OCD at OGD and the BioSImilar Two-Step

08/04/2011 07:18 AM |

The FDA is reorganizing it’s Office of Generics Drugs (OGD) to “improve coordination, communication, efficiency and to enhance the Office’s ability to assure that all generic drugs are safe, effective, high quality and interchangeable to the brand name drug product/reference listed drug.”

“Interchangable?”  Hm.

OGD adds another Division to both the Bioequivalence and Chemistry programs, and converts the Microbiology and Clinical Review Staffs into Divisions.  It also formalizes the position of Deputy Director for Science and Chemistry.  Here is the new structure.

OFFICE OF GENERIC DRUGS 
Division of Bioequivalence I 
Division of Bioequivalence II 
Division of Labeling and Program Support 
Division of Microbiology 
Division of Clinical Review 
Division of Chemistry I 
Division of Chemistry II 
Division of Chemistry III 
Division of Chemistry IV

On a biosimilar note, BioCentury reports that the FDA reiterated plans for a two-step regulatory process for biosimilars that includes evaluating similarity and then interchangeability, according to an article published in the New England Journal of Medicine on Wednesday.

 

The agency will first review the similarity of a biologic to an FDA-approved reference product using a "totality-of-the-evidence" approach, which includes a review of comparative analytic data and in vitro data. Based on the initial review, FDA will determine if additional animal or human studies are required for approval as a biosimilar.

 

To meet the higher hurdle of interchangeability with a reference product, a manufacturer must demonstrate that the biosimilar is expected to produce the same clinical result in patients, and that switching between the two products does not increase a patient's risk compared with continued use of the reference product.

 

Earlier this year, senior FDA officials began outlining plans for the two-step process. The agency expects to issue a series of guidance documents on the process

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And that's not even counting what's behind the couch cushions in Peggy's office

08/03/2011 10:51 AM |

According to the Pink Sheet, FDA has amassed a large reserve fund throughout PDUFA IV, but will likely be forced to spend most of it in the next fiscal year as application volume declines and more personnel move to the agency's new headquarters.

 

FDA ended FY 2010 with more than $150.6 million in the carryover account, a 252% increase from the $42.8 million in the account as of June 30, 2006. The agency said aside from excess fee collections during PDUFA III, the fund also included fees saved for the agency relocation to its White Oak campus that were allocated during PDUFA IV and a reserve for refunds.

 

Spending the carryover funds will allow FDA to reduce the size of the user fee the upcoming fiscal year, although that will probably feel like a small comfort to sponsors facing a more than 19% increase in FY 2012, which brings the total fee for a full application requiring clinical data to $1.842 million.

 

Two of the largest carrover balance expenses will be personnel-related. The agency said in the notice it expects to spend about $37.9 million to move the Center for Biologics Evaluation and Research to its White Oak campus in Silver Spring, Md. by FY 2014. CBER now is located in nearby Rockville, Md.

 

The General Services Administration already approved the project and President Obama allocated $23.68 million in his FY 2012 budget request to complete the lab and ensure it is ready for use by FY 2014. FDA said without the investment, it would be forced to pay rent for a new lab it cannot use and for an old lab it cannot vacate.

 

FDA also plans to spend $29.8 million from the carryover balance in FY 2011 and FY 2012 to pay for 53 new full-time equivalent employees. The hires were authorized in FY 2009 to handle additional work created by the FDA Amendments Act's drug safety provisions.

 

Another $2.5 million of the carryover fund must be reserved for refunds, the agency said in the notice.

 

FDA also is obligated by PDUFA rules to use some carryover money to offset industry fee overpayments made from FY 2002 and FY 2007. That nearly $31 million payment lowered the revenue target and, subsequently, the fees sponsors will pay. The agency said in the notice fee payments from FY 2008 through FY 2011 will be more than $27.2 million less than the amounts appropriated, which would not require an offset payment.

 

The agency also will be forced to cover two years of shortfalls with the carryover balance that were caused by slumping submissions. The agency projected an $8.4 million revenue shortfall in FY 2011 and an $8.7 million shortfall in FY 2012, according to the notice.

 

After receiving 17 fewer full paid applications in 2010, the agency adjusted its assumptions for annual volume to be 5.5 submissions fewer in FY 2011 and FY 2012, which caused the shortfalls. FDA received 118.4 fee paying full application equivalents in FY 2010 and estimated it would receive 102.5 through the end of FY 2011. Both were near record lows since PDUFA was implemented in FY 1993.

 

The shortfalls also likely influenced an FDA decision to lower its revenue target for operational reserves in early FY 2013. The agency is allowed to add three months of operating expenses to its fee calculations for the final year of a PDUFA cycle so it can "assure sufficient operating reserves" at the start of the next fiscal year, which would be the first for PDUFA V.

 

Three months of revenue at that time would be $169.2 million. When the remaining $32.4 million in the carryover balance is subtracted out, the agency would need another $136.9 million.

 

But FDA decided it would "assume more risk" and only require two months of operating expenses for the reserve, lowering the required amount to $80.4 million. The agency said including the full three months in FY 2012 "poses a substantial burden on the regulated industry at a time when it is undergoing significant financial strain," according to the notice.

 

The decision will save sponsors of applications requiring clinical data about 8% in fee payments. The fee would have been about $1.99 million if FDA demanded a three-month reserve.

 

When the House approved the FY 2012 Agriculture/FDA appropriations bill in June, it gave the agency a miniscule $3 million budget increase. User fee revenue increases were used to offset a $285 million cut in federal funding.

