46-Love

05/11/2012 06:10 AM |

The U.S. House Energy and Commerce Committee voted 46-0 on Thursday to pass the PDUFA reauthorization   Read More & Comment...

CDC Plays Politics With Whooping Cough Outbreak

05/10/2012 06:00 PM |

Washington State is a hotbed of anti-vaccine sentiment.  It had a very liberal policy that allows parents not to immunize their kids for religious or philosphical reasons..  Here's a copy of the exemption form

http://www.doh.wa.gov/cfh/immunize/documents/coe2011.pdf

So why is the CDC punting on the whooping cough outbreak in Washingon?  Here's the CDC spokesperson on the epidemic:

'Adults and teens need booster shots so they don't give pertussis to the babies in their lives, said CDC spokeswoman Alison Patti
"We want to create a cocoon of protection around them," she said. "We're really worried about keeping babies safe."

 Patti emphasized that pertussis isn't spreading because of an anti-vaccine movement. Among possible reasons for the recent spike are that diagnoses in teens and adults are getting better and doctors are doing a better job with reporting, she added.'

This is nonsense.   While outbreaks go in five year cycles, the incidence has skyrocketed in places where anti-vaccine sentiment is strongest: including Idaho, New Mexico, Madison, WI,  Woodstock, NY and Vermont.  

Under Obama, the CDC has been ducking the source of vaccine resistance and in some cases, caving into it.  Perhaps its because their anti-sugar allies are also anti-vaccine or because the states essential to the president's re-election are also the one's where the anti-vax movements are strongest..  

This is pure speculation on my part.  But in any event, the lack of leadership on immunizations at the CDC is troubling.  Bad enough that CDC is using cost-effectiveness analysis to decide whether it's worth paying for new vaccines under Obamacare.  Ignoring a principal threat to herd immunity is even worse. 


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FDA Impatience Is Great News For Patients

05/10/2012 05:37 PM |

The FDA signaled that it supported a new accelerated pathway for approval of new drugs for serious diseases that use biomarkers and show high response in early trials.    

U.S. may speed approval of 'breakthrough' drugs


When the Critical Path was launched, the goal was to align the approval of drugs with the science of drug development.  That process was derailed after the Vioxx panic.  It is now back on track thanks to a bi-partisan effort on Capitol Hill, support from the Obama administration and the persistence of Janet Woodcock and Peggy Hamburg at the FDA.   This is a great day for innovation and patients, at least in principle.   

There will be those who see this reform as a sop to evil, profit making drug companies...  Such as Merrill Goozner:  "Pfizer last week won Food and Drug Administration “accelerated approval” for Xalkori (crizotinib) for a rare form of metastatic lung cancer that strikes about 3 percent of people — almost always non-smokers — who come down with the disease. That’s about 10,000 patients per year in the U.S., and perhaps a similar number abroad. Multiply that times the $115,200 a year the company plans to charge for Xalkori, and they’ve got themselves a billion dollar blockbuster.  Through this period where there will be no concrete proof of efficacy, Pfizer will get to charge an outlandishly high price for the drug. Is there a way to put pressure on the company to complete the trials in a more timely fashion, so that the paying public knows that it is truly getting value for its health care dollar and not subjecting patients to some pretty rough side effects (nausea, diarrhea, etc.) for no benefit?"

Goozner is either ignorant or willfully misleading.  By definition accelerated approval will be reserved for treatments that show significant benefits in specific groups of patients through well-characterized biomarkers and clinical endpoints.   That will cut the time required for approval and reduce costs. (If Goozner can figure out a way to reduce the cost of producing new biologics for small groups of people, he should start his own biotech company.)  The price of drugs will be relative to their value.  Or should be.   

The question is, as accelerated approval is adopted will CER just add back delay and access.   Let's hope the courageous impatience of the FDA, the Obama administration, Congress and the public is shared by the CER crowd and PCORI.    


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The Scoop on PDUFA

05/10/2012 06:43 AM |

It’s time to excise the Ben and Jerry’s Clause from the Senate HELP Committee’s draft of PDUFA.

That would be the amendment (adopted this April) that would require an evaluation of the so-called “prize fund approach” to drug development.

It’s same old and tired and false proposition put forth by Bernie Sanders – the independent Senator from the great state of Ben and Jerry’s – since time immemorial.

It’s not a new idea. The “prize” model has been used in the past—in the old Soviet Union. It didn’t work. The Soviet experience was characterized by low levels of monetary compensation and poor innovative performance.

