What can we do about the delay of TDM-1

[Barbara H.]

Hi Everyone,
I spoke to my oncologist and obviously he is not in a position to get this decision reversed. He is quite upset with the decision because metastatic HER2 patients who have been waiting for the drug are going to die. In fact, there were a number of patients who were scheduled to receive it next week through the expanded access program that will be denied that opportunity.

The reason for the denial is ludicrous. Why would a HER2 cancer patient choose to take awful chemo treatments when more effective target therapies are available? Although the members of the FDA have medical backgrounds, they are not necessarily oncologists, and presumably do not understand the ramifications of the decision they made considering the track record of TDM-1. Obviously, they do not realize the number of patients who will die as a result of their decision.

As many of you know, I was on a phase 1 trial of TDM-1 for two years and it was a miracle drug for me. This drug did not stop working, but I developed lung inflammation. That does still not mean I couldn't try it again in the future.

I am not writing on behalf of myself. It pains me to know that many of you are being denied access to this treatment.

Apparently this decision can be reversed if many of us write and voice our opinions and experiences. The FDA needs to understand why this drug needs to be approved ASAP. They need to know about the number of patients who will lose their lives if they do not reverse this decision. They need to understand their responsibility for these patients.

Unfortunately, I do not have experience being an advocate and do not even know how to get this message out so that the essential people read it. For example, President Obama's 60, 000 letters he receives each day are screened by readers that decide which ten letters he will read on that day. I am also not sure to whom we should send our letters. My oncologist will hopefully let me know if the advocates at the Dana Farber have any ideas.

Please post any suggestions or ideas you may have about this critical situation. Remember, we may be able to make an impact if many of us write.

Thanks for reading and thinking about this issue.

Kind regards,
Barbara Holz

[Jackie07]

Well, Health Care Reform has been a hot topic. I'm sure our e-mails to the White House will get some kind of attention. President Obama's Mother died of a gynecological cancer and their experience with her insurance company was a traumatic one.

Let's get on with it!

www.WhiteHouse.gov (http://www.WhiteHouse.gov)

[Nancy L]

I do not believe Obama cares in the least about this decision. This is the start of what Obamacare is all about.

I get disgusted every time I hear him say how hard it was for him to watch his mother argue with insurance companies. I say, Where was he? He was a grown man with a law degree and the one who should have been arguing and then maybe he would understand our plight. I took care of these issues for my mother and my sister when they were ill. This is what family does when they truly care. I think he is a phony.

[ElaineM]

Maybe finding out who the "movers and shakers" at the FDA are and writing to them is a good idea. I think every president has a staff that decides which pieces of mail he sees and what he doesn't.

[Chelee]

Barbara,
I would love to do something about this T-DM1 nightmare. Sign me up...lets do whatever it takes to be heard. Waiting till 2012 is unacceptable! There are so many women that were depending on this drug...myself included. We need to find a way to get the ball rolling--so lets all put our heads together and be as pro-active as possible.

Nancy...AMEN to everything you said...I couldn't of said it better myself! I'm not sure who we need to direct our letters too...but I do not believe for one minute it's the White House. Now Elaine might be on to something? The FDA might be one good place to start. Lets figure this out and get going on it...T-DM1 has already proven to be a life saver so we need to make sure it's available to all that need it asap. Any other ideas on who we write too please jump in here. (Thanks Barbara for bringing this issue back up to the top.)

Chelee

[Pam P]

In honor of Joe's memory and work, please let's not let this discussion get into political views for or against Obama etc. Let's just focus on the best ways to help accomplish our goal of getting tdm1 to people who need it. I'm going to send an email to the white house link right now and I'll also send letters to others if any of you come up with names/ addresses within the FDA or other influentials. I don't have the advocacy or research expertise but I can write letters.

[schoonder]

If Roche files for T-DM1 approval by mid-2012 using Emilia's trial results, then earliest approval for this drug would be onset of 2013.

How about Congressional FDA Oversight Committee for those letters?

FDA refused to look at "any" of T-DM1's trial results because Roche failed to include "all possible" mbc therapies in this trial.
Since T-DM1 a "targeted" therapy has shown to be an effective treatment against HER2+ cancers, extending this trial, looking for candidates that failed those other "much less effective", thus "much less prescribed" treatment options, would've been a "waste of time".
However the FDA, overly risk averse, opted to use this omission, one that really had no bearing on trial outcome of HER2+ candidates, to issue a Refusal To File (RTF) letter. This RTF will delay approval of this very promising drug by at least two years.

[Barbara H.]

Thank you to all who have responded. I agree with Pam that in Joe's honor as well as considering the purpose of this website that we should not discuss politics. My purpose for starting this thread is to try to get this decision reversed. I will also write a letter, but I was hoping for ideas about how we can get our letters noticed. I also hope to be considerate about not putting down the FDA. However, I just do not understand how they could have rejected T-DM1.
Hopefully, we can change this decision.
Barbara H.

[Barbara H.]

Schoonder's reply is absolutely correct. FDA's refusal was based on the technicality that she describes. Their decision is shortening lives and the quality of the lives of HER2 patients that need it.
Barbara H.

[Sheila]

I agree, we need to do all we can...too many of us cannot wait until 2012 or 2013 to get this drug. I was ahead of my time for the Herceptin in the adjuvant setting, it was only for stage IV when I was diagnosed. Now, I am stage IV, running out of options, and still unable to get the TDM-1... Does the FDA really think it matters when we have nothing else to try?
If they were in our shoes.............

