Latest Drugwonks' Blog
Here's what the FDA has said about the benefits and risks of various EPOs:
"FDA and the manufacturer of these products have agreed on revised product labeling that includes updated warnings, a new boxed warning, and modifications to the dosing instructions. The new boxed warning advises physicians to monitor red blood cell levels (hemoglobin) and to adjust the ESA dose to maintain the lowest hemoglobin level needed to avoid the need for blood transfusions. Physicians and patients should carefully weigh the risks of ESAs against transfusion risks.
Recently completed studies describe an increased risk of death, blood clots, strokes, and heart attacks in patients with chronic kidney failure when ESAs were given at higher than recommended doses. In other studies, more rapid tumor growth occurred in patients with head and neck cancer who received these higher doses.
In studies where ESAs were given at recommended doses, an increased risk of death was reported in patients with cancer who were not receiving chemotherapy and an increased risk of blood clots was observed in patients following orthopedic surgery."
Now here is how the BMJ -- what an appropriate acronym -- covers it:
"Concerns about the safety of a class of drugs used to treat anaemia have triggered a federal alert in the United States. The alert comes after recent studies reported a higher incidence of fatal cancers and cardiovascular events among patients who were treated with drugs known as erythropoiesis stimulating agents when the drugs were used to raise haemoglobin concentrations to more than 120 g/l.
The US Food and Drug Administration issued the alert on 16 February, urging doctors "not to [give doses] to exceed haemoglobin levels of 12 g/dl" when treating patients with anaemia resulting from chronic renal failure, cancer or cancer chemotherapy, or HIV or AIDS."
See how BMJ traverses from proceeding with caution to prescribing beyond a specific hemoglobin level? Notice how things have moved from an advisory to a triggering a federal alert, like some terrorist warning? And note the inconsistency in metrics. Can we stick with 120g/l or 12g/dl from one graf to the next please?
Oh and leave it BMJ to allege a plot of some sort: conflicted scientists stuffed with cash hiding results about how plying patients with EPO kills them...as if there were no independent drug safety monitoring board reviewing the study as it progresses and if there were no mechanistic reason to explore the use of EPO in other cases...
"Issues of financial conflicts of interest and unpublished data have led to considerable controversy around the drugs. The New England Journal of Medicine decided not to publish an opinion piece it commissioned from Robert Steinbrook, one of the journal's senior physician writers, who questioned whether guidelines issued by the National Kidney Foundation to achieve higher concentrations of haemoglobin may have been tainted by multimillion dollar contributions from the manufacturers of the drugs, says a report in the Wall Street Journal (26 Dec 2006, p B1)."
Ok. I'm back. Had to shower after reading THAT again.
Let me state that I have always thought that the nextgen approach to EPO dosing - or dosing of any kind -- should be of the personalized sort, finding which dose works best with what subpopulation of patients. This is the right approach for any number of reasons.
Having said that, I am so sick and tired of this allegation of bribery regarding guidelines. If anyone -- including -- the lazy author of the first WSJ article on this subject had taken the time to read the NKF involvement in guideline development with respect to dialysis (a distinction that BM-J does not make in it's J'accuse) and use of EPO type meds they would see it is as transparent as BMJ's hatred of the pharmaceutical industry.
http://www.kidney.org/PROFESSIONALS/kdoqi/nkfAdvisory.cfm
And moreover they would see that in the past Medicare reimbursement rates had kept EPO doses artificially low to the point that people were not getting sufficient hemocrit levels. Dosing is higher and so is survival. At least in anemia. We are finding that this is not always the case in other clinical situations. Absent biomarker driven or more aggressive use of observational studies, reliance on RCTs will cause us to stumble our way to better or more targeted care. That's the way science proceeds. It is not a function of malfeasance.
"FDA and the manufacturer of these products have agreed on revised product labeling that includes updated warnings, a new boxed warning, and modifications to the dosing instructions. The new boxed warning advises physicians to monitor red blood cell levels (hemoglobin) and to adjust the ESA dose to maintain the lowest hemoglobin level needed to avoid the need for blood transfusions. Physicians and patients should carefully weigh the risks of ESAs against transfusion risks.
Recently completed studies describe an increased risk of death, blood clots, strokes, and heart attacks in patients with chronic kidney failure when ESAs were given at higher than recommended doses. In other studies, more rapid tumor growth occurred in patients with head and neck cancer who received these higher doses.
