Switzerland Creates Secure Test Site for GM Crops

…Swiss researchers running recent GM trials spent 78% of their research funds on security. 

That is shocking – almost 80% of scarce Swiss research funds are wasted to prevent criminals such as Greenpeace from destroying research that benefits everyone – research which especially benefiits the Bottom Billion. Well, the Swiss government is going implement a more efficient centralized security scheme at the Reckenholz research station near Zurich. The goal is remove the drain of security expenditures from research grants – distributing the cost to all taxpayers. Sadly it won’t eliminate the need for the security in the first place.  

(…) GM crops are controversial in Europe, and European law requires scientists to notify the public about the precise locations of the fields where they are running experiments. This has led to protests and sometimes vandalism at more than 100 European trials since 2010. One result is that the number of GM field experiments conducted in the European Union dropped from about 250 per year in the late 1990s to fewer than 50 in 2011, the researchers report. In Switzerland, researchers have submitted just six applications for field experiments with GM plants since the late 1990s; authorities rejected two in 1999 because “the social and environmental impacts compared to any possible economic benefits were clearly too high.”

In a bid to make such experiments easier, the Swiss Federal Council approved spending €600,000 annually from 2014 to 2017 to create a protected field site of approximately three hectares at the Reckenholz research station, 10 kilometers north of Zurich. Researchers will initially use it to test GM wheat with resistance to powdery mildew, a fungal disease, but they could ultimately plant other crops such as potatoes. 

Every attack by the masked vandals is a crime against humanity. I wonder if criminal prosecution of Greenpeace in all of the OECD would stop this nonsense? Possibly not – as Greenpeace is so successful with fund-raising based upon blocking evil GMO and evil nuclear power.

This Is Your Global Food Supply On Climate Change

I think that Steve Savage expresses the climate change connection about right. We won’t know for twenty years if the 2012 weather extremes are expressing a climate change signal. Regardless, the extremes are signaling how important it is to make the global food supply more robust under the stress of combined extremes of heat and drought.  Here’s a snippet from Dr. Savage:

OK, I’m going to go out on a limb and say that I think that this year’s climate extremes are linked to human-caused climate change.  We might not really have the definitive answer on whether that is true for 20 years, but I would like nothing better than to be proven wrong about the linkage I’m making today.  From a global food supply perspective, the effects of weather on 2012 food production is problematic no matter what its cause.  As bad as it seems, it might just be a ‘shot over the bow’ relative to what me might expect in the future. The unfilled corn cob pictured above is a relatively decent example of what the US corn crop is yielding this year.

How Hot Is It?

This isn’t just about low rainfall.  There is a recent graph about temperature extremes on the NOAA (National Oceanic and Atmospheric Agency) site that is striking. The 2012 difference from average is off the chart!


When it is both hot and dry, our dominant, rain-fed crops suffer the most.

Please do read Steve’s complete analysis.

Scott Andes: Why California’s GMO Labeling Proposition Should be Defeated

I was planing to write an article on California Proposition 37. Now I don’t need to, because Scott Andes has done the job nicely with his essay at ITIF’s Innovation Files.

To frame the discussion we need prof. Kevin Folta’s tabulation of the available methods for altering the DNA of a plant. I recommend that you read Kevin’s article before continuing. Click the image for the full-size table:

Here are the ways that plants are genetically altered.  Note that all of them are acceptable to most people, despite having no idea what the heck is being changed, and the huge number of genes affected. 

Scott Andes explains that the activists promoting GMO labeling have no scientific basis – this is nothing like Trans-fat labeling. This is about politics. As David Tribe put it, this is about the financial interests of “Big Quacka and Big Organic“. And let us not forget the Trial Lawyers, which I discussed here: California proposition 37: Trial Lawyers, Bootleggers and Baptists

Here’s selected snippets from Scott Andes’ essay: 

This November, California voters will be asked to decide whether food that has been ‘genetically modified (GM)’ should come with a special GM label.  Proponents of proposition 37, or the ‘Right to Know’ initiative, argue that ‘in a democratic, free-market society, consumers get to make informed choices about what we eat and feed our families,’ i.e., a GM label will help consumers make informed choices. Sounds simple enough. What could possibly be the downside to a small label that presumably enables greater consumer decision making?

