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Information About Early Access
Since December 2, when I was diagnosed I have been trying to manage the process of applying for early access to Tofersen. Right after my diagnosis, the FDA delayed the decision. I was expecting the process to take a month to a month and half. It was more like 4 months and just when I was expecting to get access, Biogen put all early access applications on hold until after the FDA made a decision. I will be starting the treatments. If you would like me to share any information just ask or let me know.
Amanda
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4 Replies
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Amanda,
You waited in the periphery of the ALS community for so many years with the knowledge that with your family history, it was inevitable. I was so sorry to hear about your diagnosis when you posted it in December, but I’m happy you finally got access to Tofersen. Four to five months is an eternity in our ALS world. Godspeed to you and all the others in your journey of hope!
Len
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Len,
Thank you so very much!! I just talked to the local news station here in Fort Myers, WINK NEWS, and they are doing a story on ALS. They are going to interview me next week. I asked if they wanted to film my first procedure (I also had to ask my doctors if that was ok). The reporter sounded interested, but I don’t know when my treatments will start,but hey, anything that draws attention to the unmet needs of our community!! My goal when I reached out was to, obviously raise awareness next month and to draw attention to ALS research which will hopefully bring in more donations!! More donations will support more research and find desperately needed treatments.
Yay, we have a treatment that looks promising to target SOD1 mutations, and that will hopefully help 300+ people! However, my dedication is to the entire ALS community, not just this one small fraction. I’m so lucky I’m in that group and I know it. It’s a gift, an prayer answered and I’m not taking that for granted. Now, we have to figure out how we can have the same opportunities available to pALS with familial and sporadic ALS. It’s been a long journey, and I will continue to pray, volunteer and work until everyone has treatment options or cures!! One for All, and All for One!!
Amanda
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How do you apply for early access to any new drugs or therapy?
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Hi James,
If you are applying for “Early Access/Compassionate Care” for an experimental treatment which is one that has not been FDA approved, the pharmaceutical company likely has an application. My doctor had to initiate the process. Biogen had an application that needed to be completed.
Many of pharmaceutical companies use a company to manage applications.
The ALS Association’s website has a lot of helpful information too!
https://www.als.org/research/expanded-access
For access to newly approved drugs you would probably have to go through your health insurance. That’s what I’m having to do for Tofersen/QALSODY. I’m not sure how that will go. If it isn’t approved the doctor can work with the Insurance company to say it is “Medically necessary” and that should over ride the initial insurance decision. At least, that is how my insurance rep explained things to me.
If you have any questions I can answer just let me know. I’d be more than happy to talk, message – whatever works to be helpful!!
Amanda
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