Latest Drugwonks' Blog
A more complete response to the politically motivated, mean spirited, and just plain wrong comments by the so-called Union of Concerned Scientists ...
http://www.tjols.com/article-350.html
Here are some snippets:
* Twenty-five years ago, the success rate for a new drug used was about 14 percent. Today, a new medicinal compound entering early-stage testing – often after more than a decade of pre-clinical screening and evaluation – is estimated to have only an 8 percent chance of reaching the market. For very innovative and unproven technologies, the probability of an individual product's success is even lower.
* Better, more current and predictable scientific research and standards must be developed and devoted to streamlining the critical path. Investment in basic research is not enough. Specifically new development tools are needed to improve the predictability of the drug development cycle and to lower the cost of research by helping industry identify product failures earlier in the clinical trials process.
* New development tools in these areas will enable better through-put to commercial product development and will act as a productivity multiplier, increasing the returns on public and private investment in basic research. With improved scientific methods and a new, shared effort by all of us, we can develop and improve standards for product characterization and product safety testing, for both traditional and innovative products.
* Today only about 1 percent of the proteins in blood have been identified. Of that 1 percent only a fifth has FDA approved diagnostic utility. These proteins, after we understand them, could help predict disease remission. Currently academics and private companies collect data and establish correlations, but no one is responsible for organizing this information into the broader knowledge that could lead to generalized principles industry and FDA could use for broader, faster, and more accurate product evaluation.
* Think about the millions of dollars that would be saved by all types and sizes of companies and governments if publicly discussed and vetted biomarkers could be used and used predictably in the drug approval process. Using the lower end of the Tufts drug development number, a 10 percent improvement in predicting failure before clinical trials could save $100 million in development costs. Similarly, shifting 5 percent of clinical failures from late-stage to early-stage trials reduces out of pocket costs by $15-$20 million.
Next up on the Critical Path hit parade -- who will be named to the Reagan/Udall board of directors. Watch this space for more details.
http://www.tjols.com/article-350.html
Here are some snippets:
* Twenty-five years ago, the success rate for a new drug used was about 14 percent. Today, a new medicinal compound entering early-stage testing – often after more than a decade of pre-clinical screening and evaluation – is estimated to have only an 8 percent chance of reaching the market. For very innovative and unproven technologies, the probability of an individual product's success is even lower.
* Better, more current and predictable scientific research and standards must be developed and devoted to streamlining the critical path. Investment in basic research is not enough. Specifically new development tools are needed to improve the predictability of the drug development cycle and to lower the cost of research by helping industry identify product failures earlier in the clinical trials process.
* New development tools in these areas will enable better through-put to commercial product development and will act as a productivity multiplier, increasing the returns on public and private investment in basic research. With improved scientific methods and a new, shared effort by all of us, we can develop and improve standards for product characterization and product safety testing, for both traditional and innovative products.
* Today only about 1 percent of the proteins in blood have been identified. Of that 1 percent only a fifth has FDA approved diagnostic utility. These proteins, after we understand them, could help predict disease remission. Currently academics and private companies collect data and establish correlations, but no one is responsible for organizing this information into the broader knowledge that could lead to generalized principles industry and FDA could use for broader, faster, and more accurate product evaluation.
* Think about the millions of dollars that would be saved by all types and sizes of companies and governments if publicly discussed and vetted biomarkers could be used and used predictably in the drug approval process. Using the lower end of the Tufts drug development number, a 10 percent improvement in predicting failure before clinical trials could save $100 million in development costs. Similarly, shifting 5 percent of clinical failures from late-stage to early-stage trials reduces out of pocket costs by $15-$20 million.
Next up on the Critical Path hit parade -- who will be named to the Reagan/Udall board of directors. Watch this space for more details.
A letter from the FDA to Peter Stark and Henry Waxman lies about the benefits of EPO.
It claims that there no quality of life benefits associated with use of EPO among cancer patients undergoing chemotherapy.
That is a lie. Andy von Eschenbach should -- if he is around -- as a cancer patient and oncologist have the guts to squash Richard Padzur's assault on EPO and stand up for the truth.
The FDA has even allowed Amgen to make QOL claims on the label. The current label for EPO allows for QOL benefits. And just because the FDA does not allow them because of Padzur's pique does not make it so. There will likely be revisions but not a total rejection.
So the claim there is no QOL benefit approved by the FDA is also a lie. The fact that the FDA sees no benefit in terms of survival at a biological level does not mean such a correlation fails to exist overall. Cancer death rates are down. They are down because of earlier screening and better drugs. Better drugs can only be used because of a reduction in RBC and a corresponding optimization of hemoglobin levels. Take away ESAs and guess what happens?
Why would the FDA capitulate to Stark and Waxman?
