Jeff's Back

03/15/2011 08:49 AM |

While many often disagree with Jeff Shuren, those of us who know him and have served with him know him as an honorable man and a dedicated public servant.

Using words like “perjury” to advance an agenda (even, in this case, one that I mostly support) is inappropriate.  Intelligent people can (indeed must!) regularly disagree on many things.  That's how progress is made. 

As background, some reportage courtesy of the talented Matt Herper at Forbes.

 
FDA Says Official Did Not Lie Under Oath

The Food and Drug Administration replied to accusations that a top official had perjured himself during Congressional hearings about direct-to-consumer genetic tests, saying that the FDA stood behind his statements.

Jeffrey Shuren, now the head of the FDA’s Center for Devices and Radiological Health, was asked at a Congressional hearing last July if direct-to-consumer genetics companies like 23andMe and DeCodeMe add to the medical literature by doing their own research. He said no.

“From the information we know,” Shuren said, “they are not doing their own research on the genetic profiles but they’re interpreting the studies that have been performed by others.”

FDA critics pointed out that Shuren had just days before those comments listened to a presentation by 23andMe founder and chief executive Anne Wojcicki during which she presented her company’s plans to do research in areas including Parkinson’s. On March 8, Michael Lee, whose FDABlog advocates less regulation for genetic tests, posted a video pairing Shuren’s comments with Wojcicki’s, and saying that Shuren was being “accused of misleading Congress.” That video is embedded below.

The charge was picked up by researcher-blogger Dan MacArthur at Wired and Duke professor and author Misha Angrist. John Derbyshire, writing for National Review, wrote that Shuren “went for Olympic Gold in bare-faced dishonesty.”Razib Khan at Discover viewed Shuren’s comments as a threat to people’s right to view their own DNA. “If they take our rights away because we’re silent, we have only ourselves to blame,” Khan wrote.

But the FDA says Shuren’s comments were accurate because the research 23andMe published was not medical, but instead related to physical traits like eye color and hair color. (23andMe’s paper is here.)

An FDA spokeswoman writes, “We stand behind the comments made by Dr. Shuren,” She adds: “At that time we were not aware of any of the more than a dozen DTC genetic tests making medical claims that were based on results of a company’s own research. Instead, the companies relied on published research in the literature.” (The full FDA statement is below.)

This statement is true. 23andMe has said it is working on using its data, collected from patient volunteers and customers, to try and understand genetic variants that are linked to disease. One area of interest is Parkinson’s disease; Wojcicki’s husband, Google co-founder Sergey Brin, watched his mother suffer from Parkinson’s and carries a gene that may raise his risk of developing the disease.

In context, Shuren’s quote was clearly about medical research, but he should have made the statement in the past tense. The fact of the matter is the anger about his statement is driven by a larger conflict: the collision between a community of genetics researchers and consumers who see access to one’s own DNA as a fundamental right and the medical community, which sees the tests as medical interventions with risks and benefits.

The main fear of those involved with DNA research is that the FDA will create regulations that prevent people from getting DNA test results without a prescription. There are many arguments that this should not happen. Research shows that people can handle the results from these tests, and it’s not clear that doctors are any more ready to understand them than consumers. Right now, these tests provide only limited information, but the technology for understanding human genetics is advancing at a breakneck pace.

My take remains that we’re set up for a huge clash between traditional medicine and genetics as these two fields, which don’t know how to talk to each other, wind up having to work together more and more often. This spat is merely a prelude.

One piece of advice from those angered by Shuren’s comments is very good: it is past time for those who believe it is important for consumers to be able to access genetic data make their views known to Congress and to the FDA.

The full FDA statement:

The FDA is responsible for reviewing clinical genetic tests. To date, direct-to-consumer genetic tests have not been reviewed by the FDA and as a result, we cannot verify that they provide safe and accurate results. The FDA just concluded an advisory panel with experts representing various aspects of the testing community, which provided us with a set of opinions on several scientific issues in DTC testing. We will review these and other public comments in the weeks ahead to determine what we think is the best path forward for addressing the complex issues related to direct-to-consumer testing products.

We stand behind comments made by Dr. Shuren in a July 2010 Congressional hearing on a GAO investigation into DTC genetic tests made by several companies. At that time we were not aware of any of the more than a dozen DTC genetic tests making medical claims that were based on results of a company’s own research. Instead, the companies relied on published research in the literature.

At a separate 2-day public meeting on laboratory developed tests held by the FDA in 2010, a panel of experts and agency officials, including Dr. Shuren, listened to a brief presentation from 23andMe CEO Ann Wojcicki about her company’s early research efforts. However, most of the described activities, including the one paper they had published, pertained to non-medical claims, such as eye or hair color.