 

For more discussion of PDUFA, have a look (and bookmark) www.modernmedicines.com. It’s an important and useful site from the folks at Eli Lilly & Co on all-things PDUFA.

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Got tonsillitis? Keep calm and carry on.

08/02/2011 06:58 AM |

Remember “SiCKO?” That was the Michael Moore “documentary” that showed how, if the US would adopt the NHS model, our own healthcare problems would vanish.

Well, as this article from the British newspaper, “The Independent,” shows – well, reality bites.

(Can you imagine President Obama or any member of Congress telling parents that their kids can't have their tonsils out until they have had seven -- that's right seven -- bouts of tonsillitis?)

Cataracts, hips, knees and tonsils: NHS begins rationing operations

Almost two-thirds of trusts affected as cuts bite

Hip replacements, cataract surgery and tonsil removal are among operations now being rationed in a bid to save the NHS money.

Two-thirds of health trusts in England are rationing treatments for "non-urgent" conditions as part of the drive to reduce costs in the NHS by £20bn over the next four years. One in three primary-care trusts (PCTs) has expanded the list of procedures it will restrict funding to in the past 12 months.

Examples of the rationing now being used include:

* Hip and knee replacements only being allowed where patients are in severe pain. Overweight patients will be made to lose weight before being considered for an operation.

* Cataract operations being withheld from patients until their sight problems "substantially" affect their ability to work.

* Patients with varicose veins only being operated on if they are suffering "chronic continuous pain", ulceration or bleeding.

* Tonsillectomy (removing tonsils) only to be carried out in children if they have had seven bouts of tonsillitis in the previous year.

* Grommets to improve hearing in children only being inserted in "exceptional circumstances" and after monitoring for six months.

* Funding has also been cut in some areas for IVF treatment on the NHS.

The alarming figures emerged from a survey of 111 PCTs by the health-service magazine GP, using the Freedom of Information Act.

Doctors are known to be concerned about how the new rationing is working – and how it will affect their relationships with patients.

Birmingham is looking at reducing operations in gastroenterology, gynaecology, dermatology and orthopaedics. Parts of east London were among the first to introduce rationing, where some patients are being referred for homeopathic treatments instead of conventional treatment.

Medway had deferred treatment for non-urgent procedures this year while Dorset is "looking at reducing the levels of limited effectiveness procedures".

Chris Naylor, a senior researcher at the health think tank the King's Fund, said the rationing decisions being made by PCTs were a consequence of the savings the NHS was being asked to find.

"Blunt approaches like seeking an overall reduction in local referral rates may backfire, by reducing necessary referrals – which is not good for patients and may fail to save money in the long run," he said. "There are always rationing decisions that have to go on in any health service. But at the moment healthcare organisations are under more pressure than they have been for a long time and this is a sign of what is happening across many areas of the NHS."

According to responses from the 111 trusts to freedom-of-information requests, 64 per cent of them have now introduced rationing policies for non-urgent treatments and those of limited clinical value. Of those PCTs that have not introduced restrictions, a third are working with GPs to reduce referrals or have put in place peer-review systems to assess referrals.

In the last year, 35 per cent of PCTs have added procedures to lists of treatments they no longer fund because they deem them to be non-urgent or of limited clinical value.

Some trusts expect to save over £1m by restricting referrals from GPs.

Chaand Nagpaul, a member of the British Medical Association's GPs committee, said he was concerned about PCTs applying different low-priority thresholds and rationing access to treatments on the basis of local policies.

He said the Government needed to decide on a consistent set of national standards of "low priority" treatments to help remove post-code lotteries in provision. "Patients and the public recognise that with limited resources we need to make the maximum health gains and so there needs to be prioritisation. What is inequitable is that different PCTs are applying different thresholds and criteria," he said.

A Department of Health spokesman said: "Decisions on the appropriate treatments should be made by clinicians in the local NHS in line with the best available clinical evidence and Nice [National Institute for Health and Clinical Excellence] guidance. There should be no blanket bans because what is suitable for one patient may not be suitable for another."

Bill Walters, 75, from Berkshire, recently had to wait 30 weeks for a hip operation instead of the standard 18. "I believe that the Government is doing this totally the wrong way," he said.

Case study: 'They changed the rules to save money'

Anne Ball, 71, is a retired business consultant who used to work in electronics

"I have bilateral cataracts and under the original NHS criteria I was entitled to have at least one of mine treated – but then the West Sussex health authorities decided to change the threshold level to save money.

"It's like looking through gauze. Everything is foggy, and I've got quite a large 'floater' in my left eye. The consultant was as distressed as me, having to tell me, and he thought with my eyesight he wouldn't be able to function.

"I've appealed because the cataracts are having a significant impact on my quality of life and it's left me depressed and fearful about my low vision, which will continue to deteriorate. The new guidelines mean that people who fall below the standard set by the DVLA still do not qualify to have surgery. My vision is not good enough to drive at night.

"I'm not a cranky old lady. I'm the chair of a local village charity and I do a lot of computer work that is affected.

"It will just store up costs for future years, putting a strain on resources as more patients will end up in falls clinics. The longer you put it off the more complex the operation becomes and the riskier it is for the patient."

  Read More & Comment...

Obamacare in the Crosshairs in 2012

08/01/2011 05:09 PM |

This article in the Washington Examiner makes an excellent point:  Obamacare will be a centrai element of the 2012 campaign.
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Turning Point or Tipping Point?

