Latest Drugwonks' Blog
ChemGenex has agreed to meet with the FDA discuss developing a validated assay test for determining which patients might benefit from its experimental chronic myeloid leukemia (CML) treatment Omapro (omacetaxine mepesuccinate).
"This is a comparative efficacy claim that somebody is making here, so the level of proof has to be there," said Richard Pazdur, director of the Office of Oncology Drug Products in the Center for Drug Evaluation and Research. "The message is that the agency is trying to get across is that attention has to be paid to these in vitro diagnostics." ChemGenex announced the initiative shortly after the FDA's Oncology Drugs Advisory Committee voted 7-1 to require such testing before approving Omapro, which is targeted at CML patients who have failed Novartis' Gleevec (imatinib).
Speaking of Novartis, the ChemGenex situation is another important example of the role companion diagnostics can play in bringing new drugs to market.
In August 2009, Novartis’ two-year effort to revive its Prexige pain pill (after it was rejected by
With a test showing that a certain drug will be safe, Nohaile said, “We can go to doctors and say it moves it out of the realm of choice into the realm of malpractice if you don’t use this drug.
Exciting stuff -- but once these diagnostics are developed, will payers reimburse? In the wake of the payer disquietude over spending $400 on a diagnostic test to determine whether a patient should be given warfarin, it’s very much an open question. (Even more peculiar considering that conservative estimates project using the warfarin diagnostic -- specifically called out in the amended FDA label -- will prevent 85,000 serious bleeding events and 17,000 strokes annually in the United States. And this “safer use” is estimated to save $1.1 billion annually.
Stay tuned.
Two headlines on the same day... March 22
Major health care changes won’t take place until 2014
Delays could help Obama’s reelection hopes
Key elements of health reform would start soon
The Pink Sheet reports on "renewed optimism" that the European Union's proposed legislation on allowing drug makers to provide information to patients on prescription-only medicines will again start moving through the legislative process. The latest thinking, however, is strongly focused on the rights of patients to receive such information, rather than industry's right to disseminate it.
An interesting and important finesse – the rights of a patient to the information but no “right” for industry to provide it. Hm.
Suggested amendments emerged on March 10 from European Parliament Member Christofer Fjellner. He's reviewing the proposed legislation for the EU parliament's Committee on the Environment, Public Health and Food Safety.
For patients, Fjellner contends that information on pharmaceuticals should only be made available to patients who are actively searching for it, i.e., information should be "pulled" by the patient rather than "pushed" by industry. Fjellner believes that companies should not be allowed to make available information on prescription-only medicines on television or in newspapers or magazines. He believes the Internet is the appropriate medium for providing information to patients.
And maybe he's right -- but is there really a difference? If a pharmaceutical company makes available information on a web page – why is that different than making it available in other media? Hm. And what about patients who do not have access to the Internet -- what about their rights?
Fjellner's stance comes as a response to a ruling from the European Court of Justice, which concluded in April 2009 that current legislation could be applied to independent journalists. One journalist, Frede Damgaard, was considered to have broken the ban on DTC advertising by writing about a particular prescription-only product, a ruling that has caused an outcry in the media.
For more on this issue, see “Eighty-Sixing Free Speech.”
After a feisty year of debate, Congress has passed healthcare reform legislation. Once enacted, it will increase the numbers of Americans with health insurance as well as both the size and scope of government. There’s also the very real danger that the legislation will further erode the stake that physicians have to practice both the art and science of medicine.
And the numbers? Staggering when you consider they are absurdly under-scored. According to Douglas Holtz-Eakin (director of the Congressional Budget Office from 2003 to 2005), if you strip out all the gimmicks and games and rework the calculus, a wholly different picture emerges: The health care reform legislation would raise, not lower, federal deficits, by $562 billion. Even with readjustment, it will make the Social Security trust fund look like Fort Knox. The day the President signs this into law could be viewed by a near-future generation of Americans as a day of infamy -- if we let it.