The experience in the United States hasn’t been much better. The federal government paid the father of American rocketry, Robert Goddard, $1 million as compensation for his basic liquid rocket patents. A fair price? Not when you consider that during the remaining life of those patents, U.S. expenditures on liquid-propelled rockets amounted to around $10 billion.

Senator Sanders wants to replace a patent system that has fueled innovation allowing the average American lifespan to increase by almost a full decade over the past 50 years with a prize program that has a solid record of failure. As the healthcare economists Joe DiMasi and Henry Grabowski have argued, under a prize program, pharmaceutical innovators would lack the incentive to innovate. DiMasi, director of economic analysis for the Tufts Center for the Study of Drug Development, and Grabowski, director of Duke University’s Program in Pharmaceuticals and Health Economics, said, “The dynamic benefits created by patents on pharmaceuticals can, and almost surely do, swamp in significance their short-run inefficiencies.”

Who could support the idea of a prize? As DiMasi and Grabowski presciently observed in 2004, “The main beneficiaries in the short-term would be private insurers and public sector purchaser of pharmaceuticals.” That’s because, as they note, governments and insurers are focused “myopically on managing health care costs” and are not likely to be “strong advocates for funding new drug development that can increase individual quality of life and productivity.”

Those who support this idea view it as a solution to all the world’s health care ills. “Research is risky, new drugs are too expensive, and industry focuses far too much of its effort on drugs of minimal medical significance,” Merrill Goozner, director of the Integrity in Science Project at the Center for Science in the Public Interest, has said. “The prize fund solves all these problems by disconnecting the incentives for generating breakthroughs from the price that individual patients or their insurers must pay.”

Not.

According to Jamie Love, director of Knowledge Ecology International, “By separating the markets for innovation from the markets for the physical goods, the Prize Fund would ensure that everyone, everywhere, could have access to new medicines at marginal costs.”

Not.

Here’s Jamie’s spin on Senator Sander’s legislation -- a highly revisionist history.

The truth of the matter is that the promotion of innovation and the creation of new medicines cannot be based on a top-down process. Rather, they should be based on bottom-up solutions by the actual players involved in this process—companies, research institutions, and the regulatory and IP authorities.

Clearly Senator Sanders and Jamie Love do not concur. And that is their privilege.

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IOM Obesity Study Weighed Down By Bad Science

05/09/2012 05:59 PM |

I will have a lot more to say about the IOM report: Accelerating Progress in Obesity in another post.  But for now, I urge anyone who intends to read it not to if you care at all about science.   The report uses BMI as a measurement of obesity.  And as the following article and segment from NPR (produced in 2009) shows, the IOM -- with the help of HBO, CDC and a bunch of other nurtitional busybodies -- is basing it's recommendation for government micromanagement of our daily lives on a measure that is politically correct but scientifically useless.  


Top 10 Reasons Why The BMI Is Bogus
by KEITH DEVLIN



The BMI Formula
BMI = weight in pounds/(height in inches x height in inches) x 703

The 703 is to convert the index from the original metric version of the formula.

CDC Recommendations:
Below 18.5 = Underweight

18.5 to 24.9 = Ideal

25.0 to 29.9 = Overweight

30.0 and above = Obese

text size A A A July 4, 2009
Americans keep putting on the pounds — at least according to a report released this week from the Trust for America's Health. The study found that nearly two-thirds of states now have adult obesity rates above 25 percent.

But you may want to take those findings — and your next meal — with a grain of salt, because they're based on a calculation called the body mass index, or BMI.

As the Weekend Edition math guy, I spoke to Scott Simon and told him the body mass index fails on 10 grounds:

1. The person who dreamed up the BMI said explicitly that it could not and should not be used to indicate the level of fatness in an individual.

The BMI was introduced in the early 19th century by a Belgian named Lambert Adolphe Jacques Quetelet. He was a mathematician, not a physician. He produced the formula to give a quick and easy way to measure the degree of obesity of the general population to assist the government in allocating resources. In other words, it is a 200-year-old hack.

2. It is scientifically nonsensical.

There is no physiological reason to square a person's height (Quetelet had to square the height to get a formula that matched the overall data. If you can't fix the data, rig the formula!). Moreover, it ignores waist size, which is a clear indicator of obesity level.