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Quote of the day

03/15/2011 01:57 AM |

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CMPI Interview with Congressman Bill Cassidy, M.D. (R-LA)

03/15/2011 01:50 AM |

CMPI recently interviewed Congressman and physician Bill Cassidy (R-LA). In our interview with the Congressman we discuss the long-term impact of the health care law on the practice of medicine, the expansion of the Medicaid program, his vote in favor of repeal, and much more.

To watch our interview with Congressman Cassidy, click here:


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Is Innovation Lost?

03/14/2011 12:07 AM |

blog.nj.com/njv_guest_blog/2011/03/losst_innovation_may_mean_lost.html


For those with life-threatening diseases or painful chronic conditions, time is not on their side. When promising treatments languish waiting for approval in a bog of bureaucracy, the cost must be reckoned in lost lives and diminished quality of life.

That’s why it’s so alarming that only 21 new drugs gained FDA approval last year. This was a significant decrease from the previous two years— there were 25 approvals in 2009 and 24 in 2008.

Not only are approvals down, so are applications for approval. This portends even further declines in annual approvals down the road. With science more cutting edge today and grants for new research — in areas from pediatrics to Alzheimer’s — at all-time highs, how is it possible that approvals and applications are both dropping?

The FDA’s review process, in the view of many in the medical and biopharmaceutical communities, has become increasingly turgid. The FDA now frequently calls for extra clinical trials, requiring detailed safety plans that necessitate additional doctor and patient education, and an extended review period.

Of the 21 drugs approved in 2010, there were 21 drug makers to take credit. Not a single company earned more than one approval. From Pfizer to Bristol-Myers Squibb to Eli Lilly to Merck — all of which were shut-out for approvals in 2010 — the FDA was an equal opportunity rejecter.

And the problem is not limited to medicines. The development of tools that tailor treatments to our individual needs are drowning in a sea of endless confusion. Tests and medical efforts that can help detect and prevent disease, and eliminate useless or even harmful care are being held up in the name of patient safety.

Government policy writ large has now begun to stifle innovation in pharmaceuticals. Obama’s health care plan levies tens of billions in taxes on new medical products through 2019. Comparative effectiveness studies, required even after FDA approval as a condition for being added to benefits, will delay progress, too.

Even worse is that FDA regulators are beginning to consider the comparative effectiveness of products and as a result are raising the bar for approval.

Sound far-fetched? It isn’t. Consider the case of $8,000-per-month Avastin, an anti-cancer wonder drug that blocks blood flow to tumors. In 2007, the FDA granted accelerated approval for the use of Avastin for treatment of metastatic breast cancer. It was clear from the Avastin studies then that while many women would not benefit from the drug, a significant minority could live longer and with less pain. The FDA asked Avastin researchers to evaluate the drug’s risks and benefits on a larger group of patients with the same standards used to approve the drug in the first place. The study confirmed the 2007 results showing benefit to specific groups of women. But the FDA revoked Avastin’s approval for breast cancer treatment because it didn’t extend life on average.

Innovation is the result, not of a top-down decision, but by learning from actually using an invention. Taking products off the market therefore undermines medical progress in many cases.

As a result of the FDA’s slow-to-act review process, the Obama health care plan’s disproportionate taxation of pharmaceutical firms and this recent Avastin decision, innovation is in a very precarious position. Most medical innovations come from start-ups with limited capital. For all the happy talk about supporting innovation and small businesses, the trifecta of government tactics is doing just the opposite. Just this month, Kenneth Kaitin, director of the Tufts Center for the Study of Drug Development, registered his concern: “The question remains whether developers can bring enough new drugs to market at the pace needed to remain financially viable.”

Meanwhile, China, India and Singapore are inviting America’s innovators to set up shop overseas. As the world’s leader — by far — in scientific research investment, the United States must change course and must do so immediately. Not only are we losing innovation, we are losing lives as well.

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Updates from "Over There"

03/11/2011 08:01 AM |

IMI Shelter

Europe's Innovative Medicines Initiative (IMI) launched eight new projects with a total budget of EUR 172 million ($240.7 million) under a five-year, EUR 2 billion program to relieve bottlenecks in drug discovery and development. The projects are focused on cancer, rheumatoid arthritis (RA) and infectious disease.

In 2009, IMI awarded EUR 246 million to 15 projects involved in improving safety prediction and pharmacovigilance; improving prediction of efficacy in diabetes and brain and respiratory disorders; and education and training in support of medicines development.

