Aloha ObamaCare

01/27/2010 11:50 AM |

  Read More & Comment...

Sustainable Healthcare Reform Generates Prosperity

01/26/2010 08:49 PM |

Something that was ignored throughout the long ponderous, hollow debate over healthcare reform is clearly articulated by Jim Pinkerton:

"Healthcare is a great driver for the economy. It's the biggest employer, and medicine--not "health insurance"--is a product that people want to consume, sometimes desperately, ravenously. And not just Americans, but people around the world. So if that's not an economic engine, what is? Isn't that what we want?

But the argument has to be made. And alas, since, 98 percent of the discussion over healthcare has been over controlling costs--that is, shrinking the healthcare sector, perhaps provoking a recession in one of the few vibrant sectors of the economy--then it's little wonder that the country doesn't think of healthcare and medicine as an economic driver.

Serious Medicine Strategy has always been an economic strategy--now is the time to make that case."

seriousmedicinestrategy.blogspot.com/2010/01/democrats-slam-brakes-on-health-care.html
  Read More & Comment...

Extraordinary Measures: Not Just A Movie

01/25/2010 03:19 PM |

The movie  Extraordinary Measures  chronicles the story and struggle of John and Aileen Crowley to find a treatment to save the lives of their two children, both of whom have Pompe Disease, an irreversible and rare disorder that prevents the body from breaking down glycogen (a form of sugar).  Glycogen accumulates around the heart and skeletal muscles leaving children unable to walk or breathe on their own.   I won't provide a movie review here since information about this new release is available everywhere, except to say that Extraordinary Measures is a family movie in two important ways.

First, it is a movie that parents and kids (13 and up in my opinion) can and should see together.  It demonstrates the power of love and the determination parents have, even under the most dire circumstances, to provide children with hope and happiness each moment of their life.   It shows that you can go up against and use the system to make a difference and that the "system" will invest in that risk and reward it.  However the movie shows that making a miracle involves going against the grain, which is why meaningful change or advances take time.  And the movie does the best job of explaining the interesection of capital, science, passion and faith the constitutes the process of biomedical innovation.  A thoughtful, impactful movie about biotech and drug development?  Extraordinary Measures is that and more because it ties the process of innovation to the ultimate purpose and people who benefit.

Second,  the movie itself is the culmination of a process that has created a family of relatives, friends, supporters around the Crowleys.   I was fortunate to have attended a post-movie reception that Geeta Anand, the gifted and gracious author of The Cure held in NYC yesterday.  The group that gathered (including the Crowleys) -- including people involved in the movie,  Geeta's literary agent (and mine) Joelle Delbourgo, former associates, etc.  shared not only in the success of her book but in the spirit the book, the movie and the release of both have created:  A sense that while anything may not be possible,  nothing great happens without passionate intensity, friendship and cooperation and support of those you love.  Such is the recurring source of hope. 

Seeing the movie and reading The Cure will allow you to share in that experience.   Few movies have been made to convey such a message.  You read more about Extraodinary Measures and The Cure at the link below. 

http://www.thecurebook.com/


  Read More & Comment...

Boston Tea Party

01/22/2010 08:39 PM |

So, what's next?

Listen to this new podcast, courtesy of the Burrill Report.
  Read More & Comment...

Prospective Healthcare: Real Reform

01/21/2010 08:23 PM |

Let the big government types defend comparative effectiveness generated decisions that exclude and undermine the health of minorities... We are supporting a different, more compassionate and equitable approach...

Prospective Health Consortium: Development and Assessment of Predictive Tools in Disease Prevention and Wellness Promotion

A core mission of the Center for Medicine in the Public Interest is to spur approaches to the problem of chronic illness in medically underserved communities that are not “one size fits all” or based on studies that ignore clinical, behavioral and genetic variations in disease progression and treatment response.    Our efforts build on the leadership and pioneering work of the National Minority Quality Forum in using data to map and identify these variations.

We are proud to support the launch of a consortium that would develop and evaluate a tool for health risk assessment and treatment selection based on predictive information relatively unique to that individual that can be used to reduce disease and promote wellness in medically underserved communities.

