March 2012 Archives


Tuesday, March 23 marked the two-year anniversary of the passage of the Patient Protection and Affordable Care Act of 2010 (PPACA) and the largest transfer of power from Congress to the Administration in the history of this nation. As the Supreme Court hears arguments about PPACA on Constitutional grounds - the individual mandate, in particular - let's not be lulled into thinking its broader assumptions and provisions are otherwise innocuous or valid. Setting aside the need to address legitimate reform, including egregious problems with pre-existing condition rules on the insurance side, there's the bigger problem of healthcare delivery and the fundamental safety of this sector of the industry.

In his March 23, 2012, "Opinionator" column for the New York Times, Dr. Ezekiel Emanuel, previously a CMS official closely tied to the White House, proudly points to the accomplishment of PPACA and suggests that even if the individual mandate is struck down, we're all better off because of PPACA. His analysis reflects an arrogance and blind faith in the power of the State.

Dr. Emanuel is very familiar with PPACA; he was frequently called upon to help craft the legislation by the Senate Finance Committee, in particular guiding language related to Accountable Care Organizations (ACOs) and Comparative Effectiveness Research (CER). He is a physician by training and his economic views, widely critiqued, emphasize making societal choices reflecting the utilitarian philosophy of John Stuart Mill... the greatest good for the greatest number. In this thinking, investments in public health benefitting society as a whole are better than investments that benefit individuals, especially late in life.

Aside from his philosophy, he applauds PPACA's Partnership for Patients "aimed at reducing hospital-acquired infections, errors and other preventable complications" for a mere $500 million. He goes on to note correctly that "the act also requires Medicare to begin posting online each hospital's rate of certain medical errors and infections, and to cut payments to hospitals with the highest rates."

As a result of the law, he points out that "hospitals across the country are working to reduce preventable hospital errors." Now that PPACA has made clear this is a priority, "significant progress can be made." He also notes that his current employer, the Hospital of the University of Pennsylvania, actually started to "pay attention to reducing preventable errors"... just a few years before PPACA was passed.

Now isn't that interesting?

Why is it that federal cash incentives are required to make it clear to healthcare delivery organizations that washing hands, giving the right medication, and operating on the correct side of the body is "a priority"? If "tens of thousands of Americans die" because of these simple-to-fix issues, just what more important priorities are distracting our hospitals? And if their priorities are so misaligned with common sense, why does it require cash payments to make the point?

When the auto industry cranks out defective cars that kill people, does the department of transportation start a multi-million dollar Partnership for Drivers program that pays manufacturers to fix the problem? Since when is it the job of the taxpayer to pay hospitals to fix the mistakes that they can't otherwise find time to pay attention to?

More than that, why was legislation needed to "authorize" taking action on this problem at all? CMS is responsible for oversight of the quality of the services provided with its money (our tax dollars) to participants, so why didn't CMS take administrative action on the issue - to stop paying for HAIs medication errors, etc., a long time ago? It's entirely within the mandate of CMS to do so. We didn't need to pass a 2700-page law to ensure hospitals would be safe. Where is the public outrage?

And finally, why is it that "tens of thousands of Americans die because of hospital-acquired infections every year, and far more are harmed by medical errors", and we haven't heard about the Congressional hearings that grill healthcare executives about why such issues haven't been a priority? Where are the multi-million dollar fines, the threats of congressional action, the public outrage?

In the 2010 Toyota brake recall, 37 people were alleged to have lost their lives, and the Congressional debate was intense. Between HHS, CMS, the Surgeon General, and Congress, why isn't someone paying attention? And don't tell me we needed 2700+ pages of legislation and a trillion dollars to fix it. We didn't and we don't.

How Dr. Emanuel and others think it is the responsibility of the taxpayer to pay for industry errors is beyond comprehension. If this was a pharmaceutical error, or medical device error, my guess is that the CEO of the offending companies would be hauled before Congress and affected families and patients would be called to testify against them - just like Congress did with Toyota.

Aha! Maybe that's it. Maybe the issue is that these hospitals have a designation of "not-for-profit." So in some people's distorted view of the world, maybe they can do no wrong, and for-profit business enterprises can do no right. If this line of thinking is out there, then no amount of regulation will make us safe.


I couldn't agree more with something Paul Howard wrote yesterday in his post on the Bennet, Hatch, and Burr "Advancing Breakthrough Therapies for Patients Act" proposal. "It's time -- long past time -- to update and expand [Accelerated Approval] for the millions of patients who could benefit from science-based reforms," says Paul.

Like the Faster Access to Specialized Treatments Act (FAST) and Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) bills recently introduced in the House and Senate respectively, the Breakthrough Act (apparently no cute acronym available) would make a "seemingly" major shakeup in the way FDA reviews and approves promising new treatments. One that, as Paul suggests, is long overdue.

According to Pharmalot, "If a drugmaker or a biotech believes its med would qualify [as a "drug intended for a serious or life-threatening disease or condition where preliminary clinical evidence indicates they may demonstrate substantial improvement over existing therapies"], the FDA could be asked to designate the med as a breakthrough. From (sic) the agency would meet with the sponsor through the development process, provide advice as development progresses, minimize the number of patients in clinical trials and shorten the duration of the trials, among other things."

In theory, those are all wonderful things that can streamline the testing process by giving developers a sense as to what endpoints the FDA believes are most important and how best to measure them, thereby helping developers better structure trial methodology to get the necessary data in the shortest time. The bill text would require the Secretary of HHS (i.e. the FDA Commissioner's boss) to take "steps to ensure that the design of the clinical trials is as efficient as practicable, when scientifically appropriate, such as by minimizing the number of patients enrolled in the trial and the duration of the trial and considering alternatives to the traditional multi-phase, sequential development approach, designed to abbreviate, consolidate, and condense clinical trials and studies."

The bill would essentially instruct FDA to be helpful and not a hindrance to new drug development. That is, it would require the agency to provide real assistance to drug firms that try to bring beneficial new treatment options to market but have difficulty knowing how to satisfy the FDA's demand for evidence of safety and efficacy. To some observers, that would seem to be a radical improvement.

One of the most frequent complaints about the FDA drug and device review process is that it is highly unpredictable, even when manufacturers meet with the agency prior to designing clinical studies. The process of developing safe and effective new medical products is difficult and uncertain in the best of circumstances. But time and time again, we hear stories about how FDA promised a developer one thing before a clinical trial but changed its mind after the results were in. A few years ago, after learning that FDA had rejected approval for one its drugs even though the expert advisory panel voted unanimously in favor of approval, Schering-Plough CEO Fred Hassan told The Wall Street Journal, "What will it take to get new drugs approved? The point is, we don't know."

So, at least in theory, the Breakthrough Act would seem to represent a huge improvement in the way things are done at the FDA -- and surely could not make things worse. For those reasons alone, I would argue that the reform proposals it includes are worth supporting. Still, I think that something far more radical is needed to shake up the agency.

In addition to the Breakthrough Act, I'd like to see the FAST or TREAT proposals or something similar be implemented as well. I'd like to see the FDA embrace adaptive trial design more fully and more swiftly. And I'd like to see the agency adopt a robust benefit/risk assessment model and formally incorporate patient-reported outcomes into the new product approval process, as the FDA has proposed in its recommendations for the current round of PDUFA reauthorization.

Fortunately, achieving all those things is not only conceivable, it's possible to do them this year. Two of the proposals are already on the table for this round of PDUFA reauthorization. The FAST/TREAT proposal is merely an extension of an existing law. And though the Breakthrough Therapies Act may seem radical to some, it really represents a fairly modest change -- only improving the way FDA goes about performing its duties, not actually changing those duties. So, by all means, let's adopt the Breakthrough Therapies proposal. But let us not stop there. There is plenty more to be done.



U.S. Senators Michael Bennet (D, CO), Orrin Hatch (R, UT), and Richard Burr (R, NC) introduced a smart, thoughtful bill yesterday (Advancing Breakthrough Therapies for Patients Act) designed to move innovative products to patients faster. The legislation is also supported by the cancer advocacy group Friends of Cancer Research and the National Venture Capital Association.

First, a little background. If enacted, this would be the first reform of the FDA's drug approval process for innovative new therapies since Accelerated Approval (AA) was enacted by the agency in 1992 and Fast Track created as part of the FDA Modernization Act in 1997.

So it's either been 15 or 20 years since the FDA or Congress has made any attempt to systematically expand pathways for new therapies.

How well has AA worked? For HIV/AIDS and cancer patients and some orphan indications, very well. The vast majority of drugs that have received AA have been for those indications because the agency has more confidence in surrogate markers for those diseases for other diseases. As a result, cancer and HIV therapies reach market years faster than if they had been required to complete full Phase III trials before marketing approval.

In cancer in particular AA has led to an explosion of investment, research, and clinical trials testing new medicines. But we can't recognize the value of AA for cancer and HIV and somehow say to patients with other serious and life-threatening diseases that "sorry, you'll have to wait at the back of the line until we sort things out for you."

It wasn't as if the FDA woke up one morning and just created AA out of thin air. It took smart and savvy AIDS protestors demanding that the FDA change how they evaluated new treatments - both through public protests and targeted (science-based) policy demands.

It's time - long past time - to update and expand AA for the millions of patients who could benefit from science-based reforms. As John Marangore, CEO of Anylam Pharmaceuticals, put it in recent Congressional testimony:

...there are several reasons why the Accelerated Approval pathway should be expanded and modernized. First, it is important that the ability to utilize an accelerated pathway is better understood by sponsors and more consistently applied by FDA. This is especially true when it comes to FDA accepting clinical endpoints, including those that can be measured earlier than irreversible morbidity or mortality, to demonstrate a reasonable likelihood of clinical benefit.

