Drug Application For A Rare Disease”
  1. Bo Allen says:

    My company , Natchez Trace Consulting Group has written over 2o orphan designation for the FDA and 7 for the EMEA. The US OOPD is wonderful to workwith and supportive of a small company’s efforts on every front.

  2. Sam Grant says:


    I am just wondering if yo have received your Orphan Drug Designation, and if so, what other requirements have you had to do?

    I am looking at applying for Orphan Drug Designation for my product, a natural Product for Multiple Myeloma, which also reduces the side effects of other MM Drug Treatments, and would really appreciate any assitence I can get.

    I thank you and hope to hear from you.

    Best regards,


  3. Dear Chris,
    Thanks for posting this information and for the copy of the application. It is helpful to know more about the process. There are many needed and useful treatments that can be of great benefit to the public and this information is valuable to those working in the life science industry.

    Craig Shimasaki, PhD, MBA

  4. Alfresco says:

    Thank you very much sir, this article is of great help. As mentioned by Decebal an update on whether you received the designation and what new lessons were learned will be definitely help.

  5. Decebal Bora says:

    Dear Sir;
    I hereby thank you for your efforts for openly posting this on the web. Very much appreciated. Hopefully, the FDA grants your product Orphan Designation. It owuld be interesting as a follow-up to have a lessons learnt exercise / debrief of the points you had to further address (of course retaining the confidential information).
    Yours sincerely,
    Decebal Bora

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