Birds In Spring: Waiting On Carfilzomib
I try to make a point about not talking in my column too much about whatever symptom, side effect, or malady is affecting me at any particular time.
But suffice it to say that lately there has been a lot going on with me, and it has brought to the forefront that inevitable discussion about what to do next should my current treatment regimen of Revlimid (lenalidomide) and dexamethasone (Decadron) start to fail.
I’ve discussed this prospect at various times over the past year with the handful of myeloma doctors who treat me as well as with other myeloma doctors I know. They are unanimous that the next best thing for me would be a drug in the pipeline called carfilzomib (Kyprolis).
The book on this new drug is that it is pretty effective and well tolerated.
The myeloma doctors I know talk about carfilzomib’s efficacy and especially about the fact that side effects seem to be relatively few and less onerous than with many treatment options.
I’d say that carfilzomib is one of the most highly anticipated new treatment drugs for myeloma – right up there with the introduction of Velcade (bortezomib) and Revlimid a few years ago.
Carfilzomib, however, is having a bit of trouble getting to the marketplace.
In December, I was watching NBC’s Nightly News when there was an item stating that the U.S. Food and Drug Administration (FDA) announced that it was not going to use the expedited approval process for carfilzomib – that it “did not see the urgency.”
In one of those talking-to-an-inanimate-object moments, I started shaking my hand at my TV and at either Lester Holt or Brian Williams (I was too distraught at the time to remember now which one) saying things like:
“Wait a minute, who says there’s no urgency?”
“I’m a myeloma patient – I’ll bet there are a lot more who think there’s an urgency here.”
“It’s urgent to me!”
So, instead of being approved – if it even gets approved – by this March, the FDA won’t reach a conclusion on carfilzomib until July 27 through the standard review process.
Not getting expedited approval was a surprise to just about everybody – even Wall Street analysts. All the myeloma doctors I spoke with in the past year told me that they expected carfilzomib to be approved right after the first of the year. Not so, now that the FDA has spoken.
It’s been a bumpy ride for this drug.
The first significant problem happened in the fall of 2010 when Onyx Pharmaceuticals, the company developing carfilzomib, moved from clinical to commercial-scale manufacturing, which resulted in “minor variations.” This required fixing, of course, and an FDA review, forcing delay in the plan to file a New Drug Application by the end of 2010.
Another problem is that research supporting carfilzomib is only an open label, single-arm Phase 2b clinical study. In a single-arm study, patients are managed with a specific therapy, and a proposed drug is systematically observed to measure outcomes among patients with the specific disease. Participants are not randomized to receive either the study drug or the standard treatment, so no direct comparison can be made.
Pretty much everything I read in researching this column says that the FDA’s Oncology Drug Advisory Committee really prefers Phase 3 trial results. Nonetheless, the FDA has given new drugs approval based on Phase 2 research on several occasions.
One of the things that Onyx did last year was gather additional safety data from other carfilzomib studies that are underway, which were included in the New Drug Application that was finally submitted at the end of last September.
The good news is that the FDA accepted the application in late November. In its filing communication letter two weeks later, one of the review concerns raised by the FDA in accepting the application, however, was whether there was appropriate balance between risk and benefit.
The letter also included the bad news: The FDA said no to a priority review.
In order to get priority review, it’s important to show significant benefit and a high degree of unmet need, especially when the supporting study is a single-arm Phase 2 trial.
So I guess what the FDA is saying is that while the results of the study show respectable benefit, they aren’t spectacular, and that those of us who are relapsed/refractory have a number of other treatment options that work.
Well, for many people that’s likely true, but for others, maybe not so.
You can get carfilzomib today under two conditions. One is through a clinical trial, which means you can only get it at treatment centers participating in the study.
For me, if the time comes when switching treatment makes sense, it wouldn’t be too bad to enter a trial – under normal circumstances. I’d have to be at Memorial Sloan-Kettering Cancer Center two days a week. Since I work in New York City, that’s pretty much a no-brainer. Heck, lots of myeloma patients have packed up and moved temporarily to another location for months in order to participate in a clinical study.
Unfortunately, if that “time” to switch is neigh upon me, my circumstances right now aren’t “normal.” I’m sort of under indefinite house arrest with a broken foot, and home is almost 200 miles away from Memorial Sloan-Kettering. Everyone there feels that trying to make that trip frequently would likely wreck any healing that’s going on with my broken bone.
You can also access carfilzomib as a last-ditch “salvage therapy.” The Carfilzomib Myeloma Access Program (C-MAP), a joint undertaking of Onyx and the Multiple Myeloma Research Foundation, makes carfilzomib available to seriously ill myeloma patients in the U.S. lacking any other treatment options.
As for the prospects of carfilzomib winning approval this summer, the jury is still out. One Wall Street analyst pegs approval chances at around 80 percent. Others aren’t quite so sure, seeing the failure to win priority review as a red flag. And these Wall Street guys aren’t ignorant about things, especially new drugs, because there’s oodles of money to be made by those who get in early in pharmaceutical developments. If you want to know what’s going on with a drug that’s under development, check with the Wall Street guys – reporters and analysts. They pretty much deal with the cold, hard facts.
If carfilzomib doesn’t get approval, Onyx would have to submit a new application supported by a Phase 3 study; the main one underway is called “Aspire.” It would move up a prospective launch to sometime in 2014, at the earliest.
Okay, just let me say that this all doesn’t seem right to me.
I know that the safety-benefit issue is important, and I have heard a few anecdotal reports of individual problems with carfilzomib. But a handful of serious problems accompanying most any drug out there seems to be a matter of course today – just listen to the disclaimers on those incessant pharmaceutical ads on television.
