Fake Avastin In Drug Supply

02/15/2012 07:10 PM |

Remember all those politicians and consumer 'advocates' pushing for the importation of drugs?  Where are they now in light of the flood of fake Avastin pouring into the United States:

http://www.reuters.com/article/2012/02/14/roche-avastin-idUSL2E8DEIYC20120214

Counterfeits of Roche cancer drug found in US

* FDA investigating counterfeit Avastin

* Bogus Avastin label in French, says Roche not Genentech

* FDA has informed 19 medical practices about counterfeits

Feb 14 (Reuters) - Counterfeit versions of Roche's multi-billion cancer drug Avastin have been distributed in the United States, the Swiss drugmaker and its U.S. biotech unit Genentech said on Tuesday.

Roche was contacted about the bogus Avastin by a health authority outside the United States and was informed that the counterfeit drug in the United States came from another country, the company said but declined to divulge which country.

"We are working with the FDA and law enforcement to aid their evaluations, determine the source of the counterfeit drug, and prevent its further distribution," Roche and Genentech said in a statement. "The counterfeit product is not safe or effective and should not be used."

The U.S. Food and Drug Administration is taking the lead on the evaluation, a Genetech spokeswoman said.

Genentech said there is an ongoing investigation by national health authorities but could provide no further information at this time.

The FDA said it has sent letters to 19 medical practices informing them about counterfeit 400mg/16ml doses of Avastin.

The company does not yet know just how much counterfeit Avastin is out in the market. Avastin is given intravenously.

But there are several obvious differences in packaging and label that should allow doctors to easily spot the bogus drug.

Among them genuine Avastin, known chemically as bevacizumab, has Genentech on the label, which is all in English. The counterfeit says Roche and the label is in French.

Lot numbers of actual Avastin are comprised of six digits with no letters, while the counterfeit lot number begins with a letter. And the counterfeit bottles of Avastin are missing information on the label, such as "for intravenous use."  Read More & Comment...

Discount!

02/15/2012 02:02 AM |

Progressive Insurance has Flo.  Progressive approvals have Janet.

The Pink Sheet reports that the FDA wants to make a better case for increasing the use of accelerated approval, potentially as an alternative to implementing a new expedited approval mechanism.

In addition to saving the agency the inevitable headaches associated with implementing a new pathway, it also could quiet discussions about placing a new “Progressive” and/or “Exceptional” approval route into the agency toolbox. Draft legislation outlining both has been circulating among industry and congressional circles for several weeks.

The progressive approval concept would allow a drug to be marketed if evidence is submitted for a candidate that is likely to predict clinical benefit for a designated population and use. Exceptional approval would be allowed using an alternative showing if the data needed couldn’t be generated ethically or feasibly, according to the draft legislation.

On the January 15^th edition of “BioCentury This Week” Center for Drug Evaluation and Research Director Janet Woodcock said that, while there are differing views on what progressive approval entails, the consensus is that no new approval pathway should lower the standards for safety and efficacy.

Woodcock also said accelerated approval is not realizing its full potential as a vehicle for qualifying drug candidates. She said it could be used more often and more consistently.

It would seem to be making the case that increased use of accelerated approval could be a viable alternative to instituting the two new approval schemes.

“[Accelerated approval has] been limited by its use, it’s not limited technically,” Woodcock said. “I think FDA could go a long way to [expanding the pathway’s use] with new guidance and new policies. I believe currently what’s codified in fast-track regulation is a little bit confusing about accelerated approval. So that could be clarified as well.”

Woodcock said more internal and external guidance on accelerated approval, such as provisions for the use of intermediate clinical endpoints, would help increase interest.

Woodcock said the concept of “staged approval” is feasible, but also potentially problematic.

“If you’re really going to have less evidence you've got to have some quid pro quo on the other side,” she said. “You can’t just toss it over the wall to the market and say go at it. And REMS doesn’t work in this situation because REMS is about a known safety problem.”

FDA also could speed up its approval of potentially breakthrough drugs by considering the best course of action early in the process, Woodcock said.

