Just Spoke To Vertex

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

[Tippyclubb]

Consider it done, and we should do it daily.

[Magnum]

Here is a direct link to the email page...

http://www.fda.gov/cder/comment.htm

Magnum

[burned74]

thats a great idea.
my first email is going out today

[FlGuy]

Real letters could be more impactful, copying a federal legislator.  Ask for a letter back in reply.  Then follow-up with phone calls to the person who writes back.  Then, repeat...and repeat

[Magnum]

Here is the email I just sent:

Regarding the Vertex drug Telepravir (VX-950) for the treatment of Hepatitis C patients, I want to add that many of us are in Cirrhosis and need this new drug NOW!

You MUST release it for “compassionate” use to us who are dying, and not force us to attend clinical trials and possibly get placebos. This is our last hope. The alternative? To burden the Medicare system with the high cost of liver transplants.

The Telepravir drug is now in phase 3 (the final phase) and has shown remarkable abilities to clear this virus and allow the liver to regenerate.

We are willing to risk it even though phase 3 has not completed. As for all the literature and clinical trials results, the risk seems non-existent for toxicity . We are the people, and as Lincoln stated in the Gettysburg address, "and the government of the people, by the people, for the people, shall not perish from the earth".

Please listen to us people and rethink this as soon as possible. Many lives could be saved NOW...

[Tippyclubb]

Well, my letter was no where near eloquently stated as yours, but I sent one.  I also requested a reply.

I think FI guy has a good point about real letters and will write one of those also.

[Andiamo1]

Great idea - wonderful thread.  Here is the email I sent:

I am requesting that you approve Telaprevir for use as soon as possible.  There are many people that have been fighting HCV for many years and are now running out of options.  I know - I was one of them.
I am 68 years old and had HCV since I was 21 years old.  I repeatedly treated with the standard treatments to no avail.  The treatments were devastating in themselves and did not rid me of the disease.  I was rapidly progressing towards cirrhosis with very little hope.
I managed to get into the Telaprevir trial and was lucky enough to get the drug and not a placebo.  It worked, and I am now free of the virus at 48 weeks post treatment.

I sincerely want all people with this disease to have the chance to rid themselves of this devastating disease and not leave it to the lucky few that get in trials and avoid the placebo.

[Willy50]

You are a marvel; way to take the bull by the horns.

I think it is a great idea.  I actually would hope that they would allow approval for past treatment failures that will be put before them this year which could allow many more people to treat that have marginal hope for re-treating NOW.

I fully support allowing the compassionate use of TVR and especially in your case where there exists a very strong possibility that waiting for approval could be waiting too long.  

I will also write them but think I may try to do some "googling" and attempt to portray the scope of the problem.  Many people are up against the wall.  They have no little hope in treating again with the same treatment, and as they wait their chances of success goes down with advancing liver staging.  Treating cirrhotics is always more difficult and as they approach decompensation the toxicities of the treatments can also push people towards liver failure.

Treating these people through allowing "compassionate care" may also free up a liver that could go to another worthy person.  They could save two lives through allowing Vertex to treat this population.

Good enough for now.  Thanks for starting this thread.

Willy

[HectorSF]

Magnum, Thanks for being proactive on this! You've help us all.

Many times I've heard people on this forum ask what can they do to
make a difference in how we are served and treated by the medical community. I think this is a great opportunity for us to advocate for ourselves by letting our voices be heard.

I hope as many people on the forum as possible and pitch in on this worthy effort.

I will be making my voice heard on this issue.

I will also be writing to my Congresswoman, Nancy Pelosi to see if we can get more support for our HCV community as part of the ongoing overall health reform efforts of the Obama administration. This is a time of big changes in our government and a good time to make others in power aware of the impact this disease is having on us as patients and our medical system.

If we don't get more effective treatments available for us now, in time many more of us will be waiting for transplants, which are personally "life changing", and far more costly then treatments. There aren't enough livers to go around for everyone who needs one now. This will only get worst as us "boomers" age and our illness progresses. This is a tragedy in the making.

Thanks all for your good work everyone!
Hectorsf

[hopeful51]

Per your instructions"continue to bombard" I will start a B-52 mission a.s.a.p


And now I have a ? if you needed rescue drugs on standard tx. Would it be safe to assume that you would also need them if you were to tx with Telaprevir and I am to understand that you can not use rescue drugs with Telaprevia.

Can anyone help> How wrong do I have this?

Hopeful51

[BelB64]

Your letter was fantastic.  I would hope it would move the right people. By the way when speaking to your Teleprevir contact could you ask him how the European trials with Genotypes 2 and 3 are going and do the interim results compare favorably with the genotype 1 trials.

Thanks

[Rockerforlife]

You are a smart cookie i see,ill be sending an email and a hard copy letter also...ive often wondered why the 'compassinate care" never was talked about in the main stream...about time my brother...the ball is rolling...its also a shame what happened to us canadians in the tainted blood scandel in the 80`s,we never did get the proper compensation... if .they can afford to waste BILLIONS on wars...im sure they can help the sick..its time to save people...not kill them....

Rocker  4  LIFE

[HCA]

I think that you'll find that Vertex and it's partners and investors,together with the FDA and the medical profession are already acutely aware that there is a desperate human and economic need for effective treatment for the Hepatitis C virus.
It is for this reason that countless billions of dollars have been spent on the R&D of telaprevir and other agents.
All y'all is really saying today is 'We want it NOW!'
Well of course you do-you have advanced liver disease.So do I!
All life saving drugs have an eager and impatient patient poulation wanting to burn away the red tape.
I think this has less to do with the Gettysburg Address than with writing a cordial personal letter to the new CEO of Vertex asking him specific questions about what plans he may have for a compassionate programme,what contingencies are involved and what timescale may be anticipated.

[viaduk]

Thanks for starting this.  My email is below:

I am requesting that you approve Telaprevir for  “compassionate” use to those patients who have not responded to the current standard of care for Hepatitis C.  For those patients, particularly ones with cirrhosis, it is crucial that we be provided with this new and highly effective treatment.  At this point in our disease, it is more risky to be forced to resort to a clinical trial and the possibility of getting a placebos. Also, the trials' restriction on the use of “helper” medication for low WBC and RBC disqualifies many patients who may otherwise be able to clear this virus.

