"Compassionate Use" for VX-950 more complicated

[Magnum]

As you know, I posted about getting a "Compassionate Use" approval for Telaprivir. The plot thickens and I will explain.

Although the FDA is surprisingly helpful and so is Vertex, here is the update. Vertex has not yet officially approved the "Compassionate Use", but... they are reviewing my case. I spoke to my contact today. They review the case in their Virology Research Dept. They will then send me an email letting me know if they approve or not.

Let me make this a little more clear, because Dr. Gish was told the same thing. Although it is not yet approved, they will consider this in an "emergency" case, perhaps being that the patient is dying and needs this ASAP. Also keep in mind that this is an “emergency use”, meaning nothing else works, the person has Cirrhosis, and their days could be numbered. This is NOT for a person just requesting the drug before the trials are finished.

The FDA still needs the doctor to fill out the forms I mentioned in my former post, and also has to write a planned protocol of: How he plans to administer the drug. What dosage. What the follow-up will be. This, the doctor will do after he gets approval from Vertex, who will instruct the use, dosage and follow-up.

My doctor called me today and has filled out the forms and was ready to send them, but he was confused as to the ambiguity of the questions. That’s why I contacted the FDA. It may be important to note that speaking to “someone” at the FDA is not like speaking to a “supervisor”, which is what I did today. Don’t let this diminish your flame of hope. Rest assured I will keep pushing for answers and post them as I get them.

I should hear from Vertex by email next week. If I don’t, my doctor will call them and push for an answer. It isn’t easy but I still feel that the pressure now (as someone on this site mentioned), has to be put on Vertex, not the FDA. This pressure has to come from everyone. Email and bombard them at ***@**** for the “Single Patient Compassionate Use”. One person doing this is one thing, a group of people doing this is another... Apply enough pressure and something has to give....

Magnum

[epiphiny]

medicalinfoatvrtxdotcom

medicalinfo at vrtx *******

[willing]

not sure whether the protocol for compassionate use bears any resemblance to FDA's waver for emergency investigatigational use, but here's a recent story about a Dr that has twice successfully petitioned the FDA for the latter in the last couple of years. He might be able to give some advice about how to proceed:

http://www.santacruzsentinel.com/ci_11432584

[epiphiny]

Well you can just send me a message through MH by holding your mouse over my name and selecting send a message or you could write the address out in full in your journal.  Here's an example:

epiphanymouseatmousevilledotcom or epiphanymouse at mouseville dotorg

[Magnum]

Give me the details on how and I will post the direct email address to the Vertex Team (where they evaluate case by case and will determine the final decision).

I can't state enough how important it is not to abuse this. I am a little reluctant in giving out this email address, but on the other hand, I want to help people in dire need. I'm not looking for sainthood, I just know what it's like to suffer and I want to help...

Magnum

[epiphiny]

You could always PM the address to someone else, and that someone else could write out the email address in full under their name.  I could be that someone else if it would help others.

[HCA]

I am pretty certain that Dr. Joshua Boger recently retired and that his successor was promoted from within.

[Magnum]

Sorry. Wrong site. This is what happens when you're tired and frustrated...

Try this:

http://www.vpharm.com/contact.html


Magnum

[Magnum]

Joshua Boger is the CEO of Vertex Pharmaceuticals.

This site does not have the email I wanted to post, but it's a possible alternative:
http://www.vertex.com/contact_us.asp

Good luck and don't be abusive...

Magnum

[Magnum]

I also have grade 2 Verices and the doctor banded some. I go a week from Monday to band more. He now predicts one or two more treatments. You should ask your doctor why he's not banding yours, but instead abanding the project. This is nothing to toy with and could be very risky if left undone. Not trying to scare you, but it was relayed so by my MD.

The pain with me lasted 9 days, especially when swallowing. Matters were worse with me inparticular because I'm a singer. I asked the doctor why the pain lasted so long. He said he put 4 rubber bands on each, but next time he will try 2. He also mentioned they are not as big as higher than grade 2 varices, but still wanted to band them. I would ask your doctor for clarification...

Magnum

[Tippyclubb]

Okay I will try,  but we really can't help you if we don't know where to send the e-mails and that's a shame.

Your right about the tricks--all they will do is delete it.

[Magnum]

AK

If I try tricks, I may never be able to post again and help those in need.

Tippy

I can't honestly remember where I got it. You can try their site.

Magnum

[AKHepper]

try spelling it out without the @ symbol, maybe?

[Tippyclubb]

Would it be listed on their website, or do you need a particular link to send the e-mail?

[Tippyclubb]

I saw that

[Magnum]

Tried that. HCA also blocks emails there... Sorry...

Magnum

[Tippyclubb]

Go to your profile, scroll down to bottom left side.  You will see All journal entries, clic on see all.  Now you should see new entry, clic on that and type in the box.  Scroll down and Clic on send or post.  I forget which one.

[Trinity4]

Fingers crossed for you Magnum.  

[Magnum]

As to posting the email, I know HCA has rules against it. If you could instruct me on the "my journal" posting of the email, I will do it. This is where I sent my email and I know they got it because they said so.

When you present your case, say it's what your "doctor" suggested, since there are no other avenues, he's tried all drugs and treatments available and you can't wait two more years.

Rest assured that Dr. Gish is also working on this and he will let me know when they let up on their strangle hold on the release for "Compassionate Use".....

Magnum

[desrt]

Better yet, I wish you continued good health so you can treat when it is approved.

[ClippedWings]

I am interested in pursuing this avenue, as I grow tired of  this seemingly endless wait for the new meds.  I am also a cirrhotic and a non-responder to SOC.  I have grade 2 varices but have not had any banding procedures yet.  At this point my cirrhosis is compensated.  I don't have acites, hepatic encephalopathy, and no history of bleeding varices. Although I know the clock is ticking, I don't know (and my doctors don't know) how long until I start to decompensate.  Is your cirrhosis still compensated?  If so, what argument does your doctor use to justify the life threatening nature of your liver disease.  I'm trying to figure out how to use this emergency use provision.  Basically I think it is safe to safe that Hep C and liver disease are killing me, but will I die tomorrow or this year? Not likely, but any varice has the capability of causing a fatal bleeding event.  I'd like to know your thoughts and how your case compares to mine as my doctor is very responsive to trying new things and would probably be very cooperative if I could make the proper argument.  Thanks for all the great work you've done and are doing.  I too know the frustration of the seemingly endless waiting game for the approval of new treatments.  Keep up the great work.  Best of luck.    

[desrt]

Companies are not allowed to charge for compassionate use medicines, otherwise it would simply become a scam to bring products to market before they're fully tested. Each compassionate use becomes a customer they lose when the product is finally approved. You will basically have to prove that you are so sick that you aren't going to be around in a year and a half to buy their product.
I'm sorry if this sounds harsh, but they have to recoup the millions they've infested developing the drug, including an obscene amount paid to the 'patent holders' of the hepatitis C virus - a company that's done nothing but buy a company that bought another company that bought a company that first cloned the virus. Not discovered the virus. Not isolated the virus. Not developed a medicine for the virus. Simply was the first to clone it. That's the way international patent law works and that's why I'm always fussing about it. I wish you the best in your endeavors.

[Tippyclubb]

Email and bombard them at ***@**** for the “Single Patient Compassionate Use”. One person doing this is one thing, a group of people doing this is another... Apply enough pressure and something has to give....
============================================

As you can see the site or link didn't come through.  I will e-mail them when I know where to send it.  Maybe you can put the website in your journal and we can get it from there.