Medical Progress Today
  Volume 2, Number 13
  April 4, 2005


In the Spotlight

Our competitors understand why America is the global leader in biotechnology.
Why don’t we?


Robert Goldberg, Ph.D., Medical Progress Today, 4-4-05

In March, I met with officials from the Health and Insurance Ministry of the People's Republic of China as part of a program to foster better understanding of America’s free market economy. I took them through the steps of drug development. I emphasized that, if they wanted to encourage private investors to put a significant amount of money into far-reaching biomedical research activities, the government shouldn’t impose price controls on new medicines.

Continue reading . . .


Featured News:
Back from the brink | Millionaire father rescues company; knew drug would help his daughter
The San Diego Union-Tribune, 3-25-05
Featured Commentary:
Kianna's Legacy
Wall Street Journal, 3-29-05
Featured Research:
Efficacy of nine-valent pneumococcal conjugate vaccine against pneumonia and invasive pneumococcal disease in The Gambia: randomised, double-blind, placebo-controlled trial
F. T. Cutts, S. M. A. Zaman, The Lancet, 3-30-05

News

MS patients plead for a recalled medication Some doctors agree that Tysabri's dramatic results outweigh risks of a rare, fatal disease contracted by two users
Newsday, 3-30-05

Editor’s Notes:

Tysabri, an important new multiple sclerosis drug, is still off the market. It may never return now, since a third case of fatal PML, or progressive multifocal leukoencephalopathy, has surfaced in a patient with Crohn’s disease who was taking the drug. Unlike the two prior patients afflicted with PML, this patient wasn’t taking Tysabri in combination with Avonex, another MS drug that was implicated in the earlier deaths. Still, researchers speculate that a combination of immunosuppressive drugs and Tysabri may be causing PML. Does this mean that Tysabri is unsafe? Researchers and physicians seem to disagree.

“For many years we have used other, more dangerous treatments for MS,” said Dr. Jeffrey Horstmyer, director of the MS Center at Mercy Hospital in Miami. “Even if Tysabri has the rare side effect of PML, it still likely safer than a lot of other things being used.”


One other expert called Tysabri’s withdrawal “perfectly ludicrous”, but said that “with product liability attorneys hiding behind every bush, there’s no alternative to pulling it.” There should be an alternative. More of an effort needs to be made to quantify the relative risks and benefits of new treatments versus standard therapies, and to take account of potential drug-drug interactions in patient groups likely to be on multiple drug therapies. And, last but not least, pharmaceutical companies that are in full compliance with FDA regulations should be given some safe harbor from the plaintiff’s bar.

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Can we limit stroke damage? Because every second counts, patients in a novel study will be treated before they reach the hospital.
Los Angeles Times, 3-28-05

Editor’s Notes:

Your mother always told you to eat your green vegetables. She didn’t know how right she was. Today, scientists are testing whether or not a key mineral (magnesium sulfate) found in most leafy green vegetables can be injected into stroke victims to help save brain cells in the first crucial hours after an attack. The effort is focused in Los Angeles County, which is launching a large, ambitious clinical trial involving paramedics and thousands of patients.

Los Angeles County paramedics in mid-January started screening patients for participation in the planned three year study, which would allow emergency personnel to administer a specially prescribed solution to patients being transported to any hospital in the county for further treatment….The treatment under study is called “Fast-Mag,” named for magnesium sulfate. Researchers believe this mineral might slow the chemical process that can kill 12 million brain cells per minute during an untreated stroke, leading to long-term disability and death.


The study will also be groundbreaking because the “the sheer scale and diversity of the population that lies within [Los Angeles County] could establish its findings quickly as relevant across the country—and possibly the world.”

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New Vaccine Said to Offer Hope Against Bacterium
The New York Times, 3-25-05

Editor’s Notes:

The most feared—and best known—diseases are not always the biggest killers. More prosaic maladies often inflict more suffering and death in developing countries than headline-grabbing ailments like AIDS and Malaria. Take, for instance, the Streptococcus pneumoniae bacterium, which kills thousands of children every year in Africa. A vaccine for this disease has been available in the U.S. for infants since 2000. The vaccine, called Prevnar, prevents “rare but serious infections” from the bacterium; a stronger version just finished clinical trials in Africa.