So here’s the good news – the solution is innovation.
We have to embrace innovative technologies for medical records and prescribing. We need innovative clinical trial designs and molecular diagnostics so that we can develop better, more personalized medicines faster and for far less then the current $1 billion plus delivery charge. We need innovation in access and reimbursement policies that rewards speed-to-best-treatment rather than more lower-cost patients per hour.
Will more people have access to health insurance? They will and that’s a good thing. But, let’s be honest, we’re not talking about erasing the word “uninsured” from the American healthcare dictionary – we’re just redefining what it means.
We have to embrace the fact that we will all pay more in taxes (yes, all of us) eventually. And, ultimately, we will be okay with that. Americans are always willing to do what’s right for their fellow citizens. As Winston Churchill said, “Americans always want to do the right thing – after they have tried everything else.” Even so, many of our fellow Americans will receive less comprehensive healthcare benefits than they are receiving now.
So we’d better start taking innovation – of both the incremental and discontinuous varieties – seriously. And that means both spending more on harder developmental R&D (with concomitant higher investment risks). In this regard, the new legislative language on the development of FOBS (follow-on biologics or, if you prefer, biosimilars) is a good thing. (And don’t ever call them generic biologics!)
There’s lip service to the need for more robust comparative effectiveness – although this is a battle yet to be either defined (comparative effectiveness or cost effectiveness or clinical effectiveness?) or fought (do we need a U.S. version of NICE?). And a battle royal it will be. In addition, there’s as yet-to-be reconciled language on a Medicare advisory board that could very well morph into a national formulary body. L’audace, l’audace, toujours l’audace. This isn’t even the end of the beginning.
Of course we bid adieu to the infamous Medicare Part D Doughnut Hole. Pax vobiscum. The Medicare prescription drug benefit is coming in hundreds of millions of dollars under budget already and consistently has 90% + approval ratings by America’s savvy seniors. Medicare Advantage programs? Don’t ask.
Now insurance companies can’t turn anyone down because of a pre-existing condition (bravo!) but they can’t charge higher premiums for people who have them? This isn’t an elegant or economically viable solution and will have to change. Otherwise it’s just a slow march to a single-payer system.
Over the past year, we spent a lot of wasted time throwing around terms like “death panels” but, at the end of the day, we didn’t even begin to address the elephant-in-the-room issue of how much of our national treasure we spend on end of life care. We will have to address this highly volatile and divisive issue – and sooner rather than later.
The legislation doesn’t do anything really significant about driving young, healthy people into the insurance pool. The anemic penalties (which don’t even kick-in right away – the demographics and politics aren’t too hard to figure out) actually disincentivize youthful participation. After all, why not pay the monthly penalty (which is less than even a very affordable monthly insurance premium) if, when you do face a medical emergency, you can’t be turned down or charged more? Nor does the bill create any sort of national insurance pool – where we can all benefit from a 50-state economy of scale insurance marketplace.
Some of the best things about the bill are what is does not do.
No drug importation. (Sorry! Senator Dorgan. Hooray! Peggy Hamburg.) And the Non-Interference Clause remains the law of the land. When originally drafted (wisely by then Senators Daschle and Kennedy), we knew then what we need to remember now, that (1) direct government negotiations for Medicare drug prices won’t (according to numerous government studies and leading economists) lower Medicare drug prices and (2) it is the next slippery step towards even broader price controls. And price controls equal choice controls.
So let’s keep our eye on the prize. No, not the November elections – the real prize: better access to healthcare for all Americans. Innovation that focuses on creating a chronic healthcare culture that embraces prevention and prophylactic care. We will not survive as a nation of obese, hypertensive diabetics. Rather than wasting time on spin, let’s redouble our efforts on innovation. Then, when we succeed through brainpower and teamwork (and, hopefully some civil bipartisanship), the circus surrounding this vote and the past year’s partisan political warfare will be but a footnote in American political history.
