Drawing a Bead on Side Effects
Business Week, 4-11-05
Drug therapy—like all medical treatments—has risks. As new technologies develop, however, researchers are giving doctors an increasing ability to mitigate those effects. Sometimes this means using diagnostic tools to monitor or identify patient groups at high risk for side effects. Other times, old drugs will be redesigned so that they are less toxic to critical organ-systems.
The battle against harmful side effects is emerging as a promising new frontier for the drug industry. Some companies..aim to remove noxious chemicals. Others hope to salvage bad drugs by improving the way the body digests them. Yet others use what they have learned about side effects to overhaul their development process. Together, these strategies could resurrect hundreds of pharmaceutical flameouts—drugs that were pulled from the market, or never made it out of the lab because of adverse reactions.
New tools for reducing toxicity gives patients and physicians more treatment options. It also helps the pharmaceutical industry by allowing it to capture revenue streams from drugs once thought expensive failures.
Let There Be Light.
Cancer drugs are by definition toxic. In fact, some of the very first cancer drugs were derived from a fearsome chemical weapon (mustard gas) used in the trenches of World War I.
Consequently, cancer oncologists face a deadly dilemma: how to use chemotherapy to kill cancer cells without killing the patient first. Researchers are addressing this problem by designing targeted cancer treatments—the medical equivalent of a precision-guided “smart bomb.” Some targeted drugs (like Gleevec) attack genetic abnormalities in cancer cells while sparing healthy organs and tissues. Other treatments use novel delivery methods to inject their fatal payloads directly into tumors. An example of this technology is a new prostate cancer drug called Tookad.
[Tookad, Hebrew for light] is an innovative twist within the established cancer drug class called photodynamic therapies. These drugs work their way through the bloodstream but become toxic only when exposed to light. When doctors shine a laser onto either the skin or an internal tumor using catheter-inserted optical fibers, the drugs kill just the illuminated tissue and leave unexposed tissue relatively undamaged.
Tookad may represent an advance over current photodynamic drugs since it “degrades easily in the liver and exits the body within hours.” According to one researcher studying the drug, “[Tookad] is one of the most promising treatments for recurrent prostate cancer after radiation I’ve ever seen.” Clinical trial results for Tookad should be available early next year.
Smart Drug for Lung Cancer May Be Pulled From Market: Iressa Helps Small Portion of Patients
The Boston Globe, 4-5-05
As we discussed earlier, targeted drugs will revolutionize cancer treatment. But is the FDA leading the revolution or slowing it down? The FDA “is geared toward finding drugs that work broadly across the population.” Targeted drugs that benefit small groups of patients “may be slipping through the cracks.”
The FDA’s regulatory gold standard—large placebo-controlled clinical trials—is designed to identify statistically significant outcomes. In this system, a very small difference between thousands of patients—half of whom take a drug, half a placebo—can be shrugged of as random variation. If this happens, the drug fails.
The cancer drug Iressa is the poster child for this problem. It “failed” in its pivotal clinical trial—even though physicians know that it is very beneficial for a small group of patients.
The Iressa saga…underscores the challenges the FDA and cancer doctors will increasingly face, as researchers churn out precision cancer drugs that benefit subsets of patients but do little or nothing for others, with delicate trial and error required to match the right patients with the right drugs. Pulling [Iressa] off the market could deny a helpful treatment to a subset of patients who have experienced remarkable recoveries with the drug.
The FDA shouldn’t hesitate to approve more drugs like Iressa. Market approval will spur companies to develop genetic tests that will help match the right drug with the right patient. Eventually, the FDA’s old “gold standard” will make way for a new one—truly personalized medicine.
Childbirth for Bargain-Hunters: Pregnant Women Take Lead In Negotiating Over Prices Amid Cutbacks in Coverage
Wall Street Journal, 4-5-05
What consumer-driven health care needs to flourish are consumers dedicated to finding the best bargains. And it would be hard to imagine a more dedicated group than expectant mothers. As maternity health care coverage becomes increasingly expensive, mothers-to-be are demanding that hospitals and obstetricians compete on price and service.
As companies boost premiums for family coverage—and in some cases drop dependents altogether—a growing group of women lack health coverage for pregnancy, one of the biggest medical costs they face. Others are in some type of consumer driven high-deductible plan, with a Health Savings Account, that gives patients incentives to shop around.
