Sunshine Supermen

03/20/2008 07:16 AM |

Check out H.R. 5605, the “Physicians Payments Sunshine Act of 2008. The sponsor is Peter DeFazio (D-OR) and one of the co-sponsors is Pete Stark (D-CA). Here's a link to the proposed legislation:

Download file

(Another of the co-sponsors is Representative Dennis Kucinich – glad he’s getting back to work after the grueling primary season.)

The bill calls for the reporting of absolutely everything (and they thought of everything) over $25.00 considered “a transfer of value” to a physician.

(What’s the AWP for a large pizza and a liter of Diet Coke these days?)

The penalties are between $10,000 - $100,000 and multiple offenders can say adios to their corporate deductions for advertising related expenses.

According to Mr. Stark, “The Sunshine Act will help enable Medicare beneficiaries to determine if their doctors are acting in patients’ best interests. It may even convince doctors to quit taking what can only be described as industry kickbacks."

Kickbacks? That’s a pretty strong accusation. Mr. Stark should apologize to America’s physicians and to the pharmaceutical industry he slanders so easily. And as far as America’s Medicare beneficiaries, Mr. Stark might also want to mention that the Medicare Drug Benefit (Part D) doesn’t "pay" doctors.

Mr. DeFazio issued some equally thoughtful and measured comments:

"If the billions of dollars drug companies spend taking doctors on trips to the Caribbean and to expensive dinners at the country’s finest restaurants are above-board, then the pharmaceutical industry should support our legislation. This bill will keep the pharmaceutical industry honest."

Speaking of “honesty,” Mr. DeFazio should own up to the fact that the aforementioned boondoggle trips and are against the rules.

But this has nothing to do with honesty and everything to do with posturing – and hidden agendas. (One of the groups supporting this legislation is Consumers Union – yes, the same organization that receives millions of dollars from foundations supported by the generic drug industry.)

The concept that big bad Pharma is to blame for everything, and that a $25.15 pizza party is at the heart of the destruction of medical ethics, isn’t just simplistic and sophistic but deleterious to a serious conversation about the issue.

H. R. 5605 is just another example of superbity from our legislative Sultans of Sanctimony.  Read More & Comment...

The Hazards of Harassing Doctors

03/19/2008 08:10 AM |

The Center for Medicine in the Public Interest will shortly publish a series of essays on the consequences of physican disempowerment in both the US and the EU. The title of this collection is "The Hazards of Harassing Doctors," and features contributions by Dr. Alphonse Crespo (a Swiss physician) and Dr. Marc Siegel (an American).

The thoughtful introduction is penned by Dr. Fred Goodwin, CMPI board member and Research Professor of Psychiatry at The George Washington University and Director of the University’s Center on Neuroscience, Medical Progress, and Society. Dr. Goodwin is the former Director of the National Institute of Mental Health

Here is a taste of Dr. Goodwin's take on the situation.

William Osler, generally regarded as the father of modern medicine wrote "If you listen carefully to the patient they will tell you the diagnosis." Arriving at a diagnosis and appropriate treatment plan has always represented collaboration between individual patients and their personal physician.

But today physicians are increasingly seeing the decisions that they and their individual patient reach about a specific treatment plan second guessed by distant “third parties” (working for government or insurance bureaucracies) who, of course do not –indeed cannot- know the physician or the unique individual circumstances of a particular patient. Lacking any knowledge of the patient or the doctor, these bureaucrats must fall back on general “guidelines” as the basis for approval or rejection of a particular treatment. Having served on guideline development bodies I can tell you that, at best, they represent the kind of “lowest common denominator” conclusions necessary to get the many different opinions represented on a committee to coalesce into a consensus. At worst guidelines represent a deliberate effort to drive down the cost of treatment by emphasizing “one size fits all” treatment approaches. While the physician-patient relationship theoretically remains the fulcrum of good medical practice and medical progress, in reality it is rapidly being replaced – on both sides of the Atlantic – by guidelines.