The idea of a consortium is based on discussions held in May at Brookings Institution at a meeting entitled “A Critical Path for Personalized Medicine”.   That meeting -- chaired by Dr. Mark McClellan and Sir Michael Rawlins -- discussed ways to demonstrate how predictive or personalized medicine could be used to improve the quality of care and health outcomes.  In particular, Ralph Snyderman MD, Chancellor Emeritus of Duke University Medical Center and CEO of Proventys, called for an evaluation of an tools  bringing together the latest technologies to predict events and enable intervention before damage occurs.    Combining personalized risk prediction and strategic health-care planning will facilitate what Snyderman calls ‘prospective health care’.

Stay tuned.
  Read More & Comment...

More Brown Breakdown...

01/21/2010 12:24 PM |

Maggie Mahar -- another liberal health care "expert" -- on why a bipartisan healthcare effort should not be pursued:

"Conservatives blame the sick for being sick, and they fault the poor for being poor.  Health care reform must be compassionate, and it must be equitable."

Read more here.
  Read More & Comment...

Krauthammer Clairvoyance

01/20/2010 11:04 PM |

Not Waterloo, Just a Tactical Retreat

  Read More & Comment...

EC ITP? Not ASAP.

01/20/2010 10:12 AM |

ITP? Not ASAP.

John Dalli, Commissioner-designate for Health and Consumer Policy (SANCO) has pledged new initiatives to break the deadlock on Commission proposals to liberalize controls on the provision of prescription drug information to patients. And we all know how fast Brussels operates.

As Pharma Times reports:

- In the new European Commission, the move of responsibility for the pharmaceutical industry from the Directorate General (DG) for Industry and Enterprise to DG Health and Consumer Safety (SANCO) has been welcomed by consumer and patient groups. One of the reasons holding back progress on the information directive has been concern that it was drafted by DG Enterprise and Industry - whose responsibilities include regulatory and industrial policy for the pharmaceutical industry – rather than DG Sanco, which is concerned with consumers and public health.

While countries including Denmark, Sweden and the UK support the Commission’s proposals, a number of others – including France, Germany and Spain - oppose any move to liberalize controls on the provision of prescription drug information, for reasons including the fact that the draft does not distinguish sufficiently between “information” and “advertising.” They also believe that the industry should have no role in the provision of information to patients and that the proposals would drive up health costs by increasing demand and because of the need to set up monitoring mechanisms.

  Read More & Comment...

What can Brown do for you?

01/20/2010 12:55 AM |

Don’t think about Scott Brown as the 41st vote against healthcare reform.  Consider him the 1st vote towards a new, more thoughtful approach.  A more honest approach.  An approach that doesn’t require Uncle Sam to become Uncle Sam, MD.  An approach that doesn’t tax the middle class, gut Medicare and abolish Medicare Advantage.  An approach that doesn’t put the power of government reimbursement decisions into the hands of a politically appointed panel without any oversight by elected officials.  Perhaps even an approach that takes into consideration reform via a partnership between government and the private sector (a la Part D). And maybe even an approach that considers allowing Americans to buy insurance policies across state lines so that we can all benefit from a significant economy of scale and radically lower premiums. A study by University of Minnesota shows that Congress could boost by more than 12 million the number of people who have health insurance without spending taxpayer dollars.  The change required is to allow people to buy health insurance across state lines, so they can shop for less expensive policies. For example, a typical health-insurance policy in heavily regulated New York costs more than three times as much as in less regulated Iowa ($388 a month versus $98 a month for the same coverage).

But – for starters – how about an approach that’s open and transparent.

  Read More & Comment...

Unassisted Suicide...

01/19/2010 10:57 PM |

What exactly about the Scott Brown victory does Ezra (the mind of moment) Klein not get?

"There's nothing about Scott Brown's victory that needs to derail health-care reform in particular, or the rest of Obama's 2010 agenda in general. But if Democrats decide to cower and hide, they can end Obama's presidency on Brown's behalf.

That said, I really wonder what the Democratic Caucus thinks will happen if they let health-care reform slip away and walk into 2010 having wasted a year of the country's time amidst a terrible recession. It won't be pretty, I imagine. If health-care reform passes, the two sides can argue over whether it was a success. If it fails, there's no argument."

voices.washingtonpost.com/ezra-klein/

I have heard that petulant "it's all about me" tone before..  It's the gift that keeps on giving to those of us seeking sensible health care reform consistent with the need to sustain job creating biomedical innovation...  I hope Klein and others keep pushing Dems to ignore the outrage of the voters...  It will produce more Scott Browns.


  Read More & Comment...