First, labels such as this are never about education and open consumer choice, but about limiting people’s interest in/exposure to? a harmful substance. Labels are one of many public policies that aim to ‘nudge’ consumer behavior away from a product. As Richard Thaler and Cass Sunstein outline in their well-known book Nudge, consumers are fickle, uncertain, and look for cues to make decisions. Thaler and Sunstein use the example of putting fruit first in cafeteria lines. Because people irrationally fill up their trays with things at the beginning of cafeteria lines, one way to ‘nudge’ people to eat healthy is to put healthy food first. Mandatory labels do the same thing. Cigarette labels do not exist to inform people that smoking leads to lung cancer—everyone knows that—they exist to nudge a consumer to think twice before purchasing a pack. The same thing goes for other mandatory labels such as Trans fat.

The question becomes, what makes an ingredient or food processing method warrant a label?  Obviously, there are many examples of products that are sold without detailed consumer information. Take generic brands. Beyond knowing a product is ‘canned tuna’ or ‘diced tomatoes’ consumers know little about the producing company or their method of production, yet we readily allow such products because they are cheaper and we are ensured that generics undergo the same health and safety requirements as name brands. Additional identifiers on generic goods add nothing  to informed decision making so we do not require them. Therefore, arguing, ‘consumers have a right to know,’ implies there is something about GMOs that make them more like Trans-fat than generic canned tuna. So what is the distinction?

The regulatory litmus test for mandatory labeling in the United States is the health impact of an ingredient. Nutritional content labeling helps consumers evaluate, for example, the number of calories and vitamins in a product while more explicit labels help consumers avoid unhealthy ingredients. Labels containing such useful, accurate information are required by law. Under the current regulatory framework, in order to justify a GMO label, GMOs would need to have different health or nutrition implications for humans than that of conventionally grown food.

While there are many ethical debates surrounding GMOs, one corner of the debate that science rightfully owns is whether or not GMOs have a unique health portfolio. The evidence clearly shows they do not. According to the Mayo Clinic, ‘A recent study examined the past 50 years’ worth of scientific articles about the nutrient content of organic and conventional foods. The researchers concluded that organically and conventionally produced foodstuffs are comparable in their nutrient content.’ The WHO states, ‘GM foods currently available on the international market have passed risk assessments and are not likely to present risks for human health. In addition, no effects on human health have been shown as a result of the consumption of such foods by the general population in the countries where they have been approved.’ And in a literature review for congress, the GAO writes, ‘To date, GM foods have proven to be no different from their conventional counterparts with respect to nutrients, allergens, or toxicity.’

If GMOs do not differ from conventional foods in terms of nutrition then why the call for a label? In part it’s because of a public misunderstanding that genetic engineering is creating unprecedented and novel organisms. As my colleague Val Giddings has noted, genetic manipulation is commonplace throughout the food system by conventional and organic farmers. What separates traditional transgenic methods  from genetic engineering is the use of recombinant DNA (rDNA) technology—a laboratory method of coordinating genetic material from multiple sources—to  confer beneficial traits to an organism. rDNA technologies are unique in that scientists can target one specific gene and monitor its impact on an organism, unlike traditional hybridization that blends two organisms in a completely unpredictable and largely uncontrollable grab-bag process.

The vastpreponderance of scientists agree that using GE rDNA techniques actually reduces the risk of surprises or undesirable results compared to traditional methods because through rDNA  one can actually see the genetic effects of a foreign gene, while traditional methods are only able to observe the phenotype implications (what a plant looks like). (…)

(…) Marchant, Cardineau, and Redick show in their book on GMO labeling that when the predicted cost of labeling is included in the questions, consumers overwhelmingly reject mandatory labels. More importantly, the reason so many consumers support labeling is because  believe GMOs are harmful. Responsible public policy should not promote this misconception but try to correct it. When cigarette labels were first debated most consumers believed they were unnecessary because people did not understand the health consequences of smoking.  Science was further along than public opinion. Similarly, with GMOs, science is ahead of public opinion.

