Regulatory Open Forum

This message was posted by a user wishing to remain anonymous

Dear Colleagues,

One of the FDA definitions of unmet need for drugs and biologics, where there are available therapies, is

"[The treatment]...Provides efficacy comparable to those of available therapy, while (1) avoiding serious toxicity that occurs with available therapy, (2) avoiding less serious toxicity that is common and causes discontinuation of treatment of a serious condition, or (3) reducing the potential for harmful drug interactions"

My question for the hive mind is whether there is any precedent for a drug/biologic receiving Accelerated Approval specifically on this basis.

When you say "specifically on this basis," I am assuming you mean if all the other conditions for Accelerated approval are met.

This has been used to accelerate the study and review of products that haven't shown improved efficacy, but have shown improved safety.

I would have expected companies that received this designation to brag about it in press releases and stock market reports, but I see now why you had trouble finding that, because so did I!

Way down in the search I found these drugs and biologics who got the designation of addressing an unmet need for cancers, psoriasis, Parkinson's, etc. from companies like Amgen, Aurinia, Carrick, ProventionBio, Shionogi, and BlueRock Therapeutics. Perhaps one could be a good example for you.

Take a look at this CBER and CDER Guidance: Benefit-Risk Assessment for New Drug and Biological Products (Sept. 2021).
FAQs: Breakthrough Therapies

FYI, here is a document on unmet needs on devices at CDRH.

Good luck!


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Andrea Chamblee Esq., RAC, FRAPS
Senior Counsel, Wilson Sonsini

This information and views expressed are provided in my capacity as an FDA and compliance professor at Johns Hopkins and George Washington universities, and as a RAPS Fellow.
This is not intended as legal advice. Consult a lawyer for your specific matter.

Maryland
United States
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This message was posted by a user wishing to remain anonymous

Hi Andrea,

Many thanks for your detailed response - this is really helpful, and in some ways reassuring that I am not the only one struggling to find specific precedent :-). I realize my question could have been clearer; what I meant, as you rightly guessed, was "...any drug that has received Accelerated Approval on the basis of improved safety vs. available therapies."
Original Message:
Sent: 16-Feb-2022 11:18
From: Andrea Chamblee
Subject: Precedent question for the RAPs community

When you say "specifically on this basis," I am assuming you mean if all the other conditions for Accelerated approval are met.

This has been used to accelerate the study and review of products that haven't shown improved efficacy, but have shown improved safety.

I would have expected companies that received this designation to brag about it in press releases and stock market reports, but I see now why you had trouble finding that, because so did I!

Way down in the search I found these drugs and biologics who got the designation of addressing an unmet need for cancers, psoriasis, Parkinson's, etc. from companies like Amgen, Aurinia, Carrick, ProventionBio, Shionogi, and BlueRock Therapeutics. Perhaps one could be a good example for you.

Take a look at this CBER and CDER Guidance: Benefit-Risk Assessment for New Drug and Biological Products (Sept. 2021).
FAQs: Breakthrough Therapies

FYI, here is a document on unmet needs on devices at CDRH.

Good luck!


------------------------------
Andrea Chamblee Esq., RAC, FRAPS
Senior Counsel, Wilson Sonsini

This information and views expressed are provided in my capacity as an FDA and compliance professor at Johns Hopkins and George Washington universities, and as a RAPS Fellow.
This is not intended as legal advice. Consult a lawyer for your specific matter.

Maryland
United States
------------------------------

Sorry to say, no, you will not find a precedent because the basis* of accelerated program of 1992 does not provide the following possibility from your clarified question: ""...any drug that has received Accelerated Approval on the basis of improved safety vs. available therapies."

The above possibility from your question would give you a regular or full approval and does not need an accelerated approval

The basis for accelerated approval is as follows (i.e. pertains only to clinical benefit/therapeutic effect (not safety) based on surrogate endpoint) :

*"A positive therapeutic effect that is clinically meaningful in the context of a given disease is known as "clinical benefit". Mindful of the fact that it may take an extended period of time to measure a drug's intended clinical benefit, in 1992 FDA instituted the Accelerated Approval regulations. These regulations allowed drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint. Using a surrogate endpoint enabled the FDA to approve these drugs faster."

Hope this helps! Contact me privately if you need further help!

------------------------------
GRSAOnline
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Original Message:
Sent: 14-Feb-2022 05:17
From: Anonymous Member
Subject: Precedent question for the RAPs community

This message was posted by a user wishing to remain anonymous

Dear Colleagues,

One of the FDA definitions of unmet need for drugs and biologics, where there are available therapies, is

"[The treatment]...Provides efficacy comparable to those of available therapy, while (1) avoiding serious toxicity that occurs with available therapy, (2) avoiding less serious toxicity that is common and causes discontinuation of treatment of a serious condition, or (3) reducing the potential for harmful drug interactions"

My question for the hive mind is whether there is any precedent for a drug/biologic receiving Accelerated Approval specifically on this basis.