Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
>>> Monane said the FDA will probably ask for 72-week data on that trial. He also said the FDA probably will suggest a large Phase III trial involving about 1,000 patients. <<<
I've been wondering about this too , except I've been thinking 72 wks. plus 24wks off-tx. , for a total of 96 wks. for the SOC arm of the P3 trial.
I don't see how it can be ethical to stop tx. on all patients in the SOC arm at 48 wks. , when there is now a body of evidence that suggests that slow-responding patients have a better chance of achieving SVR by extending tx. to 72 wks. If such a protocol was adopted , it would mean 96 wk. registration trials to allow for completion of the SOC arm , regardless of how fast the experimental tx. might work.
It's possible that all of the recent studies on extended tx. times by Roche and S-P may have been done for the express purpose of slowing down VRTX.
New Canadian open-access medical journal
Open Medicine @ http://www.openmedicine.ca/
Vol 1 , # 1 includes a review of the book : " Pharmaceutical ethics ? "
Here's one paragraph of the rather unfavorable review , written by Dr. Jerome P. Kassirer:
"There is virtually no mention of the pharmaceutical industry's major ethical lapses, such as hiring ghostwriters to write favourable journal articles, rigging study designs to produce favourable results, hiding unflattering results, failing to publish negative findings, promoting off-label drug use, giving bribes and kickbacks in return for promises to prescribe, and intimidating researchers whose results counter a company's interests. There is also little mention of shameless attempts by manufacturers to extend their monopolies, to block the production and sale of generic drugs, to put undue influence on the US Food and Drug Administration (FDA), to buy off large cadres of doctors, to promote drugs to treat social conditions, and to spend more money on marketing than on research - and, at the end of the day, to produce a shrinking list of truly innovative, clinically useful drugs."
( Dr. Kassirer doesn't leave much doubt as to how he'd answer the question posed in the book title , does he ? )
>> Quiz: What drug had an advisory-panel meeting set up before the NDA/BLA was even submitted? <<
Viagra , because the company said they would be handing out free samples ?
Beneficial health effects of exercise - the role of IL-6 as a myokine.
http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?itool=abstractplus&db=pubmed&cmd=Retrieve&...
Trends Pharmacol Sci. 2007 Apr;28(4):152-156. Epub 2007 Feb 28
Pedersen BK, Fischer CP.
Centre of Inflammation and Metabolism, Department of Infectious Diseases and Copenhagen Muscle Research Centre (CMRC), Copenhagen University Hospital, Rigshospitalet, University of Copenhagen, Faculty of Health Sciences, Blegdamsvej 9, DK-2100 Copenhagen, Denmark.
It is not clear how contracting skeletal muscles mediate the numerous and diverse metabolic and physiological effects that are beneficial for health. Researchers have searched for a muscle-contraction-induced factor - an 'exercise factor' - that mediates some of the exercise effects in other tissues such as the liver and adipose tissue. In our search for such a factor, we encountered the cytokine interleukin (IL)-6, which is produced by contracting muscles and released into the blood. We propose that muscle-derived IL-6 meets the criteria of an exercise factor and that such classes of cytokine should be named 'myokines'. The discovery of contracting muscle as a cytokine-producing organ creates a new paradigm: skeletal muscle as an endocrine organ. By contracting, it stimulates the production and release of myokines that can influence metabolism in tissue and organs. Newly identified myokines and their receptors could serve as targets in the treatment of metabolic disorders and other diseases.
PMID: 17331593 [PubMed - as supplied by publisher]
Re : Great article on DNDN/Provenge/Cancer
I agree , it was an excellent response and pretty much dissected Scher. It's a shame that they didn't make one author's text in bold , so it would have been easier to follow who was saying what.
Here's another response to Scher posted on iV today :
http://www.investorvillage.com/smbd.asp?mb=971&pt=msg&mid=1936164
EDIT : LOL. Ask , and thou shalt receive , or something like that.
Here's the reformatted letter by David , just posted on iV :
http://www.investorvillage.com/smbd.asp?mb=971&mn=82709&pt=msg&mid=1936692
Quiz : I'd guess that it's an ADHD drug , maybe Adderall or Concerta.
