Heart is Half a Prophet

03/23/2012 03:38 PM |

Eric Topol and colleagues at Scripps have developed a gene test that quickly and accurately predicts who is at risk for sudden heart attacks. Now here's the question: will we have to wait years for AHRQ and health plans to pay for this diagnostic? How many avoidable deaths will be caused by a delay or underpayment in reimbursement? http://stm.sciencemag.org/content/4/126/126ra33   Read More & Comment...

PDUFA and the Critique of Pure Reason

03/23/2012 07:20 AM |

Immaturity is the incapacity to use one's intelligence without the guidance of another. -- Immanuel Kant

Is "Kant" in the FDA vocabulary?

Here’s the latest House PDUFA discussion draft being circulated by the House Energy and Commerce Committee.

As pointed out in the Pink Sheet, guidance development would take on a much larger and likely more extensive role at FDA if provisions in the draft are ultimately approved. The draft, would require FDA to give at least three months’ notice before it intends to issue a certain type of draft guidance and solicit public comment.

New, more rigid guidelines for public participation prior to issuing some draft guidance documents, as well as a requirement that they be finalized within a year, likely would require an increase in agency resources devoted to those efforts.

The more predictable schedule also would favor industry by ensuring the waiting period for FDA thinking on a subject would not be potentially unending. Industry typically complains about the uncertainty of FDA policy on many subjects.

The rule would apply to a guidance that “sets forth initial interpretations of a statute or regulation, sets forth changes in interpretation or policy that are of more than a minor nature, includes complex scientific issues, or covers highly controversial issues,” according to the draft.

A clause in the proposal allows the agency to skip the advance notice if it is impracticable, unnecessary or contrary to public interest, but it would still be required to meet with stakeholders during a three-month period beginning the day the guidance is issued.

Once a draft guidance is released, FDA would be required to finalize it no more than 12 months later or it would be considered null and void, according to the discussion draft.

Comprehensive reviews of final guidances also would be required every five years under the bill to ensure they are not “outmoded, ineffective, insufficient or exceedingly burdensome.”

The provision would subject FDA to a much more formal schedule of guidance development. While the intention appears to exclude routine guidance updates from the rule, it would be easy to place most, if not all, new agency guidance, into one of those categories, especially “highly controversial” and “more than a minor nature.”

The schedule would create more pressure on the agency to finalize guidances before the one-year deadline, which could leave them incomplete or rapidly outdated. A more structured guidance development process ideally would prevent situations where industry has been penalized for violations of FDA policy, yet still waited for years to receive formal agency thinking on the subject.

Sponsors have complained the agency changed its guidance on clinical trial endpoints in the midst of a development program, making continuation more complicated.

FDA is clearly being forced to battle against congressional efforts to adjust its mission to focus more on issues of innovation … and PDUFA’s first principle of predictability.

A provision would add to FDA’s mission that it would strive to establish a regulatory system that “protects the public health and enables patients to access novel products while promoting economic growth, innovation, competitiveness, and job creation among the industries regulated by this Act,” according to the draft.

The package includes reauthorizations of the prescription drug and medical device user fees, as well as new programs for generic drugs and biosimilars.

The four user fee cycles would run from FY 2013 through FY 2017.

The user fee package is expected to be ready for passage soon. Health Subcommittee Chairman Joe Pitts, R-Pa., already has stated he wants the bill signed by President Obama by June.

He alone is free who lives with free consent under the entire guidance of reason. -- Baruch Spinoza   Read More & Comment...

Downton AbbVie

03/22/2012 08:16 AM |

Abbott Laboratories’ pharmaceutical spinoff will be called AbbVie and will debut by the end of the year.

  Read More & Comment...

Prometheus Bound

03/21/2012 08:45 AM |

Abraham Lincoln wrote that patents, “add the fuel of interest to the passion of genius.”

The Supreme Court of the United States has just doused a lot of that passion – and, along with it, the hopes that personalized medicine (via advanced diagnostics) can become reality sooner rather than later. 

Or, as the Wall Street Journal judiciously puts it, “The ruling sparked uncertainty about the booming field of personalized medicine, in which some of the world's largest drug companies are vying to tailor treatments to patients' unique makeups by using diagnostic tests.”

The Supremes have rules that telling doctors of a new scientific discovery and recommending they use it to treat patients is not patentable. Per Justice Stephen Breyer, that’s all Prometheus Laboratories did when it patented a test to help doctors set drug dosages for patients with Crohn's disease. The inventions claimed in the patents "consist of well-understood, routine, conventional activity already engaged in by the scientific community.”

Justice Breyer said the Prometheus patents recited laws of nature connecting the level of certain chemicals in the blood to a thiopurine dosage that is too high or too low. Then, he said, the patent walked doctors through several obvious steps—such as measuring the chemical levels—to turn that natural law into recommendations for particular patients. He rejected the company's contention that, taken together, the steps represented a patentable method for treating patients.

But isn’t that what “intellectual property” is all about?

Justice Breyer emphasized that patent law should not inhibit future innovations by "tying up the future use" of natural laws. He said Prometheus's patent claims "threaten to inhibit the development of more refined treatment recommendations."

But where there is no patent protection there is no investment.  And where there is no investment there is no development. 

