Regulatory Open Forum

Dear all,

I am doing some research around Written Request for voluntary pediatric studies. I understand that a sponsor can request a Proposed Pediatric Study Request (PPSR) in order to have a Written Request issued by the FDA. As the incentive is pediatric exclusivity, there is no real rush to do that upfront but I was wondering if this is possible to initiate the action at the time of the IND? Or is it better to do it once the NDA is cleared?

Thank you,
Best Regards

------------------------------
Charlene Revel
Associate Director, Regulatory Science
Cambridge MA
United States
------------------------------

Hi Charlene,

You can submit the PPSR at any time. Whether you do it during the IND phase or post-marketing it would still be helpful, and preferred by FDA, to have a discussion with the review division as early in development as possible.

------------------------------
Glen Park PharmD
Vice President, Regulatory Affairs and Quality Assurance
New York NY
United States
------------------------------

Original Message:
Sent: 24-Jan-2022 15:28
From: Charlene Revel
Subject: BPCA Pediatric voluntary studies

Dear all,

I am doing some research around Written Request for voluntary pediatric studies. I understand that a sponsor can request a Proposed Pediatric Study Request (PPSR) in order to have a Written Request issued by the FDA. As the incentive is pediatric exclusivity, there is no real rush to do that upfront but I was wondering if this is possible to initiate the action at the time of the IND? Or is it better to do it once the NDA is cleared?

Thank you,
Best Regards

------------------------------
Charlene Revel
Associate Director, Regulatory Science
Cambridge MA
United States
------------------------------

Thank you, Charlene for starting the thread, and Glen for weighing in on the topic.

I am interested in learning more about global pediatric drug development and was wondering, if you're planning to pursue a pediatric indication and hoping for a WR from FDA, would you want to wait until the NDA to begin the discussion? If I understand the regulation on this, PREA obligates the sponsor to submit the PSP in order to clear the NDA. 

A follow-up question, could you utilize the Type F meeting to discuss pediatric plans? 

I don't have direct experience with WRs so appreciate the discussion and input. 

Olesya

------------------------------
Olesya Jaganathan
Cambridge MA
United States
------------------------------

Original Message:
Sent: 25-Jan-2022 07:35
From: Glen Park
Subject: BPCA Pediatric voluntary studies

Hi Charlene,

You can submit the PPSR at any time. Whether you do it during the IND phase or post-marketing it would still be helpful, and preferred by FDA, to have a discussion with the review division as early in development as possible.

------------------------------
Glen Park PharmD
Vice President, Regulatory Affairs and Quality Assurance
New York NY
United States

Original Message:
Sent: 24-Jan-2022 15:28
From: Charlene Revel
Subject: BPCA Pediatric voluntary studies

Dear all,

I am doing some research around Written Request for voluntary pediatric studies. I understand that a sponsor can request a Proposed Pediatric Study Request (PPSR) in order to have a Written Request issued by the FDA. As the incentive is pediatric exclusivity, there is no real rush to do that upfront but I was wondering if this is possible to initiate the action at the time of the IND? Or is it better to do it once the NDA is cleared?

Thank you,
Best Regards

------------------------------
Charlene Revel
Associate Director, Regulatory Science
Cambridge MA
United States
------------------------------

Thank you Glen for your input, this is useful. 

Hi Olesya,
For this specific case, this is an Orphan drug so they are exempt from PSP requirements. Also, they already included some pediatric groups in their ongoing studies. They are mostly interested in the pediatric incentives, but I agree with you if they plan to perform pediatric studies for exclusivity to support their indication, then it has to be discussed upfront in their development rather than post-marketing.

------------------------------
Charlene Revel
Associate Director, Regulatory Science
Cambridge MA
United States
------------------------------

Original Message:
Sent: 25-Jan-2022 08:33
From: Olesya Jaganathan
Subject: BPCA Pediatric voluntary studies

Thank you, Charlene for starting the thread, and Glen for weighing in on the topic.

