An UFA by any other name

12/14/2011 07:45 PM |

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.
 

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Ever metaanalysis you didn't like?

12/13/2011 09:40 AM |

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."

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Me and Newt and 21st Century Healthcare

12/12/2011 07:57 AM |

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.

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Trade and health: a new agenda for the WTO

12/09/2011 02:28 PM |

At CMPI's second policy round table lunch in Geneva, we were lucky enough to have Fredrik Erixon, the head of Brussels trade think tank ECIPE, as speaker. He spoke on the need for a new international trade compact focused exclusively on health.
 
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.

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The Wheels on the Bus go 'Round and 'Round

12/09/2011 01:20 PM |

Solid reporting from Matt Herper over at forbes.com:

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.

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Making Pharma More Productive

12/09/2011 09:57 AM |

This essay on how to improve R&D productivity rightly focuses on the need to be more targeted and value driven. But it begs the question of what to do about an FDA unwilling to adjust regulatory requirements that reward value driven development? http://www.oliverwyman.com/media/OW_EN_HLS_Publ_2011_Beyond_the_Shadow_of_a_Drought(2).pdf  Read More & Comment...

Katy -- Bar that door!

12/08/2011 01:29 PM |

Whether or not you feel that Plan B (aka -- "The Morning After Pill") should be available OTC to adolescents, the bigger issue (MUCH bigger) is the dangerous precedent of politicizing the FDA process.
 
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!  Read More & Comment...

FDA Slowing Progress on Progressive Approval

12/07/2011 12:30 PM |

The "word" is that FDA wants to  delay progressive approval by defining clinical benefit on a case by case basis and by front loading Phase I with all sorts of endpoints and metrics that it would be tantamount to going through all the hoops.   The point of the Critical Path initiative was to increase certainty and reduce the need for extensive human experimentation.  Instead, patients and doctors would tell innovators and the FDA how treatments are working in the real world.

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.  Read More & Comment...

Social Media, Pharma, and Cats in Sweaters

12/07/2011 07:59 AM |

Yesterday I was pleased to chair Day 1 of the Social Media for Pharma event in New York City.

First off, this and similar events can no longer (and should no longer) be identified as “pharma” events. Witnessing today’s attendance by and active participation of members of the medical technology (née “device”) community along with a full table of dietary supplement marketers, makes it pretty clear this and similar symposia are healthcare events. 

And that’s a good thing.

A few memorable moments:

Greg Kueterman and Amy O’Connor of Eli Lilly & Company gave a splendid presentation on their health policy blog Lillypad (www.lillypad.lilly.com).  One of their most important points (not rocket science, but rarely discussed) is that, when it comes to healthcare in the social media space – it can be about things other than product marketing and that metrics are more than about volume.  It’s not about the top ten, but rather the right ten. Amen.

Greg asserted that he's “the Justin Bieber of healthcare social media.”  Not sure what that means, but it does seem to be an off-label claim.

William Martino (Saatchi & Saatchi Wellness) and Shermon McMillan (Chattem) – henceforth forever known as Shermon/William – painted an interesting portrait of social media and the OTC launch (and subsequent marketing) of Allegra. 

Ned Russell (Saatchi & Saatchi Wellness) pointed out that the oft-quoted FaceBook development maxim of “move fast and break things,” couldn’t be more antithetical to regulated healthcare communicators (note – not marketers, but communicators).  My comment was that this might explain the tone deafness of the world’s largest social media platform has to issues such as moderated user-generated commentaries.  (Also note that the FCC didn’t find FaceBook’s swiftness to cast aside privacy issues so kosher either.)

And, while we’re on the subject, why does FaceBook allow “drugs from Canada” pages.  Did they not get the memo from Google and the DOJ?

Tai Spargo of NORD discussed their new social media efforts in creating a plentitude of orphan disease sites minus any participation of the biopharmaceutical industry.  She also noted (and rightfully so) that there is no rare disease too small for social media.  To illustrate her point she displayed the FaceBook page “Cats in Sweaters.” Meow.

Check it out if you are so inclined at http://www.facebook.com/pages/Cats-in-sweaters/183921988289175. Idle hands indeed do the devil’s work.

Sharon Landa (Johnson & Johnson) and Erica Wittes Schlack (Communispace) discussed the design and utility of private (read, “closed”) social media communities.  Fascinating.  Two comments to pass along, “Feedback is about the brand, but discovery is about the consumer” (utility) and “Diversity is for conversation but similarity is for cohesion (design).

