I am still recovering from Wednesday evening. The ever gracious Grace-Marie Turner (of Galen Institute fame) hosted a memorable launch party for the Center for Medicine in the Public Interest (the public policy home of drugwonks.com.)

Memorable for so many reasons but mostly because of those who attended — FDA Deputy Commissioners Janet Woodcock and Scott Gottlieb, NCI Deputy Director Anna Barker, Julie Goon, the new White House health care policy guru, along with my former FDA colleagues Dan Troy and John Taylor, former NIMH director (and CMPI board member) Dr. Fred Goodwin — and our keynote speaker, Dr. Mark McClellan — the hardest working man in health care. Here are some of the kind things Mark had to say:

“… CMPI is focusing on the right issues at the right time. And with the right people.”

“… CMPI is about great ideas and effective communication. CMPI begins at the right time, promoting ideas that make a difference in medicine that is personalized, predictive, preventative, and therefore effective in the 21st century.”

“… It’s nice to see ideas, communicated effectively, that translate into policies that change the way healthcare is being delivered in this country. That is happening because of what Peter and Bob and all of you here tonight are doing day in and day out, tirelessly, often thanklessly — but it is making a huge difference.”

“… I’m certain that CMPI will be an important new force in health care policy — a force that will help to get better healthcare for all Americans.”

Well golly.

That’s a lot to live up to — and we intend to exceed expectations.

New op-ed, “Putting Clinical Trials in the Dock.”

Have a read:

Putting clinical trials in the dock
The Roanoke Times
June 27, 2006

BY: Peter Pitts

Pitts, a former Food and Drug Administration associate commissioner, is director of the Center for Medicine in the Public Interest.

“One size fits all” rarely does. From clothes to shoes to hats, few people find that items carrying that label work with their individual bodies. So why do we entrust the health of our bodies — one of the most important assets we have — to a one-size-fits-all mentality?

Unfortunatly, that’s exactly what the influential movement known as “evidence-based medicine” does. It sounds like a good thing, does’t it? Of course we want our health care based on evidence. But the phrase is misleading. As it turns out, “evidence-based medicine” often ignores the most critical evidence of all — the individual patient.

Advocates of EBM urge doctors to base their clinical decisions on research findings. Randomized clinical trials become crucial under this regime: The results of these trials are what doctors overwhelmingly base their suggested treatment on.

The problem is that clinical trials aren’t the only thing doctors need to take into account when making decisions. Evidence-based medicine emphasizes just one aspect of the clinical pie over all the others. This model, which began taking shape in the 1970s, is now broken and outdated.

EBM is stuck in the past. For the most part, it is a retrospective look at clinical studies and head-to-head comparisons of medicines and medical procedures. EBM may involve a careful look at the science. But in practice, it’s very limited. All of these studies are population-based, have rigid exclusion criteria and can’t integrate new information or innovations.

The result is decidedly and transparently a narrow one: to eliminate “practice variation.” Indeed, that’s one of the primary aims of EBM — to standardize medicine around what is known to work.

Practice variation is any treatment that varies from the norm that EBM prescribes. The canard of evidence-based medicine is the belief that practice variation is bad and that one-size-fits-all medicine is good. EBM presupposes that all people respond precisely the same way to all medicines. But that’s simply not true. Disease varies by individual, and selection of treatment must be driven by diagnostics, not just guidelines.

Today, the science of genomics is ushering in an age of personalized medicine. Clinical outcomes can be monitored with increasing precision. Computers can control for hundreds of variables to help doctors and researchers identify what treatment steps matter most in improving care.

People can be screened with a variety of molecular diagnostics to reduce side effects, increase compliance, improve outcomes, and even prevent various forms of cancer, depression, hypertension, Alzheimer’s and immune disorders.

This is 21st-century evidence-based medicine — patient-centric and cost-efficient.

The triumph of modern medicine is that it can be so precisely targeted to a single patient’s needs. It is a dramatic leap forward from EBM’s sweeping approach, which sees only through the broad lens of population-based studies, and the individual patient is kept out of focus.

