Asked and Answered

07/14/2011 11:08 AM |

A Drugwonks reader writes (relative to the recent Pew report on FDA's need for more authority for overseas inspections and related matters):

There is another answer here.  Section 801 of the FDCA, which has been revised recently to reflect the global reality, establishes an algorithm for FDA review of imports that priorities scarce resources according to product type, existence (or not) of registration, and other factors.  The provision even addresses importation in cases of medical emergency and personal-use importation.  It reflects careful attention from Congress – and makes clear that products originating ex-US get heightened scrutiny precisely because they’re not coming from inspected facilities.  Asked and answered, people.  Or should I say “by the people.”
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Drug Importation? Pew!

07/14/2011 10:34 AM |

Did somebody say “drug importation?”

Didn’t think so.

According to the Pew Health Group, the FDA needs much more power to protect the U.S. supply of drugs as more and more are made in other countries.

 

The new study found that increased outsourcing of manufacturing, a complex and globalized supply chain, and criminals all help to create the potential for counterfeit or substandard drugs to reach patients.

 

Well, duh – but important to regularly reinforce.

 

 “It is clear the FDA was set up to deal with a domestic industry,” Allan Coukell, the director of medical programs at Pew Health Group, told National Journal. “But drugs are increasingly manufactured globally and are outside of the oversight of the FDA. There is a real need to update legislation to reflect the realities of the industry.”

 

The FDA is bound by a 1938 law that only gives the agency the authority to inspect products manufactured in the United States. “There’s only so much the FDA can do under the current law,” FDA Office of Compliance Director Deborah Autor said in a statement.  

 

As Peggy Hamburg said at a recent meeting of the Council on Foreign Relations:

* The new reality of food and drug regulation is that it’s global. In fact, it should be a topic for conversation at the next meeting of the G20.

* The recent crises in both food and drug safety will only repeat themselves unless regulatory agencies from around the world work in closer and more regular partnership.

* There is a responsibility on the part of the FDA and other more developed regulatory agencies around the world to help build “regulatory capacity” for those nation’s that want and need assistance.

* Part of a closer working relationship means a more regular and robust sharing of global intelligence on issues of counterfeiting.

* And lastly, “We can’t inspect our way out of this problem."

All good things – progressive things -- but, short of a regulatory Marshall Plan, things that will have to rely (at least initially) on personal relationships between senior officials at various regulatory agencies and a focus on what’s best for global public health writ large is convergent with what’s best for any given nation.

It’s not as easy as it sounds.

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Deck Chairs or Decisiveness?

07/13/2011 05:43 PM |

And now a message from Peggy Hamburg:

Dear Colleagues,

I am writing today to let you know about some changes that I will be making to the agency’s management structure. As you probably recall, back in January, I told you that I was initiating a review of the Office of the Commissioner.  As I explained at that time, this review was driven by the expanding and rapidly changing nature of the Agency’s responsibilities, and the need for a management structure that reflects these changes and best supports your efforts. 

I consulted with former Commissioners, as well as with HHS Secretary Sebelius, and considered many options before arriving at the structure that I am announcing today.

The most important thing driving my consideration of this is the changing nature of both the Agency and the job of Commissioner.

Today, the Agency faces several key challenges: 

First, we are a very large agency, with an incredibly broad span of responsibility.  We regulate products that account for between 20 and 25 percent of every consumer dollar spent in the U.S. and that total more than a trillion dollars annually.  For the most part, these are products that people rely on in fundamental ways every day.

Second, as technology and science continue to evolve, we are faced with the challenge of making sure that new ideas translate into the products and opportunities that people need and count on to protect their health.   Innovative products that are truly transformative create unique scientific and regulatory challenges, and FDA must be a consistently powerful catalyst for innovation.

Third, we have seen the dramatic transformation of globalization – more products, more countries, more access by consumers and companies to global supplies – and this presents an enormous challenge to FDA in ensuring the safety and quality of the products we regulate.

Finally, we continue to be faced with administrative challenges.  In these difficult economic times, our agency’s budget requires constant attention.  And, simply providing the support and services for our 12,000 plus employees – everything from phones to IT to office space on our beautiful, growing White Oak campus – is a daunting job.

I take very seriously my responsibility to lead FDA along a path that will meet these challenges.  One crucial part of this responsibility is to create a structure in the Commissioner’s Office that best supports your efforts and reflects the changing nature of the Agency. 

The structure of the Office of the Commissioner that I inherited was created in 1970, when the FDA consisted of three Centers and a field office.  By 2011, we had grown to seven Centers, and a Commissioner’s Office with more than 1,600 staff.  Over the years, as Congress created new programs that cut across Center responsibilities, those programs were placed by default in the Office of the Commissioner. 

The new organizational alignments more accurately reflect the agency’s responsibilities, subject matter expertise and mandates in an ever more complex world, where products and services do not fit into a single category.

Let me begin by saying that, for most of the FDA, this organizational alignment will likely not have a significant impact on you or your day-to-day work. 

The most obvious change you will see is that the Agency’s programs, in terms of a reporting chain to me, will be divided into “directorates” that reflect the core functions and responsibilities of the Agency. This new management structure will enable the Office of the Commissioner to better support the agency’s core scientific and regulatory functions, and help tie together programs that share regulatory and scientific foundations.  I will rely on the leadership of these directorates to help provide the necessary direction and coordination needed by an Agency of this scope.

I am establishing a new Deputy Commissioner for Medical Products and Tobacco, who will provide high-level coordination and leadership across the Centers for drug, biologics, medical devices, and tobacco products.  The Centers will, of course, remain as discrete management entities under their current expert leadership.  In addition to this strategic role with the Centers, this position will oversee our Special Medical programs. 

I am pleased to announce that Dr. Steven Spielberg, former Dean of Dartmouth Medical School and currently Director of the Center for Personalized Medicine and Therapeutic Innovation at Children’s Mercy Hospital in Kansas City, has accepted this position. In this role, Dr. Spielberg will serve as both advocate and a support for Center Directors in their important work for FDA.  

 I will also be creating a directorate focused on grappling with the truly global nature of today’s world -- food and drug production and supply, as well as the science that undergirds the products we regulate -- so that the FDA can move from being a regulator of domestic products to one overseeing a worldwide enterprise.  

To oversee this transformation, I have asked Deborah Autor, now Director of CDER’s Office of Compliance, to take on the role of Deputy Commissioner for Global Regulatory Operations and Policy.  In this position, Deb will provide broad direction and support to the Office of Regulatory Affairs and to the Office of International Programs, with a mandate from me to make response to the challenges of globalization and import safety a top priority in the years to come. Dr. Murray Lumpkin, who has served with dedication and accomplishment as Deputy Commissioner for International Programs and Director of the Office of International Programs, will take on a new role as Senior Advisor and Representative for Global Issues.  In this role, he will be charged primarily with special projects that draw on his expertise working with counterpart regulatory agencies on issues of global regulatory harmonization, governance and capacity-building.