 A third call for project proposals is under way and will focus on developing treatments for autism, tuberculosis and diabetes, as well as drug and vaccine safety. IMI is a joint undertaking by the EC and the European Federation of Pharmaceutical Industries and Associations (EFPIA) that is similar to FDA's Critical Path initiative.  

NICE News

While we do not always agree with him, we are pleased to share the news that our friend and colleague Sir Michael Rawlins has been reappointed Chairman of NICE for an additional one-year term. Rawlins, whose new term runs through March 31, 2012, has served as chairman since the agency was established in 1999.  

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The "Unsales Force"

03/10/2011 08:13 AM |

Via forbes.com:

 

What Standards Will Apply To Federal Drug ‘Unsales’ Force?

 

 

Posted by Cory L. Andrews

 

Proponents of health care “reform” have invested an enormous amount of time and effort proclaiming how pharmaceutical company sales representatives (colloquially known as “detailers”) and their visits to medical professionals increase the cost of patient treatments and, in turn, health care in general. 

 

For this reason, state and federal regulators have piled on laws and rules dictating what these detailers can and cannot say and do to educate physicians about their products.  Some states, most prominently Pennsylvania, have programs where purportedly independent clinicians, pharmacists, and others pay visits to doctors to essentially counter what they hear from drug detailers.

 

This counter-detailing, or academic detailing, is now poised to move forward at the federal level as a way to circulate government-manufactured “comparative effectiveness” research funded by the 2010 Recovery Act and advanced in ObamaCare.  The aim of comparative effectiveness is to reduce health care costs, an especially compelling goal for our government, the number one purchaser of medical treatments in America. 

 

This fact begs the question: can academic detailers, paid by federal taxpayers and distributing federally-funded research be considered unbiased or neutral?  And, if that is the case, what safeguards exist to ensure that this federal “unsales” force isn’t sacrificing the highest quality patient care in favor of saving money?

 

In fact, at this point, no such safeguards exist.  Peter Pitts, a former FDA Associate Commissioner who is currently President of the Center for Medicine in the Public Interest, explores this question and puts forward many more important queries in a Washington Examiner op-ed, “What a Difference Two Words Can Make in Health Care.”  Those questions include:

 

* “Will these studies be peer-reviewed before release?”

* “Who will decide what these detailers can say or not say? Will these government “reps” have to play by the same   rules as their pharmaceutical counterparts?”

* “If academic detailers stray into off-label conversations, to whom does Food and Drug Administration send a letter?”

* “Who will determine the difference between “communicating” these findings and “promoting” them?”

 

These are all issues worthy of consideration by those who oversee federal implementation of the health care reform law.

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CMPI Interview with Congressman Paul Broun, M.D.

03/10/2011 02:41 AM |

CMPI recently interviewed Georgia Congressman and physician Paul Broun.

In our interview with the Congressman, we focus on the health care law's impact on the practice of medicine in the long-term and plans by the House of Representatives this year to delay implementation of the law.

Watch the interview here:



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Crossing a chasm in two leaps

03/09/2011 08:57 AM |

As Disraeli said, “The most dangerous way to cross a chasm is in two leaps.”

Lengthy article in today’s edition of the Wall Street Journal (In Health Law, Rx for Trouble) focusing on the many (and immediate) unintended consequences of ObamaCare.

The article’s focus is on the law’s treatment of OTC medicines vis-à-vis flexible spending accounts.

It’s a very lengthy article.  What follows are some pieces thereof that relate to the negative impact on physicians.

Sandy Chung is grappling with a new kind of request at her pediatrics office in Fairfax, Va.: prescriptions for aspirin and diaper-rash cream.

Patients are demanding doctors' orders for over-the-counter products because of a provision in the health-care overhaul that slipped past nearly everyone's radar. It says people who want a tax break to buy such items with what's known as flexible-spending accounts need to get a prescription first.

The result is that Americans are visiting their doctors before making a trip to the drugstore, hoping their physician will help them out by writing the prescription. The new requirements create not only an added burden for doctors, but also new complications for retailers and pharmacies.

"It drives up the cost of health care as opposed to reducing it," says Dr. Chung, who rejected much of a 10-item request from a mother of four that included pain relievers and children's cold medicine.

Though the new rules on over-the-counter drugs amount to a small part of the massive overhaul of the health-care system, the unintended side effects show how difficult it can be to predict how such game-changing legislation will play out in the real world.

Some doctors, irked by the paperwork and worried about lawsuits, are balking at writing the new prescriptions. Pharmacists and retailers say the changes mean they have to apply a personalized label on some 15,000 different everyday products for customers paying with certain debit cards.