This movement towards guideline-driven medicine to which access to healthcare and physician reimbursement is increasingly linked, is based on the assumption that variations in medical practice are not only wasteful, but also potentially dangerous. Eliminating variations in clinical practice, we are told, will increase quality and save billions that could be poured into covering more uninsured or increasing coverage. The key to standardizing treatments – and outcomes – therefore are guidelines based on a combination of retrospective analysis of randomized clinical trials and the results of large prospective studies that compare the cost and effectiveness of established treatments or drugs in certain disease areas.

I bring a special perspective to this subject because of my professional background. On the one hand I have been a practicing physician for nearly 40 years and during almost all of those 40 years I was medical researcher and policy maker at the National Institute of Mental Health (NIMH). Incidentally my long experience in a part time private practice came about as a consequence of government salaries at the time not being competitive with academia so that a private practice was allowed in order to retain talent.

I was director of NIMH during the health care reform efforts of the first Clinton administration and, as such, I participated in some high level meetings of groups that were part of the effort. It is now well known that the perspective of practicing physicians was not included in this health care reform effort and my involvement was as the government’s principal mental health official, not as a clinician. Indeed I doubt that any of the other participants in the meetings I attended even knew that outside of my government job I was a physician in the private practice of a medical specialty.

I present this history so that the reader can understand what I am about to say. The meetings I attended had a surreal “Alice in Wonderland” quality. Here were all of these intelligent, well educated, well intended professionals discussing meta-analyses of controlled clinical trials, outcomes research, etc, etc, and yet it seemed that that none of them really understood what clinical practice was all about – it’s about enormous individual differences even among patients with the same diagnosis, it’s about cross-over trials where the physician uses each individual patient as their own control, trying treatment B when treatment A isn’t working, etc, etc Today’s advocates for coercive guideline driven medicine (as opposed to guidelines which are advisory to the doctor and the patient) seem to be cut from the same cloth as the people I encountered on those committees in the early 90s, except that now the lure of saving money and increasing the profits of managed care companies provides a new level of passion and intensity to these efforts.

In Europe, doctors are limited by reimbursement patterns and practice guidelines designed to control costs. This trend is growing in the United States. Yet there is no evidence that limiting access based on reviews of clinical literature or large scales trials either improves outcomes or saves money. On the contrary, the outcomes evidence suggests that seeking to elimination variations in practice and prescribing is actually more costly and contributes to morbidity.

The practice guidelines themselves are not the problem, it is how they are applied and for what purpose. Voluntary guidelines provide the intelligent physician with a benchmark from which to mark a patient’s progress or the impact of prevention. I have been involved in the development of practice guidelines for the treatment of mental illness for nearly three decades. It is a given that by the time there are developed, the guidelines themselves are outdated as new clinical insights and biomedical discoveries inform and shape both diagnosis and prescribing. The rigid imposition of guidelines regarding what to prescribe and how to treat insure that doctors cannot tailor treatments to the patient or deliver the best care.

Finally, the emphasis on cost-containment undermines continuity of care. The recognition that someone with mental illness is twice as likely to suffer from heart disease, diabetes or hypertension reveals the interaction of disease pathways which themselves have been show to be highly individualized. The evidence-based medicine movement fails to incorporate such insights. Rather, by emphasizing studies that evaluate the treatment of one aspect of a particular disease in a vacuum, the EBM movement is contributing to fragmentation of care.

Clinical decision-making is becoming increasingly centralized and the domains of economists or physicians who crunch numbers but never practice medicine.

Watch this space for more excerpts from this new and timely publication.  Read More & Comment...

Spare Change?

03/18/2008 11:14 AM |

According to the RPM Report:

“FDA Commissioner Andrew von Eschenbach expressed rare public irritability last week at the American Enterprise Institute.

What brought an edge to the voice of the usually unflappable, on-message commissioner? He bristled at the suggestion that the re-appointment of Janet Woodcock to head of the Center for Drug Evaluation & Research represents an attempt to rebuild the old ways of running the agency’s drug regulatory operations."

“I do not see her going back to CDER as business as usual,” von Eschenbach shot back in response to a question from a reporter during a Q&A session. She “is going back as a very, very strong change agent, and we have mapped and discussed many of those changes that she is embarking upon.”