The only people who are going to love this ruling are those who agree with  Jamie Love that separating the markets for innovation from the markets for physical goods will ensure that everyone, everywhere, will have access to new medical technologies at marginal costs.

Baloney.

Prometheus Bound is, after all, a Greek tragedy.
 

  Read More & Comment...

Survival of the Most Personalized

03/20/2012 08:20 AM |

According to Alan Ashworth, head of the Institute of Cancer Research in London, "Today, despite the best efforts of the drug companies, we still use medieval methods to attack most cancers: cutting [surgery], burning [radiotherapy] and poisoning [non-specific chemotherapy]." But, says Ashworth, "Once we understand the Darwinian process by which cancer cells evolve in the body, we should be able to control even advanced disease through combinations of specific drugs."

  Read More & Comment...

Biosimilar Music Theory

03/19/2012 01:18 PM |

Overheard at event bio break:  “EPO is the new Lipitor.”

Last week in Boston I was honored to give a presentation at the 10^th Biosimilars America conference. But, as is often the case at good events, it was more about what I learned than what I had the chance to say.

Or, as my father used to remind me, “Listening is not just waiting for your turn to talk.”

Of the many fine presentations, I refer specifically to the talk given by John Pakulski, head of US Biopharmaceutical Regulatory Affairs at Sandoz, and the current chair of the GPhA Biologics Task Force.

John made many interesting and important points, but the one that resonated the most was that, in an age of technology, we must use technical data more and rely on clinical trials less. In short – when it comes to understanding the complicated concept of biosimilarity (from both regulatory as well as clinical perspectives) it’s a brave new world. But will we have the courage to move beyond “the gold standard.” Perhaps we should say, “the 20^th century gold standard.”

Claude Debussy said, “Music is between the notes.” And the same can be said for biosimilarity. We now have many thoughtful guidance documents but, in many respects, it’s just theory. And just like with music theory, the words on the page are one thing – but when talented performers sit down at different pianos in disparate venues the result is both similarity and uniqueness.

In other words – creativity happens. 

Another difference between biosimilars and small molecule generics is that the developers of biosimilars are very much in the innovation business. 

Innovation happens.
  Read More & Comment...

Second Look At Statin Risk

03/16/2012 02:18 PM |

A couple of weeks ago I posted on the 'statin scare',  and referred to a study that discussed the increase risk of diabetes for people taking cholesterol lowering meds in primary prevention. 

I was skeptical of a single study as the basis for concern (and certainly panic).    Eric Topol's recent videoblog on the issue is underscores the need for careful review of all data, tailoring treatment to patients to maximize benefits and the urgent need for whole genome research on response and metabolism of all statins.

I am on a low dose statin.  I monitor my liver enzymes pretty regularly (normal for someone who works out as regularly as I claim to!).  It never occured to me that the slight elevation in my glucose levels -- something my doctor has always kept an eye on --  was a 'side effect' of statin use.   Since we have a family history of high cholesterol and heart attacks statins are indicated and my risk of a heart attack were probably greater if I didn't take statins compared to taking them and getting diabetes.   But a more precise and individualized measure of risk and benefit would be greatly appreciated!

Dr. Topol raises important questions about the risks and benefits of using statin in primary prevention absent other risk factors.  And he again makes a persuasive case about the value of genomic testing.  As he notes in his excellent book   The creative destruction of medicine: How the digital revolution will create better healthcare   30 percent of people being treated for diabetes are non-responders to metformin, which is pushed as first line therapy.   Why not a test that measures response and reaction to statins and diabetes meds.  Why not make that test available to consumers in the most convenient way possible?

The impact on compliance and health outcomes would be significant in my opinion.  And as for my use of statins,  I will be a bit more careful going forward.

Thanks Dr. Topol. 

http://blogs.theheart.org/topolog
  Read More & Comment...

Progressive Approvals "Over There"

03/16/2012 09:09 AM |

Many tough questions. No easy answers.

From the latest edition of the British Medical Journal ...

Speeding up access to new drugs
Nigel Hawkes
 

The prime minister promised at the end of last year to give some patients quicker access to new drugs. Was this just an empty aspiration?

No. A lot of thought has been given to a faster access scheme since David Cooksey recommended it in his review of UK health research funding in December 2006. A working group from industry and government produced a plan by November 2009. It sat on a shelf until resurrected two years later.

How would it work?

Medicines that have completed phase III trials (or in exceptional circumstances phase II) and that will treat or prevent life threatening, chronic, or seriously debilitating conditions that lack adequate existing treatments would qualify. Manufacturers would have to apply, and a decision would be promised in 75 days.

How would that be any quicker than licensing, if phase III trials have already been completed?

It usually takes a year or more to get licensing approval after a successful phase III trial. The new process is expected to get these medicines to patients a year earlier than otherwise would be the case.

But doesn't it mean that proper risk assessment will be skimped?

There's a danger of that. The burden of risk will be shifted towards the doctor and the patient and away from the manufacturer. Good information will be vital; patients will need to be fully informed and give active consent. Legally, the position will be the same as that for any unlicensed medicine, and the working group believes-but cannot guarantee-that primary care trusts or clinical commissioning groups will not be liable should anything go wrong. And it says that if the decision by a doctor to treat a patient was reasonable in all the circumstances and all relevant information was provided, a successful claim for negligence is unlikely.

