In the Spotlight
Hotel California Health Care
Sally C. Pipes, Medical Progress Today, 7-21-05
Single-payer health care is making news in California thanks to State Senator Sheila Kuehl, whose bill to have California’s government run the state’s entire health care system has passed the Senate and will surely pass the Assembly. Her supporters and Californians should look at what is happening north of the 49th parallel, where single-payer health care is also hitting the headlines. If Ms. Kuehl and company are successful, it provides a preview of what’s to come.
Continue reading . . .
U.S. Affirms Support for Vaccines Amid Worries Over Autism Link
Wall Street Journal, 7-20-05
The incidence of autism in the U.S. population appears to have risen sharply in recent years. Experts are largely at a loss to explain whether the increase can be attributed to increased efforts to identify and treat autistic children, or to other unknown genetic or environmental factors. The problem is that too little is known about the causes of the disease. What mainstream experts seem to agree on is that a mercury-based preservative—thimerosol—formerly used in childhood vaccines isn't the source of the problem.
Dr. Gerberding [Director of the Centers for Disease Control] said the predominance of the evidence shows no link between thimerosal-containing vaccines and autism—a report last year by the Institute of Medicine found none—though she adds, "I can't tell you with 100% certainty that there will never be a link." Dr. Gerberding recently met with parents of autistic children and says she understands their frustration at the lack of a known cause. …
Peter Hotez, a vaccine researcher at George Washington University and father of a 12-year-old with autism, said he doesn't believe that there is any link between the condition and vaccines. "We need a war on autism, not a war on childhood vaccines," he said.
It may take several more years and more epidemiological studies (there have already been four) before distraught parents are convinced. In the meantime, there are plenty of firebrands—like environmental activist Robert Kennedy, Jr—who allege that there is an international thimerosol conspiracy (including the Institute of Medicine and the CDC) to protect vaccine manufacturers.
This effort is reminiscent of an attack on the pertussis vaccine that began in 1974 with an alleged link to brain damage. Hundreds of lawsuits and millions of dollars later, the number of vaccine companies making the important vaccine had dropped from four to one—despite the fact that the link was eventually refuted by science. There are currently hundreds of thimerosol suits pending in U.S. courts that may, yet again, push the vaccine industry to the brink of extinction and put millions of children's lives at risk.
Who's fighting cancer in kids?
USA Today, 7-20-05
Making vaccines for children is, as we noted above, an extremely risky proposition, given the enormous potential liability exposure facing companies that vaccinate large populations of (apparently) healthy children.
Making cancer drugs for children is equally difficult for a diametrically opposed reason—because there are so few children who contract cancer. Pediatric cancers are, in fact, so rare that it is unprofitable for pharmaceutical companies to design drugs specifically for pediatric patients.
A government report in April found a "near absence" of research into pediatric cancer drugs. About half of the oncology drugs used to treat children are at least 20 years old, according to the report by the Institute of Medicine, a non-profit group that advises the government on health policy. Most drugs given to children were developed for adults, then passed down to children. In the past 10 years, only one cancer drug, Clolar, has received initial approval for children. …
…adults attract more cancer research than children because they are a far larger and more lucrative market. Patients younger than 20 make up 12,400 of the nearly 1.4 million Americans stricken with cancer each year. Drug companies are generally unwilling to invest hundreds of millions of dollars into such a small market...
One promising approach for developing new therapies for pediatric cancer is to catalogue the genetic abnormalities involved. That is exactly what is being done now in the National Cancer Institute's Pediatric Oncology Preclinical Protein -Tissue Array Project (POPP-TAP). Once these targets have been identified, drug companies can use them to help develop new cancer drugs and identify which adult cancer therapies will work best in children.
