Yes CER

08/31/2010 09:43 AM |

Take a breath.  Some news about the FDA and comparative effectiveness.  Relax – it’s not what you think (or what the people at Consumer’s Union want).

By the end of September, the FDA will launch several initiatives to aggregate data on medical products, assess the information and eventually answer a myriad of questions on patient populations and class-wide issues, according to agency officials.

The new efforts will not affect product approvals and instead focus on simply answering a slew of outstanding questions by leveraging the abundance of collected data stored at the agency.  And it’s about time.  The FDA sits at the nexus of vast amount of untapped and highly important data. And while data is great, knowledge is power. 

The American Recovery and Reinvestment Act (aka, “the stimulus package”), the FDA received funding (via AHRQ) to develop a CER infrastructure with outside assistance.

The agency's multi-step CER approach that includes creating the Janus data repository and developing at least one external center through the Partnership in Applied Comparative Effectiveness Science (PACES) initiative to examine the collected data, assess the information and make recommendations to answer questions on different therapies and patient groups. The data will include information from new product submissions and previously submitted product applications.

The Janus data repository will serve as the crux of the program and a "hub" that aggregates a substantial portion of collected agency information. One of the FDA officials described the data aggregation design as a "federated model" that will take advantage of the wealth of agency data collected for decades.

The data will come from standardized electronic product applications, previously submitted products, the Sentinel post-market analysis system, the MedWatch program, the common electronic document room, the automated laboratory management system and other data sources, officials said.

In parallel to the creation of the Janus data repository, the agency will also convert legacy data into a standardized format that can be inputted into the system. Agency officials acknowledged that the data retrofit will be costly, and the ARRA funding will focus on piloting the initiative to determine whether the program's benefits outweigh its costs. But that depends, largely, on how the agency chooses to define "benefits" -- and over what period of time?

The program will not aggregate data on unapproved drugs, and only focus on new submissions and retrofitting information from approved products that were previously introduced on the market.

It’s a smart move.  The more information the agency has on both individual and class MOAs the better it can understand how things work in the real world (beyond the neatly designed, gold standard world of RCTs).

FDA science adviser Vicki Seyfert-Margolis said the program is not focused on directing regulatory actions, restricting randomized controlled clinical trials or limiting access to healthcare services.

Instead, she said the program will address the real world impact of therapies, help improve consistency and transparency in the approval process and identify healthcare gaps. For example, the examination of data could identify sub-populations that are not impacted by certain classes of drugs, with those patients potentially obtaining improved health outcomes from different therapies, such as certain devices.

Pulse check – this is not CER as part of the PDUFA process.

Janet Woodcock: "I think the science is still too early to be able to really design comparative trials that stand much chance of being conducted, at least pre-approval.”

According to FDA Week, “Agency sources said the Janus data repository and the PACES centers are not intended for use in making product-specific decisions in the premarket arena, and instead will simply enable the agency to understand the science behind certain classes of drugs, how they compare to other treatment options in patient subpopulations and assess the effect of genetics on therapies.” This is about personalized medicine [which is] a major new area of investment," an agency official stressed. "We're not using this to do cost assessments."

Personalized medicine?  Well, yes – if you consider the use of outcomes data to be personalized medicine.  And it is.  It’s certainly an important first step towards realizing the “four rights” (right medicine in the right dose for the right patient at the right time).

The FDA will not release product-specific data from this initiative to the public, but the agency hopes to publish information on general CER methods and strategies developed through the PACES program. The agency could also (and should) release answers to questions on the impact of genetics on certain therapies and class-wide observations.

How does this relate to the Critical Path initiative? 

FDA Chief Scientist Jesse Goodman: "In the long run for FDA and the sponsors, this will make everything more efficient. … Moving towards identifying what's the right way to do it does take some maturation of technology.”

Oh yes. And to that end the FDA will strive to implement “modern analytical tools” to examine the data. Easier said than done – but it’s money well spent.

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The Victims of "Bad Science"

08/30/2010 08:19 AM |

"Bad science conducted to support litigation."

Via the AP -- and without mincing words ...

A federal appeals court on Friday upheld a ruling that vaccines are not to blame for autism.

The U.S. Court of Appeals for the Federal Circuit upheld a decision last year by a special vaccine court, which concluded there's little if any evidence to support claims of a vaccine-autism link.

Scientist years ago reached that conclusion, but more than 5,500 families sought compensation through the government's Vaccine Injury Compensation Program.