More on Ariad court battles
http://www.biopharma-reporter.com/news/printNewsBis.asp?id=75862
Ariad and Amgen in battle over biotech patent
By Emilie Reymond
18/04/2007- Ariad Pharmaceuticals has filed a counterclaim against pharma giants Amgen and Wyeth accusing the two companies of infringement of one of its patents.
The claim comes in response to a patent infringement lawsuit filed by biotech titan Amgen against Ariad 12 months ago.
In April last year, Amgen filed a offensive lawsuit against the Massachusetts-based firm in a bid to protect two of its products from the threat of patent infringement proceedings.
Amgen, the world's biggest biotech company, was seeking declaratory judgment that one of Ariad's patents - No 6,410,516 - was invalid and that it had not infringed any part of it with its arthritis drugs Enbrel (etanercept) and Kineret (anakinra).
The patent in question - which was awarded in 2002 and expires in 2019 - covers the methods of treating human disease by regulating NF-(kappa)B cell-signaling activity.
It is based on the discoveries made by research groups from the Massachusetts Institute of Technology, The Whitehead Institute for Biomedical Research, and Harvard University. Ariad is the exclusive licensee of the technology and patents all three institutions have joined the lawsuit alongside the biotech company.
"Our assertion of an infringement claim against Amgen and Wyeth highlights our strong belief in the validity and enforceability of our NF-kB patent," said Harvey Berger, chairman and CEO of Ariad.
Amgen's other arthritis drug, Kineret, is also named in the dispute.
In addition, Ariad included Wyeth as a defendant as the drug maker markets Enbrel with Amgen.
The patent has also been the subject of a previous lawsuit between Ariad and another drug maker, Eli Lilly, although Ariad said that the claims at issue against Amgen and Wyeth are different from those found valid and infringed by Lilly.
In the Lilly case last May, Ariad alleged that Lilly's septic shock drug Xigris (drotrecogin alfa) and Evista (raloxifene), a treatment for osteoporosis, both break its intellectual property (IP), and that it should get receive royalty payments. Lilly was claiming that the NF-(kappa)B signalling pathway is a natural biologic process and therefore cannot be patented. But Ariad won the case as a jury found certain claims of the patent were indeed valid and infringed by Lilly.
In addition to the $65m (€48m) in damages for previous sales, the jury decided Lilly had to pay Ariad a royalty of 2.3 per cent on future sales of Evista and Xigris.
If a similar scenario is replayed with this latest lawsuit, Ariad could bag itself a nice windfall as Enbrel generated $2.1bn in revenue in North America in the first nine months of last year.
>> I'd go a step further and argue that "major" short-position holders ought to have to be identified <<
I'd take it one step further and set up a web page with full-frontal photos of all naked shorts.
You'd need to have age-verification for site access , of course , to protect minors.
Re : Cancer Risks
Good find !
Full credit PLUS a gold star !
;)
Re : Quiz -- Ok , let's end this so you guys don't spend all day googling.
The answer is : AIDS.
The defect in adaptive immunity that is caused by AIDS causes huge increases in risk for viral-associated diseases , like Kaposi's sarcoma , and we were all aware of that , I'm sure.
More surprising is that the same mechanism confers a reduced risk for several cancers because of a dampening of the inflammatory response that can lead to cancer.
Transplant patients on immunosuppressives have a similarly reduced risk for certain tumor types.
I learned this from the webcast of the AACR presentation by de Visser , available on page 2 at this link :
http://www.aacr.org/home/scientists/meetings--workshops/annual-meeting-2007/webcast-sessions.aspx#
I recommend these webcasts -- I've listened to a couple and they're well done. Most are about 30 min , I think.
You can also find the quiz info in this pdf of a paper by de Visser :
"Paradoxical roles of the immune system during cancer development"
http://cancer.ucsf.edu/coussens/deVisser_et_al__NRC_2006+S.pdf
>> GHD? <<
Not the disease I'm referring to , but if you can show me the data , I'll give you full credit.
