Latest Drugwonks' Blog
For those of you following the Plan B saga, here’s the transcript of the April 27th, 2012 hearing in New York Federal Court.
The April 27th hearing was supposed to be about whether Teva could intervene (to protect its 3 year exclusivity based on additional actual use/label comprehension studies FDA had acquired) and the court's order to show cause why the USG should not be held in contempt, but it also touched on the plaintiff's motion for a preliminary injunction/summary judgment and the government's motion to dismiss the case (which the judge denied orally at the hearing).
The most amusing part of the transcript is when the government tries to argue that none of the plaintiffs are 13, so the court can't give relief as to them. There were 13 years old when the case began, but the case has gone on so long they have reached the age of majority.
The judge is scathing of the government's argument, saying "Do you know how long this agency -- your agency has done nothing and then you have the chutzpah to come in here and say that thirteen-year-olds who started the case are of age now." (See page 55.)
The judge is pretty scathing of Secretary Sebelius, referring to her logic as "totally ludicrous" (page 12). He says that if Teva had appealed Sebelius's decision they would have won (page 24), but then later the DOJ lawyer points out that the Sebelius decision is not appealable, since it resulted in a complete response, which is not final agency action -- which the court finds odd (pages 38-43), at one point saying it seems like "blowing smoke."
Later on the judge says, "this whole thing is an artificial construct at odds with common sense” (page 54) and wonders how the argument could be made with a“straight face” (page 55). The judge also talks about how the no one can satisfy the Sebelius criteria because there are not enough 11 year olds who are having sex (pages 43-44). Ouch.
The judge also calls the FDA to task for not denying the citizens petition as soon as he remanded it to them (in 2009), and he thinks that as soon as the agency told Teva they needed to do more studies, FDA should have denied the Citizens petition. (This conversation begins on page 50.)
Yep.
http://www.doh.wa.gov/cfh/immunize/documents/coe2011.pdf
So why is the CDC punting on the whooping cough outbreak in Washingon? Here's the CDC spokesperson on the epidemic:
'Adults and teens need booster shots so they don't give pertussis to the babies in their lives, said CDC spokeswoman Alison Patti
"We want to create a cocoon of protection around them," she said. "We're really worried about keeping babies safe."
Patti emphasized that pertussis isn't spreading because of an anti-vaccine movement. Among possible reasons for the recent spike are that diagnoses in teens and adults are getting better and doctors are doing a better job with reporting, she added.'
This is nonsense. While outbreaks go in five year cycles, the incidence has skyrocketed in places where anti-vaccine sentiment is strongest: including Idaho, New Mexico, Madison, WI, Woodstock, NY and Vermont.
Under Obama, the CDC has been ducking the source of vaccine resistance and in some cases, caving into it. Perhaps its because their anti-sugar allies are also anti-vaccine or because the states essential to the president's re-election are also the one's where the anti-vax movements are strongest..
This is pure speculation on my part. But in any event, the lack of leadership on immunizations at the CDC is troubling. Bad enough that CDC is using cost-effectiveness analysis to decide whether it's worth paying for new vaccines under Obamacare. Ignoring a principal threat to herd immunity is even worse.
U.S. may speed approval of 'breakthrough' drugs
When the Critical Path was launched, the goal was to align the approval of drugs with the science of drug development. That process was derailed after the Vioxx panic. It is now back on track thanks to a bi-partisan effort on Capitol Hill, support from the Obama administration and the persistence of Janet Woodcock and Peggy Hamburg at the FDA. This is a great day for innovation and patients, at least in principle.
There will be those who see this reform as a sop to evil, profit making drug companies... Such as Merrill Goozner: "Pfizer last week won Food and Drug Administration “accelerated approval” for Xalkori (crizotinib) for a rare form of metastatic lung cancer that strikes about 3 percent of people — almost always non-smokers — who come down with the disease. That’s about 10,000 patients per year in the U.S., and perhaps a similar number abroad. Multiply that times the $115,200 a year the company plans to charge for Xalkori, and they’ve got themselves a billion dollar blockbuster. Through this period where there will be no concrete proof of efficacy, Pfizer will get to charge an outlandishly high price for the drug. Is there a way to put pressure on the company to complete the trials in a more timely fashion, so that the paying public knows that it is truly getting value for its health care dollar and not subjecting patients to some pretty rough side effects (nausea, diarrhea, etc.) for no benefit?"