While the pathway, which was codified in 1997, allows for approval based upon effects on clinical endpoints that are reasonably likely to predict clinical benefit, in practice the lack of clarity surrounding such approval options has led to very limited use by sponsors and FDA.

Additionally, the Accelerated Approval pathway has been largely limited in practice to drugs that treat cancer and HIV/AIDS, along with a handful of other situations, leaving many other rare and serious conditions effectively excluded from the pathway and creating confusion among sponsors on how to apply the pathway to these indications.

The Breakthrough Therapies bill (Pharmalot links to a copy of the legislation here) would, in a nutshell:

Create a flexible pathway for the FDA to evaluate products to "treat serious or life threatening disease or conditions" when preliminary clinical evidence "indicates that the drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints."

Require the FDA to take action to help facilitate the development and review of breakthrough products, including holding additional meetings, offering advice, facilitating cross-disciplinary reviews, and having a project lead "facilitate an efficient review of the development program and to serve as a scientific liaison between the review team and the sponsor."

The legislation also encourages the FDA to ensure that the design of clinical trials is as efficient as possible (and scientifically appropriate). This is particularly important since clinical trial costs and development times are skyrocketing, helping to drive up drug prices and delay access to innovative therapies. (Smaller, leaner, faster ought to be the agency's motto when it comes to helping to develop breakthrough therapies.)

The legislation also sets deadlines (always helpful for focusing regulators' attention) for draft and final guidance for sponsors on how to seek breakthrough, accelerated approval, and/or fast track designation.

The most important section of the legislation may be the clause that requires the Secretary of HHS to commission an independent entity to asses the "quality, efficiency, and predictability" of how the FDA has applied the directives in the legislation no later than four years after the bill passes.

That may the best way to ensure that we won't have to wait another 15 or 20 years to understand how well the FDA is utilizing the authority granted it by Congress.

The core ideas in this legislation are, as Senator Hatch observed, "common sense" reforms. They should only help Commissioner Hamburg and senior FDA staff institute the management and scientific reforms that they've been talking about for several years now. But the track record of the FDA is that it only innovates under pressure - from a public health crisis or at explicit Congressional direction.

Hopefully, the core elements of this legislation will be incorporated into the upcoming PDUFA reauthorization. But PDUFA is also a double edged sword, because without the legislation the FDA will have to start firing reviewers and slowing down new drug and device approvals, something no one (industry, patients' groups, Congress) wants.

PDUFA is, indeed, a "must pass" bill.

But getting a timely PDUFA bill passed is no excuse for Congress to neglect its fundamental responsibility of ensuring that the FDA is reviewing innovative new therapies as quickly and effectively as possible, using the best scientific and management tools at its disposal. Losing or neglecting this opportunity may push off critical FDA reforms for another 5 years - when we'll just have to battle another tight PDUFA deadline.

And that's how 15 or 20 years pass by without anyone driving the kinds of "common sense" FDA reforms suggested by Senators Bennet, Hatch and Burr.


Manhattan Institute senior fellow Avik Roy and Columbia University's Gillian Metzger discuss the Supreme Court's hearing of the case with WSJ Legal reporter Ashby Jones.



In an earlier posting, I described how India is trying to become a free rider, gaining the benefits of drug development without having to share in the costs.

The Indian government violated the intellectual property rights of Bayer and Onyx by breaking Nexavar's patent and giving a new license to Natco Pharma, an Indian company, to manufacture and market a generic version of Nexavar in India.

Roche has been paying close attention and sees the writing on the wall. Roche just announced that it would launch rebranded versions of Herceptin and MabThera/Rituxan in India at lower prices. Roche will, of course, sell a higher volume of these products at the lower price, but it is clear that Roche primarily aims to avoid "compulsory licenses." Facing the prospect of earning nothing or something, Roche has chosen the latter.


A little-noticed event (non-event, really) over the past year may be giving us a glimpse of what really lies ahead. Not the absurd "death panels" from a few years ago, but something much more subtle. Yet it could could have pretty much the same result.

My op-ed in today's New York Post examines what seems to be a case of health care rationing at the expense of the elderly. A very effective vaccine (Pfizer's Prevnar 13) against streptococcal pneumonia, already in use in children, will not be recommended for use by the CDC for 50+ adult use, despite the fact that the FDA given it fast-track approval for this use.

If the vaccine is recommended by the CDC, Medicare must pay for it. But they refuse to even vote on it, even though pneumonia kills 25,000 seniors over 65 every year and hospitalizes 300,000.

This would seem to be a clear case of rationing health care on the backs of seniors. The implications are frightening.


While technological advances have provided us with an abundance of innovative and useful products, many of these enter the market with limited or no clinical data. Medical devices are becoming increasingly complex, and the means of reviewing them for safety have become severely inadequate. Over the past several years, critics have repeatedly pointed to the urgent need for the FDA to strengthen the approval process for medical devices - particularly the 510(k) process - and to increase and improve safety surveillance requirements. Innovation - crucial to the US maintaining its role atop the medical device world - must share the podium with a focus on protecting the consumer. The FDA must carefully balance both interests, allowing products to move through the process in a predictable manner, while still thoroughly collecting safety data.

So what is the right amount of regulation to create optimal levels of safety and innovation? While there is no definitive answer that covers every product - medical devices must be examined on a per-product basis - the FDA is tasked with ensuring safety and limiting risk, but it also has a duty to help foster - or at least not get in the way of - innovation.

On the one hand, safety is the original mandate behind the FDA. Protecting the public from harmful products should remain an essential priority. But at some point, too much regulation may impede technological advances in medicine, and ironically inhibit the public's access to new devices. The FDA must first and foremost take a consistent, predictable approach so that manufacturers can readily meet their requirements. The process must be logical and easy to follow, and clearly communicated - with constructive information exchanges between manufacturers and the agency. This means that product review processes must be overhauled, as currently, they are riddled with inconsistencies.

Product recalls and adverse events have consistently been in the news for the past decade. The FDA indicates that approximately 2,500 medical devices were recalled for potential safety problems in fiscal 2008, nearly double the number reported in 2007, and a 164 percent increase since 2000. In fact, more than 1,000 recall notices were sent out in the first seven months of 2009, 100 of which were ranked as "Class 1" recalls, which indicate a significant risk of injury or death. Of the devices recalled between 2005 and 2009, 80% were cleared through the 510(k) process or exempt from any approval process.

The DePuy ASR Hip Replacement helps to highlight this issue and the need for better regulatory balance by the FDA. DePuy created two products that utilized a metal-on-metal "cup and ball" implant, but differed in that one required bone resurfacing. The ASR XL Acetabular System - deemed similar enough to a predicate (previous) device that used metal-on-plastic - went through the 510(k) process of review, which requires minimal pre-market studies, and was approved. The ASR Hip Resurfacing System - although it used similar materials - was deemed to be too dissimilar from predicates, given the additional procedure of bone resurfacing - and went through the more stringent Pre-Market Approval process (PMA). Ultimately, the ASR Hip Resurfacing System was not approved for U.S. use due to a lack of clinical data demonstrating safety. DePuy decided not to pursue approval in the U.S. - even though the product was approved for use in Europe and Australia - given their belief that interest was waning in the resurfacing technique.

Both devices were later voluntarily recalled worldwide after DePuy determined (through post-market surveillance) a high rate of premature failure. Though the FDA's tough PMA process restricted one device from getting to the market, they still approved the other through the 510(k) process. Consequently, the FDA succeeded and failed at the same time!

Even though one device required bone-resurfacing, most of the failure issues were linked to problems with the materials they both had in common. Why is it that two devices using the same materials - ultimately recalled for the same reason - went through two different approval processes and met two different fates? While the specifics are beyond the scope of this article, the example points to a large gap in the way the FDA balances safety and innovation.

The process seems flawed in that two very similar - though not identical - devices would receive two different levels of scrutiny. The FDA must reassess the criteria it uses to determine when a product needs a tougher review, and when it can ease safety requirements to increase innovation. In the case of the hip implants, the only clear answer is that the 510(k) process did not do an adequate job of finding that balance.

Our current example shines a light on a much bigger picture than just one product recall, and that is that most devices are ultimately approved via the 510(k) process. Such products are not likely to have had clinical studies performed pre-market, resulting in insufficient data on safety issues prior to launch. Even so, pre-market clinical trials (even large ones) have limited power to identify the potential for post-market adverse events or failures, especially given the relatively short lifecycle of these products. Consequently, this minimizes the likelihood of identifying low-frequency adverse events.

As NAI discusses in Post-Market Safety Surveillance and Medical Devices , this results in a greater burden on a passive post-approval surveillance system, which has not proven effective. Accordingly, ongoing (active) review of patient registries may be increasingly necessary. While the concept of registry reviews makes sense, current U.S. use of registry data may not be adequate. But if the FDA made monitoring and auditing of registries a part of their review processes, this could be a viable option.

But this type of analysis is a two-way street. The FDA must work closer with companies throughout the product lifecycle to identify safety concerns. This will enable the FDA to effectively communicate a consistent message balancing both safety and innovation. Presently, discussions to reauthorize the Medical Device User Fee and Modernization Act (MDUFA) are focused on building a more collaborative process between the agency and manufacturers, which I believe represents a significant move in the right direction.

MDUFA was originally enacted in 2002 to enable the FDA to collect user fees for pre-market reviews of certain medical device submissions, which would in turn allow the FDA to increase resources and reduce the time it takes to approve a product. FDA committed to implementing a more aggressive and interactive review process, and MDUFA was extended in 2007. The current reauthorization aims to further solidify these goals, as well as add some additional efforts to improve the process.