>> "some studies inversely associate statin use with breast and colorectal cancers" <<
Nope. In that case , it's the treatment for the 'disease' , hypercholesterolemia , that's associated with reduced risk. As another hint , think about the MOA of statin cancer risk reduction.
In the disease I'm referring to , it's the characteristics of the disease itself that confers the protection.
The statin stuff is interesting , although there's been some conflicting data. At one point it seemed like statins cured everything. ;)
>> If by risk you mean the risk of death, then pancreatic cancer fits the bill. <<
No. Risk of occurrence.
This is not a trick question , BTW . I'm talking about a reduced risk of occurrence per unit time for the diseased cohort vs. the overall pop.
Here's a hint : The answer is extremely counterintuitive.
Quiz : Patients with which disease have about half the relative risk of breast or prostate cancer , compared to the overall population ?
>>> They note that the Department of Veterans Affairs is allowed to negotiate and gets cheaper prices for some drugs than Medicare programs. <<<
Either the formulary / price negotiation system is the best way to go , or it's not. It can't be best for the V.A. and not best for Medicare , or vice versa.
Since Bush had 5 years of a lap-dog Congress to change the V.A. system , and didn't , it strikes me as illogical and a little odd to hear these arguments from him now.
Actually , not odd at all , considering the source.
OT : OK , stuck , you got me , but I bet it was not before you drew a blank on a PubMed search for alcapone. The guys on the COR board are probably still looking for references.
:)
AACR : DC vaccine for pancreatic cancer
( Another study that highlights the possibility of improving results of DC vaccine therapies by selective depletion of reg-Ts. )
http://www.aacr.org/home/about-us/news.aspx?d=740
Analysis of the immunological response to a MUC-1 loaded DC vaccine for human pancreatic cancer: Abstract 4896
Results from a Phase I study of a pancreatic cancer vaccine may offer clues toward promoting long-term survival from the disease, according to researchers from the University of Pittsburgh Cancer Institute.
The researchers gave a dendritic cell vaccine to 12 pancreatic cancer patients. Four of the subjects have shown no signs of recurrence in the three years since the study began.
"The trial was a look at the toxicity and feasibility of using a dendritic cell-based vaccine against pancreatic cancer," said Andrew Lepisto, Ph.D., post-doctoral researcher in the University of Pittsburgh's Department of Immunology. "While we are unlikely to run large-scale trials with this particular form of the vaccine due to difficulty in its manufacturing, we have learned a tremendous amount from the subjects that benefited from the trial, which may translate well into more practical vaccine formulations."
The dendritic cell vaccination strategy combines a cancer protein with the patient's own dendritic immune cells. These cells are antigen presenting cells that, in effect, advertise the presence of the antigen molecule to the rest of the immune system. The antigen, MUC-1, is a protein that is over-produced by pancreatic cancer cells. By presenting patients with MUC-1 on dendritic cells, the researchers expected that they could influence the white blood cells to attack pancreatic cancer cells.
The study data suggests that the key to the effectiveness of the vaccine could be in controlling the regulatory T cells, which suppress the immune system, says Lepisto. Prior to vaccination, the pancreatic cancer patients had significantly more regulatory T cells than normal, which then increased following each injection. Likewise, the patients also experienced an increase in effector T cells, white blood cells that respond against antigen.
"Our next step is to create a strategy that allows us to downplay the regulatory T cells while still benefiting from the increase of effector T cells," Lepisto said.
Each year, pancreatic cancer kills approximately 32,000 people in the United States alone. Pancreatic cancer is notoriously resistant to conventional cancer therapies and has one of the lowest five year survival rates of all cancers.
Possible ADHD approach you may find interesting
see :http://www.investorshub.com/boards/read_msg.asp?message_id=18855883
Nonstimulant ADHD candidate ?
I'm posting this link for Dew because I know he has an interest and because the link comes courtesy of one of Dew's favorite posters , nerdseeksblonde. ;)
"Tolcapone Improves Cognition and Cortical Information Processing in Normal Human Subjects"
http://www.nature.com/npp/journal/v32/n5/full/1301227a.html#bib15
P.S. They had previously discontinued research on an analog - alcapone- that triggered violent behavior.