Friday's ruling came in the case of Michelle Cedillo of Yuma, Ariz., who is disabled with autism, inflammatory bowel disease and other disorders that her parents blame on a measles vaccine given at 15 months.

In the 2009 ruling Special Master Denise Vowell wrote that the evidence "is weak, contradictory and unpersuasive. Sadly, the petitioners in this litigation have been the victims of bad science conducted to support litigation rather than to advance medical and scientific understanding" of autism.

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That obscure object of desire

08/27/2010 09:58 AM |

What is that obscure object of desire?  Could it be ... risk information?

In the belief that facts drive guidance and oversight on behalf of the public health, some interesting and important data on attitudes towards risk and benefit in DTC ads.

(Note – key word is “ads” – not social media.)

Attitudes toward Risk/Benefit Info in DTC Ads

TV       Magazine      Online

 Risk

Seen/heard                                            79%         48%              37%

Pay a lot/some attention                         76%         66%              69%

Say it is very/somewhat useful                76%          75%              75%

Benefits

Seen/heard                                           73%           52%              54%

Pay a lot/some attention                        63%           63%              57%

Say it is very/somewhat useful                75%           76%              76%

Source: Rodale, "2010 DTC Study," July 15, 2010

What does this tell us?  Well, on the “benefit” side, it seems to suggest that consumers rank all three media equally when it comes to utility (“say it is very/somewhat useful”).  And while TV and print still seem to have an edge in the “pay a lot/some attention” department (at 63%), online ads are a very close show at 57%.  Decimal dust?  Not when you’re doing ROI calculations -- but it’s really only a half-game lead – and the momentum is shifting online's way.

In the “seen/heard” department, TV leads both print and online – but online beats print – further adding to the “death of print” argument.

With “utility” almost even across the board (76%/75%/75%), it’s the risk stats that give pause for reflection. 

While 79% have “seen/heard” risk information on TV ads (not surprising since the viewer passively receives it whether they want to or not), and print (48%) out-strips online (37%). 

But the race tightens in the “pays a lot/some attention" – where TV still leads (we are, after all, glued to the couch), but online (69%) out paces print (66%).  Among other conclusions, this points to (1) the well-documented inadequacies of the brief summary (i.e., neither brief nor a summary) and (2) the greater desire of the online ad viewer to click to risk information.  Put another way –nearly 70% of online ad viewers actively click-through to risk information.  Pretty impressive – and even more so considering this data was collected well after the November 2009 FDA letters on sponsored Google links.

Food for thought.

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Ties that bind = Ads that stick

08/27/2010 08:30 AM |

Hello Muddah.  Hello Faddah.

A new study in the August issue of the Journal of Applied Communication Research (conducted by researchers at the Universities of Pennsylvania and Georgia) shows that direct-to-consumer ads that make mention of family history as a significant risk factor for a disease resonates strongly with their target audiences.

Those who viewed the ads indicated a stronger intent to adopt healthy behaviors as well as a stronger intent to seek the medications that were advertised. The researchers concluded consumers were willing to mix pharmaceutical and behavioral changes to minimize their risks to diseases that could be related to genetics.

The study involved a group of 395 adults who viewed four magazine ads, including three for drugs: Bayer aspirin for heart disease, Vytorin (ezetimibe/simvastatin) for high cholesterol, and Actonel (risedronate sodium) for osteoporosis. The fourth was a "masking" ad for ThermaCare , an over-the-counter product for joint and muscle pain.

All participants who saw the ads with familial risk cues, regardless of the level of family disease history, showed a strong intent to purchase medications. The results also indicated that that the consumers who saw the ads with familial risk cues had stronger intentions to engage in healthy lifestyles than those who did not.

"Exposure to familial risk cues in DTC prescription drug ads enhanced behavioral intentions for both healthy lifestyles and for pharmaceutical choices," the article states.

Funding was provided entirely through a grant from the National Cancer Institute.   Read More & Comment...

AARP Drug Price Study Getting Old

08/26/2010 01:58 PM |

Does anyone pay retail or sticker price for medicines and does no one buy generic drugs?

Only in AARP's real world.

