FDA's Pathway Roadblock

05/17/2010 11:14 AM |

The FDA scotched the Pathway and Walgreens partnership by asking Pathway to prove it didn't need FDA approval as a medical device.  Someone should explain to me how gene tests are a medical device..I guess soap and bathroom scales are also medical devices (David Kessler once said the former was!) and should have FDA approval of some sort.   Meanwhile, clinical labs that are part of programs designed to test, diagnose and sell quackish treatments abound...

www.sciencebasedmedicine.org/

The lame protectionist statement from the genetics counseling trade group sounds a lot like the whining from HIV clinics who opposed home HIV tests (protecting patients, not their monopoly on testing).  "National Society of Genetic Counselors issued a statement saying that receiving genetic information without input from a doctor "increases the chance for misunderstanding or misinterpretation of results."

Really?  Seems to me that it will lead people to contact doctors and make lifestyle changes...just as the spread of the home HIV test.  Paternalism persists. 

I guess the agency has nothing better to do...

articles.latimes.com/2010/may/13/business/la-fi-dna-kits-20100513




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Into the Valley of Death

05/17/2010 09:03 AM |

A last gasp of sanity from Newsweek:

From 1998 to 2003, the budget of the NIH—which supports such research at universities and medical centers as well as within its own labs in Bethesda, Md.—doubled, to $27 billion, and is now $31 billion. There is very little downside, for a president or Congress, in appeasing patient-advocacy groups as well as voters by supporting biomedical research. But judging by the only criterion that matters to patients and taxpayers—not how many interesting discoveries about cells or genes or synapses have been made, but how many treatments for diseases the money has bought—the return on investment to the American taxpayer has been approximately as satisfying as the AIG bailout. "Basic research is healthy in America," says John Adler, a Stanford University professor who invented the CyberKnife, a robotic device that treats cancer with precise, high doses of radiation. "But patients aren't benefiting. Our understanding of diseases is greater than ever. But academics think, 'We had three papers in Science or Nature, so that must have been [NIH] money well spent.'?"

More and more policymakers and patients are therefore asking, where are the cures? The answer is that potential cures, or at least treatments, are stuck in the chasm between a scientific discovery and the doctor's office: what's been called the valley of death.

And wither the Reagan/Udall Foundation?

Just sayin ...
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Gene Genie

05/17/2010 08:12 AM |

According to the Pink Sheet, “A particularly public pre-emptive stance” by FDA's in vitro diagnostics oversight office halted Pathway Genomics' plans this week to start selling genetic screening test sample collection kits out of Walgreens stores, but the office's director stresses the move signals no changes to its enforcement policy for laboratory-developed tests.

Walgreens' decision came after FDA sent Pathway a letter questioning the approval status of the tests and after Alberto Gutierrez, director of FDA's Office of In-Vitro Diagnostic Device Evaluation and Safety, provided explicit statements to national media outlets that Pathway was flouting FDA regulations.

The firm, which already sells similar sampling kits and services to consumers over the Internet, says it does not need FDA go-ahead because the offerings qualify as traditionally non-agency-regulated laboratory-developed test services and the company is not making diagnostic claims that require FDA oversight.

But, per Gutierrez, the crucial factor is that once a patient, rather than a physician's office, is tasked with taking the sample, all arguments that a product is not an FDA-regulated IVD go out the window.

He added, however, that there is no need for the agency to buck precedent on lab-developed test oversight to go after Pathway, or, likely, other companies that sell similar genetic screening services over the Internet, including 23andMe and Navigenics.

Hm.

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From the Taste of One’s Own Medicine Department

05/14/2010 04:11 PM |

Mr. Pot meet Ms, Kettle.

The Food and Drug Administration has again denied Teva's citizen's petition asking the agency to block approval of a generic version of its Copaxone multiple sclerosis treatment.

 

The Israeli drug giant filed the letter in November after its initial attempt failed earlier in 2009. The FDA rejected the second petition for the same reason as the first, because it would be "premature and inappropriate" to grant Teva's requests, but gave a detailed response that rejects many of the company's arguments against the generic approval.

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Dublin Down

05/13/2010 08:30 AM |

The Irish Medicines Board has decided to support generic substitution.

According to IMB Chief Executive Pat O'Mahony, "While the IMB ... has no involvement in product pricing structures or agreements, we welcome any initiative that improves value for money and ensures the continued provision of innovative medicines as they become available.”

“Value” to whom?  And, last time we looked, such a policy actually strikes against innovation.

Mr. O’Mahony would be wise to remember the old Irish proverb, “Everyone is wise until he speaks.”

In the meantime, just across the Irish Sea in Great Britain, our regulatory cousins at the MHRA have are concerned about generic substitution as well – specifically for oral tacrolimus. The MHRA says it has received 12 case reports of prescribing or dispensing errors associated with the immunosuppressant, oral tacrolimus, which has a narrow therapeutic index.

