REMS & Preemption. E&C Redemption?

06/19/2007 08:05 AM |

CongressNow reports that,

“House Democrats relented and removed two of the most controversial provisions from pending Food and Drug Administration legislation, withdrawing language overturning federal preemption and a requirement for mandatory post-market reviews, Congressional and other sources say.”

According to an aide to Rep. Michael Burgess (R-Texas), the preemption language and a provision for mandatory risk evaluation and mitigation strategies “have been dropped.”

More as more develops.  Read More & Comment...

Word of the Day: "Febrile"

06/18/2007 05:07 PM |

Lead graph from a post by Trevor Butterworth on The Huffington Post:

Among the many febrile statements that followed the publication of a study in The New England Journal of Medicine (NEJM) contending that a diabetes pill carried an unacceptable risk of heart attack, one comment stood out, not so much for the scale of prognostication (deaths from Avandia may "dwarf 9/11," said study author Dr. Steven Nissen on ABC), or the degree of vitriol over the way drugs are regulated in the U.S., but because it managed the remarkable feat of sounding both stupifyingly naive and disingenuous at the same time. "We are a scholarly journal, not a news outlet," NEJM executive editor Gregory Curfman told BioCentury, a publication covering the drugs industry. 'What happens in the media is beyond our control."

The full post can be found at

http://www.huffingtonpost.com/trevor-butterworth/medical-journal-malpracti_b_52677.html

"Febrile." Great Scrabble word.  Read More & Comment...

Toxic Train

06/18/2007 09:59 AM |

No, not Thomas the Tank Engine.

The toxic train referred to herein is the one leaving the station on comparative effectiveness.

Or as Jill Wechsler of Pharmaceutical Executive writes, "The comparative-research bandwagon is gathering steam in Washington health-policy circles."

Except that a bandwagon doesn't "gather steam" -- it gathers hangers-on.

But choose your metaphor, it's a free country.

Gathering steam? Perhaps. Generating a lot of hot air? Definitely.

Two sample paragraphs to whet your interest:

"The danger is, of course, that effectiveness studies could be used to limit coverage and treatment options to low-cost products. And additional research requirements for sponsors could be costly. Prospective studies cost hundreds of millions of dollars and are vastly different from relatively low-cost retrospective data reviews, points out health economist Bryan Luce of United BioSource at a March seminar on comparative-effectiveness research sponsored by the Center for Medicine in the Public Interest."

"Variations in patient response to treatments and the desire to link provider payments to quality measures speak to the need for more valid comparative healthcare information."

Here's a link to the article:

http://www.pharmexec.com/pharmexec/article/articleDetail.jsp?id=429153

Comparative Effectiveness. The wrong train leaving the station in the wrong direction.  Read More & Comment...

Pharma Should Not Get Sick Over Sicko

06/18/2007 09:48 AM |

Not much in "Sicko," Moore's film advocating "free" universal health care for America. Rather, he spends a few minutes claiming that drug companies -- because Billy Tauzin wound up becoming head of PhRMA -- got the government to spend $800 billion on a new drug entitlement. Yawn. (PS/ He doesn't mention that Part D works and that seniors love it.)

In fact, there is not much of anything new in the movie as a whole. If you are a Moore loyalist you will rave how great it is. But mainly Moore recycles tired old arguments for national health care, abeit with more charm, humor and poignancy than most leftists can ever muster even when high. And Moore's fact and anecdotes are mostly incorrect, outdated and easily rebutted -- something we will take pleasure in doing here.

Does this mean that health care shouldn't more affordable or available or that 9-11 workers shouldn't have been given comprehensive care immediately? Of course not. But there are tradeoffs too. Moore keeps referring to "free" universal health care. Health care is neither free nor universal in the UK, Canada or France. But on the whole our system provides better care for more conditions with less rationing and about the same out of pocket spending (yes, folks) than all others.  Read More & Comment...

No Soap

06/18/2007 08:32 AM |

In the movie “Marathon Man,” Lawrence Olivier’s Mengele-inspired dentist threateningly asks, “Is it safe?” That same question, as Congress debates PDUFA reauthorization and FDA reform, has the real threat of crippling medical progress and the freedom physicians have in deciding which treatments are best for their patients. These are frightening thoughts — and attention must be paid lest we find ourselves, micron-by-micron, abdicating the hope of 21st century medicine to politically expedient measures that serve only to further the political aspirations of sound-bite hungry politicians and the voracious appetites of trial lawyers.

