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(When I served at FDA we signed an MOU with PhRMA that required its member companies to inform the agency when instances of counterfeit medicines came to the attention of manufacturers. It got the job done – minus additional federal regulation.)
Shortly afterwards, Senators Amy Klobuchar (D, MN) and Bob Casey (D, PA) introduced legislation to require drug manufacturers to give “proper prior notification” to FDA when there is going to be a drug shortage.
Watch the Federal Register on Monday for an interim final rule from the FDA that requires manufacturers that are the only producers of critical drugs to report to FDA all interruptions in manufacturing processes, regardless of whether a drug shortage occurs. Previously, such manufacturers were only required to notify the agency when a drug shortage occurred. The rule goes into effect 30 days after being published.
This would have been a better and more interesting story if industry had taken the initiative rather than being directed to change its behavior.
What if we’re all wrong? What if prevention doesn’t save money?
Read Sarah Kliff’s piece in the Washington Post here.
According to Plato, “They certainly give very strange names to diseases.” Well, he clearly never encountered PDUFA -- otherwise the might have hit the hemlock too.
Ask me and I'll tell you that PDUFA ought to stand for the "Predictability Deposit User Fee Act." According to Tim Franson, one of the "Fathers of PDUFA," it means “Pharma’s Dollars Underwriting Federal Appropriations.” And when Paul Kim was on the Hill, he wanted to call it the “Giant Omnibus Drug and Medical Device Amendments Act” or GODDMAA.
Alas – cooler heads prevailed.
But PDUFA is more than just a name -- it's a philosophy. And as that well-known expert in FDA policy, William Shakespeare said, “There are more things in heaven and earth, Horatio, than are dreamt of in your philosophy.”
Well, neither the Bard nor Horatio could make it to “PDUFA Without the Politics,” CMPI’s Capitol Hill seminar on the fifth iteration of the original UFA, but many hornblowers and FDA cognoscenti did. I was the moderator and was honored to be joined by a panel of peerless nonpareils that included Vincent J. Ventimiglia, Jr (Former Assistant Secretary for Legislation at the US Department of Health and Human Services), Paul T. Kim (Former Deputy Staff Director for health policy for Senator Edward M. Kennedy), Michele J. Orza (Former Assistant Director of the Health Care Team at the Government Accountability Office), and Tim Franson (Former Vice President, Global Regulatory Affairs, Eli Lilly & Co., President, USP Convention).
The Honorable Michael C. Burgess, M.D. (Vice-Chairman, Subcommittee on Health, U.S. House of Representatives) offered remarks as well – specifically on the issue of FDA advisory committee members and conflicts of interest.
But – see for yourself. Full videos of all the presentations can be found here.
Why PDUFA without the politics? Well – watch the presentations and I challenge you to identify any of the speakers’ politics from their astute commentary on the future of the FDA.
Which is as it should be.
A study published in the Journal of the American Medical Association (JAMA) shows that Ritalin, Adderall and other drugs widely used to treat attention disorders didn't increase the risk of serious heart problems. The findings, from an analysis involving nearly 500,000 adults, come on the heels of a separate study that reached a similar conclusion about the medications' effect in 1.2 million children and young adults.
According to an article in the Wall Street Journal, “Researchers and doctors who treat the condition -- known formally as attention deficit-hyperactivity disorder, or ADHD—said that together the reports should generally allay worries about heart risk that have stirred confusion among doctors and patients for several years.”
Fears about potential heart risk for ADHD medicines were sparked in 2005 by reports of about a dozen deaths from sudden cardiac arrest among young users of Adderall. The reports prompted the Canadian government to temporarily remove Adderall from the market, though it later lifted the suspension after it was determined that the patients who had died had heart defects or other underlying heart disease.
The FDA held advisory panels to discuss the matter in 2006 and subsequently required an update to product labeling to mention cardiac risk. The American Heart Association in 2008 urged that children being considered for ADHD drugs undergo a thorough heart exam, possibly including an electrocardiogram, before taking the medicines.
