Latest Drugwonks' Blog
Cost-Effectiveness of Cancer Drugs Is Question
Read Wall Street Journal article here"The widespread use of expensive cancer drugs to prolong patients’ lives by just weeks or months was called into question by an article published Monday in the Journal of the National Cancer Institute.
Crunching data from published studies, the authors found that treating a lung-cancer patient with Erbitux, a drug that costs $80,000 for an 18-week regimen, prolongs survival by only 1.2 months.
Based on that estimate, extending the lives of the 550,000 Americans who die of cancer annually by one year would then cost $440 billion, they extrapolated."
Let it be noted that the authors Tito Fojo and Christine Grady work at the National Cancer Institute where, we hope, they are nowhere near patients or even lab rats. This is multiplication by morons... Most cancer drugs don't cost that much, extend life by more, often add to quality of life even if they don't add to length, and by doing so add value and wealth to the economy. And then there is the obvious -- one would hope -- shift in cancer care towards personalized treatment, providing medicines to those who can benefit, which brings the 1.2 survival way up.
"How to control escalating spending on end-of-life care is one of the thorniest questions facing lawmakers working on the overhaul of the U.S. health-care system.
Some countries, like the United Kingdom, agree to pay for expensive drugs only if they meet a certain threshold of efficacy, but no such rationing exists in the U.S."
Here's how Tito and Company -- trained bioethicists as well as having advanced degrees in multiplication - deal with these sensitive issues...
“Many Americans would not regard a 1.2-month survival advantage as ‘significant’ progress,” the authors wrote. “But would an individual patient disagree? Although we lack the answer to that question, we would suggest that the death of a mother of four at age 37 years would be no less painful were it to occur at age 37 years and 1 month, nor would the passing of a 67-year-old who planned to travel after retiring be any less difficult for the spouse were it to have occurred one month later.”
How does one respond to such wisdom and compassion? Perhaps by suggesting that the authors follow their own sagacious advice when faced with a similar situation and voluntarily refuse treatments that -- on average -- increase survival by 1.2 months but could also do so by 4 years or more.
They would be making such a contribution to science and humanity.
From the IOM report:
"This focus of CER reflects the growing potential for individualized and predictive medicine—based on advances in genomics, systems biology, and other biomedical sciences—through the analysis of subgroups with demographic, ethnic, physiologic, and genetic characteristics that could be useful factors in clinical decisions."
Then there is this:
"A common misapprehension is that CER will lead to uniform, “one-size-fits-all” care that ignores the ways that patients differ. In fact, CER done well should give providers the means to tailor the choice of treatment to the individual patient’s characteristics and preferences. Better comparative effectiveness studies will make it possible to measure the implications of individual differences in disease severity and the presence of comorbidities, to identify predictors of response to treatment, and to incorporate other aspects of a person’s health and preferences. For example, CER might assess the added value of using genomic information in addition to traditional clinical predictors to determine the best treatment for a cancer in a particular patient."
There is no misapprehension. The fact is CER has used data from medical claims and randomized trials to compare drug A to drug B to figure out -- on average -- which one provides the most "benefit" for the least amount of money. And CMS is stil proposing such studies today. Moreover, CER is still framed as head to head comparisons conducted largely through randomized clinical trials that take forever and are biased by the cost contaiment goals of government and by definition exclude patient variation as opposed to continuous feedback loop for improving clinical practice.
Read more here
My letter to the editor of The Hill is a start....
The fact that there have been fewer patent challenges to biologics than for pharmaceuticals is not a rationale for weakening patent protection for biologics..... Is that the new standard for economic and science policy for the Obama administration? Or is it all about cost?
Eshoo drug legislation better than Waxman alternative bill
By Robert Goldberg, Ph.D., co-founder, Center for Medicine in the Public Interest
Posted: 06/30/09
An important part of the healthcare debate is the two competing pieces of House legislation that give the Food and Drug Administration the authority to create modernized pathways for the creation of generic forms of biologic medicine drugs.
Known as follow-on biologics, or biosimilars, the FDA currently cannot approve these important medicines because of safety concerns over the difficulty in replicating these types of drugs.
House Energy and Commerce Committee Chairman Henry Waxman (D-Calif.) believes the language in his follow-on biologic bill should be included in the broader healthcare legislation in order to reduce the cost of one of President Obama’s top policy agenda items.
On the other hand, Rep. Anna Eshoo (D-Calif.) believes her legislation should go through the standard process of committee markup and onto the House floor on its own, allowing for full vetting and debate on one of the key healthcare reforms of this generation.
Both pieces of legislation have their merits and both members should be commended for their willingness to take a lead on this important issue, but Eshoo’s bill has the right prescription for getting follow-on biologics to the market.
First, the Eshoo legislation puts a priority on patient safety by requiring appropriate and stringent clinical trials and testing. This is necessary because biologic drugs are created from living organisms such as proteins and carbohydrates, and are not as simple to replicate as traditional drugs like aspirin and antihistamines.
Second, by protecting adequate data exclusivity, innovator companies will not be forced to charge more for their biologic treatments.
