Latest Drugwonks' Blog

Geeta Anand had another sob story about the high price of biotech drugs. This time it was about Revlimid, Celgene’s drug for treating multiple mylema, Myelodysplastic syndrome (MDS) an illness associated with the underproduction of blood cells and possibly ALS if the results with transgenic mice are replicated in human trials. Geeta is upset that Revlimid retails for about $6400 a month. Mind you, that’s for a drug which appears to extend for a year or more the lives of about 6000 people who are facing certain death. The cancer stopped progressing in most patients after seven treatments and they were disease free 9.6 months after followup. People with multiple myeloma can live disease free for years. A true breakthrough. Is that worth $6400 a month. A house in the Hamptons is. So is a month at Harvard for the matter.

Revlimid is also approved to treat patients with transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities. That is, it’s a genetically targeted therapy for MDS which itself affects about 10000 people a year.

Both diseases are devastating, either fatal or life crippling. But they are not blockbuster in size. They are orphan diseases. Revlimid is a medicine that will control them with specific regimens and in specific quantities. As it is studied further, funding for which will come from Celgene’s well deserved profits and not any idiotic Cracker Jack prize competition suggested by Jamie Love or Bernie Sanders in lieu of real rewards (let’s divide .5 of our GDP by the contribution of the increase to the quality of life years divided by the number of patients, less the total cost of care, less the NIH investment which equals the reward less the 90 percent tax rate…) and in the wake of patent seizures, we might see even wider and newer uses, including ALS.

And one more thing. What if Celgene is a one hit wonder? Celegene now has a full pipeline of products in development. What if none of them get approved? Revlimid might have a 97 percent gross margin but that goes to pay for all the other research. That’s the company’s venture capital for future biomedical discovery activity. You can’t finance the future of medicine on WSJ criticism or Jamie Love’s Cracker Jack prizes. And by the way, isn’t worth $72k to beat back cancer?

You know how we feel about “evidence-based medicine” — that quotation marks are required since what it really means is “cost-based medicine.” Here at we are firm believers in patient-centric medicine with no quotes required because that’s what it means — putting the patient at the center of the health care equation.

We are also apostles of the Critical Path, of personalized medicine, of the Biomedical Century. And so it is with great interest that we recommend an article from The New York Times, “Imperfect, Imprecise But Useful: Your Race,” by Denise Grady. Here’s the link:

The article addresses the question, “Is race medically relevant?” According to Dr. James P. Evans, director of adult genetics at the University of North Carolina, “The question remains, does any of the differential distribution of gene forms have potential medical significance. I think the answer is sure. There may be differentially distributed genotypes that put a group, in aggregate, at increased or decreased risk for certain diseases or affect their responses to certain medications.”

Yes indeed. More information is better.

Are we there yet?

Ms. Grady writes, “Ideally, he said, doctors would like to know precisely which genes in each individual explain susceptibility to disease. But for most diseases the science is not there yet.”

No better argument exists for the need to accelerate down the Critical Path.

“In the meantime,” says Dr. Evans, “If we can glean some hint from someone’s ancestry, we should use it if it can help us treat them better.”

Drugwonks Exhibit A: The urgent need to aggressively identify and exploit biomarkers.

Yes — there are Luddites. Consider the statement of one editorialist, “In medicine there is only one race — the human race.” Nice sound-bite but, as the NEJM reports, important racial differences exist in the way people react to various medicines, including drugs used to treat high blood pressure, heart failure, depression, and pain.

How does this relate to patient-centric medicine? Ms. Grady writes, “The differences could affect the dose a person needed, or whether a particular drug should be used at all.”

The battle for using “continent of ancestry” (the PC term used in place of “race”) is similar in tone and temperament to the “evidence-based” versus patient-centric debate. Just as “cost” must not be the only issue in determining the most appropriate treatment for an individual patient, so too must race (pardonnez-moi — continent of ancestry) not be excluded because of political considerations.

Patient-centric, n’est pas?

Ms. Grady writes, “As a patient I would like to know about this kind of thing. More important, I would really like my doctor to know about it. If information linked to race could help somebody even a little, it would seem worth having. But it could be lost if researchers become wary of studying this subject or even talking about it.”

Is there even a debate here? Do we want doctors to use all relevant information so that their recommendations can be as targeted, effective and safe as possible? Or do we want them to close their eyes and write the prescription?

This just in about the dangers of taking high doses of Tylenol, especially if you are knocking a couple back this holiday week. I fully expect the new darling and David Graham of the media, FDA Drug Safety numbers cruncherRosemary Johann-Lang, who complained “How does one justify balancing the risk of fatal liver failure against one day less of ear pain?” about Ketek — the last line of defense for many against community acquired pneumonia — to rise up and leak a memo claim “how does on justify balancing the risk of fatal liver failure against one day less of fever?” which in turn will be interpeted by members of Congress and the media as a call to take Tylenol away from everybody… (More on Rosemary’s obsession with insignificant safety signals in other areas in another post)

High Tylenol doses linked to liver woes

By CARLA K. JOHNSON, Associated Press Writer

Healthy adults taking maximum doses of Tylenol for two weeks had abnormal liver test results in a small study, researchers found, raising concerns that even recommended amounts of the popular painkiller might lead to liver damage

Lots of polls and pols are reminding us almost daily that confidence in the FDA is on the decline. How much of this is caused by posturing and putting politics in front of the public health? A lot. But the numbers don’t lie.