The third directorate is the previously established Office of Foods, which we created to make our oversight of FDA’s food and feed program a more seamless enterprise. That task is even more important today as Mike Taylor leads the implementation of the Food Safety Modernization Act.

The fourth directorate will be a new Office of Operations, headed by a Chief Operating Officer.  The COO will oversee the agency’s administrative functions, such as human resources, facilities, information technology, finance, and other activities that provide support to your organizations.  Within this Office, I am bringing the budget formulation and budget execution functions together under a CFO position.  We have initiated a search to fill the Chief Operating Officer position.

The Office of the Chief Scientist, charged with our important efforts to improve FDA’s science and address issues of cross-cutting scientific concern, will continue to do so. The National Center for Toxicological Research will report to the Chief Scientist, Dr. Jesse Goodman, and, like the other Centers, will remain a discrete management entity within this new directorate model.

Within the new, smaller, immediate office of the Commissioner, John Taylor will remain as Counselor and will have the additional responsibility to oversee the policy and planning functions, the Office of Legislation, and the Office of External Affairs. I want to thank John for serving as acting Principal Deputy these past months, in addition to his duties as Counselor.  He has tirelessly supported me and the Agency with enthusiasm, energy, expertise, and good humor.

You can find revised organizational charts, reflecting this realignment here. In addition, I will share a video message of this announcement shortly.  Your managers will be available to answer any questions you might have in the coming days.

In closing, I want to take a moment to thank you so much for all that you do.  FDA is an extraordinary place, with so many highly-dedicated professionals and support staff who are committed to promoting and protecting public health. You accomplish a tremendous amount every day and I am grateful for all of your work. These organizational changes are intended to help further your important work and the mission of this remarkable Agency.

Sincerely,

Margaret A. Hamburg, M.D.

Commissioner of Food and Drugs

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Avastingate Redux

07/13/2011 01:00 PM |

A first test of whether the drive to require larger numbers of patients enrolled in clinical trials to measure overall survival as an endpoint (rather than progression free survival) in the wake of FDA's Avastingate is due this week:

"Seattle Genetics Inc. (SGEN) and Takeda Pharmaceutical Co.’s drug brentuximab for Hodgkin’s lymphoma and a less common type of the disease may require more data on benefits compared with treatments already on the market, U.S. regulators said.

The Food and Drug Administration is trying to determine whether the experimental treatment given the trade name Adcetris should receive accelerated approval in an agency staff report released today. An FDA panel of outside advisers on July 14 will weigh applications for the medicine to treat anaplastic large cell lymphoma and Hodgkin’s lymphoma.

The agency is seeking more patient data to be able to weigh more clearly the drug’s benefits. The FDA may decide whether to approve by Aug. 30. The experimental treatment could generate peak sales of $850 million in 2020, according to a note last month from Rachel McMinn, a research analyst with Bank of America Merrill Lynch.

“Small size limits the benefit-risk analysis,” the FDA said in questions to outside advisers released with its report. “For this application, consideration for accelerated approval would be consistent with regulatory actions taken in the past decade for similar hematology applications based on single arm clinical trials.”

We know what that might mean for cancer patients in Pazdur-land. 

Meanwhile as I blogged a couple of weeks ago, the Kevorkian Center for FDA Reform run by Harvard's resident anti-innovation scold Jerry "Use My Academic Detailing Without Evidence of Improved Outcomes" Avorn is pushing for longer and larger studies that would run small firms like Seattle Genetics into bankruptcy...

You can read Avorn and his colleague Aaron Kesselheim's blueprint to ration innovations by expanding clinical trials here:

Characteristics of Clinical Trials to Support Approval of Orphan ...

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Liz and other biz

07/13/2011 09:26 AM |

As reporter in BioCentury, HHS will appoint Elizabeth Dickinson as acting FDA chief counsel, effective Aug. 8, according to an internal memo from acting HHS General Counsel William Schultz. Dickinson, who has been at FDA's Office of Chief Counsel since 1994, will replace Ralph Tyler, who is departing. The memo noted that HHS plans to convert the chief counsel position into a career job; currently, it is filled via political appointment.

And on a more therapeutically personalized note, FDA released draft guidance on the development of in vitro companion diagnostics. The document clarifies FDA's definition of a companion diagnostic, reiterates the agency's intention to conduct simultaneous reviews of a product with its companion diagnostic, and identifies situations where the agency may approve a targeted product in the absence of an approved companion diagnostic. However, the guidance does not address the design or conduct of clinical trials of products in combination with companion diagnostics.

FDA defines a companion diagnostic as a device that provides information essential for the safe and effective use of a corresponding therapeutic. The agency added that the definition does not include clinical laboratory tests intended to provide "useful" information where that information is not a determining factor in the safe and effective use of a therapeutic.

The guidance notes that product labeling should only identify a type of approved companion diagnostic device, rather than a specific manufacturer's device. FDA said this will facilitate the development and use of more than one approved diagnostic of the type described in the companion product's label. Comments are due 60 days after publication in the Federal Register, which is expected Thursday.

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Innovation Reborn

07/12/2011 03:34 PM |

Janet Woodcock gave an excellent overview of the resurgence in novel treatments getting the FDA approval.   The years of effort to validate drug targets based on disease mechanisms is now yielding important, first in class products are paying off.  There is still a lot of room for increased productivity, particularly in the area of orphan drug development and molecular diagnostics.  But it is clear that the model of development Dr. Woodcock envisioned when she lead the Critical Path Initiative is paying off.

Imagine what industry and the FDA could do if it expanded on that model by creating more Critical Path Institutes, for example. 

And imagine how these breakthrough products will languish if CER holds up or delays access. 

You can read Dr. Woodcock's testimony here:

Testimony of Dr. Janet Woodcock, July 7, 2011
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Taking a shot at misinformation

07/12/2011 08:07 AM |

From the op-ed pages of the Baltimore Sun:

Vaccine safety: Misinformation about vaccine risks is making us less safe

By Sandeep Rao

Last month, the Maryland Department of Health and Mental Hygiene reported the first case of measles in the state since 2009.

This development demonstrates that even Maryland, which has one of the highest vaccination rates in the U.S., is not immune to a larger trend facing the nation. This past year, the U.S. has seen the largest increase in measles cases in almost two decades, according to the U.S. Centers for Disease Control and Prevention. The rise in measles cases over the first half of this year is double the rate typically seen compared with previous years.

Most of those diagnosed with the disease did not receive the measles, mumps, and rubella (MMR) vaccine.

