Latest Drugwonks' Blog
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."
"We started during the Clinton administration to transition the VA system to a paperless system....The VA is leading the way in reducing medical errors, improving patient safety, and delivering high quality care; now this is a lesson about what can be done when we have a plan. A plan that is evidence-based, a plan that uses what we know works, and a system that we can actually get to respond to that evidence-based planning."
Hillary Clinton
"If you take a look at how the VHA system works, it's much more flexible getting drugs that are not on the list than the private plans are. And so people get the worst of all worlds: They have a more rigid formulary under Medicare, and the prices are much higher."
Ron Pollack FUSA
"Who do you think receives higher-quality health care. Medicare patients who are free to pick their own doctors and specialists? Or aging veterans stuck in those presumably filthy VA hospitals with their antiquated equipment, uncaring administrators, and incompetent staff? An answer came in 2003, when the prestigious New England Journal of Medicine published a study that compared veterans health facilities on 11 measures of quality with fee-for-service Medicare. On all 11 measures, the quality of care in veterans facilities proved to be "significantly better."
Philip Longman, Washington Monthly, 2005 from an article The Best Care Anywhere that boasted: "Ten years ago, veterans hospitals were dangerous, dirty, and scandal-ridden. Today, they're producing the highest quality care in the country. Their turnaround points the way toward solving America's health-care crisis."
Hillary Clinton
"If you take a look at how the VHA system works, it's much more flexible getting drugs that are not on the list than the private plans are. And so people get the worst of all worlds: They have a more rigid formulary under Medicare, and the prices are much higher."
Ron Pollack FUSA
"Who do you think receives higher-quality health care. Medicare patients who are free to pick their own doctors and specialists? Or aging veterans stuck in those presumably filthy VA hospitals with their antiquated equipment, uncaring administrators, and incompetent staff? An answer came in 2003, when the prestigious New England Journal of Medicine published a study that compared veterans health facilities on 11 measures of quality with fee-for-service Medicare. On all 11 measures, the quality of care in veterans facilities proved to be "significantly better."
Philip Longman, Washington Monthly, 2005 from an article The Best Care Anywhere that boasted: "Ten years ago, veterans hospitals were dangerous, dirty, and scandal-ridden. Today, they're producing the highest quality care in the country. Their turnaround points the way toward solving America's health-care crisis."
Today the Senate reintroduce legislation to require the FDA insure that packages, wholesalers and manufacturing plants in Europe -- all old drugs of course -- are safe. The bill also requires the FDA to claim that drugs with different coatings, formulations, doses but same active ingredients are as safe and effective as products made to US spec.
Earlier this week, the Senate introduced a bill that would require the FDA to come up with a standard and process for approving -- I am not making this one up -- a safe cigarette.
Previously, Congress introduced a bill requiring the FDA to approve certain knock-off versions of biotech drugs as generic and as interchangeable without any safety tests or risk management or life cycle management strategies,
Previously still, legislation was introduced which required the FDA to set up a risk management program for every drug on the market and every new one being developed...except for imported drugs and generic biologics.
The FDA is still responsible for approving new medicines. I think.
But not until they improve spinach inspection and beef up labeling on peanut butter and organic foods.
Earlier this week, the Senate introduced a bill that would require the FDA to come up with a standard and process for approving -- I am not making this one up -- a safe cigarette.
Previously, Congress introduced a bill requiring the FDA to approve certain knock-off versions of biotech drugs as generic and as interchangeable without any safety tests or risk management or life cycle management strategies,
Previously still, legislation was introduced which required the FDA to set up a risk management program for every drug on the market and every new one being developed...except for imported drugs and generic biologics.
The FDA is still responsible for approving new medicines. I think.
But not until they improve spinach inspection and beef up labeling on peanut butter and organic foods.
Let's say you are a leading clinician researcher in the area of auto-immune disorders and a new drug for rheumatoid arthritis has a risk management program attached to it that limits prescribing to patients with RA and to RA specialists. However because of the drug works on a particular pathway you, because of previous research, have a hunch, it will work fine on patients with lupus. Or what if you want to use a new antibiotic to suppress an infection in CF patients but the RIskmap plan limits it to organ rejection use. If you use it (or if you can even get it) for an off-the-map purpose are you liable, criminally or civilly? Could you lose your medical license? Do patients have to wait to enroll in a clinical trial for every novel use? Will medical innovation grind to a halt? Will patients die because they are not on The MAP?
For all those eager to kill off the US pharma industry, no need to worry, Cuba is filling the void with an ambitious program to find a use for coca leaves for every ailment. Who needs stem cells when you have the precursor for cocaine from Castro's labs?
Scientists seek coca's medical benefits
By DAN KEANE, Associated Press Writer Tue Mar 6, 6:54 PM ET
LA PAZ, Bolivia - Cuban scientists are studying the possible medicial benefits of the coca leaf, a Bolivian official said Tuesday, signaling a possible expansion of President Evo Morales' plans to develop more legal products from a plant that is the chief ingredient of cocaine.
http://news.yahoo.com/s/ap/20070306/ap_on_sc/bolivia_cuba_coca
Scientists seek coca's medical benefits
By DAN KEANE, Associated Press Writer Tue Mar 6, 6:54 PM ET
LA PAZ, Bolivia - Cuban scientists are studying the possible medicial benefits of the coca leaf, a Bolivian official said Tuesday, signaling a possible expansion of President Evo Morales' plans to develop more legal products from a plant that is the chief ingredient of cocaine.
http://news.yahoo.com/s/ap/20070306/ap_on_sc/bolivia_cuba_coca
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.
