Getting It Right on Clinical Trials: Honesty is the Best Policy

10/25/2007 09:51 AM |

Sanofi's conduct in the testing of Ketek underscores the importance of trust in shaping perceptions of risk. The relationship between drug companies and consumers must change. It will have to be more honest, more transparent, more individualized and enduring. It can't end after the FDA approves a product either.

According to the WSJ the FDA said it was "unable to find evidence" that the company either fixed the problems or threw the problematic doctors out of the study and told the FDA. The FDA also faulted Aventis for failing to make sure the study was properly conducted and for allowing unqualified investigators to participate in the trial.

These problems are rare and will happen. However when they are detected they should be reported promptly and the unqualified investigators should be barred from participating in clinical trials as a matter of industry policy.

http://online.wsj.com/article/SB119328145497070973.html?mod=googlenews_wsj  Read More & Comment...

Stark Raving Anti-Semite...

10/24/2007 12:17 PM |

Who says sorry seems to be the hardest word? Here's a sample of some a Pete Stark's other gentle musings about those who differ with him on healthcare policy issues... From Jonathan Weisman of the Washington Post (get a look at the photo the WP selected for the article..)


2003: As Stark belittled House Ways and Means Chairman Bill Thomas (R-Calif.), then-Rep. Scott McInnis (R-Colo.) told him to "shut up."

"You think you are big enough to make me, you little wimp?" Stark taunted. "Come on. Come over here and make me, I dare you. You little fruitcake."

2001: At a Ways and Means subcommittee hearing on abstinence promotion, Stark referred to then-Rep. J.C. Watts (R-Okla.) as the "current Republican Conference chairman, whose children were all born out of wedlock."

1995: In a private meeting, Stark called then-Rep. Nancy L. Johnson (R-Conn.) a "whore for the insurance industries."

"He didn't call her a 'whore,' " defended Stark press secretary Caleb Marshall. "He called her a 'whore of the insurance industry.' "

1991: Stark singled out "Jewish colleagues" for blame for the Persian Gulf War, referring to then-Rep. Stephen Solarz (D-N.Y.) as "Field Marshal Solarz in the pro-Israel forces."

1990: Stark called then-Health and Human Services Secretary Louis Sullivan, who is African American, "as close to being a disgrace to his race as anyone I've ever seen."

http://www.washingtonpost.com/wp-dyn/content/article/2007/10/23/AR2007102302165.html  Read More & Comment...

Evidence of an Evidence-Based Double Standard at CMS

10/24/2007 09:48 AM |

Maybe someone can explain the difference between the risks of off-label use of drug-eluting stents -- which CMS will still cover -- and the risks of appropriate ESA use -- which CMS will is banning. In both cases there is no evidence from randomized controlled trials demonstrating safety or quality of life benefit which CMS claims is the standard for reimbursing things these days.

Last time I checked there was no randomized placebo controlled study of aspirin with safety being the primary endpoint. Maybe Medicare should not pay for that either.

http://online.wsj.com/article/SB119318921736669347.html?mod=health_home_stories  Read More & Comment...

People Leak When They Are Losing or it is When They Are Losers?

10/24/2007 08:00 AM |

Gee, I wonder who leaked this to the WSJ...Let's see, when you don't have the science or the facts on your side, presenting the sort of observational data in public to defend your position that you derided as evidence in support of the safety of other drugs and when everyone else in the agency is pretty much responsible...my guess is David Graham and that it is part of a well-orchestrated effort. We know it's not the European Medicine Agency which just tighted prescribing data. Let's hope with better post market data we won't have this hysteria in the future. My guess is, with tort lawyers and web-based interest groups profiting from fear we will see more of this, not less.

http://online.wsj.com/article/SB119318921736669347.html?mod=health_home_stories  Read More & Comment...

Andy's Legacy

10/24/2007 02:18 AM |

Andy von Eschenbach comments on the issue of "legacy drugs" via a letter in today's edition of the New York Times ...

To the Editor:

The Food and Drug Administration thanks you for emphasizing our continued efforts to protect the public and raising consumers’ awareness that certain drugs are marketed without F.D.A. approval. In June 2006, the F.D.A. announced its renewed emphasis on solving the problem that many drugs lack F.D.A. approval, often because they were introduced into the market long before the F.D.A. had its current regulatory authority.

To deal with the unapproved drugs that remain in use, we give highest priority to drugs that have safety concerns, lack evidence of effectiveness or are health frauds. This approach is carried out without imposing undue burdens on health care and is applied uniformly against all companies, large and small, that market these drugs.

The F.D.A. is committed to proactive action to assist companies with finding opportunities to legally market their products. As part of this commitment, the F.D.A. appointed an unapproved drugs coordinator and conducted a full-day workshop in January 2007 to educate companies about the drug approval process, the over-the-counter monograph process and user fee waivers. Our unapproved drugs coordinator consults frequently with companies, and many of them have begun the approval process for their products.