3. It is physiologically wrong.

It makes no allowance for the relative proportions of bone, muscle and fat in the body. But bone is denser than muscle and twice as dense as fat, so a person with strong bones, good muscle tone and low fat will have a high BMI. Thus, athletes and fit, health-conscious movie stars who work out a lot tend to find themselves classified as overweight or even obese.

4. It gets the logic wrong.

The CDC says on its Web site that "the BMI is a reliable indicator of body fatness for people." This is a fundamental error of logic. For example, if I tell you my birthday present is a bicycle, you can conclude that my present has wheels. That's correct logic. But it does not work the other way round. If I tell you my birthday present has wheels, you cannot conclude I got a bicycle. I could have received a car. Because of how Quetelet came up with it, if a person is fat or obese, he or she will have a high BMI. But as with my birthday present, it doesn't work the other way round. A high BMI does not mean an individual is even overweight, let alone obese. It could mean the person is fit and healthy, with very little fat.

5. It's bad statistics.

Because the majority of people today (and in Quetelet's time) lead fairly sedentary lives and are not particularly active, the formula tacitly assumes low muscle mass and high relative fat content. It applies moderately well when applied to such people because it was formulated by focusing on them. But it gives exactly the wrong answer for a large and significant section of the population, namely the lean, fit and healthy. Quetelet is also the person who came up with the idea of "the average man." That's a useful concept, but if you try to apply it to any one person, you come up with the absurdity of a person with 2.4 children. Averages measure entire populations and often don't apply to individuals.

6. It is lying by scientific authority.

Because the BMI is a single number between 1 and 100 (like a percentage) that comes from a mathematical formula, it carries an air of scientific authority. But it is mathematical snake oil.

7. It suggests there are distinct categories of underweight, ideal, overweight and obese, with sharp boundaries that hinge on a decimal place.

That's total nonsense.

8. It makes the more cynical members of society suspect that the medical insurance industry lobbies for the continued use of the BMI to keep their profits high.

Insurance companies sometimes charge higher premiums for people with a high BMI. Among such people are all those fit individuals with good bone and muscle and little fat, who will live long, healthy lives during which they will have to pay those greater premiums.

9. Continued reliance on the BMI means doctors don't feel the need to use one of the more scientifically sound methods that are available to measure obesity levels.

Those alternatives cost a little bit more, but they give far more reliable results.

10. It embarrasses the U.S.

It is embarrassing for one of the most scientifically, technologically and medicinally advanced nations in the world to base advice on how to prevent one of the leading causes of poor health and premature death (obesity) on a 200-year-old numerical hack developed by a mathematician who was not even an expert in what little was known about the human body back then.

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AMA and Personal Injury Bar On Opposing Sides Of Expanded Rx-OTC Switch Proposal

05/09/2012 03:58 PM |

As I noted yesterday  (AMA Seeks To Derail Consumer Health Revolution) the AMA and other lobbying organizations for medical professions sounded the alarm against the FDA's Rx-OTC proposal ( http://tinyurl.com/18r).     

You would think (if you accept the stereotype) that trial lawyers would warn against the proposal as well.  But Greg Webb, a blogger for the Injury Board Blog Network has penned a measured and thoughtful piece on the FDA's safe use proposal for expanded OTC access.. 

"Representatives of the American Medical Association (AMA) and the American College of Allergy, Asthma and Immunology (ACAAI) have gone on record objecting to the proposed new safe-use class of medications. Roland Goertz, MD, president of the American Academy of Family Physicians (AAFP), stated that the safe-use designation where pharmacists dispensed medications without a patient having to see a doctor first, “could seriously compromise the physician's ability to coordinate the care of multiple problems of many patients.”[1]

Health insurers reportedly have tentatively "approved" of the measure. Without proper safeguards, there is some concern that patients may obtain drugs that they may not need. The insurers also would have to determine how to cover drugs that fell into this category. Additionally, the health insurers were in favor of "expanding access" to helpful medications.

Because pharmacists are more easily accessible than physicians who require appointments for frequently costly office visits, the FDA believes that the “safe-use” designation may benefit many Americans who presently go untreated with legitimate medical needs. Doubtless the collection of comments and the promulgation of a new regulation on safe-use medicines by FDA (requiring additional debate) will cause more lines to be drawn in the sand of looming health issues. Is this the wave of the future for certain medications? Much of it makes sense, and ultimately would save money by preventing potentially unnecessary doctors' visits. What are the unintended consequences?"

