Lies About Generics and Access to Medicines

03/14/2007 11:53 PM |

I have been going back and forth in my own mind about my initial reaction to Abbott's decision to not sell new medicines in Thailand pending a shift in the policy of patent theft on the part of the junta-run government there. At first I thought, this will only make the perception worse. But then it struck me it is not as if compulsory licensing has actually made any medicines any more available in any country. The only people who benefit from compulsory licensing appear to be the small circle of generic companies, government bureaucrats and media-thirsty NGOs who use the tactic to use drug companies to divert attention away from the real problems causing disease. Just as free food does not solve the problem of world hunger and free measles shots has not eradicated measles (500,000 African kids a year still die from measles) the short sick history of CL has proven that low cost copycats of drugs do not improve health in third world countries. Just the opposite, it usually makes things worse, encouraging piracy, profiteering and counterfeiting. I have said it before and I will say it again, the NGOs who support CL are more interested in killing drug companies than in saving lives.

Good for Abbott and Novartis for refusing to sell new medicines. Other companies should follow suit... and set up their own distribution programs as necessary.  Read More & Comment...

A Breakthrough: Pharma CEO Talks About Science

03/14/2007 11:46 PM |

Sid Taurel, Lilly CEO has an editorial in the Boston Herald about the mission of drug companies, of all things. More CEOs should talk about the science they are investing in and do it often.


Taurel writes:

"We are now beginning to understand biology as a set of information processes, and we’re developing realistic models of how disease and aging progress. And, we are developing the tools to reprogram them.
I believe that this represents the most exciting and important story in any technology field in this new century.
It might take decades for biologists to map all of this territory . . . and fully solve these new puzzles. But the good news is that patients will not have to wait decades to see the medical benefits of this massive effort. "

Unless military juntas, backed by certain members of Congress, make a hash of IP globally.



http://news.bostonherald.com/editorial/view.bg?articleid=187903&format=&page=1  Read More & Comment...

FDA Puts a Black Box Around Sleeping While Driving

03/14/2007 11:25 PM |

This from fark.com...

FDA and drug makers claim that patients who eat and drive while under the influence of sleeping pills are actually asleep, rather than awake, intoxicated, and making bad decisions.  Read More & Comment...

Henry Howls. Public Pays.

03/14/2007 07:07 AM |

What's the cost of playing politics with health care? Well, in this case, it seems to be $10 billion -- charged to the American tax payer by Representatives Waxman, Barton, and McCrery.

Lawmaker Inquiry May Bump Medicare Costs
By KEVIN FREKING, Associated Press Writer

WASHINGTON -- A lawmaker's inquiry could slightly increase the cost of the Medicare drug benefit if its results are publicized, congressional auditors said Tuesday.

Rep. Henry Waxman, D-Calif., has asked several insurers sponsoring Medicare drug plans to submit information about price concessions they wring from drug manufacturers. That pricing information is given to the Centers for Medicare and Medicaid Services, but cannot be disclosed.

Waxman's request has pharmaceutical companies worried, though he has not said what he would do with the information once he gets it.

The Congressional Budget Office estimates that disclosing the discounts means the cost of the drug benefit would likely increase by less than $10 billion over a 10-year period, and possibly much less.

The program is expected to cost about $45 billion this year alone.

Under the drug program, manufacturers typically give insurers a discount if they agree to cover a particular drug for their customers. The manufacturers would be less likely to offer the large rebates if they had to provide it to other types of insurance plans in the private sector, which would increase costs for the government and for enrollees, auditors said.

The publication of the prices also could encourage "tacit collusion" among the manufacturers of comparable medicines, they said. Such collusion occurs if drug makers offer similar discounts rather than trying to outdo each other in their pricing.

The CBO had originally projected in 2003 that disclosing discounts would increase the cost of the program by $40 billion over 10 years. The CBO lowered its estimate, in part, because Medicare plans are not getting substantially different discounts than what commercial plans get. The CBO had originally projected that Medicare plans would get substantially larger discounts, but the difference has been narrower than expected.

Two Republicans, Reps. Joe Barton of Texas and Jim McCrery of Louisiana, requested the CBO analysis.  Read More & Comment...

Denial is more than just a river in ... Thailand

03/13/2007 10:09 AM |

And what did the military junta expect, a big wet kiss?