08/01/2011 08:11 AM |

According to FDA Commissioner Peggy Hamburg there  is “a lot of energy” spent by industry and FDA on DTC issues … I think it’s a missed opportunity to do something much better if we’re going to be talking about drugs and health to the public at large. Certainly one of the discussions that I’ve been engaged in with industry is how can we use some of the time and money that they’re putting into this to really address broader public health concerns and broader education with the public about important health issues.”

I agree!  Have a look at this article from Health Affairs for more on this issue.

The article’s conclusion –

“Working together with industry, we can make a difference. We can make DTCA a more potent, precise, and persuasive tool on behalf of the public health. And rather than rubbing the lamp and wishing, we need to burn the midnight oil and work harder to make it a reality—because an educated consumer is our best customer.”

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Is Big Pharma a Social Media Nin-compoop?

08/01/2011 07:50 AM |

There came a time w hen the risk to remain tight in the bud was more painful than the risk it took to blossom.  ~Anaïs Nin

Just back from the ePharma West conference where I was pleased to give a keynote address on the future of social media.

Some snippets:

Social media is communications at the speed of life. As Marshall McLuhan wrote, “"At electric speed, all forms are pushed to the limits of their potential." 

(Replace “electric” with “digital” and it’s amazing how prescient McLuhan was.  That’s genius.) That’s a wonderful challenge, to be pushed to the limits of our potential. But wait, it gets more complicated.

Healthcare social media has precious few rules.  But there’s only one Golden Rule -- transparency. 100% transparency. 100% of the time.  You can’t airbrush social media.

Social media for regulated industry is a wonderful green field of opportunity. But to maximize the opportunity, we must accommodate the reality of a messier world.  Social media, almost by definition, is messy – and the regulatory framework (or lack thereof) is equally so. And it’s not likely to get much better. Get used to it.

Embracing social media means embracing regulatory ambiguity. And that’s a paradigm shift for an industry that has (in a post-Vioxx world) been going in precisely the opposite direction.

Social media (and its game-changing  opportunities) demands a move away from the cautious tactics of the Vioxx Populi towards a better understanding of the digital Vox Populi. And that means more than sponsored Google links and branded Facebook pages with the interactivity turned off.

It means mixing it up with real people in real time. And when it comes to FaceBook, it means – turn the interactivity on! 

It’s not going to be easy, or risk-free, or inexpensive.  And whatever social media “marketing models” companies build will have to be elastic – just like the media environment in which they are designed to operate.

Benjamin Franklin once said: “Every problem is an opportunity in disguise.” While Facebook strategies and approaches have to be reexamined, Mr. Zukerberg’s medical mandate provides pharmaceutical marketers with an excellent opportunity to finally acknowledge and embrace the full capabilities of two-way social communication writ large.

FaceBook’s changes represent an opportunity for regulated industry to learn, understand and embrace the three key tenets of Pharmaceutical Marketing 3.0:

1. The Rise of the “Face of Pharma”

For the past 20 years, the overwhelming majority of pharmaceutical marketing budgets were dedicated to promoting specific products.

Now, due to both a less robust drug development pipeline and an increase in the rates of patent expiry, the next era of pharma marketing will put the company – and it’s corporate reputation – front and center.

When you think about it (if you allow yourself to think about it), its a perfect match for social media where transparency is the most urgent, non-negotiable and magnificent mantra.

Not third party groups, not KOLs (although these traditional avatars have their place) – but the company speaking on behalf of itself and its products.  What a concept!

2. The Role of Social Media in the Era of Post-Patent Medicine

I believe that the blockbuster era of the pharmaceutical industry will be replaced by the Era of Post-Patent Medicine. To compete in an era of generics and biosimilars, Pharma companies will need not only a robust portfolio of lower cost medications, but an army of brand loyalists.

Communications programs, supported by social media must be one tool. Why? Because it’s where the people are.

3. Social Media Can Help Increase Patient Education and Prescription Compliance

You know the numbers. It’s estimated that Pharma loses $30 billion a year in patient non-compliance. True two-way social media has the potential to serve as a new and puissant health education platform by helping to keep patients informed of the dangers of non-compliance by earning their trust through transparent dialogue. And that’s twice as true when it’s mobile-based.

As another conference presenter, Dr. James Fowler, of the University of California at San Diego opined, “Pharma must realize their own network power.”

Amen.

PS/ I am also pleased to announce that I have joined the board of the Digital Health Coalition - a nonprofit corporation - seeks to promote responsible innovation via digital marketing and communications in a connected world. Their first initiative is focused on the rapidly evolving space of social media and seeks to promote a world where patients, physicians, and brands can connect, empower, and drive positive health outcomes. Individuals interested in learning more about the Digital Health Coalition are encouraged to visit:

http://www.digitalhealthcoalition.orgwww.digitalhealthcoalition.org

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To Burr, with Love

07/29/2011 07:46 AM |

To Burr, with Love

BioCentury reports that Senator Richard Burr (R-N.C.) threatened Thursday to delay reauthorization of medical device and prescription drug user fee legislation unless FDA implements steps to improve and speed product reviews. Speaking at a Senate committee on Health, Education, Labor and Pensions hearing, Burr said user fees, especially for medical devices, have not improved FDA performance and expressed skepticism that increasing user fees would improve the situation. He said reauthorization will become "a very slow and laborious process" unless the new legislation has measurement tools to track whether a fee system produces better outcomes

HELP committee Chairman Sen. Tom Harkin (D-Iowa) pushed back, saying that safety and efficacy are more important than speed to market. He also said FDA is understaffed and underfunded, so more money could improve its review performance.