There are three different oral formulations of tacrolimus available in the UK, but they are not interchangeable, and switching between them should only be performed under the close supervision of a transplant specialist, the MHRA recommends. The reports have included four cases of acute rejection reaction, three cases of increased drug levels, and two cases of increased creatinine.

Generic drugs are a hugely important part of the healthcare paradigm – but bioequivalence cannot be ignored.

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Pathway Genomics and Walgreens: New Path to Personalized Medicine?

05/12/2010 11:22 AM |

Anti-pharma Luddites -- in an ideologically induced haze -- can't decide whether to call for additionally safety studies (PPIs) or to call them off (Avandia) consistently ignore the value of using gene testing to personalize drug response and optimize care.  The reason is obvious.  Keeping people in the dark about whether a medicine will help a lot or a little, or help or hurt breed uncertainty and uncertainty is the wellspring of fear and fear is what allows the Luddites to continue their very profitable campaign against commercialization.  If people know or knew the relative risks and benefits of medicines, then the fearmongering would be ineffective..

Hence, the launch of Pathway Genomics gene test for drug response and disease risk via Walgreen's (and CVS) is most welcome.  From what I can tell, Pathway's approach is medically and scientifically responsible.  Their scientific management is top-notch and careful.  And spreading the knowledge of what drugs or doses can help or hurt is, like cholesterol levels, information that can advance the public health.

But of course it will have it's critics.  And enemies. 

Which is why the FDA "investigation" of the Pathway product - now -- after it has been on the market for over a year should raise eyebrows.  Given Peter Lurie's connection with Luddites like Goozner, Wolfe, Mahar, Diane Zuckerman etc. it would and will be interesting to see how these "experts" who of course have no bias whatsoever weigh in on the Pathway-Walgreen's alliance.  And it will be interesting to see if the FDA, which seems to be involved in everything but advancing The Critical Path and regulatory science these days,  makes a federal case out of a science-based effort to educate patients about the important role genes and genetic response plays in response to medicine and prospective health.

I have a hunch how Maggie Mahar will react. 

www.pathway.com/more_info/health_test#q6
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Ipcress File Part 2

05/12/2010 10:42 AM |

Just because you’re paranoid doesn’t mean they’re not out to get you.

In keeping with yesterday’s announcement by the FDA that they’re going to enlist physicians in ferreting out “bad ads,” it’s worth revisiting the recent JAMA article by National Institute of Mental Health director Tom Insel, “Psychiatrists’ Relationships with Pharmaceutical Companies” (March 24-31, 2010).

To combat Pharma’s nefarious “culture of influence,” Dr. Insel wants the NIMH to develop an online curriculum that will sensitize psychiatric residents to Big Pharma’s nefarious “culture of influence.”

And, while we’re on the subject of content creation, maybe the FDA should consider publishing an annual report entitled, “Best ads, worst ads.”

On the other hand, maybe I shouldn’t give them any ideas.

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And in your spare time ...

05/11/2010 01:09 PM |

Now that's what I call counter-detailing.

FDA: ‘Bad Ad Program’ to Help Health Care Providers Detect, Report Misleading Drug Ads

The U.S. Food and Drug Administration today launched a program designed to educate health care providers about their role in ensuring that prescription drug advertising and promotion is truthful, and not misleading.

The Bad Ad Program is an FDA-sponsored educational outreach effort administered by the agency’s Division of Drug Marketing, Advertising, and Communications (DDMAC), in the FDA’s Center for Drug Evaluation and Research.

“The Bad Ad Program will help health care providers recognize misleading prescription drug promotion and provide them with an easy way to report this activity to the agency,” said Thomas Abrams, director of DDMAC.

The program will be rolled out in three phases. In Phase 1, DDMAC will engage health care providers at specifically-selected medical conventions and partner with specific medical societies to distribute educational materials. Phases 2 and 3 will expand the FDA’s collaborative efforts and update the educational materials developed for Phase 1.

The FDA’s traditional regulatory activities for monitoring prescription drug promotion primarily rely on review of promotional pieces submitted to the agency by sponsoring drug companies, industry complaints, and field surveillance at large medical conventions. Although these efforts are effective, the agency has limited ability to monitor promotional activities that occur in private.

Health care professionals are encouraged to report a potential violation in drug promotion by sending an email to badad@fda.gov or calling 877-RX-DDMAC. Reports can be submitted anonymously; however, the FDA encourages providers to include contact information so that DDMAC officials can follow-up, if necessary.
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They laughed when I sat down at the piano ...

05/11/2010 10:53 AM |

Two years and change ago, drugwonks raised the issue of companion diagnostics as part of the NDA process. The result was a collective yawn.

