FDA Reform and Risk Perception

02/02/2012 02:15 PM |

According to an article in Reuters by Anna Edney, there has been a dispute among large and small biotech companies about how specific FDA reform legislation should be.  

The plan, drafted by Democratic Senator Kay Hagan of North Carolina, was supported by biotechnology companies to speed up approvals and make the process less expensive. It would have created a program with the flexibility to allow drugs to be cleared based on any testing that shows they may be effective. This may include early trial results, interim data and use of so-called biomarkers that measure the effects of a molecule on biological functions linked to a disease.

The unusual dispute pitted biotechnology companies struggling to finance extensive trials against larger drugmakers who often trade financial aid in return for sharing sales later, said Erik Gordon, a business professor at the University of Michigan in Ann Arbor.

“Smaller biotech companies often can’t raise enough money on their own to do the currently-required safety trials, so they want to dilute the rules to let them get approvals without having to give big pharma a cut of the action in return,” said Gordon, who is based in Ann Arbor, in an e-mail. “Big pharma says ‘don’t dilute safety measures for new drugs.’ That sounds better than ‘we want to keep our cut of the action.’”

I don't agree with Gordon.   He assumes that small and big firms have different interests.  In fact, the larger firms are partnering with or buying smaller biotech companies to sustain product development.  Why would a company invest in a product that will go through the FDA faster only to oppose reforms that would slow down approval? 

Rather,  larger companies view new approval measures and endpoints as causing uncertainty about how FDA will respond.  Smaller companies see the regulatory path as risky to begin with and regard specific requirements to approve based on a number of measures as reducing uncertainty.

It's a difference of perspectives, both of which have merit and are not at odds with the other.   I tend to side with the small companies and favor approval benchmarks that are based on a variety of response measures and post market experience.  But companies of all sizes and disease foci want to get highly effective products to market more quickly. 

My question is: how do we insure that what a product does -- as well as it's risks and benefits -- reflect consumer values more directly?

You can read the entire article here:  http://www.businessweek.com/news/2012-02-02/drugmakers-upset-approval-system-plan-biotechs-sought.html

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Ready. Aim. PCORI.

02/02/2012 08:28 AM |

Representative Brett Guthrie (R-KY) introduced a bill (H.R. 3827) that would repeal the Patient-Centered Outcomes Research Institute and funding for comparative effectiveness research. The bill was referred to the Committee on Ways and Means, the Committee on Appropriations, the Committee on the Budget, and the Committee on Energy and Commerce. It was co-sponsored by Reps. Dan Benishek (R-Mich.), Joe Barton (R-Texas), Marsha Blackburn (R-Tenn.), Cathy McMorris Rodgers (R-Wash.) and Mike Rogers (R-Mich.).

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Another AHRQ Report, Another Wasted Million Dollars

02/01/2012 05:56 PM |

Yet another AHRQ review of decades old data concluding that there is no gold standard evidence (randomized clinical trials) to determine whether stem cell treatments help or hurt kids who need organ transplants or who have juvenile diabetes.  

Other feautres of the study that now characterize most AHRQ 'research.'

No mention of genetic tests or variations that can stratify and optimize treatment.

Conducted by the same people who dole out money to other AHRQ grantees and sit on the PCORI board and who also sit on CER boards of HMOs. (Naomi Aronson).  If someone from a device or drug company was a co-author of a study funded by an agency whose decisions they would derive benefit from, Merrill Goozner and Gary Schwitzer (the wholly owned subsidiary of Health Dialog) would mutate into another life form.  Oh, I forgot to mention that AHRQ is also hiring drug reps to peddle this stuff to doctors and hospitals. 

How about some oversight on AHRQ's activities en route to cutting its budget or reordering it's mission?

http://www.effectivehealthcare.ahrq.gov/index.cfm/search-for-guides-reviews-and-reports/?pageaction=displayproduct&productid=945  Read More & Comment...

National Pharmaceutical Council Measuring Whether CER Measures Up to Personalized Medicine

02/01/2012 02:32 PM |

http://www.npcnow.org/Public/Newsroom/Press_Releases/2012pr/2012five_research_projects_pr.aspx  Read More & Comment...

Redlining Vaccines

01/31/2012 07:42 PM |

If the people who make the decisions are the people who will also bear the consequences of those decisions, perhaps better decisions will result.

-- John Adams

On the last working day of the year (December 30, 2011), the FDA approved Prevnar 13 (a pneumococcal 13-valent conjugate vaccine) for people ages 50 years and older to prevent pneumonia and invasive disease caused by the bacterium, Streptococcus pneumoniae. In fact, the new use for Prevnar 13 was approved under the agency’s accelerated approval pathway, which allows for earlier approval of treatments for serious and life-threatening illnesses.

(The Centers for Disease Control and Prevention reports that 5,000 adults die from pneumonia every year.)

And to drive home the importance of this action, the FDA issued a press statement on the approval before heading home for the long weekend:

“According to recent information for the United States, it is estimated that approximately 300,000 adults 50 years of age and older are hospitalized yearly because of pneumococcal pneumonia,” said Karen Midthun, M.D., director of FDA’s Center for Biologics Evaluation and Research. “Pneumococcal disease is a substantial cause of illness and death. Today’s approval provides an additional vaccine for preventing pneumococcal pneumonia and invasive disease in this age group.” 

Not so fast.

Although it’s quite a high hurdle to have a vaccine approved by the FDA (and appropriately so), it’s not the final hurdle in getting it to patients.  That final hurdle resides with the Centers for Disease Control’s Advisory Committee on Immunization Practices (ACIP).

ACIP’s charge is to “provide advice and guidance to the Secretary, HHS, the Assistant Secretary for Health, and the Director, CDC, regarding the most appropriate selection of vaccines and related agents for effective control of vaccine-preventable diseases in the civilian population.”

The ACIP meets three times a year, and during these meetings newly licensed vaccines are discussed and a vote is taken to include (or not include) the new vaccine on the adult immunization schedule. ACIP’s recommendations become a basis for reimbursement by public and private payers who will pay for vaccinations that are part of the committee’s recommendation -- but generally not otherwise. The CDC schedule plays an important gatekeeper role for vaccines that goes well beyond the scope of FDA approval. Vaccines approved by the FDA but not appearing on the CDC routine vaccination schedule are likely to gain little traction because of a lack of guidance to providers on how to use the vaccine -- and lack of payer coverage.