As a cirrhotic person who twice relapsed from previous treatments, I am anxiously waiting for the opportunity to treat with Telaprevir. I urge you to release this medication for “compassionate” use to those of us who may be facing a transplant or death.

[desrt]

Is anyone actually familiar with the waiver the patient has to sign for "compassionate" use and what the financial implications for your post-tx health care costs would be? As someone who takes half a dozen Rx I never had to take prior to FDA approved SOC, I would be more than a little curious.

[Willy50]

Your reply is a good wake up call.  I'm sure that the FDA is not about to release the drug prematurely.  I'm sure that none of us can talk them into doing so.  But consider.....

The drug approval may be at a tipping point.  The FDA may have ample proof to approve the drug FOR ALL past TX failures.  If the don't they very well may have it in weeks.  We don't have the data....the final data but from the SVR12's provided by Vertex months (and months) ago there was a comfortable superiority of the SVR rates respectively between triple therapy and SOC.  That is all that is required for the FDA to approve this drug since there is nothing superior available (barring safety issues, that is).

This is a drug that deserves an early approval.  We are talking about many lives on the line here.  In such a circumstance.....should we NOT even ASK?  LOL: should we just wait and hope that they do the right thing?  They always do the right thing after all; right?  
I don't always think so.  I think that some drugs have been held up (re-visit the morning after pill in the abstinence era), some have been approved too easily (my father died after being treated with Vioxx, but before the recall broke).  This is not a organization that always does things right.  Approving the wholesale use of the drug for past treatment failures or at the minimum for compassionate use could save many lives.  It is pretty much a no-brainer that many lives would be saved.  This is something that they could do right.  If you recall there was a great need for action during the AIDS epidemic.  The FDA has to look at the scope and magnitude of this drug and our situation and factor that into their timetable.

Speaking of HIV and AIDS.  That group of infected people really shook things up.  They got some media attention, they wrote letters, petitions, their congressmen and they took to the streets.  
...........And the government acted.

Then there is us with HCV; the SILENT KILLER.  
It's true.  The disease takes many people because they wait until it's too late to act.  I think that it speaks of us....the HCV infected.  All too often we remain silent.  We don't speak up.  We don't write our congressmen.  We choose to remain invisible and silent.  It's one reason that HCV gets about....what is it....5% of the funding that HIV has gotten for years although we outnumber them 2:1  More of us will die from HCV each year than from HIV.  What will it take for us to break our silence?

Should we ask for something?  GOD forbid; they might say no....... so .....lets not.  Let's don't.
Lets stay silent.  

I think it's called the silent killer for more than one reason.  

I say; lets do.
     Lets ask.  Lets explain what we want and why we want it.  Lets say who we are......  what we do for a living, where we live, that we have family.  That they can save our lives.  That in doing so..... for each person that they save by allowing compassionate care that they can save a life and reduce one person from the rolls who would need a liver transplant.  By reducing the rolls one person awaiting a transplant a second person can *also* be saved.  That person may be a veteran who acquired the virus serving our country.  It could be a mother who needed blood while giving birth to a child 30 years ago, or a first responder.  It could be a child now grown infected through vertical transmission.  It could be a hemophiliac who received tainted blood.  The FDA can give the gift of life by recognizing the need and by acting.  We have been waiting and we have been dying.

But first..... we may need to act.  
All that we need to do is ask.

Well...... that's my stimulus package.
  
If they are going to save some taxpayers lives they are going to need to act and quickly.  They are going to need to take some actions that convey that they will finally grant this population some of the care that was given to HIV infected citizens.  They can act and save lives..... or they can wait.

I only know that I will not wait to ask.  ; )

best,
Willy

[fretboard]

I just wrote mine, the quicker telaprevir comes out, the sooner other things will come out.  You never know what's gonna happen when you write an email to a federal agency, but it's certainly not gonna hurt anyone and it just may help.  Besides that I signed up for a couple of their email newsletters while I was there.  latr

[pjhep80]

excellent work mag i will email today also

[smaug48]

sent the first...

smaug

[Isobella]

Heartfelt thanks to you for this thread.  I am SO on-board.  

Working today, so I'll have plenty of time to send e-mail(s).


Nicely said, Willy =)

[portann]

Fantastic what you made happen here. Something's coming of this, I can feel it.

Maybe they'll send you home with loads of extra gift boxes of P.I.'s to acknowledge your major role in successfully lobbying for these meds ahead of schedule!

A really constructive move - thank you for being so much on the ball.

[nygirl7]

fyi when sending them an email always open a new browser up and don't just click it off this page otherwise if they do look (because of a surge in letters) they will see that all the letters are coming from this website - that makes it look  more like we are spamming them then if individuals are just writing.

It does make a difference.  Don't ask me how I know these stupid things........but it's true.

And keep it to as few words as possible, that helps too.

>>>Use bullet points if you can they stick out more than long paragraphs.

(Yeah I've been involved in some serious heavy duty email campaigns before). I'll post what else I remember if anything is important but that should help.

[portann]

.

[Willy50]

OK.... you made your point.      (.)     ; )

That has to be the briefest post ever; even more abbreviated than replying with a "smiley".

Willy

[Magnum]

As I stated in my opening, we must bombard the FDA daily with emails. I won't post my daily emails here to them, but here's my second day's email. Please make it a habit to email DAILY...

Regarding the drug Telepravir (VX-950) by Vertex Pharmaceuticals, the drug with enormous promises to rid the Hepatitis C virus. There are MANY of us who either have Cirrhosis or at the beginning of Cirrhosis (as myself) who desperately need this drug released to us NOW as a “compassionate” exception before the clinical trials are over. As you may know, it is in the final (Phase III) of clinical trials with astounding results, and no deaths reported.

When the Hepatitis C virus reduces the liver to Cirrhosis, it becomes more and more difficult to treat with drugs. At some point, when the liver is at such a damaged state, treating the liver with drugs could be fatal.