There was also much chatter about how to make social media programs work along side of “traditional communications initiatives.”

Rather than calling them “traditional,” perhaps we should label them as “legacy” programs.

And we all know what happens to those.

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AHIP on HSAs

12/06/2011 05:56 PM |

Politico notes that AHIP’s new report on health-savings accounts contain some interesting findings:
 
America's Health Insurance Plans is out with a new report on the state of health savings accounts. Among the findings: Lower-income individuals open HSAs at a greater rate than do higher income groups - with an average income in the mid-$50,000 range - and the amount of money that individuals have accumulated in their accounts has grown over time, and the amount of money being rolled over from one year to the next also has increased.
 
Click here to read the report.  Read More & Comment...

Just in Time for the Holidays: FDA Approves Hangover Drug

12/05/2011 02:16 PM |

FDA Approved Hangover Medication Hits Market.

The New York Daily News (12/3, Pesce) reported, "A new FDA-approved hangover cure has hit the market." The over-the-counter medication, dubbed Blowfish, "combines 1,000 milligrams of aspirin, 120 milligrams of caffeine and a stomach-soothing agent into two effervescent tablets taken the morning after a night of heavy drinking." Blowfish "costs $2.99 for a single dose, or $11.99 for a six-pack" and "is currently available in Ricky's NYC stores or online at ForHangovers.com."

        The website of WCBS-TV New York (12/5) reports, "Just in time for the holidays, the FDA approves a purported hangover remedy. Blowfish, created in the West Village, is promised to get rid of all your hangover symptoms within 15 to 30 minutes." FOX News Radio (12/4, Rappoport) added that that the drug "combines aspirin, caffeine and a stomach soother into two effervescent pills to be taken the morning after a night of partying." The remedy's creator Brenna Haysom "says the pill is only intended for people who had one too many, not binge drinkers."


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PDUFA: Process or Provision?

12/05/2011 07:15 AM |

At CMPI’s “PDUFA without the Politics” Capital Hill Briefing, Dr. Tim Franson (President, USP Convention, former Vice President, Global Regulatory Affairs, Eli Lilly & Co., and one of the “father’s of PDUFA”) mentioned the Hawthorne Effect – “that which is measured gets accomplished.”

Which begs the question – should more things in PDUFA V be measured or measured with more robust deliverables?

Are “meetings” sufficient promises – or are actions more desirable?  Specifically – are meetings about a benefit/risk grid enough or should there be a date-certain for a regulatory instrument to be operational?

Should PDUFA V be exclusively about process? What about provision?

Speech is conveniently located midway between thought and action, where it often substitutes for both.  – John Andrew Holmes

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PDUFA -- All the rest is commentary

12/02/2011 07:45 AM |

Always listen to the experts. They’ll tell you what can’t be done and why. Then they do it.  – Robert Heinlein

On Tuesday the Center for Medicine in the Public Interest (www.cmpi.org ) hosted a Capitol Hill panel entitled, “PDUFA without the Politics.” 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.

The participants were pithy, poetic, and pragmatic.

We will shortly post videos of all the aforementioned presenters.  In the meantime, here’s a short news story from Drug Information Daily:

Lawmakers Plan to Use PDUFA V Bill to Ease Conflict Rules for Advisory

Panels

Lawmakers plan to attach a bill easing financial conflict rules for FDA advisory panel members to legislation reauthorizing the Prescription Drug User Fee Act (PDUFA), a congressional leader says.

Rep. Michael Burgess (R-Texas) introduced H.R. 3206 last month and said Tuesday it would be attached to the measure authorizing PDUFA V, yet to be introduced to Congress. H.R. 3206 would eliminate the existing financial conflict rules for panelists, opening the door for committee members with greater expertise on the products they discuss.

Burgess, vice chair of the House Energy and Commerce Health Subcommittee, offered similar legislation during consideration of PDUFA IV in 2007, but it was voted down along party lines. With his Republican party now in control of the House, he may have an easier time gaining approval of the bill.

Speaking at a PDUFA V briefing for Capitol Hill staffers hosted by the Center for Medicine in the Public Interest (CMPI), Burgess said there are about 10 strong candidates for legislation to tack on to PDUFA V. One possibility is a bill addressing the issue of drug shortages, he said.

Another likely candidate is a bill to increase incentives for antibiotic makers, according to Foley Hoag lobbyist Paul Kim.

