Drug Development for Dummies

11/16/2007 09:36 AM |

Jonathan Cohn, in an otherwise excellent and thoughtful piece in The New Republicv on how to shift health care systems to a value based reimbursement approach (from a single payer perspective) , resorts to the shibboleth that drug companies are not very innovative as if this is somehow their fault and by design. He relies on the totally useless work of Marcia Angell and Merrill Goozner, neither of whom know much about drug discovery and development and what they do write is selective and misleading.

First, if drug development and discovery were so easy it wouldn't be so expensive. Success rates have fallen not be design. Would any one want to invest $800 billion in a drug only to have it fail. But it happens all the the time. Incrementalism by the way is the norm of science and all things whereas the ability to use one medicine or insight to transform or extend life is rare indeed. Yet Cohn falls into the trap all amateurs fall into and believes that if drug or biotech companies tried a little harder they could nail it. That's arm chair quarterbacking at it's worst. If you think it's so easy to hit a Jaba Chamberlain splitter, why don't you try out for the Bosox and hit it not once every 1000 times but one out of three times. Hit .300 and you are in the Hall of Fame. My brother has worked for a drug company for 20 years and has 3 of the drugs he has worked on approved. That's a lot.

Now as to the heavy lifting. This notion that NIH discovers everything is just not true. The amount of collaboration is astounding and is what separates the US from other parts of the world. Angell and Goozner lie outright to avoid describing the partnerships that shape innovation in America and depict a Golden Age that never existed. Angell asserts that every breakthrough drug started without drug company involvement. She claims that Gleevec, the first cancer drug to target cancerous cells without side effects, was developed without any real input from Novartis, the company that makes the product. Angell says that Brian Druker, a cancer researcher at Oregon Health and Science University, said that Novartis showed little interest in the cancer compound until he discovered its tremendous properties. The real story--from the Journal of the National Cancer Institute--reflects the risky and collaborative nature of drug development, which requires massive capital and biopharmaceutical know-how to turn discoveries into effective treatments. An academic researcher and private company, working together, launched a revolution in the treatment of cancer. You wouldn't know it by reading Angell.

Goozner makes the same claim about drugs developed by Amgen. Everyone involved knows better.

Similarly both Goozner and Angell disdain the revolution in personalized medicine thereby ignoring genomic based science, something that Senator Obama has taken leadership on in the Senate along with Senator Richard Burr.

For instance, The Truth About the Drug Companies also claims there is no real evidence that any one drug is better than another or that most medicines really do much at all. And Angell goes as far as to say: "the idea that patients respond differently to me-too drugs is merely an untested and self-serving hypothesis." Rather, she says, "one or two drugs will do" for most medical conditions. And Goozner still can't figure out the difference between a surrogate end point like blood pressure and a genetic marker which both predicts and controls disease progression.

Cohn's article in the New Republic tries to argue that value based reimbursement can let people have the best of both worlds in a single payer system. I disagree. But he gets the point that health care is an investment and that innovation is valuable. I would recommend that he go beyond Goozner and Angell to understand the role the private sector plays in promoting innovation and just how difficult real invention is.

http://www.tnr.com/politics/story.html?id=53e206dd-c286-43b1-9c5b-079e81ab3474  Read More & Comment...

Compulsory Hypocrisy

11/16/2007 09:07 AM |

The recent session of the WHO’s Intergovernmental Governmental Working Group (IGWG) ended without consensus. And that’s a good thing considering that many of the issues on the table would have resulted in a screeching halt to medical progress.

The IGWG discussions were completely void of innovators -- with pharmaceutical researchers relegated to the sidelines. In their place were activists who are unwilling (and seemingly unable) to engage in any discussion that does not begin and end with removing systems of intellectual property (IP) protection for medicines.

As with many of their ilk, these activists believe in freedom of speech – as long as what you say supports their position. Otherwise you’re a capitalist tool. Their grasp on the truth is, well, questionable.

Consider this statement from our comrades over at Médecins Sans Frontières:

“Patents are not a relevant factor in stimulating R&D and bringing new products to the market.”

Really? What about HIV/AIDS, one of the WHO’s “neglected diseases,” where all of antiretrovirals currently in existence were developed under patent protection.

If their grasp on the truth is questionable, their chutzpah is unlimited.

Consider a recent editorial the latest publication of The Lancet where officials of the Thai military junta attack comments offered by patient organizations and other groups to the open forum of the IGWG.

“The issue that attracted the most responses was intellectual property (IP), which was cited in 43 of 68 submissions. Although we were not surprised to see that 11 of 12 organisations directly affiliated with the pharmaceutical industry supported strong IP protection, it was surprising that 14 patient advocacy groups took a similar position, which in several cases was the only point raised in their submissions; three professional associations also took similar positions … We declare that we have no conflict of interest."

This last comment is made after they state that, "We strongly suggest that contributors to public hearings must disclose any conflicts of interest, as required of authors submitting papers to peer-reviewed journals."

