Latest Drugwonks' Blog
From today's edition of the Wall Street Journal ...
Faster FDA Cures
Whoever controls Congress next year, the Food and Drug Administration is sure to be a political battleground. The controversial Prescription Drug User Fee Act of 1992 is scheduled for reauthorization, and the conventional political wisdom holds that the FDA is too "cozy" with Big Pharma and thus overeager to rubber-stamp dangerous new drugs.
So allow us to draw attention to an important and undernoticed study, "Assessing the Safety and Efficacy of the FDA," published by the National Bureau of Economic Research. Its surprising conclusion is that the FDA does a pretty good job managing "the central speed-safety trade-off" involved in drug approvals -- and might even do well to move faster.
The authors looked at 662 drugs approved between 1979 and 2002, a period that included the passage of the Prescription Drug User Fee Act that allowed drug companies to help fund the FDA budget in exchange for faster reviews. They found that the resulting modest gains in drug review times have produced unambiguous public benefits.
In monetary terms, the authors estimate that the law improved consumer welfare by as much as $19 billion. But more importantly, they found that more rapid access to beneficial drugs saved between 180,000 and 310,000 "life-years." By contrast, assuming that every risky drug withdrawn in that period was the fault of the Prescription Drug User Fee Act produces an upper-bound estimate of only 56,000 life-years lost.
This study deserves a lot more attention than it's been getting, since the Washington debate is dominated by bad ideas about how to make FDA regulation much more onerous. In particular, a recent report from the National Academy of Science's Institute of Medicine (IOM) is being spun as evidence that the FDA is somehow "broken" and in need of fundamental reform.
We've looked at the IOM report in detail, and it's hardly as damning of the FDA as media reports suggest. For example, the report explicitly rejects the idea that high-profile drug withdrawals -- such as Vioxx -- "represent de facto failures of the drug safety regulatory system. . . This is not so." The IOM also rejects the basic idea behind legislation sponsored by Senators Chuck Grassley and Chris Dodd that the FDA should have a drug safety office separate from the office that approves drugs in the first place: "Risk and benefit cannot be considered in isolation from one another."
But despite these sensible observations, the IOM still wants more regulation. Among its recommendations are vastly increased levels of post-approval safety monitoring. That might make sense if the goal is making regulators comfortable with the idea of approving drugs more quickly. But that doesn't appear to be what the IOM and its cheering section want.
The IOM also proposes draconian restrictions on new-drug advertising and tighter conflict-of-interest standards for FDA advisory panel members. This despite the fact that the most qualified scientists and doctors have substantial sources of private income, and despite a study by the Naderite Public Citizen group that failed to find one instance of a panel recommendation that would have changed if allegedly conflicted members had been excluded.
Most fundamentally, the IOM presents little evidence that there is even a problem to remedy. By contrast, the National Bureau authors at least attempt to put some real facts on the table. They note that the absence of other attempts at hard, quantitative analysis in the great FDA debate is "somewhat paradoxical, since despite the agency's strict adherence to evidence-based evaluation of products overseen, there is less evidence on its own safety and efficacy. Put differently, no product application would pass the FDA approval process with the quality and type of evidence that currently exists for evaluating the FDA policies themselves."
The upshot of the economists' report is that the 1992 Prescription Drug User Fee Act deserves reauthorization. But the evidence also suggests Congress should probably be looking for other ways to make FDA approvals still faster. Despite the high-profile Vioxx panic, the FDA is far more likely to kill by depriving you of a drug than allowing you to take a dangerous one.
Faster FDA Cures
Whoever controls Congress next year, the Food and Drug Administration is sure to be a political battleground. The controversial Prescription Drug User Fee Act of 1992 is scheduled for reauthorization, and the conventional political wisdom holds that the FDA is too "cozy" with Big Pharma and thus overeager to rubber-stamp dangerous new drugs.
So allow us to draw attention to an important and undernoticed study, "Assessing the Safety and Efficacy of the FDA," published by the National Bureau of Economic Research. Its surprising conclusion is that the FDA does a pretty good job managing "the central speed-safety trade-off" involved in drug approvals -- and might even do well to move faster.
The authors looked at 662 drugs approved between 1979 and 2002, a period that included the passage of the Prescription Drug User Fee Act that allowed drug companies to help fund the FDA budget in exchange for faster reviews. They found that the resulting modest gains in drug review times have produced unambiguous public benefits.
In monetary terms, the authors estimate that the law improved consumer welfare by as much as $19 billion. But more importantly, they found that more rapid access to beneficial drugs saved between 180,000 and 310,000 "life-years." By contrast, assuming that every risky drug withdrawn in that period was the fault of the Prescription Drug User Fee Act produces an upper-bound estimate of only 56,000 life-years lost.
This study deserves a lot more attention than it's been getting, since the Washington debate is dominated by bad ideas about how to make FDA regulation much more onerous. In particular, a recent report from the National Academy of Science's Institute of Medicine (IOM) is being spun as evidence that the FDA is somehow "broken" and in need of fundamental reform.
We've looked at the IOM report in detail, and it's hardly as damning of the FDA as media reports suggest. For example, the report explicitly rejects the idea that high-profile drug withdrawals -- such as Vioxx -- "represent de facto failures of the drug safety regulatory system. . . This is not so." The IOM also rejects the basic idea behind legislation sponsored by Senators Chuck Grassley and Chris Dodd that the FDA should have a drug safety office separate from the office that approves drugs in the first place: "Risk and benefit cannot be considered in isolation from one another."