Follow On About Face

01/19/2010 10:14 AM |

Michael McCaughan's blog in RPM First Take reveals that the operational definition of a follow on biologic is simply someone else's product that we want to copy...
therpmreport.com/Free/4e767e88-bc84-40e1-871a-4025e307e300.aspx

Saturday, January 16 2010

Teva’s Hedge on Complex Generics
By Michael McCaughan

Generic applications for Lovenox and Copaxone have become bellwethers for the emerging follow-on biologics pathway in the US. Teva is on both sides.
 

Who says you can’t have your cake and eat it too?

When it comes to the follow-on biologics space, there are plenty of companies who are likely to try. As we’ve written before, the looming new pathway for abbreviated approval of biosimilars opens up opportunities for lots of companies—brand, generic and biotech—to consider whether to play as a true “follow-on” supplier, focus on improved “biobetters,” or do both.

But no one is in quite the same position as Teva. Not only is the company doing that analysis for the longer term biosimilar opportunity (you can read more about its latest thinking in “The Pink Sheet” DAILY here), but it is on both sides of the issue for two near-term decisions on complex molecules that are regulated as drugs.

Teva is one of three companies (along with Momenta and Amphastar) with pending applications to market a generic version of Sanofi Aventis’ enoxaparin (Lovenox). In that case, Teva wants FDA to agree that, while enoxaparin is a relatively complex molecule, it is not too complex to allow for a fully substitutable generic approval.

On the other hand, Teva’s largest and most important product is the branded multiple sclerosis therapy glatiramer (Copaxone). There are two would-be generics pending, Momenta’s and Mylan's.

In that case, Teva is arguing that Copaxone is far too complex a molecule to be copied closely enough to allow for substitutability—and is even suggesting that other manufacturers may have trouble even getting a non-interchangeable product approved without full clinical studies.

Teva, of course, is aware that this may sound like trying to have your cake and eat it too. But they don’t see a contradiction. Here is how Teva CEO Bill Marth put it during the Goldman Sachs CEO “unplugged” conference Jan. 6:

“When you think about Copaxone, many people try to equate it to Lovenox. It is
much, much different—vastly more complex—than Lovenox will ever be. We have not characterized it. We don't believe it can be fully characterized.

If one cannot fully characterize Copaxone, I'm not sure how you get it approved without a clinical study since the method of action is not well understood and exactly what the active sequence is. And, by the way, we think there are multiple methods of action, and potentially multiple reasons for that. We think that it is virtually impossible to prove your efficacy without a clinical study. So it really falls into more of that sweet spot of the biologics....

I think it is much different with Lovenox than it is with Copaxone because with Lovenox, the active sequence has been identified by us. It hs been identified by the
innovators. It has been identified of course by Momenta and Amphistar.

So it is defined. It is a sugar. When you look at those sugars and you look at the active sequence, then what you really have to do is understand what is the other stuff or junk that is within your protein or sugar, and there make sure that you don't have improper immunogenicity. They have asked us for immunogenicity testing. We have done that. And it seems to be acceptable so far.”

In other words, Teva thinks it can kept its Copaxone cake and take a huge slice of Sanofi-Aventis’ Lovenox cake too.

But there are other outcomes. From Teva’s perspective, an outright rejection of substitutable Lovenox wouldn’t be so bad, since it would underscore the company’s position that substitutable Copaxone is a pipe dream.

And while Teva would dearly love to tap into the $2 billion Lovenox market, at best it will only get a percentage of a big generic opportunity. With Copaxone, Teva hopes to maintain its own multi-billion dollar brand in something like perpetuity.

How important is that to the company? During its January 7 investor day, Teva’s bullish forecasts for growth for 2015 included what the company called a “conservative” forecast for Copaxone, with sales peaking at $3 billion and then eroding over time to $2 billion in 2015. But the erosion, in Teva’s view, will come only in the face of competition from other MS agents (including Teva’s own oral product)—not from any substitutable generic competition.

So call it a hedge: if generic Lovenox is rejected, Teva’s Copaxone franchise is more secure. If Copaxone can’t be protected, at least Teva will have some generic enoxaparin revenues to fill in the hole.

There’s really only one scenario where Teva loses. If one of the other applicants (Momenta being the most likely candidate) actually has superior technology, it could potentially get approval for generic Lovenox and generic Copaxone, while no one else can.