Goozner is either ignorant or willfully misleading. By definition accelerated approval will be reserved for treatments that show significant benefits in specific groups of patients through well-characterized biomarkers and clinical endpoints. That will cut the time required for approval and reduce costs. (If Goozner can figure out a way to reduce the cost of producing new biologics for small groups of people, he should start his own biotech company.) The price of drugs will be relative to their value. Or should be.
The question is, as accelerated approval is adopted will CER just add back delay and access. Let's hope the courageous impatience of the FDA, the Obama administration, Congress and the public is shared by the CER crowd and PCORI.
It’s time to excise the Ben and Jerry’s Clause from the Senate HELP Committee’s draft of PDUFA.
That would be the amendment (adopted this April) that would require an evaluation of the so-called “prize fund approach” to drug development.
It’s same old and tired and false proposition put forth by Bernie Sanders – the independent Senator from the great state of Ben and Jerry’s – since time immemorial.
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 experience in the United States hasn’t been much better. The federal government paid the father of American rocketry, Robert Goddard, $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, U.S. expenditures on liquid-propelled rockets amounted to around $10 billion.
Senator Sanders wants to replace a patent system that has fueled innovation allowing the average American lifespan to increase by almost a full decade over the past 50 years with a prize program that has a solid record of failure. As the healthcare economists Joe DiMasi and Henry Grabowski have argued, under a prize program, pharmaceutical innovators would lack the incentive to innovate. DiMasi, director of economic analysis for the Tufts Center for the Study of Drug Development, and Grabowski, director of Duke University’s Program in Pharmaceuticals and Health Economics, said, “The dynamic benefits created by patents on pharmaceuticals can, and almost surely do, swamp in significance their short-run inefficiencies.”
Who could support the idea of a prize? 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.” That’s because, as they note, governments and insurers are focused “myopically on managing health care costs” and are not likely to be “strong advocates for funding new drug development that can increase individual quality of life and productivity.”
Those who support this idea view it as a solution to all the world’s health care ills. “Research is risky, new drugs are too expensive, and industry focuses far too much of its effort on drugs of minimal medical significance,” Merrill Goozner, director of the Integrity in Science Project at the Center for Science in the Public Interest, has said. “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.”
Not.
According to Jamie Love, director of Knowledge Ecology International, “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.”
Not.
Here’s Jamie’s spin on Senator Sander’s legislation -- a highly revisionist history.
The truth of the matter is that the promotion of innovation and the creation of new medicines 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 Jamie Love do not concur. And that is their privilege.
Top 10 Reasons Why The BMI Is Bogus
by KEITH DEVLIN
The BMI Formula
BMI = weight in pounds/(height in inches x height in inches) x 703
The 703 is to convert the index from the original metric version of the formula.
CDC Recommendations:
Below 18.5 = Underweight
18.5 to 24.9 = Ideal
25.0 to 29.9 = Overweight
30.0 and above = Obese
text size A A A July 4, 2009
Americans keep putting on the pounds — at least according to a report released this week from the Trust for America's Health. The study found that nearly two-thirds of states now have adult obesity rates above 25 percent.
But you may want to take those findings — and your next meal — with a grain of salt, because they're based on a calculation called the body mass index, or BMI.
As the Weekend Edition math guy, I spoke to Scott Simon and told him the body mass index fails on 10 grounds:
1. The person who dreamed up the BMI said explicitly that it could not and should not be used to indicate the level of fatness in an individual.
The BMI was introduced in the early 19th century by a Belgian named Lambert Adolphe Jacques Quetelet. He was a mathematician, not a physician. He produced the formula to give a quick and easy way to measure the degree of obesity of the general population to assist the government in allocating resources. In other words, it is a 200-year-old hack.
2. It is scientifically nonsensical.
There is no physiological reason to square a person's height (Quetelet had to square the height to get a formula that matched the overall data. If you can't fix the data, rig the formula!). Moreover, it ignores waist size, which is a clear indicator of obesity level.