Some refer to these unapproved marketed drugs as “legacy drugs.” A legacy is not a substitute for drug approval. Individual experience, anecdotal evidence and marketing history are insufficient bases for concluding that a drug is safe and effective. In fact, experience demonstrates that unapproved drugs are often unsafe, ineffective, inappropriately labeled and poorly manufactured. This is not the legacy that we want for our families.

I, and the entire F.D.A., remain committed to ensuring that safe and effective drugs are available to protect and promote the health of the American people.

Andrew C. Von Eschenbach, M.D.
Commissioner of Food and Drugs, Food and Drug Administration
Washington, Oct. 22, 2007  Read More & Comment...

FDA Cough Medicine Decision Puts Pediatricians In Silent Mode

10/23/2007 06:18 AM |

Here's the bottom line reason pediatricians love the decision to ban cough medicines: fewer 2 AM phone calls from parents about dosing, less anxiety about lawsuits...

Benjamin Brewer MD who blogs for the WSJ on medicine:

The dose provided some relief — the boy’s sniffles and cough abated long enough for the Brewers to get some shuteye. “I have found that the advice to hold off on a kid’s cold medicine is much easier to give than it is to heed,” Brewer writes.

The crackdown on cold medicines for kids may have an unintended benefit for the doctor. “With infant cold medications off the market, I won’t miss the parents calling in the middle of the night for recommendations on how much to use,” he writes. “I won’t miss meticulously documenting how many tenths of a milliliter I advised a person to give or the liability risks associated with my advice.”

Thanks for the dose of honesty doc.

I just wish the FDA experts were as freaked out about MRSA and diminishing antibiotics to treat them as they were about cough medicines. Ironically, the "safety uber alles" movement has made it likely kids will see fewer antibiotics for killer bugs or coughs. Reminds me of when I was a kid. It would make me feel a little bit better if Mickey Mantle were still batting clean up for the Yankees.

http://blogs.wsj.com/health/2007/10/23/a-taste-of-his-own-medicine/

For those who want to see Mantle's stats as opposed to the number of kids who died from infectious diseases in the 1960s...

http://www.baseball-reference.com/m/mantlmi01.shtml  Read More & Comment...

Bone Débarras

10/23/2007 03:23 AM |

Just in from the Beeb ...

Bone drug rationing 'must end'

Campaigners are appealing against a decision they claim is restricting doctors from prescribing osteoporosis drugs on the NHS.

Currently only one drug - alendronate - is approved by the National Institute for Health and Clinical Excellence. But the drug can cause a severe reaction in a quarter of patients.

The National Osteoporosis Society (NOS) says hundreds of thousands of people are missing out on potentially life-saving treatment as a result. NOS says the decision by NICE to recommend the cheapest drug is a false economy and leaves many patients at increased risk of painful and life-threatening fractures.

Professor Ignac Fogelman of the NOS said: "We are challenging the financial model that was used to look at the cost effectiveness of the various treatments for osteoporosis." GP Rosemary Leonard said: "Alendronate is the one osteoporosis drug which is now off patent so it is a lot cheaper than the others which is why there is this push to prescribe it. "But unfortunately one in four people who are on it can get bad reactions to it, particularly inflammation of the oesophagus.

"There is a choice available but NICE guidelines say that primary care trusts only have an obligation to provide alendronate."

Postcode lottery

In some primary care trusts, this means alendronate is the only drug choice offered, she said.
"We are heading for a postcode lottery."

Professor Tim Spector, a consultant rheumatologist at St Thomas' Hospital, London said: "It is vital for clinicians and patients to have alternative treatments available so we can maximise patient choice, reduce avoidable drug side effects and reduce the risk of osteoporotic fractures."

NICE has not yet released its final guidance.

It said in a statement: "It is disappointing that the appeal will delay final guidance on use of drugs for osteoporosis and delay publication of our clinical guideline which will then set out the best use of drugs and non-drug treatments."

A spokeswoman said NICE would report back in due course once the appeal had been heard.
Osteoporosis literally means "porous bones" and makes it more likely for them to fracture as they lose their density.

Over 1m women in the UK have been diagnosed with the disease, although experts say many more probably suffer from the condition.

Is this what we really want in the US? That's just, well, SiCKO.  Read More & Comment...

March of the Paternalists

10/22/2007 05:19 AM |

Seichel. In Hebrew it means discretion and in Yiddish it means "common sense" or as Joseph Aaron puts it...good sense, street smarts, the kind of wisdom you don’t get from getting a Harvard diploma.

Seichel tells you what’s right and what’s smart better than any book or guide or intellectual.

Or any FDA panel.

Seichel tells you that when millions of parents over 40 years have used cough and cold medicines responsibly they have a better handle of their kids needs than the media seduced know it alls who gathered in Rockville.

Did any of them consider that, because of the ban, parents will simply water down or dose adult cough meds to help their kids stop coughing or sniffling? Or that they will turn to even less effective or untested "natural" remedies that may be even more dangerous?

