Latest Drugwonks' Blog
As we continue to furrow our collective brows pondering the eternal quandary of risk/benefit, I thought you’d enjoy the comments below (which I received this afternoon via e-mail from a respected physician who shall remain nameless):
TO ALL THE KIDS WHO SURVIVED THE 1930s, 40's, 50's, 60's & 70's
First, we survived being born to mothers who smoked and/or drank while they were pregnant.
They took aspirin, ate blue cheese dressing, tuna from a can, and didn't get tested for diabetes.
Then after that trauma, we were put to sleep on our tummies in baby cribs covered with bright colored lead-based paints.
We had no childproof lids on medicine bottles, doors or cabinets and when we rode our bikes, we had no helmets. Not to mention the risks we took hitchhiking.
As infants & children, we would ride in cars with no car seats, booster seats, seat belts or air bags.
Riding in the back of a pick up on a warm day was always a special treat.
We drank water from the garden hose and NOT from a bottle.
We shared one soft drink with four friends, from one bottle and NO ONE actually died.
We ate cupcakes, white bread and real butter and drank Kool-Aid made with sugar, but we weren't overweight because we were always outside playing.
We would leave home in the morning and play all day, as long as we were back when the streetlights came on.
No one was able to reach us all day. And we were O.K.
We would spend hours building our go-carts out of scraps and then ride down the hill, only to find out we forgot the brakes. After running into the bushes a few times, we learned to solve the problem.
We did not have Playstations, Nintendo's, X-Boxes, no video games at all, no 150 channels on cable, no video movies or DVD's, no surround-sound or CD's, no cell phones, no personal computers, no Internet or chat rooms.
We had friends and we went outside and found them!
We fell out of trees, got cut, broke bones and teeth and there were no lawsuits from these accidents.
We ate worms and mud pies made from dirt, and the worms did not live in us forever.
We were given BB guns for our 10th birthdays, made up games with sticks and tennis balls and, although we were told it would happen, we did not put out very many eyes.
We rode bikes or walked to a friend's house and knocked on the door or rang the bell, or just walked in and talked to them!
Little League had tryouts and not everyone made the team. Those who didn't had to learn to deal with disappointment. Imagine that!
The idea of a parent bailing us out if we broke the law was unheard of. They actually sided with the law!
These generations have produced some of the best risk-takers, problem solvers and inventors ever!
The past 50 years have been an explosion of innovation and new ideas. We had freedom, failure, success and responsibility, and we learned HOW TO DEAL WITH IT ALL!
FDA and International Serious Adverse Events Consortium Release First Data on Genetic Basis of Adverse Drug Events
The first data offering health care professionals a better look into the genetic basis of certain types of adverse drug events was released today by the FDA and the International Serious Adverse Event Consortium (SAEC). The data are focused on the genetics associated with drug-induced serious skin rashes, such as Stevens-Johnson syndrome and toxic epidermal necrolysis, and helps better predict an individual’s risk of developing these reactions.
Both skin conditions appear as allergic-like skin reactions associated with blistering and peeling, and are considered life-threatening. Medications causing these serious allergic reactions should be discontinued; and if such signs and symptoms are not quickly recognized, these reactions can be fatal.
“The SAEC has fulfilled a key goal of the Critical Path Initiative by providing the research community with public access to new genomic data on adverse drug events,” said Janet Woodcock, M.D., director, the FDA’s Center for Drug Evaluation and Research. “This consortium has taken a significant step forward by promoting open sharing of drug safety data. This type of cooperation has the potential to lead to more personalized approaches to medicine that can reduce a patient’s risk for experiencing an adverse drug event.”
The SAEC is a nonprofit partnership of pharmaceutical companies, the Wellcome Trust, and academic institutions focused on research relating to the genetics of drug-induced serious adverse events. The samples from the initial serious skin rash cases and matched controls were collected by GlaxoSmithKline plc, London, U.K., and donated to the consortium for this research.
By pooling these samples, the SAEC has identified numerous genetic associations that may contribute to an individual’s risk of developing serious drug-induced skin reactions. The data was compiled and analyzed just 16 months after the consortium was launched.