I think the key matter here is that, as best I can tell, the benefit of this drug is clear.
It works. Not for everyone. Maybe not spectacularly. But the key thing is: carfilzomib is effective.
The fact is, and we all know it, there’s no cure for myeloma. And no single treatment works forever.
We need in the arsenal every possible and practical drug intervention to keep us alive.
I think the failure to give priority review to carfilzomib, and an FDA decision that the drug's Phase 2 study is insufficient for approval, would deny myeloma patients an apparently effective and overall safe treatment option.
Carfilzomib is the wrong drug for the FDA’s Oncology Drug Advisory Committee to use just to make a point about Phase 2 research.
I think that doing so would be unconscionable.
Forcing relapsed myeloma patients to continue to wait for the wide availability of carfilzomib – perhaps another two years or more depending on how this drama plays out – would be just plain wrongheaded.
Lou Ganim is a multiple myeloma patient and columnist at The Myeloma Beacon.
If you are interested in writing a regular column to be published by The Myeloma Beacon, please contact the Beacon team at .
Actually the odds of Carfilzomib getting approved on the basis of Phase 2 trials with no comparison group, after showing a modest ~20% response rate in late stage (post-Velcade) patients, was always bad and, given the recent FDA decision, is now very bad. The drug will be approved eventually, but probably not until >6 months after a 'proper' randomized, controlled Phase 3 trial is completed, i.e. ~2015.
I'm certainly not saying this is a good thing, but it is reality. Wall Street Analysts who have said otherwise were sell-side, meaning their job is basically to hawk Onyx Pharma stock to the clueless. And many, though not all of them, are rather clueless themselves.
Thanks for another great column, Lou.
Although I think there is something to Mark's assessment, I would not take it too seriously. I think he has a vested interest he's not revealing.
His language and arguments sound a lot like those I have read elsewhere online by someone well known for relentlessly trying to drive up the value of Celgene's stock. I think he also made comments here at the Beacon last year during the Revlimid secondary cancer controversy. Although he posted under a different name.
Approval of carfilzomib this year could create competitive pressure for Celgene. So Mark and other Celgene investors want very much to convince everyone that there is no reasonable argument in favor of carfilzomib being approved this year.
So take what he says with a big grain of salt.
Hi Lou, thanks for the informative column, and hope your foot injuries heal up soon also! I see from the Myeloma Canada website that clinical trials involving carfilzomib are also ongoing in Calgary, Winnipeg, Toronto and Montreal.
The National Cancer Institute of the NIH has two ongoing trials involving carfilzomib and, according to Dr. Landgren's recent interview with the Beacon, will soon have a third for SMM this Spring. I have a gut feeling this bodes well for carfilzomib's early, rather than later, approval. I would imagine the FDA logically consults with the NCI/NIH, being part of the same government, and its researchers for its input on approval. Maybe I am wrong.
Marty,
I don't see how carfilzomib would be bad for Celgene.
I think all "customers" will use all of the drugs available in their battle with mm. We might even consume more Revlimid, Thalidimide or Pomalidimide in combination with carfilzomib.
I put a small amount of Celgene stock into my retirement account. More of a good karma investment than anything else.
I agree with you that Mark has a vested interest and is probably shorting Onyx.
Is this the first time MMRF hasn't been able to get FDA to expedite a MM drug?
Marty,
Thanks so much for your post!! ICAM!!
I have had the same experience here at the Beacon, where a post is clearly, 'promoting' Celgene and/or MIRT. but let me not digress.
The FDA Advisory panel, is funded today primarily by the pharmaceutical industry, due to government cut backs to the FDA under the Bush administration. This creates a serious conflict of interest and makes the approval process far more political. The bigger the company the more clout they have with the advisory panel.
It is very much in Celegene's interest to lobby for approval delay of carfilzomib, it is also in the interest of Millenium, who is bringing out an oral protesome inhibitor which as present insurance stands is not coverable. So you have 2 companies who are likely using their resources towards a common goal.
Like Terry, I am in an NIH trial.
NIH clinical triasl provide the imprimateur of "unbiased" (i.e. not pharma funded) and will have more of an impact on the advisory panel. I heard yesterday that the results of the trial I am in are so significant (100% response) that the data will be used to re-expedite FDA approval of carfilzomib that has thus far been denied.
So Lou, hang in there...help is on the way sooner than you may know.
If MM patients want to become pro-active, what they should do is learn every single name of the oncology FDA advisory panel...and lobby them. Start a letter writing campaign. We have patient power.
Lori, to your point. I do not know how much money is backing MMRF or how they wield their influence but it is also worth investigating and lobbying those who are vested. I suspect that Onyx, has less clout as a fairly new company pushing a drug through the Oncology advisory panel, vs. Celegene...
But let's not despair.
We have power as patients if we direct and focus it on the areas where decisions are made for approvals. Yes, we are up against big pharma. But collectively, we are a diverse group of backgrounds. We can make this a public issue. Write letters, get a spot on the news, advance the cause of cancer. People respond when they hear drugs are denied or being witheld from cancer patients.
KNOW the world of MM expertise is small. The individuals who are on that advisory panel are already likely known to us !! Why do you think those disclosures are made prior to presentations at made medical meetings. We can influence and bring pressure on them. The pharma checks may be good, but typically they value the hippocratic oath, and their personal ethical reputations more.
Let's marshall our energies..acquire the names..and bring pressure to bear.
The HIV community successfully used this approach to expedite approval of drugs during the 90s.