But despite these sensible observations, the IOM still wants more regulation. Among its recommendations are vastly increased levels of post-approval safety monitoring. That might make sense if the goal is making regulators comfortable with the idea of approving drugs more quickly. But that doesn't appear to be what the IOM and its cheering section want.
The IOM also proposes draconian restrictions on new-drug advertising and tighter conflict-of-interest standards for FDA advisory panel members. This despite the fact that the most qualified scientists and doctors have substantial sources of private income, and despite a study by the Naderite Public Citizen group that failed to find one instance of a panel recommendation that would have changed if allegedly conflicted members had been excluded.
Most fundamentally, the IOM presents little evidence that there is even a problem to remedy. By contrast, the National Bureau authors at least attempt to put some real facts on the table. They note that the absence of other attempts at hard, quantitative analysis in the great FDA debate is "somewhat paradoxical, since despite the agency's strict adherence to evidence-based evaluation of products overseen, there is less evidence on its own safety and efficacy. Put differently, no product application would pass the FDA approval process with the quality and type of evidence that currently exists for evaluating the FDA policies themselves."
The upshot of the economists' report is that the 1992 Prescription Drug User Fee Act deserves reauthorization. But the evidence also suggests Congress should probably be looking for other ways to make FDA approvals still faster. Despite the high-profile Vioxx panic, the FDA is far more likely to kill by depriving you of a drug than allowing you to take a dangerous one.
Yesterday I was privileged to participate in the latest in a series of Senator John Breaux’s “Ceasefire†debates. (Ceasefire events bring together a variety of speakers with opposing viewpoints in an effort to find common ground. Past speakers include former Speaker of the House Newt Gingrich, Senator Hillary Rodham Clinton, Former Secretaries of HHS Donna Shalala and Tommy Thompson, Press Secretaries Ari Fleischer and Mike McCurry, and former Senators Tom Daschle and Ron Nickles. The Ceasefire on Healthcare series is made possible by a grant from Pfizer Inc.)
I was paired with David Kendall, Senior Fellow for Health Policy and Director of the Health Priorities Project at the Progressive Policy Institute.
We had, what’s the right word here, a robust conversation. The entire debate will shortly be posted (as a podcast) at http://www.ceasefireonhealthcare.org/podcast, but I wanted to share two things that seemed especially germane and interesting –
(1) We debated whether or not physician “pay-for-performance†is a good thing. (My position is that it all depends on what “performance†means.) Consider this – if we limit what medicines doctors can prescribe based on cost-centric rather than patient-centric models, how can we possibly measure “performance†outcomes?
(2) Senator Breaux asked if we thought health care would play a major role in the up coming Presidential election. David said, “yes.†I said “no.â€
If the current debate over health care is any indication, what we’re in store for is too many months of sounds bites, finger pointing, and pharma bashing.
And that is not a debate over health care.
I was paired with David Kendall, Senior Fellow for Health Policy and Director of the Health Priorities Project at the Progressive Policy Institute.
We had, what’s the right word here, a robust conversation. The entire debate will shortly be posted (as a podcast) at http://www.ceasefireonhealthcare.org/podcast, but I wanted to share two things that seemed especially germane and interesting –
(1) We debated whether or not physician “pay-for-performance†is a good thing. (My position is that it all depends on what “performance†means.) Consider this – if we limit what medicines doctors can prescribe based on cost-centric rather than patient-centric models, how can we possibly measure “performance†outcomes?
(2) Senator Breaux asked if we thought health care would play a major role in the up coming Presidential election. David said, “yes.†I said “no.â€
If the current debate over health care is any indication, what we’re in store for is too many months of sounds bites, finger pointing, and pharma bashing.
And that is not a debate over health care.
Maclean's, Mon 16 Oct 2006
Byline: DANYLO HAWALESHKA
In the pivotal courtroom scene from A Few Good Men, Tom Cruise shouts, "I want the truth!" at Jack Nicholson, whose truculent character is testifying. "You can't handle the truth!" Nicholson famously retorts. Today, in a real-life courtroom drama, CanWest MediaWorks Inc. has, in a manner, assumed Cruise's role, with a somewhat less altruistic goal in mind than simple verity.
In a civil lawsuit now before Ontario's Superior Court of Justice, CanWest contends Canadians are being denied important truths about prescription medications because of strict limits on drug advertising. Canada's largest media company claims the regulations thwart its constitutional right to freedom of expression.
CanWest's suit against the attorney general of Canada casts the country's Food and Drugs Act as the villain. The act forbids direct-to-consumer advertising (DTCA) that ties a prescription drug to a treatment, cure or disease. (It is illegal for CanWest to, for instance, run an ad to promote Viagra for impotence, but quite legal to separately publicize the brand, or the condition; there are no such restrictions on ads that target physicians exclusively in, say, trade journals.) In a sworn statement, Arturo Duran, a CanWest MediaWorks president, claims permitting DTCA in Canada would educate the public about drug risks and benefits. "There is no evidence to justify a ban on truthful advertising of prescription drugs," Duran says.
