Aa
Perseverance Pays Off
Constant pressure through constant emails pays off, regardless of the doubters...
I received this email today from the FDA, outlining in detail what your doctor has to do to apply for the “compassionate” use of Telepravir. They point to Form 1571 for your doctor to fill. Follow the link... This is proof that if you put on the pressure, something has to give.....
Magnum
Thank you for writing to the Division of Drug Information in the Center
for Drug Evaluation and Research (CDER).
We understand your urgency in this matter. The FDA has created a
"compassionate use" or single patient IND program that allows patients
to receive investigational products before they are approved for
marketing.
A physician may file a single patient IND directly with the FDA. This
must be done with the cooperation and permission of the drug supplier.
The FDA has created a page specifically addressing the process of
developing and submitting a single patient IND,
http://www.fda.gov/cder/cancer/singleIND.htm. Please share this
information with your physician as they would be responsible for filing
out and submitting the required paperwork and forms to the FDA.
Please note that the physician (not the patient) must file this
information with the FDA and the drug manufacturer must agree to supply
the product to the physician. Please read through the attached single
patient IND webpage as you may be able to gather some of the required
information or forms for your physician and move the process along.
While this may seem to be bureaucratic busy work, the FDA has set up
this specific process to make sure that patients receiving
investigational drugs not subject to a clinical trial are not being put
at additional risk for harm.
Sincerely,
Division of Drug Information CJ
Center for Drug Evaluation and Research
Food and Drug Administration
I received this email today from the FDA, outlining in detail what your doctor has to do to apply for the “compassionate” use of Telepravir. They point to Form 1571 for your doctor to fill. Follow the link... This is proof that if you put on the pressure, something has to give.....
Magnum
Thank you for writing to the Division of Drug Information in the Center
for Drug Evaluation and Research (CDER).
We understand your urgency in this matter. The FDA has created a
"compassionate use" or single patient IND program that allows patients
to receive investigational products before they are approved for
marketing.
A physician may file a single patient IND directly with the FDA. This
must be done with the cooperation and permission of the drug supplier.
The FDA has created a page specifically addressing the process of
developing and submitting a single patient IND,
http://www.fda.gov/cder/cancer/singleIND.htm. Please share this
information with your physician as they would be responsible for filing
out and submitting the required paperwork and forms to the FDA.
Please note that the physician (not the patient) must file this
information with the FDA and the drug manufacturer must agree to supply
the product to the physician. Please read through the attached single
patient IND webpage as you may be able to gather some of the required
information or forms for your physician and move the process along.
While this may seem to be bureaucratic busy work, the FDA has set up
this specific process to make sure that patients receiving
investigational drugs not subject to a clinical trial are not being put
at additional risk for harm.
Sincerely,
Division of Drug Information CJ
Center for Drug Evaluation and Research
Food and Drug Administration
http://www.thestreet.com/_yahoo/story/10474626/1/vertex-hep-c-drug-improves-cure-rate-in-study.html?cm_ven=YAHOO&cm_cat=FREE&cm_ite=NA
Final Prove 3 results;
SOC 14% SVR Telaprevir triple therapy 51%
Keep in mind that this was the first go round for past treatment failure patients. A 4 week SOC lead in such as boceprevir does is currently in trials.
Note in the second Page Feuerstein mentions that Telaprevir is the ONLY compound in trials that has shown meaningful success of the past treatment failures.
Willy
Final Prove 3 results;
SOC 14% SVR Telaprevir triple therapy 51%
Keep in mind that this was the first go round for past treatment failure patients. A 4 week SOC lead in such as boceprevir does is currently in trials.
Note in the second Page Feuerstein mentions that Telaprevir is the ONLY compound in trials that has shown meaningful success of the past treatment failures.
Willy
sounds like you have a very good case to receive the trial meds and should be ok. Are you trying just for Telaprevir or Boceprevir as well? Best of luck
It remains to be seen, but my contact did not say or hint of anything negative. Of course, he's not the final decision maker, but according to my contact at the FDA, he sees this as a straight ahead proposition that Vertex should comply with, especially IF the doctor presents a strong case on a patient’s behalf and has all the medical proof, lab results, failed trials, etc...