That last comment is directed at any naysayers. There is a model..it has been done and it was successful.
Do not discount that this is an election year either!!
We can have a real impact.
Let's move not moan!!!
Thank you Lou for pointing us in the direction we need to move.
Lou
Great article with some things that really needed to be said, and very timely for me. It is very frustrating for those of us with advanced disease now refractory to velcade. The truth is with all the hype about new drugs nothing new has been FDA approved for multiple myeloma since Revlimid in 2007. As for cMap expanded access program this protocol does not allow for Carfilzomib to be combined with any other drug except dex. Since as other have mentioned carfilzomib only works in about 20% of velcade refractory patients its best bet for those of us relapsing is in combination with other drugs. Not an option on cMap
O and one other thought on this. Millenium also manufactures Velcade and they are getting the SQ route expedited..that will considerably impact carfilzomib. Millenium then has an even bigger interest than Celgene then in delaying approval of carfilzomib.
I wouldn't be surprised if that is why
http://www.myelomabeacon.com/news/2012/01/25/questions-and-answers-about-the-fda-approval-of-subcutaneous-velcade-bortezomib/
Bortezomib has a longer history of use, which along with the SQ, considerably more clinical trials...that is a winning marketing message against carfilzomib. Doctors are far more inclined to use a drug with longer years of experience as there are fewer unknowns, and far less surprises for them to manage. Millenium will iwin the marketing message with these points in the local oncologists office.
Which means more folks will receive bortezomib than carfilzomib even when it does receive approval.
Point of fact, what will likely happen is docs, will use carfilzomib in relapsed/refractory patients while bortezomib will claim the newly diagnosed patients despite carfilzomib having received an indication for the newly diagnosed.
Not to mention that even the MM experts have far more experience with bortezomib.
In addition, all these new statements about not using up all the available therapeutic options at once, (another pharma marketing message, if I ever heard one) seems to be creeping into the expert's presentation. This too, was a common message during the HIV battle for approval. Effectively what this does is relegate one mfgrs agent to 'second line use' DESPITE it having approval for first line use.
Gotta give it to Millenium..they are working on multiple fronts to hang on to their profits.
I bet those power point presentations on the decline in market share, if carfilzomib hit the market prior to their SQ approval, must have been a real wallop in terms of profit, with a finite market.
I hate to be the bearer of bad news but Carfilsomib may not be the next wonderdrug. After experiencing rigors, Sinus Ventricular Tachycardia (High high rate) one day and Bradycardia (low heart rate) the next requiring hospitalization, it is clear that non toxic dose regimens have yet to be established. Equally disappointing was the lack of efficacy of the Carfilsomib on my myeloma. Chatting with other patients in the clinical studies my experiences are by no means unique. It is the worst drug that has been administered to me to date. I hope that FDA examines the data thoroughly before approving the drug to an unsuspecting myeloma patient population.
Kevin,
Just to affirm those reports out of ASH. I have a VGPR after 4 cycles.
So Gary,
is Millenium paying you or what?
O that's right your check probably doesn't come from Millenium but rather from one of those consulting marketing firms they hire to send out to post in forums and hold focus groups with community physicians.
Sorry buddy, but your experience is an outlier and it is not by any means deterring those who ARE having an outstanding response and NOT looking for a wonder drug.
The only wonder drug in the world is FAITH.
Have a great day!!
I was also shocked when the FDA said it did not see the urgency in doing a priority review. My first thought was whether they would change their mind if one of them had MM and was relapsed with limited options.
As anyone who's read my posts knows, I'm what I consider one of the fortunate that have been able to get into a clinical trial with Carfilzomib. As far as I'm concerned the responses have been as good or better than any other treatment available, though the mean time to relapse has yet to be established. Reports from ASH this past December indicated that for patients having received 12 or more cycles of treatment, 100% achieved VGPR, and 79% reached nCR or CR. To me, that seems to "show significant benefit".
I realize this isn't totally relevant to the recent submittal for approval since that was only for relapsed patients, and the results for relapsed patients are not as high, but I see this approval being a stepping stone to getting it approved for newly diagnosed patients as well.
Bottom line - given a terminal disease, any drug with promise of keeping the disease at bay, particularly with very tolerable side effects, should not be considered low priority.
Gary, I am sorry to hear that you have had such serious problems with carfilzomib. I feel extremely lucky that Dr Landgren's NIH carfilzomib trial for newly diagnosed MM patients was available at the right time for me. Carfilzomib has worked for me: I am in a near CR after 4 cycles and I have had no serious side effects, no neuropathy, no nothing, only occasional phlebitis from the infusion. I want to note that I have had no heart issues or high blood pressure before starting or during the treatment, and I am in a rather good physical condition otherwise. My sincerest hope is that carfilzomib will be approved soon so it can help you Lou and all MM patients. Suzierose is super in researching the names and will find the names to lobby ! I will certainly sign on to lobby.
It's great to see this spirited discussion, and we hope it continues.
However, let's be a bit careful about the accusations we make about the motivations of other people commenting in this thread. Also, going forward, perhaps we could agree to disclose any reasons for possible bias that we might have, in the spirit of full disclosure. If people do that -- and many have -- it probably is best to take them at their word, unless there is a sound reason for questioning what they have said (which should be explained).
If Gary says he had a really bad reaction to carfilzomib, then it's entirely plausible. He said something similar in a comment on another posting (assuming it's the same Gary).
Isn't his experience as valid as those who have had positive results with carfilzomib? There are other people who have commented on Beacon articles who have had severe negative reactions to myeloma drugs that, for many people, have been very effective. It can happen.