The authors did not declare that the Thai Ministry of Health owns the Government Pharmaceutical Organization (GPO) -- and that the GPO stands to significantly benefit financially from a proposed IGWG agenda strongly supports compulsory licensing.

Well, what’s good for the goose is good for the gander. Transparency should cut both ways – even in Geneva.  Read More & Comment...

A HIPAA-SIzed Problem With Patient Reported Outcomes

11/15/2007 10:23 AM |

Ed Silverman, the brains behind pharmalot ( www.pharmalot.com) has written a great piece on the trend towards patient reported outcomes in clinical trials. Must reading on this subject for anyone interested in personalized medicine and understanding how and why FDA yanked the QOL indications from Amgen's anemia drugs.

http://www.the-scientist.com/article/home/53617/

Now it seems that accessing this data can often be a problem for the researchers who want to use this research to find out what works. An editorial in JAMA by Norman Frost of the Univ of Wisc. Medical School writes that application of HIPAA rules has shut down research or scared off otherwise important studies in the name of privacy:

"The sources of these problems include Office for Human Research Protection and the FDA because they appear to threaten institutions with draconian penalties for minor infractions; institutional (university and other) administrators acting out of fear that their institution could be the next to have its entire research operation suspended by 'getting caught' in one of these minor infractions; and credentialing and certifying agencies for supporting these excesses by including them in their criteria for accreditation."

http://www.sciencedaily.com/releases/2007/11/071113165648.htm  Read More & Comment...

USA Today Adds Its Own Avandia Warning

11/15/2007 09:10 AM |

Here's USA Today crawling through the mud -- past Janet Woodcock who officially speaks for the FDA -- to talk to David Graham about Avandia:

"This is a warning that's not a warning," said Graham, speaking for himself. "The other thing that's missing is a statement that … there are other alternatives available that work as well as Avandia and don't have this cloud hanging over them."

So Graham wants the FDA to engage in comparative effectiveness which is clearly not it's statutory authority and for which there is no evidence anyways that could be slapped on a label. USA Today runs with this statement instead of the scientific one made by Dr. Woodcock based on the evidence.

No wonder patients are confused and afraid.

http://www.usatoday.com/news/health/2007-11-14-diabetes-heart_N.htm?loc=interstitialskip  Read More & Comment...

FDA's Final Word on Avandia

11/14/2007 10:01 PM |

Forget the black box for now..here's FDA's Janet Woodcock speaking clearly and confidently on what the agency decided and what the new labeling means:

"We are keeping Avandia on the market because we have concluded that there is not enough evidence to conclude that the risk for heart attack or cardiac ischemia is higher than for other type-2 diabetes drugs," the FDA's Dr. Janet Woodcock told reporters in a phone briefing.

The studies have been inconclusive and those that appear to show a risk all compare Avandia to a placebo, she said."

Thus the FDA is closer to the European Medicine Agency than Canada. I am keeping a count to see how many news accounts or blogs note that the EMEA kept Avandia on the market without relabeling. Big zero so far.

http://www.reuters.com/article/governmentFilingsNews/idUSN1421392120071114  Read More & Comment...

Prize Turkey

11/14/2007 02:32 PM |

John Edwards must know something -- like his campaign is dying. Why else would he decide to jump on the bandwagon of Bernie Sanders (the Senator from Ben & Jerry's) and come out in support of the year's worst idea in health care reform -- prizes rather than patents.

Here's what Senator Edwards had to say to a group of medical professionals at Dartmouth-Hitchcock Medical Center on the subject of prizes for pharmaceutical innovation:

"It would also create a different dynamic for drug companies and particularly for breakthrough drugs in big areas like Alzheimer’s, cancer, etc. We’d offer a cash prize for the research and development of these drugs, but they don’t get a patent. So we eliminate the monopoly…The idea is you’ve got to give the financial incentive for the companies to do it but on the flip side you get the products to the market quicker, available quickly and at a much lower cost.”

Okay -- once more with feeling -- this is just not true. As Joe DiMasi (Tufts University) and Henry Grabowski (Duke University) have argued, under a prize program, pharmaceutical innovators would lack the incentive to innovate. To quote DiMasi and Grabowski, “The dynamic benefits created by patents on pharmaceuticals can, and almost surely do, swamp in significance their short-run inefficiencies.”

For more reasons this is a crackpot idea see our October 22nd blog entry ("Et tu, Bernie") here:

http://drugwonks.com/2007/10/et_tu_bernie.html

Note to Senator Edwards: You should look into how much money is currently being spent by the biopharmaceutical industry on Alzheimer's research and development. (We assume you have already done so on the issue of breast cancer.)  Read More & Comment...

Improving Health Care Quality and Value

11/14/2007 07:01 AM |

On March 29th, CMPI and Old Dominion University sponsored a conference on evidence-based medicine and its implications for a cost-centric vs. patient-focused health care future.