As Medicaid Balloons, Watchdog Force Shrinks
The New York Times, 7-19-05
This is the second article in a well-researched series by the Times cataloguing the enormous waste and fraud riddling New York’s $44 billion dollar state Medicaid budget:
New York's Medicaid program pays more than a million claims a day, feeding a $44.5 billion river of checks to radiologists and ambulance drivers, brain surgeons and orderlies, medical centers and corner pharmacies. Many who get those checks pocket more money than they deserve, and millions of taxpayer dollars are believed to be lost every day to theft and waste. Yet the state, charged with protecting those dollars, has done little to stop them from draining away. …
Many experts say that it is likely that at least 10 percent and probably more of New York Medicaid dollars are stolen or wasted.
Albany policymakers have used a lavish system of Medicaid spending to court votes from politically connected health care unions and other providers. Today, New York's Medicaid program is—astonishingly—more expensive than those of Texas and California combined. In response to the Times' expose, Governor Patacki has announced the creation of an independent inspector general's office to help police the program, and appointed a former federal prosecutor to recommend reforms for how the state monitors Medicaid fraud.
Manhattan Institute Senior Fellow E.J. McMahon said in response to the Governor's reforms that "There's no guarantee that a third office dedicated to policing Medicaid will accomplish anything more than the two agencies that were already supposed to be overseeing this area. What we still need, above all, are far more fundamental Medicaid reforms that would weaken the ability of providers to scam the system by giving patients themselves an incentive to spend wisely. The problem may prove to be that the state itself is far too involved in Medicaid administration and should devolve more of this responsibility to private insurers."
Ex-shrimper at vanguard of 'personalized medicine'
Associated Press Newswires, 7-15-05
Oncologists looking for better treatments for pediatric cancer confront the same challenge facing proponents of personalized medicine: how to best uncover the molecular roots of disease and develop the tools to fight it. This challenge is as easy to say as it is difficult to do.
Brad Margus, CEO and co-founder of Perlegen Science, knows this better than anyone. Margus' interest in personalized medicine started when he discovered that two of his three sons had a rare and fatal genetic disorder called ataxia-telegiectasia (A-T) that afflicts only about 500 U.S. children every year:
Since doctors told Margus in 1993 that his toddlers Jarrett and Quinn would soon be confined to wheelchairs and probably dead before they were old enough to vote, the Harvard Business School graduate has been on a mission to save the boys from their genetic fate. …
In the process, he has become a self-taught genetics expert, a lobbying fixture on Capitol Hill and head of his own startup biotech company [Perlegen], making him a learned colleague alongside the field's leading scientists. Margus has helped raise $17 million for the nonprofit patient advocacy group he created called the A-T Children's Project, and he sits down for an occasional national television interview with the likes of Barbara Walters.
Perlegen helps companies personalize their medicines, matching drugs with patients' genetic backgrounds.
A lot is riding on the underlying technology. A more precise understanding of the genetic basis of disease should not only help accelerate the search for new cures, it should lower the cost of finding and testing new medicines, bringing hope to small groups of patients with rare diseases like pediatric cancer or A-T.
Industry Concerned Drug Watch Will Cause Disruptions, Lawsuits
Food & Drug Letter, 7-15-05
As we've seen in articles on autism, pediatric cancer, and A-T, quantifying the underlying mechanisms of disease is extremely difficult, time-consuming, and expensive. What, then, should we make of the FDA's new public communications strategy for adverse drug events, i.e. letting the public know about potential adverse drug reactions early on, when there may be little hard evidence on whether or not the therapy or some other factor was to blame?
Communication of premature and unvalidated safety observations not only has the potential to confuse healthcare professionals, but it could also unintentionally disrupt beneficial treatment, according to Alan Goldhammer, associate vice president of regulatory affairs at PhRMA.
"Well-founded clinical decisions may be compromised as a patient is moved off of one therapy to a second whose therapeutic risk-benefit profile is less favorable," Goldhammer told a June 8 committee meeting of the National Academies' Institute of Medicine (IOM), which has been charged with making recommendations to improve the oversight system of marketed drugs.
The pharmaceutical industry obviously is a stakeholder in this dispute, but they do have a point: correlation of a drug with an adverse event doesn't prove causation. Trumpeting potential risks will, however, spark a lot of litigation while FDA is still hashing out the science. This doesn't serve the cause of patient health or improve our understanding of real risks.