Third, Eshoo’s legislation rewards new biologic innovation by drug companies because it grants them a longer period of data exclusivity to continue research and development to fight other diseases.
Fourth, Eshoo’s legislation gives hope to those suffering from rare diseases or conditions. If drug companies think they will have a short time before a generic version of their product is on the market, they will only focus on the drugs for major diseases and conditions, potentially ignoring ailments that are less common, but equally as serious, to those suffering.
Follow-on biologic legislation must be about balancing patient safety and cost reduction. To ignore either one — or to unnecessarily rush creating this pathway — will only hurt those patients who depend on follow-on biologics the most.
Consider what Steve Usdin of BioCentury has to say on the subject of biosimilars in the wake of the recent Federal Trade Commission (FTC) report.
“A Federal Trade Commission (FTC) report released last week, and the responses to it from members of Congress and industry, demonstrate that the prolonged debate has not diminished the controversy over the best ways to balance incentives for biomedical innovation with the economic benefits to patients and payers that could be produced by a robust biosimilars market.
The FTC flatly rejected arguments the Biotechnology Industry Organization (BIO) has made to justify its contention that a 14-year exclusivity period for pioneer biologic products is essential to ensure the future ability of companies to attract capital and to invest in new product development.
Indeed, all four FTC commissioners signed off on the report, “Emerging Health Care Issues: Follow-on Biologic Drug Competition,” which argues there is no need for any market exclusivity for pioneer biologics beyond that provided by patents, save for products for Orphan diseases and unpatentable drugs.”
Ouch.
According to the FTC, “There is no evidence that patents claiming a biologic drug product have been designed around more frequently than those claiming small molecule products.”
Double ouch.
Usdin’s complete article is worth a careful read.
Samson, my yellow labrador and constant companion for 14 years, passed away this morning. Just wanted to share a couple of photos and one of my favorite poems to let everyone know who he was and what he meant...
The House Dog's Grave (Haig, an English bulldog)
I've changed my ways a little; I cannot now
Run with you in the evenings along the shore,
Except in a kind of dream; and you, if you dream a moment,
You see me there.
So leave awhile the paw-marks on the front door
Where I used to scratch to go out or in,
And you'd soon open; leave on the kitchen floor
The marks of my drinking-pan.
I cannot lie by your fire as I used to do
On the warm stone,
Nor at the foot of your bed; no, all the night through
I lie alone.
But your kind thought has laid me less than six feet
Outside your window where firelight so often plays,
And where you sit to read--and I fear often grieving for me--
Every night your lamplight lies on my place.
You, man and woman, live so long, it is hard
To think of you ever dying
A little dog would get tired, living so long.
I hope than when you are lying
Under the ground like me your lives will appear
As good and joyful as mine.
No, dear, that's too much hope: you are not so well cared for
As I have been.
And never have known the passionate undivided
Fidelities that I knew.
Your minds are perhaps too active, too many-sided. . . .
But to me you were true.
You were never masters, but friends. I was your friend.
I loved you well, and was loved. Deep love endures
To the end and far past the end. If this is my end,
I am not lonely. I am not afraid. I am still yours.
Robinson Jeffers, 1941
The right answer is to understand the medium and use it to advance both the public health and specific marketing objectives. The two are not mutually exclusive.
Let's take blogs as our example.
Blogging is a potent weapon in both the war of ideas and the marketplace of communications. Blogs are wonderful tools when used appropriately. They are pithy, witty, and timely. A wonderful triple play – but counter-intuitive to many pharmaceutical marketing cultures. Brevity is the soul of wit – but not necessarily of medical marketing.
The tools of the blogosphere are immediacy, emotion, humor, asperity and acerbity. “Let me ponder the nuances of your argument” is just so 20th century. The best bloggers are both incisive and sassy. Blogs act as message accelerators. While a good Op-Ed is absorbed, a timely blog is injected, main-streamed into the collective consciousness of a variety of interconnected audiences. Good blogs resonate.
How can healthcare companies take advantage of this phenomenon? One word – engage. But engage carefully. Legal and regulatory issues abound, but a smart place to start is to spend quality time identifying which blogistas are the most relevant to a product or an issue or a company and then read them every day throughout the day. The best blogs are updated more than once a day. It's hard work, but it's worth it. And, relative to metrics, it isn't the top 10 – it's the right 10.
Blogs are the new frontier of pharmaceutical communications. The best bloggers are activist, educated, unpaid, unprompted and uncontrolled. Blogs are real-time focus groups without either the design or permission of the sponsor. Its voyeuristic marketing – wonderfully, terribly, brutally naked in both honesty and utility. The medium is the message.
And the message is being regularly trawled by the mainstream media for “what's next.” I blog every day and regularly get calls from “traditional” journalists to discuss what I'm saying and where I think its going. And if your cognitive mapping is having a hard time accepting this proposition – ask Maureen Dowd.
Some interesting tidbits from the FDA’s June 24th meeting on the issue of transparency.