Abbott Won't Launch New Drugs
In Thailand After Patent Revocation
By NICHOLAS ZAMISKA

Abbott Laboratories has decided against launching any new medicines in Thailand in response to the military-installed government's decision to revoke the company's patent for its blockbuster AIDS drug, according to people familiar with the matter.

The U.S. drug maker has also withdrawn its current drug applications from the government review process, these people said, adding that Abbott has no plans to stop selling drugs that are currently on the market.

Abbott's move, which could leave patients in Thailand with fewer options for treatment of certain conditions, raises the stakes in a battle between multinational pharmaceutical companies and the Thai government that took power following a military coup last year.

In January, the government said it would suspend patent protections for two drugs to make them more widely available to patients who need them. These included the HIV treatment Kaletra, made by Abbott, of Abbott Park, Ill., and Plavix, a blood-thinning drug originally developed by Sanofi-Aventis SA of Paris and co-marketed in several countries by New York-based Bristol-Myers Squibb Co.

In certain situations, including national emergencies, World Trade Organization rules allow a government to unilaterally make or sell patented drugs without the permission of the drug companies. However, pharmaceutical companies have criticized Thailand for stretching the scope of those rules beyond widely accepted boundaries.

Abbott has withdrawn its application for seven medicines, according to a person familiar with the matter, including a new formulation of Kaletra, the AIDS treatment. Abbott notified the Thai government a few weeks ago, after talks between the two sides broke down, the person familiar with the matter said.

A decision that may keep critical, life-saving drugs away of patients who need them could prove controversial for Abbott. "It's not good for anyone, even the American company because they will lose the market," says Thawat Suntrajarn, director general of the Ministry of Health's department of disease control, who says that he was unaware of Abbott's decision.  Read More & Comment...

Beyond REMS for Dems

03/13/2007 08:15 AM |

Mark McClellan remains the hardest working man in American health care -- and one of the most astute.

Consider his recent remarks at the IOM symposium on the drug safety challenges faced by the FDA. Mark makes a very strong case that the agency should make more use of the pooled information contained in large health-care databases, like those run by private insurers that include information about 100 million Americans.

According to McClellan, surveillance, for problems like the increased risk of heart attack and stroke that led to the withdrawal of the popular painkiller Vioxx in 2004, could broaden the FDA’s view of drug safety problems as they emerge while respecting patient confidentiality.

Here's a link to the media report on Mark's comments:

http://www.msnbc.msn.com/id/17583122/

That's FDA reform for all -- not just REMS for Dems.  Read More & Comment...

News from the Oversighter-and-Chief

03/13/2007 08:06 AM |

Scott Gottlieb, in this Forbes.com piece, weighs in on the latest lunacy from Mr. Waxman, Congress' new Oversighter-and-Chief.

Medicine & Markets
Democrats Opt For Socialism Over Success

Scott Gottlieb, M.D.

A series of sharply worded letters fired off by a California congressman to health insurance companies, demanding that they disclose proprietary price data on what they pay for drugs they dispense to Medicare members, could undermine the government’s new drug benefit--a program that by all measures is working.

Medicare’s new program relies on networks of private drug plans, all competing to offer attractive benefits and discounted drugs in order to sign up new members. Most of the health plans have enrolled millions of members, and they have used this purchasing clout to extract deep discounts from the drug makers, translating into cheaper health coverage for Medicare members.

But even a successful program, so long as it relies on elements of the private market, is anathema to politicians if the political aim is health care run solely by Washington.

Enter Rep. Henry Waxman, D-Calif., the new head of the House Committee on Oversight and Government Reform. Under the guise of "oversight," Waxman sent letters to the 12 largest prescription drug plans, including Aetna, Humana and Wellpoint.

He demanded that they divulge data on the prices that they are paying for the drugs they offer beneficiaries, along with their administrative costs, negotiated price discounts and other price concessions obtained from drug makers. Waxman said he wants to determine how profitable they are and how much of the savings they negotiate is passed on to Medicare beneficiaries.

Rest assured, the Waxman dispatches wont push the plans off the cliff, so do not expect near-term impacts. But disclosing this commercial and confidential data could slowly erode the competitive activities that enable the Part D plans to save consumers' money and the new benefit program to lower drug costs.

Competition between the plans to lower costs turns on the negotiations that take place between the health plans and drug makers, and the ability of a drug maker to offer a preferred health plan more favorable pricing, often bundling together different drugs in one negotiation, without having to offer the same price to everyone else. This kind of price discrimination enables health plans use leverage and dealmaking to extract the lowest price for the panels of drugs that are most suitable to their members.