I urge you to consider this as a dire need for us with Cirrhosis to have this drug available NOW! Many lives could me saved, many millions of dollars could be saved by Medicare and other insurance companies to bypass a liver transplant alternative.

As the AIDS epidemic arose, the FDA put the appropriate drugs through a fast track to approval. Be aware that there are millions infected with Hepatitis C. Many are soldiers back from war. America is the most humane country in the world. The “compassionate” release of the drug Telepravir (VX-950) is your duty to your citizens. Please act NOW!

Magnum

[Rockerforlife]

http://www.fda.gov/comments/webform.html


This is a good link to contact th FDA

This the letter i sent...not the most detailed..but i made my voice known.


This is a topic i think deserves special attenttion,We have people dying left and right just because treatment for Hep C is not aprroved by the FDA yet,i do believe as human being we have to be compassionate in cases where our fellow brothers and sisters need these newer drugs ....the protese inhibitors i am referring too...these drugs are currently in ongoing trials with amazing SVR rates...with little side effects or the same as SOC  treatments...Please help the ones who need these drugs now...its a do or die situation for some....imagine if this was one of your loved ones...???

[Rockerforlife]

http://www.fda.gov/comments/webform.html

[Emilio44]

Could I please start by thanking you for initiated this initiative you are indeed a champion. Today I intend to put a backgrounder and a link to this information on my home site of 'hep c australasia' .  I think it's important that we also get this information to all sites like Janis, Nomads and others. Great letters everyone I'm sure you will make the difference.  Lastly can I say to everyone who reads this thread is that your letter/email does not have to be extensive, spell checked or grammatically correct, just to the point.  It's okay to write something like 'Hi my name is Emi and I have hep c, I've been battling this disease for a long time, please please can you approve this drug now for special cases so I don't have to watch anymore friends die of end stage liver disease and to cure myself'.  So anything that is to the point will do. REgards Emi

[Rockerforlife]

How bout im dying and i need help....no beating around the bush with me

[Tippyclubb]

THIS IS THE RESPONSE I RECEIVED FROM FDA :

Thank you for writing to the Division of Drug Information in the Center
for Drug Evaluation and Research (CDER).  
  
Please note that under the Freedom of Information Act (FOIA), we cannot
comment on products that have not been approved.  However, we can
provide information on the drug review process.  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.  For more
information accessing unapproved products and single patient/emergency
IND, please visit the sites at http://www.fda.gov/cder/cancer/access.htm
and http://www.fda.gov/cder/cancer/singleIND.htm  

If you have further questions on this information, please contact our
Division of Drug Information at 1-888-463-6332 or our direct line is at
301-796-3400.

Sincerely,

Division of Drug Information LL
Center for Drug Evaluation and Research
Food and Drug Administration

This communication is consistent with 21 CFR 10.85 (k) and constitutes
an informal communication that represents our best judgment at this time
but does not constitute an advisory opinion, does not necessarily
represent the formal position of FDA, and does not bind or otherwise
obligate or commit the agency to the views expressed.

[Magnum]

I received the same “ore-prepared form”statement form the FDA as Tippyclub. Doesn't matter to me, I will still bombard them. That's the whole point. Passing the buck to your doctor is a way of sometimes saying "don't bother us, talk to your doctor to do something about this".

We all know doctors are very busy with dozens of patients a day. Can we all count on them to write this necessity letter to Vertex? I say bombard the FDA into submission. No letup, no mercy, no giving in, no capitulating...no, no, no... You don’t win battles by quitting..........

Magnum

[fretboard]

You should have your doctor involved in the issue of getting “compassionate use of telaprevir” b/c although it’s a good idea to send the fda emails, it’s time to focus and work on a sure thing.  Having worked for a regulatory agency you absolutely must follow all guidelines that are in place.  I pulled this information off of a cancer site, I left the link as well.  good luck

"Patients who are not eligible for either clinical trials or an expanded access program (if one exists) may be able to get the unapproved new drug by applying for single patient access. In this case, the patient's doctor must first request permission for access to the drug from the drug company. If the company agrees, the patient's doctor works with the drug company to ask the FDA to approve the drug for use by this one patient. The length of time it takes to get single patient access varies. But if it is an emergency, the FDA can complete the paperwork in 24 hours".

http://www.cancer.org/docroot/ETO/content/ETO_1_2x_Compassionate_Drug_Use.asp

[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.

[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]

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.

[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.

[HCA]

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

[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

[can-do-man]

Seems everyone is thinking only about the FDA or the doctors, I'm not so sure the drug company would be real interested in this and really why should they??? They are just now doing trials on us cirrhotics, they have a lot of money tied up in this drug. At this point do they really want to risk maybe tying this FDA approval for what could be years if just 1 case went bad whether it would be the drugs fault or out of their control? I just don't see the benifit to the drug company when they are just now testing cirrhotics.

Plus what insurance is going to cover it, showing the shelf life of telaprevir, pricing of the drug, alot of things involved in this when their into trials and about two years out for FDA approval................. Just my thoughts.

[desrt]

That would certainly make the reference to "a definite timeline for compassionate release" in the original posting make more sense. The FDA can't give off-label approval to something doctors don't have access to. I guess all these letters should have been going to Vertex instead of the FDA.

[Magnum]

Yes, but can the FDA persuade the drug companies to simplify this request for "compassionate" release. Let's be careful to not lose thought of what we’re trying to achieve and instead look at all the down sided negative possibilities. I keep referring back to the AIDS crisis. It was the pummeling, no let up persistence that forced both the FDA and the drug companies onto a fast track release. Otherwise, it's up to you...

Magnum

[can-do-man]

I agree with you, vertex is the one to pressure,

As for the FDA to "pressure" a drug company into early release with NO data on people with cirrhosis......... Hmmmmm

[fretboard]

"It was the pummeling, no let up persistence that forced both the FDA and the drug companies onto a fast track release". Otherwise, it's up to you...