In the third world, the same germ is a major killer, and the new vaccine, tested in Gambia, “exceeded our expectations,” said Dr. Orin Levine, head of the pneumonia program at the Global Alliance for Vaccines and Immunization, which backed the trial. “Most people don’t know it, but pneumoccocal disease kills more people every year than malaria. In this trial we prevented one death for every 200 children we vaccinated. That’s a whopping public-health contribution.”


Wyeth, the company that developed the vaccine, is “negotiating a price for standard Prevnar with the Gambian government and would work with GAVI in the future on the strengthened vaccine.” The new vaccine, however, doesn’t come cheaply: the trial for the vaccine tested in Gambia involved some 17,000 children and cost $30 million. While citizens in developed countries complain about the high price of medicines, sales of drugs in the first world subsidize cheaper prices in developing nations. For instance, a vaccine “used in poor countries that protects against diptheria, tetanus, whooping cough, hepatitis B and heamophilius influenza B in one shot is purchased by the Global Alliance for $3.65 a dose, a fraction of what similar shots cost in the West.” Policymakers in the U.S. should consider this reality the next time ideologues demand price controls here.

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Back from the brink | Millionaire father rescues company; knew drug would help his daughter
The San Diego Union-Tribune, 3-25-05

Editor’s Notes:

Allen Andersson is an “angel investor” who saved the drug company Amylin Pharmaceuticals from bankruptcy by investing millions of dollars of his own wealth when the FDA rejected the application for its flagship drug. Last week, the drug was finally approved and “Amylin pharmaceuticals…credits Andersson with keeping Symlin alive for the six years it needed to obtain FDA approval.” The root of Andersson’s passion: his daughter, Rachel, is a diabetic. Amylin’s drug, Symlin, is the first new diabetes drug in about 80 years.

Amylin’s trouble started when it submitted the results of two late stage (Phase III) clinical trials to the FDA in 1999.

The FDA said the results were “statistically insignificant,” meaning that the results could not conclusively prove that the drug’s success was not due to chance. Wall Street’s reaction was unfavorable. Amylin shares took a dive…Andersson looked at the same statistics with the eyes of a mathematician and concerned father. “Anyone with an understanding of diabetics or statistics could see this was a good drug that did the job and was safe and effective,” he said.


Still, it is sobering to remember that the hopes of thousands of patients can hinge on the generosity and faith of a single investor—who sees something FDA’s statisticians can’t.

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India Adopts Patent Law Covering Pharmaceuticals
International Herald Tribune, 3-24-05

Editor’s Notes:

India recently passed legislation which allows drug compounds to be patented for the first time; “previously, only the process of making the drug was patentable.” Critics of the law are already saying it is a devastating blow to developing countries in need of inexpensive generics, since India is one of the world’s leading producers. According to one generic company’s spokesperson: “The passage of the patent bill, for me personally and for India as a whole, is a very tragic and a very sad day…It will be the start of a predictable, long term tragedy for the country.”

This jeremiad seems like a gross exaggeration. India produces cheap generics not only because it can avoid the expensive R&D that burdens research pharmaceutical companies, but also because it has a low-cost, highly skilled labor force. Most of the drugs that are off patent will remain off patent, and those medicines will stay cheap. Conversely, India’s competition with established pharmaceutical companies in the U.S. and Europe will help spur innovation and drive down prices, since Indian companies can use their lower capital costs to pioneer new medicines at a fraction of the cost. More innovative drugs at lower prices is a win-win scenario for rich and poor nations alike.

The Campaign for Fighting diseases (www.fightingdiseases.org) also points out that patents are not the primary barrier keeping poor countries from accessing needed medicines.

