Regulatory Open Forum

Hi Colleagues, 

According to the Orphan Drug Designation guidance, after you get designated for certain subset, you are supposed to submit AR for that Orphan Drug Designation within 14 months of approval. If someone as experienced, please provide what and how this request needs to be done.

Thanks,

Masa  


------------------------------
Masa Razic
Regulatory Affairs Specialist
Lexington MA
United States
------------------------------

Hello Rasa,

Submit on a single compact disk (CD) with a signed cover letter attached (as you would with an original orphan drug designation request). 

For the content, consult 21 CFR 316.30 

Kind regards,
Michelle

------------------------------
Michelle McGuinness
Consultant - Quality Assurance, Regulatory Affairs & Commercial Compliance
Glenmoore PA
United States
------------------------------

Original Message:
Sent: 07-Jun-2019 14:55
From: Masa Razic
Subject: Orphan Drug Designation-Annual report

Hi Colleagues,

According to the Orphan Drug Designation guidance, after you get designated for certain subset, you are supposed to submit AR for that Orphan Drug Designation within 14 months of approval. If someone as experienced, please provide what and how this request needs to be done.

Thanks,

Masa


------------------------------
Masa Razic
Regulatory Affairs Specialist
Lexington MA
United States
------------------------------

Yes - you'll need to submit an Annual Report each year to OOPD. Check OOPD site to see if they have a template or format for the AR. Typically consists of an Introduction, Progress on Development of Drug (which includes a list of al non-clinical and clinical studies ongoing or completed during the reporting period), Investigational Plan for upcoming year, and Any Changes that may Affect Orphan Status.  Also note that if you also have an IND open, the 1571 form includes a box for the Orphan Status and Designation #. Hope this helps.​

------------------------------
Tom
Senior Director, Regulatory Affairs CMC
Chicago
------------------------------

Original Message:
Sent: 07-Jun-2019 14:55
From: Masa Razic
Subject: Orphan Drug Designation-Annual report

Hi Colleagues,

According to the Orphan Drug Designation guidance, after you get designated for certain subset, you are supposed to submit AR for that Orphan Drug Designation within 14 months of approval. If someone as experienced, please provide what and how this request needs to be done.

Thanks,

Masa


------------------------------
Masa Razic
Regulatory Affairs Specialist
Lexington MA
United States
------------------------------

An Annual Report for Orphan Drug Designation contains;

1. Pre-Clinical & Clinical Development 
2. Investigation Plan for Next Year
3. Orphan Drug Status; A brief discussion of any changes that may affect the orphan-drug status of the product. For example, for products nearing the end of the approval process, sponsors should discuss any disparity between the probable marketing indication and the designated indication as related to the need for an amendment to the orphan-drug designation pursuant to §316.26

Send this with cover letter as an attachment to an email OPDAR@fda.hhs.gov (Corresponding with the OOPD Drug Designation Programs)  

Thank you,

------------------------------
Shaishav Shah

------------------------------

Original Message:
Sent: 07-Jun-2019 14:55
From: Masa Razic
Subject: Orphan Drug Designation-Annual report

Hi Colleagues,

According to the Orphan Drug Designation guidance, after you get designated for certain subset, you are supposed to submit AR for that Orphan Drug Designation within 14 months of approval. If someone as experienced, please provide what and how this request needs to be done.

Thanks,

Masa


------------------------------
Masa Razic
Regulatory Affairs Specialist
Lexington MA
United States
------------------------------

Thank you all so much for all these helpful answers.
This was much appreciated.

Unfortunately, no templates are displayed on the OOPD website. (If anyone find any templates, please send it my way)
For each and every FDA submission, one of the forms needs to be submitted, and so far I have not found which form is requested for this submission. So I am still slightly confused.

