FDA's Coalition of the Willing

06/21/2011 08:07 AM |

Back in February, Peggy Hamburg made some interesting comments at the Council on Foreign Relations:

* The new reality of food and drug regulation is that it’s global. In fact, it should be a topic for conversation at the next meeting of the G20.

* The recent crises in both food and drug safety will only repeat themselves unless regulatory agencies from around the world work in closer and more regular partnership.

* There is a responsibility on the part of the FDA and other more developed regulatory agencies around the world. (MHRA certainly comes to mind.) to help build “regulatory capacity” for those nation’s that want and need assistance. (Nigeria comes to mind.

* Part of a closer working relationship means a more regular and robust sharing of global intelligence on issues of counterfeiting. (And, for that matter – many other things too.)

* And lastly, “We can’t inspect our way out of this problem."

All good things – progressive things -- but, short of a regulatory Marshall Plan, things that will have to rely (at least initially) on personal relationships between senior officials at various regulatory agencies and a focus on what’s best for global public health writ large is convergent with what’s best for any given nation.

It’s not as easy as it sounds.

Beyond the generally difficult nature of international regulatory harmonization (yes friends, it is a lot harder than it looks) is the profoundly difficult issue of domestic economic interests and trade.

For example, every nation (from the United States to Nigeria) is concerned about counterfeit drugs and unsafe food coming across its borders.  That’s obvious. But are all nations equally concerned about controlling the export of counterfeit, unsafe, and substandard products to other nations?  That’s trade.

Principles aren’t principles, as my father used to say, until they hurt.

Today the FDA released a new report designed to monitor the rising tide of goods from abroad. It includes planned partnerships with counterpart regulators in other countries and better data-sharing between those regulators.

 

According to the report, imports of FDA-regulated products are growing at an estimated 15% annual rate.

 

Peggy Hamburg said it will likely take many years to implement its new approach, which also includes building up its IT capabilities to try to identify risks in the food and drug supplies before they pose serious harm and using public and private third parties to conduct audits and inspections on the agency’s behalf.

 

Hamburg said that the new approach “will require some up-front investments in terms of dollar resources, human resources and time,” she expects better sharing of data and strategies between regulators in different countries will eventually mean using fewer resources to achieve the same end.

 

“All of us are confronting the same set of challenges,” said John Taylor, acting principal deputy commissioner of food and drugs.

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The Audacity of AFSSAPS

06/20/2011 10:30 AM |

Not all business travel is arduous.  I’ve just returned from the 11^th annual France-USA Conference on pharmaceuticals and health policy.  The meeting was in Paris.  Sometimes being a policy wonk has its perks.

My role was to debate NYU professor, Dr. Victor Rodwin on “The Current State of Obama’s Health Reforms.” It was fun and feisty – especially the parts where we both spoke in French. (Shout out to my high school French teacher, Linda Diaz!)

Dr. Rodwin regularly cited Commonwealth Fund research,  ‘Nuff said. I focused on the legality of the individual mandate, the growing “idealism gap” of those who want to improve our healthcare system – but not via the approaches of the Affordable Care Act, the increasing (and increasingly contradictory) roles of comparative effectiveness and patient-centered outcomes research, IPAB (and the creeping fear of Uncle Sam, MD), the dangerous vagaries of academic detailing, and the war against medical innovation (personified by President Obama’s call for a roll-back of biologics patent life from 12 to 7) and the issue of data exclusivity.

My summation – “L’audace, l’audace. Toujours l’audace.”

Another fascinating presentation was offered by Dominique Maraninchi, the relatively new (since the end of February 2011) General Manager of AFSSAPS. That means he’s the French equivalent of the Commissioner of the FDA.

France is going through it’s own “Vioxx Moment,” having recently rescinded the market license for Mediator (a diabetes drug that was widely used off label as a weight loss treatment). “L’Affaire Mediator” is a big deal and has created a public outcry for change and reform.  Sound familiar?  (FYI – for more detailed information on the Mediator issue, see here.)

Much of what Dr. Maraninchi had to say will sound very familiar to FDA watchers:

AFSSAPS must:

·      Reestablish trust

·      Enhance pharmacovigilance and do a better job analyzing data and drawing conclusions

·      Be more transparent (a truly radical concept for the French bureaucracy)

·      Be more accountable

·      Offer more information and explanations about its decisions via “bon usage” (safe use)

These might sound rather derivative to a US audience, they are not. While they certainly do share much of what the FDA is trying to accomplish, (1) the French system is very different and these initiatives will require different strategies and tactics, (2) as such, their efforts will deliver different results, and (3) there will be much for the Wizards of White Oak to learn from the AFSSAPS experience.

Another echo of FDA came from Yves Burr (a member of France's National Assembly and chair of that body’s working group on medicines and health products), who asked, “Do we have the courage to put the patient in a role where they can be heard?”

Dr. Maraninchi ended his presentation with a statement of principle that should be widely discussed inside the FDA:

“It is better to make a decision than to have no decision at all.”

Perhaps he should testify at the upcoming Congressional hearings on PDUFA?

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What's Wrong With Paying KOLs?

06/17/2011 05:03 PM |

Ed Silverman at pharmalot ran a three part series of blogs on physician compensation for being key opinion leaders, consulting with pharma, etc that is very balanced and factual. 