That was then, this is now.

At last week’s Windy City BIO bar mitzvah, Nicholas Dracopoli, VP-biomarkers at Centocor commented that all new drug candidate programs within J&J/Centocor (home to J&J's biotechnology, immunology and oncology drug R&D programs) will include a strategy for linking the medicine from its earliest inception to a biomarker that identifies the most appropriate treatment population.

According to Dracoploi. "We are now firmly committed in new programs entering into our portfolio that there is an integrated diagnostics strategy that is evaluated ... from preclinical into early clinical testing and into approval …We are looking to do selective partnering with companies, often for specific platforms, technologies and assays.”

And, per the Pink Sheet, “On the other hand, Dracopoli stressed that the company is not, in general, attempting to incorporate diagnostics into mid-stage development programs that got off the ground previously without a biomarker program. The history of trying to retrofit tests into already established R&D programs is not very successful, he said."

On the FDA front, Peggy Hamburg has raised expectations that a draft guidance addressing drug-diagnostic co-development will be issued by year-end. At BIO, FDA in vitro diagnostics office staffer Elizabeth Stafford said she and her colleagues are "very serious" about meeting that deadline.

Can you hear me now?

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Rash of Fearmongering About Diapers

05/10/2010 05:33 PM |

As someone who changed his share of diapers (with one hand, no less) I can attest to the shock and horror of dealing with your baby's diaper rash.  Back in the day it was a matter of if, not when, an infant would get a terrible rash, usually caused by way too much juice, not enough breast feeding, or being lazy about changing the diaper (I justified it by assuming that super asorbent diapers could hold 20 gallons of fluid).  And when the rash hit, it was a matter of keeping baby dry, using the right ointment and being more vigilant about changing diapers when wet.  

Along the way, diapers did get better at helping reducing the incidence and severity of diaper rash. " Infant diapering practices have evolved from the use of cloth (covered with plastic, impermeable over pants), to disposable diapers with a cellulose core and a plastic outer cover, to disposable diapers with highly absorbent polymers (known as absorbent gelling material and referred to as AGM diapers) and to AGM diapers with a permeable or 'breathable' outer cover."

The bottom line (no pun intended) is convenience.  A recent WSJ blog about the displacement of cloth diapers with disposables put the shift in perspective:

"Cloth diaper options have also proliferated, as manufacturers make cloth diapers that are easier to change, with Velcro tabs like disposables. The jury is still out, however, on whether cloth is truly greener. While cloth diapers don’t hog up space in landfills like disposables, they suck up energy and water to launder. (A look at some of the research on the impact of cloth vs. disposables, and a review of some of the new easier-to-use cloth options, can be found in this Boston Globe article and this Wall Street Journal review.)"

So P&G launched a leaner diaper called DryMax to take up less landfill space and is more absorbent than Cruisers or Swaddlers. 

In advance of the nationwide product launch, a handful of parents used facebook and twitter to claim that the new product was causing terrible diaper rash, and nothing else... not the poop, the pee or parental forgetfulness...  Because parents have never tried to shift responsibility for things out of their control onto other people or corporations...   It was probably not smart strategy for PG to simply switch a product parents like and trusted without a lot of advance notice or leaving the old one on the shelf.   People don't like abrupt transitions. 

But that has nothing to do with the specious claims that the new diaper is designed to cause diaper rash or the willingness of people to spread fear through the Web or the media to whip the fear up without putting the real risks or veracity into context....

Here's an example of one of 718 complaints on one website:  "Both my kids had this same thing, but it was all over! Their leg area, butt, front end, just everywhere where the diaper touched."

A classic example of how a large corporation (P&G) is willing to produce and market a product that harms babies just to make a proft!

Except that complaint wasn't about Pampers Dry Max.  It was about Huggies.  In 2008. 

Here's another:  "My middle child had a similar problem with Huggies! However, he would get a rash in his entire diaper area. We used Pampers instead. After a few weeks I got brave and tried the Target brand, and they were fine.
My middle child was the only one that had a reaction to Huggies. (I have 3) So, yes, it is strange!
Since your son's rash is only around the elastic, maybe it's because he's hot. We've been in such a heat wave lately. "

www.mamapedia.com/article/anyone-tick-s-kids-have-rash-from-huggies

Want more?  Here's one of a bunch blaming diaper wipes.