In studies where ESAs were given at recommended doses, an increased risk of death was reported in patients with cancer who were not receiving chemotherapy and an increased risk of blood clots was observed in patients following orthopedic surgery."
Now here is how the BMJ -- what an appropriate acronym -- covers it:
"Concerns about the safety of a class of drugs used to treat anaemia have triggered a federal alert in the United States. The alert comes after recent studies reported a higher incidence of fatal cancers and cardiovascular events among patients who were treated with drugs known as erythropoiesis stimulating agents when the drugs were used to raise haemoglobin concentrations to more than 120 g/l.
The US Food and Drug Administration issued the alert on 16 February, urging doctors "not to [give doses] to exceed haemoglobin levels of 12 g/dl" when treating patients with anaemia resulting from chronic renal failure, cancer or cancer chemotherapy, or HIV or AIDS."
See how BMJ traverses from proceeding with caution to prescribing beyond a specific hemoglobin level? Notice how things have moved from an advisory to a triggering a federal alert, like some terrorist warning? And note the inconsistency in metrics. Can we stick with 120g/l or 12g/dl from one graf to the next please?
Oh and leave it BMJ to allege a plot of some sort: conflicted scientists stuffed with cash hiding results about how plying patients with EPO kills them...as if there were no independent drug safety monitoring board reviewing the study as it progresses and if there were no mechanistic reason to explore the use of EPO in other cases...
"Issues of financial conflicts of interest and unpublished data have led to considerable controversy around the drugs. The New England Journal of Medicine decided not to publish an opinion piece it commissioned from Robert Steinbrook, one of the journal's senior physician writers, who questioned whether guidelines issued by the National Kidney Foundation to achieve higher concentrations of haemoglobin may have been tainted by multimillion dollar contributions from the manufacturers of the drugs, says a report in the Wall Street Journal (26 Dec 2006, p B1)."
Ok. I'm back. Had to shower after reading THAT again.
Let me state that I have always thought that the nextgen approach to EPO dosing - or dosing of any kind -- should be of the personalized sort, finding which dose works best with what subpopulation of patients. This is the right approach for any number of reasons.
Having said that, I am so sick and tired of this allegation of bribery regarding guidelines. If anyone -- including -- the lazy author of the first WSJ article on this subject had taken the time to read the NKF involvement in guideline development with respect to dialysis (a distinction that BM-J does not make in it's J'accuse) and use of EPO type meds they would see it is as transparent as BMJ's hatred of the pharmaceutical industry.
http://www.kidney.org/PROFESSIONALS/kdoqi/nkfAdvisory.cfm
And moreover they would see that in the past Medicare reimbursement rates had kept EPO doses artificially low to the point that people were not getting sufficient hemocrit levels. Dosing is higher and so is survival. At least in anemia. We are finding that this is not always the case in other clinical situations. Absent biomarker driven or more aggressive use of observational studies, reliance on RCTs will cause us to stumble our way to better or more targeted care. That's the way science proceeds. It is not a function of malfeasance.
Is cigarette smoking deleterious to America's health. Absolutely. Should Americans who currently smoke quit? Absolutely. Should the FDA regulate tobacco products? Absolutely not.
Pending legislation (Kennedy-Cornyn and Waxman-Davis) aims to give the FDA broad authority over tobacco products, providing the agency with oversight over virtually every aspect of tobacco company operations. The knee-jerk reaction is "great!" But, when you ask ""Would such legislation result in fewer smokers and reduced death and debilitation from tobacco? the answer is very unclear -- with the weight of logic and evidence saying otherwise.
For example, proposed legislation would set a very high bar (both scientific and procedural) before the FDA could approve a claim of "modified risk." The impact here would be to reduce any tobacco company's ability (or, most probably, desire) to promote their brands that are lower in nicotine content or, indeed, to even develop such products.
Or consider this, adult smoking has been declining since 1997 -- due to a number of things including clean air laws, media campaigns, and youth access programs. Who did these things? Largely the states. If FDA became the nation's tobacco czar, it would become difficult if not impossible to convince state legislators to continue to allocate the funds required for robust state-level tobacco control programs.
And then, of course, there's the question of both FDA resources and expertise. Let's take the latter first. What is the current level of FDA expertise in tobacco regulation? Let me put it this way -- none. As far as resources are concerned, the FDA's tobacco program would be funded by user fees. And, considering the current PDUFA-IV negotiations, you have to ask yourself if this is really the way we want to be going.
