Today I am a REMS plan

10/18/2010 07:52 PM |

Today I chaired the third annual Risk Management and Drug Safety Summit where I was joined by (among others) CDER Director, Dr. Janet Woodcock and MHRA Chairman, Sir Alasdair Breckenridge.

It was a risk management bar mitzvah in the sense that it felt like REMS was finally coming-of-age – although not yet entirely mature.

Here’s how I kicked off the conference and introduced Janet:

It’s been quite a year on the REMS watch. As Walter O’Malley – the man who moved the Brooklyn Dodgers to Los Angeles once commented, “The future is just one damn thing after another.”

During the course of 2010 there have been both bouquets and brickbats.

Last month the Infectious Diseases Society of America suggested that overuse of antibiotics could be controlled via a REMS-like approach – or even an actual REMS.  If it takes a REMS to drive the safe and appropriate use of antibiotics, then so be it.  Burdensome on docs, yes – but you know the drill.

In July -- Janet Woodcock said “We don't have the kind of standardization and consistency of REMS programs that would be ideal."  Then she announced that the FDA would begin to develop a standard REMS system. Perhaps this will even include a Periodic Table of the Elements to Assure Safe Use.

In June -- John Jenkins, director of the Office of New Drugs -- and the best dressed man at the FDA – admitted that having to develop and then assess the impact of medication guides as part of the REMS program is a burdensome administrative task for the agency.

He said, "We are looking to try and be creative in how we interpret that part of the statute, so stay tuned to see if we're able to find some creative ways around this," he said.

In the meantime, said Dr. Jenkins, "until we work through this further, there are a lot of medication guide-only REMS - a lot of burden on us, a lot of burden on you - that we'd like to try to get out of."

As of June 3^rd, FDA had listed 123 REMS on its website. Eighty-four consist only of a MedGuide, while another 25 involve a medguide and communication plan. The other 14 REMS require sponsors to adopt elements to ensure safe use. Five of those also involve a MedGuide; three also have a communication plan; and the other six also require both a MedGuide and a communication plan.

If FDA does not find a solution to the current MedGuide Malaise, the next reauthorization of the Prescription Drug User Fee Act in 2012 offers an opportunity for legislative change. Industry and other stakeholders already have cited REMS as an area for focus during PDUFA V.

In May -- FDA began designing a five-item grid as a management tool to explain its risk-benefit decisions in a new more concise format.

The grid has five basic factors that need to be addressed. The top two are the seriousness of the condition addressed and the need for a new treatment of the condition. Then comes the traditional heart of the NDA package: analyses of clinical data on the benefits of the drug and the risks associated with its use.

Significantly, the fifth fundamental factor is explicitly the level of risk management associated with the product. FDA is going to take it into consideration in every decision; and sponsors who ignore or underplay the identification of who should use the product and who might use it will have a gap in their filings.

The grid proposal does not call for a fixed mathematical formula behind each approval. Net/Net, the agency has not tried to reduce the role of judgment in approval decisions.

Judgment?  You mean FDA decisions aren’t black and white?  Egad! Someone had better tell Congress.

In the words of John Jenkins, disagreement "happens a lot in the decisions that we have to make. Very few of the decisions that we make on drugs are easy. Very few of the drugs we see have a dramatic overwhelming benefit with relatively no risk. We see that most drugs have marginal to moderate benefits on a population basis and they have general safety but they have the risks of serious toxicities at some low levels." In other words, every decision is "very complex."

Really?

Key take-away is that the FDA is officially moving risk management into the list of key factors affecting new products. And, for better or worse, "judgment" is in the eye of the beholder.

In April -- Biogen Idec announced that it is developing a test that can tell patients their odds of getting PML from Tysabri.

The screening tool could be marketed as early as 2011 if clinical trials involving 9,000 people show a low rate of false findings.

With a false-negative rate of 2 percent, patients would lower their risk of getting the brain disorder PML to 1 in 25,000 for the first three years of their Tysabri therapy. That’s 21^st century risk management.  REMS par excellence.

In March -- Josh Sharfstein suggested, during a House Energy and Commerce Health Subcommittee hearing, that FDA could use more authority to bring negotiations over a drug's Risk Evaluation and Mitigation Strategy to a swifter conclusion.

Josh said, "It's very important for us to work with companies to come up with something that works. There's no question there's a lot we learn from the interchange with companies, but it sometimes can take a long time to come to agreement. Well – that’s a bit of the pot calling the kettle black – but at least it’s an acknowledgement of the problem.

And, of course, there was Avandia.

Let me introduce our first keynote speaker, CDER Director, Dr. Janet Woodcock, by reminding you of what she told this conference last year. Janet said that, “Safety means doing the right things for patients. FDA must consider post-approval issues as part of a drug’s lifecycle.”

Importantly, Janet understands that there’s a real difference between “headlines and help.”  In other words, REMS and other safety mechanisms can be viewed as either “headlines” about “unsafe” drugs or in a more appropriate context of “safe use.”

According to Janet, “FDA does not control the health care system, so our improving the use of marketed drugs, to a great extent, is going to involve influence rather than control.”

“Influence rather than control” is a savvy and sophisticated concept -- one that many of our elected members of Congress could learn from, and one in which REMS plays an important role.