Suzierose, I understand your position with Gary's response but the ambassadors for pharmas are not contracted nor paid to defend them and write articles. They are contracted and trained in a required FDA set guideline to go out to tell their story offering inspiration and hope and they are not allowed to push the drug regimens they are on. They are allowed to thank the pharma for making it possible for them to be there (airfare/hotel/honoraria) and if 'branded' can mention they are on the drug the company makes in their story. Having spoken at one official and one unofficial (invitation of a group where I was visiting in the area) I can tell you that it is inspiring to share and have a dialog, not on the drugs, but how we cope with myeloma in our lives. So I just wanted to clarify that point as it seems to be sometimes misunderstood.
Also, the FDA has had conflicts of interest in its approval process for the better part of it's existence. Doctors who approve drugs are on advisory boards or even work for the pharmas involved in the drugs. I know this as long before myelomaville I was involved in protesting this very point with the onset of drugging school children and Eli Lily. It has nothing to do with any recent "cuts". FDA relies on experts to help them facilitate the approval process and those experts generally don't work inside the government but outside. Its an incredibly frustrating system. I could go off on a long tangent of how pharmas can do 7 trials and only present FDA committee the two that show the best results and hide the other five. But that's another subject for a different argument.
Whoa ... Is Gary actually a paid spokesperson, "ambassador" or whatever you want to call it, for Millennium?
If so, he really should have disclosed that. I'm assuming you are, Lori, given what you just wrote. If so, it seems like you should be a bit more open about that when you're making comments where someone might think that's relevant to your opinion -- even if you don't think it's relevant to your opinion.
I agree and thought I was.
Lou,
Thank you for your thought provoking article! I certainly have learned a lot.
In the interest of full disclosure (which seems to be an issue in this discussion), I am a DES Daughter. My mother took a drug called diethylstilbesterol when she was pregnant with me. DES has since been shown to cause cancer and other problems in the daughters and granddaughters of women who took the drug. I have been part of a study on the effects of DES since I was about 12 years old. Because of the problems it caused, DES was also one of the first major class action lawsuits against a drug manufacturer. The other major lawsuit was, of course, against Thalidomide.
I say this because I appreciate what the FDA is trying to do. Their system is laborious and is in desperate need of an overhaul, but still too many drugs get on the market only to be pulled later after they have proven to be ineffective or harmful. Think about all the problems with the breast cancer drug Avistan, the pain killer Vioxx, or even the diet drug Phen-fen.
I'm not a beliver in "big bad pharma" and I don’t really think that Celgene or Millenium is overly concerned that a competitor is introducing a newer or better product. As business people, I think they expect that this will happen! They also know that their patent protection only lasts for 8 – 10 years after the drug is approved (Velcade’s was extended to 2017), at which point other companies can market generics, and/or encroach on their scientific processes. So if they want to continue to make money off of their product, they had better have other plans.
And they do. Once a drug manufacturer gets a drug approved by the FDA for a particular disease, it is easier for them to get that drug approved for use in treating another disease because they have already proven that the drug is safe. I believe that manufacturer’s real goals are to find as many applications for their product as they possibly can. If you do a search on clinicaltrials.gov you’ll see that both velcade and revlimid are being studied for possible use in treating a host of cancers including lung, ovarian, and pancreatic cancers. With over 500,000 new cases of these cancers diagnosed in 2010 they are a far more lucrative market than multiple myeloma.
To me, it seems that Onyx tried to rush a drug without the necessary information to prove that an expedited approach was in the best interest of the patient or consumer. Shame on Onyx. But as a consumer, I want assurances that the drugs I take are safe AND effective. I don’t want to be in the same position that breast cancer suffers who took Avistan are. Hopefully the NIH studies will be able to show carfilzomib is effective and it will be available before the end of the year.
Lyn
I am appalled by some of the comments here regarding FDA, Celgene and Millennium. Many of these comments were made based on erroneous understandings of the drug approval process.
Hey Anon,
I'm not sure if you are responding to my post, or others. My comments are part personal experiences (the DES and needing a drug that worked but wasn't approved for a certain illness), part an odd mix of professional experiences (intellectual property and healthcare). I will be the first to admit that my A's and B's don't always add up to C. If I said something to offend you, please feel free to correct me, my shulders are big enough.
But it sounds like you might have something constructive to add. I hope you'll share.
Lyn
Lori
You wrote:
I understand your position with Gary’s response but the ambassadors for pharmas are not contracted nor paid to defend them and write articles
What basis do you have for this? I worked in pharma for over 20 years. I know what they do, and your assumptions are false. I know the process. I understand the process. And I can assure you had I not worked for 4 MAJOR pharmaceutical companies I might also belabor under your false assumptions. I see clearly what is happening here.
Which is why I directed our focus to those involved in the decision making process. Identify the physicians on the ONCOLOGY FDA advisory panel and lobby them.
That is our job as MM patients. NOT to support the profit making tactics of pharma.
Anon, I agree with Christa's Mom. Could you please share with us exactly what is so appalling about some of the things people have said here?
I've seen some errors in the details of what people have said. For example, if I'm not mistaken, the members of the Oncology Drug Advisory Committee are not changed from one meeting to the next just to reflect the kind of drugs being considered.
But I'm not sure that is such a major error. It's certainly doesn't qualify as something "appalling".
So what exactly is so objectionable?
Suzierose ... Just so there is no misunderstanding, I think that Lori is referring to Millennium's "patient ambassador" program, or whatever they exactly call it. She is not using "ambassador" as a synonym for salespeople, which is what I think you are thinking.