In other words, minus a positive ACIP recommendation, a disease that is responsible for approximately 200,000 emergency room visits a year will continue to harass patients and haunt our healthcare system. Minus a positive ACIP vote, new and potentially life-saving vaccines are redlined and another nail is hammered into the coffin of innovation.

The FDA recognized the importance of the adult indication for Prevnar 13 (currently the only vaccine for pneumococcal bacteria approved in the United States for adults 50 years of age or older is Pneumovax which is only effective against invasive pneumonia and not effective on the more common, pneumococcal pneumonia. Prevnar 13 is a conjugate which means it contains a pneumococcal bacteria bound to a protein to help the body’s immune system recognize the bacteria and will have a longer lasting immune response), but at the upcoming ACIP meeting (February 22-23), there is only a discussion of 13-valent pneumococcal conjugate vaccine. Just discussion.  That’s important – but a positive recommendation is crucial.  Otherwise it is, in many unfortunate respects, just talk.

The need for this patient population exists.  The vaccine is safe and effective. Without a recommendation the vaccine will not be available to a large swath of Americans. It’s time for ACIP to call the question.

The battle against the “dangerous idiots” of vaccine denial is dangerous enough, we must avoid the equally daunting danger of … inertia.

As the saying goes, “Truth fears no questions.”

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Ken Abramowitz on the Pitfalls Inherent in Obamacare

01/31/2012 02:13 PM |

Remember when Obamacare was topic in chief in Washington?  Today, the administration and single payer supporters are avoiding the subject like the proverbial plague.

Hence,  Ken Abramowitz, our guest blogger reminds us of the pitfalls (and pluses) of Obamacare.   Ken is a co-founder and Managing General Partner of NGN Capital. He joined NGN Capital from The Carlyle Group in New York where he was Managing Director from 2001 to 2003, focused on U.S. buyout opportunities in the healthcare industry. Beginning July 2003, he transitioned to Senior Advisor at Carlyle in order to devote the time necessary to create a dedicated healthcare fund on behalf of Carlyle. Prior to joining Carlyle, Mr. Abramowitz worked as an Analyst at Sanford C. Bernstein & Co. where he covered the medical-supply, hospital-management and HMO industries for 23 years, after which he was an EGS Securities Healthcare Fund Manager. 

We look forward to his future posts on health care reform and medical innovation.

ObamaCare imposes European socialism “lite” on the U.S.  The plan involves 2,800 pages of legislation and, soon, 10,000-15,000 pages of regulation.  It is a massively underfunded $900 billion program that relies on stealing $500 billion from a grossly underfunded Medicare program and it does not recognize the $200 billion cost of offsetting the 21% Medicare physician cut.

Rather than making medicine more affordable, It will bend the cost curve upward by taxing insurance carriers, medical device companies, and pharmaceutical companies.  It also raises costs in the individual market by imposing insurance mandates (guaranteed issue, narrow underwriting bands, no lifetime benefit limits).

Worse it sets up grossly underfunded individual insurance exchanges that will quickly exceed projected spending.  therefore they eventually seek to control cost under dysfunctional price controls.  Thereafter insurance carriers will exit the market, tempting the government to take over as it did after the collapse of the housing market. These exchanges offer a costly defined benefit that is 65% subsidized by Federal and State governments, but should have been financed by a more affordable defined contribution.

On the positive side, the bill does provide genuine subsidies to finance and facilitate the “meaningful” use of EMRs and IT integration.  On the really positive side, the massive government overreach we will see voters continue to reject Obamacare as they did in 2010.   Hence, while the plan is guaranteed to blow up by 2015 fortunately 50% of the bill will be repealed before then. Though no one knows which 50%.

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Zeke Emanuel's Faith in ACOs

01/31/2012 12:30 PM |

Former Obamacare czar Zeke Emanuel boldly predicts that health insurance companies will be obsolete in 2020 and be replaced by ACOs that focus on managing disease and health outcomes

Didn't we hear that in the 1970s when people like Emanuel were predicting insurance companies would become obsolete and be replaced by HMOs that would manage health and improve outcomes?

The faith in administrative changes or addition of regulation to improve, shape, control human behavior is particularly strong among health care policy experts of all stripes.   I believe that it is technological progress that makes certain types of care --  fee driven, intermediate or palliative treatments produced through hardware or in hospitals -- obsolete.   Think of how infectious diseases were generally treated less than 50 years ago,  the use of medicines instead of institutions for people with mental illness, same day surgery vs. the 5 day stay.   

ACOs were designed to be conduits for government produced guidelines that are biased against new technology.  They are organized against innovation, not to capitalize on it.  

As a result, I believe by 2020 not only will the current form of underwriting be obsolete -- because of advances in personalized medicine and direct to consumer delivery of healthcare --  but so will ACO's and Obamacare.   If you think it was surprisingly easy to overthrow Arab dictators wait till you and I know more about our health sooner than government bureaucrats who produce outdated guidelines and mandates. 

Here's a link to Zeke's article:

http://opinionator.blogs.nytimes.com/2012/01/30/the-end-of-health-insurance-companies/?nl=opinion&emc=tya1  Read More & Comment...

Why Roche Wants Illumina

01/31/2012 12:18 PM |

Why does Roche want Illumina?

It's true that the cost of sequencing continues to follow Moore's law --  exponential decline in material and equipment costs and increased computing power -- so that what Illumina does with supercomputers is something that doctors and patients will be able to do with smartphones.   But Roche is not interesting in the hardware of sequencing.  If it did, it could just buy a bunch of machines for a lot less that $5.7 billion.

In fact, Roche knows that sequencing will become commonplace.  I think it wants Illumina because of it's partnerships with clinical labs worldwide to perform sequencing.  Distribution for Roche diagnostics and products -- as the firm focuses on targeted therapy -- is more valuable than equipment or sequencing.  Roche also sells glucose kits too and find that marketplace pretty lucractive. 