Disclosing the price breaks would probably quash the ability to work these bundling deals, which are at the heart of many discounts. With all the prices made public, every plan will get the same deal regardless of what they are willing to offer. You can bet the public price they are offered will be higher than the private deal they might have been able to cut.

The irony is, by all measures, this competition is working for consumers. Why would Washington want to abate it?

The average monthly premium that seniors pay is $24, far lower than the $37 originally estimated by government actuaries. While Democrats have hammered away at the idea that having seniors choose among competing drug plans is too "confusing," recent polls show satisfaction with the benefit at about 80 percent.

In 2007, insurers are marketing more than 50 different drug plans in every state except Alaska and Hawaii--up from an average of about 40 in 2006. Seventeen insurers are selling nationwide plans, up from nine last year. That compares with the one or two that critics of including private plans predicted would be available in many markets.

The Medicare program agrees that disclosing the price breaks would undermine the ability of health insurers and pharmacy benefit managers like Caremark Rx and Medco Health Solutions, who also administer drug plans, from obtaining discounts.

The program cites an FTC analysis concluding that whenever competitors know the actual prices charged by other firms, tacit collusion and thus higher prices may be more likely. Separately, the Congressional Budget Office said a proposal to disclose the price data would add $40 billion over 10 years to the cost of the Medicare drug benefit.

Right now, Medicare has held firm and is not disclosing the data. The Medicare law prevents public disclosure of the prices that Medicare drug plans pay drug makers for their medicines. Waxman’s letters to the private plans are a clever attempt at end-running this law. These plans are harder pressed to hold out, since the Waxman request carries the threat of a subpoena.

But they too would be wise to hold firm and let the courts decide just what Congress has a right to access. At least one company has sent a box of files with a nice note attached, but has not yet sent what the congressman asked for. Worst case, a judge rules the raw data must be turned over and empowers a monitor with explicit restrictions on how Congress uses the information.

The only prices that really matter, the ones that beneficiaries pay, are all publicly disclosed on Medicare’s Web site so that consumers can be smart shoppers. The broader lesson that bears reminding is that the government makes a bad business partner, especially when it comes to health care. Just ask the HMOs, who signed on a decade ago to treat Medicaid and Medicare beneficiaries only to see reimbursement rates slashed when the plans turned profitable. Now drug companies are seeing the consequence of their own Faustian bargain.

In Washington, the only health care businesses that continue to get honest funding by Congress are the ones that do not work. As soon as something turns a profit, Congress looks to take it away. The fear about Part D was always that the drug program would devolve into price controls, thus destroying incentives for research and development as European governments have done.

Ironically, the politicians working the hardest to hobble the successful Part D plans are those like Waxman who are also advocating a universal health care system. Given the political track record they are laying, what health care venture is going to ever trust them? If Waxman succeeds, Part D will be added to a long list of cautionary tales for health companies contemplating work with Washington.  Read More & Comment...

Time to Let Vets Go AWOL From the VA

03/12/2007 11:40 AM |

The Washington Times
www.washingtontimes.com
The VA 'model'
By Robert Goldberg
THE WASHINGTON TIMES
Published March 12, 2007