If unlicensed medicines can be marketed and sold, what's the point of licensing?

The medicines under this scheme would be exceptional and few in number, perhaps only one or two a year. The NHS already uses unlicensed or off-label medicines in some cases, on the authority of the prescribing doctor (for example, bevacizumab for age related macular degeneration). Patients consulted by the working group were confident that they were competent to make a proper assessment of the risks. Doctors were not so sure.

What happens when the medicine is licensed?

The approval will last a year and can be renewed if necessary. When the medicine gets a licence it will become part of the normal process and the National Institute for Health and Clinical Excellence (NICE) will examine its cost effectiveness. Arrangements for continued funding of early access patients will need to be agreed for each medicine in advance.

So these fast access medicines will bypass NICE?

Yes. NICE deals with licensed medicines; these are unlicensed, so NICE does not have a role.

So how can we tell if manufacturers are overcharging?

We can't. They will set the price. It will be up to commissioning organisations, without NICE input, to decide if it's a price they want to pay.

Where's the money coming from?

An important question, the answer to which could yet put a spanner in the works. Since these medicines will not be approved by NICE, there will be no obligation for primary care trusts or clinical commissioning groups to pay for them. There will be no additional money available, unless the Department of Health or the drug companies provide it. Commissioning bodies will have to fund the drugs out of their normal allocations and in hard times may be reluctant to do so. This could lead to "postcode prescribing," as the working group conceded. But it hoped that if the process were applied only to medicines providing "significant clinical benefit in areas of current unmet need" the variation could be minimised.

Has anybody else attempted a similar scheme?

Yes, the French Temporary Authorisation for Use (ATU) scheme is quite similar and has operated for 15 years. To qualify for this, the medicine must have no satisfactory alternative and patients cannot access the scheme if they could become part of a clinical trial of the same medicine. The scheme is restricted to hospital specialists, and companies must agree to submit an application for a full licence, usually within a year, of the temporary authorisation being granted.

Experience shows that companies usually set high prices, but if the price fixed after market authorisation is lower they can be asked to refund the difference.

What about surveillance of side effects?

Applications for faster access would have to include plans for drug surveillance. "Collecting safety data is essential to protecting patients receiving the medicine," the working group concluded and is also an important way of developing a better understanding of the medicine. But the demands should not be so burdensome as to discourage companies from applying; nor should data gathering be seen as a clinical trial.

What's next?

The Medicines and Healthcare Products Regulatory Agency plans to launch a consultation on the scheme at the end of March. It will allow 12 weeks for responses.

  Read More & Comment...

A Clinical Trial wrapped in a mystery, inside an enigma.

03/15/2012 09:41 AM |

You mean central planning doesn’t work?
 
Russia: Russian Companies Receive Lowest Number of Permits for Clinical Research in Seven Years 
 
Russian companies have received the lowest number of permits for clinical research in seven years, according to a report from Remedium. In 2011, the Ministry of Public Health issued 571 permits for clinical studies, of which 369 were for multicenter studies (an increase of 49% from 2010), and 117 for local studies (a decrease of 27% from 2010).
 
The share of research by Russian sponsors was below average for the last seven years, with domestic producers receiving 14.1% of permissions for drugs research testing—against the seven-year average of 20.6%. According to the report, the number of local studies has fallen following the 1 September 2010 "law on treatment of drugs", which specified that local studies be included in the registration of medicines, meaning that Russian companies must apply for a drug's registration and pay a state fee before testing. There is no such requirement for foreign companies in Russia.
 
The Federal Antimonopoly Service (FAS) is currently preparing an amendment to the law proposing that the requirement to register be cancelled. They are also proposing to cancel the obligation for foreign companies in Russia to conduct local research if not including the country in a multi-center trial.
 
Significance: Russia's healthcare and pharmaceutical industry is currently going through regulatory changes that aim to simplify the registration process and speed market entry for drugs, among other goals. The report will come as a setback to Russia's efforts to expand the pharmaceutical industry and stimulate domestic manufacturers. The changes proposed by the FAS are likely to form a part of the regulatory reforms that are currently being formulated.

  Read More & Comment...

PCORI Spends $9 million and 18 months to define what's in the organization's name

03/14/2012 11:25 AM |

It's the Patient-Centered Outcomes Research Institute.    It will collect $3.5 billion of our insurance premium payments over ten years -- with 20 percent going straight to the Agency for Healthcare Research And Quality -- a federal agency that can now bypass congressional oversight thanks to the pass through. 

PCORI has devoted 9 million taxpayer dollars and nearly two years focusing exclusively on figuring out what patient-centered outcomes research is.   That's like the Department of Homeland Security or Defense Department doing nothing but trying to define what "security" and "defense" is.  

Here's the full defintion of patient-centered outcomes research:

Patient-Centered Outcomes Research Working Definition


Patient-Centered Outcomes Research (PCOR) helps people and their caregivers communicate
and make informed health care decisions, allowing their voices to be heard in assessing the
value of health care options. This research answers patient-centered questions such as:

1. “Given my personal characteristics, conditions and preferences, what should I expect
will happen to me?”
2. “What are my options and what are the potential benefits and harms of those options?”
3. “What can I do to improve the outcomes that are most important to me?”
4. “How can clinicians and the care delivery systems they work in help me make the best
decisions about my health and healthcare?”

