Regulatory Open Forum

This message was posted by a user wishing to remain anonymous

Hello all, 

I would appreciate your help and advice on the following. 

we are working on the clinical evaluation of a new IVD device that has no predicate available on the market. the device as per intended use will be for diagnosis of a rare disease. clinical/ patient samples required to run clinical studies are very hard to provide due the low prevalence of the disease and also the stability of the sample. is there any alternative to patient sample that we can use? is there any guidance explaining such situation that i can look at. 

Kind Regards, 
Asma

Hi Asma,

Since your disease is rare, your sample size should be low and manageable. For sample stability, you should employ suitable lab technique to enhance this.

PS: Since your device seems novel and you mentioned that no equivalent devices are available, their data cant be used. Of course you can use similar device clinical data but it depends upon NB to accept it or not depending upon closeness of technology, risk benefit ratio and clinical utility.

Hope this helps
Rahul

------------------------------
Rahul Pandey
PhD
New Delhi
India
------------------------------

Original Message:
Sent: 15-Nov-2022 10:57
From: Anonymous Member
Subject: Clinical Performance for IVD

This message was posted by a user wishing to remain anonymous

Hello all,

I would appreciate your help and advice on the following.

we are working on the clinical evaluation of a new IVD device that has no predicate available on the market. the device as per intended use will be for diagnosis of a rare disease. clinical/ patient samples required to run clinical studies are very hard to provide due the low prevalence of the disease and also the stability of the sample. is there any alternative to patient sample that we can use? is there any guidance explaining such situation that i can look at.

Kind Regards,
Asma

This message was posted by a user wishing to remain anonymous

Hi Rahul, 

thank you so much for your reply. 

as it is a rare disease it is not easy to get samples for clinical study. can we use simulated samples/ QC panel instead? is there any reference/standard that we can rely on to explain use of such alternative? 

thanks, Asma
Original Message:
Sent: 16-Nov-2022 01:08
From: Rahul Pandey
Subject: Clinical Performance for IVD

Hi Asma,

Since your disease is rare, your sample size should be low and manageable. For sample stability, you should employ suitable lab technique to enhance this.

PS: Since your device seems novel and you mentioned that no equivalent devices are available, their data cant be used. Of course you can use similar device clinical data but it depends upon NB to accept it or not depending upon closeness of technology, risk benefit ratio and clinical utility.

Hope this helps
Rahul

------------------------------
Rahul Pandey
PhD
New Delhi
India

Original Message:
Sent: 15-Nov-2022 10:57
From: Anonymous Member
Subject: Clinical Performance for IVD

This message was posted by a user wishing to remain anonymous

Hello all,

I would appreciate your help and advice on the following.

we are working on the clinical evaluation of a new IVD device that has no predicate available on the market. the device as per intended use will be for diagnosis of a rare disease. clinical/ patient samples required to run clinical studies are very hard to provide due the low prevalence of the disease and also the stability of the sample. is there any alternative to patient sample that we can use? is there any guidance explaining such situation that i can look at.

Kind Regards,
Asma

Hello Asma,

The Clinical Laboratory Standards Institute (clsi.org) document EP39: A Hierarchical Approach to Selecting Surrogate Samples for the Evaluation of In Vitro Medical Laboratory Tests will likely be very helpful for you. It walks through the thought process and options for selecting surrogate samples when validating an IVD. I'm not sure which regulatory agency you interact with, but it is recognized by FDA as a consensus standard as well. 

https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfStandards/detail.cfm?standard__identification_no=42930

------------------------------
Shannon Bennett
Director of Regulatory Affairs
Rochester MN
United States
------------------------------

Original Message:
Sent: 16-Nov-2022 04:28
From: Anonymous Member
Subject: Clinical Performance for IVD

This message was posted by a user wishing to remain anonymous

Hi Rahul,

thank you so much for your reply.

as it is a rare disease it is not easy to get samples for clinical study. can we use simulated samples/ QC panel instead? is there any reference/standard that we can rely on to explain use of such alternative?

thanks, Asma
Original Message:
Sent: 16-Nov-2022 01:08
From: Rahul Pandey
Subject: Clinical Performance for IVD

Hi Asma,

Since your disease is rare, your sample size should be low and manageable. For sample stability, you should employ suitable lab technique to enhance this.

PS: Since your device seems novel and you mentioned that no equivalent devices are available, their data cant be used. Of course you can use similar device clinical data but it depends upon NB to accept it or not depending upon closeness of technology, risk benefit ratio and clinical utility.

Hope this helps
Rahul

------------------------------
Rahul Pandey
PhD
New Delhi
India

Original Message:
Sent: 15-Nov-2022 10:57
From: Anonymous Member
Subject: Clinical Performance for IVD

This message was posted by a user wishing to remain anonymous

Hello all,

I would appreciate your help and advice on the following.

we are working on the clinical evaluation of a new IVD device that has no predicate available on the market. the device as per intended use will be for diagnosis of a rare disease. clinical/ patient samples required to run clinical studies are very hard to provide due the low prevalence of the disease and also the stability of the sample. is there any alternative to patient sample that we can use? is there any guidance explaining such situation that i can look at.

Kind Regards,
Asma

This message was posted by a user wishing to remain anonymous

Hi Shannon, 

Thank you very much for your reply.

This is a very useful standard and has lots of info that I was looking for. I appreciate sharing this!