Health-policy experts predicted that new insurance pools for high-risk patients would attract so many expensive enrollees that funding would be quickly exhausted. In fact, enrollment is running at just 6% of expectations, partly because of high premiums.

A provision preventing insurers from denying coverage to children with pre-existing health conditions prompted insurers in dozens of states to stop selling child-only policies altogether.

Only after the president's signature was dry did the American Medical Association realize what had happened and send a letter to the government warning of unintended consequences, including more office visits and extra paperwork.

Sure enough, when the change took effect Jan. 1, patients began bringing lists of over-the-counter drugs to office visits and also requesting over-the-counter prescriptions by phone, doctors says.

Among those most upset by the changes are pediatricians, who say that small sizes of children's medicines and multiple children per family make the requests particularly burdensome.

"It's an amazingly disruptive policy," says Jesse Hackell, a Pomona, N.Y., pediatrician who is charging $5 to fill such requests via phone. "I am now doing the IRS's work, and that's what I resent most."

After writing two over-the-counter prescriptions free of charge in January, pediatrician Richard Schwartz of Vienna, Va., says he began imposing a $10 surcharge for each prescription, on top of the office co-payment. That is likely to discourage some patients from asking for a prescription, as the surcharge could outweigh the tax savings from using a flexible-spending account.

Doctors are also concerned about malpractice lawsuits, since a prescription potentially puts them on the hook for any problems a patient suffers from over-the-counter drugs.

In the Nielsen survey, 37% of flexible-spending account users said they would ask their doctor about prescription drugs that could replace their over-the-counter medicines.

This is what happens when you look before you leap (translation – when you vote for a bill you have not read).

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CMPI Interview with Senator Dan Coats

03/09/2011 12:37 AM |

CMPI recently interviewed Senator Dan Coats (R-IN) to discuss his vote in favor of repealing the health care law and the health care law's negative impact on Indiana's pharmaceutical and medical device sectors.

Watch the interview here:



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Is Mitch Daniels A Socialist?

03/08/2011 08:50 PM |

"Conservatism is the antithesis of the kind of ideological fanaticism that has brought so much horror and destruction to the world. The common sense and common decency of ordinary men and women, working out their own lives in their own way—this is the heart of American conservatism today. Conservative wisdom and principles are derived from willingness to learn, not just from what is going on now, but from what has happened before." - Ronald Reagan, CPAC, 1977

In recent weeks many pundits and think tank types have raised questions about whether Governor Mitch Daniels is a 'real' conservative. Or more transparently put: there are many conservatives who don't agree with Daniels on specific issues and therefore categorize him as not conservative enough.

I won't go into pre-positioning around his so-call called for a truce on social issues.  (He didn't say surrender or truce but a timeout  and he suggested that the most immediate threat to our nation's future is the rocketing debt.)  

And I will only note in passing that playing to most ideological elements of a party is par for the course of running for president.

My concern is the allegation that Daniels health care programs in Indiana is either creeping socialism or warmed over Medicaid.  This argument is advanced by Michael Cannon’s article: “ Mitch Daniels’s Obamacare Problem: His state’s health-care plan promotes dependence on government.”  www.nationalreview.com/articles/261285/mitch-daniels-s-obamacare-problem-michael-f-cannon

Cannon’s major claim is that under Daniels the number of people on Medicaid have increased and the creation of a program to provide health savings accounts for Hoosiers that cant afford insurance (and have not had it for 60 days) and do not qualify for Medicaid.  The Healthy Indiana Plan is funded by a combination of Medicaid dollars and an increase in the state tobacco tax. 

Cannon’s complaint is “Like Obama, Daniels increased cigarette taxes to expand government-run health care. Whereas Obamacare requires states to open their Medicaid programs to families of four earning $31,000 (138 percent of the federal poverty level), Daniels expanded Indiana’s Medicaid program to families of four earning $44,000 (200 percent of poverty). From 2008 to 2010, Indiana’s Medicaid enrollment spiked: Adult enrollments grew 21 percent, a rate nearly double the national average. By 2010, Daniels had enrolled another 62,000 Hoosiers in government-run health care. “

First, Cannon should get his facts straight.  Indiana’s adult Medicaid enrollment increased 17 percent from 2008-2010, slightly higher than the national average of 14. 6 percent.  And between 2009-2010 the rate of increase in Indiana's adult Medicaid enrollment declined (as it did in only 16 other states) and did so more dramatically than most.   Second, Cannon cites Indiana’s income eligibility levels for families when he should have cited the income levels for adults.  But that would undermine his argument since Indiana caps eligibility at 50 percent of poverty. 

