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April 30, 2008

IMI Shelter

by Peter Pitts

While we dither about funding for the Reagan/Udall Center, the European Commission and the pharmaceutical industry have unveiled a €2 billion partnership to reverse the EU's declining international role in medical research. According to a report in the Financial Times, teams of commercial and not-for-profit researchers will be able to seek support for work on a range of medical projects, on condition that their findings are publicly shared in an effort to stimulate faster and safer drug development.

The Innovative Medicines Initiative (IMI), jointly supported by the Commission and members of the European Federation of Pharmaceutical Industries and Associations (EFPIA), will shortly release details of its first 18 priorities, which will be funded by €123 million in awards to be given out by the spring of 2009.

This is a chance for us to really get back on the right foot," said Arthur Higgins, president of EFPIA and head of Bayer Healthcare of Germany. It was important for patients and the wealth and prosperity of Europe. "We're sending a signal to the US, Japan, China and India that Europe is taking its bioscience sector really seriously."

The first wave of projects includes the development of measurable biomarkers in the body to gauge the side-effects of new medicines on the kidneys and liver, as well as research into the basic mechanisms of pain, severe asthma, psychiatric disorders and neurodegenerative diseases.

The Commission has given €1 billion in funding for the seven-year program, which will be matched by support from pharmaceutical companies through loans of staff, laboratory equipment and libraries of compounds.

It’s time that we take the issue of 21st century medicine just as seriously.

Funding for Reagan/Udall now!

Posted by peterpitts at 08:26 AM
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April 29, 2008

Follow-on O Ship of State

by Peter Pitts

Excellent piece by Bryan Liang in the Los Angeles Times:

Don’t Compromise the Safety of Biotech Drugs

By Bryan A. Liang
April 28, 2008

A toy plane has a handful of parts. A Boeing 747 has several million. This makes sense. Toy planes are small, simple models, while 747s are large, high-performance aircraft that travel more than 500 mph with thousands of component systems acting together. The model costs a few dollars because it's easy to manufacture. The 747 costs about $225 million because of its highly complex nature, testing and the need to ensure safety.
The comparison is worth keeping in mind as the debate heats up over "follow-on" biologics. Biologics are today's most advanced medicines, fully tested biotechnology protein drugs that provide targeted therapy to victims of cancer and other diseases. Follow-on biologics are the second or subsequent versions, but they are not identical.

U.S. spending on them reflects the importance of these drugs in medicine's arsenal. Biologics represent the fasting-growing sector in the medicines market, with more than $30 billion spent on these drugs each year. Indeed, the top five drugs in terms of Medicare expenditures administered in physicians' offices are biologics. By 2010, worldwide spending on biologics is estimated to grow to $10 billion, and biologics will make up nearly half of all newly approved medicines. Hence, many policymakers are focused on reducing the costs associated with these drugs.

Congressional legislation is pending that would allow the sale of follow-on biologics without requiring extensive testing -- essentially following the same model used for approval of generic forms of traditional prescription drugs.

But most drugs we're familiar with, like the pills we get from the pharmacy, are "small-molecule" drugs -- simple chemical compounds. They can be easily manufactured and identically copied. The anti-convulsant drug valproic acid, for example, has a total of just 26 atoms.

Identical to the brand-name version, these generics can "piggyback" on a brand-name company's testing. That's reasonable. These small-molecule drugs -- which typically are made up of a total of 20 to 100 atoms -- can be copied perfectly. So they don't need independent safety testing, cost less to make and are cheaper -- allowing more patients to obtain the medical benefits.

But biologics are far more complex. The brand-name drug Herceptin, a biologic that's widely used to treat cancer, is made up of a total of roughly 25,000 atoms. Large biologics can have millions of atoms.

Biologics aren't made by combining chemicals in a flask. They're made by life forms such as cells, yeast and bacteria. Like humans, these life forms exhibit diversity in metabolism and composition, making the final product a unique, heterogeneous mix that cannot be copied exactly. So follow-on biologic forms of a drug can only be similar to the original, not identical.

Because of the complexity of biologics, there's more regulation. In comparison with common chemical drugs that can have generic versions -- such as penicillin -- which only require 50 to 60 manufacturing tests for safety and quality, biologics require at least four times that number.

So the policy challenge is to provide incentives for innovation while also ensuring that any follow-on forms of biologics that enter the market are safe.

The key lies in something called "data exclusivity," which is a legal mechanism for allowing a company to keep confidential the data associated with a drug's development. Data exclusivity usually lasts for several years and spurs innovation by protecting new inventions. This is the current rule for small-molecule drugs, and it should be applied to biologics as well. Strong data exclusivity is critical for biologics, which are about 50% more expensive to develop than small-molecule medicines.