So what can the FDA do to regain the confidence of the American public? I think that a large part of the answer is to tell the truth — that the agency is the world’s gold standard — and they’ve got the proof to back up that statement. As a mentor of mine once opined, “He that tooteth not his own horn, that horn shall go untooted.”

As Dizzy Dean said, “It ain’t bragging if you can do it.”

Just doing the right thing, day after day isn’t enough. Just addressing some of the most difficult and important regulatory issues of the day isn’t enough. Just working likes dogs for government pay isn’t enough. FDA must go on the offensive, knocking down untruths, mistruths, and half-truths one-by-one everytime they are floated in the media, in press releases, in public statements by grandstanding public officials, by vendetta-driven “whistle blowers.”

All of the research that I’ve seen makes it clear that the pubic wants to have faith in the FDA. They are willing to believe the FDA. But the FDA must go out and get it. Mix it up. Engage in the debate. The FDA has the bona fides while those basking in the Splendor in the Grassley are but paper tigers.

And the time is now. With the pending release of the Enzi/Kennedy bill and PDUFA reauthorization, now is the time to set the agency on an aggressive, accelerated Campaign of Confidence.

What better way to commence the FDA’s second hundred years.

Mark. My Words.

  • 06.30.2006

I am still recovering from Wednesday evening. The ever gracious Grace-Marie Turner (of Galen Institute fame) hosted a memorable launch party for the Center for Medicine in the Public Interest (the public policy home of

Memorable for so many reasons but mostly because of those who attended — FDA Deputy Commissioners Janet Woodcock and Scott Gottlieb, NCI Deputy Director Anna Barker, Julie Goon, the new White House health care policy guru, along with my former FDA colleagues Dan Troy and John Taylor, former NIMH director (and CMPI board member) Dr. Fred Goodwin — and our keynote speaker, Dr. Mark McClellan the hardest working man in health care. Here are some of the kind things Mark had to say:

“… CMPI is focusing on the right issues at the right time. And with the right people.”

… CMPI is about great ideas and effective communication. CMPI begins at the right time, promoting ideas that make a difference in medicine that is personalized, predictive, preventative, and therefore effective in the 21st century.”

… It’s nice to see ideas, communicated effectively, that translate into policies that change the way healthcare is being delivered in this country. That is happening because of what Peter and Bob and all of you here tonight are doing day in and day out, tirelessly, often thanklessly — but it is making a huge difference.”

“… I’m certain that CMPI will be an important new force in health care policy — a force that will help to get better healthcare for all Americans.”

Well golly.

That’s a lot to live up to — and we intend to exceed expectations.

Here’s a press release from the Office of Senator Chuck Grassley which, along with the Senator, a edging towards a complete disconnect with reality when it comes to Ketek….

Sen. Chuck Grassley issued the comment below in response to the
announcement today about Ketek and a new bold warning label. Sen.
Grassley has been investigating allegations about the FDAâs handling of
Ketek and failure to ensure the integrity of a pivotal study about the
benefits and risks of this drug. The FDA continued to cite the study in
safety information despite its own determination that the study was
riddled with fraudulent information.

Grassley comment —-

âThere are questions about whether this drug should stay on the market,
and thereâs great legitimacy to those questions
. Ketek is another
example where the FDA accommodated a drug maker and turned a blind eye
to serious safety concerns. …..â

Is there anyone who questions whether Ketek should stay on the market? Who are they and what are there reasons? Does Senator Grassley have sources? And if he does, will he provide them or will we at drugwonks have to send someone over to his over to demand them?

We hope to have answers soon.

New op-ed, “Putting Clinical Trials in the Dock.”

Have a read:

Putting clinical trials in the dock
The Roanoke Times
June 27, 2006

BY: Peter Pitts

Pitts, a former Food and Drug Administration associate commissioner, is director of the Center for Medicine in the Public Interest.

“One size fits all” rarely does. From clothes to shoes to hats, few people find that items carrying that label work with their individual bodies. So why do we entrust the health of our bodies — one of the most important assets we have — to a one-size-fits-all mentality?

Unfortunatly, that’s exactly what the influential movement known as “evidence-based medicine” does. It sounds like a good thing, does’t it? Of course we want our health care based on evidence. But the phrase is misleading. As it turns out, “evidence-based medicine” often ignores the most critical evidence of all — the individual patient.

Advocates of EBM urge doctors to base their clinical decisions on research findings. Randomized clinical trials become crucial under this regime: The results of these trials are what doctors overwhelmingly base their suggested treatment on.

The problem is that clinical trials aren’t the only thing doctors need to take into account when making decisions. Evidence-based medicine emphasizes just one aspect of the clinical pie over all the others. This model, which began taking shape in the 1970s, is now broken and outdated.