For Teva, that would be more like a pie in the face.
  Read More & Comment...

What does a Scottish health official wear under his kilt?

01/19/2010 08:09 AM |

This blessed plot, this earth, this realm ... this reimbursement decision.

From the UL newspaper, The Telegraph:

The Government’s drugs rationing body, the National Institute for Health and Clinical Excellence (Nice), has provisionally said that it does not intend to recommend the use of the drug, called Tocilizumab, or Roactemra.Nice claims that the £9,000 a year drug, for rheumatoid arthritis, has not proved that it is cost effective.But patients in Scotland are to receive the treatment after it was recommended by the body which regulates drugs on the Scottish NHS, the Scottish Medicines Consortium (SMC).

The move will reopen accusations of medical ‘apartheid’ within Britain.

It follows an outcry after patients in Scotland were given access to expensive cancer drugs denied on the NHS in England and Wales.

Roactemra has been described as a “life changing” drug because it can be taken after other medications have failed, a common problem in the treatment of rheumatoid arthritis.Patients groups last night said that denying the medication to tens of thousands of patients with the crippling condition in one part of the country was “cruel”.

Ailsa Bosworth, chief executive of the National Rheumatoid Arthritis Society (NRAS), said: “I have heard patients stories that would make you weep.

“People are virtually suicidal because they have nowhere else to go and yet they know that there are other drugs out there that they could have access to but cannot because of Nice.” She added that it was “ludicrous” that the drug would be available in Scotland “and yet two miles on over the border you can’t get it.”

The drug - the first new arthritis treatment for a decade - is already used in most other European countries, including France and Germany.

It offers another option for patients for whom other treatments have failed or no longer work and is used in combination with a standard anti-inflammatory drug, called methotrexate.Currently many rheumatoid arthritis patients receive methotrexate as a first-line treatment to ease their symptoms. In later years they are offered another class of drugs, called anti-TNFs, together with methotrexate, but even combined the effects of the drugs can wear off.

In combination Roactemra has been found to improve the rates of remission of the illness sixfold in comparison with just methotrexate alone.

The SMC - set up in the aftermath of devolution to make decisions about drugs north of the border - has agreed that the drug can be used for patients suffering from moderate to severe forms of the disease for whom other medications no longer work.

Prof John Isaacs, from Newcastle University, said: “This is fantastic news for people in Scotland who suffer from this disabling, lifelong disease.

“However, it also highlights the disparities in accessing treatments between Scotland and the rest of the UK. “Because Roactemra works in a completely different way to the existing drugs it is likely to be effective in some patients where the other drugs don’t work or have stopped working, providing an extremely important option for these individuals.”

Neil Betteridge, chief executive of Arthritis Care and vice president of the European League against Rheumatism (EULAR), said: "There are a number of treatments for RA currently available but they simply don't work for everyone. "There are people who are most severely affected by this debilitating condition – living in intense pain, unable to work, often struggling even to walk – who have been failed by existing treatments, and it's for them that tocilizumab could provide real hope.” He called on Nice to follow the lead of the SMC and approve the drug for use in England and Wales.

Up to 37,000 patients across Britain would be eligible for the drug.

But local health care trusts do not have to pay for drugs which have not been approved by Nice.

In December Nice took the unusual step of challenging Roche, the drug’s manufacturers, to provide more evidence of that the drug was cost effective.

A final Nice appraisal of the drug is expected later this year. Around 646,000 people in Britain are though to suffer form rheumatoid arthritis, in which their own immune systems start to attack their joints. The condition can be extremely painful and debilitating. Around half of sufferers are too disabled to work within a decade of the disease taking hold. The direct and indirect costs of the disease are estimated at £3.8 billion to £4.75 billion a year in the UK alone.

Nice has previously come under fire for refusing drugs which have been approved by the SMC.

Herceptin, a £21,000-a-year drug for breast cancer, was initially turned down by Nice but available in Scotland, which has its own health budget.

A climb-down, ordered by Patricia Hewitt, the then health secretary, allowed the drug in England and Wales. Patients in Scotland also had access to Tarceva, a lung cancer treatment, which costs about £1,700 a month, two years before the rest of the country. Nice also provoked outcry by turning down Lucentis, a £20,000-a-year treatment available in Scotland for wet age-related macular degeneration, one of the most common causes of blindness, although it later also reversed that decision.

  Read More & Comment...