Speakers included Carolyn Clancy (AHRQ), Gail Wilensky (Project Hope), Scott Gottlieb (AEI), John Bridges (Johns Hopkins), Peter Elkn (Mayo Clinic), among others.

Here is the final report on the event -- and it's really must reading for anyone concerned about the precarious balance between value and quality -- in short, the future of the American health care system.

Here's a link to the final report:

http://cmpi.org/archives/2007/11/improving_health_care_quality.php

Here's an enticing tidbit from the conclusion of the report ...

"We're not at the end of this debate. We're not at the beginning of the end of this debate. But we are at the end of the beginning where at least we can all agree that this is not, and must not be exclusively a debate about saving money. It must be about patient care."

An important read.  Read More & Comment...

Waxman's (non) Inferiority Complex on MRSA

11/13/2007 10:14 AM |

MRSA slowing spreading to the community. No drugs in the pipeline in large part to Congressman Waxman's jihad against using clinical trial designs that have brought HIV drugs to market to accelerate approval of medicines for MRSA that kill more people than HIV. Waxman's response? Read his sanctimonious speech on infectious disease control which essentially urges everyone to wash their hands.

http://oversight.house.gov/story.asp?ID=1606

Read this article on how Waxman's attack has helped kill investment and scared the FDA into inferior antibiotic drug approval rates.

http://www.morgenthaler.com/.../Ventures/Articles/  Read More & Comment...

Where are all the dead Canadians? Quadra Island for starters.

11/13/2007 04:24 AM |

People who rob banks only steal money. And they only steal it once. But when intellectual property is stolen, the rip-off is ongoing -- and the results can be deadly.

Today, one of the biggest rip-offs going threatens to cripple the U.S. pharmaceutical industry, which faces unprecedented losses from the theft of intellectual property via the mass production of copycat drugs. These drugs are manufactured in places like China, the former Soviet Union, the Philippines and other places where the U.S. Food and Drug Administration has no effective oversight authority — and where the safeguards designed to protect public health taken for granted in America are virtually nonexistent.

Amazingly, some lawmakers in Congress don't recognize the extent of the threat, and are pushing legislation that would make it legal to import prescription drugs from abroad.

Such a measure would lead to a deluge of copycats entering the legitimate supply chain, since it would be virtually impossible to track what's real and prevent phony medications from entering the U.S. market.

Senator Bernie Sanders (I, B&J -- Ben and Jerry's) is fond of taunting the opponents of drug importation by asking, "Where are all the dead Canadians?" Unfortunately, the most recent answer to this question is -- Quadra Insland.

Earlier this year in Canada, a woman who purchased Xanax and Ambien from what she thought was an online Canadian pharmacy died after taking her pills. It turns out the drugs were fake — and were filled with aluminum, tin and even arsenic. They had actually been purchased from a Web site in the Czech Republic and produced in Southeast Asia. But because the medication, in all likelihood, came with labeling and packaging that was indistinguishable from the real thing, there was no way for the victim to know her pills were contaminated with dangerous impurities.

Toxicology tests found three well-known drugs in her system: alprazolam -- more commonly recognized as the anti-anxiety drug Xanax; zolpidem -- which most are acquainted with as the brand-name sleeping pill Ambien; and acetaminophen. Zolpidem is not available in Canada, so it's understandable why Bergeron turned to the internet to try to get the drug.

As the prevalence of counterfeit drugs increases, the long-term viability of legitimate pharmaceutical companies is threatened.

Estimates vary, but the cost of developing a new drug ranges from a low of around $800 million to as much as $2 billion. Much of that investment goes to cover the cost of clinical trials to assure the new drugs are safe and to meet the federal government's many regulations.

Copycats, of course, don't have to worry about any of that; they just take the proverbial ball and run with it — straight to the bank. In fact, it's estimated that counterfeit drug sales will account for $75 billion globally by 2010. According to a recent article in U.S. News & World Report, the counterfeit drug trade is already worth some $35 billion annually.

Anyone who doubts the effect such losses have — both in real terms today and in how the American pharmaceutical industry chooses to invest its dollars and its human talent tomorrow — must have slept through Econ 101.

Here's the rest of the story from today's edition of the Florida Sun-Sentinel:

http://www.sun-sentinel.com/news/opinion/sfl-copycat13forumnbnov13,0,2801251.story

Opening the floodgates to imported drugs of unknown origin is a terrifying proposition. It poses a real risk today, and it threatens our health and well-being tomorrow  Read More & Comment...

ADHD Drugs Don't Work In All Kids: Media Engages in Fearmongering

11/12/2007 12:46 PM |

Here's the straight story:

Preschoolers With Three or More Coexisting Disorders Show No Response to ADHD Medication Treatment

BETHESDA, MD -- November 5, 2007 -- Preschoolers who are diagnosed with attention-deficit hyperactivity disorder (ADHD) are not likely to respond to treatment with the stimulant methylphenidate, regardless of dosage, if they also have three or more coexisting disorders, according to a recent analysis of data from the Preschoolers with ADHD Treatment Study (PATS).

