What can we do about the delay of TDM-1 - Page 3

ElaineM · 09-07-2010, 10:24 AM

Thanks Brenda and Chrisy. I am going to start composing a letter this week. Are we also going to start a general petition for everyone to sign? I hope Chrisy or another representative can be our media contact. I applaud your efforts Brenda. I am looking forward to reading your interview in Cure. I signed up for a free subscription, so I get that regularly.
I agree Brenda. It seems silly for researchers and authorities to say targeted drugs are the future of cancer treatment and then try to stop promising targeted therapies from getting to the people who need them makes me wonder what are they really doing or saying? I thought the news about Avastin was bad enough. Slowing down T DM1 on top of that is unacceptable. I hate to make this a gender thing, but does the FDA have something against women with breast cancer? Maybe we should find some men who are Her2 positive to write some letters too. By the way, during my own personal research I found her 2 is not limited to breast cancer. I read It can affect other cancers too, but I have not researched that further, because I am trying to learn all I can about breast cancer and I have limited time.
By the way, if you go to the pipl link I included previously you will find Lilly Tartikoff. I tried it. It works. It should be easy enough to find Dr. Slamon's contact information. Do you think we should contact the editor of Cure Magazine? Do you think Cure could do an article about the FDA activities and/or TDM1?

Jackie07 · 09-07-2010, 10:32 AM

Be sure to send a letter/e-mail to the Congressman/Senator of our own district. Remember Joe had said it one time, each of us represent one vote and that's what politicians most concerned about.

ElaineM · 09-07-2010, 10:45 AM

I am trying to figure out who I can send a letter to in congress. The person who I could have sent it to just resigned from the U. S. congress to campaign for governor of Hawaii. He is a long time friend of the Obama family and would remember President Obama's mother's fight with cancer. The other congress people from Hawaii have other major concerns they are trying to get through congress. I might have to wait until after the primary elections in Hawaii and then write to the person who has a good chance of replacing the Obama friend in congress. The Obama friend might know whoI I should write to. I will think about it.

VirginiaGirl · 09-08-2010, 10:37 AM

As one of those people who were looking to T-DM1 as my next promising treatment option, I too am very disappointed, and scared, that it may well be another 2 years before it's approved. I was initially dx right before (but not in time to get) Herceptin was approved for early stagers.

I was hoping to find support for this issue from Breast Cancer Action, a bc advocacy group. Unfortunately, what I found on their website (bcaction.org) was a letter to the FDA OPPOSING fast-tracking T-DM1 (and their position against approval of Avastin for mbc). Their T-DM1 letter (copied from their website) is below. At least we can write to/email the same person and express a different viewpoint!

"
March 23, 2010
Richard Pazdur, M.D., F.A.C.P.
Director
Office of Oncology Drug Products
U.S. Food and Drug Administration
richard.pazdur@fda.hhs.gov

RE: Opposition to TDM-1 Fast Track Application

Dear Dr. Pazdur:

As you are no doubt aware, Genentech intends to seek fast track approval of TDM-1 based on the results of a single-armed, Phase II trial.

It is our understanding from the sponsor that the FDA was asked two years ago about what would be required for fast track approval of TDM-1, and that the agency took the position then that a randomized trial would be necessary.

Despite this clear direction from the agency, the company has completed -- and will seek FDA approval based on the results of – one single-armed trial, TDM4374g.

Breast Cancer Action believes, as the FDA does, that only sufficiently large, randomized trials will give us the information we need about drug efficacy and safety. We urge you, in the strongest possible terms, to require the sponsor to submit Phase III data before you consider approval of TDM-1 for breast cancer.

We understand that there is an important unmet medical need for people with advanced breast cancer whose treatment options have run out. We desperately hope that TDM-1 proves to be a beneficial option for these people. But we oppose sacrificing drug approval standards on the altar of hasty access.

As you know, the FDA used to require the results from two Phase III trials before considering approval of a drug for the market. More recently, a single randomized trial has been found by the agency to be sufficient in some cases. We think this weakens standards for drug approval. Please do not weaken them further by allowing marketing based on a small, single-armed Phase II trial.

We are in conversation with the sponsor about an expanded access program for TDM-1. While we applaud the company’s desire to make sure that those who might benefit have access to the drug, we are very concerned that the company intends to use what they are calling the “Expanded Access Study” as a substitute for a Phase III trial.

The Expanded Access program will not provide Phase III data. The FDA must insist on sufficient randomized results before approving TDM-1 for the market,

Sincerely,

Barbara A. Brenner
Executive Director

Note: As a matter of policy, in order to avoid the fact or appearance of a conflict of interest, Breast Cancer Action does not accept funding from Genentech or from any other pharmaceutical or biotech company."