In today’s edition of the Wall Street Journal, Scott Gottlieb addresses the fact that …

“A decade ago, we were mostly focused on uncovering the remote risk that certain drugs could, in rare cases, cause immediate, unexpected and potentially life-threatening problems such as liver damage or disturbed heart rhythms. Surfacing these problems was made easier by the fact that the side effects occurred in close proximity to administration of the medicine, so it could be deduced when the drug was at fault.

Now we're focused on unearthing cases where prolonged use of some drugs may prompt small elevations in the risk of otherwise naturally occurring and even common events, like heart attacks. This was the issue that caused the withdrawal of the painkiller Vioxx and the premature flap around the diabetes drug Avandia.

Our heightened vigilance will surely improve the information people have to make informed choices. But finding and proving these kinds of rare and latent problems will often take many years and entail large and rigorous clinical testing. Even then, deciding if such remote kinds of risk outweigh benefits requires an often subjective calculus that is best left for patients and doctors to decide individually.”

In short, rhetoric and politics, and now actual legislation in the form of REMS, are forcing the FDA ever more towards adopting the Precautionary Principle. That’s the one-dimensional dogma that dictates that nothing should be done until everything is understood. Prudent? No, puerile. And the unintended consequences are fatal. Fatal like in no new medicines.

A little harmless politicking? Hardly. Just ask the people who may soon longer have access to the medicines they need, or to those who will suffer needlessly in the wake of Tropical Storm Safety — since the inevitable result of the Precautionary Principle is a dearth of new medicines in the pipeline.

Is it time to recall Ivory Soap? Is it safe? After all, it’s only 99 44/100% pure.

We must not allow safety to be hijacked.  Read More & Comment...

Nulla mensa sine impensa

06/15/2007 03:45 PM |

"There is no free lunch.” (A big “thank-you” to Grace-Marie Turner of the Galen Institute for the Latin translation.)

More to the point, there is no “cheap” lunch. In other words, there are rarely simple answers to complex questions.

For example -- John Edwards’ suggestion that the government take away patent rights for companies that develop breakthrough drugs and instead, reward them with “prizes” from the government.

Except that, er, it doesn’t work and has significant unintended consequences.

Details. Details.

The “prize” model has been used in the past – in the old Soviet Union. It didn’t work. The Soviet experience was characterized by low levels of monetary compensation and poor innovative performance. The US experience isn’t much better. The federal government paid Robert Goddard (“the father of American rocketry”) $1 million as compensation for his basic liquid rocket patents. A fair price? Not when you consider that during the remaining life of those patents, US expenditures on liquid-propelled rockets amounted to around $10 billion.

Certainly not what Schumpeter had in mind when he wrote about “spectacular prizes … thrown to a small minority of winners.” Creative destruction indeed!

Does Candidate Edwards really want to replace a patent system that has allowed the average American lifespan to increase, over the past 50 years, by almost a full decade with a prize program that has a solid record of complete failure.

As Joe DiMasi (Tufts University) and Henry Grabowski (Duke University) have argued, under a prize program, pharmaceutical innovators would lack the incentive to innovate. To quote DiMasi and Grabowski, “The dynamic benefits created by patents on pharmaceuticals can, and almost surely do, swamp in significance their short-run inefficiencies.”

As DiMasi and Grabowski presciently observed in 2004, “The main beneficiaries in the short-term would be private insurers and public sector purchaser of pharmaceuticals … Governments and insurers are focused myopically on managing health care costs. They are not likely to be strong advocates for funding new drug development that can increase individual quality of life and productivity."

Sound familiar? Correct. Europe. Sound familiar? Correct. Evidence-Based Medicine.

To be sure, there will be other unworkable, ill-considered, and precarious suggestions for ways to “fix” the U.S. health care system. But a prize system may be the worse of them all.   Read More & Comment...

Tort Lawyers Take on Health Care Reform

06/15/2007 11:11 AM |

John Edwards wants to end long term patents on breakthrough drugs for Alzheimer's and cancer and replace them with cash prizes for inventors. So reducing the incentives for investing in life saving medicines will help improve health care how exactly?

I have a better idea...let's reduce the cash prizes currently available for tort lawyers like Edwards that now go to pay for 16 acre spreads like his and use it to buy health insurance for hundreds of people.

http://www.nytimes.com/aponline/us/AP-Edwards-Michigan.html?_r=1&oref=slogin  Read More & Comment...