By paying $595 million over the next five years, the medical device industry hopes that MDUFA reauthorization will reduce review time, but also improve the efficiency and consistency of the process. Some of the current issues that are being considered include shorter cycle times for 510(k) submissions, greater predictability and transparency (including an additional "Product Development Fee" to allow for more collaboration before submission), stopping the "review clock" to allow for more questions, and better rationing of resources per review.

MDUFA reauthorization has the potential to benefit the industry and regulators alike. The issues discussed in connection with hip implants are not uncommon across the device spectrum. Encouraging greater collaboration among stakeholders represents an excellent way to achieve better balance between safety and innovation. Manufacturers want reduced cycle times for 510(k) submissions, but this can only be done safely if the FDA sets clear requirements, and both parties communicate frequently throughout the process. The revenue created by MDUFA should provide the means for enhanced review and better analysis, ultimately enabling good products to come to market faster.


Many things in life make me crazy. It doesn't take much, as anyone who knows me will confirm. So, it is no surprise that the feature article in last week's Times called "Is It Safe to Play Yet?" did the job. But it happened before the end of second sentence. And that takes something special.

Things certainly have changed since I was a kid. People had a slightly different idea of the meaning of the word "safe." Biking to school (no helmet) through a Superfund toxic waste site with a bottle of Jack Daniels in one hand and boa constrictor wrapped around your neck seemed perfectly fine. Who knew? Perhaps we are much safer now. But also much less sane.

The Times article first described the efforts made by a pregnant woman to prevent her baby from being exposed to any chemical. So, she tossed out pretty much everything in the house, including her makeup, shampoo and detergents. She was even worried that the plastic stickers decorating the nursery were toxic and irritating to the lungs, because they were made out of polyvinyl chloride (PVC). Except it's all wrong.

A little fact check: Vinyl chloride is a toxic, irritating gas used to make PVC. I know this. I got a whiff of it a few times during my career--most unpleasant. But vinyl chloride and polyvinyl chloride are not even remotely the same, except for the names. PVC is a white powder that can be made into all kinds of plastics, including your plumbing, vinyl siding, and a good deal of your car. It has been in use since the 1950s. It is impossible to avoid, which is fine, since it is harmless.

Naturally, the more the woman read, the scarier every label got, leading her to the internet, which, in turn led her to the web site of the Environmental Working Group (EWG)--a very vocal and extremely activist organization, that appears to be against all physical matter on earth.

I don't know what their charter says, but I suspect it looks something like this:
1. Assume that chemicals cause cancer unless you can prove that they don't.
2. It is not possible to prove that a chemical doesn't cause cancer.
3. See #1.

And this woman is not alone. People who apparently listen to too many TV celebrities are flocking to sites like healthychild.org, and healthystuff.org, to ensure that their babies are exposed to absolutely nothing that sounds threatening. Some of the big no-nos these days are rubber ducks, perfume, plastic and steel (!) utensils, pajamas and frying pans.

Even cribs are now dangerous--untreated, "formaldehyde-free" wood cribs handmade by the Amish are the latest rage. Sorry guys, but the amount of formaldehyde coming out of a normal crib, if any, would be so miniscule that it could never be measured. And even if a smidgen of formaldehyde somehow made it into your body, it is gone in 5 minutes.

Some environmental changes have been real success stories, like removing lead from gasoline. That was a real risk. But this notion that we are constantly are being bathed in toxins, and that all accumulate in our bodies and do some kind of harm is farcical. The reason that miniscule amounts of chemicals can be measured in our bodies is due to better analytical techniques--not a wholesale poisoning of the American public.

My current favorite fad is the obsession with "green," non-toxic detergent made from baking soda and vinegar. When the two are mixed another chemical is formed called sodium acetate. Is that dangerous? No. But it doesn't clean anything either. And if you're tempted to make some, consider the experiment below (not recommended):

1. Take a half-full bottle of vinegar
2. Pour in a bunch of baking soda
3. Close bottle and shake thoroughly, making sure you hold it really close to your face
4. Retrieve your head from the tree above you

Your kids are going to spend a lifetime being exposed to chemicals: auto exhaust, smoke, soot, chlorine, soap, perfume. They will be fine. Just calm down. The groups with a vested interest in keeping you afraid are not a credible source of information.


I've just returned from a week long family hiatus in Florida where I tried (mostly successfully) to unplug from the health care Matrix. Still, there've been a number of interesting develoments in the health care/biopharmaceutical space that caught my eye over the last several days:

People are starting to pay attention to the the "hard truths" of health care reform. As Politico noted on Friday, President Obama's many promises about health care reform look likely to haunt him over the next few months. In a nutshell, people are going to lose coverage that they like, health care costs are going up, not down, and the more people learn about the Affordable Care Act, the less they seem to like it. As Jonathan Adler points out at the Volokh Conspiracy, these "hard truths" were noted early and often by the ACA's critics - with the media now playing catch up.

There's a terrific article on the challenges to R&D productivity in the drug industry in Nature Reviews Drug Discovery by Jack Scannell et al. It's eminently worth reading, and Derek Lowe has also been offering a sustained commentary at his blog, In the Pipeline.

Finally, Luke Timmerman at Xconomy notes that while the Euro may be imploding, American venture capital firms seem to be growing more bullish on the future of the European biotech sector:

--Domain Associates, a top life sciences venture firm in Princeton, NJ and San Diego, struck a deal with Rusnano, a Moscow-based nanotechnology firm owned by the government of the Russian Federation, to invest as much as $760 million in biotech companies in both the U.S. and Russia. Rusnano and Domain said they will work together to transfer technology into Russia, and build up biotech development and manufacturing capabilities.

--Sofinnova Ventures, which raised a new $440 million biotech-only venture fund last year, said last week that it plans to open an office to look for investments in Ireland. That move came after Enterprise Ireland and the National Pensions Reserve Fund put $37.5 million into the latest Sofinnova fund.

--And today, Versant Ventures, a $1.6 billion Silicon Valley-based life sciences fund, is announcing that it's opening a new office in Basel, Switzerland, where it will look to make new seed and early stage biotech investments. Versant has brought in Guido Magni, Roche's former global head of medical sciences, to work out of the new office with part-time colleagues Tom Woiwode and Brad Bolzon.

There's nothing new about various European governments or economic development agencies attempting to build up their biotech clusters. Even while the industry has become increasingly networked around the world, and some tremendous innovations have come out of Europe, the U.S. has dominated biotech since the beginning in the 1970s. Last year, U.S. biotech companies raised about four times as much venture capital as companies in Europe, according to Ernst & Young's Beyond Borders report. Public biotech companies in the U.S. generated about $61.6 billion in revenue last year, about four times the revenue of their counterparts across the Atlantic.

What is happening is that the U.S. venture business is in crisis, and the firms who are left standing are being forced to be more creative than ever to stay viable.

Crisis or not, Timmerman finds it hard to believe that Europe will outshine the U.S.-based industry any time in the near future:

No question, biotech is a global industry and the trend toward outsourcing that relies heavily on low-cost vendors in China, India, and Eastern Europe is likely to continue. But when you're talking about where the intellectual property really comes from, and the networks of entrepreneurs and investors that it takes to start companies that create real drugs or devices, there are only a small handful of clusters in the U.S. that really do it well.

While I don't think the U.S. can take its leadership for granted, I wouldn't count on any place in Europe becoming the next Boston or San Francisco anytime soon. Europe has its opportunities, and a few VCs will surely find a way to capture them. But it's hard to imagine a real movement toward one of the riskiest industries on the planet, right when people there will probably be licking their financial wounds for decades.

Finally, the IPAB repeal bill in the House (H.R. 5) is set to come up for a vote later this week. Stay tuned for more developments on that front....


We at MPT have written extensively about how the FDA's and other groups' conflict of interest rules, which have the effect of discounting or ignoring information connected in any way to the drug and device industry, are not only short-sighted, but counterproductive (see here, here, and here for just a few of the more recent examples). Now, via Richard Epstein, we learn that some of the biggest critics of these rules are patients themselves:

"In addition to the endless regulatory hoops mandated by the FDA for marketing approvals, a significant, and wholly unnecessary, part of this protracted slow-down [in new drug development] stems from sharp limits on conflict of interest waivers for the agency's vital advisory committees that help the FDA review new medicines, which Congress imposed on the agency, with excessive zeal, in 2007. Rather than coming from industry, complaints about onerous conflict-of-interest rules have come primarily from patients groups representing the users and consumers of pharmaceutical products, for whom new drugs and devices often spell the difference between life and death. ...

"This prohibition has not only left about 20 percent of the FDA's many committee positions vacant but also led to a pool of 'experts' less qualified than those disqualified, by virtue of the simple fact that they are so pre-eminent in their fields that industry seeks out their advice and services. ...

"Quite simply, the current FDA conflict of interest rules regard doctors and scientists with any financial connections with drug and device manufacturers as corrupt shills, who should be banished from its sacred precincts. Yet it takes an all-too-tolerant position to such 'pure' advisors like Sidney Wolfe, from Public Citizen, who has served on the FDA's Drug Safety and Risk Management Advisory Committee since 2008, despite being an avowed enemy of the industry and a close ally to plaintiff's lawyers who launch multibillion dollar class action suits against drug companies - and who contribute generously to Public Citizen's coffers."