{snip snip}

Definitely read the whole thing. Also be sure to read Hank Campbell The Mercenary Intent Behind Proposition 37’s GM Food Labeling

California proposition 37: Trial Lawyers, Bootleggers and Baptists

“When I used to go and talk about Prop 65 when it was on the ballot, I would say the biggest beneficiaries would be lawyers. I think that goes double for Prop 37,” said Michele Corash, a environmental defense partner with Morrison & Foerster. — from The Recorder, Defense Lawyers Say Prop 37 Will Bring Bumper Crop of Litigation, 7/27/2012

Courtesy of Andrew Apel @AgBioEye, an excellent little article on the developing California proposition 37 train-wreck. Snippet: 

Proposition 37, which will be on the ballot this November, will all but guarantee payday after payday for trial lawyers in the Golden State. Also known as the Right to Know Genetically Engineering Food Act, the law would require labels on foods and beverages that include ingredients produced with biotechnology. Never mind that biotechnology can produce crops more resistant to disease or with greater yields. Oh, and they’ve been on the market for almost two decades, and there’s no credible evidence that biotech crops have produced even a case of sniffles in humans.

So who stands to benefit, other than dubious nutrition supplement peddlers? If you guessed trial lawyers, you win an organic cookie. They have already attacked food companies for using biotechnology in the past, so having the law in their favor will turn these seemingly frivolous cases into sure winners.

Never mind that biotech crops could change the world and avert the global food crisis; ignore the fact that biotech food has helped nations develop. The American Medical Association’s declaration that “there is no scientific justification for special labeling of bioengineered foods” is irrelevant to the debate, it seems. Facts don’t matter when there’s money to be made.

This should all sound familiar, because Prop 37 isn’t too many steps away from Proposition 65. It was a decade ago when we first noted the outrageous results of Prop 65—when French fries were under attack. It’s a rule that California has onlymade even worse over the years by adding more and more substances to the list.

So is it any surprise that the man who helped craft Prop 65 is leading the charge for Prop 37? Would you be any less surprised if he tried to deny the blatantly obvious similarities

Read the whole article – lots of potentially useful resources. I’ve not had time to do any checking on the article source: “Center for Consumer Freedom“. This group may be baptists fronting for other bootleggers. See Bruce Yandles Bootleggers and Baptists if you are not familiar with this economic analysis.

Some background on the other bootleggers hiding behind Prop 37. E.g.,

Quackwatch FDA Orders Dr. Joseph Mercola to Stop Illegal Claims, Mercola is biggest $$$ at $800,000. Mercola is a large anti-vaccine, organic, “natural stuff” moneymaker. They are part of the Big Organic lobby.

Station KCET is tracking prop 37 donors. The bootleggers were way in front on the funding last week (~3x the opposition who had only two small contributors). I’m happy to see some contributors coming along to oppose. The challenge here is that Prop 37 sounds harmless to people who don’t know anything about the food supply chain, and don’t appreciate the powers that are behind the sorts of propositions.

Anne Glover, EU chief scientific advisor: “No risk with GMO food”

Jeremy Fleming at Euractiv.com recently posted a remarkable interview with University of Aberdeen biologist Anne Glover – who was appointed the European Commission’s Chief Scientific Advisor Jan 1, 2012. Dr. Glover certainly does not sound like any EU official that we have heard – she is talking evidence-based policy! I will speculate that the EU politicians are not going to like listening to Glover one bit. We sure hope they do listen and that she keeps her appointment!

EXCLUSIVE: Genetically modified organisms (GMOs) are no riskier than their conventionally farmed equivalents, the European Commission’s Chief Scientific Advisor Anne Glover has told EurActiv in an exclusive interview, calling for countries impeding GMO use to be put to proof.

The endorsement of GMO safety will rattle member states where bans are in place (see background), and represents the CSA’s highest-profile policy intervention since Glover became Commission President José Manuel Barroso’s scientific advisor last December.

“There is no substantiated case of any adverse impact on human health, animal health or environmental health, so that’s pretty robust evidence, and I would be confident in saying that there is no more risk in eating GMO food than eating conventionally farmed food,” Glover told EurActiv, saying the precautionary principle no longer applies as a result.

Glover said she was not promoting GMOs, and added that “eating food is risky”, explaining: “Most of us forget that most plants are toxic, and it’s only because we cook them, or the quantity that we eat them in, that makes them suitable.”