Recently, John Stossel of ABC had lunch with Sen. Hillary Clinton. Mr. Stossel writes that when he launched into one of his usual libertarian rants about free markets, Mrs. Clinton cited the VA as an example of government success. Indeed, under her husband's administration, the Veterans Health Administration came to provide the "best care anywhere," according to the Washington Monthly. It was clear in the Monthly piece and in Mrs. Clinton's other remarks about the glories of the VA health-care system that it was a model for expanding coverage to all Americans. Similarly, Democrats and the media could not contain their enthusiasm for the way the VA doled out drugs at bargain-basement prices as an alternative to the current Medicare drug benefit.
Democrats beat a strategic retreat from imposing restrictions on access to medicines that veterans deal with when it became clear that seniors valued freedom of choice. Mrs. Clinton might want to reconsider her recent depiction of the VA as a medical paradise she created in light of growing dissatisfaction and poor treatment of the mental-health needs of the members of the armed forces returning from Iraq. We know accepting responsibility is too much to ask.
The VA is having a hard time handling all the most common battle-related injuries: traumatic brain injury and post-traumatic-stress disorder. It is not a matter of lack of funding or staffing or compassion. It is a matter of trying to do what a single-payer government system does anywhere in the world: force as much of the treatment of specialized care of complex and chronic illnesses down to the primary care doctor in order to hold down costs.
The VA system has made much of the fact that some of its outpatient centers outperformed commercial managed-care organizations on certain process measures (screening for diabetes and diabetes-related illnesses, checking cholesterol levels) more than seven years ago. And no one should dispute that the VA has made strides to improve the ability to follow patients through the system, something that makes it easier to reduce errors and improve care.
But there are simple measures for judging the quality of care. If you need care, you get it when you need it. If you're sick, when you get treatment you get better. Finally, what is done is based on total well-being and for the long haul, not to meet a short-term budget goal. And the way to guarantee quality is simpler still. If you don't get care that meets those standards, you should be free to seek it out and get it somewhere else.
And when it comes to treating traumatic brain injury and post-traumatic-stress disorder, we have very good evidence about that earlier admission that newer medications and more aggressive treatments are essential to better outcomes. Both illnesses are also associated with other psychiatric disorders and substance-abuse problems. Meanwhile, on average, it takes six months for the VA to process a new claim for coverage. And once enrolled, it takes nearly a month to even get a primary care doctor, let alone to see a specialist for complex conditions. The VA is often in the forefront of research in such areas, but efforts to translate findings into clinical practice are stymied by the one-size-fits-all approach to doling out care.
And no amount of funding will change this approach fast enough to provide veterans the kind of care they deserve. More choice and the freedom to go outside the VA system rather than die waiting could make the difference. But don't hold your breath. Under the visionary leadership of Rep. Pete Stark, California Democrat, Congress is already seeking to reduce the amount of money spent on Medicare managed health plans to pay for other social programs. Since the so-called savings go to plans with the sickest seniors, siphoning that cash will not only hurt the elderly but would also deny veterans another choice of health care.
Congress is eager to give airplane passengers the right to a refund if they wait longer than three hours on the tarmac before takeoff. Why can't it give returning veterans the same right if they have to wait longer than what is medically prudent for care?
The failure of the VA system to meet the mental-health needs of returning soldiers is a direct result of the fact that they are not consumers but captives. If we are asking them to fight for our freedoms, the least we can do is give them the freedom to seek care when they return wounded from the battlefield. They truly deserve to receive the best care, anywhere they can get it.

Robert Goldberg is vice president of the Center for Medicine in the Public Interest.  Read More & Comment...

McClellan on Follow-on Biologics: It's the Science

03/12/2007 09:54 AM |

Mark McClellan's spoke to the generic trade group about follow on biologics. In a word, focus on working with the FDA to develop the science for FOBs instead of just ramming through the current legislation which could, because it is devoid of science, could set FOBs back a decade. In this regard, Hillary Clinton appears to have moved into the science-based camp and has called for more clinical testing and post marketing monitoring of FOBs. Good for her. It shows she is serious about the subject.  Read More & Comment...

The EPO Wars

03/12/2007 09:11 AM |

Here's what the FDA has said about the benefits and risks of various EPOs:

"FDA and the manufacturer of these products have agreed on revised product labeling that includes updated warnings, a new boxed warning, and modifications to the dosing instructions. The new boxed warning advises physicians to monitor red blood cell levels (hemoglobin) and to adjust the ESA dose to maintain the lowest hemoglobin level needed to avoid the need for blood transfusions. Physicians and patients should carefully weigh the risks of ESAs against transfusion risks.

Recently completed studies describe an increased risk of death, blood clots, strokes, and heart attacks in patients with chronic kidney failure when ESAs were given at higher than recommended doses. In other studies, more rapid tumor growth occurred in patients with head and neck cancer who received these higher doses.

In studies where ESAs were given at recommended doses, an increased risk of death was reported in patients with cancer who were not receiving chemotherapy and an increased risk of blood clots was observed in patients following orthopedic surgery."

Now here is how the BMJ -- what an appropriate acronym -- covers it:

"Concerns about the safety of a class of drugs used to treat anaemia have triggered a federal alert in the United States. The alert comes after recent studies reported a higher incidence of fatal cancers and cardiovascular events among patients who were treated with drugs known as erythropoiesis stimulating agents when the drugs were used to raise haemoglobin concentrations to more than 120 g/l.
The US Food and Drug Administration issued the alert on 16 February, urging doctors "not to [give doses] to exceed haemoglobin levels of 12 g/dl" when treating patients with anaemia resulting from chronic renal failure, cancer or cancer chemotherapy, or HIV or AIDS."