Unfortunately, EBM continues to enjoy broad support in the policy community. Why? Because at its core, evidence-based medicine is cost-based rather than patient-based. In other words, its standardized approach supposedly saves money. But this is extremely short-sighted.

Evidence-based medicine may provide transitory savings in the short term, but the same patient who takes the cheapest available statin today may very well be the patient costing you — the taxpayer, the policymaker, the thought-leader, the sister, the spouse — big bucks when that patient ends up in the hospital because of improperly treated cardiovascular disease.

The repercussions of choosing short-term thinking over long-term results and cost-based medicine over patient-based are pernicious to both the public purse and the public health.

We need a new approach. The health care community must work together to develop new cost-efficient programs that account for modern genomics and individual screening. Because “one size fits all” treatments are dangerously outdated in this era of patient-centric medicine.

The FDA has decided to shelve its plans of opening an Indian office to help expedite regulatory clearances for Indian drug exporters to US.

Sources said Dr David Lepay, the FDA’s Senior Advisor for Clinical Science and Director, GCP Programs revealed at a session on Asian clinical trials at the 42nd annual meeting of the Drug Information Association (DIA) in Philadelphia, USA, that the US food and drug regulator was not planning to set up offices neither in India or anywhere else in Asia, at present. The announcement from the FDA official came in the presence of Dr Ashwini Kumar, Drug Controller General of India (DCGI), who was also a speaker at the program.

Pharmabiz had reported about two years ago that the FDA was planning to start its Indian office at New Delhi with an initial investment of about $5 million, considering the fact that India has the maximum number of FDA-approved facilities outside the US. This office was to facilitate Indian companies with the procedures of filing for marketing approval for products to be launched in US, application for site inspection and other procedures dealing with the FDA. The office was envisaged to facilitate the exchange of communication between Indian companies and the US authorities, thereby saving a lot of time and resources, sources had told Pharmabiz.

Dr Leepay told the seminar, attended by a good contingent of Indian participants, that it was not mandatory that a new drug marketing application in the US be supported by a US study and there had been instances of marketing applications wholly supported by non-US studies. However, the FDA had criteria that non-US studies were expected to meet US standards and that non-US data was reviewed to the same standards as data from the US. Though the FDA had a long history of inspection of clinical trials outside the US, its experience in the Asia/Pacific region was limited, and the region currently accounts for only 5 per cent of FDA’s international inspections. He also clarified that the FDA does not certify clinical investigators, clinical sites, sponsors or ethics committees, nor does it approve study protocols or informed consent documents.

The latest broadside against the FDA is by Congressman Waxman who argues the FDA is doing a lousy job by not prosecuting and attacking companies who are producing inferior products, engaging in mislabeling or selling suspicious goods. This is the sort of after fact enforcement activity that pols love since it gives them something to hold hearing about. I call it regulation by body count since it requires people to die or be harmed in order for a regulatory or enforcement act to take place. In contrast, since Mark McClellan became commissioner in 2003, the FDA has sought to improve overall product quality through manufacturing efficiencies, risk management programs and other quality improvement efforts that are not politically sexy and often take years and millions of dollars to implement. (Not to mention cooperation. ) Indeed manufacturing is part of the Critical Path lest anyone forgets.

Of course, the drive by media ignores all of this. And the FDA has not done enough to get the message out about these important initiatives. One more thing: Why does Gardiner Harris refer to the ‘conservative’ American Enterprise Institute and the ‘watchdog’ Public Citizen? Public Citizen is nothing but ‘liberal’ and as for watchdog, that is a matter of opinion, not absolute truth. Even a Rockefeller can figure that one out.

At a time when the social scientists are telling us that post 9/11 Americans are more safety-conscious/risk-averse then ever before — while at the same time looking to medical science for ways to live longer, healthier lives at lower costs — the announcement that the FDA is going to lead the charge towards a 21st century model for clinical trials is good news — very good news.

Here’s the announcement. Further detail can be found at www.fda.gov.