Where is the leadership? Politics as usual should not apply when lives are on the line. Stark, Waxman and Padzur are distorting science and -- working together -- seeking to gain control over regulatory processes for political purposes. Where is the courage to put a stop to it.
Andy, your phone is ringing.
It claims that there no quality of life benefits associated with use of EPO among cancer patients undergoing chemotherapy.
That is a lie. Andy von Eschenbach should -- if he is around -- as a cancer patient and oncologist have the guts to squash Richard Padzur's assault on EPO and stand up for the truth.
The FDA has even allowed Amgen to make QOL claims on the label. The current label for EPO allows for QOL benefits. And just because the FDA does not allow them because of Padzur's pique does not make it so. There will likely be revisions but not a total rejection.
So the claim there is no QOL benefit approved by the FDA is also a lie. The fact that the FDA sees no benefit in terms of survival at a biological level does not mean such a correlation fails to exist overall. Cancer death rates are down. They are down because of earlier screening and better drugs. Better drugs can only be used because of a reduction in RBC and a corresponding optimization of hemoglobin levels. Take away ESAs and guess what happens?
Why would the FDA capitulate to Stark and Waxman?
Where is the leadership? Politics as usual should not apply when lives are on the line. Stark, Waxman and Padzur are distorting science and -- working together -- seeking to gain control over regulatory processes for political purposes. Where is the courage to put a stop to it.
Andy, your phone is ringing.
After Alex Berenson's sob story about the growing dental health crisis in America due to greedy dentists we have this from that health care paradise, Great Britain.
English 'pull own teeth' as dental service decays
Mon Oct 15, 7:19 AM ET
LONDON (AFP) - Falling numbers of state dentists in England has led to some people taking extreme measures, including extracting their own teeth, according to a new study released Monday.
Falling numbers of state dentists in England has led to some people taking extreme measures, including extracting their own teeth, according to a new study released Monday."
Maybe Jon Cohn of TNR wants to nibble on this one. I thought universal coverage made health care more accessible and affordable. (Don't forget the UK has nearly doubled spending on health care in the past decade so don't use the resource problem as an excuse.) When you pay people nothing and make services free you wind up with people expecting everything and getting nothing.
http://news.yahoo.com/s/afp/20071015/wl_uk_afp/britainhealthdentists
English 'pull own teeth' as dental service decays
Mon Oct 15, 7:19 AM ET
LONDON (AFP) - Falling numbers of state dentists in England has led to some people taking extreme measures, including extracting their own teeth, according to a new study released Monday.
Falling numbers of state dentists in England has led to some people taking extreme measures, including extracting their own teeth, according to a new study released Monday."
Maybe Jon Cohn of TNR wants to nibble on this one. I thought universal coverage made health care more accessible and affordable. (Don't forget the UK has nearly doubled spending on health care in the past decade so don't use the resource problem as an excuse.) When you pay people nothing and make services free you wind up with people expecting everything and getting nothing.
http://news.yahoo.com/s/afp/20071015/wl_uk_afp/britainhealthdentists
The Republicans are winning the battle on SCHIP and should not mistake the carpet bombing from the Left as a sign they are in trouble. It used the same tactics to run down Part D and now they see that in politics it is hard to add something, even harder to take it away. More to the point, Americans know when their tax dollars are being wasted. Part D to help seniors and reduce hospital visits, yes. Government subsidies and government run health plans to middle income families who could get insurance on their own, no.
But look at how USA Today tries to spin the war of ideas as a war of attrition against the GOP and Bush....
"Slim majorities back two positions at the core of the president's opposition to the expansion:
•52% agree with Bush that most benefits should go to children in families earning less than 200% of the federal poverty level — about $41,000 for a family of four. Only 40% say benefits should go to such families earning up to $62,000, as the bill written by Democrats and some Republicans would allow.
•55% are very or somewhat concerned that the program would create an incentive for families to drop private insurance. Bush and Republican opponents have called that a step toward government-run health care.
Taken together, the results show that while Bush may be losing the political battle with Democrats, he may be doing better on policy."
Slim majorities? I bet 50 dollars that if the poll was on Iraq and the same numbers signaled for withdrawal the adjective "slim" would nowhere to be found.
How can you lose the policy battle and not the political battle . Who said anything about losing? See how that assumption just...crowds out the reality?
PS
The poll should have asked about the precedent settting $83K with no asset test ripoff and the numbers would have been lower. And all this despite the media painting the contest as one of Bush vs poor kids.
http://www.usatoday.com/printedition/news/20071016/a_chippoll16.art.htm
But look at how USA Today tries to spin the war of ideas as a war of attrition against the GOP and Bush....
