Big dosage, big profit: Does Genzyme encourage larger dosages than we need?
Filed under: Company News, Healthcare
Pharmaceutical drugmaker Genzyme (GENZ), based in Cambridge, Massachusetts, specializes in creating drugs for rare diseases. It's a small market, but it's highly lucrative: the Orphan Drug Act gives the company tax credits and enhanced patent protection, virtually guaranteeing that its drugs become blockbusters.Consider Genzyme's drug Cerezyme, which treats a rare, chronic genetic disease called Gaucher, and which has an astonishing price tag that netted the company $1.24 billion last year -- more than a quarter of its total revenue. But when a recent manufacturing problem threatened a potential Cerezyme shortage, Genzyme's response seemed to confirm some critics' long-held suspicions that the company may be encouraging inflated dosage -- and therefore higher sales -- of its drug, which costs patients hundreds of thousands of dollars a year.
After announcing weeks ago that it would shut down a Boston-area plant through July, Genzyme said it would ask some consumers to conserve their stocks of the drug by temporarily skipping or reducing doses. The missed doses will not cause significant health problems, Genzyme's chief executive, Henri Termeer, said in a recent conference call: "It is not a life-and-death, immediate acute situation we're dealing with here."
What's troubling is that Genzyme has a monopoly on treatment for Gaucher, among other rare diseases. Without significant competition for drug treatment, the medical community is left to provide its own checks and balances on an intricate, delicate symbiosis that benefits patients and doctors as much as it benefits Genzyme's bottom line.
Gaucher affects fewer than 10,000 people worldwide, many of them Ashkenazi Jews and northern Swedes. Sufferers lack enough glucocerebrosidase, the enzyme that breaks down lipids which would otherwise amass in the liver, spleen, bones and bone marrow, preventing cells and organs from working properly. Symptoms include enlarged spleen and liver, anemia, low blood platelets, painful skeletal disorders and bone lesions, osteoporosis, and neurological complications.
Genzyme has monopolized Gaucher treatment: some 5,000 patients take Cerezyme -- roughly half of the people with the disease. (Rival drugmaker Actelion Pharmaceuticals markets a medicine called Zavesca for patients who can't take Cerezyme; many patients take no drug at all.)
The drug's astronomical cost is related to the common dosage of 60 units per kilogram every two weeks. Median prices are $1,225 for a 200-unit supply and $2,781 for a 400-unit supply, according to the Canadian Patented Medicine Prices Review Board. The average cost for treatment is $200,000 a year. Approximately 10 percent of Cerezyme users receive the drug free from Genzyme.
"Recent analyses of data from the Gaucher Registry established that treatment with Cerezyme leads to a dose-dependent improvement in key bone, hematologic, and visceral parameters in patients with Gaucher disease type 1," says John Yee, M.D., Genzyme's vice president of Global Medical Affairs. But not everyone agrees such high dosage is necessary. Ari Zimran, M.D., is director of the Gaucher Clinic at Shaare Zedek Medical Center in Jerusalem, which treats nearly 200 Gaucher patients and monitors more than 700. Zimran administers roughly a quarter of the average dosage given in the U.S., arguing that no clinical evidence suggests that a higher dosage is more effective in treating the symptoms or preventing bone disease.
Zimran says the higher dosage isn't necessarily harmful, but he argues that a patient should take what he believes is the minimal efficient dosage. Zimran says dosage should depend on the severity of symptoms, the patient's age -- and the cost. "Gaucher is a benign disease," he says. "Unlike cancer or bacterial infection, which must be eradicated -- often at the expense of making the patient sick in other ways, such as chemotherapy, which kills healthy cells too -- there is no need to go that length with Gaucher."
Genzyme's Yee says, "Studies show that higher doses generally lead to better clinical response and greater achievement of published therapeutic goals." But he acknowledges: "Evidence regarding dose in the peer-reviewed, published medical literature is not consistent," and adds that some studies show little difference in outcomes for Gaucher patients treated with lower Cerezyme doses.
Zimran was a part of a clinical trial in the early 1990s to examine the impact of the frequency of the the low-dose regimen. The results for the patients were the same, he says, for high and low frequency, and similar to the high-dose study in the U.S. While the trial results, published in leading medical journal The Lancet, are not often cited, Zimran says, other studies support its conclusion on a clinical basis.