At the point a dramatic treatment effect is discovered, whether it is in a small population or any development phase, she suggested everyone “ought to all stop, take a deep breath and figure out how can we evaluate that drug as rapidly as possible.”

She said in some cases years could be cut off a drug’s development period if the larger treatment effect is verified.

“Even at that point, if it’s real, that’s a drug that offers something that no other drug’s offered before in that disease,” Woodcock said. “So you have to think how do we verify if that is real or not and then how do we evaluate the safety profile in the most efficient way possible?”

How, indeed.

Did somebody say “molecular diagnostics?” Did somebody say “personalized medicine?”
   Read More & Comment...

Feet First

02/14/2012 09:10 AM |

Yesterday I wrote in the Washington Examiner that, “A Grand Canyon exists between reality and hope in our new age of personalized medicine. Today, former FDA Commissioner Andy Von Eschenbach writes in the Wall Street Journal that, ”Breakthrough technologies deserve a breakthrough in the way the FDA evaluates them

Von Eschenbach points to a troubling statistic that, according to the Tufts Center for the Study of Drug Development clinical trials from 2003-2006 were nearly 70% longer than those from 1999-2002. “Longer (and more complicated) trials have led to skyrocketing drug-development costs. High costs discourage investment in much-needed new therapies for conditions like obesity, diabetes and heart disease.”

Is this regulatory leadership? That’s debatable. Not according to a new California Biomedical Industry study that reports about 80% of life sciences CEOs surveyed don’t believe the FDA regulatory approval process "is the best in the world." Of equal import is that 81% of those surveyed believe that "within five years, another country could conceivably recreate the ecosystem that has made the U.S. the leading biomedical region in the world.” Investors talk with their feet.

Andy writes that, “Other countries such as Israel, Singapore and China are already preparing to leapfrog the U.S. for leadership of the global life-sciences industry.”

“Preparing” is one thing, doing it is another.  But it is a real threat that we ignore at our own peril.  As Sanofi CEO Chris Viehbacher said during  his closing remarks at last year’s annual PhRMA meeting, “The question isn’t will our companies be successful? It’s will they continue to be successful in the United States?”

In January of 2010, I was part of a group of a group government regulators, health care policy experts, industry leaders, health economists, health care attorneys, patient advocates, and academics convened to study and offer new solutions to the hurdles to patient access and to develop high-impact global solutions.

(The report from this meeting, “Expediting Patient Access to New Medicines: A Call to Action,” was published in Drug Information Journal and can be found here.)

One of the ideas that discussed was for a new Asia-Pacific pan-regional regulatory agency that would provide centralized regulatory support outside of North America, Japan, and Europe. The goal would be to create an agency with sufficient resources and scale to accelerate drug approval in a region for which drugs are not usually designed. We suggested that the first embodiment of the idea could be an Asia-Pacific regulatory agency in Singapore that serves Australasia and Asia (ex-Japan). The creation of an additional new agency (similar in scale to the FDA and EMA) would continue to drive regulatory excellence but, more importantly, serve the needs of geographies that are underserved or dependent on guidance from the developed countries. This would encourage biopharmaceutical companies to invest in innovative medicines that may have a different regulatory pathway to approval, resulting in faster access to critical medicines in emerging markets.

Game on.

View PDF Here  Read More & Comment...

Andy von Eschenbach on FDA Reform and Innovation

02/14/2012 10:55 AM |

Andrew von Eschenbach has an excellent piece in the WSJ today about how FDA reform could support innovation. 

http://tinyurl.com/83emomv

Andy is a visionary who transformed the National Cancer Institute and then went on to become fine FDA Commissioner under extremely difficult circumstances.  He will be chairing MI's Project FDA and we wish him and MI the best of luck in this important endeavor!

As an historical footnote, I was chair of MI's 21st Century FDA Task Force from 2004-6.  Peter and I wrote the Task Force  report "Prescription for Progress: The Critical Path To Drug Development", which you can find here along with links to articles about Andy's participation in the report's release:

http://www.manhattan-institute.org/html/fda_task_1_members.htm  Read More & Comment...