Now, it's not completely true that FDA has never ruled out someone for a so-called "intellectual conflict". Indeed, Sidney Wolfe himself was once prevented from voting on the disposition of a class of birth control pills containing the hormone drospirenone because a Public Citizen publication had warned against using those products. And a prominent cardiologist had once been invited and then "disinvited" from participating in an advisory committee hearing on the blood thinner prasugrel because the doctor had made disparaging comments about the drug.

Still, in the latter case, FDA later acknowledged that it should not have excluded the cardiologist for having a possible "intellectual bias". And though Wolfe was only permitted to sit on the advisory committee panel considering drospirenone as a non-voting member, he continues to vote on numerous other products despite having campaigned for more than 30 years against the evils of the pharmaceutical industry.

The point remains, conflict of interest allegations almost invariably run in one direction: against drug and device manufacturers and not their critics. The only time it seems the critics are willing to overlook a drug industry conflict is when the otherwise conflicted party is joining in on the criticism of another manufacturer's product.

Consider one particularly ironic case that I wrote about previously: U.S. Sen. Charles Grassley (R-Ia.) has been a frequent critic of FDA for not being harsh enough on industry conflicts of interest. But, at the same time, Grassley has frequently relied upon Cleveland Clinic cardiologist Steven Nissen to pursue their joint campaign against the diabetes drug Avandia. But it turns out that Nissen just happens to be one of the investigators on a study purporting to show that Avandia's closest competitor, Takeda Pharmaceutical's drug Actos, is superior to Avandia because it doesn't increase cardiac risk. And, as it turns out, Takeda provided $25,000 in funding to Nissen's Cleveland Clinic team to conduct the Actos study.

If the tables were turned, and it was Takeda's product that Grassley wanted banned, would he have relied so easily on Nissen's judgement that Actos was safe? Steven Nissen is a highly regarded scientist, and I am aware of no credible evidence that his views on Avandia or Actos have been biased by the funding from Takeda. But you could bet your bottom dollar that, in such a case, Nissen's views would not only be discounted, they'd probably be ridiculed as nothing more than "bought and paid for science".

Ultimately, conflicts of interest can never be eliminated, though they can and should be managed. But it seems clear that the way Congress and the FDA are trying to manage them has been a dismal failure.

Two Faces of Cancer
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According to a paper in yesterday's Journal of Clinical Oncology, the 5 year survival rate of children with acute lymphoblastic leukemia (the most common form) has continued its upward trajectory, and now stands at 90 percent--fairly amazing considering that it was almost always fatal as recently as the 1960s.

This should not be taken as evidence that the end of cancer is around the corner--it is not. Although there have been a few cancers that are either curable (testicular, for example), preventable (cervical) and more treatable (breast), progress against the disease has, for the most part, been incremental and slow. A recent paper in the New England Journal of Medicine offers an peak into why this may be.

When Dr. Marco Gerlinger of the Cancer Research UK London Research Institute and his group examined the genetic makeup of kidney tumors and compared it to that of metastases from the same tumor, they found unexpectedly large differences between the genetic makeup of the original tumor and the cancer that had metastasized. Oncology researchers have long known about mutation of cancer cells, but until this week they didn't appreciate the magnitude of the process. According to Dr. Merlinger, in real life "...a serious flaw in the imagined future of oncology is its underestimation of tumor heterogeneity."

The authors logically concluded that a single needle biopsy of a cancer mass will tell you little or nothing about the genetics of any other masses within the body, and that this would make personalized treatments very difficult. But the implications of this are worse.

The use of specifically targeted cancer therapies (called kinase inhibitors)--new drugs that have been designed to attack a specific growth pathway in the cancer cell--has been the holy grail of oncology research for some time. But much of original hype and promise about this revolutionary approach has not been realized. Ironically, part of the problem is that the very specificity that was desired was too much of a good thing.

Even if you could wave a magic wand and obtain all the genetic information of all cancer cells, there is still not that much you can do about it. The increased specificity of kinase inhibitors limits their damage to non-cancerous cells (fewer toxic side effects), but also decreases their ability to kill mutated cells from the same tumor.

This may explain why it is not uncommon to see impressive responses at the beginning of treatment, with profound tumor shrinkage. However, the mutated cells--which are either part of the original tumor or form later on--will eventually thrive in the space left behind by the original tumor. When the cancer returns it is much less treatable.

This heterogeneity points out one of the big obstacles in oncology research--the lack of a good animal model. Certain mice are bred to have no immune system. This enables scientists to implant cultured human cancer cells under their skin, and the cells will grow into a tumor.

But the implanted cells are much more homogeneous and more susceptible to attack by the new drug. When treated with the experimental drug, the tumor will often disappear entirely. When applied to real life, things are not so simple. This tumor heterogeneity will be a major barrier to overcome to take this approach to the next level.

It is unlikely that we will see groundbreaking advances in oncology comparable to what happened with other deadly diseases, such as AIDS and hepatitis C, which can now be effectively controlled or cured. Virtually all chemotherapy regimens use one or more drugs that were first used in the 1960s. Reliance on 50 year old drugs as the first line of defense is a good indicator of how difficult the problem really is. There is a long way to go.



The Indian government was upset at Bayer and Onyx for not supplying Nexavar (sorafenib) in India at a "reasonably affordable price." India was also miffed that Nexavar was imported into India, rather than being manufactured there. Instead of just stewing, India took action. The government broke Nexavar's patent with a "compulsory license" and gave a new license to Natco Pharma, an Indian company, to manufacture and market a generic version of Nexavar in India until Bayer's patent expires in 2021.

What's really going on here?

Many governments have monopsony power when buying pharmaceuticals--that is, monopoly power on the buyer's side--and they use this power to get good deals. These governments are, in effect, saying that if they can't buy a medicine cheaply, their citizens won't have access to it. This ultimatum has two aspects. One, if the price is "too high," the pharmaceutical company won't sell a single bottle in that country. And two, if the government feels its citizens really need that medicine, it will break the drug company's patent. This threatened violation of intellectual property rights can bring a seemingly powerful drug company into quick compliance. When faced with a choice between making nothing or something, most drug companies choose the latter.

It is in everyone's interest to give drug companies an adequate incentive to invest in new drugs. To do so, drug companies must be able to price their drugs well above production costs to a large segment of customers to cover the expense of R&D. However, each individual government's narrow self-interest is to demand a low price on drugs--closer to the manufacturing cost--and let people in other countries pay the high prices that generate the return on R&D investments. Each government, in other words, has an incentive to be a free rider. And that's what many governments, like India, are doing.


Yet another major pharmaceutical company is now being persecuted prosecuted for off-label promotion. This time it's Johnson & Johnson, being pursued over allegations that the company illegally promoted its antipsychotic drug Risperdal for unapproved uses. As The Wall Street Journal reports, though, settlement negotiations have hit a snag.

Federal prosecutors in Philadelphia reached a tentative agreement under which J&J would pay a whopping $1 billion in fines. But Justice Department officials in Washington rejected the deal and are now seeking an even higher payment. According to the Journal:

"The Justice Department prosecutors are seeking a settlement of around $1.4 billion, the sum that Eli Lilly & Co. agreed to in 2009 to resolve allegations it had improperly promoted its antipsychotic drug Zyprexa, according to one of the people familiar with the matter. Yet J&J officials have resisted paying that amount.

"An outright rejection of a deal is unusual, according to Shelley Slade, a former Justice Department health-care fraud lawyer who now represents whistleblowers suing drug makers. Ordinarily, prosecutors in a U.S. Attorney's Office work with counterparts in Washington, D.C., to find mutually agreeable terms, Ms. Slade said."

As with many of these off-label promotion cases, the allegations are broad ranging and appear to include a mix of ethical and unethical behavior - ranging from the promotion of Risperdal to treat pediatric patients with attention-deficit disorder prior to 2006 when the drug was approved for that use to the payment of kickbacks to pharmacies to push Risperdal over alternative drugs. And the company is being pursued not only by the feds, but also by several state attorneys general.

But, while J&J thought its position in the state lawsuits was strong enough to fight in court, the federal government has a tool at its disposal that makes an out of court settlement almost guaranteed: the potential exclusion of any of the company's products from Medicare and other federal health programs. Federal anti-fraud laws permit government programs to exclude, suspend, or "debar" corporations that have merely been accused in an indictment of fraudulent behavior from doing business with the federal government. No guilty verdict is necessary.

As you can imagine, with the federal government picking up the tab for more than a quarter of all US health spending, exclusion from federal health programs would be tantamount to a corporate death sentence. "It is thus not surprising," notes Ropes & Gray attorney Joan McPhee, "that virtually all rational corporations ... conclude, as a business matter, that they cannot incur the risks associated with taking an indictment and going to trial, even when, in the corporation's assessment and that of its seasoned counsel, the threatened case is without factual or legal merit."

Because I have argued in both the popular and scholarly press that much (though not all) off-label promotion is protected by the First Amendment, I have been asked repeatedly why few defendants have challenged the off-label promotion ban in court. In part, it is because the high profile cases brought against drug firms often include allegations other than constitutionally defensible speech - such as the kickback allegations in this case. The flip-side of that is that the Food and Drug Administration and Department of Justice have been very good at selecting the cases they pursue, so as not to set up clean constitutional questions. And, when backed into a corner, the FDA and DOJ have been able to dispense with problematic cases before a clear ruling that the off-label promotion ban is unconstitutional (see my descriptions of the WLF and Allergan cases in this paper, for examples).

But arguably the most important reason why more of these cases don't make it to court is the fact that an indictment, what McPhee calls the "admission ticket" to the courtroom, all by itself spells potential doom for a drug firm. "[W]hile there are strong legal and constitutional defenses to the government's attempted criminalization of truthful, nonmisleading off-label dissemination, there is no available avenue for targeted corporations to gain access to a judge or jury without risking corporate death."