When people mention David & Goliath, maybe so, but David did win...
Keep in mind that the doctor has to show an "emergency need". In my case, at the beginning stages of Cirrhosis, with liver and kidney cysts, Esophageal Varices, low platelets, etc... he considers this to be an emergency case in which I cannot wait for two more years.
I have no doubt that others on this board also have pressing physical issues, probably worse than mine, which should tug on the heart strings of Vertex. If they deny, I will appeal through whatever means, starting with the FDA, Surgeon General, and more. To deny me is start a storm of activity. I'm pretty determined, but I need the backing of everyone here and I need others to try as well... This is not after all, a one man battle... David was healthy when he slew Goliath, Unfortunately, I’m not...
Magnum
When people mention David & Goliath, maybe so, but David did win...
Keep in mind that the doctor has to show an "emergency need". In my case, at the beginning stages of Cirrhosis, with liver and kidney cysts, Esophageal Varices, low platelets, etc... he considers this to be an emergency case in which I cannot wait for two more years.
I have no doubt that others on this board also have pressing physical issues, probably worse than mine, which should tug on the heart strings of Vertex. If they deny, I will appeal through whatever means, starting with the FDA, Surgeon General, and more. To deny me is start a storm of activity. I'm pretty determined, but I need the backing of everyone here and I need others to try as well... This is not after all, a one man battle... David was healthy when he slew Goliath, Unfortunately, I’m not...
Magnum
Sorry for piece mealing this info to all of you, but I'm trying hard. There is a third form for your doctor to fill out...
http://www.fda.gov/cder/regulatory/applications/EIND.htm
This is the: Emergency Investigational New Drug Application (EIND) Information Request Form
That should hopefully do it. Now, it's up to you to present all this to your doctor and get the ball light speeding down the bureaucracy highway and to the final destination.... Good luck...
Magnum
http://www.fda.gov/cder/regulatory/applications/EIND.htm
This is the: Emergency Investigational New Drug Application (EIND) Information Request Form
That should hopefully do it. Now, it's up to you to present all this to your doctor and get the ball light speeding down the bureaucracy highway and to the final destination.... Good luck...
Magnum
I can understand HCA's concern and everyone else's about this jaw-dropping, astonishing, bewildering and shocking development. You read the FDA’s email to me...
Don't think I'm home free of concern either. Let's just see what transpires in the next week. By the way, I forgot to mention, the FDA contact stated the doctor has to file for an "emergency IND". Remember that...
Magnum
Don't think I'm home free of concern either. Let's just see what transpires in the next week. By the way, I forgot to mention, the FDA contact stated the doctor has to file for an "emergency IND". Remember that...
Magnum
Without reviewing exactly what was said in past threads I think that there is a general sense of happiness for a few reasons;
Magnum had been told before that he would have to wait before Telaprevir was approved, which could be about 2 years away...
While waiting due to advanced liver damage he had several impending HCV related health issues always hovering. Would some related health issue get to him before he could treat?
Could waiting the 2 years cause him to progress beyond the point of being able to treat? (maybe low platelets or decompensation?) One doctor told him it could happen.
An Co-writer told him that she very very rarely saw drugs approved for compassionate care. The odds of getting it approved seemed bleak. To be fair this whole process is not yet complete. We will still have to see it it happens and when, and if there are downsides to treating with compassionate care versus under FDA approval. At first glance however it might be that Magnum has about a 2 year head start. We will see what transpires....
I think you may be right that this may or may not be a case of david vrs Goliath. Maybe they are doing what they were always going to do.(if asked)
IF I recall correctly however Magnums first query ended with a dead end, however. It looks like a green light for now; at least proceed to the next step.
If nothing else this could be a useful path for other patients in certain circumstances. Illuminating the process could help a number of people.
Seems like there is much more to be happy about than the reverse......
just my 2 cents.
Willy
Magnum had been told before that he would have to wait before Telaprevir was approved, which could be about 2 years away...