Previously reported PATS results showed that overall, low doses of methylphenidate were safe and effective in treating 3-5-year-olds diagnosed with ADHD.

This most recent study, one of seven new PATS articles published November 5, 2007, in the Journal of Child and Adolescent Psychopharmacology, sought to identify individual characteristics that may affect how a child would respond to treatment. The other articles examine topics such as the effectiveness of methylphenidate over a follow-up phase, the effects of the medication on functional outcomes for the preschoolers, and others.

"This new data is an important step forward in bridging the gap between research results and clinical practice, bringing potentially valuable information to clinicians about ways to better customize treatments for their patients," said NIMH Director Thomas R. Insel, MD. "It also identifies a group of young children who have significant and multiple problems, and for whom more research is needed to identify effective treatments."

http://www.docguide.com/news/content.nsf/news/852571020057CCF68525738A0075CA73

Here is how the media mangled and engaged in fear mongering right off the bat:

BBC

Drugs for ADHD 'not the answer'Treating children who have Attention Deficit Hyperactivity Disorder (ADHD) with drugs is not effective in the long-term, research has shown.

FoxNews.com

" Drugs used to treat attention deficit hyperactivity disorder (ADHD) have no long-term effectiveness and could stunt your child's growth, News.com.au is reporting.

And scientists have conceded that test results that prompted the parental craze to dole out the drugs to their kids, may have been exaggerated.

In what is sure to generate debate, BBC's Panorama program Sunday aired the results of a long-term monitoring program of 600 children across the U.S. since the early 1990's.

The Multimodal Treatment Study of Children with ADHD study concluded that while drugs such as Ritalin and Concerta worked in the short term, there was no demonstrable improvement in children's behavior after three years of medication."

FoxNews.com uses the BBC as the authority! Parental craze...not too much editorializing.

Disgusting.

http://www.foxnews.com/story/0,2933,310596,00.html

http://news.bbc.co.uk/2/hi/uk_news/7090011.stm  Read More & Comment...

Who Overstated the Risk of Drug Coated Stents??

11/12/2007 09:40 AM |

A year after safety questions about drug-coated heart stents prompted doctors to change treatment for hundreds of thousands of cardiac patients, many physicians say the medical community overreacted and should now reverse course.

The alarm was caused by medical studies suggesting that drug-coated stents might be causing deadly blood clots. But with benefit of additional data and further analysis, many doctors say drug-coated stents may not be so risky after all, at least compared with various alternatives whose drawbacks may outweigh the risks of clotting.

Because the safety fears were widespread, however, even those rooting hardest for a rebound — the companies that make stents — are not expecting a quick resurgence for the drug-coated devices. Worldwide, stent sales have fallen by about $1 billion since last year, to $5 billion this year."

The alarm was not caused by the studies but by media accounts that ignored dozens of other studies and the absolute risk of death followed by trial lawyers fanning flames of fear...

From an Oct 21 2006 article by Feder

Doctors Rethink Widespread Use of Heart Stents

..."But now stent sales are falling and some doctors are rethinking their faith in the devices, driven by emerging evidence that the newest and most common type — drug-coated stents — can sometimes cause potentially fatal blood clots months or even years after they are implanted.

The Food and Drug Administration said yesterday that it would hold hearings in early December to consider whether to issue new stent safety guidelines.

The evidence indicates that overuse of stents may be leading to thousands of heart attacks and deaths each year, whether because stents are being used in relatively mild cases where drugs should be prescribed instead, or because patients are receiving drug-coated versions where simpler, cheaper bare-metal devices might work just as well.

There is no question that stents have saved countless lives in the short term by preventing impending heart attacks or opening arteries while an attack is being treated. But neither type of stent, no matter how much better it may make a patient feel, has been shown in rigorous clinical trials to improve long-term survival compared with other forms of treatment.

“In the past we’d say, ‘Why not?,’ ” said Dr. William O’Neill, a well-known cardiologist at the University of Miami and longtime advocate of using drug-coated stents. But the new safety data, he said, amounts to “a big why not” for many patients.

The new evidence has added to a long-simmering debate over whether doctors have been too quick to prescribe stenting — whether because drugs would work as well for healthier patients or because bypass surgery might help the sickest ones live longer."

http://www.nytimes.com/2006/10/21/business/21stent.html?ex=1319083200&en=0ad3debe72a4dc90&ei=5088&partner=rssnyt&emc=rss

http://www.nytimes.com/2007/11/12/health/research/12stent.html?n=Top/Reference/Times%20Topics/People/F/Feder,%20Barnaby%20J.  Read More & Comment...

Pitcher-Centric Medicine

11/12/2007 07:11 AM |

Advising policy-makers, taking on pundits, and fighting the good fight for patient-centric medicine is one thing -- getting quoted in Sports Illustrated is something else. Here's story that talks about how one-time Yankee great Mel Stottlemyre has had his life returned to him because of the miracles of modern medicine.