Here’s the complete story:

http://therpmreport.com/Free/41678225-dbfe-4f3a-972e-c223f74843d8.aspx?utm_source=RPMel

Some more aggressive retorts from the Commissioner:

Just because she has a long tenure in FDA management and is can be termed an “insider”, the commissioner said, “does not in any way, shape or form mean that she is not a change agent.”

Woodcock offers “understanding of the historical perspective as well as the external realities that are now impacting upon the agency,” the commissioner says (see here). She is “absolutely, the most extraordinarily gifted and talented person to lead” change at CDER.

Give 'em hell Andy -- it's about time.  Read More & Comment...

Falling into FDA's Web

03/18/2008 10:48 AM |

New website that is.

Check it out at http://www.fda.gov

(We'll take FDA reform in any form.)  Read More & Comment...

Inhuman Healthcare Economics

03/18/2008 09:19 AM |

Here's Alan Greenspan on the economic models used by the Fed and the banking community that "justified" the deep exposure of some financial institutions in subprime mortgage instruments:

"The models used by the finance industry to determine risk and measure economic strength are too simple to fully account for human responses. We cannot hope to anticipate the specifics of future crises with any degree of confidence."

http://money.cnn.com/2008/03/17/news/economy/greenspan/index.htm?postversion=2008031707

Translation: the rational models of utility and discounting activities are outdated because they fail to capture rapid and wide variations in response to risk and uncertainty.

Similarly, comparative effectiveness research, complete with QALYs and the assumption that standardizing care will lead to lower costs and optimal health are essentially ersatz consumer welfare models based again on the "rational actor" view of economics. There is more variability in healthcare because valuations and value do not correspond to particular price but rather to individual perceptions of risk and uncertainty that are part and parcel of the effort to treat and prevent disease. The simple models developed to predict and shape human responses in the healthcare system will anticipate changes or outcomes with any accuracy. Nor have they.


We have come a long way from when the RAND experiment was conducted.  Read More & Comment...

Foul-Up on Posting About Follow-On Biologics

03/17/2008 01:15 PM |

A post on the wsj.com healthblog by Heather Won Tesoriero claims

"Late last night, a pair of congressmen introduced a bill aimed at creating a pathway for approval of generic biotech drugs.

Finally, you say. But, wait, there’s a catch, and it’s a big one. The legislation would also give brand-name biotech drugs an extra 14 1/2 years of patent protection. (Read a summary of the bill, or the complete text.) That almost defeats the purpose of establishing a route to market for generic biotech drugs, advocates for them say. "

Well I read the summary and the text and it sounds like someone is buying the generic trade group's spin on the patent protection. No one is getting 15 years of additional patent protection. Rather, it gives companies going forward 12 years of data exclusivity and 2.5 years of patent life that of course begins well before a biologic enters clinical trials. The wailing is part of a larger strategy by the generic trade group to argue for the right to challenge biologic patents early and get market exclusivity as generic firms receive if they do make a successful challenge. Wailing as in: "Waah! Innovators get solid patent protection so I can't start copying their products from day one...waah!!

I am sure the anti-capitalists who attack pharma and BIO will go wild...


http://blogs.wsj.com/health/2008/03/14/want-generic-biotech-you-might-wait-14-more-years/?mod=googlenews_wsj  Read More & Comment...

Uncertainty MD

03/17/2008 08:45 AM |

Those who think a comparative effectiveness institute will generate super savings by imposing standardization on clinical practice should read Benjamin Brewer's latest blog in the WSJ entitled "Uncertainty is My Co-Pilot"

http://online.wsj.com/article/SB120527311006228441.html?mod=djempersonal  Read More & Comment...

A Dose of Science About Drug Dosing and Off-Label Uses

03/17/2008 08:18 AM |

Two articles in the NY Times -- one about the discussion of off-label uses for Zyprexa and another accusing drug companies of launching meds at a higher dose than necessary -- are devoid of scientific context.

The failure to report on off-label uses without describing the process of arriving at them and the fact that most are the product of trying to solve a clinical problem is a consistent and glaring gap in reporting. And burying the science only empowers trial attorneys and federal prosecutors who are constantly prodded by Senator Grassley to use the threat of prosecution to shake down drug companies for settlement fees.

