It was live but they may archive it later like they did with the JP Morgan one for a while.Is there a link to the presentation?
It was live but they may archive it later like they did with the JP Morgan one for a while.Is there a link to the presentation?
Compassionate use is not up to the FDA. It's solely up to the company. On their website they said they are waiting until they can produce enough to meet demand and implied they are also waiting for further testing to show what dosage they would need.I am desperate for tinnitus relief and I'm sure other people are too! Do you think there's any way we could organize a group of individuals to convince Frequency Therapeutics/FDA to allow us to take FX-322 under "compassionate use" before it is released to the market?
Is anybody here friends with Bill Gates?Compassionate use is not up to the FDA. It's solely up to the company. On their website they said they are waiting until they can produce enough to meet demand and implied they are also waiting for further testing to show what dosage they would need.
Personally, if you can convince a philanthropy organization to fund their production ramp up, that might be the only thing that would further it along quicker.
I am desperate for tinnitus relief and I'm sure other people are too! Do you think there's any way we could organize a group of individuals to convince Frequency Therapeutics/FDA to allow us to take FX-322 under "compassionate use" before it is released to the market?
Maybe we should contact the Gates Foundation. One of its goals is to enhance health care.Personally, if you can convince a philanthropy organization to fund their production ramp up, that might be the only thing that would further it along quicker.
In Europe at least the national authority needs to approve compassionate use of the drug. Companies can't go out giving people unapproved drugs without government permission.Compassionate use is not up to the FDA. It's solely up to the company. On their website they said they are waiting until they can produce enough to meet demand and implied they are also waiting for further testing to show what dosage they would need.
Personally, if you can convince a philanthropy organization to fund their production ramp up, that might be the only thing that would further it along quicker.
Sorry to hear this, like you say it sounds awful. Not sure how I'd cope in that situation. Masking is the only way for me to escape it. Guess in that sense I feel lucky. It does seem like some form of hyperacusis as you said. Hope it gets better and/or you find a way to deal with it. I mainly posted because once I found effective masking techniques my life became so much better.Unfortunately, masking reactive tinnitus makes it worse for a lot of people (myself included). Any consistent external noise causes my tinnitus to react. I'm best in quiet rooms, but I try to have some exposure to reasonable sound levels to avoid further sound sensitivity. Some believe this condition is a subset of hyperacusis. Regardless, reactive tinnitus is no fun.
In the US, they can for conditions that greatly affect quality of life for which there are no good treatment options. Your doctor can apply for it for you if you fit the bill but the company has to approve it.In Europe at least the national authority needs to approve compassionate use of the drug. Companies can't go out giving people unapproved drugs without government permission.
Yes, but the FDA has to approve it as well. Companies can't go handing out unapproved drugs to patients without the FDA approving it.In the US, they can for conditions that greatly affect quality of life for which there are no good treatment options. Your doctor can apply for it for you if you fit the bill but the company has to approve it.
From what I understand, all diseases with high morbidity are approved. The FDA is not the road block here. High morbidity and no available treatment is the reason it got Breakthrough status in the first place.Yes, but the FDA has to approve it as well. Companies can't go handing out unapproved drugs to patients without the FDA approving it.
https://www.fda.gov/news-events/expanded-access/expanded-access-information-patients#fda-consider
Or Bill Clinton. He has hearing loss from guns so you'd think he'd be all over this.Is anybody here friends with Bill Gates?
We'll have to wait and see how the FDA will look at this since there aren't any approved hearing loss drugs on the market. My understanding is that authorities like the FDA are careful when considering compassionate use. And for hearing loss at least there's the, albeit unattractive, alternatives of hearing aids or cochlear implants that the FDA will consider when making a decision.From what I understand, all diseases with high morbidity are approved. The FDA is not the road block here. High morbidity and no available treatment is the reason it got Breakthrough status in the first place.