Mr. Webb's article is well worth reading because it lays out the FDA proposal clearly.  It is also noteworthy because it shows that the AMA and other groups are more hysterical about the Rx-OTC issue than the trial bar.     Read More & Comment...

Living off the Land of the Fat

05/09/2012 06:48 AM |

The FDA has proposed a benefit-risk assessment system for weight loss devices that would use available safety data for a device to set efficacy thresholds for a pivotal trial of that device. BioCentury reports that the proposal came in briefing documents issued to FDA's Gastroenterology and Urology Devices Panel ahead of a meeting on Thursday and Friday to discuss issues in the development of devices placed endoscopically or laparoscopically.

Devices would be classified into one of three risk levels based on the percent of patients who experience mild, intermediate and severe adverse events over one year. For example, to qualify for the lowest risk category, devices can have high rates of the most mild events (up to 100%), low rates of events of intermediate severity (2-5%), and very low rates of the more severe events (<0.1%).

Each level of risk would set corresponding efficacy endpoint targets for pivotal trials. Targets for the least risky devices would be based on endpoints used for weight loss drugs, while targets for the highest risk devices would be based on the two FDA-approved obesity devices, which the agency said fall into the high risk category. Those are the Lap-Band adjustable gastric banding system from Allergan and the Realize adjustable gastric band from the Ethicon Endo-Surgery.

FDA also proposed that efficacy be measured using percent total body weight loss instead of the current standard of percent excess weight loss. The panel is part of the Medical Devices Advisory Committee.

  Read More & Comment...

All for One and One for PDUFA

05/09/2012 06:45 AM |

Yesterday the U.S. House Energy and Commerce Committee's health subcommittee voted unanimously to pass the PDUFA reauthorization bill. A full committee vote on the draft is expected Thursday and a full House vote is expected later this month.

  Read More & Comment...

AMA Seeks To Derail Consumer Health Revolution

05/08/2012 02:20 PM |

Are you afraid of your drugstore and what you might do there?  The AMA is....  

 Medscape Medical News

Pharmacists Dispensing Without Rx 'Scary,' Say Med Societies
Robert Lowes

May 2, 2012 — Imagine a patient walking into a pharmacy, self-diagnosing a cough at a kiosk with medical software, and then getting a drug from a pharmacist in the back of the store, all without a physician's prescription.

The US Food and Drug Administration (FDA) is imagining this scenario, much to the alarm of the American Medical Association (AMA) and other medical societies, which see the beginnings of an end-run around physician authority.

Sandra Fryhofer, MD, chair-elect of the AMA Council on Science and Public Health, said her group continues to "strongly support" the status quo of 2 drug classes — prescription and traditional OTC. In exploring how new technologies and new roles for pharmacists might expand the portfolio of OTC drugs, the FDA should not "undermine the relationship that a patient has with his or her physician."

"In our view, lack of oversight from a practitioner could be a serious concern," said Dr. Fryhofer.

The FDA has not provided any evidence showing that patients with hypertension, hyperlipidemia, or asthma can diagnose and manage these conditions by themselves, she noted. "The balance of medical evidence strongly suggests otherwise." She also estimated that patients would pay more out-of-pocket for drugs that insurers would shift into the OTC safe-use category.

Bobby Lanier, MD, speaking for the American College of Allergy, Asthma and Immunology (ACAAI), raised similar concerns, but more pointedly.

"The new proposals here being discussed are chilling and scary," said Dr. Lanier. "You are facilitating bad behavior by not having (patients) talk to a clinician. In our mind, there is no substitute for a clinician."

Which brings to mind the fact that when the FDA decided to move painkillers and allergy meds OTC, you heard the same dire warnings.  Now the same groups are seeking to change an Obamacare law requiring a doctor's prescription for OTC drugs purchased through a Flexible Spending Account.  Not necessary and too much of a hassle according to the AMA and other medical lobbies.   In Dr. Lanier's words,  it's ok to facilitate bad behavior with existing OTC products but not new ones.  

The FDA should be commended for promoting more consumer-driven care.  As Peter and I demonstrated in our recent survey on cough medicine usage, the vast majority of Americans like OTC access because it makes health care simpler and therefore more likely.   Doctors are behind the curve on the consumer revolution.  Rather than trying to derail it, doctors should embrace new ways of connecting with patients and finding ways to help consumers help themselves.   http://cmpi.org/uploads/File/CMPI_CoughSurvey_KeyFindings.pdf


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Measuring the Marigolds

05/08/2012 07:08 AM |

BioCentuty reports:

House moves closer to Senate on PDUFA

The U.S. House Energy and Commerce Committee posted a new draft PDUFA reauthorization bill that is more closely aligned with the Senate PDUFA bill than previous versions. Changes that reflect the Senate bill include dropping sections that would add jobs creation and economic development language to FDA's mission and adding a section that would create an expedited development pathway for breakthrough drugs.