Re : DNDN / CEGE
>>> The article described detailed analyses of the immune response to the immunotherapy in three out of 14 patients who were long-term survivors and who also demonstrated strong T cell responses to mesothelin, a tumor- associated protein found in the majority of pancreatic cancers and in the GVAX immunotherapy for pancreatic cancer cells. The specificity of the T cell response to mesothelin was shown to be unique to each responding patient providing further scientific proof-of-concept for the company's GVAX immunotherapy strategy. <<<
This puts CEGE infinitely ahead of DNDN in the MOA race. Was DNDN unaware of the SEREX technology and similar methods , or did they try everything and not find anything that made any sense ?
Information comparable to what CEGE just reported would have helped DNDN counter critics at the AC meeting. It's a real weak spot for DNDN.
>>> The agency (and ethics) virtually require the sponsor to build in a crossover component to the trial. If the crossovers then survive substantially longer than the non-crossovers, thus improving the control arm's performance and rendering the overall survival p value non-stat sig, then it's not fair to penalize the sponsor for running the trial in an ethical manner. <<<
I'd like to see a quiz on this , but the part I'd be interested in might not be answered honestly so it probably wouldn't work :
Question #1 : When the FDA requires a crossover design in oncology trials for ethical reasons , do you think they should also allow the use of statistical methods to correct the results of the trial for the adverse effects of crossover ?
a) Yes , obviously. The results are obscured by crossover , thru no fault of the sponsor , so common sense and fairness dictates that some allowance be made.
b) No , obviously. That's data-mining. Jeez!!
Question #2 - to be answered only by those who chose answer (b), above :
Do you now , or have you ever , made your living as a government bureaucrat ?
a) Yes.
b) No.
I'm not sure how question #1 would turn out , but the (truthful) results on question #2 would be 100% (a) , guaranteed.
>> The Epic trial that you point out is actually a success because it did it's job. <<
jbog,
I'm sure you're correct about that. I only picked those data because they were handy and fit well in the hypothetical situation I was creating. It would have been better if I'd called Erbitux " New Drug X " and called metastatic CRC " deadly cancer with unmet need Y " to make it a more generic argument.
>>> The hypothetical scenario you describe has some features in common with the Iressa case. And we all know what the result was there. <<<
Indeed. In the case of Provenge , there seems to be a concern that approval would result in a flood of junk BLAs , and attempts by pharma to game the system. The example I described shows that the surrogate marker approval process is tailor-made for gaming and junk submissions.
This discussion of efficacy determination is directly analogous to the one we had on safety issues associated with running combo trials of experimental HCV drugs. If the FDA is not consistently rigorous both before and after approval on efficacy and safety metrics , gaming of the system will occur , to the detriment of the drug development process. The decisions the FDA makes should be easily explainable to any reasonably intelligent person. Right now , each decision they make seems like a spin of the roulette wheel. A rigged wheel , at that.
Surrogates vs. OS : Once more , with numbers
The IMCL EPIC trial results released today provides a context for a thought experiment about oncology trial data.
Erbitux in metastatic CRC yielded the following results:
Surrogate markers:
PFS : p<.0001 ( WOW! )
Tumor response : p<.0001 ( WOW! )
Clinical benefit marker:
OS : p= 0.71 ( unwow , unwow )
Now , this was not a pivotal trial , but rather a trial for label expansion purposes on an approved drug. Imagine that it was , however. Lets say they were going for accelerated approval based on one or both of the above surrogates , based on prior P2 results which were excellent for those markers. They power the trial for the surrogate(s) , and they get p=.01 for both , say. They file the NDA , and we all know they get accelerated approval. The FDA mandates a P4 and they run a trial sized like the one above , 1300 patients , and get the p=0.71 for OS.
What to do ? Well , it's not really too clear is it ? Like most trials of this sort , the data was confounded by crossover. It appears that OS was significant if you compare crossovers to noncrossovers , but that's post hoc , exploratory , hypothesis- generating data-mining , right? Pull it from the market ? The docs think it helps some patients , the patients want access to it. A real dilemma.
