Just Spoke To Vertex

[Magnum]

I have a contact at Vertex. We spoke at length about Telepravir and the "compassionate" use. That means use now (without placebo) for Cirrhotic victims. While he couldn't give me a definite time line for the compassionate release, he did say that telepravir being in Phase III, they are seeking approval by the FDA in the second half of 2010, and making it available through prescription instead of the current clinical trials.

Interestingly, he added a comment that we could hopefully expedite the "compassionate" use approval by the FDA if we write them an email stating the urgency for those who need this NOW.

Here is a direct link, with phone number to the FDA: http://www.fda.gov/comments.html

Write to them and continue to bombard them with emails until they give in. This is a battle to be sure, but if the government sees that we are desperately seeking a compassionate release of Telepravir, they could rethink this whole matter. Write an email now...

Magnum

[Magnum]

What HCA said makes sense. I wrote yet another email along those lines that may inspire you to be creative and non-abusive by writting this type of email and continue to elaborate slowly but surely on the subject:

I received a pre-written form response to my question of releasing the Vertex Pharmaceutical's drug Telepravir (VX-950) for the eradication of the Hepatitis C virus, for us who have Cirrhosis. This would be a “compassionate” exception. Many of us are dying now. We cannot wait until the final release on the second half of 2010. Soon, our livers will be too sick to accept drugs. At that point, as many of us are over 62 and with Medicare, the enormous cost to Medicare for liver transplants would be prohibitive.

Your reply was to ask the doctor to submit a special need letter to the drug company. Makes sense. However...

As you well know, doctors see dozens of patients per day. The mountainous paperwork they must fill out at the end of the day with blood shot eyes, does not propel a doctor to do this. He’s tired, wants to be with his family and have dinner, have a couple of martinis, plan his next day, maybe watch a show and hit the hay.

If however, you were to make the paperwork simpler and more to the point, they may capitulate and write such a letter. I'm not saying I don't trust doctors, but they have been known to accidentally kill people and as you know, there are some in federal prisons now for other devious deeds.

Help us who are Cirrhotic and in dire need of this drug now. Please simplify the process for the “compassionate” exception form for the doctors to fill out. Another advantage may be to allow us to download the forms so we may print them and present them to our doctors face to face or dropped off at their office. That will save one major step of them downloading them.

Another thought, is to allow us patients to fill out a questionnaire and a release form, give it to the doctor, and have him fill in the minimum he has to, before sending it to the pharmaceutical company. This could help us get this done in the fast track...

Magnum

[HCA]

Very good input from desrt above.
His final paragraph seems to me to be the definitive statement and is worth heeding.

[desrt]

"There is a mechanism to
access products that have not been approved.  However, this would
involve a physician willing to file a single patient or emergency
Investigational New Drug (IND) application and this must be done with
the cooperation and permission of the drug supplier."

This agrees with everything I've read, meaning VX-950 is already available for compassionate use.
Perhaps you could get your doctor to clarify what he meant by " a definite time line for the compassionate release".
As far as I can tell, the compassionate use procedure is fairly straight forward:
1) Nothing else works.
2) Your doctor arranges access to the drug through the drug company.
3) Your doctor fills out a mountain of paper work, which the FDA must approve on a case by case basis.
4) You sign a waiver stating you understand and are willing to assume the risks of an unapproved medication.

The following is a good model/explanation of the process.

http://www.research.ucsf.edu/chr/Guide/chrEmerUSE.asp

As far as I can tell, there is no 'blanket release for compassionate use'. You either go through this process or wait for FDA approval.

[Tippyclubb]

You should post this on the forum at Janis & Friends also. Bet there's plenty of people there who would send e-mails and letters.

[oneatenuff]

Definitely write letters and definitely definitely get your doc involved to push for you.   Mine told me  (in 2006 when I did not make the tx naive cut and was waiting on another tx naive trial with a PI or another VX group)  that within a few years  (2 - 3 yrs before "market approval" of VX)  that for certain patients meeting certain criteria, VX would be available by prescription.  At that time, I was under the impression that in order to meet that criteria I would need to be a lot worse off, would need to have been unsuccessful with conventional SOC treatment, repeated treatments, real sick, etc.   (lotta hoops jumped through and in dire need of treatment to save my life.)   Letters to FDA are great and needed and do help those with hep c,  but for you personally  (or anyone in your same shoes)  if you are at the end of the line and have no options left and need and want VX before it's approved,  definitely get that clinical / trial doc behind you on the chance at getting VX sooner.

I've been reading your trials and tribulations - you've jumped through a lot of hoops!  Best of luck to you.

[Tippyclubb]

Oh yes, I will still send e-mail and will write some letters for snail mail.  I just wanted you to see the part about your physician willing to file a single patient or emergency
Investigational New Drug (IND) application and this must be done with
the cooperation and permission of the drug supplier.

Your doctor should get involved too.