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If a pharmaceutical company funds a double-blind clinical trial performed by highly respected medical researchers, are the results relevant?
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Does anyone take Sid Wolfe and Public Citizen seriously anymore? In a pathetic attempt to block the OTC marketing of fat-blocking drug Xenical, Sid and his group — who peddle a scare a day screed called “Worst Pills, Best Pills” as a Christmas gift — are actually arguing that the drug should be pulled because…….the drug allegedly caused precancerous colon lesions in animal studies!!! Sid Vicious forget to mention the mounting evidence that Xenical exhibits antitumor properties towards prostate and breast cancer cells by virtue of its ability to block the lipogenic activity of fatty acid synthase. Oh. I guess bumps in a rat’s rump are more compelling than the ability to block cancer to the good folks at Public Citizen. What dopes.
Why is this week different from all other weeks? Well, it’s Passover for one thing but moreover BIO is meeting in Chicago where plenary sessions are discussion some very critical questions about whether, when personalized medicine matures, government regulators, trial attorneys, and the pols won’t suffocate in pursuit of their own agendas. Cases in point:
The Agency for Health Care Quality and Research — under orders from Congress — is supposed to compare the cost-effectiveness of certain drugs and treatments. (This is a Hillary Clinton pet project.) Bad enough that every day more and more research comes in for specific subgroups that AHQR isn’t looking at. Medical knowledge is not just doubling it is transforming as molecular diagnostics will help tailor preventive strategies for care to the individual or specific pathway. And what if reimbursement decisions are made based on these head to head studies while ignoring genetic variations? I can see a system where people are dying or sicker than they should be under Hillarycare’s new manifestation because it’s cheaper to simply rule on the old data and ignore the new.
What about a federal prosecutor or HHS that makes it a crime to tell patients about novel uses for medicines that are apparent at the molecular or biomarker level but not aproved by the FDA? Does that make sense? Where is the safe harbor for companies? The fact is, docs and researches and patients are already way ahead of the bureaucrats and lawyers in the effort to match targeted treatments to high responders. BUt will the threat of litigation and the refusal to pay for these treatments slow down progress?
Finally, the Critical Path opportunities list has been launched. We need a new commissioner to drive change through the agency and to create momentum for change throughout the regulatory system. The agency needs more resources to retain and hire talented and dedicated scientists to bring about this change and it needs to be involved in the development of the scientific consensus required for such an effort. Technological and societal change requires leadership. Requlations that stifle it will will not fall without such efforts.
Let’s call it like it is, the canard of evidence-based medicine is one-size-fits-all medicine. At its core it is cost-based rather than patient-based.
According to Greg Scandlen of the Heartland Institute, the concept of evidence-based medicine is founded on a few key ideas:
* First is that there is too much variation in medical practice, and variation is a bad thing. We should know what to do and do it in all cases, the idea goes. Medicine should be standardized around what is known to work.
(Of course this also presupposes that all people respond precisely the same way to all medicines. Sure. And if you believe that one, let me introduce you to my pet rabbit Harvey.)
As Mark McClellan said, “Looking at a gigantic uniform solution for everything is never going to work.” (JD Kleinke, Health Affairs, May/June 2004; 23(3): 177-185.)
* Next, there is only one way to determine what works and what doesn’t — using randomized, double-blind studies and measuring the effects on large populations to develop guidelines and practice protocols.
(I suppose that, after all, without our traditions our lives would be as shaky as … a fiddler on the roof!)
* Third, physicians who fail to follow those guidelines should be punished.
Scandlen opines, “Under this scenario, the only room for debate is around the severity of the punishment. People have argued that noncompliant physicians should be paid less, have higher premiums for malpractice coverage, lose their hospital privileges, be kicked out of insurance networks, and/or have their medical licenses revoked. I haven’t yet heard anyone argue that noncompliant doctors should be thrown in jail, but it is only a matter of time.”