Retail prices for some of the most widely used brand-name prescription drugs shot up more than 8 percent in 2009, even as inflation plummeted to a record low, according to a new AARP analysis of retail drug price trends released today. The AARP Rx Price Watch Report also looked at retail drug prices over the past five years and found some huge increases for popular drugs including the prostate drug Flomax, which nearly doubled in price; Advair and Aricept saw price hikes of 40 percent. During the same period, the consumer price index rose 13.3 percent. The findings show that the cost of prescription drugs—many widely used by those on Medicare—far outstripped the price increases for other consumer goods and services. … The AARP report found that all but six of 217 brand-name prescription drugs had retail price increases exceeding general inflation last year.

Conducted by long time pharma-price schlockmeister, Stephen Schodelmeyer,  the "study"  ignores the fact that the 217 brand name drugs were actually a smaller number of medications with different doses and that for most, if not all of the drugs, there was a generic alternative.   It also ignores the fact that almost no one pays retail or the full cost of a medication or that the retail price includes drugstore mark ups.. 

Meanwhile, another reliable survey, this from Consumers Union, claimed that 27 percent of people skipped or split medications to save money.  Then again, up to half of people who actually have drug coverage skip or stop taking medications.   Did Consumers Union consider other reasons than money? 

And by the way, it's survey showed that "a whopping 81 percent said they are concerned about the rewards drugmakers give to doctors who write a lot of prescriptions for a company’s drugs. And 72 percent were displeased with payments pharmaceutical companies give to doctors for testimonials or for serving as a company spokesperson for a given drug."

Rewards?  Do they mean the pens or the calendars?  And how much of a drug do you have prescribe to get such a nifty prize.    You see, that's what passes for rewards these days since companies no longer hand out bags of jewelry and cash. 

The coverage of these studies in the media was free of any analysis or context.  Instead the journalists simply rewrote the press releases of the two organizations and linked to them in blogs... (You can read about both in the Kaiser Healtn News service which also substitutes for health care reporting in major newspapers around the country.)

Way to go. 

www.kaiserhealthnews.org/Daily-Reports/2010/August/26/Drug-prices.aspx
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Sullivan's Travails

08/26/2010 08:51 AM |

In a 33-page opinion, Judge Emmet Sullivan of U.S. District Court in Washington, D.C., declined to issue a preliminary injunction blocking the sales of the generic Lovenox.

 

sanofi-aventis sued the FDA after the agency granted Sandoz approval to make enoxaparin (the generic form of the special heparin).

 

s-a contended that the FDA didn’t follow its own regulations in granting the approval. s-a, interestingly, didn’t claim that the Sandoz product was unsafe. Rather, they argued that the enoxaparin made by Sandoz is not exactly the same drug as Lovenox and therefore should not be approved or marketed as a copycat version of the blockbuster.

 

But has anyone ever argued that either traditional Hatch-Waxman generics or FOBS need to be 100% bioequivalent?  Indeed, is that even possible?  No and no. It is equivalence of functionality based on activity. The generic or the FOB has to work the same way and be designed in ways that produce such functional equivalence. But that doesn’t mean that interchangeability in prescribing is always appropriate.  That, however, isn’t a legal matter.  It’s a therapeutic one.

 

Judge Sullivan ruled that the FDA had not acted inappropriately in approving enoxaparin. "Just because the FDA … reached a conclusion at odds with the position advanced by Sanofi does not mean that the FDA's decision was arbitrary and capricious," he said, noting that it has been seven years since s-a filed a citizen's petition at the FDA objecting to generic versions of Lovenox.

 

He said, the FDA had presented "a satisfactory explanation for its decision." Judge Sullivan noted that none of the parties had challenged the safety of the generic enoxaparin.

 

And that’s the best victory for both the FDA and the public health.

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Between a Roche and a Hard Place

08/25/2010 04:32 PM |

A social media platform from Roche?  Well, kinda sorta. But it's certainly a step in the right direction.

For more on the issue, see here.
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A Label Fable?

08/25/2010 07:48 AM |

Yesterday the FDA released a preliminary draft of the restaurant menu labeling rules that will go into effect on March 23, 2011. A section of the health care legislation passed in March mandates that restaurants clearly display calorie information on menus, including sit down and drive through menus.

The new rules will apply to many different types of eateries. The general rule is that any restaurant with twenty or more locations is required to label products with calorie information.  The rule also includes vending machines.

Senator Tom Harkin (D-IA) and Congresswoman Rosa DeLauro (D-CT) co-authored the legislation upon which the draft guidelines are based back in 2003.