Proactive Lawmakers

Also at the briefing, Peter Pitts, former FDA associate commissioner and president of CMPI, urged Congress to be more proactive in changing the technical letter the FDA and drugmakers agreed to during PDUFA negotiations earlier this year. Specifically, Pitts called for more programs and agency action to address transparency and striking a better risk-benefit balance in product reviews.

Following these recommendations might mean altering the package the FDA and industry ratified, which has only happened once in the previous four PDUFA bills.

Recently, David Wheadon, PhRMA's senior vice president for scientific and regulatory affairs, said the package includes a "robust set of programs" to provide an objective measure of risk-benefit analysis at the agency.

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Homeless and Living in Cars

12/01/2011 11:46 AM |

I found it very hard to watch this 60 Minutes piece on kids whose parents were forced by extended joblessness and sudden, sustained cuts in pay to live out of their cars.   Having been unemployed and down to my last dollar many years ago (with the added burden of finding out a month before my son was born that my previous employer had not paid my health insurance premiums and was not going to give me my last paycheck) the segment not only resonated and brought back some sad and unpleasant memories, it reminded me that for millions of people the difference between a decent life and having to depend on government is very slim indeed.  A robust economy will go far to restore opportunity.  In the meantime, we all must do what we can to stop more people from winding up in the same situation share by those interviewed by 60 Minutes.

http://www.cbsnews.com/8301-18560_162-57330802/hard-times-generation-families-living-in-cars/


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Event Horizon

11/30/2011 10:14 PM |

BioCentury Reports:

EC proposes new research program

The European Commission proposed a new EU research and innovation program for 2014-20 called Horizon 2020, which would have a budget of EUR 80 billion ($107 billion). The proposal includes allocations of EUR 24.6 billion ($32.8 billion) for science research in Europe, including an increase in funding of 77% to EUR 13.2 billion ($17.6 billion) for the European Research Council (ERC); EUR $13.7 billion ($18.2 billion) for industrial technology development and small and medium-sized enterprises; and EUR 31.8 billion ($42.4 billion) to address societal concerns in areas such as climate change, energy and resource scarcity, and health and aging.

The EC said Horizon 2020 will have a simpler set of rules and procedures for obtaining funding than those of previous research programs, with the goal of reducing time to funding following a grant application by about 100 days. The European Parliament and EU Council will now review the proposal, with the goal of adopting it by year end 2013. The current research program, Framework Programme 7 (FP7), has a budget of EUR 54 billion and runs from 2007-13.   Read More & Comment...

The Biomedical Century?

11/30/2011 04:37 PM |

Joseph Rago recently sat down for an insightful interview with John Lechleiter, the CEO and chairman of Eli Lilly & Co.

Here are a few key excerpts:

But above all, Mr. Lechleiter explains, "There's no better investment that we can make than in biomedical research and in our health. This is not something that we're trying to steal away from someone else. This is not a nascent industry." Pounding his desk on each word—"America leads the world, okay?"

"I believe this will be the biomedical century," he continues. "We'll look back a hundred years from now and say the 20th century was the century of chemistry and physics, and the 21st century was the century of biomedicine."

Mr. Lechleiter adds that "The challenge or the opportunity we have is that never before has the science and our knowledge base been riper for exploitation." For most of the pharmaceutical industry's existence—since Civil War veteran Col. Eli Lilly began to improve on the patent medicines of the day—"it was akin to feeling your way around a dark room and trying to make sense of what's what. Suddenly the lights are on and we can see, aha: In a cell, this pathway and that pathway both contribute to, say, tumor formation."

Not only is there an ongoing revolution in genomics and systems biology, Mr. Lechleiter continues, we increasingly have the tools to make use of this basic research and commercialize it. "A process that used to take years and years and rely too much on serendipity and conjecture can now be accomplished in a period of time that looks closer to months and months." Researchers are more "mission driven and deliberate" and, with a biological target, can "come up with a viable clinical candidate, something that we could hope to take into human testing" faster and with more confidence than ever before.

Read the full piece here.

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CMPI health conference in Beijing – reform of the General Practitioner system

11/28/2011 12:24 AM |

While health reform has become a focus for the Chinese government over the past three years, there is still no national system of primary care, in particular general practice.  Reform in this area is currently a hot topic of discussion, as without proper primary care it is impossible to manage access to specialist care, as well as the whole range of preventative and consultative services that keeps people healthy and out of hospital.
 
To help shape thinking in this area, Beijing think tank the Cathay Institute for Public Affairs invited me to discuss with officials and experts how the system works in Britain at a symposium and workshop in Beijing, with additional comment from local policymakers and experts on general practice reform.
 