They’re both right.

PhRMA wants dedicated biosimilars funding

The merits of creating a dedicated appropriation for biosimilars reviews has emerged as a point of contention in closed door FDA-hosted biosimilars user fee stakeholder discussions. In a July 24 letter from the Pharmaceutical Research and Manufacturers of America (PhRMA) to FDA, the trade association came down solidly on the side of creating a funding stream for biosimilar reviews that is separate from PDUFA-funded drug reviews.

 

PhRMA also called for a separate biosimilars user fee "trigger," or minimum amount Congress must allocate for biosimilars reviews to enable FDA to spend user fees. A trigger was built into PDUFA with the goal of making user fees supplement, not replace, federal funding. The law creating a biosimilars pathway called for FDA to fund biosimilars reviews from PDUFA funds until October 2012, when biosimilars user fees are expected to kick in. Applying PDUFA to biosimilars past October 2012 would drain resources from reviews of innovative medicines, according to the PhRMA letter.

Lack of money is the root of all evil.
-- George Bernard Shaw

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Part D Hearty

07/29/2011 07:30 AM |

This week the mainstream media discovered patent expirations and the headlines rang, “drug prices plummet!”

 

But they missed the real story.

 

From JAMA:

Implementation of Medicare Part D and Nondrug Medical Spending for Elderly Adults With Limited Prior Drug Coverage

 

1.      J. Michael McWilliams, MD, PhD;

2.      Alan M. Zaslavsky, PhD;

3.      Haiden A. Huskamp, PhD

 

Author Affiliations

 

1.  Author Affiliations: Department of Health Care Policy, Harvard Medical School (Drs McWilliams, Zaslavsky, and Huskamp); and Division of General Internal Medicine and Primary Care, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School (Dr McWilliams), Boston, Massachusetts.

Abstract

Context Implementation of Medicare Part D was followed by increased use of prescription medications, reduced out-of-pocket costs, and improved medication adherence. Its effects on nondrug medical spending remain unclear.

Objective To assess differential changes in nondrug medical spending following the implementation of Part D for traditional Medicare beneficiaries with limited prior drug coverage.

Design, Setting, and Participants Nationally representative longitudinal survey data and linked Medicare claims from 2004-2007 were used to compare nondrug medical spending before and after the implementation of Part D by self-reported generosity of prescription drug coverage before 2006. Participants included 6001 elderly Medicare beneficiaries from the Health and Retirement Study, including 2538 with generous and 3463 with limited drug coverage before 2006. Comparisons were adjusted for sociodemographic and health characteristics and checked for residual confounding by conducting similar comparisons for a control cohort from 2002-2005.

Main Outcome Measure Nondrug medical spending assessed from claims, in total and by type of service (inpatient and skilled nursing facility vs physician services).

Results Total nondrug medical spending was differentially reduced after January 1, 2006, for beneficiaries with limited prior drug coverage (−$306/quarter [95% confidence interval {CI}, −$586 to −$51]; P = .02), relative to beneficiaries with generous prior drug coverage. This differential reduction was explained mostly by differential changes in spending on inpatient and skilled nursing facility care (−$204/quarter [95% CI, −$447 to $2]; P = .05). Differential reductions in spending on physician services (−$67/quarter [95% CI, −$134 to −$5]; P = .03) were not associated with differential changes in outpatient visits (−0.06 visits/quarter [95% CI, −0.21 to 0.08]; P = .37), suggesting reduced spending on inpatient physician services for beneficiaries with limited prior drug coverage. In contrast, nondrug medical spending in the control cohort did not differentially change after January 1, 2004, for beneficiaries with limited prior drug coverage in 2002 ($14/quarter [95% CI, −$338 to $324]; P = .93), relative to beneficiaries with generous prior coverage.

Conclusion Implementation of Part D was associated with significant differential reductions in nondrug medical spending for Medicare beneficiaries with limited prior drug coverage.

 

http://jama.ama-assn.org/content/306/4/402.full.pdf+html

 

http://jama.ama-assn.org/content/306/4/402.short

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Progressive Improvements

07/28/2011 03:37 PM |

Recently BIO released a white paper on post-PDUFA FDA reform.  (Full details can be found here.)

Many good ideas – but one that gets the blood flowing (whether you’re for or against):  “progressive approvals.”

Sounds good (at least in theory) to many. But there are some tough questions. As an FDA insider opined, What exactly would the standard for progressive approval be? And would every one of the progressive approvals come with some sort of access control?”

 

Attempting to address some of these unknowns (via an op-ed in the Wall Street Journal) are Michael Boldrin (chair of the economics department at Washington University in St. Louis) and S. Joshua Swamidass (medical professor at Washington University).

 

They write:

 

“We can reduce the cost of the drug companies' bet by returning the FDA to its earlier mission of ensuring safety and leaving proof of efficacy for post-approval studies and surveillance.”

 

I’m not sure where they get the “earlier mission” statement, but let’s allow them to continue.

 

“In exchange for this simplification, companies would sell medications at a regulated price equal to total economic cost until proven effective, after which the FDA would allow the medications to be sold at market prices.”

 

Leaving the difficulty of determining what such a “regulated price” might be (and don’t for a minute believe that the devil isn’t in the details), an even tougher question (and a real rabbit hole of one) is the issue of “until proven effective.”