In the U.S., most children receive the MMR vaccine series by age 2. Of those patients diagnosed with measles, most survive; however, fatal brain and lung complications can occur.

A survey released earlier this month by the CDC showed almost 80 percent of parents are uncomfortable with the concept of childhood vaccination. Among the reasons provided, roughly 30 percent cited the potential for learning disabilities, such as autism, for their hesitation to vaccinate.

Some of the fear of vaccination is driven by disease-driven litigation tied to junk science.

The landmark research linking the MMR vaccine to autism was initially published in 1998 by Dr. Andrew Wakefield in the Lancet, a respected British medical journal. Other researchers' inability to replicate his findings spawned further investigation. Subsequent inquiry into Dr. Wakefield's research demonstrated that his subjects were recruited by a plaintiff's lawyer preparing a lawsuit against vaccine manufacturers.

Additionally, not only had Dr. Wakefield received payment from these attorneys two years prior to initiating his research study, but he also had a patent application for a rival measles vaccine. In addition to the numerous conflicts of interest, Dr. Wakefield's research was also found laden with altered data. Finally, the Lancet took the unprecedented step of retracting the original published peer-reviewed article.

The best medical research suggests no link between vaccination and autism. Nonetheless, the absence of data has not stopped families from pursuing their claims of disability through available legal channels.

Lawyers intent on creating a mini-industry out of lawsuits against vaccine makers have threatened the supply of vaccines to the American public. Many drug companies in the U.S. were pushed out of the vaccine business in the 1980s from large settlements related to whooping cough-tetanus-diphtheria (DTP) vaccine reaction lawsuits. In response, Congress in 1986 created an alternate legal system of "vaccine courts," compensating patients financially based on known vaccine-related side effects. Payments on the judgments of these tribunals are funded by a tax levied on each vaccine.

Despite the lack of solid research confirming a causal link between vaccines and autism, there are petitions from more than 5,000 families pending before the courts, arguing otherwise. However, as per the common scientific axiom, the plural of "anecdote" is not "data."

In February, the Supreme Court heard a challenge to the legality of the federal law that created this no fault, nonjuried tribunal system, which shields drug companies from product liability lawsuits. In a 6-2 decision, the court reaffirmed the success of these vaccine courts, whose judgments are based on known science rather than fickle juries, which are often swayed by personal stories of hardship.

Writing in the majority opinion, Justice Antonin Scalia found the law "reflects a sensible choice to leave complex epidemiological judgments about vaccine design to the [Food and Drug Administration] and the National Vaccine Program rather than juries."

While scientifically baseless claims of disability have no weight in these courts, they have had a monumental effect on the lay public.

The response following Dr. Wakefield's initial study in the United Kingdom was palpable. MMR vaccination rates in Britain dropped from 92 percent in 1996 to 84 percent by 2002. Measles and mumps cases subsequently grew at rates tenfold to thirtyfold compared with periods prior to the study. A decade of suboptimal levels of vaccination in the U.K. has now compromised herd immunity to measles, (that is, the immunity gained by an individual susceptible to a disease through the critical mass of the surrounding immunized community).

According to the UK Health Protection Agency, their equivalent to the CDC, the U.K. now faces endemic levels of measles within its population, allowing for the continuous spread of the disease.

In a viral age, the persistence and prevalence of vaccine misinformation among the public may take some time to correct. However, regaining the health protection afforded by effective vaccination programs will take longer.

Dr. Sandeep Rao is a fellow at Johns Hopkins Hospital. His email is srao28@jhmi.edu.

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Drop and give me 20

07/11/2011 10:01 AM |

FDA currently is approving two-thirds of critical drugs in the first review cycle, CDER Director Janet Woodcock told the House Energy and Commerce Health Subcommittee July 7, disputing complaints that the agency's approval process is stifling innovation and capital investment in the pharmaceutical industry.

FDA approved 20 new medications during the first half of 2011, one shy of the 21 approved in all of 2010, she added.

(True, but FDA approves about 60% of priority reviews within a single review cycle -- by pulling staff away from standard reviews.)

The rate of first-cycle drug approvals is at the highest level seen in 20 years, she pointed out during a hearing that opened debate on reauthorization of the Prescription Drug User Fee Act with a look at how FDA's oversight of drug development impacts investment in new therapies.

Subcommittee Chairman Joe Pitts, R-Pa., said at the hearing that he wants to avoid a last minute rush to pass PDUFA V, and plans to have the reauthorization completed and signed by the president by June 30, 2012, well ahead of the Sept. 30, 2012, expiration date of the current law.

Details of the agreement are to be published on Sept. 1, with final recommendations sent to Congress by Jan. 15, 2012.

Despite the quick timetable for reauthorization and implication that the legislation would be relatively "clean," both subcommittee Chairman Joe Pitts, R-Pa., and full committee Chairman Fred Upton, R-Mich., used the hearing to voice concerns that uncertainty in the FDA approval process are stifling medical innovation and delaying access to new therapies.

Upton said the committee will examine the lack of predictability and certainty at FDA, two issues that "appear to be stifling American innovation, costing American jobs and hurting American patients."

"What we have heard," Pitts said, is that the approval process often fails in terms of certainty, predictability and transparency, and this is "frustrating both the drug sponsors and the public, who are waiting for treatments and cures to everyday maladies, chronic illnesses and terminal diseases."

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Sometimes Sanity

07/08/2011 09:15 AM |

During my tenure at the FDA I was the senior official in charge of advisory committee oversight and the final decision-maker on who got a COI waiver and who did not. Many did not — but those who did received their waivers because FDA professional career staff made a strong case that these people weren’t just important to the advisory committee — but critical.

And we should all pay attention to the nomenclature.  It’s not about “conflict of interest” – it’s about (as Secretary Sebelius correctly says) “interest.”  And having an “interest” is not necessarily a bad thing – as long as you’re transparent about it.

If we allow FDA adcomms to become the realm of the second best and the almost brightest –what have we done to the advancement of America’s health? The answer is a significant disservice.

In the February 7, 2010 edition of The Lancet, Richard Horton points out that the battle lines being drawn and between clinician, medical research and the pharmaceutical industry are artificial at best -- and dangerous at worst.  Dangerous, because all three constituencies are working towards the same goal -- improved patient outcomes.

Horton’s main point is that we must dismantle the battlements and embrace of philosophy of "symbiosis not schism."  It's what's in the best interest of the patient.

And so it is with this in mind that I share some promising news (as reported today in The Hill.)

GOP wants FDA bill to boost drug industry's role

Republicans want to roll back new conflict-of-interest rules they say are depriving the Food and Drug Administration of needed expertise from the drug industry.

Democrats, meanwhile, will focus largely on the safety of imported drugs as Congress begins work on a five-year FDA reauthorization bill.