"Slim majorities back two positions at the core of the president's opposition to the expansion:
•52% agree with Bush that most benefits should go to children in families earning less than 200% of the federal poverty level — about $41,000 for a family of four. Only 40% say benefits should go to such families earning up to $62,000, as the bill written by Democrats and some Republicans would allow.
•55% are very or somewhat concerned that the program would create an incentive for families to drop private insurance. Bush and Republican opponents have called that a step toward government-run health care.
Taken together, the results show that while Bush may be losing the political battle with Democrats, he may be doing better on policy."
Slim majorities? I bet 50 dollars that if the poll was on Iraq and the same numbers signaled for withdrawal the adjective "slim" would nowhere to be found.
How can you lose the policy battle and not the political battle . Who said anything about losing? See how that assumption just...crowds out the reality?
PS
The poll should have asked about the precedent settting $83K with no asset test ripoff and the numbers would have been lower. And all this despite the media painting the contest as one of Bush vs poor kids.
http://www.usatoday.com/printedition/news/20071016/a_chippoll16.art.htm
On September 23, 2005 we blogged on a grandstanding measure put forward in the DC City Council by David Catania — a lawyer at the mega-DC firm of Akin, Gump, Strauss, Hauer, Feld (“What does DC stand for?â€).
(Mr. Catania is also known by many inside-the-Beltway as “Rahm Emanuel, Jr.")
At the time Councilman Catania was calling for pharmaceutical price control legislation in the District of Columbia (since overturned in court). This prompted drugwonks to ask just what “DC†stood for. We posited that it stood for “drug catastrophe.â€
Well, Mr. Catania is at it again.
This time he’s proposing the District of Columbia “Safe Rx Act of 2007.†Besides the fact that many of its provisions are illegal (perhaps Mr. Catania should have asked one of his junior associates to do some background work), what’s most important is that the bill is contrary to both the public health and to common sense.
In other words, it’s all about politics.
I know – shocking.
Some sample content:
* Any person who educates physicians without a license shall be subject to a fine of $10,000. (This is in the section that calls for the licensing of pharmaceutical representatives.)
What about free speech, Mr. Catania? Were you absent during that law school lecture? And what about the fact that the FDA already regulates all educational efforts between physicians and sales reps? Inconvenient truths.
Also, nothing in the bill requires that evidence based “detailers†be held to any of the same standards as pharmaceutical representatives.
* The DC Department of Health will create a “Pharmaceutical Education Fund†for the “sole purpose of establishing and funding an evidence-based research, outreach, and education program within the DC DoH designed to provide information on the therapeutic and cost-effective utilization of prescription drugs.
Gadzooks, a DC DERP. Gee – I wonder who Mr. Catania has been talking to inside the majority Congressional leadership. Do the people of the District of Columbia really want cost-based rather than patient-centric care by statute?
* Pharmaceutical companies must disclose specific information on any clinical trials related to any drug or biologic product sold, delivered, dispensed, offered for sale, or given away in the District.
Oops again. Looks like Mr. Catania missed The FDA Amendments Act of 2007 (FDAAA), passed in September 2007, which expands the existing federal clinical trial registry administered by NIH by including more trials, more comprehensive information on each trial, and establishing a clinical trial results databank
Once rules for the federal expanded clinical trial registry and results database are promulgated the federal law will preempt any state clinical trial registry or results requirements.
Sloppy legal prep, counselor.
* Pharmaceutical companies must provide DC with FDA correspondence.
Um, all warning and untitled letters are already posted on the FDA website. But, hey, demanding such already publicly available letter it makes for good headlines, right?
Here’s my favorite …
* The bill requires prescribers to obtain written informed consent from patients when prescribing a prescription medication for a medically accepted indication.
So much for the concept of the “learned intermediary.â€
And as far as off-label use is concerned …
* Prescriber must explain to the patient that the medication is being prescribed outside of the indications for that medication as approved by the FDA and provide the patient with information commonly known by the medical profession regarding potential risks and side-effects
Um, according to the Medicaid statute medications prescribed for a medically accepted indication may only be restricted if, based on the compendia, the drug does not have a significant, clinically meaningful therapeutic advantage in terms of safety, effectiveness, or clinical outcome.
So much is wrong about so many things in this bill that it makes H.R. 380 (Rahm Emanuel’s “Pharmaceutical Market Access and Drug Safety Actâ€) look like the next coming of the Magna Carta.
“DC†may mean “David Catania,†but it mustn’t be allowed to mean “Drug Catastrophe†-- again.
(Mr. Catania is also known by many inside-the-Beltway as “Rahm Emanuel, Jr.")
At the time Councilman Catania was calling for pharmaceutical price control legislation in the District of Columbia (since overturned in court). This prompted drugwonks to ask just what “DC†stood for. We posited that it stood for “drug catastrophe.â€
Well, Mr. Catania is at it again.