Scarce resources

But she said that scientific evidence needed to play a stronger role in policymaking, firing a warning shot at countries that have banned GMOs. “I think we could really get somewhere in Europe if when evidence is used partially, there were an obligation on people to say why they have rejected evidence,” she said.

GMOs and other scientific advances must be explored in order to head off the increasing scarcity of energy and other resources and competition for land use, Glover suggested.

“If we are using land to produce biofuels, we are not producing food, and that that means we have to intensify food production,” she said.

Glover, a former professor of biology at the University of Aberdeen, served as chief scientific advisor for Scotland before from 2006-2011. She joined the Commission on 1 January.

Her role is to bolster scientific evidence by saying things that politicians and officials are sometimes uncomfortable with, she said, adding: “The evidence with which I work is independent, the evidence with which I work does not change according to political philosophy. And that should give people a lot of confidence.”

Glover said that discomfort around the subject of GM crops in the 1980s and 1990s was “a generation ago, we’ve moved on and the challenges are completely different”.

She said that the precautionary principle was appropriate when applied properly, but added: “We should not … somehow tie our hands behind our back in such a way that we will be so precautionary that we will wait for everyone else to use our knowledge before we use it.”

“That would be my worry, because knowledge is an international currency, and we are amongst the slowest in taking advantage of the knowledge we create, and that cannot be right.”


Please read the entire interview. Then read the comments. The vast majority are content-free Greenpeace talking points. There was one important exception I noted, by Melinda Miller 24/07/2012. She is commenting on the weak reference posted by one of the anti-GMO types:

As a Scientist and a family Farmer I am always interested whenever someone posts a research paper to defend their viewpoints. I took time to go directly to the research itself (Spiroux de Vendômois1, et al, A Comparison of the Effects of Three GM Corn Varieties on Mammalian Health, Int’l Journal of Biological Sciences, 2009; 5(7):706-726) and reviewed their findings. I thought it was striking that the Authors themselves repeatedly admitted in the paper that they did not have enough information from the study groups of rats to draw statistically relevant conclusions. Even more shocking was that while they used a control group of over 300 rats, their experimental group (being fed GM-corn) was only 80 rats and of those animals they only included 10 into their data. Why not use all 80 animals since they were already starting with a shockingly low numbered group study?

Being open-minded, I assumed that since this article is already outdated in the scientific community (3 years), I searched for more recent works by the Authors. Perhaps they have filled in some of the blanks in their research since such a simple feeding study should be easily replicated in 3 years time to include more animals? I could find none, but I did discover that Greenpeace was the original study’s funding source.
As a Scientist, it is critically important that research be done through an independent, peer-reviewed system. It maintains an objective viewpoint which reduces chances of data being used to further any specific predetermined viewpoint. I am open to looking through any other studies that I can find that meet these requirements. Unfortunately, none of the previous links provided meet those standards.

In Gene Sequencing Treatment for Leukemia, Glimpses of the Future

Second Chance: Lukas Wartman, a leukemia doctor and researcher, developed the disease himself. Facing death, his colleagues sequenced his cancer genome. The result was a totally unexpected treatment.

Thanks to Vinod Khosla who tweeted the URL for this article. This is an important case – we know that cancer is definied by its DNA and RNA – not by the invaded organ or tissue. Lukas Wartman was lucky to be at one of the top cancer institutes, Washington University, and part of a team who had the knowledge and facilities to do whole genome sequencing and analysis. The team found via his RNA sequence a rogue gene that was responsible for the high rate of cancer cell production. Wartman’s luck was further enhanced when the team identified a drug approved for kidney cancer that might shut down the malfunctioning gene.

Here is a snippet of the first paragraphs:

ST. LOUIS — Genetics researchers at Washington University, one of the world’s leading centers for work on the human genome, were devastated. Dr. Lukas Wartman, a young, talented and beloved colleague, had the very cancer he had devoted his career to studying. He was deteriorating fast. No known treatment could save him. And no one, to their knowledge, had ever investigated the complete genetic makeup of a cancer like his.

So one day last July, Dr. Timothy Ley, associate director of the university’s genome institute, summoned his team. Why not throw everything we have at seeing if we can find a rogue gene spurring Dr. Wartman’s cancer, adult acute lymphoblastic leukemia, he asked? “It’s now or never,” he recalled telling them. “We will only get one shot.”

