Selected news articles which highlight important policy issues.

News: Weekly Archives

News for the week of 12-12-2006

Mother's Tale: As their Babies Tested New Drug, A Friendship Grew
Wall Street Journal, 12-12-06

Editor's Notes:

This is a moving story about two children, from very different families, suffering from Pompe disease—a rare and fatal genetic disorder. Their parents meet after they enroll their children in a small clinical trial testing an experimental treatment. It is a story of hope and tragedy, but also of learning how to confront death with dignity and grace. Read the whole thing.

Deb Koncel, a Chicago office administrator, and Jean Kelly, a bank manager from a small Iowa town, met seven years ago, as their sons fought for their lives in a tiny clinical trial of a drug at Duke University Medical Center. The women forged a bond even as their babies veered in different directions—one growing stronger and the other rapidly weakening.

They returned to Duke this year to celebrate regulatory approval of the first treatment for Pompe disease, a genetic condition that causes muscles to rapidly waste away. A dozen other families who participated in the pioneering trials came too, from as far away as South Africa. Some brought their children who were saved. Others brought only memories.

For readers who would like to learn more about Pompe disease, Geeta Anand, the author of this powerful article, has written an entire book on the subject: The Cure: How a Father Raised $100—and Bucked the Medical Establishment in a Quest to Save His Children.

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Novartis Test Drug Shows Promise in Blood Cancer; Tasigna Aids People Who Don't Respond to Gleevec Therapy
Wall Street Journal, 12-12-06

Editor's Notes:

Gleevec, made by Novartis, is a breakthrough cancer treatment for chronic myeloid leukemia. But researchers from Novartis and other companies have improved on the drug's original design and created new medicines to treat the small cohort of patients whose disease is Gleevec–resistant. So far, Novartis' new drug Tasigna is living up to expectations:

Swiss drug maker Novartis AG said an intermediate–stage study showed that its experimental drug Tasigna helped many blood–cancer patients who no longer respond to treatment with its Gleevec drug.

Patients in the chronic phase of chronic myeloid leukemia were given Tasigna for six months. In about half of 279 patients, Tasigna significantly reduced or eliminated the presence of blood cells containing a defective chromosome that characterizes the disease. About three–quarters of patients achieved normal white blood cell counts.

All of the patients had developed resistance to Gleevec—the standard treatment for the condition—or didn't tolerate Gleevec. The study was presented at an American Society of Hematology meeting in Orlando, Florida.

Hagop Kantarjian, chairman of the leukemia department at the University of Texas M.D. Anderson Cancer Center, who helped carry out the Tasigna study, said the data show "we have a very safe and effective second–line therapy for people who have failed" on Gleevec.

Gleevec, launched five years ago as one of the first of so–called targeted cancer drugs, is Novartis's second–best selling drug with sales of $2.17 billion in 2005.

Last summer, the FDA approved Sprycel, from Bristol-Myers Squibb, which is also designed to help Gleevec–resistant patients. The rapid development of drugs for Gleevec resistant leukemia is a powerful example of how new sciences are helping researchers to quickly improve on existing cancer therapies. For more on how researchers are developing follow–on therapies for Chronic Myeloid Leukemia go here.

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Detecting Alzheimer's Early: Scientists Make Progress Toward Test for the Disease
Wall Street Journal, 12-12-06

Editor's Notes:

The Alzheimer's Association says that "detecting biomarkers specific to a disease can aid in the identification, diagnosis and treatment of affected individuals." The hope is that if researchers can find a biomarker (for instance, a protein in the blood) for Alzheimer's, then we can start treating patients earlier, when it may do more good, and could perhaps even accelerate the search for a cure.

The Journal reports that researchers in the U.S. and Europe are narrowing their search for Alzheimer biomarkers to several likely candidates.

Over the past two years, rival scientists in the U.S. and Europe have identified a series of proteins, known as biomarkers, whose presence in blood or spinal fluid may indicate whether a patient has Alzheimer's, the most common form of dementia. In the short term, that could lead to better and earlier diagnosis of Alzheimer's patients. In the longer term, it could yield more–effective drugs and even reduce the cost of developing them.