So, J&J may believe that the Justice Department's demand for a $1.4 billion settlement is wholly unwarranted. But I'd be willing to bet they eventually take that deal.


Yesterday the FDA's Arthritis Drugs Advisory Committee voted 21-0 that anti-nerve growth factor (NGF) agents should continue to be developed to treat osteoarthritis pain.

From Bio Century:

Because the joint destruction was increased in patients taking anti-NGFs with NSAIDs, the panel felt studies in OA could continue if concomitant NSAID use is excluded and additional safeguards are put in place. These could include measuring biomarkers of joint destruction and increasing use of MRI. The panel did not feel anti-NGFs should be restricted to refractory OA patients despite an increase in joint destruction seen in clinical trials.

Read Paul Howard's analysis of the FDA's anti-NGF in his blog post, "Chronic pain relief: too much of a good thing?"


Margaret Hamburg recently posted a piece on the FDA's blog reflecting on the thalidomide tragedy's 50th anniversary. Her post describes how this particular tragedy was well-managed in the U.S. compared to other countries, and champions "smart, science-based regulation."

As we reflect on this anniversary, it's worthwhile to consider the ultimate results of the thalidomide tragedy. In 1962, the Kefauver-Harris Amendments to the Food and Drug Act were passed as a consequence of the backlash over thalidomide (they were actually proposed two years earlier, but had floundered due to lack of popular support). The amendments expanded the FDA's role in ensuring drug safety by requiring adverse event reporting and increasing the agency's control over human experimentation. They also expanded the mandate of the FDA to include ensuring drug efficacy -- not just safety.

The issue with thalidomide wasn't that it didn't work -- it did, in fact, relieve morning sickness. But it wasn't safe. And since the thalidomide that had been taken in the U.S. was part of a clinical testing program, expanding the FDA's role in controlling human trials made a lot of sense. Dr. Hamburg's blogpost says that the "Harris-Kefauver Amendments created a culture of quality and innovation that laid the foundation for our current regulatory environment which fosters a domestic pharmaceutical industry that is second to none." But is the FDA really doing enough?

Consider the multibillion dollar nutriceutical industry, including vitamin supplements, which are reviewed by the FDA for safety, but not for efficacy. There's a lot we don't know about these products - like how they might interact with other drugs, which is a major safety issue. And setting aside the safety issue, we don't even know if these products work! The placebo effect could ultimately be the reason consumers feel any benefit from these supplements. Is the FDA still meeting its mandate of making sure products work, or are these nutriceuticals just 'modern day snake-oil'? More importantly...are they truly safe?

The agency should return to prioritizing its original mandate - focus on safety - while still fostering the advance of medicine. FDA involvement in balancing safety and innovation will require more stringent requirements for post-market studies and real-world evidence. Coupled with a streamlined process reducing the time it takes to get products to market, this approach would create a climate more hospitable to innovation and ensure products remain safe and effective. But celebrating an achievement from 50 years ago - albeit, a very important one - runs the risk of not continually trying to improve a process...especially one that clearly still has some significant gaps.

Good science is a must going forward, and the FDA should continue to direct its attention there so that another "thalidomide" doesn't happen. With unsafe and untested products still getting to the market, the FDA can't afford to rest on its laurels. It's time to overhaul both the agency's regulatory process and its focus, which I will address in greater detail in my next post.


(This article is re-posted from Megan McArdle's blog at TheAtlantic.com where Avik Roy will be guest blogging over the next two weeks.)

Avik S. A. Roy

Chapin White of the Center for Studying Health System Change has published an important new paper in Health Services Research, a journal of health economics, which suggests that a critical part of the Affordable Care Act--its expansion of Medicaid coverage to 16 million more Americans--may actually reduce those individuals' access to health care.

White's report comes on the heels of numerous studies that show that patients on Medicaid, our national government-run health-care program for the poor, do far worse on health outcomes than do those on private insurance, and in some cases, worse than those with no insurance at all. (For an extremely deep dive into these studies, see my three-part series on the topic.)

1) Medicaid underpays doctors for their expenses

Why does this occur? The main reason is that Medicaid underpays doctors and hospitals to care for Medicaid beneficiaries. Medicaid's reimbursement rates are around half of those paid by private insurers. In many cases, Medicaid pays doctors less than it costs to care for Medicaid patients, meaning that doctors face the choice of caring for the poor, and going broke, or shutting their doors to Medicaid patients. One survey found that internists were 8.5 times as likely to accept no Medicaid patients at all, relative to those with private insurance. Another found that two-thirds of kids on Medicaid were denied a doctor's appointment for a serious condition, relatively to only 11 percent for the privately-insured. . .



The House Energy and Commerce Subcommittee on Health today is hearing testimony on a wide range of topics related to the pharmaceutical industry and FDA regulation of medical products, including the integrity of the pharmaceutical supply chain.

But for my purposes, I wanted to focus on one part of the discussion: opening up the FDA's accelerated approval (AA) process for new medicines to new indications. Since its creation in 1992, in the midst of the AIDS crisis, AA has led to a surge in new medicines for HIV and AIDS, and some orphan diseases. Beyond that, it has largely remained off limits for other classes of drugs.

In a nutshell, the AA pathway allows early market access (typically after Phase II trials) for drugs that show efficacy against a "surrogate endpoint" that is reasonably likely to improve clinical outcomes. The classic surrogate marker is viral load in HIV patients, where 24 weeks of viral suppression is sufficient to grant AA; 48 weeks of viral suppression beyond that is sufficient to grant full FDA approval.

Sponsors of drugs that are granted AA are expected to complete confirmatory trials with "due diligence". Companies that do not complete those trials, or whose trials fail to confirm a clinical benefit can be withdrawn by the FDA. In point of fact, as far as I know, only a handful of drugs granted accelerated approvals have been withdrawn from the market, and almost all voluntarily by the sponsor. Only one drug - Avastin, for metastatic breast cancer - has had withdrawal initiated by the FDA.

What does this have to do with the price of tea in China? Not to mix metaphors, but in areas where surrogate markers are either well established (HIV) or at least accepted (tumor shrinkage or progression free survival in oncology) companies have a viable pathway for rapid market access.

This gives patients rapid access to new therapies (often several years faster than if the drugs had to go through Phase III trials first) and allows companies to reach market faster - which, considering limited patent time, is a very valuable financial incentive driving R&D in these areas.

Not surprisingly, manufacturers have gravitated towards AA. As CDER Director Janet Woodcock testified today:

Over 80 new products have been approved under Accelerated Approval since the program was established, including 29 drugs to treat cancer, 32 to treat HIV, and 20 to treat other conditions such as pulmonary arterial hypertension, Fabry disease, and transfusion-dependent anemia. Three of the 30 new molecular entities (NMEs) approved in 2011 were approved under Accelerated Approval.

A little basic math will tell you that about 75% of the products approved through AA have been for HIV and cancer. In 2011, for instance, two of the three drugs that received AA were for cancer indications. As patent expirations have hit more "blockbuster" drugs, and the FDA has toughened safety testing for primary care drugs, the industry has shifted even more of its research dollars towards cancer and other rare indications that are eligible for AA. Dr. Woodcock underscores this point as well:

Therapies for rare diseases--those affecting fewer than 200,000 people in the United States--represent the most rapidly expanding area of drug development. Although each disease affects a relatively small population, collectively, rare diseases affect about 25 million Americans. Approximately one-third of the NMEs and new biological products approved in the last five years have been drugs for rare diseases.

Now, this is all to the good. New targeted cancer drugs have made impressive inroads against some cancers, like Gleevec for CML, Herceptin for breast cancer, and Xalcori for lung cancer. Patients with rare genetic diseases have also seen the first glimmers of hope in decades. HIV positive patients, thanks to a flood of powerful new AIDS cocktails, can look forward to a nearly normal lifespan.

But other indications - like Alzheimer's, diabetes, obesity, to name a few - that afflict millions of Americans have relatively little chance of getting through accelerated approval because the FDA hasn't been able to agree on surrogate endpoints that would make those medicines eligible for AA.

It is critical - if we want to improve patient health, lower U.S. health care spending, and sustain U.S. based medical innovation - to make AA a routine pathway for cutting edge medicines for these indications.

New legislation, the FAST Act in the House, and the TREAT Act in the Senate, would do much to expand the FDA's regulatory science base to help identify new surrogate markers - and, equally importantly - set timelines for the FDA to issue guidance for expanding the AA pathway.

We've done this before. We did it with AIDS. We did it with cancer. And despite many reservations and criticisms about using surrogate markers (both for cancer and AIDS) the long term results have been enormously beneficial for patients.

We didn't wait until the science was perfect. The science will never be perfect, and it will evolve over time. But having the pathway available for new indications will, in itself, help to drive innovation for more targeted therapies that can show more powerful efficacy in early stage testing.

Build a better AA pathway. Better medicines will come.

That's message we should be sending to Congress, the White House, industry, and the FDA.


I had to read something twice yesterday to convince myself that someone didn't spike my Cap'n Crunch with LSD. Turns out they didn't. Which is for the most part good, except that I'm still having trouble explaining what I saw.

Sam Waksal, the founder of ImClone, probably best remembered for the insider trading scandal of 2002, yesterday published an opinion piece in The New York Times, entitled "Pay Only for Drugs That Help You," in which he suggested an "interesting" way of making drug companies more innovative.

While his idea certainly gets high marks for creativity and novelty, it also ranks near the top of the "You Must Be Kidding Me" scale.