See how BMJ traverses from proceeding with caution to prescribing beyond a specific hemoglobin level? Notice how things have moved from an advisory to a triggering a federal alert, like some terrorist warning? And note the inconsistency in metrics. Can we stick with 120g/l or 12g/dl from one graf to the next please?

Oh and leave it BMJ to allege a plot of some sort: conflicted scientists stuffed with cash hiding results about how plying patients with EPO kills them...as if there were no independent drug safety monitoring board reviewing the study as it progresses and if there were no mechanistic reason to explore the use of EPO in other cases...

"Issues of financial conflicts of interest and unpublished data have led to considerable controversy around the drugs. The New England Journal of Medicine decided not to publish an opinion piece it commissioned from Robert Steinbrook, one of the journal's senior physician writers, who questioned whether guidelines issued by the National Kidney Foundation to achieve higher concentrations of haemoglobin may have been tainted by multimillion dollar contributions from the manufacturers of the drugs, says a report in the Wall Street Journal (26 Dec 2006, p B1)."

Ok. I'm back. Had to shower after reading THAT again.

Let me state that I have always thought that the nextgen approach to EPO dosing - or dosing of any kind -- should be of the personalized sort, finding which dose works best with what subpopulation of patients. This is the right approach for any number of reasons.

Having said that, I am so sick and tired of this allegation of bribery regarding guidelines. If anyone -- including -- the lazy author of the first WSJ article on this subject had taken the time to read the NKF involvement in guideline development with respect to dialysis (a distinction that BM-J does not make in it's J'accuse) and use of EPO type meds they would see it is as transparent as BMJ's hatred of the pharmaceutical industry.

http://www.kidney.org/PROFESSIONALS/kdoqi/nkfAdvisory.cfm

And moreover they would see that in the past Medicare reimbursement rates had kept EPO doses artificially low to the point that people were not getting sufficient hemocrit levels. Dosing is higher and so is survival. At least in anemia. We are finding that this is not always the case in other clinical situations. Absent biomarker driven or more aggressive use of observational studies, reliance on RCTs will cause us to stumble our way to better or more targeted care. That's the way science proceeds. It is not a function of malfeasance.  Read More & Comment...

Thank you for not regulating

03/12/2007 07:58 AM |

Is cigarette smoking deleterious to America's health. Absolutely. Should Americans who currently smoke quit? Absolutely. Should the FDA regulate tobacco products? Absolutely not.

Pending legislation (Kennedy-Cornyn and Waxman-Davis) aims to give the FDA broad authority over tobacco products, providing the agency with oversight over virtually every aspect of tobacco company operations. The knee-jerk reaction is "great!" But, when you ask ""Would such legislation result in fewer smokers and reduced death and debilitation from tobacco? the answer is very unclear -- with the weight of logic and evidence saying otherwise.

For example, proposed legislation would set a very high bar (both scientific and procedural) before the FDA could approve a claim of "modified risk." The impact here would be to reduce any tobacco company's ability (or, most probably, desire) to promote their brands that are lower in nicotine content or, indeed, to even develop such products.

Or consider this, adult smoking has been declining since 1997 -- due to a number of things including clean air laws, media campaigns, and youth access programs. Who did these things? Largely the states. If FDA became the nation's tobacco czar, it would become difficult if not impossible to convince state legislators to continue to allocate the funds required for robust state-level tobacco control programs.

And then, of course, there's the question of both FDA resources and expertise. Let's take the latter first. What is the current level of FDA expertise in tobacco regulation? Let me put it this way -- none. As far as resources are concerned, the FDA's tobacco program would be funded by user fees. And, considering the current PDUFA-IV negotiations, you have to ask yourself if this is really the way we want to be going.

So, when you consider all of these issues (and there are many others)the answer to "Will FDA regulation of tobacco help to reduce tobacco use in America?" is very much an open one.

So for now, thank you for not regulating.

FYI -- the Center for Medicine in the Public Interest (the sponsor of drugwonks.com) does not accept funding from the tobacco industry.  Read More & Comment...