I believe we can also hear the voice of Janet Woodcock in the white paper the agency released a few weeks ago on “Advancing Regulatory Science for the Public Health.

“There is no single discovery — no magic bullet — to address our unique set of modern scientific regulatory challenges. But one thing is clear: if we are to solve the most pressing public health problems we face today, we need new approaches, new collaborations and new ways to take advantage of 21st century technologies. And we need them now.

Ladies and Gentlemen, I am pleased to introduce Dr. Janet Woodcock.

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Chile and The Least Costly Alternative

10/18/2010 10:32 AM |

How would comparative effectiveness be used to justify the Least Costly Alternative in the case of the Chilean miners.

It cost $33 million to rescue 33 Chilean miners.  Each made $12000 a year.   If we use the QALY approach and assume $50000 per QALY it was obviously a waste of Chile's time and money to undertake the successful operation.  Couldn't that money be better spent on disease management programs?  There would be money left over to pay the families of the miners after the mine had been sealed up.  Too bad that Health Dialog didnt have a shared decision making tool to discourage the miners from asking for such an expensive, invasive and untested procedure.   At least Chile should have waited to let AHRQ conduct a CER review before deciding to pay for the rescue. 


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Exchange Rates

10/18/2010 07:07 AM |

When it comes to mandated health insurance exchanges, state officials must strive to ensure that they don’t crowd out free market mechanisms.  Preserving a vibrant private insurance market will maximize choice and enable people to find the insurance plan that best fits their needs.

Healthcare coverage isn’t a one-size-fits-all proposition. 

When President Obama said that people who are happy with their insurance “can keep it,” we should keep him to his word.  Policymakers and private healthcare stakeholders need to work together so that state exchanges don’t become the only way to get health insurance.

Choice is crucial.

See here for a more detailed examination of the slippery slope towards a single payer system.

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What Chile Can Teach Us About Obamacare

10/17/2010 09:27 PM |

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What does Pharma want?

10/15/2010 08:04 AM |

During a past episode of Mad Men, the creative team at Sterling/Cooper is hard at work ideating on a “women’s product” campaign when someone asks, “What do women want?”

Strolling by, Roger Sterling quips, “Who cares!”

Well, when it comes to social media, what does pharma want -- and who cares?

Many will say “regulation from the FDA -- in fact, a great many.  But is that really what pharma wants?

Yesterday I participated in a small roundtable (sponsored by AstraZeneca) on “Examining the Roles of the FDA and the Pharmaceutical Industry in Social Media.” 

(Full disclosure: I ate two small eggplant and tomato tea sandwiches and drank 2.5 cups of organic coffee. I did not offer to reimburse AZ for the “gift.”)

What does pharma want? One of the opening comments was that pharma wants the “ability to engage” in social media.  My response to that was to ask whether pharma has the “will” to engage – because they certainly have the ability if they choose to use it.  And where there’s a will, there’s a way.

Another issue that came up early – and that generated a lot of conversation – was the need to bifurcate the discussion of digital advertising from that of social media. There are rules for digital advertising, paid digital advertising. Social media, on the other hand, is the New Frontier.  It’s the crucial gray zone that exists between regulated speech and user-generated content.  It’s where the rubber meets the road.

What pharma wants (or should want) is specific areas of clarification from the FDA on this new and exciting zone of opportunity.

What of the empowered digital healthcare consumer that we hear so much about?  Well – there were a few of them in attendance at the AZ confab and they had some interesting things to say.

What struck a chord for me was when one of the civilians in the room (by which I mean a patient) said that she really had no idea why pharmaceutical companies chose to absent themselves from disease-related social media conversations.  She assumed it was because Big Pharma is afraid of mixing it up with real people in real time dialogue.

And she’s right, of course – but for reasons she didn’t suspect. The ensuing explanations of adverse event reporting and other compliance-related issues didn’t cause her to nod her head, but rather to say (indeed, almost insist) that “pharma should explain to people why they’re not there.”

Blame the FDA! was the knee-jerk reaction.  But that’s not fair and it’s not true.  How can the agency be blamed for industry’s reluctance to push the boundaries – even a little?   Fear of warning letters?  Fear of unearthing adverse events? I say, where there’s a will, there’s a way. If you won’t blaze the path – even a little -- then don’t expect anyone to know where you want to go.

Unfortunately, blazing new territory through real-time learning is not, shall we say, historically a tradition of the pharmaceutical industry.  Everyone wants to do new and exciting things – second.

Here’s an even more basic question – what’s the right thing to do?  I submit that it’s irresponsible to actively avoid participating in the social media healthcare conversation.  It is, to directly quote CDER Director Dr. Janet Woodcock, “where the people are."  Healthcare begins at search.

 

But, someone pushed back, that’s why we need more directive regulation from the FDA.  I fundamentally disagree (1) that’s what’s needed and (2) that’s what’s coming.  Let me explain.

(1)  IMHO, “We need more regulation” just doesn’t cut it. Since there is no direct “ask” from industry, it’s impossible to expect the FDA to offer direct guidance. It’s not like requesting guidance for DTC advertisements.  That was a precise request for a tangible deliverable that resulted in direct and specific rules and regulation. More regulation?  Be careful, that may be precisely what you get.  Also, “more” guidance means nothing without a more precise reference.  “More” relative to what aspects of social media?  These details were lacking at last November’s Part 15 hearing and (alas) equally so in the lengthier (but equally non-specific) docket submissions.

