Latest Drugwonks' Blog
Why did Willy Sutton rob banks? “That’s where the money is.” This apocryphal aphorism led to what is called “Sutton's Law,” that when diagnosing, one should first consider the obvious.
President Obama and Medicare directors nationwide should pay heed. If we want to lower health care costs (or, more honestly, health care spending), we need to look at where the spending takes place. It’s a classic case of Occam’s razor.
According to the Agency for Healthcare Research and Quality (AHRQ), the top 1 percent of health care spenders is responsible for more than 20 percent of all health care spending. And the bottom 50 percent represent only 2.9 percent of the nation's health care bill.
And while this is not news, it seldom seems news fit to print. After all, it just doesn’t justify a lot of political agendas and editorial opinions that prefer a simplistic, one-size-fits-all single payer national healthcare “solution.”
The National Journal opines, “The data suggest that health care policies designed to reduce spending of healthy populations may be of limited value in reducing overall costs. The big savings can be achieved by reducing the costs of the biggest health care users.”
Entia non sunt multiplicanda praeter necessitatem.
This raises an important question, why didn’t Florida maintain its “Healthy State” program where high-cost Medicaid recipient care was being managed and overall costs were going down? And why aren’t more states aggressively pursuing solutions like the Hoosier State’s “Healthy Indiana” program, where Governor Daniels made personal responsibility central? Instead public and private payers continue to rely on population-based approaches when science and data – and best practice -- suggest that what we require are more personalized and preventative approaches.
To address the 800-pound gorilla in the room, it also demands a thoughtful national dialogue on end of life care. (77% of Medicare decedents’ expenditures occur in the last year of life – 52% in the last two months of life and 40% in the last month.)
And what better time to raise this issue than during a Presidential election cycle.
Will December 7th, 2011, be a day that will live in regulatory infamy?
That’s when Secretary of Health and Human Services Kathleen Sebelius overruled a decision of the Food and Drug Administration (FDA) on the over-the-counter (OTC) status of emergency contraception.
By reversing an FDA decision, the Secretary set a dangerous precedent for all-comers to lobby Congress, the Department of Health and Human Services and the White House on any and all FDA decisions—directly inserting politics into what must be a scientifically driven process.
"Should the HHS Decision to Overrule FDA on Plan B be Reversed?" is addressed head on in the latest issue of the Food and Drug Law Institute’s Policy Forum (Volume 2, Issue 1, January 11, 2012).
I think it’s a timely and provocative read. (After all, I wrote it.)
Statistics are like a bikini. What they show you is interesting, but what they conceal is essential.
Nevertheless, someone at the FDA owes the Pink Sheet a thank-you note.
The Pink Sheet reports that:
The agency has been trumpeting the 30 new molecular entity and new biologic entity approvals from CDER in 2011 – the most since 2004 and a welcome sign of recovery after the approvals doldrums in between – but it also deserves recognition for achieving an unprecedented, near-perfect rate of PDUFA review goal compliance (96.7%) while maintaining healthy rates of first-cycle approvals (63.3%), and time to approval (an average of 15 months)
|
CDER Review Performance In The PDUFA IV Era |
|||||
|
Year |
Approvals (average time to approval) |
User fee compliance |
First-cycle review share |
First-cycle share, Standard |
First-cycle share, Priority |
|
2011 |
30 (15 mo.) |
96.7% |
63.3% |
43.8% |
85.7% |
|
2010 |
21 (15.1 mo.) |
86% |
76.2% |
75% |
77.8% |
|
2009 |
25 (17.3 mo) |
72% |
58% |
47% |
66.7% |
|
2008 |
24 (17.9 mo.) |
70% |
67% |
53.3% |
88.9% |
Consistency is becoming a hallmark of FDA’s review activity, with nearly identical average times to approval in the two years following the adjustment period for implementation of the FDA Amendments Act. The striking difference between 2010 and 2011 is the number of approvals, not the parameters of the reviews – showing FDA can deliver the same performance with a higher volume.
Innovative drugs drove the positive review trends, with 12 first-in-class agents among the CDER NMEs and NBEs, representing 40% of calendar year 2011 approvals.