EBM is stuck in the past. For the most part, it is a retrospective look at clinical studies and head-to-head comparisons of medicines and medical procedures. EBM may involve a careful look at the science. But in practice, it’s very limited. All of these studies are population-based, have rigid exclusion criteria and can’t integrate new information or innovations.

The result is decidedly and transparently a narrow one: to eliminate “practice variation.” Indeed, that’s one of the primary aims of EBM — to standardize medicine around what is known to work.

Practice variation is any treatment that varies from the norm that EBM prescribes. The canard of evidence-based medicine is the belief that practice variation is bad and that one-size-fits-all medicine is good. EBM presupposes that all people respond precisely the same way to all medicines. But that’s simply not true. Disease varies by individual, and selection of treatment must be driven by diagnostics, not just guidelines.

Today, the science of genomics is ushering in an age of personalized medicine. Clinical outcomes can be monitored with increasing precision. Computers can control for hundreds of variables to help doctors and researchers identify what treatment steps matter most in improving care.

People can be screened with a variety of molecular diagnostics to reduce side effects, increase compliance, improve outcomes, and even prevent various forms of cancer, depression, hypertension, Alzheimer’s and immune disorders.

This is 21st-century evidence-based medicine — patient-centric and cost-efficient.

The triumph of modern medicine is that it can be so precisely targeted to a single patient’s needs. It is a dramatic leap forward from EBM’s sweeping approach, which sees only through the broad lens of population-based studies, and the individual patient is kept out of focus.

Unfortunately, EBM continues to enjoy broad support in the policy community. Why? Because at its core, evidence-based medicine is cost-based rather than patient-based. In other words, its standardized approach supposedly saves money. But this is extremely short-sighted.

Evidence-based 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 ends up in the hospital because of improperly treated cardiovascular disease.

The repercussions of choosing short-term thinking over long-term results and cost-based medicine over patient-based are pernicious to both the public purse and the public health.

We need a new approach. The health care community must work together to develop new cost-efficient programs that account for modern genomics and individual screening. Because “one size fits all” treatments are dangerously outdated in this era of patient-centric medicine.

Generic Competition

  • 06.29.2006

Some people, like Senator Schumer, thinks it is wrong for drug companies to reduce their prices and co-market generic version of products nearing the end of patent life. As is Hatch-Waxman is a law that turns Para 4 challenges into an annuity. Sorry. That’s not what the law or the federal court says. And moreover, more competition means lower prices over the life cycle of the drug. As for the idea that Merck has “forced” managed care companies to impose a higher copay on generic Zocor, an allegation that Schumer served up, the fact is HMOs use higher copays to drive people to lower priced drugs all the time. I don’t like the practice because drug choice should be based on what’s best but that’s the game. Whining about it won’t change a thing and it shouldn’t.
Ultimately, the opposition to the Zocor price shift is just an effort to protect the generic drug “industry” and not consumers. It is ironic that the same people who belittle Rx and bio industry arguments about the impact of price controls on incentives for future investment are now arguing that the Zocor price shift and authorized generic movement —which is temporal and depends on blockbusters by the way — will undermine the long term incentive of generic companies to….initiatie patent challenges. What a loss. Fewer lawsuits.

Goodbye Delhi

  • 06.28.2006

The FDA has decided to shelve its plans of opening an Indian office to help expedite regulatory clearances for Indian drug exporters to US.

Sources said Dr David Lepay, the FDA’s Senior Advisor for Clinical Science and Director, GCP Programs revealed at a session on Asian clinical trials at the 42nd annual meeting of the Drug Information Association (DIA) in Philadelphia, USA, that the US food and drug regulator was not planning to set up offices neither in India or anywhere else in Asia, at present. The announcement from the FDA official came in the presence of Dr Ashwini Kumar, Drug Controller General of India (DCGI), who was also a speaker at the program.

Pharmabiz had reported about two years ago that the FDA was planning to start its Indian office at New Delhi with an initial investment of about $5 million, considering the fact that India has the maximum number of FDA-approved facilities outside the US. This office was to facilitate Indian companies with the procedures of filing for marketing approval for products to be launched in US, application for site inspection and other procedures dealing with the FDA. The office was envisaged to facilitate the exchange of communication between Indian companies and the US authorities, thereby saving a lot of time and resources, sources had told Pharmabiz.

Dr Leepay told the seminar, attended by a good contingent of Indian participants, that it was not mandatory that a new drug marketing application in the US be supported by a US study and there had been instances of marketing applications wholly supported by non-US studies. However, the FDA had criteria that non-US studies were expected to meet US standards and that non-US data was reviewed to the same standards as data from the US. Though the FDA had a long history of inspection of clinical trials outside the US, its experience in the Asia/Pacific region was limited, and the region currently accounts for only 5 per cent of FDA’s international inspections. He also clarified that the FDA does not certify clinical investigators, clinical sites, sponsors or ethics committees, nor does it approve study protocols or informed consent documents.

Here’s one of CMPI’s Scientific Adviser Peter Hotez, (and recipient of a Gates grant for his work on the development of a vaccine against hookworm) on how the Bill and Melinda Gates Foundation does it’s good work…


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.

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