“FDA should make it obvious, to those who are interested, how scientific data leads to its approval decisions.” -- PhRMA Assistant General Counsel Jeffrey Francer. As an example, Francer pointed to FDA approval decisions that do not track with advisory committee recommendations. "That's often confusing, not only to those of us in industry, but people generally."
Absolutely. But, since transparency cuts both ways, wouldn’t it be equally beneficial for pharmaceutical companies to proactively release complete response letters received from the agency once a final action has been taken?
Transparency thrives in an atmosphere of clarity. And that was also a topic of conversation.
BIO’s Director for Science and Regulatory Affairs Andrew Emmett, commented that the FDA does not meet and communicate with firms on a consistent basis early in the development and review process. He urged, and appropriately so, that the agency work with industry to minimize barriers to such meetings.
Emmett also called for the agency to provide more information so industry can understand how FDA makes decisions. There needs to be "regulatory transparency and clear articulation of FDA's policies and expectations."
Since it can take several years for FDA to finalize guidance, "This leaves companies to ascertain FDA policy by interpreting the agency's regulatory decisions and enforcement actions, which is an inefficient way for industry to understand and meet the agency's regulatory expectations."
If we’re going to go “open kimono” on this, then the real 800 pound gorilla in the room is the fact that many inside the FDA prefer to leave guidances in draft form – because that ambiguity gives them almost unlimited and perpetual power. That’s why interpretation of FDA actions is such a vibrant cottage industry. Industry, on the other hand, seeks clarity. They want bright lines. They want to know the rules. They want predictability. This may sound simple, but it has proven to be a fractious bureaucratic kulturkampf within the FDA.
Regulators change industry behavior by changing the rules of the game. But changing the minds of regulators, having them embrace bright lines rather than broad definitions, is a distinctly more challenging proposition, because changed minds must begin with change agents within the agency itself.
FDA transparency cannot be achieved without FDA predictability. Predictability is power in pursuit of the public health.
On a related note (and at a different venue – the annual DIA meeting in San Diego), Sandy Kweder (Deputy Director, FDA Office of New Drugs) and Gerald Dal Pan (Director, Office of Surveillance and Epidemiology) discussed the need to begin REMS conversations as early as Phase II. Dal Pan commented that "REMS are as new for us] as they are for all of you. It's not that we hold some secret and we're just not telling anybody about it. We're all navigating through this and trying to figure it out.”
Looks like the FDA is getting down to some serious social science
According to the Pink Sheet, a study proposed by FDA to evaluate how benefit information is conveyed through direct to consumer advertisements could end up showing drug marketers how to describe "high" and "low" efficacy drugs.
The proposed study will evaluate how consumers' interpretations of a drug's efficacy after viewing DTC ads compare to the efficacy data in the product's label.
The study will be conducted in two concurrent parts - one looking at print ads and one at TV ads - and examine three factors: drug efficacy, visual format, and type of statistic.
The agency defines drug efficacy as a quantifiable, objective metric that can be conveyed in graphical representations of the drug versus placebo, with "high efficacy" meaning "noticeably better than placebo" and "low efficacy" meaning "minimally better than placebo."
The agency traditionally has discouraged companies from making such qualitative claims - or even making too much of quantitative numbers - in its advertising. But the proposed study endpoints suggest a growing FDA comfort with, and even an interest in, consumers receiving this kind of information.
Testing Pfizer-like pictographs
The study will examine graphs, pictographs and pie charts as ways to visually present that efficacy information, and whether statistical information is best conveyed as frequency, relative frequency, or percentage.
The test product will be for a cholesterol treatment and modeled on an actual drug, such as Pfizer's Lipitor (atorvastatin), with labeling used as the reference for defining efficacy levels and the objective metrics for clinical performances.
The study will include 4,500 participants, 2,250 in each group (television or print). The subjects will read or view one advertisement version, and then make a series of judgments about the drug in a 20-minute interview.
FDA first will test whether, within each format, the participants were able to distinguish between low- and high-efficacy drugs. Then, it will look at whether the participants' efficacy estimates differ across formats and how accurate the estimates are.
Comments on the proposed study, called "Experimental Study of Presentation of Quantitative Effectiveness Information to Consumers in Direct-to-Consumer Television and Print Advertisement for Prescription Drugs," are due by Aug. 21.
All to the good. And it sure beats the ignorant "drug facts box" – the a la nutrition facts panel idea,that’s being kept alive via legislation introduced by Senators Jack Reed (D, RI) and Barbara Mikulski, (D, MD) which, BTW, would also require comparative effectiveness information to be included in product labeling.
Vey
Roche.
Roche is leaving the Pharmaceutical Research & Manufacturers of America trade association effective June 30.
Roche describes the action as a "business decision" by "the newly combined Genentech-Roche US commercial organization."
According to the Pink Sheet, "As a practical matter, the departure will add to the strain on PhRMA for resources as it works on all fronts to build alliances and promote its reform agenda. The pending mergers of Pfizer/Wyeth and Merck/Schering-Plough will further reduce the group's core member base. The association traditionally adjusts dues following periods of consolidation."
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.