 

(2)  What are the odds, lacking direction, expertise and experience, that DDMAC will deliver some kind of deus ex machina solution?  Expecting the Holy Grail will only lead to disappointment and frustration.  And blaming the FDA when that happens won’t make anything better or move the social media agenda any further ahead.  If industry is expecting to climb the steps of White Oak on its knees, kiss an FDA relic and miraculously throw away the crutches hobbling their ability to participate in social media, well, there had better be a Plan B.

 

Where there’s a will there’s a way.

 

Then there’s the question of language and syntax.  For example, what does “sponsored” mean? Let’s do a brief thought experiment.  Consider a televised PGA tour event.  When a product logo for an erectile dysfunction medicine appears on the screen and the announcer intones, “This portion of the Masters is sponsored by DRUG NAME HERE,” nobody out there in the viewing audience takes that to mean the “sponsor” has chosen the speed of the greens, the height of the rough, or the pairing of golfers in the tournament.  But say “sponsored” on a social media site and watch the sparks fly at internal regulatory review. Fore! This also leads to the still vague regulatory distinction between property owner and property user – an issue in dire need of FDA clarification.  Discussion of this important social media issue in FDA docket submissions?  Try and find it.

 

Of course, there’s the subtle but crucial differentiation between “permissible” and “appropriate.”  And this returns us to where we started.  What does pharma want?  Do they want social media, primarily, as a new channel for marketing or do they see it as a new and exciting and robust and dynamic mechanism for advancing the public health through real-time interactive communications?

Indeed – why not both?  Where there’s a will, there’s a way.

 

And AZ – thanks for the sandwiches and kudos for a job well done.

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A new wrinkle

10/14/2010 05:02 PM |

The FDA has approved a clinical trial of Botox as a treatment for vaginal spasms that can block sexual intercourse and gynecological examinations.

 

clinicaltrials.gov also lists a similar study that already is under way at the University of Tehran in Iran.

 

The full article can be found here.

I leave the rest to your imagination.

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Advise and Dissent

10/13/2010 08:51 AM |

As Harry Truman opined, “I have found the best way to give advice to your children is to find out what they want and then advise them to do it.”

If only it were that easy with FDA advisory committies.

Matt Herper (Forbes) reports that an analysis of FDA advisory committee recommendations compared to actual FDA actions (from 2007 through 2010) shows that FDA followed adcomm advice 74% of the time. The study, by Concept Capital, looked at a total of 120 product-specific advisory committee votes and the ensuing FDA actions.

Interestingly, the FDA overruled “no” votes only three times: (Tarceva for maintenance therapy in lung cancer, Avastin for breast cancer, and Micardis to lower blood pressure.)

As Herper writes, “In other words, a no vote from an advisory panel is likely to stick, but a yes vote does not mean the product will be approved.”

“Advice is judged by results, not by intentions.”

                            -- Cicero

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Is The Critical Path in Critical Condition?

10/12/2010 04:08 PM |

An article in RPM,  Regulatory Science: A Biodefense Backlash? by Michael McCaughan    tinyurl.com/2byhfxr suggests that the effort to remake product development around 21st century science is stalling or even on life support:

"Securing funding for "Regulatory Science" has been a top priority for Commissioner Margaret Hamburg, highlighted most recently during an October 6 luncheon address to the National Press Club, coinciding with a white paper outlining the agency’s vision for enhancing its science base.

The initiative may be the last hope for the agency to play a central role in encouraging drug development as a core part of its mission, after the underwhelming results from prior efforts like the Critical Path Initiative and the Reagan/Udall Foundation. For industry, "Regulatory Science" funding may be the only hope to secure a pool of resources for the agency to craft policy in areas like companion diagnostics, surrogate markers, etc. without another significant step-up in user fees."

The problem is what money that has been spent is being steered to academic institutions that are not collaborating with either other, with other industry consortium or things on the Critical Path opportunities list.  Further,  lip service is being paid to adoption of new tools and pathways even as the FDA puts out guidances that ignore or fail to encourage their use. The muddled 510k guidance discussion and recent FDA guidance on submission of Phase I safety data are examples of guilt by omission. 

I find myself agreeing with Senator Tom Harkin who expressed concerned about the FDA's increasing set of duties and inability to establish common protocols to speed development of anti-infectives and vaccines within the bio-defense sphere:

Stressing that he was “just thinking out loud,” Harkin suggested that maybe “we need to take something out of FDA, something out of Defense, that would be put under BARDA, and let BARDA be the lead agency.” (Harkin previously noted the “hard work” he and others in Congress put into creating BARDA, and wondered how the new HHS initiative fit within that framework.)

Harkin noted his ongoing frustration with the failure to achieve licensure of cell-based flu vaccines. “FDA just—institutionally, I don’t know if they can do it,” he said. “It is just that they have so much to do and they have other responsibilities, and mostly they are focused on drugs that we take for illnesses.”

That's quite a statement. Unfortunately the organized interests in Washington are more interested in rent-seeking than getting things done. 