"This is a great step forward towards ensuring that Americans will be able to make more informed choices about the food they are eating, which will help to combat obesity, cut health care costs, and improve and enhance our lives. The FDA's efforts in making this a reality are admirable, as is the cooperation and participation of the restaurant industry as we move forward," said DeLauro in a press release from her office. "With childhood obesity rates tripling over the last 30 years, this legislation is absolutely imperative to the health of our nation."


Well – except that the research to back up the childhood obesity claims is, well shall we say – inconclusive.


"The issuance of today's guidance regarding menu labeling is an important step in efforts to empower consumers by giving them the information necessary to make sound decisions about their health," said Harkin in a statement.  

Well – except that the research to back up the claim that people alter their habits after reading such labeling (if they, in fact, read them at all) is, shall we say – inconclusive.

We shall see. Stay tuned for the open public comment period.

The draft guidelines can be found here.

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The Ambassador of Death

08/24/2010 05:58 PM |

Suddenly end-of-life care is back in vogue.

Here’s the New York Times explaining why: 

In a study that sheds new light on the effects of end-of-life care, doctors have found that patients with terminal lung cancer who began receiving palliative care immediately upon diagnosis not only were happier, more mobile and in less pain as the end neared — but they also lived nearly three months longer.

The findings, published online Wednesday by The New England Journal of Medicine, confirmed what palliative care specialists had long suspected. The study also, experts said, cast doubt on the decision to strike end-of-life provisions from the health care overhaul passed last year.

 

“It shows that palliative care is the opposite of all that rhetoric about ‘death panels,’ ” said Dr. Diane E. Meier, director of the Center to Advance Palliative Care at Mount Sinai School of Medicine and co-author of an editorial in the journal accompanying the study. “It’s not about killing Granny; it’s about keeping Granny alive as long as possible — with the best quality of life.”

 
Here’s what the study found and what the New York Times conveniently ignores: People with end stage lung cancer who were given palliative care at diagnosis and  simultaneously with standard cancer care  “had a significantly better quality of life and significantly lower rates of depression than those who received only standard care.

They also lived longer — median survival for patients in the simultaneous-care group was 11.6 months and in the standard-care group was 8.9 months (P = .02). This survival benefit of 2.7 months is similar to that achieved with standard chemotherapy regimens.”  (www.nejm.org)

The New York Times  reporting makes it seem that palliative care alone was better and that it was therefore wrong to eliminate end of life counseling from Obamacare by calling it a death panel.

In fact, end of life counseling in the original version of Obamacare was not about  “keeping Granny alive longer” or even palliative care.

 

Section 1233 of the health-care bill drafted would have paid doctors to give Medicare patients end-of-life counseling “every five years -- or sooner if the patient gets a terminal diagnosis.” 

 

And the counseling was to include advanced care planning, including key questions and considerations, important steps, and suggested people to talk to about” living wills and durable powers of attorney, and their uses …a list of national and State-specific resources to assist consumers and their families."   Not a word about living longer. To suggest now that’s what Democrats meant is absurd: If spending more money to let Granny live longer after a terminal diagnosis  why keep reminding people every five years about “living wills”?

Because it’s a way of telling seniors as they get older that living longer is not very valuable.  Here’s Victor Fuchs, an Obamacare advocate, economist and long time consultant to Donald Berwick and Obama’s health policy adviser Ezekiel Emanuel on technologies that extend life:

 

“..further gains in life expectancy will mostly mean keeping more Americans alive while they are retired and dependent on indirect transfers of funds from younger workers for much of their living expenses, health care, and social services.”   Because keeping people alive longer is so…wasteful Fuchs suggests government discourage “ innovations that increase life expectancy”  in favor of innovations, such as joint replacement, that improve the quality of life for both the elderly and the near-elderly.”

 

This is ideology masquerading as science.  In fact, advances that improve quality of life also tend to improve survival especially when it comes to diseases associated with aging.  And it winds up reducing or slowing the cost of treatment.   Since 1996, the average per patient costs for cancer, heart disease and mental illness have declined in inflation adjusted dollars.  And life expectancy continues to increase as well. 

 

But that’s not good enough for Fuchs,Berwick and others.   And just because of end of life counseling is gone, Obamacare has other tools to shorten life.  One way to do it is to have the government not pay for any new technology that doesn’t meet this goal.  Another is to not count spending on such innovations when determining if a health plan spent the federally required 80-85 percent of it’s premiums on medical care.   Still another is paying doctors to discourage people from using new technologies by discussing their risks and lack of value.   