No, because the panel and the American Academy of Pediatric were not guided by seichel but by the "safety uber alles" principles pushed by the trial lawyers (websites now coming to a computer near you) have pushed medicine past science and common and into the panic room.

The decisions we make out of fear and to eliminate risk completely will always lack common sense and reek of mob rule.  Read More & Comment...

Et tu, Bernie?

10/22/2007 05:00 AM |

The Junior Senator from Ben & Jerry’s, Bernie Sanders, has just introduced a bill that would replace our current patent system for pharmaceuticals with a “Medical Innovation Prize Fund.” Here we go again.

It’s not a new idea. The “prize” model has been used in the past – in the old Soviet Union. It didn’t work. The Soviet experience was characterized by low levels of monetary compensation and poor innovative performance. The US experience isn’t much better. The federal government paid Robert Goddard (“the father of American rocketry”) $1 million as compensation for his basic liquid rocket patents. A fair price? Not when you consider that during the remaining life of those patents, US expenditures on liquid-propelled rockets amounted to around $10 billion.

Certainly not what Schumpeter had in mind when he wrote about “spectacular prizes … thrown to a small minority of winners.” Creative destruction indeed!

Senator Sanders wants to replace a patent system that has allowed the average American lifespan to increase, over the past 50 years, by almost a full decade with a prize program that has a solid record of complete failure.

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.”

In other words (and to paraphrase Winston Churchill) our pharmaceutical patent system is the worst way to stimulate and support health care innovation – except for every other system. On a list of 100 ideas for ways to improve innovation and access, a prize program shouldn't even on the list.

Who could support such a crackpot idea? Nobody? Wrong! Dangerously wrong. Again, as DiMasi and Grabowski presciently observed in 2004, “The main beneficiaries in the short-term would be private insurers and public sector purchaser of pharmaceuticals … Governments and insurers are focused myopically on managing health care costs. They are not likely to be strong advocates for funding new drug development that can increase individual quality of life and productivity."

Cui bono indeed.

Here is a link to the legislation(courtesy of one of its biggest supporter, Jamie Love):

http://www.keionline.org/misc-docs/SandersRxPrizeFundBill19Oct2007.pdf

Those who support this idea are so blinded by their own propaganda, that they view it as a solution to all the world’s health care ills. Consider some of the following statements:

Merrill Goozner (CSPI), “Research is risky, new drugs are too expensive, and
industry focuses far too much of its effort on drugs of minimal medical significance. The prize fund solves all these problems by disconnecting the incentives for generating breakthroughs from the price that individual patients or their insurers must pay."

Sorry Merrill. Wrong on all counts.

Jamie Love (KEI), “By separating the markets for innovation from the markets for the physical goods, the Prize Fund would ensure that everyone, everywhere, could have access to new medicines at marginal costs.”

Er, Jamie – time for some remedial economics classes.

And the timing of Senator Sanders’ bill is interesting too. It coincides with the upcoming meeting of the WHO’s Intergovernmental Working Group on Public Health, Innovation and Intellectual Property (IGWG in short) whose goal is "to prepare a global strategy and plan of action on essential health research to address conditions affecting developing countries disproportionately.”

As Meir Pugtach (Haifa University) and Helen Disney (Stockholm Network have argued:

After all, how can we expect that an international body will be able to secure the implementation of recommendations, such as "promote the active participation of developing countries in innovation", "provide support for national health research programmes in developing countries through political action and long-term funding", "promote transfer of technology and the production of health products in developing countries" and "monitor the impact of intellectual property rights and other factors on innovation and access to health-care products"?

The truth of the matter is that the promotion of innovation and the creation of new medicines for the sake of developing countries cannot be based on a top-down process. Rather they should be based on bottom-up solutions by the actual players involved in this process - companies, research institutions, and the regulatory and IP authorities.

Clearly Senator Sanders and the Jamie Love-ites do not concur. They want the philosophy of the IGWG to become the law of the land in the United States -- hard facts, economic theory, and historical precedents not withstanding.

Hopefully the US delegation in Geneva will strongly argue against this philosophy and refuse to enter into “consensus.”

A prize in every box does not a Crackerjack idea make.   Read More & Comment...

Comparative Effectiveness Continued

10/19/2007 01:01 PM |

First, I am glad we finally got the posting problem fixed. I do appreciate the comments and criticisms (most of them.)

Second, with respect to Merrill's comments. CMPI is a separate 501 c 3 and we would be happy to send you our 990.

Third, I appreciate Dr. Posen's comments too. I think we are all on common ground...what comparative effectiveness should look like. I would be more than happy to debate/discuss with Merrill you or anyone and provide a forum to do so. Here's our view of comparative effectiveness:

Comparative effectiveness research as currently constructed consists of centralizing coverage decisions for entire groups of people using population-based studies. It looks a single treatment or device in isolation, rather than an integrated focus on personalized, predictive and prospective medicine. Comparative effectiveness only looks at the bottom line of insurers. Tailored treatments rely on combining information about individual differences in genetic and clinical responses to improve wellbeing and measuring outcomes and the value of care to patients, employers and families.

