Latest Drugwonks' Blog
Patient choice: strike one.
Donald Berwick as CMS Administrator? By all accounts a good choice. But what does it mean for the upcoming battle royale over comparative effectiveness and patient choice?
According to Robert Pear, “Dr. Berwick could help shield the White House from Republican charges that Mr. Obama’s policies would lead to the rationing of care or even a government takeover.”
This conditional shield comes courtesy of remarks Dr. Berwick made last December. Speaking at the annual conference of the Institute for Healthcare Improvement (which he heads), he challenged the audience thus, “Over the next three years, reduce the total resource consumption of your health care system, no matter where you start, by 10%. Do that without a single instance of harm, without rationing effective care, without excluding needed services for any population you serve.”
Okay – sounds good and we should give Dr. Berwick the benefit of the doubt that when he says he’s against the rationing of “effective care,” he means effective care that’s defined by an MD in the field – and not Uncle Sam, MD inside the Beltway.
But vigilance is required, especially now that it’s precisely Uncle Sam who is going to be paying more and more of the bills for this “effective care.” And vigilance is even more important considering that AHRQ (now the nation’s leading practitioner of comparative effectiveness studies) plans to hire a PR firm to help create a “publicity center” for comparative effectiveness reports and materials.
If you recall the debacle that followed the government’s “publicity center” efforts behind CATIE and ALLHAT – you know why vigilance is the order of the day.
The “angry itch” is the desire for payers (both public and private) to opt for the least expensive treatment rather than the one that’s best for the patient. An itch that’s often penny-wise and pound-foolish. An itch that’s dangerous when scratched.
Let’s all wish Dr. Berwick great success. He has big shoes to fill and a tough road ahead.
The UK has launched a pilot of its "Innovation Pass" process, which will provide £25 million in funding for medicines that treat very rare diseases but are not evaluated at launch by the National Institute for health and Clinical Excellence (NICE).
Innovation passes were first proposed in the UK's office for life sciences' Life Sciences Blueprint, issued last year. The government has run a public consultation on the proposals since then.
There’s a maximum spending cap of only £8 million per year for each individual product – and uncertainty over long-term funding. The pilot is to run for three years, but government funding of £25 million has only been arranged for the first year.
Products included in the pilot Innovation Pass scheme will automatically be appraised by NICE after the end of three years, raising the suspicions that they could be rejected for use in the UK national health service (NHS) at that stage.
NICE giveth and NICE taketh away since it will set up and run an advisory committee that will select products based on defined criteria. These criteria include the medicine being a significant medical innovation (acting at a new target receptor, for example), it should satisfy an unmet clinical need, and is expected to have a substantial impact. Additional studies to gain further clinical data should be planned.
The government will pay an amount to the pharmaceutical company for supplying the medicine based on a price-volume agreement (the number of patients multiplied by the price), rather than paying for each dose of drug dispensed. This is to ensure a financial return for the company. Products would normally be submitted for consideration at around the same time as they are filed for marketing approval.
BUT … the sum asked for by the company will be judged to be reasonable or not by a governmental/NHS panel that will look at the cost of therapeutically similar medicines, the actual cost of the medicine in other European countries, and the cost of its research and manufacture.
This judgment on whether the cost is reasonable or not will then be passed to NICE's advisory committee, which will produce a list of drugs for funding, which will be approved by government ministers.
Sounds familiar.
NICE work … if you can get it.
FDA Chief Scientist Jesse Goodman to Representative Rosa DeLauro (D/CT and Chairwoman of the Agricultural Appropriations Subcommittee), "I think what FDA really needs is a 5- to 10-year building effort/re-building effort. And it's not just rebuilding to what was. I think it's being a part of building the science of the future."
Bravo. By all means. Rather than look backwards to “the good old days” (whatever that means) let’s improve and move forward. It’s not rocket science – but it’s good to hear the agency’s Chief Scientist say it.
And change starts from within. According to Goodman, "What I want to do is begin to use the resources we have and the leadership we have to encourage and identify and free-up some of the time of our promising junior and mid-level people to beef up their education. Because if we just do it as leaders of the center or agencies, that doesn't have all the transformational power."
Absolutely right. When I served at the FDA (along with Jesse), one of the most valuable lessons I learned was that dictates from “on high” don’t get the job done. Real change happens because all levels of the agency understand and embrace the philosophy of those changes. Change may begin at the top – but success or failure is determined by the agency’s 11,000+ career professionals.
Change is never easy but, as W. Edwards Deming commented, "Change is not required. Survival is not mandatory."
Goodman: “"It's very challenging. It's almost like taking the current state of the agency from sort of always swimming to keep our heads just above the water to something that is a truly outstanding scientific partner and really has the power and relationships it needs. And by power I don't mean power over people, but the mental and scientific tools to have the ability to make decisions. So it's a process and it's going to take a while and we need to get the best and the brightest."