(Note that for all the hot air about personalized medicine, there is no specific mention of genetic characteristics)

By way of comparison, it took less time to draft and ratify the Constitution of the United States.  The excuse given for this over-cost and over-due delivery of a definition that leaves everyone more confused than ever is the following: " the difficulty in managing large numbers of healthcare providers with busy schedules and divergent interests."   You see, these board members are much, much busier than Benjamin Franklin, John Adams, and Thomas Jefferson ever were. 
And patient centered outcomes research is supposed to timely and scientifically up to date?  If it took all this time just to agree on a definition,  what can we expect about the effort to develop a research agenda that meets the definition?

As I have noted before, it will take less time to commercialize whole genome sequencing systems that produce same day results for clinical use then it will for PCORI, AHRQ, etc to decide what to study and to pay the groups who control the PCORI and AHRQ apparatus to do the research for the very same organizations.     Did I forget to mention the incredible amount of cronyism involved in the entire CER process?   But more on that in a future post.

A couple oversight hearings on what PCORI is really doing, how it's being done and whether it should be abolished would be beneficial.  I mean, what's a couple more meetings and two days on the Hill when you spend two years defining what your organization should do.   And I bet all those busy providers could clear their calendars pretty quick if called to testify.   Read More & Comment...

PDUFA Predictions Department

03/14/2012 09:03 AM |

Last November, the Center for Medicine in the Public held a confab entitled, “PDUFA without the Politics.” Many of the predictions from the event’s expert panelists seem to be coming true.

AS PREDICTED, the current conversation surrounding the reauthorization of the Prescription Drug User Fee Act (PDUFA) must focus on (among other things) the First Principle of Predictability as well as ensuring that the FDA can fulfill its role as an important ally in advancing healthcare in America.

The draft bill extends FDA's mission to include: advancing medical innovation; promoting economic growth and job creation; promoting predictability and allowing flexibility; identifying and using the most innovative and least burdensome tools for achieving regulatory ends; and incorporating a "patient-focused benefit-risk framework that accounts for varying degrees of risk tolerance, including for people living with a life-impacting chronic disease or disability."

AS PREDICTED, the House Republican discussion draft incorporates the Generating Antibiotic Incentives Now (GAIN) Act, which would extend market exclusivity by five years for drugs that treat antibiotic-resistant pathogens

AS PREDICTED, by Representative Michael Burgess at the CMPI conference, the bill also completely removes limits on issuance of conflict-of-interest waivers for FDA advisory committee participation that were enacted as part of the FDA Amendments Act.

The discussion draft also includes language intended to expand accelerated approval. The accelerated approval provisions are similar to those in the FAST and TREAT acts.

Is there still room for a few more Christmas Tree ornaments?  Perhaps permanent renewal of the Best Pharmaceuticals for Children’s Act and PREA?

  Read More & Comment...

Health Exchange Regs Leave Consumers Short Money, Care and Doctors

03/13/2012 12:54 PM |

Where else but in Washington would the release of 600 pages of regulations be hailed as promoting greater choice, efficiency and reduce cost?

In this regard, the final rule regulating the establishment of "Affordable Health Exchanges" does not disappoint.

Most of the regs deal with how to qualify people living on Indian reservations and setting up systems to determine eligibility and award tax credits to the 9 percent of Americans who might actually get them.   But there are some interesting discussions that crop up which might surprise those who regard exchanges as another step towards utopia:

1.    If your income changes after you buy a health plan with some tax credits,  your costs will go up or down without warning.   And you may have the IRS knocking at your door asking for repayment of the tax credits you planned on having but no longer do as your income changes. 

2.    If your income changes so does your cost sharing.  Even a few bucks a month can make a huge difference.  Hence: A five percent decrease in income
from $47,018 is $44,667 (190 percent of the FPL), would correspond to a total premium, after advance payments, of $217 per month, for a total difference in premium of around $360 per year.  And there's more "... based on the same figures cited above, the difference in cost-sharing between a family at 190
percent FPL and a family at 200 percent FPL is $1,000 per year, due to the change in eligibility for cost-sharing reductions at 200 percent FPL. The difference is $2,000 around 250 percent FPL, which is the upper limit for cost-sharing reductions based solely on household income.

So if your pre-tax income goes up by $2351 you wind up paying an additional $1360.  That's an effective marginal tax rate of 68 percent.

3.   And since such changes do not keep up with tax information, exchanges will be required to create a system to monitor what we make in other ways.  To wit: "the Exchange must use information other than tax data to verify income in cases in which an applicant attests that a change has occurred or is reasonably expected to occur..."   What might that information be?  Bank accounts?  Pay stubs from employers?  Undercover informants?  

4.   If you or I decide to stop paying premiums for three months.  Under Obamacare, we still are able to receive health care services.  Coverage is terminated retroactively after three months and health plans don't have to pay doctors and other professionals for services rendered.   In otherwords, health providers are on the hook for the cost of providing free care if we stop paying premiums.  Or as the regs state: Qualified health plan (QHP)  issuers.. must notify providers who submit claims for services rendered during the second and third months of the grace period that any such claims will be pended, and potentially not reimbursed by the QHP issuer if the individual does not settle outstanding premium payments."