However, the current state of science makes ensuring safety of follow-on biologics difficult. Currently, the technology to map out the exact chemical structure and function of one large biologic versus another is not available. That makes safety reviews inexact. And follow-on forms may induce unpredictable adverse reactions.

Several years ago, a fully tested biologic created in the U.S. was cooperatively licensed overseas to be made in Europe. But the new version caused patients to suffer "pure red cell aplasia," whereby their bodies could not make red blood cells. This may have contributed to the deaths of some patients and permanent injury to others. Yet today, after eight years of research, the cause of these reactions is still unknown.

If even cooperative company efforts can result in unpredictable adverse reactions, any follow-on product that does not undergo full testing should be of concern. Recognizing this reality, the European Union has developed a system of assessment that requires clinical testing of follow-on products in Europe before approval.

U.S. policymakers should take note. Relevant clinical data and testing should be required to ensure safety of any follow-on biologic product being considered for patient use in the U.S. And appropriate data exclusivity should be put in place to foster innovation.

If chemical drugs were toy planes, biologics would be 747s, the cutting edge of medicine. Because of their complexity, any follow-on forms must be held to a higher safety standard. As we have learned in aviation, safety is no accident.

Bryan A. Liang is executive director of the Institute of Health Law Studies, California Western School of Law and co-director of the San Diego Center for Patient Safety, UC San Diego School of Medicine.

Posted by peterpitts at 08:05 AM
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April 25, 2008

EU Considers Merging onto the Healthcare Information Superhighway

by Peter Pitts

Maybe it has something to do with the strength of the Euro -- or maybe it has something to do with a patient's right to know. And just maybe things are beginning to change.

Have a look at this new article (from the Journal of Life Sciences) on how the EU is pondering changing what's allowable vis-a-vis what they call "Information to Patients" and we in the US call "Direct to Consumer Communications:

http://www.tjols.com/article-640.html

Bottom line -- knowledge is power.

Posted by peterpitts at 08:30 AM
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April 18, 2008

Slamma JAMA

by Peter Pitts

What ever happened to dialogue and ... ethics?

In it's reporting/editorializing on the recent "what-did-Merck-know-and-when-did-it-know-it" saga, JAMA accuses some pretty high-powered healthcare professionals of shilling for shillings.

Now it comes to light that the folks JAMA denounced weren't even given the chance by the Sultans of Science to defend themselves.

Here's what the Washington Post says,

"Although the two studies question the integrity of dozens of physicians and scientists, the JAMA authors did not seek responses from them. Several of those people yesterday called the conclusions incorrect, incomplete or unfair."

Don't "real" journalists" seek out both sides of the story? Don't real scientists?

Posted by peterpitts at 02:22 PM
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April 15, 2008

"Evidence-Based" Rx Miscues

by Peter Pitts

From today's Washington Times ...

"Evidence-based" Rx miscues

Hillary Clinton, Barack Obama and John McCain have been traveling the country laying out their solutions to the problem of escalating health-care costs. One plan they all favor is ramping up federal funding for so-called "evidence-based" medicine.

The theory behind evidence-based medicine is simple: If the government were to run clinical trials testing the effectiveness of drugs and medical technologies, and then use the results to determine what to cover, taxpayers would avoid paying for treatments that aren't effective enough to justify their price tag.

Sounds great, right? Too bad that in practice, evidence-based programs are largely driven by the political imperative to cut costs — not the medical imperative to give patients the best care possible.

That was certainly the case for CATIE, or the Clinical Antipsychotic Trials in Intervention Effectiveness. This federally funded, $40 million study concluded new "atypical" nonpsychotic drugs are no more effective at treating schizophrenia symptoms than are older drugs. Because this finding flatly contradicted psychiatrists' real-world observations, CATIE had no impact on which drugs were prescribed for schizophrenia.

Like most large-scale trials, CATIE took a one-size-fits-all approach to medicine. Evidence-based programs encourage this approach. The underlying assumption is that the same care can be applied to every patient suffering from the same disease.

Modern science disproves that notion. Everyone has a unique biological makeup. Health-care professionals need to be given the autonomy to tailor their treatments to the specific needs of their patents.

Evidence-based programs rarely provide that autonomy. In the long run, this often results in higher costs.

Consider an overweight man who is forced to take a cheaper, less effective anti-cholesterol drug. If he ends up in the emergency room because of undertreated cardiovascular disease, this could end up costing the health-care system significantly more money.

All too often, denying patients access to the right medicines early on means paying for more invasive and expensive procedures later.

The evidence confirms this. A study from the University of Utah examined the relationship between cost-containment programs and the total cost of health care for a number of medical conditions. It found that the tighter the restrictions on which treatments a physician could administer, the higher the overall cost of care.