Paying doctor as consultants or to speak about products/treatments is problematic when the physician in question is not an expert or not disclosing the payment.   And it's no more problematic than a doctor who is paid to write or speak against a product or even does so because of an ideological or cultural belief that any new drug is inherently useless or overhyped.  In fact, the latter case is even more troubling since it is shaped not by science or clinical practice but an agenda.

Second, having attended sessions where KOL have spoken (and disclosed support) it is obvious that doctors are not drinking kool-aid.  They are curious, skeptical, interested in getting additional information about the risks and benefits of drugs and devices.  

Finally, the ongoing effort to ban pharma payments to doctors is part of a larger effort to substitute information provided by or supported by industry by groups that want to impose their views and control all medical information. 

Diffusion of innovation is evil to the opponents of pharma supported KOL.  So is any research or clinical finding funded by drug companies.  This is a purist and paranoid view of science. 

Meanwhile, pharmalot is hosting a webinar on a subject every drug company and doctor should be  paying attention to.  I encourage everyone to tune in.

How Can Pharma Use Social Media for Real-Time Intelligence and Research?

Tuesday, June 21, 2011
2:00 p.m. ET | 11:00 a.m. PT
Webcast Duration: 1 hour
Registration: $59
Register Now - Click Here!

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Over here first. Over there second.

06/17/2011 12:58 PM |

As reported from the kind folks over at lilypad ...

FDA Data on Cancer Drug Approvals

June 16th, 2011 | By: Greg Kueterman

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Swiss (en)Guard

06/17/2011 12:22 PM |

Research-based pharmaceutical industry launches collaborative framework to tackle NCDs
 
Geneva, New York, 16 June 2011 – The research-based pharmaceutical industry launches today a Framework for Action to respond to concerns that cardiovascular disease, cancer, diabetes and chronic respiratory diseases are posing mounting threats to public health as well as public and private finances worldwide. The industry’s ten-point Framework scopes out specific areas of action to tackle non-communicable diseases (NCDs) including innovation, access and affordability, prevention and health education. It also underscores the essential role of partnerships and dialogue in implementing the Framework. The NCD Framework for Action brings together the global research-based pharmaceutical industry in support of the World Health Organization’s (WHO) Action Plan on NCDs and the Moscow Ministerial Declaration. It will be presented today at the United Nations (UN) Civil Society Hearings in New York, held to prepare for the UN High Level Meeting on NCDs to take place in September, which aims to secure an action- oriented UN response to the rise of NCDs. The aim is that the NCD Framework for Action will provide a sound basis for the research-based pharmaceutical industry to partner with the people on the ground, governments and the WHO to find ways to address prevention, care and treatment for NCDs in the developing world.

NCDs are the leading cause of death and disease worldwide, killing more than 36 million people in 2008, with nearly 80% of these deaths occuring in low- and middle-income countries. NCD deaths are projected to increase by 15% globally between 2010 and 2020. In part, this is due to progress made in combating infectious diseases through economic growth, development, and better treatment options; but it is also largely linked to lifestyle choices. For most infectious diseases, rapid access to diagnosis and treatment is an imperative for patient survival. But for NCDs the “lifecycle” is different, and there may be many actions that can take place, such as prevention, before medicines or other treatments need to be prescribed.

The Framework for Action on NCDs focuses on the areas where the research-based pharmaceutical industry can make the most significant difference, such as innovation, access and affordability, but also prevention and health education. Eduardo Pisani, IFPMA Director General explains: “The framework is just the beginning; our vision is to work with others to identify what can be done in practice to help poor people to access the care and treatment they need. Together we want to find ideas for concrete actions to put on the table in the aftermath of the UN NCD Summit in September. The Framework is our roadmap for this work.” The IFPMA also pledges to report regularly on industry’s progress and share updates with stakeholders, such as the WHO and other interested agencies.

The Framework confirms the industry’s crucial role in continued investment in R&D programmes dedicated to the development of innovative medicines for the prevention and treatment of NCDs to enhance the lives of patients. There are currently over 1,500 medicines in the pipeline for major NCDs. The industry will also endeavour to address its innovation towards the specific needs of developing world populations and settings. To this end, in addition to the Framework, the pharmaceutical industry will launch a programme of research that will improve its understanding of the specific needs of developing world populations.

Actions on access and affordability of NCD treatments include promoting the right policy, regulatory and supply chain environments that secure optimal quality of care for patients and enable companies individually to implement commercially sustainable access and pricing strategies for the supply of NCD vaccines and medicines to the developing world.

It is estimated that half of deaths caused each year by NCDs are preventable[1], and lifestyle choices that often accompany improvements in living standards, such as unhealthy diet and tobacco use, are part of the explanation. The Framework for Action highlights the importance of prevention so that individuals can make informed lifestyle choices. Industry is also working with the World Health Professions Alliance to develop an NCD scorecard that will be shared with over 26 million health care professionals in more than 130 countries to help encourage patients to identify and prevent risky behaviors. Reducing disability or deaths through increased investment in prevention programs will contribute to higher economic growth and allow limited resources to be focused efficiently on patients most in need.

Eduardo Pisani, IFPMA Director General explains: “This NCD Framework for Action represents a paradigm shift for the research-based pharmaceutical industry. It puts our industry’s collective global health responsibilities firmly at the forefront of how we see our role in the global health community. Let’s be clear: it is not about altruism, but rather about revolutionising our relationship to others. Times are tough for governments, business and patients. In order to tackle the rise of NCDs, and stay the course, we need to look at sustainable new approaches to global health which have prevention at its core.”