With respect to the allegation that Avastin, Herceptin, Cerazyme, etc., are overdosed on purpose is completely without merit. Dosing is -- by the nature of clinical trials -- one size fits all. Achieving an appropriate dose in the real world goes on all the time and hitting the right dose -- as the warfarin genetic labeling effort suggests -- is a matter of genetic variation and other factors that cannot be integrated into drug development. And consider this: in some instances changing the timing and dosing of certain drugs can be regarded as off-label use and therefore subject to prosecution and tort lawyer litigation.

When Avastin was being developed it's initial "failure" in early trials was -- according to Judah Folkman who pioneered angiogenesis drugs -- linked directly to the fact that the FDA wanted every patient to get the same dose even though he believed varying dosing to a number of criteria -- would have led to better and more precise outcomes.

Many drugs don't work in most people at the marketed dose. The failure of HMOs not to provide coverage of the most expensive and innovative medicines should have nothing to do with dosing. The right dose for the right patient at the right time for the best outcome is the goal of any doctor. The Critical Path leading to personalized medicine or tailored treatments reflects the commitment of companies and the FDA to move away from the one-size fits all approach.

The media continues to ignore the underlying science shaping medicine and it leads to the arrogant notion that prosecutors and policy analysts can strip doctors of their discretion about how to prescribe and use medicines to advance the health of their patients.

http://www.nytimes.com/2008/03/16/business/16gaucher.html?pagewanted=1&_r=1&ref=todayspaper

http://www.nytimes.com/2008/03/15/business/15drug.html?scp=1&sq=berenson&st=nyt  Read More & Comment...

The Tainted Veil

03/17/2008 07:51 AM |

Last August I commented on the Lou Dobbs program that it was unlikely that Congress would take FDA reform seriously “until there were dead bodies.”

Unfortunately, I was right.

On Friday, in the shadows of tainted Heparin, the Senate passed a budget resolution to give the F.D.A. an additional $375 million, a 20 percent increase over this year.

Some representative quotes on this issue from an article by Gardiner Harris in today’s edition of the New York Times:

“Congress has a responsibility to close the glaring gaps in food and drug safety that have begun to overwhelm the F.D.A.,” Senator Edward M. Kennedy, Democrat of Massachusetts.

“F.D.A. needs a serious infusion of resources and strong leadership dedicated to reforming the agency,” said Representative Henry A. Waxman, Democrat of California.

And, of course, everyone’s favorite FDA expert, Representative Rosa DeLauro, Democrat of Connecticut, “I don’t want to throw money at an agency that doesn’t have the infrastructure to carry out its mission.”

Some top agency officials are simply “incompetent,” she added, and real change can occur only with a new administration.

Really, a new administration? Note to Representative DeLauro – the head of every center at the FDA is a career government employee. At the FDA, an agency of roughly 10,000, there are fewer than 10 “political” appointees. Does Ms. DeLauro, the chair of the House appropriations subcommittee with authority over the agency, believe that Dr. Janet Woodcock is “incompetent?” What about CBER’s Dr. Jesse Goodman – widely considered one of the finest scientists in government?

And then, of course, there’s the usual ranting from our favorite Sheep in Wolfe’s Clothing.

Here’s a link to the complete article:

http://www.nytimes.com/2008/03/17/health/policy/17fda.html?scp=2&sq=gardiner+harris&st=nyt

Alas, the title of the article says it all –“Tainted Drugs Put Focus on the F.D.A.”

And it isn’t even a done deal.   Read More & Comment...

There Will Be Blood: Part II

03/14/2008 08:56 AM |

Here's a link to a a video that shows how atherosclerosis happens. This arterial tour gives you a graphic view of how unhealthy habit and genes can conspire to clog the major routes that supply blood to the heart and back again. No butter with your popcorn when watching this movie. Steve Nissen makes a cameo appearance as an avenging LDL molecule..(Not really.)

Read Full Story



  Read More & Comment...

When is an industry trial not an industry trial?

03/14/2008 06:25 AM |

The Center for Medicine in the Public Interest (the think tank home of drugwonks.com) is part of a transatlantic public policy institute consortium on the future of healthcare technology assessment (HTA). One of the member organizations, the German Institut fur Unternehmerische Freiheit (the Institute for Free Enterprise) held a seminar in Berlin yesterday entitled, "Cost Pressures on the German Health System -- Is Health Technology Assessment the Solution?"