Further, the introduction of the use of “quality adjusted life year” as a bench mark for comparative effectiveness, coverage and reimbursement flows from a method and model of analysis that is similarly outdated and which fails to take into account the value that personalized and targeted therapies provide individuals and their families. In general, the default value of a QALY appears to be $50000 US dollars though that figure has little empirical evidence and was developed to assess the value of dialysis for end stage renal patients in 1979.

CMPI has set up a Patient Centric Health Leadership Forum.

In contrast to the reliance on meta-analyses and large trials that exclude patient variation, we hope to advance the use of individual patient level information from conventional clinical assessments, genomic and biomarker analyses, and, where appropriate, advanced imaging studies.

Such information will be used to increase the adoption of the use of real time updates and refinement of the risk prediction algorithms and health plan strategies that are supported by data-mining techniques, filtered through expert panels at the patient level.

Third, we want to work with policymakers, insurers and government to ensure that value of personalized evidenced, integrated care and targeted medicine is fully articulated at all policy considerations about comparative effectiveness.  Read More & Comment...

Paid Advocate: Look Who's Talking

10/19/2007 09:36 AM |

Let's be clear: Goozner works for Center for Science in the Public Interest. We run Center for Medicine in the Public Interest. Both groups deal with science and medical issues. Both are agenda driven research based organizations. We both get money from different sources that reflect two very different views about the relationship between science, innovation and the private sector.

CSPI gets $16 million a year from a variety of left wing groups and foundations and from a newsletter that over the years has told people that transfats were good (1980s) and that they were bad (2000s) and that wine, soda, popcorn, Chinese food, cookies, and anything other than raw carrots and celery can kill you. It has a vested interest in pumping out bad news and selling it. It has taken money from foundations to promote campaigns against Olestra, antibiotics in agriculture, wine consumption, gelatin, food additives because it causes ADHD, acrylamide (because it "causes" cancer) in bread and social drinking.

We get less then ten percent of that from individuals, foundations, biotech and pharma companies to discuss, promote and develop strategies and approaches to personalize the delivery of medicine. The blog and our oped writing is a small part of who we are and what we do. We blog and write on comparative effectiveness and its limits because no one else is and no one else seems to have the willingness to do so.

In this context simply calling me or Peter a "paid advocate" is tired and intellectually shallow. If we toed the party line and trashed "Big Pharma" no one would care where we got our money from, even if it came from Soros or Chavez or third generation tobacco scions.

This is the last time we will deal this issue because if our critics lack the self awareness and self-honesty to accept that no one group can claim to speak on behalf of the enlightened public interest because of where they get their support. Calling oneself a public interest group is, as Wildavsky noted, not just convenient, it is flattering. " Perhaps getting money from newletters and liberal foundations puts one on a higher moral plane and less conflicted We just think it makes others more...liberal.  Read More & Comment...

Unfinished Business Over Comparative Effectiveness

10/19/2007 08:43 AM |

Drugwonks has been attacked over it's views on comparative effectiveness. The attacks break down into two lines of "thinking".

1. We receive funding from drug companies.
2. We receive funding from drug companies.

Apparently if we supported comparative effectiveness as a wonderful tool for achieving optimal prescribing and reimbursement decisions our funding from drug companies would not be an issue. But because we criticize it and offer the collection of patient-centered data to support improved outcomes and productivity, our views are both wrong and tainted. I guess taking money from George Soros, trial attorneys and left wing foundations or just hating drug companies -- which some bloggers do -- in no way biases the views of others. So people who receive pharmaceutical firm support are tainted but everyone else is objective. Yeah, right.

Indeed, the biomarker and patient-centric approach we support is now being attacked as..no surprise... as another venue for industry to enrich itself. See our previous posts regarding the unwarranted attack on the Reagan Udall Institute.

Follow this logic. Big Pharma used the FDA to push through me-too drugs of limited efficacy so it could market blockbuster drugs to an unthinking public. Critics clamored that companies should invest in breakthroughs based on new genetic research that targeted important diseases and developed drugs that really advanced care.

So now companies are doing just that or trying. They are now being criticized for investing in efforts to develop targeted medicines using new science. Instead, critics want "hard" evidence that people are actually cured or better before a drug is approved though the predictive accuracy of genetic markers is precisely what is revolutionizing health care.

When someone keeps changing the standards and the goalposts it tells you that what they dislike is not the goal but the target of their criticism. Comparative effectiveness, as applied in every health system, is used to control costs and limit access to new medicines. Meanwhile studies show new medicines improve productivity and extend life.