Congress tightened the FDA's conflict-of-interest rules in 2007, as part of the last FDA reauthorization. But Republicans on the House Energy and Commerce Committee said they may try to loosen the standards in the next reauthorization, which needs to pass next year.

Committee Chairman Fred Upton (R-Mich.) said the upcoming bill should reverse "rigid, unrealistic conflict-of-interest provisions" that have delayed drug approvals. The rules govern who can participate in FDA advisory panels, which study safety and effectiveness issues.

"No longer can we afford to sideline experts simply because of their ties to the pharmaceutical industry," Rep. Phil Gingrey (R-Ga.) said Thursday at an Energy and Commerce health subcommittee hearing.

Janet Woodcock, the director of FDA's drug center, said the limits have slowed down the advisory committee process. The agency sometimes goes through the long haul of finding experts in a given field only to discover ties to the pharmaceutical industry toward the end of the process, she said.

The FDA has already begun negotiating with drugmakers and consumer advocates over the reauthorization bill. Thursday's hearing marked the first formal involvement from Congress.

Technically, the purpose of the FDA bill is to reauthorize the programs through which drug and medical device companies pay the FDA to review their products for approval. But because it's a must-pass measure — the people and offices that approve new products are paid for almost entirely by industry fees — it consistently becomes a magnet for broader policy changes.

Upton said Thursday that he also wants to reexamine a piece of the last reauthorization that gave the FDA more power to regulate drugs after they've been approved. The FDA can now require drugmakers to study certain safety issues and add new warnings to drug labels.

Upton and other committee Republicans say FDA overregulation is stifling innovation and preventing drug and device companies from creating new jobs. Rep. Henry Waxman (D-Calif.) argued that while getting new products to market is important, the FDA's mission should be protecting public health rather than fostering job creation.

Energy and Commerce Democrats indicated that the safety of imported drugs will be their biggest policy focus. Rep. John Dingell (D-Mich.) began working on an import safety bill in 2007, the food-safety portion of which passed on its own in 2009.

The FDA inspects foreign factories far less often than domestic ones, and it can't make a surprise visit outside of the United States. Those limitations received extra scrutiny following the 2007 heparin contamination, which Dingell cited repeatedly at Thursday's hearing.

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Keeping the Spark Alive

07/08/2011 08:57 AM |

From today's Wall Street Journal:

An ObamaCare Drug Preview in Germany

Do you want penny pinchers nixing safe medicines before they've had a chance in the marketplace?

 

By John C. Lechleiter

 

Germany was in many ways the birthplace of the pharmaceutical industry. But today, German policies place the industry's future progress at risk.

More than a century after Eli Lilly's founding, many of us still trace our company's greatest "spark" to Germany, and to a scientist born in 1877 named George Henry Alexander Clowes. An Englishman, Clowes was determined to become a great chemist and was dedicated to improving human health. In the late 19th century, such a goal meant one thing: Go to Germany. Clowes left Germany not only with a Ph.D. in chemistry but also with a conviction that breakthroughs against disease depended on the collaboration of industry, academia and medicine.

Twenty-six years later in 1922, Clowes had crossed the Atlantic and become the head of research at a small company in Indianapolis. He helped to spearhead the commercial manufacture of insulin for the treatment of diabetes—turning what was once a death sentence into a manageable condition.

Today in Germany, it's doubtful that George Clowes would find much inspiration. The ethos of hard work certainly still exists, but intrusions of bureaucracy, misunderstandings of science, and an exaggerated emphasis on short-term cost savings are jeopardizing the country's legacy as a pharmaceutical powerhouse.

Of particular concern is a new law passed last year that imposes a complex new assessment mechanism to determine the added benefit of new pharmaceutical products, which in turn is used to set prices. The situation in Germany bears close scrutiny in the U.S., as health-technology assessment processes grow in significance under the recent health-care overhaul.

Our concerns begin with the timing of Germany's assessment: at the point of launch. Pharmaceutical companies invest hundreds of millions of dollars into clinical trials to prove the efficacy and safety of new medicines—but efficacy and safety are not the same things as added benefit. We introduce products into the market with well-founded hypotheses about their unique benefits, but rarely with definitive evidence. By insisting that we provide such evidence before a single patient has used a medicine in regular medical practice, German authorities are devaluing the final and arguably most important stage of pharmaceutical development.

Contrary to popular mythology, so-called blockbuster medicines are not the result of slick marketing campaigns but of demonstrated success in the actual practice of medicine. At Lilly, we wonder how many of our earlier blockbusters—which might have appeared very similar to existing medicines at their launch but proved more effective in practice—would have survived the requirements of Germany's new early assessment requirement. Similarly, we wonder if incremental innovations in the treatment of cancer, for example, or new medicines that limit side effects and improve patient compliance with doctors' orders in diabetes care, will be considered beneficial under Germany's new system.

Even that bleak conclusion assumes that the early assessments will be carried out in a fair and transparent manner, which is far from assured based on what the industry has seen so far. Companies such as Lilly are looking at the all-important "comparators" being assigned to our new molecules in the assessment process. In many cases, German authorities appear to be comparing cutting-edge products with decades-old generic products and still pricing the new products just pennies above the generics. Our conclusion is that we are being set up for negative decisions on added benefit and ruinous price controls as a result.

The wisest decision that a company can make in such circumstances may be to delay the launch of a new medicine in Germany until evidence can be generated elsewhere. The other alternative may be to keep new drugs off the German market entirely to prevent the global reputational damage of a slap from the German authorities.

Meanwhile, the broader uncertainties created by the new German law—literally any product can be drawn into the new evaluation process without advance notice—wreak havoc on our industry's business planning.

Now is the time to step back and consider the unintended consequences of Germany's new law, and make sure we don't repeat the same mistakes in the U.S. We share Berlin's goals of generating value and limiting unnecessary costs in health care. We see ways of harnessing and improving health-outcomes research.

Most of all, we believe that dialogue between government and industry is the only way to ensure that the cradle of the pharmaceutical industry remains a nurturing environment for new medicines in the 21st century.

Mr. Lechleiter is chairman and CEO of Eli Lilly & Co.

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Junk Science on Autism

07/07/2011 07:50 PM |

A new study purports to show that environmental factors are more likley to cause autism than genetic mutations.  I won't get into the weeds on the methodological problems of the research since genetic association studies are, absent analysis or sequencing of genes, full of statistical assumptions that vary from paper to paper.  Moreover, when discussing the contributions of genes and 'environmental' factors to a behavior or disease, the term environment really means "anything we can explain by genetics that is not explained by demographics." 

http://archpsyc.ama-assn.org/cgi/content/full/archgenpsychiatry.2011.76#YOA15046T2

Which begs this question:  Since so many of the likely 'environmental' factors alleged to be linked to autism (, inflammatory diseases, mitochondrial disorders, vaccines, water supply, chemicals) have been discredited, how reliable is a study -- one that is at odds with every other genetic association study regarding autism -- that claims environmental factors are twice as likely to trigger autism than genes?  How good is a model that predicts things at odds with what experience demonstrates?