The search for Alzheimer's biomarkers "is a hot field, and the goal is a very big one," says Simon Lovestone, a professor of geriatric psychiatry at King's College London, who led a team that recently found two such biomarkers in blood. "It's a step along the way to better treatment."

Both the European Commission and the U.S. National Institutes of Health are funding large–scale projects on Alzheimer biomarkers. In a new paper appearing today in the Annals of Neurology, researchers from Cornell University in Ithaca, N.Y., and Weill Cornell Medical College in New York describe how they have identified 23 biomarker proteins for Alzheimer's in a trial of nearly 100 patients. The results need to be replicated in a larger clinical trial—and plenty of challenges remain before the findings can become the basis for a reliable clinical test.

Still, the study is a "good start," says Susan Molchan, who leads a new $60 million project at the NIH's National Institute on Aging, which is also seeking to identify Alzheimer's biomarkers. "We want to identify patients earlier and treat them earlier," she adds. Dr. Molchan has read the Annals of Neurology paper but wasn't involved in the research.

Alzheimer's is a progressive brain disorder that can cause memory lapses, fuzzy thinking and difficulty in performing simple activities, such as dialing a phone number. Existing drugs only treat symptoms; there is no cure. About four million mostly elderly Americans have the disease. As the population ages, the figure isexpected to soar to about 14 million by 2050, unless new ways are found to prevent or treat the illness, according to the NIH.

Validating these biomarkers is the next important step. Once that happens, an effective diagnostic test may only be a few years away.

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The Rush to Biomarker Tests
Wall Street Journal, 12-12-06

Editor's Notes:

While the previous Journal article focused on the quest for Alzheimer's biomarkers, this one expresses some concerns about the "rush" to discover (and market) biomarkers for a wide variety of ailments, from cancer to kidney stones.

Physicians and patients have long dreamed of a simple blood test that could instantly detect cardiovascular disease, cancer or other serious illnesses in their earliest, most treatable stages. No more probes up the colon or down the esophagus. No more waiting for symptoms to show up, by which time it's often too late. And unlike much–hyped DNA tests, a diagnostic blood test would reveal the definite presence of an illness, not just whether you have a chance of one year in the future.

Biomarker–based tests aim to detect proteins and other substances in the blood that, at elevated levels, suggest the presence of disease. The latest advance in this arena—a study published today showing that 23 proteins may be used to diagnose Alzheimer's disease—is still years away from a practical, consumer test. But other biomarker-based tests are already being offered. The best known is the prostate specific antigen (PSA) test for prostate cancer, but hundreds more are in various stages of development.

Federal agencies, cancer centers and biotech companies are making big bets on biomarkers, with the National Cancer Institute spending $135 million to identify new cancer biomarkers, and medical labs such as Roche Diagnostics, a division of the Basel, Switzerland–based drug giant, investing millions more.

Thousands of proteins can be detected in serum, says Tim Jaeger, head of medical and scientific affairs at Roche Diagnostics, "and a major effort is under way to identify and associate the level of these proteins with disease."

First out of the blocks is a diagnostic blood test based on 250 proteins whose levels indicate the presence of disease ranging from autoimmune conditions and cancer to osteoarthritis and liver problems, according to the test's manufacturer. Developed and marketed by privately held Biophysical Corp. of Austin, Texas, the "Biophysical250" test was first offered in February. So far, about 500 people have ponied up the $3,400 for it. The company sends a nurse to customers' home or office, or customers can have blood drawn and send it in to the company.

One concern—that the market will be flooded with unreliable tests—is probably overblown. Insurers will only cover the tests once they have been validated and have shown some medical utility—i.e., cost effectiveness.

In the meantime, if wealthy consumers want to spend several thousand dollars on a dubious medical test they can—along with plastic surgery or two weeks at Canyon Ranch playing golf. Companies that want to enter the mass market and have their products embraced by mainstream physicians and insurers will have to have their tests validated by independent laboratories. If entrepreneurs are pouring money into biomarker research in the hopes of catching the market early, so much the better.