However, several items in the House draft bill are still not in the pending Senate version, including provisions to increase public participation in crafting guidance documents and creation of a new priority review voucher system to encourage development of therapies for rare pediatric cancers. The House draft also requires that FDA provide Congress a 60-day notice prior to issuing guidance on the regulation of laboratory-developed tests. The Energy and Commerce Committee's health subcommittee is scheduled to meet Tuesday (today) to deliberate on the draft bill.

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"Safe Use Drugs" -- the Saga Continues

05/08/2012 07:03 AM |

Per today’s Washington Times, “Health insurers gave a tentative thumbs-up Monday to the Food and Drug Administration’s proposal to make drugs treating chronic conditions available without a prescription by classifying them in an all-new category.”

Insurance companies cautiously support the idea but warned Monday in a letter to the FDA that a host of complications could arise.

Without the right safeguards in place, patients could obtain drugs they don’t need and it’s unclear who would be held liable if they do, said Robert Zirkelbach, spokesman for America’s Health Insurance Plans.

Insurance companies also would have to figure out whether and how they would cover drugs that fall into the new category.

“Expanding access is something that we support, but these other issues would have to be addressed for this to work if they decide to move forward with that,” Mr. Zirkelbach said in an interview.

If the FDA decides to move ahead with the plan, it would create a third category for classifying drugs.

Called “safe use” drugs, patients wouldn’t need a prescription but neither could they obtain them over the counter. Instead, people could only buy the drugs after diagnosing their ailments online or in the pharmacy.

Seeking a way to expand access to drugs for Americans who struggle with common conditions such as high cholesterol, migraines and diabetes, the FDA has raised the idea several times over the past decade and brought it up again in March, asking the public to weigh in.

It’s uncertain whether the agency will sign off on the new policy since the idea has faltered in the past under opposition by doctors and other medical providers. The American Medical Association has said it could open the door to drug misuse and expects that out-of-pocket costs will rise for patients.

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Loggerheads, Wiser Heads and Troubled Intermediaries

05/08/2012 06:44 AM |

When it comes to addressing and solving the drug shortages, what are the most important variables to consider?

According to Healthcare Intermediaries: Competition and Healthcare Policy at Loggerheads? -- a new white paper by the American Antitrust Institute, a key question “is whether high levels of consolidation in intermediary markets and potentially exclusionary conduct have caused or exacerbated shortages.”

“One major protection against shortages is a stable supply chain, which is largely determined by the number and diversity of suppliers. The concept of supply chain “fragility” is increasingly relevant in operations research, marketing, economics, and even sociology. Supply chains featuring only a few competitors and high entry barriers at critical junctures are excessively exposed to the risk of disruption and collapse following an exogenous shock … but a fragile supply chain can also be inefficient when it “fails” because of excessive consolidation that leaves few suppliers.”

Intermediary conduct can, “threaten to impair the achievement of healthcare policy goals such as affordable healthcare, choice in medical products, a stable supply chain, and diversity of supply.”

The paper points out that sterile injectables. In 2010, 60 percent of sterile injectables (which accounted for 80 percent of the drugs in shortage) were sole-sourced. Markets for specific drugs are likely to be even more concentrated because only one or two firms produce them. Markets for generic drugs – which accounted for 60 percent of sterile injectables and 50 percent of all shortages – are also concentrated. In 2010, the top three firms accounted for about 70 percent of the generic sterile injectable market and 90 percent of the generic sterile injectable oncology segment of the market.

Indeed, the FDA notes that while demand in the generic and oncology segment of the market is robust, the supply system is “vulnerable to drug shortages because a large supply disruption is difficult to make up with alternative suppliers.” This is compounded by low demand and supply elasticities for certain drugs, stringent product manufacturing quality controls, dedicated production lines, and “just-in-time” manufacturing and inventorying practices.

Among other suggested next steps, the paper recommends that regulatory initiatives designed to address drug shortages “should focus less on reporting requirements and more on the analysis of competition in intermediary markets and upstream markets for drugs, and medical devices and supplies.”