So true, the pummeling plus a lawsuit that was won.lol  The clip I leave gives one the idea that "compassionate drug use" is limited.  I'm sure other people/drugs have been approved, but off of wikipedia, this was all I could find.  The compassionate drug use idea was made possible in 1978 when this man was trying to get medical marijuana from his pharmacy for use on his glaucoma.  Oh, the article says the program was shut down by George Bush.  If he shut it down, I don't think it's shut down anymore.  latr

The settlement in Randall v. U.S. became the legal basis for the FDA's Compassionate IND program. Initially only available to patients afflicted by marijuana-responsive disorders and orphan drugs, the concept was expanded to include

HIV-positive patients in the mid-1980s. However, because of the growing number of AIDS patients throughout the late 1980s and the resulting numbers of patients who joined the Compassionate IND program, the George H. W. Bush administration closed the program down in 1991. At its peak, the program had thirty active patients.

[can-do-man]

Why does a good thread like this have to turn to polictics??? Its quite clear from the response tippyclub got from the FDA it can still be done. Its just up to the drug company and your doctor.

FROM THE FDA

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

[portann]

I didn't even know the FDA's position until this thread got started. I'm pretty happy to see it in black and white:

Just to cut and paste again from above posts:
------------------------------------------------------------------------------------------------------
FROM THE FDA

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.
------------------------------------------------------------------------------------------------
So there's a policy in place to make it do-able NOW.  

I wonder how many docs have enough experience with cirrhotics to sign off on this? It seems the best docs would have the confidence and expertise to proceed but others would hesitate. And the best docs would have the application process streamlined, perhaps able to make it happen within twenty-four hours.

[HectorSF]

"Emergency Use of an Investigation Drug or Biologic" For more info go to
http://www.fda.gov/oc/ohrt/irbs/drugsbiologics.html#emergency

UNITED STATES

Prepared by the Food and Drug Administration, Office of Special Health Issues

Introduction The acronym "IND" refers to an Investigational New Drug application, which is an application to the Food and Drug Administration (FDA) to administer an investigational new drug or biologic product to humans. Since a biologic is also considered a drug, the term "drug" as used in this document refers to either a drug or a biologic product.

Most human use of investigational drugs takes place in clinical trials conducted to assess safety and efficacy of those drugs. Consequently, FDA's IND processes are designed primarily for drug companies and medical researchers engaged in clinical development of new drugs. However, some patients not eligible to participate in a clinical trial may benefit from treatment with an investigational drug under study. For such cases, FDA has developed numerous regulatory mechanisms to make investigational drugs available outside of clinical trials. These mechanisms include single-patient INDS, emergency INDS, and treatment INDs/protocols.

Single-Patient INDs and Emergency INDs

In many cases, the investigational drug will not be available through an expanded access program from the manufacturer. For such cases, the discussion below addresses how a physician can file a single-patient IND or an emergency IND for treatment of a patient with an investigational drug. In the case of a single-patient IND or an emergency IND, all of the following conditions must be met before FDA can allow a physician to administer an investigational drug under a single-patient or emergency IND:

    * a) The patient must be informed about the drug and consent to be treated with it.
    * b) The physician must be properly licensed and must agree to administer the product and be responsible for monitoring and reporting to FDA on the patient's use of the product (See forms 1571 and 1572.)
    * c) The local Institutional Review Board (IRB) must approve the proposed use of the investigational drug (please note that in emergency situations it may be possible for the physician to notify the IRB promptly after treating the patient),
    * d) The manufacturer must be willing to provide the product without charge (unless the sponsor has applied for and FDA has allowed charges for cost recovery as provided for under Federal Regulations), and
    * e) FDA has allowed the transfer of the product from the manufacturer to the physician.

All Biological IND submissions must be made in triplicate and should be addressed as follows:

Center for Biologics Evaluation and Research
HFM-99, Room 200N
1401 Rockville Pike
Rockville, MD 20852-1448
Phone: [phone] or 301-827-1800

Additional information and and all forms may be obtained at the FDA's Center for Bioloics Evaluation and Research (CBER) web site

http://www.fda.gov/cber/ind/ind.htm

Additional FDA information about the IND program can be found at
Investigational New Drug Application process


Hectorsf

[fretboard]

"It seems the best docs would have the confidence and expertise to proceed but others would hesitate".

Unfortunately due to the stigma that surrounds HepC I have to agree that alot of doctors would hesitate and even shy away from getting involved.  Also unfortunate is that we don't have truckloads of supporters like the HIV movement did back when they forced the fda's hand.  latr  

[Upbeat]

There has to be something we don't know about.  This is by far the slowest FAST TRACK drug ever developed.  I have heard of several drugs approved long before Phase 3 is completed.  If and when this is approved will it give them more slack on their other pi's and the combo they are working on? In other words will they be able to forgo the rigorus phase one and two data requirments for next generation pi's.?

                                                                                                                     Ron

[Magnum]

Monday morning I'm going in for banding of some Esophageal Verices by my Gastro, who is also a surgeon. I'm going to run this by him and see how easy or difficult it will for him be to ask permission for a "compassionate" release of the drug by Vertex, for me, to be picked up by prescription. Should be interesting. I will report...

Magnum

[Rockerforlife]

Another point i want to bring up...the daily injections of inerfergen is punishmet, brutal in my opinion....the PI drugs are not even close to it in terms of SX....so why is ok to almost kill your self with interfergen,but its not ok to save yourself with the unapprovved PI`s....

[Willy50]

As mentioned...... Telaprevir has a unique opportunity to be approved or treatment failures only based on, at least in part the results of Prove 3 (which was for past treatment failures).  I'm sure that they look at the toxicology reports of other trials; not only the single trial.  Currently over 2000 patients have been treated with Telaprevir, so the FDA has a decent file on it's safety profile.

My understanding is that the FDA will look at the safety records and efficacy rates and at least has the opportunity to provide early release for past treatment failures based on the fact that no other form of treatment exists with a close efficacy/safety profile.  We still don't have the official SVR rates but the SVR12 rates suggested a comfortable improvement over current SOC.  

INSTEAD of causing doctors to write letters, investigate "compassionate care" (which I wager is a means of treating that a fraction of a decimal point of doctors are familiar with) the FDA could simply approve the drug for the wholesale use without special appeals, paperwork, the learning curve in filing compassionate care papers.  I don't think the current system is favorable to accommodate the vast numbers of people who could benefit from an improved form of treatment.