schoonder · 09-08-2010, 01:28 PM

The FDA was fully justified in their ruling to issue a Refuse to File (RTF) letter re T-DM1. When questioned they surely will be able to identify directive(s) that support that decision. But therein lies the problem, what happens if particular directive(s) have flaws, are outdated, lack specificity, fail to take into account technological advances, or even more important, don't reflect the spirit of FDA's mandate?
Here we have an instance where FDA ruled that trial was incomplete because candidates that partook in this evaluation were not subjected to all possible metastatic breast cancer (MBC) remedies approved for this disease. Let's not deny that outcome of this scrutiny by FDA of patient population is factual; indeed not all available MBC treatment options were accounted for. However,let's look at the other side of the coin, are there mitigating reasons why Genentech decided not to include this missing collection of infrequently prescribed, low in its effectiveness, yet highly toxic medication as part of their trial? The answer of course is a resounding YES:
This trial was all about finding new options for “HER2- positive” women whose breast cancer progressed while on Herceptin or Tykerb, in third-line or greater setting, for which today there are no standard treatments or guidelines. FDA was fully aware of trial’s intent to address an unmet medical need strictly for “HER2-positive” patients, a small subset of overall MBC population. This study included patients that had failed on average seven (range of 3-13) regimens including both Herceptin and Tykerb which are known to work best in HER2-positive breast cancer. Not all regimens were accounted for, but the most predominant, most meaningful, most effective and all these therapies were no longer therapeutic.
One can only surmise that Genentech ruled in favor of the most immediate and timely considerations to bring what they observed to be an astonishingly effective, targeted drug to Her2-positive patients who were so desperately in need of new, viable treatment options. This urgency to save lives, led them to stop their time consuming, probably inconsequential and wasteful search to find further candidates that had been medicated with these for Her2-positive MBC rarely prescribed, ineffective therapies. As the Biologics License Application (BLA) Priority Review filing based on just phase II results strongly suggests, time considerations to market what promises to be a very effective drug was a strong driving force behind Genentech's motivation.
FDA’s uncompromising position to refuse to accept this filing based on a minor technicality, most likely brought on by shortcomings in their directive(s) dealing with newly “targeted” drugs like T-DM1, that are designed specifically for a small, Her2-positive overexpressing MBC patient population, is not only incomprehensible, but it is totally unacceptable. The consequence of this ruling, which appears to delay T-DM1’s approval process by at least two years, were instantaneous, the hope for a possible effective treatment option went poof, it disappeared into thin air and ultimately, it will result in premature demise for many an ill Her2-positive MBC patient.
Yes, this was such harsh and inappropriate ruling by FDA.

chrisy · 09-08-2010, 02:18 PM

Very well articulated. One factual error I think. Recheck the number of failed therapies. I think it was AVERAGE of seven with range of 3-13!

Add a call to action to reconsider the needs of the patients who tdm1 was made for and sent THAT to the FDA (All parties onn my earlier list) and their congressional counterparts

ElaineM · 09-08-2010, 03:34 PM

I called Genetech to try to get information today. The person I talked to told me they have not canceled the extended access program for T DM1 yet. They still have 9 sites open and are still planning to set up more. He also told me the phase 3 Emila study is still in operation. Other her2 studies involving Herceptin and/orTDM 1 such as the TDM4450g, MARIANNE, and the CLEOPATRA are still active.
I asked for more information and was directed to someone else. That person directed me to the Genetech website (www.gene.com) and suggested I look in the media section regularly for updates. I followed her directions.
The latest press release can be found at
http://www.gene.com/gene/news/press-...etail&id=12947
I think it is a good idea to check for press releases regularly.
http://www.gene.com/gene/news/index....btn=Web+Search

Patb · 09-08-2010, 04:05 PM

Thank all of you for your work on this matter. I know
little about it but am willing to help send e-mails, etc.
because my dear friend stage IV was declared all
tumors gone today at the Indiannapolis trials. She
is elated. They will contine the treatment for awhile.
Her lungs and nodes were clear.
patb

schoonder · 09-09-2010, 09:56 AM

Thanks Chrisy for pointing out that it was an AVERAGE of 7 prior treatments ranging from 3-13, subject post has been corrected.
Note of interest, compilation of T-DM1 data Genentech is looking at continuous to be promising and must have convinced them to broaden their attack on Her2-positive cancer. A new multinational trial in both adjuvant and neo-adjuvant settings is about to get underway, more details can be found in the clinicaltrials.gov registry.

Chelee · 09-13-2010, 02:14 AM

I posted to this thread & lost it. Ugh! Too late to start over now..have an early morning appt with onc/Herceptin.
But I want to keep this thread up at the top...it's such an important issue we have to stay on top of. I'm so impressed with all of you. Lots of great idea's you have all come up with.
I plan on writing my letter this wk...then figure out which places to send it too. Like Elaine asked...are we going to write up a petition for all to sign on top of these other ideas?

We need to be heard...and with what I've read here so far I think we can make it happen.

Chelee

ElaineM · 09-13-2010, 09:40 AM

Can something be done on You Tube? Any volunteers?
I can put a link to any petition we may want to start on my website.

Emelie B · 09-13-2010, 09:55 AM

Did we get a petition drafted yet? Elaine-that is a great idea. How can we get this done?
Let me know what I can do and I will be glad to help.

Chelee · 09-17-2010, 03:22 PM

Elaine...I never thought of You Tube...another good avenue to use. I really don't want any of us here to drop the ball on this one. We need to strike asap. My onc had not even heard about this, but did say we should be pro-active and jump on it. I told her that's what were doing. We want to get our voices heard.