The FDA and other U.S. agencies funded both the JAMA study and an earlier one published last month by the New England Journal of Medicine. The earlier study, by the same research team, reached a similar conclusion among patients 2 to 24 years old.
The study in JAMA compared about 150,000 adult users of ADHD medications between 25 and 64 years old to nearly 300,000 nonusers. Researchers reviewed medical records from four health insurance plans to look at the number of serious cardiovascular events such as sudden cardiac death, heart attack and stroke among both groups.
Despite the findings, the FDA said Monday that patients treated with ADHD medications should continue to be monitored for changes in heart rate or blood pressure as well as for other side effects that include decreased appetite, weight loss and trouble sleeping. The agency also said the drugs "should not be used by patients with serious heart problems, or for whom an increase in blood pressure or heart rate would be problematic."
In August 2005 I had the opportunity to interview Newt Gingrich on a variety of healthcare issues. The questions and answers are as fresh as if we spoke this morning – and perhaps even more relevant considering the Speaker’s current position as one of the Republican front-runners.
Peter Pitts: Hello Mr. Speaker. Thank you for taking the time to chat with me.
Speaker Gingrich: I’m delighted to have the chance to talk with you.
Peter Pitts: Mr. Speaker, a lot of people talk about personalized medicine – but what do you think we need to do to make it a reality?
Speaker Gingrich: In the long run, it’s going to involve DNA data because there are so many things we can learn, particularly with regard to cancer. In the short run, it’s already starting to evolve. Take a look at the iHealth record product, for example, where you could keep your personal information and send it to a doctor using encrypted email. It’s an example of a baby step in the right direction. I just saw a diabetes cell phone that is being developed in Korea and has actually been submitted to the FDA. It will allow those who have diabetes to monitor their condition on a cell phone, send the data to their personal health record and to their doctor, and even schedule a set time to remind them to check their blood sugar. It sounds like a bit of magic, but it’s all coming down the road right now.
Peter Pitts: Do you think that pharmaceutical companies have the economic models to make personalized medicine a reality, and does the FDA have the tools to review them and approve them?
Speaker Gingrich: Part of the reason why we are going to see more personalized, narrowly focused drugs by both pharmaceuticals, and even more important, biotechs, is that we are now entering a period where drug companies will be able to reach you in a targeted way. When a drug company rolls out a drug to certain people, it has to go to every doctor’s office in the hope that it will find the 12 doctors that actually need the specific drug. But if it can actually target electronically – for example, because we actually have electronic records now, we could contact directly the 30,000 people who are on Vioxx the morning there was a problem. That’s the kind of step towards the model that I am describing, where you would know that there are x number of thousand people who have Lou Gehrig’s disease, and you can reach them instantly without an ad campaign. You could also reach the doctors instantly and therefore, you would actually dramatically increase the net value of certain drugs because you would so dramatically lower both the loss time and the distribution time.
Peter Pitts: What would be the broad implications for that in terms of changing the overall health care model – to evolve from an acute care model to more of a chronic care model?
Speaker Gingrich: Well there is a difference, let me draw a distinction here. In acute care, you’re going to get more rapidly focused, narrow delivery of precisely what you need for your acute problem. A chronic condition will require lifetime management, which in some cases could mean diet, exercise, and attitude. In other cases, it will be the application of appropriate medications. It will be less profitable per unit, but there will be massively more units. Businesses will either use traditional acute care or orphan drugs.
Peter Pitts: How do you get physicians and patients in the mindset that they can be cared for individually and precisely, as opposed to waiting until something bad happens, then go for treatment?