The statistical purists would say that the accelerated approval was perfectly legitimate because the company should be allowed to market the drug while they wait for the OS results. If the OS trial had been done first , those same purists would say " No way ! p=0.71 ? Are you kidding ? " And I think the purists would be mixed , and certainly uncomfortable , about whether or not to pull the drug after the P4 failure.
Same drug , same results , just in a different order.
Here's what I think. I think the AA was reasonable. On the follow-on P4 , I'd look at the totality of the data , and mine it for all it was worth. Then I'd weigh the risk / benefit and might recommend a restricted label with a request for continued data collection , or I might ask them to pull the drug. Nothing in any rulebook would make this decision clear-cut because too many factors have to be weighed , not the least being the reluctance to deny access to a drug that both patients and docs want continued access to.
I can only hope that FDA folks have the ability to think beyond the rulebook and statistical tests , and apply some common sense in their decisions.
Re : response to Schers letter on seeking alpha
Nice job , walldiver et al.
Someone will write a book about all this one day.
Interesting times !
>>> the real SVR20 that would playout with larger study numbers could be assumed to be 0.666*0.799 = 0.532 ie 53.2% <<<
I don't think you can look at it that way. The only people who would stop tx. at 12 wks would be those neg at both 4 and 10 (or 12 , for our purposes here ) wks. Assume that the 70% who were neg at 12 wks were also neg at 4 , and use the 66.6 % SVR rate from group D , and you get .666 x .7 = 46.6%. Of course , that doesn't include SVRs from patients who would continue tx. past wk 12 after failing the cutoff criteria. That is academic though , since no SOC would stop tx. at 12 wks. with only a 66% chance of SVR , though many individual patients would , I'm sure. :)
EDIT :
JR beat me to the punch this time.
Re : DNDN
>>> Absolutely no immune response to a physiological antigen <<<
The anti-GMCSF ab response wasn't so bad. ;)
Only half-kidding about that part , but I agree that the MOA is MIA. Some tumors secrete GMCSF , and some people think GMCSF at the wrong time and place can promote a regulatory phenotype , so an anti-GMCSF response could be the proverbial pony in the crap pile. I would find that hilarious , if it turned out to be the case.
The DNDN stuff I've looked at reveals research that would get a person fired in any decent lab. Maybe they held back all the interesting stuff , like results for controls. The graphs of immune response seemed designed to confuse or hide something , or both.
That said , MOA is not important if the 3-yr survival results are anything close to being real , and I don't think they're smart enough to have pulled off a complete hoax.
The upreg of CD54 could be a hint of something , and if P11 shows significant effects on PSA velocity that could hint at something also. One reason I'd like to see it approved is because I think some smart researchers out there would figure out what's going on long before DNDN does.
>>> I say VRTX goes all out for SVR superiority in phase-3, come what may. <<<
Yes , no doubt. I forgot about the Boger Ego Effect.
:)
In one of the earlier VRTX studies , out of 12 patients on triple therapy for 4 wks., all of whom were HCV-neg at that time , 2 became positive when switched to SOC , and maybe a third who was lost to follow-up. So 17-25% became positive after switching to SOC. Also questionable from that study is the sensitivity of the assay used. I know they used 10 iu/ml for the on-VX part , but I've seen charts that suggest they might have used a higher cut-off for the follow-on part. I'd like to see more detail on that.
Now , things might not be as bad after 12 wks. of triple therapy as opposed to only 4 as above , but who knows ? Assume that about 15% of the 70% ITT who were neg at 12wks. become pos on SOC , and you're down to about 60% neg. Is that number likely to grow with continued SOC ? Probably not too much, it might even shrink further. Then you get the loss to relapse when tx. stops. You're left with less-than-heroic SVR rates.
An interesting question is whether the FDA would consider a non-inferiority trial of (triple therapy for 12 wks. plus 12 wks. SOC ) vs. SOC for 48 wks. I guess it would be a question of whether the safety detriment of adding a third agent for 12 wks. is less than the detriment of the extra 24wks. of SOC.