Just thinking out loud...maybe everyone needs to redirect efforts towards the development of collaboration to produce the science and regulatory culture required to sustain advances in clinical development and the goal of increasingly effective use of products once on the market.  That's what the Critical Path is and was about.  Discussions about what can be done with fees collected under The Prescription Drug User Fee Act are starting.  

www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/default.htm

That might be a good time and place to introduce changes in priority and purpose. 

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Britannia Rules

10/12/2010 03:48 PM |

I beat up the NHS often enough so it's only cricket that I point to something transformational that it is undertaking, system wide cancer gene testing that will guide treatment selection and use.  This is something NICE Chair Sir Michael Rawlins has suggested is critical to real health reform anywhere.. 

www.nature.com/news/2010/101012/full/467766a.htm

Cancer-gene testing ramps up
Thousands to get personalized medicine in Britain's National Health Service.

Ewen Callaway

In an approach that many doctors and scientists hope will form the medical care of the future, Massachusetts General Hospital in Boston has for the past year and a half been offering people with cancer a novel diagnostic test. Instead of assessing tumours for a single mutation that will indicate whether a drug is likely to work or not, the hospital tests patients for some 150 mutations in more than a dozen cancer-causing genes, with the results being used to guide novel treatments, clinical trials and basic research. This form of personalized medicine tailors treatments on the basis of the molecular and genetic characteristics of a patient's cancer cells, potentially improving the treatment's outcome.

Now Britain is set to test whether an entire health-care system is ready for the approach. Plans were unveiled this week to deploy broad genetic testing for selected cancer patients in Britain's government-run health-care provider, the National Health Service (NHS). This form of 'stratified medicine' uses genetic information to group patients according to their likely response to a particular treatment.

"The United Kingdom is really the ideal place to do this," says James Peach, who heads the programme for Cancer Research UK, the charity that is leading the effort. As the NHS treats millions of people each year, unprecedented numbers of suitable patients could be enrolled in the genetic-profiling programme. "The idea is to scale this up to every patient in the NHS," says Peach. In its first phase, the programme will be rolled out to as many as 12,000 NHS cancer patients over two years, beginning in early 2011. By contrast, Massachusetts General has tested about 1,600 patients, and other hospitals' efforts each number in the hundreds.

The tests, which will look for several dozen mutations in about a dozen genes linked to cancer, will be carried out on people with lung, breast, colorectal, prostate or ovarian cancers, or metastatic melanoma, who are being treated at six NHS hospitals. Therapies that target specific tumour-causing mutations have already been approved, or are on the verge of approval, for most of these conditions, says Peach.

Testing a clinical sample for so many mutations at once is a challenge in itself. Because most existing clinical tests probe individual genes, the NHS programme is working with the Technology Strategy Board, a government agency that supports technology development, and several companies to design a customized test that detects all of these mutations in one go. The partnership, which includes the pharmaceutical multinationals Pfizer and AstraZeneca, will also design software to make the results useful to researchers and clinicians. By genotyping patients for a broad array of cancer-causing mutations, the new tests will make it easier to assign subjects to clinical trials, Peach says.

That is already happening at Massachusetts General, where the test is helping to establish clinical trials that wouldn't otherwise have happened, says Leif Ellisen, a geneticist who helps lead the hospital's cancer testing programme. For example, its broad genetic test detects a mutation in a gene called BRAF that is already known to be commonly mutated in metastatic melanoma. Finding such mutations in people with lung and colon cancer made it possible to put them in a trial of an experimental treatment targeting that gene, Ellisen explains.

Basic research should also benefit from the NHS programme, says Peach. Researchers will have access to consenting patients' genetic data as well as to medical records of the outcomes of the treatment. These data could reveal how drugs targeting one molecular pathway are affected by mutations in another gene, says Andy Futreal, a cancer geneticist at the Wellcome Trust Sanger Institute in Hinxton, UK, and an adviser to the programme.

Peach hopes that the first phase of the cancer programme will pave the way for expanding genetic testing to more patients and other conditions, such as diabetes, AIDS and even psychiatric disorders. Cancer offers a good testing ground for personalized medicine, because numerous targeted therapies already exist, but "there's no reason why this should be restricted to cancer", says Peach.

Fabrice André, who runs a similar cancer diagnostic programme that has so far been offered to about 100 patients at the Gustave Roussy Institute in Villejuif, France, says the NHS programme could point the way to implementing personalized medicine across an entire population. "It can really change the landscape of how molecular testing is being done for cancer," he says. "If they succeed, then it's going to be a major step forward."


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Up Close and Personnel

10/12/2010 07:53 AM |

The Office of Personnel Management (OPM) is planning a new database that will store health care claims information from three federal programs - the Federal Employees Health Benefits Program, the National Pre-Existing Condition Insurance Program and the forthcoming Multi-State Option Plan.

 

OPM (in a 10/5 Federal Register notice, says the database will allow OPM to "actively manage all three programs to ensure the best value for the enrollees and taxpayers." The database will be effective Nov. 15 "unless comments are received that would result in a contrary determination."

 

OPM?  Healthcare “value?”  This raises two issues:  (1) expertise and (2) mission creep. Are people being hired to do this?  Who are they? Who’s choosing them? Transparency is required – if not a Congressional hearing.

 

Information collected will include personal identifying information, address, dependent information, employment information, health care provider details including debarred provider information, health care coverage information, health care diagnosis information and provider changes and reimbursement on the aforementioned coverage, procedures and diagnosis.