*  It could be a speedy, or relatively speedy process.
*  Inherent risk would be outweighed by the greater good.
*  It could cause a reduction in the number of people awaiting TP.  
*  It could in addition to reducing liver damage reduce other co-morbidities/ extra hepatic diseases and damages associated with advanced liver damage.
*  Provide the most cost effective means of treating this group- curing them instead of treating the effects of cirrhosis.
*  Dramatic reduction in healthcare; it is far easier to treat a patient and cure them than to allow them to proceed into liver failure, transplant them, then keep them on anti-rejection meds for life and THEN retreat them for HCV.  That is folly.  It's inhuman.  It is unacceptable.
*  Early approval for past TX failures is a humane and compassionate act.
*  The early approval of Telaprevir would make a drug available for a large group of people with minimal paperwork and with no other reasonable means of treating.

It's the right thing to do and the right time to do it.

best,
Willy

[portann]

I'm entirely out of my depth here but went to school with a thalidomide boy. Doctors in Europe, Australia, New Zealand and Canada were prescribing thalidomide left and right to pregnant women with morning sickness.  The U.S. stood alone in deciding it was potentially dangerous to approve it and avoided countless tragedies.

I'm very excited about the PI's and wish I'd waited for them myself.  I think until the final results are in, though, the current policy is the most reasonable one. It serves people with cirrhosis very efficiently, at the same time that it makes clear the final results are still in the making. Emotions and anecdotes don't make a drug safe.

[Rockerforlife]

PI drugs have been used for HIV patients for years now...i dont think the SX from these drugs are  dangerous...i say lets "push" the PI`s now.

[Rockerforlife]

Little  of topic...but still interesting


Some herbs and supplements, such as St. John's Wort and garlic supplements, can affect the level of protease inhibitors in your blood.

http://www.aegis.org/factshts/network/simple/protease.html

[Rockerforlife]

Now i just found out the PI`s used for HIV are different than the PC`s used for HCV...different animal all together...

[Willy50]

Good point and of course, Vioxx was much more recent.  One needs to keep in mind that this is a disease or treatment in which there are NO other elective therapies which exist with any significant hope of cure.  There exists no path such as maintenance therapy which provides a means of prevention of continued damage.

People with advanced liver staging face a daunting list of hurdles.  Since the cure rate drops for that population they often must do higher dosing or much longer treatments.  They have generally speaking less reserve in the ability to take drugs as they approach decompensation, indeed, doing chemotherapy could cause them to decompensate.  They face a myriad of complications as they move towards ESL and even more issues after (and if) they get a transplant.  For this group there is a window of time which they can treat and after which are too liver compromised to treat.  Allowing the natural course of FDA approval when there exists a solution is a death sentence for some of these people,  For others it may force them to go thru the ESLD (encelepathy, banding of ascites, draining of fluid from stomach, ect) before getting a transplant, after which one is on anti-rejection meds for life and constant medical monitoring.

Some of these patients could merely be allowed to treat with a drug which by most estimates will certainly be approved and offers dramatic efficacy improvements over current treatments.  Making some of this group of people wait 2 years will certainly allow some to die and others to await a liver.

Early approval is allowed by the FDA in it's own guidelines.  It has to meet standards of safety, efficacy (I think these two are virtually guaranteed with current data), and finally..... the need.  Does the NEED exist?  Do the rewards outweigh the risks?  Perhaps we cannot answer that question without being doctors, having actuarial tables and projections for the population group ate risk who could benefit.

From the trenches..... from where I sit.... the early approval looks like a wise and humane thing to consider.  It looks like it could save lives and untold amounts of dollars through the early CURING of people instead of treating them in ESLD or post TP.

Sometimes the standard can be used; what would you want for your child....for your parent if given this choice?

This equation is not about exposing unborn children to risk.  It is about refusing to allow people a drug that could save their lives or forestall the risk of entering into ESLD.  The FDA does not have to allow this drug to proceed down the normal pathways and chutes which will almost certainly make it approved in 2 years.  They have the latitude to allow it's early approval.  At minimum they have the ability to expedite or simplify "compassionate care" provisions.

best,
Willy

[Rockerforlife]

Cool article you published...well written.

[can-do-man]

Then why isn't VERTEX pushing for this??? Because they are not. Hmmmmm

[Willy50]

"Then why isn't VERTEX pushing for this??? Because they are not. Hmmmmm"
------------------------------------------------------------

I can't read their minds.  I can't "read" the FDA.  I don't know even what you are thinking.  : )

Are you suggesting that Vertex should?  And in the absence of Vertex doing so it means something?

We are *reading tea leaves* here but here's my take on it;

1)  They don't cause they can't
2)  they don't cause they shouldn't
3)  they don't because the risks of seeming "pushy" could cause the FDA to slow things down further
4)  It might even protect them if early approval came and some unforeseen issue came up, such as Portann alluded to with Thalidomide or I mentioned with Vioxx.

Understand; the fact that they are not doing it is open to interpretation.  Reading between the lines if a *Vertex rep* suggested that Magnum write the FDC then you might have a glimpse at what they may hope for.

I personally think that it would be a double edged sword.  They may not actually be in favor of it because they are not quite ready.... not set up to manage a "commitment to care" type operation.  On the other hand I would guess that their stock would SOAR.  It would be a wonderful "problem" to have; OH DRAT!!!! Profit during a depression..... D*mn; first to market!!!  : (

....  Just my guess.

While guessing...... I just feel that they have run their trial process very carefully.  They didn't allow rescue drugs in phase 2 when other companies did.  They ran a far more blinded study than any of the other companies did; at least for Scherring (boce) or the recent Roche (r1626) studies.  Could it be that they are bending over backwards to get approval?  If Roche or Scherring got delayed or thrown out of trials their company would continue.  It would be a catastrophe of the first order for Vertex.  My FEELING is that they will not risk offending the FDA nor would they risk in any way compromising their current favorable position by applying pressure to the FDA.  It would probably be counter productive.  What's your opinion?