DNDN --- Warning : Myriad Posting Alert
>> The FDA already has trouble enforcing post-marketing trials, and if we leave it to big pharma, they're going to work at a snail's pace, and game their trials <<
This is what big pharma does right now with surrogate markers--- in early studies they look for indications and surrogate markers where they can reliably demonstrate a benefit , which is much easier and cheaper than if they're limited to looking at markers of real clinical benefit. It's front-end data-mining. They know they'll make big bucks after approval regardless of how the confirmatory trial comes out , assuming it's even done in the first place. The FDA is a strict arbiter of a travesty.
For anyone who has stayed awake thru a biochem lecture or two , the following will be obvious , but others may benefit by giving it some thought :
Every biological process or pathway is , directly or indirectly , related to every other , and to Kevin Bacon. For any marker of disease , say tumor volume , there are many , many pathways , with many intermediates in each , that might provide a target for a drug to effect shrinkage of that tumor. The problem is , because of the interconnectedness , that drug-induced tumor shrinkage can be accompanied by an earlier requirement for funeral arrangements. For each drug , you're never sure about the real benefit of surrogate marker effects until you confirm that benefit in controlled trials.
Everyone readily accepts HCV viral load as a surrogate for clinical benefit , but , as Thomas said , it's not so clear-cut. Many people live for years , even decades , with HCV without even knowing. They know immediately when they've been dosed with interferon plus ribavirin , and they wonder if HCV is really so bad after all. When they get ifn-induced autoimmune disease they really begin to wonder. We may be nearing the time when HCV nonresponders will go on lifelong anti-HCV therapy. That will be another travesty if there is not convincing evidence that there is a clinical benefit to doing so , for each new approach. If a trial is done showing that new HCV drug "A" is not as effective in suppressing virus long-term as new drug "B" , but that it improves survival ( not an endpoint , it was noted as "dropout due to death" ) compared to "B" or to no treatment , the FDA will grant full approval to "B" and will tell the makers of "A" to find a drug that suppresses virus better. Sorry , but that's just nuts.
Re : DCGN
I have a little but I must admit I don't keep up with what's going on with them. It looks like a good time to add more but I'm so bearish on the overall economy that I'm reluctant to add more of anything. Taking reasoned short-term bets on biotech 'binary events' has more appeal to me right now.
>>> So if a drug gets on the market because of its effect on a surrogate, then it is reasonable to provide a framework to allow the sponsor to test survival. <<<
And according to the argument advanced by yourself and Dew , the best framework to accomplish this would be a CU program while the confirmatory trial is ongoing , so as not to allow full marketing of drugs that may turn out to have no clinical benefit.
For the life of me I can't see what I'm missing here.
A surrogate marker trial success is a " hypothesis generating " result that has a reasonable chance of predicting a clinical benefit.
A close miss on a survival result is exactly the same thing , but with a better chance of ultimately demonstrating that benefit , because no assumptions have be be made about the connection between the outcome measured and the desired outcome.
>> If anything, today’s announcement was somewhat disappointing because of the high dropout rate. <<
It doesn't bode well for Prove 3 , either. If 11% dropped out knowing they faced only 12-wk. of the VX-950 regimen , how many will bail knowing they've got 24 wks. of tx. ahead ?
They could be looking at a 15% or more hit to ITT SVR rates right off the top in Prove 3 unless they get a handle on SE management.
Re : VRTX
I agree. Expectations were too high , including mine , and so results that are very respectable will probably be seen as disappointing by most.
It seems like the focus will be on 12 wks. of triple therapy followed by 12 wks. of SOC for the P3 , maybe with some arms with a somewhat shorter SOC phase. The fact that 6 of 9 who met the viral load hurdle at 4 and 10 wks., then went on to achieve SVR after stopping tx. at 12 wks. is still a major advance. After VX-950 is approved , they will no doubt develop viral load measurement algorithms that will allow certain patients to stop at 12 wk. with a high probablility of achieving SVR , similar to what is done now with SOC , which allows some to stop at 24 wks.