Should Illumina sell?  I don't know and have no opinion.  However, I remember when Yahoo snubbed a Microsoft acquisition for a hefty premium over it's share price.  Now Yahoo is scrambling to find a buy at a share price far below that offer.   Information is quickly becoming a commodity in health care.   In 10 years drug companies will be delivering individual content directly to consumers to treat illness.   Perhaps Roche believes it can be a source of revenue growth.  In any event, acquiring Illumina is about how medical care will be organized in the future, not about it's microarrays now.   Read More & Comment...

Ode to Bob. ODE1 to Ellis.

01/31/2012 07:57 AM |

Update on the Office of the Center Director and Office of Drug Evaluation1

CDER Staff:

I want to update you on recent changes in my Immediate Office to further enhance medical and scientific decision-making across CDER.  These changes also affect the Office of Drug Evaluation 1 (ODE1), Office of New Drugs.

I have asked Robert (Bob) Temple, M.D., to give up his role as the Acting Director of ODE1, so that he can devote more of his time and vast expertise to his other role as the Deputy Center Director for Clinical Science.  Ellis Unger, M.D., currently the Deputy Director of ODE1, will serve as Acting Director of ODE1.  Bob will continue as Acting Deputy Director of ODEI and plans to maintain substantial (but somewhat less) involvement in ODEI activities.

In November 2009, due to the growth of the Center’s responsibilities, I appointed Bob as the Deputy Center Director for Clinical Science with primary responsibility for high level initiatives and programs related to clinical science and clinical trial methodology issues.  At that time, Bob agreed to wear two hats and continue as Acting Director of ODE1.

As Deputy Center Director for Clinical Science, Bob has played and continues to play a major role in directing and executing many cross-cutting CDER functions, which enhance the quality and consistency of our operations.  Now he will have more time to further provide valuable regulatory input and methodological assistance across the Center.

I am grateful to Bob for serving ODE1 with excellence and superb leadership. I also am grateful that we have someone like Ellis to step in and assume the Office Director position on an Acting basis.  Ellis has been with CDER since 2004, serving in various leadership roles, and in the ODE1 Deputy Director capacity since August 2008.  I am confident that he will continue to serve ODE1 exceptionally well.

Janet Woodcock

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Quad Erat Demonstration

01/31/2012 07:48 AM |

Really interesting issue brief from CBO regarding approaches taken in various projects that help in attainment of demonstration goals:

Gather timely data on the use of care, especially hospital admissions. Programs that collected timely data on when their patients’ health problems developed or became exacerbated and where they were treated seemed better able to coordinate and manage their patients’ care. Those efforts could be strengthened if CMS improved its capability to provide programs with timely data on their patients’ use of services.

Focus on transitions in care settings. Programs that smoothed transitions (for example, by providing additional education and support to patients moving from a hospital to a nursing facility or between a primary care provider and a specialist) tended to have fewer hospital admissions.

Use team-based care. Demonstrations that provided close collaboration between care managers and physicians -- especially those with larger teams that included pharmacists, who could help patients manage their medications-- appeared to have fewer hospital admissions.

Target interventions toward high-risk enrollees. Programs that targeted interventions to beneficiaries they identified as being at greatest risk of being hospitalized—on the basis of medical condition, prior hospitalization, or predictive modeling—appeared to have fewer hospital admissions.

Limit the costs of intervention. To achieve federal budgetary savings, a program’s fees or bonuses must be smaller than its reductions in regular Medicare expenditures. There was nearly a threefold difference in the Medicare fees paid to different organizations that combined telephone and in-person contact to coordinate care, an indication that some organizations were able to deliver such interventions much more efficiently than others.

The complete CBO brief can be found here.

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PCORI Board Member Rails Against Screening For Babies Like Bella Santorum

01/30/2012 11:17 PM |

First, here's a blog from the Washington Post about two children diagnosed genetic disorders, one of them being Rick Santorum's 3 year old daughter Bella.

Bella Santorum, Amelia Rivera and ‘marginal’ children who prove no child is marginal

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The Wyden-Ryan Express

01/30/2012 08:03 AM |

The Wyden–Ryan proposal—a foundation for realistic Medicare reform

Joseph Antos | The New England Journal of Medicine

The need for significant Medicare reform is increasingly evident, even to policymakers long accustomed to avoiding this politically explosive topic. A host of commissions and expert groups, ranging from the President’s National Commission on Fiscal Responsibility and Reform to the Heritage Foundation, have argued that the United States is on an unsustainable fiscal path. Medicare is at the center of our fiscal crunch, with outlays that have grown about twice as fast as the economy over the past decade, according to the Congressional Budget Office (CBO).

Even if the substantial reductions in payments to health care providers included in the Affordable Care Act (ACA) are fully implemented and Congress allows the 27.4% reduction in physician payments required under current law to go through, Medicare spending will continue to grow at unsustainable rates. It is more likely that Congress will not enforce such large reductions in provider payments, making Medicare’s drain on the budget that much greater. With the retirement of 76 million Baby Boomers over the next two decades, the program will consume an ever increasing share of the federal budget unless policies are adopted to bend Medicare’s cost curve.

On December 15, 2011, Senator Ron Wyden (D-OR) and Representative Paul Ryan (R-WI) released a Medicare reform proposal based on the concept of premium support.[1] Under their proposal,

Medicare would be converted from a defined-benefit to a defined contribution program. Instead of guaranteeing to pay for services as they are rendered, as fee-for service Medicare does, the program would give beneficiaries a subsidy (“premium support”) to purchase coverage from one of multiple competing health plans. The motivation behind the approach is to give plans a clear incentive to provide necessary services in a cost-effective manner, which can result in lower premiums or other beneficiary costs, attracting enrollees and increasing the plan’s share of the market.