Speaker Gingrich: Well part of it is an incentivization program. I’ll give you two examples. I was just at Des Moines University in Des Moines, Iowa, where they actually gave bonuses to faculty and students for being compliant on exercise, blood pressure, and the management of diabetes. It’s a terrific program. And they are beginning to have real impact because people are focused on their own behavior. I also know that in the Mercy Health system in St. Louis, 76% of the blue-collar workers in a factory signed up for a personalized contract and an incentive plan – 93% of their diabetics are compliant. So those are examples of focused incentives.
We are working in Georgia with 14 corporations and state government to begin to put together a Bridges to Excellence diabetes program that pays the doctors $100 in advance for managing diabetes. With the patients in Cincinnati and Mobile, they’ve been saving about $150 a patient for a net of $250 per patient per year by incentivizing the doctor. We believe that if you combine that with incentivizing the patient, you’ll get an even better response. And with this new diabetic phone, you are going to get dramatic improvements in compliance.
Peter Pitts: That’s an interesting point – the convergence of technology on the one side, and drugs and biologics on the other. On the technology front, what role will medical devices or medical technology inside the body play in the future?
Speaker Gingrich: I believe almost certainly, in the next decade, you’ll have the equivalent of a pacemaker/cell phone. Your pacemaker will call 9/11 before you know anything is happening. It will also send routine monitoring data. I’m sure you’ve seen the shirts people wear that automatically keep track of vitals. What will happen at one level is that all of this will become ubiquitous invisibly. You won’t notice, but it will be constant. It will involve medicine with a wireless capability and a massive bandwidth, matched up with expert systems that analyze data. Some of this is happening now.
A facility in Oregon now puts a GPS locator on a band on Alzheimer patients’ arms. They can walk all over the area without having to worry about getting lost. They have wired their beds in such a way that the staff knows when they are laying down and sleeping soundly. If they have a walker, the walker is wired. If they can go to bed and in the middle of the night, go to the bathroom and later their walker doesn’t move, there is a presumption that they fell down and within two minutes the nurse comes to check. It’s a very fascinating place and I strongly recommend that you look at it. It’s an example of some of the things that you are asking about.
Peter Pitts: That’s definitely going to avoid a lot of problems and a lot of complications down the road. What can the FDA, the National Health Institute, or the Centers for Disease Control, or government in general do to help?
Speaker Gingrich: Well, you have to remember that government is the largest payer. It’s up to the government to make purchases intelligently, and to incentivize. It’s up to the government to help incentivize patients with Type II diabetes. It’s up to the government to transform Medicaid, and to have the discipline of change. That’s part one. For part two, it’s up to the FDA to accelerate the introduction of entrepreneurial new products that are not life threatening. The diabetes phone may be one of these examples. You want to have very rapid flow of approval for things that are relatively easy to do.
Peter Pitts: And for those areas where approval doesn’t exist, how can the FDA reform itself to make that happen?
Speaker Gingrich: Part of it is changing minds and to say, if we don’t believe that there is something significantly dangerous about this, the bias should be in favor of approving it. When you get to a very sophisticated electronic health record system with expert analysis, you’ll be able to approve things more rapidly because if something goes wrong, you can recall them more decisively. So that increases the level of risk you can take.
Peter Pitts: The pharmaceutical industry is in such a hole right now in terms of their reputation, what can it do to dig itself out of the hole to become a more positive player?
Speaker Gingrich: I think it should be clear to the pharmaceutical industry that they should focus on reaching and linking to an electronic health system, rather than producing massive commercials. If you look at the amount of money they spent on commercials in the last two years, you could probably provide an electronic health record for every person in the country. I think the more people see the pharmaceutical industry genuinely worried about people’s health, the better off we are. Second, I think the more we can move to a Travelocity model of selling drugs, where you have full information, know the strengths and weaknesses of each drug, what the side effects are, and the pricing, you are more in control. I think that would dramatically improve the image of the drug industry and give people a sense again that they are in charge as customers.
Peter Pitts: That’s an interesting concept, to have the pharmaceutical industry adopt and fund electronic records for all Americans.