This time he’s proposing the District of Columbia “Safe Rx Act of 2007.†Besides the fact that many of its provisions are illegal (perhaps Mr. Catania should have asked one of his junior associates to do some background work), what’s most important is that the bill is contrary to both the public health and to common sense.
In other words, it’s all about politics.
I know – shocking.
Some sample content:
* Any person who educates physicians without a license shall be subject to a fine of $10,000. (This is in the section that calls for the licensing of pharmaceutical representatives.)
What about free speech, Mr. Catania? Were you absent during that law school lecture? And what about the fact that the FDA already regulates all educational efforts between physicians and sales reps? Inconvenient truths.
Also, nothing in the bill requires that evidence based “detailers†be held to any of the same standards as pharmaceutical representatives.
* The DC Department of Health will create a “Pharmaceutical Education Fund†for the “sole purpose of establishing and funding an evidence-based research, outreach, and education program within the DC DoH designed to provide information on the therapeutic and cost-effective utilization of prescription drugs.
Gadzooks, a DC DERP. Gee – I wonder who Mr. Catania has been talking to inside the majority Congressional leadership. Do the people of the District of Columbia really want cost-based rather than patient-centric care by statute?
* Pharmaceutical companies must disclose specific information on any clinical trials related to any drug or biologic product sold, delivered, dispensed, offered for sale, or given away in the District.
Oops again. Looks like Mr. Catania missed The FDA Amendments Act of 2007 (FDAAA), passed in September 2007, which expands the existing federal clinical trial registry administered by NIH by including more trials, more comprehensive information on each trial, and establishing a clinical trial results databank
Once rules for the federal expanded clinical trial registry and results database are promulgated the federal law will preempt any state clinical trial registry or results requirements.
Sloppy legal prep, counselor.
* Pharmaceutical companies must provide DC with FDA correspondence.
Um, all warning and untitled letters are already posted on the FDA website. But, hey, demanding such already publicly available letter it makes for good headlines, right?
Here’s my favorite …
* The bill requires prescribers to obtain written informed consent from patients when prescribing a prescription medication for a medically accepted indication.
So much for the concept of the “learned intermediary.â€
And as far as off-label use is concerned …
* Prescriber must explain to the patient that the medication is being prescribed outside of the indications for that medication as approved by the FDA and provide the patient with information commonly known by the medical profession regarding potential risks and side-effects
Um, according to the Medicaid statute medications prescribed for a medically accepted indication may only be restricted if, based on the compendia, the drug does not have a significant, clinically meaningful therapeutic advantage in terms of safety, effectiveness, or clinical outcome.
So much is wrong about so many things in this bill that it makes H.R. 380 (Rahm Emanuel’s “Pharmaceutical Market Access and Drug Safety Actâ€) look like the next coming of the Magna Carta.
“DC†may mean “David Catania,†but it mustn’t be allowed to mean “Drug Catastrophe†-- again.
Article today on efforts to develop a biomarker based test to predict early onset of Alzheimer's underscores the valuable role the Reagan Udall Foundation can play and the extent to which its "critics" are engaged in ad hominem attacks that flow from rage instead of science.
Blood Test Might Spot Alzheimer's Early
By Jeffrey Perkel
HealthDay Reporter 2 hours, 10 minutes ago
MONDAY, Oct. 15 (HealthDay News) -- An international team of scientists has developed a blood test that could reveal which patients with mild cognitive impairment will go on to develop Alzheimer's disease.
ADVERTISEMENT
If replicated and validated -- and assuming the development of effective treatments against Alzheimer's in the future -- such a test could open the door to medicating at-risk patients earlier and slowing or limiting neurological damage, explained Dr. Allan Levey, chair of neurology at Emory University, Atlanta.
"If it can be replicated, then we will find out how important [the study] really is," said Levey, who was not involved in the research.
Reagan Udall would help not only in replication and validation but set up standards for its approval by the FDA. Sharing data from the test would lead to more specific and early treatment of people and new medicines. As a recent CMPI study found, if we delay onset of Alzheimer's by 5 years it would be worth nearly $2 trillion to the United States.
How sad that so many interest groups and professional cranks (like David Ross) have lined up so quickly to attack an institute with such a profound and humane mission and have condemned its biomarker based work as dangerous.
http://news.yahoo.com/s/hsn/20071015/hl_hsn/bloodtestmightspotalzheimersearly
Blood Test Might Spot Alzheimer's Early
By Jeffrey Perkel
HealthDay Reporter 2 hours, 10 minutes ago
MONDAY, Oct. 15 (HealthDay News) -- An international team of scientists has developed a blood test that could reveal which patients with mild cognitive impairment will go on to develop Alzheimer's disease.