Of course the media never examines this question, never puts this study into context and never looks at the toxicological studies showing that certain toxins some swear cause autism, impotence, cancer, etc. are not even present in concentration levels necessary to cause of biological reaction (benign or not).      Not suprisingly, BPA is now being held up as the next new culprit causing autism.  Thus the recent EPA review of BPA levels in humans will go unmentioned:

EPA Study Findings:

·         Consistent with previous human and animal studies, this new study confirms that human exposure to BPA is extremely low, and BPA is efficiently metabolized and rapidly excreted in urine. Of particular importance, these results confirm the validity of the controlled human volunteer pharmacokinetic studies that have been used by regulators in BPA risk assessments. 

 

o   Based on how much total BPA (after hydrolysis of metabolites) was found in urine (average exposure was 21% greater than the 95^th percentile exposure determined by CDC’s population-scale urine biomonitoring data), the authors concluded that “BPA exposures in this study can therefore be viewed as representative of, or exceeding, the high end of the U.S. BPA exposure distribution.”

 

o   Total BPA was detected in only 14% of the 320 blood samples, only one of which was above 1 ppb. Total BPA was below the sensitive limit of detection (0.3 ppb) for 86% of the samples.

 

o   Estimates of peak BPA levels in the blood were 1 to 3 orders of magnitude below levels associated with potentially adverse health effects in the most sensitive experimental rat models. 

 

o   Free BPA was below the limit of detection in all 320 blood samples analyzed by the CDC lab, even for samples with detectable total BPA. Based on their results the authors note that reported high levels of BPA in blood are unlikely to be valid. (“Furthermore, the current results obtained using analytical methodology 10-45 times more sensitive than the previous human study by Voelkel et al. suggest that reported BPA concentrations in human blood of 1.4-19.2 nM [~0.3-4.4 ppb] (Vandenberg, Chahoud et al. 2010) are highly unlikely in the general population exposed orally to amounts as much as ~4 times greater than the 95^th upper percentile of aggregate exposure in the general U.S. population.”)

 

o   While subjects of this study did not include pregnant women, the authors also add that “recently reported mean urine concentrations in pregnant women are low enough that blood and serum levels are likely to be below current detection limits.”

 

o   Samples with detectable total BPA were further analyzed in the FDA lab to confirm the findings. Very low but detectable levels of free BPA were found in 3 of these samples, but further analysis revealed that contamination was most likely the source of the free BPA. The authors note, “the evidence presented here and elsewhere for low-level contamination (Markham et al., 2010; Twaddle et al., 2010), even in the face of extraordinary attention to this problem, suggests that these infrequent positive determinations near the detection limit should be suspect” and “Thus, some attributions of high blood BPA concentrations from oral exposure seem implausible.”

 

24-Hour Human Urine and Serum Profiles of Bisphenol A During High Dietary Exposure  tinyurl.com/3efsdw8

Will we -- and the media -- unlearn the lessons of Wakefieldism?

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Blame Innovation!

07/07/2011 09:08 AM |

Blame innovation!

According to an editorial in today’s New York Times, “A prime driver of our escalating health care costs is the advance of medical technology …”

Indeed. 

The driver of the Gray Lady’s oration is the decision by Medicare to pay for both Provenge (advanced prostate cancer) and Avastin (for breast cancer – even if the FDA removes this as an on-label indication).

The Times objects to both treatments “staggering high cost” and “modest help to the typical patient.”

Are these treatments “too expensive?” Well, as they say inside the Beltway, where you stand depends on where you sit.”  Too expensive for whom?  And there lies the rub.

When our nation’s biggest health insurance provider – Uncle Sam – is footing the bill, the real payer is the American tax payer.  You and me.

That being the case, how do we feel about spending our tax dollars on end-of-life care for our fellow citizens?  After all, these treatments aren’t cures.  They extend patient life. Some gain months, others years. 

This is a tough and timely question for both public and private debate – and not just amongst policy wonks and editorialists. Do we want a health care system that is cost-based or patient-centric? Should end-of-life care be rationed?  If so, by whom and by what measure?

And if we decide not to pay for new medicines, the clear signal to the pharmaceutical industry is “cease research and development for new treatments for killer diseases."

Blame innovation? Consider:

Innovation is slow.  As any medical scientist will tell you, there are few "Eureka!" moments in health research. Progress comes step-by-step, one incremental innovation at a time.

Innovation is hard.  Today it takes about 10,000 new molecules to produce 1 FDA-approved medicine. And if that's not frightening enough, only 3 out of 10 new medicines earn back their research and development costs. And here's the kicker -- unlike other R&D-intensive industries, pharmaceutical investments generally must be sustained for over two decades before the few that make it can generate any profit.

Innovation is expensive.  In 2003, researchers at Tufts Center for the Study of Drug Development estimated the costs to bring a new medicine to market to be $802 million, and others suggest that the total cost is closer to $1.7 billion

Innovation is under attack.  From accusations of the “me-too” variety, to crackpot schemes to replace pharmaceutical patents with a “prize” system, life for innovator pharmaceutical companies is rough and tough. 

But innovation is important – and not just for pharmaceutical industry profits. Increases in life expectancy resulting from better treatment of cardiovascular disease from 1970 to 1990 have been conservatively estimated as bringing benefits worth more than $500 billion a year. In 1974, cardiovascular disease was the cause of 39 percent of all deaths. Today it is about 25 percent. Cerebrovascular diseases were responsible for 11 percent of deaths back then. In 2004 they caused 6.3 percent of deaths. Kidney diseases were linked to 10.4 percent of deaths and now they are associated with 1.8 percent. And that’s just for the United States.

As Harvard University health economist (and Obama healthcare advisor) David Cutler has noted: "The average person aged 45 will live three years longer than he used to solely because medical care for cardiovascular disease has improved. Virtually every study of medical innovation suggests that changes in the nature of medical care over time are clearly worth the cost."

Blame innovation – because now we are living long enough to suffer in larger numbers from cancer and Alzheimer’s disease. Diseases that cannot be cured (at least not yet) and are expensive to treat.

And what of the “typical” patient?  The current gold standard of drug testing, the randomized controlled clinical trial, isn’t specifically designed to identify patient subpopulations who might benefit from a treatment that does not aid the general population. According to the folks at our nation’s newspaper of record, Avastin “provides almost no benefit for the typical woman with advanced breast cancer.” Well – true, except for the subpopulation of women for whom it does.  The problem isn’t that Avastin “doesn’t work,” it’s that we have no way of identifying which women it will work for.