Citing the $2.6 trillion that the US spends on health care with little to show for it (which I don't buy at all) Waksal proposes that individuals and insurance companies should only be held responsible for paying for drugs that "work" for them. Waksal calls this a "pay-for-response model."

He cites as an examples cancer drugs, which would have to meet criteria established by the FDA for any given patient, or the company would not be paid for the drug. He also applies this standard to new drugs for hepatitis C, which would have to cure the patient for the companies to be paid.

While the concept behind this idea might be intruiging to some, to me it is simply insane. Here are a few reasons why.

First, response to cancer therapy is not a simple measurement. It involves multiple factors, including tumor growth, or shrinkage, increased survival time, increased time without disease progression (these two are not the same), and ancillary issues, such as pain relief, weight gain and time in the hospital. Good luck trying to come up with a set of standards at all, and even better good luck trying to apply them to individual patients.

If a tumor shrinks by 30% instead of a theoretically required 35%, is there really a difference? What do you do then? Yet, some numbers will need to be used to make this call. What will you do if the tumor grows, but the patient survives a few months longer than expected. One could spend hours arguing about whether the response of one patient meets criteria to determine if something "works." Now imagine trying to do this on a national scale. Nightmare.

And if Waksal thinks that this will spur innovation by drug companies, he couldn't be more wrong. Things are bad enough right now. It is impossible to determine how well a drug really works until it is marketed and widely taken. But if you add this subjective measurement to the mix why on earth would a company spend $1.3 billion and 15 years to get something approved, and still not have the slightest idea if they'll get compensated for their efforts.

No- this would not spur innovation. It would spur the end of drug discovery entirely.

And why apply this concept solely to drugs? If a neurosurgeon removes a herniated disk from my neck and I still experience some pain later on, should he be paid? If my investment adviser fails to meet my expectations for my portfolio, do I get me fees back? If I go to a lousy Broadway show or the Yankees lose, do I get my money back?

Aside from being medically counterproductive, this concept is illogical at its roots. There are no guarantees in life, especially in the medical world. You pays your money, you takes your chances.


Alzheimer's Disease-related dementia is the next public health "time bomb." And it's not just a bomb of human suffering; in addition, AD dementia is also an already-exploding bomb of fiscal red ink.

"The figures speak for themselves. We are really going into the next global health time bomb"
--those are the words of the Belgian-born Dr. Peter Piot, now director of the London School of Hygiene and Tropical Medicine. Piot co-discovered the Ebola virus in Africa back in the 1970s, and, since then, he has been a leading voice on AIDS issues; he was president of the International AIDS Society, then took leadership roles at the World Health Organization and the United Nations. In other words, he has been a witness--both as a front-line worker and as a policy-maker--to epidemics in the past. And so when he espies a new "global health time bomb" on the horizon, he deserves a close hearing.

As Piot explains, the 36 million dementia sufferers in the world today--increasing a rate of one new case every seven seconds--will double in just eight years. And yet, he argues, it's a "myth" that dementia is an inevitable part of aging. That is, medical science could and should address the issue in a direct problem-solving manner. But unfortunately, the pipeline for AD drugs is drying up.

Here in the US, some six million Americans have Alzheimer's, costing annually, according to the Alzheimer's Association, $172 billion annually, reaching a cumulative cost of $20 trillion by 2050. Yet in American politics today, the focus of eldercare is on finance--Obamacare vs. Republican insurance alternatives.

In other words, AD is being seen as a "demand-side" problem; people are afflicted with AD, and then some financing mechanism kicks in, public or private. In other words, it's an accounting issue. The thought of dealing with AD on the "supply side," however--the cure side--is simply not being discussed in Washington DC or on the presidential campaign trail.

Yet if AD is handled the way we handle many other diseases--with an emphasis on paying for it, as opposed to curing it--then we face fiscal calamity, as well as medical calamity. In the past, healthcare was seen as a medical issue; doctors tried to cure the disease. Now increasingly, and perversely, healthcare is seen as a financial issue; that is, policy experts grapple with issues of coverage and cost-control. And so, not surprisingly, the idea of curing AD falls through the cracks of political and governmental indifference.

But the compassion factor aside, AD is not a finance issue.  It is a science issue.  Either we come up with an effective treatment for AD/dementia, or we face a future of enormous costs that will likely be uncompassionate in the extreme. That is, future leaders will either consign millions of elderly Americans to long-term 24/7 care, hiring millions more to care for them--and with brutally crushing taxes to pay for this bureaucracy of chronic suffering--or else concoct drastic measures to reduce those long-term costs.   And we do mean "drastic," in a way that could unhinge the idea of a safety net and a decent society.

Faced with the AIDS epidemic a quarter-century ago, America and the world resolved to focus on a cure, which in many cases today--not enough, but many--is the functional equivalent of a cure.  The medical-science approach to AIDS, as opposed to the finance approach, has been a bonanza for human compassion, for the advance of medical science, and for the economies of countries around the world.

Surely we should want the same sort of positive solution for this fast-approaching medical time bomb.


A few weeks ago, Senator Kay Hagan (D-NC) introduced the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act, a bill designed to accelerate the approval process for new drugs. When she released a draft of this bill in November, I praised her efforts to expand this process in several posts (Avastin, Accelerated Approval and Playing God, and Finding the Right Balance at the FDA) because they reflect a certain logic -- if the accelerated approval process is sufficient to ensure safety and efficacy for some products, then it follows that it should be good enough for others. But the final legislation that Senator Hagan proposed didn't go nearly as far as the first draft.

The first draft of TREAT would have extended the accelerated approval process for a significant number of new therapeutic areas, including first-to-market treatments, drugs for patients who are unresponsive to the standard of care, orphan drugs, and drugs that offer a significant improvement in outcomes over existing treatments for genetic subpopulations of patients, or otherwise satisfy an unmet medical need. If subsequent confirmatory studies didn't reproduce that benefit, the FDA could revoke its approval, as it did for Avastin's breast cancer indication.

In contrast, the final version of the bill that Senator Hagan introduced earlier this month cut the number of groups of drugs that would be eligible for accelerated approval in half, only offering the accelerated process to drugs that target genetic subpopulations of patients with a given condition, offer a significant improvement in outcomes over existing treatments, or otherwise satisfy an unmet medical need. Now, this is still a big step in the right direction, albeit notably less expansive than the first draft.

The real story, though, is the reason for the changes. According to Bloomberg , PhRMA -- the industry organization representing pharmaceutical companies -- lobbied heavily to limit the use of an accelerated approval process. Now why would Big Pharma want to make it harder for new drugs to get to market?

While it may appear strange that large manufacturers would want to make it harder for new drugs to get to market, especially when their pipeline to new drugs is barely dripping, not approving drugs until after stage III clinical trials makes it easier for big companies to maintain their monopoly. In the current system, getting a drug to market can prove to be quite expensive. Even though big pharmaceutical companies have not been generating sufficiently robust late-stage pipelines to replace products facing generic competition, they have built an empire on the fact that their competition is prohibited by the cost as well. So late-stage products can hang on longer and continue to generate a profit.

And because the cost of drug trials remains high, smaller companies are forced to sell or license novel compounds to the major players, further ceding control of the industry to Big Pharma. If the requirements for pre-market data were less comprehensive, it would be much easier for small biotech companies to produce the drugs themselves, rather than partnering with a bigger company. But accelerated approval would level the playing field, and as such, the threat of competition has led to the impediment of potential technological advances in medicine. And while the pharmaceutical companies - big and small - continue to profit, the consumer ends up as the big loser and manufacturers assume the role of the 'man in the black hat' (a moniker they can't seem to escape).

Accelerated approval does not have to mean the end of success for Big Pharma. There is opportunity for pharmaceutical companies and consumers to benefit equally, all the while increasing the level of innovation within the industry and maintaining safety. For some thoughts on how Big Pharma can overcome its R&D issues and improve its reputation, check out my industry briefs: Transforming a Broken Development Engine and Restoring Pharma's Reputation for Ethical Leadership.


Last summer, Pfizer announced that it would begin experimenting with a new, "virtual" clinical trial design in which patients would "participate remotely without having to visit trial sites." The Research on Electronic Monitoring of OAB Treatment Experience (REMOTE) trial was designed to use smart phone and other Internet tools that would enable enrollees and their doctors submit data on the safety and efficacy of Pfizer's Detrol LA (tolterodine tartrate), an already approved treatment for overactive bladder problems.

Pfizer touted the virtual design as having "the potential to widen the available trial population, speed up clinical development while cutting costs (which weigh heavily in patient recruitment and retention), improve patient compliance, reduce withdrawal rates and deliver better-quality, more reliable 'real-time' data."

Unfortunately, the company had to admit recently that enrollment didn't live up to expectations, leading some to question whether the concept is realistically feasible. The good news is that Pfizer's senior director of clinical sciences, Miguel Orri, believes the company has learned enough from the early setbacks to salvage the idea. And the company is planning to launch a re-designed REMOTE 2 trial with tweaks that it believes will address the enrollment problems.

Whether the re-designed trial will succeed remains to be seen, of course. But there is no doubt that this kind of radical reinvention of the standard, 20th Century clinical trial design will be necessary if the research-intensive pharmaceutical industry is going to remain sustainable in the 21st Century. As science journalist Malorye Allison explained in a January Nature Biotechnology article (subscription required), "The expanding timelines, size, failure rate and cost of trials have finally reached a point where, like the towering US debt, nobody can pretend it is viable."