So as one big part of this are we going to draft a petition and have everyone sign it? I know we could get alot of signatures for this with no problems. So many good idea's here...so what should we all be doing now that will have the most affect?

There are so many of us that don't have that option of waiting till 2012 or worse yet 2013. Any other great idea's pls throw them out here. If Joe was here he would be all over this...so lets make him proud of us. That's a big part of why he and Christine built this board. So lets get it done...I know we can. This is too important not to give it our best shot.

Chelee

ElaineM · 09-17-2010, 09:33 PM

I agree about the petition. I think Chrisy was going to ask Christine about it. There is a site where anyone can start a petition. I think I put a link to it somewhere in this thread. I will try to look for it over ther weekend.
Is anyone up to doing a You Tube project?

ElaineM · 09-18-2010, 02:18 PM

As promised here is the link to the petition site for anyone who wants to start a petition about the recent FDA decision about the T DM1. I am not the one to start a petition, because I prefer not to disclose my health problems to some family members, friends, co workers and associates. I have only shared my health problems with a small group of people who will keep it confidential. This has worked well for me for almost 12 years.
http://www.thepetitionsite.com/create.html
You Tube, interviews, news articles, or media appearances are better left to those who don't mind going public about their health problems. I prefer behind the scenes kind of activities like research etc.
I still want everyone who is eligible for T DM1 to receive it in a timely manner. It is one of my greatest wishes, because it is such a promising drug.

schoonder · 09-19-2010, 09:59 AM

Quote from interesting article
"Critics say that science behind some medications has eclipsed old rules — and ethics — of testing them"

http://www.msnbc.msn.com/id/39253556...new_york_times

Trish · 09-21-2010, 07:18 PM

Thank you for all the work you are doing to get the TDM-1 decision reversed. My liver mets progressed on both Herceptin and Tykerb after initial positive responses (despite liver mets growing and sky high tumour markers I feel ridiculously healthy) so was really disappointed about the recent FDA decision. I live in Australia and Genentech won't apply to our Pharmaceutical Benefits Scheme until they get FDA approval. Hence my gratitude for the work you are doing. I am so frustrated by the one size fits all mentality. I hope Personalised Medicine is gaining ground (I see there is a big conference at Harvard Medical School on November) and just hope it can have some influence on bodies such as FDA and PBS here in Aust.
I will keep racking my brains about anything I can do from here.
I find it so sad to see the excited post from Joe about the expanded access for TDM-1. He tried so hard for us and I am ever grateful.
Trish
Trish

Chelee · 09-22-2010, 04:02 AM

Trish,
I so agree with your comments about Joe and his post. I can't help but think of him every single day! If he was here he would be ALL OVER this T-DM1 battle for us...he had the connections and would of known exactly who to talk too.. and what we all should be doing. So many of us were depending on TDM1. I just get angry when I think about how we are denied access to TDM1. It makes no sense at all to have us run through toxic chemo's before we can have a targeted therapy. Are we still going to do something about this TDM1 issue? I know it's so hard when many of us are seeing doctors, doing chemo and trying to keep up with all life hands us...but I hope some how we can make a difference for the sake of so many that need this drug now...or will in the very near future.

Schoonder,
Great link...it says it all. Thanks for sharing that.

Chelee

schoonder · 09-22-2010, 06:25 AM

To know Super-Herceptin, a very effective Her2+ MBC drug exists, but won't be made available for approx. 3 years because of some technicality that probably has no real bearing on drug's therapeutic activity, that ranks right up there with cruel and unusual punishment (Eighth Amendment to the United States Constitution) for the effected patient population.
http://thehill.com/blogs/congress-blog/healthcare/119305-a-plea-to-the-fda
http://abcnews.go.com/Site/page?id=3271346&cat=World News with Diane Sawyer
http://www.cbsnews.com/stories/1998/08/01/eveningnews/main15218.shtml
http://www.msnbc.msn.com/id/32359544/

Sheila · 09-23-2010, 05:22 AM

As far as a "campaign" slogan for us to base our voices on...I know when I tried to get TYKERB, and kept getting denied, I finally wrote my own appeal, and titled it
I AM DYING TO TRY THIS DRUG

It got their attention, unfortunately, by the time I got it, things had progressed and it didnt offer the punch we had hoped for.

They now have the TDM-1 where I go for early access....I was one of the first to be able to get it...waiting in the wings with the Metronomic chemo.

I went Tuesday for treatment, and here is what happened:
They have it where I go for treatment now in Chicago. I have been waiting, and since i now have progression in my neck nodes yet again, causing left facial numbness, thought my prayers had been answered. But, I was sent for a MUGA, and it shows 39%...spent all day yesterday at the cardiologist, echo cardiagram...will find out today...as of right now i have been taken off everything, Herceptin, chemo, etc.....maybe some fairy dust and hocus pocus will be my new treatment. Waiting to hear from the Dr today. Sometimes it seems like I just cant catch a break! I was diagnosed too early for Herceptin adjuvant, and now after 7 years of Herceptin and various chemos, the muga is too low for TDM-1...

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