Indeed. Perhaps this will encourage Consumer Reports to move beyond offering “best buys” on medications for Alzheimer’s Disease (see blog “Crash-Test Dummy Medicine,” 3/6/06) to legal advice on how to sue physicians for practicing patient-based medicine.
(Sadly, I’ll bet the folks at Consumer Reports don’t find this concept so outrageous.)
One-size-fits-all medicine may provide transitory savings in the short term, but the same patient who takes the cheapest available statin today may very well be the patient costing you — the taxpayer — the policymaker — the thought-leader — the sister — the spouse — big bucks when that patient (otherwise known as a “person”) ends up in the hospital because of improperly treated CVD.
And make no mistake, by “improperly treated” I mean treated with the least expensive rather than the most effective medication.
The reprecussions of short-term thinking vs long-term results, of cost-based over patient-based, of one-size-fits-all medicine, is pernicious to both the public purse and the public health.
For those out there who think that Andy von Eschenbach isn’t serious about his job at the FDA or question the Administration’s desire for his Senate confirmation, today’s news that he is officially resigning his post at the NCI should provide cause for reflection.
You may have missed many of these facts. I’m sorry, that’s wrong. What I meant to say is that you probably haven’t read about these facts.
So, without further ado, here’s some Sunny-D.
* Enrollment So Far Has Almost Surpassed The Entire Year’s Goal.
27.6 million of the 42.4 million eligible seniors are now covered by the prescription drug benefit. Of the 14.8 million who are not enrolled, 5.8 million are covered by alternative drug benefits like Veterans’ Administration or their current employers. (CMS, 3/23/06)
* Enrollment Is Well Ahead Of Schedule.
The benefit has now almost met its goal of enrolling 28 million seniors by December 2006. (CMS, 3/23/06)
* More Seniors Are Enrolling Every Week.
Since the start of March, seniors have been enrolling in the plan at an average rate of 416,000 per week, and enrollment has increased 29 percent since February. (CMS)
* A Majority Of Seniors Approve Of The Plan.
79 percent of seniors approve of the drug benefit, and 50 percent approve “strongly.” (Harris, 2/7-2/9/06)
* And Seniors Who Haven’t Yet Enrolled Plan To.
47 percent of seniors who aren’t yet in the plan say they plan to enroll by May 15; an additional 16 percent said they would sign up soon when reminded that costs will be higher after then. (KRC Research, 3/15-3/20)
4 Out Of 5 Seniors Who Signed Up For The Plan Are Satisfied.
78 percent are now satisfied with their prescription drug coverage, while just 13 percent are not. Furthermore, 77 percent now say they have peace of mind about their coverage and 67 percent say they’re better off than they were before. (KRC Research, 3/15-3/20)
* The Longer They’re In The Plan, The More Positive Seniors Feel About It.
66 percent of seniors say the effort they put into evaluating different drug plans was worth it, while only 13 percent say it wasn’t. The number who says it was worth it has increased by a net of 12 points since December 2005, when 57 percent thought it was worthwhile and 16 percent disagreed. (Ayres McHenry, 3/6-3/9/06)
* Seniors Have Been Using The Plan Successfully.
84 percent of seniors who enrolled themselves in the plan had no troubles signing up and 85 percent had no problems using it. (Ayres McHenry, 3/6-3/9/06)
* Seniors Are Able To Choose The Plan Right For Them.
Almost three-quarters of those in the plan, 72 percent, are confident they chose the drug plan that best fit their needs. (Pew Research, 3/8-3/12)
* Seniors In The Plan Are Saving Money On Their Drugs.
The average senior in the plan will save more than $1,100 on their prescription drugs this year. Furthermore, the plan’s premiums now average $25 per month, down from a projected $37 per month. (CMS Final Rule Regulatory
Impact Analysis, 1/28/05)
* And They Would Recommend It To Others.
65 percent of those in the plan say they would recommend other seniors enroll; just 8 percent said they would not. (Ayres McHenry, 3/6-3/9/06)
* Seniors Think The Plan Is A Step In The Right Direction.