Dr. Ley’s team tried a type of analysis that they had never done before. They fully sequenced the genes of both his cancer cells and healthy cells for comparison, and at the same time analyzed his RNA, a close chemical cousin to DNA, for clues to what his genes were doing.

The researchers on the project put other work aside for weeks, running one of the university’s 26 sequencing machines and supercomputer around the clock. And they found a culprit — a normal gene that was in overdrive, churning out huge amounts of a protein that appeared to be spurring the cancer’s growth.

Even better, there was a promising new drug that might shut down the malfunctioning gene — a drug that had been tested and approved only for advanced kidney cancer. Dr. Wartman became the first person ever to take it for leukemia.

And now, against all odds, his cancer is in remission and has been since last fall. While no one can say that Dr. Lucas is cured, after facing certain death last fall, he is alive and doing well. Dr. Wartman is a pioneer in a new approach to stopping cancer. What is important, medical researchers say, is the genes that drive a cancer, not the tissue or organ — liver or brain, bone marrow, blood or colon — where the cancer originates.

Do We Really Need More Scientists?

Please do not miss Derek Lowe’s guest post – very well-informed education commentary by an insider scientist with decades of experience in drug discovery research:

(…) There’s surely an upper bound to the proportion of students who could usefully study the hard sciences. We can argue about what that number is, but not, I think about its existence. Stipulating that, the question becomes whether we should find ways to get the smartest and hardest-working students into (or back into) these fields, which would mean dragging some of them away from business and law careers. But there’s a potential problem there, too: if money and social standing are your motivating factors, you’ve probably ruled out the sciences for those reasons alone. Now, I make a good living in the pharmaceutical industry. But my salary is pocket change to the hedge fund people. I definitely did not go into science to become rich.


My solution? Well, I agree with Tyler Cowen in The Great Stagnation that we would be well served by making science and technology jobs more prestigious (says the guy who has one), although I’ve no idea of how we would go about that. But that only helps the fields that have a labor shortage – exalting the shrinking number of medicinal chemistry jobs seems rather pointless. In the end, the best advice I have is Virginia Postrel’s view, in her column “How Art History Majors Power the U.S. Economy.” It’s futile, she says, to try to make the labor markets flow in the directions you think they should go:

The argument that public policy should herd students into Stem fields is as wrong-headed as the notion that industrial policy should drive investment into manufacturing or “green” industries. It’s just the old technocratic central planning impulse in a new guise. It misses the complexity and diversity of occupations in a modern economy, forgets the dispersed knowledge of aptitudes, preferences and job requirements that makes labor markets work, and ignores the profound uncertainty about what skills will be valuable not just next year but decades in the future.

Be light on your feet, in other words. Learn how to learn, and don’t assume that you’ve ever won some sort of lasting job security, because lasting job security isn’t something that the world’s economy is built to deliver these days. You may feel, like Evelyn Waugh’s Mr. Scott-King, that outfitting someone to survive in the modern world is a rather wicked thing to do, because this isn’t very comforting advice. But we don’t owe people comfort when the truth would serve them better.

Read the whole thing »

Virginia Postrel’s piece is also well worth a read.

SRT1720: Good (And Confusing) News for Obese Mice

An insider analysis of a new Nature Scientific Reports paper by Derek Lowe:

Readers of this blog will be fairly familiar with the long, interesting story of sirtuin activators. Today we will speak of SRT1720, of which we have spoken before. This molecule was described in 2007 as an activator of Sirt1 with beneficial effects in rodent models of diabetes. But both of those statements were called into question by a series of papers which found difficulties with both the in vitro and the in vivo results (summarized here). The GSK/Sirtris team fired back, but that paper also served as a white flag on the in vitro assay questions: there were indeed artifacts due to the fluorescent peptides used. (Another paper has since confirmed these problems and proposed an off-target mechanism).

But that GSK response didn’t address the in vivo assay questions at all – we still had a situation where one group said that these compounds (SRT1720 in particular) were beneficial, and another said that it showed no benefit and was toxic at higher doses. Adding to the controversy, another paper appeared late last year that went back to nematodes, and found the SRT1720 did not extend their lives, either. The state of this field can be fairly described, then, as “extremely confused”.