The solution isn’t to carp on cost – it’s to develop better diagnostics to help physicians identify the “four rights” – the right drug in the right dose for the right patient at the right time.  We need to focus not on cost effectiveness but on clinical effectiveness.

But nowhere in the Times’ editorial is there a call for increased FDA funding for the development and regulation of molecular diagnostics – something that is urgently needed and, alas, rarely discussed.

Rather the Times’ editorial ends as follows, “Whether the drug is worth its high price is a question our health care system is currently unprepared to answer.”

Really?  It seems that Medicare is not only prepared to answer the question – but that, via their decision to reimburse for both Provenge and  Avastin, they have.

At least for now.  After all, we're entering into a Presidential election cycle.

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More Science, Less Salt

07/06/2011 09:10 PM |

A "systematic reviews" about the clinical effectiveness of reducing salt intake on high blood pressure showed no effect. 

Of course many experts weighed in on the study:

"Critics say this new review is based on just seven studies that were not designed to test the effects of sodium reduction interventions on CV events and mortality and is therefore unreliable. "

One physician, Lawrence Appel, was particularly harsh: "The authors truncated the best set of trials, TOHP1 and TOHP2, which has extended follow-up of >10 years, to just the trial period, 1.5 years for TOHP1 and three years for TOHP2. This set of trials, with extended follow-up, documented a significant reduction in CVD events."

This is the same Lawrence Appel who continues to push ALLHAT and a diuretic alone as the gold standard for treating hypertension to the exclusion of several new, large studies demonstrating that a combination of blood pressure meds reduce the risk of death from stroke and other ailments more effectively. 

A little more consistency in how medical studies are evaluated and applied would be helpful.  The totality of evidence matters.  But what matters more is moving away from population based conclusions towards individualized and mechanism specific approaches to care. 

Epidemiology, the foundation of comparative effectiveness research, is so 19th century.

See: Advising people to eat less salt is not best approach to reduce CVD at theheart.org  Read More & Comment...

Duals in the Sun

07/06/2011 09:57 AM |

And now a cry for “mandatory rebates.”

Really?  Why don’t we just call it what it is – a tax.  More precisely, an excise tax imposed by Uncle Sam on drug sales to dual eligibles (based on the price difference between Medicaid prices and those of the private market).

But wait, it gets worse. The revenues from this tax don’t lower costs for a single patient.  Not one.  The cash would go into the general fund.  It’s not a “rebate” – it’s a tax, plain and simple.  A levy imposed on price.

And it’s a tax with a hidden purpose – the introduction of backdoor price controls.  And price controls equal cost controls – with or without IPAB.

Forewarned is forearmed.

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Post Facto

07/05/2011 08:49 AM |

Editorial from today's edition of The Washington Post:

Editorial Board Opinion

High court highlights a gap in drug safety regulation

GLADYS MENSING’S doctor prescribed the brand-name drug Reglan to treat a gastrointestinal problem. When the Minnesota resident went to fill the prescription, the pharmacist — as he was compelled to do in Ms. Mensing’s state — dispensed a cheaper, generic version of the drug.

Ms. Mensing now suffers from tardive dyskinesia, an irreversible neurological condition linked to the drug and characterized by “grotesque involuntary movements of the mouth, tongue, lips, and extremities, involuntary chewing movements, and a general sense of agitation.” Ms. Mensing sued the manufacturer under a state law for failing adequately to warn of this potentially severe side effect.

Last month a 5-4 majority of the Supreme Court threw out her case. The reason: She used the generic version of the drug. Had Ms. Mensing suffered the same harm after consuming the brand-name drug, her case would have been allowed to proceed.

The majority, led by Justice Clarence Thomas, had valid reasons for its decision, but even Justice Thomas admits the result “makes little sense.” We would go further: The result is a dangerous one that exempts some 75 percent of the drugs dispensed in this country from essential safeguards. This situation should be rectified — and quickly — through federal regulation.

Risks associated with long-term use of Reglan and its generic version were long known, but neither the brand-name nor the generic version carried a “black box” warning — the most serious kind — until years after Ms. Mensing began using the product. Ms. Mensing sued the generic’s manufacturer under a Minnesota law that dictates that “where the manufacturer . . . of a product has actual or constructive knowledge of danger to users, the . . . manufacturer has a duty to give warning of such dangers.” She argued that the generic drug’s maker should have changed its label to reflect heightened risks.

But federal law foreclosed this possibility. Brand-name producers are typically the first to be alerted to adverse affects of their products and are allowed to — and in some instances are obligated to — make unilateral changes to warning labels. Generics producers, on the other hand, do not enjoy such flexibility — or responsibility — and are tasked only with parroting the brand-name’s warning labels. The court concluded that federal law required dismissal of Ms. Mensing’s suit since it was “impossible” for the manufacturer to comply simultaneously with the state and federal rules.

Writing for the four dissenters, Justice Sonia Sotomayor correctly noted that “as a result of today’s decision, whether a consumer harmed by inadequate warnings can obtain relief turns solely on the happenstance of whether her pharmacist filled her prescription with a brand-name or generic drug.”

This should not stand. The Food and Drug Administration should require manufacturers of generic drugs to track adverse effects and reach out to the FDA to initiate label changes to reflect new or heightened dangers. A fair right to sue is important, but even more so would be compelling full disclosure so that patients are not harmed in the first place.

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Unclear and Present Danger

07/05/2011 08:07 AM |

Never express yourself more clearly than you are able to think.
--Niels Bohr

Think regulatory headaches about FaceBook is a hot topic??  Here are some communications issues that make social media guidance pale in comparison.

Today Eli Lilly, Johnson & Johnson, Novartis, Novo Nordisk, Pfizer, and Sanofi-Aventis will file a Citizen Petition with the U.S. Food and Drug Administration (FDA) asking the agency to clarify its policies on how truthful, non-misleading scientific information not included in approved product labeling can be communicated.

The petition will state that communicating accurate scientific information about new research would enhance health care quality and potentially lead to better patient outcomes, but that companies lack precise guidance on how to communicate such information.

Specifically, the companies requested that FDA issue clarifying regulations or guidances on four issues:

* “Scientific exchange”
* Sharing information with formulary committees and payers
* How to provide independent third-party clinical practice guidelines
* Responding to unsolicited requests for information

“Scientific exchange,” broadly defined, is the sharing of research and clinical information about investigational medical products or new information on approved products without representing the product as safe and effective for that use.  FDA said in a 1963 regulation that it does not intend to restrict “scientific exchange.”  The concept of “scientific exchange” however, is not precisely described in FDA’s regulations and
therefore leaves ambiguity about the limits of what is permitted.