A study by Amir Attaran has shown that in 65 low- and middle income countries, where four billion people live, patenting is rare for the 319 products on the World Health Organization’s Model List of Essential Medicines. Only seventeen essential medicines on the list are on patent in any of the countries, so that overall patent incidence is low (1.4 percent) and concentrated in larger markets…many companies choose not to enforce their patents in certain lower-income countries. Of the 969 cases surveyed by Attaran where companies probably could have obtained and maintained patents for these essential medicines, they did so only 31 percent of the time.


In other words, companies don’t patent drugs in countries that are too poor to pay for them in the first place. Activists want more medicines available in poor countries and attack intellectual property rights in order to bludgeon companies into supporting the activists’ agenda. In the long run, however, undermining support for R&D by attacking patents will inhibit innovation as investors become afraid that new medicines will be compulsorily licensed by governments (like Brazil) or international agencies that want to cure diseases without paying the full cost of the cure.

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Commentary

Congress Should Get Serious About Medicaid
Nina Owcharenko, Heritage Foundation, 3-30-05

Owcharenko notes that the U.S. Senate has excised the very modest Medicaid reforms proposed by President Bush in his budget. The Senate’s refusal to trim Medicaid even at the margins bodes ill for future Congressional efforts to bring entitlement spending under control.

Medicaid, the joint federal-state program for the poor and indigent, is a fiscal disaster. Like Social Security and Medicare, Medicaid consumes an ever-growing portion of the federal budget: Today, these three programs account for 44 percent of all federal spending, and Medicaid alone accounted for 13 percent of mandatory spending in 2004. In future years, Medicaid and other entitlement costs will explode.


The President’s budget contains two very modest proposals for containing Medicaid costs: reducing state financial gimmicks to leverage increased federal payments, and tightening rules for asset transfers whereby individuals can give their assets to their heirs and declare bankruptcy, thereby triggering Medicaid enrollment. The President’s reforms are far from severe. All told, they would only slow future Medicaid growth from a full gallop to a fast trot (7.4 percent down to 7.2 percent). The President is asking for Congress to take a few baby steps today—and if Congress ignores the President’s call for reform it is sure to stumble when more serious measures are needed later on.

TOP^

Kianna's Legacy
Wall Street Journal, 3-29-05

This editorial follows up on last week’s Journal article on Kianna Karnes, a kidney cancer patient who couldn’t get access to potentially life-saving experimental treatments because of the FDA’s “fetish for placebo-controlled testing of cancer drugs.” The companies mentioned in the article contacted Ms. Karnes almost immediately after the article ran to try and get her access to their treatments but, unfortunately, Ms. Karnes succumbed to her disease this week. The Journal writes that it is a

…national scandal that cancer sufferers should have to be written about in The Wall Street Journal to be offered legal access to emerging therapies once they’ve run out of other options. The FDA’s oncology division has proven to be essentially incorrigible on this point in recent years, so it’s time for Congressional action mandating the use of 21st-century science and statistical methods to get these therapies to patients sooner.

TOP^

A Malaria Success
The New York Times, 3-27-05

The Times chronicles one of the few programs to successfully blend economic development and health care infrastructure in Africa.

In 1998, the Australia-based mining company BHP Billiton began building a huge-aluminum smelter outside Maputo, the capital of Mozambique. The company knew that malaria plagued the region. It gave all its workers mosquito nets and free medicine, and sprayed the construction site and workers houses with insecticide. Nevertheless, during the first two years of construction there were 6,000 cases of malaria, and at least 13 contractors died. To deal with the problem, the company did something extraordinary. It joined an effort by South Africa, Mozambique, and Swaziland to eradicate malaria in a swath of the three countries measuring more than 40,000 square miles.


In this region, called the Lubombo Spatial Development Initiative, employed insecticide spraying, disease surveillance, and state of the art treatment to control the mosquito borne disease. The result was that “malaria incidence dropped in one South African province by 96 percent.” The national governments involved encouraged these efforts—“probably the best antimalaria program in the world”—not only to save lives but to “encourage tourism and foreign investment.” Other governments and international agencies that are serious about improving African health care should push for more public-private ventures like this one that link doing-well with the desire to do good.