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Jerry Avorn and Co: Harvard's Kevorkian Corp

06/16/2011 04:32 PM |

Jerry Avorn has been a long time malignant presence on the health policy scene.  Among his contributions has been a frontal assault on one of the most effective drugs developed to treat heart failure in African Americans (BiDil) by claiming it shouldn't be approved because we didn't know the mechanism of action.  That brought a swift response from the FDA's Bob Temple who noted that if the FDA only approved drugs where the mechanism of actio was known, a lot of people alive today would be dead.

Now Avorn and part of his crew, Aaron Kesselheim and Jan Myers are arguing that drugs for rare diseases are not studied long enough and don't have enough people in clinical trials AND should be studied in randomized controlled trials.

Avorn, Kesselheim and Myers to kids dying of rare disease:  Drop dead while we study you as long as we deem it appropriate. Note to Kesselheim and the rest of the Kevorkian Corp: orphan drug trials are designed to reflect the size and needs of patients, not the use of CER as a rationale for rationing.

Orphan drugs add more years to  life than any other class of treatments.  Two-thirds of the people who benefit are kids who can now live longer instead of dying painfully.   But CER advocates like Avorn and Kesslehim want   longer and more complicated trials because they know, as with Avastin , average results will reduce the number of treatments. CER will be a death sentence to many children.  People like Kesselheim and Avorn raise the bloody shirt of drug safety to justify their position.  Here's my view: let them say no to orphan drugs when it might save the life of their loved ones out of safety concerns and leave the rest of us alone.  Please.

Thankfully, Congress is putting it’s foot down in support of innovation and personalized medicine.  Sen. Robert Casey, D-Penn., has introduced legislation last March to support this research. The Creating Hope Act of 2011, will provide incentives “to develop treatments for rare diseases that are often less profitable than treatments for more common medical conditions."  Meanwhile, Senators Jon Kyl R-TX and Mitch McConnell R-KY are sponsoring  The Preserving Access to Targeted, Individualized, and Effective New Treatments and Services (PATIENTS) Act of 2011.  The PATIENTS Act would bar the federal government from using “comparative effectiveness research” to deny or delay coverage of a health-care treatment….”

Today medical innovatons can use the understanding of how we get sick to prevent disease or death.  Such advances lead to longer, better lives, enriching us all.  CER, by design, censors these biological insights.  It deliberately delays progress by demanding studies that, by ignoring individual differences,  conclude no one benefits from medical progress.   It is used to justify rationing, not make individuals more sustainable. To save ourselves and children dying of rare diseases we have to pull the plug on CER and it’s adherents.   Starting with Kessleheim, Myers and Avorn -- Harvard's Kevorkian Krew -- is a great place to start.

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The Last Mile Towards First-to-File

06/15/2011 10:14 AM |

From today's edition of The Washington Times:

Protect inventors with strong patent reform:

Shift to ‘first-to-file’ would make system more efficient, less costly

By Peter J. Pitts

 

While there are many problems with the U.S. health care system, it's undeniable that our country leads the world in producing the most advanced medical techniques, drugs and lifesaving technology.

 

Today, our leadership in this field is being threatened - not just by taxes, regulations or the new health care reform law - but also by a patent system that is increasingly ill-equipped to do its job of rewarding inventors and innovation.

Now that we are tantalizingly close to reforms that will modernize and strengthen the U.S. patent system, a small group of misguided ideologues is threatening to derail the effort.

 

There's no question that innovation is the lifeblood of our economy and strong patents are innovation's backbone. But over the years, the U.S. patent system has failed to keep pace with changes in the economy that demand stronger patents, faster approvals and patents that are recognized by our key trading partners.

 

There are numerous problems. For starters, the Patent and Trademark Office (PTO) faces an astonishingly large backlog of 700,000 patent applications, and inventors typically wait three years before getting a patent. Inventors then face the prospect of lawsuits challenging their patents, which cost an average $400,000 to defend.

 

Because the U.S. patent system is different from those of most other industrialized countries, inventors can find themselves with patent rights in the United States, but not abroad, cutting the value of their patents.

 

As Rep. Lamar Smith, Texas Republican, put it: "The current patent system is outdated and bogged down by frivolous lawsuits and uncertainty regarding patent ownership."

 

Legislation pending in the House would go far to fix these problems. It would, for example, let the PTO set its own fees so it has the funds needed to do its job, ending congressional raids on the PTO's budget while protecting small, independent inventors with heavily discounted fees.

 

It would more effectively weed out wrongful patents and settle disputes through a vastly improved "supplemental examination" process.

 

But most important, the legislation would shift the United States from a "first-to-invent" system to a "first-to-file" system.

As things stand, if an inventor is the first to file a patent, he still can be challenged by someone who claims to have invented the same thing earlier but failed to file it with the PTO. That results in too much uncertainty and far too much litigation over patent rights, which is why so many other countries moved to a first-to-file system. Under that system, the first to file with the Patent Office is the rightful patent holder, making patents easier and less costly to defend.

Most lawmakers in the United States realize the need to make these changes, and earlier this year, the Senate voted 95-5 to pass the America Invents Act. A similar bill passed the House Judiciary Committee by an equally strong 32-3 vote.