Byline: DANYLO HAWALESHKA
In the pivotal courtroom scene from A Few Good Men, Tom Cruise shouts, "I want the truth!" at Jack Nicholson, whose truculent character is testifying. "You can't handle the truth!" Nicholson famously retorts. Today, in a real-life courtroom drama, CanWest MediaWorks Inc. has, in a manner, assumed Cruise's role, with a somewhat less altruistic goal in mind than simple verity.
In a civil lawsuit now before Ontario's Superior Court of Justice, CanWest contends Canadians are being denied important truths about prescription medications because of strict limits on drug advertising. Canada's largest media company claims the regulations thwart its constitutional right to freedom of expression.
CanWest's suit against the attorney general of Canada casts the country's Food and Drugs Act as the villain. The act forbids direct-to-consumer advertising (DTCA) that ties a prescription drug to a treatment, cure or disease. (It is illegal for CanWest to, for instance, run an ad to promote Viagra for impotence, but quite legal to separately publicize the brand, or the condition; there are no such restrictions on ads that target physicians exclusively in, say, trade journals.) In a sworn statement, Arturo Duran, a CanWest MediaWorks president, claims permitting DTCA in Canada would educate the public about drug risks and benefits. "There is no evidence to justify a ban on truthful advertising of prescription drugs," Duran says.
Open Letter from the Ontario Pharmacists' Association
TORONTO, Oct. 11
Hon. Tony Clement
Minister of Health, Government of Canada
Minister's Office, Health Canada
Brooke Claxton Building, Tunney's Pasture
Ottawa, Canada K1A 0K9
Dear Minister,
Ontario pharmacists are gravely concerned about the imminent threat to Canada's prescription drug supply, and the corresponding public health and safety threat to Canadians, arising from legislative changes and relaxed enforcement measures in the United States that re-open America's border to the importation of prescription drugs by U.S. patients.
As you know, Congressional leaders struck a deal on September 29 to allow Americans to carry back from Canada a 90-day personal supply of prescription drugs through "foot traffic" importation. The Department of Homeland Security compounded this change by ceasing confiscation by Customs and Border Protection agents of drugs ordered over the Internet and mailed to Americans, effective October 9.
We appreciate that these developments represent political deal-making by American lawmakers to take high U.S. prescription drug costs off the table as an issue to protect incumbents in the U.S. mid-term elections. However, the impact on Canada is clear: we are back to a free-for-all for cross-border drug importation.
We are deeply disturbed that in spite of the serious implications for Canada's drug supply and the health and safety of Canadians, there is no evidence of consultation with the Government of Canada, or of any effort to ask how our pharmacists and drug experts view American patients raiding Canada's medicine cabinet.
Our pharmacists do not want to become America's drug store. Our job is to provide medications and expertise to Canadian patients, not provide solutions or the shortcomings of the U.S. health care system and its problem with high drug costs.
U.S. demand is more than ten times the size of Canadian supply. We do not have the capacity to feed America's need for lower-cost drugs, and unimpeded depletion of our supply poses a serious threat to public health and safety in Canada.
We are further concerned that the legitimizing of Internet drug purchases by Americans encourages fraud by offshore criminals posing as Canadian pharmacists and selling counterfeit drugs. This is a health and safety threat to both American and Canadian patients who rely on the reputation of Canadian pharmacists and buy drugs from what they believe are Canadian Internet pharmacies, believing them to be safe and genuine.
In fact, U.S. customs statistics on mail-order seizures show at least 10 per cent of packages purportedly from Canadian Internet pharmacies contain counterfeit drugs.
We believe it is prudent and reasonable to also call attention at this time to the renewed emergence with these U.S. legislative and enforcement changes of a national security vulnerability in both the U.S. and Canada arising from the re-opening of the door to the potential for drug terrorism, with drugs sent by mail used as a vehicle of attack on innocent members of the public.
In this respect, an April 2005 report to Congress warned "the nation's medicine supply is vulnerable to exploitation by organized criminals, drug traffickers and terrorists. We should not contemplate opening our borders to threats to our medicine supply when in all other aspects we are searching for ways to tighten the security of our borders."
While the legislative changes enacted to date do not support bulk importation, we note that proponents of the changes in Congress have indicated their interest and intent to bring forward "full-blown" importation legislation in the future. We must warn unequivocally that bulk importation would quickly deplete the Canadian drug supply and cause a crisis in drug
availability for Canadians - in one credible U.S. analysis, within 38 days.
We also have a more fundamental concern that these developments further encourage the use of Internet pharmacies by patients who bypass their own community pharmacists. Patients who do so jeopardize their health and risk dangerous drug interactions by relinquishing expert consultation and crucial pharmacist-patient interaction.
We are disappointed that the Government of Canada has stood idly by while the U.S. has made changes that threaten the Canadian prescription drug supply, pose a danger to the health and safety of Canadians, raise national security vulnerabilities and undermine the important, long-standing relationship between pharmacists and patients.
We respectfully call on the Government of Canada to take immediate action to protect Canada's prescription drug supply by banning prescription drug sales to U.S. patients by all means, including "foot traffic", Internet and mail order.
We look forward to your reply at your earliest convenience.
Marc Kealey
Chief Executive Officer
Ontario Pharmacists' Association
For further information: or to set up an interview, please contact: Mary-Anne Cedrone, Manager of Communications, Ontario Pharmacists' Association, (416) 441-0788 ext.4266, Cell: (416) 278-5582
TORONTO, Oct. 11
Hon. Tony Clement
Minister of Health, Government of Canada
Minister's Office, Health Canada
Brooke Claxton Building, Tunney's Pasture
Ottawa, Canada K1A 0K9
Dear Minister,
Ontario pharmacists are gravely concerned about the imminent threat to Canada's prescription drug supply, and the corresponding public health and safety threat to Canadians, arising from legislative changes and relaxed enforcement measures in the United States that re-open America's border to the importation of prescription drugs by U.S. patients.
