Getting drugs released prior to trials

[garyisleofwight]

Have been following AB Science and their drug Masitinib. Just noticed today on their press release that the FDA are, and have , released this drug for compassionate use in ALS prior to clinical trials. Is this new? First time I have seen it and obviously that's exciting . Is it not time we all got together and stopped just waiting ?- I am in the uk but happy to start kicking butt with the government to change things!! Of all the trials there are a few which are very exciting but waiting years is not acceptable . Let's attack this part of our fight get the drugs to us !!!

 

[Nikki J]

The FDA of course only has jurisdiction in the US. Your possible access would be through the procedures of your regulatory agency.

Compassionate use is by a case by case basis and your doctor has to initiate it by filing paperwork. There was a concerted compassionate use effort back in 2012 for dex which was in phase 3 trial and looked so promising. My sister got it. I know ALS Worldwide helped her get the paperwork , her doctor filled it out and she went on a waiting list - I think the FDA approved a certain number and then they had to reapply to add more. She eventually got it ( did not help and when the trial failed and she stopped it she realized it made her worse). It was very expensive- unlike trials you are generally responsible for costs

This ties into the right to try movement and there are organized groups for that where you might want to join. If you do not already know who regulates drug access in the UK worth looking into. It might be EU ??? So going to change soon if so

 

[garyisleofwight]

THankyou- yes the EMA has a compassionate use scheme, thanks for helping to highlight this for all regards gary