Why Health Care "Reform" Is Imploding

01/18/2010 11:16 PM |

This is prompted by the  latest posting of one of the "leading" liberal health care "experts", Ezra Klein.  Expert not because he has practiced medicine (he has not), or published anything close to an original piece of research on health care delivery or economics (nothing there either) or worked in health care (no)..  Rather he is an expert by virtue of his position as a blogger he has helped shape liberal opinion and policy that went into the current malformation of a proposal that is causing Democrats to go down in flames. 

Thanks Ezra. 

Part of Klein's contribution to Democrat demise is perpetuating the belief that all people cared about was the total cost of the bill, which he once again argues, is really not so big when you consider say, what it would cost to build the entire fleet of Starships for the Federation.  Well, okay, maybe not that much but here is Klein at his misleading and misinformed best:

"by the standards of the health-care system, it's not that big at all. It goes two-thirds of the way on covering the uninsured. It makes a courageous, but insufficient, start on cost control. This is the beginning, not the end, of reform.

Let's begin by breaking down the numbers. The $900 billion price tag is repeated with the regularity of a rooster's crow. That's a shame, as the number is, somewhat impressively, misleading in both directions.

On the one hand, that $900 billion is stretched over 10 years. But people don't think in 10-year increments. They don't pay taxes once a decade. Put more simply, the bill will cost an average of $90 billion a year.

But that number is meaningless without context. Ninety billion is a lot more than you probably paid for, say, your house. But is it a lot of money in the context of national health-care spending? Not really. In 2008, we spent $2.3 trillion on health care. Ninety billion is about 4 percent of that. In other words, a drop in the bucket."

voices.washingtonpost.com/ezra-klein/

I know that The Economist named Klein "minds of the moment" (there is a joke in that but I'll resist) but isn't there a difference between what we choose to spend on health care and what government will force us to spend?  Put another way,  that $90 billion a year drags along the rest of the spending by forcing people into Medicaid, choose only government approved plans with government approved benefits, paying taxes (not if you are a union member) for higher cost plans that you have no say in shaping which in turn will force you to spend money for premiums, deductibles, etc you may not want to pay or might have to if the services you really want are not covered.  

Here is what Michael Cannon, who is a lot smarter and has done a lot more original research than Klein has said about this $90 billion scam:

"The CBO explains it will not count mandatory premiums as federal revenues if the individual mandate leaves consumers with what the CBO considers a "sufficient" or "meaningful" or "substantial" degree of choice among health plans. That rule is both amorphous and arbitrary. (For example, it presumes that the freedom not to purchase health insurance — which an individual mandate would eliminate — is not "meaningful." Millions of Americans would disagree.) More important, evading that rule doesn't make an individual mandate any less compulsory, or any less costly. It just hides those costs by pushing them off-budget.

In Massachusetts, which has enacted what is essentially the Democrats' health plan, mandatory premiums account for about 60 percent of overall costs, according to the Massachusetts Taxpayers Foundation. On-budget government spending is just 40 percent. By my count, mandatory premiums accounted for a similar share of the Clinton health plan's projected cost.

So while the CBO estimates that the coverage expansions in the House Democrats' legislation would trigger about $1 trillion of new federal spending over ten years, the actual cost of those coverage expansions is more like $2.5 trillion."

www.cato.org/pub_display.php

Actually, that $2.5 trillion over 5 years or so.  Which is $5 trillion over ten years, both which equal $500 billion a year or so.  Not to mean the loss of freedom and the dumb central planning involved to make this all happen.

Regular people -- amateurs -- get this.  Experts like Klein who talk to other experts in DC do not.  Their detachment from what we, the little people, can feel and perceive, formed by either arrogance or Economist-induced self-regard is stunning.  And contributory to the growing sense across this nation that Democrats actually believe that health care is a system established to serve Washington interests instead of a series of human relationships that should sustain our lives and well-being. 

 

  Read More & Comment...

Pharma Giveth, Congress Taketh

01/15/2010 06:39 PM |

  Read More & Comment...

The Desperate Hours

01/15/2010 04:07 PM |

Now that the unions are getting a tax break on their health plans the rest of us will not get, the President is dialing for dollars to cover the cost. He is actually picking up the phone and calling members of Congress to shorten the amount of time before generic companies can try to produce their own version of biotech drugs from 12 years to  8 years and allow a short cut around the FDA’s efforts to come up with an approach that insures that medicines meant to heal don’t maim.