 

What does “effective” mean? Does it mean “cure” or “remission?” Does it mean "cost effective?" Or extension of life? And, if so, for how long?  5 years?  5 months? 5 minutes?  And who makes the call? 

 

According to Boldrin and Swamidass, “Doing so will improve all of our lives, decrease the cost of health care, and unleash the next wave of medical innovation.

 

It’s a weak argument in support of an important discussion.

 

Do we really want to open the door for tacit price controls and healthcare technology assessment in return for a very questionable upside?

Safety without efficacy?  Really?

Better to pursue a path last publicly discussed in April 2009 when Merck agreed to peg what the insurer Cigna pays for the diabetes drugs Januvia and Janumet to how well Type 2 diabetes patients are able to control their blood sugar.

 

Now that’s progressive.

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Media Drops BPA Ball

07/27/2011 04:01 PM |

Scary stories about Bisphenol-A (BPA) over the past  year?  About 100

Stories reporting on the study that demonstrated that even when human cram themselves with BPA-heavy diets scientists "find the substance (in urine and blood)  below our ability to detect them, and orders of magnitude lower than those causing effects in rodents exposed to BPA"?  17

As Trevor Buttorworth points out in his blog on Forbes: " the media have ignored the stunning finding – double checked before publication – that overturns pretty much everything the press has told the public about this common chemical."  tinyurl.com/3wcgbl6

The Enviromental Protection Agency's response to the study?

EPA considers new call for toxicity testing of BPA

"The Environmental Protection Agency solicited public comment, July 26, about whether to require new toxicity testing and environmental sampling of bisphenol A, an ingredient in many plastics and food-contact resins." 

All of which will be duly reported by the media in he-said, she-said fashion. 
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Is FDA A Deadend for Medical Devices and Diabetic Kids??

07/27/2011 10:15 AM |

Another depressing example of how the path for approval of  medical devices has become constipated at the FDA in response to the so-called consumer advocates such as Public Citizen, Consumers Union, Merrill Goozner, Diana Zuckerman.    To quote British philosopher Isaiah Berlin, "disregard for the preferences and interests of individuals alive today in order to pursue some distant social goal that their rulers claimed is their duty to promote has been a common cause of misery of people throughout the ages."   The groups above have been a constant source of misery in our time.


www.sfgate.com/cgi-bin/article.cgi

Lives wasted as FDA stalls on diabetes care

Pam Sagan

Wednesday, July 27, 2011

For more than 20 years, my daughter Piper has lived with the constant, frightening, deceptive and malicious disease called type 1 diabetes. Piper has always been prone to the kind of hypoglycemic - low blood sugar - life-threatening attacks that come on hard, fast and without warning. She almost drowned as a youngster after becoming unconscious from low blood sugar. In college, she went into hypoglycemia while she slept and didn't wake up in the morning. Fortunately, she was discovered and emergency care saved her life.

Unfortunately, the Food and Drug Administration has been dragging its feet on technologies that could revolutionize diabetes care and make these kinds of episodes a thing of the past. Key trials are on hold and it looks to be years more before these proven, life-saving technologies are available for patients in the United States. Meanwhile, kids are dying.

Every hour of every day, individuals with type 1 diabetes have to balance insulin, food and activity to try to prevent low and high blood sugars, and the devastating and costly complications: seizures, comas, kidney failure, heart disease, blindness and amputations. The human cost is incalculable; the economic cost isn't: Diabetes costs our nation more than $174 billion a year and $1 in $3 of Medicare spending goes to care for people with diabetes.

Perhaps the most gut-wrenching story of diabetes is the specter of "dead in bed" - kids found dead in the morning after a completely normal evening. Dead in bed occurs because blood sugar levels can suddenly change. When this happens while sleeping you are unable to adjust insulin to right the body's blood sugar, which can be life threatening.

We know how to prevent these attacks, but we don't - at least not here in the United States. Breakthrough technologies that protect against dangerous diabetes episodes are already available elsewhere, but not at home. Low-glucose suspend systems have been approved for nearly three years and used safely in more than 40 countries worldwide, but they are not available in the United States because of the FDA's unnecessarily slow process.

These pumps stop delivering insulin automatically when a monitor indicates that the body's glucose levels are low. The low-glucose suspend technology is the first phase of an artificial pancreas, a combination of a continuous glucose monitor and an insulin pump with software that would communicate between the two to automatically monitor glucose levels and administer insulin doses. The artificial pancreas would address both high and low blood sugar levels. In 2006, the FDA recognized the importance of this technology and placed the artificial pancreas on its Critical Path Initiative. But now key trials are on hold until the FDA provides a roadmap for outpatient studies. A draft is promised in December.

It should not have taken this long, and must not take any longer. When I testified before Congress, my message was simple: this technology could revolutionize diabetes care and it is imperative that the FDA provide reasonable guidance immediately. Waiting is not an option. My daughter's life, and those of millions of people with diabetes, depends on it.

Pam Sagan of Los Altos is a former board member of the Juvenile Diabetes Research Foundation International.

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Waive this!

07/26/2011 01:01 AM |

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FDA Mission Creep: There's an App for that

07/25/2011 08:37 AM |

Mission creep is a worrisome thing – especially at the FDA.

Awhile back there were some folks at CDRH who believed that the mobiles that you hang over a baby’s crib should be classified as a medical device because they can impact vision development.

No – really.