Thanks,

Masa

------------------------------
Masa Razic
Regulatory Affairs Specialist
Lexington MA
United States
------------------------------

Original Message:
Sent: 10-Jun-2019 13:15
From: Shaishav Shah
Subject: Orphan Drug Designation-Annual report

An Annual Report for Orphan Drug Designation contains;

1. Pre-Clinical & Clinical Development 
2. Investigation Plan for Next Year
3. Orphan Drug Status; A brief discussion of any changes that may affect the orphan-drug status of the product. For example, for products nearing the end of the approval process, sponsors should discuss any disparity between the probable marketing indication and the designated indication as related to the need for an amendment to the orphan-drug designation pursuant to @316.26

Send this with cover letter as an attachment to an email OPDAR@fda.hhs.gov (Corresponding with the OOPD Drug Designation Programs)

Thank you,

------------------------------
Shaishav Shah


Original Message:
Sent: 07-Jun-2019 14:55
From: Masa Razic
Subject: Orphan Drug Designation-Annual report

Hi Colleagues,

According to the Orphan Drug Designation guidance, after you get designated for certain subset, you are supposed to submit AR for that Orphan Drug Designation within 14 months of approval. If someone as experienced, please provide what and how this request needs to be done.

Thanks,

Masa


------------------------------
Masa Razic
Regulatory Affairs Specialist
Lexington MA
United States
------------------------------

I don't believe a form is needed.

------------------------------
Tom
Senior Director, Regulatory Affairs CMC
Chicago
------------------------------

Original Message:
Sent: 10-Jun-2019 17:17
From: Masa Razic
Subject: Orphan Drug Designation-Annual report

Thank you all so much for all these helpful answers.
This was much appreciated.

Unfortunately, no templates are displayed on the OOPD website. (If anyone find any templates, please send it my way)
For each and every FDA submission, one of the forms needs to be submitted, and so far I have not found which form is requested for this submission. So I am still slightly confused.

Thanks,

Masa

------------------------------
Masa Razic
Regulatory Affairs Specialist
Lexington MA
United States

Original Message:
Sent: 10-Jun-2019 13:15
From: Shaishav Shah
Subject: Orphan Drug Designation-Annual report

An Annual Report for Orphan Drug Designation contains;

1. Pre-Clinical & Clinical Development 
2. Investigation Plan for Next Year
3. Orphan Drug Status; A brief discussion of any changes that may affect the orphan-drug status of the product. For example, for products nearing the end of the approval process, sponsors should discuss any disparity between the probable marketing indication and the designated indication as related to the need for an amendment to the orphan-drug designation pursuant to @316.26

Send this with cover letter as an attachment to an email OPDAR@fda.hhs.gov (Corresponding with the OOPD Drug Designation Programs)

Thank you,

------------------------------
Shaishav Shah


Original Message:
Sent: 07-Jun-2019 14:55
From: Masa Razic
Subject: Orphan Drug Designation-Annual report

Hi Colleagues,

According to the Orphan Drug Designation guidance, after you get designated for certain subset, you are supposed to submit AR for that Orphan Drug Designation within 14 months of approval. If someone as experienced, please provide what and how this request needs to be done.

Thanks,

Masa


------------------------------
Masa Razic
Regulatory Affairs Specialist
Lexington MA
United States
------------------------------

Hi Masa,

I've submitted several Orphan Annual Reports. There is no form or template for the US - you basically just create a document that covers the annual reporting requirements (21 CFR 316.30) and e-mail it to OOPD (OPDAR@fda.hhs.gov). While the original orphan drug designation must be submitted on a compact disk and mailed to OOPD, annual reports should be e-mailed (the FDA website specifies that these should be e-mailed).
https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/corresponding-oopd-drug-designation-programs

The EMA used to have an orphan annual report template that covered the same topics requested by the FDA (and more). If you have orphan designation in both the US and EU, you could fill out the EMA template and send it to both agencies. However, the EMA has since updated their orphan annual reporting process so the template is no longer easy to find and actually isn't required anymore. I've attached it here, along with the corresponding EMA guidance, in case you may find it helpful for what to include in your US report. Click around on the EMA form - more spaces appear depending on what you click.

------------------------------
Marissa Berry, Ph.D., RAC
Manager, Global Regulatory Affairs
Cary, NC
United States
------------------------------

Original Message:
Sent: 07-Jun-2019 14:55
From: Masa Razic
Subject: Orphan Drug Designation-Annual report

Hi Colleagues,

According to the Orphan Drug Designation guidance, after you get designated for certain subset, you are supposed to submit AR for that Orphan Drug Designation within 14 months of approval. If someone as experienced, please provide what and how this request needs to be done.