ADVERTISEMENT
If replicated and validated -- and assuming the development of effective treatments against Alzheimer's in the future -- such a test could open the door to medicating at-risk patients earlier and slowing or limiting neurological damage, explained Dr. Allan Levey, chair of neurology at Emory University, Atlanta.
"If it can be replicated, then we will find out how important [the study] really is," said Levey, who was not involved in the research.
Reagan Udall would help not only in replication and validation but set up standards for its approval by the FDA. Sharing data from the test would lead to more specific and early treatment of people and new medicines. As a recent CMPI study found, if we delay onset of Alzheimer's by 5 years it would be worth nearly $2 trillion to the United States.
How sad that so many interest groups and professional cranks (like David Ross) have lined up so quickly to attack an institute with such a profound and humane mission and have condemned its biomarker based work as dangerous.
http://news.yahoo.com/s/hsn/20071015/hl_hsn/bloodtestmightspotalzheimersearly
The great news; Cancer rates are declining once again and falling faster than ever before thanks to advances in screening and treatment of lung, breast and colorectal cancer.
The bad news: The CMS decision -- made in an effort to position itself as Hillary Clinton's Best Practice Institute -- will deny cancer patients access to blood boosting drugs that make such treatments -- and the increased survival -- possible.
Here's what prospective randomized trial of colorectal cancer patients found:
"Anaemia was a strong predictor for activity of first-line 5FU-based chemotherapy especially in those groups that showed the best responses, for example high performance status, infusionally treated, higher 5FU dose and those with liver secondaries. Patients with higher haemoglobin levels recorded a greater response rate and a longer time to progression and survival than anaemic subjects. Prospective evaluation of role of correcting anaemia on response to therapy is justified by these results."
Br J Cancer. 2006 Jul 3;95(1):13-20. Epub 2006
CMS -- with it's decision to withhold access to ESAs that correct anemia in cancer patients - is sure to join the ranks of the UK is insuring that more people die of cancer than medical science would permit.
The bad news: The CMS decision -- made in an effort to position itself as Hillary Clinton's Best Practice Institute -- will deny cancer patients access to blood boosting drugs that make such treatments -- and the increased survival -- possible.
Here's what prospective randomized trial of colorectal cancer patients found:
"Anaemia was a strong predictor for activity of first-line 5FU-based chemotherapy especially in those groups that showed the best responses, for example high performance status, infusionally treated, higher 5FU dose and those with liver secondaries. Patients with higher haemoglobin levels recorded a greater response rate and a longer time to progression and survival than anaemic subjects. Prospective evaluation of role of correcting anaemia on response to therapy is justified by these results."
Br J Cancer. 2006 Jul 3;95(1):13-20. Epub 2006
CMS -- with it's decision to withhold access to ESAs that correct anemia in cancer patients - is sure to join the ranks of the UK is insuring that more people die of cancer than medical science would permit.
The Reagan-Udall Foundation was created to promote new tools to accelerate the development a new generation of targeted medicines. The emphasis is on tools, meaning biomarkers, meaning coming up with validated measures and standards at a time when we have a lot of data and information but very little knowledge.
I hope the Foundation focuses on very mundane and important scientific work and provides hub for achieving consensus on measures and markers for qualifying and ap approving drugs based on actual biological differences and mechanisms. And I hope pharma does begin to more money into credible scientific projects that will help change the regulatory paradigm. The scientists in the companies want to do the right thing. I am not always so sure of the marketing types who still cling to the one size fits all blockbuster mentality and who have not yet realized that if drug companies don't get into the business of tracking the safety of their drugs in the post market -- using these tools in research and clinical settings -- they will continue to get clubbed by the likes of Nissen and Graham. If they don't seize the opportunity given to them in Reagan Udall then well....it's their own fault.
Of course, the fact that this might make drug development more efficient years from now (i.e. fewer clinical trials, more subpopulations targeted) and might help drug companies will drive the Marcia Angells, Kassirers, Avorns, etc crazy. Matt Perrone has an excellent piece that captures the knee-jerk "corporations are evil" bleating from the likes of The Union of Conceited Scientists.
Matt Perrone ought to brush up on what a biomarker is however otherwise he will continue to be misled by likes of David Ross who gave him this example:
"For example, Ross, who left the FDA last year over a drug safety dispute, is concerned, as are others, that the FDA might be persuaded to quickly adopt a controversial drug-testing process that uses biological indicators, such as blood pressure and cholesterol levels, to determine a drug's safety.
Drug companies have taken the unusual step of sharing research data on these indicators, called biomarkers, to see if the size and duration of patient drug trails can be reduced. Some critics call their use a high-risk gamble.
"Biomarkers are a bit like dynamite in that they can be extremely useful but they can cause a lot of damage," said Ross.