While waiting due to advanced liver damage he had several impending HCV related health issues always hovering. Would some related health issue get to him before he could treat?
Could waiting the 2 years cause him to progress beyond the point of being able to treat? (maybe low platelets or decompensation?) One doctor told him it could happen.
An Co-writer told him that she very very rarely saw drugs approved for compassionate care. The odds of getting it approved seemed bleak. To be fair this whole process is not yet complete. We will still have to see it it happens and when, and if there are downsides to treating with compassionate care versus under FDA approval. At first glance however it might be that Magnum has about a 2 year head start. We will see what transpires....
I think you may be right that this may or may not be a case of david vrs Goliath. Maybe they are doing what they were always going to do.(if asked)
IF I recall correctly however Magnums first query ended with a dead end, however. It looks like a green light for now; at least proceed to the next step.
If nothing else this could be a useful path for other patients in certain circumstances. Illuminating the process could help a number of people.
Seems like there is much more to be happy about than the reverse......
just my 2 cents.
Willy
This news you are posting is making me feel good....to be honest...im feeling depressed about losing my job....even tho i got my financial help from my employer...thank God ...im still thnking of what i had and now its all gone...its tough...i was getting paid well and all...20 years gone down the drain...sorry for venting...but like i said...news like yours helps me cheer up
Whilst I really hope that within a short time Magnum will have telaprevir surging through his veins,I don't quite understand what all the triumphalism is about.
Unless I have got this wrong all I believe has happened is that the FDA have referred Magnum to a pre-existing rescource whereby a treating physician may,in concert with a drug company submit a pettition for a compassionate exemption.
I shall be happy and enlightened if I have misunderstood and some sort of amazing event has occurred ,but at present I am somewhat baffled.
Unless I have got this wrong all I believe has happened is that the FDA have referred Magnum to a pre-existing rescource whereby a treating physician may,in concert with a drug company submit a pettition for a compassionate exemption.
I shall be happy and enlightened if I have misunderstood and some sort of amazing event has occurred ,but at present I am somewhat baffled.
Never give up Magnum you are a champ.
A real inspiration.
I'm usually quite flip but I mean that literally.
Debby
A real inspiration.
I'm usually quite flip but I mean that literally.
Debby
Just got a call from my contact at the FDA. The doctor has to also fill out form 1572.
http://www.fda.gov/opacom/morechoices/fdaforms/cder.html
Also, doctor FIRST has to get permission from Vertex for the "compassionate use" of Telepravir. Your doctor should fax or phone them with "why" the patient needs this drug now, which means Cirrhotic and no other option. Keep in mind that you will waste your time and your doctor’s time if you have him just ask for the drug if you do not fit the protocol...
When the doctor gets approval from Vertex, he then contacts the FDA who will send him a packet to fill out. Again, he has to state the drug, the reason, patient's condition, blood work, etc...
After the FDA approves that, they will then contact Vertex for the release approval of the drug you can then pick up at your drug store, go home, do the drug, get cleared, and in a year, have a bottle of champagne to celebrate... so there it is...
Magnum
http://www.fda.gov/opacom/morechoices/fdaforms/cder.html
Also, doctor FIRST has to get permission from Vertex for the "compassionate use" of Telepravir. Your doctor should fax or phone them with "why" the patient needs this drug now, which means Cirrhotic and no other option. Keep in mind that you will waste your time and your doctor’s time if you have him just ask for the drug if you do not fit the protocol...
When the doctor gets approval from Vertex, he then contacts the FDA who will send him a packet to fill out. Again, he has to state the drug, the reason, patient's condition, blood work, etc...
After the FDA approves that, they will then contact Vertex for the release approval of the drug you can then pick up at your drug store, go home, do the drug, get cleared, and in a year, have a bottle of champagne to celebrate... so there it is...
Magnum
Just sent an email to my Vertex contact, of the proposed FDA form for the doctor to fill out. He and the Vertex Team will look over this and see what role Vertex will play and get back to me.
At first, he was a bit upset that I directly plead with the FDA for an early release, until I told him about the "compassionate use". Then his whole attitude changed.