Fortunately, cooler minds prevailed and sanity won the day.

Today, the issue is whether or not some mobile apps can be considered medical devices.  It’s important for many reasons, not the least of which is that over-regulation or the threat of FDA action will slow both the development and adoption of mobile technologies for a variety or urgent public health purposes.  Adherence and compliance come to mind as well as safety issues relative to appropriate use/safe use.

To that end, an interesting audio interview in the Burrill Report. It's with Joe Smith, chief medical and science officer for the Gary & Mary West Wireless Health Institute about new draft guidance from the FDA on medical apps, how the agency is approaching these products, and whether this provides the clarity needed to promote investment and innovation in this new world of digital health.

The interview can be found here.

This issue, BTW, is yet another reason why the name of CDRH (the Center for Devices and Radiological Health) needs to change to the Center for Medical Technology.

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Friday reading

07/22/2011 12:35 PM |

More here.

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FDA Diabetes Advisors Wimp Out

07/20/2011 08:00 PM |

A drug that reduces blood suger by removing through the urine would be huge. 

But FDA advisors are too scared about the possible risks of the drugs to allow diabetics and doctors to see how they work in the real world.

"Several committee members said they could have voted either way.

“I changed my mind about four times in the last 10 seconds,” said Erica H. Brittain, a statistician at the National Institutes of Health who voted no. "

The fate of a new drug should be  decided by FDA's Hamlets?


 

The biggest safety concern was that in clinical trials, patients who got the drug were more likely to develop breast and bladder cancers than those in the control groups.

About 0.4 percent of women taking the drug got breast cancer, compared with 0.1 percent of the women in the control groups. About 0.3 percent of men getting the drug got bladder cancer, compared with about 0.05 percent of men in the control groups.

The numbers were very small, however, making it hard to draw definitive conclusions. Bristol-Myers and AstraZeneca argued that many of the cancers occurred too soon to have been caused by the drugs."

And get this:

"The committee members agreed that more study of the possible cancer risks and other safety questions would be needed. Those who voted no mainly believed that the studies needed to be done before approval, even though that might delay approval by years. "

How many people will die from diabetes related complications because this medicine is not approved in order to organize trials that will likely never resolve the issue? 

This is pathetic. The advisory committee members are being haunted by the ghost of Steve Nissen.

www.nytimes.com/2011/07/20/business/diabetes-drug-dapagliflozin-rejected-by-fda-panel.html  Read More & Comment...

Total Eclipse of Baltimore Sun By Anti-Vax Stupidity

07/19/2011 05:19 PM |

The Baltimore Sun has become a safe haven for anti-vax pseudo science and it's lethal stupidity.   And it does so by repeating the cardinal sin of most science or health care reporters: treating conspiracy driven theories (consisting of  an alleged link between alleged harms  and the profit-driven actions and products of BIG PHARMA.)  as scientific as, well, real science without demanding to see any scientific proof of said theory.  And by proof I mean real science: hard evidence that biological mechanisms alleged to trigger alleged harms can be shown to cause said  harms.  

Stephen Salzburg, who runs genomic research at the University of Maryland scores a direct hit on the Sun and the quacks whose crap it published in Fighting Pseudoscience

I also wrote a letter to Michael Cross-Barnet who is in charge of op-Eds at the Sun.  Here it is in the likely case they don't print my piece:

The Baltimore Sun published two articles that ignore the scientific evidence about the importance and safety of evidence-based vaccination and then make discredited claims about how to make vaccines and immunization safer.   It should be ashamed of itself for doing so.

Medical science is not a he-said, she-said process.  It is an incremental process of proving and disproving hypothesis based on biological evidence established through experimentation.   When facts don’t fit a theory or an assertion, it’s the latter that is wrong.  

By giving two pseudo-scientists, Margaret Dunkel and Mark Geier, access to it’s press, the Sun has legitimized misleading and dangerous claims about vaccine safety and about the role vaccines play in causing all sorts of childhood disorders, particularly autism.  It perpetuates assertions that contribute to the rise of vaccine preventable diseases such as measles, whooping cough and cervical cancer.  And it has legitimized the idea that wild claims about a product causing autism are “science” even if such claims have never been proven scientifically or have been disproven.   To the Sun, just raising the possibility of danger is enough to merit publication. 

I will not restate the scientific evidence about the significant benefits and incredibly small risks associated with vaccines.  One can read Stephen Salzberg’s editorial in Forbes for a concise discussion. 

http://blogs.forbes.com/stevensalzberg/2011/07/17/the-baltimore-sun-sinks-deep-into-anti-vaccination-quicksand/

The problem is not with the Geiers and Dunkels of the world who peddle their conspiracy theories and lethal prescriptions for assuring vaccine safety.   The problem is with newspapers, new shows and politicians who promote fearmongering.  

Would the Sun allow those ‘scholars’ who deny the Holocaust or claim astronauts never landed on the moon access to its editorial page?  Both types of conspiracy driven twaddle exist in spite of the facts, not because of them.  Yet the Sun, in giving Geier and Dunkel a platform, has given the scientific equivalent of Holocaust denialism legitimacy and renewed strength.  

In so doing, it has shamed itself and empowered quacks to endanger the lives of children.

 

 

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Apres Josh, le deluge

07/19/2011 01:52 AM |

But in a good way.

 

Learn more about it via an excellent analysis in this week’s edition of BiocCentury which begins thus:

 

FDA organizational changes and personnel appointments announced last week could help depoliticize the agency’s decision making, increase its understanding of the way industry operates, and, possibly, improve coordination of drug, biologics and device oversight.