He cites a biomarker that suggested irregular heart rhythms were a key predictor of fatal heart attacks. Using the assumption, doctors prescribed heart-regulating drugs to millions of patients in the 1980s but abruptly halted the practice in 1989 when a long-term study revealed patients were actually more than twice as likely to die of sudden heart failure."
Note to Perrone and Ross: The reason people died was because the drug was prescribed to patients outside of the clinical trial guideline. Ross is using the "generic" definition of a biomarker, meaning biological measure that is linked to a disease state. A biomarker that captured genetic variations explaining why people more likely to die from the drug is now possible to develop using a blood test. I guess we shouldn't use that. Ditto, all those tests that can now screen to see which people respond to certain cancer drugs and avoid needless chemotherapy or which HIV drugs will work for them.
It should also be noted that Ross was one of the biggest loudmouths about the dangers of SSRIs which have been overhyped to the point that prescriptions are down and suicides are up.
This effort to eliminate measures like cholesterol, glycemic index, bone density, blood pressure as conditions of approval is just an effort to slow down drug approval and move it into the dark ages. It is being engineered by those who believe that the current approach to clinical evaluation is corrupt and corporatized. Supporters offer this more rigid and unreachable methods not because they are based on better science but because they reduce the number of new medicines that companies develop. The attack is political, pure and simple.
The battle lines are already being defined: The opponents of the Foundation and Critical Path oppose both because it might advance industry and industry is evil. (Even though most of the research will be done in collaboration with academia and NIH). Because corporations are involved it is inherently evil and corrupt. WHich allow the other side to distort the mission and the science all they want.
The headline in the WP "New FDA Research Center Rife WIth Risks" tells you that Reagan Udall has already been spun. How sick and sad is that?
http://www.washingtonpost.com/wp-dyn/content/article/2007/10/14/AR2007101400722.html
I hope the Foundation focuses on very mundane and important scientific work and provides hub for achieving consensus on measures and markers for qualifying and ap approving drugs based on actual biological differences and mechanisms. And I hope pharma does begin to more money into credible scientific projects that will help change the regulatory paradigm. The scientists in the companies want to do the right thing. I am not always so sure of the marketing types who still cling to the one size fits all blockbuster mentality and who have not yet realized that if drug companies don't get into the business of tracking the safety of their drugs in the post market -- using these tools in research and clinical settings -- they will continue to get clubbed by the likes of Nissen and Graham. If they don't seize the opportunity given to them in Reagan Udall then well....it's their own fault.
Of course, the fact that this might make drug development more efficient years from now (i.e. fewer clinical trials, more subpopulations targeted) and might help drug companies will drive the Marcia Angells, Kassirers, Avorns, etc crazy. Matt Perrone has an excellent piece that captures the knee-jerk "corporations are evil" bleating from the likes of The Union of Conceited Scientists.
Matt Perrone ought to brush up on what a biomarker is however otherwise he will continue to be misled by likes of David Ross who gave him this example:
"For example, Ross, who left the FDA last year over a drug safety dispute, is concerned, as are others, that the FDA might be persuaded to quickly adopt a controversial drug-testing process that uses biological indicators, such as blood pressure and cholesterol levels, to determine a drug's safety.
Drug companies have taken the unusual step of sharing research data on these indicators, called biomarkers, to see if the size and duration of patient drug trails can be reduced. Some critics call their use a high-risk gamble.
"Biomarkers are a bit like dynamite in that they can be extremely useful but they can cause a lot of damage," said Ross.
He cites a biomarker that suggested irregular heart rhythms were a key predictor of fatal heart attacks. Using the assumption, doctors prescribed heart-regulating drugs to millions of patients in the 1980s but abruptly halted the practice in 1989 when a long-term study revealed patients were actually more than twice as likely to die of sudden heart failure."
Note to Perrone and Ross: The reason people died was because the drug was prescribed to patients outside of the clinical trial guideline. Ross is using the "generic" definition of a biomarker, meaning biological measure that is linked to a disease state. A biomarker that captured genetic variations explaining why people more likely to die from the drug is now possible to develop using a blood test. I guess we shouldn't use that. Ditto, all those tests that can now screen to see which people respond to certain cancer drugs and avoid needless chemotherapy or which HIV drugs will work for them.
It should also be noted that Ross was one of the biggest loudmouths about the dangers of SSRIs which have been overhyped to the point that prescriptions are down and suicides are up.
This effort to eliminate measures like cholesterol, glycemic index, bone density, blood pressure as conditions of approval is just an effort to slow down drug approval and move it into the dark ages. It is being engineered by those who believe that the current approach to clinical evaluation is corrupt and corporatized. Supporters offer this more rigid and unreachable methods not because they are based on better science but because they reduce the number of new medicines that companies develop. The attack is political, pure and simple.