Kind Regards, Asam


Original Message:
Sent: 16-Nov-2022 09:07
From: Shannon Bennett
Subject: Clinical Performance for IVD

Hello Asma,

The Clinical Laboratory Standards Institute (clsi.org) document EP39: A Hierarchical Approach to Selecting Surrogate Samples for the Evaluation of In Vitro Medical Laboratory Tests will likely be very helpful for you. It walks through the thought process and options for selecting surrogate samples when validating an IVD. I'm not sure which regulatory agency you interact with, but it is recognized by FDA as a consensus standard as well.

https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfStandards/detail.cfm?standard__identification_no=42930

------------------------------
Shannon Bennett
Director of Regulatory Affairs
Rochester MN
United States

Original Message:
Sent: 16-Nov-2022 04:28
From: Anonymous Member
Subject: Clinical Performance for IVD

This message was posted by a user wishing to remain anonymous

Hi Rahul,

thank you so much for your reply.

as it is a rare disease it is not easy to get samples for clinical study. can we use simulated samples/ QC panel instead? is there any reference/standard that we can rely on to explain use of such alternative?

thanks, Asma
Original Message:
Sent: 16-Nov-2022 01:08
From: Rahul Pandey
Subject: Clinical Performance for IVD

Hi Asma,

Since your disease is rare, your sample size should be low and manageable. For sample stability, you should employ suitable lab technique to enhance this.

PS: Since your device seems novel and you mentioned that no equivalent devices are available, their data cant be used. Of course you can use similar device clinical data but it depends upon NB to accept it or not depending upon closeness of technology, risk benefit ratio and clinical utility.

Hope this helps
Rahul

------------------------------
Rahul Pandey
PhD
New Delhi
India

Original Message:
Sent: 15-Nov-2022 10:57
From: Anonymous Member
Subject: Clinical Performance for IVD

This message was posted by a user wishing to remain anonymous

Hello all,

I would appreciate your help and advice on the following.

we are working on the clinical evaluation of a new IVD device that has no predicate available on the market. the device as per intended use will be for diagnosis of a rare disease. clinical/ patient samples required to run clinical studies are very hard to provide due the low prevalence of the disease and also the stability of the sample. is there any alternative to patient sample that we can use? is there any guidance explaining such situation that i can look at.

Kind Regards,
Asma

This message was posted by a user wishing to remain anonymous

Hi Shannon, 

Thank you very much for your help. This is what I was looking for. Very useful CLSI. Really appreciated!
Original Message:
Sent: 16-Nov-2022 09:07
From: Shannon Bennett
Subject: Clinical Performance for IVD

Hello Asma,

The Clinical Laboratory Standards Institute (clsi.org) document EP39: A Hierarchical Approach to Selecting Surrogate Samples for the Evaluation of In Vitro Medical Laboratory Tests will likely be very helpful for you. It walks through the thought process and options for selecting surrogate samples when validating an IVD. I'm not sure which regulatory agency you interact with, but it is recognized by FDA as a consensus standard as well.

https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfStandards/detail.cfm?standard__identification_no=42930

------------------------------
Shannon Bennett
Director of Regulatory Affairs
Rochester MN
United States

Original Message:
Sent: 16-Nov-2022 04:28
From: Anonymous Member
Subject: Clinical Performance for IVD

This message was posted by a user wishing to remain anonymous

Hi Rahul,

thank you so much for your reply.

as it is a rare disease it is not easy to get samples for clinical study. can we use simulated samples/ QC panel instead? is there any reference/standard that we can rely on to explain use of such alternative?

thanks, Asma
Original Message:
Sent: 16-Nov-2022 01:08
From: Rahul Pandey
Subject: Clinical Performance for IVD

Hi Asma,

Since your disease is rare, your sample size should be low and manageable. For sample stability, you should employ suitable lab technique to enhance this.

PS: Since your device seems novel and you mentioned that no equivalent devices are available, their data cant be used. Of course you can use similar device clinical data but it depends upon NB to accept it or not depending upon closeness of technology, risk benefit ratio and clinical utility.

Hope this helps
Rahul

------------------------------
Rahul Pandey
PhD
New Delhi
India

Original Message:
Sent: 15-Nov-2022 10:57
From: Anonymous Member
Subject: Clinical Performance for IVD

This message was posted by a user wishing to remain anonymous

Hello all,

I would appreciate your help and advice on the following.

we are working on the clinical evaluation of a new IVD device that has no predicate available on the market. the device as per intended use will be for diagnosis of a rare disease. clinical/ patient samples required to run clinical studies are very hard to provide due the low prevalence of the disease and also the stability of the sample. is there any alternative to patient sample that we can use? is there any guidance explaining such situation that i can look at.

Kind Regards,
Asma

Hi Asma,

As Sharon suggested, do review the CLSI document. I would also suggest that you reach out to your regulatory agency early on. For example, you can request a pre-submission meeting with the US FDA. It will be helpful, in case you are looking at a De Novo classification process. You can also seek clarification on the acceptable surrogate sample, especially because your physiological sample is not very stable.

Good luck

Ashwin

------------------------------
Ashwin Nair, PhD
TX, USA
------------------------------

Original Message:
Sent: 15-Nov-2022 10:57
From: Anonymous Member
Subject: Clinical Performance for IVD

This message was posted by a user wishing to remain anonymous

Hello all,

I would appreciate your help and advice on the following.

we are working on the clinical evaluation of a new IVD device that has no predicate available on the market. the device as per intended use will be for diagnosis of a rare disease. clinical/ patient samples required to run clinical studies are very hard to provide due the low prevalence of the disease and also the stability of the sample. is there any alternative to patient sample that we can use? is there any guidance explaining such situation that i can look at.

Kind Regards,
Asma