As you know, Congressional leaders struck a deal on September 29 to allow Americans to carry back from Canada a 90-day personal supply of prescription drugs through "foot traffic" importation. The Department of Homeland Security compounded this change by ceasing confiscation by Customs and Border Protection agents of drugs ordered over the Internet and mailed to Americans, effective October 9.
We appreciate that these developments represent political deal-making by American lawmakers to take high U.S. prescription drug costs off the table as an issue to protect incumbents in the U.S. mid-term elections. However, the impact on Canada is clear: we are back to a free-for-all for cross-border drug importation.
We are deeply disturbed that in spite of the serious implications for Canada's drug supply and the health and safety of Canadians, there is no evidence of consultation with the Government of Canada, or of any effort to ask how our pharmacists and drug experts view American patients raiding Canada's medicine cabinet.
Our pharmacists do not want to become America's drug store. Our job is to provide medications and expertise to Canadian patients, not provide solutions or the shortcomings of the U.S. health care system and its problem with high drug costs.
U.S. demand is more than ten times the size of Canadian supply. We do not have the capacity to feed America's need for lower-cost drugs, and unimpeded depletion of our supply poses a serious threat to public health and safety in Canada.
We are further concerned that the legitimizing of Internet drug purchases by Americans encourages fraud by offshore criminals posing as Canadian pharmacists and selling counterfeit drugs. This is a health and safety threat to both American and Canadian patients who rely on the reputation of Canadian pharmacists and buy drugs from what they believe are Canadian Internet pharmacies, believing them to be safe and genuine.
In fact, U.S. customs statistics on mail-order seizures show at least 10 per cent of packages purportedly from Canadian Internet pharmacies contain counterfeit drugs.
We believe it is prudent and reasonable to also call attention at this time to the renewed emergence with these U.S. legislative and enforcement changes of a national security vulnerability in both the U.S. and Canada arising from the re-opening of the door to the potential for drug terrorism, with drugs sent by mail used as a vehicle of attack on innocent members of the public.
In this respect, an April 2005 report to Congress warned "the nation's medicine supply is vulnerable to exploitation by organized criminals, drug traffickers and terrorists. We should not contemplate opening our borders to threats to our medicine supply when in all other aspects we are searching for ways to tighten the security of our borders."
While the legislative changes enacted to date do not support bulk importation, we note that proponents of the changes in Congress have indicated their interest and intent to bring forward "full-blown" importation legislation in the future. We must warn unequivocally that bulk importation would quickly deplete the Canadian drug supply and cause a crisis in drug
availability for Canadians - in one credible U.S. analysis, within 38 days.
We also have a more fundamental concern that these developments further encourage the use of Internet pharmacies by patients who bypass their own community pharmacists. Patients who do so jeopardize their health and risk dangerous drug interactions by relinquishing expert consultation and crucial pharmacist-patient interaction.
We are disappointed that the Government of Canada has stood idly by while the U.S. has made changes that threaten the Canadian prescription drug supply, pose a danger to the health and safety of Canadians, raise national security vulnerabilities and undermine the important, long-standing relationship between pharmacists and patients.
We respectfully call on the Government of Canada to take immediate action to protect Canada's prescription drug supply by banning prescription drug sales to U.S. patients by all means, including "foot traffic", Internet and mail order.
We look forward to your reply at your earliest convenience.
Marc Kealey
Chief Executive Officer
Ontario Pharmacists' Association
For further information: or to set up an interview, please contact: Mary-Anne Cedrone, Manager of Communications, Ontario Pharmacists' Association, (416) 441-0788 ext.4266, Cell: (416) 278-5582
If you want a concise explanation as to why the drug safety issue is spiraling out of control and how the media's reporting on such issues contributes to a sense of danger in every drug, read this excellent post from The Scientist.....
A few years ago Princeton health economist Uwe Reinhardt, Dartmouth geriatrician Joann Lynn, and I filmed a documentary on the effect of advancing biomedical technology on affordable healthcare in the United States. Five hours of interviews were reduced to 10-minute bookends for a set of short, emotional stories that obliterated the complexities of the issues. As Reinhardt quipped in our cab, repeating the oft-cited quote, the plural of anecdote is not data. Except on television.
We've all had this experience: You bring work home and talk about concepts central to biomedical research such as evidence-based medicine, controlled trials, equipoise, peer-review, or impact factor. Friends' eyes roll up into their skulls in boredom. Yet the same topics come up in everyday conversation all the time, just framed in a different way: "I know a person who lost his house to the cost of drugs," and "you know a guy who is alive because of Lipitor." Heated arguments ensue about real problems in science, but driven by someone's single story.
Stories are not the enemy of good science and evidence-based medicine. Physicians make crucial but subtle changes in their practices based on individual experiences. Scientists all use intuition and inductive reasoning in the nascent period of an investigation. But anecdotes cannot substitute for either ethnography or controlled study. When Terri Schiavo became the world's test case for diagnosing persistent vegetative state (PVS), the emotional intonations about Ms. Schiavo waking up began to sound like Intelligent Design.