One of the speakers was Dr. Christian Behles, Director and Professor of Drug Regulatory Affairs at the University of Bonn and an advisor to the German government.

Professor Behles pointed out that while IQWiG casts a suspicious eye on industry-designed pharmaco-economic studies, they use industry-sponsored RCTS as the basis of their comparative effectiveness findings.

He also noted that these RCTS were not designed to be used for head-to-head comparisons -- further reinforcing the recent comments made by NICE's Sir Michael Rawlings in front of the British House of Commons that HTA "is not based on empirical research."

In other words, IQWiG embraces industry-sponsored RCT data that was not designed to be used comparatively, while rejecting industry-sponsored data that was specifically designed to show the value of a new innovative medicine.

When is an industry study not an industry study? It seems that, for IQWiG, the answer is "when it's convenient."

It's interesting to note that the title of Professor Behles' presentation was, "HTA and Political Interference -- the Case of Germany's IQWiG."  Read More & Comment...

ODAC Jumps Off Padzur's Railroad and Refuses to Join Media Circus

03/13/2008 04:47 PM |

The best part of the ODAC meeting was when the committee as a whole dismissed Richard Padzur question -- designed to prop up an increasingly suspect coverage decision by CM -- asking to slap a one-size fits all dosing limit on ESAs. To quote one panelists: "This is silliness." Other comments could be characterized as dismissive.

In general the panel appeared perturbed that the FDA essentially did not provide it with either an objective or complete picture of the overall risks and benefits of ESA use in chemotherapy. The same goes with Padzur's lame attempt to misconstrue his negative opinion of the nature of quality of life data because most of it is observational study as the final word on the subject. He fooled the media who failed to look deeply into the data but not practicing oncologists on the panel.

Of course the media had all but predicted the demise of ESAs and is treating the ODAC decision as some sort of upset. Read the CNN.com piece below. You can just feel the shock and disappointment....Ultimately companies and doctors will have to work together to come up with more patient-centric data on who the drugs work for. If they had done this in the first place, the generalized concerns about safety could have been mitigated. Let's hope they do so going forward.

The committee did the right thing by swatting away Padzur's thinly veiled attemtp to manipulate them into affirming CMS' wrongheaded coverage decision. It did the right thing by giving doctors flexibility and recommending limiting use in areas where no benefit seems to exist.

http://money.cnn.com/2008/03/13/news/companies/amgen/?postversion=2008031316  Read More & Comment...

ODAC Live

03/13/2008 01:16 PM |

The committee voted against restricting the use of ESAs to small cell lung cancer.

Question 2--Should FDA require that product labeling be modified? Please address each of four potential approaches to mitigating risks through revised labeling separately.

a. To date, only clinical trials in small cell lung cancer have reasonably excluded an increased risk for death among patients receiving ESAs. Trials have demonstrated an increased risk of death and/or tumor promotion in head/neck, non-small cell lung cancer, breast (neoadjuvant and metastatic settings), lymphoid malignancies, and cervical cancers. Tumor types, other than those listed above, have not been adequately studied. Should the current indication be modified to restrict use only to patients with small cell lung cancer?

Vote: YES-- 6 NO-- 8

Question 2--Should FDA require that product labeling be modified? Please address the following potential approaches to mitigating risks through revised labeling.

b. Vote: The PREPARE trial demonstrated decreased relapse-free and overall survival in breast cancer patients receiving neoadjuvant chemotherapy. The risk/benefit assessment is different for patients receiving neoadjuvant and adjuvant chemotherapies than for patients with metastatic or incurable cancers. Should the current indication be modified to include a statement that ESA use is not indicated for patients receiving potentially curative treatments?

Vote: YES--11 NO--2
The current indication should be modified to say that it should not be used in the adjuvant setting.



c. Vote: Although increased tumor promotion and/or decreased survival have been demonstrated in several tumor types, adverse findings have been duplicated in two malignancies-breast cancer and head and neck cancer Should the current indication be modified to include a statement that ESA use is not indicated for patients with breast and/or head & neck cancers? (If yes, please specify breast and/or head & neck cancer).

