Latest Drugwonks' Blog
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.”
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.
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.
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.
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?
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
The National Institutes of Health and the Food & Drug Administration dole out $9.4 million in grants as part of a three-year program to promote "regulatory science"
The National Institutes of Health and the Food & Drug Administration will dole out $9.4 million over three years to support a quartet of research projects in so-called "regulatory science."
The program is aimed at improving data for scientists and regulatory reviewers on medical device safety and at improving the "evaluation and availability of new medical products to the community," according to a press release.
The FDA will put about $950,000 toward the grants, according to the release. It's part of a joint effort announced early this year designed to speed innovative medical technologies to market. When the cooperative effort was announced, the agencies said their total kitty was about $6.75 million over three years.
Regulatory science is the "development and use of new tools, standards and approaches to more efficiently develop products and to more effectively evaluate product safety, efficacy and quality," according to the February announcement of the program.
Here are the details on the recipients of the four grants:
- Dr. Donald Ingber of Harvard University Medical School in Boston — "Heart-Lung Micromachine for Safety and Efficacy Testing"
- Dr. William Barsan, Donald Berry and Roger Lewis of the University of Michigan in Ann Arbor — "Accelerating Drug and Device Evaluation through Innovative Clinical Trial Design"
- Daniel Cerven and George DeGeorge of MB Research Laboratories Inc. in Spinnerstown, Pa. — "Replacement Ocular Battery (ROBatt)"
- Dennis Hourcade of Washington University in St. Louis — "Characterization/Bioinformatics-modeling of Nanoparticle: Complement Interactions"
"These four projects were chosen among many applications because they were the most meritorious proposals for addressing high-priority areas in cutting-edge biomedical research and regulatory science. This partnership marks the beginning of our work with FDA to use new scientific and technological tools to aid/enhance the review of new drugs and devices. It is one facet of our shared commitment to speed the delivery of new medical products to patients," NIH director Dr. Francis Collins said in prepared remarks.
"These projects show the potential breadth of opportunity that comes from advancing regulatory science. The results are likely to have broad application to researchers across scientific disciplines and will result in better-informed regulatory decision-making and faster drug development and approval processes," added FDA commissioner Dr. Margaret Hamburg.
During the initial debate on healthcare reform, those who spoke about “death panels” were called fear mongers. And, to a large degree that’s what they were trying to accomplish. But the issue is a real one. We spend a disproportionate share of our on what is loosely termed “end of life care.” And it’s an issue we have to address. Now. Unless we are willing to surrender to Uncle Sam, MD and the $50,000 QALY and adopt a lowest common denominator of “care we can afford,” we’d better place this issue near the top of our national healthcare conversation.
And so, kudos to Marilynn Marchione of the Associated Press, who asks the question nobody wants to address … what’s a life worth?
BOSTON — Cancer patients, brace yourselves. Many new drug treatments cost nearly $100,000 a year, sparking fresh debate about how much a few months more of life is worth.
The latest is Provenge, a first-of-a-kind therapy approved in April. It costs $93,000 and adds four months' survival, on average, for men with incurable prostate tumors.
For the last decade, new cancer-fighting drugs have been topping $5,000 a month. Only a few of these keep cancer in remission so long that they are, in effect, cures. For most people, the drugs may buy a few months or years. Insurers usually pay if Medicare pays. But some people have lifetime caps and more people are uninsured because of job layoffs in the recession. The nation's new health care law eliminates these lifetime limits for plans that were issued or renewed on Sept. 23 or later.
Unlike drugs that people can try for a month or two and keep using only if they keep responding, Provenge is an all-or-nothing $93,000 gamble. It's a one-time treatment to train the immune system to fight prostate tumors, the first so-called "cancer vaccine."
I'm fearful that this will become a drug for people with more resources and less available for people with less resources," said M.D. Anderson's cancer research chief, Dr. Christopher Logothetis.
When is a drug considered cost-effective?
