Latest Drugwonks' Blog
I had lunch today with Sir Michael Rawlins (Chairman, National Institute for Health and Clinical Excellence) in his office today. Finger sandwiches were served – but the conversation was meaty.
Rather than sharing any particulars of a private conversation (Chatham House rules, you know), I will say that we were of a mind on many things -- not the least of which is the need for better tools for clinical effectiveness research.
My only “ask” was for Sir Michael to consider traveling to the U.S. to speak at a Center for Medicine in the Public Interest conference on the future of clinical effectiveness. He readily agreed.
Watch this space for more details.
While waiting in the NICE lobby, I picked up a brochure entitled, “How to Change Practice.” There is much in it to debate, but one thing must be stipulated – the quote at the bottom of page 4:
“Change is not made without inconvenience, even from worse to better.”
The words (via theologian Richard Hooker, 1554-1600) are right, but it’s frightening that NICE should choose to quote from Hooker, who is (arguably) best known for his belief in the doctrine of “Justification by Faith.”
But I digress.
In the meantime, have a look at this new op-ed (from the Newark Star-Ledger), "President Obama, health care and "comparative effectiveness research."
Here are the concluding paragraphs to whet your wonkish appetite:
“Another way to make sure comparative effectiveness research is used properly is to follow the Food and Drug Administration's lead in creating a Critical Path Initiative for CER. The FDA's current Critical Path Initiative aims at using the latest scientific advancements to modernize the process through which treatments are turned from laboratory discoveries into useable medical technologies.”
“This model would be just as helpful in creating a modernized comparative effectiveness research program. By utilizing the most up-to-date scientific knowledge, treatment potency could be assessed in a manner that gives the utmost attention to the genetic, clinical, and demographic factors that affect how different patients react to different treatments.”
“President Obama is right to see the potential benefits associated with comparative effectiveness research. As he moves forward with his plan, however, it's imperative that he also be aware of the serious risks that this research poses.”
Blumenthal is also head of an organization called The Institute on Medicine as a Profession (IMAP) which according to it's website "aims to set forth a vision for professionalism in the 21st century and to promote that vision through research and policy initiatives. "
IMAP received a $7.5 million grant from George Soros who made his money in part from wrecking currencies. IMAP is part of a new venture called The Prescription Project, which is funded by Community Catylst, which in turn is funded by the same group that funds the liberal Families USA which also receives money from Soros. The Prescription Project is being funded by the Pew Charitable Trust to the tune of $6 million but is also linked to the Prescription Access Litigation Project through its affiliation with Community Catalyst. That project is comprised of the largest tort lawyers suing drug companies for a variety of reasons.
The Prescription Project is designed to end companies from having any contact with doctors or patients whatsoever. As the project notes: "Public and private payers spend billions of dollars a year on prescription drugs. When these payers rely on information from industry marketing campaigns rather than unbiased scientific studies, the result is higher cost and poorer quality."
Maybe we will only connect to doctors by computer using messages screened for commercial content and judge consistent with materials produced by the Federal Comparative Effectiveness Coordinating Council.
Jesse Goodman, the well-respected CBER director is getting a new job. Actually, two new jobs. He’s been promoted to be FDA’s Chief Medical Officer and will also serve (as the agency’s only representative) on the Federal Coordinating Committee on Comparative Effectiveness – where his ability to think big, think smart, and think fast will be a tremendous asset.
It's good news for Dr. Goodman -- and better news for the public health.
Unnamed "lobbyists" say one thing -- I say, let's give the guy a chance.
Here's what CongressDaily has to say:
Drug industry is wary of Obama's pick for FDA deputy
By Anna Edney, CongressDaily
Food, pharmaceutical and medical device groups have lauded President Obama's pick to lead the Food and Drug Administration, but his selection for second in command is giving the drug industry heartburn.
Industry insiders describe their colleagues as cautiously optimistic about FDA Administrator-nominee Margaret Hamburg, a former New York City health commissioner who works at a nuclear nonproliferation think tank, and nervous in varying degrees about Baltimore City Health Commissioner Joshua Sharfstein.
"
"Companies are clearly going to get more scrutiny."
The drug industry is viewing the dual picks, as one FDA lawyer put it, as a "power-sharing agreement."
Obama emphasized food safety when he announced the selections of
Sharfstein, on the other hand, has a long and sometimes contentious history with the pharmaceutical industry.
"Anyone who spent their career under Henry Waxman ...," said one lobbyist, trailing off with a shudder.
