|Selected news articles which highlight important policy issues.||
News: Weekly Archives
News for the week of 12-06-2004
New Leukemia Drugs May Prove More Potent Than Gleevec
Gleevec is a miracle drug that provides hope for patients with a rare and deadly form of blood cancer called chronic myeloid leukemia (CML). However, the drug isn’t perfect – some genetic variants of CML are resistant to the drug, and some patients who have initials success with the it may see their cancers re-emerge months later, as their cancers develop drug resistance.
However, thanks to new forms of molecular imaging and DNA sequencing, scientists can test for genetic variations in Gleevec-resistant cancers and discover exactly how the disease differs from its standard version – and then design new drugs to attack other vulnerabilities in the disease.
Two new drugs, one from Bristol-Myers Squibb, the other from Novartis, do just this. “These drugs emerged from a new line of research that examines the molecular structure of resistant tumors to see how they evolve to defy Gleevec. Scientists say the findings provide compelling evidence that such research could lead quickly to the next generation of precisely targeted weapons against a variety of tumors.” One drug, developed by Bristol-Myers, has shown remarkable success in small, early stage human trials. “Of 36 CML patients in the so-called chronic phase of their disease, 31, or 86%, had a complete remission after up to nine months on the Bristol-Myers drug. In addition, 13 of 29 patients evaluated had reductions in the number of cells containing the genetic defects that cause the disease.”
Celebrex Less Risky Than Vioxx
The war against so-called “me-too drugs” and Cox-2 inhibitors, the poster children for the me-too phenomenon symbolizing drug company greed and cynicism, may have hit a snag. Researchers are discovering that the class of Cox-2 inhibitors (including Vioxx ) “don’t all carry the same risk for heart attacks” according to findings from a study involving more than 8,000 people.
Researchers at the University of Pennsylvania have found that “Vioxx was almost three times as likely to be associated with a nonfatal first heart attack as Celebrex”. In fact, Celebrex users had a “57% lower risk of a heart attack” than nonusers, which raises the question (unanswered in this article) of whether or not this particular Cox-2 drug might actually be cardio protective. Pfizer, the maker of Celebrex, is currently funding its own study to see if, in fact, the drug protects users from heart disease.
This finding explodes the myth of the “me-too” drug, which clinicians and researchers have long suspected is really baseless. The fact is that different patients can respond very differently to similar drugs because genetics and small molecular difference in drug composition produce different responses across patient groups. If Celebrex does turn out to be cardio protective, it might just be the silver lining in the Vioxx cloud hanging over the FDA and industry.
A new tests holds the promise of identifying the earliest signs of cancer in the human body – well before tumors even begin to form. The test looks for “substances in blood platelets that indicate cancer is in the works. Armed with such information, the researchers involved say, doctors could potentially offer cancer therapies that would prevent tumors from developing – and save patients from having to undergo much more toxic and aggressive treatment [later on].”
The research holds even more promise given the ongoing proliferation of targeted drugs that specifically attack cancer proliferation mechanisms without harming normal tissue. These drugs – Gleevec, Tarceva, Iressa – and other drugs may even have cancer preventative properties (like aspirin or statins) could then be taken by patients who are at very high risk of cancer or cancer recurrence after initial treatment.
Although there are other cancer biomarkers currently used by physicians (such as the PSA count for prostate cancer) they are very unreliable, and often require a biopsy to confirm the disease. The platelet test could avoid this problem because it identifies a specific substance involved establishing new blood vessels that feed tumors. Currently, “more than 30 drugs that specifically try to inhibit the process of making more blood vessels [for tumors] are working their way through clinical trials to get Food and Drug Administration approval.”
NIH licensing performance under par
If a company stumbles on a promising anticancer compound, you can bet your last venture-capital dollar that it would rush it into testing and clinical trials. If the government stumbles over that same compound – it may sit on a shelf for years before ever seeing the light of day.