 

Fuchs states: Obamacare should only pay for   “innovations whose main effect is to substantially decrease cost while holding quality constant or reducing it only slightly.”   Many Obamacare advocates endorse his view with enthusiasm.  Yet by that standard, a combination of palliative and standard care that increases well-being and extends lives would be discouraged by government.   Maybe the term “death panel” understates the problem.

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Hyper(tension) Drive

08/24/2010 08:34 AM |

According to three new studies in the latest issue of the American Journal of Hypertension, almost half of the 50 million Americans with hypertension haven't been prescribed the drug that would work best for them.

 

"Our current prescribing methods are very primitive. We haven't increased the success rate [in treating hypertension] in 35 years," says Michael Alderman, a blood-pressure expert at the Albert Einstein College of Medicine in New York City, and a co-author of one of the new studies.

 

One study shows some drugs work better in certain ethnic groups than in others. The two other studies recognize the importance of testing patients' levels of renin, a hormone produced by the kidneys, as a guide in prescribing blood-pressure medicine. Researchers in each of the studies emphasized that larger-scale trials would be necessary before the findings could become part of official treatment guidelines.

 

Welcome to what “personalized medicine” really means.  Not “bespoke” or tailored medicines, but rather a focus on the “four rights” – the right medicine in the right dose for the right patient at the right time.

 

One of the studies, co-authored by Ajay Gupta of Imperial College London, looked at drug responses among 5,425 patients in various countries and across different ethnic groups. For example, in the U.K., south Asians are often given ace inhibitors as a first-line treatment, though the effectiveness of such prescriptions isn't based on any hard evidence. Dr. Gupta's study, for the first time, confirms that south Asians respond especially well to such drugs.

 

U.K. medical-treatment guidelines say that first-line drug therapies should be guided by a patient's age and race. (Guidelines in the U.S. don't include such suggestions – but since we’re looking at the NHS to learn about comparative effectiveness, maybe we should broaden our field of study, especially considering that our new CMS chief is such a fan of the UK model.)

And, while we're on the subject of the NHS, a brief digression.  Did you see today's news that NICE has rejected Avastin for use in patients with colerectal cancer that has spread?  It's the second time that the agency has issued such guidance. At the same time, NICE Chairman Sir Michael Rawlins has suggested that the best way to counteract the dearth of real science behind HTA is for drug companies to just lower their prices.  Sir Michael is a bright guy and needs to do better than such a simplistic answer to the problem of 21st century HTA's lack of scientific integrity.

The two other studies focused on the hormone renin. Medical experts say few doctors today measure a patient's renin level, despite a study in the 1970s that suggested it might be used as a biomarker for prescribing the drugs. One of the new studies, involving 363 patients, confirmed the 1970s finding, showing that measuring the renin level can be an effective method for selecting a blood-pressure medication.

 

"These are not fundamentally novel biological discoveries," says Morris Brown, professor of clinical pharmacology at the University of Cambridge, U.K., who wasn't involved in the studies. But they constitute "a wake-up call that we should be using renin measurements as a systematic form of help" for prescribing hypertension drugs.

And it should also serve as a wake-up call to those who aren't paying close enough attention to the future of diagnostics development and approval. 

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Hello Cleveland

08/23/2010 07:09 AM |

From the pages of the Wall Street Journal:

 

Cleveland Clinic CEO Worries Comparative Effectiveness Could Stifle Innovation

 

By Katherine Hobson

 

The last time we looked at consumer effectiveness research — basically defined as identifying which health care services work best under which circumstances — it was

consumers who were skeptical.

 

Now it’s the CEO of the Cleveland Clinic who’s expressing concern, as the

Cleveland Plain Dealer reported yesterday. In response to a question after a speech, Toby Cosgrove said he wanted to ensure that manufacturers and investors would still be willing to make financial bets on unproven devices and drugs. He used the example of a heart valve, saying it now takes two decades to bring a new valve product to market and then assess the effectiveness.

(Cosgrove knows of which he speaks; his bio says he has 30 patents filed, including heart valves and surgical instruments.)

 

The concern he’s raising isn’t so much over the comparative research itself, but what the government does with the results. If regulations deem that only the treatment judged most effective is paid for, “my concern is that .. we begin to limit what people are willing to do in terms of developing new products,” Cosgrove said, according to the paper.

 

Two health-care experts interviewed by the Plain Dealer said they thought the government and insurance companies understood the need to safeguard innovation.