Those who support comparative effectiveness have yet to show me one study they support they demonstrates the better value of new medicines. That's because for the most part they are design by government agencies and others with a bias towards cost containment and against medical innovation. That goes for the ALLHAT and CATIE studies.

And as for the ALLHAT study, Health Care Renewal does not want to accept the fact that the ALLHAT design was bizarre and structured to produce excess mortality in blacks. Don't believe me, believe Michael Weber who was one of the investigators....

"The reality of ALLHAT – it was poorly designed, the interpretations were disingenuous, it violated appropriate scientific reporting, and most frightening, it did something that was so unethical that if a pharmaceutical company had done it or any of us as individual academics had done it, we would not only be thrown out of our jobs, we would be pilloried and maybe even be facing criminal charges: They exposed African-American patients for several years to treatments they knew would not be effective in controlling their blood pressure.

And one thing that did show up in favor of diuretics, the fact that they cause fewer strokes than one of the other drug classes, was driven entirely by a 40% excess stroke rate in black patients that was predictable before the study began. And they used that as their reason to claim superiority of the diuretic."

I want to know if Health Care Renewal would treat his African American patients with high blood pressure and congestive heart failure without using BiDil and according to the ALLHAT guidelines?

http://hcrenewal.blogspot.com/

Some proponents of comparative effectiveness might because they are ideologues. And that's the difference. For a lot of people and policymakers comparative effectiveness analysis -- from the design of studies right down to the reimbursement -- it's political and a way to wound drug companies.

Finally, I apologize to all who have tried to post comments and have not been able to. It was not intentional. We are making a technical fix to clear this problem up.  Read More & Comment...

Dosing For Dummies

10/19/2007 08:41 AM |

Instead of banning medicines, how about banning people from using drugs until they can follow directions. These are actual cases of how people used OTC and Rx drugs from the Oklahoma Poison Control Center.

Dosing cup errors accounted for 3.8% of all therapeutic errors, but was the fifth leading cause for errors in the less than 5 year-old patient group. The most common reasons for therapeutic errors in all age groups involved taking or giving the wrong formulation or concentration, inadvertently taking/giving medication twice and another incorrect dose. Typical examples of some of these errors are as follows.

Incorrect Formulation or Concentration:

1. A mother mistakenly gave 2.5 ml of lindane 1% shampoo to 6 month-old, 20 pound child instead of promethazine DM cough syrup due to similarity in the appearance of the bottle.
2. A mother gave 2 ml of a sibling’s baclofen suspension instead of acetaminophen syrup to her 8-month-old son.
3. A grandparent gave 5 ml of Benadryl ® Maximum Strength Itch Stopping Gel 2% to a 10 year-old child after being instructed by parents to give the girl her dose of “Benadyl ® syrup”. Final dose of diphenhydramine equaled 100 mg instead of 12.5 mg.
4. Four capsules of Hartz Mountain ® Dog Wormer containing piperazine adipate were taken by a 42 year-old woman instead of 4 diphenhydramine 25 mg capsules for sleep. She also gave 4 capsules to her 15 year-old son as well.


Other Incorrect dose:

1. Parents misunderstood prescription directions and double dosed 10 year-old daughter’s dextroamphetamine sulfate® 10 mg for two weeks.
2. Parents gave 2 year-old daughter doses of Tylenol® Syrup for Children and Dimetapp® Nighttime Flu for 3 days before checking the labels and finding acetaminophen in both products.
3. Alendronate sulfate 70 mg was taken daily for 3 days instead of once weekly for 3 weeks by 86 year-old cardiac patient.
4. Six tablets of Triphasil® birth control pills were taken at one time because 34 year-old woman has missed 6 of her doses.


Maybe we should cough medicines for children under the age of 34.  Read More & Comment...

Cough, Sniffle, Sneeze -- No OTC Meds for Children, Please

10/19/2007 07:36 AM |

Excellent/frightening article from today's Newark Star-Ledger. (And not frightening because of safety concerns -- frightening because of creeping Precautionary Principle-ism.

Doctors target remedies for kids, Urge wider restrictions for cough and cold drugs
BY ROBERT COHEN

STAR-LEDGER WASHINGTON BUREAU

SILVER SPRING, Md. -- A group of leading pediatricians said yesterday the drug industry's recent decision to stop selling over-the-counter cough and cold remedies to children under 2 is inadequate and should be extended to age 6.

"The Food and Drug Administration did not approve these products on the basis of evidence of safety and effectiveness, but FDA has permitted widespread marketing that is not supported by scientific evidence," Maryland Commissioner of Health Joshua Sharfstein told an FDA advisory panel that is holding two days of hearings on the issue.

Sharfstein, a pediatrician by training, said the drug industry spent more than $51 million advertising pediatric cough and cold medications from July 1, 2006, to June 30, promoting them in a "misleading" way as pediatrician-recommended and safe for young children.

He said the over-the-counter medicines are no more effective than placebos, have resulted in serious side effects, including death, because of misuse and should be barred in the "vulnerable" population of children under 6 -- points strongly disputed by the drug industry.