Managed care formulary committees and other payers make important coverage and reimbursement determinations based on clinical evidence and other data.  FDA has not issued a formal policy with respect to what information manufacturers can provide to formulary committees about new uses of approved medical products so that patients can be promptly reimbursed.

Third-party clinical practice guidelines are produced by leading medical organizations or the government to inform and guide diagnosis, management and treatment decisions.  These guidelines are based on the most up-to-date clinical evidence and data and are intended to guide health care professionals in improving quality of care and optimizing clinical outcomes.  There is no formal FDA policy on when and how it is appropriate
for companies to disseminate third-party guidelines that mention off-label uses of their products.

“Unsolicited requests” are unprompted questions from physicians or patients about the off-label uses of medical products.  For years FDA has not taken enforcement action when companies respond to unsolicited requests for off-label information in a balanced and scientific manner.  However, there is no written regulation or policy providing a comprehensive description of FDA’s policies on unsolicited requests.

 The absence of alternatives clears the mind marvelously.
--Henry Kissinger

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The Avastin Spring

07/04/2011 01:07 PM |

Sometimes profound change happens so swiftly that is unrecognized and unappreciated. Such is the case with the response to the Food and Drug Administration advisory committee’s decision to withdraw the agency’s approval for using Avastin to treat metastatic breast cancer. After the FDA Oncological Drug Advisory Committee declared Avastin to be unsafe and ineffective for women suffering from that incurable form of the disease the headlines and pundits spun the decision – and the discussion – as one where science triumphed over emotional and desperate women. Gary Schwitzer, a self-styled expert on objective medical reporting quoted the representative from the National Breast Cancer Coalition who at the ODAC hearing: "This decision can't be driven by anecdotes. It must be driven by science."

This is an unfair and lazy characterization of the debate over Avastin. The ODAC panel was stacked with members who already voted against Avastin’s use in MBC. The conflict was over the interpretation of clinical information and whether the FDA can pull an approval because, as it said in December of last year, Avastin doesn’t provide “a sufficient benefit in slowing disease progression to outweigh the significant risk to patients.” But several other groups that looked at the same evidence came to a different conclusion including The National Comprehensive Cancer Network and the European Medicines Agency (Europe’s FDA) that expanded approval Avastin based on the same “science” ODAC used in rescinding the drug’s FDA endorsement.

In any event, the speed with which both the Obama administration and private health plans announced they would still cover the cost of Avastin for the use the FDA rejected was stunning. When the FDA decided to yank Avastin’s approval in December 2010, several healths plans and regional Medicare reimbursement contractors told women that Avastin would no longer be covered. (Such decisions were reversed only after the FDA said it would review its revocation.)

Last week the same organizations – along with the Obama administration – rushed to distance itself from the FDA and assure women it would cover the drug.

The difference in response is a result of the rapid organization of patients through social media. While it culminated in the presence of hundreds of women at the ODAC hearing, that participation was only a part of an extensive, sustained and scientifically informed movement.

The FDA officials and the interest groups urging Avastin’s revocation were outgunned by individual patients who joined together much as HIV patients did in the 1990s and MS patients did in pushing for the return of Tysabri to the market a few years ago. This time however, the use of social media to share clinical information, organize support and express opinions was rapid, sustained and, above all surprising. The cancer advocacy ‘establishment’ was outgunned and reduced to irrelevancy. This also happened after the Obama administration’s recommended not to cover mammograms for women under 50. The National Breast Cancer Coalition sided with the administration and cancer patients, oncologists and radiologists everywhere told them go stick it. That decision, like the FDA’s rendition of Avastin, is being ignored in practice.

We see in this recent uprising against the Avastin decision a clear shift in power from the Beltway groups that claim to represent patients to individuals organizing on their own and in their own best interest. More to the point, this movement is well informed and shaped by what the science says. The attempt to demean or diminish it as purely anecdotal and emotional reflects the arrogance of bloggers and pundits who have but a thimble full of the clinical understanding of the movement’s participants. And it suggests contempt for any use of social media that does not seek to spread fear or skepticism of innovation.

Companies who develop new products will have to contend with consumers that demand credible and substantive clinical information. Health plans and the government will have to expect ongoing challenges to their ‘expertise’ and claims that it is using the best evidence in determining what to cover. In particular the apparatus and funding supporting comparative effectiveness research will find that consumers will be skeptical of one-size fits all judgments made by so-called ‘experts.’

The Avastin Spring suggests that resistance against the use administrative means to tell the rest of us what treatments we can and cannot use will only grow. We don’t know how it will all play out. But we do know that it will lead to more individual choice and less authority for self-styled advocacy groups, government and corporate entities who claim that only they have science on their side en route to restricting access to innovations.

 

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Don't Dig in Against Gibson (Or innovation)

07/01/2011 12:54 PM |

As I suggested in my earlier posts,  Medicare will continue to pay for Avastin to treat women with metastatic breast cancer. 
What I didn't expect, but should have, was how quickly the Obama administration would make that decision and how it distanced itself from the FDA ODAC vote to revoke Avastin's indication.   From Reuters:

"The FDA decision, when it comes, does not affect CMS," Don McLeod, a spokesman for the Centers for Medicare and Medicaid (CMS), said on Thursday."The drug will still be on the market, doctors will still be prescribing it, and we will continue to pay for it," he said, adding that CMS often pays for off-label uses of drugs."  www.reuters.com/article/2011/06/30/roche-avastin-cms-idUSN1E75T17U20110630

Gee,  what happened to the effort to coordinate reimbursement decisions and FDA approvals in order to "bend the cost curve."

The comparative effectiveness crowd must be comparing in their pants..

And if that wasn't enough, Team Obama trotted out Donald Berwick to extol the virtues and value of Provenge, a drug it delayed paying for by 18 months.  Here's what Berwick said in a prepared statement that sounded like a forced confession from the lover of Britain's single payer health system.

“We are optimistic that innovative strategies may improve the experience of care for our beneficiaries who have cancer,” said CMS Administrator Dr. Donald M. Berwick.  “CMS is dedicated to assuring that these patients can seek the treatments they need in accordance with their wishes.”
www.latimes.com/health/boostershots/la-heb-prostate-cancer-provenge-medicare-20110701,0,7641053.story


Here's Berwick in 2009: 

"We can make a sensible social decision and say, “Well, at this point, to have access to a particular additional benefit [new drug or medical intervention] is so expensive that our taxpayers have better use for those funds.” We make those decisions all the time. The decision is not whether or not we will ration care — the decision is whether we will ration with our eyes open. And right now, we are doing it blindly."

www.ncbi.nlm.nih.gov/pmc/articles/PMC2799075/

Every time a new cancer drug is approved in Britain, the NHS gets hammered for rationing care.   But what happens in Britain is tee-balll compared to what CER types and the institutions that will produce or use CER will go through every time it tries to restrict or delay access to treatments that benefit a segment of the population.