Marty,
I hear you!! Sales people are promoters, but all the other names such as 'ambassadors' are simply acceptable euphemisms for folks who do the same thing withOUT the sales title, thus they are far more deceptive and subtle.
And I what follows is being more aggressive, I am on steroids...just finished 2 days of infusion which includes steroids.
HowEVER, let me assure you. What the public sees and believes is NOT what occurs. The public is SUPPOSE to perceive PHARMA as Lori does. That is their job. The behind the scenes is a whole LOT different. The corporate stratgies are not public knowledge and they are conceived to achieve the very public responses we hear. How they deploy their marketing objectives are not perceptive to the public, unless they work there and watch HOW it is done. Prior to working the jobs I did, I could not see it.
Why else would pharma have an Ambassador program other than to promote their interests? The trick is making their connection and association deep enough to not be readily apparant..but pharma is paying the bills!! Their objectives are so interconnected that it is seamless.
So, while I try to listen to the other side, Lori gives, I worked on the other side. I know what is transpiring and more importantly I know HOW those objectives are achieved using the long handled spoon. No different than when the parent pretends to like the uncouth GF or BF so that their child does not rebel and stick with the varmint. Hoping and praying their child will see the light if they do express opposition, the parent however sees what the child can't, all because of their life experience the child lacks.
This is America and capitalism is KING!! It is all about profit. Anyone who loses sight of that is simply naive and unrealistic. All that feel good stuff simply makes them more profitable.
Listen to what Romney says if you do not believe me. The deepest cuts to FDA were under the GOP administrations because anything government does EATS their profits. I hate to get politically but it is the bald truth. Those who choose to defend politics simply are skipping over the real culprit CAPITALISM which has no politics other than politics for profit. All you have to do is pull up the historically data to see the truth. We have allowed 'free market" capitalism to reign and it has resulted in the poor being poorer and the rich being richer. None of these folks who proclaim to be for less taxes pay 13% in taxes, YET they believe those policies work for them? GMAFB!! No they do not. As long as you have earned income you will always pay 30% or more. To get to that 13% level of taxes you have to have UNEARNED income and as long as jobs are deported for taxes, you have a snowballs chance in hell of EVER reaching such...but I am digressing.
So, just let me say, this pharma is about PROFITS, nothing they do is for any other reason. AND as I said previously when Millenium saw how much market share they would lose, the dollars to prevent that and influence the FDA advisory committee PALED in comparison to the cost to buy the influence to get Bortizemib approved SQ. The trials supported that. No 'ambassador' or MM expert was 'comprised' by supporting that, as the medical evidence was there.
Pharma rules and I understand their strategies.
Hi Lori,
I love your optimism to death. But no, you do not understand me. I worked for pharma for over 20 years. Your view is from the outside in. My is from the inside out.
Your opinion is valid from that perspective. We will have to agree to disagree.
I was a Caregiver Ambassador and they don't write papers nor promote the pharma. I don't think that conflicts with your view of pharma's agendas with 20 years on the inside. Patients/Caregivers, social workers, support groups all have questions about drugs. Prior to a program the pharma has researchers, doctors, etc., to send. Putting a face with same condition to tell their own personal story without selling, promoting or pushing an agenda in treatment is of value and has very distinct FDA guidelines. On the one hand I can see how it looks from a cynical perspective. On the other hand I can see that it is a good will effort. All I know is, I told our story and sometimes answered questions about our experience and our lives.
I also worked with a group putting together questions regarding bisphosphonates. Helping them to understand the questions and concerns patients/caregivers have about them. They will be doing PSAs. And yes, I'm pretty sure that all of that is being funded by the pharma that makes them. I wanted to help. Help them to understand what I'm hearing on the ground. Help them to be more clear and precise in getting questions answered so patients/caregivers can make informed decisions. I suppose I could see how some would view this as some sort of treasonist act? I guess it would depend on how you feel about pharmas. It's not an easy situation we all find ourselves in with this disease. I'm frustrated for instance, with talk of optimism with all the new drugs in the pipeline to handle those who are now resistant. Is that the best we can do? Move from one drug to another to another to another. It's not a pleasant thought. Or I can remain hopeful that there are groups made up of individuals trying to do the right thing for the right reasons. Of course you know me by now, I would choose the latter.
Hi Everyone! This thread is getting kind of 'heated', but is still interesting to read. We just want effective treatments, and the drugs already available are marketed by 'big pharma', of course. I know a little about the world of drug reps and marketing of drugs...the reps have to try to make a quota, they are in a sales position. They try to convince the doctors to prescribe their particular brand of cure...the trouble is with these cancer drugs, is that there just aren't that many available, and also they are marketed to cancer boards and institutions, not so much to individual doctors. The profit motive there is pretty obvious, and it is a captive market. At least that is my impression..and the drugs are very expensive also! I know there must be flaws in this system , but to an extent we are stuck with it. Hope the new drug carfilzomib gets approved in a timely fashion...if the clinical trials are doing really well, then it should be approved as soon as those wrap up, don't you think? And if there are at least three drug companies involved in making myeloma drugs, then that should create some competition, and also incentive to do research. The original, university type research, that led to the development of the proteosomase inhibitor type drugs, led to those scientists winning a Nobel Prize in Chemistry (2004 I think). That is how amazing these new drugs are...like nothing ever seen in medicine before.
Thanks for all the info, about the drug companies, the Ambassador programs, and more. Just having fallen into this 'journey' less than three years ago, a lot of it is still new to me. Part of this 'journey' is about learning all we can about myeloma, its treatments and even the politics of the thing!