EU Goes Apple-plectic Over ITunes IP

03/11/2007 10:52 AM |

In another sign that the Europe is shrinking by virtue of self-inflicted acts of self-loathing and irrelevancy...

BERLIN (Reuters) -
"European Union consumer chief Meglena Kuneva has hit out at Apple Inc.'s (Nasdaq:AAPL - news) bundling of its popular iPod music players and its iTunes online music store, according to German weekly magazine Focus.

"Do you think it's fine that a CD plays in all CD players but that an iTunes song only plays in an iPod? I don't. Something has to change," EU Consumer Protection Commissioner Kuneva was quoted as saying in a preview of an interview to be published on Monday."

http://news.yahoo.com/s/nm/20070311/tc_nm/eu_apple_dc

The last time I was on-line I found tons of music sites where you can download music that play on any number of MP3 players. So what's the problem?

What has to change is the EU's attack on anything that is both innovative and American.
Like new drugs, Ipods, Microsoft, etc. Too bad American innovators don't join forces HERE to stop the assault on innovation in Congress and in Europe. Self-loathing you see, is a global epidemic.  Read More & Comment...

Caution on the biologics knockoff front

03/09/2007 04:47 PM |

The copy biologics brigade got a dose of reality yesterday. Turns out all those drugs they would just deem interchangeable aren't and all the biologics they could mix up in Jo Ann Emerson's bath tub like fertilizer need a bit of testing. An excellent article by Diedtra Henderson of the Boston Globe on the recent FOB hearing in the Senate HELP committee shows that for some folks it money first and safety second...

http://www.boston.com/business/healthcare/articles/2007/03/09/biotechs_nod_as_senators_urge_caution_on_generics/  Read More & Comment...

Want an off-label combination of drugs...Not under Kenzi you don't

03/09/2007 04:27 PM |

Great piece in Technology Review about how bioinformatics wil lead to tailored combos of drugs to treat diseases according to who were are and what we need...

"A biotech company called CombinatoRx has found that at the right doses, thousands of counterintuitive drug pairs are synergistic. The Cambridge, MA, company has eight drug combinations in clinical trials and several more in preclinical development. In a few years, diabetics, instead of injecting insulin, might be prescribed a cholesterol drug and a pain medication to help control their blood sugar. People suffering from chronic pain might find relief through a combination of a steroid and an antidepressant, with fewer side effects than they experience with current therapies.

Alexis Borisy, founder and CEO of the company, says his researchers take a brute-force approach to finding fruitful drug combinations. In the lab, they test combinations of several thousand drugs at several different doses on cellular models of diseases including cancer and arthritis--regardless of what diseases the drugs are currently approved for, if any. Then they feed the data into software that looks for synergies."

The entire article and link thereof can be found here:

http://www.technologyreview.com/Biotech/18280/

Just one problem....for every combo you will need.....a Risk management plan under the enlightened regime of Kennedy Enzi. Otherwise you and your doctor will be subject to some sort of liabiity and likely be denied reimbursement by an equally enlightened insurance or health plan.

Meanwhile Big Pharma lobbyists work with the staffers in Enzi and Kennedy's office for minor changes in the bill. Don't they realize that in a matter of days the Waxmaniac is going to drop his mutated version of Kenzi, complete with a comparative effectiveness study requirement?

What's counterintuitive is that people are still failing to ask just how Kenzi will affect access to lifesaving medicines and cripple the doctor patient relationship...  Read More & Comment...

Kids R'nt Us ... Yet

03/09/2007 03:22 PM |

Here's the lead from a story that ran earlier this week in the Philadelphia Inquirer on a new study showing that nearly 80 percent of the children cared for at academic children's hospitals got at least one medicine outside the age parameters approved by the FDA:

"Most children treated at major pediatric hospitals are given medicines not approved by the Food and Drug Administration for use in patients so young. The study, in today's Archives of Pediatrics & Adolescent Medicine, found that the sickest children and those undergoing surgery were most likely to get a so-called off-label drug. But altogether, nearly 80 percent of the children cared for at academic children's hospitals got at least one medicine outside the age parameters approved by the FDA."

Why is this so? Well it's because of the big, bad, evil pharmaceutical industry don't ya know:

"The risk and benefits of many drugs in children are poorly studied, often because the drugmakers had little financial incentive to do so."