Sharfstein worked under the now-House Energy and Commerce chairman from July 2001 to December 2005, a Waxman spokeswoman said.
Waxman has been tough on drug companies, particularly given several scandals involving contaminated drugs or unheeded warnings in the recent years. Sharfstein has carried the torch on many of his former boss' public health priorities, ranging from cracking down on medication use for illnesses not approved by FDA to criticizing pharmaceutical companies for gifts handed out to physicians.
Sharfstein led the Obama transition team's FDA evaluation and was thought by many to be a shoo-in for the top post at the agency. He raised warning bells as well that led the agency to re-evaluate the safety of cough and cold medicine use for children.
The pediatrician could play a key role implementing 2007 FDA legislation that set up a system for pharmaceutical companies to assess risks associated with their products and come up with a plan to mitigate those risks -- hence the term risk evaluation and mitigation strategy, or REMS. The law affords FDA the discretion to determine how stringent a product's REMS should be, and the drug world fears potentially burdensome REMS could become the norm.
"I think companies are really nervous based on his track record," an industry insider said.
Peter Pitts, co-founder of the Center for Medicine in the Public Interest and a former FDA associate commissioner for external relations, argued the drug lobby's fears are unfounded.
"I don't think you'll see more strident regulations," Pitts said. "I think you'll see a more effective way to use existing regulations."
He anticipates FDA will experience an influx of resources this year that will have the underfunded agency running smoother and more efficiently.
One lobbyist called the new funding on the horizon "a blessing and a curse," adding, "With that money, the question is: What type of additional oversight or regulatory burden [is] not going to be put on just pharma-companies but biotech and medical device companies as well?"
Waxman is a leader in trying to move legislation that would grant FDA the authority to oversee tobacco, a bill Sharfstein worked on with the chairman, as well as his most recent legislation that would allow FDA to approve generic versions of biologic drugs.
Did you miss that? I sure did. I am sure you did too. Know why? Because the New England Journal of Medicine, the New York Times, USA Today, etc, all ignored the findings and ran with this:By initially screening men 55 to 69 years with the PSA marker and offering regular follow up, this led to an increase in early detection. Deaths due to metastasized disease were then reduced. Exact data showedthat on average for every 1,408 men screened, 48 had cancer diagnosed and received treatment, resulting in saving one life. Screening took place on average every four years with a mean follow-up over nine years. The cut-off value was a PSA level of 3.0 ng/ml or more. Men with this reading were then offered a biopsy.
The study shows that PSA screening delivers a 20% reduction in mortality from prostate cancer. This provides decision makers on screening policies with important new data on the effectiveness of PSA testing in preventing deaths.
"The PSA blood test, used to screen for prostate cancer, saves few lives and leads to risky and unnecessary treatments for large numbers of men, two large studies have found."
Mortality Results from a Randomized Prostate-Cancer Screening Trial (The New England Journal of Medicine)
"The findings, the first based on rigorous, randomized studies, confirm some longstanding concerns about the wisdom of widespread prostate cancer screening. Although the studies are continuing, results so far are considered significant and the most definitive to date ..."
Continued Here
"What the European study tells us is that, if you are a man who chooses screening, you are 47 times more likely to be harmed by screening than to have your life saved," said Dr. Otis W. Brawley, chief medical officer of the American Cancer Society.
Where to begin?
Unnecessary and harmed by screening?
What is Brawley thinking? Does the ACS really pay him to say things to reduce prostate cancer survival rates? Does he realize that urological oncology has learned how to segment and stage prostate cancers based on risk and PSA velocity? That the number of cancers detected that undergo watchful waiting have increased even as the more dangerous tumors can be removed successfully to increase overall life expectancy at ever age and that this is possible because of the increase in early and widespread detection in combination of better treatment?
Oh, and did he read the methodology section of the study which controlled for mortality caused by treatment? Guess not.
Read More
Now let's turn to Kolata who's article is as balanced as the Dreyfus Affair. The American study likely understates the impact of screening because the design is biased against minorities who would have higher detection rates of cancers that progress faster and have an oversampling of asymptomatic patients whose slow growing tumors are likely to have a a good prognosis. This bias was further compounded in the American study because the treatment rates in the untreated group were about the same as those in the treatment group:
The other half of the men on the trial were offered “usual care” - meaning ‘whatever their health insurers considered appropriate’. Crucially, this meant that the control group for the study contained men who could also potentially be screened for prostate cancer.
"According to this analysis, over half of men in the ‘unscreened’ group actually received some form of prostate cancer screening (compared to over eight-out-of-ten men in the ’screened’ group)."