5.  There is other mind-numbing stupidity too.   HHS still hasn't figured out how to get accurate information about income and family size without tax information.  And it will take the word of people that they are poor enough to get tax credits without checking first. 

I am not making any of this up.   I wish I were.
  Read More & Comment...

Mainstream Media Misreads Research on Personalized Medicine

03/12/2012 11:16 AM |

Last week there was doom and gloom about the potential and pace of personalized medicine:  

'Personalized Medicine' Hits a Bump
Wall Street Journal‎ -

    Tumor Evolution More Complex than Imagined
    MedPage Today‎

   Personalized cancer treatment: Genetic differences abound in tumors   Los Angeles Times‎


The source of this pessimism?  A study in the NEJM: Intratumor Heterogeneity and Branched Evolution Revealed by Multiregion Sequencing

http://www.nejm.org/doi/full/10.1056/NEJMoa1113205?query=featured_home

Here's Ron Winslow of the WSJ passing sentence on why the study is a blog to targeting treatments:

"Tumor's genetic makeup can vary significantly even within the same tumor sample, researchers said, a finding that poses new challenges to the personalized-medicine movement in cancer.

One big implication of the new research, being published Thursday in the New England Journal of Medicine, is that analyzing only a single sample of a patient's tumor—the current practice—may miss important genetic mutations that affect the course of the disease.

That, in turn, could hinder emerging efforts to match patients with drugs that target the mutations affecting their tumors, a basic strategy of personalized medicine.

The findings don't diminish enthusiasm for the idea that genetic knowledge about tumors can transform cancer care, the researchers said. But it could make personalized treatment more complex—and more costly."


Winslow and others -- following the bias of the researchers who did the study --  overstate the novelty of finding differences in tumor progression because of differences in mutations identified by exome sequencing and appear to suggest that such subtle changes always mislead.

First,  exome sequencing is becoming commonplace, as is deep sequencing of the type provided by Life Technologies and Oxford Nanopore Technologies.  The costs and speed of such analysis is dropping rapidly.  So cost is not a factor.   Second,  such sequencing will become commonplace precisely because clinicians know that every cancer progresses differently.    Mutations are not always associated with changes in tumor progression.  Sometimes they are, sometimes they are not. 

Winslow and others present personalized medicine as a single gene-single step process of individualizing treatment.  No one who knows anything about the clinical and functional application of sequencing believes that.  Hence, the media -- where many 'thought leaders' still get the primary source material on health and science -- perpetuates both an outdated view of personalized medicine and artificially overstates the costs and barriers of the same. 

  Read More & Comment...

No end in sight

03/12/2012 10:56 AM |

Politico correctly notes that the Supreme Court ruling on the ACA will do little to end the debate on President Obama’s health care law:
 
When the Supreme Court's ruling comes down, probably in June, whoever wins will try to make it sound like the final word on the ACA. But everyone knows that just isn't the case. For Republicans, a win would validate their two-year fight against "Obamacare." For Democrats, a win means the ultimate stamp of approval on Obama's signature policy achievement and the chance to accuse Republicans of wasting the past two years fighting a law that's constitutional. If the court strikes the individual mandate but leaves the rest of the law in place, that would leave policymakers in a tough situation. Or, even if the mandate stays, it does nothing to erase the provision's unpopularity.  Read More & Comment...

Living in a post IPAB world

03/09/2012 07:14 AM |

Those who opposed the health care bill are debating whether “repeal and replace” should be the clarion call in the coming election. Regardless of how this debate turns out, those in favor of doing better should focus their immediate attention on identifying and fixing the most harmful parts of the bill. The Independent Payment Advisory Board, or IPAB, should be a top target.

Sometimes dreams do come true.

IPAB needs to go, but the goal of reigning in Medicare expenses is a worthy one. As is, the program’s costs are spiraling out of control: Medicare is projected to accumulate a $38 trillion budget shortfall during the next 75 years.

Not only does IPAB further grease the slippery slope towards government price controls and rationing -- it doesn’t even have any authority over the biggest cost-drivers in Medicare.

Medicare Part A, for instance, is so expensive its reserves will be empty by 2017, according to the Medicare Trustees. Part A covers in-patient hospitals stays. By 2035, the program’s revenues will only finance about half of promised benefits.

Medicare Part B, which covers out-patient services, has similar cost problems. Administrators just raised Part B premiums on nearly a quarter of beneficiaries because expenses have gotten so high. And an analysis from the Congressional Research Service found that without substantial hikes in Part B premiums, the program’s finances are “at risk of exhaustion.”

Yet the IPAB has no power over Part A or Part B.

If left unaltered, Medicare could literally bankrupt this country.

Last June, HHS launched two important new health care cost-saving initiatives.

First, the HHS announced it would make $42 million available to enhance coordination efforts between primary care physicians and other health care providers treating Medicare patients. The potential savings are estimated at $125 billion over the next 10 years.

Second, the HHS launched a $40 million effort to help states combat chronic disease. Chronic diseases are responsible for 75 percent of our health care costs—diabetes, heart disease and strokes alone account for nearly $1 trillion in medical spending annually.