"Both of my girls developed diaper rash, I was using Huggies Natural Care Baby Wipes hoping they were gentle enough. My girls would scream and bleed when I would change their diapers and I just thought the diaper rash really hurt. We quit using these wipes and their rashes have started to heal up. We are using organic wipes, and they really seem to be helping. I think the Huggies may have alcohol in them or something.  "

www.viewpoints.com/Huggies-Natural-Care-Baby-Wipes-review-b908

If you want to read the complaints about Pampers you can go to, of all places a pro-cloth laundry site or the website of a law firm seeking clients who want to sue P&G for bottom abuse:

dirtydiaperlaundry.com/pampers-dry-max-being-investigated-by-the-cpsc/

www.krclassaction.com/Default.aspx

And here is the AP's very helpful reporting on the matter:

"The Consumer Product Safety Commission started an investigation this week following complaints of babies and toddlers suffering severe and persistent diaper rashes and blisters that resemble chemical burns..."

I am not a doctor or play one... but blisters like that on a baby's tuchus are likely impetigo.   Much like the festering pustules that blister and persistently eat away at objective science, impetigo is very common.  But at least impetigo it treatable.  The fearmongering and willingness to transmit it as "fact" by the media seems to be spreading. 

What's next?  Blaming disposable diapers for autism?

You bet..

"If the increase of autism started in the 70''s then you should also consider what other medications, additives etc was introduced at the same time. Most children get vaccines so sure that would be on the list but since the 70s more children are in daycare, wear disposable diapers etc. These are just things that are involving the child not the home as a whole. Microwave ovens could be on the list too. Fact is that it''s an idea but by no means a smoking gun. A lot of children are forgoing immunizations because fear of autism. Whether they are making the right choice or not is yet to be seen. My son has a mild case of PDD-NOS. He wasn''t beaten or traumatized. He got his regular shots like my other two children did and they experienced no problems at all. For all I know living by the airport might have caused his PDD-NOS. It''s not the only disorder to have skyrocketed over the last 20 years."

www.cbsnews.com/stories/2008/01/07/health/main3684105.shtml
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Wax On, Wax Off

05/10/2010 03:56 PM |

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Hypocrisy on FDA Conflicts

05/10/2010 12:53 PM |

Merrill Goozner, Peter Lurie and Sid Wolfe.  All three have made it clear they believe FDA is simply a tool of Big Pharma, the monolith that according to all three produce huge profits and little public health by pumping drugs into the marketplace with little oversight from FDA on their making or marketing.

All three profit from promoting this position.  Goozner peddles his wares via book sales, lectures, etc.  And Lurie and Wolfe have co-authored something called Worst Pills, Best Pillsm which ,I kid you not , was once offered as a the perfect holiday gift. 

"All three have or had direct influence on the FDA's decisions on medicines and establishing the risk-benefit tolerance of the agency.  Gooz and Wolfe were or are on FDA advisory committees.   Lurie is now in the FDA's Office of Policy in Peggy Hamburg's shop.  

Lurie, a physician, will be the third person with ties to Public Citizen to be engaged by the FDA. Sidney Wolfe, former head of Public Citizen's Health Research Group and editor of Worst Pills, Best Pills (http://worstpills.org/; Nat. Biotechnol. 26, 149, 2008) has been a member of the FDA Drug Safety and Risk Management Advisory Committee since 2008. Also Joshua Sharfstein, the FDA's current principal deputy commissioner, has early ties to Public Citizen from an internship in 1992. Before joining the FDA, Sharfstein developed a reputation as a reformer and an industry critic through his advocacy work in the field of HIV/AIDS and his efforts to limit marketing of pediatric cold remedies."

No conflict or coverage thereof?

Only sweet sounds are heard about their appointments: 

“It can't hurt to have smart people—who may be a little controversial—in government. People [in government] can go into this deep inertia of making only small, incremental movement...you can take [Lurie's] energy and his desire to improve the process to help move things forward.”

Which brings me to the comments of Wolfe and Goozner and the manufactured hit job they help lead against another smart, controversial person, Scott Gottlieb, who is a real doctor who sees real patients, unlike Wolfe or Goozner. 

Here's what Goozner said about Scott's appointment to Alicia Mundy, who is now the go to source for those inside FDA seeking to make life miserable for Janet Woodcock, John Jenkins and others:

"I've never heard of anything like this," said Merrill Goozner, a director at the liberal Center for Science in the Public Interest.

"If he's had dealings regarding companies whose products are up for review at the agency, it strikes me as a potential conflict of interest. You want a barrier between the regulated and the regulators. It's fundamental," Goozner said.