As we become more alert to such concerns as cardiac- and hepato-toxicity, and as scientific advances reveal more and more about the individualized effects of various drugs in smaller and smaller patient sub-populations, regulators are demanding additional testing and more data. This is forcing drug firms to design trials with larger enrollment and increasingly more interventions per patient. In turn, it is taking sponsors longer to enroll trials and longer to run them, with costs skyrocketing as a consequence. And, as we move into a world of more personalized medicine, where each new drug can treat a smaller and smaller population of patients, the conventional, randomized, placebo-controlled trial will have to become a thing of the past. Innovative trial designs may be the only way to ensure a steady stream of new and beneficial treatment options into the future.

Fortunately, the entire drug and biotech industry, along with academic and public sector researchers, are now fully aware that trial methodology cannot continue on as before. Pfizer may be a pioneer in this space, but it is not alone. There are over two dozen companies participating in the Critical Path Institute's Electronic Reported Outcome Consortium, which among other things, is working to develop innovative electronic data collection technologies for clinical trials. And even patient groups have begun experimenting (to a limited extent) with virtual clinical trial designs without drug firm participation.

Eventually, I expect to see much more interest in so-called adaptive trial designs as well. Standard trial methodologies enroll a few thousand patients all at once and administer the identical treatment protocol or placebo. But if, half way through, the administered dose isn't working for some patients, or others experience unacceptable side effects, researchers must go back to the drawing board and re-design the methodology to account for the new information. Adaptive trials, on the other hand, use smaller cohorts of patients in a way that lets researchers adjust the methodology from cohort to cohort to respond more quickly to what they learn along the way.

To date, the Food and Drug Administration has been receptive to adaptive trial proposals, though the agency insists that these trials must be designed more carefully than conventional trials in order to prevent biases from being introduced into the statistical analysis. If the rules for adaptive trials are too rigid, it could prevent firms from reaping the full benefits of the innovative methodologies. Many firms have therefore been reluctant to experiment with these innovations until more is known about how the agency will evaluate them after the fact.

The important thing is that innovative clinical trial approaches are now being given serious attention. Without them, the drug and biotech industries will find it difficult to turn cutting edge science into approved, real world products. As Raymond Woosley, president and CEO of Critical Path Institute, notes, "Randomized controlled trials are out of date, and it's time to use the tools of the future."



Since I wrote about knee replacements for arthritis yesterday, now seems to be a good time to note a puzzling development with a new class of pain medicines known as anti-nerve growth factors that are being developed to treat everything from osteoarthritis to cancer pain.

The need for new pain meds - especially non addictive, non-opiate drugs - is high, and this would be the "first important new class of drugs for general pain since the prototype non-steroidal anti-inflammatory drug (NSAID), came into general use at the end of the 19th century," according to Nature Biotechnology.

The anti-NGF drugs have a lot of promise for severe osteoarthritis, and seem to have offered good pain control in ongoing trials - but those trials have also been halted by the FDA over worries that the drugs may be causing rapid joint necrosis and joint failure in some patients. (Clinical trials for other indications, like cancer pain, are ongoing.)

The FDA is holding an advisory committee meeting next week to review the full data on the medicines and to try and parse exactly what is happening.

One theory is that the drugs actually worked so well at dulling pain that patients became much more physically active - and basically exercised to the point that they did extensive damage to their own joints. If this theory bears itself out, the FDA will likely let trials on the drugs continue, perhaps with a warning that patients should limit their physician exertion after beginning treatment so as not to overstress their joints.

Earlier, Phase II studies with one drug in 2006-07 didn't uncover any joint problems, which only emerged after 16 (out of 6800) patients in Phase III trials developed the problems in 2011.

The drug won't be out of the woods until the FDA has some assurance that joint necrosis isn't an on-target (or off-target) effect of the entire class (several drug companies are developing competing anti-NGF monoclonal antibodies). Some researchers (quoted in the same Nature Biotechnology article) suggested that joint necrosis might be directly related to the drugs' mechanism of action.

Even if the question isn't totally resolved at the advisory committee meeting, the FDA also has additional tools at its disposal to monitor potential adverse effects in the post-market environment (if one or more anti-NGF's are eventually approved for marketing), where patients could be tracked to see if their joint failure rates exceeds the background rate in the osteoarthritis population, and why.

We'll have a much better picture of the benefits and risks of the anti-NGF class after the FDA's advisory committee meeting.

For more background on the drug class, and the FDA's clinical hold, see this Bloomberg article.


The folks at Center for Science in the Public Interest (CSPI) have apparently run out of poisons to scare us with because they are now recycling some oldies. Their scare du jour is called 4-methylimidazole (4-MEI). Yawn.

Old, like in 1951--the earliest paper I could find in which the compound was studied for toxicity in rats. The rats are most likely no longer alive, but it wasn't because of the 4-MEI. Nothing happened to them during the experiment.

In fact when the authors tried to find the minimum amount of 4-MEI required to show toxicity, it was estimated to be 5 grams per day per rat, a crazy high number. Applied to humans, that comes to 700 grams per day. To put this in perspective, this is weight of 3 boxes of Kraft macaroni and cheese before cooking. That's how much chemical you would have to eat. It's not clear whether that much mac and cheese would also kill you.

Having worked (and played) with chemicals for 30 years, I developed a pretty good idea which ones are bad (e.g. getting one drop on the back of your lab glove and dying) and ones that you could pretty much bake into a creme brûlée without anyone noticing much of a difference. 4-MEI is quite a bit closer to the latter.

Which isn't all that unexpected, since it is one of components of caramel coloring, which has been used in a variety of food and drinks since before the Civil War. It gives color and flavor to soda, potato chips, beer, ice cream and whiskey to name a few. Doesn't really sound like much of a poison.

So if they can't convince people that a can of Pepsi is going to make you take an immediate dirt nap, the old cancer scam is a fine substitute. Cancer is really scary. And we all know from watching TV celebrities that we are just swimming in a vast ocean of carcinogens as a result of living in modern times. That explains why cancer rates are dropping every year! Wait a minute. No, it doesn't.

Here's how most things get labeled carcinogens: Rats or mice get fed ridiculous amounts of a chemical for their lifetime, and are then examined for tumors. Some of them get tumors. What this has to do with human health in not obvious. Does this chemical really cause cancer? The answer is "who knows?" These experiments have so little to do with mimicking a real life situation that they are essentially worthless.

This is not to say that there aren't real carcinogenic chemicals. Of course there are. All chemists have worked with them at one time or another and you have to be really careful. But this ain't one of them. The "evidence" of human carcinogenicity of 4-MEI is nonexistent. Yet that doesn't stop CSPI issuing the headline "Lab Tests Find Carcinogen in Regular and Diet Coke and Pepsi" today. Please.

While these guys are busy measuring barely-detectable amounts of chemicals in this and that, they are doing us all a disservice by taking attention from real risks, like smoking, drunk driving, obesity and The Real Housewives of New Jersey. Give us a break guys. We have enough to worry about.



Last week's article by Tara Parker-Pope in the New York Times on the potential benefits of total knee replacements for patients with arthritis was a welcome respite from the litany of doom and gloom stories about rising health care costs - which often ignore the health and quality of life benefits from advancing medical innovation.

Parker-Pope reports on a massive study of 135,000 patients with new diagnoses of osteoarthritis from 1997-2009. Some 54,000 patietns opted for knew replacement after their diagnosis, with the rest declning surgery. She reports that

Three years after diagnosis, the knee replacement patients had an 11 percent lower risk of heart failure. And after seven years, their risk of dying for any reason was 50 percent lower.

The study, presented this month at the annual meeting of the American Academy of Orthopedic Surgeons, was financed with a grant from a knee replacement manufacturer. It was not randomized, so it may be that these patients were healthier and more active to start with.

Still, the researchers did try to control for differences in age and overall health. And the findings are consistent with large studies of knee replacement and mortality in Scandinavia. Given the big numbers in the study and the size of the effect, the data strongly suggest that knee replacement may lead to improvements in health and longevity.

In many ways, this is unsurprising. Any kind of intense, chronic joint pain is likely to impact mobility and, consequently, negatively impact cardiovascular health and weight.

Now, the cautions to interpreting this data at face value are well taken. The study wasn't radomizzed, and people who were more likely to excercise before they had a knee replacement are also likely to be more physicially active post-operatively.

But on some level, I'm not sure it matters. Improvements in population health are always going to be skewed towards people who are more motivated to watch their weight, exercise, take their meds regularly, eat well, be non-smokers, etc. But insofar as new medical innovations help keep those people active and able as they age, those health gains (and, presumably, the lower medical costs associated wtih them) can be maintained.

I'm also curious as to whether health gains through hip or knee replacement translate into other areas: slower declines in cognitive functioning, lower rates of nursing home enrollment, etc, that may pay dividends many years after the initial joint replacement.

Answering questions like these may not only give us a better sense of the value of health care spending, but also to help us focus on promoting interventions that have the most potential to improve quality of life over the long term.


There's been a lot of press coverage over the past several weeks of the problem with prescription drug shortages. And, as you might expect, members of Congress are eager to be seen as doing something -- anything really -- to help alleviate the crisis. A bi-partisan group of House members led by Rep. John Carney (D-Del.) introduced a bill at the end of January. That came on the heels of another House bill introduce by Reps. Diana DeGette (D-Col.) and Tom Rooney (R-Fl.) in June. And Sens. Amy Klobuchar (D-Minn.) and Robert Casey (D-Penn.) recently moved to attach their drug shortage bill, first introduced last year, to a piece of transportation legislation moving through the Senate.

Unfortunately, most of the proposed action will have little to no affect on the fundamental underlying problems associated with drug shortages. And one of the bills very well may make the shortages worse.