"When a treatment is ineffective, its risks, if not zero, will always exceed its benefits," added Michael Shannon, a professor of pediatrics at Harvard Medical School.

The comments were made to back up a petition by the pediatricians asking the FDA to restrict use of the medicines for children. They said they chose age 6 because of safety concerns, but do not believe they are effective for older children, either.

The FDA advisory panel will vote on recommendations today that could include restrictions on marketing of cough and cold medicines for children under 12, adding warnings to labels and allowing only single-ingredient products. The FDA usually follows the recommendations of its panels, although it is not required to do so.

The panel is meeting a week after Johnson & Johnson, Wyeth, Novartis and Prestige Brands voluntarily recalled 14 nonprescription "infant" cough and cold medicines targeted for children under 2 years of age.

At yesterday's hearing, industry representatives continued to insist the drugs can be used safely and effectively at recommended doses for children 2 and over.

"Reported serious adverse events are very rare when cough and cold medicines are administered at therapeutic doses," said Edwin Kuffner of McNeil Consumer Healthcare, a unit of Johnson & Johnson.

Kuffner said side effects tend to result from overdose or accidental ingestion and represent a very small percentage of the tens of millions of children who effectively use the medicines every year.

The Centers for Disease Control and Prevention reported earlier this year at least 1,500 children younger than 2 suffered complications from cough and cold remedies in 2004 and 2005.

A recent FDA staff review described dozens of cases of convulsions, heart problems, troubled breathing, neurological complications and other reactions, including at least 54 deaths involving decongestants and 69 deaths involving antihistamines from 1969 to 2006.

FDA officials said they believe adverse events from the medicines are greatly underreported.

Richard Dart of the University of Colorado School of Medicine, speaking for the industry, said while more research is needed, there are "some pediatric studies that have shown effectiveness in children even as young as 6 months old ... and we know from adult studies that their medicines work."

Asked later by FDA advisor Leon Dure of the University of Alabama School of Medicine if there is "objective data about the benefits of these drugs," Phil Walson, an industry expert from the University of Cincinnati, said, "Not that I am aware of."

FDA Medical Officer Lolita Lopez also told the panel "published clinical studies in children did not establish efficacy of cough and cold medicines."

Linda Suydam, president of the Consumer Healthcare Products Association, the industry trade group, said the industry wants to strengthen labels to stress "do not use" for children under 2 and "do not use to sedate children." She also said companies will undertake an education campaign to ensure proper use of the drugs.

George Goldstein, an industry consultant on the advisory panel, asked that if the medicines are not effective or safe, "how is the purchase of millions -- hundreds of millions -- of doses by parents explained?"

Dan Levy, president of the Maryland chapter of the American Academy of Pediatrics, said parents buy the medicines for emotional reasons, such as fear and caring. He said he recommends to parents they would get better results by "throwing it down the toilet rather than administering by mouth."  Read More & Comment...

VA and Nissen Team Up To Leave Diabetes Uncontrolled

10/18/2007 12:26 PM |

The VA yanks Avandia from the formulary...which means more people will just tough it out on one less diabetes drug.

If this is evidence-based medicine give me science fiction. Where's Dr. McCoy when you need him?

Here's a question from Stephanie Saul's piece on the VA's Avandia hook...

Dr. Jon LeCroy, a senior pharmaceuticals analyst for the investment and research firm Natixis Bleichroeder, said that before Dr. Nissen’s article last May, about one million prescriptions were being written each month for Avandia.

As of September, Avandia prescriptions had declined about 60 percent, to 426,000 a month, according to Dr. LeCroy.

But he said there had not been a corresponding increase in the use of other drugs for diabetes, indicating that some patients who had stopped taking Avandia had not replaced it in their drug regimens. Diabetes patients often take more than one medication at a time for their conditions.

Actos, a drug similar to Avandia that some studies indicate does not carry the same heart risk, has picked up 100,000 prescriptions a month, but that does not account for the drop of almost 600,000 in Avandia prescriptions.

Ms. Rhyne, of Glaxo, said a survey had shown that 50 percent of patients who discontinued Avandia did not begin another therapy as a substitute. She questioned whether those patients were placing themselves at risk for uncontrolled diabetes.

Here's Nissen's number at the Cleveland Clinic. (216) 445-6852. Someone should call and ask him. He's giddy over his "success."

http://www.nytimes.com/2007/10/18/business/18drug.html?_r=2&ref=health&oref=slogin&oref=login  Read More & Comment...

Rules of the Game

10/18/2007 05:42 AM |

According to an article in today’s edition of the New York Times …

“The F.D.A. has begun to crack down on the thousands of drugs that have never had to go through the agency’s stringent approval process, many of them made by small companies … and those companies are crying foul." According to one such manufacturer, “It has no regard for the cost or damage they do to small businesses. There are estimates that only a few of us will make it.”