A New Progressive Era in Personalized Medicine

02/13/2012 08:15 AM |

From today's edition of the Washington Examiner:

America needs more breakthrough “progressive” medicines

A recent global cancer conference was abuzz with discussion of a potential breakthrough therapy in the treatment of Alveolar Soft Part Sarcoma, a rare form of cancer afflicting only about 100 patients per year in the U.S.

If ASPS is not eliminated through surgery, it is always fatal. A new therapy for ASPS, called cediranib, was found in early testing to melt away the tumors in up to 70 percent of treated patients.

Yet there is no development pathway for FDA approval for such small populations of patients, so the world's next Gleevec lies on a shelf, with fingers crossed that years from now, after larger, longer clinical trials in higher-prevalence cancers are performed with cediranib, ASPS patients might get access through indirect means.

Simply put, at the FDA, there is no official process for the approval of a product developed to attack a condition afflicting just 100 patients. A Grand Canyon exists between reality and hope in our new age of personalized medicine.

Enter Sen. Kay Hagan, D- NC. As a member of the Senate Health, Education, Labor and Pensions Committee, she has proposed legislation known as the TREAT Act, which calls for FDA reform that would fast-track drugs that show promising early data in treating deadly diseases.

The bill is an acknowledgement of the failings of the 1990's accelerated approval regulations, which rely on "surrogate endpoints" for a clinical trial to represent and reflect the endpoint that is usually used for a drug's approval.

The result is an anemic armamentarium for our nation's number-one killer, and completely empty medicine chests for many rare cancer types.

The TREAT Act reforms the accelerated approval pathway to allow state-of-the-art clinical endpoints that are "reasonably likely to predict clinical benefit" as the basis for approval for life-threatening diseases.

For cancer, this change could allow a return to the notion of simple, universal measures such as significant tumor death, or delay in disease progression, as the basis for rapid release of new products to desperately ill patients. Additional data following confirmatory studies would then allow progression to full approval.

Safeguards are included in the bill to remove the product from the market should it not live up to its initial promise. Also, as a humanitarian gesture for micro-populations with deadly disease, there is the creation of an "Exceptional Approval" pathway.

This would authorize approvals when the usual data "cannot ethically, feasibly or practicably be generated." This pathway would be a game-changer for U.S. rare disease populations of extraordinarily small numbers.

Thanks to Hagan, there can be a new way forward for development of therapies not just for rare cancers, but for thousands of life-threatening rare diseases where there are no realistic opportunities for drug development. Such reforms go beyond helping only the forgotten orphans.

By fostering innovation in new arenas of drug development, jobs are created and businesses prosper, and innovation accelerates the boundaries of medical knowledge and patient possibilities.

Now is the time to embrace a 21st century FDA -- and progressive approvals is a good place to start.

Peter Pitts, a former FDA Associate Commissioner, is president of the Center for Medicine in the Public Interest. Dr. Mark Thornton, a former FDA Medical Officer, is president of the National Organization Against Rare Cancers and is employed in the biotechnology industry.

  Read More & Comment...

The High Cost of Innovation Is Killing People

02/10/2012 04:14 PM |

Matt Herper has a great piece on  how expensive it is to develop new drugs...

http://www.forbes.com/sites/matthewherper/2012/02/10/the-truly-staggering-cost-of-inventing-new-drugs/

I like how Herper compared the cost of developing small molecule meds to biologics.  What's more, he neatly shows that no matter how you slice it or estimate costs they are too high to be sustainable and to sustain the advances in health and life expectancy people have enjoyed over the past 50 years or so.   Herper writes:


Read  How to improve R&D productivity: the pharmaceutical industry's grand challenge by Steven M. Paul, Daniel S. Mytelka, Christopher T. Dunwiddie, Charles C. Persinger, Bernard H. Munos, Stacy R. Lindborg & Aaron L. SchachtNature Reviews Drug Discovery 9, 203-214 (March 2010)doi:10.1038/nrd3078 http://www.nature.com/nrd/journal/v9/n3/suppinfo/nrd3078.html

 Sir Michael Rawlins was warning about the crisis in R&D back in 2003:

  Read More & Comment...