So, when you consider all of these issues (and there are many others)the answer to "Will FDA regulation of tobacco help to reduce tobacco use in America?" is very much an open one.
So for now, thank you for not regulating.
FYI -- the Center for Medicine in the Public Interest (the sponsor of drugwonks.com) does not accept funding from the tobacco industry.
Pending legislation (Kennedy-Cornyn and Waxman-Davis) aims to give the FDA broad authority over tobacco products, providing the agency with oversight over virtually every aspect of tobacco company operations. The knee-jerk reaction is "great!" But, when you ask ""Would such legislation result in fewer smokers and reduced death and debilitation from tobacco? the answer is very unclear -- with the weight of logic and evidence saying otherwise.
For example, proposed legislation would set a very high bar (both scientific and procedural) before the FDA could approve a claim of "modified risk." The impact here would be to reduce any tobacco company's ability (or, most probably, desire) to promote their brands that are lower in nicotine content or, indeed, to even develop such products.
Or consider this, adult smoking has been declining since 1997 -- due to a number of things including clean air laws, media campaigns, and youth access programs. Who did these things? Largely the states. If FDA became the nation's tobacco czar, it would become difficult if not impossible to convince state legislators to continue to allocate the funds required for robust state-level tobacco control programs.
And then, of course, there's the question of both FDA resources and expertise. Let's take the latter first. What is the current level of FDA expertise in tobacco regulation? Let me put it this way -- none. As far as resources are concerned, the FDA's tobacco program would be funded by user fees. And, considering the current PDUFA-IV negotiations, you have to ask yourself if this is really the way we want to be going.
So, when you consider all of these issues (and there are many others)the answer to "Will FDA regulation of tobacco help to reduce tobacco use in America?" is very much an open one.
So for now, thank you for not regulating.
FYI -- the Center for Medicine in the Public Interest (the sponsor of drugwonks.com) does not accept funding from the tobacco industry.
In another sign that the Europe is shrinking by virtue of self-inflicted acts of self-loathing and irrelevancy...
BERLIN (Reuters) -
"European Union consumer chief Meglena Kuneva has hit out at Apple Inc.'s (Nasdaq:AAPL - news) bundling of its popular iPod music players and its iTunes online music store, according to German weekly magazine Focus.
"Do you think it's fine that a CD plays in all CD players but that an iTunes song only plays in an iPod? I don't. Something has to change," EU Consumer Protection Commissioner Kuneva was quoted as saying in a preview of an interview to be published on Monday."
http://news.yahoo.com/s/nm/20070311/tc_nm/eu_apple_dc
The last time I was on-line I found tons of music sites where you can download music that play on any number of MP3 players. So what's the problem?
What has to change is the EU's attack on anything that is both innovative and American.
Like new drugs, Ipods, Microsoft, etc. Too bad American innovators don't join forces HERE to stop the assault on innovation in Congress and in Europe. Self-loathing you see, is a global epidemic.
BERLIN (Reuters) -
"European Union consumer chief Meglena Kuneva has hit out at Apple Inc.'s (Nasdaq:AAPL - news) bundling of its popular iPod music players and its iTunes online music store, according to German weekly magazine Focus.
"Do you think it's fine that a CD plays in all CD players but that an iTunes song only plays in an iPod? I don't. Something has to change," EU Consumer Protection Commissioner Kuneva was quoted as saying in a preview of an interview to be published on Monday."
http://news.yahoo.com/s/nm/20070311/tc_nm/eu_apple_dc
The last time I was on-line I found tons of music sites where you can download music that play on any number of MP3 players. So what's the problem?
What has to change is the EU's attack on anything that is both innovative and American.
Like new drugs, Ipods, Microsoft, etc. Too bad American innovators don't join forces HERE to stop the assault on innovation in Congress and in Europe. Self-loathing you see, is a global epidemic.
The copy biologics brigade got a dose of reality yesterday. Turns out all those drugs they would just deem interchangeable aren't and all the biologics they could mix up in Jo Ann Emerson's bath tub like fertilizer need a bit of testing. An excellent article by Diedtra Henderson of the Boston Globe on the recent FOB hearing in the Senate HELP committee shows that for some folks it money first and safety second...
http://www.boston.com/business/healthcare/articles/2007/03/09/biotechs_nod_as_senators_urge_caution_on_generics/
http://www.boston.com/business/healthcare/articles/2007/03/09/biotechs_nod_as_senators_urge_caution_on_generics/
Great piece in Technology Review about how bioinformatics wil lead to tailored combos of drugs to treat diseases according to who were are and what we need...
