Regulatory Open Forum

Hi all,

I am working on a project with a client who wishes to consult FDA on his development protocol. The client has put together a synopsis for his study however they have included two different options with different endpoints /sample size calculations one for accelerated approval and one for "normal" pathway. 

In my experience with other agencies, there should be one clear strategy and one "protocol" to discuss during the consultation. Does this apply to FDA as well or is FDA open to discuss multiple protocol scenarios in a consultation? 

Thanks

My strategy has always been to propose to a regulatory agency the regulatory pathway that the client wants to do as the primary preference. Present the argument for that pathway, justify it, and then ask for agreement. Of course, also be ready to discuss the alternative pathway.  However, my experience with FDA is that I don't think they would revert to the "normal" pathway by default without seriously considering the accelerated pathway if that would be the pathway they could accept. So, I don't see any downsides to presenting both possibilities, particularly if the program qualified for the accelerated pathway as an unmet medical need.

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Glen Park PharmD
Consultant
Burien WA
United States

Original Message:
Sent: 22-Feb-2024 10:08
From: Anonymous Member
Subject: FDA pre-IND meeting

This message was posted by a user wishing to remain anonymous

Hi all,

I am working on a project with a client who wishes to consult FDA on his development protocol. The client has put together a synopsis for his study however they have included two different options with different endpoints /sample size calculations one for accelerated approval and one for "normal" pathway. 

In my experience with other agencies, there should be one clear strategy and one "protocol" to discuss during the consultation. Does this apply to FDA as well or is FDA open to discuss multiple protocol scenarios in a consultation? 

Thanks

Thank you Glen for your response. So would you attach to the meeting package two different protocol synopsis for the phase 3 confirmatory trial? One for the accelerated approval and one for the standard pathway? 

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


Original Message:
Sent: 23-Feb-2024 07:08
From: Glen Park
Subject: FDA pre-IND meeting

My strategy has always been to propose to a regulatory agency the regulatory pathway that the client wants to do as the primary preference. Present the argument for that pathway, justify it, and then ask for agreement. Of course, also be ready to discuss the alternative pathway.  However, my experience with FDA is that I don't think they would revert to the "normal" pathway by default without seriously considering the accelerated pathway if that would be the pathway they could accept. So, I don't see any downsides to presenting both possibilities, particularly if the program qualified for the accelerated pathway as an unmet medical need.

------------------------------
Glen Park PharmD
Consultant
Burien WA
United States

Original Message:
Sent: 22-Feb-2024 10:08
From: Anonymous Member
Subject: FDA pre-IND meeting

This message was posted by a user wishing to remain anonymous

Hi all,

I am working on a project with a client who wishes to consult FDA on his development protocol. The client has put together a synopsis for his study however they have included two different options with different endpoints /sample size calculations one for accelerated approval and one for "normal" pathway. 

In my experience with other agencies, there should be one clear strategy and one "protocol" to discuss during the consultation. Does this apply to FDA as well or is FDA open to discuss multiple protocol scenarios in a consultation? 

Thanks

Hi all,

I am working on a project with a client who wishes to consult FDA on his development protocol. The client has put together a synopsis for his study however they have included two different options with different endpoints /sample size calculations one for accelerated approval and one for "normal" pathway. 

In my experience with other agencies, there should be one clear strategy and one "protocol" to discuss during the consultation. Does this apply to FDA as well or is FDA open to discuss multiple protocol scenarios in a consultation? 

Thanks

Proposing a primary plan (e.g., Accelerated Approval) and a back-up plan ("normal") is perfectly reasonable and I have taken this strategy previously a number of times.  This helps the company get regulatory information important for decision making in 1 agency interaction rather than having to go back for a follow-up discussion.

I would use 1 question on the design of the trial to support an Accelerated Approval pathway. Then, the subsequent question could be something like "If the Agency does not agree on an opportunity for Accelerated Approval, could the proposed design support a standard approval pathway?"  This way, if FDA believes AA is not supported by the company's development plan, the company can still get feedback on a pathway to registration.

DM me if you'd like to chat informally.

Good luck!

Hi all,

I am working on a project with a client who wishes to consult FDA on his development protocol. The client has put together a synopsis for his study however they have included two different options with different endpoints /sample size calculations one for accelerated approval and one for "normal" pathway. 

In my experience with other agencies, there should be one clear strategy and one "protocol" to discuss during the consultation. Does this apply to FDA as well or is FDA open to discuss multiple protocol scenarios in a consultation? 

Thanks

Dear Mark,

Thank you for your response.

So would you present two different trial designs for a meeting (actually, this is what client is planning). Spontaneously, I would stick to one study design for the development program and ask if it supports accelerated approval and complete with the question you propose above: "If the Agency does not agree on an opportunity for Accelerated Approval, could the proposed design support a standard approval pathway?" Does this make sense to you? 

Thanks

Proposing a primary plan (e.g., Accelerated Approval) and a back-up plan ("normal") is perfectly reasonable and I have taken this strategy previously a number of times.  This helps the company get regulatory information important for decision making in 1 agency interaction rather than having to go back for a follow-up discussion.