Vote: YES--9 NO--5
Modified to say that ESA use not indicated for metastatic breast and head and neck cancers.  Read More & Comment...

Evidence-Based Medicine Takes a Flying Leap

03/13/2008 08:07 AM |

http://www.bmj.com/cgi/content/full/327/7429/1459?eaf


Hazardous journey
Parachute use to prevent death and major trauma related to gravitational challenge: systematic review of randomised controlled trials

Gordon C S Smith, professor1, Jill P Pell, consultant2

1 Department of Obstetrics and Gynaecology, Cambridge University, Cambridge CB2 2QQ, 2 Department of Public Health, Greater Glasgow NHS Board, Glasgow G3 8YU

Correspondence to: G C S Smith gcss2@cam.ac.uk

Abstract

Objectives To determine whether parachutes are effective in preventing major trauma related to gravitational challenge.

Design Systematic review of randomised controlled trials.

Data sources: Medline, Web of Science, Embase, and the Cochrane Library databases; appropriate internet sites and citation lists.

Study selection: Studies showing the effects of using a parachute during free fall.

Main outcome measure Death or major trauma, defined as an injury severity score > 15.

Results We were unable to identify any randomised controlled trials of parachute intervention.

Conclusions As with many interventions intended to prevent ill health, the effectiveness of parachutes has not been subjected to rigorous evaluation by using randomised controlled trials. Advocates of evidence based medicine have criticised the adoption of interventions evaluated by using only observational data. We think that everyone might benefit if the most radical protagonists of evidence based medicine organised and participated in a double blind, randomised, placebo controlled, crossover trial of the parachute.

I am sure CBO's Peter Orzag, who has been pushing comparative effectiveness as a way to reduce costs by denying access to new technology will be the first to volunteer....  Read More & Comment...

How many economists does it take to screw in a lightbulb?

03/13/2008 07:06 AM |

If the lightbulb is meant to shine a light on the value of new healthcare-related technologies in the context of healthcare technology assessment (HTA) -- then the answer is "one."

And the "one" is Dr. Frank Lichtenberg of Columbia University.

According to Frank, for HTA to yield valid decisions in practice, it is necessary to have reliable estimates of:

ΔCOST
ΔQALY
and VSLY (Value of a Statistical Life Year)

And his main point is that the devil is in the details.

He believes that incorrect estimates of some or all of these key inputs are often used:

ΔCOST is frequently overestimated
ΔQALY and VSLY are frequently underestimated

And due to these estimation biases, health technologies that are truly cost-effective may often be rejected as cost-ineffective.

Per the recent debate over the utility of new cancer treatments, he makes a very interesting point -- that even though, over the past 30 years, the U.S. Mortality Age-Adjusted Rates for cancer have remained relatively constant -- (leading to such mainstream media headlines as Fortune Magazine's "Why have we made so little progress in the War on Cancer?” and NEJM articles like "The effect of new treatments for cancer on mortality has been largely disappointing” -- the often ignored reality is that 5-year relative survival rates, for all cancer sites, have increased from 50.1% in 1975 to 65.9% in 2000.

For more specifics on both the economic impact of new treatments and their impact on cancer survival, please see the paper that Dr. Lichtenberg wrote for the Center for Medicine in the Public Interest in 2007:

Click here:

http://www.cmpi.org

Then go to the heading "Reports" and click on "Value of Cancer Drugs."

Lichtenberg cites two crucial studies, pointing out how health care economists must seriously reconsider the outdated estimates of a QALY:

Viscusi and Aldy: The value of a statistical life for prime-aged workers has a median value of about $7 million in the United States

Viscusi, W. Kip and Joseph E. Aldy, “The Value of a Statistical Life: A Critical Review of Market Estimates Throughout the World,” The Journal of Risk and Uncertainty, 27:1; 5–76, 2003.

and

Murphy and Topel: The value of a life year is $373,000.

Murphy, Kevin M., and Robert H. Topel, “The value of health and longevity,” Journal of Political Economy, 2006.

Attention must be paid. Hello NICE. Hello IQWiG. Hello Senators Baucus and Conrad.