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Medicare Links Doctors’ Pay to Practices
The New York Times, 12-12-06

Editor's Notes:

Shortly before it adjourned, Congress passed legislation forestalling a cut in Medicare physicians' fees. The legislation also included a provision offering a small payment bonus to doctors who report quality data to the Center for Medicare and Medicaid Services—a tactic known as "pay for performance."

After years of trying to rein in the runaway cost of the Medicare program, Congress has decided to use a carrot instead of a stick to change doctors' behavior.

Doctors had been fearing a pay cut under Medicare, the health care program for 43 million elderly and disabled, but Congress instead has offered doctors a small bonus with big strings attached. To get the money, doctors will have to report how often they provide quality care, as defined by the government. Lawmakers approved the change as one of their final acts before adjourning early Saturday morning, and proponents said it would improve the quality of medical care.

But the plan immediately raised concerns among some doctors and lawmakers who specialize in health issues. They said they worried that it could be a step toward cookbook medicine and could erode the professional autonomy of doctors.

Doctors had been facing a 5 percent cut in Medicare payments in 2007. Congress deferred the cut, freezing doctors’ payment rates instead.

Now, doctors can qualify for a 1.5 percent bonus in the second half of 2007 if they report data on the quality of their care, using measures specified by the government. For example, doctors could be asked to report how often they prescribe a particular drug after a heart attack or how well they control blood pressure in patients with diabetes.

With these statistics, Medicare officials say, they will, in the near future, be able to reward doctors who follow clinical guidelines and perhaps penalize those who flout such standards without justification.

For several years, Medicare officials have advocated a pay–for–performance system, noting wide regional variations in the practices of hospitals and medical specialists. The idea was supported by the Bush administration and by Senators Charles E. Grassley, Republican of Iowa, the chairman of the Finance Committee, and Max Baucus, the Montana Democrat who will be chairman next year.

"Medicare now pays the same amount regardless of quality," Mr. Grassley said. Indeed, he said, Medicare "rewards poor quality," paying doctors to treat complications caused by their own mistakes.

Some critics are concerned that government will encourage too much rote medicine. The easiest way to alleviate concerns about "cookbook" medicine is to set broad, risk–adjusted parameters for conditions like diabetes and heart disease, and then let providers decide how to best achieve those goals. This would encourage innovation without impairing doctors' autonomy.

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The more you pay, the better the care? Think Twice
The New York Times, 12-17-06

Editor's Notes:

The New York Times reviews evidence showing widespread disparities in health care costs across the U.S., including studies indicating that additional health care spending does not necessarily result in better health.

EXPERTS have long been puzzled by the existence of large regional disparities in medical care in the United States. Even for diseases for which the appropriate treatment is widely accepted, doctors across the country take vastly different approaches, often leading to enormous expense without making any appreciable improvement in their patients' health.

Consider heart attacks. Prescribing beta blockers immediately after a heart attack is a well–established, cheap and efficient treatment. In Iowa, nearly 80 percent of victims in 2000 received the drugs within 24 hours of a heart attack. In Alabama or Georgia, by contrast, fewer than 6 out of 10 patients received the drugs.

"What makes the lag in beta–blocker adoption puzzling is that the clinical benefits have been understood for years," wrote Jonathan S. Skinner and Douglas O. Staiger, economists at Dartmouth, in a recent study about these regional patterns.

Congress has decided that some treatment decisions may be best taken out of doctors' hands. In one of their last acts this year before adjourning, lawmakers passed a bill entitling doctors to a bonus from Medicare if they report data on the quality of their care, using criteria like whether they prescribe aspirin or beta blockers to heart attack victims.

In the future, this data would permit Medicare to reward doctors who followed government guidelines. Many doctors criticized the decision, saying it would impose a form of medicine by cookbook that could endanger patients. Still, some experts contend that this form of accountability is a necessary step to deal with inefficiencies that riddle the health care system and fuel much unnecessary spending on care.