The new proposal gives minimal bipartisan credibility to a concept that has long been championed by Ryan and that, in various forms, has been supported by experts on both sides of the political aisle.[2] It also represents a considerable compromise from Ryan’s earlier proposal, which was advanced as part of the House budget resolution last spring.[3]

Under the Wyden–Ryan proposal, seniors would have a choice of private plans competing alongside traditional fee-for-service Medicare. All plans, including traditional Medicare, would bid against each other, with premiums based on the plan’s cost of providing the full package of Medicare-covered services. The federal subsidy would be tied to the cost of the second-least-expensive plan in each market, which means that beneficiaries would have the option of enrolling in a less expensive plan and receiving a cash rebate. Anyone choosing a more expensive plan would pay the full additional premium out of pocket.

The subsidy would be greater for beneficiaries with greater health needs or lower incomes, and the average payment would increase with the growth in the gross domestic product (GDP) plus 1%. In addition, low-income beneficiaries who qualify for both Medicare and Medicaid would be protected from premium increases and would receive additional subsidies to cover their out-of-pocket expenses. As has been the approach with other reforms, this premium-support system would not take effect until 2022.

Wyden–Ryan is more realistic in some respects than Ryan’s earlier reform proposal. The most important change is allowing beneficiaries to opt for traditional fee-for-service Medicare if they so choose. Some conservatives criticize this change as backsliding.[4] They correctly see the traditional

Medicare program in its current form as inefficient and anticompetitive. But pretending that the program will disappear in 10 years feeds the worst tendencies of politicians, who would avoid making important but difficult decisions needed to set traditional Medicare on a fiscally sustainable path.

The reality is that traditional fee-for-service Medicare will probably have about 57 million enrollees in 2022, and it could remain a dominant force in the health sector for decades if seniors continue to enroll.[5] That is likely in rural locales and other markets that are dominated by a small number of providers. In such cases, health plans have little bargaining power and thus little ability to compete on price. In other markets where there is less concentration and more competition among providers, health plans should be better able to contract selectively and offer lower-cost options to seniors.

Ryan and Wyden hint at the need for commonsense reforms to traditional Medicare, including a new structure of deductibles and copayments, a cap on catastrophic costs, and a new physician-payment system. They skirt the central problem: a disorganized fee-for-service system and top-down limits on prices paid for services drive the use of more, and more complicated, services. The program’s survival depends on our willingness to make substantial changes over the next few years — before the major reform is implemented — so that traditional Medicare can provide cost-effective care without draining the Treasury.

The current proposal also offers a more politically palatable fiscal target at the cost of achieving fewer “scoreable” savings. Under Ryan’s earlier proposal, the federal subsidy would grow only with general inflation (1.5% in 2012, according to the CBO) instead of the more generous target of GDP plus 1% (a rate projected to total 4.8% in 2012). Not coincidentally, that is the same fiscal target established for the Independent Payment Advisory Board (IPAB) under the ACA.

A 3.3-percentage-point difference in fiscal targets translates to a 1-year increase in program spending of about $20 billion, or about $300 billion over 10 years. Adopting the weaker target means a substantial loss of budget savings, but only if Congress would actually enforce the stricter limits. That may be unlikely given recent history. Over the past 8 years, Congress has overridden even relatively small reductions in physician payments called for by the sustainable growth rate formula. Clearly, a favorable score from the CBO does not guarantee lower program spending.

Will this premium-support proposal based on full competition among private plans and traditional Medicare work? Some critics argue that premium support simply shifts the cost of care to seniors without improving the efficiency of health care delivery. That would be true only if there were no room to improve health care efficiency or if plans ignored opportunities to cut costs, increase market share, and improve their bottom lines.

Under a premium-support system, each additional test or procedure would not generate additional reimbursement from the government. Most Medicare beneficiaries live on fixed incomes and are not in a position to pay more. That reality will force health plans and providers to coordinate patient care and find other efficiencies rather than perpetuating the current fragmented system.

The Wyden–Ryan proposal offers a safety valve. If its critics are correct, then traditional Medicare, with its price controls and government regulations, will be the low-cost plan in every market. Beneficiaries will shift back to traditional Medicare when the cost differences become apparent, and the competition experiment will be declared a failure.

But one should not be fooled. If the alternative to market incentives is price controls wielded by the IPAB, access to necessary services will inevitably be limited, as providers seek more lucrative business.

Given the serious fiscal problems facing this country, slowing the growth of Medicare spending is no longer optional. The only question is how to do it. The Wyden–Ryan proposal outlines a strategy for Medicare reform that harnesses market forces to control costs. It provides a real alternative to the top-down controls favored in the ACA. Paul Ryan and Ron Wyden have defined the policy parameters that could be the basis for real Medicare reform in 2013.

Antos is the Wilson H. Taylor Scholar in Health Care and Retirement Policy at AEI. He also is a commissioner of the Maryland Health Services Cost Review Commission and a health adviser to the Congressional Budget Office.

[1] Wyden R, Ryan P. Guaranteed choices to strengthen Medicare and health security for all: bipartisan options for the future. December 15, 2011 (http://budget.house.gov/UploadedFiles/WydenRyan.pdf).

[2] The Debt Reduction Task Force. Restoring America’s future: reviving the economy, cutting spending and debt, and creating a simple, pro-growth tax system. Bipartisan Policy Center, November 2010 (http://www.bipartisanpolicy.org/sites/default/files/BPC%20FINAL%20REPORT%20FOR%20PRINTER%2002%2028%2011.pdf).

[3] House Committee on the Budget. The path to prosperity: restoring America’s promise. Fiscal Year 2012 budget resolution (http://budget.house.gov/UploadedFiles/PathToProsperityFY2012.pdf).

[4] Suderman P. Paul Ryan’s Medicare compromise. Reason.com. December 15, 2011 (http://reason.com/blog/2011/12/15/paul-ryans-medicare-compromise).

[5] 2011 Annual report of the boards of trustees of the Federal Hospital Insurance and Federal Supplementary Medical Insurance Trust Funds. May 13, 2011 (https://www.cms.gov/ReportsTrustFunds/downloads/tr2011.pdf).

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Hike Soda Tax To Cut Child Obesity? Fat Chance

01/26/2012 01:41 PM |

Yet another example of shoddy 'research' confirming biases that are not established by evidence.

Tax on sugary beverages projected to have broad health benefits
A penny-per-ounce excise tax on beverage makers would mean reduced sales and rates of type 2 diabetes and associated health problems, a study says.