For every 10 percent shaved off the life of patent protection, biotech investment declines by 10 percent. Over the next 15 years, the Obama deal would cut biotech investment, heavily concentrated in the Bay State, by 33 percent.  Every biotech dollar invested has a ripple effect in terms of jobs, spending and investment in related businesses. Cut biotech spending by a third and the impact on economic growth will be magnified in reverse.

Then there is the human element. For cancer an increase in the number of biotech drugs iis associated with an increase in both the one-year and the five-year survival rate for all forms of the illness. For orphan disease, it has meant longer lives for people with lupus, cystic fibrosis, MS, Gaucher’s disease, HIV, etc. Less investment, fewer drugs, more deaths sooner. That’s one way to keep costs down and pay for the union tax break.  

Finally, Obama wants to save a few million by cutting corners on drug safety. Current proposals require genetic companies to demonstrate patient safety by requiring appropriate and stringent clinical trials and testing. This is necessary because biologic drugs are created from living organisms such as proteins and carbohydrates, and are not as simple to replicate as traditional drugs like aspirin and antihistamines. Even changing the size of the molecule of the same protein can turn of biosimilar from avatar of health into an avalanche of deadly side effects. But Obama wants safety to meet budgetary, not scientific standards.
  Read More & Comment...

Ted Med

01/15/2010 01:33 PM |

Having been rebuffed in his own committee on follow-on biologics, Mr. Waxman is trying to roll the President. That’s petty politics and bad public health policy.

Consider what Senator Ted Kennedy had to say on the subject of follow-on biologics in March of 2007:

We are in a remarkable period of discovery in the life sciences. Unprecedented advances are taking place, and patients have already begun to see the benefits of this new era through new wonder drugs that can make the difference between life and death for patients afflicted with serious illnesses.

These miracle medicines, called biologics, are complex molecules whose healing power has been brought to patients by dynamic biotechnology companies. Such drugs were once a rarity in the medical arsenal, but each day seems to bring new hope from new breakthrough biologics.

Now Congress must consider whether to authorize FDA to accept applications for follow-on versions of these path breaking medicines.

The stakes riding on the answer to this question are enormous, both for patients and for our economy, and the interest among our committee colleagues in this question is intense. One of our colleagues, Senator Clinton, has a proposal to allow FDA to approve follow-on biologics. I look forward to hearing her views on this question, and to receiving the testimony of the legislation’s co-sponsor, Senator Schumer.

Our committee should be guided by three basic principles.

First, we must be led by science. Acceptable legislation on follow-on biologics must not pre-judge science, but should enable the FDA to make the best decisions based on the most complete science reasonably available.

Second, protecting patient safety is essential. Congress must make certain that any drug given to patients – whether a conventional drug, an innovative biologic, or a follow-on product – is safe and effective.

Third, innovation must be valued and promoted. Just as it is essential to help patients afford the medicines of today, so too it is vital to provide incentives for the innovations that will bring the medical miracles of tomorrow.

Mr. Waxman should show some respect. And so should the President.

  Read More & Comment...

Phase 0.1

01/14/2010 08:57 AM |

FDA's Endocrine and Metabolic Drugs Advisory Committee unanimously agreed that a retrospective analysis of 23 case studies provided substantial evidence to support approval of Orphan Europe's Carbaglu, but recommended collection of long-term safety data as the drug could be used for years as maintenance therapy.

Did somebody say precedent?

  Read More & Comment...

The Tonight Show ... with Peggy Hamburg

01/13/2010 11:36 AM |

The FDA is launching a new Web site explaining its operations to consumers as part of a broader effort to rebrand itself as a more accessible agency. (For more information, go to www.fda.gov.)

The Web site, “FDA Basics,” features short videos about the agency as well as conversations with FDA leaders.
Must see TV.  Maybe it can hosted by Conan O’Brien.

The website is a good idea.  A positive step in the right direction.  If “the people” don’t know what the FDA does, it’s impossible to build a broader base of support. In that respect, it’s more than just a good idea – it’s a crucial one.
  Read More & Comment...

FDA Commish New Year's List

01/13/2010 09:22 AM |

A lot on the plate in the coming year...

