Latest Drugwonks' Blog
Get ready Krug. Spring training is nearly over. http://www.nytimes.com/2008/11/03/opinion/03krugman.html?ref=opinion
To view the interview, click here.
And to view Dr. Sarkozy's PowerPoint presentation, click here.
This issue is becoming ever more urgent when you consider today's news from the UK that NICE (the National Institute for health and Clinical Excellence) has extended until January its assessment of four kidney cancer drugs, none of which was deemed cost-effective in a draft appraisal issued in August .
NICE, which "assesses" technology for the national health service in England and Wales, said that bevacizumab (Roche's Avastin), sorafenib (Bayer's Nexavar), sunitinib (Pfizer's Sutent) and temsirolimus (Wyeth's Torisel) did not represent the best use of NHS resources when used to treat advanced and/or metastatic RCC, despite acknowledging their clinical effectiveness in some settings and the narrow choice of other treatment options.
Is this what we want in the US? Because this is what (at least in the current context) "comparative effectiveness" means.
What we need are 21st century assessment tools for a personalized approach to the "4 rights" -- the right medicine in the right dose for the right patient at the right time. In other words, tools for patient-centric rather than cost-based comparative effectiveness.
Consider some of the arguments from the pages of the Journal of Life Sciences.
Attention must be paid.
http://www.cnn.com/2008/HEALTH/10/30/ep.doctors.prescribe.placebos/
Next, rather than paying doctors for keeping people healthy or simply being there, health plans are -- even as legislative and ideological forces seek to eliminate the role of drug, biotech and device companies in discussing products with physicians -- filling the void with dollars and demands to swtich patients or start them on generic drugs. Nothing wrong with generic drugs, they are saving Medicare billions, but ultimately we want to get to point where prescribing is based on value, not just cost. But getting information about that will require close and open communication between everyone.
http://ap.google.com/article/ALeqM5hsuaRf6mJu1lKskznFkkD9tHg6MAD944C4HG0
So you see, both power and knowledge abhor a vaccuum. And it will be filled by those pushing a financial and political agenda, such as trial attorneys and the consumer groups that benefits from their largesse, medical quacks and shakedown artists (like Steve Nissen).
How does this pentultimate preemption affect patients?
The most recent study from my former perch at the Center for Medical Progress at the Manhattan Institute (home to many solid Yankee fans!) tells the tale. The analysis, conducted by Columbia University Frank Lichtenberg is entitled:
Alive and Working: How Access to New Drugs has Slowed the Growth in America’s Disability Rates
It concludes that "access to pharmaceutical innovations has been responsible for keeping large numbers of U.S. residents off disability rolls who otherwise would have joined them."
The study shows that if " the average vintage of drugs prescribed since 1995 and paid for by Medicaid had not become more recent, the rate of increase at which working-age people were classified as disabled would have been 30 percent higher than it actually was, resulting in 418,000 additional people receiving disability payments in 2004. Social Security benefits paid to this population would have been an additional $4.5 billion."
Are generics really the best value for Americans? For millions of us, of course they are. But to say it's better to prescribe generics in all cases is to suggest that the only valuable thing doctors do is not to prescribe new drugs...
Better drugs (the right drug for the right person!), more time with doctors. That's a much better bang for the buck than what the social engineers and health policy elite have in mind.
http://www.manhattan-institute.org/html/mpr_07.htm
(I represented the "pro"/public health side of the argument. The "anti"/tort lawyer argument was proferred by Brian Wolfman of Public Citizen.)
Here's a link to the segment.
Have a look and judge for yourself.
Janet Woodcock, MD, Director, CDER, FDA
“Safety means doing the right things for patients.”And you would have missed this penultimate statement from the flurry of memos (I read them all)
"I agree with the idea that we should preempt state requirements for labeling of drugs. It makes no sense for us not to have a federal system for labeling approved drugs that is based on a careful scientific review of the available data and a consistent application of labeling policies across products. I see this as a legitimate FDA area of involvement given our statutory authority over the drug approval process."
That was John Jenkins. And not one of the memos take issue with the position that FDA should preempt state requirements, especially those dredged up from tort proceedings.
The issue was and is about whether the FDA had enough control over labeling and safety monitoring in a coordinated fashion to produce the appropriate balance between risk and benefit, patient rights and company behavior. The goal is to encourage companies to do more to make sure labeling reflects the most up to date science and is communicated clearly and quickly to patients and everyone else to improve the use of the product and public health. The idea that FDA officials and their words are being twisted to promote a trial attorney agenda is disgusting beyond imagination...
Jenkins seeks a compromise which is now part of the fabric of the Critical Path and FDA authority under FDAAA:
"If we are going to go this route to protect sponsors, the appropriate balance in the favor of public health may be to give FDA more authority over labeling."