Approaching Clarity on Biosimilarity

02/10/2012 08:31 AM |

FDA issued three draft guidance documents on biosimilar product development. These documents provide FDA’s current thinking on approaches to demonstrate that a proposed biological product is biosimilar to an FDA-approved biological product (the “reference product”) using the abbreviated pathway under section 351(k) of the Public Health Service Act (PHS Act).

The draft guidance documents are:

Scientific Considerations in Demonstrating Biosimilarity to a Reference Product
 

Quality Considerations in Demonstrating Biosimilarity to a Reference Protein Product

Biosimilars:  Questions and Answers Regarding Implementation of the Biologics Price Competition and Innovation Act of 2009

FDA is accepting public comment on these draft guidance documents. Instructions on how to submit comments will be announced in an upcoming Federal Register notice.

Some higlights:

FDA intends to use a risk-based totality-of-the-evidence approach to evaluate all available data and information submitted in support of the biosimilarity of the proposed product.  The type and amount of analyses and testing that will be sufficient to demonstrate biosimilarity will be determined on a product-specific basis.

FDA will be able to provide meaningful advice on the scope and extent of necessary animal and human testing after a thorough review of data from structural and functional analyses.  Additional animal and clinical studies should be tailored to address residual uncertainty regarding the biosimilarity between the two products to ensure such testing is appropriately targeted.

This draft guidance provides an overview of analytical factors that may be relevant to assessing whether a proposed therapeutic protein product and a reference product are highly similar, as required for a determination of biosimilarity under the BPCI Act.

The guidances discuss general scientific principles, including the importance of extensive analytical, physicochemical and biological characterization.

The type, nature, and extent of any differences between the proposed biosimilar product and the reference product, and the potential effect of any differences on the safety, purity, and potency of the proposed product should be clearly described and discussed by the sponsor in the 351(k) application. 

The agency recognizes that advances in analytical sciences and manufacturing technology, including integration of Quality by Design approaches, may facilitate a “fingerprint”-like analysis of therapeutic protein products, and thus may provide appropriate bases for a more selective and targeted approach to subsequent animal and/or clinical studies to support a demonstration of biosimilarity.

And a solid definition of what it means for a biological product to be “interchangeable”?

The agency defines  “interchangeable” biological product is biosimilar to the reference product, and can be expected to produce the same clinical result as the reference product in any given patient.  In addition, for a biological product that is administered more than once to an individual (as many biological products are), the risk in terms of safety or diminished efficacy of alternating or switching between use of the biological product and the reference product is not greater than the risk of using the reference product without such alternation or switch.  

No surprises.  But it's good to see it on paper.  So let it be written, so let it be done.

  Read More & Comment...

Contraception Edict and Obamacare

02/09/2012 12:46 PM |

http://spectator.org/archives/2012/02/09/the-contraception-edict-an-ass

The Contraception Edict: An Assault on Liberty


We're seeing Obamacare's future and how it works.

The Obama administration's decision to force religious institutions to cover contraception is a case study on how Obamacare and its implementation have politicized medical decisions. Here's how it works. First, health care choices are overruled if they do not flow from the state and do not require taking rights from one group to give to another in the name of fairness. Next, the same administrative apparatus engages in politicking and deal making to appease groups angered by the original decision.

The contraception mandate was issued unilaterally and without regard to the deep feeling and anger it would generate. Now the administration is rhetorically backpedaling and seeking to find a way to respond to the quickly spreading attacks on the action. Claims to the contrary, this was and is not about assuring all women have access to contraception. It was about eliminating the choices and overruling the preferences of one group to enforce the "rights" of another.