The hcv-neg data at 4 and 12 wks. is pretty impressive when compared to the SOC control arm data. This was expected , of course , but seeing it on larger numbers of patients on an ITT basis makes it more real.
A critical and as yet unanswered question is : Do you lose much of the benefit of VX-950 after the patients move on to the 12 wks. of follow-on SOC ? In other words , will the 24 wk. EOT viral loads look more like VX-950 data , or more like SOC data ? That data and whatever 12 plus 12 SVR data is available will be presented at AASLD , apparently , and should provide a pretty clear picture of what the P3 will yield.
A lot of focus will have to be put on managing SEs to prevent drop-outs -- 11% so far.
A 24 wk. tx. regimen will still make VX-950 a money-maker.
Biowatch , I think your commentary on the AC panel discussion really cuts to the chase.
What cancer patients are saying is that they want access , now , to treatments that are less-than-perfectly characterized , or those that might be statistically imperfect , as long as there is a reasonable chance of benefit and little chance of harm. The panel reflected that in a tempered way since they consider the issue from a more detached viewpoint and consider other things , like the possibility of wasting healthcare dollars on ineffective treatments , for example.
The important thing to remember is we're talking about patients with essentially no options. Taxotere is such a poor option that it is often refused. Nobody is suggesting lowering the standards for the next hot new laxative. If ten drugs like Provenge are approved and one eventually fails confirmation , I would consider that a huge overall success. Many more people would have been helped , sooner , as a result of this progressive policy and more would be learned about all ten drugs in a short time after approval than was known in total beforehand , as various researchers test their own theories on them. One of the FDA stat people originally estimated the false positive probability ( for survival advantage ) of Provenge at 1 in 40. In this situation , that's a slam dunk , IMO.
The CVA issue is a red herring. If I was a cancer patient I would be happy if I knew I was going to live long enough to have a chance of a stroke. If I had the stroke , I would look on it as an opportunity to learn a new language.
The CU issue is another red herring. It's a way for the FDA to blame the failure to allow access to the drug on the company. If it's safe enough and effective enough to allow CU , it's safe and effective enough for a conditional approval. It's not the patients' faults that the FDA hasn't figured out yet how to do a conditional approval. It's about time they do.
>> It is unlikely that Dr. Scher's participation in the discussions on March 29 of a cellular therapy for prostate cancer will have a direct and predictable effect on his financial interest <<
Who the (DELETED) do they think they're (DELETED) kidding ??
>>> There is no evidence that CVA is a side effect of Provenge seperate from being a "side effect" of living longer in this patient population. <<<
Someone please tell me that the FDA routinely considers the effects of increased survival on the numbers of AEs ; for example ,that they would look at the # of events per patient-year.
If not , we're all wasting our time discussing this. Science is not involved in the process. A huge bank of brain fog has moved downwind from the White House , enveloping the FDA , and all hope of rational thought taking place there is lost.
>>> I was referring to your not mentioning a CU program in any of your myriad posts on this topic. <<<
A CU dictate would only be logical if it was also mandated in place of surrogate approvals , a point that should be obvious from my myriad posts on this topic.
Besides , that's a solution that solves nothing , because DNDN couldn't possibly manage giving away a couple hundred million dollars/yr. for the next 3 yrs. until 02b is done , and that's about what it would take if all eligible patients requested compassion.
Try this :
http://www.investorvillage.com/smbd.asp?mb=971&mn=76393&pt=msg&mid=1899788
Post 76393 on iVillage DNDN.
>> You seem to be trying really, really hard to avoid thinking about this. <<
I've expressed the opinion that there appears to be no easy way for the FDA to do what I think should be done , in this and similar cases. As I said before , they may need some new rules in the rulebook.
In this case , I think they have all the leverage they need with DNDN to assure completion of 02B , since DNDN will be going to them for label expansions , new indications , etc. They don't need a legal hammer. If 02B fails they will deal with it.
Again , though , the comparison with FDA action on surrogate approvals would show that the outcome with Provenge could not possibly be worse that what routinely occurs now : no confirmatory trial completion ten years after approval , confirmatory trials that are enrolling a dozen patients a year , failed confirmatory trials , etc.