[hutchibk]

The hopefully 'fatal flaw' in their written decision was the sentence "regardless of HER2 status" ~ you can't approach efficacy of potential new treatments for HER2 cancer from a "regardless of HER2 status" standpoint... IT IS ALL ABOUT THE HER2 STATUS. That is why they are developing these targeted treatments, because if the other available treatments that "have not been exhausted by the study patient population, regardless of HER2 status" worked for us, then there would be no need for intense research and focus on treating this subgroup. It's sounding like a "one size fits all" mindset that is so scary in the bigger picture.

Chrisy knows the woman at Genentech that Joe was very close to... perhaps she can connect us with lobbying folks who know the inner workings of this insane bureaucracy and we can offer our powerful, survivor/patient voice to a coordinated push for the FDA to reconsider their flawed thinking.

As well, if anyone has meaningful connections to Komen, they are the most powerful lobbying group for breast cancer and they are currently applying a full court press in coordination with Genentech to the FDA about Avastin... I am guessing if we hit them with our outrage about this, they would be very interested in helping, if they aren't already in the early stages of it.

Anyone here close to Komen? Or does anyone have an in to Nancy Brinker?

[ElaineM]

Yes. We need to do something that will help all her2 positive people and honor Joe, but still not be too political.
Sorry I mentioned the White House, but I have personal experience with important requests not getting through to the president in a timely manner. I don't think it is President Obama's fault. He is a very busy person and can't take care of every e-mail request personally. He probably gets hundreds every day. His staff may be in a position to decide what he sees and what he doesn't.
I will go to the FDA website to see if I can find out where we can send something to.

[Debbie L.]

Good luck at the FDA site, Elaine. I just spent a LONG time there and couldn't find anything. They started this big "transparency" initiative and if there's anything there about TDM-1 or about inviting public input, it's opaque, not transparent. Jeeze.

They do have a blog about their transparency initiative, where comments can be posted. I wonder if that might be a way to get their attention. Although the ruling on TDM-1, as I understand it, is a done deal - it's not like Avastin where they still have the final meeting coming up.

So here's the blog page if anyone wants to try there:
http://fdatransparencyblog.fda.gov/

The other thing I noticed, while googling all over the place for news of some organized reaction to the TDM-1 accelerated approval denial, was that posts here on the HER2 list show up in a google search. Isn't there a way to make forums private? I'm on several others that don't show up in google.

Debbie Laxague

[hutchibk]

Elizabeth Thompson, Senior Vice President of Medical and Scientific Affairs for Susan G. Komen for the Cure.

5005 LBJ Freeway, Suite 250
Dallas, TX 75244
1-877 GO KOMEN

She is the one quoted about Komen's reaction to the FDA/Avastin situation.

Perhaps we can write her a letter outlining our concerns and see what their position is... see if there is any help they can offer.

I don't have time right now to start a letter, but I can certainly contribute to it.

[Rich66]

Brenda,
I like your ideas on identifying larger entities/avenues to force the message through.
Another powerful avenue might be news organizations. We saw how media picked up on Herceptin years ago. Regardless of political spectrum, health care issues are hot now in terms of getting viewership or readers. Getting a few TDM1 success story patients in front of cameras..maybe picketing in front of FDA headquarters with a media notification could be powerful. I see some of that with Avastin now.

Regarding google searching, I think having forum topics searchable enhances information access. If concerned over privacy, maybe it means being more discreet with names and e-mails in profiles.

[ElaineM]

Good idea Brenda. I just did a search for "writing to the FDA". Here is what I found so far. I am still checking and might find more later.
http://www.fda.gov/AboutFDA/ContactFDA/default.htm
Please see the left side bar, right side bar, middle employee directory, FDA centers and offices and other information you might be interested in searching.
Even if the TDM1 decision is a done deal we may wish to comment on it as consumers of health care. It might be good for the FDA to hear from consumers once in awhile since FDA decisions may affect our health care. I do not think this is political. The FDA is an agency that is supposed to advocate for us as consumers by checking the safety of the products we use and the medicines we take.

[ElaineM]

I like your contacting the media idea Rich. We have to pick a good one if we want to do that though. Some of us may not want to be in the spotlight. I am one of those. I am better at behind the scenes work. What about writing to some oncology organizations? Is that a good idea? Going back and reviewing how Herceptin and Tykerb got approved might help us get some ideas too.

[Emelie B]

I like all the ideas. I think we should at least start a petition type thread and we can all sign it and possibly print it and send to whomever we think would help assist us. I would be glad to do whatever I can.
I also think we Rich's idea of media is excellent. With all the c--- I see on the morning shows, our issue would surely enlighten someone who needs this drug too.
My daughter lives in D.C. and I can always go visit her and try to do something there.
Let's get this thing rolling!!!!

[ElaineM]

All the ideas are good. See what we can do when we put our heads together !!
One more idea. We could team up with another organization like Susan Komen or others and do something.
By the way, "Stand Up To Cancer" will be airing on most of the T. V. channels on September 10th. That is a program about raising money and awareness for cancer research. I think they have a website. Should we look around there for ideas?

[schoonder]

What might have lead Genentech astray.