What about intellectual bias?  Note how the Gooz cut himself yards of slack, with Mundy giving him room to do so.  I guess neither knew or cared about the FDA's equally strict restriction on using one's position to skew or bias proceedings to fit a particular, uh, narrative.  Or book sales. Or continued existence as Project Director at certain advocacy groups...Or whatever:

But intellectual bias is a big problem.  Bigger than financial conflicts because, as Aaron Wildavsky observed in Risk and Culture, the adherents of organizations that individuals such as Lurie, Goozner, Wolfe and Alicia Mundy have worked for and work for need enemies, corporate enemies to unify and motive a base sharing a deep cultural bias about the inherent dangers of technology.   To suggest this bias does not exist or is an articulation of objective reality is to cede control of the political and social institutions that regulate all human activity to the aforementioned since their goal is not this regulation or that, but to "break the stranglehold which they consider such interests have on society."  tinyurl.com/33vyzj5

All the more reason that the FDA in it's small way has sought to put a leash on such totalitarian behavior:

"Within the Center for Drug Evaluation and Research, there are provisions stating that members participating on advisory panels should be free from intellectual bias. According to the FDA document, the appearance of intellectual bias is an issue if the member is identified as a "primary advocate" or is "so strongly associated with a position on a matter" that concerns could be raised about his or her impartiality and objectivity.

Also, intellectual bias may exist if "statements of record have been made by that member concerning an issue to be considered by the committee that draw conclusions or strongly appear to draw conclusions to a degree that would appear, to informed experts, to preclude an impartial and objective evaluation of information on that matter presented to the committee." These statements could be taken from legal cases, other regulatory agencies, or even from the media.

As Jenkins told heartwire last week, members are welcome to bring differing perspectives and to ask tough questions, "But at the same time, we want the committee members to come to the table with an open mind, so they can give us advice based on the data, the presentations, and the discussions that are held at the committee meeting itself."

www.theheart.org/article/943511.do

And finally here is Lurie essentially shooting himself in the foot by acknowleding that financial "conflicts" are easily spotted whereas intellectual bias can be disguised as just scientific disagreement:

"Frequently, one hears that there are both financial and intellectual conflicts of interest; somehow this argument is offered as evidence to downplay the importance of the financial conflicts. While intellectual conflicts are important, they can readily be distinguished from financial ones. Financial conflicts of interest are extrinsic to the scientific endeavor, whereas intellectual conflict is the very way science moves forward. Financial conflicts can occur at variable levels – some people have them, some people don’t – and they can be quantified, whereas intellectual conflicts are ubiquitous and not susceptible to quantification in the same way. Moreover, in the context of debate on an advisory committee, for example, it is unlikely that the financial conflict information will naturally emerge, whereas it is likely that any relevant intellectual one will. There are relatively straight-forward methods to alleviate financial conflicts, whereas it’s not nearly as clear how one should approach intellectual conflicts. Finally, our legal system has long recognized the distinctions between the two.
brodyhooked.blogspot.com/2008_03_01_archive.html

Indeed, it has.  The intellectual bias is much more problematic: 

"Two of these definitions are: an individual has a COI when there is a conflict between his/her private or institutional interest and his/her official duties in a position of responsibility or trust; or, an individual is in conflict if he/she owes a duty of loyalty or responsibility to two distinct entities or individuals, both of which are likely to be affected by the scientific activity in which the individual is engaged. The term "interest" can refer to a financial (i.e., employer, employee, consultant, stockholder, investor, etc.) or a non-financial interest/relationship (i.e., family member, mentor, mentee, professional colleague, co-author, etc.). Most existing COI policies address financial but not non-financial conflicts of interest; however, non-financial conflicts of interest are equally common and important, and they warrant, and will likely receive, more attention and a higher degree of scrutiny in the future."

toxsci.oxfordjournals.org/cgi/content/full/87/1/11

The trashing of Scott Gottlieb was part of a jihad against a particular approach to science and risk led by the same people who populate and are influencing the agency Scott served in.   Their cultural biases will and can have consequences for the public health if they are not made as transparent as financial biases. 








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Getting the Finger

05/10/2010 12:00 PM |

I just returned from my annual physical where my internist learned a lot about me and I learned how unhappy she is about ObamaCare. While I was getting it where the sun don't shine, she recognizes that it's America's physicians who are getting the finger.  Read More & Comment...

From USTR ... with Love

05/10/2010 11:34 AM |

The U.S. Trade Representative's most recent report on global intellectual property rights suggests compulsory licensing may be receding somewhat as a worry for pharmaceutical companies, but inadequate data protection remains a strong concern.

The USTR's 2010 Special 301 Report, which reviews U.S. trading partners' protection and enforcement of intellectual properties, cited 19 countries (the same number as last year) where there appears to be a lack of adequate protection against unfair commercial use and unauthorized disclosure of test or other data generated to obtain approval of pharmaceutical products.

The USTR has faced pressure from Congress and public advocacy groups to modify its stand on compulsory licensing and other measures advocated by the pharmaceutical industry.

Representative Henry Waxman (D, CA), chair of the House Energy and Commerce Committee, sent a letter to U.S. Trade Representative Ron Kirk advocating that the Special 301 Report support international access to medicines.