A paradox of biomedical research is that huge controlled trials, meta-analyses, and reviews of the literature are ubiquitous, but the number of "case reports" - and journals comprised entirely of incidental "findings" - is growing.
The media has no idea how to deal with case reports. The worst example of this in recent times was a case study of Zolpidem, the nonbenzodiazepine-branded 'Ambien' and approved by the US Food & Drug Administration for the treatment of insomnia. Physicians Ralf Clauss and Wally Nel have published, a few cases at a time, their very different use of the medication. The Guardian carried a breathless report of Clauss et al's August report in the journal NeuroRehabilitation of three cases involving patients who have been in PVS, they report, for more than three years. Claus and Nel grabbed the front page with the Guardian's report that they used Ambien to wake up these patients.
The 'investigators' had administered Zolpidem for between three and six years and saw each of the three 'treated' patients wake up each day as a result of the medication; one even "caught a baseball." When the medication wore off, the patients dropped back into PVS each evening.
Stunning science? It seemed so, too, back in 2000, when Claus and a different set of South African colleagues published in the South African Medical Journal on a single case with essentially the same outcome. In 2001 they made the same claim in a letter to the same journal. At no point did the investigators conduct an actual study of the phenomenon, with an IRB-approved research protocol or informed consent. Again and again they "wrote up" their "cases," describing their work as innovative medical management rather than research. Journal editors, asleep at the switch, have been derelict in publishing bad research disguised as cases - in this instance a case with the impact of finding a life-extending potion or the presence of extraterrestrial life.
Investigators who jumpstart their programs with case reports are often in search of research support, as was Claus - who as a result of the case report is now funded. In this respect they, and the journals who publish nothing but these case reports, are like the television producers I worked with: They aim at using the power of stories to make the claims and reap the rewards that come from research, but without doing the research. The victims are journalists, readers, and in the Claus case the patients and their families, who are exposed to uncontrolled experiments framed as good medicine. It is time for editors, journalists, the FDA, and the US Health & Human Services Office for Human Research Protections (OHRP) to clamp down on the "case study."
Glenn McGee is the director of the Alden March Bioethics Institute at Albany Medical College, where he holds the John A. Balint Endowed Chair in Medical Ethics.
gmcgee@the-scientist.com
A few years ago Princeton health economist Uwe Reinhardt, Dartmouth geriatrician Joann Lynn, and I filmed a documentary on the effect of advancing biomedical technology on affordable healthcare in the United States. Five hours of interviews were reduced to 10-minute bookends for a set of short, emotional stories that obliterated the complexities of the issues. As Reinhardt quipped in our cab, repeating the oft-cited quote, the plural of anecdote is not data. Except on television.
We've all had this experience: You bring work home and talk about concepts central to biomedical research such as evidence-based medicine, controlled trials, equipoise, peer-review, or impact factor. Friends' eyes roll up into their skulls in boredom. Yet the same topics come up in everyday conversation all the time, just framed in a different way: "I know a person who lost his house to the cost of drugs," and "you know a guy who is alive because of Lipitor." Heated arguments ensue about real problems in science, but driven by someone's single story.
Stories are not the enemy of good science and evidence-based medicine. Physicians make crucial but subtle changes in their practices based on individual experiences. Scientists all use intuition and inductive reasoning in the nascent period of an investigation. But anecdotes cannot substitute for either ethnography or controlled study. When Terri Schiavo became the world's test case for diagnosing persistent vegetative state (PVS), the emotional intonations about Ms. Schiavo waking up began to sound like Intelligent Design.
A paradox of biomedical research is that huge controlled trials, meta-analyses, and reviews of the literature are ubiquitous, but the number of "case reports" - and journals comprised entirely of incidental "findings" - is growing.
The media has no idea how to deal with case reports. The worst example of this in recent times was a case study of Zolpidem, the nonbenzodiazepine-branded 'Ambien' and approved by the US Food & Drug Administration for the treatment of insomnia. Physicians Ralf Clauss and Wally Nel have published, a few cases at a time, their very different use of the medication. The Guardian carried a breathless report of Clauss et al's August report in the journal NeuroRehabilitation of three cases involving patients who have been in PVS, they report, for more than three years. Claus and Nel grabbed the front page with the Guardian's report that they used Ambien to wake up these patients.
The 'investigators' had administered Zolpidem for between three and six years and saw each of the three 'treated' patients wake up each day as a result of the medication; one even "caught a baseball." When the medication wore off, the patients dropped back into PVS each evening.
Stunning science? It seemed so, too, back in 2000, when Claus and a different set of South African colleagues published in the South African Medical Journal on a single case with essentially the same outcome. In 2001 they made the same claim in a letter to the same journal. At no point did the investigators conduct an actual study of the phenomenon, with an IRB-approved research protocol or informed consent. Again and again they "wrote up" their "cases," describing their work as innovative medical management rather than research. Journal editors, asleep at the switch, have been derelict in publishing bad research disguised as cases - in this instance a case with the impact of finding a life-extending potion or the presence of extraterrestrial life.
Investigators who jumpstart their programs with case reports are often in search of research support, as was Claus - who as a result of the case report is now funded. In this respect they, and the journals who publish nothing but these case reports, are like the television producers I worked with: They aim at using the power of stories to make the claims and reap the rewards that come from research, but without doing the research. The victims are journalists, readers, and in the Claus case the patients and their families, who are exposed to uncontrolled experiments framed as good medicine. It is time for editors, journalists, the FDA, and the US Health & Human Services Office for Human Research Protections (OHRP) to clamp down on the "case study."