3. It is physiologically wrong.
It makes no allowance for the relative proportions of bone, muscle and fat in the body. But bone is denser than muscle and twice as dense as fat, so a person with strong bones, good muscle tone and low fat will have a high BMI. Thus, athletes and fit, health-conscious movie stars who work out a lot tend to find themselves classified as overweight or even obese.
4. It gets the logic wrong.
The CDC says on its Web site that "the BMI is a reliable indicator of body fatness for people." This is a fundamental error of logic. For example, if I tell you my birthday present is a bicycle, you can conclude that my present has wheels. That's correct logic. But it does not work the other way round. If I tell you my birthday present has wheels, you cannot conclude I got a bicycle. I could have received a car. Because of how Quetelet came up with it, if a person is fat or obese, he or she will have a high BMI. But as with my birthday present, it doesn't work the other way round. A high BMI does not mean an individual is even overweight, let alone obese. It could mean the person is fit and healthy, with very little fat.
5. It's bad statistics.
Because the majority of people today (and in Quetelet's time) lead fairly sedentary lives and are not particularly active, the formula tacitly assumes low muscle mass and high relative fat content. It applies moderately well when applied to such people because it was formulated by focusing on them. But it gives exactly the wrong answer for a large and significant section of the population, namely the lean, fit and healthy. Quetelet is also the person who came up with the idea of "the average man." That's a useful concept, but if you try to apply it to any one person, you come up with the absurdity of a person with 2.4 children. Averages measure entire populations and often don't apply to individuals.
6. It is lying by scientific authority.
Because the BMI is a single number between 1 and 100 (like a percentage) that comes from a mathematical formula, it carries an air of scientific authority. But it is mathematical snake oil.
7. It suggests there are distinct categories of underweight, ideal, overweight and obese, with sharp boundaries that hinge on a decimal place.
That's total nonsense.
8. It makes the more cynical members of society suspect that the medical insurance industry lobbies for the continued use of the BMI to keep their profits high.
Insurance companies sometimes charge higher premiums for people with a high BMI. Among such people are all those fit individuals with good bone and muscle and little fat, who will live long, healthy lives during which they will have to pay those greater premiums.
9. Continued reliance on the BMI means doctors don't feel the need to use one of the more scientifically sound methods that are available to measure obesity levels.
Those alternatives cost a little bit more, but they give far more reliable results.
10. It embarrasses the U.S.
It is embarrassing for one of the most scientifically, technologically and medicinally advanced nations in the world to base advice on how to prevent one of the leading causes of poor health and premature death (obesity) on a 200-year-old numerical hack developed by a mathematician who was not even an expert in what little was known about the human body back then.
You would think (if you accept the stereotype) that trial lawyers would warn against the proposal as well. But Greg Webb, a blogger for the Injury Board Blog Network has penned a measured and thoughtful piece on the FDA's safe use proposal for expanded OTC access..
"Representatives of the American Medical Association (AMA) and the American College of Allergy, Asthma and Immunology (ACAAI) have gone on record objecting to the proposed new safe-use class of medications. Roland Goertz, MD, president of the American Academy of Family Physicians (AAFP), stated that the safe-use designation where pharmacists dispensed medications without a patient having to see a doctor first, “could seriously compromise the physician's ability to coordinate the care of multiple problems of many patients.”[1]
Health insurers reportedly have tentatively "approved" of the measure. Without proper safeguards, there is some concern that patients may obtain drugs that they may not need. The insurers also would have to determine how to cover drugs that fell into this category. Additionally, the health insurers were in favor of "expanding access" to helpful medications.
Because pharmacists are more easily accessible than physicians who require appointments for frequently costly office visits, the FDA believes that the “safe-use” designation may benefit many Americans who presently go untreated with legitimate medical needs. Doubtless the collection of comments and the promulgation of a new regulation on safe-use medicines by FDA (requiring additional debate) will cause more lines to be drawn in the sand of looming health issues. Is this the wave of the future for certain medications? Much of it makes sense, and ultimately would save money by preventing potentially unnecessary doctors' visits. What are the unintended consequences?"