 

But with the finish line in sight, a small group of conservative lawmakers want to derail the bill, claiming it is an unconstitutional attempt to subvert our patent system in deference to European and Asian governments. Phyllis Schlafly has called it an "un-American" effort by liberals to "put us on the road to a borderless patent system." Those arguments are misguided at best.

 

First, the fact that the reforms so far have received overwhelming support from GOP lawmakers shows that it's not some liberal conspiracy. The House bill's sponsor, Texas' Mr. Smith, has a lifetime American Conservative Union rating of 92 out of 100.

 

Second, the measure is clearly constitutional. In fact, Michael Mukasey, who was President George W. Bush's attorney general from 2007 to 2009, reviewed the bill and concluded that it was "both constitutional and wise."

 

Third, the change from first-to-invent to first-to-file isn't as revolutionary as critics make it seem. PTO Director David Kappos notes that of the 3 million patents filed in the past seven years, just 25 were granted to inventors who weren't the first to invent, with just one granted to an independent inventor.

 

"In the last seven years," he told a congressional panel in March, "only one independent inventor's filing would have received a different outcome under the first-inventor-to-file system. That's one in 3 million."

 

What these reforms would do is make the patent system stronger, more transparent, fairer, faster, less costly and more in line with our major trading partners. Does that seem un-American?

 

In the past, our patent system served our inventors and our country as well. Now we have the opportunity to renew our patent system so it can effectively encourage, protect and reward inventors and keep the United States on the cutting edge of health care well into the next century.

 

Let's not blow it.

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The Death of Hope

06/14/2011 03:45 PM |

Add people suffering from mental illness to the body count from the war of fear waged against drug companies:
tinyurl.com/3rkxvkp

Psychopharmacology in crisis

Researchers warn of 'withdrawal of hope' as funding shrivels.

Daniel Cressey

Many people affected by mental illness are facing a bleak future as drug companies abandon research into the area and other funding providers fail to take up the slack, according to a new report.

Produced for the European College of Neuropsychopharmacology (ECNP), the report warns that "research in new treatments for brain disorders is under threat". With current treatments inadequate for many patients, it says, "withdrawal of research resources is a withdrawal of hope for patients and their families"¹.

A number of formerly big players in neuroscience have all but abandoned the area recently as the pharmaceutical industry has undergone massive restructuring. AstraZeneca and GlaxoSmithKline have both cut research funding and closed down entire teams dedicated to developing drugs for psychiatric disorders.

Although some of the problems faced by the field also apply to other sections of the pharmaceutical industry, many are specific to researchers trying to hit targets in the brain.

David Nutt and Guy Goodwin, who authored the report following a recent ECNP meeting on the topic, note that it can take much longer to develop medicines for psychiatric disorders than for better-understood conditions such as cancer, and that potential drugs for psychiatric conditions have higher failure rates. These failures sometimes become apparent only late in the development process, making neuroscience an expensive and risky prospect for industry.

The coming crisis

Nutt, a neuropsychopharmacologist at Imperial College London, told reporters at a press conference in London on 13 June that "these are dark days for brain science".

Both authors add that, in addition to the dearth of pharmaceutical funding, there is still a stigma surrounding conditions such as depression. This feeds through into the money donated to advocacy groups. "Almost nothing" comes from charity groups for mental-health research, compared with huge charity funding in areas such as cancer, notes Goodwin, head of psychiatry at the University of Oxford, UK.

He warns of a "generational crisis" in terms of both training and capacity to develop new drugs for conditions such as depression and dementia, unless the withdrawal of pharmaceutical funding is addressed.

This warning is echoed by an editorial published last week in the British Journal of Clinical Pharmacology (BJCP). 'Vanishing clinical psychopharmacology', written by Joop van Gerven and Adam Cohen of the Leiden University Medical Centre in the Netherlands, outlines the perilous state of the field². Over the past year, the authors write, the BJCP has published only five papers in this area, none of which involved novel drugs.

At the 2011 meeting of the American Society for Clinical Pharmacology and Therapeutics, only 13 of 300 abstracts related to psychopharmacology and, again, none related to novel drugs. This situation mirrored that at the 2010 Collegium Internationale Neuro-Psychopharmacologicum, where 8 of 870 abstracts were on human psychopharmacology and four were on "new or relatively new mechanisms of action", they report.

The road ahead

Cohen says that much of the problem derives from a failure to develop the underlying science. Depression is a complex disease, yet to assess whether drugs work, researchers have to rely on crude tools such as questionnaires.

"People have not paid enough attention to how to measure depression, how to measure psychosis," he says. "When we develop new drugs, we still measure on these basic scales."

Developing new ways of assessing brain function and disease will reduce the risks in developing new drugs, van Gerven and Cohen argue in their editorial.

Nutt and Goodwin also suggest a number of ways forward. Patents could be longer-lived for drugs that take longer to develop, such as those for brain disorders, to encourage companies to work in the area. And researchers should lobby for European funding — such as that available from the Framework programme initiative — to set brain research as a priority.

Academia could also develop more creative relationships with industry in order to fill the gap in drug development, they argue. The ECNP is pushing the idea of a 'medicines chest', to which companies can assign compounds they are no longer actively developing to be taken forward by researchers in academia or elsewhere. Nutt says that several companies have already expressed an interest in this idea.

If the current research base is allowed to evaporate, Nutt warns, it will be decades before it can be built up to start again.