The most widely quoted figure is $50,000 for a year of life, "though it has been that for decades — never really adjusted — and not written in stone," said Dr. Harlan Krumholz, a Yale University expert on health care costs.
Logothetis said Provenge and other experimental cancer vaccines in development need "a national investment" to sort out their potential, starting with Medicare coverage.
"It's no longer a fringe science. This is working," he said. "We need to get it in the door so we can evolve it."
The complete AP story can be found here.
From the op-ed pages of the Baltimore Sun:
On R&D tax credit, Obama does Reagan one better
President serves up a plan even conservatives can love
President Barack Obama's economic agenda has been controversial, to put it mildly. Tea Partiers are marching on Washington to demand reduced government spending, while meek and mild-mannered Nobel Prize-winning economist Paul Krugman criticizes the administration's stimulus as too small. Nobody seems to agree on what will create jobs and get the economy back on track.Fortunately, the president recently put forward an economic proposal that the whole country should be able to get behind. Its core component is expanding the research and development tax credit.
This move builds on the legacy of Ronald Reagan, who introduced the tax credit in 1982. However, the credit has always been temporary, requiring congressional reauthorization roughly every two years since.
Mr. Obama's proposal would allow companies to take a 17 percent deduction above 50 percent of R&D costs. That's a significant increase over the previous 14 percent rate. And the credit would be made permanent, allowing businesses to make better long-term plans and invest in new research with confidence.
More to the point, the Obama tax credit addresses an urgent economic need. Today, the size of America's Research and Development Tax Credit ranks 17th among the 30 nations measured by the Organization for Economic Co-operation and Development. A decade ago, our credit was the biggest in the world. Even France now has an R&D credit four times larger than ours.
We can't afford to lose our advantage in innovation. Research and development are at the heart of America's most vibrant industries. Case in point: The biopharmaceutical sector.
Biopharma innovation is usually noteworthy for saving lives. Three years after introducing the first antiretroviral treatments, AIDS deaths dropped 70 percent, and new pharmaceuticals are largely responsible for cutting cancer death rates in half.
Less well known are the tremendous economic benefits of the industry. Biopharma directly employs some 700,000 Americans and indirectly supports 3.2 million more jobs. The average biopharma salary in 2008 was $77,595. That's $32,000 more than the average private-sector job.
From 1996 to 2006, the rate of job growth in biopharma was twice the national average. Even through the first year of the current recession, when overall private sector unemployment fell, the biopharmaceutical sector grew by 1.4 percent.
Globally, America dominates this rapidly expanding, high-wage industry. In 2007, America had 2,700 new drugs under development, compared with 1,700 being developed by all other countries combined.
But with other countries offering huge tax credits for research and development, America can't count on biopharmaceutical dominance forever.
On average, a new biotech drug costs more than a billion dollars to develop. And for every new drug that gets FDA approval and makes it to market, 5,000 to 10,000 unsuccessful drugs get tested. America's R&D tax credit provides significant support for this industry.
A bigger and permanent credit would boost the biopharmaceutical industry, help create new, life-enhancing drugs, and drastically cut down on overall medical expenses. It's estimated that each additional dollar spent on new pharmaceuticals saves $7 elsewhere in the economy.
At first glance, a bigger R&D tax credit might seem like just more government spending. But the evidence suggests the credit would create jobs and pay for itself.
According to a 2010 study by the Milken Institute, increasing this credit by 25 percent and making it permanent would add $206.3 billion to real GDP and raise total employment by 510,000 jobs within a decade.
By comparison, the Research and Development Tax Credit is currently estimated to cost just $7 billion a year.
At a time when there's a lot of partisan rancor over economic policy, Mr. Obama's proposal is the expansion of an existing policy that has had bipartisan support for decades. There are precious few points of agreement on the economy these days. A bipartisan solution with almost no economic downside rarely comes along. We shouldn't miss the opportunity to expand and make permanent the Research and Development Tax Credit.
Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.