'Strong as a horse'
Treatment allows Stottlemyre to return to baseball

SEATTLE (AP) -- Mel Stottlemyre was getting his white blood cells counted for yet another month. Unsolicited, his doctor said he could go back to full-time work again, back to the ballpark routine he had followed just about every summer for 41 years.

Then came another unexpected offer.

Stottlemyre returned to the major leagues this week as the pitching coach of the Seattle Mariners, accepting the first of what he hopes is a series of one-year contracts. It's the only job that could get him back into a dugout.

"I certainly hope it lasts for more than one year," Stottlemyre said. "Whatever happens, at my age and certainly with my health issue, I'm excited for the opportunity."

The 65-year-old former pitching coach for the Yankees and Mets got cleared to return to the bigs during a visit to his Seattle-area doctor this summer. Just as she had each month for years, the doctor told Stottlemyre his blood-cell count would allow him resume the three-weeks on, one-week off pill cycle he takes to combat multiple myeloma.

But this time, she surprised the old right-hander-turned-fisherman.

"You have no restrictions to go back to work full time, if you want," she told him a few months ago.

"I wasn't even looking for that," Stottlemyre said Monday night.

The former five-time All-Star with the Yankees left them in 2005, after 10 seasons and four World Series titles as New York's pitching coach. He said he was tired of criticism from owner George Steinbrenner. Raised in Mabton, Wash., he returned to his home in the Seattle suburb of Issaquah and interviewed that fall to become manager Mike Hargrove's pitching coach with the Mariners. Hargrove chose relatively inexperienced Rafael Chaves instead.

Stottlemyre dabbled in spring training and instructional league work with the Arizona Diamondbacks last year. He golfed, fished and helped his son Todd, one of two sons who also pitched in the majors, begin his new career as a financial adviser.

One of the first clients he lined up for Todd was John McLaren, the Mariners bench coach who became manager when Hargrove abruptly resigned July 1.

The dividends from that arrangement arrived this month. McLaren called to ask Stottlemyre to replace Chaves, after Seattle's starters had a 5.12 ERA this past season -- 12th in the AL. They were the main reason for the remarkable September collapse that doomed Seattle's unlikely contention for a playof spo.

"Mel was my No. 1 choice," McLaren said. "His reputation speaks for itself."

Stottlemyre was content fishing and golfing.

"I wasn't really anxious to get back into the game -- until the Seattle job came open," Stottlemyre said. "I've always wondered what it would be like to be in baseball but still be able to come home every day."

McLaren had been golfing with Stottlemyre over the last year and noticed that his friend's health was not an issue.

"I sure wouldn't want to mess with Mel. He's strong as a horse," McLaren said.

Stottlemye credits the cancer drug lenalidomide, marketed under the brand name Revlimid, for repelling his disease. Revlimid isn't for everyone. Its retail price can reach $6,400 a month, according to the Center for Medicine in the Public Interest.

"It's very expensive, but it's very effective," Stottlemyre said. "With the type of cancer I've had it's something where they never use the word 'cure.' Right now, it's not curable. It's treatable.

"I'm doing absolutely super. I have no signs of the disease. I wouldn't call it a 'remission' so much as I would say that I'm on a tremendous maintenance program."

Stottlemyre will have a far different pitching staff than the one he enjoyed with the Yankees. Instead of tutoring the likes of Roger Clemens, Mike Mussina and Andy Pettitte, he'll be demanding that Seattle's 21-year-old ace Felix Hernandez plus veteran holdovers Jarro Washburn and Miguel Batista pitch inside more -- something Stottlemyre focuses on. He also will advise McLaren and general manager Bill Bavasi as they seek another veteran starter this winter.

"At first glance, I see a very challenging job," Stottlemyre said of his new group of pitchers. "I hope that I have something to add to each that will help each one."

Talk about personalized!  Read More & Comment...

The Final Facts on Rudy Cancer Ad: Death Rates, Not Survival Rates

11/09/2007 02:48 PM |

I guess I shouldn't be tossing numbers around after being flagged for bad math just a few minutes ago. But you still have to take a turn at bat even after a strike out, so here goes.

Everyone is missing the point on the UK-US cancer comparison because everyone is too lazy to do real research. Which is how this fight over facts instead of philosophy started in the first place. If anyone had bothered to check with me -- and they didn't -- I could of told them straight up that five year survival rates can be tinkered with and explained away but age-adjusted death rates by cancer and by stage...well, they don't lie. But you actually have to do a little work with the numbers, which most health care policy wonks don't do. They regurgitate or pick and choose the facts that fit their perspective. Right and Left.

Survival rates do have some value in measuring differences.

Survival rates for lung, breast and prostate cancer – which means your chance of being alive five yeas after being diagnosed with cancer has increased more rapidly in the US for lung, prostate, breast and cervical cancer. Breast cancer five-year survival rates are higher and death rates are lower for women in the US regardless of age or stage of cancer. The age adjusted survival rates for lung cancer in the UK is about 8 percent, half that of the United States.