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Safe use via Smart Labels

06/14/2011 02:40 PM |

FDA NEWS RELEASE

For Immediate Release: June 10, 2011
Media Inquiries: Morgan Liscinsky, 301-796-0379, morgan.liscinsky@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA

FDA approves redesigned labels for some Merck drugs
Changes developed under Merck’s Label Standardization Project

The U.S. Food and Drug Administration today is announcing the approval of Merck’s redesigned drug container labels that include a new standardized format to improve readability and provide better information on product and strength differentiation.

Merck’s Label Standardization Project includes the revision of 34 container labels for 16 solid oral drug products regulated by the FDA’s Center for Drug Evaluation and Research (CDER). Drugs affected by the revisions include: Cozaar, Crixivan, Hyzaar, Isentress, Janumet, Januvia, Mevacor, Noroxin, Prinivil, Prinizide, Propecia, Proscar, Singulair, Zocor, and Zolinza.
 
“We commend Merck for their efforts,” said Janet Woodcock, M.D., director of CDER. “This was no small undertaking, and we are hopeful that Merck’s new standardized labels will aid in reducing pharmacy selection errors.”

Merck's project included evaluating the proposed label content and layout, selecting new packaging design, and obtaining regulatory approval to implement the new packaging design. The Label Standardization Project process included:

• a scientific approach to label design through Human Factors Engineering and Usability Studies
• incorporation of feedback received from the FDA and from label surveys
• a bundled supplement regulatory approach to ensure that labels were acceptable across CDER’s eight clinical divisions.

To better access the impact of these label changes, the FDA encourages health care providers to report medication errors related to the products included in Merck's Label Standardization Project to MedWatch, the FDA’s adverse event reporting program.

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Part D gets an A while Chuck Schumer gets a D

06/13/2011 05:19 AM |

The bad news is that safest place to be in Washington these days is between Chuck Schumer and the facts about Part D.  Safe -- because it’s the path less traveled. Bad news because he has recently begun trumpeting the need for government price controls and extended rebates for duel eligibles as the “solution” to increased Medicare spending.

New York’s senior senator is living in talking point fantasyland.

The good news is that accumulating evidence shows that Part D is succeeding beyond all expectations, delivering needed prescription drugs to Medicare beneficiaries for less money than anyone expected—driven by strong competition among plans.

Consider:

Current CBO Estimates Show Part D Is Costing Far Less than Initial Estimates 

* The 2011 CBO Medicare Part D baseline forecasts and actual recorded spending show costs for Part D benefit payments have declined by 46%, for the 2004 to 2013 period compared with initial estimates of the 10-year cost projections for those years.[i]

* Also this year, CBO reduced its baseline 10-year spending projections for all of Medicare by $186 billion, mostly due to lowered drug spending forecasts. CBO cites that “[a]pproximately two-thirds of the change comes from reducing the projected growth rate for Part D (prescription drug) spending per enrollee on the basis of an updated analysis of national trends in spending for prescription drugs.”[ii]

Part D Plan Bids Declined, Even as the Value of the Benefit Increased

The Affordable Care Act (ACA) significantly enhanced the value of Part D benefits by providing:

* a one-time $250 tax free check for beneficiaries with any spending in the gap in 2010
* a 50% discount on branded drugs while in the coverage gap beginning in 2011; and
* a phased-in closing of the remaining coverage gap by 2020 for both brand and generic drugs.

Despite these significant enhancements to the value of the benefit, Part D plan bids, for which plans need to be accurate since they are at risk, decreased for the 2011 plan year. According to CMS, the Part D National Average Monthly Bid Amount for 2011 is $87.05, a decrease of $1.28 (or -1.4%) compared with the 2010ii] Further, the Medicare Trustees estimate that “[f]or 2011 and beyond, the bids are projected to ultimately converge to between 1 and 2 percent lower than actual spending due to aggressive plan bidding.v]

Medicare Trustees Find Continued Robust Negotiation of Drug Rebates by Part D Plans

In analyzing Part D spending, the Medicare Trustees cite significant levels of rebates across the universe of drugs in the program, including brands and generics. In 2011 the Trustees reported, “rebates for 2009 were approximately 11.1 percent of total prescription drug costs, which was somewhat higher than the plans estimated in their bid submissions. However, some of the drugs with the highest Part D rebate amounts will be losing patent protection in the next several years. As a result, rebates are projected to decrease from 10.7 percent in 2010 to 9.7 percent in 2020.”[v]

The Trustees clarify that the reported rebate levels “are average rebate percentages across all prescription drugs. Generic drugs, which represent about 72 percent of all Part D drug use in 2009, typically do not carry manufacturer rebates. Many brand-name prescription drugs carry substantial rebates, often as much as 20-30 percent.”[vi]

Recent Research Shows that Competition among Part D Plans Lowers Drug Spending in the Private Sector  

Economists from the University of Southern California and Boston University have found that because Part D resulted in more people being covered by private insurance plans, the plans’ negotiating powers were increased such that “[o]n average, Part D lowered retail prices for commercial insureds by 5.8% to 8.5%. The cost-savings to the commercial market amount to $3 billion per year, which approximates the total annual savings experienced by Part D beneficiaries who previously lacked drug coverage.”[vii]