Consumers are learning that if you can bargain with a car dealer for a $30,000 Mercedes, you can bargain with a hospital for a $5,000 maternity bill. One patient received a line-item breakdown of “what her previous insurer paid for her last pregnancy” and demanded that her hospital and doctors give her the same discounts. They did. Policymakers should take note of this development and encourage it by giving consumers easy access to information on quality and costs across providers and insurers.
Directing the Poor to Free Medicine
Wall Street Journal, 4-5-05
Pharmaceutical companies are expanding access to critical prescription drugs for low-income Americans without health insurance.
The new initiative, called the Partnership for Prescription Assistance, will direct patients or people who know them to a toll-free number or an Internet site for help. After a phone interview, callers will receive forms by mail for the drugs sought, while those completing a questionnaire online can print out forms. The forms are then taken to a doctor for final approval.
Standards for the program vary depending on the company that makes the drug; “generally, the income cutoff is about two times the poverty threshold, which for a person younger than 65 was $9,827 last year.” In 2004, the industry gave away over $4 billion in drugs (wholesale cost) and is launching a national ad campaign to help expand the program.
Will this solve the problem of affordable health care? No. But America’s health care woes aren’t the fault of industry.
Doctors Lobbying to Halt Cuts to Medicare Payments
The New York Times, 4-4-05
Medicare and Medicaid were engineered in an era when constituents in the programs were few and the services they used cheap. Now they serve millions of Americans, and their payment model rewards physicians and hospitals for treating patients without regard for the quality or appropriateness of the treatment provided.
Policymakers could head off the fiscal crisis facing both programs by changing how the nation reimburses care for the poor and elderly. If they do, they will face enormous opposition from entrenched interests.
Doctors are mobilizing a nationwide lobbying campaign to stave off cuts in their Medicare fees as Congress hunts for ways to rein in the soaring costs of the insurance program…Doctors said that if the cuts took effect, they would be less likely to treat Medicare patients because the payments would not cover the costs of care.
Neither across-the-board cuts nor across-the-board increases are appropriate in a system where quality varies widely. Indeed, “the chairman of the Medicare Payment Advisory Commission, an independent panel, said [the formula for paying doctor’s fees] had severe flaws.” In fact, it mandates “a national goal for spending with ‘no incentive for individual physicians to control the volume of services’ they provide.” Policymakers should leverage this crisis to make quality a national health care priority.
Is free health care worth a 3-month wait? Ask Canada
Chicago Tribune, 4-3-05
Canada’s universal health care program seems like a panacea to some policymakers in Congress. But for many Canadians, the system promises much more than it ever delivers.
Americans who flock to Canada for cheap flu shots often come away impressed at the free and first-class medical care available to Canadians, rich or poor. But tell that to [Canadian] hospital administrators constantly having to cut staff for lack of funds, or to the mother whose teenager was advised she would have to wait up to three years for surgery to repair a torn knee ligament.
Canada’s “free” health care system is supported by draconian cost-controls that ration health services. This means that patients may have to wait weeks or months for medical services that Americans take for granted. According to one Canadian think tank, “the average wait for surgical or specialist treatment is nearly 18 weeks, up from 9.3 weeks in 1993.” If you’re a patient with chest pains who needs an EKG to diagnose a potential heart attack, that wait may turn out to be a death sentence.
Canadians, of course, are proud of their system, no matter what its flaws. It is, in many ways, more a symbol of national character than anything else. Americans, however, should remember that that Canada’s “free” health care comes with a long, long wait.
New Ethics Rule Cost NIH Another Top Researcher
Washington Post, 4-2-05
Another respected NIH researcher has resigned from the agency in the wake of restrictive new conflict-of-interest rules.
James F. Battey, chief of the [NIH’s] high-profile human-stem-cell program and director of that agency’s deafness institute will retire in September after more than 20 years…citing his inability to comply with strict new conflict-of-interest rules that have roiled the NIH internally and prompted a backlash in the broader science and business community.
Battey manages a family trust that contains assets—bequeathed to him by his father—that he will be forced to divest under the current rules. Sensible conflict of interest rules are one thing—and invaluable for a public institution with enormous financial clout like the NIH. The current rules go too far, however, and should be relaxed. Otherwise, the NIH will continue to bleed talent.
Medicare Puts Data Comparing Hospitals Onto Public Web Site
Wall Street Journal, 4-1-05
Consumer driven health care can only succeed if consumers can access critical information about provider quality. Thankfully, the Bush administration and the Centers for Medicare and Medicaid Services are helping to provide that information.
