New Approved Medications/Drug Clinical Trials

@AffyAvo I was used in those researchs when i was younger, and it was a terrible experience. many of those have never got approved/ got rejected as result of those researchs

@AffyAvo if there were new medications out that had shown to be helpful during trials I would be willing to take it

@AffyAvo

I think it all depends on the current sitiation that I wass in. Sometimes it would be easy to grasp at anything that might help, another it would be no way!

What about you?

For me it depends on the medication, the condition, and how bad it already is. I agreed to be in a clinical trial for the HAE medication I used to be using, but a more concentrated version and injecting it subcutaneously instead of via IV. I only go through the initial period of stopping for a bit and testing though, because one of my blood tests was normal which is actually abnormal for my type of HAE. About a year later I got to do it off-label anyway which was nice, as then I didn't have to worry about taking a placebo for a while which was guaranteed as it was a crossover study. So I was concerned about having attacks during that phase.

There is a new HAE medication that is upcoming. It's a monoclonal antibody that targets kallikrein. It would mean less injections as the frequency of use is much lower. I have concerns though about side effects, especially as it's not the lowest level of the pathway that creates swelling (although neither are the plasma products I use) and isn't just replacing what I don't make properly (that is what the plasma products do).

WIth new medications, the potential side effects and drug-drug interactions aren't always known, plus long term effects are a concern.

I'm not even sure when this will be approved and once it is there's still the consideration of coverage - many HAE drugs cost well into the $100s or $1000s per dose. In Canada, patients cannot pay for blood products, so coverage via goverment insurance (which is mandatory and often doesn't even have a direct fee, depending on province, it's mostly covered by taxes) is how those costs are covered. For this new medication, if I use it at home it would most likely be only private insurance, and it might not be covered at all. One of my attack meds is over $3000/dose and my current plan covers 80% and only 12 doses/year.

My current prophalaxis treatment is also working really well. There was a huge change in the last year and I suspect certain things to be a factor in that but I don't totally know why. So switching I would also be concerned about it not being as effective for me, although trials have shown that most do really well on it.