If I'm understanding it correctly, the purpose of a Compassionate Use Program (aka Expanded Access Program) is to allow access to a potentially beneficial therapy to patients who don't meet the inclusion criteria for a clinical trial.
I assume physicians are free to decide whether or not a particular patient is likely to benefit from a therapy available via a Compassionate Use program, assuming the funding is there, but I'd welcome input from anybody who knows for sure.
How many patients are there in the US alone who stand to benefit from an antibiotic which controls NTM infection whilst causing negligible systemic toxicity?
Insmed quotes an estimated 250,000 in the US with Non-CF Bronchiectasis. The estimated 30,000 in the US with CF are also at risk of NTM infection (but are barred from participating in the ongoing NTM study even if they have a history of NTM infection).
It's well worth bearing in mind that the physicians see people who are ill with Bronchiectasis, and their primary aim is to improve the patient's quantity of life and quality of life. Until they get the results of culture tests back from the laboratory they don't know what the actual infection is.
The Non-CF Bronchiectasis workshop arranged by the FDA yielded some illuminating observations by leading pulmonologists -
..... "once people are colonized with many of these bugs, they're generally colonized for a long time, and maybe it's for the rest of their life. You don't always find them every time you look for them. But I'm pretty convinced that some of these bugs are fairly recalcitrant, and they stay there. You may control them. You may beat their population down with antibiotics, but you don't eliminate them. And I'm thinking of NTM; I'm thinking of pseudomonas; I'm thinking about some of these bugs more than others when I say that, but I think it's certainly a concept worth keeping in mind."
..... "So I labeled this, Pathogens of Interest. I mean these aren't just ones I'm interested in, but generally speaking these are the bugs we deal with. And this is not a complete list by any means, but I have to say just off the top of my head, I deal with gram negative bacilli and I deal with mycobacterium a lot"
..... "I think you can say that more severe bronchiectatic patients tend to end up getting pseudomonas at one point, compared to the milder forms"
..... "it's very important that we recognise many patients have concurrent infections. And it is, as a clinician, sometimes difficult to tease apart which one today is causing that exacerbation."
..... "in the unit, in the hospital, we frequently double coverage people with pseudomonal pneumonia, for example, or gram-negative pneumonia, or just empirically before we know what anything is. And a patient with a ventilator-associated pneumonia, or just a rip-roaring severe pseudomonal pneumonia, often gets two drugs of a different class"
..... "There's a lot more adverse events when you use two drugs versus one. I think in the end, probably you should use two drugs if someone's really sick, because it improves the chances that you're going to actually have one of them working"
..... "we use antibiotics in this disease not only to treat acute and chronic infections, but we also use inhaled antibiotics to try to prevent infections"
..... "But in terms of infection prevention and maintenance therapy and suppressive therapy, certainly inhaled antibiotics offer at least a very nice option for many patients, if they can take them and tolerate them and afford them"
..... "some of my patients are on inhaled TOBI, for example, for maintenance therapy"
That last observation is of particular interest - TOBI is not approved as a therapy for Non-CF Bronchiectasis.
TOBI was studied at Phase II, and it was reiterated in the workshop that the benefit from the reduction in bacterial load was perceived to be cancelled out by the side effects of the therapy. Seems to me pretty much the same story as its approved (CF) use nowadays - kills the pseudomonas, but doesn't deliver a noticeable improvement in quality of life.
As usual, form your own opinion of how widely Arikace is likely to be used under the NTM Expanded Access program next year.
oh, fud...another thing about compassionate use. It is intended for the treatment of diseases that have no alternate therapy. Or, if there is a therapy, that individual is refractory. All those people that have CF or other disease will be treated by FDA approved therapies until they do not respond...then the physician can consider using Arikace.
Now, the bombastic sort that you are will not realize the following two things. 1) I know what I'm talking about and 2) I'm a long. The lies you tell here are not acceptable.
I'm encouraged by the fact that not a single user of this forum thus far has responded to my invitation -
"I assume physicians are free to decide whether or not a particular patient is likely to benefit from a therapy available via a Compassionate Use program, assuming the funding is there, but I'd welcome input from anybody who knows for sure."
One of the more prolific contributors to this forum did pull me up on my suggestion (in the title, but not in the post itself) that EAP might effectively stand for Early Access Program.
Should we take that as an implicit agreement that pulmonologists will be free to use Arikace under the EAP if they feel that their patients would benefit from the therapy?
Compassionate use will generate just about zero profit. And the outcome of the patients that are treated will not be part of any study. Compassionate use is nice for the patient if it works...but it doesn't do much for the bottom line or approval.
Fud, for some reason or another, sees this as a major milestone or development. It isn't.
In response to a question about the ideal target of permanently ridding patients of the offending "bugs" -
"Environmental bugs get into surfaces in your body or procedures. And everyone's all stuck on the fact that there's just one of them there. You just have the, you know, the black curly haired bugs there.
But the bottom line is, when you have an environmental source of a - there's multiple different strains living in that environment. So it makes sense to me that you're often infected with multiple different strains. They'll have different susceptibility patterns. They'll behave differently. So you might get rid of one, but you don't get rid of the other, or maybe they're competing with each other in there. And so you don't really know what's going on. I think it's a very dynamic situation.
But I do think it's a good goal. I share your goal. We try that with NTM. We try to get rid of it. You know we often don't, and even if we get rid of it, or we think we get rid of it, we don't find it for many years. You look at their CT scans and it's hard to believe it's not in there still. I think probably if everyone lived long enough, we'd find it again.
But it's a noble goal and it may be possible if we have the right drugs. That's another thing to think about."
Btw - it was suggested that perhaps a more suitable approach would be a similar therapeutic regimen to that used with Cystic Fibrosis - permanent maintenance therapy involving antibiotic rotation.