"A biotech company called CombinatoRx has found that at the right doses, thousands of counterintuitive drug pairs are synergistic. The Cambridge, MA, company has eight drug combinations in clinical trials and several more in preclinical development. In a few years, diabetics, instead of injecting insulin, might be prescribed a cholesterol drug and a pain medication to help control their blood sugar. People suffering from chronic pain might find relief through a combination of a steroid and an antidepressant, with fewer side effects than they experience with current therapies.
Alexis Borisy, founder and CEO of the company, says his researchers take a brute-force approach to finding fruitful drug combinations. In the lab, they test combinations of several thousand drugs at several different doses on cellular models of diseases including cancer and arthritis--regardless of what diseases the drugs are currently approved for, if any. Then they feed the data into software that looks for synergies."
The entire article and link thereof can be found here:
http://www.technologyreview.com/Biotech/18280/
Just one problem....for every combo you will need.....a Risk management plan under the enlightened regime of Kennedy Enzi. Otherwise you and your doctor will be subject to some sort of liabiity and likely be denied reimbursement by an equally enlightened insurance or health plan.
Meanwhile Big Pharma lobbyists work with the staffers in Enzi and Kennedy's office for minor changes in the bill. Don't they realize that in a matter of days the Waxmaniac is going to drop his mutated version of Kenzi, complete with a comparative effectiveness study requirement?
What's counterintuitive is that people are still failing to ask just how Kenzi will affect access to lifesaving medicines and cripple the doctor patient relationship...
"A biotech company called CombinatoRx has found that at the right doses, thousands of counterintuitive drug pairs are synergistic. The Cambridge, MA, company has eight drug combinations in clinical trials and several more in preclinical development. In a few years, diabetics, instead of injecting insulin, might be prescribed a cholesterol drug and a pain medication to help control their blood sugar. People suffering from chronic pain might find relief through a combination of a steroid and an antidepressant, with fewer side effects than they experience with current therapies.
Alexis Borisy, founder and CEO of the company, says his researchers take a brute-force approach to finding fruitful drug combinations. In the lab, they test combinations of several thousand drugs at several different doses on cellular models of diseases including cancer and arthritis--regardless of what diseases the drugs are currently approved for, if any. Then they feed the data into software that looks for synergies."
The entire article and link thereof can be found here:
http://www.technologyreview.com/Biotech/18280/
Just one problem....for every combo you will need.....a Risk management plan under the enlightened regime of Kennedy Enzi. Otherwise you and your doctor will be subject to some sort of liabiity and likely be denied reimbursement by an equally enlightened insurance or health plan.
Meanwhile Big Pharma lobbyists work with the staffers in Enzi and Kennedy's office for minor changes in the bill. Don't they realize that in a matter of days the Waxmaniac is going to drop his mutated version of Kenzi, complete with a comparative effectiveness study requirement?
What's counterintuitive is that people are still failing to ask just how Kenzi will affect access to lifesaving medicines and cripple the doctor patient relationship...
Here's the lead from a story that ran earlier this week in the Philadelphia Inquirer on a new study showing that nearly 80 percent of the children cared for at academic children's hospitals got at least one medicine outside the age parameters approved by the FDA:
"Most children treated at major pediatric hospitals are given medicines not approved by the Food and Drug Administration for use in patients so young. The study, in today's Archives of Pediatrics & Adolescent Medicine, found that the sickest children and those undergoing surgery were most likely to get a so-called off-label drug. But altogether, nearly 80 percent of the children cared for at academic children's hospitals got at least one medicine outside the age parameters approved by the FDA."
Why is this so? Well it's because of the big, bad, evil pharmaceutical industry don't ya know:
"The risk and benefits of many drugs in children are poorly studied, often because the drugmakers had little financial incentive to do so."
Well, not precisely. It's not the financial incentives so much as the difficulty relative to the return. I know, that sounds like verbal gymnastics. Try this -- the real issue is that FDA permitted pediatric clinical trial design makes it difficult if not impossible to precisely determine which medicines in what dosages work in specific circumstances in specific pediatric patient populations. In other words, if personalized medicine in adults is difficult, in children it is nearly impossible.
