Right to try refused

[JAM]

Help please.
Our neuro just responded to my husbands request for an Ibudilast prescription. He indicated that under Right to Try we have to contact the pharmaceutical company directly and ask them for the drug. He also indicated they will probably deny him & refer him to the Expanded Access Trial and they do not have one here so he could try in Minnesota- then went on to tell him Expanded Access takes awhile to set up. He ended it with in a small trial patients dropped off bc GI issues.
That was it. No help on how to set up this account, where to go to get help. Honestly- it is outrageous ImO. I know second opinion but my husband can’t go through hearing all the hopelessness again so unfortunately it seems we are stuck. Can anyone help with how we might be able to get access to medication beside clinical trials? For instance, if he wants to try Prime C? (Not saying he does, but if he did). He prefers to just pay out of pocket then be put in a trial as he is going through bouts of depression rn.

 

[Nikki J]

Did you reference right to try in your message to them? Because that isn’t the mechanism for what you want to do. If you want to buy a drug overseas then it is under a provision that in a serious inadequately treated disease you cam import with proper paperwork a drug that is unavailable in the US. The everyone dot org site can help with that but not of your doctor won’t cooperate at all. Btw I doubt he qualifies for expanded access It is only for people who are ineligible for all trials usually due to time from onset or advanced disease

Your second opinion may be more cooperative

 

[JAM]

I am so confused. We did mention right to try- I guess that’s our fault then. Also unfortunately he has my husbands outset at 2 years ago bc that is when he started having twitches. Altho a year after twitches started he went to neuro & had an EMG that was clean. He was diagnosed at that time with BFS. It wasn’t until 6 months after BFS diagnosis that he was unable to button his cuff. Now it seems as a result he may be eliminated from some trials? I am just shocked. He still only is unable to button cuff. All else is unchanged (altho he’s lost weight and is tired- but depressed so that could also be cause).
We meet 10/16 with our clinic so I hope they help us. We are still exploring which drug to try, but sure would like to know exactly what needs to be done when we decide.
Ty for direction here.

 

[Nikki J]

Healey which I think is going to start a new regimen is 3 years. It would be up to the screening doctor to call onset. If you are going for a trial I would say . His weakness started x. If more detail twitching before but normal exam and emg. And I would lead with the weakness with your next doctor. And again can say some twitching before Have you seen his diagnosis note? Mine specifically said onset date I think for possible trial

Right to try is frankly a red herring. It would work as your doctor said and needs the drug company buyin and the doctor to do a lot of work. I only know one very special case where an individual with ALS got something If you want to take something you buy overseas that is different. And if you want to take something off label that is available here you just need a prescription from your doctor ( and your drug benefit to cover). I take metformin for c9. My neurologist prescribes it and my drug benefit covers without a PA because it is cheap.

In your place I would research upcoming trials and read critically about things you are considering outside of trials. Do not go by press releases. Read criticisms of the way results are presented in the press releases and news stories You need both sides. When you see the next doctor you will be able to ask them. The fact that he appears to have slow progression means you have time to make a balanced decision

 

[JAM]

How do we know what medication offer“off label”? Is the over seas option the “everyone.org”? I guess it’s not worth getting too upset about if it won’t affect his ability to get into a trial (should he decide he wants to at some point). I guess I was just confused how if the twitches were diagnosed a year after onset as BFS based on EMG results how later they can be considered beginning ALS symptoms. I mean wouldn’t they be either BFS or not BFS? How can they be both? Again, probably doesn’t make a difference. It’s just one more frustration with this ugly disease.