"In recent years, despite the United States' commitment to the Doha Declaration, Special 301 reports have been used to pressure developing countries to adopt pharmaceutical protection rules that go beyond their obligations under the TRIPS agreement, limiting the scope of actions such countries could take to promote access to medicines," Waxman wrote.

Mr. Waxman also said he was concerned that anti-counterfeit measures may be used to enforce patents. Looks like the Chairman has Jamie Love on speed dial.

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Innovative if not Transformative ...

05/07/2010 08:41 AM |

From today's edition of the Wall Street Journal and the pen of CMPI board member Mark Thornton:

Three years and more than 80,000 deaths ago I wrote in these pages about a travesty that had occurred at the Food and Drug Administration.

In May of 2007 two unique cancer therapies for the treatment of prostate cancer and osteosarcoma (a type of bone cancer) came under review at the FDA on the same day. Both the new agent for prostate cancer, Provenge, and the new agent for osteosarcoma, Mepact, had shown the ability to prolong lives to a significant degree. And both drugs were summarily rejected. Provenge and Mepact were tossed back to the companies developing them with the directive to do more clinical studies.

This was easy enough for Provenge, due to the return on the risk of investment possible with a new prostate cancer drug and the large number of men with the disease available for another clinical trial. The Dendreon company, makers of Provenge, worked as quickly as possible to redo their already successful trial. The results of the new trial turned out the same as the original, and the drug was finally approved by the FDA last week.

In the three years that it took to duplicate what was already known, upwards of 80,000 men lost their lives to prostate cancer. This is equal to the number of men killed in combat in the Korean, Vietnam and Iraq wars combined.

Those FDA staffers who had a role in preventing the approval of Provenge in 2007 will have to live with this sin of omission. But at least now a powerful new weapon is available to help prostate cancer patients.

Not so for those of us in the American sarcoma community waiting for Mepact. Osteosarcoma effects only 900 Americans, mostly children and young adults, each year. The FDA's stunningly cavalier demand for another clinical trial for Mepact was made with the full knowledge that the repeat effort would require about 900 new patients.

Imagine if a clinical trial for a new asthma or diabetes drug required every single patient in the country to be included not once but twice and you will begin to appreciate the profound predicament those of us in the rare-disease community face when it comes to the FDA's irrational standards.

Recently, hope has emerged that a more humane approach might be taken at the FDA to approve new drugs for exceedingly rare diseases. In an amendment to the 2010 FDA Appropriations law, Sen. Sherrod Brown (D., Ohio) and Sen. Sam Brownback (R., Kan.) inserted language that requires the FDA to explain the unfairness of its policies, which have resulted in about 200 treatments for the over 8,000 diseases categorized as rare.

In March, the new FDA Commissioner, Dr. Margaret Hamburg, appeared before the Senate to discuss the issue. In emotionally moving testimony about her own experiences as a doctor dealing with patients in desperate need of new therapies, she promised "innovative if not transformative" approaches to the problem, saying that "new regulatory pathways could be developed . . . to catalyze activity in areas where there are limited markets." She added that this issue is "of the highest priority in the White House."

In Europe, such creative pathways for regulatory approval exist, and they have resulted in the approval for Mepact in the EU. Mepact is the poster child for all that is wrong at the FDA regarding treatments for rare diseases, and many have hope that Dr. Hamburg is the right leader to address this health crisis.

The approval of Provenge, a revolutionary agent that taps the body's own immune system to fight cancer, ushers in the dawn of the age of cancer immunotherapy. Mepact similarly stimulates the immune system to prolong osteosarcoma patients lives, and it is doing so as we speak for children in Europe with this bone cancer.

Oncologists in the FDA Center for Drugs should show penance for their fatal error and join their European colleagues in approving Mepact without the need for another 10-year long clinical trial. The Americans currently living with osteosarcoma can't wait any longer.

Dr. Thornton worked as a medical officer at the FDA for six years. He is the president of the Sarcoma Foundation of America, which has received modest contributions from a U.S. subsidiary that is owned by the makers of Mepact.

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We Are the World

05/06/2010 10:36 PM |

Some patently absurd claims have been made in the wake of health care's passage, but New York Times writer Thomas Friedman and Senate Majority Leader Harry Reid are tied for First Place.

Senate Majority Leader Harry Reid is campaigning in Nevada for re-election and continues to tout the health care law. He recently said, “The most important thing we've done for the country and the world is health care.”

Last month, Friedman posited that passage of the health care law strengthened President Obama’s hand in foreign policy:

“Our enemies surely noticed, too. You don’t have to be Machiavelli to believe that the leaders of Iran and Venezuela shared the barely disguised Republican hope that health care would fail and, therefore, Obama’s whole political agenda would be stalled and, therefore, his presidency enfeebled. He would then be a lame duck for the next three years and America would be a lame power.”