The International Collaborative Gaucher Group Gaucher Registry (supported by Genzyme) reveals the symbiotic relationship among the company, health-care professionals, and Gaucher patients. The largest cooperative, observational registry on Gaucher was established in 1991 as a longitudinal database tracking outcomes of routine clinical practice. Genzyme's access to the registry's patient and treatment data gives the company highly unusual clout in following up with -- and potentially influencing -- doctors treating Gaucher patients. At times, Genzyme funded full-time data-entry personnel for Gaucher centers.
Genzyme says its registry is meant only to benefit the patients, an important study tool that benefits patients and medicine with longterm tracking and with treatment options that help optimize patient care. But it's also a brilliant marketing tool for the for-profit drugmaker -- and a controversial one. The medical community would probably not accept such a registry for treating other diseases; doctors and patients typically calibrate a dosage without the intervention of a pharmaceutical company. Indeed, the registry has been criticized for monopolizing treatment of the disease and for breaching ethical boundaries on patient information.
Of course, Genzyme has a duty to maximize shareholder value, and it does so with great science, a high-quality product, and superb marketing, as Zimran says. But Genzyme's ability to question a doctor's dosage, and its access to patients' consumption of Cerezyme, makes the registry a minefield of potential conflict.
"Data from the Gaucher Registry have contributed to more than 20 peer-reviewed publications on Gaucher disease," Yee says. Nevertheless, Zimran counters that this data isn't often used to the fullest.
Some also question the practice of treating Gaucher patients with minimal or no symptoms, Zimran says. Gaucher patients may have some natural immunity, due to elevated levels of glycollipid, a fat molecule, in their plasma, he says. "While Cerezyme itself does not cause diabetes, heart disease or cancer," he says, "mild patients who take it may lose a natural selective advantage -- thus making them more susceptible to common diseases they had better natural immunity against."
Genzyme strives to appear uninvolved in the treatment process. Officially, at least, the company recommends individualized dosing for all patients. "Numerous studies have supported the need to determine dose on an individual basis," Yee says.
Physicians, as a profession, generally claim they're immune to pharmaceutical company marketing. But evidence suggests that drug-company marketing, including promotional materials and free samples, influences doctors' attitudes and increases drug sales. (Such marketing is feeling pressure from Congress, under this year's bipartisan Physician Payment Sunshine Act.) The mission statement of NoFreeLunch, a non-profit watchdog organization founded by health-care providers, states: "We discourage the acceptance of all gifts from industry by health care providers, trainees, and students. Our goal is improved patient care." A World Health Organization database also documents the impact on health professionals of biased marketing information and gifts and other inappropriate promotions.
Zimran, it should be noted, admires Genzyme for the medical advances it's made. He gets research grants from the company, and he has contributed to Britain-based Shire and Israel-based Protalix Biotherapeutics developing enzyme therapies similar to Cerezyme. He predicts that both new enzymes will get approval from the U.S. Food and Drug Administration, which is moving to speed up access to these drugs to help offset likely Cerezyme shortages. However, he doesn't anticipate the company's severing ties with its network of physicians.
With the expected Cerezyme shortage, Genzyme's critics will watch to see whether patients' taking a "vacation" from the higher dosage notice any health effects. And if many patients don't feel any worse on a lower, cheaper dosage, then many Cerezyme consumers could decide to make it a permanent vacation.
Fed stalls on Chinese bank deal, costs taxpayers $1.7 billion
Dell in a handbasket: Should founder Michael Dell resign?
TARP saved the banking system, but failed at everything else
Reader Comments (Page 1 of 2)
7-10-2009 @ 12:56PM
jgc711 said...
Drug companies are the scum of the earth. They take advantage of the fact that people need their products. And they collaborate with doctors and encourage them to prescribe drugs that people will take for their lifetime. I have high cholesterol and before I started taking prescription drugs that are really, really expensive even with insurance, I used to take oat bran with niacin. Then, they took over and lo and behold, oat bran with niacin could no longer be found. Who's responsible? The drug manufacturers. Why? Because oat bran with niacin is so effective in lowering bad cholesterol and if it is available, nobody would buy their drugs.
Reply
7-10-2009 @ 1:28PM
Watcher said...
http://seniorjournal.com/NEWS/Health/4-12-21FDAonAleve.htm
“Patients should not exceed the recommended doses for naproxen (220 milligrams twice daily) and should not take naproxen for longer than ten days unless a physician directs otherwise.
FDA Issues Advice on Use of Naproxen (Aleve) After Trial Halt
Read their instructions, not only deceptive about being an anti-inflammatory at the dosage but also instructs to take up to 5 per day. And the FDA nor FTC does anything about it.