Thanks,

Masa


------------------------------
Masa Razic
Regulatory Affairs Specialist
Lexington MA
United States
------------------------------

Original Message------

Hi Masa,

I've submitted several Orphan Annual Reports. There is no form or template for the US - you basically just create a document that covers the annual reporting requirements (21 CFR 316.30) and e-mail it to OOPD (OPDAR@fda.hhs.gov). While the original orphan drug designation must be submitted on a compact disk and mailed to OOPD, annual reports should be e-mailed (the FDA website specifies that these should be e-mailed).
https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/corresponding-oopd-drug-designation-programs

The EMA used to have an orphan annual report template that covered the same topics requested by the FDA (and more). If you have orphan designation in both the US and EU, you could fill out the EMA template and send it to both agencies. However, the EMA has since updated their orphan annual reporting process so the template is no longer easy to find and actually isn't required anymore. I've attached it here, along with the corresponding EMA guidance, in case you may find it helpful for what to include in your US report. Click around on the EMA form - more spaces appear depending on what you click.

------------------------------
Marissa Berry, Ph.D., RAC
Manager, Global Regulatory Affairs
Cary, NC
United States
------------------------------

Original Message:
Sent: 07-Jun-2019 14:55
From: Masa Razic
Subject: Orphan Drug Designation-Annual report

Hi Colleagues,

According to the Orphan Drug Designation guidance, after you get designated for certain subset, you are supposed to submit AR for that Orphan Drug Designation within 14 months of approval. If someone as experienced, please provide what and how this request needs to be done.

Thanks,

Masa


------------------------------
Masa Razic
Regulatory Affairs Specialist
Lexington MA
United States
------------------------------

Hi Masa,

You're welcome, I'm glad it was helpful!

As for the submission date, I would plan for no later than 12/22 (or maybe 12/23/19 since 12/22/19 is a weekend this year), but you can always submit earlier.  Keep in mind that the end of the reporting window/data cutoff date in your case will be on 10/21/2019 even if the submission is due in December, so if you have all your ducks in a row you could submit as early as 10/22/19.  That being said, I don't think the OOPD is very strict in enforcing the annual reporting deadlines - I've seen a program be 3 years delinquent in submitting an orphan annual report and the OOPD never said a word.  Of course, we should never plan to do this!

Cheers,
Marissa

------------------------------
Marissa Berry, Ph.D., RAC
Manager, Global Regulatory Affairs
Cary, NC
United States
------------------------------

Original Message:
Sent: 18-Jun-2019 16:28
From: Masa Razic
Subject: Orphan Drug Designation-Annual report

Hi Marissa,

Information you have provided have been extremely helpful.

Thank you so much. 

Our drug got designated on 10/22/2018. It said that I need to submit Annual report within 14 months. Would I target my submission on 12/22/2019 exact date, or?

Thank you so much for templates and everything-Regulatory is really challenging especially when I am on beginning of the beginning :)

Masa

Maša Razić

Regulatory Affairs Specialist

m:339.223.7784 I masa.razic@partnertx.com 

PARTNER THERAPEUTICS

Original Message------

Hi Masa,

I've submitted several Orphan Annual Reports. There is no form or template for the US - you basically just create a document that covers the annual reporting requirements (21 CFR 316.30) and e-mail it to OOPD (OPDAR@fda.hhs.gov). While the original orphan drug designation must be submitted on a compact disk and mailed to OOPD, annual reports should be e-mailed (the FDA website specifies that these should be e-mailed).
https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/corresponding-oopd-drug-designation-programs

The EMA used to have an orphan annual report template that covered the same topics requested by the FDA (and more). If you have orphan designation in both the US and EU, you could fill out the EMA template and send it to both agencies. However, the EMA has since updated their orphan annual reporting process so the template is no longer easy to find and actually isn't required anymore. I've attached it here, along with the corresponding EMA guidance, in case you may find it helpful for what to include in your US report. Click around on the EMA form - more spaces appear depending on what you click.

------------------------------
Marissa Berry, Ph.D., RAC
Manager, Global Regulatory Affairs
Cary, NC
United States

Original Message:
Sent: 07-Jun-2019 14:55
From: Masa Razic
Subject: Orphan Drug Designation-Annual report

Hi Colleagues,

According to the Orphan Drug Designation guidance, after you get designated for certain subset, you are supposed to submit AR for that Orphan Drug Designation within 14 months of approval. If someone as experienced, please provide what and how this request needs to be done.

Thanks,

Masa


------------------------------
Masa Razic
Regulatory Affairs Specialist
Lexington MA
United States
------------------------------