Two-thirds of seniors, 66 percent, believe the drug benefit was a step in the right direction, while just 20 percent think it was not. (KRC Research, 3/15-3/20)
* And They Believe The Benefit’s Critics Are Playing Politics.
46 percent of seniors in the plan believe that politicians criticizing the prescription drug benefit are just trying to score political points, while only 14 percent think they are sincerely trying to fix it. (Ayres McHenry, 3/6-3/9/06)
The bi-partisan health care bill passed by the Mass. legislature requires deadbeats who don’t get health insurance only to ask taxpayers to foot the bill of their uncompensated care to the tune of three quarters of billion a year (more than Manny Ramirez makes ) to buy their own insurance. It gives people help to buy insurance on sliding scale according to income. The money comes from the uncompensated care fund most states have to pay for folks to use health care but don’t have or pay for insurance. It expands HSAs, allows consumes and small businesses to purchase health insurance at the same rate as big corporations through association health plans, relies on competition based on price and quality to drive down costs. These are all principles pushed by President Bush.
And by the way, the Mass. bill was applauded by my good friend Ron Pollack of FUSA. Now the question is: if market-based competition is good enough to expand coverage for the 65 and under crowd, why does Godfather Ron oppose the same principles when applied to Medicare?
Here’s a link to a powerpoint presentation of the Mass. plan elements
Lots of discussion today about Governor Mitt Romney’s initiative to provide universal insurance coverage to all residents of Massachusetts. But what’s really unusual is that the debate is over the program rather than politics. The “tease” from Pam Belluck’s article in today’s edition of the New York Times reads, “Combining ideas from across the political spectrum, the state would be the first to require its citizens to have insurance.” And consider this — the bill was passed by a legislature that is 85 percent Democratic and was hammered out with proposals and input from state Democratic legislators; Mr. Romney, a Republican; Senator Edward M. Kennedy, a Democrat; insurers; academics; businesses; hospitals; and advocates for the poor, including religious leaders.
When pigs fly indeed!
Let the debate begin.
I recently had the privilege of discussing the future of drug development with CMPI advisory council member, Joseph A. DiMasi, PhD, Director of Economic Analysis Tufts Center for the Study of Drug Development, Tufts University.
Here are some tidbits you may find of interest —
Q: How will 21st century science help advance the future of drug development?
A: There are hopeful signs that new technologies and analytical approaches will improve the pharmaceutical R&D process in the 21st century. If successful, they will reduce costs and facilitate getting the right drugs to the right people at the right time. There is a growing recognition that genetic and other biomarkers that predict efficacy and toxicity responses or measure disease progression need to be developed, validated, standardized and included in drug development programs. The resulting increase in predictive power can eventually permit smaller and more informative clinical trials. Sharing of blinded clinical outcomes data from failed as well as successful trials across companies can also improve the efficiency of the clinical development process. Bioinformatics, data mining, and Bayesian statistical analysis can also help. Given the high costs of investigating the numerous drugs that eventually fail in testing, advances in discovery and preclinical development technologies that result in a higher hit rate for successful drugs can substantially reduce development costs.
Q: Will these advances impact cost?
A: All of the above technologies and techniques can lower the overall cost of drug development. It is hard to know how much they will do so, but reductions of one-quarter or one-third in average costs seem attainable from increases in success rates or reductions in development times.
Q: What can the FDA do to facilitate these improvements?
A: There is much that the FDA can do, in conjunction with industry, academia, and other government agencies, to modernize the development and regulation of new medicines. These include collaborations to develop and validate biomarkers, facilitating the analysis of pooled data on outcomes across clinical trials, developing guidance on how pharmacogenetic data can be used in labeling, how to utilize outcomes data from post-marketing drug surveillance, where and when it is appropriate to use Bayesian analysis and observational studies, and the evaluation of and standards for electronic medical records submissions.
As we’ve discussed before — how about a standing FDA advisory committee on communications issues? See attached article from this week’s Pink Sheet.