I am interested in learning more about global pediatric drug development and was wondering, if you're planning to pursue a pediatric indication and hoping for a WR from FDA, would you want to wait until the NDA to begin the discussion? If I understand the regulation on this, PREA obligates the sponsor to submit the PSP in order to clear the NDA.

A follow-up question, could you utilize the Type F meeting to discuss pediatric plans?

I don't have direct experience with WRs so appreciate the discussion and input.

Olesya

------------------------------
Olesya Jaganathan
Cambridge MA
United States

Original Message:
Sent: 25-Jan-2022 07:35
From: Glen Park
Subject: BPCA Pediatric voluntary studies

Hi Charlene,

You can submit the PPSR at any time. Whether you do it during the IND phase or post-marketing it would still be helpful, and preferred by FDA, to have a discussion with the review division as early in development as possible.

------------------------------
Glen Park PharmD
Vice President, Regulatory Affairs and Quality Assurance
New York NY
United States

Original Message:
Sent: 24-Jan-2022 15:28
From: Charlene Revel
Subject: BPCA Pediatric voluntary studies

Dear all,

I am doing some research around Written Request for voluntary pediatric studies. I understand that a sponsor can request a Proposed Pediatric Study Request (PPSR) in order to have a Written Request issued by the FDA. As the incentive is pediatric exclusivity, there is no real rush to do that upfront but I was wondering if this is possible to initiate the action at the time of the IND? Or is it better to do it once the NDA is cleared?

Thank you,
Best Regards

------------------------------
Charlene Revel
Associate Director, Regulatory Science
Cambridge MA
United States
------------------------------

This message was posted by a user wishing to remain anonymous

Thank you for this thread. I have a related question.

My company is developing a drug for 2 orphan indications, 1of which is exclusively in pediatric populations. Based on PREA, as an drug with an orphan designation in this indications, the program is exempt from the requirement to submit an iPSP. 

However, the guidance on PSPs (https://www.fda.gov/regulatory-information/search-fda-guidance-documents/pediatric-study-plans-content-and-process-submitting-initial-pediatric-study-plans-and-amended), also states on pg 4, that "for drugs that are being developed specifically for use in pediatric populations, the sponsor should submit an iPSP".

As an orphan drug being developed specifially for use in pediatric populations, is our program still exempt from the requirement to submit an iPSP?

My read is yes, since the orphan exemption is clearly written in the law [505B(k)(1) of the FD&C Act; 21 U.S.C. 355c(k)(1)], while the requirement for an iPSP for exclusively pediatric populations is not. But I'm curious to hear if any of you have experience with this situation.

Thanks!
Original Message:
Sent: 25-Jan-2022 08:41
From: Charlene Revel
Subject: BPCA Pediatric voluntary studies

Thank you Glen for your input, this is useful.

Hi Olesya,
For this specific case, this is an Orphan drug so they are exempt from PSP requirements. Also, they already included some pediatric groups in their ongoing studies. They are mostly interested in the pediatric incentives, but I agree with you if they plan to perform pediatric studies for exclusivity to support their indication, then it has to be discussed upfront in their development rather than post-marketing.

------------------------------
Charlene Revel
Associate Director, Regulatory Science
Cambridge MA
United States

Original Message:
Sent: 25-Jan-2022 08:33
From: Olesya Jaganathan
Subject: BPCA Pediatric voluntary studies

Thank you, Charlene for starting the thread, and Glen for weighing in on the topic.

I am interested in learning more about global pediatric drug development and was wondering, if you're planning to pursue a pediatric indication and hoping for a WR from FDA, would you want to wait until the NDA to begin the discussion? If I understand the regulation on this, PREA obligates the sponsor to submit the PSP in order to clear the NDA.

A follow-up question, could you utilize the Type F meeting to discuss pediatric plans?

I don't have direct experience with WRs so appreciate the discussion and input.

