Regulatory Open Forum

We have recently received orphan drug designation from FDA and have been asked to "provide assurance of availability of sufficient quantities to meet the needs of patients under 21CFR316.36." This is the first time our company has received ODD, and we are not quite sure what they're looking for to provide this assurance. Is it sufficient to say that based on expected demand and manufacturing schedules, we are confident we can meet demand, or do they want to see those calculations? How far into the future would we need to go?

Thanks for your help.

OP here with a bit of clarification. Specifically, the letter from FDA states the following:

As the holder of exclusivity, the sponsor is required to assure the availability of sufficient quantities of this drug to meet the needs of patients. Failure to do so could result in the withdrawal of the drug's exclusive approval as stipulated under 21 CFR 316.36(b).

They give no further information on how they expect us to do this and no deadline is attached. Are they even expecting a response? Or are they just letting us know that if we should encounter a supply shortage during the exclusivity period, they could revoke it? We would appreciate any insight you might have.

We have recently received orphan drug designation from FDA and have been asked to "provide assurance of availability of sufficient quantities to meet the needs of patients under 21CFR316.36." This is the first time our company has received ODD, and we are not quite sure what they're looking for to provide this assurance. Is it sufficient to say that based on expected demand and manufacturing schedules, we are confident we can meet demand, or do they want to see those calculations? How far into the future would we need to go?

Thanks for your help.

You just said your drug received ODD but did not clarify whether the drug is approved? If you are still in the development and received ODD then this is just a standard language from the FDA. You can respond in your annual status update, nothing urgent if you are still in development!

OP here with a bit of clarification. Specifically, the letter from FDA states the following:

As the holder of exclusivity, the sponsor is required to assure the availability of sufficient quantities of this drug to meet the needs of patients. Failure to do so could result in the withdrawal of the drug's exclusive approval as stipulated under 21 CFR 316.36(b).

They give no further information on how they expect us to do this and no deadline is attached. Are they even expecting a response? Or are they just letting us know that if we should encounter a supply shortage during the exclusivity period, they could revoke it? We would appreciate any insight you might have.

We have recently received orphan drug designation from FDA and have been asked to "provide assurance of availability of sufficient quantities to meet the needs of patients under 21CFR316.36." This is the first time our company has received ODD, and we are not quite sure what they're looking for to provide this assurance. Is it sufficient to say that based on expected demand and manufacturing schedules, we are confident we can meet demand, or do they want to see those calculations? How far into the future would we need to go?

Thanks for your help.

Sorry, I was not clear. We have received the orphan drug exclusivity after approval. So not just the designation, but the exclusivity. Our question is, are the expecting us to send them some sort of documentation at this point? Or are they simply pointing out that, as stated in the CFR, we could lose the exclusivity if we cannot meet demand? 

You just said your drug received ODD but did not clarify whether the drug is approved? If you are still in the development and received ODD then this is just a standard language from the FDA. You can respond in your annual status update, nothing urgent if you are still in development!

OP here with a bit of clarification. Specifically, the letter from FDA states the following:

As the holder of exclusivity, the sponsor is required to assure the availability of sufficient quantities of this drug to meet the needs of patients. Failure to do so could result in the withdrawal of the drug's exclusive approval as stipulated under 21 CFR 316.36(b).

They give no further information on how they expect us to do this and no deadline is attached. Are they even expecting a response? Or are they just letting us know that if we should encounter a supply shortage during the exclusivity period, they could revoke it? We would appreciate any insight you might have.

We have recently received orphan drug designation from FDA and have been asked to "provide assurance of availability of sufficient quantities to meet the needs of patients under 21CFR316.36." This is the first time our company has received ODD, and we are not quite sure what they're looking for to provide this assurance. Is it sufficient to say that based on expected demand and manufacturing schedules, we are confident we can meet demand, or do they want to see those calculations? How far into the future would we need to go?

Thanks for your help.