Britain presents a case study of how not to organise a health system. Since britain nationalised healthcare in 1947, subsequent governments have struggled to improve productivity, contain costs and improve the quality of care. Up until 1991, the system was mainly managed through central targets and control, with a chain of command going right up to the minister's office in Whitehall. By the late 80s, this system was beginning to look rather tired.
 
As part of the Thatcher government reforms, competition was introduced into the health service, in the form of an internal market. Providers and purchasers were split for the first time, and GPs were given the power and budget to buy care on behalf of their patients from any NHS provider. The aim was to use their market power to drive down costs and promote innovation. While this system achieved some success in reducing patient waiting times, Tony Blair’s government on election in 1997 decreed that this created a two tier system in which some received faster treatment than others.
 
This nascent market was therefore abolished and replaced with the old command and control system based on centrally-mandated targets. But by 2004, it was clear that this system was failing to manage an increasingly complex and massive health system, so the government reintroduced a diluted form of the internal market it had abolished, wherein GPs could form voluntary groups to commission care on behalf of patients. This time the budgets were notional instead of real, and any notional savings would be reimbursed by the ministry for spending on developing innovative services.  Not surprisingly, few doctors found they had the time to engage with this new bureaucracy, so it has remained something of a sideshow to the main modus operandum of the NHS which is targets and central dictat.
 
Finally, the new government of David Cameron proposes reinvigorating the internal market by making it compulsory for GPs to commission care on behalf of their patients, and extending it to the majority of areas of care. This met with fierce opposition from opposing parliamentarians, as well as doctors unions and most of the medical profession, who fear that moves to a more market based system might undermine their not inconsiderable pay and generous employment conditions (as well as having ideological objections to the involvement of markets in healthcare). Pro reformers worry that the new bureaucratic apparatus required to manage this quasi market will work against physician autonomy, and could act as conduit for central government to reassert control - health civil servants are notorious jealous of their status as guardians of such a significant part of the body politic.
 
The key lesson for China, then, is that once they embark down a route in which central government is the primary provider and guarantor of care, it is very difficult to inject market force to tackle the problems that always arise from monopolies - interest groups, once entrenched, are very difficult to tackle. As a result of these failings, GP care in the UK is no way comparable to that in the US, as they have little incentive to develop the doctor patient relationship beyond responding to a narrow set of clinical and waiting time targets.
 
While things are bad in the UK, in China they are several orders worse. At our workshop Qin Jiangmei from the Ministry of Health outlined the major problems with the GP system, including the poor quality of doctors and service providers, and the fact that patients (especially in rural areas) normally have to pay out-of-pocket to poorly trained GPs.  And as GPs derive most their income from sellling medicine or hosptial procedures, a visit to the doctor for even a mild ailment can end up with a huge bill as a result of over-prescribing. As a result, the majority of Chinese simply avoid the healthcare system altogether.
 
Professor Gu Yuan of the General Practictioner Association of Chinese Medical Association outllined her vision of the future system, which included training 300,000 extra doctors by 2020, and creating a new system of contract between patient and doctor that would inject some accountablity into the system.  She also pointed out that General Practice is a very unpopular career choice for medical doctors, the majority of whom aim for a clinical specialism. This is hardly surprising when General Practitioners earn less than average civil servants. As a result, GPs are few and far between, particularly in rural areas, and they are often only in possession of a sketchy knowledge of medicine. In order to increase their wages to something reasonable, GPs routinely overprescribe, and make unnecessary use of expensive diagnostics (when for instance the patient may only have a cold). This is exacerbated by the fact that doctors receive a large proportion of their salary from profits they generate for the hospital, which leads to overprescribing, and not enough focus on preventative care and basic diagnostics (such as checking blood pressure).
 
Prof Wang Zhen on the Institute of Economics noted the problems inherent in expanding a system of coverage to a rapidly ageing population, in the face of rapidly changing technology and medical knowledge.  The NHS experience shows that state-monopolies are not well-placed to foster innovation either in managerial techniques or the use of new medical technologies.  China therefore needs to embrace choice and competition in its design of coverage systems if it is to avoid making the same mistakes as Britain. He noted that the government’s role should be restricted to supervision or the provision of additional services that cannot be provided by the market.
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ITP Values

11/23/2011 07:20 AM |

Excellent article in the always excellent RX Compliance Report on the Europe’s continuing debate over the dissemination of healthcare information.