 

Last year both the NIH and IOM published lists of things they’d like to see studied under CER. Included were expensive biologic drugs for auto-immune diseases, treatments for atrial fibrillation, school-based anti-obesity programs and pregnancy-prevention strategies. The co-chair of the IOM committee told the Health Blog back then that CER includes figuring out what works best for certain subgroups — say by age or gender or ethnicity.

 

Comparative effectiveness bonus: Read why one expert is concerned that the emphasis on CER

might present some tough obstacles for cancer research.

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Is There A Sunny Side To Egg Safety?

08/22/2010 01:19 PM |

Q:  How many eggs are laid each year  in the United States?

A:  72 billion

Q.  How many chickens are in the egg producing business?

A.   About 340 million..  According to USDA "U.S. egg production totaled 7.45 billion during June 2010, up 1 percent from last year.  Production included
6.38 billion table eggs, and 1.07 billion hatching eggs, of which 1.00 billion were broiler-type and 69 million
were egg-type.  The total number of layers during June 2010 averaged 338 million, up 1 percent from last
year.  June egg production per 100 layers was 2,203 eggs, unchanged from June 2009."

Q.  How many eggs are being recalled, give or take a few yolks..

A.   360 million.   According to my math, that's about 5 percent of all eggs and about 15 million chickens...


That's a lot of omelettes.. 

Throughout all this the outcry from Congress has been nearly non-existent.   Compared to the bashing FDA took under the previous president, the slience has been... welcome.

Salmonella happens.. all the time.   There is more food produced more efficiently than ever before.   We will recover from our egg deficit in no time.  Tracking outbreaks requires more than increasing the size of the food police.   It requires better tools and coordination.   The last thing we need is a Food Safety Czar or a separate Food Safety Agency.. 

That would really be laying an egg..

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HHS Seeks To Create Govt-Run Drug Company

08/20/2010 02:24 PM |

If running the banks, healthcare and car companies wasn't enough, the Obama Administration apparently wants to get into the biotech business.

It already set up a venture capital firm in Dept of Treasury to dole out  $1 billiion to small companies to the projects of it's choosing.  This, at a time when private VC financing for life sciences is actually up.  But no matter, the biotech genuises at Treas. who I am sure have extensive investment and biotech backgrounds, will hand out tax credits that can be used to offset losses or as collateral for other funding.  Sound familar?

Now comes the administration's response to their botching of the H1N1 production and distribution effort.  By insisting on single doses to avoid the fearmongering of vaccine critics, HHS delayed the roll out of the vaccine by months.   The snafu forced a re-examination of the government's role in preparing the nation for both pandemics and bioterrorism.  Thus HHS just released " The Public Health Emergency Medical Countermeasures Review" where Secretary Sebelius said “Our nation must have a system that is nimble and flexible enough to produce medical countermeasures quickly in the face of any attack or threat, whether it’s a threat we know about today or a new one,”

It's solutio for becoming nimble and flexible?   More government control over the production of biotech products.   There will be the Centers of Innovation for Advanced Development and Manufacturing where the government -- which not only has no expertise in either but has failed miserably -- will be building and running vaccine production facilities.  As for new products, moving from the mistaken idea that all new discoveries come from NIH, " HHS will be creating new teams at the National Institutes of Health to identify promising research and facilitate its translation  into vaccines, drugs, and treatments that keep Americans safe."

Once government picks winners and losers it will invest in pet projects through a strategic investment firm and give it $200 million.  

Will all this  make biologic countermeasure development more nimble and flexible?   The government track record in product development is terrible.  Worse, there is no acknowledgement of the real barriers to innovation:  the failure to apply the same science of discovery to accelerate the evaluation and development of new products overall.  The  funding for regulatory science pushed by Sebelius misses the mark.  It focuses on studying the potenetial of early stage products when in fact the FDA needs more money for later stage evaluation and more efficient ways to monitor production.  

The report claims HHS will reposition government as a "strategic partner."  The current proposals put the government into the biotech and vaccine business.  If you like ObamaCare you will love ObamaShots.

http://www.phe.gov/Preparedness/mcm/enterprisereview/Pages/default.aspx

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Post Haste?

08/20/2010 08:42 AM |

Subsequent to the July adcomm on Avandia, the FDA asked GSK to send a letter to doctors detailing what took place.