In the US trial, some men who were in the unscreened group actually ended up having a PSA test - probably as part of their health insurance or because of suspected prostate cancer symptoms.This may have significantly affected the results, by cutting deaths from prostate cancer in this group, and reducing any differences the trial was designed to show."
Finally, both studies took a one size fits all approach to screening and to PSA levels. In fact, PSA use and levels in the real world are tailored to family history, age and race. Increasingly, algorithms that combine clinical data, PSA levels and other markers such as fusion genes and sarcosine, a metabolite found in urine can be used to more precisely determine whether prostate cancers were benign, localized or agressive.
Read More
Here is info on the PSA Test
So the take away is this: to make policy or impose reimbursement from one clinical trial or even several is fool hardy. To dictate clinical practice and ration screening based on press releases or sound bites is irresponsible.
Then again both the NY TImes and the American Cancer Society are desperately seeking to show the flag on comparative effectiveness. They have done the job well on prostate cancer screening. At the expense of cancer patients and medical progress.
By Robert M. Goldberg
Comparative effectiveness—which is supposedly the “science” of comparing two treatments for the same illness and determining which one provides the best outcome for the least amount of money—is something that at least on the surface should be a process we can all agree on. Who wouldn’t want to use baking soda in warm water for an upset stomach instead of a four-dollar pill?
But these homely comparisons are not why a collection of interests—including insurance companies, managed care plans, government bureaucrats, advocates of single-payer health plans and experts from government-run health systems from Canada, Britain and Australia—have spent millions lobbying for the inclusion of $1.1 billion to create a government run agency to conduct comparative effectiveness studies. And it is not because, as the advocates for this mega-agency promise ( with a budget exceeding the Food and Drug Administration’s allocation on regulating new drugs, vaccines and devices) any decisions would be legally binding on doctors, Medicare, Medicaid or any health plan that would be regulated under Obamacare. In fact, Britain’s National Institute for Health and Clinical Excellence (NICE) has no authority to control what doctors do or what its National Health Service (NHS) pays for. (The managed care lobby, America’s Health Insurance Plans or AHIP, supports giving a comparative effectiveness agency such authority.)
Yet, the NHS now obliges itself to follow NICE comparative effectiveness decisions. And so doctors and patients have to wait years for NICE judgments or more accurately for NICE to say that paying for drugs for osteoporosis, Alzheimer’s, arthritis and cancer just isn’t worth it. Even worse, the whole comparative effectiveness decision-making process now overshadows everything. Any evidence, biological or otherwise that does not go through the government comparison mill lacks “kosher certification” and is regarded as not authoritative. To express pain, pose scientific questions, challenge questionable or sloppy assertions, one must become part of the bureaucratic apparatus or the parasitic lobbying necessary to obtain a “seat at the table.” The establishment of such an agency is dangerous if not done with great humility and humanity. My opposition is based not only on this concern but my experience on how it grinds human life into dust.
Several years ago my daughter battled bulimia. She was hospitalized three times for one month or less. Her discharge had nothing to do with treatment success. It had everything to do with her managed behavioral health plan following the logic of the comparative effectiveness review of the Agency for Healthcare Research and Quality (AHRQ) which will be responsible for turning the $1.1 billion into more reports. Or rather, it was how such reports are written and used that gave the insurer the running room to toss Sara and others like her out of the inpatient setting irrespective of whether they were clinically ready.
For instance, an AHRQ technology assessment, “Management of Eating Disorders,” published in 2006 concluded that the evidence for the effectiveness of combination of treatments for bulimia remains “weak.” It also went on to note that “few factors were found to be consistently related to outcomes.”
It was the process of taking the opinion of a group of health care consultants that evidence was “weak” and giving it the imprimatur of government authority that allowed health plans to limit coverage for eating disorders, claiming they are psychological instead of biological or truly measurable. This happened even without a law linking reimbursement to an explicit recommendation.
In fact, the reasons that “factors” are not “related to outcomes” have nothing to do with the implication that longer treatment is ineffective. The “technology assessment” never states what is well known: that the disease is still not well understood. Worse, it never acknowledges that the 30 day or less discharge contributes to the problem. When Sara was discharged she found herself fighting the urge to binge or purge the sense of shame and drive to perfection common among people with bulimia was amplified and was, sadly, reinforced by parents anxious to avoid a relapse or doing anything wrong.