“We are pleased to be able to provide these invaluable data to the research community to both improve the productivity of drug development and to begin the critical process of developing validated biomarkers to forecast patients who may be at risk for drug-induced serious adverse events,” said Arthur Holden, founder and chairman of the SAEC. “We continue to believe the application of genomics to research the genetic basis of serious adverse events will prove to be one the most productive early applications of this technology.”
The consortium will publish its initial research results later this year.
Researchers who enter in to a data use agreement can obtain free access to the data to generate custom data inquiries and obtain immediate results on the genetic basis of adverse drug events.
For more information on the International Serious Adverse Event Consortium see www.saeconsortium.org.
For information on the FDA’s Critical Path Initiative see http://www.fda.gov/oc/initiatives/criticalpath/
Hmm, I guess the doctor-patient relationship doesn't quite cut it for Mr. Pearlstein. Or put another way, if Mr. Pearlstein's doctor had access to 21st century tools that made medicine predictiive and prospective -- which is increasingly possible -- he would still opt for cookbook decisions rendered by researchers who are largely selected from HMO sponsored research institutions using research methods that by definition exclude the variations in treatment effect and response that his doctor can detect...or could detect if those said researchers would ever get around to evaluating the value of those 21st century tools...
Pearlstein asserts that critics of comparative effectiveness don't have "any shred of evidence that the professionals who do this research are incompetent or have any but the best intentions in trying to figure out what treatments are the most effective for patients. There is no reason to believe that once this clinical research is completed, it cannot be used in a disciplined, scientific way by physicians, economists and medical ethicists to determine whether there are drugs, tests, surgical procedures or devices that simply don't deliver enough benefit to justify their cost."
He is quick to claim that England is special and doesn't count. What about Canada, or Australia or Germany? How about Netherlands or Italy or Israel? Is there any entity that uses comparative effectiveness that does not restrict access to new technologies based on what it is worth to the institution without regard to the consumer? What about the VA which restricts access to new drugs? Or the Medicaid formularies that restrict access to cancer medicines and drugs for mental illness based on the comparative effectiveness research churned out by the Drug Effectiveness Research Project which is paid for by the Agency for Health Care Research and Quality and is conducted by HMO supported institutes? Forget about intentions. In healthcare, it's the outcomes that matter. It's not belief, it's evidence. And the best evidence is biological and mechanistic, not probability.
Pearlstein then introduces a straw man when he writies:
"But ours is an economy that is sinking under the weight of a health-care system that costs twice as much as any in the world while delivering poorer health outcomes. The cost of health care has crippled entire industries, disadvantaged our companies in international competition and brought millions of families into bankruptcy. Worst of all, in denying vital medical services to the 40 million Americans without health insurance, we engage in the most immoral kind of medical rationing imaginable -- rationing by the ability to pay. "
The reasons for our rate and intensity of health care spending has little do with medical device and drug expenditures, the smallest part of the health care budget. It is multifactorial. And the assertion that our outcomes are poorer is wrong in any event. In any event, health systems that ration have done little to control the rate of health expenditures. They merely shift spending into other categories. Ultimatley the solution to health care problems, including spending, is innovation, which comparative effectiveness is used to kill.
Pearlstein tries to slime those who criticize comparative effectiveness as political arsonists supported by drug and medical devicemakers. So be it. If those fires had been quenched decades ago where would society be. And where will it go if Pearlstein fails to fully understand the consequences of the approach being proposed the comparative effectivenesss zealots.
See his article here.
"America did too much of this and that's why their medical costs have grown," said Masaharu Nakajima, a surgeon and former director of the Health Bureau at the Ministry of Health, Labor and Welfare.
Since Japan enacted universal health insurance in the early 1960s, the emphasis has been on a minimum standard of care for all. People must pay a monthly health-insurance fee, and large companies pay also. Coverage decisions, doctors' pay, and other rules are set by the central government.
Japanese doctors complain that they have no time to spend with patients. The experience of seeing a doctor is summarized as "a three-hour wait for a three-minute visit."
Somebody should send this to AAJ’s former president Kathleen Flynn Peterson.
It is becoming increasingly un-PC (“Pharma Correct”) to say that $1.1 billion earmark for a “Federal Coordinating Council for Comparative Effectiveness Research” is a bad idea.
Well, it’s a bad idea.