Both initiatives have the promise to save money and lives. Unfortunately, they represent the opposite approach to Medicare cost control set forth in the Affordable Care Act.

On one hand, the two new HHS initiatives show that the Obama administration is making credible efforts to target the areas of the health care system that could produce the most savings. On the other hand, the Affordable Care Act through IPAB seeks to devalue efforts such as these in favor of squeezing doctors and other providers.

The right way forward is to get rid of IPAB and substitute for it a Medicare cost-savings plan that encourages long-term strategic thinking along the lines of these HHS studies.

Support for IPAB is rapidly and rightly collapsing as citizens become better informed about the danger this all-powerful panel of unelected bureaucrats poses to their health care. It’s time to urge Congress to get rid of IPAB and stand up for real Medicare reform.

  Read More & Comment...

Counterfeit Jail Bait

03/08/2012 08:45 AM |

In 2004 I was a member of the FDA’s inaugural counterfeit drug taskforce. Our draft report called for stiffer criminal penalties for the purveyors of false profits – but we were told to remove that particular codicil because it was “outside of our jurisdiction.”

Now? Well, better late than never. Here’s the press release from Senator Grassley’s office:

Senate Approves Leahy-Grassley Bill To Increase Penalties For Counterfeit Drugs

WASHINGTON – The Senate Tuesday night unanimously approved a bill authored by U.S. Senators Patrick Leahy (D-Vt.) and Chuck Grassley (R-Iowa) to increase penalties for trafficking counterfeit drugs.  The legislation responds to recommendations made by the U.S. Intellectual Property Enforcement Coordinator and the administration’s Counterfeit Pharmaceutical Inter-agency Working Group.

The Counterfeit Drug Penalty Enhancement Act will increase penalties for the trafficking of counterfeit drugs to reflect the severity of the crime and the harm to the public.  While it is currently illegal to introduce counterfeit drugs into interstate commerce, the penalties are no different than those for the trafficking of other products, such as electronics or clothing.  The Counterfeit Drug Penalty Enhancement Act will target violators that knowingly manufacture, sell or traffic counterfeit medicines to the United States.

“We cannot allow the counterfeiting of life-saving medicine to be just one more low-risk venture from which international organized criminals can profit,” said Leahy.  “While we should not expect that enactment of this or any legislation will completely deter the serious problem of counterfeit medication entering the American supply chain, it is an important step in the fight.  I urge the House of Representatives to act quickly on this legislation.”

“Worldwide counterfeit medicines are a multi-billion dollar industry, and growing at an alarming pace, especially over the internet.  These medicines pose a serious threat to the health and safety of unsuspecting Americans,” Grassley said.  “The House should act as quickly as possible to ensure that counterfeit drug traffickers are punished accordingly for putting people’s lives at risk with this serious crime.”

The legislation is cosponsored by Senators Michael Bennet (D-Colo.), Richard Blumenthal (D-Conn.), Sheldon Whitehouse (D-R.I.), Dianne Feinstein (D-Calif.), Jon Kyl (R-Ariz.), Christopher Coons (D-Del.), Amy Klobuchar (D-Minn.), and Robert Casey (D-Pa.).  Companion legislation in the House of Representatives was introduced last year by Representatives Patrick Meehan (R-Pa.) and Linda Sánchez (D-Calif.).

It has been reported that counterfeit drugs result in 100,000 fatalities globally each year, and account for an estimated $75 billion in annual revenue for criminal enterprises   Read More & Comment...

Fixing a hole in the ocean

03/07/2012 08:51 AM |

Its old news that expanded access to healthcare doesn’t correlate to broader (or wider) use of services. This lesson from (among other places) the United Kingdom hasn’t gotten a lot of attention on this side of the pond. In fact, the reverse is true. Government-mandated access results in a reduction in usage.

Why? The reason is the role of the gatekeeper – otherwise known as the physician. As Nadine Reibling and Claus Wendt (University of Mannheim) write in their paper, Access Regulation and Utilization of Healthcare Services:

“Since cost containment is at the top of the political agenda, efficiency considerations dominate the political discussions on instruments that regulate access to care. This, however, neglects the fact that such measures also have implications regarding equity of access (Saltman and Busse 2002).”

In other words, equity of access isn’t the same as availability of access. What happens, for example, when physicians choose not to accept Medicare or Medicaid patients?  What value is equity when there is a physician shortage? And what does this say about how we value – and compensate – our healthcare providers. What unintended consequences arise when we ignore the growing gap between equity and access?

In November 2010, the Physician Foundation released the results of a national survey of physicians intended to gauge American physicians’ initial reaction to the passage of health reform and to learn the ways in which they plan to respond to it.

Key research findings include:

* The majority of physicians (60%) said health reform will compel them to close or significantly restrict their practices to certain categories of patients. Of these, 93% said they will be forced to close or significantly restrict their practices to Medicaid patients, while 87% said they would be forced to close or significantly restrict their practices to Medicare patients.

* 40% of physicians said they would drop out of patient care in the next one to three years, either by retiring, seeking a non-clinical job within healthcare, or by seeking a non-healthcare related job.

* The majority of physicians (59%) said health reform will cause them to spend less time with patients.

