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The UK has launched a pilot of its "Innovation Pass" process, which will provide £25 million in funding for medicines that treat very rare diseases but are not evaluated at launch by the National Institute for health and Clinical Excellence (NICE).
Innovation passes were first proposed in the UK's office for life sciences' Life Sciences Blueprint, issued last year. The government has run a public consultation on the proposals since then.
There’s a maximum spending cap of only £8 million per year for each individual product – and uncertainty over long-term funding. The pilot is to run for three years, but government funding of £25 million has only been arranged for the first year.
Products included in the pilot Innovation Pass scheme will automatically be appraised by NICE after the end of three years, raising the suspicions that they could be rejected for use in the UK national health service (NHS) at that stage.
NICE giveth and NICE taketh away since it will set up and run an advisory committee that will select products based on defined criteria. These criteria include the medicine being a significant medical innovation (acting at a new target receptor, for example), it should satisfy an unmet clinical need, and is expected to have a substantial impact. Additional studies to gain further clinical data should be planned.
The government will pay an amount to the pharmaceutical company for supplying the medicine based on a price-volume agreement (the number of patients multiplied by the price), rather than paying for each dose of drug dispensed. This is to ensure a financial return for the company. Products would normally be submitted for consideration at around the same time as they are filed for marketing approval.
BUT … the sum asked for by the company will be judged to be reasonable or not by a governmental/NHS panel that will look at the cost of therapeutically similar medicines, the actual cost of the medicine in other European countries, and the cost of its research and manufacture.
This judgment on whether the cost is reasonable or not will then be passed to NICE's advisory committee, which will produce a list of drugs for funding, which will be approved by government ministers.
Sounds familiar.
NICE work … if you can get it.
FDA Chief Scientist Jesse Goodman to Representative Rosa DeLauro (D/CT and Chairwoman of the Agricultural Appropriations Subcommittee), "I think what FDA really needs is a 5- to 10-year building effort/re-building effort. And it's not just rebuilding to what was. I think it's being a part of building the science of the future."
Bravo. By all means. Rather than look backwards to “the good old days” (whatever that means) let’s improve and move forward. It’s not rocket science – but it’s good to hear the agency’s Chief Scientist say it.
And change starts from within. According to Goodman, "What I want to do is begin to use the resources we have and the leadership we have to encourage and identify and free-up some of the time of our promising junior and mid-level people to beef up their education. Because if we just do it as leaders of the center or agencies, that doesn't have all the transformational power."
Absolutely right. When I served at the FDA (along with Jesse), one of the most valuable lessons I learned was that dictates from “on high” don’t get the job done. Real change happens because all levels of the agency understand and embrace the philosophy of those changes. Change may begin at the top – but success or failure is determined by the agency’s 11,000+ career professionals.
Change is never easy but, as W. Edwards Deming commented, "Change is not required. Survival is not mandatory."
Goodman: “"It's very challenging. It's almost like taking the current state of the agency from sort of always swimming to keep our heads just above the water to something that is a truly outstanding scientific partner and really has the power and relationships it needs. And by power I don't mean power over people, but the mental and scientific tools to have the ability to make decisions. So it's a process and it's going to take a while and we need to get the best and the brightest."
Now maybe Representative DeLauro will finally embrace the Reagan/Udall Foundation.
- 24 March 2010, The Washington Post (By Ruth Marcus)
In fact, the occasion called for more humility than hyperbole, however unlikely that may have been given the setting. If I were a member of Congress, my floor speech before casting a yes vote would have boiled down to:
Gee, I hope this works.
One of the astonishing aspects of the health-care debate is how little is actually known about the implications of a change this far-reaching. Everyone has a theory, and a model to match, but even some of the most fundamental questions remain the subject of debate.
On the most basic of all -- does having health insurance lead to better health? -- the evidence is solid but not unanimous. The Institute of Medicine , reviewing the literature in 2009, found that "the body of evidence on the health consequences of health insurance is stronger than ever before. . . . Simply stated: Health insurance coverage matters ."
But a study that same year by Richard Kronick, a former health-care adviser to President Bill Clinton, found "little evidence to suggest that extending insurance coverage to all adults would have a large effect on the number of deaths in the United States ." Kronick's study has been criticized because it did not adjust for the fact that those in poor health are more likely to seek insurance. But the disagreement underscores the difficulty of knowing precisely what changes are in store.
To take another example, one common assertion has been that the uninsured end up getting health care -- just more expensive health care, in emergency rooms and when conditions have worsened, with the costs passed on to the rest of the population. The notion that the tab is being picked up one way or another makes intuitive sense.