And cozying up to the powers-that-be isn’t going to change the fact that this is a giant first step towards a U.S. version on NICE.
Whether or not you agree that such a notion is a bad idea, we should ALL agree that it’s important enough to debate on its merits – and not become law through legislative legerdemain; hidden deep within the bowels of the stimulus package.
Wither transparency?
Senator Feinstein says that, “the purpose of the council is to coordinate comparative effectiveness research activities with the goal of reducing duplicative efforts and encouraging coordinated and complementary use of resources.”
Senator Baucus promises that, “Its charge should not go beyond that.”
Indeed, Senate Finance Committee documents detailing health provisions in the Senate's economic stimulus package say that the bill "specifically prohibits the government from making any coverage decisions based on this research, or even from issuing guidelines that would suggest how to interpret the research results."
But the House language has no such limitations in mind:
“By knowing what works best and presenting this information more broadly to patients and health care professionals, those items, procedures and interventions that are most effective to prevent, control and treat health conditions will be utilized, while those that are found to be less effective and in some cases, more expensive, will no longer be prescribed.”
The problem is that “comparative effectiveness,” as it is currently designed, places into conflict the short-term budgeting dilemmas of governments elected for relatively short periods of time with the ever-lengthening life spans of their electorates. Us.
As currently organized, comparative effectiveness will be used to increase government control over the practice of medicine and introduce price controls.
Let's get real folks. All rhetoric to the contrary -- this is the first step towards allowing Uncle Sam to push a restrictive formulary on more and more Americans. Step Two is to do away with the Non-Interference Clause so that comparative effectiveness measures can be used to offer a VA-style formulary. And Step Three is to make that the model for the "universal care" we'll all be paying increased taxes to support.
(Note: The VA formulary offers 1,300 drugs, compared with 4,300 available under the average Part D plan -- prompting more than one-third of retired veterans to enroll in Medicare drug plans.)
Broader access to mediocre care? “Just like in
We need a new model. We need to develop proposals that modernize the information used in the evaluation of the value of treatments. Just as the key scientific insights guiding the FDA Critical Path program are genetic variations and biomedical informatics that predict and inform individual responses to treatment, we must establish a science-based process that incorporates the knowledge and tools of personalized medicine in reimbursement decisions: true evidence-based, patient-centric medicine.
For instance, the FDA, in cooperation with many interested parties, has developed a Critical Path opportunities list that provides 76 concrete examples of how new scientific discoveries in fields such as genomics and proteomics, imaging, and bioinformatics could be applied during medical product development to improve the accuracy of the tests used to predict the safety and efficacy of investigational medical products.
We need a Critical Path for Comparative Effectiveness to begin the process of developing a similar list of ways new discoveries and tools (such as electronic patient records) can be used to improve the predictive and prospective nature of comparative effectiveness.
It’s a complicated proposition—but such a goal is as simple as it is essential—cost must never be allowed to trump care, and short-term savings must not be allowed to trump long-term outcomes. Just as we need new and better tools for drug development, so too do we need them for comparative effectiveness measurements.
A comparative effectiveness model for the 21st Century should reflect and measure individual response to treatment based on the combination of genetic, clinical, and demographic factors that indicate what keep people healthy, improve their health, and prevent disease. A rapidly aging society demands a new healthcare paradigm capable of providing for its needs in the 21st Century. Equality of care must be matched with quality of care.
In an era of personalized medicine, one-size-fits-all treatments and reimbursement strategies are dangerously outdated. We are early in this debate, but at least we can all agree that this is not, and must not be exclusively, a debate about saving money. It must be about patient care.
For more on this issue, have a look at this new policy paper from the Washington Legal Foundation.
If you like the legislation -- you'll love the movie.
Have a look at our new 16 minute documentary, "Off Label: Universal Healthcare." Just click here and then click on the picture of our very own Uncle Sam.
Please share this video with all your contacts, friends, relations -- and elected representatives. And send along your comments as well.
Increasing choices, decreasing costs
The Medicare Prescription Drug Savings and Choice Act of 2009 proposes increasing beneficiary choice and driving down costs by creating a Medicare-administered drug plan as an alternative to privately administered Part D plans. Additional provisions include:
- The Medicare-operated drug plan or plans would be available nationwide with a uniform monthly premium.