By Carolyne Krupa, amednews staff. Posted Jan. 24, 2012.

From the article reporting on the 'study'

"Forty states have sales taxes on soda, but they are too low to impact sales, said Dr. Y. Claire Wang, lead study author and assistant professor at Columbia University's Mailman School of Public Health in New York. California's tax is the highest at 7.3%. In many states, profits from the taxes are used to benefit health-related programs and low-income families."

Dr. Wang fails to tell us that while California has the tax rate on food as well as soda the prevalence of  kids classified as either obese or overweight (10-17) increased in the state while it decline in other states where the tax on food and soda is non-existent or lower.  

Social engineers like Wang want us to believe -- absent clear evidence -- that raising taxes on soda will reduce obesity and healthcare costs.  A glance at tax rates and obesity prevalence shows that it not so. 

http://archpedi.ama-assn.org/cgi/content/full/164/7/598

And of course every assumes an increase in obesity.  But is that absolutely true?  It turns out the prevalence of obese adults has remained the same from 2003-2008 with differences in the change of prevalence within enthnic and racial groups (as well as within states among similar ethnicities) according to a most recent CDC study that received almost NO media attention.

Prevalence of Obesity and Trends in the Distribution of Body Mass Index Among US Adults, 1999-2010.

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Liz Szabo's Piece Puts PFC Impact on Vax Response In Proper Perspective

01/26/2012 10:47 AM |

In my last post I wrote about heuristic biases that lead to wrong and even dangerous conclusions.  The one bias that runs rampant in media reporting of science, (Gary Schweitzer -- who claims he is objective -- is the worst offender) is relying upon single studies with small sample sizes that claim statistical significance (internal validity) to make a sweeping judgement about the dangers of chemicals, soda, medicines, etc.    

This week a single study, "Serum Vaccine Antibody Concentrations in Children Exposed to Perfluorinated Compounds" was published in JAMA http://jama.ama-assn.org/content/307/4/391 this week claiming that "elevated exposures to PFCs were associated with reduced humoral immune response to routine childhood immunizations in children aged 5 and 7 years. "    The research is the only study to look at PFC levels over time in humans.  The handful of other studies have only looked at the relationship betwee immune suppression in rats at human PFC levels.    Blood samples in this study were  taken to measure PFC levels in maternal pregnancy serums and then at age of 5 and 7.    There was no regular monitoring of serum levels from birth to age 5 or 7 nor from age 5 to 7.   The group was not compared to controls with different serum levels and antibody response.   So the results are just as likely to be random as they are to be suggestive.  

But that didn't stop JAMA or media outlets to sound the alarm using the heuristic of representativeness and small sample sizes to draw conclusions about probablities that are not supported by the evidence:


Chemical Contaminants Linked To Low Immune Response To Vaccines  MedicalNewsToday


Chemicals linked to lower vaccine response in children‎   AFP

Vaccine effectiveness reduced by common environmental toxin .   Boston Globe

However, Liz Szabo at USAToday provides the most balanced evaluation of the study and frames the coverage by calling to the limited sample size and the absence of causality.

http://yourlife.usatoday.com/health/story/2012-01-24/Chemical-exposure-may-compromise-vaccine-response/52774114/1

Chemical exposure may compromise vaccine response

"Children exposed to chemicals called PFCs — used in some non-stick cookware, stain-resistant coatings, fast-food packaging and microwave popcorn bags — have a reduced response to vaccines, raising the possibility that the compounds could prevent children from being adequately protected against disease, a new study shows..."

She goes on to cite Paul Offit, a straight shooter and great guy (even though he is a Phillies fan) and to point out that PFC levels are decreasing:

More vaccine coverage:

Continuous coverage by health reporter Liz Szabo on the myths and benefits of vaccines.

• Common myths about vaccines
• Measles returns as some parents refuse vaccines
• Parents describe pain of losing children to vaccine-preventable diseases
• Adult vaccines can hold down health costs
• More kids skip school shots in 8 states

  Read More & Comment...

PCORI's Information Imbalance

01/25/2012 06:04 PM |

I received a bunch of emails from people regarding my previous post on PCORI and I was also quoted in a recent Politico article on PCORI punting on looking at diseases and instead spending more money and hiring consultants to yet again decide what it should hire more consultants and hold more meetings about what it should study.   I received a form letter from PCORI's executive director Joe Selby that reads in part:

National Priorities for Research and Research Agenda. The priorities and agenda provide a framework and identify the broad questions that must be addressed so that patients can make better and more personalized decisions in partnership with their clinicians across all areas of health.

Our initial research agenda is not prescriptive about which conditions or treatments to study. It is a starting point. We hope that patients and other stakeholders will join with us in the coming months as we apply this framework to identify the specific questions that are most important for PCORI to address.

An initial research agenda with the goal of deciding what broad questions a neither research nor starting point, it's group therapy.

In my previous post I wondered by the National Pharmaceutical Council would hold a conference on asymmetries in information coming out of PCORI and made a somewhat snarky comment about not knowing what asymmetry is with respect to PCORI unless it mean dividing up it's grant money to stakeholders.

It was explained to me that 'asymmetry' has to do with an extremely important issue:  Namely while  that PCORI, AHRQ, etc will not only determine what information is sent out to doctors, patients, health plans  but will also control the means of production and distribution.  Unlike NIH, what PCORI will put out can and will be used in determining what health plans should and will pay for.  But given PCORI and AHRQs financial focuse on dissemination it begs the question of whether the mostly outdated information that will be distributed via government web sites, conferences and academic detailers will crowd out other forms of information and studies.  

The NPC event on asymmetry will be important if it raises these questions  http://www.npcnow.org/Public/Education___Events/Events/2012_events/Asymmetry_2012/asym12.aspx

Further, PCORI is loaded with what are called a heuristic biases.   I encourage anyone who doesn't think that PCORI is designed to establish asymmetries to read Judgment under Uncertainty: Heuristics and Biases  Amos Tversky; Daniel Kahneman Science, New Series, Vol. 185, No. 4157. (Sep. 27, 1974), pp. 1124-1131.  http://links.jstor.org/sici?sici=0036-8075%2819740927%293%3A185%3A4157%3C1124%3AJUUHAB%3E2.0.CO%3B2-M

Tversky and Kahneman describe how subjective assessment of probabliities leads to systematic and severe errors in judgment.   Even more so if the subjective assessement is also systematic. 