Average Beneficiary Part D Premiums in 2011 Are 43% below Original Projections

The average monthly beneficiary premium for Part D coverage will be $30 in 2011, far below the $53 forecast originally, and an increase of only $1 over the 2010 average premium of $29. According to CMS Administrator Don Berwick, “[t]hese very modest increases in premiums, along with the new discounts...are going to make medications more affordable to Medicare beneficiaries”[viii] CMS officials report that in 2011, over 99% of Part D enrollees will have access to a plan with a premium that is the same or lower than their 2010 premium.[ix]

Beneficiaries Are Highly Satisfied with Part D

Recently released polls show that Medicare Part D enrollees are overwhelmingly satisfied with their Part D coverage. It shows 84 percent of Part D enrollees are satisfied with their coverage, and 95 percent say their coverage works well. Additionally, vulnerable beneficiaries who are dually eligible for both Medicaid and Medicare exhibited the highest satisfaction.[x]

Senator Schumer should check the facts before he points the finger.   Read More & Comment...

Warning Shot

06/12/2011 09:47 AM |

From the pages of the New York Times:

Letter

Warning Labels on Drugs
June 10, 2011

“Side Effects? These Drugs Have a Few” (Week in Review, June 5) rightly ridiculed the growing number of warning labels on prescription drugs. But it’s the Food and Drug Administration’s job to promote the safe and effective use of medications and medical technology.

Unfortunately, the current liability system encourages well-financed tort lawyers to view new product warnings or withdrawal decisions as signals to file lawsuits in hopes of securing a quick payday. Such irresponsible litigation doesn’t make America’s health care system safer — it just helps a small number of lawyers get rich.

PETER PITTS
New York, June 6, 2011

The writer is president of the Center for Medicine in the Public Interest and a former F.D.A. associate commissioner.

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But some are more equal than others

06/10/2011 12:29 PM |

According to Politico, “Consumer advocates are blasting the FDA for the process it has used to renegotiate the terms of the drug approval process following news that a deal has been reached with industry on proposed changes to the process.”

The FDA had been holding two sets of meetings in working out an agreement on the Prescription Drug User Fee Act, which Congress must reauthorize in 2012. One set has been held with representatives of PhRMA and the Biotechnology Industry Organization, the other with stakeholders including patient groups, consumer advocates and provider organizations.

According to an email obtained by POLITICO, industry accepted an FDA proposal “shortly before Memorial Day weekend” and forwarded an agreement to HHS last week. But stakeholders did not receive details on the deal until Wednesday morning. The email, which was forwarded to POLITICO by a source, was sent by Theresa Mullin, the Director of the Office of Planning and Informatics in the FDA Center for Drug Evaluation and Research.

 “I was not surprised that we were left in the dark,” said one rueful consumer advocate participating in the stakeholders’ meetings on background. “We went through the motions but the real deal was over there with industry and the process was opaque.”

FDA spokeswomen Karen Riley declined to comment on any of the specific details of this process.

"The package of proposed recommendations is currently undergoing further administration review and that we cannot comment on the details of the package," Riley wrote in an email back.

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More Signals. Less Noise.

06/10/2011 02:04 AM |

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Jenny’s Vaccine Fearmongering

06/08/2011 02:54 AM |

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A Mighty Minyan and the Lords of Discipline (Letters)

06/07/2011 08:47 AM |

Terrific review of PDUFA V (via BioCentury’s always insightful Washington Editor, Steve Usdin), “PDUFA:  A lot of talk.”

The selected verbiage below is from Mr. Usdin’s article – the subheads are not.

Thy rod and thy staff, they comfort me

High quality reviews performed on a predictable timetable has been the industry’s primary objective for PDUFA since the program’s inception in 1992. But as user fees have exploded over the decades and have been allocated to a widening range of activities, success in achieving their principal goal has been elusive. FDA has consistently managed to approve about 60% of priority NDAs for new molecular entities (NMEs) and original BLAs within a single review cycle, but has done so only by pulling staff away from standard reviews.

The 33 1/3^rd % Solution

Although about 80% of all NDAs and BLAs are eventually approved, there has never been much more than a one-in-three chance that a standard NME or BLA would be approved in the first cycle.

Subjective Meeting Objectives Meetings

To increase first-cycle approval performance, the PDUFA V agreement adds a two-month “filing period” to the start of the review process for NME NDAs and original BLAs, establishes a mid-cycle meeting to allow sponsors to attempt to get errant reviews back on track, and sets up a late-cycle meeting to smooth any advisory committee meetings or negotiations over labels or risk evaluation and management strategies (REMS).

In addition to improving communications with sponsors, the agency plans to create new fora for patients, along with formal procedures for integrating patient perspectives into review criteria.

According to CDER Director Janet Woodcock, “I would like to pursue patient-centered drug development where the regulators and the developers really understand where the patients are coming from in that disease,” She said that “no one, the medical profession or the FDA, has done a good enough job listening from the patients how drugs feel to them and how they feel about benefits and risks.

The BRAT Pack

Woodcock added that FDA is developing a semi-quantitative benefit-risk framework that will “standardize how we think and talk about benefits and risks and residual uncertainty,” and that the agency plans to make it part of the review process during PDUFA V.

Begging the question – or just begging?