Now maybe Representative DeLauro will finally embrace the Reagan/Udall Foundation.
- 24 March 2010, The Washington Post (By Ruth Marcus)
In fact, the occasion called for more humility than hyperbole, however unlikely that may have been given the setting. If I were a member of Congress, my floor speech before casting a yes vote would have boiled down to:
Gee, I hope this works.
One of the astonishing aspects of the health-care debate is how little is actually known about the implications of a change this far-reaching. Everyone has a theory, and a model to match, but even some of the most fundamental questions remain the subject of debate.
On the most basic of all -- does having health insurance lead to better health? -- the evidence is solid but not unanimous. The Institute of Medicine , reviewing the literature in 2009, found that "the body of evidence on the health consequences of health insurance is stronger than ever before. . . . Simply stated: Health insurance coverage matters ."
But a study that same year by Richard Kronick, a former health-care adviser to President Bill Clinton, found "little evidence to suggest that extending insurance coverage to all adults would have a large effect on the number of deaths in the United States ." Kronick's study has been criticized because it did not adjust for the fact that those in poor health are more likely to seek insurance. But the disagreement underscores the difficulty of knowing precisely what changes are in store.
To take another example, one common assertion has been that the uninsured end up getting health care -- just more expensive health care, in emergency rooms and when conditions have worsened, with the costs passed on to the rest of the population. The notion that the tab is being picked up one way or another makes intuitive sense.
A new National Bureau of Economic Research paper by Michael Anderson, Carlos Dobkin and Tal Gross questions this assumption. The researchers examined health-care consumption by 19-year-olds who had just been dropped from their parents' coverage. They found that not having insurance resulted in a 40 percent reduction in emergency room visits -- "contradicting the conventional wisdom that the uninsured are more likely to visit" the emergency room and a 61 percent drop in hospital admissions.
"Overall, these results suggest that an expansion in health insurance coverage would substantially increase the amount of care that currently uninsured individuals receive and require an increase in net expenditures," the authors write. Emergency room visits could increase by 13 million annually, and hospital admissions by 3.8 million, they project.
So prudence is in order when tinkering with such an interconnected system and when making confident predictions about the effects of reform, for good or ill. Will younger adults, who account for about half the population of uninsured non-elderly adults, sign up for coverage -- or will they pay the fine instead? How will that decision affect premium levels and the adequacy of federal subsidies?
Will the expansion of coverage create a shortage of health-care providers and result in higher prices, or will, for example, higher Medicaid payments for primary-care doctors stem an exodus of doctors from the program? Will employers add coverage because workers facing the mandate to obtain insurance will press for it, or will they drop it because it will be cheaper to pay the penalty and let employees fend for themselves?
Will increased coverage of preventive care save money because diseases will be caught earlier -- or will the added cost of widespread screening exceed the economic benefits? The Congressional Budget Office has concluded that, "for most preventive services, expanded utilization leads to higher, not lower, medical spending overall ."
The legislation is a risk worth taking. Millions of Americans are without insurance, a national scandal that should have been addressed long ago. Rising health-care costs threaten the nation's fiscal security, and the new law holds the promise of beginning to stem the increases.
The status quo is unsustainable. A new study by the Urban Institute shows how, without reform, the numbers of the uninsured will rise, employers will continue to drop coverage and premiums will climb.
Still, for those who express cocky certitude about how this is going to turn out, the best prescription is a generous dose of caution
Lynne Newhouse Segal was the picture of robustness. At 59, the slim former lawyer was an avid runner, golfer and yoga practitioner. Segal, who lives in San Francisco, was healthy by nearly every measure — except her cholesterol level, which a routine test four years ago revealed was high. High cholesterol is a key risk factor for heart disease, especially in a patient Segal's age and with her family history (several close relatives had had heart attacks), so her doctor put her on a cholesterol-lowering statin drug as a preventive measure.
Oh, but dark clouds soon gather...
But Segal's statin ended up preventing her from living a heart-healthy lifestyle. A month after she started taking the drug, she suffered muscle pain so severe, she had to stop all physical activity and was unable to sleep at night. Although her husband, who was worried about her risk of heart attack, pleaded with her to stay on the drug, she discontinued using it. The muscle pain receded. "My husband was scared for me. Doctors scare you. But I was in so much pain, I told him I would have rather died than stay on them," says Segal.
That grim situation could have been avoided, researchers say.
How about asking for another type of statin? That's what Marilyn Goldberg, age 75, did after suffering muscle pains so severe she complained to her husband Mort and son Bob (that's me) almost daily... And she did, about 4 or 5 times until she found one that worked. And now the pains are gone and her cholesterol is really low.