The battle lines are already being defined: The opponents of the Foundation and Critical Path oppose both because it might advance industry and industry is evil. (Even though most of the research will be done in collaboration with academia and NIH). Because corporations are involved it is inherently evil and corrupt. WHich allow the other side to distort the mission and the science all they want.
The headline in the WP "New FDA Research Center Rife WIth Risks" tells you that Reagan Udall has already been spun. How sick and sad is that?
http://www.washingtonpost.com/wp-dyn/content/article/2007/10/14/AR2007101400722.html
I appreciate Jonathan Cohn's thoughtful response to my post on SCHIP and the Frost family being the poster for SCHIP expansion/renewal. It is obvious we have two different views of how to make health care convenient, predictive and prospective. Indeed, the fight over the bona fides of the Frost family have less to to do with their income or wealth or mine for that matter but really about whether entitlements should be expanded to cover and crowd out private insurance and whether government should be the payor and provider of health care or not.
I supported Part D -- the largest expansion of Medicare in a lifetime -- while most Democrats who supported the same approach in 1999 did not. I did because I thought that adding drug benefits would reduce health care spending overall and that giving people choice would create competition and lower costs. I have always opposed SCHIP because it addressed a problem that did not exist with a solution that made matters worse. We have 4 -5 million kids eligible for Medicaid in 2006 and that was about the same number in 1997. Meanwhile the single payer approach if you will has made it hard for people to enroll in SCHIP. All the while, we have ignored the issue of coverage for illegal workers who make up a huge chunk of the uninsured and the fact that lots of people are just to cheap and irresponsible to buy their own insurance.
But that's the heart of the debate. I think everyone should have access to health care coverage and decent care. I don't think we can guarantee results or equal outcomes and I don't think government does a great job delivering health care coverage. Cohn does. This debate is not over. SCHIP will get refunded but not expanded. And then we can have a debate about whether we want Hillary to run health care (setting up a false set of choices that lead to a single payer system) or let people decide for themselves through insurance deregulation, HSAs, tax credits and retail clinics.
I think conservative ideas on health care will win the day. Why? Because we value freedom as an essential ingredient for excellence in medical science and sustaining dignity in the doctor patient relationship. Without freedom, the freedom to innovate, to prescribe, to choose, to err, medicine suffers.
My thanks again to Mr. Cohn for reshaping the debate around what matters. Here's a link to his post and to his book about the health care system.
http://www.tnr.com/
blog/the_plank?pid=151407
http://www.sickthebook.com
I supported Part D -- the largest expansion of Medicare in a lifetime -- while most Democrats who supported the same approach in 1999 did not. I did because I thought that adding drug benefits would reduce health care spending overall and that giving people choice would create competition and lower costs. I have always opposed SCHIP because it addressed a problem that did not exist with a solution that made matters worse. We have 4 -5 million kids eligible for Medicaid in 2006 and that was about the same number in 1997. Meanwhile the single payer approach if you will has made it hard for people to enroll in SCHIP. All the while, we have ignored the issue of coverage for illegal workers who make up a huge chunk of the uninsured and the fact that lots of people are just to cheap and irresponsible to buy their own insurance.
But that's the heart of the debate. I think everyone should have access to health care coverage and decent care. I don't think we can guarantee results or equal outcomes and I don't think government does a great job delivering health care coverage. Cohn does. This debate is not over. SCHIP will get refunded but not expanded. And then we can have a debate about whether we want Hillary to run health care (setting up a false set of choices that lead to a single payer system) or let people decide for themselves through insurance deregulation, HSAs, tax credits and retail clinics.
I think conservative ideas on health care will win the day. Why? Because we value freedom as an essential ingredient for excellence in medical science and sustaining dignity in the doctor patient relationship. Without freedom, the freedom to innovate, to prescribe, to choose, to err, medicine suffers.
My thanks again to Mr. Cohn for reshaping the debate around what matters. Here's a link to his post and to his book about the health care system.
http://www.tnr.com/
blog/the_plank?pid=151407
http://www.sickthebook.com
Why is the Union of Concerned Scientists putting politics in front of the public health?
http://www.washingtonpost.com/wp-dyn/content/article/2007/10/14/AR2007101400722.html
Consider the statement of Francesca Grifo, a director at the UCS, “Given FDA's track record in the past, I'm not confident in their ability to create something that is free of influence from industry."
Believe it or not, this statement was aimed at, of all things, the bi-partisan crown jewel of current FDA reform legislation – the Reagan/Udall Foundation (aka “the Crtitical Path Foundationâ€), approved by Congress and signed into law late last month, allowing the FDA to work with both industry and academe to accelerate the nascent sciences of genomics and proteomics to help realize the very real potential of a new kind of medicine – personalized medicine -- capable of tailoring highly effective treatments against the underlying causes of specific diseases in individual patients – and perhaps even preventing those diseases from occurring or progressing in the first place
According to Senator Ted Kennedy, the Reagan-Udall Foundation "will make new research tools and techniques available to the entire research community, shortening the time it takes to develop new drugs and reducing costs for patients."