Trying to go up against innovation in the US?  It could be compared -- if the Avastin and Provenge sagas are any indication -- to trying to challenge St. Louis Cardinal pitcher Bob Gibson, know for his take no prisoner attitude against any hitter.  What Hank Aaron said in that regard might explain Team O's response to the FDA decision:

'Don't dig in against Bob Gibson, he'll knock you down. He'd knock down his own grandmother if she dared to challenge him. Don't stare at him, don't smile at him, don't talk to him. He doesn't like it. If you happen to hit a home run, don't run too slow, don't run too fast. If you happen to want to celebrate, get in the tunnel first. And if he hits you, don't charge the mound, because he's a Gold Glove boxer.' I'm like, 'Damn, what about my 17-game hitting streak?' That was the night it ended."

Don't dig in against innovation. 








 

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FDA Dooms Cancer Patients

07/01/2011 11:29 AM |

Chalk one up to the anti-medical progress crowd.. for now.

The Public Policy

FDA Decision Dooms Cancer Patients

By Robert M. Goldberg on 7.1.11 @ 6:08AM

Opponents of medical progress are celebrating the decision of a Food and Drug Administration panel to revoke approval of the use of Avastin in treating advance breast cancer.

Not so fast.

As Scott Hensley, a health care reporter for NPR, wrote:

The day after a panel of experts advised the Food and Drug Administration to go ahead with plans to revoke approval of Avastin to treat breast cancer, European authorities moved in the opposite direction.

The European Medicines Agency (EMA) gave the OK to an expansion of Avastin's approval to include using the drug in combination with Xeloda, a chemotherapy drug, to treat metastatic breast cancer, Genentech parent company Roche said Thursday.

The Europeans considered the same information that hasn't proved persuasive to U.S. regulators. Among other things, a study called RIBBON 1 found Avastin plus Xeloda increased progression-free survival or PFS -- (living longer without tumor growth) of women by 2.9 months over women getting Xeloda alone.

This improvement seems small but is actually quite significant. Gains in PFS among patients with cancers that spread through the body are hard to achieve because when the disease spreads it is considered incurable. And over time, the small victories over incurable cancers extend and improve life.

The EMA believes 2.9 months is a great beginning. The FDA sees nothing less than 6 months to be worth it and worked hard to make sure Avastin would never hit that mark. 

The FDA's Richard Pazdur, who heads up FDA's Office of Cancer Drugs, had approved Avastin's use in breast cancer on the condition that future studies confirmed the improvement in PFS. Genentech conducted three studies that hit that mark. But then Pazdur claimed that PFS was not enough. He wanted evidence that median overall survival increased. 

While an increase in overall survival has been considered the gold standard for cancer drugs, progression-free survival has been used as a benchmark for longer and better quality of life.

That's because a standard like overall survival only tells you what the median survival is for all patients -- it doesn't take into account individual differences in response based on genetic, clinical and other factors.

Yet when results of additional studies this summer concluded that Avastin doesn't extend life on average, an FDA advisory committee suddenly decided that Avastin should be measured by overall median survival -- not progression-free survival.

Before this measurement switch, the FDA admitted that the new studies showed a statistically significant improvement in PFS with Avastin. But then it said, "the magnitude of treatment effect, as commonly assessed by clinicians based on differences in median PFS, was marginal."

Pazdur seems to want to toughen standards based on a one-size-fits-all measure that requires longer and more expensive clinical trials. In doing so he has been supported by such left-leaning and anti-innovation entities as the Center for Science in the Public Interest's Merrill Goozner and Public Citizen, a Naderite group that has warned against most new medicines for cancer and diabetes since the 1970s. These and other Avastin opponents -- who have long regarded the drug as a symbol of corporate greed -- have lobbied for median survival as the key standard. They know that applying that standard would harm the perception of Avastin's effectiveness.

Goozner -- who has no medical background -- was appointed to an FDA advisory committee on pharmaceutical science. Two senior Public Citizen operatives, Peter Lurie and Larry Sasich, now set policy for the FDA. Fran Visco, the head of the National Breast Cancer Coalition, applauded the FDA decision after lobbying for it over the past year. Visco, a Democrat, is also on Experts Advisory Panel for the Universal Health Insurance Program at the New America Foundation, a left-wing think tank supporting Obamacare. The NBBC also supported the administration's decision not to cover mammograms for women under 50 though many breast cancers grow faster and earlier in African-American women.

To these groups, the FDA decision was a triumph. But their effort to manipulate the FDA will backfire. The EMA and every major group of cancer providers support Avastin's use. Cancer patients moblilized spontaneously to keep Avastin's label. They will take on the anti-innovation establishment and the FDA with greater intensity and vigor.

Medicare and some health plans will try to stop paying for Avastin. But Congress will hold oversight hearings on the Avastin decision en route to amending FDA's statute through one of many bills calling for faster access to new treatments.   

The administration will have to respond to show it's not using FDA to ration care or slow innovation. 

In the long term this is all well and good. But meanwhile many women dying of breast cancer will have to go to Europe to get a drug that could keep them alive. 

Robert M. Goldberg is vice president of the Center for Medicine in the Public Interest and founder of Hands Off My H ealth, a grass roots health care empowerment network. His is new book,  Tabloid Medicine: How the Internet is Being Used To Hijack Medical Science For Fear and Profit, was published last month by Kaplan.
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A "Yes" from CMS

07/01/2011 08:50 AM |

On CNBC this Tuesday, I called on HHS Secretary Sebelius to promise that CMS would continue to pay for Avastin as a treatment for breast cancer.

(The complete CNBC interview can be viewed here.)

And, late yesterday, Medicare confirmed it would do just that. According the CMS spokesman Don McLeod, “The label change will not affect our coverage.”  

At least for now. As Andrew Pollack reports in today’s New York Times, “Mr. McLeod’s statement could allay those concerns, at least for women covered by Medicare. He said that Medicare commonly paid for off-label use of cancer drugs.”

McLeod said that while there were no plans for one right now, he could not totally rule out that Medicare might one day undertake a national coverage determination to decide whether to pay for Avastin. That process would take at least a year and involve public input.

In other words, the administration understands that, had CMS withdrawn reimbursement for Avastin’s use in breast cancer, it would have become a front-and-center election issue.  Proof positive that ObamaCare equals denial of care.

A unique situation (at least as of late) where the right public health decision is also the savvy political move.

And hopefully it won’t be the last.

(Can you say “Adieu IPAB?")

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