Reply
7-10-2009 @ 2:14PM
kenhp1 said...
This article might explain the resistance of some to pass healthcare reform.
This company is one who rapes the consumers financial well being all the while patients are being denied basic medical care.
What is more disturbing is the fact the credents who run this company have no ethics, are greedy money hungry scumbags who could care less about the wellbeing of their fellow americans.
Reply
7-10-2009 @ 2:37PM
Nancy said...
Drug companies make the drugs, doctors push them. My husbands cousin is on so many drugs they fill two shopping bags full. Yet, when she was in the hospital for two weeks, she NEVER got one of those prescribed for home. Does that make sense? I don't think, yet her doctors say she needs them. Why would she need them at home, but not while two weeks away from home?
Reply
7-10-2009 @ 2:43PM
arentany said...
"jgc711" is so very right. A paradigm is Lilly. Once they were a model of much that was good in American business -- the source of insulin, so valuable for the life of diabetics. Now, they are hucksters of the worst kind -- like the other pharm firms. Interesting note, the dominant factor in psychiatric pharm agent choice has been shown by acceptable research to be the time proximity of the last visit by a company's pharm rep! Such is the danger of these hucksters. But we can't compete with their massive lobby funds.
Reply
7-11-2009 @ 3:09PM
Jill Marie said...
Dr. Joel Fuhrman's Eat to Live or recent Health for Health way of life is the only medically proven diet in the world but is more for reversing disease. This NJ-based M.D. has reversed heart disease, diabetes, no matter how old the person, the weight of the person, or the severity of the disease. The Am. Diabetes Assoc. wanted to publish an article on Dr. Fuhrman's success but Eli Lilly stopped publication because it actually CURES diabetes and would cut into their profits!
7-10-2009 @ 3:18PM
jeff said...
TALK ABOUT STATIN DRUGS THOSE ARE THE REAL DRUG THAT IS BEING OVER PRESCRIBED AND HAS TONS OF SIDE EFFECTS THEY COVER UP!! 5% OF ALL PEOPLE WHO TAKE STATINS HAVE SEVERE MUSCLE PAIN WHICH CAN BE PERMANENT OR KIDNEY AND LIVER DAMAGE!! DOCTORS TAKE BILLION$ IN BRIBES FROM THE DRUG COMPANIES AS DOES CONGRESS AND THE FDA SO DO NOT TAKE ANYTHING UNLESS YOU RESEARCH THE REAL TRUTH ABOUT THE DRUG FIRST ON THE INTERNET!! SEE ALL SIDES OF IT NOT JUST WHAT YOUR BRIBED DOCTOR IS TELLING YOU!!
Reply
7-10-2009 @ 3:02PM
SCHUE said...
IT HAS BEEN KNOWN FOR QUITE SOME TIME THE PHARM. COMP. AND MED. INS. INDUST. HAVE BEEN GOUGING JOHN Q. FOR AGES. I THINK WE ARE LONG OVERDUE FOR GOVT. INTERVENTION. BY THE WAY I TRASHED ALL MY EXPENSIVE MEDICINE BUT TWO . I FEEL BETTER AM LOSING WEIGHT AND MORE ACTIVE, REST MY CASE RIGHT THERE.
Reply
7-10-2009 @ 3:24PM
tommytortugam said...
F. D. A. = Federal Death Administration
Reply
7-12-2009 @ 3:49PM
jimon said...
Amen !!!
7-10-2009 @ 4:47PM
Cartoon Pig Dog said...
Very few people have been made aware of the medicinal uses for marijuana and its derivatives (cannabinoids and thc). The reason for this is because the pharmaceutical companies actually pay for research on marijuana and then pay the researchers to post findings that are false because many disorders, especially those associated with dementia (such as Alzheimer's) are more successfully treated with marijuana with fewer side effects than those drugs manufactured by the pharmaceutical companies, whose drugs often have very bad and sometimes fatal side effects, not to mention the fact that many of the chemical drugs are extremely addictive, where it has been shown that marijuana has no addictive qualities, contrary to what the Governments propoganda style reports would have you to believe.
Go ahead and check it out, you will find that all negative reports on marijuana were funded either by pharaceutical companies, or by the DEA. All honest research on it shows very different findings than those funded by these two groups.
Reply
7-10-2009 @ 8:24PM
travelergtoo said...