Speaker Gingrich: If you look at what Pfizer is doing in Woodbury, Connecticut, they are in fact sponsoring information technology health care in that town. I think if the drug companies are focusing on reaching the customer in an informed way, designed to maximize health rather than profit, people would, in fact, understand that and be responsive to it.
Peter Pitts: What’s the big idea to reform health care in this country?
Speaker Gingrich: Well, I don’t know that it’s a single big idea. We propose a Twenty- first Century Intelligent Health Care System. We propose three very large changes. First, moving away from acute care to focusing on wellness, prevention, and early detection. Second, from focusing on the provider, to focusing on the individual, so we get you involved in your health before you ever become ill. Third, that we take advantage of information technology, so that we maximize productivity, accuracy, speed – what people see everyday in other aspects of lives. We think those changes together really would make a dramatic difference in both saving lives and saving money.
Peter Pitts: How do you get Americans involved in their health care when they are not ill?
Speaker Gingrich: By moving to Health Savings Accounts and Health Reimbursement Accounts – when you start getting bills, you start getting directly involved. Second, if we should have a right to know pricing and quality legislation -- 93 percent of the country likes the idea of knowing the price and quality before making decisions. I think the country is ready for change, but the politicians aren’t. And I think 80 percent of the country favors electronic private health insurance for example.
Peter Pitts: What does your crystal ball tell you about legislation and price controls?
Speaker Gingrich: I think price controls are such a stunningly dumb idea. It has failed totally almost everywhere in the world. It has ruined the research components of pharmaceuticals in Canada. The danger is that if we don’t move to a Travelocity model of individual control of purchasing, somewhere down the road, the government will start setting prices for what it pays for health care just because the prices are so staggering. For four thousand years of recorded history, when politicians have to choose between their own survival and your property, they choose your property and their own survival.
Peter Pitts: That’s interesting, so basically replacing the debate over price with a practical movement forward in personal involvement. Thank you.
During the lunch, which was attended by a 25 high ranking Geneva diplomats and officials, Fredrik set out the case that such a new trade compact would invigorate trade negotiations, while helping countries drive down costs.
Fredrik argued that the Doha Round is dead and it looks unlikely that the WTO will ever again prove to be a useful forum from which to negotiate global trade liberalisation. As mercantilism is still a dominant philosophy throughout the world, trade is unlikely to be liberalised further unless it is clear there will be clear gains in trade volumes. Trade negotiations need therefore to become less ambitious and focus on specific, clear issues.
Meanwhile, both developed and emerging economies are facing huge fiscal pressures as their populations age. Healthcare already comprises 20 per cent of the service sector of most higher and upper-middle income countries, with costs rising all the time due to factors such as technology, labour costs and so on. In other sectors, these inflationary pressures have been tempered via international trade, as countries are able to trade across borders to exploit their comparative advantage and lower costs. However, almost no trade exists in healthcare outside of pharmaceuticals and other medical goods.
At the same time, emerging economies such as China are developing new healthcare systems. As they are starting from a very low base, it requires enormous amounts of capital. They too would benefit from international trade in healthcare in order to attract investment and capital from overseas.
All these factors underline the need for a new trade compact in healthcare. Richer countries would derive great advantage from utilising communications technologies to have diagnostics and other analysis performed in countries where such things can be done more cheaply. There is also great potential to use such technologies to keep patients at home rather than in capital-intensive hospitals. Emerging markets could tap into this demand to help them mobilise the capitals and skills necessary to help them develop modern healthcare systems.
So what can be done? First, there needs to be a thorough reworking of the rules surrounding trade in services. Only a few (mainly developing) countries have made firm commitments under the WTO's General Agreement on Trade in Services (which governs the rules on trade in healthcare). Rich countries therefore need to put healthcare services at the front of their trade strategies. Second, healthcare needs to be included in the WTO’s Government Procurement Agreement to ensure that state healthcare systems are part of this international process. Third, work needs to be done to reduce non tariff barriers to trade in healthcare, such as technological and regulatory barriers.