 

The reorganization, which was set in motion by the January departure of Joshua Sharfstein as principal deputy commissioner, also could deepen the bench of managerial talent at an agency that has traditionally valued technical competence over management skills. Radiological Health (CDRH), and the Center for Tobacco Products.

 

In a memo to FDA staff, Hamburg said one of her goals is to “enable the Office of the Commissioner to better support the agency’s core scientific and regulatory functions, and help tie together programs that share regulatory and scientific foundations.”

The complete article can be found here.

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Brooks was here

07/18/2011 05:06 PM |

From Sunday’s Washington Post:

David Brooks: The scary and sloppy case for rationing

By Jennifer Rubin

 

David Brooks of the New York Times likes to fancy himself as a truth-seeker, bringing social and hard sciences to the masses. But in his Friday column on health care and death, he makes some shocking and inaccurate assertions. Given his coziness with the Obama administration one has to wonder if he is test-driving some Obama administration rationalizations for rationing.

 

Brooks is enamored of Dudley Clendinen’s “splendid” essay, as he describes, “The Good Short Life.” Brooks thrills to this definition of a life worth living:

 

Instead of choosing that long, dehumanizing, expensive course, Clendinen has decided to face death as one of life’s “most absorbing thrills and challenges.” He concludes: “When the music stops — when I can’t tie my bow tie, tell a funny story, walk my dog, talk with Whitney, kiss someone special, or tap out lines like this — I’ll know that Life is over. It’s time to be gone.”

Well that “dehumanizing, expensive course” allows millions of Americans who would have died in past years to “kiss someone special.” But is someone confined to a wheelchair (no dog walking) or who needs help dressing not living a life of value? Clendinen, and in turn Brooks, begin down a slippery slope as they decide that, really, is it worth it to keep grandpa around for years if he can’t tie his tie?

 

Brooks then embarks on a flight of misinformation to suggest we’re wasting much of that money. He finds other useful sources:

 

As Daniel Callahan and Sherwin B. Nuland point out in an essay in The New Republic called “The Quagmire,” our health care spending and innovation are not leading us toward a limitless extension of a good life. Callahan, a co-founder of the Hastings Center, the bioethics research institution, and Nuland, a retired clinical professor of surgery at Yale, point out that more than a generation after Richard Nixon declared the “War on Cancer” in 1971, we remain far from a cure. Despite recent gains, there is no cure on the horizon for heart disease or stroke. A panel at the National Institutes of Health recently concluded that little progress had been made toward finding ways to delay Alzheimer’s disease.

Much of this is flat-out wrong or misleading. We may not have “cured” all cancers (Brooks is misinformed if he thinks “cancer” is one disease). But survival rates for many types of cancer have soared, especially for breast, prostate and lung cancer. Five-year survival rates for the range of cancers went from 50.1 percent to 65.9 percent in 2000. Peter Pitts of the Center in the Public Interest told me in a phone interview that for many cancers ”early detection and aggressive treatment” can now extend life or result in effective “cures,” that is long-term remission.

 

A recent report from the Center for Disease and Prevention control explained:

As a result of advances in early detection and treatment, cancer has become a curable disease for some and a chronic illness for others; persons living with a history of cancer are now described as cancer survivors rather than cancer victims . From 1971 to 2001, the number of cancer survivors in the United States increased from 3.0 million to 9.8 million … The number of cancer survivors increased from 9.8 million in 2001 to 11.7 million in 2007. Breast, prostate, and colorectal cancers were the most common types of cancer among survivors, accounting for 51% of diagnoses. As of January 1, 2007, an estimated 64.8% of cancer survivors had lived 5 years after their diagnosis of cancer, and 59.5% of survivors were aged 65 years. Because many cancer survivors live long after diagnosis and the U.S. population is aging, the number of persons living with a history of cancer is expected to continue to increase.

In other words, in just six years the number of cancer survivors increased nearly 20 percent. Interestingly, women and seniors have benefited the most. “Women are more likely to be survivors because cancers among women (e.g., breast or cervical cancer) usually occur at a younger age and can be detected early and treated successfully; in addition, women have a longer life expectancy than men. Among men, a substantial number of cancer survivors had prostate cancer, which is diagnosed more commonly among older men. The large proportion of cancer survivors aged 65 years reflects the increase in cancer risk with age and the fact that more persons with diagnoses of cancer are surviving 5 years.” Put differently, millions more Americans are alive because of progress in cancer research and treatment. I don’t know how one would put a price on the value of lives saved, the contributions those survivors continued to make to society and the children they gave birth to and raised.

 

Brooks likewise bizarrely claims that there is no “cure” for a heart attack. He surely picked the worse example possible. A heart attack used to be a death sentence or a recipe for permanent convalescence. Now with the advent of beta-blockers, new medical technology and surgical innovations survival rates have risen dramatically. (Researchers, for example, found “rates [of in-hospital mortality] decreased among all patients from 1994 to 2006, falling more markedly in women than men. The steepest drop, 52.9%, occurred among women younger than 55. The mortality rate for men in the same age group decreased by 33.3%.)

Alzheimer’s hasn’t been cured, but drugs to slow the rate of deterioration provide building blocks needed for continued progress. For diabetes the results are stunning. (“People diagnosed with diabetes between 1965 and 1980 lived approximately 15 years longer than those diagnosed between 1950 and 1964 (53.4 years vs. 68.8 years).