The first question is … why didn’t the FDA do this itself?  It was, after all, an FDA event.  Was a written synopsis, perhaps, too hot to handle for the folks at White Oak?

To nobody’s surprise, a certain agency employee is unhappy with GSK’s letter.

 “This summary is biased, misleading and not truthful,” Dr. Graham told the New York Times. “The whole purpose of this letter is so that they can reassess whether this is an ethical trial going forward, but the step-by-step ethical flaws and problems with the Tide trial are not even referenced.”

Some panel members agree with Dr. Graham. Panel member Dr. Curt D. Furberg, described the letter as “very Avandia friendly. Panel member Dr. Sanjay Kaul, disagreed, saying the letter “faithfully reflects the deliberations of the Avandia advisory meeting.”

Erica Jefferson, an F.D.A. spokeswoman, said that after ordering GlaxoSmithKline to send a summary of the hearing to the Tide trial investigators, the agency had relied on the company to provide a balanced account. “F.D.A. did not pre-clear or approve the content,” she said.

Mary Anne Rhyne, a GlaxoSmithKline spokeswoman, said the company had only one week to write the 10-page summary, which was necessarily brief. But the company and the leader of the Tide trial agreed that the letter “reflected the science and data discussed at the advisory committee meeting,” Ms. Rhyne said.

Avandia?  Controversy?  Who could have guessed? 

Why didn’t the FDA write this letter?

"Please give me some good advice in your next letter.  I promise not to follow it."

-- Edna St. Vincent Millay

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The Oil Spill of Alzheimer's

08/19/2010 09:49 AM |

If we learn nothing else from BP’s recent unpleasantness, it’s that being able to identify an obvious problem (like when oil is gushing uncontrolled into the Gulf of Mexico) is one thing.  Identifying that there is a potential problem is tougher.  And toughest of all is designing a solution that addresses a need early in the curve.

Case in point, Alzheimer’s Disease. As Gina Kolata writes in today’s New York Times, “The failure of a promising Alzheimer’s drug in clinical trials highlights the gap between diagnosis — where real progress has recently been made — and treatment of the disease.”

Alzheimer’s Disease is a healthcare oil spill of draconian proportion.

Recent significant steps forward in early diagnosis of the disease are important.  And frustrating -- because there is still precious little that can be done when this devastating condition is identified either late in the game or in its nascent stages. 

To say that the science is “hard” (while true) is not particularly helpful.  What needs to be addressed are the twin issues of drug development and regulatory science.  Both are lagging.  Biomarkers notwithstanding, more needs to be done.  We need better tools.  Too many programs  (almost 50%) are failing in late Phase III. Too many programs are mired in regulatory treacle.  The economics are unsustainable from a corporate R&D standpoint and the impact of Alzheimer’s Disease on patients, their families and American healthcare economics is devastating.

Better, more current and predictable scientific research and standards must be developed and devoted to streamlining the critical path. Investment in basic research is not enough. Specifically new development tools are needed to improve the predictability of the drug development cycle and to lower the cost of research by helping industry identify product failures earlier in the clinical trials process.

 

25 years ago, the success rate for a new drug used was about 14%. Today, a new medicinal compound entering Phase 1 testing—often after more than a decade of preclinical screening and evaluation—is estimated to have only an 8% chance of reaching the market. For very innovative and unproven technologies, the probability of an individual product’s success is even lower. We have got to work together to turn that around.

 

When Thomas Edison was asked why he was so successful he responded, “Because I fail so much faster than everyone else.” Consider the implications if FDA could help companies to fail faster. Using the lower end of the Tufts drug development number ($802 million):

 

* A 10% improvement in predicting failure before clinical trials could save $100 million in development costs.

 

* Shifting 5% of clinical failures from Phase 3 to Phase 1 reduces out of pocket costs by $15-$20 million.

 

* Shifting of failures from Phase 2 to Phase 1 would reduce out of pocket costs by $12-$21 million.

 

Investment in basic research is not enough. Specifically new development tools are needed to improve the predictability of the drug development cycle.

 

For all that modern science has to offer, developing new treatments is still very much an art—in which hunches, intuition, and luck play a critical role. The odds are long. For medicine that is affordable and innovative, we need more well-understood science and we need regulatory predictability. And that’s precisely the mission of the FDA’s still moribund Reagan-Udall Foundation.

 

To quote the late Senator Ted Kennedy, the Reagan-Udall Foundation “will make new research tools and techniques available to the entire research community, shortening the time it takes to develop new drugs and reducing costs for patients.”