The industry also has agreed that FDA can use PDUFA money to hire over 10 staff to bring new scientific expertise into the agency. Nevertheless, the total tab for PDUFA V over five years is expected be about $3 billion, compared to $2.9 billion for PDUFA IV. Thus the begging question will be whether the added review time and communications commitments, rather than money, will achieve the kind of agency performance that has not been reached in prior user fee agreements.

Unpresent Danger

Moreover, the agency’s Office of Surveillance and Epidemiology (OSE) has not been present in the negotiations, which could be a sign the agreement will not resolve poor coordination and bureaucratic in-fighting.

A Mighty Minyan

Industry agreed to pay for 10 FTEs to implement the enhanced communications program, as well as $100,000 annually for internal training and industry outreach. FDA has agreed that the “liaison staff. The new liaison staff will “serve as a point of contact for sponsors who have general questions about drug development or who need clarification on which review division to contact with their questions,” according to the goals letter.

Better Late Than Never

PDUFA V also will add a late-cycle meeting for new molecular entity NDAs and new BLAs that is intended to be a comprehensive assessment of the agency’s review, including descriptions of issues that might be raised at an advisory committee meeting.

The late-cycle meeting addresses complaints from sponsors that they have learned about critical issues FDA will raise with an advisory committee only a few days before the meeting, and that often companies learn about agency concerns for the first time in a complete response letter.

For applications that will be discussed by an advisory committee, the late-cycle meeting will occur at least 12 calendar days before the panel meets. FDA will provide the sponsor with a draft of the questions the agency plans to ask the committee.

The Lords of Discipline (Letters)

FDA has stated it will provide the briefing package at least 20 calendar days prior to a scheduled advisory committee meeting and at least 12 calendar days prior to the late-cycle meeting if a panel meeting is not planned. FDA told industry it aims to have “discipline review” letters ready in advance of the late-cycle meeting. Discipline review letters note any deficiencies in specific sections of applications, such as the clinical, chemistry, manufacturing and controls, non-clinical pharmacology and toxicology, and the human pharmacokinetics and bioavailability sections.

A New Broom?

In the end, FDA and the industry groups agreed on a sweeping policy that applies to all companies and is intended to make the drug review culture more interactive and collaborative.

Amen.

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When PMI is TMI

06/06/2011 08:48 AM |

Ah, the brief summary.  Inside the FDA many joke that it is like the Holy Roman Empire – neither brief nor a summary.  And if you want to throw in “fair balance and adequate provision,” well, go right ahead.  Same issue – too much information equals not enough comprehension.

It’s not a new problem. FDA took a crack at addressing it in January 2004 with a draft guidance on new ways to approach the brief summary.  (I was proud to have been part of that effort.) Then, in January 2006, came the agency’s New Labeling Rule – an attempt to make the PI more user-friendly (the “user” being the physician). More recently the FDA has launched its "safe use" initiative, trying to reconfigure patient medical information (PMI) in more potent and health literate ways to plural constituencies. To date, all of these efforts met with only modest success.

This is an important public health issue. (In 2006, Dr. Jerry Avorn and Dr. William Shrank of Harvard Medical School wrote a paper in The New England Journal of Medicine calling it “linguistic toxicity.”)

The issue comes front and center again in a short article that appeared in Sunday’s edition of the New York Times, “Side Effects?  These Drugs Have a Few.”

Some snippets:

Dr. Jon Duke of Indiana University was trying to figure out why his patient’s blood platelets were abnormal. Could it be a side effect of one of the dozen drugs the man was taking, a number that is not uncommon among elderly people?

He began reading the label of each and every drug. “I was just overwhelmed,” Dr. Duke said. The lists of possible adverse reactions went on and on. Now he knows why. In a new paper in the Archives of Internal Medicine, Dr. Duke and two colleagues report that the average drug label lists 70 possible side effects and some drugs list more than 500. “This was beyond even what I’d expected,” he said. 

For anyone who has ever had to watch an entire Flomax commercial, the listing of a drug’s side effects is almost a joke. But the question is, why does the list continue to grow?

That same year, the Food and Drug Administration suggested making labels clearer with a new format and advised drug makers: “Exhaustive lists of every reported adverse event, including those that are infrequent and minor, commonly observed in the absence of drug therapy or not plausibly related to drug therapy should be avoided.” 

But, Dr. Duke found, instead of decreasing in the years after the agency issued guidelines, the average number of side effects rose to 94, as compared to 67 for those whose labels predated the new format. Some potential complications are weird, like “compulsive gambling.” Others, like “nausea” are so common — it’s listed on 75 percent of drug labels — that they almost seem like a universal issue. 

Listing every inkling of an adverse reaction can help drug companies in lawsuits, Dr. Duke said. If someone sues about a side effect that is listed in the drug’s package insert, the company can say patients had been warned. 

The Pharmaceutical Research and Manufacturers Association says the companies are just complying with the F.D.A.’s requirement that they reveal all of a drug’s risks, “even if a clear causal connection between the medicine and the observed adverse event cannot be fully established,” a spokesperson for the group wrote in an e-mail. 

And the F.D.A., in an e-mail, said “extensive lists of rare and minor adverse events for which there are no data to support a causal relationship” are not useful. 

That last statement from the FDA is pretty quixotic when you consider both the volume of warning letters and their content. Perhaps the above e-mail hasn’t yet made it down to DDMAC.