Olesya

------------------------------
Olesya Jaganathan
Cambridge MA
United States

Original Message:
Sent: 25-Jan-2022 07:35
From: Glen Park
Subject: BPCA Pediatric voluntary studies

Hi Charlene,

You can submit the PPSR at any time. Whether you do it during the IND phase or post-marketing it would still be helpful, and preferred by FDA, to have a discussion with the review division as early in development as possible.

------------------------------
Glen Park PharmD
Vice President, Regulatory Affairs and Quality Assurance
New York NY
United States

Original Message:
Sent: 24-Jan-2022 15:28
From: Charlene Revel
Subject: BPCA Pediatric voluntary studies

Dear all,

I am doing some research around Written Request for voluntary pediatric studies. I understand that a sponsor can request a Proposed Pediatric Study Request (PPSR) in order to have a Written Request issued by the FDA. As the incentive is pediatric exclusivity, there is no real rush to do that upfront but I was wondering if this is possible to initiate the action at the time of the IND? Or is it better to do it once the NDA is cleared?

Thank you,
Best Regards

------------------------------
Charlene Revel
Associate Director, Regulatory Science
Cambridge MA
United States
------------------------------

I would agree with your assessment that the orphan exemption takes precedence over PREA, but it is easy to confirm that with your PM. Of course, we are only talking about the US and not EU where you would have to still do a PIP.

------------------------------
Glen Park PharmD
Vice President, Regulatory Affairs and Quality Assurance
New York NY
United States
------------------------------

Original Message:
Sent: 26-Jan-2022 08:57
From: Anonymous Member
Subject: BPCA Pediatric voluntary studies

This message was posted by a user wishing to remain anonymous

Thank you for this thread. I have a related question.

My company is developing a drug for 2 orphan indications, 1of which is exclusively in pediatric populations. Based on PREA, as an drug with an orphan designation in this indications, the program is exempt from the requirement to submit an iPSP.

However, the guidance on PSPs (https://www.fda.gov/regulatory-information/search-fda-guidance-documents/pediatric-study-plans-content-and-process-submitting-initial-pediatric-study-plans-and-amended), also states on pg 4, that "for drugs that are being developed specifically for use in pediatric populations, the sponsor should submit an iPSP".

As an orphan drug being developed specifially for use in pediatric populations, is our program still exempt from the requirement to submit an iPSP?

My read is yes, since the orphan exemption is clearly written in the law [505B(k)(1) of the FD&C Act; 21 U.S.C. 355c(k)(1)], while the requirement for an iPSP for exclusively pediatric populations is not. But I'm curious to hear if any of you have experience with this situation.

Thanks!
Original Message:
Sent: 25-Jan-2022 08:41
From: Charlene Revel
Subject: BPCA Pediatric voluntary studies

Thank you Glen for your input, this is useful.

Hi Olesya,
For this specific case, this is an Orphan drug so they are exempt from PSP requirements. Also, they already included some pediatric groups in their ongoing studies. They are mostly interested in the pediatric incentives, but I agree with you if they plan to perform pediatric studies for exclusivity to support their indication, then it has to be discussed upfront in their development rather than post-marketing.

------------------------------
Charlene Revel
Associate Director, Regulatory Science
Cambridge MA
United States

Original Message:
Sent: 25-Jan-2022 08:33
From: Olesya Jaganathan
Subject: BPCA Pediatric voluntary studies

Thank you, Charlene for starting the thread, and Glen for weighing in on the topic.

I am interested in learning more about global pediatric drug development and was wondering, if you're planning to pursue a pediatric indication and hoping for a WR from FDA, would you want to wait until the NDA to begin the discussion? If I understand the regulation on this, PREA obligates the sponsor to submit the PSP in order to clear the NDA.

A follow-up question, could you utilize the Type F meeting to discuss pediatric plans?

I don't have direct experience with WRs so appreciate the discussion and input.