Sara was discharged the first time after she did not binge or purge in a controlled setting for a few days in a row. Twice more she was discharged not because she was able to get control of her illness but because her blood pressure had been stabilized. Similarly, one of her friends at her eating disorders program had a perforated stomach from so much vomiting. Against the medical advice of her doctor, her insurance company kicked her out after a week because her heart rate was “normal.”
In 2007, Magellan, Aetna and Blue Cross and Blue Shield, three groups leading the push to expand AHRQ’s reach (and whose comparative effectiveness centers would do much of AHRQ's work) were sued. Susan Pisano, the spokeswoman for AHIP, said “there is no research that shows that longer treatment produces better results. In today's environment, the real question has to be: What does the evidence show?" I wonder where she got that excuse.
Advocates of comparative effectiveness claim that studies will be used differently here than in Britain. That’s a lie. In every other setting in the United States, comparative effectiveness reviews have been used to restrict access to new drugs or deny coverage to life-saving treatment more often than not. Comparative effectiveness research as currently construed is not about what’s best for people. It’s about saving money for political purposes. Don’t take my word for it. Ask my daughter.
Here's what Representative Henry Waxman (D, CA) told the American Medical Association the other day: "We all know that we have to get costs under control, but the way to do that is not to tell physicians what they can and cannot do or put them in a position where they cannot put the needs of their patients first.”
Mr. Waxman said this publicly at the AMA’s National Advocacy Conference.
Further:
“Don’t let anyone tell you that what I’m interested in is socialized medicine. I flatly tell you that is not the case … I am not interested in trying to put a public plan in place that would drive out competition.”
Thanks for that – but just what does Mr. Waxman define as “socialized medicine?”
We’ll see soon enough.
Good quotes from Mr. Waxman – but the winner of the drugwonks healthcare reform quote of the week (yes, even though it's only Wednesday) goes to Senator Ron Wyden (D, OR):
"Nobody disputes the fact that there's going to be some startup costs."
Thank you Senator Wyden.
However, there are limits that Connolly's piece did not discuss and which likely will be silence by the IOM panel which is stacked with people who believe that the one size fits all, literature review, take it or leave approach of comparative effectiveness is the key to universal coverage. This (with some rare and sensible exceptions) is a panel of patronage appointees intended to suppress responsible, science based alternatives to evaluating treatment effects. I will have much, much more on this subject in a forthcoming post. But for instance, don't expect the IOM panel, which is stacked to simply implement an AHRQ agenda that they have developed and receive millions from to address the following issues:
Which drug or treatment works best for an individual based on a variety of factors including genetic variation, co-morbidity, life style preference, stage of life?
Moreover, will comparative effectiveness force additional studies before a product is paid for? If so, that will inevitably delay access, raise prices or reduce rates of innovation. And if trials have to be randomized for each subpopulation the cost goes higher still.
The drug effectiveness review studies conducted by DERP fail to control for subpopulations and the literature they look at themselves exclude adjustments for severity of illness, genetic variation, etc. So most "studies" show no difference in drugs for a particular illness when in fact there are substantial variations, particularly in the areas of depression, schizophrenia, hypertension.
Therefore, shouldn't money be spent on tools for personalized medicine instead of one-size fits all guidelines?
That being said, the issue of whether or not food safety and security remains inside the FDA or becomes its own agency within HHS remains as contentious as ever, with Representative Waxman and Representative DeLauro agreeing to disagree.
Mr. Waxman (D-CA) believes that the "first step on the legislative path" should be shoring up food safety and finding ways to prevent further incidents. According to the Pink Sheet, “Waxman did suggest that he would entertain the concept of a bifurcated agency in the future. Once reforms are passed and implemented, he suggested Congress could consider "whether a reorganization is necessary" for food safety enforcement.”
But Representative Rosa DeLauro, (D-CT), the chair of the House Agricultural Appropriations Subcommittee which oversees FDA's budget, has introduced the Food Safety Modernization Act, which would move some divisions out of FDA and form a Department of Health and Human Services agency for food safety. The bill, H.R. 875, has 40 co-sponsors.
That being said, a dangerous bifurcation that must be avoided at all costs is a differentiation in the vision for the future of the FDA between appointees and career staff. Drs. Hamburg and Sharfstein should listen and learn from the agency’s senior staff – most specifically as to how the agency can be both regulator and colleague with the industries it regulates. A fine line to walk -- but a journey worth the effort.
Exhibit A: The Reagan/Udall Foundation and the Critical Path Initiative.
After all, the FDA must work to both protect and advance the public health.
A shared vision, crafted together will succeed.
Unity, yes. Bifurcation, no.
Yes we can.
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.