Was omission of certain non-HER2+ therapies in their T-DM1 trial design a glaring oversight by Genentech that compelled FDA’s RTF response, or was this a misunderstanding by Genentech attributable to lack of specificity by FDA during preceding trial discussions?

Did FDA’s acceptance of trial requirements that did end up receiving Accelerated Drug Approval mislead Genentech in T-DM1's trial construct?
Case in point a recent accelerated approval ruling re: Tykerb where, according to FDA Tykerb WAS NOT compared to Trastuzumab-containing chemo regimen for treatment of MBC. This compare could be done later, why not allow Genentech to correct their shortcoming in the "compassionate" trial setting?
FDA approves TykerbR for use with letrozole to treat certain postmenopausal women with hormone receptor positive metastatic breast cancer


On January 29, 2010, the U.S. Food and Drug Administration granted accelerated approval to lapatinib tablets (Tykerb®, GlaxoSmithKline) for use in combination with letrozole (Femara®, Novartis Pharmaceuticals Corp.) for the treatment of postmenopausal women with hormone receptor positive metastatic breast cancer that overexpresses the HER2 receptor and for whom hormonal therapy is indicated. Lapatinib, in combination with an aromatase inhibitor, has not been compared to a trastuzumab-containing chemotherapy regimen for the treatment of metastatic breast cancer. The approval was based on a clinically meaningful increase in progression-free survival (PFS) observed in a single trial (EGF30008). As a condition of accelerated approval, subsequent randomized trials are required to verify and describe the clinical benefit of lapatinib in patients with metastatic breast cancer who have received prior treatment with trastuzumab.

EGF30008 was multinational, randomized, placebo-controlled trial of lapatinib plus letrozole versus placebo plus letrozole in patients with hormone receptor-positive metastatic breast cancer who had not received prior therapy for metastatic disease. Patients were randomly assigned to receive lapatinib (1,500 mg once daily) plus letrozole (2.5 mg once daily) or to placebo plus letrozole (2.5 mg once daily). There were 219 patients (17%) who were HER2-positive, 952 (74%) patients who were HER2-negative, and 115 (9%) patients did not have their HER2-receptor status confirmed.

Accelerated approval was based on the results from the group of postmenopausal women with metastatic breast cancer that overexpressed the HER2 receptor. The primary efficacy endpoint was PFS, defined as the time interval between randomization date and the date of either first documented disease progression or death due to any cause. The lapatinib plus letrozole combination had a median PFS of 35.4 weeks compared to 13.0 weeks for the placebo plus letrozole arm (HR = 0.71, p = 0.019). The overall survival data are not mature at this time.

Safety data was evaluated in 1278 postmenopausal women with hormone receptor-positive metastatic breast cancer. The safety profile of lapatinib in this trial population was consistent with previous safety data. The most common adverse reactions (=10%) in the lapatinib plus letrozole arm were diarrhea, rash, nausea, and fatigue. Elevated liver enzymes and bilirubin were reported in 53% and 22% of the patients receiving lapatinib, respectively. Among 654 patients who received the lapatinib plus letrozole treatment, 26 patients experienced Grade 1 or 2, and 6 patients experienced Grade 3 or 4 decreases in left ventricular ejection fraction.

Full prescribing information, including clinical trial information, safety, dosing, drug-drug interactions and contraindications is available on the FDA website (http://www.accessdata.fda.gov/drugsatfda_docs/label/2010/022059s007lbl.pdf).

http://www.asco.org/ASCOv2/Practice+&+Guidelines/Practice+Management+&+Reimbursement/FDA+Drug+Alerts/FDA+approves+TykerbR+for+use+with+letrozole+to+tre at+certain+postmenopausal+women+with+hormone+recep tor+positive+metastatic+breast+cancer (http://www.asco.org/ASCOv2/Practice+&+Guidelines/Practice+Management+&+Reimbursement/FDA+Drug+Alerts/FDA+approves+TykerbR+for+use+with+letrozole+to+tre at+certain+postmenopausal+women+with+hormone+recep tor+positive+metastatic+breast+cancer)
Roche surely isn't shirking their responsibility in completing evaluating T-DM1, presently company is conducting two large, international MBC phase III trials. The issue is that, what appears to be a minor technicality, an inconsistent ruling by FDA, is keeping this drug for at least two years out of hands of critically sick people, of which a good many no longer have viable options for efficacious treatment on hand.

[hutchibk]

Two voices we need to have as support of this are Dennis Slamon and a spokesperson from Genentech. We need to be sure we are approaching the media, the FDA, lobbiest, etc with a very concise and scientifically factual message.

[ElaineM]

I agree Brenda.

[Darlene Denise]

Anyone have an in with Lilly Tartikoff? She is friends with Dr Slamon, helped him raise money with Revlon for Herceptin and has partnered with Catie Coruic (there is our news connection) on cancer issues. She played a large role in bringing Herceptin to us with all of the money and drug company issues. I understand she is still very involved in cancer projects. I think she could be very powerful if we could get to her. What about some of our ladies who see Dr Slamon. Perhaps he could get us on track with something?

[ElaineM]

Here is a number to see if the T DM1 expanded access
program has been affected by the FDA's decision.
1-888-662-6728
This is the TDM1 Expanded Access Trials Support number.
We might also like to check genentechclinicaltrials@druginfo.com
or the genentech website for updated information.
clinicaltrials.gov
Trial number NCT00558 103 Glaxco Smithkline.
Good point Darlene. Some of our ladies who see Dr. Salmon might want to ask him about it and if Lily can give us some advice that would be good too.

