CMPI TO HOST HILL BRIEFING ON FOLLOW-ON BIOLOGICS WITH REP'S ESHOO AND ROGERS 

Cost-Effectiveness of Cancer Drugs Is Question

"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.  

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

Some interesting tidbits from the FDA’s June 24^th 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 izmir.

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."