Policy is People. People are Policy

06/15/2007 09:37 AM |

We sent our roving reporter on the streets of New York City and asked following question:

"What do you think of a doctor who compares the adverse events of a diabetes drug to the events of 9-11?"

A non-scientific representative sample of these comments (on video) can be found at http://www.cmpi.org Just go to the section on podcasts and click on "Dr. Weber."

Yes, that Dr. Weber. Well-respected cardiologist, FDA maven, and Chairman of the Center for Medicine in the Public Interest. He comments (a bit more knowledgably) on the same question.

This podcast can also be found on YouTube and other social networking sites. We're trying to bring health care policy to the people.  Read More & Comment...

Another Bissl on Nissen

06/14/2007 05:08 PM |

From http://www.stat.org Trevor Butterworth, this time his trenchant comments from his blog on Huffington Post:

http://www.huffingtonpost.com/trevor-butterworth/the-avandia-nightmare-it_b_50596.html  Read More & Comment...

Where Were the Weight Watchers on the Accomplia Panel?

06/14/2007 01:41 PM |

Yale University's David Katz, whose website http://www.davidkatzmd.com is a valuable tool for anyone interested in nutrition or learning ponders the rejection of Accomplia in a recent post on ABCnews.com. He thinks the panel -- devoid of physicians who actually treat people with obesity problems -- went too far in rejecting the drug. He asks a question the FDA did not pose to the AdComm:

"That's not trivial, but how great is two times your current risk of depression if you're not depressed? And how much does uncontrolled obesity increase the risk of depression, anxiety and sleep disturbances in comparison?"

Oh.

Read the entire post here: http://www.abcnews.go.com/Health/Diet/Story?id=3277937&page=2  Read More & Comment...

The Critical Path Gap

06/14/2007 08:30 AM |

After all the non-stop claptrap about there being too many new drugs comes a sobering fact: even as pharmaceutical companies poured a record $43 billion into research and development in 2006, U.S. Food and Drug Administration statistics show that the agency approved only 18 new drugs last year. That's down from 36 in 2004 and nearly as low as it's been at any time during the past decade.

According to Dr. Scott Gottlieb, the agency's former deputy commissioner for medical and scientific affairs, "Our concern is that the development process itself is not keeping up at a fast enough pace to match the progress on the discovery end."

Which begs the question—wither the 21st century drug development tools that would make possible a swifter path from bench to bedside? More importantly, where's the outrage? Where are the scathing articles about the "critical path gap?" Where's the outcry from the halls of Congress about "who lost the critical path?"

Here's the rest of the story from The Journal of Life Sciences:

http://www.tjols.com/commentary/jun13_fda.jsp

Thoughts and comments most welcome.  Read More & Comment...

Misdiagnosis of conflict

06/14/2007 08:10 AM |

Daniel Carlat has a near hysterical piece about that equates pharmaceutical and biotech funding of CME to money laundering. Which I guess makes all the academics, researchers and clinicians who particpate...criminals? Carlat needs to take a dose of the trazadone he defends in one of his newletters that are available for a hefty fee. Which leads me to another point: nothing is stopping self-righteous types like Carlat from going into business themselves to offer CME programs. Oh, and if I were Carlat, I would drop the characterization "Unbiased" from the description of his expensive newsletter. He is as biased as anyone. Anyone who calls his colleagues money launderers is biased.  Read More & Comment...

Fat Chance

06/13/2007 11:38 PM |

I was incredibly wrong about the Adv Comm vote on Accomplia. The FDA's questions to the panel clearly indicated skittishness about approval and the committee simply went with the flow. Sanofi could have presented better -- more stratified-- data to identify who was more likley to have adverse events with Accomplia. Companies are simply going to have be more proactive in this regard.  Read More & Comment...

Is the Web Patient Friendly

06/13/2007 03:35 PM |

Laura Landro has an article on how the web empowers patients. I am not so sure. Unless companies, academics, and other responsible third parties take great pains to roll out solid material, most of the space is taken up by misfits,manipulators and opportunistic feeders as Jay Byrne of V-influence describes them who engage in client shopping, fearmongering and peddling of alternative health solutions. The worst offenders: Consumers Union with their blog campaigns that scare people into noncompliance and hidden campaign to fatten the pocketbooks of trial attorneys, Joseph Mercola and his numerous snake oil solutions and well...the tort sharks themselves who hire firms to set up fake "consumer health site" that offer crappy health info but really collect data that they use to fill the FDA with questionable adverse drug events.