Although medical tourism will become increasingly popular amongst richer people it is unlikely to become a mass market phenomena as it is paid for mainly out of pocket. Rather, using technology to allow patients and their physicians to communicate with low cost, foreign service providers -without having to go to a hospital- could be a potential game changer, driving down costs and improving the quality of care. To make this happen, there needs to be a new global compact for trade in healthcare.
Did The Obama Administration Throw The FDA Under The Bus?
Susan Wood quit her
job as an assistant commissioner for woman’s health at the Food and Drug
Administration in 2005 because she thought political, not scientific, forces
were delaying efforts to make the Plan B emergency contraceptive available
without a prescription. Four years later, she rejoiced when she was present to
watch President Barack Obama sign a statement that said decisions
in his administration would be based on science, not politics.
Now she says she is
beyond disappointed. Obama’s Secretary of Health
and Human Services, a member of his cabinet, Wednesday made a new decision to
prevent a further expansion of Plan B’s over-the-counter use, this time to
adolescents, overruling not only FDA staffers but also the drug agency’s
commissioner, Margaret Hamburg. Although the HHS secretary,
Kathleen Sebelius, was within her legal authority under the 1938 law that
created the FDA, this is the first time a presidential administration has ever
publicly overruled the FDA in this manner.
“This is contrary to
the scientific integrity memo signed by Obama,” Wood says. “It’s contrary to
the whole principal of making decisions based on science and evidence.”
Wood says the
decision sets “a terrible precedent for the whole breadth and depth of the FDA”
and that the “medical and scientific expertise to make such decisions and
recommendations truly resides in the FDA,” not elsewhere in Sebelius’
department. The only silver lining, Wood says, is that by publicly standing up
to Sebelius with a letter of protest posted on the FDA’s web site, Hamburg may
have prevented the decision from demoralizing rank-and-file FDA staffers.
President Obama
asserted yesterday that he “did not get involved in he process” but backed
Sebelius’ decision. Whatever the merits of the plan B decision, using Sebelius’
power in this way may tarnish what has been one of the Obama administration’s
great, pro-business achievements: Hamburg’s successful transformation of an
embattled FDA, scarred and damaged by the controversies over big pharmaceutical
industry scandals like Vioxx and Avandia, into an agency that is approving new
medicines and a near-record clip and clearly and efficiently deals with new
controversies as they crop up.
The question is
whether this decision is a one-off, or a change in the way medicines are
regulated. Could future negotiations with drug companies be weakened by the
fear that the FDA commissioner would be overruled? And even if Sebelius is
always going to back up Hamburg on every other regulatory decision, what about
future administrations?
David A. Kessler, who
headed the FDA between 1990 and 1997, praises the Sebelius and Hamburg for
being transparent by airing their disagreement in public. And he says that for
many decisions like Plan B, even if they are based on science, reasonable
people can disagree. “There’s no scientific textbook where you can look up the
answer,” he says. “You can respect people coming out on different sides.” His
worry is not about whether 11-year-olds should need a prescription for Plan B,
but about what happens in the future if decisions traditionally made by FDA
scientists become cabinet-level political issues.
“My concern is just
that it’s lousy precedent,” says Kessler. “The last thing you’d want is to have
this be seen as precedent so any new drug applicant sees the decision-maker as
the secretary. That’s not the way it’s ever worked or should work.”
Others put their
opinions even more bluntly. “The decision on Plan B by the secretary represents
an unprecedented level of interference in the FDA process and puts the country
on a slippery slope toward politicization of science,” says Steven Nissen, a
physician who has been critical of the FDA in the past. “Future political
leaders may choose to overrule the FDA on much more critical decisions.” Nissen
said he was not speaking for his employer, the Cleveland
Clinic, where he is chairman of cardiology.