 

OPM?  Really?  Sounds like a CMS program – or even AHRQ.  Also, will this data be shared with other federal agencies?  And if so, to what end. 

 

Per the FR notice, "the data will be de-identified for specific analysis that provide flexible queries of the data set for general demographic queries, risk-adjusted profiles, and comparison of chronically ill patients and other useful analytics; and engage in econometric modeling of, among other things, health trends, risk adjustment methodologies, pharmacy pricing and negotiation."

 

Hm – “econometric modeling?”  That sounds menacing.

 

Yes – definitely Congressional hearing material.

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Health Affairs Skews Survival Statistics To Support Obamacare

10/11/2010 02:56 PM |

Compared to other developed nations, United States has better survival and life expectancy rates for cancer, heart disease and stroke according to analysis of validated registry and clinical data.   When absolute differences in smoking are controlled for, life expectancy after age 50 in the US is better still.  The critical difference: Americans are receiving more effective treatments more often.

So if you want to claim just the opposite: that spending on health care is not related to life expectancy or want to show that the US healthcare system needs to control costs by allowing the government to "coordinate care?" 

You do what Sherry Glied and Peter Muening did in their widely publicized article in Health Affairs, "What Changes In Survival Rates Tell Us About US Health Care".   You come up with a 15 year survival rate which understates the effect of treatment since most studies track 5 year survival rates after diagnosis and treatment and overstate behavioral issues.   Then you simply assert -- because there is more spending in the US than Europe -- that the difference is the result of our lousy, inefficient system of care. 

content.healthaffairs.org/cgi/content/full/hlthaff.2010.0073v1#SEC1

How do the authors get away from making the exact obvious conclusion that the rest of the epidemiological literature has shown?  One reason is that Health Affairs is carrying a torch for Obamacare and whatever peer review took place was passive or unconcsious.  Another reason:  such statistical skewing is easy to get away with because most in the media simply report the conclusion without looking at the methods.

The authors state: "We measured fifteen-year survival rather than life expectancy because the latter can be biased by the survival experiences of a small number of elderly people, among whom coding errors are common. Focusing on survival also allowed us to distinguish between the experiences of specific cohorts. We explored fifteen-year survival for men and women separately because risk-factor profiles differ greatly by sex and country.

By looking at overall survival after 15 years the authors can go back to a time when medical innovations essential to survival by disease were non-existent but detection in the US was more prevalent   The effect of higher levels of detection -- in the absence of innovations -- are what appears to be lower rates of survival.   At the same time they ignore mortality rates because the US had a faster decline in mortality from major diseases (many of which Glied and Muenning ignore) than in other countries. 

This hatchet job has yet to be questioned by anyone in the media.  If anyone is interested they can compare the Health Affairs j'accuse with other studies that are less biased. In particular, look at Low Life Expectancy in the United States: Is the Health Care System at
Fault? by Samuel H. Preston and Jessica Y. Hoy of UPenn.


repository.upenn.edu/cgi/viewcontent.cgi


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Death Ray

10/11/2010 08:29 AM |

Been warning about this from the very beginning, and now it’s starting in earnest:  Cost-centric strategies leaving patient-focused medicine in the dust.

Last week the Medicare Payment Advisory Commission met to consider recommendations that empower Medicare to reinstate the option to base Part B drug reimbursement on the least costly alternative (LCA) among products.

Note please, that “least costly” in no way means “best for the patient.”

On October 7^th, MedPAC heard two proposals outlined by commission staffer Nancy Ray.

The first was that Congress should give CMS authority to apply least costly alternative policies in setting payments for items and services covered under Medicare Parts A and B, and CMS should periodically assess the clinical similarity of Medicare-covered services and apply LCA policies for those services deemed clinically similar.

The second was that Congress should direct CMS to set the payment rate for a newly covered service that lacks evidence demonstrating better outcomes than existing treatment options at a level that is no higher than the LCA.

The policy could end up relying heavily on data from comparative effectiveness research conducted under the auspices of the Patient-Centered Outcomes Research Institute, which is being created under the Affordable Care Act.

A provision of the ACA states that HHS cannot deny coverage of items based solely on the results of comparative clinical effectiveness research, presenting a possible gray area should LCA determinations favor one product over another.

Ray predicted that both of the LCA recommendations would decrease spending relative to current law and also would lower beneficiary cost sharing in the short term and Medicare premiums in the long term.

While the commission did not vote on either recommendation, comments from members were generally supportive. Surprised?

If you needed another reason to understand why the upcoming elections are so crucial to 21^st century patient care – you’re welcome.

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Obama Repeals Obamacare

10/08/2010 02:34 PM |

By granting waivers to avoid forcing nearly 3 million people to lose coverage, the Obama administration has started towards the repeal of Obamacare. Note that this is only a waiver to increase annual minimum coverage levels for mostly part time, young and temporary workers.  The next tsunami to hit will be when plans inform HHS it will drop coverage because of the mandate to spend up to 85 percent of medical premiums on what Obama defines as quality care.    If the administration continues down this road it can take credit for "creating or saving" coverage for 20 million Americans...

You can see the first evidence of Obamacare repeal by Obamacare here:

www.hhs.gov/ociio/regulations/patient/appapps.html
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510 Kay doesn't play in the WNBA

10/08/2010 10:33 AM |

Who "lost" medical technology? 