 

[Nikki J]

I don’t know what the investigator would say. I think your case has wiggle room and could go either way. And sometimes the trial standard is specifically onset of weakness. But healey at least is 3 years. Maybe others. You can look at clinical trials if there are listings and it will say. I also don’t know of there is anything worthwhile offlabel at the moment except methylcobalamin but the prime c components would be an example. Everyone is a place you could buy ibudilast. I know people who used it for earlier drugs that didn’t work out but people wanted to try. As far as I know the organization fulfilled the orders and helped with paperwork

 

[JAM]

Is there someplace that lists which medications are available off label? I don’t know much about Prime C yet. I think we will wait a month to see what reports come out on various drug trials (the Herv-k, the Prime C, I think Ibudilast ends mid November) then decide which to attach our wagon too. The thing is we need direction on HOW to do that. ?’s
1. Where do we find what drugs are off label? Are the off label drugs FDA approved?
2. What is an orphan drug?
3. Do we need a prescription to get drugs at Everyone.org?
4. At what point are medications available before FDA approval? And how/where do we get them??
I’m sorry to bombard - but what good are experimental medications for someone who has nothing to lose if the patient can’t get them?
I am tired. Ty.

 

[Nikki J]

Off label just means you are prescribed a drug that is approved for one indication and take it for another. As in the metformin example. It is approved for diabetes but it is perfectly legal for my neurologist to prescribe it for my c9. Taking the two components of prime c which are an fda approved antibiotic and an approved anti inflammatory would be off label. Off label prescribing happens frequently for all kinds of conditions

Being an orphan drug is just a designation that gives its pharma company some financial benefit to encourage drug development for rare diseases

3 yes

4 generally they are not available before approval unless they are available overseas, you are in a trial or happen to be in expanded access. Most of us have to wait

Please try to calm down. Experimental drugs are just that unproven and experimental. And from your description your husband has a great deal to lose if they turn out unsafe. Some trials find the tested drug is not only ineffective but made the participants worse.

 

[JAM]

The reason we all feel so desperate to try these experimental drugs is bc of time/ as we all know, time is not something ALS patients can afford to lose. That’s why they look for sooner rather than later even if risks are involved. But Ty. I understand what you’re saying

 

[Nikki J]

Look at my tag line I have lost virtually my whole family. I will die from this.

But if you have your heart set on ibudilast he should qualify for the trial.

 

[JAM]

I’m sorry I did not mean to imply you don’t understand , I was just trying to explain why I seem so anxious. I am anxious- very anxious You have been so helpful! I will try to do my best to calm down. We aren’t set on any particular meditation, but we are set on trying something bc we can’t just do nothing. I guess that’s what I was mostly trying to say. Sorry for any misunderstanding.

 

[Nikki J]

He will qualify for several trials. All but one I looked at clearly had time limits on dx date( one) and weakness onset ( the rest) except for one that was symptoms

I suggest before your appointment go through the listings and choose ones to discuss with the doctor. You may have to travel a bit for some. If you decide against trials then you can look at supplements or overseas drugs

Spend your time preparing for the next visit so you can be super organized and ask the questions you need to

 

[Marieb425]

@JAM you can also ask the neuro about Radicava ORS which is approved for ALS. Your insurance should cover it too although you might have to file an appeal of whatever if they deny it at first. I’ve learned that everything requires a fight with insurance over the past two years.

My PALS tolerated it just fine with no side effects. I don’t know if it did anything, but taking it made him feel like he was doing something. As well, his limb progression did progress more after he stopped it. But it’s impossible to know was this caused by stopping the Radicava or was it simply time for more limb progression.

 

[ahl39]

<discussion of illegal pharmacies removed>

Consider these promising drugs that do not require prescription and are widely available on Amazon or your local pharmacy. They all have a trials grade of D but have A or B mechanism of action.

Acetyl-l-caritine
L-serine
Basis (pterostilbene and nicotinamide riboside)

These are part of the 8 most promising alternative treatments to ALS in the Neals presentation in June. The other 5 need prescription.

 

[Landis02]

See ALS untangled dot com for more about these treatments.
Dr Bedlack started my PALS on Basis in the spring. He’s had no side effects but who knows if it’s making any difference.