European Commission proposal on patient information draws divergent views

The European Commission’s (EC) revised proposals on information to patients, released last month, drew divergent assessments across the Atlantic. The European Federation of Pharmaceutical Industries and Associations (EFPIA), which represents research-based pharma companies operating in Europe, cautiously welcomed the amendments, urging all member states to adopt a “pragmatic approach” to address what it calls “the existing inequality” of access to quality data. “The revised proposals put rights, interests and safety of patients first,” insisted John Dalli, European Commissioner for health and consumer policy.

However, former FDA Commissioner for External Relations, Peter Pitts, spoke for many on this side of the Atlantic when he said the proposals appear to slow what had been a movement on the part of the EC to liberalize the industry’s ability to provide information about prescription drugs to patients. “That effort was on the ‘slow track’ before last month’s announcement,” says Pitts, who heads the Center for Medicine in the Public Interest in New York. “Now it’s on the ‘super slow track.”

International compliance expert, Paul Woods, says he is hopeful that the revised proposal will eventually be enacted in a form that will enable citizens all across Europe to have equal access to high quality non-promotional information about their prescription medicines. “The outcome, however, remains uncertain,” he says.

The complete RX Compliance Report article can be found here.

And a happy Thanksgiving to all.

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This One Goes to 11

11/22/2011 08:22 AM |

By now it should be clear to regulated healthcare communicators that delaying robust entry into the world of social media due to lack of FDA guidance is an empty excuse. And, as more and more people turn to social media as their first and primary portal for healthcare information, absence from the playing field isn’t only a bad business decision – it’s irresponsible.

Regulated healthcare industry must participate in social media –not because of its potency as a marketing vehicle – but because it’s the right thing to do.  That being said, here are 11 principles that must serve as the basic substrate of regulated social media participation.  (Why eleven?  Because, in the immortal words of Spinal Tap’s lead guitarist, Nigel Tufnel, “It’s one louder.”)

1.     We engage in social media to help improve the lives of patients and advance the public health of our nation.

2.     We will thoughtfully engage in social media while remaining in compliance with both the letter and the spirit of FDA regulations.

3.     Our social media engagements will have both strong public health themes and appropriate marketing communications.

4.     All social media messages and partnerships must be accurate, appropriate and transparent.

5.     We believe that social media presents multiple opportunities to learn more about how our products impact the lives of patients.

6.     We believe that social media engagement allows us to correct errors and misperceptions about both our company and our products.

7.     We believe in using social media discover adverse drug experiences, which will then be addressed off-line.

8.     We will strive to interact in a timely manner, appropriate to the general expectations of social media.

9.     We believe that social media must be regularly monitored and our programs measured in real time to gauge effectiveness.

10.  We respect but are not responsible for user-generated content that resides on sites we do not control.

11.  We believe the path to engagement is through useful and thoughtful content and commentary.

One principle that runs as a red thread throughout all of these 11 principles is transparency.  Real, honest transparency – not the usual translucency that “in compliance” often brings.

It’s time for action.  As Friedrich Engels said, “An ounce of action is worth a ton of theory.

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With Supporters Like Those Accelerated Approval Doesn't Need Enemies

11/21/2011 08:45 PM |

There has been a lot of coverage about the Food and Drug Administration's decision to revoke approval for the use of Avastin in metastatic breast cancer.  Some commentators – who have at most a third hand understanding of the FDA’s handling of Avastin and how it squares or doesn’t with other cancer drugs granted accelerated approval – have lauded the withdrawal as good for innovation.  Most of it takes at face value FDA Commissioner's Hamburg 69 page decision in which she claims that the 'objective' evidence of benefit did not exist to support approval relative to the serious side effects of the drug. 

In particular, people who should know better let the FDA's spin affect their judgment.  For example, Avik Roy who writes for Forbes and is a fellow at The Manhattan Institute who in turn salutes another MI fellow for his support of the Avastin withdrawal:


"Paul Howard is the guy who gets it right:

        If you think (as I do) that the FDA should be expanding the accelerated approval pathway and allow more drugs to get to market based on promising         early studies. rather than waiting for large Phase III clinical trials that can take years to complete, you can argue that this outcome actually strengthens AA. Critics have charged that AA is sop to industry, and that companies never do the follow up studies to support AA. Avastin proves them wrong.