[hutchibk]

I spoke to the Genentech clinical trial support line on Thur and Fri (at 4 pm cst on Friday) and they say they have heard no word at all that the EAP will be curtailed or discontinued, and they are still pre-screening folks for the current locations. One other thing I seem to remember that I learned is that they are only planning on accruing 200 total across the country for the EAP. I can't for the life of me remember who told me that, but it was on Thur (since I talked to Genentech support and to an EAP location coordinator... can't remember who said it). The last person I spoke to at Genentech support told me she had 8 new locations that were pending for EAP, awaiting full approval...

I wouldn't be at all surprised to hear word soon that the EAP might be suspended. I certainly wouldn't blame them if they decide to suspend for now and re-open it around the time in 2012 that they plan to submit the BLA again... I believe it is designed as a 'bridge' for mets patients to help carry them over to full market approval, and now that that has been set back, it would be an expensive proposition to continue accruing the program for 2-3 years as opposed to 6-8 months that I think they believed would be their accelerated timeline to market.

Just one girls opinion.

[Darlene Denise]

Lilly Tartikoff lives in Beverly Hills. Could our Dear Flori get us any scoop about how to reach her?

[Barbara H.]

If we can be effective, The FDA does procedures to reverse their decision.
Barbara H.

[Rich66]

As far as success stories, I notice Chrisy had liver mets brought to NED via TDM1. Whatcha say Chrisy..ready for your closeup?

[evlin75]

Hi folks, my daughter was also watching the trials of TDM1 as she is her2+ and does not want to do all the hard chemos before getting a medication targeted to her diagnoses.
I would try GMA as they seem to be more open to publicising problems cancer patients have with health coverage. She is on Xeloda and Tykerb.....and regular Herceptin but her markers are starting to move up. She does not want to take Taxol and is considering a clinical trial that has TDM1 with GDC 0941.
Good Morning America aired her story when she was having trouble with her company giving her disability and I feel they would be sympathetic to this difficult situation, explore it and get some attention to the problem.
My daughter volunteers with Komen and yes they are trying to help.
And yes, I was sorry to hear about Joe.
Courage!
Ev

[Rich66]

Hmmm...those seem like ready made connections. Could get Chrisy as success story and Ev's daughter and others for the wannabees. Slamon and or other docs to anchor the medical side. Wasn't Dr. Sledge going to participate on this site? Maybe he would be another medical voice to pursue.

[schoonder]

T-DM1 was developed during the days that Susan Desmond-Hellman presided over product development at Genentech, presently she's chancellor at UCSF. Her support would strengthen this case from the technical perspective. Chrisy could possibly get to see her, win her over on this subject matter when at UCSF.

[ElaineM]

If you want to contact Susan Desmond-Hellman or
Lily Tartikoff this might help.
http://www.pipl.com. I find this search engine helpful when I want to find people.
If you want to work with ABC's Good Morning America you might want to contact Anchor Robin Roberts. She is a breast cancer survivor,so she might be willing to do a segment on TDM1.

[hutchibk]

Sounds like Chrisy is our linch-pin to a few good ideas...

And just a recap of some of the ideas:

"get ducks in a row"
1. reach out to Genentech
2. reach out to Dr. Slamon (Dr. Sledge or Dr. Julie Gralow in Seattle at SCCA, she is running a TDM1 study and could advise us.)
3. reach out to Susan Desmond-Hellman
4. reach out to Komen, (Dr. Winer is on Board of Science Advisors)


"media outreach"
1. reach out to GMA/national TV talk shows
2. reach out to local TV in cities where survivors live
3. reach out to cable news channels
4. reach out to women's magazines

"Lobbying/advocacy"
1. reach out to KOMEN for lobbying and support to cut through bureaucratic walls.
2. reach out to our local Senators and Congressfolks.
3. reach out to Lily Tartikoff.


(I am scheduled to be the subject of a monthly spotlight on breast cancer survivors on our local ABC affiliate in either Oct or Nov, will highlight the TDM-1 problem, as well as a follow-up article on me in CURE Magazine before SABCS, in which Kathy LaTour also knows I will highlight the TDM-1 refusal...)

[ElaineM]

Thanks for helping us stay organized Brenda. Thanks for highlighting TDM1 in your upcoming interviews.
Keeping T DM1 in the spotlight will help. If each one of us "talks it up" to our medically related contacts that will help too. Let's keep T DM1 in the spotlight whatever way we can.
"The squeaky wheel gets the oil."
Smile.

[chrisy]

Brenda - well organized indeed. I've spent all morning researching the discussion on other websites and sending emails.

Your earlier post on this thread, about needing to have clear, concise and scientifically factual info is critical. Talking points if you will.

I also found some good discussions on other boards so far, including this one:

http://www.inspire.com/groups/advanced-breast-cancer/discussion/tdm-1-recent-setback/

[Midwest Alice]

10/16/10 Metastatic Breast Cancer Network conference in Indiana
I am 3 hours away and will be going.

We should be able to launch a media campain from this conference.

I can write letters to the editor about the people I meet there and the effect the FDA rulling will have on their life. I will try the Wallstreet journal and the Indy Star.

If we could do video interviews of women who would have started theatment we could put it on youtub. I don't know how to do that. I can run a vido camera and maybe interview willing women at the conference.