Cannon believes Daniels ultimate sin is that he “made Medicaid more attractive: Under his plan, the government hands out coverage plus something a lot like cash.”

Do Cannon and other Daniels denialists regard the governor’s proposal to convert public education funding into a statewide school voucher program an example of making “public education” more attractive by handing out coverage and cash.  Cannon dismisses the shift in personal responsibility, the creation of competition, the cost savings and the qualitative differences that vouchers and health savings accounts bring.  

Daniels can push such a change because he has control over state funding of schools. As of now, Daniels and other governors that would want to turn Medicaid into a voucher program must wait years and face a hostile Obama administration. 

I know Cannon works for the Cato Institute and is therefore more likely to object to any expansion in government.  But governing is not ideology.  And policy disagreements should be based on facts.

And facts matter. And the facts show that Indiana's Medicaid population did not double compared to the national average. Healthy Indiana has enrolled the most chronically ill and chronically un-insured Hoosiers since 2006.  Over that time costs have stabilized and not exploded. Those who have found jobs don’t forfeit the coverage they have. They can keep it, along with the health savings accounts they have created. Which might be one reason that Indiana has more business startups than other states and its workforce and population has increased.  

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Media Matters Has Obamacare Derangement Disorder

03/07/2011 03:57 PM |

Media Matters and CMPI have an honest difference of opinion on Obamacare.  Media Matters believes that the majority of the country rejects the increase in government regulation, higher taxes and reduced choice in plans (including dumping up to 80 million Americans into Medicaid) because of Republican 'lies' that distort the true beauty of the law.  

CMPI believes in and supports patient-centered, portable and affordable health care coverage.  We don't accept the status quo.  We believe we can do better but that Obamacare is an obstacle to innovations in medicine, technology and funding that are essential to improving the healthcare system.  That doesn't mean some features of the law shouldn't be preserved.   It means that to preserve them will likely mean repeal takes the form of substantial reform. 

That's an honest difference of opinion that we are welcome to debate and discuss anytime. 

So when MM responds to our interview with Michele Bachmann (See the Media Matters response and the video at politicalcorrection.org/blog/201103040003 ) by first accusing us of being paid to be part of a "front" to kill healthcare reform it suggests that Media Matters is more interested in silencing CMPI rather than engaging in debate.   It would be easy to respond by pointing out that MM's funding is from George Soros, whose non-profit empire could be considered a "front" of sorts.

Conflict of interest is a canard that cuts both ways.  It scores points and generates heat but little light.  

It's fine for MM to take issue with Congresswoman Bachmann and CMPI's interest in her views.  However it seems that Media Matters is more interested in repeating talking points than in substantive discussion.  

I will debate or discuss Obamacare with anyone from Media Matters or ThinkProgress any time or any place.  But I don't think they will take up the challenge.  They seem too obsessed with reassuring themselves that the rejection of Obamacare is the result of a well-financed campaign to lie about how wonderful the new healthcare law is.

If and when Media Matters and ThinkProgress snaps out of that disassociative state, I will be happy to engage them.


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Detail-Safe

03/07/2011 11:01 AM |

The proposition of the FDA’s “Safe Use” initiative is that the way to make a drug “safer” is to better educate prescriber, dispenser, and user about the product.

At a meeting of the FDA’s Risk Communications Advisory Committee (February 26-27, 2009), there was open public comment on the issue of how to improve the written information currently provided to patients about the medicines they receive (aka “consumer medical information” or “CMI).

(PS/ per full disclosure, while I did not participate in this meeting I am a Special Government Employee consultant to this advisory committee.)

The meetings complete comments can be found here.

I found the remarks of Pam Budny, a regulatory affairs manager at Eli Lilly of particular interest.

Some extracts from her comments:

Patients should be able to locate, interpret, and act upon information in written patient labeling. It should facilitate or reinforce the communication between the patient and the prescribing and/or dispensing healthcare professional. 

Patient labeling should be prepared by the sponsor just as is the case for physician labeling. Physician and patient labeling are inextricably linked in terms of the information they contain on benefits and risks. 

Sponsor-prepared patient labeling ensures consistency of information provided to patients in multiple venues. 

For example, patient labeling received at the time the medication is dispensed would have the same content as the “full disclosure” accompanying promotional materials for patients.

Testing with patients and or caregivers prior to submission is a critical way to determine the usefulness of patient labeling prior to patient use.

Patient labeling should be developed by sponsors, tested with patients, approved by FDA, and delivered in ways that are compatible with pharmacy dispensing workflows. This information should be made available to patients each time they receive their medication. 

These comments reinforce the concept of a sponsor-developed, FDA approved, “label detailing guide.” Assuming that healthcare providers both read and understand the PI is, well – wrong.