Peter Pitts, a former
FDA associate commissioner who now works at the Center for Medicine in the
Public Interest, a right-leaning think tank that receives drug industry
funding, echoed those views, calling the decision “outrageous.” Pitts says: “It
opens up the hornet’s nest of companies lobbying HHS, of members of Congress
lobbying HHS. It’s a Pandora’s box of a decision.”
Not everyone agrees
Sebelius’ move will have such far-reaching effects. For one thing, political
pressures have in the past been brought more privately on the FDA – it’s not
clear that the public argument between Hamburg and Sebelius really changes
things. Ramsey Baghdadi, a founder at Prevision Policy,
a consultancy, and an editor at the RPM Report, says Plan B is “a unique
situation that demonstrates how FDA has to grapple with the intersection of
science, politics and policy all at once.”
Baghdadi says it is
possible that the Plan B controversy could lead Hamburg to exit the FDA. He
calls this “highly speculative,” but notes that Hamburg was appointed when
former Senator Tom Daschle was expected to head HHS; she was not a Sebelius
pick.
Plan B is thought to
generate only about $200 million for its maker, the generic drug giant Teva,
and the new regulation was seen as only a minor issue in terms of public
health. But the emergency contraceptive, which reduces the chance of pregnancy
after unprotected sex by half, is one of a series of issues that have made the
FDA into a political football over the past decade, often at times when the
agency was without leadership.
The controversy over
Merck’s painkiller Vioxx, which increased patients’ risk of heart attacks,
resulted in congressional hearings that, in turn, resulted in tougher new drug
approvals and voluntary industry limits on drug promotion. Safety issues and
badly conducted clinical trials with the antibiotic Ketek helped make
antibiotic approvals so tough that drug companies seemed to abandon the field
en masse. The backlash over the diabetes drug Avandia led to new hurdles for
heart safety for medicines to lower blood sugar. The drama has hurt the drug
industry.
Hamburg’s leadership
has more or less turned this around. She handled the controversial decision
over whether to withdraw Roche’s approval to market Avastin for breast cancer
with aplomb, backing her scientists that the data did not support the drug’s
use. Although bad for Roche, many in the drug industry saw this as a positive
step, because there were fears that not revoking Avastin’s approval would have
made it harder for experimental drugs to be approved in the future.
In the fiscal year
that ended Sept. 30, the FDA approved 35 new medicines, the second-highest
count this decade. New initiatives appear to be making it easier for companies
to get clear guidance as to what they need to get new drugs approved. For the
first team in years, the FDA seems anything but rudderless – a change that is
great for the pharmaceutical industry, which depends on strong, clear, and
consistent regulation. The Plan B decision may have set back that turnaround,
and, if saner heads don’t prevail in rebuilding the wall around the FDA, it
could do long-term damage to medicine in the years to come.
The morning after Secretary Sebelius' unprecedented overruling of the FDA's decision one thing is clear -- the door is now wide open to anyone who has a beef over any decision rendered by Peggy & Co.
The line forms to the left.
When asked directly if the White House had weighed in on this matter, the HHS press office refused to comment. In other words, yes. Can you imagine the hue and cry if this had taken place during the previous administration?
Having served as Associate Commissioner at the FDA during the first round of Plan B hysteria, I can personally attest to the heat and scrutiny it generated. And appropriately so. The significant difference is that debate was internal to the agency. There were differences of opinion -- but the decisions (whether you agree with them or not) were FDA decisions.
Leaving aside the peculiar politics of reproductive health, this action by the Secrerary must be reversed. Left standing it will severely undermine the authority of the FDA and embolden those who think that political arm twisting should be used to influence agency decisions.
Katy -- bar that door!
The FDA would have a hand in writing regulations and guidances for accelerated approval. But it seems to me that the agency should not be the one to determine clinical benefit especially when the scientific community and patients can do that. It should just verify that products are doing what they were designed to do. The pushback on progressive approval suggests that legislative action may be required to truly bring the FDA into the 21st century.
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.