But the big problem is that the current liability system doesn’t reward lawyers who focus on real public health concerns. Instead, the most experienced and well-financed law firms know that the biggest payouts regularly go to those who take advantage of the FDA’s best efforts to promote the safe and effective use of medications and medical technology. More and more often, these “mass tort” firms specialize in taking a new product warning label or withdrawal decision by the FDA, and view it as a signal to go forward with all guns blazing. Their bullets, unfortunately but not unpredictably, hit multiple innocent targets and result in a wounded American health care system.

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Push Peggy's Buttons!

06/03/2011 10:07 AM |

What Are Your
FDA Hot Buttons?

Help BioCentury
tell Commissioner Dr. Margaret Hamburg
what's important to you.

In an Exclusive Interview
on
"BioCentury This Week" TV
 

Email your comments or questions
to viewercomments@biocenturytv.com
(sender identity will not be disclosed)

Your questions/comments must be received
by Tuesday, June 7

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Join us in Geneva

06/03/2011 08:52 AM |

Chronic diseases such as cancer, heart disease and diabetes have overtaken infectious diseases as the biggest health problem facing developing countries.  While there are many cost-effective ways of tackling these diseases, they often require access to well-trained medical professionals and high quality health infrastructure - something that is sadly in short-supply in many parts of the world.

Please join us in Geneva for an important lunchtime conversation with Eric de Roodenbeke, Chief Executive of the International Hospital Federation. Dr. de Roodenbeke will address the challenges facing developing countries as they adapt to this new epidemiological landscape – and provide some context ahead of the UN Summit on non-communicable diseases in September 2011. 

 When: Wednesday 13th July, 12:45-2:30pm

Where: Hotel Intercontinental, Geneva

Buffet lunch will be included - attendance is free of charge

About Eric de Roodenbeke

Dr. de Roodenbeke is Chief Executive of the International Hospitals Federation, the global association of hospitals and healthcare associations. He has held senior positions in the World Health Organisation, the World Bank, and the French Ministry of Health.  He has extensive hospital management experience, gathered both in Africa and France, and has published widely on hospital organisation, health systems reform and health policy insurance and financing in developed and developing countries.

The e-vite (along with RSVP form) can be found here.

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Harlan Speaks!

06/02/2011 09:18 AM |

“Beware of the tension between CER and personalized medicine.”

-- Francis Collins

Some interesting and thoughtful comments from the interesting and thoughtful Harlan Krumhotz.  One thing to note, is that he is a member of the Patient-Centric Outcomes Research Institute (PCORI) board – an organization tasked via the Patient Protection and Affordable Care Act (aka, “ObamaCare,” aka, “healthcare reform”) to study comparative effectiveness research. 

No, that’s wrong – what they’ve been tasked to study (by specific legislative language) is comparative clinical effectiveness research.  And that’s not rhetoric. Comparative means which treatment (or healthcare technology if you prefer) is “better” (subjective) versus data on real world clinical outcomes. To put it bluntly, “comparative” is subjective. Clinical is outcomes-driven. It’s important to remember both the letter and the spirit of the stature.

And now let's hear directly from Harlan K.

Five Lessons From Niaspan’s Disappointing Study

By HARLAN KRUMHOLZ

Comparative effectiveness research — investigations that determine which treatments are best — has attracted attention in the health care debate. Critics charge that these studies are designed to restrict choice. The Center for Medicine in the Public Interest released a report that suggested that they would stifle innovation. Often they are framed as studies to support efforts to keep useful but expensive therapies from patients.

But what if these studies, done well, revealed that some medications were better than others? What if they overturned conventional wisdom about understudied drugs, demonstrating that many patients were receiving ineffective treatments? What if they showed that some patients were actually being harmed? What if more knowledge about the benefit and risk of treatments in medicine, compared with their alternatives, is just what patients need?

His complete comments can be found here – and they’re worth a read.

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Comparative Harmfulness?

06/02/2011 08:56 AM |

An amendment to the House Agriculture/FDA appropriations bill that appears intended to block FDA from restricting agricultural use of antibiotics could stymie agency efforts to curtail the use of unsafe products. Farmers and ranchers have been concerned that FDA will limit the use of antibiotics in animals in order to reduce the incidence of resistance to the drugs.

Montana Republican Denny Rehberg insisted his proposal is motivated by interest in the animal sciences. "If I'm looking at it from the perspective of all the rules and regulations I'm required to conform to on my farm and my ranch, it has nothing to do with anything other than trying to make a determination, is the Food and Drug Administration going ... into the social sciences as opposed to the hard science?"

But amendment (approved 29 to 20 by the full House Appropriations Committee as part of the fiscal 2012 Ag/FDA funding package) does not limit its effect to the animal sciences.

Rather, it states that FDA may not spend money from the bill "to write, prepare, develop or publish a proposed, interim, or final rule, regulation, or guidance that is intended to restrict the use of a substance or a compound unless the Secretary bases such rule, regulation or guidance on hard science (and not on such factors as cost and consumer behavior), and determines that the weight of toxicological evidence, epidemiological evidence, and risk assessments clearly justifies such action, including a demonstration that a product containing such substance or compound is more harmful to users than a product that does not contain such substance or compound, or in the case of pharmaceuticals, has been demonstrated by scientific study to have none of the purported benefits."

The latter two conditions would put FDA in the position of examining the comparative harm of two options, or in the case of drugs, having to prove a negative. Further, the amendment would seem to prevent FDA from issuing any regulations or guidances related to REMS.

Oops.

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