From:   A Message from the Commissioner 
Sent:   Thursday, January 07, 2010 7:12 AM
To:     FDA-Wide
Subject:        A New Year

The beginning of a new year is a good time to take stock of what we have done over the past 12 months and where the next year will take us. Upon my arrival at FDA in May, I found myself distinctly impressed by this agency’s enormous impact, both as a regulator of so much of the American economy and as an organization upon which so many depend for the safe use of a wide array of products critical to their daily lives.

I recently observed to Secretary Sebelius that I have found FDA’s employees to be a wonderfully talented and dedicated group that I believed, if adequately resourced and supported, could solve virtually any problem that comes your way.  The Center directors, ORA, and Commissioner’s staff offices recently shared with me their accomplishments for 2009, and it’s a remarkably impressive list of product reviews, inspections, enforcement activities, rulemaking, outreach to the public and those we regulate, reaction to crises and so many other activities that enable the agency to be an effective public health protector.

All of that was done as a cascade of new challenges were thrust upon us – H1N1 influenza, implementation of the new FDAAA and animal drug legislation, new food contamination and drug registration systems, and an entirely new Center to regulate tobacco for the first time in the nation’s history.  I should also note that lurking out there are new requirements in the health care bill moving through Congress (e.g., “follow on” biologics and restaurant menu labeling).

Of course, we launched a number of new things ourselves – new foreign offices, a safe use initiative for drugs, a new food labeling effort, a reexamination of the process for reviewing medical device 510(k)s, new procedures for emergency response, a new policy with regard to antimicrobial resistance for animal drugs, and a rejuvenation and integration of the food safety program, to name just a few.  New facilities also came on line in 2009, most notably the medical device office and laboratory complex at White Oak and the Bio-Imaging facility at NCTR. 

For my part, I am proud of the new emphasis that Josh Sharfstein and I have placed on ensuring FDA’s reputation as a public health agency, as an organization more transparent to the outside world, and as a regulator intent on its scientific integrity and on enforcing the safety standards we have been charged with implementing. 

All of the things that I have mentioned above will, of course, be a priority for 2010 as well.  But I also intend to dedicate myself to giving you more and better tools to do your jobs.  This will include seeking Administration support to improve our regulatory science, ensuring passage of the food safety legislation now before Congress, seeking new authorities to better regulate imports, and identifying changes in our medical device statute that are needed to ensure that program has 21^st century capabilities.  I also intend to urge the Administration and Congress to complete the long-awaited consolidation of our headquarters facilities at White Oak and College Park.  And, of course, getting you the resources and staffing necessary to be successful will be a constant imperative, despite the demands to reduce Federal spending.

I have gone on long enough, even though I have barely touched upon the hundreds of discreet activities that FDA staff carry out every day. I will close simply by saying that I consider myself privileged to serve as your Commissioner in this great enterprise we are about.  I pledge to you the same dedication that you have shown to the American people.  I am proud to be associated with you and with the Food and Drug Administration, and that I hope the new year is as filled with accomplishment and progress as the old.

With all best wishes for a happy, healthy and productive new year.

Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs

 

  Read More & Comment...

Phase 0

01/12/2010 08:59 AM |

The Pink Sheet reports on a very urgent ethical question:

FDA's Endocrinologic and Metabolic Drugs Advisory Committee on Jan. 13 will consider whether Orphan Europe can use positive results from patient experience with carglumic acid to demonstrate efficacy of the firm's Carbaglu for treating hyperammonemia associated with NAGS deficiency, given the difficulty of conducting a clinical study in the orphan setting.

A controlled clinical trial in this target patient population "cannot be conducted because the disease has an extremely low incidence, it is life-threatening, severely symptomatic, and hyperammonemic decompensation leads to quick deleterious neurological/psychomotor consequences," Orphan Europe maintains in briefing material for the committee meeting.

A deficiency of N-acetyl-glutamate synthase is one of the rarest of the urea cycle disorders, which have an overall occurrence rate of approximately one per 30,000 live births. It results in hyperammonemia - high blood ammonia levels - that can lead to death or neurological impairment.

Instead of conducting a trial, the sponsor submitted a retrospective review of the effect of carglumic acid on both short-term and long-term plasma ammonia levels in 23 patients diagnosed with NAGS deficiency. Carbaglu is the pharmaceutical grade of carglumic acid, which has been used as a chemical grade product by clinicians. Also submitted in support of the NDA were interim data for three patients in an open-label, Phase II clinical trial of three days duration.

  Read More & Comment...