 

Shortly before his death, I had the privilege of a private meeting with Nobel Laureate Joshua Lederberg. The topic of conversation was the future of the FDA and the agency’s Critical Path initiative. We talked about the state of applied research and “the texture” of the agency, the prioritization of development science, biomarkers and a host of other future-oriented issues. He talked. I took a lot of notes. At the end of the meeting he put everything into perspective in a single sentence. He leaned over the table and said, “The real question should be, is innovation feasible?”

 

I hope so and so should we all.  Innovation = Hope.

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A Seminole moment

08/18/2010 07:59 AM |

A new Orange revolution?

The Florida Medical Association decided Sunday after two days of heated debate not to break off relations with the American Medical Association, officials and delegates said at the conclusion of the event.

Instead, FMA will send AMA a letter describing just how unhappy it is with the national group’s actions on health reform. 

In a formal written statement, Executive Vice President Timothy J. Stapleton said that the FMA letter will convey a vote of “no confidence.”  “The FMA House of Delegates strongly believes that the American Medical Association has failed to represent practicing physicians on the issue of health care reform.”

One of the delegates, Tampa surgeon Michael Wasylik, said, "Doctors are really, really, really upset with the AMA.  Doctors are so angey they can't see straight.

And not seeing straight is a bad place for a surgeon to be.

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FDA's Crude Tools

08/17/2010 04:52 PM |

The FDA makes binary decisions in the main while medical science is quantum knowledge --  which means it's capacity to regulate drugs and devices by adding language to labels or simply pulling a drug because it doesn't benefit -- on average -- all patients relative to risks is outdated.  From Avandia to Avastin the old school view of FDA regulation -- from the right and left -- is prehistoric.    Why is the FDA frittering away millions on discovery work masked as regulatory science and playing footsie with CER when the future is in digitizing and personalizing product development and treatment selection?  And why is the industry funded NIH Foundation lauded for promoting Alzheimer's advances through collaboration and the Reagan-Udall Critical Path Institute is attacked?     I just don't get it. 

marketplace.publicradio.org/display/web/2010/08/16/am-fda-may-revoke-breast-cancer-drug-q/


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Desperate times call for desperate measures

08/17/2010 02:36 PM |

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Of Danes and Deutschland

08/16/2010 07:23 AM |

German authorities have filed charges against pharmacies in the north of the country that have been suspected of purchasing cheap and illegal active ingredients, which they have used to manufacture in-house cancer treatment preparations and then sold them to the general public.

 

Frank Keller, chief investigator of the Technician's Health Insurance Fund (TKK), as saying that the fund had been tipped off that a Danish wholesaler had been supplying German pharmacies with an unauthorized active ingredient for the preparation of an infusion used in cancer treatment. Some 100 of the 400 pharmacies authorized to manufacture the preparation in-house are suspected of having purchased the cheap, illegal product from the wholesaler.

 

Separately, Alexander Retemeyer from the Office of Public Prosecution in Osnabruck, revealed that he had tracked a local wholesaler, which had sourced illegal products from Eastern Europe and then sold them to pharmacies.

 

In the latest revision to the Pharmaceutical Act, the German government has put in place measures to sanction those who introduce counterfeits into the health system. The European Union is also concerned about the increase of falsified medicines emerging in the Member States. Figures from the Directorate General for Taxation and Customs show that counterfeit medicines accounted for 10 percent of goods seized upon entering the EU. Almost 75 percent of these were shipped from the United Arab Emirates.

 

In April, the European Parliament's Environment Committee approved the European Commission's proposal for fighting counterfeit medicines. The committee added a stipulation to the draft, which insists that in addition to preventing counterfeits from entering the legal supply chain and introducing mandatory safety features for prescription medicines, the legislation should prohibit internet sales of “detrimental products.” The EU Parliament will vote on the draft proposal in October.

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The Morning After the Morning After

08/16/2010 07:09 AM |

Per the FDA’s approval of the 5-day emergency contraceptive ella, some perspective. When the FDA reviewed (and reviewed and reviewed) moving the Plan B “morning after” pill OTC in 2003 it was reported as “political interference.” Well it wasn’t true then and it isn’t true now with the approval of a new improved (safe and effective) Rx alternative.  The FDA reviews the science and makes it’s decision. The agency is often imperfect and sometimes contentious.  But it never lacks for passion or intellectual honesty.

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