Here is Dr. Lichtenberg's presentation on these issues -- spelled out and supported by both facts and examples:

Download file

If the devil is in the details (and it is) -- it's time for a deep dive beyond simplistic and self-serving "comparative effectivess."  Read More & Comment...

ESAs Under the Gun and Off the Critical Path

03/12/2008 12:22 PM |

After reading the FDA's documents prepared for the ODAC review of ESAs I am struck by how primitive and incomplete the brief about the safety problems associated with the anemia drugs are and how short-sided the FDA is in how to assess risk and benefit of the drugs going forward.

1. The FDA memo ignores quality of life benefits and it's risk management of the drug ignores the opportunity to use electronic medical records and observational studies to determine which dose works for what patients. It rewrites the standard for demonstrating quality of life to require randomized controlled trials to demonstrate such benefits....



2. The FDA memo ignores patient preferences and would radically limit the freedom of doctors to prescribe drugs based on their real world experience as opposed to the results of clinical trials focusing on higher than label doses.

3. The FDA ignores the fact that there are no randomized clinical trials assessing the impact of transfusion on survival or mortality.

4. Rather it cites the decline in transfusion-related infections even though the principle reason for using ESAs in chemo-related anemia was to reduce fatigue and sustain hemoglobin levels more efficiently in tandem with newer and more powerful cytotoxic agents/regimens.

5. The FDA ignores the fact that requiring RCTs to establish safety would entail studies of such power that doing so will be nearly impossible. Imposing this standard on all drugs would eliminate many drugs from regular use.

The fix is in. To a large extent the companies have themselves to blame for not tracking the risk and benefits of these medicines more consistently. However denying access to patients who feel better on the drug and who know the relative and absolute risks associated with their use is wrong. And it sends a message to companies that efforts to demonstrate risks and benefits in the post market consistent with the Critical Path will be rejected. The FDA's use of unsophisticated arguments and models in pressing for ESA restrictions underscores that trying to create a patient-centered pathway is simply not worth it. And if it isn't, how serious can the agency be about Critical Path and including patient preferences in its evaluations?
http://www.fda.gov/ohrms/dockets/ac/08/briefing/2008-4345b2-00-FDA-index.htm  Read More & Comment...

Acronyms of Denial

03/12/2008 04:48 AM |

I am currently "on the road" in Europe with Dr. Frank Lichtenberg of Columbia University. We're speaking on the topic of healthcare technology assessment (HTA). We've already spoken in Brussels (cold and wet) and Rome (sunny and fattening). Today we are in Berlin.

When it comes to health care there are a lot of acronyms. When it comes to cost-containment strategies the main ones are:

HTA: Healthcare Technology Assessment

EBM: Evidence-Based Medicine

CER: Comparative Effectiveness Research

RUM: Rational Use of Medicine

But no matter the acronym, these are all cost-based practices designed to reduce costs and restrict patient care. They are acronyms of denial.

Today, Healthcare Technology Assessment is a short-term, short-sighted, politically-driven policy that results in one-size-fits-all medicine. And while it may provide transitory savings in the short-term, current HTA strategies result in a lower quality of care that result in higher health care costs over time.

Restrictive formularies and health care systems that deny patients access to the right medicine in the right dose at the right time but pay for more invasive and expensive procedures later on have their priorities upside down.

So why is the current HTA model enjoying such wide support? Because it drapes a veil of pseudo-science around the blunt instrument of one-size-fits-all price controls. Consider what Sir Michael Rawlings of NICE said about comparative effectiveness in front of the British House of Commons:

“It is not based on empirical research, there is no empirical research anywhere in the world, it is really based on the collective judgment of the health economists we have approached across the country. It is elusive."

IQWiG in Germany claims that it makes its decisions based on "international standards." But such "standards" do not exist. Nice try though.

HTA, as it is currently designed, places into conflict the short-term budgeting dilemmas of governments elected for relatively short periods of time with the ever-lengthening life spans of its electorate.

HTA is a creature not of health care professionals, but of economists being paid by governments (aka: "payers"). Hardly a group of disinterested academics.

HTA is widely based on the concept of “patient variation,” that the same care should be applied to every patient suffering from the same disease based on evidence derived from RCTs.