First-in-class status correlates closely but imperfectly with priority review status, another marker of innovation that incorporates not only pioneer mechanisms but also unmet medical needs. By the metrics, the 2011 class did not vary significantly from the level of innovation in recent years, though the significance of some of the innovative products cleared in 2011 certainly makes the class notable.
The difference in time to approval between the first-cycle and second-cycle approvals is stark. On average, the penalty for going to the second review cycle was 19.6 months, the difference between the 7.8 month average time to approval for all NMEs and novel BLAs (CDER and CBER) approved in one review cycle and the 27.5 month average for products requiring two review cycles.
FDA could not have posted a good year for new drug approvals without plentiful applications based on well-constructed clinical programs – an aspect in the hands of industry. But the agency can also make its own luck. The case studies of drug approvals published each month by Pharmaceutical Approvals Monthly suggest that the agency will show great creativity and flexibility in analyzing and acquiring data for new products that address unmet medical needs.
The Pink Sheet ends it’s analysis with the comment, “As the agency heads into PDUFA reauthorization, FDA’s review success will be a powerful argument that the agency knows what it is doing. Just look at the numbers.”
Just so, but as Vince Ventimiglia, Former Assistant Secretary for Legislation at the US Department of Health and Human Services, commented at a recent Center for Medicine in the Public Interest conference on PDUFA, numbers are very much determined by who provides them. He called one myth about PDUFA, “Lie to Me." When it comes to numbers, he asks, “Are they always true? And the answer is no. It behooves you to look very carefully at FDA numbers, Department of HHS numbers, and OMB numbers as a package. They’re not always in sync with each other."
As Mark Twain said, “Facts are stubborn things, but statistics are more pliable.”
According to executive director Guido Rasi, the European Medicines Agency will issue final guideline on biosimilar monoclonal antibodies in March or April with draft guidelines on the approval process for copies of other drugs to follow in May or June. (These will include low molecular weight heparin and modern analogues of insulin.)
Rasi said he believed biosimilars would play an important role in future European healthcare and also noted that many pharmaceutical companies are developing next-generation versions of medicines.
Rasi, "I don't really have concern about the level of safety. What is a question-mark is efficacy, since many biotechnology products are now being improved."
“Biobetters” anyone?
BioCentury reports:
CDER creating medical policy board
FDA is creating a medical policy board at the Center for Drug Evaluation and Research to enhance the consistency of medical policies, CDER Director Janet Woodcock told BioCentury. Woodcock, who announced the initiative to CDER staff on Friday, said the new board is a response to complaints from drug sponsors that review divisions often take different, sometimes contradictory, approaches to similar issues.
Examples of issues the board will work on include creating a CDER-wide policy for obtaining approval based on a single trial, and the types of evidence that can be used to supplement a single trial. The board also will establish standards for use of the Animal Rule to obtain approvals of bioterrorism countermeasures based on efficacy studies in animals, Woodcock said.
In addition to consistency, the new body will also examine whether approval standards are infeasible, and if they are, establish new standards. The board's conclusions will be published and will be subject to public comment procedures, Woodcock said. It will be headed by Rachel Sherman, director of CDER's Office of Medical Policy
Is the free market the cause or the solution to the problem of drug shortages.?
Yes.
According to a story in today’s Detroit Free Press:
“The most common causes are manufacturing violations, production delays, shipping problems or ingredient shortages … Shortages grow as generic manufacturers have consolidated and fewer plants are left making certain drugs.”
All of these issues can be addressed and resolved through smart free market solutions and the helping hand of economic incentives.
As Scott Gottlieb commented in front of the Senate Finance Committee:
The is a clear correlation between price and availability – with many of the cheapest infused medicines also being the ones that seem most likely to be in shortage. These drugs are often sold for very low prices, sometimes just several dollars for a single dosage vial of a medicine. As a result, the cost of manufacturing ends up comprising a sizable proportion of the overall price of the finished medicine. In some cases, these drugs are being sold at a loss to their manufacturers once all the production and distribution expenses get fully loaded into the cost. The economic problems are widespread, and deeply embedded in the markets for these drugs.
We should consider policy constructs that would give manufacturers a financial incentive to develop intellectual property that improved the manufacturing characteristics of generic medicines even if these changes it didn’t alter the clinical properties of a drug.