One argument frequently touted by critics of industry is that the government could – and should – perform the bulk of drug development, and could do so much more cheaply than the profit-driven industry. But the problem is that without the incentive of profits, government researchers and bureaucrats are more likely to ration time and resources rather than bet them on promising new drugs. For instance, in September, a biotech startup licensed a “class of anticancer compounds called macrocyclic lactones” originally discovered by the National Institutes of Health (NIH) more than 7 years ago. Why the long wait? “They have fallen through the cracks multiple times”, says one of the former NIH researchers who originally discovered the compounds.
At the NIH’s Office of Technology Transfer (OTT), the director, Mark Rohrbaugh, said that “Given the resources that we have to put to bear on technology licensing, I’m satisfied with the expeditiousness of our efforts.” Of course, if new compounds aren’t reaching patients for years after they are first identified, lives are lost waiting for government resources and “expeditiousness” to improve.
Academic research universities, with their own OTT offices, fare much better – presumably because universities directly profit from licensing and have powerful incentives to market and develop promising compounds as aggressively as possible. For instance, in the last five years, NIH’s OTT has seen its licenses grow by 11.1%. Impressed? During that same period, the University of Wisconsin – with a research budget less than a third that of the NIH – has seen its licenses grow by 294%. The University of California – with a slightly bigger budget than that of the NIH – is also a lagging performer compared to Wisconsin, but still comes in at five year growth of 33.7%, more than 3 times the NIH rate.
The market may not be perfect – and people are always trying to game markets (because, let’s face it, human nature will cheat when it can), but for the improvement of human health and well-being there is no better mechanism than private initiative.
Importing of drugs approved by AMA; Support increases pressure on FDA
The AMA is supporting price controls on the pharmaceutical industry. Wait – isn’t this argument about importation of medicines, not price controls?
Well, the reason that drugs from other industrialized nations are cheaper than in the U.S. is because those nations impose price controls on them. Importing drugs therefore means importing price controls. Besides, Canadian drug importation is a red-herring at best, since the Canadian government has already announced that it won’t – and can’t – become America’s drugstore. The problem is that the Canadian market for prescription drugs is miniscule compared to the U.S., and couldn’t accommodate American demand without denying Canadians access to their own supply.
So what the AMA is really fronting for is price controls. Fair enough. However, to test their sincerity on this issue, everyone should take a doctor out to lunch and ask them how they like the reimbursement schedule for Medicare and Medicaid. Chances are the doctors will grumble that the government chronically underfunds them for the work they do, and that they’ve had to drop Medicare patients from their practices just to survive. In other words, when applied to them, doctors hate price controls.
The time has come for Americans – and their policymakers – to ask themselves if they want price controls on prescription drugs. We suspect the answer will be no, but having a frank debate is better than trying to slip Americans a Mickey Finn called “drug importation” when they’re not looking.
Report warns of drug price controls
On Tuesday, the Manhattan Institute for Policy Research (the parent organization for Medical Progress Today) released its first original Medical Progress Report, cataloging the effects that increased government influence on drug prices has on pharmaceutical innovation and R&D. The report takes its bearings from the fact that the largest expansion of a government program in nearly 50 years, the Medicare Modernization Act of 2003, which gives prescription drug coverage to seniors starting in 2006, has a non-interference clause that prohibits the government from negotiating with drug manufacturers – a clause that has drawn quite a bit of fire from critics who see it as a give-away to industry.
Extrapoliting from prior government interference in pharmaceutical markets, the report “predicted that lower prices…would reduce research and development spending in the industry by 39.4 percent, or $372 billion over the lifetime of the [MMA].” It is readily accepted by the vast majority of economists that R&D is a function of prices – reduced profits means reduced R&D. While this premise seems acceptable when it comes to classroom economics, it suddenly becomes fodder for the political mill when people start talking about prescription drugs.
The study only focuses on costs of price controls – not the benefits of the government lowering prices on all drugs for everyone now, which would presumably subsidize broader access to lifesaving medicines – that is, the medicines we have today.
However, additional research conducted by the same authors suggests that the gains from innovations in medical progress dwarf the benefits from subsidized access. In other words, consider this question: would you freeze prices and innovation in 2004 and relinquish discoveries that might otherwise happen in 2010? How about if you knew that you were at high risk of developing cancer in 10 years?