Last week, our very own Paul Howard wrote an article for the Washington Examiner explaining how "a combination of market forces and government price controls have reduced incentives for companies to either enter the market for critical drugs or make manufacturing investments to keep their plants up to date - and running safely." Former Obama administration health policy advisor Ezekiel Emanuel delved more deeply into the price control issue in this New York Times op-ed.

The gist of the problem is that the profit margins for the generic drugs that comprise the vast majority of drugs in short supply are so small that there often are just two or three manufacturers (and occasionally just one) for a particular critical drug. And Medicare price controls contribute substantially to these profitability issues.

Health care scholar John Goodman summarized some of these and other points nicely in a post on his blog last summer. As Goodman pointed out, another contributing factor is the FDA's increasingly strict regulation of drug manufacturing facilities, which he calls a "zero tolerance regime" that is "forcing manufacturers to abide by rules that are rigid, inflexible and unforgiving." This is exacerbated by the fact that FDA not only approves drugs as safe and effective, but also regulates the production facilities themselves, including the quantities produced and the timing of production schedules. So, when one manufacturer's plant is closed, competitors need to get FDA approval before they can ramp up their own production to meet the shortfall.

Waiting for FDA to get through the approvals takes time, and that contributes to some of the drug shortages we've seen. The Carney bill would instruct FDA to expedite these reviews -- both to bring shuttered facilities back on-line more quickly and to let competitors increase production. And all three of the bills would also require drug makers to warn the FDA in advance if they decide to discontinue manufacturing a "critical" drug or if they anticipate an "interruption" in production. To some extent, these measures may help, but the FDA has taken some modest steps on its own to address some of the most problematic shortages. And nothing in the legislation is likely to change the fundamental nature of how the agency operates. Nor would any of the bills address the fundamental underlying economic considerations that are the primary cause of the shortages.

Worse still, The Carney bill would, to some degree, actually intensify the problem. It would try to stabilize prices by banning so-called "stockpiling" and "price gouging". But it's Economics 101 that, when the supply of something falls below demand, the price will rise. What every politician who complains about price gouging forgets, ignores, or just doesn't realize, is that rising prices are a signal (a) for consumers to conserve the supply of a scarce product, and (b) for manufacturers to produce more of it. That's exactly what stockpiling is, an effort to conserve a drug that's in short supply.

When prices of these drugs rise, doctors and hospitals start asking themselves, "Do we really need to use this drug in this situation, or is there a reasonable substitute?" That in turn means that the patients who most need the drugs are more likely to get them, and that patients who could make do with a lower dose or an alternative use less of the product that is scarce. No consumer likes sharply rising prices, particularly when they're seen as an effort by some sellers to profit unfairly. That's why politicians always propose these kinds of measures. But the problem with preventing so-called price gouging is that it means no signal gets sent to consumers indicating that conservation is necessary, and no signal gets sent to manufacturers that it's worth ramping up production. In short, anti-stockpiling and anti-price gouging policies almost inevitably make a shortage worse, not better.

In short, drug shortages are a real problem. But the way to alleviate them is not to eliminate the market signals that incentivize adequate production. In the end, we would be better off if Congress did nothing at all than if they enacted these bills. Better still, though, would be for Congress to lift the rules that have contributed to the shortages in the first place.



The FDA announced this week that it is adding new safety information and warnings to the labels of all statin drugs. In a nutshell, the FDA is adding new warnings that suggest that statin may slightly increase the risk of developing diabetes, and (also very rare) may lead to some transient memory problems or confusion in some patients.

Here's what the FDA says:

We want health care professionals and patients to have the most current information on the risks of statins, but also to assure them that these medications continue to provide an important health benefit of lowering cholesterol.

The irony here is that obesity is a risk for both heart disease and diabetes, and obese patients may now wonder whether or not the drug they are taking to prevent a heart attack will give them diabetes.

Still, (as the FDA says) the risk is small. And patients and doctors can now take additional steps to watch for changes in blood sugar in otherwise healthy patients after they begin statin therapy.

This is all to the good. Knowledge, after all is power.

So why is it that the FDA is so much more forthcoming about the details of potential drug risks than benefits?

The FDA's announcement underscores a fundamental imbalance between how it conveys (and allows companies to convey) risk and benefit information to the public.

If, as in this case, a meta-analysis of studies shows a likely safety problem, the FDA will add it to the label and make an announcement that (through no fault of the FDA) may lead to a rash of lawsuits against companies.

On the other hand if companies do a meta-analysis of studies on their own products and discover a benefit, they can't talk about it. In fact, they will be fined and prosecuted if they promote these "off-label" uses to physicians, even if they are true or very likely to be true.

Instead, they will have to invest many millions of dollars in new clinical trials to prove to the FDA that the "signal' from their initial studies turns out to be correct. This may take a decade or more and in the meantime patients who would have benefitted from the new use of drug that is already FDA approved will suffer - and perhaps die - because the company's hands are tied from promoting off-label use.

Of course, allowing off label promotion might encourage companies to peddle snake oil using misleading statistics or poorly designed trials. A sensible compromise would be to allow the companies to distribute non-industry sponsored, peer reviewed research on off-label use to physicians with the expectation that physicians have the knowledge and expertise to use the information appropriately.

The benefits wouldn't be certain, of course, and physicians would have to use their judgment in a case by case basis that the drugs' benefits outweighed its risks.

Isn't this exactly the situation that the FDA is offering us today, by informing us about rare statin risks?

So why not create a parallel safe harbor for describing a drug's benefits?


I have written in the past that, although pharmaceutical sales reps are usually considered to be one step above head lice on the food chain of life, they actually perform quite a valuable service in educating physicians, many of whom have absolutely no time to time to keep up with the literature on new drugs.

Little surprises me any more, but that changed last week--in a big way.

A friend of mine who has been going through some rough times decided to give drug therapy a chance. He was put on one of the standard SSRI antidepressants by a very expensive New York psychopharmacologist.

Within two weeks, he was feeling nauseated much of the time, and had lost a lot of weight, so he called me for advice. It didn't take House to figure out this one. Nausea is one of the very common symptoms of SSRI use. Fortunately, it can be controlled with Zofran, an anti-emetic drug originally developed to treat chemotherapy-induced nausea and vomiting (CINV). It did this so well that oncologists maintain that it revolutionized the field, enabling patients to complete their chemo, even with the most emetogenic drugs, such as cisplatin. And to do so with far more comfort than before.

Zofran (generic name ondansetron), one of the most important pharmaceutical discoveries in a generation, is now also used to treat nausea and vomiting caused by general anesthesia, morphine, pregnancy, viral gastroenteritis and SSRIs.

So, it took no special wisdom for me to suggest that he call his doctor and see if he would call in a script for some. Which was a perfectly fine plan until he called me back, saying that the doctor had never heard of it.

After removing my jaw from my desk, I tried to come up with any explanation of why a $400 per hour New York psychopharmacologist had never heard of a drug that had not only been on the market since 1991, but had transformed the fields of oncology, obstetrics and pediatric care.

The best I could do was "he was having a bad day." But I don't really believe that.

Being in the pharmaceutical universe for my entire career, it's not unusual that I sometimes know more about certain drugs than the doctor I'm visiting. They are generally pleased to learn something new. But this is not my job.

In the absence of any Continuing Medical Education requirement for physicians about new drugs, it becomes the job of the reps to inform physicians about new products.

This is a valuable service, and perhaps it is time that this side of the story is considered. Although this system certainly has its flaws and the potential for abuse, on the whole, I believe it is far more useful than harmful.

I told my friend to get another doctor, and this time to check whether there was actually a license behind the desk. I feel badly for the other patients. Unless there are shrunken heads on the wall, or bottles of chloroform lying around, they may not know that they are seeing someone with a frighteningly limited knowledge of modern pharmaceuticals.




Vaccines are undoubtedly the most successful medical intervention in the history of modern medicine. From smallpox to polio and measles, vaccines have managed to either eradicate or sharply reduce deaths and disability from diseases that once ravaged human communities for thousands of years.

The notable exceptions, however, are viruses like influenza (and AIDS) that mutate so frequently that the human immune system is unable to generate a lasting and effective immune response against new strains. As a result, a vaccine that works perfectly well against this years' flu strain may be useless next year.

Researchers and companies have, however, suspected for some time that it may be possible to create a universal flu vaccine that would target the deep structures of influenza, leading to a robust immune response against multiple strains simultaneously. Ideally, a universal vaccine would eliminate the need (and expense) associated with companies guessing which strains will dominate the flu season in any given year.

Recently, Princeton University researchers confirmed that - at least in theory - a universal flu vaccine could work by providing "herd immunity" to new flu strains:

On a very deep level, these viruses are all related," said Nimalan Arinaminpathy, a postdoctoral research associate in Princeton's Department of Ecology and Evolutionary Biology and the lead author of the study. "The seasonal flu vaccine just works on the surface proteins -- these vaccines would penetrate to the inner part of the virus."

The Princeton team analyzed whether the universal vaccine would protect against a widespread flu, such as the Spanish influenza of 1918, which killed an estimated 50 million people worldwide, or the recent 2009 swine flu.

The researchers determined a universal vaccine could stop such pandemics in their tracks.

"By vaccinating just a part of the population, you reduce the risk of transmission," Arinaminpathy added. "We've never been able to contemplate herd immunity with flu vaccine before."

Of course we won't know if this strategy will really work until we try it en masse.

But if it does work, it will help reduce hospital costs associated with severe flu cases and save thousands (perhaps tens of thousands) of American lives every year.

Despite the fact that vaccination is a two hundred year old technology, researchers are still just beginning to scratch the potential of vaccines to harness the human immune system to fight everything from seasonal flu to metastatic cancer.


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