Consider the report out of the University of Chicago (a national random sample mail survey of 599 primary care physicians and 600 psychiatrists from November 2007 to August 2008) indicates there is confusion among physicans about what is or is not “on-label.”

According to the abstract, the average respondent accurately identified the FDA-approval status of just over half of the drug-indication pairs queried (mean 55%; median 57%). Accuracy increased modestly (mean 60%, median 63%) when limited to drugs the respondent reported having prescribed during the previous 12 months. There was a strong association between physicians' belief that an indication was FDA-approved and greater evidence supporting efficacy for that use.

The study’s senior author, Dr. G. Caleb Alexander (assistant professor of medicine at the University of Chicago) said a concern was that off-label uses often did not have the same level of scientific scrutiny as FDA-approved uses.

All the more reason for the FDA and the pharmaceutical industry to jointly develop (as part of the agency's Safe Use initiative) better ways to make the PI more user-friendly.

The time for sponsor-created and FDA-approved label “detailing aids” is now.

 “Sometimes the questions are complicated and the answers are simple.

Dr. Seuss  Read More & Comment...

Communicating Cost-Think

03/04/2011 08:46 AM |

“Newspeak,” as you Orwellian cognoscenti know, is the official language of Oceania – the land ruled by Big Brother.  Newspeak was designed “not to extend but to diminish the range of thought” (author’s original italics).  Its goal was to “make all other modes of thought impossible.”

Further, per Mr. Orwell, “A Newspeak word was simply a staccato sound expressing one clearly understood concept.”

Some well-known examples of Newspeak are Bellyfeel (a blind, enthusiastic acceptance of an idea), Duckspeak (meaning literally to quack like a duck or to speak without thinking), Crimethink (the Newspeak word for thought crime) and Goodthink (or “political orthodoxy”).

All of which brings us from the nightmare fantasy of 1984 Newspeak to the healthcare debate of 2011, the concept of comparative effectiveness research, and a new term we must all become familiar with -- “Cost-think” (which defines everything that reduces short-term costs as a benefit to the patient).

The Recovery Act of 2010 (aka – “the stimulus package”) gave the Agency for Healthcare Research and Quality (AHRQ) $1.1 billion to conduct (according to the HHS press release) “comparative effectiveness research” into various “healthcare interventions.”

Except that’s not what Congress funded.  Per the Recovery Act, that $1.1 billion was earmarked for clinical comparative effectiveness not comparative effectiveness research. And this is not splitting hairs. Enter cost-think.

Those in favor of comparative effectiveness research favor large scale trials to "compare" drugs and other healthcare “technologies, striving to show which medicines are most effective for any given disease state. Is there a “more effective” statin? A “more effective” treatment for depression?

 

But how do you compare two molecules (or three or more) that have different mechanisms of action for patients that respond differently to different medicine based on their personal genetic make-up?

 

Comparative effectiveness relies heavily on findings from randomized clinical trials. While these trials are essential to demonstrating the safety and efficacy of new medical products, the results are based on large population averages that rarely, if ever, will tell us which treatments are “best” for any given patient. 

 

Two such studies, the Clinical Antipsychotic Trials in Intervention Effectiveness (CATIE), study and the Antihypertensive and Lipid-Lowering Treatment to Prevent Heart Attack Trial (ALLHAT) study, were two such “practice-based” clinical trials, sponsored in part by the National Institutes of Health, to determine whether older (cheaper) medicines were as effective in achieving certain clinical outcomes as newer (more expensive) ones. 

 

The findings of both CATIE and ALLHAT were highly controversial, but one thing is not: even well-funded comparative effectiveness trials are swiftly superseded by trial designs based on better mechanistic understanding of disease pathways and pharmacogenomics. And, since most comparative effectiveness studies are underpowered, they don’t capture the genetic variations that explain differences in response to medicines by different patients. Comparative effectiveness in its current form leads to a “one-size-fits-all” approach to healthcare, which means that it doesn’t fit anyone all that well.

 

Clinical effectiveness, on the other hand, measures outcomes on an individual patient level.  Clinical effectiveness studies help us to understand how to design treatments based on patient variation rather than cost.  The very definition of personalized medicine.

 

All this to say that the differences between comparative and clinical effectiveness studies are profound and that by changing the actual legislative verbiage, the legislative intent is likewise altered.

 

As Mr. Orwell wrote, no word in the Newspeak vocabulary was “ideologically neutral” and a great many were “euphemisms.” Welcome to cost-think, where anything that has to do with healthcare reform cannot be spoken about in terms of cost but must be entirely based on the philosophy of reducing short term costs.