Let’s adopt a free-market solution and free American physicians, pharmacists, and patients of the problem of needless drug shortages.
What do Avastin and Plan B have in common?
The answer is that both products received attention from both the FDA and HHS. One was a policy decision, the other a political.
During the FDA’s hearing on Avastin, HHS announce that “The label change will not affect our coverage.” (Actually, he should have said “potential label change,” but neither here nor there.)
That “Yes from HHS” was a policy decision that was also a savvy political one (had CMS withdrawn reimbursement for Avastin’s use in breast cancer, it would have become a front-and-center election issue).
On the other hand, Secretary Sebelius’ override of the FDA’s decision to allow unfettered OTC access for Plan B was a political decision thinly (and awkwardly) couched as a policy decision.
That’s the difference between policy and politics.
The institute is trying to advance the cause of CER without creating a political flashpoint.
By BRETT NORMAN | 12/29/11 9:32 AM EST
So far, the new institute that’s supposed to run comparative effectiveness research has avoided the harsh political rhetoric of health care rationing and “death panels” — but its supporters worry that its luck could be about to change.
The Patient-Centered Outcomes Research Institute, set up by the health care reform law, is walking a fine line, trying to advance the cause of comparative effectiveness research without creating a political flashpoint. And that means getting as much buy-in as possible from stakeholders throughout the health care sector.
In January, PCORI will lay down two markers — its draft research agenda and its national principles — outlining the framework of a national program for comparative effectiveness research. And a lot of people are watching closely.
“They are really operating under the shadow of the death panel crazies,” said Jerry Avorn, professor at Harvard Medical School and author of "Powerful Medicines." “There’s still a lot of fear that even the most reasonable and necessary research — say, the best approach to lowering blood pressure or cholesterol, for instance — will be demagogued and demonized by those on the right.”
But tempers have cooled at least somewhat since the arguments flared in 2009 after the stimulus bill included $1.1 billion for CER. The calmer environment is leading supporters to be guardedly optimistic that PCORI will be able to proceed — albeit cautiously — without excessive political distraction.
“My belief is that much of the storm has subsided — not to say it can’t come back,” said Robert Dubois, chief science officer of the National Pharmaceutical Council. Dubois wrote a paper this month in Health Affairs with advice for how PCORI should prioritize treatments for study — transparently and based on explicit evidence, with input from all stakeholders.
“The combination of the way the legislation was written, the board of governors and the tone that they have set has satisfied most people,” he said.
The point of comparative effectiveness research is to compare two or more different ways of treating the same condition to see which one works best. The idea is that if definitive best practices can be established, they will be widely adopted by providers and may be preferentially reimbursed by payers.
Cheaper treatments that are effective would be favored.
It may sound harmless — like common sense, even, to the uninitiated — but it’s a menacing prospect to some pharmaceutical companies and medical device-makers who are concerned that their products may wind up on the wrong side of the ledger.
For this reason, Michael Cannon, director of health care studies at the Cato Institute, says good comparative effectiveness research is almost suicidal.
“The whole point of [comparative effectiveness research] is to find out what doesn’t work,” Cannon said in an email. “Every time the government has tried to do CER, the guys who provide the stuff found not to work successfully lobby to have the offending agency defunded. I see no reason to think this time will be any different. The moment it produces useful CER, PCORI is toast.”
And that’s just one source of opposition. The other includes general foes of the Affordable Care Act, who have seized on elements like the Independent Payment Advisory Board, and end of life counseling, suggesting that health reform will lead to government “pulling the plug on grandma.”
Betsy McCaughey, former lieutenant governor of New York and author of “Obama Health Law: What It Says and How to Overturn It,” fueled some of the most heated claims in 2009 about threats of government health care rationing.
McCaughey said she won’t prejudge PCORI’s agenda, but that generally “there’s an intense politicization of medical research to support an agenda that wants to save money by denying care to the elderly and sick. … The term ‘comparative effectiveness’ is code for giving care based on age and life expectancy of the patient.”
Supporters say that objection is off base, and that the research is designed to find the most effective treatment for every population.