Olesya

------------------------------
Olesya Jaganathan
Cambridge MA
United States

Original Message:
Sent: 25-Jan-2022 07:35
From: Glen Park
Subject: BPCA Pediatric voluntary studies

Hi Charlene,

You can submit the PPSR at any time. Whether you do it during the IND phase or post-marketing it would still be helpful, and preferred by FDA, to have a discussion with the review division as early in development as possible.

------------------------------
Glen Park PharmD
Vice President, Regulatory Affairs and Quality Assurance
New York NY
United States

Original Message:
Sent: 24-Jan-2022 15:28
From: Charlene Revel
Subject: BPCA Pediatric voluntary studies

Dear all,

I am doing some research around Written Request for voluntary pediatric studies. I understand that a sponsor can request a Proposed Pediatric Study Request (PPSR) in order to have a Written Request issued by the FDA. As the incentive is pediatric exclusivity, there is no real rush to do that upfront but I was wondering if this is possible to initiate the action at the time of the IND? Or is it better to do it once the NDA is cleared?

Thank you,
Best Regards

------------------------------
Charlene Revel
Associate Director, Regulatory Science
Cambridge MA
United States
------------------------------

This message was posted by a user wishing to remain anonymous

Thank you, Glen!
I will definitely run this by our RPM. We had written the exemption into a briefing package (in the common sections at the front of the document), but still received boilerplate language about the PSP in our meeting minutes, which got us wondering and delving deeper.
And, yes, PIPs are in the works.
Thank you!
Original Message:
Sent: 27-Jan-2022 07:49
From: Glen Park
Subject: BPCA Pediatric voluntary studies

I would agree with your assessment that the orphan exemption takes precedence over PREA, but it is easy to confirm that with your PM. Of course, we are only talking about the US and not EU where you would have to still do a PIP.

------------------------------
Glen Park PharmD
Vice President, Regulatory Affairs and Quality Assurance
New York NY
United States

Original Message:
Sent: 26-Jan-2022 08:57
From: Anonymous Member
Subject: BPCA Pediatric voluntary studies

This message was posted by a user wishing to remain anonymous

Thank you for this thread. I have a related question.

My company is developing a drug for 2 orphan indications, 1of which is exclusively in pediatric populations. Based on PREA, as an drug with an orphan designation in this indications, the program is exempt from the requirement to submit an iPSP.

However, the guidance on PSPs (https://www.fda.gov/regulatory-information/search-fda-guidance-documents/pediatric-study-plans-content-and-process-submitting-initial-pediatric-study-plans-and-amended), also states on pg 4, that "for drugs that are being developed specifically for use in pediatric populations, the sponsor should submit an iPSP".

As an orphan drug being developed specifially for use in pediatric populations, is our program still exempt from the requirement to submit an iPSP?

My read is yes, since the orphan exemption is clearly written in the law [505B(k)(1) of the FD&C Act; 21 U.S.C. 355c(k)(1)], while the requirement for an iPSP for exclusively pediatric populations is not. But I'm curious to hear if any of you have experience with this situation.

Thanks!
Original Message:
Sent: 25-Jan-2022 08:41
From: Charlene Revel
Subject: BPCA Pediatric voluntary studies

Thank you Glen for your input, this is useful.

Hi Olesya,
For this specific case, this is an Orphan drug so they are exempt from PSP requirements. Also, they already included some pediatric groups in their ongoing studies. They are mostly interested in the pediatric incentives, but I agree with you if they plan to perform pediatric studies for exclusivity to support their indication, then it has to be discussed upfront in their development rather than post-marketing.

------------------------------
Charlene Revel
Associate Director, Regulatory Science
Cambridge MA
United States

Original Message:
Sent: 25-Jan-2022 08:33
From: Olesya Jaganathan
Subject: BPCA Pediatric voluntary studies

Thank you, Charlene for starting the thread, and Glen for weighing in on the topic.