Why?  As T and K explain: judgments are all based on data of limited validity, which are processed according to heuristic rules.
What are these heuristics?  The first one of representativeness is used to assess a person, product, or event based on how similar it seems to be of stereotypes or preconceptions we already have in mind.   PCORI uses representativeness by assessing, funding and disseminatiing CER in terms of how well it fits the subjective belief that all CER will lead to lower costs and better health.   That is, if a particular approach to research and dissemination fits the pre-existing stereotype of CER then PCORI assumes it will improve health delivery.  Moreover, any objectiion raised to CER by introducing information basedon  the prior probability, or base-rate frequency, of the outcomes of CER is ignored.   Simply stated, PCORI never looks at the actual relevance and impact of CER to determine if it effective.  Rather, the problem is one of poor dissemination, not design or scholarship.  This insensitivity to prior outcomes is a hallmark of representative bias and explains the total lack of PCORI's self criticism.

Second, PCORI and AHRQ institutionalize insensitivity to sample size.   If a study compares two products and is representative of the desired CER outcome then it is accepted.  It adheres to the law of small numbers," according to which even small samples are highly representative of the populations from
which they are drawn. PCORI's is framed by the expectation that a valid hypothesis about a population will be represented by a statistically significant result in a sample- with little regard for its size.  So for instance Steve Pearson and Sean Tunis, two people who's organizations stand to be enriched by PCORI cash cite clinical trials that 'concluded' injection of medical cement into compression fractures of the spine produced no better pain relief than "sham" injections and the FDA's withdrawing approval of  Avastin for advanced breast cancer (claiming there were no benefits given the risks) as examples of good CER.  

But both ignore the extremely small sample sizes of studies for both issues.  Rather, the small studies are not only highly representative of the population as a whole but representative of what good CER is.    And this exposes another, more profound bias institutionalizd by PCORI, name that of availability.    We remember and regard as true based on what information happens most often or is more available.   Hence, because PCORI and AHRQ will produce and deliver most of the information on medical treatments and will control the outlets for such information it uses the heuristic of availability to create the perception that the CER it produces is the "right" path.  Or as Kahneman quips: "What we see is what it is."

Finally, PCORI reinforces the view that only CER and evidence based medicine it produces should be talked about, shared and acted upon.  So what if consumers with their own genetic information, companies that produce novel results, or physicians want to learn more about individualize care want to share and use such knowledge.   It turns out that they can't:  Under the health care law all the actionable information for benefit coverage and reimbursement is channelled through AHRQ and PCORI.   It's ok for AHRQ funded academic detailers to wine and dine docs about the blessings of CER but it is illegal for innovators to talk about and share scientific knowledge about the novel uses of their products. In otherwords some medical knowledge (PCORI/AHRQ produced CER generated by the people who have a subjective bias that CER is wonderful) is more equal than others.


There is more to discuss regarding asymmetry and there will be more to come.   But as you can see, I believe I was wrong to dismiss asymmetry as a Beltway slogan.   Asymmetry is a real problem.   As Tversy and Kahneman note: "The rational judge will nevertheless strive for compatibility,
even though internal consistency is more easily achieved and assessed. In particular, he will attempt to make his probability judgments compatible with his knowledge about the subject matter, the laws of probability, and his own judgmental heuristics and biases."

The way PCORI Is structured and the way in which AHRQ generates and produces CER, incentivizes the use of these biases.  By seeking to generate acceptance of CER and embedding results into clinical decision tools, PCORI and AHRQ makes "internal consistency" of the research it's funds the foundation of medical decsionmaking.    It thereby institutionalizes several errors in judgment that will leave patients less healthy.

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Senate Judiciary takes on Medicaid Rx rates

01/25/2012 02:13 PM |

Senate Judiciary takes on Medicaid Rx rates
Deborah Weinstein
January 24, 2012
 
Medical Marketing & Media


Senator Chuck Grassley (R-IA) put 34 Medicaid programs on notice that they are not doing enough to rein in over prescription of certain drugs and he wants to know why. He has issued a 12-point questionnaire that includes questions about how and if the states track their prescriptions and or provider prescription rates. They have until February 13 to reply, but the letter does not indicate whether there are consequences for not responding.

“Mental health drugs continue to be prescribed at astounding rates and pain management clinics are turning into a hotbed for black market painkillers. When these drugs are prescribed to Medicaid patients, it is the American people who pay the price for over-prescription, abuse, and fraud” he wrote.

The targeted drugs fall into three categories: anti-psychotics, anti-anxiety and pain-killing medications. Most of the cited programs had two to four over-prescribed medications. Some states, like Nevada, which was cited for eight drugs, and Illinois, which was cited for five, were outliers. The painkiller oxycontin and the anti-psychotic Abilify were among the most overprescribed among the 33 states and the District of Columbia.

Grassley says the prescription rates are astounding – and expensive. His letter to Illinois says doctors cost the state Medicaid program $2.9 million in prescriptions for Geodon, Xanax, Abilify, Zyprexa and Seroquel, and that high prescription rates cost Florida's Medicaid program over $4 million in 2009.
The senator's request is actually part two of an inquiry he kicked off in 2010, when he asked these same programs to identify their top 10 Medicaid prescribers of Abilify, Geodon, Seroquel, Zyprexa, Risperdal, Oxycontin,Roxicodone and Xanax. He said his inquiry, as the ranking member of the Senate Finance Committee, was to ensure the $317 billion states spend on Medicaid annually were legitimate expenses, as opposed to being applied to fraudulent prescriptions.
Monday's letter  letter to California explained the appeal; the senator explained that addicts use the antipsychotic drug Zyprexa to enhance and lengthen an opioid's effects Two doctors wrote 2,300 scripts for the drug in 2009. Cost: $879,000.