* While over half of physicians said health reform will cause patient volumes in their practices to increase, 69% said they no longer have the time or resources to see additional patients in their practices while still maintaining quality of care.

A brand new study from the Health Research and Educational Trust brings the matter to our own shores via a study of the Children’s Health Insurance Program (CHIP).

A Comparison of Two Approaches to Increasing Access to Care: Expanding Coverage versus Increasing Physician Fees

Objective

To compare the effects of a coverage expansion versus a Medicaid physician fee increase on children's utilization of physician services.

Primary Data Source

National Health Interview Survey (1997–2009).

Study Design

We use the Children's Health Insurance Program, enacted in 1997, as a natural experiment, and we performed a panel data regression analysis using the state-year as the unit of observation. Outcomes include physician visits per child per year and the following indicators of access to primary care: whether the child saw a physician, pediatrician, or visited an ER in the last year, and whether the parents reported experiencing a non-cost-related access problem. We analyzed these outcomes among all children, and separately among socioeconomic status (SES) quartiles defined based on family income and parents' education.

Principal Findings

Children's Health Insurance Program had a major impact on the extent and nature of children's insurance coverage. However, it is not associated with any change in the aggregate quantity of physician services, and its associations with indicators of access are mixed. Increases in physician fees are associated with broad-based improvements in indicators of access.

Conclusions

The findings suggest that (1) coverage expansions, even if they substantially reduce patient cost sharing, do not necessarily increase physician utilization, and (2) increasing the generosity of provider payments in public programs can improve access among low-SES children, and, through spillover effects, increase higher-SES children as well.

With all the discussions of equity, perhaps it’s time we asked “Who lost access?” And, more importantly, what can we do about it? Otherwise we’re just (Doc) fixing a hole in the ocean.

Net/Net? If you want more access you have to pay for it.

Nothing fixes a thing so intensely in the memory as the wish to forget it.

-- de Montaigne

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Soda Bad, Magnets...Not So Bad?

03/06/2012 04:26 PM |

The Center for Science in the Public Interest..not to be confused with The Center for Medicine in the Public Interest found yet another aspect of everyday life that causes cancer.

This time it's soda, or as we call it in Rochester NY: pop.

Add soda to the list of other things CSPI believes are dangerous including prescription drugs, french fries, Chinese food, plastic, food coloring and transfat (the latter was something CSPI pushed to get introduced in the food supply during the 1980s.)

What CSPI does is find things that sound scary, link them to cancer and other ills in RATs. It then raises money through newsletters and lawsuits.

There is no evidence, but for the CSPI study, that any of the above cause cancer or heart disease or anything else.  As one FDA official said about the soda scare study:

"A consumer would have to consume well over a thousand cans of soda a day to reach the doses administered in the studies that have shown links to cancer in rodents," said Doug Karas, an FDA spokesman, in a statement.

http://www.reuters.com/article/2012/03/06/us-us-regulators-dispute-idUSTRE8250W620120306

Meanwhile,  little infants can swallow dozens of magnets and, thankfully, survive:
PORTLAND — A 3-year-old girl was recovering Monday at Legacy Emanuel Hospital after doctors removed 37 'Buckyballs' magnets from her intestines.

Payton Bushnell complained to her parents of symptoms that resembled the flu, Legacy spokeswoman Maegan Vidal told KGW. Then, they took her in to get checked.

Doctors took an X-ray and found the balls, clustered in her stomach. She was expected to fully recover and was listed in good condition Monday morning. She has been in the hospital since Feb. 21.

The Oregon toddler was fortunate. In 2006 the government warned about risks from magnets used in toys after at least one child died and almost 19 were injured. As a result, the Consumer Product Safety Commission recalled almost 4 million Magnetix building sets and magnets were included in holiday warnings about dangerous toys. The risk occurs when a child swallows one or more small magnets, which can link together in the digestive tract and perforate the intestines.


http://today.com

Could it be that swallowing buckeyballs is less toxic than swallowing junk science from CSPI?  

I wouldn't even try that experiment on rats.


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PCORI's 20/20 Night Vision

03/06/2012 08:23 AM |

BioCentury reports:

PCORI board votes on research definition

The Patient Centered Outcomes Research Institute (PCORI) board voted 17-2, with one abstention, to adopt a definition of patient-centered outcomes research that begins with a broad summary followed by a list of characteristics. According to the definition, such research "helps people and their caregivers communicate and make informed health care decisions, allowing their voices to be heard in assessing the value of health care options." The research evaluates preventive, diagnostic, therapeutic, palliative or health delivery system interventions; focuses on outcomes such as survival, function, symptoms and quality of life; incorporates a variety of settings and diversity of participants; and "investigates optimizing outcomes while addressing burden to individuals, resource availability, and other stakeholder perspectives."

The definition does not mention "comparative effectiveness research." PCORI's methodology committee, which drafted the definition, said the research includes many components of comparative effectiveness but is intended to be "broader."

Several board members voiced concerns about the definition's focus on communication and decision-making. NIH Director Francis Collins said the definition does not emphasize research, which he called the "primary function" of patient-centered outcomes research. However, the members also expressed a desire to adopt the definition to allow PCORI to focus on other goals such as developing research priorities and launching an annual research conference. Created by the Patient Protection and Affordable Care Act, PCORI expects to have about $112 million in funding for 2012, of which $90 million will be used for grants.