The FDA’s response? “This is a public health initiative,” said Deborah A. Autor, director of the Office of Compliance at the F.D.A.’s Center of Drug Evaluation and Research. “Some of these drugs may not be safe. In all likelihood, these companies knew from Day 1 that they were producing illegal drugs.”

A trade group representing about 50 small to medium-size companies has submitted a bill to Congress that would create a cheaper and simpler process for gaining F.D.A. approval. Mr. Blansett claimed the cost could run up to $5 million for a new drug application.

Here’s a link to the complete article:

http://www.nytimes.com/2007/10/18/business/18hunt.html

My contribution to this reportage, two quotes:

“Many of these drugs were grandfathered in when the current approval process was instituted,” he said. “However, that doesn’t give these companies carte blanche — they still have to play by the rules.”

And

“The earlier you can get all parties to the table, including manufacturers, the better.”

Important issue.  Read More & Comment...

A Test of Bad Health

10/18/2007 04:48 AM |

From today's edition of the New York Times ...

A Test of Bad Health

By PETER PITTS

IF Congress overrides President Bush’s veto of the State Children’s Health Insurance Program, a little-known provision of the original House bill could be revived.

As written, the provision would allocate $300 million to create a Center for Comparative Effectiveness that would test whether newer, more expensive drugs work better than their older and cheaper counterparts. Medicare would use the center’s findings to help decide which drugs to cover. If the center found that a newer, pricier pill was no more effective than the older, cheaper version, Medicare would probably refuse to pay for it.

This sounds reasonable. But it will most likely result in Medicare covering fewer breakthrough medicines, which would, in turn, force doctors to prescribe only the drugs that Medicare will pay for — not the ones that are best for the patient.

Why? Drugs must be tested on large, representative populations that must be monitored for years. Because conducting these studies is so tricky, their findings are regularly overturned or modified by further research. In fact, some are so off the mark that doctors ignore them.

But if Medicare starts using flawed studies like these to determine its list of covered drugs, doctors will have to give them respect they probably don’t deserve. There’s also an inherent conflict of interest when the government conducts comparative-effectiveness studies and then uses those studies to determine which pills are worth buying. The more drugs the government classifies as “wasteful,” the more money it saves.

Look what happened in Britain. In 2001, contrary to expert findings by licensing authorities around the world, the British comparative-effectiveness agency cited “insufficient evidence” for recommending the use of Gleevec in both early- and late-phase chronic leukemia patients.

In 2002, the United States approved Gleevec for another purpose: to treat a rare stomach cancer. It wasn’t until 21 months later that Britain approved the use of Gleevec for victims of the disease.

What aside from cost concerns could explain such restrictions? And what could stop something like this from happening here? The center’s supporters say it will be financed through an independent “trust fund.” But this won’t solve the problem. The center would still be part of the government — and still get in the way of medical innovation.  Read More & Comment...

Gooz Cruise

10/17/2007 01:01 PM |

Merrill Goozner is a nice guy. We rarely agree -- but he's a nice guy.

Now he shows his colours as a NICE guy with a statement like, ""Congress should leave...life-and-death medical decisions to the professional, objective physician-scientists at our nation’s health agencies."

At least he's honest about where his path leads -- not to "universal" health care but rather to "government" care.

Bad idea.  Read More & Comment...

Power to the Pinheads

10/17/2007 09:52 AM |

Here's Merrill Goozner of the Center for Science in The Tort Lawyer's Interest giving full voice to a totalitarian view of regulation:

"Congress should leave...life-and-death medical decisions to the professional, objective physician-scientists at our nation’s health agencies."

Regular doctors -- those who are not scientists at our nation's health agencies are, by definition, too stupid, corrupt, tainted by Big Pharma to be trusted and consumers, well, why have consumer groups if you could trusts you and me to act on our own behalf. Thank goodness we have people like Merrill Goozner to lead the way.

Mind you,these are the same consumer groups that were screeching during the run up to PDUFA renewal that the FDA is a tool of Big Pharma and can't be trusted to protect the public.

I guess if you say you are a consumer group you can be as hypocritical all you want.

Here's the the post:



Dr. Congress Makes a House Call
GoozNews: October 17, 2007
As I mentioned yesterday, the Food and Drug Administration wrote a strong letter to Capitol Hill last week backing the Center for Medicare and Medicaid Services' restrictions on the use of erythropoietin-stimulating drugs like Aranesp and Procrit during cancer chemotherapy. The FDA black box warning, the letter pointed out, called on oncologists to use the lowest possible dose for avoiding blood transfusions since higher doses of the drugs to boost energy -- which, of course, results in higher sales for their makers, Amgen and J&J -- leads to more deaths and faster tumor growth in cancer patients.

Despite this evidence, Congress is considering a resolution to overturn the CMS payment decision, which went into effect late last month. It has at least 26 co-sponsors already and the drug companies' lobbyists are out in force trying to get it passed. So is the American Society of Clinical Oncologists, whose members profit from greater sales of the drugs.