That applies to the goal of carving up Medicare and controlling healthcare spending through greater government regulation on access to and higher taxes on innovation, with the objective and/or on consequence of discouraging investment overall in order to expand healthcare entitlements:
Washington State, which has made much of it's position as leader in biomedical innovation is a bellweather of what it to come:
tinyurl.com/ydpvubs
Non-Profits, Investors Worry About Proposed Washington State Tax Surcharge Increase
NEW YORK (GenomeWeb News) – Washington state lawmakers are considering more-than-doubling the business and occupation tax surcharge imposed on non-profit research institutes engaged in R&D, as well as their startup spinouts and other biotech businesses — part of an $800 million package of taxes intended to balance a $2.8 billion budget shortfall.
The current B&O tax surcharge on qualified research and development expenditures, other than for capital improvement purposes, is the gross income derived from R&D, multiplied by 0.484 percent. That tax would rise by half a percentage point, to 0.974 under the measure, which passed the state House of Representatives earlier this month by a 52-45 vote, with one representative excused.
ESSB 6143, a bill to modify the state excise tax law, is pending in the state Senate's Rules Committee, which on Thursday placed the bill on a third reading. State lawmakers have extended their 60-day regular session with a special session that began Monday.
"All the R&D community is up in arms about this — as well as the investment community," Robert Nelsen, a co-founder and a managing director of Arch Venture Partners, told GenomeWeb Daily News. "It seems like innovation is under fire at the federal and state level. There is a whole lot of talk from all levels of government about how great innovation is, and the response is to tax it. It is absolutely insane."
Nelsen has joined the state's life sciences trade group, the Washington Biotechnology and Biomedical Association, and the Washington Global Health Alliance, which promotes collaborations in global health research and programs across the state, in fighting the proposed B&O tax hike.
Leroy Hood, president of the Institute for Systems Biology, told GWDN a review of the bill by ISB's senior vice president for finance and operations, James Ladd, found the bill is not likely to hurt the institute in the short term because it can more than make up the tax through several tax credits.
"The longer term is very unclear. I think the biggest impact that it will have is that it will reduce the attractiveness of startup companies in Seattle," said Hood.
Hood has co-founded more than a dozen companies, including five spun out of ISB that are based on its technologies. He recalled having "a long and difficult fight" with the funding venture capitalists and CEO candidates to locate one of the ISB spinouts — Integrated Diagnostics — in Seattle. That firm, which raised $30 million in Series A venture capital financing last fall, aims to develop tests for monitoring organ-specific proteins that appear in the earliest stages of diseases, using genomic and proteomic technologies and discovery data licensed from ISB.
"I won — just barely," said Hood. "This probably won't happen in the future."
He added, "I think the bill is unbelievably short sighted — just what I have come to expect from a state government that does not seem to understand that the future for Washington will be embedded in information-based jobs. This is exactly the wrong approach to facilitating the information—based economy.
"I must say it was one of the big debates I had with myself when contemplating moving to Seattle — namely is the state populist and not forward looking? It looks like my skeptical self was correct," said Hood.
Also joining in the effort is the Seattle Biomedical Research Institute, which has recently re-branded itself as Seattle BioMed. Lynn Zimmerman, Seattle BioMed's director of finance, told GWDN the institute now more than recoups what it pays in B&O taxes by participating in an employee commuter trip reduction program.
"The increase will probably put us over the commuter trip reduction credit amount, and as a result, we are planning to apply for the High Technology Business and Occupation Tax Credit enacted in 2004, of which we currently do not take advantage," Zimmerman said.
Zimmerman noted that most of Seattle BioMed's revenues came from two sources not subject to the B&O tax: contributions from donors and grants.
According to Seattle BioMed's annual report for the fiscal year that ended June 30, 2009, the institute derives 49.5 percent of its revenues from private grants and contracts, another 35 percent from government grants, and 9 percent from contributions.
Private grant revenue grew 20 percent year-over-year, to almost $22.9 million in FY 2009; while government grants rose about 22 percent, to $16.2 million; and contributions slid 16 percent, to $4.2 million.