Which is exactly what has happened since 2003 when the memos were written. Where oh where have the self-serving solons at JAMA and NEJM been the past five years. They keep flogging VIoxx as an example of why tort lawyers should have control over labeoling. But within the FDA Vioxx was and is regarded as a safe drug under certain conditions that no longer apply since the drug was withdrawn:
Jenkins again:
"Had Merck not decided to voluntarily withdraw the product it would have been necessary for them to make labeling changes to add warnings about ,the new findings. We probably would not have issued an approval letter on the labeling changes since we would not have had access to the full study report for review so we could evaluate the actual data that would be added to the labeling. So, we probably would have agreed to interim labeling and asked Merck to submit it as a CBE and we would have made more official determinations later after we reviewed the full study report. "
It was the withdrawal of Vioxx and the failure to work with the FDA to make labeling changes that triggered the "Vioxx scandal" and the ensuing mess. Waxman wants us to believe FDA officials want more of the same instead of a science-based approach to label changes.. The Waxman report is all about manipulating the truth and is an example of how "facts" are treated in the courts that he and others would want to determine what is said about medicine and how it's practiced.
I am still colelcting my notes and my thoughts and will report on what the various speakers (including the FDA's Janet Woodcock, Jane Axelrad) had to say. In the meantime, let me commence this reportage with my own opening remarks:
When I told my wife -- who is not in this line of work -- that I was chairing a conference on REMS, she asked me what I was doing at an event about rapid eye movement.
A common question I get about REMS is – how is it different from what we used to call RiskMAPS? I see two main differences. The first, obviously and importantly, is that REMS has actual legislative language. And that’s an important detail – but it’s one-dimensional.
While I agree that REMS does indeed represent an expansion of the FDA's authority, I do not agree that it is either ill advised or an over-extension of the agency’s purview.
Their latest attempt to muddy the waters of FDA labeling authority comes in the form of internal FDA documents that, Waxman staffers claim, show career officials oppose agency regulations that weaken consumers' ability to sue drug makers.
Here’s one quote they’re using to make the case for “the FDA doesn’t believe in preemption" ...
"Much of the argument for why we are proposing to invoke preemption seems to be based on a false assumption that the FDA approved labeling is fully accurate and up-to-date in a real time basis," the report quoted Dr. John Jenkins, who oversees FDA's new drug reviews, as saying. "We know that such an assumption is false."
Nobody ever claimed that any FDA label is fully accurate. Such an assumption is indeed false – but nobody has ever said different. What supporters of preemption do say is that the FDA carefully considers the scientific evidence relating to any proposed warning, as well as the public health consequences of including or omitting particular language from drug labeling or advertising. Those who support preemption believe that a drug’s FDA mandated label deserves deference from courts and juries applying state tort law.
John Jenkins is right. But that doesn’t make preemption wrong.
The next “smoking gun” statement comes via Jane Axelrad, an associate director for policy at the agency, who wrote: "We rarely find ourselves in situations where sponsors want to disclose more risk information than we think is necessary," she said. "To the contrary, we usually find ourselves dealing with situations where sponsors want to minimize the risk information."
Jane’s right too. But that doesn’t change the argument for preemption. Of course drug companies would prefer to minimize risk information – and they make their arguments based on sound science. Sometimes the FDA agrees, other times they do not.
Labeling can and must be a valuable tool for improving and protecting America’s health. That’s the law. The FDA is concerned that current labeling practices are in direct counterpoint to the principle objective of current regulations, to facilitate a doctor’s ability to “rationally prescribe” any medical product approved by the FDA. Rational prescribing occurs when a health care professional orders an approved prescription drug or biological product in circumstances where the risk/benefit profile of the product is optimal. The FDA’s most potent weapon in the battle for accurate, timely, “rational,” prescribing is clear, approved labeling.
The FDA’s legal and legislative authority over labeling for prescription drugs and biological products is plenary. The Federal Food, Drug, and Cosmetic Act establishes mandatory and prohibited labeling content and manufacturers have no choice but to comply with these requirements. Less obvious, but equally important, is the principle that the Act also constitutes a “ceiling.” In other words, a manufacturer cannot add risk information to labeling unless the FDA has specifically granted its permission.
The FD&C Act clearly gives the FDA the authority to decide whether or not a product, when used properly, is safe, effective, and properly labeled. A product, used as described in FDA-approved labeling, should be considered safe and effective throughout the United States. Preemption is a well-recognized doctrine of constitutional law derived from the Supremacy Clause and analyzed by the Supreme Court in several cases. Federal law preempts state law here because FDA intends for federal regulation to be exclusive and preemption does not exceed statutory or constitutional limitations.
The “leaked” documents being aggressively peddled to the media change the sound legal and public health argument for preemption not a whit. What it does show is that there is a robust and collegial debate within the agency on this matter. Taking these conversations out of context, as is now being done by members of Mr. Waxman’s staff, just shows how desperate they are – and how weak their intellectual arguments.
"Honest differences are often a healthy sign of progress. "
-- Mahatma Gandhi
But – and it’s a big but -- NICE, the National Institute of Clinical Excellence, is expected to introduce a big increase in the threshold to assess whether such treatments for relatively rare conditions are cost-effective.
In other words, NICE is going to even further restrict access to critical care – particularly for cancer patients.
Alan Johnson, the health secretary, told the Daily Telegraph at the weekend he wants "a fair system that doesn't deny people essential treatment unduly.”
Welcome to “benevolent government healthcare.”
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.