That's true but I also think from the way that Frequency's worded things they are very much framing their drug as meeting an unmet need, e.g. the emphasis on clarity when hearing aids are amplification devices. Cochlear implant surgery is also not really a viable alternative if you don't have severe loss IMO, it's very expensive and of course irreparably damages the cochlea and in no way restores natural hearing function.We'll have to wait and see how the FDA will look at this since there aren't any approved hearing loss drugs on the market. My understanding is that authorities like the FDA are careful when considering compassionate use. And for hearing loss at least there's the, albeit unattractive, alternatives of hearing aids or cochlear implants that the FDA will consider when making a decision.
With regard to cochlear implants that's the catch-22 isn't it? If your hearing loss is severe/profound, which might be an illness for which compassionate use is meant, you can get a CI. If you have mild hearing loss it's probably not considered for compassionate use in the first place. And you could still get hearing aids for that.That's true but I also think from the way that Frequency's worded things they are very much framing their drug as meeting an unmet need, e.g. the emphasis on clarity when hearing aids are amplification devices. Cochlear implant surgery is also not really a viable alternative if you don't have severe loss IMO, it's very expensive and of course irreparably damages the cochlea and in no way restores natural hearing function.
Breakthrough Status means the FDA feels this is a serious condition with an unmet need. Expanded Use is specifically for those conditions. The only reason they would deny it is safety concerns but the FDA has a history of allowing even unsafe last ditch cancer drugs to be used under the law.We'll have to wait and see how the FDA will look at this since there aren't any approved hearing loss drugs on the market. My understanding is that authorities like the FDA are careful when considering compassionate use. And for hearing loss at least there's the, albeit unattractive, alternatives of hearing aids or cochlear implants that the FDA will consider when making a decision.
FX-322 hasn't gotten Breakthrough Status. Frequency probably has their policy listed because they also have to approve compassionate use. It does not mean the FDA will just write a blanc cheque should Frequency open up access to their drug. You also can't compare cancer drugs to hearing loss drugs.Breakthrough Status means the FDA feels this is a serious condition with an unmet need. Expanded Use is specifically for those conditions. The only reason they would deny it is safety concerns but the FDA has a history of allowing even unsafe last ditch cancer drugs to be used under the law.
There is a reason Frequency worded their Expanded Use policy on their website the way they did. They know it's up to them.
It doesn't have to be a cancer drug. It does not even have to be life threatening, it just has to be a drug with high quality of life implications for which people don't qualify for a clinical trial. The only reason it would have been declined is safety issues of which there weren't any.FX-322 hasn't gotten Breakthrough Status. Frequency probably has their policy listed because they also have to approve compassionate use. It does not mean the FDA will just write a blanc cheque should Frequency open up access to their drug. You also can't compare cancer drugs to hearing loss drugs.
It doesn't have to be a cancer drug. It does not even have to be life threatening, it just has to be a drug with high quality of life implications for which people don't qualify for a clinical trial. The only reason it would have been declined is safety issues of which there weren't any.
That being said, cancer drugs are certainly easier because your doctor can just say "they have cancer" where with this, your doctor has to describe in detail the poor quality of life you have and why you need the drug. I think this will be easier for tinnitus after phase 2a results support its use for that.
If that happens, it's probably a good idea to gather some evidence that you've tried other treatments to no avail, or you might be excluded.Really it'd be nice if we could confirm with Frequency that they plan on ramping up production & opening expanded use if phase 2a is promising. Maybe we really could help somehow.
This is the thing I am most concerned about - would you have had to exhausted all other treatment options to be permitted compassionate use? Say if we discover that FX-322 helps hyperacusis patients, for example, would they have had to have tried out sound therapy (which some people may be reluctant about understandably in case of worsening)?If that happens, it's probably a good idea to gather some evidence that you've tried other treatments to no avail, or you might be excluded.
I'm not going to argue tinnitus can be life-threatening, but the thing is, how does one prove it to the outside world and thus he or she should be entitled to compassionate use?
I was thinking about this too. I assume your doctor could say along the lines that you have tried sound therapy through the use of speakers but unfortunately it made the patient worse because it's similar to hearing devices. If you can't cope with speakers then you can't cope with hearing devices.This is the thing I am most concerned about - would you have had to exhausted all other treatment options to be permitted compassionate use? Say if we discover that FX-322 helps hyperacusis patients, for example, would they have had to have tried out sound therapy (which some people may be reluctant about understandably in case of worsening)?