Yes, because I’m sure our health care system is an issue very dear to Iranian president Mahmoud Ahmadinejad.

A majority of Americans stood opposed to the law. More than 20 states have joined in a lawsuit against the federal government challenging the law, with particular emphasis on the individual mandate. And polls continue to show no improvement in public opinion of the health care law.

In this country we don’t pass life-changing domestic legislation so that a president may convey political strength overseas.

With that said, it’s highly doubtful that the legislation had the impact Friedman suggests.

Fidel Castro applauded the health care law as “a miracle.” Castro also said, “It is really incredible that 234 years after the Declaration of Independence ... the government of that country has approved medical attention for the majority of its citizens, something that Cuba was able to do half a century ago.”

Here are some recent pictures smuggled out of Cuba highlighting that medical attention Cubans receive.

After praising passage of health care, Castro then attacked President Obama as a “fanatic believer in capitalist imperialism.”

The goodwill didn’t last long.

Now the World Health Organization Director-General Margaret Chan just returned from North Korea with words of admiration for their health care system. Apparently North Korea does an excellent job of keeping its citizens thin.

“They have something which most other developing countries would envy,” said Chan.

Who would have thought?

Then again, most of the world has been praising Cuba’s health care system for the last 40 years.

At any rate, the point here is that we should not decide health care policy in the US based on the misguided perceptions of our system by other countries. And we should most definitely not use domestic policy as a means of strengthening our influence on foreign policy matters.

Senator Reid and Thomas Friedman ought to know better.


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Do Vaccines Cause Autism?

05/06/2010 10:26 PM |

Ed Morrissey at Hot Air has posted an excellent video from Reason TV on the Autism-Vaccine hoopla.

The lesson: Correlation is not causation.

The video is worth checking out:







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A Betting Man

05/06/2010 10:22 PM |

Having missed the televised grilling of Goldman Sachs’ executives last week, I just now happened to come across a stunning statement made at the Senate hearings.

Arkansas Senator Mark Pryor went on the attack in front of the cameras accusing Goldman Sachs executives for “betting with other people's money and other people's futures.”

Senator Pryor failed to recognize the blatant hypocrisy in his accusation.

Here is a United States Senator who voted for the health care bill before he voted against it.

In voting against the reconciliation bill in March, Pryor acknowledged the adverse impact of the legislation:

"I believe the package falls short of the criteria of making health care more affordable, reliable, and accessible. As more and more details of the package were released, I spent considerable time weighing the benefits and drawbacks to Arkansas. In the end, I believe this legislation is a step we don’t need to take."

And this man dares lecture financial sector executives?

Where was his concern for “other people’s money” and “other people’s futures” when he voted in favor of the Senate health bill last year?

Mind you, Pryor had a fiduciary duty to his constituents to read and understand this far-reaching and costly legislation. He failed to do that and instead gambled with one-sixth of the US economy.

Some might argue that is far worse than any transgressions by Goldman Sachs.

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BIO con brio

05/06/2010 09:55 AM |

At the BIO conference yesterday I had the pleasure of chairing a panel entitled, “Advertising or Freedom of Expression?  Cross-Border Communications and the New World Order.” And a high-powered panel it was.  I was joined by Jack Bierig (Sidley Austin), Marie Kennedy (Baxter) and Ray Kerins (Pfizer). 

An audio recording of the panel can be found on the BIO site (www.bio.org).  

Here are my remarks:

If healthcare is a “right,” then is access to information about healthcare also a right?

Well, where you stand depends on where you sit and, for biopharmaceutical companies, it determines how you act.

And nowhere at BIO 2010 will you find a more lively, engaging, and expert panel to debate this timely and crucial issue than right here and right now.  Welcome to BIO con brio.

What’s going on in the US?  Does the First Amendment still count?  What’s going on in the EU?  Does “information to patients” really mean anything?  In the complex and hyper-bureaucratic world of Brussels, is reform going anywhere beyond just words?

And when it comes to healthcare communications, wither social media on either side of the pond?

Let’s start with a look at what’s going on in Europe.

The situation “over there” can be summed up by James Copping of the European Commission’s Enterprise & Industry Directorate. According to Mr. Copping:

“We have an unsustainable mix of regulations, and the legislation on advertising was largely drafted in the 1980s, before the Internet became a daily feature of many people’s lives.”

In 2006 I debated Mr. Copping. He said, “We must find new ways to regulate healthcare information to patients.” I then suggested that a better way to frame that statement would be to say that “the EU needs to find a better way to facilitate healthcare information to patients.”  To which Mr. Copping replied, “Yes, that’s right.”

Well, that was then and this is now.  But before we see what’s sprouting in pursuant to the shifting of ITP authority from Enterprise & Industry to DG SANCO, let’s turn our attention northwards to Denmark for a disturbing twist and a reminder that “free speech” means something very different in Europe then it does here in the US.