Just what track record is Ms. Gifo referring to anyway?
The hard truth is that many of the most dramatic scientific advances in basic research are difficult to translate into safe and effective medical treatments for patients. Isn’t this something that “concerned†scientists should be concerned about?
Despite the increase in R&D spending, the number of new innovative products being submitted to the FDA for approval is decreasing and the rate of failure is increasing. Almost 50% of applications are failing in late-stage Phase 3 trials. This costs companies millions of extra dollars and is driving up the cost of successfully bringing a new drug to market. In 2003, researchers at Tufts Center for the Study of Drug Development estimated these costs to be $802 million, and some sources suggest that the total cost is closer to $1.7 billion.
As the late Senator Everitt Dirksen said, “A billion here and a billion there, and pretty soon you’re talking about real money.â€
Think about the millions of dollars, pounds and Euros that would be saved by all types and sizes of companies and governments if publicly discussed and vetted biomarkers could be used and used predictably in the drug approval process.
IP questions? You bet – it’s the 800 pound gorilla sitting in the corner. But we must acknowledge, address, and overcome this and other obstacles. Bumps along the road of all sizes must not allow the Critical Path to be the path less traveled.
FDA must assume the lead organizational role, because the agency is at the crossroads of the translational process. FDA is uniquely suited to take a major role in this effort because of their unique cross-industry and cross-cutting knowledge of the hurdles companies and products encounter that are causing them to fail in late stage clinical trials.
The Critical Path must be blazed in partnership. Regulators and industry, patient groups and legislators, FDA and EMEA must work together to help bridge the widening canyon between bench and bedside.
The Union of Concerned Scientists thinks the FDA has “an agenda†– and they’re right – it’s to protect and advance the public health.
http://www.washingtonpost.com/wp-dyn/content/article/2007/10/14/AR2007101400722.html
Consider the statement of Francesca Grifo, a director at the UCS, “Given FDA's track record in the past, I'm not confident in their ability to create something that is free of influence from industry."
Believe it or not, this statement was aimed at, of all things, the bi-partisan crown jewel of current FDA reform legislation – the Reagan/Udall Foundation (aka “the Crtitical Path Foundationâ€), approved by Congress and signed into law late last month, allowing the FDA to work with both industry and academe to accelerate the nascent sciences of genomics and proteomics to help realize the very real potential of a new kind of medicine – personalized medicine -- capable of tailoring highly effective treatments against the underlying causes of specific diseases in individual patients – and perhaps even preventing those diseases from occurring or progressing in the first place
According to Senator Ted Kennedy, the Reagan-Udall Foundation "will make new research tools and techniques available to the entire research community, shortening the time it takes to develop new drugs and reducing costs for patients."
Just what track record is Ms. Gifo referring to anyway?
The hard truth is that many of the most dramatic scientific advances in basic research are difficult to translate into safe and effective medical treatments for patients. Isn’t this something that “concerned†scientists should be concerned about?
Despite the increase in R&D spending, the number of new innovative products being submitted to the FDA for approval is decreasing and the rate of failure is increasing. Almost 50% of applications are failing in late-stage Phase 3 trials. This costs companies millions of extra dollars and is driving up the cost of successfully bringing a new drug to market. In 2003, researchers at Tufts Center for the Study of Drug Development estimated these costs to be $802 million, and some sources suggest that the total cost is closer to $1.7 billion.
As the late Senator Everitt Dirksen said, “A billion here and a billion there, and pretty soon you’re talking about real money.â€
Think about the millions of dollars, pounds and Euros that would be saved by all types and sizes of companies and governments if publicly discussed and vetted biomarkers could be used and used predictably in the drug approval process.
IP questions? You bet – it’s the 800 pound gorilla sitting in the corner. But we must acknowledge, address, and overcome this and other obstacles. Bumps along the road of all sizes must not allow the Critical Path to be the path less traveled.
FDA must assume the lead organizational role, because the agency is at the crossroads of the translational process. FDA is uniquely suited to take a major role in this effort because of their unique cross-industry and cross-cutting knowledge of the hurdles companies and products encounter that are causing them to fail in late stage clinical trials.
The Critical Path must be blazed in partnership. Regulators and industry, patient groups and legislators, FDA and EMEA must work together to help bridge the widening canyon between bench and bedside.
The Union of Concerned Scientists thinks the FDA has “an agenda†– and they’re right – it’s to protect and advance the public health.
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.