But even the institute’s name bears the imprint of the red-hot controversy the subject fuels. During the health care reform debate, the very term “comparative effectiveness research” was abandoned in favor of “patient-centered outcomes research.” The latter has an explicit emphasis on the wishes of the patient, rather than hinting that the true goal is cheaper care.
And the ACA specifically states that PCORI will have no authority to mandate coverage or reimbursement, although it doesn’t forbid public or private payers from using the research to inform their coverage decisions, John McDonough, who worked as an aide to Sen. Ted Kennedy, reports in his book "Inside National Health Reform."
PCORI is led by Joe Selby, a widely respected former executive at Kaiser Permanente, who is well aware of the delicate political balance he has to strike.
“Everyone at PCORI, our board and our staff, recognizes the great need for research that compares different prevention, diagnosis or treatment options to see which ones work best for different people with a particular health problem,” Selby said in an emailed response to questions. “That’s at the heart of PCORI’s mission, to provide patients and those who care for them with the information they need to support health care choices.”
After the release of its draft agenda in January, PCORI will award 40 grants totaling $26 million that will fund methodological and agenda-setting research. Selby said PCORI will award another $100 million in primary research funds by the end of the year. The majority of funds will go to comparative effectiveness research studies, he said.
The ACA established a trust fund for PCORI, so it isn’t subject to the annual appropriations process, which provides a measure of security. It’s funded by the government but is independent of it, and so far, in the various debt reduction negotiations this year, it has not emerged as a target.
But laws can change, and even PCORI foes suspect the agency will tread lightly next year.
Robert Goldberg, vice president of The Center for Medicine in the Public Interest, believes that money being spent on PCORI would be better spent elsewhere. “I don’t think there’s any hard evidence that comparative effectiveness actually improves health at a reduced cost,” he said.
He co-authored a paper with John Vernon, an economist at the University of North Carolina, arguing that if a comparative effectiveness standard were built into the FDA’s premarket approval process, the impact on innovation would be severe. “To the extent that it’s just another link in the chain from the time a product is developed 'til the time it’s approved, it’s going to delay access and discourage innovation,” Goldberg says.
Harvard’s Avorn calls this “crying wolf,” because comparative effectiveness has never been a part of FDA review and including it is not in the cards.
And Goldberg acknowledges it is a purely hypothetical scenario — one that can’t be allowed to become reality.
Despite his suspicions, though, he thinks of PCORI as mostly harmless.
“I don’t see PCORI as a real threat. I think they’re more interested in shoving the money out in a neutral way not to upset anybody and see if they can build support for it after 2012,” Goldberg said. “Depending how the elections go, they could be an early target, so they’d like to build some consensus.”
Supporters agree on the last part — the need for consensus — and generally applaud the efforts to bring as many people to the table as possible. Consensus is needed, they believe, not just to keep political foes at bay, but also to advance the cause of comparative effectiveness research beyond the institute.
Large-scale comparative effectiveness studies are extremely expensive. Despite PCORI’s substantial funding, it will never be able to tackle all of the clinical issues that need to be studied.
The goal is to set up a framework that will allow others — insurance companies, research institutes and others — to build on the experience and carry out studies of their own, Dubois said.
Rita Redberg, a comparative effectiveness expert and professor at the University of California San Francisco, said she believes PCORI is doing great work under very trying circumstances.
“I think they’re going to move the needle in terms of what we need to know,” Redberg said. “They’re really doing great with the restrictions they have, but there are a lot of masters to please.”
According to a new report by University of Southern California researchers in the American Journal of Managed Care," "Though some doctors stick to a few highly advertised medications, only a few physicians prescribe them exclusively.”
No surprise there, but useful and timely as we enter into the New Year with the specter of government detailing (aka “academic detailing”) hovering over both patient choice and the overall ability of physicians to practice medicine as they see fit.
"Narrow prescribing patterns may be optimal when one drug is clearly superior to the others, or if all the drugs in the class act in a similar way."
“Superior” in individual patient performance (clinical performance) to be sure.
The researchers also noted “although most physicians have a favorite drug, they are not reluctant to try new therapies.”
Which begs the question – how and from whom are physicians to learn about new treatments?
A tough and important question for 2012. And it’s not, strictly speaking, academic.
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.