I linked to Jacob Goldstein's blog...one of the more reasonable sources

http://blogs.wsj.com/health/2007/06/13/in-a-switch-online-patient-groups-inform-researchers/

So if we shut down the right of drug companies to advertise then we will be left with all this responsible behavior AND Michael Moore.  Read More & Comment...

A NICE Example of Comparative Effectiveness CMS Style

06/13/2007 03:18 PM |

AEI's Joe Antos in The American on how quickly and conveniently CMS is using post market evidence on cancer drugs to suit it's needs and screw patients: "The CMS action is breathtaking for its speed and its incursion into the practice of medicine. As a long-time observer of CMS, and as a former official in the agency during the 1980s, I have never seen a proposal with this level of detail emerge from the thicket of clearances in such a short period of time. Perhaps CMS has found some new efficiency that permits it to release rules in record time?"

Want an idea of how patients will fare under this farce called a federally funded R and D center for comparative effectiveness, read Balancing the Budget on The Backs of Cancer Patients.

http://american.com/archive/2007/june-0607/balancing-the-budget-on-the-backs-of-cancer-patients  Read More & Comment...

Matt Release

06/13/2007 10:13 AM |

Note: This is Peter's blog...I am posting for him because his computer is experiencing technical difficulties that were not detected by meta analysis.

FDA reform, real reform, requires hard work, partnership, and candor -- not politics-as-usual.

As Matt Herper over at Forbes writes:

"One change that seems inevitable is the creation of more Steven Nissens--that is, more outside scientists looking over the FDA's shoulder. A Senate-passed bill from Senators Edward Kennedy (D--Mass.) and Michael Enzi (R--Wyo.) seeks to force manufacturers to make all study results public. Nissen was able to do his Avandia analysis because Glaxo, in order to get then New York attorney general Eliot Spitzer off its back, agreed three years ago to put drug-trial results up for public viewing. The bill would put all drug firms in this boat.

Think of this as the open-source FDA. Already outsiders have pinpointed valid safety issues, such as Vioxx. But open source can be an invitation to a lynching. AstraZeneca's blockbuster cholesterol pill Crestor was originally maligned over kidney worries but has since been cleared. Expect "a free-for-all" as academic researchers get more access to data from companies and from electronic health records, says FDA Deputy Commissioner Janet Woodcock. “You could imagine tort lawyers running those analyses," she says."

Matt’s article (“Lynch ‘Em”) can be found at www.forbes.com

Herper continues:

"Still, big pharma is backing the Kennedy-Enzi plan, which would also give the FDA more power to change drug labels and force companies to conduct studies. New Pfizer Chief Jeffrey Kindler praised the bill in a speech at the Economic Club of Washington, saying drug firms should absolutely support a bill that requires candor about clinical trials."

And candor is something this debate needs. And honesty and a focus on advancing the public health rather than settling private scores (both within the FDA and elsewhere).

Nobody said it was going to be easy.  Read More & Comment...

Paging Dr. Moore ... Dr. Michael Moore

06/13/2007 07:18 AM |

Welcome to the Age of YouTube.

We will be doing regular podcasts, where we will ask experts as well as average folks what they think about some of the most pressing health care issues we face as a nation.

Oh yes, and some that aren't so pressing -- like whether or not Michael Moore should be trusted to direct American health care.

To view our initial effort, click on the link below and follow it to the "Podcast" box:

http://www.cmpi.org

Enjoy. (We did.)  Read More & Comment...

Accomplia

06/12/2007 01:23 PM |

Before you read any media reports, read Derek's balanced analysis of both the FDA and Sanofi's documents on the risks and benefits on Accomplia. As usual, he tees up the issue with insight and objectivity:

The incidence of suicidality – specifically suicidal ideation – was higher for 20 mg
rimonabant compared to placebo. Similarly, the incidence of psychiatric adverse events,
neurological adverse events and seizures were consistently higher for 20 mg rimonabant compared to placebo. . ."

They're also concerned about other neurological side effects, and seizures as well. The seizure data don't look nearly as worrisome, except in the obese diabetic patients, for whom everything seems to be amplified. And all of this happens at the 20-mg dose, not at the 5 (which doesn't do much for weight, either, as noted above). And for those who are wondering, yes, on my first pass through the data, I find these statistics much more convincing than I did the ones on the Avandia (rosiglitazone) association with cardiac events.