Yesterday, a coalition of consumer groups including the Center for Medical Consumers, the Center for Science in the Public Interest, Consumers Union,
National Research Center for Women & Families, National Women’s Health Network, the TMJ Association and U.S. PIRG wrote every member of Congress asking them to vote no on H.J. Res. 54, which would overturn the CMS decision. I thought it worth reprinting here because of the principles at stake, which are outlined in the letter:

October 16, 2007
Dear Member:
We urge Congress not to interfere in the efforts of the Centers for Medicare & Medicaid Services and the Food and Drug Administration to use the best available science to determine the proper dosing of erythropoietin stimulating agents (ESAs). The safe and proper dosage of this drug in very vulnerable cancer patients is an extremely technical issue. Congress should leave these life-and-death medical decisions to the professional, objective physician-scientists at our nation’s health agencies.

H.J. Res. 54 or similar proposals in the Senate are a direct violation of this principle. The FDA has issued a “black box” label warning against excessive use of ESAs. There is significant evidence that the overuse of ESAs can actually speed tumor growth and early death in cancer patients. After more than a year of study, CMS issued a national coverage decision (NCD) in July that took this latest evidence into account. Congress should not substitute its own judgment for that of CMS and the FDA on these issues.

While it is true that the American Society of Clinical Oncologists, which represents the nation’s cancer physicians, protested the CMS decision, we cannot help but note that companies and physicians make enormous windfall profits from the sale and use of ESAs. Now they are trying to convince Congress that Medicare is denying a needed medical service.

This is not the proper venue for their objections. In late September, CMS invited ASCO to submit evidence to support the agency reopening its coverage decision. It is altogether fitting and proper that physicians in community practice and physicians at CMS who determine payment policy adjudicate their differences in this manner, rather than through Congressional intervention.

We also must note that some provider groups opposed previous reductions in the windfall profits that came from the reimbursement system of various cancer drugs. They said it would radically reduce treatment options and hurt patients. Those statements have been proven untrue. Self-serving arguments about the negative consequences of a proposed payment policy is no substitute for objective, scientific evidence.

Congress should set broad policy objectives and standards for Medicare, but Congressional interference regarding coverage policies for specific medical products would set a terrible precedent. It would encourage companies making medical products as well as medical specialty organizations to constantly ask Members of Congress to override scientific evidence and spend taxpayer dollars needlessly on products whose sale would benefit those companies or specialties more than they benefit patients. In some cases, such overrides could promote the use of medical products in ways that are potentially dangerous to patients because they are unsafe or ineffective.

Health care costs are the leading domestic consumer issue. Congressional interference on individual reimbursement decisions at CMS will just feed those health inflation fires while possibly causing harm to patients.

Please reject H.J. Res. 54.
Sincerely,

http://www.gooznews.com  Read More & Comment...

Critical Wrath

10/17/2007 05:04 AM |

A more complete response to the politically motivated, mean spirited, and just plain wrong comments by the so-called Union of Concerned Scientists ...

http://www.tjols.com/article-350.html

Here are some snippets:

* Twenty-five years ago, the success rate for a new drug used was about 14 percent. Today, a new medicinal compound entering early-stage testing – often after more than a decade of pre-clinical screening and evaluation – is estimated to have only an 8 percent chance of reaching the market. For very innovative and unproven technologies, the probability of an individual product's success is even lower.

* Better, more current and predictable scientific research and standards must be developed and devoted to streamlining the critical path. Investment in basic research is not enough. Specifically new development tools are needed to improve the predictability of the drug development cycle and to lower the cost of research by helping industry identify product failures earlier in the clinical trials process.

* New development tools in these areas will enable better through-put to commercial product development and will act as a productivity multiplier, increasing the returns on public and private investment in basic research. With improved scientific methods and a new, shared effort by all of us, we can develop and improve standards for product characterization and product safety testing, for both traditional and innovative products.

* Today only about 1 percent of the proteins in blood have been identified. Of that 1 percent only a fifth has FDA approved diagnostic utility. These proteins, after we understand them, could help predict disease remission. Currently academics and private companies collect data and establish correlations, but no one is responsible for organizing this information into the broader knowledge that could lead to generalized principles industry and FDA could use for broader, faster, and more accurate product evaluation.

* Think about the millions of dollars that would be saved by all types and sizes of companies and governments if publicly discussed and vetted biomarkers could be used and used predictably in the drug approval process. Using the lower end of the Tufts drug development number, a 10 percent improvement in predicting failure before clinical trials could save $100 million in development costs. Similarly, shifting 5 percent of clinical failures from late-stage to early-stage trials reduces out of pocket costs by $15-$20 million.

Next up on the Critical Path hit parade -- who will be named to the Reagan/Udall board of directors. Watch this space for more details.  Read More & Comment...