Now everyone is focusing on "accommodating" concerns that Obamacare would force employers and organizations to cover birth control. And in doing so, by turning an individual health choice into a government edict we must obey subject to Obamacare's equivalent of plenary indulgences -- a waiver -- we move one step away from liberty and closer to centralized control. That's why one idea for "accommodating" will never be implemented: 'Allowing' religious organizations and employers not to offer contraception as part of health insurance as long as they give employees who want it a choice of plans that do. That's because giving people choices of health plans also comes at the expense of enforcing everyone's "right" to medical care.

The contraception edict is but one of a series of Obamacare judgments that have angered millions of Americans because of its one size fits all nature. From mammograms, to coverage requirements, to end of life planning, the administration's actions rankle not because they are completely dumb (they usually are) but because they violate people's sense that such decisions should not be made by government and should not be based on meeting a political or policy goal.

The contraception decision is more disturbing than these previous enactments of Obamacare because freedom of religion is a deeply cherished freedom that allows us the liberty to establish a relationship with God, family, and lives in ways that government can never replace. As a result, more than any other action, the restriction of Catholic and other religious-sponsored organizations or employers to exercise choice reflects the belief that freedom of religion come at the expense of "reproductive rights of women" underscores how Obamacare regulators will restrict choice and access in the future. In this warped world, birth control medicines and devices are mandated and essential but not Avastin or genetic testing or treatments for cancer or rare diseases. Remember how the administration tried to make end of life planning a requirement? The inner logic of Obamacare is that life-saving therapies and choice inhibit the growth of the welfare state and the appropriate distribution of resources. It is the underlying rationale of the Independent Payment Advisory Board and the Patient Centered Outcomes Research Institute, which focus on "social decisions" rather than individual benefit, decisions that produce winners and losers based on a political calculus.

The action also exposes -- at the beginning of this presidential election season -- the fundamental view of liberty enshrined in Obamacare. Thomas Jefferson wrote "The God who gave us life, gave us liberty at the same time." Another political leader noted, "Liberty is precious. That is why it must be rationed." That was Lenin. You tell me which worldview shapes the contraception edict.  Read More & Comment...

The Sinews of Safety

02/09/2012 08:10 AM |

In his 1946 Sinews of Peace speech Winston Churchill famously remarked, “From Stettin in the Baltic to Trieste in the Adriatic an iron curtain has descended across the Continent.

Has an iron curtain today descended on drug safety and quality on many other continents?

Word has it a new WHO study reports that only 20% of member nations have drug regulatory capacity to properly ensure the safety of their national drug supplies.  Yikes.

To paraphrase the British Bulldog, the safety of the world’s drug supply, ladies and gentlemen, requires a new unity from which no nation should be permanently outcast.

It’s time to actively and aggressively pursue FDA Commissioner Peggy Hamburg’s call for a regulatory Marshall Plan to help build (nation-by-nation) global systems for both quality and safety.

Unarguably, two of the most important health advances of the past 200 years are public sanitation and a clean water supply. Those achievements helped to control as many public health scourges as medical interventions helped to eradicate them. A high tide floats all boats.

Working together to raise the regulatory performance of all nations will help all nations (even the 20% deemed “capable” by the WHO) to create sound foundations to address a multitude of quality and safety dilemmas such the manufacturing of biosimilars, the control of API and excipient quality, pharmacovigilance and, yes, even counterfeiting.

With scare resources, perhaps the best place to start is by committing to common standards for data capture and reporting. After all, knowledge is power.

Difficult? To be sure. But, as Winnie reminds us, “A pessimist sees the difficulty in every opportunity; an optimist sees the opportunity in every difficulty.”   Read More & Comment...

PDUFA in the Red Zone

02/07/2012 07:37 PM |

  Read More & Comment...

Bailing out drug shortages

02/07/2012 10:50 AM |

In Greece wise men speak and fools decide.
-- George Santayana

Poor Greece.

According to a new article in The Lancet:

The Greek Government's austerity drive has inadvertently triggered problems with the country's drug supply, causing shortages of hundreds of medicines.

But it’s not just Greece; drug shortages have been in the news in the United States and the EU. Also, in Saudi Arabia, some 2000 medicines are in short supply since 2008 and in Turkey some 350 medicines have been at risk of stock outs since arbitrary changes in pricing and reimbursement last November. 