A paper worth reading with conclusions worth debating.

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Massachusetts Doubles Down On Obamacare

05/07/2012 11:11 AM |

Language from Bill H.4070 "An Act relative to Health Care Quality Improvement and Cost Reduction Act of 2012" introduced in the Massachusetts House of Representatives:

" The division shall have the power to design and to revise, consistent with this chapter, a basic schedule of health care services that enrollees in any health insurance program implemented by the division shall be eligible to receive. Such covered services shall include those which typically are included in employer-sponsored health benefit plans in the commonwealth. The division may promulgate schedules of covered health care services which differ from the basic schedule and which apply to specific classes of enrollees. The division may promulgate a schedule of premium contributions, co-payments, co-insurance, and deductibles for said programs, including reduced premiums based on a sliding fee, and other fees and revise them from time to time, subject to the approval of the division of insurance; and provided, however, that such schedule shall provide for such enrollees to pay one hundred per cent of such premium contributions if their income substantially exceeds the non-farm poverty guidelines of the United States office of management and budget."

Compared to this bill, Obamacare is free market reform.    Read More & Comment...

Amazing Pace

05/07/2012 01:06 AM |

According to FDA Commish Peggy Hamburg, since President Obama gave the agency new authorities to address drug shortages new shortages have fallen (year-to-year) by more than half.  There have been 42 new shortages in 2012 compared to 90 at the same time last year.

But is this decline really due to the FDA’s ability to demand earlier information about potential shortages from manufacturers? 

“I am both amazed and delighted to see the progress that’s been made, said Hamburg.  Key word:  “Amazed.”

While the FDA’s new authorities are both timely and important, there are many pieces to the drug shortages problem – not the least of which is that (when it comes to hospital injectables) 30% of manufacturing capacity is off-line due to FDA inspection issues. That’s a lot of capacity. In fact, according to the agency, 43% of reported potential shortages were due to manufacturing issues.

Greater cooperation between agency and manufacturers is required so that this gap can be corrected before problems arise.  A 30% hole in manufacturing is more than a hint that something’s amiss on both sides.

Is it a victory when you are solving the problems you create?


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The Calendar Days of our Lives

05/04/2012 07:18 AM |

Peggy Hamburg:  Calendar Girl

Big hat tip to http://www.fdalawblog.net for the excellent reporting.

GAO Report Says That FDA Has Met Most PDUFA Performance Goals; Agency Plans to Take Steps to Address Lingering Stakeholder Concerns

A report released earlier this week by the Government Accountability Office (“GAO”) says that FDA has met most of the Agency’s PDUFA performance goals for priority and standard original NDA and BLA submissions and for priority and standard original efficacy supplements to approved NDAs and BLAs, although in each case FDA review times have increased slightly.  The GAO’s analysis covers applications in the Fiscal Year 2000 to 2010 cohorts, as well as preliminary information for applications submitted in Fiscal Year 2011. 

The GAO report was sent to Senators Richard Burr (R-NC) and Tom Coburn (R-OK).  Both Senators have long criticized FDA, saying that the Agency‘s “regulatory malaise” harms patients and manufacturers.  Senator Burr has also threatened to delay the passage of PDUFA and other user fee legislation unless FDA speeds up application approval times.  In addition, Senator Burr was successful in getting an amendment added to the FDA appropriations bill passed last year that seeks to “improve the transparency and accountability of the FDA in order to encourage regulatory certainty and innovation on behalf of America’s patients.”  That amendment requires the submission of information to Congress on, among other things,

• “the average number of calendar days that elapsed from the date that drug applications (including any supplements) were submitted to such Secretary under [FDC Act § 505] until the date that the drugs were approved under such section 505;” and
• “the average number of calendar days that elapsed from the date that [BLAs] (including any supplements) were submitted to such Secretary under [PHS Act § 351] until the date that the biological products were licensed under such section 351.” 

The GAO’s analysis shows that except for Fiscal Year 2008, FDA met PDUFA goals in all of the Fiscal Year 2000 to 2010 cohorts.  (FDA recently provided similar statistics in testimony before Congress.)  Moreover, the GAO found that an average of 44% of all original NDAs and BLAs submitted to FDA in Fiscal Years 2000 to 2010 were approved during the first review cycle and 75% were ultimately approved.  FDA and industry stakeholders the GAO interviewed suggested that FDA failed to meet Fiscal Year 2008 goals as a result of implementation of the Risk Evaluation and Mitigation Strategy (“REMS”) requirements added to the FDC Act by the 2007 FDA Amendments Act.