Several new studies suggest that there is no relationship between the amount spent on treating a patient and the quality and outcome of the care.

Consider chronically ill elderly patients in the last two years of their lives. According to a comparison of hospitals across the country done by researchers at Dartmouth, if the patients die in a hospital in New York State, the average cost of those two years would be $38,369. In Florida, by contrast, it would be $29,604, while in Iowa it would be only $23,746.

The entire article is worth reading. But we should keep in mind that U.S. health care markets are dominated by third-party payers. This means that the link between health and health care spending is severed because the end user of the system—that is, the consumer—has no incentive to limit consumption or seek out the most cost effective treatments. At the same time, government price controls in Medicare give physicians perverse incentives to game the system, over–using some services while shortchanging others.

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The Future of the FDA
The Scientist, 12-18-06

Editor's Notes:

The Scientist offers an insightful look at the future of the FDA—at least for the next year. And there are plenty of regulatory clouds on the horizon.

While Americans are broadly unhappy with rising health care costs, the pharmaceutical industry has become public villain No. 1 because millions of Americans without health insurance find themselves paying retail prices for prescription drugs. Congress is sensitive to this backlash, and drug safety is a convenient scapegoat issue for politicians eager to show that it can "get tough" with industry through increased regulations.

Some new initiatives—particularly new FDA funding, and looking at better ways to detect adverse drug events postmarket—are well worth looking at. Others may make drug development even harder and more expensive, without offering real safety improvements.

The Food and Drug Administration celebrated its first century in 2006, but as 2007 begins, it is also stepping into the cross–hairs of a new Democratic Congress. Bolstered by a public that seems increasingly worried about the FDA's ability to protect it, the Congress is eager to leave its mark on the agency. "There is a confluence of legislation in the coming year," says Scott Gottlieb, deputy FDA commissioner for medical and scientific affairs. "There are some big, must–pass bills, and that will create an environment where a lot of people will be proposing a lot of different ideas."

Key among the FDA reform bills will be legislation by Senators Chuck Grassley (R–Iowa) and Chris Dodd (D–Conn.) to give additional resources to the FDA's drug safety office and make it structurally independent of the agency's drug–approval division. This would remove conflicts of interest, a problem identified in several government studies but which the FDA denies exists. The Grassley–Dodd bill (S 930) also would give the agency more teeth to demand that manufacturers conduct postmarketing surveillance and other measures to track safety issues of newly approved drugs.

Reform seems probable because of another development: The Democrats likely to assume leadership of key congressional committees (and therefore amendable to reform measures) include perennial FDA critics Rep. John Dingell (D–Mich.) for the House Energy and Commerce committee, and Sen. Edward Kennedy (D–Mass.) for the Senate Health, Education, Labor & Pensions committee. The PDUFA bill will go through these two committees. Another FDA critic, Rep. Henry Waxman (D–Calif.), is in line to head the House Government Reform committee, from where he will confront the FDA on several issues, including a slackening of enforcement efforts and elevating what he calls "politics over science," as evidenced by delayed approval of the Plan B contraceptive. "It is safe to say he will be pursuing his initiatives to reform the FDA," says Waxman spokesperson Karen Lightfoot.

Senate staffers say other likely areas of reform next year will address the managing conflicts of interest among FDA scientific review committee members, and giving the agency greater oversight of clinical trials. Review committee members recommend whether new drugs can be marketed, and they are often academics and researchers who have received money from drug companies for clinical investigations or expert opinions. This has led to the perception, at least, of conflicts and bias and in some cases has eroded public perception.

Rep. Maurice Hinchey (D–NY) is likely to reintroduce legislation that would prohibit FDA from convening any financially conflicted reviewers, although passage of such a draconian measure is doubtful. The IOM recommends that 60% of these panelists be free of significant financial conflicts, while Furberg and colleagues say 50% would suffice.

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Project FDA.


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