Plainly speaking, artificially low prices cause drug shortages.

In the past few months, according to the PanHellenic Association of Pharmacists, 500 commonly used drugs are in short supply. Among them, there are drugs for hypertension, gastroenterological disorders, cancer, kidney diseases, and painkillers.

What’s causing Greek’s drug shortage problem? The same issue that causes it everywhere else – perverse market incentives.

In the United States, a government analysis of average sales prices showed that oncology sterile injectable drugs that experienced shortages since 2008 decreased in price from $56.17 per unit in Q1 2006 to $37.88 per unit in Q1 2011. Oncology sterile injectable drugs that have not experienced shortages have had relatively stable prices over this period.

Last year, the Greek government mandated lower drug prices to cut down its medical expenses. Medical expenses were €2·4 billion in 2004, €5·2 billion in 2009, and dropped to €1·65 billion in 2011, after the measure was taken.

However, the government's decision has fed the parallel trade market since
wholesalers prefer to sell their products in other countries where the profit is higher.

“Due to medicine shortage, the population's health is in great danger,” says Kostas Kouvaris, president of Pireaus Pharmacists Association. Theodore Abatzoglou president of the PanHellenic Association of Pharmacists points out that the recent government law for the prices of medicine has also caused hospitals to run out of drug stocks. And because of the extremely low prices set by the government, international companies are not interested in supplying Greek hospitals with medicine. Health institutes are now mainly being supplied by companies that sell generic drugs which, in many cases, represent older medical technologies.

But, generic drugs aren’t often what the doctor ordered.  Replacing an old generic drug for a modern innovator product may save a government money in the short term, but as patient outcomes worsen and hospitalization occurs, health costs skyrocket.

According to Greg Conko of the Competitive Enterprise Institute,

"Essentially all of the drug shortages that occur in the U.S. arise in the generics market, where profitability is fairly low … Compounding the problem is the fact that the 'buyers' of the drugs in question aren't patients but third-party payers -- insurance firms, pharmaceutical benefit managers, and government health programs -- all of which have a strong incentive to drive the prices paid for these products down as low as possible … So, for many of these drugs, the market can really only sustain a handful of manufacturers -- sometimes just one or two. So, when supply disruptions occur -- caused by a shortage of raw materials or more often a quality control problem in a manufacturing facility -- there aren't a lot of additional producers in the market to take up the slack.”

"The main cause of drug shortages is economic," says Mandy L. Gatesman, PharmD, of Virginia Commonwealth University in Richmond, and Thomas J. Smith, MD, of Johns Hopkins. "If manufacturers don't make enough profit, they won't make generic drugs.”

Let’s adopt a free-market solution and free physicians, pharmacists, and patients of the problem of needless drug shortages.

The government solution to a problem is usually as bad as the problem.              
-- Milton Friedman  Read More & Comment...

No personalized medicine please, we're British

02/06/2012 08:55 AM |

NICE has issued draft guidance recommending against the use of three breast cancer diagnostics to guide decisions about chemotherapy use in women with estrogen receptor-positive, lymph node-negative, HER2-negative breast cancer.

According to NICE, the tests aren’t cost effective because evidence for their clinical efficacy is "limited."

But isn’t that the whole idea of personalized medicine?  And “limited” for whom?

Molecular diagnostics are what make medicines personal. Diagnostics are how drugs can be made safer through safe use. They make the “four rights” (right medicine for the right patient in the right dose at the right time) possible. And the four rights lead to lower costs through better outcomes.

Studies show that testing for HER2 breast cancer delivers savings that are 65 times its cost. That reimbursement should be based on value rather than activity is an essential paradigm shift.

This message is finally reaching a mass audience. Consider the recent USA Today story, “Genetic testing boosts efficacy in cancer care,” which reports that “tailoring cancer therapies to fit a person’s genetic makeup could spare thousands of patients from harmful side effects and save millions of dollars a year.”