Although FDA met most PDUFA goals for the Fiscal Year 2000 to 2010 application cohorts (and is on track to meet Fiscal Year 2011 goals for the applications submitted in that cohort), the GAO’s analysis (reflected in the tables below) shows that average FDA review times (i.e., the time elapsed from when FDA received a submission until it issued an action letter) hve increased slightly from Fiscal Year 2000 through Fiscal Yeat 2010 for both priority and standard NDAs and BLAs and priority and standard original efficacy supplements to approved NDAs and BLAs.

With respect to Senator Burr’s request for the average number of calendar days that elapsed from the date of NDA or BLA (including supplement) submission to final FDA action, the GAO says that it was unable to calculate average FDA review times in any meaningful way because most cohorts were still open; that is, “fewer than 90 percent of submissions had received a final action such as approval, denial, or withdrawal.”  Specifically, for priority original NDAs and BLAs, only four cohorts had at least 90% of submissions closed (Fiscal Years 2001, 2002, 2005, and 2006), and for standard original NDAs and BLAs, only one cohort had at least 90% of submissions closed (Fiscal Year 2002).  For priority efficacy supplements, only four cohorts had at least 90% of submissions closed (Fiscal Years 2000, 2001, 2004, and 2007), and for standard efficacy supplements, only one cohort had at least 90% of submissions closed (Fiscal Year 2005).

Stakeholders the GAO interviewed identified some issues that they believe hamper the NDA and BLA approval process, including REMS implementation, the use of outside expertise for reviewing applications, insufficient communication between FDA and stakeholders, and a lack of predictability and consistency in FDA reviews.   FDA commented in the Agency’s response to the GAO report that it is taking or has agreed to take steps (as part of PDUFA V) that may address these issues, including issuing new guidance, establishing new communication-related performance goals, training staff, and enhancing scientific decision making.

  Read More & Comment...

Heavy Meddle

05/03/2012 09:12 AM |

The American Recovery and Reinvestment Act (aka, “the stimulus package”) provided AHRQ with $29.5 million for a program on academic detailing and the “communication of CER results to physicians.”

One contract, for $11.7 million, went to Total Therapeutic Management (TTM) and is specifically intended for physician outreach and education.

(TTM is a company that focuses on chart abstraction, data mining, and physician and patient education for a predominantly commercial client base -- health plans, pharmacy benefit managers, employers, and pharmaceutical companies, etc.)

The goal of this contract is to integrate AHRQ’s comparative effectiveness research, products, and tools into clinical practice through 9,000 on-site, face-to-face visits with clinicians, nurses, health plan formularies, benefit managers, and other healthcare professionals.

I recently interviewed Barry Patel, the president of TTM.  Here are some snippets from our conversation:

How will the government decide which doctors are to be visited? Will ‘‘high prescribers’’ of on-patent medicines be on a priority list?

TTM’s top priority is ‘‘high volume’’ practices across 150 Metropolitan Statistical Areas (MSAs). So, rather than focusing on offices with disproportionately high negative patient outcomes, the government is directing its efforts against those doctors who are high prescribers—which is a pretty good indicator about what government detailing is all about—decreasing cost rather than improving care.

When it comes to government detailing (at the taxpayers’ expense), what are the metrics for success?

According to Mr. Patel, the only metrics are whether or not a physician says the sessions have been useful and asks the detailer to come back to discuss other topics. In other words, the metrics are subjective and anecdotal -- not clinical.

Interestingly, Mr. Patel doesn’t even agree with either the term academic detailing or counter detailing. ‘‘We aren’t counter anything. We’re not there to undo anything. It’s not good versus bad. Our visits aren’t details, they’re the beginning of a process.’’ And, as far as ‘‘academic’’ goes, Mr. Patel uses that term because “that’s the phrase AHRQ uses and placed in the contract. Our people are patient-centered outcomes consultants, PCOCs.” And “his people” are largely pharmacists and nurses.

A former Merck employee, Patel likens his PCOCs more to pharmaceutical company Medical/Science Liaisons (MSLs) than field representatives. ‘‘They’re not discussing product-specific information, but the findings of comparative effectiveness studies.”

How does TTM schedule their appointments with targeted physicians?