Finally, keep in mind that ones personal goals are not always perfectly aligned with either Vertex or the FDA.  They want a smooth transition.  
(and on the other hand) ...Some of us might want to not slide into decompensation.  Both Vertex and the FDA have to take a detached and "long" view.  That doesn't mean that one cannot or should not try to impact upon the approval process.  IF indeed it is at a tipping point public demand could allow it to move one way.  If the end point audience doesn't care...... well..... perhaps no one else will either.  If the drug is not safe, then no amount of banners or slogans will make it become approved.

My opinion only....

Willy

[Trinity4]

Wonder if Pegylated Interferon went through the same approval processes and how it was viewed by those treating with alpha interferon only and how and when the introduction of Ribavirin was considered during that time.


Treatment Timelines

1991 - FDA approves first alfa interferon (Schering's Intron A) to treat hepatitis C.
1992 - FDA approves first interferon (Schering- Intron A) to treat hepatitis B.
1996 - FDA approves alfa interferon (Roche- Roferon A ) to treat hepatitis C.
1997 - FDA approves consensus interferon (Amgen- now InterMune-Infergen) to treat hepatitis C.

The general treatment protocol was to inject 3 million units of interferon, three times a week for 48 weeks. Sustained virological response rates (negative viral load 6 months post-treatment) were approximately 9% for genotype 1 and 30% for genotypes 2 and 3.

Treatment Breakthrough

1998 - FDA approves Rebetron (Schering's Intron A plus ribavirin) for the treatment of hepatitis C.

Ribavirin is a synthetic nucleoside analogue with a broad spectrum of antiviral activity that was initially developed as a possible treatment of HIV. As it turned out, ribavirin was not effective against HIV, but it was found that it did have antiviral activity against several flaviviruses (a family of viruses that includes hepatitis C), and it was studied as a single agent for the treatment of hepatitis C. In some small studies, ribavirin was found to reduce serum ALT levels, but that it had no effect on the hepatitis C virus. The clinical findings that ribavirin reduced ALT levels led to the studies of combination ribavirin and interferon therapy. It was found that ribavirin when combined with interferon produced a snygery that proved to be a major breakthrough for treating hepatitis C. Ribavirin (in a mist form) is also approved for the treatment of respiratory syncytial virus (RSV) infection in children.

The treatment with combination therapy consists of interferon (Intron A - 3 million units thrice weekly) plus ribavirin (800-1200mg/day). The clinical trials conducted on combination therapy also determined the duration of treatment for genotype 1 as 48 weeks and 24 weeks for genotypes 2 and 3. Overall sustained virological response rates are genotype 1 - 29% (high viral load - 27%); genotypes 2 and 3 - 62% (high viral load - 60%).

A New Era in the Treatment of Hepatitis C

Synthetic interferon is a protein that is broken down rapidly by the body within 12 to 24 hours after injection. The standard protocol for interferon was to inject 3 times a week. The synthetic interferon was eliminated by the body, and, without further interferon available, the body could not suppress or kill the virus.

Pegylation is a process that attaches polyethylene glycol (a biologically inert compound) strands to the interferon molecule making it less likely to be cleared from the bloodstream. The benefit of increased concentrations of interferon levels is that these help to constantly suppress the virus and increase the likelihood of a sustained virological response.

2001

Peg-Intron (Schering’s pegylated interferon alpha-2b) was the first pegylated interferon FDA approved to treat hepatitis C. Peg-Intron is a powder that needs to be reconstituted (with a sterilized solution) before it can be injected. Peg-Intron also needs to be dosed by a person's body weight.

The sustained virological response rates for Peg-Intron monotherapy are 14% for genotype 1, and 47% for genotypes 2 and 3.

PEG-Intron plus Rebetol (ribavirin) was also approved in 2001 to treat hepatitis C. Sustained virological response rates are 42% for genotype 1 (high viral load - 30%) and 82% for genotypes 2 and 3.

2002

Pegasys (Roche's pegylated interferon alpha-2a) was approved to treat hepatitis C in 2002. Pegasys comes in a ready made solution (does not need to be reconstituted) and in a dose fixed at 180 micrograms regardless of a person's weight.

The sustained virological response (SVR) rate for Pegasys is 28% for genotype 1, and 56% for genotypes 2 and 3. People with advanced fibrosis or compensated cirrhosis (a group that is more difficult to treat) achieved a SVR of 20%. This clinical trials on cirrhotic patients also showed that Pegasys reduced liver inflammation and scarring in treatment responders and, to a lesser degree, in non-responders. Data from this trial and other conventional interferon clinical trials led to the NIH HALT C trial that is studying the role of interferon in reducing liver inflammation and slowing or 'stopping/halting' liver disease progression.

In 2002 Pegasys plus Copegus (Roche's brand of ribavirin) was also approved for treatment of hepatitis C. Sustained virological response rates for genotype 1 are 46-51% (high viral load 41-45%) and 76-78% for genotypes 2 and 3.

[can-do-man]

I have talked to vertex, have you??? Back in july when magnum first posted about the trial me and kathy from here were interested, as time went on nothing so i talked to my hepatologist, he said that both pi's would be starting a new trial soon. Told me the critria and vertex was not allowing rescue drugs. He would not choose one drug over the other.

I had read about this time last year where vertex might go for early approval, knowing i needed procrit early on and was a relapser and not wanting a dose reduction i called vertex and talked with two different people. I was told by them that vertex plans was now to do this prove 3 trial. After explaining being stage 4 and needing rescue drugs i asked about this compassionate care. (Which magnum had also posted about back then) I was again told to try and get in a trial as that was how they were proceeding. I took that as a NO.

So if vertex is not interested how can the FDA force this on them???

What i don't understand if you have a good contact at vertex and a doctor with the status of dr Gish who says treat with telaprevir WHEN it comes to market why not go through them and get this done??? The FDA allows it.

[can-do-man]

prior post.......What i don't understand if you have a good contact at vertex and a doctor with the status of dr Gish who says treat with telaprevir WHEN it comes to market why not go through them and get this done??? The FDA allows it.
-------------------------------------------------------------------

With Dr Gish and help from a contact to get this done now maybe would open the door for other hepatologists to go for it as it seems dr. has alot of respect.