 

And nowhere is cost-speak more crucial than when it comes to publicly bankrolled dissemination of the findings of tax-payer-bank funded and AHRQ-fielded comparative effectiveness research.

 

The first question to ask is whether or not these studies will be peer-reviewed before they are allowed to be released? (CATIE and ALLHAT were not.) Another query is to whom will the studies be communicated – and how?  Will physicians be “academically detailed?” And if so, will they be required to be detailed? Will physicians be given incentives to spend time with AHRQ’s comparative effectiveness angels (i.e., CME credits) and punished if they do not (via Medicare and Medicaid restrictions)? And how will Uncle Sam decide which doctors are to be visited?  Will “high prescribers” of on-patent medicines be on a priority list, identified by mechanisms being developed to enforce multiple state physician “sunshine” laws?

 

Congressional oversight must be required for the $42.3 million that AHRQ has already awarded for public and physician outreach.

And relative to academic detailing (aka, “counter-detailing”), what safe guards are in place to certify that physicians are being presented information that is unbiased? Previous government detailing efforts have often focused on demonstrating their own value by highlighting the cost effectiveness of initiatives through savings generated from the increased utilization of generics and other low cost therapies.

Asked another way – how can an “academic detailing” program funded by our nation’s largest payer (Uncle Sam) be considered neutral? Just like detailing programs run by pharmaceutical companies, there is an inherent “interest.” And that’s okay – as long as that “interest” is transparent.  Who will be the arbiters of transparency?

Who will decide what these detailers can say or not say? Will these government “reps” have to play by the same rules as their pharmaceutical counterparts?  And, importantly, what is the oversight mechanism?  If academic detailers stray into off-label conversations, to whom does DDMAC send a letter?  Whom does the Department of Justice investigate? Who pays the fine?

Most importantly, who will determine the difference between “communicating” these findings and “promoting” them?  Alas, such finesse is unlikely under a regime of cost-think. As Orwell commented, Newspeak was constructed as to “give exact and often very subtle expression to every meaning that a Party member could properly wish to express, while excluding all other meanings and also the possibility or arriving at them by indirect methods.”

 

As currently organized, comparative effectiveness research will be used to increase government control over the practice of medicine and is a slippery slope towards the introduction of price controls.

Government sponsored comparative effectiveness research is the first step towards allowing Uncle Sam to push a restrictive formulary on more and more Americans – with step one in the process being unfettered (and unregulated) communications efforts. Unless we are aware and vigilant, such cost-think may very well lead to a single-payer system referred to in cost-think as “universal coverage” – but in reality will be nothing short of healthcare rationing.

As Rudyard Kipling said to London's Royal College of Surgeons in 1923, "Words are, of course, the most powerful drug used by mankind ... They enter into and colour the minutest cells of the brain."

 

We allow them to be usurped and corrupted at our own peril.

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Don with the wind?

03/04/2011 08:40 AM |

Via BioCentury ...

Republicans urge Obama to withdraw Berwick CMS nod

Sens. Orrin Hatch (R-Utah) and Mike Enzi (R-Wyo.) and 40 other Republican Senators sent a letter to President Obama urging him to withdraw his nomination of Donald Berwick as administrator of the Centers for Medicare & Medicaid Services (CMS). Berwick was re-nominated for the position in January. Obama appointed Berwick to the position last July while Congress was in recess, allowing Berwick to serve until Dec. 31, 2011, without a Senate confirmation vote.

The Senators said Berwick's "lack of experience in the areas of health plan operations and insurance regulation raise serious concerns about his qualification for this position." Berwick's nomination can be prevented if at least 40 senators vote against it during the confirmation process.

Berwick has started to assemble his own team at CMS. Patrick Conway is on his short list of possible replacements for Barry Straube, who stepped down as the chief medical officer of CMS in January. Conway is currently chief medical officer in the Office of the Assistant Secretary for Planning and Evaluation at HHS. He is the former director of HHS' Agency for Healthcare Research and Quality.

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Testing the Test

03/03/2011 09:58 AM |

The WARFARIN (Warfarin Adverse Event Reduction for Adults Receiving Genetic Testing at Therapy Initiation) study, designed to demonstrate the value of genetic testing for guiding warfarin dosing, has received the thumbs-up from CMS and is a major step forward for future reimbursement decisions.

(The randomized, blinded, parallel-group study was designed and funded by the privately held Iverson Genetic Diagnostics, Inc., which sells a test that can be used to predict response to the generic injectable anticoagulant warfarin and help guide dosing.)