In 2003, Danish journalist Frede Damgaard published information on his website about Hyben Total, a treatment for a wide range of conditions—including gout, kidney and bladder disorders, sciatica, diarrhea, and diabetes. The Danish national regulator in 1999 refused it a marketing authorization. It is still sold as a medicine in Sweden and Norway.

 

Mr. Damgaard’s positive description of Hyben Total’s effects on the symptoms of gout and arthritis led to his being prosecuted in the Danish courts on the grounds that it constituted advertising of a medicinal product whose sale was not authorized in Denmark – and the Danish court specifically cited Article 86 of European Union Directive 2001/83/EC, which defines the concept of medicines advertising.

 

Mr. Damgaard appealed to the European Court of Justice, claiming that his discussion of Hyben Total could not be held to constitute advertising since he had no interest in selling the product, and also that the court decision contravened European Union protections of freedom of expression.  And the ECJ agreed.

 

According to the opinion handed down by the ECJ Advocate General, “a lack of connection between the author of the information and the sellers or manufacturers of the medicinal product and the non-commercial or non-industrial nature of the activity of that independent third party may…be strong indications that a message does not have promotional content.”

 

The advocate general added that this was not an isolated case; similar situations had arisen recently over statements about melatonin in Spain, and in the Czech Republic, over a collection of media features entitled “Yesterday Viagra, today Cialis.”

 

The advocate general concluded that it’s up to individual EU member states “to ensure the correct balance between, on the one hand, the objectives of protecting health and promoting the rational use of medicinal products and, on the other, the right of the party concerned to freedom of expression, taking into account the special protection afforded to the party concerned, if it is established that he is a journalist.”

 

But how do you “establish” who is and who is not a “journalist?”

 

Not a complete victory for the Fourth Estate and uncomfortable silence from the court about free speech for non-journalists. What about free speech for physicians and patients and, yes – even biopharmaceutical companies?

 

And so we return to Brussels.

 

Recent reports have spoken about "renewed optimism" that the EU’s proposed legislation on allowing drug makers to provide information to patients on prescription-only medicines will again start moving through its treacle-like legislative process. The latest thinking, however, is strongly focused on the rights of patients to receive such information, rather than industry's right to disseminate it.

That’s an interesting and important finesse – the rights of a patient to the information but no “right” for industry to provide it.  Hm.

Suggested amendments to the most current consultation document emerged on March 10^th from M.E.P. Christofer Fjellner, who’s reviewing the proposed legislation for the EU parliament's Committee on the Environment, Public Health and Food Safety.

Fjellner’s position is that information on pharmaceuticals should only be made available to patients who are actively searching for it.  In other words, information should be "pulled" by the patient rather than "pushed" by industry.

 

Fjellner believes that companies should not be allowed to provide information on prescription-only medicines on television or in newspapers or magazines. He believes the Internet is the appropriate medium for providing information to patients.

And maybe he's right -- but is there really a difference? 

 

If a pharmaceutical company makes available information on a web page – why is that different than making it available in other media?  And what about patients who do not have access to the Internet -- what about their rights?

 

And what are the ramifications for social media? “The Internet” is, after all, only the substrate.

 

On the upside, Fjellner is calling for patient organizations to be actively involved in implementing the new legislation, working to help create guidelines and a code of conduct. Bravo.

 

But needless to say (and important to share) is the hue and cry this last suggestion elicited from the anti-ITP crowd who immediately objected to the participation of patient groups because of the financial support they receive from “interested parties.  And you know who you are.

 

The environment committee will vote on the patient information proposals this June. After that a plenary vote of all European Members of Parliament will be held in September. It will then go to the Council of the EU for further consideration. Process as proxy for action.

 

The battle lines are drawn. Brussels moves in mysterious ways and “consensus” is a very un-American concept. 

 

EU president Herman Van Rompuy is often referred to as “Haiku Herman” frequently ending his speeches with a composition of this own. And so, in keeping with the transatlantic nature of the topic at hand, let me do the same:

 

Health information.

When will Brussels move forward?

We watch and await.

 

Thank you.

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USP's USP

05/05/2010 07:25 AM |

USP also means "Unique Selling Proposition."

Pleased and proud to share the news that CMPI senior fellow Tim Franson, MD has been elected President of the USP Convention.

 

As most of you already know, USP is a scientific nonprofit organization that sets standards for the quality of medicines, dietary supplements and food ingredients. USP’s drug standards are enforceable by the Food and Drug Administration in the United States and USP standards are used in more than 130 countries around the world. USP standards designate the quality, purity, strength and consistency of a medicine, food ingredient or dietary supplement for the benefit of patients, practitioners, manufacturers, and consumers. 

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