|Selected news articles which highlight important policy issues.||
News: Weekly Archives
News for the week of 05-02-2005
Team effort is best medicine for athlete's sight
Off-label prescribing is a common practice in the U.S. whereby physicians give patients a drug that has not been officially approved by the FDA for that particular disease or disability.
Some researchers worry that off-label treatments are just plain useless or turn patients into guinea pigs for drugs that may have serious side-effects. This is because off-label prescribing is often driven by anecdotal evidence or a handful of small studies, rather than by the large scale random clinical trials required for FDA approval. On the other hand, clinical trials can be enormously expensive and time consuming. Requiring clinical trials for every new off-label indication would rob patients and physicians of a many beneficial treatments.
This article, for instance, chronicles one physician’s struggle to prescribe an off-label treatment to her 19-year-old patient, Lamar Register, who has a rare inflammatory disorder known as sarcoid uveitis. The first-line drug used to treat his disorder, steroids, “was building up pressure inside Lamar’s eyes, threatening him with glaucoma and blindness.”
Lamar’s doctor wanted to prescribe him the drug Remicade off-label. Remicade, made by Centocor, is FDA-approved for treating Crohn’s disease and rheumatoid arthritis, not sarcoid uveitis. Lamar’s insurer, consequently, labeled it an investigational treatment and denied coverage. Thankfully, the FDA, Centocor, and Lamar’s doctor came up with a novel solution:
After weeks of negotiations… [Lamar’s doctor] would be allowed to conduct a one-patient experimental investigation of the use of Remicade for sarcoid uveitis. …Centocor would provide, at no cost, all the medication the young man needed. St. Barnabas [Medical Center] would provide the "infusion" - an intravenous drip that takes several hours - also at no cost.
Many thousands of patients like Lamar could be helped if the FDA and insurers joined forces and used physicians real-world clinical experience to validate off-label treatments. The evidence validating new drug indications is out there, if we want to take the time to find it.
German Curbs On Drug Costs Rile Big Brands
We have two articles this week on the issue of how price controls affect pharmaceutical innovation. The lesson to be learned from both articles: There is no antidote for success like complacency.
About ten years ago, Europe led the U.S. in the discovery of new medicines, and many of those medicines were launched in Europe first. Patients routinely complained to the FDA that they were denied access to treatments that were available in Europe. Today, the U.S. leads Europe in every significant category of biopharmaceutical innovation--from biotech venture capital, to developing new medicines.
The European Commission has called for studies on the cause of the decline (see the Financial Times article below). But it’s really no mystery. European health care systems inflicted price controls, rationing, and parallel importation on pharmaceutical companies. For instance,
Under the tax-funded German health-care system, a special commission set up by the health ministry decides how much the state will pay for drugs. The ministry decided last year it would no longer cover the higher prices of branded drugs that it deemed to have the same medical efficacy as available generics. So the commission drew up a list of drugs that it won't pay full price to cover, including many popular treatments for stomach acid and high blood pressure, as well as Lipitor, the world's best-selling drug. Legislation reflecting the ministry’s new stance went into effect in January; since then, demand for many of these once popular medicines has plummeted.
One executive for AstraZeneca likened Germany’s pricing policy to “losing your patents because you are forced to reduce your price…Basically, this methodology puts you in the same bucket with generics...”
The net result is that Germany—and Europe as a whole—penalizes pharmaceutical companies for investing in medicines that are more effective or have fewer side effects than older treatments. Adopting an age-old tradition, those companies are now refugees flocking to America’s shores.
People often labor under the mistaken impression that the science of drug development is like building a Lego castle. It’s not. It is a very unpredictable, expensive art that fails many thousands of more times than it succeeds.
Even when, it does succeed, human genetic diversity humbles multi-billion dollar companies by throwing up side-effects and other unintended outcomes that no one can predict or control. As this article correctly notes, “most mass-marketed prescription drugs typically produce the desired effect for only 40% to 60% of the patient population.” Raising those rates, and increasing the predictability of medicine, is the challenge facing companies today.
Thankfully, the unraveling of the human genome and powerful new computing and diagnostic technologies are tipping the balance towards personalized medicine:
Discoveries in the human genome are helping scientists identify the genetic origins of disease and gain a deeper understanding of how patients of a particular genotype respond to treatments. The payoff? Safer and more effective remedies. "We help patients by soothing symptoms, but rarely can we cure a disease," says Andre Terzic, professor of medicine and pharmacology at the Mayo Clinic. "The real future will go to the foundation of the disease and remove the problem."
Medical miracles that can “remove the problem” are undoubtedly still decades away. In the meantime, targeted cancer drugs like Iressa, Gleevec and Hercpetin are giving us a glimpse of the day when drug development will become more like the science of engineering than the haphazard endeavor it is today.
HSAs More Than Double in Six Months, New AHIP Study Shows
Health Savings Accounts are gaining market momentum just two years after they were created by the Medicare Modernization Act of 2003.
America’s Health Insurance Plans reports that, based on a survey of its members (“representing almost all the health insurance plans offering HSA-eligible plans”)
As of March 2005, 1,031,000 people were covered by HSA/HDHP products. This is more than double the HSA/HDHP coverage of 438,000 reported by AHIP members in September 2004.
Even more encouraging were AHIP’s findings that 37% of individual policies were purchased by formerly uninsured individuals; that 27% of policies in the small group market were sold to employers who didn’t previously offer coverage; and that “nearly half of people covered by HSA-eligible insurance are over the age of 40.” More information on HSA policies can be found at www.HSADecisions.org.