The trial design was authorized by Centers for Medicare and Medicaid Services under its coverage with evidence development program. The agency decided in 2009 that Medicare would not cover the genetic testing for routine use, but it would for randomized controlled trials. In issuing the decision, CMS made clear that it wanted to see prospective results examining use of the tests.

The 18-month WARFARIN study will include 7,000 patients over the age of 65 and assess the practical value of testing for these genetic mutations, based on rates of severe hemorrhage and thromboembolic adverse events. Per request by CMS, the study will be monitored by an independent data coordinating facility.

Via molecular diagnostics specifically called out in the amended FDA label, the health benefits and the resulting savings in health care costs generated by using personalized warfarin dosing decisions are estimated to prevent 85,000 serious bleeding events and 17,000 strokes annually – and that’s just in the United States. And estimates predict the reduced health care spending from integrating genetic testing into warfarin therapy to be $1.1 billion annually.  And that’s the mid-range.

“Safety means doing the right things for patients.”

Janet Woodcock

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Critical Path Showing Signs Of Life

03/02/2011 10:31 PM |

Development of Novel Combination Therapies

Janet Woodcock, M.D., Joseph P. Griffin, J.D., and Rachel E. Behrman, M.D., M.P.H.

February 16, 2011 (10.1056/NEJMp1101548)

www.nejm.org/doi/full/10.1056/NEJMp1101548
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Lending a hand with regulatory science

03/02/2011 09:16 AM |

Well -- not exactly lending.  More like paying.  In fact, exactly like paying.

It seems like there’s momentum to fund the FDA’s Sentinel Initiative via PDUFA. Although, according a report in the Pink Sheet, “Industry is not interested in blindly pumping user fee revenue into the project.”

Per industry suggestions, only specific Sentinel projects would be funded by user fees and an open process would be required to make the determinations,

FDA and industry also talked about possibilities of establishing a process for determining which Sentinel projects were eligible for user fees, according to minutes of a January 10 meeting.

(Ain't transparency grand?)

The proposal would limit user-fee funded Sentinel projects to those that would “emphasize safety issues that affect classes of drugs or multiple products,” according to the minutes.

Also required would be an assessment of Sentinel’s value to regulatory decision-making on safety issues and adjustments to resource needs accordingly, with the evaluations targeted for fiscal years 2015 and 2017.

The minutes indicated both sides have agreed to the proposal and will present it to the full committee for consideration.

FDA also agreed to better describe how regulatory science upgrades would be relevant to the review model and PDUFA.

Saying publicly what has to-date only been whispered privately, both sides seem to be reaching agreement that funding for the agency’s initiative on advancing regulatory science (the Advancing Regulatory Science Initiative or ARSI) is a PDUFA-worthy proposition.

Does this foreshadow new and robust agency/industry collaboration?

Stand by.

"One must have a good memory to be able to keep the promises one makes."

-- Friedrich Nietzsche


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CMPI Interview with Congressman Joe Walsh (R-IL)

03/01/2011 05:08 PM |

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NMEs: A Love Story

02/28/2011 08:56 AM |

Statistics, as the saying goes, are like a Speedo. What they show you is interesting, but what they conceal is essential.

Consider the recent comments by Ezra Klein in the Washington Post.  Mr. Klein first asks us to all shed a tear for the health insurance industry, telling us that it’s profit margin is only 4.54%. That sounds like the same math used by Hollywood studios to explain why blockbuster movies that gross hundreds of millions actually lose money.

But Mr. Klein (in a blog post widely circulated by AHIP) is not only chief apologist for the private payer community, but is also its finger pointer-in chief.  Why are healthcare costs so expensive?  According to Mr. Klein the problem is caused by “new and incredibly expensive treatments.”

Per Mr. Klein the problem is … innovation ... of the pharmaceutical and medical technology varieties.  In other words what we need are fewer new molecular entities (NMEs) and not too many cutting edge medical technologies. Really?

As Albert Einstein said, “Only two things are infinite, the universe and human stupidity, and I’m not sure about the former.”

This strategy of projection and finger pointing misdirection is not only wrong – it will boomerang against those making the argument.  Every poll on the issue of healthcare reform makes it abundantly clear that patients and physicians are keenly aware and unhappy with those who support a short-term cost-based payer-friendly paradigm over a more holistic patient-centric health care solution. 

As Harvard University health economist (and Obama healthcare advisor) David Cutler has noted, "Virtually every study of medical innovation suggests that changes in the nature of medical care over time are clearly worth the cost." The repercussions of choosing short-term stock prices over long-term results are pernicious to both the public purse and the public health.
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CER experts know best

02/25/2011 01:59 PM |

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