My daughter is a marketing person. Maybe I can get her to go with

The November elections are not far off. Go to rallys and debates and hang around till the end and meet the people who our running for office. They need to see are faces and feel our hand in theirs. We need them to remember us and our story.

[chrisy]

All of these ideas are great. With Pinktober just around the corner, there should be opportunities to generate conversation. However, that is a month away... When placed in context with the FDA's pending decision on Avastin, it's clear to me that the voices of both metastatic bc patients and Her2+ patients are not being heard.

I suggest, based on some conversations I've had in the past day or so, that if we want to have impact we need to act now, while the issue is "fresh". And continue to keep the volume up after that.

The most immediate impactful action we can take is to individually, as a group, and in concert with others (the outreach to Komen and other groups with interests in common) is to write to the FDA, copy our representatives in Congress - as well as those with aspirations to become our congresspeople and senators.

Who remembers the end of Miracle on 34th street??? Our response needs & deserves the "put it here on my desk" treatment. Since we probably won't be invited, we'll have to put it there ourselves - via email, snail mail, fax. But just as many bags. Again, they need to hear our voice.

I agree with I think Brenda's comment; we must be succinct and factually accurate; but the issue is not only a scientific one, it is a perspective.

Let's hear it - what are the salient points we would need to include in such a letter? If we can design a basic format then we can pass it on and people can send, amend, personalize or whatever...

Here are a few points (NOT succinct, yet!) we might be able to use:

We are Stage IV breast cancer patients. We have benefited from TDM1 or need access to TDM1 because we are running out of options or want options that offer good QOL. Our need is now...not 3 years from now.

We are Her2+ breast cancer patients. Science is moving very fast towards targeted and personalized treatment. Her2 overexpression in BC became a prime therapeutic target, with game changing results.

We are asking the FDA to consider the unique needs of the metastatic breast cancer community, and the Her2 cancer community. (how many people IS that, anyway?)

For people living with metastatic disease, it is important to have options. (what does that mean for you?)

Options that enable us to live more fully and with greater quality of life are a big deal. Phase II studies indicate TDM1 has a low incidence of significant side effects, especially compared to traditional chemo. Although I'm just a single datapoint, I personally have enjoyed great QOL on TDM1

Time is also a big deal - what Stage IV patients need MOST is faster access to therapies that can extend life and improve quality of life. True, we don't want to sacrifice good science on the alter of expedience - but more importantly, we mustn't sacrifice LIVES on the alter of caution. Stage IV patients simply cannot wait.

Emerging science is our best hope and tailoring therapy to the individual's disease is key. Requiring patients to run the gauntlet of "one size fits all" therapies as a precondition to try the "custom made therapy" seems completely the wrong direction.

The approval process needs to keep up with the science.



What have I missed? (I deliberately left out any personal opinions and tried to be rational - after all if I'm trying to influence someone at the FDA I'd rather have the letter actually have a chance of being read!)

If you've already written, or are writing a letter could you share it here?

[hutchibk]

The sentence "regardless of HER2 status" disturbed me the most in the decision... ~ you can't approach efficacy of potential new treatments for HER2 cancer from a "regardless of HER2 status" standpoint... IT IS ALL ABOUT THE HER2 STATUS. That is why they are developing these targeted treatments, because if the other available treatments that "have not been exhausted by the study patient population, regardless of HER2 status" worked for us, then there would be no need for intense research and focus on treating this subgroup. By the time we exhaust those other treatments, it is often the end of our road... with progression to the point that it's potentially too late to try a 'new targeted treatment'....

It's sounding like a reversion back to that "one size fits all" mindset that is so scary in the bigger picture.

[schoonder]

FDA will probably say that T-DM1 from what they have heard so far is a very effective, relatively easy to tolerate drug for Her2+ mbc patients. They will also say how disappointed they were that due to incomplete patient population in trial, they had to issue that RTF letter and that they are now anxiously looking forward to day that this issue is resolved so they can begin to evaluate the data.

I agree with hutchibk, focus should be on "judgment error" by FDA to take into account, or assign sufficient value to fact that Genentech moved this drug into clinic to provide new options, where few or none remained, for "strictly" Her2+ mbc patient population.
Underlying reason for RTF issuance "regardless of HER2 status" is totally misguided, yes, IT IS ALL ABOUT THE HER2 STATUS.
This trial by design was not for every patient suffering from metastatic breast cancer, this was a "targeted" approach, new medication for a small subset of patients. Targeted therapy, a somewhat new and upcoming process, shouldn't require that FDA look at how "all" approved mbc medicines compare to candidate drug. Participants in this trial had failed pretty much all treatment options that provided them with "any" chance of relief.
In a follow up study Genentech showed that T-DM1 was most efficacious on those candidates with strongest Her2+ expression.

[chrisy]

Well said, Brenda and Schoonder. And on point. I know you won't mind if I plagiarize:) I think the FDA is applying old rules to a new game, and it just doesn't work.

When we say it to the FDA, cc your congresspeople and your newspapers. I'm working on my letter now. Just the other day, the FDA reversed position on another drug they were about to pull, based on overwhelming response.

Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 20993



Commissioner Margaret Hamburg, M.D.
301-847-3531 (fax)
margaret.hamburg@fda.hhs.gov


Deputy Commissioner Joshua Sharfstein
301-796-5040
joshua.sharfstein@fda.hhs.gov


Director Dr. Janet Woodcock
Center for Drug Evaluation and Research
Building WO51
Room 6133
Mail stop HFD-001
301-847-8752 (fax)
janet.woodcock@fda.hhs.gov


Director Richard Pazdur, M.D.
Office of Oncology Drug Products
Building WO22
Room 2212
301-796-9909 (fax)
richard.pazdur@fda.hhs.gov

Contacting congress:
http://www.contactingthecongress.org (http://www.contactingthecongress.org/)/




Also of note, check out the attached link to the petition re the FDA pulling Avastin. Could be another path to make our voices heard. Some of the issues are the same and we have many people on this board benefiting from Avastin who potentially could lose this also!
http://www.thepetitionsite.com/3/stop-the-fda-from-disproving-avastin-to-treat-metastatic-breast-cancer/

[ElaineM]

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]

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]

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]

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]

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]

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]

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 (http://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-releases/display.do?method=detail&id=12947
I think it is a good idea to check for press releases regularly.
http://www.gene.com/gene/news/index.jsp?p=www.+gene.com&fr=ush-everythingy&ygmasrchbtn=Web+Search

[Patb]

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]

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]

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]

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]

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]

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]

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]

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]

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/ns/health-the_new_york_times

[Trish]

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]

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]

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/blo (http://thehill.com/blogs/congress-blog/healthcare/119305-a-plea-to-the-fda)gs/congress-blog/healthcare/119305-a-plea-to-the-fda (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.cbsnews.com/stories/1998/08/01/eveningnews/main15218.shtml)
http://www.msnbc.msn.com/id/32359544 (http://www.msnbc.msn.com/id/32359544)/

[Sheila]

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...

__________________

[Jean]

Sheila,
I am hoping that your cardo dr. will have a plan to raise your muga. I feel your frustration and I am praying that we will hear a solid strong plan so you can get on to the TDM1....

Sending you love,
Jean

[michka]

Oh Sheila! You have been waiting so long. I hope you will have good news coming from your cardiologist and that you will be able to jump in the TDM1 wagon. Waiting to read your next post and thinking of you. Michka

[Trish]

Oh Shelia, hoping, hoping, hoping you catch this break.....
Trish

[DeenaH]

I just heard about this last night and can't believe the FDA did this!

I'm not sure what is happening now with the letters, media, etc, but I have Dr. Slamon's contact info (or his Nurse Practitioner's) and have a consult with him November 22nd. I know that's really far away, but I can bring it up face to face if nobody gets a hold of him sooner.

His nurse practitioner is Kimberly Podsada. She can be reached at (310) 829-5471 and kpodsada.mednet.ucla.edu. I know it's not direct contact to Dr. Slamon, but she works directly with him.

If I can hand deliver a letter or any info in November when I see him, I can do that too. I am not stage IV, but I am scared to death of a recurrence because I still had alot of cancer after chemo, at time of surgery. Before chemo it showed on the PET/CT as being in my mammory nodes as well as my neck, 7-8 axillary nodes and a 7cm tumor in my breast. At surgery, the tumor did shrink to 3.2, but I still had 18/51 positive nodes and cancer in the fatty tissue between levels 1 and 2. Even though I would show as NED on a PET/CT now, I don't believe I am for one second. Even though I don't qualify for TDM1 now, I might need it in the future. Even if I don't, I want to fight for those who need it NOW!

This is just so frustrating!

[Jackie07]

Not sure what Dr. Slamon can do about it. But perhaps you can print this thread and show our 'outcry' to him?

[Chelee]

DeenaH,
I'm still willing to do anything we can about this TDM-1 nightmare. If we are pro-active enough I believe we can make changes & speed this process up. There were so many of you here with great ideas...I thought we were off to a great start.
TDM-1 needs to be available for women with metastatic & earlier stage bc. I was looking forward to at least some more EAP sites..but we now know that's not going to happen either.

The ramifications from denying us access to TDM-1 is serious...it will cost some of us our lives. Why should women have to beat up their bodies first with all these toxic chemo combo's first to make the FDA happy. I'd sure like to get the ball rolling on this some how?

Jackie's idea is a good one. I've talked with Dr. Slamon and he is very geninue...he cares. He might be able to give us the best direction to go in? It's worth a try. I'm also open to Elaine's idea of a petition...and I know of another bc board where there are lots of women upset about this very same thing, & will gladly join in to sign petitions, write letters...or whatever it takes. I'd also like to "steal" Shelia's line that she used to get attention when she was fighting for Tykerb. She said to them, "I am dying to try this drug!" Perfect...gotta love it. :) Remember the news on TDM-1 has been shown to work great as a 1st line therapy...this isn't just for metastatic bc...this is to benefit all of us.

Chelee

[ElaineM]

Thanks for reviving this subject. I was wondering what if anything we have decided to do about it. If I remember correctly some people were willing to be interviewed. Some people were going to write letters.
Somebody was going to ask Christine for her opinion.
I said I would sign a petition and offered to do research. I included several links to important information during our discussions. I thought October would be perfect timing, because it is breast cancer awareness month.
It seems the subject has been dropped. I don't know why. I wish I knew. Maybe we all got too busy, but this subject is an important one for every one of us, even those of us who are NED, because unfortunately cancer can return at any time, even years later.
I heard expanded access will end October 31st. However, some of the T DM1 clinical trials will continue.
If interested people go to clinicaltrials.gov and do a search for T DM1 they will find available trials. I guess that is the only option until the FDA fully approves T DM1.