Big Pharma strikes again. He was on so much DOPE it's no wonder he couldn't tell girls from boys and tried to mate with children. Instead of the press painting him as a pediphile, if they would have said how much DOPE he was on maybe he would have changed his DOPEY ways. Instead, even after knowing that there MAY have been inapropriate behavior with teenagers the response of the 'doctors' was to give him even more DOPE so much so that a pharmacy sued him for $100,000 for not paying his DOPE bill. He was so DOPED up I'm surprised he didn't try to mate with a tree or watermelon. The 'doctors' who gave him all that DOPE should be executed.
The FDA should also be executed for permitting this.
Reply
7-10-2009 @ 10:06PM
Jerry said...
Where in earlier time a Doctor was self motivated to provide real care for the patients. He was a general practioner able to understand all of the bodies interaction and used this to make a reliable prognosis. Todays Doctor wants to restrict his "expertise" to as little a part of the body as possible. He is a specialist!. Can't diagnose without tons of expensive tests. Relies on "Physcian Assistants and Nurse Practioners to do much of the actual doctoring. Likes to be called "Dr Paul or Dr Ben" like some Disney character. Charges the crap out of his patients and lives high on the hog--lots of golf and trips. Doesn't realize that people sue when they feel that their finances have been destroyed. Funny nobody asks why medical costs are so high--they just search for ways to continue paying the costs, whatever they are. And the politicians are the fleas on the backs of this disgusting practice.
Reply
7-10-2009 @ 10:29PM
Sonja said...
My mother passed away in Feb. at the age of 101. She never took ONE prescription drug until she was 95 - her blood pressure spiked and she was put on it. She got disgusted and didn't want to take it after awhile and didn't. I checked her BP often and she was fine without it. The last two years of her life she spent in a nursing home and she was given it there. She didn't use Medicare until she was 93! She had been told several times by nurses that it was interesting that when very old people come in for check ups they are seldom taking any prescription drugs. Those darned things are killing people left and right, not extending lives. Yes, some are needed, but there are way too many people taking drugs long term and too many. I'm 67 and take none - have no intention of taking any unless I absolutely have to! What is pathetic is how many young people are taking numerous prescription drugs.
Reply
7-11-2009 @ 2:09PM
Jay said...
Good for you and your mom. Way to go !!
7-13-2009 @ 2:21AM
Bill said...
Sounds like someone I know and love myself.
7-11-2009 @ 11:17AM
Luciano said...
Las mejores palabras de aliento
Reply
7-11-2009 @ 8:05AM
aawindoze said...
Oh yeah dude I think you hit that one spot on bro!
RT
www.anonymize.tk
Reply
7-11-2009 @ 9:54AM
angela klinger said...
I work in a pharmacy and the stories I could tell would "make you ill". Though I do not hold a "degree", I am nevertheless self informed and am able to read, write, investigate and observe. There are so many factors involved when dispensing medications along with many areas of professionalism, (doctors, pharmacists, enviromentalists, lobbyists, lawyers, manufacturers, to name a few) with the pharmacutical companies being the top over-seer and controller in charge of it all. We are not a third world nation, yet we rank higher than most third world countries with the highest level and rates of disease. Part of it is due to our own personal lack of healthcare, yet another part is due to long term effects of medications which eventually kill us or destroy other vital organs of our human system. I believe if healthcare professionals would use the brain God gave them and inform those under their care the hidden long term effects of medications, people would perhaps think more and make decisions for themselves. I'm not saying that people should assume and diagnose their own illnesses, but the medical professionals seem to be extrememly passive with information and overly aggresive with dispensing medication. We live in a time of "total re-call"..Never thought I'd see the day a pharmacutical company, after reaching their profit goal for a drug, put the brakes on it and stop its marketing by calling for "re-calls" due to "whatever" disaster they know will scare people.. If people knew the amount of medications with certain lot numbers being recalled from pharmacies daily they would be shocked! I see it EVERYDAY. There is so much more I could write a book. People need to investigate and ask questions, especially to their doctors prescribing and pharmacists who dispense. Don't be afraid. Any doctor who does not do frequent lab work, and whose goal is to keep you on medication for the rest of your "short" life is only practicing medicine, not healthcare.
Reply
7-11-2009 @ 10:34AM
marshall said...
I'm a 70 year old pharmacist. I think there might be something to this story. Why else would the very pretty Pfizer sales woman that sells us Viagra suddenly be so nice to me.
Reply