I would use 1 question on the design of the trial to support an Accelerated Approval pathway. Then, the subsequent question could be something like "If the Agency does not agree on an opportunity for Accelerated Approval, could the proposed design support a standard approval pathway?"  This way, if FDA believes AA is not supported by the company's development plan, the company can still get feedback on a pathway to registration.

DM me if you'd like to chat informally.

Good luck!

Hi all,

I am working on a project with a client who wishes to consult FDA on his development protocol. The client has put together a synopsis for his study however they have included two different options with different endpoints /sample size calculations one for accelerated approval and one for "normal" pathway. 

In my experience with other agencies, there should be one clear strategy and one "protocol" to discuss during the consultation. Does this apply to FDA as well or is FDA open to discuss multiple protocol scenarios in a consultation? 

Thanks

Dear Mark,

Thank you for your response.

So would you present two different trial designs for a meeting (actually, this is what client is planning). Spontaneously, I would stick to one study design for the development program and ask if it supports accelerated approval and complete with the question you propose above: "If the Agency does not agree on an opportunity for Accelerated Approval, could the proposed design support a standard approval pathway?" Does this make sense to you? 

Thanks

Proposing a primary plan (e.g., Accelerated Approval) and a back-up plan ("normal") is perfectly reasonable and I have taken this strategy previously a number of times.  This helps the company get regulatory information important for decision making in 1 agency interaction rather than having to go back for a follow-up discussion.

I would use 1 question on the design of the trial to support an Accelerated Approval pathway. Then, the subsequent question could be something like "If the Agency does not agree on an opportunity for Accelerated Approval, could the proposed design support a standard approval pathway?"  This way, if FDA believes AA is not supported by the company's development plan, the company can still get feedback on a pathway to registration.

DM me if you'd like to chat informally.

Good luck!

Hi all,

I am working on a project with a client who wishes to consult FDA on his development protocol. The client has put together a synopsis for his study however they have included two different options with different endpoints /sample size calculations one for accelerated approval and one for "normal" pathway. 

In my experience with other agencies, there should be one clear strategy and one "protocol" to discuss during the consultation. Does this apply to FDA as well or is FDA open to discuss multiple protocol scenarios in a consultation? 

Thanks

Hi all,

I am working on a project with a client who wishes to consult FDA on his development protocol. The client has put together a synopsis for his study however they have included two different options with different endpoints /sample size calculations one for accelerated approval and one for "normal" pathway. 

In my experience with other agencies, there should be one clear strategy and one "protocol" to discuss during the consultation. Does this apply to FDA as well or is FDA open to discuss multiple protocol scenarios in a consultation? 

Thanks

To specifically answer your question, its not an issue presenting two different regulatory pathways for feedback, of course justified with rationale!

I am just curious it appears you are at a stage seeking FDA feedback on pivotal protocol for registration purposes, which means you already have active IND with early stage data, why would you call it 'Pre-IND meeting? You meant Type B?

Original Message:
Sent: 22-Feb-2024 10:08
From: Anonymous Member
Subject: FDA pre-IND meeting

This message was posted by a user wishing to remain anonymous

Hi all,

I am working on a project with a client who wishes to consult FDA on his development protocol. The client has put together a synopsis for his study however they have included two different options with different endpoints /sample size calculations one for accelerated approval and one for "normal" pathway. 

In my experience with other agencies, there should be one clear strategy and one "protocol" to discuss during the consultation. Does this apply to FDA as well or is FDA open to discuss multiple protocol scenarios in a consultation? 

Thanks

Hello,

Thanks for your message. No there is no early stage clinical data yet. The plan is to have a confirmatory two parts trial à run-in phase and a confirmation part. 

Original Message:
Sent: 23-Feb-2024 09:33
From: Anonymous Member
Subject: FDA pre-IND meeting

This message was posted by a user wishing to remain anonymous

To specifically answer your question, its not an issue presenting two different regulatory pathways for feedback, of course justified with rationale!

I am just curious it appears you are at a stage seeking FDA feedback on pivotal protocol for registration purposes, which means you already have active IND with early stage data, why would you call it 'Pre-IND meeting? You meant Type B?

Original Message:
Sent: 22-Feb-2024 10:08
From: Anonymous Member
Subject: FDA pre-IND meeting

This message was posted by a user wishing to remain anonymous

Hi all,

I am working on a project with a client who wishes to consult FDA on his development protocol. The client has put together a synopsis for his study however they have included two different options with different endpoints /sample size calculations one for accelerated approval and one for "normal" pathway. 

In my experience with other agencies, there should be one clear strategy and one "protocol" to discuss during the consultation. Does this apply to FDA as well or is FDA open to discuss multiple protocol scenarios in a consultation? 

Thanks

Hi all,

I am working on a project with a client who wishes to consult FDA on his development protocol. The client has put together a synopsis for his study however they have included two different options with different endpoints /sample size calculations one for accelerated approval and one for "normal" pathway. 

In my experience with other agencies, there should be one clear strategy and one "protocol" to discuss during the consultation. Does this apply to FDA as well or is FDA open to discuss multiple protocol scenarios in a consultation? 

Thanks