FDA Announces New Initiative to Modernize the Regulation of Clinical Trials and Bioresearch Monitoring

The Food and Drug Administration today announced a series of new policy and regulatory developments to strengthen the Agency’s oversight and protection of patients in clinical trials and the integrity of resulting data in an effort to modernize the agency’s approach to bioresearch monitoring as part of the Critical Path Initiative. The Human Subject Protection and Bioresearch Monitoring (HSP/BIMO) Initiative will facilitate the modernization of the regulation of clinical trials and bioresearch monitoring, specifically the protection of human subjects and the integrity of data in clinical trials, and encompasses devices, foods, human drugs, biological drug products and veterinary medicine.

The new effort is part of an HHS-wide initiative to employ recent advances in basic science, including genomics and molecular analysis, in order to bring about more effective development and review of therapies, and to enable increasingly targeted and individualized care management for patients.

“As clinical trials continue to evolve, in particular becoming increasingly large, decentralized and global, the FDA’s approach to bioresearch monitoring and human subject protection must also evolve and modernize,” said Janet Woodcock, FDA Deputy Commissioner for Operations at this year’s Drug Information Association annual meeting. “BIMO will help FDA modernize biomedical research monitoring making the most efficient use of its resources to help ensure the safe conduct of clinical trials, including taking appropriate opportunities to leverage existing oversight done by private entities to accomplish the Agency’s risk minimization goals.”

Clinical trials have evolved dramatically since FDA first began inspecting them in 1977. In an effort to protect the rights and welfare of human subjects and to verify the quality and integrity of data submitted for review, FDA established over time a bioresearch monitoring program that included the development and implementation of compliance programs to provide guidance for inspections of investigators, sponsors, contract research organizations, institutional review boards and bioequivalence facilities. With the expansion of clinical trial studies and sites, electronic record-keeping in the studies, and greater participation by vulnerable subjects in clinical trials, the role of FDA’s bioresearch monitoring compliance programs must expand and evolve as well. The HSP/BIMO Initiative addresses that need.

Over the past year and a half, FDA has carefully inventoried its programs and identified issues to launch the HSP/BIMO Initiative. As this initiative moves forward, FDA will continue to gather additional issues for the initiative and related information from internal and external stakeholders, e.g., industry, academic, and government activities and programs, and intends to conduct workshops and create other opportunities for public input.

Janet Woodcock, M.D., Deputy Commissioner for Operations, will chair the HSP/BIMO steering committee which is comprised of representatives from the Center for Biologics Evaluation and Research (CBER), Center for Drug Evaluation and Research (CDER), Center for Food, Safety, and Nutrition (CFSAN), Center for Veterinary Medicine (CVM), Office of Regulatory Affairs (ORA), and the Office of the Commissioner (OC).

Great piece today in the NY Post by Dr. Marc Siegel about how the fearmongering by the media and certain members of Congress is driving his patients away from taking medicines that can keep them alive. Marc has written well about how we should use science — not fear — to guide policy and medical decisions.

Here’s the entire article:

POLARIZED PILLS
By MARC K. SIEGEL

June 23, 2006 — MEDICINES are too often portrayed as either life savers or killers - a polarization of our pills that serves neither science nor health care.

Thanks to two high-profile lawsuits, my patients are now asking me if Lipitor - the cholesterol-lowering drug - is still safe. With the suits claiming that Lipitor can cause brain and nerve damage, my old comeback - “I take the drug myself” - is no longer sufficient to calm fears. One patient tells me that I’m blindly ignoring the risks of serious side effects.

In fact, Lipitor, the country’s top-selling prescription drug, has been shown to prevent the progression of coronary plaques in patients with heart disease, and is likely to be as useful in patients who are at risk for heart attack and stroke from these same plaques.

The most potent drug in the class known as statins, Lipitor has been successfully administered to millions. In very rare cases, it can trigger a severe muscle breakdown known as rhabdomyalysis. It has never been proven to cause memory loss or nerve damage, and I and most other physicians believe it to be a safe and effective drug.