But often there are holes in survival rates because of time lags or gaps in regional reporting. The recent survival rates are a case in point. The UK doesn't include them for England and Wales. Just Scotland and Ireland. The Lancet study -- which uses old data too -- says it does not have age adjusted five year survival rates for those two important part's of Her Majesty's sinking healthcare system. But the UK does have year to year death rates for major forms of cancer by age. And boy do they stink.

Death rates from prostate cancer in America declined an average of 4 percent a year from 1994 to 2004 while they increased by 1 percent a year during the same time frame in England and Wales. And for man 65 or over, while the death rate from prostate cancer also declines about 4 percent year – regardless of race in America, men of the same age in England saw their chances of dying climb by 20 percent.

Screening rates climbed at about the same rate in both countries. So the difference is explained by access to medicines. For instance, NICE refused to approve reimbursement for Taxotere for men with prostate cancer that were failing to respond to hormonal treatment that improved survival. Taxotere not only was shown to extend life by up to a year but also reduce in pain and fatigue of those with no chance of cure to maximize their remaining time with their loved ones. It took months of lobbying from doctors and patient groups until NICE approved cleared its use in June of 2006. Yet today many NHS health trusts refused to cover it because it is not “cost effective.” In America, Taxotere is standard of care.

You can't find the death rate stuff just anywhere. You have to dig for out and run the numbers yourself. Easier when you don't have a real job like David Gratzer who is a real doctor and treats patients full time in Canada. Unlike the dishonest folks at Commonwealth who have billions to go with plenty of free time. And unlike Paul Krugman who gets paid to dump on people like David who has done more in a single day to advance healthcare than Krugman's attacks on anyone who doesn't agree with him has ever done.

Gratzer was making a point. If anything the death rates show that he was probably being conservative in his claims. He should be praised for being restrained and even more so for being willing to correct the record so publicly. The same cannot be said for his critics.

A recent article in Britain’s Daily Mail makes the same point the Giuliani ad tried to get across: “I won't let Daddy die: Girl of six raises £4,000 for life-saving drugs the NHS won't provide” When Britain’s National Health System said it would not pay for Tarceva, the drug her father needed to fight lung cancer, six year old Chantelle Hill put up posters throughout her neighborhood asking for donations so she could buy the drug herself.

Tarceva is not a cure but it does extend life and improve quality of life. And it’s use widely by cancer doctors in America. But Britain’s National Institute for Health and Clinical Excellence (NICE) – which evaluates what the NHS should pay for -- found it was not cost effective.

The fact is, cancer patients in the UK do worse than they do in America not because of statistical manipulations by the Giuliani campaign but because cancer care in Britain is rationed out of cost considerations.

Chantelle Hall is going door to door in England to raise the funds she needs to buy the drug keeping her father alive because her health system thinks it isn’t cost effective. Rudy Giuliani ran an ad essentially making the point that he is glad America has a health system that, while in need of change, doesn’t force kids to make that choice on a regular basis. His numbers might be off but his heart was in the right place. So is David Gratzer's. The same can’t be said for his critics.  Read More & Comment...

Affairs of The Heart: Continued

11/09/2007 11:29 AM |

Which of these is likely to be the per client amount individuals who entered the Vioxx litigation sweepstakes are going to get:

a. $1million
b. $400k
c. $75000
d. $5300

If you picked d) you get a tube of John Edwards' hair gel. That's right. Take the $4.8 billion Merck will fork over, give half of it to all those well meaning trial attorneys and then divide the rest among the 45000 plaintiffs.

CORRECTION: My friend, and consigliere Paul Windels nails me on the math, my estimate of the tort take and my cheap calculator that only runs into the hundreds of milions and wins a case of hair grout with the following email...

Bob -- I think you have a decimal point error here. If there are 45,000 plaintiffs, $5,000 per plaintiff is around 225 million. Your number is correct if there are 450,000 plaintiffs -- on the other hand any plaintiff with a decent case would opt out of the settlement if it were only worth $5,300. I would expect a fee max 33% meaning $3.2 billion net, which would be around $70K per, a much more attractive number. Cheers. Paul.

Did Merck ignore safety signals of Vioxx in a way that made it liable for people dying. No. There are lots of safety signals. Are there better ways to figure out which ones reflect real risk. You bet and that means not having to wait four years to find out about them, which was the point of Eric Topol's original article about Vioxx. Are we any closer to knowing how to manage the risks of COX-2s because of the grandstanding and litigation? No. Could Vioxx been remarketed and sold with tighter restrictions in a saner, less politicized environment. Yes. What will help patients more, better monitoring of risks and benefits of meds post market using 21st century science or a system gripped by fear and controlled by trial lawyers and self proclaimed consumer advocates who dredge data for danger?