Call me naive, but what should be foremost, is what is in the best interest of the patients. I truely hope that any pharma political jockying will be seen for what it is and be ignored. We need more options and Carfilzomib along with some other new drugs in development should be approved as soon as possible. The only issue for me is safety. If the drug shows a benefit and is safe it should be approved. The fact that no new MM drugs have been approved since 2007 took me by surprise, and if actually true, is inexcusable. We all need to voice our opinions to the FDA since our lives depend upon it.
Ron H
Susie,
Good old capitalistic Big Pharma has saved my bacon. My young children might even graduate from high school before I die thanks to Big Pharma.
Please refrain from the political attacks. Many people don't agree with you but they are too classy to respond to your political accusations (on a cancer forum). Stan, who will not post his Avitar in case Suzie finds it ugly.
Lou--this is all your fault-ha.
I really like your writing. Please keep it up. Stan
Just the facts Stan. Nothing to do with class.
"The new Republican leadership in the House has been previewing across-the-board budget cuts since the party’s victory in November. What took many FDA stakeholders by surprise is the size of the cut.
The proposed cuts are a large number for FDA, an agency that has been chronically underfunded to meet its rapidly expanding authorities and responsibilities
.
Now stakeholders are wondering, if the cuts were to go through, what areas at FDA would be hardest hit.
“This would be prorated across existing appropriations. So for drugs, generics and foreign inspections in particular would be hard hit. Also drug safety,” says one official.
Cuts that big would almost certainly impact FDA operations across the board. If enacted, newer initiatives such as the food safety authorities, tobacco, and FDA’s regulatory science push would likely be at highest risk. FDA’s Sentinel active surveillance initiative also would be an area that could lose resources.
Democrats on the House side appeared surprised by the $78 million reduction and few had figured out exactly which FDA operational programs would be targeted.
On the drug/generic/medical device review side, the budget reductions would tilt some leverage toward regulated industry in ongoing user fee negotiations as the agency could be more dependent on private sector dollars.
Nevertheless, it would be hard to see a scenario where new drug, generic, and medical product review times aren’t substantially increased in the future if the agency’s budget is shrunk by the magnitude the House leadership is proposing.
“A significant cut like what is being proposed will hinder the agency’s ability to fully implement recently-established programs. This problem of adding responsibilities without sufficient resources is one that for years has eroded the scientific capacity of the agency, which directly impacts new treatments getting to patients,” said Ellen Sigal, Chair of the patient advocacy group Friends of Cancer Research.
Stan
just stay focused and post classy facts...this cancer forum blog thread topic is about FDA approval. Not avatars.
"FDA commissioner Margaret Hamburg recently told Public Citizen that the agency may loosen a three-year-old policy aimed at keeping industry-funded physicians off FDA advisory panels because the resulting vacancies are gumming up the approvals process.
An FDA spokesperson told MM&M: “The new conflict of interest rules have, in some cases, made it more difficult for FDA to find expert advisors. In most cases, FDA has been successful in finding experts to meet our needs. However, in a few cases it has been necessary to delay holding advisory committee meetings in order to allow more time to contact and screen more experts for consideration or to begin the screening process with many more experts. This increases the work requirement for advisory committee preparation.”
As of March, 23% of the agency's advisory committee seats – 138 of them – were vacant.
FDA CDER director Dr. Janet Woodcock said at a conference in May: “There is no doubt it is difficult finding highly experienced people who do not have conflicts.”
Bull, said the Project on Government Oversight (POGO), arguing that the number of conflict-of-interest waivers granted FDA advisory members has never exceeded 5% and that the percentage of seats vacant is falling, having sunk from 30% in 2009.
“These rules do create an additional hurdle, but that is exactly the point,” said POGO head Danielle Brian in a letter to Dr. Hamburg. “We want expert advice that is as free as possible from the influence of industry.”
"Cancer research funding remains flat-funded at FY2010 levels and it may soon face deep cuts.
Making good on election promises to reduce federal spending, the Republican-led House of Representatives recently approved a spending package (H.R. 1) that contains massive reductions in discretionary spending for the remainder of the current fiscal year 2011, which will expire Sept. 30.
H.R. 1 would cut current funding levels for the National Institutes of Health (NIH) by $1.6 billion, reducing the agency's total budget to $29.6 billion, a level last seen in FY2008. The full $1.6 billion is not across-the-board, but rather portions of it target specific line items such as the Global AIDS Transfer fund, the Project Bioshield Special Reserve Fund Transfer, funds for buildings and facilities, inflation for non-competing grants and the Common Fund. However, approximately $640 million is proposed as a “general reduction to 2008 levels,” and would directly impact the National Cancer Institute (NCI)."
Thanks for all the background info, Suzie. The straight facts make interesting reading! I will follow this with interest...not being an American, am doubtful that your regulatory agency would want a letter from me, but will stay focussed on the 'carfilzombib' issue. Hopefully Onyx would also apply to have their drug approved by Health Canada, and then would be the time to follow it up at home.
Yes, Stan, I am also extremely grateful for the use of the newer drugs to give me a better life. They worked really well , and for whatever the complicated processes are that brought them onto the market, I give thanks. I feel that God has given me some extra time and with some of that time, I should try to help others too. Of course, my children are grown up now and i do have more free time than when they were young (and am not yet a grandparent either!). So writing a few letters is not a problem, but I just need to feel comfortable with the facts and that I am qualified to have an opinion at all! Best wishes to you all!!