[Willy50]

One does not need to "talk" to anyone at Vertex.  They have been saying for about a year that they plan to provide the FDA with the data from Prove 3 as a registration run and enter into talks with the FDA on the possibility of an early release for past TX failures.  That should occur by mid year.  They have thus far refused to speculate further on much more about those talks.  My *assumption* is that if they didn't want an early release or that if they didn't think it possible or desirable that they would not initiate talks.

Maybe I'm missing something......

Willy

[HCA]

Willy and all,
I think the overiding concern for Vertex and their partners,Johnson&Johnson,Roche ect is providing shareholder return on investment.
In order to achieve maximum profit the telaprevir brand needs to achieve proprietory superiority over it's rival boceprevir.
I think both parties are playing cat and mouse over the data,each wanting to gain the edge.The fact that their are two leading contenders neck and neck may have slowed things a little
Vertex,the FDA and everyone in the industry know about the pressing need for the drug to get to market-they are quite well aware of the human cost of passing time and don't need to be reminded by patient groups.
As business corporations who have invested billions of dollars in getting this incredible drug this far down the line they now want to reap the rewards in an orderly and sustainable way and I don't blame them.There is additionally the safety diligence.
Anyone who has a problem with this is quite entitled to invent their own cure.
As has been stated numerous times the market expects Vertex to file for approval late next year.
Compassionate release for those who cannot wait can be petitioned on a case basis by individual doctors,although I don't believe Vertex has yet referenced a compassionate programme.
The drug companies know that the boom sales time for the protease inhibitors is quite a small window and want the patients to have the drug and spend the money rather than to progress to a stage where they can't treat.
I don't think there is much of a conflict of interest here,and those who are worried will get treatment in time as the natural history of Hepatitis C cirrhosis is slow with upto 70% of compensated cases remaining compensated ten years post cirrhosis diagnosis.
These are my last words on this thread although I'll continue to follow the debate.

[CoWriter]

"Some herbs and supplements, such as St. John's Wort and garlic supplements, can affect the level of protease inhibitors in your blood. "
--------------------------

St John's Wort should NOT be used together with any type of interferon since it interferes with its effect.  And garlic can cause/increase bleeding.

Co  

[CoWriter]

I worked in Clinical Research as a Study coordinator for 11 years so perhaps I can shed some light on the "compassionate use" process.

In 11 years that I worked in research, we obtained FDA approval for compassionate use of a medication only twice.  

The first case was a patient who had severe gouty arthritis which was causing severe deformities to feet and hands...worse case ever seen by any of the universities that were consulted and the patient even had several toes amputated because the gout had caused huge lumps.  The patient was severely allergic to all gout meds (anaphylactic reaction/respiratory failure) so there was nothing else that could be used.

The doctor wanted to use an old gout medication that wasn't even used anymore.  He  contacted the drug company and convinced them to let him have the medication to treat the patient.  Then we had to take the case to our hospital's IRB (Institutional Review Board) and get them to approve treating the patient with it.  

An Institutional review Board is an ethics committee.  Their goal is to protect the rights and welfare of research subjects (they're the ones that approve any studies done at a  research facility along with the consent that will be used).

We had to prove to them that we were doing everything in our power to make sure the patient would be safe.  Since she was so allergic to all other gout meds, the doctor made arrangements to have the patient be hospitalized to receive the first few doses of the med.  Also have a Pulmonary/Allergist doctor be on the case.  He would take care of allergic reactions if they happened.  

We had to get the "protocol" approved and also a consent that the patient had to sign saying that the hospital, doctors and drug company were not responsible if anything happened to her and would not pay for anything if she had any problems.  Mind you, that doesn't mean that if something bad happens the doctor is off the hook.  The doctors were responsible for taking care of side effects, etc.   We were also responsible for giving updates to the IRB and letting them know immediately (within 24 hours) if the patient had any side effects.

Once we had the IRB approval, we submitted it to the drug company...got FDA approval....and the drug company then sent us the drug.

By the way, drug for compassionate use is FREE to the patient.

------------------------------------

As you can see, the process for getting a drug approved for compassionate use is not as simple as you thought.  First of all, you have to prove that the drug you want to use is the ONLY option because there are no other available treatments that are satisfactory.  That would be very difficult to prove in the case of Telaprevir.  There ARE other drugs that can be used to treat Hep C, like Infergen, etc.

The doctor is the one that starts the process to obtain FDA approval for compassionate use of a drug.  And most doctors are not willing to go through all the hassle when they're not getting anything out of it.  Even if you sign a consent saying that you don't hold anybody responsible, the doctor is still responsible for your well being, stopping the drug if there are problems, getting IRB approval (which BTW, costs money if an IRB not associated with a hospital is used.  We're talking HUNDREDS of dollars), maintaining records, keeping drug accountability records, sending updates to the IRB and FDA, and also sending letters to the IRB and FDA regarding any serious adverse events caused by Telaprevir in any drug trials (the drug company would be responsible for letting the doctor know about them).


I read some of the emails you're sending to the FDA and with all due respect, some of the things you said will work against you.  When you said.....

"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. "

You basically told the FDA that doctors don't have the capability or the time to comply with the guidelines for obtaining a drug for compassionate use.  You truly did yourself no favors by saying all that.


I think you misunderstood your contact at Vertex.  What you should be trying to do is get the FDA to FAST TRACK approval for cirrhotics to user Telaprevir.  In other words, to give approval for only cirrhotics to use it before it's approved for everybody else.  Sort of like they did with eltrombopag (the drug that raises platelets).  The FDA granted approval for use ONLY on ITP patients thanks to the unanimous recommendation by the FDA ADVISORY PANEL.

And that is who I think you should be sending all your emails to.  They're the ones that make recommendations to the FDA to fast track a certain drug for a specific group of patients.  

http://www.fda.gov/oc/advisory/default.htm

Best of luck to you.

Co
P.S.  Do I believe that Telaprevir should be fast tracked?  No.  I think there's still alot about that drug that we don't know about.  For example, Tippyclubb and Bklnboy are both in the same Telaprevir trial and they both have high uric acid levels which is associated with insulin resistance, oxidative stress, high blood pressure (and Bklnboy recently developed high blood pressure needing medication) and renal disease.