At an FDA-sponsored public meeting in Irvine, California, CDRH Director Jeff Shuren shared that new regulations for approving medical devices may include a new category for medium-risk products.  Products in this category would require more studies on safety to win agency clearance. According to Shuren, changes will be phased in and that changing the rules may not result in more studies being required of device makers initially. “We are still addressing that on a case-by-case basis where it’s appropriate and necessary, he said.

 

Is more data always better?  Sometimes.  But how does this weigh against the growing “device gap” between the US and the EU?  It seems that what’s old is new again – and not necessarily for the better.

 

When the agency determines it needs more data for an approval or that a device application should fall under a new designation, consideration must also be given to what’s going on “over there.”  We cannot and must not create a regulatory environment for medical technology (or, for that matter, pharmaceuticals) that exists in isolation from the rest of the world.  Harmonization mustn’t mean “do it our way,’ but rather, “let’s talk.”

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Expletive Deleted

10/07/2010 08:31 AM |

The FDA's new framework for "Advancing Regulatory Science for Public Health" has it's heart in the right place.

It even discusses the need for collaboration:

Support for mission-critical applied research, both at FDA and collaboratively

Support within the FDA is critical to expanding the field of regulatory science. An active research program, directly connected to the FDA review process, will not only bring needed advances in regulatory science straight to FDA review, product development, and evaluation but will also add value to guidance and policy development.

In addition, the discipline of regulatory science must be developed though support from both partnerships and external research and collaboration. There are substantial opportunities to enhance and expand current FDA programs and to develop new ones that support effective, more robust, external and collaborative efforts to advance regulatory science. Some projects are already under way:

• A Joint Leadership Council recently created by FDA and NIH to promote the expansion of regulatory science through enhanced scientific collaboration and jointly supported and administered extramural research grants in regulatory science.

• Creation and support of academic Centers of Excellence in Regulatory Science to carry out applied regulatory science research both independently and in collaboration with the FDA and as a locus for scientific exchange and training opportunities for both FDA and academic scientists

• Enhanced strategic collaboration and coordination with other governmental agencies to develop new programs to advance regulatory science and innovation

• Enhanced support and focus for the Critical Path Initiative to catalyze and enable partnerships and consortia that advance regulatory science and public health through innovation and modernization of the medical product development and evaluation process

• Partnership with the Reagan-Udall Foundation on projects in support of regulatory science

Good words.  But two important words are missing ... "with industry."

Obviously, the one-line reference to the Reagan/Udall Foundation means "with industry" and it's possible that these words which must not be spoken also reside within "partnerships and consortia."  But why the strange silence about such a valuable partner?  Advancing regulatory science without regular and robust collaboration from the regulated?  Not likely.

Fortunately, Peggy Hamburg often speaks about working with industry on Critical Path issues.  Enquiring minds want to know if any references to "industry" were blue-penciled out prior to publication -- and if so, by whom.

The white paper intelligently comments that:

There is no single discovery — no magic bullet — to address our unique set of modern scientific regulatory challenges. But one thing is clear: if we are to solve the most pressing public health problems we face today, we need new approaches, new collaborations and new ways to take advantage
of 21st century technologies. And we need them now.

All said, this paper is a step in the right direction and people who work in (expletive deleted) should read it.
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A (Minor) League of Their Own

10/06/2010 09:30 AM |

There’s no crying in healthcare reform.

In today’s New York Times, David Leonhardt (discussing the Presidential rhetoric used to assuage citizen uncertainty) writes, “Mr. Obama went so far as to suggest there would be no disruptions, saying that people could keep their current plan if they liked it. But that’s not quite right. It is not possible to change a system as huge, and as hugely flawed, as ours without some disruptions.”

 Now that the legislation has passed, it seems as though the new talking point is, “It’s gonna get worse before it gets better.”  And whether or not it’s going to get better is still very much theory (at best) and hoping (at worst).

Remember all that money that healthcare reform was going to save us?  Well, since a swiftly deprogrammed Peter Orszag put that shibboleth to bed (again, in the pages of the New York Times), the debate seems to be about how insurance is going to be made more affordable.  One way we’re supposed to achieve this, according to Leonhardt, is that “people will be required to buy insurance, to spread costs among the sick and the healthy.”

Sure – except that this mandate (if it doesn’t turn out to be unconstitutional) (1) doesn’t even kick in until 2014 (the same time the theoretical state exchanges come in to play) and (2) will likely penalize offenders less than even a low-cost health insurance premium.  Oops.

Specific to the state exchanges that are the foundation of the theory, Leonhardt writes, “the new markets for health insurance, known as exchanges, won’t be up and running until 2014. This timetable has its problems, and the Obama administration will probably need to grant some more temporary exemptions.”

Call me when the revolution starts.

Not to say that it’s at all about politics!  No penalties until 2014?  If we’re in such dire straits – why not, say, right now.  “It’s coming and it’s going to be great,” as a rallying cry is, plainly speaking, tanking since the reform plan is already leaking like a sieve. And it’s not going to cut it before the November elections. The truth is that it’s likely to get worse.  Whether or not it gets “better” (whatever that means) depends largely on “fixes” that will necessarily be made in the next Congress.