So why the worry? Part of the problem is the way the drug industry hypes its products, setting them up as some kind of panacea. But if it’s sold as a magic elixir, the discovery of any flaw rings alarm bells.

Not all drugs that are victims of the pendulum swing from panacea to panic are as famous or as successful as Lipitor. This month, an FDA safety panel also cancelled a study where 4,000 children were to receive the antibiotic Ketek, an effective treatment for bronchitis and sinus infections. Why stop the study? Ketek, a new drug, has been prescribed to over 5 million people over the past two years, but 12 have sustained liver failure (in four cases, fatally), while 23 others have suffered damaged livers.

Should Ketek be restricted, labeled with ominous warnings or taken off the market entirely? Not without much better evidence of danger. At a time when few new antibiotics are being developed, drug-resistant bacteria continue to emerge, drugs like Ketek are important tools.

Unfortunately, when the media and the lawyers target a drug, they overlook the fact that the side effects are rare, and/or alternative treatments more problematic. Sober statistics-based analysis gets tossed aside. The drug-maker’s stock price and the number of prescriptions written plummet.

Decisions on drug safety should be based on real facts - a weighing of the real risks and benefits. Hysteria doesn’t belong in the drug-safety equation.

Dr. Marc K. Siegel is an internist at NYU Medical Center and associate professor at the NYU School of Medicine.

I should also add that the VA doesn’t pay pharmacy costs. And that’s anywhere between 20%-40% of the purchase price. One more time folks — a half truth is a whole lie.

The battle continues apace. Please have a look at this new op-ed (penned by myself and Dr. Bob) that appears in today’s edition of The Washington Times.

And please pass it along.

Here’s the link:

http://www.washingtontimes.com/op-ed/20060621-085649-7788r.htm

How shameless can a politician be…Chuck Schumer attacking Merck for lowering it’s Zocor prices in response to impeding generic competition..Interestingly, when he introduced changes to the law promoting generic drugs he lauded his proposals as “all about promoting competition in the drug industry…” Except when it comes to creating competition for generic drug firms? Since when did they become a protected class and since when did protecting them become more important than consumers. Since about 15 percent of consumers stick with a generic after a switch, a Zocor price slash will save them money, Schumer rather than later.

June 20, 2006, 2:03PM
Senator Raps Drug Giant Over Zocor Price

By DEVLIN BARRETT Associated Press Writer
© 2006 The Associated Press

WASHINGTON â A New York senator accused the drug giant Merck & Co. on Tuesday of conspiring to undercut a cheaper generic alternative to its cholesterol-lowering drug Zocor just days before it becomes available to patients.

Sen. Charles Schumer, a Democrat, charged that Merck is quietly collaborating with health insurance companies to create lower copays for customers buying Zocor than for those buying the generic equivalent.

Zocor generated $4.4 billion in sales last year, and generic alternatives will be available for the first time on Friday. A company executive scoffed at Schumer’s allegations.

“It appears that Senator Schumer is criticizing us because he says that our prices are too low. That’s a new one,” said Ian Spatz, Merck’s vice president for public policy.

“The truth is that we support generic competition and the generic competition for Zocor is good for patients. It’s good for people who have to pay for medicine, which include health plans. We’re going to continue to price it competitively,” he said.

Can you say penny-wise and pound foolish? This from Health IT News.. The GOP stalls a bill to encourage the diffusion of health IT because it might — I repeat might — drive up health care spending in the short term as people invest in new hardware and systems to achieve interoperability and patient-centered. Would these Republicans have stalled investment in the highway system or the transcontinental railroad because it drove up costs in the short term?

http://www.healthcareitnews.com/story.cms?id=5109

House Republicans stall healthcare IT bill
Healthcare IT News
By Bernie Monegain, Managing Editor 06/20/06

WASHINGTON â A healthcare IT bill the U.S. House of Representives was expected to vote on this week is expected be put on hold.

Congress Daily reports the move to hold off on the bill came after the Congressional Budget Office forecast the legislation would increase spending and reduce revenue.

The bill bill promotes the adoption of healthcare information technology and calls for national standards for implementing electronic health records.