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

[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.

[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

[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.

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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.

[CoWriter]

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

[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.

[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