A new National Bureau of Economic Research paper by Michael Anderson, Carlos Dobkin and Tal Gross questions this assumption. The researchers examined health-care consumption by 19-year-olds who had just been dropped from their parents' coverage. They found that not having insurance resulted in a 40 percent reduction in emergency room visits -- "contradicting the conventional wisdom that the uninsured are more likely to visit" the emergency room and a 61 percent drop in hospital admissions.
"Overall, these results suggest that an expansion in health insurance coverage would substantially increase the amount of care that currently uninsured individuals receive and require an increase in net expenditures," the authors write. Emergency room visits could increase by 13 million annually, and hospital admissions by 3.8 million, they project.
So prudence is in order when tinkering with such an interconnected system and when making confident predictions about the effects of reform, for good or ill. Will younger adults, who account for about half the population of uninsured non-elderly adults, sign up for coverage -- or will they pay the fine instead? How will that decision affect premium levels and the adequacy of federal subsidies?
Will the expansion of coverage create a shortage of health-care providers and result in higher prices, or will, for example, higher Medicaid payments for primary-care doctors stem an exodus of doctors from the program? Will employers add coverage because workers facing the mandate to obtain insurance will press for it, or will they drop it because it will be cheaper to pay the penalty and let employees fend for themselves?
Will increased coverage of preventive care save money because diseases will be caught earlier -- or will the added cost of widespread screening exceed the economic benefits? The Congressional Budget Office has concluded that, "for most preventive services, expanded utilization leads to higher, not lower, medical spending overall ."
The legislation is a risk worth taking. Millions of Americans are without insurance, a national scandal that should have been addressed long ago. Rising health-care costs threaten the nation's fiscal security, and the new law holds the promise of beginning to stem the increases.
The status quo is unsustainable. A new study by the Urban Institute shows how, without reform, the numbers of the uninsured will rise, employers will continue to drop coverage and premiums will climb.
Still, for those who express cocky certitude about how this is going to turn out, the best prescription is a generous dose of caution
Lynne Newhouse Segal was the picture of robustness. At 59, the slim former lawyer was an avid runner, golfer and yoga practitioner. Segal, who lives in San Francisco, was healthy by nearly every measure — except her cholesterol level, which a routine test four years ago revealed was high. High cholesterol is a key risk factor for heart disease, especially in a patient Segal's age and with her family history (several close relatives had had heart attacks), so her doctor put her on a cholesterol-lowering statin drug as a preventive measure.
Oh, but dark clouds soon gather...
But Segal's statin ended up preventing her from living a heart-healthy lifestyle. A month after she started taking the drug, she suffered muscle pain so severe, she had to stop all physical activity and was unable to sleep at night. Although her husband, who was worried about her risk of heart attack, pleaded with her to stay on the drug, she discontinued using it. The muscle pain receded. "My husband was scared for me. Doctors scare you. But I was in so much pain, I told him I would have rather died than stay on them," says Segal.
That grim situation could have been avoided, researchers say.
How about asking for another type of statin? That's what Marilyn Goldberg, age 75, did after suffering muscle pains so severe she complained to her husband Mort and son Bob (that's me) almost daily... And she did, about 4 or 5 times until she found one that worked. And now the pains are gone and her cholesterol is really low.
But you see, Marilyn is not a set piece or prop in some Obama-esque sob story about how we the people are screwed by uncaring corporations. She is a responsible person taking action on her own behalf...
Rather died?
I don't want to harp on this self-pitying because I am half-convinced that it is only half-true. That's because much of the "reporting" in the Time article on women and statins is half-baked. You read the piece sensing that women receive NO benefit from statins and tons of risk. In fact, there are many sub-groups and many situations where that is not the case.
The issue at hand is whether statins should be given to women who do not have high cholesterol at all but have other risk factors because cholesterol is not a very good marker for heart disease in women... (my emphasis)
The JUPITER study was designed to test whether other markers of heart disease in relatively healthy older women and men predicted heart disease by giving them statins to reduce a protein called CRP. If CRP was reduced and nasty events that lead to hospitalizations or death were reduced after taking a solid dose of the statin then the study could claim to show that CRP testing in combination with statins reduced the incidence of heart disease in relatively healthy women and men. And since women do not seem to benefit from reduced cholesterol levels (in the absense of other risk factors) the CRP -statin link is a pretty big deal for primary prevention.
As for the side effects.. When you draw the blood for CRP you can also draw blood to see if you will metabolize specific statins in ways that cause muscle pain, blurry vision, etc. There are tests for that. The question is whether the statin used in the JUPITER study (Crestor) can be substituted with other statins.
Now that is what the TIME article should have been about. But I guess the magazine had to devote resources to another hit piece on Israel.
Read more: http://www.time.com/time/magazine/article/0,9171,1973295-1,00.html#ixzz0j6iJUqhy
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.
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.