He thought I was pleading for an early release of Telepravir, period.., as opposed to the “compassionate use”. After the explanation, he got it.
He laughed when I told him I made a pain in the a$$ of myself with the FDA, but concluded he will try to help all he can.
So, hang in there and I will keep pushing for the fastest track I can for the "compassionate use" release of the drug...
Magnum
At first, he was a bit upset that I directly plead with the FDA for an early release, until I told him about the "compassionate use". Then his whole attitude changed.
He thought I was pleading for an early release of Telepravir, period.., as opposed to the “compassionate use”. After the explanation, he got it.
He laughed when I told him I made a pain in the a$$ of myself with the FDA, but concluded he will try to help all he can.
So, hang in there and I will keep pushing for the fastest track I can for the "compassionate use" release of the drug...
Magnum
Hahaha... thanks...
Yes, this is the beginning of something that will benefit those in fear and in dire need. We ALL need to keep the pressure on...
Magnum
Yes, this is the beginning of something that will benefit those in fear and in dire need. We ALL need to keep the pressure on...
Magnum
There you go....very good news indeed...like i said,that was very smart of you to think of writing letters to the FDA....btw...i recieved the same letter in my email 2 days after i wrote them my letter....i did post it on your other thread....CONGRATULATIONS...i would go with the Boceprevir if you have a choice
Good for you!! Here is a tribute to you, look at my new avatar. His name is Magnum (LOL) and he is just wonderful!
Denise
Denise
The dot at the end was the problem. Sorry... let's try it again
Magnum
http://www.fda.gov/cder/cancer/singleIND.htm
Magnum
http://www.fda.gov/cder/cancer/singleIND.htm
Correction of web address for the FDA IND application...
http://www.fda.gov/cder/cancer/singleIND.htm.
Magnum
http://www.fda.gov/cder/cancer/singleIND.htm.
Magnum
Good work!!!
Let us know how things develop with your doc and Vertex.
I agree that the benefit vs. risk is a no brainer. The safety was shown in Phases I and II.
The studies are out there. From what we've seen, Telaprevir + SOC will give you/us the best odds of clearing the virus and then the best odds of SVR. Before it gets too late to treat due to the progression of your liver disease.
...There are always critics. It is a whole lot easier to sit around and gripe then actually do something. As Mr. Bob Dylan said in 1965...
“It’s Alright Ma, I’m only Bleeding”
...Cares not to come up any higher
But rather get you down in the hole
That he's in.
Thanks for being a leader on this issue, Magnum.
Hectorsf
Let us know how things develop with your doc and Vertex.
I agree that the benefit vs. risk is a no brainer. The safety was shown in Phases I and II.
The studies are out there. From what we've seen, Telaprevir + SOC will give you/us the best odds of clearing the virus and then the best odds of SVR. Before it gets too late to treat due to the progression of your liver disease.
...There are always critics. It is a whole lot easier to sit around and gripe then actually do something. As Mr. Bob Dylan said in 1965...
“It’s Alright Ma, I’m only Bleeding”
...Cares not to come up any higher
But rather get you down in the hole
That he's in.
Thanks for being a leader on this issue, Magnum.
Hectorsf
Congratulations! I'm so happy for you. You have been fighting for this so hard and it all seems to be falling into place nicely, due to your persistence!!!
Thanks for keeping us all informed and paving the path for others.
Hugs, Marcia
Thanks for keeping us all informed and paving the path for others.
Hugs, Marcia
Have an Answer?
You are reading content posted in the Hepatitis C Community
Learn about this treatable virus.
Getting tested for this viral infection.
3 key steps to getting on treatment.
4 steps to getting on therapy.
What you need to know about Hep C drugs.
How the drugs might affect you.
These tips may up your chances of a cure.
A list of national and international resources and hotlines to help connect you to needed health and medical services.
Herpes sores blister, then burst, scab and heal.
Herpes spreads by oral, vaginal and anal sex.
STIs are the most common cause of genital sores.
Condoms are the most effective way to prevent HIV and STDs.
PrEP is used by people with high risk to prevent HIV infection.