On a lighter front, Crestor just got a label for atherosclerosis, a label which simvastatin generic Zocor, Stephanie Saul's drug of choice does not have. Simvastatin also seems to cause sleep problems in some patients which I am surprised Stephanie didn't know about since she is the NYT in house expert on sleep.

I hearken back to her article about Pfizer trying to maintain market share in the face of simvastatin competition and the firm trying to use the fact that patients respond differently to different drugs as a way to keep more people on Lipitor if its good for them in the long run. How about this as a proposition: not what's cheapest, but what is best and has the fewest side effects given our specific health needs. As we move towards personalized medicine those will be the claims that count. Or is that marketing too? In a politicized environment our choice is as follows: A drug that is marketed by drug companies is bad even if it is personalized. A drug dispensed according the musings ofJerry Avorn or some researcher who ignores individual differences in drug response to justify the cheapest (the Soros funded approach of the Institute for Medicine as Profession)..that's great.  Read More & Comment...

And not one cent for tribute

11/09/2007 09:46 AM |

By now you will have surely have heard about Merck's $4.85 billion Vioxx settlement. That comes out to something more-or-less like $100,000 per plaintiff -- and not one cent for tribute to trial lawyers.

To paraphrase -- a billion here, a billion there and pretty soon you're talking about a settlement. My sources confirm that Merck had been spending about $1 billion a year on Vioxx litigation -- and, with no end in sight, $4.85 billion to make it go away is the smart fiduciary play.

That being said, it must stick in the craw of the folks at Merck to pony up this kind of do-re-mi for having done nothing wrong -- money that could (and should) have been spent on R&D.

If anything positive can come out of this travesty it's this -- Merck stood up for what it thought was right, won more than they lost -- and showed the trial bar, pundits, and pols that they weren't going to be doormats for cheap shots and SiCKO soundbites.

And that's a valuable lesson for the rest of the Big Pharma brethren.  Read More & Comment...

Moore-ish Behavior

11/09/2007 07:55 AM |

In case you missed the news, SiCKO is now out on DVD – and it seems that New York Times columnist Paul Krugman made a beeline to Blockbuster.

Call it an early Hanukah present for a true believer.

His column, “Health Care Excuses,” replays the same tried and untrue arguments made in the Moore-ish cult classic. Here’s a link:

http://www.nytimes.com/2007/11/09/opinion/09krugman.html?ref=opinion

Looks like he read our November 5th blog, “Debunking Some Health Care Urban Myths" --

http://drugwonks.com/2007/11/debunking_some_health_care_urban_myths.html

-- where we take many of the more infamous Moore/Krugisms to task.

Well, at least he's reading -- if not learning.

(And we'll keep trying.)  Read More & Comment...

CMS Stands Alone on ESA Dosing

11/08/2007 02:29 PM |

ASCO/ASH, ACCC, EMEA and now the FDA all say the following with respect to ESA dosing

"The dosing recommendations for anemic patients with chronic renal failure have been revised to recommend maintaining hemoglobin levels within 10-12g/dL."

CMS stands alone -- along with insurers who jumped on board to save money -- as the only government agency to limit access based on safety.

Why does CMS think that ESAs are less safe at anything more than 10g/dL when everyone else thinks otherwise? Why does CMS think it can limit doctor discretion?

Here is the FDA label update.

http://www.fda.gov/cder/drug/infopage/RHE/default.htm  Read More & Comment...

Hatikvah: CMPI Co Sponsors Biotech Boot Camp In Israel

11/08/2007 10:07 AM |

Peter and I are leaving for Israel today where will be participating in and co-sponsoring a three day program for Israeli biotech startups designed to help them improved their ability to become full fledged companies. Israel is home to some of the best translational science in the world and providing companies insights to the policy and regulatory environment in the US can help enhance value and improve efficiency. The conference is entitled Health Care Technological Innovation: From Idea to Commercialization. It's co-sponsored by the Israel Life Science Industry, The International Institute for Biotechnology Entrenpreneurship, Tel Aviv University's Recanti School of Business and CMPI.

It's Peter's first trip and as for me, I get to see my son who, as some of you know, is serving in the IDF.



Here's the conference agenda:

http://biomedmanagement.tau.ac.il/

We will blog from Israel!  Read More & Comment...

If it ain't broke -- don't send it to Geneva

11/08/2007 08:56 AM |

Per the current shenanigans going in at the IGWG in Geneva, here's a breath of fresh air -- a thoughtful analysis on the actual state of affairs vis-à-vis drug development and patents. It's by Philip Stevens of the International Policy Network and appears in Investors Business Daily. It's a potent rebuttal of the various and sundry half-truths put forward by Jamie Love, the Pancho Villa of Patents, and his fellow travelers.

If R&D Ain't Broke, Why Break It?

BY PHILIP STEVENS

There is a long list of complaints against the current system of drug development:

• Patents and profits have failed to produce new medications for the diseases of poor countries.

• There are not enough groundbreaking new therapies.