Just what we need, healthcare sausage.

 All this to say that we’d better all stop crying and start trying to think about what needs to be done beyond the talking points.  After all, if it was easy, anyone could do it.

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Payback?

10/04/2010 12:27 PM |

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The Peter (Orzsag) Principle

10/04/2010 12:11 PM |

Both the left and the right hew to narratives about healthcare that comport with their worldview rather than mechanism-based analysis of treatment outcomes or evaluation of the impact of technological innovation on such interventions.  Both make the assumption, badly mistaken, that quality reduces cost.   Both base this mistaken assumption on the flawed Dartmouth Study that has peddled the falsehood that less care is actually better.  

Peter Orszag was the peddler in chief of this notion, stating that the Dartmouth research demonstrates that at least $700 billion in health care spending is wasted each year.   CMS commissar Berwick, with usual hyperbole, says that Dartmouth research shows that HALF of all health care could be eliminated and that doing do would improve outcomes. 

Such statements are made in the face of research that both cite.  And when they encounter a finding that conflicts with their narrative, they simply ignore it, even when citing the literature.

The most recent example is Orszag's oped in the NY Times today (Health Care’s Lost Weekend) tinyurl.com/27sfn7v

"The Dartmouth data also indicate that the N.Y.U. patients received no clear benefit for the higher cost. They saw, on average, more than 14 different doctors, compared with fewer than 10 for patients at the most efficient hospitals. But the extra visits did not seem to produce better outcomes. In fact, seeing more doctors may have caused harm, as patients ran the risk of side effects and complications from additional tests, treatments and medicines."

But Orszag cites an article in the NEJM that proves the opposite, en route to making his point. 

First, weekends. It’s never good to be hospitalized, but you really don’t want to be hospitalized on a weekend. There are fewer doctors around, and people admitted on Saturdays and Sundays fare relatively poorly.

One study in 2007 found, for example, that for every 1,000 patients suffering heart attacks who were admitted to a hospital on a weekend, there were 9 to 10 more deaths than in a comparable group of patients admitted on a weekday. The weekend patients were less likely to quickly receive the invasive procedures they needed — like coronary artery bypass grafts or cardiac catheterization.

Except that the study concludes the more care leads to better outome not that more people receiving care quickly:

Overall, mortality 30 days after admission was significantly higher for patients admitted on weekends than for those admitted on weekdays...Patients admitted on weekends were less likely to undergo invasive cardiac procedures than were those admitted on weekdays....the worse outcome of weekend admissions may be due in part to a lower rate of invasive intervention.

www.nejm.org/doi/full/10.1056/NEJMoa063355#t=articleTop

This article is consistent with other studies , including those by PCORI governor Dr. Harlan Krumholz, showing that the rate of invasive intervention, once severity of illness is adjusted for, saves lives and improved outcomes

The key variable shaping re-admissions:  intensity of interventional care.   The shorter the stay and the fewer the procedures, the more readmissions there were.  

jama.ama-assn.org/cgi/content/short/303/21/2141

When it comes to invoking quality, both the right and the left should be more circumspect about clalming it can save money.   And Orszag, who perpetuated this myth, ought to lead the charge.

 
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The Rational Pessimist

10/01/2010 06:44 PM |

I couldn't sleep on the way back from Israel.  So I read Matt Ridley's new book, The Rational Optimist.  www.rationaloptimist.com/   Ridley's thesis is that innovation -- broadly defined as technological advances that reduce the time required to develop goods and services that enrich life --  is the process of human evolution and uniquely so.  The huge gains in life expectancy, declines in infant mortality, growth in income are the product of innovation and the rational optimist bases his or her hope for a better world on these advances, not faith.  To him, innovation is always worth the time and investment precisely because the decisions and actions leading to specific innovations reflect a collective exchange of ideas, interests and needs across continents, civilizations and time.  By contrast, Ridley derides the efforts of government to substitute it's "wisdom' in favor of this collective and iterative process.

But one also has reasons to be a rational pessimist about innovation in large part because our society is hamstrung by free lance regulators who are able to use the government to impose the precautionary principle or their own biases on the public as a whole.  We have become a nation of tattle-tales, whiners and instant experts who confuse a foot-noted declarative statement with wisdom. 

Take this most recent example:

"Impressionable utes weren't the only ones watching when Cincinnati Reds owner Bob Castellini (right) passed out victory cigars and then lit up after his team clinched the NL Central title at Great American Ball Park on Tuesday.

Also watching at home on television were at least five whistle-blowers who noted that the Reds were violating Ohio's indoor-smoking ban. They called Cincinnati's health department to report the team and now the Cincinnati Enquirer reports the club is under investigation. 

In case you're wondering, the answer is yes — I'm extremely sorry to report that there are people living among us who would actually do this."

Me too.   Here is a list of the people and organizations among us who are now enriching themselves or making themsevles famous by using government to impose their views on the rest of us, crippling innovation in the process.

1.   The anti-Avandia axis of Gardiner Harris, Steve Nissen and David Graham who have use innuendo and meta-analysis to drive Avandia into near extinction.  Perhaps the FDA's decision to keep the drug on the market can be considered a victory of sort, but as someone said, another victory like this and we are done for.