- The Agency for Healthcare Research and Quality would assess clinical effectiveness and safety of drugs and recommend medications that should be included on the plan formulary.
- Drugs cannot be removed from a formulary during the plan year except in the case of safety concerns.
- An advisory committee would review petitions and make recommendations on whether to add drugs to the formulary.
- For drugs that provide similar benefits, the formulary would use incentives to encourage Medicare beneficiaries to choose the drug for which the HHS secretary was able to negotiate the lowest price.
http://www.ama-assn.org/amednews/2009/02/09/gvsc0209.htmBack to top.
Here’s a colloquy that includes Senators Baucus, Enzi, Hatch, Roberts, and Feinstein. The date, February 6, 2009. The topic, $1.1 billion for a “Federal Coordinating Council on Comparative Effectiveness.” The highlights are mine. The words are their own.
Mr. BAUCUS. I understand Senator ENZI has comments regarding the provisions for comparative clinical effectiveness research included in The American Recovery and Reinvestment Act of 2009 which is being considered in the Senate this week.
Mr. ENZI. I am pleased to see that in its consideration of this bill, the Appropriations Committee made sure this research will evaluate comparative clinical effectiveness, not comparative cost-effectiveness. In addition, the committee’s report language references provisions of the existing comparative effectiveness research program at HHS that ensure that the agency developing comparative information does not use it to set national practice standards or coverage restrictions. I also believe that comparative effectiveness research must be conducted using an open and transparent process, and must consider differences in how people respond to treatment. It is my understanding that the Comparative Effectiveness Research Act of 2008, which you introduced with Senator CONRAD last Congress, is consistent with these principles. I would like to see the $1.1 billion used consistently with these principles, and ask that you advocate for these principles in conference.
Mr. HATCH. I agree that the primary focus of comparative effectiveness research should be clinical effectiveness not cost. We can all agree that the ‘‘one size fits all’’ approach is the wrong approach for the American health care system. Based on our own personal experiences we all know that what works best for one person, does not always work the same for another. I look forward to working in a bipartisan and inclusive manner to come up with prudent legislation that will not only help us realize the true potential of comparative effectiveness but also preserve patient choice and innovation—the two hallmarks of our health care system.
Mr. ROBERTS. I would associate myself with the remarks of Senator ENZI, and would underscore that it is very important to require full openness, transparency and accountability in how research priorities are set and how studies are conducted and communicated. Without this openness, patients have no assurance that their voice will be heard in the process, and no ability to understand how results are being used in decisions that directly affect their health. I look forward to working with my colleagues to ensure that strong provisions for openness, transparency, and accountability are put in place.
Mrs. FEINSTEIN. I thank my colleagues for their efforts on this issue. I agree that comparative effectiveness research holds great promise to improve medical care by giving physicians and patients valuable information on treatment options. It is my understanding that the new Federal coordinating council included in the language is intended to coordinate the comparative effectiveness research efforts taking place across Federal agencies and with funds we are providing in this bill. However, there is some concern that the language, as currently written, allows the council to expand its activities beyond mere coordination. I think my colleagues would agree that the purpose of the council is to coordinate comparative effectiveness research activities with the goal of reducing duplicative efforts and encouraging coordinated and complementary use of resources.
Mr. BAUCUS. I thank Senator FEINSTEIN for pointing that out. I agree. The coordinating council should look across agencies to coordinate resources and activities of the federal government with respect to comparative effectiveness research. Its charge should not go beyond that. The language of the bill could be clarified to make that point clear. And I will support clarification of it in conference.
(Here’s the complete colloquy.)
$1.1 billion to “coordinate” resources and activities? That’s a lot of mazuma. And not a single job created. Well, maybe one coordinator and a few assistants. And at $1.1 billion, they’ll be earning more than any GS-14 I ever met.
And that’s with benefits.
And speaking of $1.1 billion, that also happens to be the mid-range savings projected for what implementation of FDA-recommended genetic testing for warfarin will deliver in one year. (The savings will be realized through the prevention of 85,000 serious bleeding events and 17,000 strokes annually.) Yet FDA is not slotted to receive a single shekel from the stimulus package.
“Clarification in conference,” indeed.
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.