• Profiteering companies routinely make tiny changes to drugs to extend patents and shut out competition.

• Not only that, the patent system allows companies to charge astronomical sums for the drugs they do produce.

These allegations come from a powerful group of ideological nongovernmental organizations, or NGOs, that abhor profit in medicine and are pushing the World Health Organization toward a global treaty that would completely change the way drugs are researched and developed.

But before taking their medicine, we should take a close look at the label — and the nasty side effects.

This Medical Research and Development Treaty, proposed by Brazil and Kenya, would have a central U.N. bureaucracy deciding what diseases to research while allocating funds, contracts and prizes accordingly. Its expert scientists would ensure that all diseases are given appropriate resources, including the handful of "unprofitable" tropical diseases in poor counties.

NGOs, including humanitarian groups such as Medecins Sans Frontieres, hope this scheme will solve the failings of the current system at a stroke.

Because intellectual property would be owned by governments, drug prices would plummet. Less would be spent on frivolous ailments such as erectile dysfunction and baldness, and more on malaria and HIV/AIDS. Resources could be concentrated on groundbreaking "blockbuster" drugs, instead of the small molecular changes that are routinely patented now.

When the WHO first mooted the treaty in May 2006, MSF called it a "breakthrough" that "would ensure that patients' needs rather than profits drove innovation."

If the aim is to punish Big Pharma's stockholders, it will probably work. But as a way of producing cheap innovative drugs for the poor, it fails on several counts.

First, giving such discretionary power to bureaucrats would politicize R&D. In a centrally directed system — as in Britain's health service — resources tend to go to the loudest pressure groups. Other diseases would be neglected in favor of politically high-profile diseases such as HIV/AIDS.

Neither is it clear how an unelected body in Geneva would be better at setting priorities than the thousands of scientists and businessmen whose livelihoods depend on getting these decisions right.

Second, using state-funded prizes as the major incentive for R&D is problematic. The prize committee can never know the true market value of the drug it is hoping to create. If the prize is too low, companies will be reluctant to compete for future prizes, leading to fewer new drugs. If the prize is too high, the new system will squander taxpayers' money and divert effort from other areas of research.

Prizes were much favored in the Soviet Union, but they never resulted in much innovation.

Third, the treaty would turn drug manufacturers into utilities, living off government contracts. Removing the freedom to decide what to sell and at what price will discourage companies from risking capital to reap rewards, which is how innovation happens.

This is a clear lesson from regulated utilities such as water, electricity, telephones and gas. In a minimal-profit sector, companies do the bare minimum to fulfill their contractual obligations.

Most fundamentally, the treaty does not solve the greatest health care problem in poorer countries: how to actually get the drugs to patients in the face of crumbling hospitals and chronic shortages of doctors and nurses.

In 2006, the director of the World Health Organization's HIV division, Kevin De Cock, said "it is very obvious that the elephant in the room is not the current price of drugs. The real obstacle is the fragility of the health systems. You have health infrastructure that is dilapidated, and supply chains that don't exist."

If prices are an issue, why not scrap taxes and tariffs on medicines, which can increase the manufacturer's price by up to 11 times? These taxes on the sick are levied by many poor countries, including Kenya.

Removing commercial incentives will make companies retreat from the difficult and expensive work on cures for cancer and the like, and try to regain lost profits in politically safe "lifestyle" ailments. And governments have yet to demonstrate that they can produce drugs themselves.

All this is worrying for the U.S., which has already allowed the draft treaty to progress too far. There are echoes of the Clinton administration's hapless negotiation of the TRIPS agreement in 1994, which is now coming back to haunt America.

That agreement on Trade-Related Aspects of Intellectual Property Rights includes a clause designed to allow emergency production of essential medicines in poor countries. In practice, its wording is so vague that it allows any government to override the patent on any drug it likes. Middle-income Brazil and Thailand are now doing exactly that.

If the negotiators from the Department of Health and Human Services do not firmly reject the proposals at the WHO's Intergovernmental Working Group on Public Health, Innovation and Intellectual Property in Geneva this week, there won't be any pharmaceutical patent rights worth the name. This will be a body blow to innovation.

The current patent-based R&D model has produced most of the drugs that exist. It has a few problems, but there is no point junking it for an ill-conceived NGO fantasy. The biggest losers will not be stockholders, but patients.

Well said, Philip.  Read More & Comment...

Tops in BioTech Blogging

11/08/2007 07:30 AM |

DrugWonks is proud to have been chosen by BioTech360 (a publication of The Scientist) as the #1 “must read” blog for those in the wonderful world of biotech.

According to Biotech360 blogger Yali Friedman, “As the forum for the Center for Medicine in the Public Interest, Drug Wonks covers policy affecting biotechnology. This blog also tracks and responds to Op-Eds and other news items, providing additional perspective on many important topics.”

His top five list can be found at:

http://www.biotech360.com/biotechArticleDisplay.jsp?biotechArticleId=100039

Thanks Yali.  Read More & Comment...