Glenn McGee is the director of the Alden March Bioethics Institute at Albany Medical College, where he holds the John A. Balint Endowed Chair in Medical Ethics.
gmcgee@the-scientist.com
Now that the Yankees were eliminated from the ALCS in humiliating fashion I have found myself channel surfing to fill the time....I stumbled upon a C-SPAN broadcast of the debate between Congressman Harold Ford, Jr. and Bob Corker, the Dem and GOP candidates for Bill Frist's Tennessee Senate seat. I might have been hallucinating but I think I heard Ford say 2-3 times that if the government negotiated with drug companies (price controls) it could save Medicare $1 trillion. Since the Part D program will spend about $500 billion over ten years, I am waiting for Ford to explain his math. Or does he have a plan to force drug companies to kick in twice what the government spends on medicines? Or maybe this is over 100 years...
In any event, Ford's koolaid drinking on price controls contrasts with the bill he co-sponsored with Cal Dooley in 2003 which itself sounds and adds up remarkably like the Part D program now in operation. In case Ford forgot or you didn't know about it, here it is:
Zero-Premium, High-Cost Protection, Part B Drug Benefit
Universal ... Progressive ... Affordable
• Zero Premium: This benefit would be incorporated into the existing Medicare Part B
program. No separate premium and no increase in Part B premium.
• Universal Eligibility: All Part B enrollees.
• Universal Protection Against High Drug Costs: Medicare will pay 80 percent of the
cost of drugs after beneficiary has total drug costs of $4,000. The individual will pay
based on a flat three-tiered co-payment: $4/$16/$24*
• Benefit Counts All Drug Spending: Negotiated drug costs Β not just the
beneficiaries= out-of-pocket spending Î’ counts toward the $4,000 deductible.
• Low-Income Benefits: Beneficiaries up to 200 percent of poverty will be eligible to
receive enhanced benefits via Medicare drug discount cards, Medicare+Choice,
Medicaid, Medigap, and other new coverage options.
Medicaid – 135% of fpl tiered co-payment: $4/$16/$24*
135% - 150% of fpl tiered co-payment: $6/$24/$36**
150% - 200% of fpl tiered co-payment: $10/$40/$60***
(State option with a federal/state match at the SCHIP rate)
• Universal Benefit Via Medicare Approved Drug Card Plans: A wide variety of
Medicare approved drug card plans including PBMs, State Assistance Plans, retiree
coverage, Medigap, Medicare + Choice, Medicaid and others that offer all seniors access
to the market-based discounts widely available to people with private insurance. These
discounts would apply to all drug purchases.
• Universal Benefit Via Current Coverage: Encourage the continuation of current
drug coverage through Employer/Retiree plans, Medigap plans, Medicaid, State
pharmaceutical assistance plans, or Medicare+Choice plans; all based on
reimbursement agreements with Medicare.
• CBO Cost Estimate: Under $400 billion
"Market-based discounts" "PBMs" "Under 400 billion" Sounds like a program we already know.
I am still waiting to see how the D's will 'negotiate' down drug prices without closing down formularies and restricting choices like the VA does. Can anyone say Third Rail?
In any event, Ford's koolaid drinking on price controls contrasts with the bill he co-sponsored with Cal Dooley in 2003 which itself sounds and adds up remarkably like the Part D program now in operation. In case Ford forgot or you didn't know about it, here it is:
Zero-Premium, High-Cost Protection, Part B Drug Benefit
Universal ... Progressive ... Affordable
• Zero Premium: This benefit would be incorporated into the existing Medicare Part B
program. No separate premium and no increase in Part B premium.
• Universal Eligibility: All Part B enrollees.
• Universal Protection Against High Drug Costs: Medicare will pay 80 percent of the
cost of drugs after beneficiary has total drug costs of $4,000. The individual will pay
based on a flat three-tiered co-payment: $4/$16/$24*
• Benefit Counts All Drug Spending: Negotiated drug costs Β not just the
beneficiaries= out-of-pocket spending Î’ counts toward the $4,000 deductible.
• Low-Income Benefits: Beneficiaries up to 200 percent of poverty will be eligible to
receive enhanced benefits via Medicare drug discount cards, Medicare+Choice,
Medicaid, Medigap, and other new coverage options.
Medicaid – 135% of fpl tiered co-payment: $4/$16/$24*
135% - 150% of fpl tiered co-payment: $6/$24/$36**
150% - 200% of fpl tiered co-payment: $10/$40/$60***
(State option with a federal/state match at the SCHIP rate)
• Universal Benefit Via Medicare Approved Drug Card Plans: A wide variety of
Medicare approved drug card plans including PBMs, State Assistance Plans, retiree
coverage, Medigap, Medicare + Choice, Medicaid and others that offer all seniors access
to the market-based discounts widely available to people with private insurance. These
discounts would apply to all drug purchases.
• Universal Benefit Via Current Coverage: Encourage the continuation of current
drug coverage through Employer/Retiree plans, Medigap plans, Medicaid, State
pharmaceutical assistance plans, or Medicare+Choice plans; all based on
reimbursement agreements with Medicare.