Propublica reports that the 13,825 Abilify prescriptions by a single Ohio provider in 2009 equals 54 prescriptions per weekday and cost the state $6.7 million.

Grassley's letters acknowledge that there is room to misinterpret the data, but that he is still concerned about “the oversight and enforcement of Medicaid abuse in your state. While I am sensitive to concerns about the potential for misinterpretation of the data you provided, the numbers themselves are quite shocking.”

His inquiry is not necessarily welcome.

“To commence an inquisition into any given physician's choice of patient care is ill-considered. Doctors should not have their choices for patient care either dictated or influenced by a politician's hunt for headlines. Senator Grassley should be smart enough to understand the unintended consequences of such an ill-considered act,” Center for Medicine in the Public Interest founder Peter Pitts wrote in an email.

John Kamp, executive director for the Coalition for Healthcare Communication, agrees, and told MM&M in an email that the Senator is off course. “I'd prefer he would help patients get the scrips they need for critical care . .. Good prescribing habits coupled with patient adherence would do more for the public health than these nasty accusations that cast shadows over health care professionals" he wrote.  Read More & Comment...

Benefit/Risk Management

01/24/2012 09:37 AM |

The need for better FDA risk/benefit methodologies isn’t just a concern for pharmaceutical companies – it’s a clinical issue for physicians.

According to a study in Medical Care (Dusetzina SB, et al "Impact of FDA drug risk communications on healthcare utilization and health behaviors: A systematic review" Med Care 2011), the attention that practicing physicians pay to FDA notices about drug risks appears is highly variable.

A literature review showed that serious adverse event warnings were the most heeded of all FDA communications, but, according co-author Caleb Alexander, MD, of the University of Chicago, responses varied.

And the cause wasn’t practice variation.

"We will need a better understanding of how to make [FDA risk communications] work, and where they can go wrong," Alexander said in a statement. "And we will need more and better studies of the successes and failures of this process."

Notices about use of drugs for certain populations tended to spill over into others, the authors reported. For instance, a warning about antidepressant use in children and adolescents led to declines in their use among adults as well.

"As with other public policies, FDA communications have the potential for unintended consequences.”

All the more reason to stop talking about "risk management" and start referring to "benefit/risk mangement."

The authors called for more research on factors associated with fast and sustained responses to risk communications, and continued work on characterizing the effects of advisories and warnings on a variety of behaviors "to enhance the science of risk communication regarding prescription drugs."

But it’s not just a problem for prescribers. A related issue is how well patients understand the risk/benefit equation of the medicines they are prescribed – and the answer is likely to be “very little.”

Why is this important? According to a Harris poll, more than one-third of Americans surveyed decided not to take a prescribed medicine because of safety concerns about risks and more than a quarter chose not to fill a prescription at all over safety concern.

Another reason to support benefit/risk management.

Per Gretchen S. Dieck, Ph.D., senior vice president, Safety and Risk Management for Pfizer, “… surveys of practicing physicians, health policy experts and the general public have revealed a lack of understanding of the fundamental safety science, processes and terminology.  This can contribute to misinformed decisions by patients about treatment options, which can lead to sub-optimal health outcomes.”

Also 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 physicians about what is or is not “on-label.”

The responsibility of risk is shared.  It must be more than what the FDA expects from industry and more than what industry expects from the FDA.  It's what all parties to the public health conversation must expect from themselves.

The responsibility of risk means appreciating and actualizing the philosophy of the safe use of drugs. For example, the responsibility of risk means not just detailing—but detailing the label.

At a February 2009 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.

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

The remarks of Pam Budny, a regulatory affairs manager at Eli Lilly were of particular interest.

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.

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

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

The importance of clear and concise and usable patient and physician medical information is recognized but more needs to done to address it. In September 2011, the FDA reorganized the Patient Labeling Team (PLT), moving it from the Office of Surveillance and Epidemiology (OSE), Division of Risk Management (DRISK) to OMPI, Division of Medical Policy Programs. That was a good move. The goal of the Patient Labeling Team is to promote the safe and effective use of prescription medications by providing accurate and easily understood patient medication information. Both physician and patient education should begin with the drug label.

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.

Janet Woodcock: “Only through coordinated interventions across all sectors of the health care system can we substantially reduce preventable injuries from using medications. All participants in the health care community have a role to play in reducing the risks and preventing injuries from medication use.”

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

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Dark Shadows

01/23/2012 08:52 AM |

An important and informed opinion from CMPI Chairman, Dr. Michael Weber.

THE PHYSICIANS SUNSHINE ACT:  MORE DARKNESS THAN LIGHT

The New Year brings in its wake the Physicians Sunshine Act and its requirement that pharmaceutical and device companies document all their payments to doctors, medical practices and teaching hospitals so that, starting in 2013, these costs can be listed by the Center for Medicare and Medicaid Services on a public website.  This action will result in unintended (but not unforeseeable) consequences on 21^st century medical progress.

The Sunshine rules are unclear and complicated – a bad combination. Not only will direct compensation to doctors be reported, but also all other costs of industry-supported physician-related research, consulting and education. 

Why this drastic and invasive step?   Follow the money.  Sunshine supporters hope that physicians, seeing their compensation made public, will curtail their interactions with industry -- thus limiting the development and use of “expensive” new products.

Understandably, government and private health insurers want to keep costs stable.  And, with prescription co-pays being patients’ most visible expense, they might find this action appealing -- for a while – until they see how innovation and new treatments are seriously delayed.

Collaborations between physicians and industry are fundamental to advancing medicine.  Academic physicians have the experience to identify unmet clinical needs, advise on potential new treatments, design and conduct innovative research and, through publications and teaching, bring key information to colleagues and patients. Without in any way diminishing the governmental and non-profit agencies that support research, it is the partnership between physicians and industry that has created many, if not most, of the major medical breakthroughs that have reduced the rates of death and other serious outcomes in recent years – as any literature search of major medical journals will quickly confirm. 

Conducting clinical research has become so rigorous and sophisticated that those of us who serve as consultants and investigators to industry recognize it as a major commitment or even a primary career path.  In addition, as studies are completed, physician researchers add to their professional commitment by becoming the teachers of this new information.