Dr. Collins’ concern isn’t new. At past meetings, when the discussion of what PCORI’s “legacy should be, the NIH Director said that if the Institute was looking for a unique and non-duplicative research agenda, it would be “a null set.”

PCORI should not forget about outcomes data. Perhaps the institute should lead the way in coordinating the many large data sets of outcomes data held by both Uncle Sam and private payers.  The use of outcomes data needs to go beyond (well beyond) well-intentioned (but relatively small) CMS pilot projects.  The United Kingdom has such a nationwide system – but hasn’t been particularly creative or aggressive in using it.  PCORI should take the lead.  After all, what’s more patient-centered than real world outcomes data?

PCORI should create a program on educating patients, physicians and payers on the use and importance of molecular diagnostics.  After all, what’s more patient-centric than early diagnosis and advancing the “four rights” of the right medicine in the right dose to the right patient at the right time?

PCORI should help to advance the nascent science of adaptive clinical trials. After all, what’s more patient-centric than adaptive clinical trial information?

PCORI should stay as far away as possible from discussions of CER as a method for cost controls – as this is a slippery slope to price controls.  And price controls equal choice controls. Moreover, PCORI is officially tasked not to pursue comparative effectiveness but comparative clinical effectiveness. 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.

(In fact, Francis Collins warned the board to “beware of the tension between CER and personalized medicine.)

PCORI should be careful in creating a databank of CER studies because (and particularly when you consider programs such as CATIE and ALLHAT) garbage in, garbage out.

PCORI should beware of information sharing via academic detailing. (Note:  20% of the PCORI budget is ear-marked to AHRQ for “information dissemination.) ‘Nuff said.

PCORI should (indeed must!) define “patient-centered” as care first and cost second – otherwise the “PC” in its acronym will only mean “politically correct.”

In May 2011, PCORI Chairman Eugene Washington introduced Joe Selby as the institute’s first executive director. To which Dr. Selby commented, “For those of you participating in this meeting via webcast, I’m the one who looks like a deer in the headlights.”

Joe – It might be time for those night vision goggles.

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Ezra Klein Contributes To the Dumbing Down About Healthcare

03/05/2012 03:22 PM |

I shouldn't have to waste my time writing a response to Ezra Klein's latest blog about The High Cost of Medical Procedures In the US compared to other countries.   I won't waste yours by summarizing it either.  You can read it at the risk of wiping out some brain cells...

http://www.washingtonpost.com/wp-srv/special/business/high-cost-of-medical-procedures-in-the-us/

His blog is a textbook case of how most health care policy research is a combination of liberal bias and mythic numbers that support a point of view.

Here's the reason medical procedures cost more in the US.   The cost per procedure does not reflect the government subsidy that artificially reduces prices and drive up demand.    Second, the cost per procedure is not the only thing that's controlled.  Waiting is part of the cost per procedure in Europe, Canada and elsehwere.   Ask someone who has to wait for cancer surgery if the lower cost per procedure is worth it.  Would people pay more to get care faster?  They do quite often.   Yeah, neonatal intensive care in the UK or Canada is cheaper.  So if infusion of biologics.  But there's a reason people come to the US from Canada to get neonatal care.  There's more of it when you need.   You see Ezra,  if price doesn't reflect both the true cost - and value -- of a product, people won't produce it.  Unless you are Solyndra or GM's Chevy Volt.  Both are -- or were heavily subsidized -- products that no one wanted.

Which is sort of like health care around the world.  And medical innovation.  Government controls also skew what will be produced.  Innovation and its diffusion is stifled by things like price controls and comparative effectiveness reviews.  Both raise the risk and cost of development and CER delays and reduces market access.  So the way to make a buck is to pump out marginally better products that will get you a slightly higher launch price. 

The US still leads in the number of new drug launches compared to other nations because we reward biomedical innovation, at least for now.   Meanwhile, medical device adoption is faster in Europe.  Cycle times matter more in medical devices and Europe is not as anal-retentive about follow on devices as is our FDA.  Add a medical device tax to the equation and the decline in innovation will accelerate. 

Klein rehashes the 'research' of Gerard Andersen, a hack who makes a living producing memos for congressional Democrats that confirm their belief that price controls would work just fine.  Andersen pushed having Medicare 'negotiate' drug prices like the Veteran's Administration. ( Here's a link to a Frank Lichtenberg study I sponsored a few years ago.  http://democrats.veterans.house.gov/hearings/Testimony.aspx?TID=59775&Newsid=470&Name=%20Frank%20R.%20Lichtenberg,%20Ph.D. )   And the association between controls on innovation and reduced health care well established.  Lichtenberg has looked at access to new cancer drugs in Europe and Australia and concluded that for most types of cancer, newer drugs save lives and displace most costly care. 

That's the same VA whose formulary and price controls reduce access to new drugs and reduce the life span of seniors.  Which I guess saves money too.  

I don't mean to single out Klein.  It's just that his blog -- a blog! -- is an example of the confirmation bias that passes for policy analysis.    Blogs are thought experiments, not science.  And when they rely upon leftwing ideologues with tenure -- like Gerard Andersen -- to prove a point they reduce the sum of human knowledge each time they are read or written.

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