When it comes to health care there is no one “quick-fix” that will solve all of our problems. But, by making basic health insurance more affordable and putting some consumer skin in the game, high-deductible Health Savings Accounts can help to slow the growth of health care costs while still expanding insurance coverage.
FDA regulators and drug manufacturers are struggling to come to grips with the reality that new “targeted” drugs work more effectively for certain patient sub-populations than others. For instance, the cancer drug Iressa appeared to be a failure in post-marketing studies designed to confirm its efficacy—until data mining showed that it may work particularly well for some Asian cancer patients.
Iressa has proved effective at treating lung cancer in Asian patients, even as it has flopped in helping Caucasians, blacks and just about everybody else. Asians respond well to the therapy because they are more likely to have a certain genetic mutation in their cancer cells that Iressa is good at targeting, scientists believe. As a result, AstraZeneca, which initially planned on big sales of Iressa in the U.S., is now adjusting its marketing plan to focus on Japan, China and other Asian markets. For years, drug companies focused on finding drugs that could be sold to mass markets around the globe. Now, as researchers invent more precise treatments that target a particular genetic cause of a disease, drug companies are finding, sometimes unexpectedly, that their products work only on certain clusters of patients.
AstraZeneca is doing more research on Iressa to better understand how it works, and for whom.
Some commentators, in the meantime, have argued that targeted drugs won’t be as lucrative for industry as mass-market blockbusters. This may turn out to be the case for certain treatments that focus on very rare genetic conditions. But drugs like Iressa are proving that new indications for targeted drugs will undoubtedly be discovered as long as companies have powerful financial incentives to find them.
Autoimmune Diseases: Trying to Shut Off the Body’s Friendly Fire
As we mentioned above, physician autonomy in how they prescribe drugs often saves lives and contributes to the progress of medical knowledge.
Take, for instance, autoimmune diseases like lupus. Lupus is a frightening disorder where the body is torn apart by in a biological civil war that can cripple or kill those it afflicts. According to the NIH, there are 80 known autoimmune diseases that afflict between 14 and 22 million Americans. Unfortunately,
Little is definitively known about the causes of autoimmune diseases or how to treat them. It has been more than 30 years since a new drug was approved for lupus, for instance, and the existing drugs have severe side effects. But scientists are now using genetics and biotechnology to gradually unravel the mind-boggling complexity of the immune system.
In the meantime, patients and physicians struggling with lupus often must make due with medicines used “off-label”—i.e. drugs that are used to treat other illnesses and that are not specifically FDA-approved for that disease. This strategy buys time for researchers to expand their understanding of the disease and develop more targeted therapies.
States and Employers Duel Over Health Care
The New York Times chronicles a growing movement among state governments to “shift more of the burden” of providing health care coverage for the working poor from state Medicaid programs to employers.
…nearly two dozen states, struggling with the growing burden of providing public assistance to people with jobs but no insurance, are looking to shift more of the financial burden onto the workers’ employers. Last month, for example, Maryland, which spends roughly $350 billion a year on health care for the uninsured, passed a bill requiring the state’s very largest employers to spend at least 8 percent of their payrolls on health benefits for their workers. …
What the article doesn’t say is that states themselves are to blame for escalating insurance premiums and Medicaid budgets. State regulations mandating insurance benefits (for instance, chiropractic care) pass added costs to small and medium sized businesses through higher insurance premiums. As premiums rise, many small employers find themselves priced out of the market for even “basic” coverage. For instance, the Council for Affordable Healthcare Insurance found that:
…mandated benefits currently increase the cost of basic health insurance from a little less than 20% to more than 50%, depending on the state. Mandating benefits is like saying to someone in the market for a new car, if you can’t afford a Lexus loaded with options, you have to walk. …
States that offer expanded Medicaid eligibility through programs like Family Health Plus can also lure low-income workers out of private insurance markets because they charge lower premiums than employer-based coverage.
The bottom line result: state regulations push or pull more people into Medicaid programs, leaving taxpayers on the hook. Before state legislators lambaste employers like Wal-Mart for dumping health care costs onto taxpayers they should first heed the old adage: Physician, heal thyself.
Next Step Toward Digitized Health Records
Experts believe that developing digitized health-care records for patients will help providers coordinate care, reduce errors, and improve overall treatment. Physicians, however, face significant start-up costs for improving IT infrastructure—for instance, “entering mountains of bulging medical charts into computers.” Patient privacy concerns, competing technical standards, and sheer inertia are also slowing down the effort.
Still, one for-profit company associated with the American Medical Association hopes that it can spur consumer demand for electronic health-records:
Medem Inc., today plans to unveil an Internet-based "personal health record" that patients can use to organize their family's medical histories, much as they might electronically manage financial records or bank accounts. …the program helps patients record emergency contacts, insurance, and medical information such as conditions, medications and allergies. Patients access their record online using a password, and they can provide access to doctors, hospitals or loved ones: In case of an out-of-town emergency, for example, a husband could provide hospital staffers with his wife's password to ease treatment; patients seeking second and third opinions could provide their password to new doctors to get them quickly up to speed.
The records are available free at ihealthrecord.org and on some doctor’s Web sites.
As personalized medical treatments and diagnostics become more prevalent, consumer demand for electronic health records should take off. Eventually, EHRs will become as indispensable to consumers as credit cards—you won’t think of shopping for health care without one.
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