Well, not precisely. It's not the financial incentives so much as the difficulty relative to the return. I know, that sounds like verbal gymnastics. Try this -- the real issue is that FDA permitted pediatric clinical trial design makes it difficult if not impossible to precisely determine which medicines in what dosages work in specific circumstances in specific pediatric patient populations. In other words, if personalized medicine in adults is difficult, in children it is nearly impossible.

Yet another important reason for the FDA to embrace adaptive clinical trial design and to become a research hub for 21st century clinical trial design. And that, of course, is part of what makes the Critical Path program so, well, critical to the future of America's health.

Here's a link to the Inquirer article:

http://www.philly.com/mld/inquirer/16841956.htm

According to Samir S. Shah, the study's lead author and infectious-disease doctor at Children's Hospital of Philadelphia, "We don't know whether in the absence of off-label use there would have been fewer deaths or more deaths. I suspect that in many instances the drugs were beneficial and in a smaller number of cases the drugs were harmful."

That's good news. What would be even better news would be getting the right drug in the right dose to the right child at the right time based on sound science and under the FDA's imprimatur.

Tempus fugit.  Read More & Comment...

Ted Kennedy's Sensible Statement on Follow on Biologics

03/09/2007 12:51 PM |

Compare this to the banshee cry of predetermined interchangeability as advanced by Waxman and the self-serving Express Scripts savings estimates from follow ons

I welcome the members of our committee and our distinguished witnesses to today’s hearing on the important question of whether Congress should give FDA the authority to approve follow-on versions of biologic medicines.

We are in a remarkable period of discovery in the life sciences. Unprecedented advances are taking place, and patients have already begun to see the benefits of this new era through new wonder drugs that can make the difference between life and death for patients afflicted with serious illnesses.

Patients with leukemia who once faced a bleak future now have new hope, thanks to an extraordinary new medicine that can slow or even halt the progression of the disease.

Until recently, a diagnosis of Gaucher’s [“go-SHAYS”] Disease meant a shorter life, full of disability and pain for the people it afflicted. Now, a remarkable breakthrough has produced drugs to treat this grave illness and extend life and reduce disability.

Similarly, a drug to stimulate the production of new blood cells is helping patients counteract the severe anemia caused by chemotherapy or renal disease.

These miracle medicines, called biologics, are complex molecules whose healing power has been brought to patients by dynamic biotechnology companies. Such drugs were once a rarity in the medical arsenal, but each day seems to bring new hope from new breakthrough biologics.

With this extraordinary progress comes a challenge to public policy. Due to the cost of developing and manufacturing new biologics, their price is often steep. They can cost patients tens or even hundreds of thousands of dollars a year, putting an extraordinary strain on the budgets of those who must pay the bills –patients, insurers and companies, or government programs.

Congress has faced similar challenges before. In the early 1980's, the cost of prescription drugs was spiraling upward. In response, Congress enacted legislation that balanced the need to reduce costs for consumers through increased competition with the requirement to promote innovation. That legislation is known universally by the names of its sponsors, Senator Orrin Hatch and Representative Henry Waxman. Our committee is honored that Senator Hatch is helping guide our deliberations. Congress and the American people are indebted to his leadership on these important issues.

When the Hatch-Waxman law was enacted, Congress did not include biologics, because at the time such drugs were not providing the major innovations that advances in the biological sciences have brought over the past 20 years.

Now Congress must consider whether to authorize FDA to accept applications for follow-on versions of these path breaking medicines.

The stakes riding on the answer to this question are enormous, both for patients and for our economy, and the interest among our committee colleagues in this question is intense. One of our colleagues, Senator Clinton, has a proposal to allow FDA to approve follow-on biologics. I look forward to hearing her views on this question, and to receiving the testimony of the legislation’s co-sponsor, Senator Schumer.

Many have recommended that the committee’s legislation on drug safety and user fees should include a proposal to allow for follow-on biologics. Today’s hearing will help to provide the information the committee needs to make the right decision on that important question.

Our committee should be guided by three basic principles.

First, we must be led by science. Acceptable legislation on follow-on biologics must not pre-judge science, but should enable the FDA to make the best decisions based on the most complete science reasonably available.

Second, protecting patient safety is essential. Congress must make certain that any drug given to patients – whether a conventional drug, an innovative biologic, or a follow-on product – is safe and effective.

Third, innovation must be valued and promoted. Just as it is essential to help patients afford the medicines of today, so too it is vital to provide incentives for the innovations that will bring the medical miracles of tomorrow.