• CBO Cost Estimate: Under $400 billion
"Market-based discounts" "PBMs" "Under 400 billion" Sounds like a program we already know.
I am still waiting to see how the D's will 'negotiate' down drug prices without closing down formularies and restricting choices like the VA does. Can anyone say Third Rail?
It is hard not to miss the mass of pink products and pink ribbon promotions, this month being Breast Cancer Awareness Month. (Pink ribbons being the symbol of the quest to treat and cure breast cancer in case anyone has been sleeping the past decade.)
Several corporations are and have allocated a share of the sales of their products to specific breast cancer screening or research activities as part of this effort or as part of a particular company's effort to be socially responsible. No one seems to mind when company's past some sort of eco-friendly sign on their packaging but apparently the idea that capitalist concerns would link the marketing of their products to supporting better cancer care for women is driving a handful of angry, socialist women types crazy.
They argue that corporations are "getting rich" by promoting breast cancer awareness and research. The case rests on one or two isolated cases where the amount to be contributed to a particular organization was not made clear or by comparing marketing costs (for the product in general) to the actual contribution.
Now excuse me, but what is wrong about using a campaign to rid the world of disease or some problem a bad thing for a company to do? When the National Campaign to End Infantile Paralysis (polio) started the March of Dimes Campaign it used tie ins galore and even got then President Roosevelt to hawk products that we donating a share of its proceeds to the effort. And no one seems to give a rip when Ben and Jerry's donates a share of its profits to eliminating low self-esteem issues working in left wing NGOs or Paul Newman kicks in part of his pasta sauce proceeds to his camp for kids dyiing of cancer. And in both cases, people probably buy the products to support the cause. I bought a particular type of matzah for Passover because a share of the dough (unleavened) went to support the Jewish National Fund.
But when some corporations decide to sponsor and promote their sponsorship of breast cancer activities, that is getting rich. The alternative I guess, if you read the bitter and disjointed screeds of the critics, is to have companies do nothing or, more precisely, contribute to their pet projects. In the case of breast cancer, it would be to fund massive studies and lawsuits about the role every food additive and greenhouse gas plays in triggering cancer instead of the effort to support treatments which just enrich drug companies anyway.
The way to deal with fringe elements in all these cases is to take the extreme thinking to its logical conclusion: when you do, you are confronted with a group of people who would rather women be denied better care in order to have their worldview adopted. (Of course they would have the government do it all with higher taxes..just like in Canada where the screening and awareness levels are lower than here). Given the choice between no corporate involvement and corporate involvement along with the funding for a variety of programs that promote early detection, treatment and research, the fringe groups attacking the pink ribbon programs would take a victory over "getting rich" any time.
Several corporations are and have allocated a share of the sales of their products to specific breast cancer screening or research activities as part of this effort or as part of a particular company's effort to be socially responsible. No one seems to mind when company's past some sort of eco-friendly sign on their packaging but apparently the idea that capitalist concerns would link the marketing of their products to supporting better cancer care for women is driving a handful of angry, socialist women types crazy.
They argue that corporations are "getting rich" by promoting breast cancer awareness and research. The case rests on one or two isolated cases where the amount to be contributed to a particular organization was not made clear or by comparing marketing costs (for the product in general) to the actual contribution.
Now excuse me, but what is wrong about using a campaign to rid the world of disease or some problem a bad thing for a company to do? When the National Campaign to End Infantile Paralysis (polio) started the March of Dimes Campaign it used tie ins galore and even got then President Roosevelt to hawk products that we donating a share of its proceeds to the effort. And no one seems to give a rip when Ben and Jerry's donates a share of its profits to eliminating low self-esteem issues working in left wing NGOs or Paul Newman kicks in part of his pasta sauce proceeds to his camp for kids dyiing of cancer. And in both cases, people probably buy the products to support the cause. I bought a particular type of matzah for Passover because a share of the dough (unleavened) went to support the Jewish National Fund.
But when some corporations decide to sponsor and promote their sponsorship of breast cancer activities, that is getting rich. The alternative I guess, if you read the bitter and disjointed screeds of the critics, is to have companies do nothing or, more precisely, contribute to their pet projects. In the case of breast cancer, it would be to fund massive studies and lawsuits about the role every food additive and greenhouse gas plays in triggering cancer instead of the effort to support treatments which just enrich drug companies anyway.
The way to deal with fringe elements in all these cases is to take the extreme thinking to its logical conclusion: when you do, you are confronted with a group of people who would rather women be denied better care in order to have their worldview adopted. (Of course they would have the government do it all with higher taxes..just like in Canada where the screening and awareness levels are lower than here). Given the choice between no corporate involvement and corporate involvement along with the funding for a variety of programs that promote early detection, treatment and research, the fringe groups attacking the pink ribbon programs would take a victory over "getting rich" any time.
You’ve got a friend in Pennsylvania? Not if you’re a low-income patient who needs access to the best medical treatment for your condition.
First Health/Provider Synergies, the PBM used by the Quaker State to manage most of its Medicaid formulary decisions, has adopted a draconian new procedure that will make it almost impossible for Medicaid-eligible Pennsylvanians to get any new, cutting-edge medicines.
Astonishingly, FH/PS will give each pharmaceutical innovator one hour per year to make clinical presentations on all their products and one additional hour per year for "continuing education" on their products. This is not one hour per product; this is one hour per company!