According to Mr. Patel, when his ‘‘outreach experts’’ phone physicians to request appointments, the fact that the meeting will result in CME credits is always mentioned. Would a pharmaceutical company be permitted to offer such an enticement? Would such an offer be ‘‘sunshine-able’’ under state and federal guidelines? And, if so, why don’t government detailers have to share the details of their valued benefactions?

Interestingly, according to the Accreditation Council for Continuing Medical Education (ACCME), government is exonerated from having a commercial interest. (A commercial interest is any entity producing, marketing, re-selling, or distributing healthcare goods or services consumed by, or used on, patients.)

Our nation’s single largest payer, Uncle Sam, is not deemed to have a conflict of interest when it comes to designing and providing physician CME.

What’s wrong with this picture?

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The Rise of the Administrative State

05/02/2012 12:01 PM |

Bob Moffitt,  The Heritage Foundation’s senior fellow in domestic and economic policy studies gave a lecture to members of Congress recently.  It addresses the historical and cultural impulse that has turned government into a mechanism for producing equality of results instead of a system the that increases opportunity by advancing liberty.   

At a time when more government agencies have been created to regulate and redistribute economy activity, with a focus on finance,  energy production and of course health care, it is useful to reflect upon how this fundamental shift in power and direction will shape our future.   Most important in my mind is the way in which the administrative state is being used to create ideological no-fly zones for those who disagree or challenge the guiding principles of the agency and the professional experts who feed off it.  

Bob Moffitt's lecture is an important contribution to this discourse.    Harry Truman noted" Once a government is committed to the principle of silencing the voice of opposition, it has only one way to go, and that is down the path of increasingly repressive measures, until it becomes a source of terror to all its citizens and creates a country where everyone lives in fear.”   Keep Truman's warning in mind as you read Moffitt's lecture. 

http://www.heritage.org/research/reports/2012/03/why-congress-must-confront-the-administrative-state?query=Why+Congress+Must+Confront+the+Administrative+State  Read More & Comment...

And speaking of benefit/risk ...

05/02/2012 06:57 AM |

BioCentury reports:

IOM: FDA should consolidate benefit-risk data for each drug

The Institute of Medicine said FDA should consolidate benefit-risk information for approved drugs into publicly available repositories created for each product. In a report released Tuesday, IOM said the agency already collects much of the information needed but that it is spread across multiple records. The report recommended the creation of a publicly available document containing information for each product from its approval throughout its entire time on market, including safety issues, regulatory actions and any restrictions. FDA said in a statement it supports the general concept, but added that it would be "very challenging to implement this recommendation within our current resources without seriously compromising other critical regulatory activities."

IOM also noted in the report that there is no universal set of criteria to determine when FDA should require a postmarketing study to evaluate a drug's safety. However, the report noted that the agency should require additional post-marketing safety research when a drug's benefits or risks are particularly uncertain, including first-in-class drugs approved based on surrogate endpoints, drugs for which endpoints provide conflicting evidence about risk or drugs with a strong biological rationale for a particular side effect. IOM, which conducted the review at FDA's request, issued a preliminary report in 2010.

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RIP RCTs

05/02/2012 01:19 AM |

An article in The Scientist makes much about how using raw data from randomized clinical trials can change results. But while the article trumpets the importance of transparency and equates more clinical trial data with better science, in fact the massive amount data being generated from randomized clinical trials is nearly beside the point because data from real world use and the collection of biological and genomic information about treatment response can be collected more quickly and at a lower cost. So why are academics and interest groups obsessing about more trial data? I think data diving gives them a reason to apply for grants and an endless supply of cherries to pick to score policy and political points. Dumping more data based on evidence generation methods of the 19th century is no way to take patients and their needs seriously. http://the-scientist.com/2012/05/01/data-diving/   Read More & Comment...

Avandia and Children With Type 2 Diabetes 1, Steve Nissen 0.0

04/30/2012 10:14 AM |

A recent article in the New England Journal of Medicine evaluating the results of an experiment on how to treat type 2 diabetes in children raises some interesting questions:

http://www.nejm.org/doi/full/10.1056/NEJMoa1109333?query=featured_home#t=articleBackground

The Treatment Options for Type 2 Diabetes in Adolescents and Youth (TODAY) study found that metformin and Avandia were superior to other treatments in controlling the disease in kids.

I have two questions:  Will Steve Nissen admit he was wrong, again?  Also,  will doctors start to use existing diagnostics that identify those who do not respond to metformin, at least in kids.  Isn't that choosing wisely?  



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