Even NICE said the test's incremental cost-effectiveness ratio (ICER) -- aka cost effectiveness ratio -- per quality-adjusted life year (QALY) was "dominant" compared to current practice -- but the lack of data on analytical reliability and clinical utility made a recommendation for widespread use unwarranted “at this time.”

In other words, no reimbursement until more data is available. Whether or not that’s a convenient excuse or a scientifically based belief –it’s the wrong decision to dissuade the use of a validated diagnostic tool that saves lives though (1) more personalized care and (2) saves money by not spending it where it isn’t going to work.

NICE noted that most studies were retrospective, which it said is associated with increased bias compared to prospective trials. Maybe so.  But tell that to the 20 percent of women whose lives are at risk.

Research shows, not surprisingly, that many breast cancer patients who might benefit from Herceptin are not receiving it, while some women on the drug had never been properly tested.

"Our review of the literature suggests that there are important knowledge gaps regarding the real world use of HER2 testing and trastuzumab," said Elena Elkin, a researcher at Memorial Sloan-Kettering Cancer Center in New York. "Filling these gaps may help optimize limited healthcare resources and improve care for women with breast cancer."

But how will data be collected if these tests aren’t being used in regular clinical practice? (And they won’t be without reimbursement.) Are these approved and validated tests to be relegated to the narrow confines of lengthy clinical trials? What message does this send to the developers of advanced diagnostics – and for those who invest it such enterprises? What message does it send to pharmaceutical companies about the future of high-risk adaptive clinical trials? What message does it send about the future of targeted therapeutics?

To borrow an over-used adjective from the world of global climate change – what does this say about our ability to protect "sustainable" innovation?

Most disturbingly, what message does NICE’s decision send about medical ethics in the age of personalized medicine? Is this but the latest malignant manifestation of choosing short-term savings over long-term results, of cost-based choices over patient-centric care? Skimping on a diagnostic test today but paying for an avoidable hospital stay later is a fool’s errand, pernicious to both the public purse and the public health. The implications for the healthcare debate in the United States are uncomfortably obvious.

And considering the white-hot debate over the Komen Foundation/Planned Parenthood imbroglio – where’s the anger and indignation?

More research?  By all means.  Better-designed research? Absolutely. But by denying reimbursement for HER-2 tests until this research is designed, fielded, studied, reported and debated many lives will be lost and many millions of dollars wasted.

  Read More & Comment...

Another fine mesh you’ve gotten us into

02/03/2012 11:04 AM |

U.S. regulators should be able to block medical devices based on past products with safety issues, said House Democrats led by Representative Edward Markey of Massachusetts.

Current law requires the FDA to clear a device for sale if it’s “substantially equivalent” to a past device, known as a predicate. Markey’s bill would let the agency say no if the predicate, or past predicates in a series of equivalent devices, were removed from the market or if the agency were in the process of pulling it. It also would require companies to explain why their products are different from recalled versions.

The legislation also requires the FDA to review previously cleared products to determine whether any have recalls in their “device lineage.” A report would be due to Congress within three years.

Manufacturers “share Congressman Markey’s interest in ensuring the safety and effectiveness of medical devices,” said Wonderful Wanda Moebius, of the Advanced Medical Technology Association. “However, we believe FDA already has abundant authority to carry out its mandate and the burdensome provisions” in the bill “will not contribute to patient safety.”

In another piece of proposed legislation, U.S. Senators Bob Casey and John McCain introduced a bill that they said would streamline approvals. The legislation would ease the “burdensome review” the FDA requires to see if a new product is similar to one already on the market, the two men said in a statement.

“The FDA’s current approval process for medical devices can be cumbersome, inconsistent and far too lengthy for patients who need innovative technologies and for the companies that develop them,‘‘ said McCain, an Arizona Republican. Casey, a Democrat, is from Pennsylvania.

The bill would also address safety issues by ensuring that medical devices on the market before current safety rules existed are properly classified in a timely manner.  Read More & Comment...

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

  Read More & Comment...

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.).

  Read More & Comment...

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...