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

[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]

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

[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]

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.

[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.

[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