Makers of Race-Based Heart Drug Warn Against Ignoring Genetics
Wall Street Journal, 7-14-05
If there are personalized medicines, are there race-based medicines? Race is an extremely slippery category; it is more accurate to talk about genes. Still, race can be used as a shorthand that can help patients ask questions about underlying genetic or familial risk factors. One new drug, BiDil, for black Americans, is helping to generate public discussion on the interaction between genes and medicine.
When a team of researchers zeroed in on studies that showed blacks responded better than whites to a heart medication, they spent another two years rechecking their work before releasing the findings.
"There was anxiety, I think, that probably made us wait a while until we were absolutely sure from our data that this was a real phenomenon, and not just a play of chance," recalled Dr. Jay Cohn, a University of Minnesota cardiologist involved in the study that was published in 1999.
But the study was clear: blacks responded better that whites to the drug that Dr. Cohn and his colleagues began investigating decades ago. That led to a 2001 clinical trial involving only black patients—and ultimately to last month's approval to market BiDil to blacks, the first medication approved by the Food and Drug Administration for a specific racial group.
NitroMed, the company that created BiDil, is also "examining genetic commonalities among blacks in the study who benefited from BiDil" which may reveal the genetic factors underlying drug response, and transform the drug into a race-neutral therapy that uses a simple genetic test to identify the patients who could most benefit from it.
DTC ADVERTISING VALUE STILL A DEBATE AMONG EXPERTS
FDA News Drug Daily Bulletin, 7-10-05
In early July, Senator Bill Frist (R, TN) called for a voluntary 2 year industry moratorium on direct-to-consumer advertising for new prescription drugs, saying that spending on DTC advertising can "lead to inappropriate prescribing and fuel prescription drug spending…[and] can also oversell benefits and undersell risks." This week, FDAnews offers a balanced look at this much debated industry practice:
Some professionals praise DTC ads for encouraging patients to visit doctors, while others contend unbranded disease awareness ads could achieve the same goal. Market research data shows DTC ads bring patients—who would otherwise go undiagnosed or untreated—into doctor’s offices… Others warn that if DTC ads were to vanish, consumers would still be bombarded by advertisements for nonpharmaceutical treatments, such as vitamins…
Changes in DTC practices are sure to come soon—whether through a voluntary industry code or additional FDA guidances. But any changes should be considered very cautiously. Ruling out branded advertising might make it substantially harder for companies to launch innovative new drugs that compete with established market leaders; ironically, this might lead to higher drug prices and worse health care in the long run.
Who's Minding Medicaid?
The New York Times, 7-20-05
follows up its three part series on New York's corruption-riddled Medicaid program with an editorial calling for reform:
New York's Medicaid program has always had a reputation as the Cadillac of state health care programs. The 4.2 million people in the state's program have access to some of the best and most complete medical coverage in the country. The very fact that it's so generous makes it particularly critical that the public is assured that money is not being wasted. Unfortunately, the opposite seems to be true. As revealed by a year's investigation by Clifford J. Levy and Michael Luo of The Times, New York's $44.5 billion Medicaid program has become a honey pot for unscrupulous practitioners. …
The Times, however, glosses over the fundamental problem with the program: once ostensibly a safety-net for poor New Yorkers, the program is now a "Cadillac" that invites fraud and waste because it has no mechanisms to control demand for services.
Editorials/Op-ED: Vaccines and autism
Washington Times, 7-15-05
The Washington Times
weighs in on the purported link between thimerosol and autism, and how ignorance about the causes of autism has led some activists to pin the blame on their favorite target: corporate America.
If true, as activist Robert F. Kennedy Jr. wrote recently in a provocative Rolling Stone article, and "our public-health authorities knowingly allowed the pharmaceutical industry, to poison an entire generation of American children," then "their actions arguably constitute one of the biggest scandals in the annals of American medicine."