I am interested in learning more about global pediatric drug development and was wondering, if you're planning to pursue a pediatric indication and hoping for a WR from FDA, would you want to wait until the NDA to begin the discussion? If I understand the regulation on this, PREA obligates the sponsor to submit the PSP in order to clear the NDA.

A follow-up question, could you utilize the Type F meeting to discuss pediatric plans?

I don't have direct experience with WRs so appreciate the discussion and input.

Olesya

------------------------------
Olesya Jaganathan
Cambridge MA
United States

Original Message:
Sent: 25-Jan-2022 07:35
From: Glen Park
Subject: BPCA Pediatric voluntary studies

Hi Charlene,

You can submit the PPSR at any time. Whether you do it during the IND phase or post-marketing it would still be helpful, and preferred by FDA, to have a discussion with the review division as early in development as possible.

------------------------------
Glen Park PharmD
Vice President, Regulatory Affairs and Quality Assurance
New York NY
United States

Original Message:
Sent: 24-Jan-2022 15:28
From: Charlene Revel
Subject: BPCA Pediatric voluntary studies

Dear all,

I am doing some research around Written Request for voluntary pediatric studies. I understand that a sponsor can request a Proposed Pediatric Study Request (PPSR) in order to have a Written Request issued by the FDA. As the incentive is pediatric exclusivity, there is no real rush to do that upfront but I was wondering if this is possible to initiate the action at the time of the IND? Or is it better to do it once the NDA is cleared?

Thank you,
Best Regards

------------------------------
Charlene Revel
Associate Director, Regulatory Science
Cambridge MA
United States
------------------------------

Hi All, 

        Great discussion. If you are working on an oncology drug, though pediatric studies are waived because of orphan designation, you are still required to conduct them if your drug appears on the "relevant molecular target list" (due to the recent amendments of FDARA 2017 to PREA - see link: https://www.fda.gov/about-fda/oncology-center-excellence/pediatric-oncology)

------------------------------
Michael Bui
Vice President, Regulatory Affairs
Arlington MA
United States
------------------------------

Original Message:
Sent: 24-Jan-2022 15:28
From: Charlene Revel
Subject: BPCA Pediatric voluntary studies

Dear all,

I am doing some research around Written Request for voluntary pediatric studies. I understand that a sponsor can request a Proposed Pediatric Study Request (PPSR) in order to have a Written Request issued by the FDA. As the incentive is pediatric exclusivity, there is no real rush to do that upfront but I was wondering if this is possible to initiate the action at the time of the IND? Or is it better to do it once the NDA is cleared?

Thank you,
Best Regards

------------------------------
Charlene Revel
Associate Director, Regulatory Science
Cambridge MA
United States
------------------------------

This message was posted by a user wishing to remain anonymous

Good reminder, Michael! 
In our case it's non-oncology. 
Thanks!
Original Message:
Sent: 27-Jan-2022 08:24
From: Michael Bui
Subject: BPCA Pediatric voluntary studies

Hi All,

        Great discussion. If you are working on an oncology drug, though pediatric studies are waived because of orphan designation, you are still required to conduct them if your drug appears on the "relevant molecular target list" (due to the recent amendments of FDARA 2017 to PREA - see link: https://www.fda.gov/about-fda/oncology-center-excellence/pediatric-oncology)

------------------------------
Michael Bui
Vice President, Regulatory Affairs
Arlington MA
United States

Original Message:
Sent: 24-Jan-2022 15:28
From: Charlene Revel
Subject: BPCA Pediatric voluntary studies

Dear all,

I am doing some research around Written Request for voluntary pediatric studies. I understand that a sponsor can request a Proposed Pediatric Study Request (PPSR) in order to have a Written Request issued by the FDA. As the incentive is pediatric exclusivity, there is no real rush to do that upfront but I was wondering if this is possible to initiate the action at the time of the IND? Or is it better to do it once the NDA is cleared?

Thank you,
Best Regards

------------------------------
Charlene Revel
Associate Director, Regulatory Science
Cambridge MA
United States
------------------------------