I had my worries about rimonabant a long time ago, but not for any specific reason. It's just that I used to work on central nervous system drugs, and you have to be ready for anything. Any new CNS mechanism, I figured, might well set off some things that no one was expecting, given how little we understand about that area."

Another important element that I think favors approval: Sanofi-Aventis is proposing labeling and prescribing limits to take into account the increased risk of a variety of pyschological side effects ranging from anxiety to suicidal thoughts (primarily among schizophrenics and others with a history of mental illness). Importantly, people in Accomplia trials had to go off antidepressants while on the drug so the label and risk management plan will limit use of the drug to people who are either on antidepressants or who are depressed. In Europe, compliance with such limits is around 95 percent.

Watch the fearmongers rise up and scream that no one should be allowed to have access to a drug that demonstrates the ability to reduce weight and glucose levels safely in a specific population. If Accomplia is NOT approved with the RISKMAP in place, with a pledge for post market studies and surveillance, no DTC ads for at least half a year, then what good is PDUFA?

Accomplia is a litmus test for the backbone of the agency and the political strength of the fearmongers.  Read More & Comment...

"Mo" Nonsense

06/12/2007 01:09 PM |

Previously we have discussed the half-baked and benighted ideas of Representative Maurice Hinchey -- author (along with Representative Bart Stupak) of a bill that would prohibit the agency from collecting fees from the companies it regulates. Instead, the money would be deposited into the general fund of the U.S. Treasury (see blog entry "Slo Mo," posted on 5/21/07).

Well, he's at it again. Here's what he had to say yesterday ...

"The FDA has essentially become the government affairs office of the pharmaceutical industry," Hinchey said in a statement, which called the relationship between the agency and industry "far too cozy and inappropriate."

This is anticipation of today's sitting of the House Energy and Commerce Subcommittee on Health.

A "Mo" untrue, unjust, and unfair statement is hardly imaginable.

The FDA is staffed with public health professionals on personal public health missions. They care, deeply, about safety and efficacy and about doing the right thing.

Mr. Hinchey, it seems, cares about headlines and hysteria.

An apology is in order.  Read More & Comment...

Rita Rubin Wrong Again

06/12/2007 12:49 PM |

I am trying to be nice... A kinder, gentler post that doesn't take media bias and laziness personally, that doesn't degenerate into childish namecalling...

Oh, screw it.

Rita Rubin's latest assault on the current PDUFA bill gets it exactly wrong.

"The FDA has essentially become the government affairs office of the pharmaceutical industry," Hinchey said in a statement, which called the relationship between the agency and industry "far too cozy and inappropriate." Hinchey is the author, and Bart Stupak, D-Mich., the chief co-sponsor of an FDA reform bill that would prohibit the agency from collecting fees from the companies it regulates. Instead, the money would be deposited into the general fund of the U.S. Treasury.

According to the Appropriations Committee, two officials of the Biotechnology Industry Organization and two officials of the Pharmaceutical Research and Manufacturers Association attended at least half of the 112 meetings."

Hey Rita, what's the punchline? So what? Did you ask how many times Hinchey and Stupak met with the lobbyists of organizations that are looking for earmarks from the Appropriations committee? Why don't Hinchey and Stupak post those numbers?

Now, unlike the done in the dark earmarking orgy, companies and the FDA are legally required to meet as part of the PDUFA reauthorization. But of course, the implication is that the companies run the show. Now if that were the case, if PDUFA were just a way to bend the drug approval process to the companies need, why have the percentage of drugs getting from Phase 1 to Phase 3 actually declined since PDUFA was implemented. Are companies deliberately conspiring with the FDA to take over the agency just to flush as much cash down the toilet as possible. Way to think through the logic, Rita!!!

Now extending that logic, should company representatives not meet with the FDA to discuss design of clinical trials, to explain endpoints, cooperate in sharing data to improve safety, design those safety trials the critics drool over while ignoring the need for new medicines?

I am one of those who actually agree that PDUFA has outlived its usefulness as a funding mechanism. It is too inflexible and keyed to one part of the drug development process. And it has not -- as I noted earlier -- done much to improve the efficiency of overall drug development (and that includes the efficiency of PM surveillance.) But don't believe - as the critics do -- that it has compromised safety. That is a silly and unscientific assertion.

I feel better now.

For the entire article go to: http://www.usatoday.com/news/health/2007-06-11-fda-drugmakers_N.htm  Read More & Comment...