As the Ol' Perfessor (Casey Stengel) would say, "You can look it up."
Here's what then-Senator Obama had to say in 2005 about reforms in the Temporary Assistance for Needy Families (TANF) welfare program that he and other Democrats opposed:
“The TANF program affects millions of American children and families and deserves a full and fair debate. Under the rules, the reconciliation process does not permit that debate. Reconciliation is therefore the wrong place for policy changes and the wrong place for the proposed changes to the TANF program. In short, the reconciliation process appears to have lost its proper meaning. A vehicle designed for deficit reduction and fiscal responsibility has been hijacked to facilitate reckless deficits and unsustainable debt.”
Yep -- he was against it before he was for it.
Bauhaus guru Walter Gropius said, "Less is more." But it was Mies van der Rohe (known to New Yorkers as the designer of the Seagram Building) who opined, "but more tastes better."
The Congressional Budget Office has released its latest estimate of the price tag of the House of Representative's healthcare reform bill. At $940 billion, this version of reform will cost more than the measures passed by the House and Senate late last year. More is not always better.
CBO also says the bill will reduce the deficit by $130 billion over the next 10 years and by $1.2 trillion over the following decade. That’s right. It will reduce the deficit by significantly increasing federal spending. Only in America.
While they’re at it, they should also predict the weather for the next decade.
Let’s face it: Uncle Sam has a poor track record of forecasting how much new programs will cost. Medicare’s progenitors, for example, stated in 1967 that the entitlement would cost $12 billion by 1990. Actual Medicare spending in 1990 amounted to $110 billion — nearly 10 times the initial estimate. Oops.
CBO’s deficit-reduction estimates are further divorced from reality because they don’t include as much as $371 billion in new spending to fix reimbursement rates for doctors who treat Medicare patients. Imagine that — health reform legislation that doesn’t include payments to doctors. Only in Washington, DC.
Absent congressional action, Medicare reimbursement rates will fall 21 percent next year. Congress has no intention of letting that happen. But the Democrats have decided that they don’t have to include this so-called “doctor fix” in their healthcare reform package — even though it’s critical to preserving Medicare.
No wonder the CBO was able to conclude that the Democrats’ health reform package would reduce the deficit by $130 billion. The bean-counters simply ignored the $371 billion in spending needed to fix Medicare reimbursement rates.
Democrats point to the favorable CBO score as proof that their health reform package is a model of fiscal responsibility. But it’s likely that the next generation of lawmakers will look back on these cost estimates with the same astonishment reserved for Medicare’s naïve forecasters back in 1967.
http://content.nejm.org/cgi/content/full/NEJMe1002322
The finding that valsartan failed to have an effect on either of the cardiovascular-disease outcomes but had a positive effect on the incidence of diabetes is surprising. Previous studies of ACE inhibitors and ARBs have suggested that these drugs have a beneficial effect on cardiovascular disease in patients with diabetes, and the lower blood pressure achieved would be expected to result in a reduction in cardiovascular disease. In the NAVIGATOR study, the high rates of loss to follow-up (13%), use of off-study ACE inhibitors or ARBs among participants assigned to placebo (24%), and nonadherence to valsartan (34% by study end) could explain the absence of an effect on cardiovascular disease.
The results from the NAVIGATOR study do not support the contention that reducing postprandial hyperglycemia has a specific role in preventing diabetes or reducing cardiovascular disease. Other than increasing the rate of hypoglycemia by a factor of two, nateglinide had little effect. Although the authors suggest that the prevention of diabetes with valsartan might make it a preferred drug as compared with antihypertensive drugs that potentially worsen glycemia, valsartan was relatively weak in preventing diabetes, and it did not lower the rates of cardiovascular disease. The prevention of diabetes remains a critical public health priority, but for now we should steer away from these two drugs and use effective lifestyle interventions and, in selected persons, metformin to combat the epidemic.
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.