In a move to provide clear, unbiased information about the quality of hospital care, Medicare is launching a Web-based database that consumers can use to see for themselves how local institutions stack up against each other. The Web site, Hospital Compare…[offers] data on 17 widely accepted quality measures in treating heart attack, heart failure, and pneumonia. It shows how most of the nation’s general hospitals perform compared with state and national averages, as well as against their [local] peers.
Mark McClellan, the CMS Administrator, hopes that this will be a “big step toward supporting and rewarding better quality, rather than just paying more and supporting more services.” The Web site may not be perfect, or cover other critical quality measures (like hospital infection rates), but it is a terrific beginning.
Is the FDA Broken?
Joseph Calfee, Ph.D., Tech Central Station, 4-30-05
Calfee takes issue with the premise that an independent drug safety board would help fix the “broken” FDA.
An independent drug safety board would make things worse precisely because it would be independent. Drug safety cannot be assessed separately from effectiveness. Even the best drugs have side effects…Information about both effectiveness and safety emerge in a continuous and highly unpredictable flow….Instead of an independent board, we need an organization that merges the safety and effectiveness information coming from clinical practice. That is exactly what the FDA is already trying to do on its own. This is one time when Congress should just let the FDA get on with its job.
Banks: The New HMOs
Dr. Scott Gottlieb, Forbes, 4-4-05
Gottlieb believes that a little known passage in the Medicare Modernization Act of 2003 will revolutionize health care markets.
A transformation is under way in health care that will displace the entrenched giants among the ranks of America's HMOs and pharmacy benefit managers. In their place will be millions of consumers linked by their financial services companies to accounts. These new accounts will allow them to manage their full range of medical benefits in the same fashion that they direct their 401(k)s. The impetus for this change is a Trojan horse buried inside the new Medicare law. Explicit language in the new law enables a health plan and drug plan to be offered by any well-capitalized outfit adept at marketing and able to bear some financial risk.
Competition drives consumer gains—and increased competition in health care markets is no exception to the rule.
To spare the pain
Elizabeth Whelan, Washington Times, 0-0-00
Whelan praises the recommendation of an FDA advisory panel to allow Cox-2 painkillers to remain on the market “despite evidence they increase heart disease in some people.”
The risks…have been well publicized. Much less publicity has been given to a spectrum of real and potential benefits that go way beyond reduced risk of stomach inflammation. These little-discussed benefits would’ve been lost, perhaps permanently, had Vioxx, Bextra, and Celebrex been driven from shelves in pursuit of perfect safety, an unattainable goal.
Whelan cites evidence that the drugs may inhibit some cancers or perhaps even the dementia associated with Alzheimer’s disease. It will be interesting to see how the FDA’s recent decision to withdraw Bextra from the market affects research into this class of drugs.
COX-2 Inhibitors Given Perioperatively Improve Surgical Outcome
Researchers have published evidence that Cox-2 inhibitors may reduce postoperative complications and help patients return to normal activities faster after certain types of surgery.
“Ten to 15 years ago, this [laparoscopic cholecystectomy] was an open procedure that required three or four days of recuperation in the hospital," says lead author Tong Joo Gan, MD, from Duke University in Durham, North Carolina, in a news release. "Now, with laparoscopic surgery, patients can go home the same day as surgery. With the growing popularity of minimally invasive surgery, the onus is on us as anesthesiologists to come up with ways to better control pain and reduce the incidence of postoperative nausea and vomiting (PONV) so patients can go home comfortably after their surgery."
This study is among the first to show Cox-2 drugs given before surgery can reduce PONV.
On the first postoperative day, nearly one third of the COX-2 inhibitor group and 7% of the placebo group resumed normal to light activity. By the second postoperative day, more than half of the COX-2 inhibitor group and 36% of the placebo group had resumed normal activities.
BNP Better Than C-Reactive Protein at Predicting Cardiovascular Events
Physicians have many options—from statins to diet and exercise—to help treat patients at high risk of coronary artery disease. The trick is finding those patients reliably and easily using tools called biomarkers. Current biomarkers include LDL cholesterol and C-reactive protein—and now “the N-terminal fragment of the prohormone brain natriuretic peptide (NT-proBNP).”
[BNP] is a stronger predictor of cardiovascular disease and death than is C-reactive protein (CRP) in older adults, according to a report in the Journal of the American Medical Association for April 6th…Using the 80th percentile as a cutoff...NT-proBNP levels above this threshold were linked to a 3.24-fold increased risk of a first major cardiovascular event. By contrast, the hazard ratios seen with CRP and the urinary albumin/creatinine ratio were lower-1.02 and 2.32, respectively.
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