Mr. Webb's article is well worth reading because it lays out the FDA proposal clearly. It is also noteworthy because it shows that the AMA and other groups are more hysterical about the Rx-OTC issue than the trial bar.
The FDA has proposed a benefit-risk assessment system for weight loss devices that would use available safety data for a device to set efficacy thresholds for a pivotal trial of that device. BioCentury reports that the proposal came in briefing documents issued to FDA's Gastroenterology and Urology Devices Panel ahead of a meeting on Thursday and Friday to discuss issues in the development of devices placed endoscopically or laparoscopically.
Devices would be classified into one of three risk levels based on the percent of patients who experience mild, intermediate and severe adverse events over one year. For example, to qualify for the lowest risk category, devices can have high rates of the most mild events (up to 100%), low rates of events of intermediate severity (2-5%), and very low rates of the more severe events (<0.1%).
Each level of risk would set corresponding efficacy endpoint targets for pivotal trials. Targets for the least risky devices would be based on endpoints used for weight loss drugs, while targets for the highest risk devices would be based on the two FDA-approved obesity devices, which the agency said fall into the high risk category. Those are the Lap-Band adjustable gastric banding system from Allergan and the Realize adjustable gastric band from the Ethicon Endo-Surgery.
FDA also proposed that efficacy be measured using percent total body weight loss instead of the current standard of percent excess weight loss. The panel is part of the Medical Devices Advisory Committee.
Yesterday the U.S. House Energy and Commerce Committee's health subcommittee voted unanimously to pass the PDUFA reauthorization bill. A full committee vote on the draft is expected Thursday and a full House vote is expected later this month.
Medscape Medical News
Pharmacists Dispensing Without Rx 'Scary,' Say Med Societies
Robert Lowes
May 2, 2012 — Imagine a patient walking into a pharmacy, self-diagnosing a cough at a kiosk with medical software, and then getting a drug from a pharmacist in the back of the store, all without a physician's prescription.
The US Food and Drug Administration (FDA) is imagining this scenario, much to the alarm of the American Medical Association (AMA) and other medical societies, which see the beginnings of an end-run around physician authority.
Sandra Fryhofer, MD, chair-elect of the AMA Council on Science and Public Health, said her group continues to "strongly support" the status quo of 2 drug classes — prescription and traditional OTC. In exploring how new technologies and new roles for pharmacists might expand the portfolio of OTC drugs, the FDA should not "undermine the relationship that a patient has with his or her physician."
"In our view, lack of oversight from a practitioner could be a serious concern," said Dr. Fryhofer.
The FDA has not provided any evidence showing that patients with hypertension, hyperlipidemia, or asthma can diagnose and manage these conditions by themselves, she noted. "The balance of medical evidence strongly suggests otherwise." She also estimated that patients would pay more out-of-pocket for drugs that insurers would shift into the OTC safe-use category.
Bobby Lanier, MD, speaking for the American College of Allergy, Asthma and Immunology (ACAAI), raised similar concerns, but more pointedly.
"The new proposals here being discussed are chilling and scary," said Dr. Lanier. "You are facilitating bad behavior by not having (patients) talk to a clinician. In our mind, there is no substitute for a clinician."
Which brings to mind the fact that when the FDA decided to move painkillers and allergy meds OTC, you heard the same dire warnings. Now the same groups are seeking to change an Obamacare law requiring a doctor's prescription for OTC drugs purchased through a Flexible Spending Account. Not necessary and too much of a hassle according to the AMA and other medical lobbies. In Dr. Lanier's words, it's ok to facilitate bad behavior with existing OTC products but not new ones.
The FDA should be commended for promoting more consumer-driven care. As Peter and I demonstrated in our recent survey on cough medicine usage, the vast majority of Americans like OTC access because it makes health care simpler and therefore more likely. Doctors are behind the curve on the consumer revolution. Rather than trying to derail it, doctors should embrace new ways of connecting with patients and finding ways to help consumers help themselves. http://cmpi.org/uploads/File/CMPI_CoughSurvey_KeyFindings.pdf
Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.