Certain Type of Breast Cancer Drug Shows Better Result in Study
Research interest continues to build in a novel class of breast cancer drugs called aromatase inhibitors, which block the production of estrogen in body tissues. A new study, published in the medical journal The Lancet, found that “aromatase inhibitors [were] more effective in preventing breast cancer recurrence in women past menopause than was tamoxifen, a medication now prescribed by many doctors.” The study tested the drug Arimidex (made by AstraZeneca) against tamoxifen (available in both brand and generic forms) in more than 9,000 women over five years. The study was financed by AstraZeneca.
Survival rates in the study were basically the same between Arimidex and tamoxifen groups. However, “the group that took Arimidex reduced the chances of their cancer spreading by 14 percent and were 42 percent less likely than women on tamoxifen to develop a tumor in the other breast. They also suffered fewer side effects like blood clots, hot flashes, and endometrial cancer.”
Genetic Mutation Tied to Depression, Drug Response
Duke University researchers have identified a gene that “appears to make people more likely to suffer major depression and less likely to respond to treatment with antidepressants. The test could lead to a gene test to identify such individuals, and possibly new ways to treat them.” The gene is called TPH2 and is involved in the brain’s production of serotonin, a neurotransmitter. The mutated version produces “80% less serotonin than normal.”
Of course finding a gene is only the first step; many people may carry the gene without major depression, and others may have major depression and not carry the gene. Even after identifying the gene, the Duke researchers checked 300 people to see if it was linked to symptoms of depression. Out of nearly 90 patients with major depression, only 9 (about 10%) had the gene; out of these, “seven had not responded to antidepressant drugs and two only responded to very high doses.”
The finding is important because it may lead to a test to identify patients whose treatment is resistant to current therapies, and lead to new treatments. This is critical because currently “about 20% of patients with major depression don’t respond to antidepressants, according to Thomas R. Insel, director of the National Institute of Mental Health.”
A new compound – with the inglorious name R207910 – has shown early promise in animal tests of becoming a powerful treatment for tuberculosis, “the second leading cause of death worldwide, surpassed only by AIDS.”
In a study reported in the journal Science, “the compound easily overcame the two biggest hurdles facing TB therapies today: their ineffectiveness against resistant strains and the long period of treatment required to achieve a cure.” The current three drug cocktail used against TB is over 40 years old, and must be taken every day for 6-9 months, leading to serious problems with patient compliance. When patients stop taking the drugs before all the TB bacteria in their bodies have been killed, drug resistant strains of the bacteria emerge. One researcher speculated that the new compound, used in conjunction with two drugs from the old cocktail, might shorten treatment duration by 50%.
The drug also doesn’t interfere with the body’s metabolism of several important AIDS drugs, a side of effect of one of the drugs in the current cocktail. That is a critical benefit since more than 11 million people worldwide have both diseases, and TB often hastens death in AIDS patients.
Making Vaccines Good Business
The authors of a new book on infectious diseases in developing nations, Strong Medicine: Creating Incentives for Pharmaceutical Research on Neglected Diseases, “argue [that] Western governments and foundations should make a legally binding promise to pharmaceutical and biotech companies: if you invent a safe and effective vaccine for malaria, tuberculosis or H.I.V., we'll buy the first (say) 200 million doses at a respectable profit-guaranteeing price. One great virtue of this scheme, the authors suggest, is that the public would pay for only a successful product. If a company invests millions in research but fails to develop a vaccine that meets the contract's specifications, no money would change hands.”
This is basically the way markets work now: no company gets a return on its investment for a drug that fails to meet FDA approval. The difference is that developing nations are often poor markets to begin with, with little money to spend on even critical medicines. Compounding the problem is that drug companies are leery of investing hundreds of millions of dollars in developing a new product only to see its patent overturned by governments eager to make the drug themselves at a huge discount.
The bottom line: the absence of financial incentives hinders research and deployment of new medicines in developing nations. Rather than berating companies for not being charities, activists and policymakers should find better ways to make markets work. Hopefully, this book will turn out to be an important first step in that direction.
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