Pre-Submission for Breakthrough Device Designation - What's the threshold?

[SSchoepel]

From the Breakthrough Devices Program guidance (18 Dec 2018): The Breakthrough Devices Program is a voluntary program for certain medical devices and device-led combination products that provide for more effective treatment or diagnosis of life-threatening or irreversibly debilitating diseases or conditions.

Does anyone have examples or real-world experience on where the FDA draws the line on what they consider life-threatening or irreversibly debilitating diseases or conditions? I have been asked to help prepare a pre-sub request for a breakthrough designation and there is no other approved or cleared alternative, and the availability is probably in the best interest of patients, however, I am wondering if the FDA will consider the condition life-threatening or irreversibly debilitating. I'd like to get a feel for the FDA view so I can determine if we need more justification or will end up in STeP regardless of justification.

 

[primavesvera]

Our company tried, we submitted multiple findings and then we were asked to provide the rationale why each of those are considered life-threatening or irreversibly debilitating. However, without any specific explanation or some threshold given to us. And we failed.

Obviously, BDP and STep are completely opposite programs, but in BDP guidance (https://www.fda.gov/media/108135/download) there are a couple of indications that might help you, such as the breakthrough technology section. Is it a breakthrough technology?
Then, you have also a bit on whether a disease or condition is life-threatening or irreversibly debilitating (with some limited examples).

Q-Sub is always an option.

 

[SSchoepel]

Thanks, we will be doing a Q-Sub to ask for the designation. I'm just not sure, even with the examples in the guidance document (read before posting and still putting me on the fence), that we would be granted the designation. If not, then we need a second Q-sub to ask questions to gain agreement on indications for use.

 

[primavesvera]

There is a forum member @Watchcat, she or he, seems to understand the FDA logic based on the work experience, so maybe you can contact that person.

 

[Watchcat]

There is a forum member @Watchcat, she or he, seems to understand the FDA logic based on the work experience, so maybe you can contact that person.

I am flattered that you think so. I like to think I have a reasonably good handle on FDA's mindset, but I don't have experience with Breakthroughs, so I'd have to research the substance of it before I could put my FDA mindset on. In other words, ka-ching. SS and have crossed paths here before, and my vague recollection is she (I know! Ha!) may well be as good at that as I am, and was just doing a reach out to see if anyone had prior experience, in case it offered additional insights.

Also, head up...maybe a month or so ago I heard FDA was just saying no to pre-subs. All of them, I can't say, but several colleagues reported having this experience recently. Speculation about limited resources due to the pandemic. Other speculation that they had originally expected to be charging user fees for them by now, and don't much want to do them until they can work through the belated MDUFA negotiations.

Watchcat is a she also.

 

[primavesvera]

I am flattered that you think so. I like to think I have a reasonably good handle on FDA's mindset, but I don't have experience with Breakthroughs, so I'd have to research the substance of it before I could put my FDA mindset on. In other words, ka-ching. SS and have crossed paths here before, and my vague recollection is she (I know! Ha!) may well be as good at that as I am, and was just doing a reach out to see if anyone had prior experience, in case it offered additional insights.

Also, head up...maybe a month or so ago I heard FDA was just saying no to pre-subs. All of them, I can't say, but several colleagues reported having this experience recently. Speculation about limited resources due to the pandemic. Other speculation that they had originally expected to be charging user fees for them by now, and don't much want to do them until they can work through the belated MDUFA negotiations.

Watchcat is a she also.

Regarding the FDA, each time when we try a new approach, we magnificently fail lately. As I said, we tried our best with the breakthrough technology, but it wasn't enough and it wasn't explained why it's not enough.

So, I really hope someone has a better insight and is willing to share their experience.

 

[Watchcat]

we tried our best with the breakthrough technology, but it wasn't enough and it wasn't explained why it's not enough.

Hoping to enlighten, rather than offend:

I will say that my general perspective on the medical device crowd is that they want FDA to figure everything out for them, tell them what to do, and how to do it. I was a writer before I was an RA professional, and struggled, as we all do, with the dictum that "the onus to communicate is on the communicator." That's harsh, but ultimately true.

I have always thought it was entirely up to the company to figure it out, then explain it to FDA in a way that FDA can understand it too. Because I don't think FDA inherently understands a lot of what industry thinks (or would like to think) it does, or "should." Maybe not even a lot of what FDA thinks (or would like to think) it does, or "should." So here, I would be inclined to say, perhaps FDA didn't explain why it wasn't enough, because FDA is in no position to know, and you failed to convince it otherwise. Or perhaps because it simply didn't meet the breakthrough criteria, and FDA understood that very well, even if you didn't.

Again, this is an effort at enlightenment, not offense. I'm in no position to know if that shoe fits you or not. Just that I see enough feet in those shoes, they are invariably the first shoes that come to mind. Certainly sometimes the FDA is just...whatever...and you are simply never going to get through to them.

 

[Watchcat]

Also, an update on pre-subs, Graematter just reported 90- to 120-day delays. So apparently still happening...for some.

 

[primavesvera]

Hoping to enlighten, rather than offend:

I will say that my general perspective on the medical device crowd is that they want FDA to figure everything out for them, tell them what to do, and how to do it. I was a writer before I was an RA professional, and struggled, as we all do, with the dictum that "the onus to communicate is on the communicator." That's harsh, but ultimately true.

I have always thought it was entirely up to the company to figure it out, then explain it to FDA in a way that FDA can understand it too. Because I don't think FDA inherently understands a lot of what industry thinks (or would like to think) it does, or "should." Maybe not even a lot of what FDA thinks (or would like to think) it does, or "should." So here, I would be inclined to say, perhaps FDA didn't explain why it wasn't enough, because FDA is in no position to know, and you failed to convince it otherwise. Or perhaps because it simply didn't meet the breakthrough criteria, and FDA understood that very well, even if you didn't.

Again, this is an effort at enlightenment, not offense. I'm in no position to know if that shoe fits you or not. Just that I see enough feet in those shoes, they are invariably the first shoes that come to mind. Certainly sometimes the FDA is just...whatever...and you are simply never going to get through to them.

Oh, we've been through that stage. We learned that you really need to put a lot of effort to communicate a clear message because a lot of it gets lost in translation and then you have a big mess to resolve. However, this is now a bit beyond that initial hurdle, so we just need to find another way or put a pause on the US market and wait, which is the least favorite option.