Yet another important reason for the FDA to embrace adaptive clinical trial design and to become a research hub for 21st century clinical trial design. And that, of course, is part of what makes the Critical Path program so, well, critical to the future of America's health.
Here's a link to the Inquirer article:
http://www.philly.com/mld/inquirer/16841956.htm
According to Samir S. Shah, the study's lead author and infectious-disease doctor at Children's Hospital of Philadelphia, "We don't know whether in the absence of off-label use there would have been fewer deaths or more deaths. I suspect that in many instances the drugs were beneficial and in a smaller number of cases the drugs were harmful."
That's good news. What would be even better news would be getting the right drug in the right dose to the right child at the right time based on sound science and under the FDA's imprimatur.
Tempus fugit.
"Most children treated at major pediatric hospitals are given medicines not approved by the Food and Drug Administration for use in patients so young. The study, in today's Archives of Pediatrics & Adolescent Medicine, found that the sickest children and those undergoing surgery were most likely to get a so-called off-label drug. But altogether, nearly 80 percent of the children cared for at academic children's hospitals got at least one medicine outside the age parameters approved by the FDA."
Why is this so? Well it's because of the big, bad, evil pharmaceutical industry don't ya know:
"The risk and benefits of many drugs in children are poorly studied, often because the drugmakers had little financial incentive to do so."
Well, not precisely. It's not the financial incentives so much as the difficulty relative to the return. I know, that sounds like verbal gymnastics. Try this -- the real issue is that FDA permitted pediatric clinical trial design makes it difficult if not impossible to precisely determine which medicines in what dosages work in specific circumstances in specific pediatric patient populations. In other words, if personalized medicine in adults is difficult, in children it is nearly impossible.
Yet another important reason for the FDA to embrace adaptive clinical trial design and to become a research hub for 21st century clinical trial design. And that, of course, is part of what makes the Critical Path program so, well, critical to the future of America's health.
Here's a link to the Inquirer article:
http://www.philly.com/mld/inquirer/16841956.htm
According to Samir S. Shah, the study's lead author and infectious-disease doctor at Children's Hospital of Philadelphia, "We don't know whether in the absence of off-label use there would have been fewer deaths or more deaths. I suspect that in many instances the drugs were beneficial and in a smaller number of cases the drugs were harmful."
That's good news. What would be even better news would be getting the right drug in the right dose to the right child at the right time based on sound science and under the FDA's imprimatur.
Tempus fugit.
Compare this to the banshee cry of predetermined interchangeability as advanced by Waxman and the self-serving Express Scripts savings estimates from follow ons
I welcome the members of our committee and our distinguished witnesses to today’s hearing on the important question of whether Congress should give FDA the authority to approve follow-on versions of biologic medicines.
We are in a remarkable period of discovery in the life sciences. Unprecedented advances are taking place, and patients have already begun to see the benefits of this new era through new wonder drugs that can make the difference between life and death for patients afflicted with serious illnesses.
Patients with leukemia who once faced a bleak future now have new hope, thanks to an extraordinary new medicine that can slow or even halt the progression of the disease.
Until recently, a diagnosis of Gaucher’s [“go-SHAYSâ€] Disease meant a shorter life, full of disability and pain for the people it afflicted. Now, a remarkable breakthrough has produced drugs to treat this grave illness and extend life and reduce disability.
Similarly, a drug to stimulate the production of new blood cells is helping patients counteract the severe anemia caused by chemotherapy or renal disease.
These miracle medicines, called biologics, are complex molecules whose healing power has been brought to patients by dynamic biotechnology companies. Such drugs were once a rarity in the medical arsenal, but each day seems to bring new hope from new breakthrough biologics.
With this extraordinary progress comes a challenge to public policy. Due to the cost of developing and manufacturing new biologics, their price is often steep. They can cost patients tens or even hundreds of thousands of dollars a year, putting an extraordinary strain on the budgets of those who must pay the bills –patients, insurers and companies, or government programs.
Congress has faced similar challenges before. In the early 1980's, the cost of prescription drugs was spiraling upward. In response, Congress enacted legislation that balanced the need to reduce costs for consumers through increased competition with the requirement to promote innovation. That legislation is known universally by the names of its sponsors, Senator Orrin Hatch and Representative Henry Waxman. Our committee is honored that Senator Hatch is helping guide our deliberations. Congress and the American people are indebted to his leadership on these important issues.
When the Hatch-Waxman law was enacted, Congress did not include biologics, because at the time such drugs were not providing the major innovations that advances in the biological sciences have brought over the past 20 years.