[Chelee]

Elaine,
Very nicely done...you summed up most of what was all discussed in this thread...pretty good girl. :) Like you I would like to know why the interest in this thread has been dropped too? Everyone was so fired up and again with great ideas and wham...it all stopped? I do know some got into the EAP sites so they just might be really busy with all the running around? I know when you join a trial your lucky if you can come up for air. But I sure hope those, and anyone else here will feel like jumping in to do their part...it's to help all of us. We can make a difference. Actually with elections coming up, along with Pink October...this is a perfect time to make ourselves heard.

Elaine makes a very good point...cancer can return at any time and it would be so nice to know we all have TDM-1 avaliable instead of all those toxic chemo's to run through! TDM-1 has been such a life saver literally to so many. I sure hope we can get those that were interested in this to come join us. We need to strike now...the sooner the better.

Chelee

[ElaineM]

Thanks for your comments Chelee. I would like to keep this discussion current for those people who reach the point where they need T DM1. Everyone has brought up important points and there were several links that might be helpful to some of our members. Maybe additional information or news about T DM1 can be added from time to time, so that people who need the information can have it in a convenient place.

[DiDi70]

How about a directed campaign of letter-writing to everyone's local newspaper with an email sent out in advance to all media outlets, particularly those in Washington? This would create buzz. Also, would there be any benefit in contacting local offices of Members of Congress, to ask their opinion on who we can contact in the hope of making an impact?

I suggest also, trying to contact celebreties, who get a ton of press and who have no qualms, usually, about speaking out. Maura Tierney, who is an actress and who starred for years on ER, has a new series out now and she is all over the media, having just battled breast cancer too. A letter to a group of celebreties who have been through what we are going through might gain some momentum - especially when this is cancer awareness month and there's a lot of press-coverage for cancer topics. It couldn't hurt to try, I think.

Is there anything that I can do from Canada to help out?

[chrisy]

I wouldn't say that the subject has been dropped...or that interest in the subject has been dropped. What has dropped is visibility and discussion of it on this thread.

What is pending is a strategy and organization to enable us to better make our voices heard, and work to be done in drafting sample letters and hopefully a position paper. I have been actively working on this with a few of the folks who had posted on this thread to get this started. If you would like to participate in this process, send me a pm.

Some have been taking individual steps, writing letters, trying to get interviews etc. I did take a vacation...but shlepped along about 4 pounds of articles, links and paper to draft letters on. (un)fortunately I didn't have access to keyboards or internet, so I had to spend much of my vacation....relaxing. Now I've been working 7 days a week to catch up but I'm not complaining because being able to have a full life is what it's all about.

I've talked, and learned behind the scenes about how to set up strategies to make our voices heard and researched where we can find allies. TDM1 is a hot issue; and each news report (such as several this week from the Milan meeting) is another opportunity to add our voices to the discussion.

Creating a sustained effective response is new territory for us as a group - we are primarily a support group, not an advocacy group. So we do need a strategy to do this - what we can do, what we should do - and most importantly, what activities have a place in Joe and Christine's home. I've asked Christine for permission to set up a room - even if it's the back porch - where those who are interested can work on these issue.

In my discussions with people who do this for a living...I've learned we are dealing with strong forces out there. Strong and sometimes difficult but not impossible to move.

The advice I've been given is that letters - both personal and as organizations - to the key FDA players and your local congresspeople and senators CAN have an impact. Factually supported letters are effective, but your personal perspective as a person living with Her2+ cancer is also impactful.

I had a long discussion with someone on my flight to Italy about TDM1 - and realized that although she considered herself a breast cancer advocate she had NO CLUE what Her2+ was, or what that meant, and certainly nothing about TDM1. I'm sure this applies to virtually all of our elected representatives (and possibly, I fear, some in the FDA!). Educate them at every opportunity.

I posted contact info for these people (FDA and how to contact your representatives) earlier in this thread. If you have meaningful input, send a letter now - don't wait. Then send one AFTER the election as well!

If you do not feel confident enough at this point to do so, one of the supportive projects we are working on is to draft some key talking points...but many of them are also already contained within this thread. Be courteous and respectful in your communications (this forum is a great model for that). It's hard to win someone over to your position by calling them names.

So, if you can do something on your own (write letters to newspapers, get an interview, deluge your elected representatives) now, DO IT! If you would like to participate in setting up a broader strategy to make our voices heard on this and other issues, send me a PM.

If you're not ready to do either of these things and need some guidance/support, hang tight. Oh, and if you've ALREADY told me you're in for the strategic part (you know who you are!), you know there's no backing out now and I'll see you on FB and in SA.

[ElaineM]

Hi Chrisy,
Thank you so much for your efforts. I think a "room" or thread devoted to T DM1 is a great idea.
That way we can consolidate our efforts, concerns, and whatever information we have to share with each other regarding T DM1.
I have been quietly doing whatever I can do as an individual. I might be able to help with a small amount of research or do something related to a petition if I can. I am thinking we might be able to put something like a petition online. Those who wish to ask more people to sign it could print copies out for that purpose and send it to a specific location choosen by the majority of the members after they have collected some signatures. I would be happy to share any information I come across related to T DM1.
I too have found many people, including health care professionals who take care of breast cancer patients who are not familiar with T DM1. I tell them about about it whenever I think it is appropriate to do so.
In my mind whatever I have read and learned about
T D M1 makes me think of it as one of the best things that has been developed since Herceptin.
I know you are very busy, but please keep in touch and share whatever you can with us whenever you can.