Critics of these collaborations see compensation of physicians as evidence of undue influence on medical practice.  But doctors, like all professionals, should be paid fairly for their time and work.  In reality, industry is now so conscious of not appearing to overpay doctors that they employ stringent “fair value” scales to set fees for research, consulting and teaching activities.  The truth is that physicians who are busy with these activities are not as well rewarded as their fellow specialists in full-time clinical practice.  Adding to this remunerative divergence is that a key part of research, writing articles for publication, takes weeks of work -- often unpaid so as to avoid any suggestion of bias.

The Sunshine Act will create troubling misconceptions for and about physicians.  Payments reported for physicians by industry will likely include funding they didn’t personally receive nor will they take into account costs incurred by these physicians in paying their own staff and covering overhead expenses.  Doctors involved in industry-supported research and education may easily get discouraged and frustrated explaining these complexities to an audience already biased and sated by sensationalistic media reports of physicians “on the take.”   

And yet the Sunshine Act, paradoxically, could have a positive effect.  Despite the near impossibility of reliably interpreting all the reported data, this information might well serve as a yardstick of cooperative achievement --identifying those physicians at the forefront of medical innovation.

Weber, a former president of the American Society of Hypertension is Editor-In-Chief of the Journal of Clinical Hypertension, professor of medicine at SUNY Downstate College of Medicine, Chairman of the Center for Medicine in the Public Interest, and a member of the Association of Clinical Researchers and Educators. 

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Patient Centered Outcomes Research Institute: So Yesterday, So Useless

01/22/2012 12:54 PM |

Last week life technologies announced it was launching the Benchtop Ion Proton Sequencer that will sequence an individual’s entire genome in less than 24 hours and for $99 a chip.   

This week the Patient Centered Outcomes Research Institute announced national priorities for comparative effectiveness research (CER).  Those ‘priorities’ included: spending money on comparative effectiveness research, spending money on communicating CER and spending money on learning how to carry out CER.  Note that no money is spent on comparing whether CER research produces better outcomes than what doctors usually do or genome informed medicine.   

Last week Life Technologies announced it was developing open-source software that doctors and patients can use to diagnose and select treatments. 

This week PCORI was discussing the idea of using a graphic comic book to explain the value of CER to consumers.  Really. 

Last week life Technologies and Eric Topol discussed how smartphones that combine genomic information with real time monitoring of illnesses (in 3-D) would be used to diagnose disease and monitor individualized response in real time in 1-2 years.

This week PCORI said that its national priority list is not final and it will take 1-2 years to come up with something final.    Carol Clancy, director of the Agency of Healthcare Research and Quality (AHRQ), said,  “We’re on a journey… (And) the process is at least as important as the final output or outcome, and it’s going to be an ongoing iterative process.”    Since AHRQ gets a 20 percent cut of what PCORI – a non-profit organization – raises through a surcharge on everyone’s health premiums (nearly a billion over ten years) no questions asked, I am sure Clancy hopes the journey is…iterative.

In five years Life Technologies will be able to sequence my genome in a couple of hours and for less than $100.    In five years, PCORI will have produced no original comparative studies but will have spent an average of $3 million on such research and nearly a billion dollars

It is already cheaper to sequence the genome of 1 million Americans than it is to for either AHRQ or PCORI to conduct reviews of old research.  And in five years Life Technologies will be able to sequence 100 million people for what PCORI will spend on studies that will be outdated by the time they are published and will still not be useful to guide individual treatment decisions. 

So why aren’t groups that are supposed to care about the disconnect between what entrepreneurs are producing to personalize medicine and what PCORI is babbling about at it’s endless series of meetings?  For instance, The National Pharmaceutical Council (NPC) is holding a conference on something called “Asymmetry in the Ability to Communicate CER Findings: Ethics and Issues for Informed Decision Making.”

http://www.npcnow.org/Public/Education___Events/Events/2012_events/Asymmetry_2012/asym12.aspx

I have no clue what asymmetrical abilities are.   The NPC brochure for the conference states:  “With billions of dollars being invested in comparative effectiveness research (CER), the good news is that the public will have increased access to health information in coming years. Yet as more and more information is communicated to patients, practitioners and payers, current regulatory policy may create asymmetries, or inequalities, in the ability of different stakeholders to convey information.” 

Now does everybody know what an asymmetry among stakeholders is?  Here’s a translation: how can stakeholders – apart from the usual suspects and the sales reps AHRQ is paying to peddle CER to doctors -- get a cut of PCORI dough?

NPC’s medical chief Robert Dubois claims: "CER has great potential to improve medical decision-making and as a result our overall health. However, the detail of how we implement and use CER is critical to achieving this goal."   http://www.sacbee.com/2012/01/20/4202757/national-pharmaceutical-council.html

It is clear that NPC doesn’t care whether PCORI and CER will impede the use and adoption of individualized genomic and clinical information.  It will not challenge PCORI raking in and spending $3 billion on the policy equivalent of shipping milk that is already past it’s expiration date. FDA or equal access to personalized medicine or not spent at all?  Why hasn’t one stakeholder (that’s Beltwayspeak for not criticizing dumb ideas for fear of not having a seat at “the table”) had the guts to ask if the money shouldn’t be spent on the FDA or making sure there is equal access to individualized medical information?

The Life Technologies breakthrough demonstrates the future is already here.   Too bad ‘stakeholders’ are propping up PCORI and pursuing 'asymmetries' instead of promoting personalized medicine.
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Caveat Emptor

01/20/2012 08:40 AM |

Christopher Viehbacher, chief executive of Sanofi and chairman of PhRMA on PDUFA reauthorization:

"To make sure we're not ignoring unmet needs in primary care, we need a lot more clarity around the risk-benefit so there's predictability when we invest in these products.”

Predictability is the philosophical core of PDUFA.  And risk/benefit is at the heart of the FDA process.

To make PDUFA V a public health victory, the most important thing is to remove (or at best minimize) the politics.

Easier said than done?  Perhaps. 

“PDUFA without the Politics” speaks to the issues inside PDUFA minus the slings and arrows of partisan politics.

But judge for yourself. Click here for the full paper.

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