In other words, if one medicine is effective for 40% of the target population and another drug within the same therapeutic category if also effective for 40% of the population – but we cannot (because of the design of these RCTs) clinically predict which 40% will respond to either treatment – what kind of evidence is that?

What’s a regulator to do? Their only alternative, as they see it, is to rely on cost-based comparisons. In other words, if two medicines are “equally effective” at 40% -- then they will opt to reimburse the one that costs less.

That isn’t evidence-based medicine. That’s bad medicine.

21st Century HTA models should reflect and measure individual response to treatment based on the combination of genetic, clinical, and demographic factors that indicate what keep people healthy, improve their health, and prevent disease. A rapidly aging society demands a new health care paradigm capable of providing for its needs in the 21st century. Equality of Care must be matched with Quality of Care.

The repercussions of choosing short-term savings over long-term results, of cost-based choices over patient-centric care, of “me-too” medicines over the right treatment for the right patient at the right time – are pernicious to both the public purse and the public health.

As Mark McClellan said, “Looking at a gigantic uniform solution for everything is never going to work.”

We're not at the end of this debate. We're not at the beginning of the end of this debate. But we are at the end of the beginning where at least we can all agree that this is not, and must not be exclusively a debate about saving money. It must be about patient care.  Read More & Comment...

Climbing the Peak of CDER Sinai

03/11/2008 06:04 PM |

After a national search for a new CDER director -- the best and the brightest was chosen.

Dr.Janet Woodcock is returning as full-time center director.

An overdue homerun for the FDA.  Read More & Comment...

But Let's Prevent Them From Working With Industry Anyways

03/11/2008 10:56 AM |

Leave it to the conflict of interest police to dig an even deeper hole for academic researchers and destroy American competitiveness: NIH funding is declining, restrictions on NIH researchers and NIH supported researchers are tighter than ever and now the conflict Kapos want to prohibit researchers from any sort of collaboration with drug companies or biotech firms.

A group of some of the most prestigious research groups in the country says that five years of flat budgets for the National Institutes of Health is threatening to deter an entire generation of young researchers. Scientists from UCLA, Harvard, Vanderbilt and four other research institutions say that the stagnant NIH budget is persuading young researchers to go into other careers or move to other countries which have proved more generous to biomedical research. To drive that point home, the report--"A Broken Pipeline"--profiles 12 young researchers engaged in groundbreaking work on stem cells, cancer and kidney disease and their difficulty finding new grants.

"This is a real problem, discussed at almost every meeting one attends on campus, that can't be simply dismissed," said Drew Faust, Ph.D., president of Harvard University. "This is about the investment that America is--or is not--making in the health of its citizens and its economy. Right now, the nation's brightest young researchers, upon whom the future of American medicine rests, are getting the message that biomedical research may be a dead end and they should explore other career options--and in too many cases, they're taking that message to heart. The president's latest budget proposal that calls for another year without an increase will only make the problem worse."

fiercebioresearcher.com

They live in a la-la- land where the government will just double NIH funding by raising taxes and -- according to the Soros-funded Institute for Medicine as Profession -- also add billions more to carry out drug development.

Why doesn't the media ever look at the implications of these ideas. I have always said, people like the folks at Healthcare Renewal, Sid Wolfe, Marcia Angell, Merrill Goozner, etc are willing to harm the public health en route to killing the private sector's role in drug development. If their conflict of inflict agenda is adopted -- which also opens doctors to the increased threat of lawsuits from trial attorneys -- they will have taken a strong stride towards that misanthropic goal....  Read More & Comment...

Disconnect on Drug Patents

03/10/2008 12:48 PM |

Two articles in the Wall Street Journal regarding the use, misuse and theft of drug patents...Of course all the drugs made by Thai-government run companies will be used for the poor and be of top quality....And none of them will ever make it onto the black market because there are SO many limits placed on selling inferior or bogus drugs to unsuspecting people in poor countries...



http://online.wsj.com/article/SB120515886199824251.html?mod=djempersonal

http://online.wsj.com/article/BT-CO-20080310-706680.html?mod=djempersonal  Read More & Comment...