2.  The comparative effectivness crowd that will be telling us how much a life or product is really worth.   Imagine if the same experts who now raise questions about whether a cancer drug is worth it because, on average, it adds a few months of life and costs $100k a year made the same determination about HIV drugs in 1987.  In today's dollars AZT would cost $21000 a year and based on initial studes added on average about 6 months of life to patients who still suffered horribly.  Today of course the cost of AZT is much lower and as Tom Philipson and co-authors found "ex-post value of hope associated with treatments for HIV patients to be as much as four times as high as standard per-capita estimates of treatment effects and as many as two and a half times as high as aggregate values across all cohorts."  (Meaning across time and society.)

www.nber.org/papers/w15649.pdf

3.  The board of governors of the so-called patient centered outcomes research institute which has as a member a chiropractor who lead the American Chiropractor Association and who believes that immunization should be "voluntary."  

4.  Those at the NIH and FDA who have hijacked The Critical Path to fund basic research that has nothing to do with creating tools that contribute to accelerating product development.   You see, 'regulatory science' now means anything that could be used down the road in the development process.   Basic research is much sexier and easier to get money for and avoid being attacked as providing aid and comfort to industy.

Lots of other countries provide generous R and D tax credits.  They provide struggling companies with cash flow in the short term.  But they are no substitute for a culture that embraces innovation.  And start ups rarely become engines of commercialization and innovation.  

So I am pessimistic because unlike Ridley so many people take innovation for granted and confuse.  Worse, unlike Ridley, as AEI's Alex Pollock writes:
"there is a danger with intellectual brightness. It is to overemphasize and develop a bias for cleverness, quickness, facility with data, and the ability to persuade."   There is a danger we all face in underestimating the pernicious influence ideas have when armed with the power of the media or government.  

Pollock adds:  Max Planck, the great physicist, made this provocative observation: “A new scientific truth does not triumph by convincing its opponents and making them see the light, but rather because its opponents eventually die, and a new generation grows up that is familiar with it.”

www.american.com/archive/2010/september/it2019s-easier-to-be-brilliant-than-right

Will we waste a generation ignoring the constants that enrich civilization in favor of shorr term policies, sound bits and cleverness with wisdom?

 

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UN phone home

09/29/2010 06:53 PM |

Between the Jewish holidays, traveling to Israel (where I am now) and the Yankees stinking up September it has been hard to focus on healthcare. Or at least blog about it.

Not that I haven't been reflecting about it from time to time..getting aggravated is more like it.

So I was about to write a blistering blog about how the media continually tries to make policy according to a narrative and if the facts aren't there to fit the narrative, well you can always make more.

And then I saw this:

UN 'to appoint space ambassador to greet alien visitors'

A space ambassador could be appointed by the United Nations to act as the first point of contact for aliens trying to communicate with Earth.

Mazlan Othman, a Malaysian astrophysicist, is set to be tasked with co-ordinating humanity’s response if and when extraterrestrials make contact.

Aliens who landed on earth and asked: “Take me to your leader” would be directed to Mrs Othman.


(Could we at least see a resume before making that decision? Coordinating humanity's response? Maybe she and the UN should start with something more pedestrian, like paying it's bills on time. )


She will set out the details of her proposed new role at a Royal Society conference in Buckinghamshire next week.

The 58-year-old is expected to tell delegates that the proposal has been prompted by the recent discovery of hundreds of planets orbiting other starts, which is thought to make the discovery of extraterrestrial life more probable than ever before.

(Let me get this straight: there is an actually a group of scientists who will sit and listen to her? Without laughing? )

Mrs Othman is currently head of the UN’s little known Office for Outer Space Affairs (Unoosa).

(Yes, I am Earth's ambassador to the United Federation of Planets, a little know office in the State Department.)

In a recent talk to fellow scientists, she said: “The continued search for extraterrestrial communication, by several entities, sustains the hope that some day human kind will received signals from extraterrestrials.

“When we do, we should have in place a coordinated response that takes into account all the sensitivities related to the subject. The UN is a ready-made mechanism for such coordination.”

(Of course it is. And you thought UNICEF was the only trick or treat program the UN sponsored.)

Professor Richard Crowther, an expert in space law at the UK space agency who leads delegations to the UN, said: “Othman is absolutely the nearest thing we have to a ‘take me to your leader’ person”.

(Wait a minute. Shouldn't we have a vote? Can't every country nominate one TMTYL type person? I would nominate William Shatner...because he's got the intergalactic experience. Or David Hasselhoff because he can do rehab and host America' Got Talent.)

The plan to make Unoosa the co-ordinating body for dealing with alien encounters will be debated by UN scientific advisory committees and should eventually reach the body’s general assembly.

(when it takes a break from bashing Israel)

Opinion is divided about how future extraterrestrial visitors should be greeted. Under the Outer Space Treaty on 1967, which Unoosa oversees, UN members agreed to protect Earth against contamination by alien species by “sterilising” them.

Mrs Othman is understood to support a more tolerant approach.

(I wonder what that might be: diversity training, a path to citizenship for extraterrestrial aliens ahead of the 12 million illegal aliens, family planning?)

Thank goodness the UN is taking on a challenge it can likely meet and on par with the level of seriousness that organization deserves.


http://www.telegraph.co.uk/science/space/8025832/UN-to-appoint-space-ambassador-to-greet-alien-visitors.html


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