[schoonder]

The 3rd line MBC T-DM1 phase II trial TDM4374g, which started mid 2008 used by Genentech to file for priority review, wasn't the only evaluation of this drug in this patient population. A similar phase II trial in this setting, TDM4258g, that got underway in July 2007 also had very positive data.
Is it possible that participants in this trial did have prior experience with treatment options Genentech per FDA failed to include in trial for which NDA was filed and if so, couldn't that data, in conjunction with positive results from multi-national 1st line MBC TDM4450g trial be used for FDA to reconsider their decision?

[chrisy]

I think the data from TDM4258g was made available to the FDA and no doubt the study participants had previously had multiple treatment combinations. But if their position was that they had not taken "all"...same answer.

That said, the FDA could decide at any time to reconsider their decision for any number of reasons.

[schoonder]

It appears a disconnect occurred between FDA and Genentech somewhere between first T-DM1 phase II trial for 3rd-line Her2+ MBC patients and second phase II trial which by design had greater emphasis on targeted aspects of prior treatments i.e. disease progression with both Trastuzumab and Tykerb/Xeloda regimens. What else could've been reason for conducting second phase II trial?

It's readily understandable that if T-DM1 showed efficacy where those prior targeted therapies, the ones most frequently prescribed because they heralded greatest promise to be effective, yet had now failed, that yes, a new life sustaining drug was brought into existence.

For Genentech to drag out their trial, searching for participants that had failed a number of infrequently prescribed treatments, ones not particularly suited for Her2+ MBC, i.e. lack of efficacy and high in toxicity, just to prove that T-DM1 was also superior to those regimens, wouldn’t that just be ludicrous?

Guess what, FDA totally failed to take into account targeted Her2+ aspect of this trial. Outdated directives didn’t provide FDA with an out to review T-DM1 3rd-line MBC Her2+ phase II trial results even though this agent clearly showed its pre-eminence over previously approved Her2+ targeted drugs.

[ElaineM]

T DM1 clinical trials that are active, not recruiting, or available from clinicaltrials.gov
http://www.clinicaltrials.gov/ct2/results?term=T+DM1+clinical+trials

[ElaineM]

In addition to what I am doing personally I am willing to put a link to a "in support of T DM1" petition we start on my website and on the personal e-mail account I use for medically related e-mail.
I did this to promote an event sponsored by another organization I belong to and found this to be a good way to promote the event without cost. Every e-mail I sent advertised the event for free. Every time someone visited the website I have for that organization they saw the event advertised for free.
__________________

[Unregistered]

I am looking for women in New England who were told they could not have this drug after being approved under compassionate care. I have a Fox News station here willing to do an investigative report but we need women to come forward.
Susan

[Barbara H.]

Hi Susan,
I suggest that you call the breast oncology unit at the Dana Farber and mention what you want to do and why you want to do it. I would also mention that you received this information from this website and that the thread was started by a patient at Dana Farber who was on TDM-1 for two years in a trial. There is currently no way for patients at Dana Farber to receive this drug for compassionate care. You are dealing with patient privacy issues so if you are unsuccessful with receiving information or help, post again and I will contact my oncologist to see if he has patients who would be willing to speak with you.

This is a major medical issue because it is a miracle drug that is very easy to tolerate, and there are cancer patients who will lose their lives if they do not receive this drug soon. They can not wait until 2012. They have used up their chemo options. After a breast cancer patient has used a number of chemo drugs, the drugs that are left do not seem to work well or at all. This targeted therapeutic drug has a good response rate for this population.

Thank you for your help.
Barbara H.

[ElaineM]

http://www.clinicaltrials.gov/ct2/show/NCT01120561?term=T+DM1+expanded+access&rank=1
and
http://clinicaltrials.gov/archive/NCT01120561

It looks like as of October 14, 2010 T DM1 Expanded Access is still available. I hope so. Please see my previous link to all T DM1 clinical trials and the phone number I posted for more information about T DM1 Expanded.

[schoonder]

Trying to work around (SURVIVE) a terrible beaurocratic state of affairs, not many will be able to.

http://www.boston.com/business/healthcare/articles/2011/01/05/testing_rules_force_patients_to_wait_for_new_drugs/?p1=News_links

[michka]

Thanks Schoonder. Very interesting article. But 2013....

[schoonder]

Michka, I believe that early 2013 is a very realistic timeframe to see T-DM1 approved in USA. Roche has indicated that they now will use progression free survival data from international her2+ mbc phase III trial Emilia, that compares this agent against Tykerb/Xeloda for approval purposes and that they expect to have this data available for FDA by mid-2012. If company can meet this schedule and if FDA is satisfied with overall contents of package and is able to complete review of this application within their usual timeframe, then yes, oncologists could be able to acquire this agent in 2013. Maybe bureaucrats in Europe will be somewhat faster in getting product's paperwork processed once Roche applies for approval over there.
I still maintain that FDA's RTF letter, their refusal to look at the data, should be reversed. Agency is 100% in the loop of development status of this agent which by now has progressed into two large phase III trials and which the FDA with much earlier phase II results had no problems with, to see it prescribed for those very needy in a compassionate use fashion.