Suzierose,
Just one question -- without capitalism (and all that goes with it), how do you expect research for new drugs to be funded?
Lyn
Christa's Mom has a point. Capitalism and shareholder's profit motive are what have driven the pharmaceutical industry to research and manufacture the new therapies that are available today. Same as all other things in life, realities are probably somewhere in the middle of a broad spectrum. In this case, the drug companies are most certainly after profits to satisfy their own economic gain and their fiduciary duties to their shareholders. But at the meantime I am also sure researchers and executives alike want sincerely to eradicate terrible diseases like MM and prevent human suffering. It's a balance act to ensure the motivation for innovation is alive and well and, at the meantime, to ensure the pharmaceutical companies take on their moral responsibilities to their fellow human beings.
Regarding the work FDA will be conducting in advance of drug approvals, we should concentrate on pushing the FDA to conduct a speedier review process while not sacrificing the quality of work needed to ensure the safety and effectiveness of the drugs. The work should be two-pronged - to expand the availability of the drugs by the manufacturers to patients in urgent need by removing limitations and to rush along the approval process. It is just that, with a downsized FDA budget, this will not be an easy accomplishment.
I wonder where we'd be in terms of new drugs for MM patients if Hillary Care had passed in the early 90's. Would the profit motive have been there? Would we have Velcade and Revlimid?
How about Tort Reform? If that passed, drug companies would spend less time covering their behinds and more time "pushing the envelope".
I hardly think Republicans are the party in the path of drug companies profiting from newer drugs.
Any interview with a government official (FDA in this case) with regards to budget cuts is going to scream bloody murder. It's how agencies fight amongst themselves for the tax money.
Most importantly, the term Big Pharma has to go (especially in this forum). It's a derogatory term for an industry that is saving our bacon. Some of the doctors who visit this board, and many others who are involved in MM research get research funding from drug companies. Why on earth would anybody with MM want to alienate, or talk down to these doctors or these companies?
I do agree that it is important to let these review panels know we're out there. We are a small group.
I would like some direction in that regard.
How do we contact these nice, nice people on the FDA review boards?
Stann,
HillaryCare? Please. Never heard of it. Was that a bill that passed the legislature? Or just a classy term you made up. Perhaps, this is why you see politics in the forum because you have personalized it derisively with classy terms like HillaryCare. For me this is about funding and the politics of funding the FDA. Not HillaryCare or TortReform. Money and funding influence what drugs are brought to market and receive approval. Which seemed to be what Lou was expressing frustration over. That's where I was focused. On that funding, how it has been cut, who cut it, who stepped in to fill that gap and how their doing so impacts drug approval.
You think BigPharma is derogatory but HillaryCare isn't? Well I am from the MotorCity and we always called it the Big Three...nothing derogatory just the coldblooded reality of how they dominated the auto industry and marketshare. Same goes for BigPharma, They dominate the pharmaceutical industry as opposed to the small companies like Onyx or ImmunTherapy etc. Again, I refer to auto industry..back in the day Lincoln and Cadillac dominated the luxury vehicle market just as today pharma companies dominate specific therapeutic/disease groups like BristolMeyerSquibbs/Celgene/Genetech/ etc do when it comes to oncology and/or MM.
Perhaps, this is as simple as you may not know the history of BigPharma and which companies dominate which disease states? In the heyday of the 80s/90s Lilly was known as the antibiotic/diabetes house, Pfizer, antihypertensives;BristolMeyerSquibbs, oncology and then there was MerckSharp&Dohme..the biggest dog on the block, with the broadest product line that stretched across multi therapeutic groups and thus made them the most dominant company (based on profits and market influence) in the industry.
You may remember the Vioxx recall and why/how that happened?
so the question becomes....
Why are you defending a process which is clearly not biased towards patients but to profits?. Yes, many doctors receive funding from the industry that is their choice and it is a two-sided street. One need not talk down to any doctor to pursue the rationale for their decision or decision making process. Surely, you are aware of that and will not defend the defenseless when it comes to how money from BigPharma has major influence on how drugs are approved and the myriad ways in which they promote their products so as to increase their market share. Sorry, if this could be a rude wake up call for you about the industry. However, in order to bring pressure to bear on BigPharma you have to understand their strategies.
I did post the names of the nice, nice, folks on the FDA review board..when you add up the honorariums from one of them and note who they were from..perhaps you will no longer feel that the objective is to alienate anyone. We, as patients, don't have money to buy influence.
As I mentioned, the model for 'moving not moaning' has been successfully done...here is some background on it:
"On 3rd October 1985, the actor Rock Hudson died of AIDS. He was the first major public figure known to have died from an AIDS-related illness.
In 1986 the Surgeon General's Report on AIDS was published. The report was the Government's first major statement on what the nation should do to prevent the spread of AIDS. The "unusually explicit" report urged parents and schools to start "frank, open discussions" about AIDS.
ACT UP’s first demonstration took place on 24th March on Wall Street in New York. The group demanded access to treatment for AIDS, public education to stop the spread of AIDS, an end to AIDS discrimination and the establishment of a national policy on AIDS.
By 1988 the group had almost 3,000 members, many of whom were infuriated that little was being done while their friends and relatives were dying.