But you see, Marilyn is not a set piece or prop in some Obama-esque sob story about how we the people are screwed by uncaring corporations. She is a responsible person taking action on her own behalf...
Rather died?
I don't want to harp on this self-pitying because I am half-convinced that it is only half-true. That's because much of the "reporting" in the Time article on women and statins is half-baked. You read the piece sensing that women receive NO benefit from statins and tons of risk. In fact, there are many sub-groups and many situations where that is not the case.
The issue at hand is whether statins should be given to women who do not have high cholesterol at all but have other risk factors because cholesterol is not a very good marker for heart disease in women... (my emphasis)
The JUPITER study was designed to test whether other markers of heart disease in relatively healthy older women and men predicted heart disease by giving them statins to reduce a protein called CRP. If CRP was reduced and nasty events that lead to hospitalizations or death were reduced after taking a solid dose of the statin then the study could claim to show that CRP testing in combination with statins reduced the incidence of heart disease in relatively healthy women and men. And since women do not seem to benefit from reduced cholesterol levels (in the absense of other risk factors) the CRP -statin link is a pretty big deal for primary prevention.
As for the side effects.. When you draw the blood for CRP you can also draw blood to see if you will metabolize specific statins in ways that cause muscle pain, blurry vision, etc. There are tests for that. The question is whether the statin used in the JUPITER study (Crestor) can be substituted with other statins.
Now that is what the TIME article should have been about. But I guess the magazine had to devote resources to another hit piece on Israel.
Read more: http://www.time.com/time/magazine/article/0,9171,1973295-1,00.html#ixzz0j6iJUqhy
ChemGenex has agreed to meet with the FDA discuss developing a validated assay test for determining which patients might benefit from its experimental chronic myeloid leukemia (CML) treatment Omapro (omacetaxine mepesuccinate).
"This is a comparative efficacy claim that somebody is making here, so the level of proof has to be there," said Richard Pazdur, director of the Office of Oncology Drug Products in the Center for Drug Evaluation and Research. "The message is that the agency is trying to get across is that attention has to be paid to these in vitro diagnostics." ChemGenex announced the initiative shortly after the FDA's Oncology Drugs Advisory Committee voted 7-1 to require such testing before approving Omapro, which is targeted at CML patients who have failed Novartis' Gleevec (imatinib).
Speaking of Novartis, the ChemGenex situation is another important example of the role companion diagnostics can play in bringing new drugs to market.
In August 2009, Novartis’ two-year effort to revive its Prexige pain pill (after it was rejected by
With a test showing that a certain drug will be safe, Nohaile said, “We can go to doctors and say it moves it out of the realm of choice into the realm of malpractice if you don’t use this drug.
Exciting stuff -- but once these diagnostics are developed, will payers reimburse? In the wake of the payer disquietude over spending $400 on a diagnostic test to determine whether a patient should be given warfarin, it’s very much an open question. (Even more peculiar considering that conservative estimates project using the warfarin diagnostic -- specifically called out in the amended FDA label -- will prevent 85,000 serious bleeding events and 17,000 strokes annually in the United States. And this “safer use” is estimated to save $1.1 billion annually.
Stay tuned.
Two headlines on the same day... March 22
Major health care changes won’t take place until 2014
Delays could help Obama’s reelection hopes
Key elements of health reform would start soon
The Pink Sheet reports on "renewed optimism" that the European Union's proposed legislation on allowing drug makers to provide information to patients on prescription-only medicines will again start moving through the legislative process. The latest thinking, however, is strongly focused on the rights of patients to receive such information, rather than industry's right to disseminate it.
An interesting and important finesse – the rights of a patient to the information but no “right” for industry to provide it. Hm.
Suggested amendments emerged on March 10 from European Parliament Member Christofer Fjellner. He's reviewing the proposed legislation for the EU parliament's Committee on the Environment, Public Health and Food Safety.
For patients, Fjellner contends that information on pharmaceuticals should only be made available to patients who are actively searching for it, i.e., information should be "pulled" by the patient rather than "pushed" by industry. Fjellner believes that companies should not be allowed to make available information on prescription-only medicines on television or in newspapers or magazines. He believes the Internet is the appropriate medium for providing information to patients.
And maybe he's right -- but is there really a difference? If a pharmaceutical company makes available information on a web page – why is that different than making it available in other media? Hm. And what about patients who do not have access to the Internet -- what about their rights?
Fjellner's stance comes as a response to a ruling from the European Court of Justice, which concluded in April 2009 that current legislation could be applied to independent journalists. One journalist, Frede Damgaard, was considered to have broken the ban on DTC advertising by writing about a particular prescription-only product, a ruling that has caused an outcry in the media.
For more on this issue, see “Eighty-Sixing Free Speech.”
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.