Medical treatment should be based on the specific genetic, clinical and demographic factors of an individual patient. That's how you keep people healthy. In an era of personalized medicine, one-size-fits-all health-care strategies are dangerously outdated.

Choosing short-term savings over long-term results has a pernicious effect on the public purse and public health. Strenuous cost controls compromise patient care.

The theory behind and the practice of evidence-based medicine just don't match up. And until politicians can show how they'll resolve that tension, they need to look elsewhere in their quest to find politically palpable solutions to the country's health-care woes.

Peter J. Pitts is a former associate commissioner of the Food and Drug Administration.

Posted by peterpitts at 10:32 AM
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April 14, 2008

Let's Talk Mandate

by Peter Pitts

Not.

Twenty-nine percent (29%) of American adults favor a national health insurance program overseen by the Federal Government. A Rasmussen Reports national telephone survey found that 39% oppose such a government-led initiative while 31% are not sure.

The survey also found that 46% believe the quality of care would decrease under a national health insurance program while 16% believe that quality would increase. Twenty percent (20%) say the quality of care would remain about the same while 18% are not sure.

At the same time, 42% believe the cost of health care would increase while 25% would expect prices to go down.

While opposing a national program overseen by the federal government, Americans support requiring companies to provide health insurance for their employees. Sixty-three percent (63%) favor such a requirement while 24% are opposed.

Posted by peterpitts at 08:50 AM
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April 11, 2008

Congress Gets Tough on ... Drug Importation?

by Peter Pitts

According to Congressional Quarterly, Chairman Dingell is aggressively pursuing efforts to require the FDA to take a much more aggressive role in monitoring food and drug production and safety abroad, with stiff penalties for companies that import tainted products. The article says: “Dingell’s plan would also crack down on companies that violate drug import regulations. Manufacturers and importers could be fined as much as $500,000 for bringing contaminated or adulterated food or drugs into the country, and individuals could be subject to fines as high as $100,000 for similar offenses.”

You may ask, How is that statement compatible with legislators’ calls for liberalized prescription drug importation? Good question.

Hopefully Mr. Dingell will point this out when his colleagues raise the subject.

Posted by peterpitts at 11:05 AM
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April 10, 2008

FDA Addresses Tort(i) Reform

by Peter Pitts

Commissioner of Food and Drugs Andrew C. von Eschenbach, M.D. announced the appointment of Frank M. Torti, M.D., M.P.H. as the FDA's Principal Deputy Commissioner and first Chief Scientist.

The newly created Chief Scientist position stems from the Food and Drug Administration Amendments Act of 2007.

"Dr. Torti's impressive clinical and scientific credentials are an excellent match for the work we do on a daily basis to promote and protect the nation's health as a science-based and science-led agency," said Dr. von Eschenbach. "FDA's Chief Scientist will ensure that the foundation of the FDA's regulatory structure will always be state-of-the-art science."

As Chief Scientist and a member of the agency's senior leadership team, Dr. Torti will support the launch of the FDA Fellowship Program, which has the potential to attract up to 2,000 professionals of varying disciplines for a two year training program. As well, the new office will work to ensure the quality and regulatory focus of the intramural research programs of the agency, and place special emphasis on the importance of clinical research trials that are a part of the foundation of the FDA's regulatory structure.

“I appreciate the confidence and trust that Secretary Leavitt and Commissioner von Eschenbach have placed in me," said Dr. Torti. “I look forward to beginning this work at this important moment for the FDA."

A prominent clinician, scientist and researcher in molecular oncology, Dr. Torti is currently serving as Charles L. Spurr Professor of Medicine, Chair of the Department of Cancer Biology, and Director of the Comprehensive Cancer Center at Wake Forest University School of Medicine in Winston-Salem, N.C.

Dr. Torti received his bachelor's and master's from Johns Hopkins University, his medical degree from Harvard Medical School, and his Master of Public Health from Harvard School of Public Health. He served as an intern and resident at Beth Israel Hospital in Boston, and a fellow of medical oncology at Stanford University where he subsequently joined the faculty and was tenured. He has written extensively on prostate and bladder cancer, designed and executed a number of cancer clinical trials, and has substantially contributed to the understanding of the molecular mechanisms that underlie inflammatory diseases and cancer.

Throughout his career, Dr. Torti has served on and chaired a number of national health and medical committees. He currently serves on the National Institutes of Health's National Advisory Council for Complementary and Alternative Medicine. He also founded and serves as President of the Cancer Biology Training Consortium, a national society of cancer biology department chairs and program directors. He is the recipient of a National Institutes of Health MERIT Award.

Dr. Torti will join the FDA in May.

Posted by peterpitts at 08:02 AM
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