This is a staggering demonstration of disinterest in clinical information, further demonstrating Medicaid’s broken process for choosing medicines -- more akin to a rug bazaar than proper science.
And it negatively impacts Pennsylvania’s neediest population. In a private plan, the consumer can make a choice to accept a higher co-pay for the medicines their doctor deem best for their condition. Medicaid recipients cannot. Typically, a prior authorization enforced by FH/PS makes it impossible for Medicaid recipients to get the medicine their doctors want them to have.
When cost-savings are put ahead of patient health, it’s not only bad process, it’s bad medicine.
First Health/Provider Synergies, the PBM used by the Quaker State to manage most of its Medicaid formulary decisions, has adopted a draconian new procedure that will make it almost impossible for Medicaid-eligible Pennsylvanians to get any new, cutting-edge medicines.
Astonishingly, FH/PS will give each pharmaceutical innovator one hour per year to make clinical presentations on all their products and one additional hour per year for "continuing education" on their products. This is not one hour per product; this is one hour per company!
This is a staggering demonstration of disinterest in clinical information, further demonstrating Medicaid’s broken process for choosing medicines -- more akin to a rug bazaar than proper science.
And it negatively impacts Pennsylvania’s neediest population. In a private plan, the consumer can make a choice to accept a higher co-pay for the medicines their doctor deem best for their condition. Medicaid recipients cannot. Typically, a prior authorization enforced by FH/PS makes it impossible for Medicaid recipients to get the medicine their doctors want them to have.
When cost-savings are put ahead of patient health, it’s not only bad process, it’s bad medicine.
On the heels of Steve Usdin's great piece on the outdated approach FDA critics are takig to drug safety comes an excellent piece by the Newark Star-Ledger's Kitta MacPherson.
Pretty inteilligible quote by yours truly on the failure of second-guessers to call for more funding for Critical Path and to accelerate use of biomarkers and other approaches to identify safety signals before drugs are on the market and to "tag" people before they are prescribed. For Bruce Psarty to say -- "yes, pharmacogenomics is an exciting tool but not ready for prime time so let's spend billions on even larger clinical trials and reviewers and risk management programs" is to simply make it even more difficult to create better tools. And since the IOM report does claim that the most important recommendation it makes is to INTEGRATE safety and efficacy evaluation, why is everyone suggesting steps that separate them instead of supporting science-based techniques that achieve that goal.
Here's the link to the Star-Ledger article
http://www.nj.com/search/index.ssf?/base/news-9/1160455110190710.xml?starledger?ntop&coll=1&thispage=2#continue
And one more thing: Curt Furberg's article on drug safety in the Archives of Internal Medicine.... Here's Furberg explaining his reasoning for putting a "black box" or additional risk warning on drugs for ADHD:
"On the surface, it is hard to believe( so many children really have ADHD)," said Curt Furberg, professor of public health sciences at North Carolina's Wake Forest University Medical School, who voted for the black-box warning. "What is also interesting is this condition is not really recognized in other countries -- you wonder what we are treating. I am sure there are patients who need these drugs, but it is not 10 percent of all 10-year-old boys."
In otherwords, Furberg decided to scare people away from using "these drugs" because he thinks most kids don't need them.... That follows the bogus cancer scare and Psaty ginned up regarding calcium channel blockers... And we are supposed to follow his lead on how to reform the FDA.
Pretty inteilligible quote by yours truly on the failure of second-guessers to call for more funding for Critical Path and to accelerate use of biomarkers and other approaches to identify safety signals before drugs are on the market and to "tag" people before they are prescribed. For Bruce Psarty to say -- "yes, pharmacogenomics is an exciting tool but not ready for prime time so let's spend billions on even larger clinical trials and reviewers and risk management programs" is to simply make it even more difficult to create better tools. And since the IOM report does claim that the most important recommendation it makes is to INTEGRATE safety and efficacy evaluation, why is everyone suggesting steps that separate them instead of supporting science-based techniques that achieve that goal.
Here's the link to the Star-Ledger article
http://www.nj.com/search/index.ssf?/base/news-9/1160455110190710.xml?starledger?ntop&coll=1&thispage=2#continue
And one more thing: Curt Furberg's article on drug safety in the Archives of Internal Medicine.... Here's Furberg explaining his reasoning for putting a "black box" or additional risk warning on drugs for ADHD:
"On the surface, it is hard to believe( so many children really have ADHD)," said Curt Furberg, professor of public health sciences at North Carolina's Wake Forest University Medical School, who voted for the black-box warning. "What is also interesting is this condition is not really recognized in other countries -- you wonder what we are treating. I am sure there are patients who need these drugs, but it is not 10 percent of all 10-year-old boys."
In otherwords, Furberg decided to scare people away from using "these drugs" because he thinks most kids don't need them.... That follows the bogus cancer scare and Psaty ginned up regarding calcium channel blockers... And we are supposed to follow his lead on how to reform the FDA.
The precise and prosaic Steve Usdin, Washington, DC editor of BioCentury, offers a prescient and penetrating perlustration on the impact and implications of the recent IOM report, the pending Enzi/Kennedy legislation, and their potential repercussions on the forthcoming battle over PDUFA reauthorization.
Must reading.
Here's a link to the article entitled, "Total Lifecycle Regulation" ...
Download file
Must reading.
Here's a link to the article entitled, "Total Lifecycle Regulation" ...
Download file
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