The first national, coordinated AIDS education campaign was not finally launched until 1988, when 107 million brochures entitled “Understanding AIDS” were mailed to every household across the country. By this point, nearly 83,000 cases of AIDS had been identified in America, and over 45,000 people had died.37 Six other nations had set up similar leaflet campaigns before America chose to do so.38
The introduction of antiretroviral treatment
In September 1986, early results from clinical trials involving AZT (zidovudine) – a drug that was first investigated as a cancer treatment – showed that it might slow the attack of HIV. The AZT clinical trial divided patients into two groups: one received AZT and the other received a placebo. At the end of six months, only one patient in the AZT group had died, while there were 19 deaths among the placebo group. The clinical trial was stopped early, because it was thought to be unethical to deny the patients in the placebo group a better chance of survival.
In March 1987 the U.S. Food and Drug Administration (FDA) approved AZT as the first antiretroviral drug to be used as a treatment for AIDS.
By 1988 frustration was growing over the length of time it had taken to approve AZT and the FDA’s slow progress in improving access to other experimental AIDS drugs. On 11th October 1988 more than a thousand ACT UP demonstrators descended on the FDA headquarters in Rockville, Maryland, demanding quicker and more efficient drug approval. Eight days later the FDA announced regulations to cut the time it took for drugs to be approved.
In 1989 results from a major drug trial know as ACTG019 were announced. The trial showed that AZT could slow progression to AIDS in HIV positive individuals with no symptoms. These findings were thought to be extremely positive; on August 17th a press conference was held, at which the Health Secretary, Louis Sullivan said:
"Today we are witnessing a turning point in the battle to change AIDS from a fatal disease to a treatable one."
The initial optimism was short-lived when the price of the drug was revealed. A year’s supply for one person would cost around $7,000, and many Americans did not have adequate health insurance to cover the cost. Burroughs Wellcome, the makers of AZT, were accused of ‘price gouging and profiteering’. In September, the cost of the drug was cut by 20 percent"
If interested, you can read rest here:
http://www.avert.org/aids-history-america.htm
Thanks Suzie for the synopsis of some of the issues surrounding medication in the '80s to do with AIDS. That era was known for its pathos, think of Freddie Mercury of 'Queen', or the movie, 'Philadelphia Story'. With better drugs available, and the advent of 'gay' marriages, hopefully it is a little better now for people with that disease. Even the practise of giving out free needles to heroin addicts (in Vancouver there is a controversial program for that) has helped to stem the problem.
Sounds like you have lived and worked through a lot of changes in the pharmaceutical industry! We need government to regulate the industry, but also need the industry to research and try out new drugs in their clinical trials. Clinical trials are really expensive to run...I doubt that governments would take that on very much. Governments are all 'cash strapped' nowadays...can't think of any country really that is flush with cash. Of course we must'nt forget the charitable sector, which also funds research.
Hope you are well, and that your health is improving too!
Hi Again..I realized after posting the above, that of course HIV/AIDS attacks people from all walks of life, and of any gender! My remarks were not inclusive enough of that disease. SORRY!! I do volunteer a bit of time every year for a fabric sale that raises funds for the Stephen Lewis fdn.. They support families in Africa which have been devastated by that disease...the grandparents looking after the kids, in many instances.
At least Myeloma isn't widely believed to literally be 'infectious' , although some people suspect that it runs in families.
Hey Lou!!
Where you at?
You brought up your dissatisfaction, why are you silent? While worked for BMS and I understand how they design clinical trial and buy FDA approval. YOU expressed how it impacts you. One of my jobs included Worldwide Safety Surveillance. I complied the reports that go to the FDA for adverse events (aka side effects). I know how that information is completed and reported.Did it.
Why are you silent?
Are you frustrated? So are many of us,
BMS also was one of the first with HIV therapy .in addition to oncology.which is why I know the story.
Whats up? Please share your thoughts.
Is the information not pertinent..or are you content to moan and not move? Either is OK,,,but in each of your other columns you have responded to posts,
Even keeping politics on the sidelines...surely you have some feedback. Don't wuss out on us. You identified a key issue.
If you have a another approach ...please share it...otherwise you are moaning...and our lives are at stake...we are talking survival and we need to MOOVE!!
Survival..I believe was your thrust no?
Hoping for feedback.
Hi Suzierose. It’s nice to be wanted. On the one hand, I’ve been intending to weigh in a bit more on this, but as I mentioned before, there’s a lot going on with me right now, which is getting in the way of my “normal” life, such as it is. I do think in the column I made it pretty clear where I stand and what I think. Nothing ambivalent there, I didn’t think.
Hi all – This was a great discussion thread, even if it did get a bit heated sometimes. It’s clear that feelings run deep on this subject and about those entities involved in the drug creation/approval process. Thanks to all for your comments, there were so many enlightening posts, and a great deal of effort spent by you all in trying to sort things out. In looking back through all the posts, I was taken again by Kevin’s comment about what might change at the FDA if one of them had myeloma and was relapsed with limited options. That fits in with Dr. Goodman, who is dealing right now with the reality of what to do when you’re running out of options. That’s a hard place to be. It’s what our little debate/discussion here is all about, really – for all that’s going on in the development of novel therapies, it’s a slow (sometimes glacially so), time-consuming process. In the back of our minds has to be that thought: who among us will survive long enough to benefit from the new stuff under development. We myeloma patients aren’t unique when it comes to the challenges faced by those with cancer. But pretty much each and every one of us faces the same fact of life: Whatever treatment we’ve taken/are taking, it’s going to stop working at some point. We all hope later rather than sooner. Worse, at some point for most of us, all those things currently in the “try this” box aren’t going to work at all.
Stan – you’re right, it’s all my fault…haha.
Another excellent column,Lou. I can emphasize with your frustration with the FDA and getting this drug on the maket. The "no urgency" comment defies understanding.
Ouch - you broke your foot?????