Now Congress must consider whether to authorize FDA to accept applications for follow-on versions of these path breaking medicines.
The stakes riding on the answer to this question are enormous, both for patients and for our economy, and the interest among our committee colleagues in this question is intense. One of our colleagues, Senator Clinton, has a proposal to allow FDA to approve follow-on biologics. I look forward to hearing her views on this question, and to receiving the testimony of the legislation’s co-sponsor, Senator Schumer.
Many have recommended that the committee’s legislation on drug safety and user fees should include a proposal to allow for follow-on biologics. Today’s hearing will help to provide the information the committee needs to make the right decision on that important question.
Our committee should be guided by three basic principles.
First, we must be led by science. Acceptable legislation on follow-on biologics must not pre-judge science, but should enable the FDA to make the best decisions based on the most complete science reasonably available.
Second, protecting patient safety is essential. Congress must make certain that any drug given to patients – whether a conventional drug, an innovative biologic, or a follow-on product – is safe and effective.
Third, innovation must be valued and promoted. Just as it is essential to help patients afford the medicines of today, so too it is vital to provide incentives for the innovations that will bring the medical miracles of tomorrow.
I look forward to the recommendations and insights of our distinguished witnesses to provide guidance to our committee as we undertake these important deliberations.
I welcome the members of our committee and our distinguished witnesses to today’s hearing on the important question of whether Congress should give FDA the authority to approve follow-on versions of biologic medicines.
We are in a remarkable period of discovery in the life sciences. Unprecedented advances are taking place, and patients have already begun to see the benefits of this new era through new wonder drugs that can make the difference between life and death for patients afflicted with serious illnesses.
Patients with leukemia who once faced a bleak future now have new hope, thanks to an extraordinary new medicine that can slow or even halt the progression of the disease.
Until recently, a diagnosis of Gaucher’s [“go-SHAYSâ€] Disease meant a shorter life, full of disability and pain for the people it afflicted. Now, a remarkable breakthrough has produced drugs to treat this grave illness and extend life and reduce disability.
Similarly, a drug to stimulate the production of new blood cells is helping patients counteract the severe anemia caused by chemotherapy or renal disease.
These miracle medicines, called biologics, are complex molecules whose healing power has been brought to patients by dynamic biotechnology companies. Such drugs were once a rarity in the medical arsenal, but each day seems to bring new hope from new breakthrough biologics.
With this extraordinary progress comes a challenge to public policy. Due to the cost of developing and manufacturing new biologics, their price is often steep. They can cost patients tens or even hundreds of thousands of dollars a year, putting an extraordinary strain on the budgets of those who must pay the bills –patients, insurers and companies, or government programs.
Congress has faced similar challenges before. In the early 1980's, the cost of prescription drugs was spiraling upward. In response, Congress enacted legislation that balanced the need to reduce costs for consumers through increased competition with the requirement to promote innovation. That legislation is known universally by the names of its sponsors, Senator Orrin Hatch and Representative Henry Waxman. Our committee is honored that Senator Hatch is helping guide our deliberations. Congress and the American people are indebted to his leadership on these important issues.
When the Hatch-Waxman law was enacted, Congress did not include biologics, because at the time such drugs were not providing the major innovations that advances in the biological sciences have brought over the past 20 years.
Now Congress must consider whether to authorize FDA to accept applications for follow-on versions of these path breaking medicines.
The stakes riding on the answer to this question are enormous, both for patients and for our economy, and the interest among our committee colleagues in this question is intense. One of our colleagues, Senator Clinton, has a proposal to allow FDA to approve follow-on biologics. I look forward to hearing her views on this question, and to receiving the testimony of the legislation’s co-sponsor, Senator Schumer.
Many have recommended that the committee’s legislation on drug safety and user fees should include a proposal to allow for follow-on biologics. Today’s hearing will help to provide the information the committee needs to make the right decision on that important question.
Our committee should be guided by three basic principles.
First, we must be led by science. Acceptable legislation on follow-on biologics must not pre-judge science, but should enable the FDA to make the best decisions based on the most complete science reasonably available.
Second, protecting patient safety is essential. Congress must make certain that any drug given to patients – whether a conventional drug, an innovative biologic, or a follow-on product – is safe and effective.
Third, innovation must be valued and promoted. Just as it is essential to help patients afford the medicines of today, so too it is vital to provide incentives for the innovations that will bring the medical miracles of tomorrow.