This is exactly the point. If you want the FDA to approve more innovative, new drugs based on promising but early clinical results, you have to give the FDA a way to revoke those approvals later on, should larger trials prove that those drugs aren't as safe or effective as they first seemed. This is why the FDA should be congratulated for the way it has handled the Avastin breast cancer saga, and why I hope we will see the FDA handle more cases like this one, not less."


http://www.medicalprogresstoday.com/2011/11/fda-did-the-right-thing-in-pulling-avastin-for-breast-cancer.php

Both of them have no clue.  

In fact the decision is less about Avastin and says more about the deep resistance at the Office of Oncology Drug Products to accelerated approval.  And those who support the reversal as some victory for medical innovation and accelerated approval do not understand either the scientific challenges of establishing clinical benefit in first line MBC care and, given how hard it is to demonstrate any clinical benefit in end stage patients, how significant the addition of Avastin to current therapies really is.   I am disappointed that observers treat the decision with the same sort of seat of the pants analysis reserved for Yankee post-game shows.

Dr. Hamburg's sympathy and support clearly lies with patients and the accelerated approval process.  However her explanation of the reason for the withdrawal of the approval reflects a combination of evasiveness and duplicity on the part of FDA staff in the Office of Cancer Drugs that has dogged the accelerated approval of Avastin from the start. 

Dr. Hamburg seeks to refute the claim that the FDA never switched the outcome measure of additional studies Genentech conducted to support ongoing approval of Avastin for MBC.   She claims (and I will guess this was written for her) that progression free survival (how long people lived without tumors growing) was always the standard and that the OODP did not demand to see an increase in overall survival  (how long people with and without Avastin treated before they died of any cause) which is a harder goal to meet.  In fact, her decision memo shows that the FDA never came straight out to say PFS of any given length would be the basis for approval.  Rather, FDA used the words "probably" or "might" and talks about the "potential" of using  PFS for approval as long as the "magnitude" of the benefit was significant given the risks of the drug.  Did FDA ever define what "magnitude" was sufficient?  Never.  Rather, as Dr. Padzur who runs OODP, told Genentech he would determine what the right "magnitude" would be when he saw the results. 

In otherwords FDA said that improvement in PFS is not adequate unless a large enough magnitude that it will decide after the fact and by the way if it doesn’t think it is big enough then a cancer drug must show a statistically significant improvement in OS to support approval.   And Dr. Hamburg calls this objective evidence? 

Additionally, Dr. Hamburg claims that the magnitude of clinical benefit has nothing to do with achieving a statistically significant benefit.  (Those words might and should be used by companies going forward as they design clinical trials.)  All well and good.  Yet the FDA ultimately revoked approval because Avastin did not show a statistically significant increase in overall median survival.   So does statistical significance  matter or not?  Apparently it does when you want to undercut approval for use of a product you opposed at the outset.  Similarly, when Dr. Hamburg claims that Genentech could never identify a subset of patients that might be more likely to benefit from Avastin in MBC she fails to point out that the FDA never asked for such data, nor did it state that it would extend approval based on response in a small group of patients.  In any event, the FDA decided that post-hoc analysis to identify super-response of a statistically significant amount is not evidence of magnitude.  

Her opinion ignores the fact that several other first line treatments for MBC were approved using PFS as an endpoint with half as much clinical benefit.   Let me put in terms that supporters of the decision can understand and let me put it in bold:  Specifically in the first-line treatment of MBC setting, two other drugs have received full approval using progression free survival or time to progression (TTP) effects of a much low magnitude than was used to grant Avastin accelerated approval.  Several second and third line drugs also were given full approval with well below the median gains in PFS of Avastin.  In all but two cases, full approval was granted without a statistical significant improvement in OS.


Dr. Hamburg offers some hints about how to show clinical benefit going forward: patient outcome data will count and designing trials to show which people might gain most from medicines will as well.  Her brief discussion about how groups that support continued use of Avastin in MBC could appear to biased because they receive unrestricted support from Genentech is a very low blow and hypocritical because the FDA is seeking to loosen its own conflict of interest rules to get more qualified experts on it's advisory committees. 

But in general Dr. Hamburg justifies the creation of higher threshold for the use of surrogate endpoints, a threshold that is arbitrary and without any empirical justification, in order to force all drugs to demonstrate OS.  All this suggests that allowing FDA to retain discretion over what is enough benefit will generate more uncertainty and ends accelerated approval as a real alternative.    The Avastin decision says less about the science of the FDA and more about how regulatory uncertainty (as a result of bias or lack of knowledge) can undermine innovation.   How can the so-called supporters of accelerated approval endorse Hamburg’s endorsement of this bait and switch?
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