[Magnum]

Thanks for your input. After PegIntron failed twice, Pegasys failed once, Infergen failed once, I bring attention to your own comment...

"First of all, you have to prove that the drug you want to use is the ONLY option because there are no other available treatments that are satisfactory.  That would be very difficult to prove in the case of Telaprevir.  There ARE other drugs that can be used to treat Hep C, like Infergen, etc."

I four failed treatments with every immaginable drug offered so far isn't proof that nothing works for me and others on this board, then?

Thanks,

Magnum

[Rockerforlife]

How about in cases like , Susan,she has treated 10 TIMES....if  she cant get the 'compassion drugs"...there seems to be a problem with this picture.

[Willy50]

In 2 years a lot can happen.......
Just data, no argument.

http://www.nationalhepatitiscinstitute.org/Data/Transmission/CDCdeathrate2010.htm

The National Association of Community Health Centers, Inc. (NACHC) There are approximately 30,000 cases of acute hepatitis C each year in the U.S. Around 55 percent to 85 percent of people who are exposed to the virus become chronically infected. The Centers for Disease Control and Prevention (CDC) estimates that the number of deaths from end-stage liver disease in the U.S. alone will reach 30,000 to 40,000 annually by the year 2010.


--------------------------------------------------------------------------------

AS MANY AS 26,000 PEOPLE WILL DIE
because of Hepatitis C in 2006

This means
EACH HOUR OF EVERY DAY 3 PEOPLE DIE

[CoWriter]

She was IR.  That is one of the biggest things that makes people fail tx.  You can't expect tx to work if you don't fix the IR.

Co

[Magnum]

Just returned from the banding of 4 Esophageal Verices. Still a little grogy, but I want to add that the doctor predicts 4 more procedures to band them all. I mentioned this before. Everyone with Hep C should get tested for Esophageal Verices. This, the doctor agrees with.

I presented the doctor with the protocol for applying for a "compassionate" release of Telepravir. Stay tuned... I will report...

Magnum

[Rockerforlife]

I recieved a reply to my letter:

Thank you for writing to the Division of Drug Information in the Center
for Drug Evaluation and Research (CDER).  Your inquiry has been
forwarded to our office for assistance.

Please note that under the U.S. Freedom of Information Act (FOIA), we
cannot comment on products that have not been approved.  However, we can
provide information on the drug review process.  There is a mechanism to
access products that have not been approved.  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.  If you know of anyone seeking
information on accessing products not yet approved for specific health
condtion, please forward the below links to them.  Thank you.

For more information accessing unapproved products and single
patient/emergency IND, please visit the sites at
http://www.fda.gov/cder/cancer/access.htm and
http://www.fda.gov/cder/cancer/singleIND.htm  

[BelB64]

The article you quote from was written in 2006. That number of 30,000 to 40000 deaths per year is an estimate and only the actual numbers next year will bear out or refute this guess. The good news(if there ever is from these type of numbers) is that it is still quite evident that the vast majority of people with this disease will die from something else. If there are 4,000,000 with the disease and thousands more each year infected it would take 100+ years for everyone to die at these rates. Also the next few years should be the peak of annual deaths as the infection rate has seriously gone down since almost noone gets it from transfusions anymore due to blood screening. Hopefully new medications and treatments will reduce deaths to near zero annually in the coming years.

[Willy50]

Sorry; got in late.

You wrote;
"Also the next few years should be the peak of annual deaths as the infection rate has seriously gone down since almost no one gets it from transfusions anymore due to blood screening."
-------------------------------------------------------
The compassionate care or the early approval specifically addresses the needs of people in the next 2 years....give or take.  I don't think we should get side tracked with the demographics of the next 10-25 years.  Yes, after the boomers die or are cured there will be many less HCV patients.  I think that is a reason that HCV is sometimes called a self limiting disease.  I think if is was transmitted more easily we would have seen more funding.  : )  

If you think 2006 is old I'm glad that I didn't post this;   (lol; but I will now.  ; ) )
(Hey; it has only been 2009 for 2.5 months....... : )

http://www.hhs.state.ne.us/dpc/hep_c.htm
(about is from about 2003)

HCV Statistics

1 out of 50 Americans are infected with HCV.

3 out of 4 don't realize they are infected with HCV.

CDC predicts 4 fold increase of chronic HCV infections by 2015.

15,000 deaths/year in U.S. caused by HCV.

3 fold increase in annual death toll expected by 2010.

HCV is 4 times more common than HIV.

1 out of 3 people infected with HIV are also infected with HCV.

Hepatitis C is the leading cause of death for those individuals infected with HIV.

U.S. averages 35,000 new infections annually.

HCV financial burden is $15 billion/year.

Disease burden predicted to increase to $26 billion/year by 2021.
----------------------------------------------------------------------------------------

I don't want to hijack a thread.  If this is off topic I would be glad to continue the discussion elsewhere.

I think the actual numbers are hard to define since deaths get coded in various ways.  For me it is somewhat besides the point; would the numbers be more *acceptable* if they were halved?  Feel free to post the *actual number* ......but I think that we are speaking somewhat about principle.

This is not about getting a special treatment for one person who has gout. It is about thousands of people who will die if they do not get treatment SOON.  A treatment exists that will about triple (I'm guessing) the success rate for past TX failures.  It might be as little as twice as effective or up to 4 times as effective as current SOC would be in retreating that group.  The stats are blinded but will not be for much longer.  Vertex is seeking entering into talks about early approval for a certain group of patients and the FDA will respond.  I don't think that Vertex would seek to enter into talks with the FDA if they were not ready to treat these people or if they didn't want to.  

My point is that there is a huge need for a drug to help this population.  (I believe) The drug exists.  There is a HUGE expense in delaying approval.  This can be quantified in lives and in dollars (and we can argue about the mortality numbers or current or projected expense but halve the amount and it's still staggering).

I'm open to another thread unless this expansion of the topic is OK with Magnum.  

I am sorry if I ended up diverting it.  (I thought that early approval might also be as good or better than compassionate care, and might still provide earlier treatment)

best,
Willy