 

Brooks, Pitts says, makes a fundamental error by setting up “cures” as the metric for assessing medical progress. “It is well-established that innovation in health care comes in incremental steps,” he explains. With increasingly personalized treatment made possible by genetic research the type and timing of drugs can be designed for optimal results. If we don’t spend money to make progress that might, for example, slow the rate of Alzheimer’s we’re not going to invest millions in one fell swoop to locate the “cure.” Pitts says, “If you don’t reward innovation,” by funding the painstaking process of step-by-step research we will cease making progress toward long-term survival rates and cures, a result that is not morally or politically acceptable in this country. He observes, “The average American male’s life expectancy has increased by a decade over the last 50 years, largely to due pharmaceuticals. We innovated our way to that.”

 

Moreover, Brooks ignores diseases such as AIDS, once a death sentence, that is now, albeit by use of expensive drugs, a manageable, chronic disease. Should we not have spent the money? Pitts, noting the dramatic improvements in drugs to treat mental illness, explains that millions of people in the past were never treated at all. “Now people with depression are functioning beautifully.”

 

Brooks says, “Most of us will still suffer from chronic diseases for years near the end of life, and then die slowly.” True, but the alternative is more dead people.

Brooks in the end doesn’t have the nerve to reach the logical conclusion of his arguments. He declares, “Obviously, we are never going to cut off Alzheimer’s patients and leave them out on a hillside. We are never coercively going to give up on the old and ailing. ” Well, then what is the point of his column? If he can’t stomach these outcomes why shouldn’t we continue to spend substantial sums to improve and elongate life?

 

Perhaps the point is to rationalize reductions in health-care dollars spent on the elderly, which by gosh is precisely what the Obama administration is trying to pull off with its Independent Advisory Patient Board. Limiting care, conscience free! After all, do all these old people really enjoy living to 90?

 

By all means we should have the debate over public and private resources. Let’s come up with market solutions that increase competition and reduce cost. Let’s minimize out unnecessary, external costs (e.g. malpractice insurance). And for the record, I am in favor of living wills and allowing those with terminal illnesses to refuse care. But let’s not kid ourselves.

 

Anyone, for example, who has had an elderly parent, a friend with cancer, or an experience with mental illness knows the difference our health-care system, warts and all, has made in the lives of millions and millions of Americans. Who of us would choose to receive only the medical care available 20 years ago? And, from where I sit, I’m not ready to throw in the towel on my loved ones (or anyone else’s) because they can’t walk the dog.

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New thinking on NCDs - CMPI’s first international event

07/15/2011 11:46 AM |

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WSJ's TM Trance

07/14/2011 11:42 AM |

Back in college, I went to a Transcendental Meditation Center, partly out of genuine interest but mainly because a girl I was interested in was going.

 

TM was touted as a technique for achieving inner peace back in the day.   I remember having to bring some flowers and fruit as an “offering” to a photograph of the Maharishi Yogi, the founder of the TM movement.  My mantra instructor (I guess that’s the term) -- a guy in a linen outfit and wire-rimmed glasses – had me kneel in front of the Yogi’s photo while he chanted and threw grains of rice at the picture.   Though I was a relatively non-observant Jew then I found the ceremony silly.  But not as silly as the claim that if I shared my mantra (ha-yam, not to be confused with Chaim or ham) with anyone it would stop working.   And of course my instructor encouraged me to sign up for some lifetime plan of mantra and TM coaching.   All that notwithstanding I did find TM helped distract and relax me.  But so did taking a nap.

 

Which leads me to a recent Wall Street Journal op-ed by director David Lynch and TM executive Norman Rosenthal: A Transcendental Cure for Post-Traumatic Stress - Wall Street Journal

 

Now claiming that TM cures soldiers with PTSD is rather audacious, especially since there is no science to back it up.   A five patient study without a control arm is not science, it is a TM retreat.   What’s more the authors claim that TM “has been found to reduce blood pressure and decrease the risk of heart attacks and strokes—other conditions in which an overactive fight-or-flight response may play a role. In a similar manner, TM may modulate nervous system responses, thereby allowing affected veterans to relax and leave behind the traumas of war.”

 

Let’s set aside the fact that the Maharishi University School of Management or TM adherents have mostly conducted the studies supporting TM’s benefits.   The research about TM as a medical treatment or “cure” is limited and conflicting.  As one review of TM’s cure claims found “ “15 trials comparing relaxation with sham therapy found a non-significant reducing in blood pressure. “   There are only small studies looking at TM’s benefit.  Even those that monitor brain function, never establish whether it’s aspects of the TM regimen or TM itself that may help people cope to PTSD, substance abuse and depression. TM is never compared to sleep for instance.  Indeed, there are other mindfulness meditation or therapy approaches that have been better studied and have shown to be beneficial.    

I believe that TM and other mindfulness modalities do help some people to the extent that they engage in it on a regular basis.  But Dr. Rosenthal is plugging a book extolling the value of TM as a cure.  He also claims TM is superior to all other forms of mindfulness-based therapies.   There is no science to back these claims up.  Rosenthal also happens to promote the kind of long term personalized sessions the TM rice thrower wanted me to sign up for when I was in college.  

 

I don’t get how the WSJ not only allowed the publication of what is essentially free advertising to Dr. Rosenthal but also promoted TM as a cure for PTSD.   Maybe the editors of the editorial page should meditate on that. 

Om.

 

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