I look forward to the recommendations and insights of our distinguished witnesses to provide guidance to our committee as we undertake these important deliberations.  Read More & Comment...

Friends of Cancer Research Drug Safety Briefing

03/09/2007 12:47 PM |

Drug Safety Briefing: "Drug Safety: Defining Safe"

On March 21st, Friends will host an educational congressional briefing on Capitol Hill entitled “Drug Safety: Defining Safe.” Featuring introductory remarks from Representative Diana DeGette (D-CO), the briefing will include expert panelists Dr. Robert Young, President of Fox Chase Cancer Center; Dr. Mark McClellan, Former Administrator of CMS & Former Commissioner of FDA; Dr. Janet Woodcock, Deputy Commissioner and Chief Medical Officer of FDA; and patient advocate Dan Perry, Executive Director of The Alliance for Aging. Susan Dentzer of The NewsHour with Jim Lehrer on PBS will moderate. For more information, please contact Heather Chaney at hchaney@focr.org or (703) 302-1540  Read More & Comment...

Does Waxman Still Stand With Thailand's Thugs?

03/09/2007 12:02 PM |

Henry Waxman sent a warm letter of solidarity when Thailand's military junta seized the patents of HIV drugs recently. The number of pharmaceutical patents rose to 11 and the range of products that the Thai government's drug company -- which has never been able to make WHO quality medicines -- would copy has expanded to include cancer and heart disease in the wake of Waxman's love letter.

Nothing like appeasement to embolden a global bully. In the January board meeting of the WHO, Thailand 's representative, Dr. Suwit Wibulpolprasert, declared that if an influenza pandemic hit, he'd counsel Bangkok to hold Western tourists hostage until those countries gave Thailand the necessary vaccines.

And if they didn't?

I wonder what sort of letter Waxman is planning to write now.  Read More & Comment...

Randomized To Death

03/09/2007 10:18 AM |

Here's what Susan Horn, one of our board members, an one of the world's experts on the science of improving outcomes in healthcare has to say about the crushing limiting randomized clinical trials have had on the quality of care for veterans with traumatic brain injury. It has implications not only for what is happening through the VA hospital system but for those who would use RiskMap as a one size fits all conduit for determining who gets access to medicines:

" The 1998 NIH consensus statement (about treating traumatic brain injury) acknowledged that individually tailored treatments provided within the context of acute rehabilitation create difficulties for efficacy studies. “This personalized approach leads to great difficulty in the scientific evaluation of effectiveness, because there is significant heterogeneity among persons with TBI and their comprehensive treatment programs”. The current level of evidence limits our ability to make firm decisions about the best therapy interventions, intensities, durations, or staffing characteristics for inpatient TBI rehabilitation. Also, randomized clinical trials severely limit the number of interventions that can be tested at any one time. Randomized trials attempt to examine an intervention in isolation from other interventions in order to detect the unique contribution to recovery of one or at best a few variables."  Read More & Comment...

(VA) Care To Repeat That?

03/08/2007 09:50 PM |

"We started during the Clinton administration to transition the VA system to a paperless system....The VA is leading the way in reducing medical errors, improving patient safety, and delivering high quality care; now this is a lesson about what can be done when we have a plan. A plan that is evidence-based, a plan that uses what we know works, and a system that we can actually get to respond to that evidence-based planning."

Hillary Clinton

"If you take a look at how the VHA system works, it's much more flexible getting drugs that are not on the list than the private plans are. And so people get the worst of all worlds: They have a more rigid formulary under Medicare, and the prices are much higher."

Ron Pollack FUSA

"Who do you think receives higher-quality health care. Medicare patients who are free to pick their own doctors and specialists? Or aging veterans stuck in those presumably filthy VA hospitals with their antiquated equipment, uncaring administrators, and incompetent staff? An answer came in 2003, when the prestigious New England Journal of Medicine published a study that compared veterans health facilities on 11 measures of quality with fee-for-service Medicare. On all 11 measures, the quality of care in veterans facilities proved to be "significantly better."

Philip Longman, Washington Monthly, 2005 from an article The Best Care Anywhere that boasted: "Ten years ago, veterans hospitals were dangerous, dirty, and scandal-ridden. Today, they're producing the highest quality care in the country. Their turnaround points the way toward solving America's health-care crisis."  Read More & Comment...