No doubt, but Mr. Kennedy's "conspiracy" so far rests on thin evidence, not to mention a certain degree of ideological spin. …
Studies done…by the National Institutes of Health and the Institute of Medicine have found no evidence that a correlation exists between thimerosal and autism, though a few suggested that further study was needed. European studies have reached similar conclusions. The journal Pediatrics conducted the most recent review of these studies in September 2004 and found that the "studies do not demonstrate a link between thimerosal-containing vaccines and Autistic Spectrum Disorder" and that epidemiological studies "that support a link demonstrated significant design flaws that invalidate their conclusions."
Small Leaps or Giant Steps
Dr. Scott Gottlieb, AEI Online, 7-13-05
Gottlieb points out that calls for more "comparative studies" between new medicines and older drugs misses the clinical differences that are relevant to real world patients:
This mandate for "comparative effectiveness" studies, where new drugs are compared to old drugs, is a standard that is used in Europe. But the European data generated from these kinds of head-to-head trials are often ignored by clinicians here, who argued to me when I was at the Food and Drug Administration that head-to-head trials are often underpowered and therefore do not take full stock of subtle but clinically important benefits such as improved dosing schedules that lead to better compliance or better side-effect profiles, which, in turn, allow safer administration of medicines.
Underpowered head-to-head trials do not reveal the small but important benefits offered by incremental advances in medicine. They often end up proving the null hypothesis, that there is no difference between two drugs, even when clinically important differences exist. Nonetheless, the policy change Dr. Angell and others advocate would reduce the total number of drugs approved, as well as the availability of back-up drugs that provide alternatives to medicines and create competition that lowers prices.
Medicaid's Untallied Costs
Michael Cannon, Cato Institute, 7-1-05
Cannon has written a thoughtful article cataloguing the parallels between welfare reform and Medicaid—and why Medicaid reform will also help empower the poor.
A significant body of research suggests that, as it exists today, Medicaid encourages recipients to become dependent on government; encourages people to behave in ways that increase the cost of government and of health care, which makes self-reliance more difficult for their neighbors; and encourages state policymakers to get more people to behave that way. A worthwhile attempt to cut Medicaid costs would look beyond state and federal budgets and seek to minimize other costs as well.
Fortunately, the federal government has a roadmap for doing just that. In 1996, it ended the entitlement to federal cash assistance; block-granted to the states funds that were previously given out in proportion to what each state spent; and gave states greater flexibility in setting eligibility and benefits. The idea was first to stop encouraging states to foster dependency, and then to give states the flexibility they needed to discourage dependency instead.
Gene Therapy for Brain Tumors Shows Promise in Animals
Gene therapy, whereby a harmless virus is used as a vector to insert new genes into patients or to attack invading bacteria or cancer cells, has been a technology that has held much promise but scant performance, until now. This article reports findings in the July 20th issue of the International Journal of Cancer that:
Recombinant adeno-associated virus expressing tissue factor pathway inhibitor (rAAV-TFPI-2) inhibits invasion, angiogenesis and tumorigenesis of human glioblastomas in culture and in murine studies…
TFPI-2, a serine protease inhibitor, is underexpressed in malignant gliomas, Dr. Niranjan Yanamandra and colleagues from the University of Illinois College of Medicine, Peoria observe. Previous work has shown that overexpression of this protein in malignant glioma cells inhibits the invasive potential of tumor cells in vitro and in vivo.
Dr. Yanamandra's group now reports that successful rAAV-TFPI-2 infection of SNB19 malignant glioma cells significantly reduced migration and invasion of these cells in a dose-dependent manner. Invasion is the primary cause of therapeutic failure in the treatment of malignant gliomas. …
"In vivo angiogenesis data substantiate the in vitro data that TFPI-2 expression effectively inhibits new vessel formation," Dr. Yanamandra and colleagues note.
These data, they conclude, suggest that "targeting angiogenesis may have therapeutic value in the treatment of malignant gliomas."
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