TOP^

A BALANCED LOOK AT COX-2 DRUGS
Timothy Hla, The Boston Globe, 3-25-05

Human nature being what it is, we are always prone avoid a known risk rather than pursue an uncertain good. Timothy Hla was one of the scientists involved in the discovery of the Cox-2 gene and he is particularly well positioned to offer up his reflections on the potential benefits and risks of these now much maligned drugs.

Because Cox-2 inhibitors have tremendous potential for the prevention and/or treatment of cancer and Alzheimer’s disease, it is extremely important that thoughtful consideration of risks vs. benefits be given to current as well as proposed future uses of these drugs. As one of the scientists involved in the discovery of the Cox-2 gene, I believe that the potential role of Cox-2 is significant enough that we must not let the commotion over adverse effects force us to ignore an entire class of drugs with tremendous potential for controlling human disease.

TOP^

Research

Efficacy of nine-valent pneumococcal conjugate vaccine against pneumonia and invasive pneumococcal disease in The Gambia: randomised, double-blind, placebo-controlled trial
F. T. Cutts, S. M. A. Zaman, The Lancet, 3-30-05

The researchers note that “Pneumonia causes an estimated 19% of the 10 million childhood deaths worldwide. Up to half of all cases of severe childhood pneumonia are caused by pneumococcus in developing countries.” This study, a large double-blind, placebo-controlled trial in Eastern Gambia tested pneumococcal conjugate vaccine against placebo and found that

…333 of 8189 children given vaccine had an episode of radiological pneumonia compared with 513 of 8151 who received placebo. Pneumococcal vaccine efficacy was 37% (95% CI 27–45) against first episode of radiological pneumonia. First episodes of clinical pneumonia were reduced overall by 7% (95% CI 1–12). Efficacy of the conjugate vaccine was 77% (51–90) against invasive pneumococcal disease caused by vaccine serotypes, 50% (21–69) against disease caused by all serotypes, and 15% (7–21) against all-cause admissions. We also found an efficacy of 16% (3–28) against mortality. 110 serious adverse events arose in children given the pneumococcal vaccine compared with 131 in those who received placebo.


The researchers concluded that

In this rural African setting, pneumococcal conjugate vaccine has high efficacy against radiological pneumonia and invasive pneumococcal disease, and can substantially reduce admissions and improve child survival. Pneumococcal conjugate vaccines should be made available to African infants.

TOP^


Medical Progress Today is published by the Center for Medical Progress at the Manhattan Institute for Policy Research.

For more information about Medical Progress Today, please contact the managing editor, Paul Howard, at phoward@manhattan-institute.org, or via telephone at 212.599.7000, x319.

Press inquiries regarding Medical Progress Today can be directed to Lindsay Young, executive director, communications at communications@manhattan-institute.org, or via telephone at 212.599.7000, x315.

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SPOTLIGHT

Our competitors understand why America is the global leader in biotechnology.

NEWS

MS patients plead for a recalled medication Some doctors agree that Tysabri's dramatic results outweigh risks of a rare, fatal disease contracted by two users
Can we limit stroke damage? Because every second counts, patients in a novel study will be treated before they reach the hospital.
New Vaccine Said to Offer Hope Against Bacterium
Back from the brink | Millionaire father rescues company; knew drug would help his daughter
India Adopts Patent Law Covering Pharmaceuticals

COMMENTARY

Congress Should Get Serious About Medicaid
Kianna's Legacy
A Malaria Success
A BALANCED LOOK AT COX-2 DRUGS

RESEARCH

Efficacy of nine-valent pneumococcal conjugate vaccine against pneumonia and invasive pneumococcal disease in The Gambia: randomised, double-blind, placebo-controlled trial
Center for Medical Progress 
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52 Vanderbilt Avenue
New York, NY 10017
(212) 599-7000
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