I look forward to the recommendations and insights of our distinguished witnesses to provide guidance to our committee as we undertake these important deliberations.
Drug Safety Briefing: "Drug Safety: Defining Safe"
On March 21st, Friends will host an educational congressional briefing on Capitol Hill entitled “Drug Safety: Defining Safe.†Featuring introductory remarks from Representative Diana DeGette (D-CO), the briefing will include expert panelists Dr. Robert Young, President of Fox Chase Cancer Center; Dr. Mark McClellan, Former Administrator of CMS & Former Commissioner of FDA; Dr. Janet Woodcock, Deputy Commissioner and Chief Medical Officer of FDA; and patient advocate Dan Perry, Executive Director of The Alliance for Aging. Susan Dentzer of The NewsHour with Jim Lehrer on PBS will moderate. For more information, please contact Heather Chaney at hchaney@focr.org or (703) 302-1540
On March 21st, Friends will host an educational congressional briefing on Capitol Hill entitled “Drug Safety: Defining Safe.†Featuring introductory remarks from Representative Diana DeGette (D-CO), the briefing will include expert panelists Dr. Robert Young, President of Fox Chase Cancer Center; Dr. Mark McClellan, Former Administrator of CMS & Former Commissioner of FDA; Dr. Janet Woodcock, Deputy Commissioner and Chief Medical Officer of FDA; and patient advocate Dan Perry, Executive Director of The Alliance for Aging. Susan Dentzer of The NewsHour with Jim Lehrer on PBS will moderate. For more information, please contact Heather Chaney at hchaney@focr.org or (703) 302-1540
Henry Waxman sent a warm letter of solidarity when Thailand's military junta seized the patents of HIV drugs recently. The number of pharmaceutical patents rose to 11 and the range of products that the Thai government's drug company -- which has never been able to make WHO quality medicines -- would copy has expanded to include cancer and heart disease in the wake of Waxman's love letter.
Nothing like appeasement to embolden a global bully. In the January board meeting of the WHO, Thailand 's representative, Dr. Suwit Wibulpolprasert, declared that if an influenza pandemic hit, he'd counsel Bangkok to hold Western tourists hostage until those countries gave Thailand the necessary vaccines.
And if they didn't?
I wonder what sort of letter Waxman is planning to write now.
Nothing like appeasement to embolden a global bully. In the January board meeting of the WHO, Thailand 's representative, Dr. Suwit Wibulpolprasert, declared that if an influenza pandemic hit, he'd counsel Bangkok to hold Western tourists hostage until those countries gave Thailand the necessary vaccines.
And if they didn't?
I wonder what sort of letter Waxman is planning to write now.
Here's what Susan Horn, one of our board members, an one of the world's experts on the science of improving outcomes in healthcare has to say about the crushing limiting randomized clinical trials have had on the quality of care for veterans with traumatic brain injury. It has implications not only for what is happening through the VA hospital system but for those who would use RiskMap as a one size fits all conduit for determining who gets access to medicines:
" The 1998 NIH consensus statement (about treating traumatic brain injury) acknowledged that individually tailored treatments provided within the context of acute rehabilitation create difficulties for efficacy studies. “This personalized approach leads to great difficulty in the scientific evaluation of effectiveness, because there is significant heterogeneity among persons with TBI and their comprehensive treatment programsâ€. The current level of evidence limits our ability to make firm decisions about the best therapy interventions, intensities, durations, or staffing characteristics for inpatient TBI rehabilitation. Also, randomized clinical trials severely limit the number of interventions that can be tested at any one time. Randomized trials attempt to examine an intervention in isolation from other interventions in order to detect the unique contribution to recovery of one or at best a few variables."
" The 1998 NIH consensus statement (about treating traumatic brain injury) acknowledged that individually tailored treatments provided within the context of acute rehabilitation create difficulties for efficacy studies. “This personalized approach leads to great difficulty in the scientific evaluation of effectiveness, because there is significant heterogeneity among persons with TBI and their comprehensive treatment programsâ€. The current level of evidence limits our ability to make firm decisions about the best therapy interventions, intensities, durations, or staffing characteristics for inpatient TBI rehabilitation. Also, randomized clinical trials severely limit the number of interventions that can be tested at any one time. Randomized trials attempt to examine an intervention in isolation from other interventions in order to detect the unique contribution to recovery of one or at best a few variables."
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.