Now we have a new paper whose title gets right down to it: “SRT1720 improves survival and healthspan of obese mice”. First time I’ve seen “healthspan” as a word, I might add, and another interesting sidelight is that this appears in Nature Scientific Reports, the publishing group’s open-access experiment. But now to the data:

{snip all the meat}

[From SRT1720: Good (And Confusing) News for Obese Mice]

mINDY Mice – No Obesity, No Diabetes?

Drug research chemist Derek Lowe:

Caloric restriction increases healthy lifespan. That’s true in a range of organisms, and probably in humans. But it’s never going to be popular – and what’s more, it’s not going to be feasible, either, given how clearly people like to eat. So the search has been on for just how it exerts its effects, with a number of interesting clues turning up.

And now there’s another one. There’s a longevity gene in fruit flies known as INDY (short for, I fear, “I’m Not Dead Yet”, and if you don’t get that reference, you should probably turn in your geek license. This would be a good time to note, as required by law, that the fruit fly people are a longstanding and apparently endless fountain of weird nomenclature). Reducing INDY expression definitely lengthens lifespan in flies and in the nematode C. elegan.

A recent paper in Cell Metabolism, from a large-multicontinent team involving the Shulman group at Yale and many others, explores the effects of the mammalian homolog, mINDY, in mice. The knockout mice are smaller, although they take in the same number of calories. They are much leaner, though, with remarkable less fat. Their metabolism seems to be ramped up, as you might figure from that situation, and they’re especially good at fat oxidation in the liver. Very interestingly, they maintain this phenotype as they age, while normal mice tend to put on more fat. They have lower basal glucose and insulin levels, and are better at clearing glucose, apparently through better uptake in skeletal muscle. They also seem resistant to the bad effects of a high-fat-chow diet, show a much reduced tendency to putting on weight and developing insulin resistance. All in all, this is what you’d call a desirable metabolic phenotype, and it fits in very well with what has been worked out in the fruit flies.

So what does this gene code for? Turns out that it’s a citrate transporter, which might not be the most obvious thing at first, but it makes sense. Citrate is converted to acetylCoA, which is the building block for fatty acid synthesis. Cutting down its availability basically starves the liver tissue, which depends on fatty acids for a good part of its energy needs, and causes it to efficiently burn off whatever fatty acids it can acquire. And this effect might just be one of the things that produce the benefits of caloric restriction – in other words, you might not have to deprive your whole body of calories, just the key parts of it. To show that I’m not overinterpreting here, I’ll let the authors say it:

These data suggest that mIndy may be a key mediator of the beneficial effects of dietary energy restriction. Since prolonged caloric restriction is very difficult to achieve in humans, our observations raise the tantalizing possibility that modulating the levels or function of mIndy could lead to some of the health-promoting effects of calorie restriction, without requiring severe caloric restriction.

And as they go on to suggest, this makes for a very interesting target for obesity, diabetes, and fatty liver disease. What about extending lifespan? Well, I’ve dug through the paper several time, and can find no mention of mice older than 8 months, and no numbers on their longevity. I assume that this will be the subject of another paper as the rodents get older – it’s too big an issue to ignore, and this paper seems determined not to say a word about it.

[From mINDY Mice – No Obesity, No Diabetes?]

FDA drug approval reform: Moving to a Safety-Only System

Thanks heaps to Tyler Cowen for this heads-up on drug development. The ideas are blindingly obvious once explained. The outmoded idea behind the laborious FDA system is now known to be false (that average efficacy is relevant to an individual patient). Read this, then contact your elected representatives about reforming the FDA. One of the lives saved could be yours.

It seems likely to me that part of the problem with the current scheme is regulatory capture. Big pharma benefits by completely eliminating any small (startup) innovators from competing. That’s a typical incumbent strategy – get the regulators to erect barriers preventing new entrants.

It now costs about a billion dollars to develop a new drug which means that many potentially beneficial drugs are lost. Economist Michele Boldrin and physician S. Joushua Swamidass explain the problem and suggest a new approach:

Every drug approval requires a massive bet—so massive that only very large companies can afford it. Too many drugs become profitable only when the expected payoff is in the billions….in this high-stakes environment it is difficult to justify developing drugs for rare diseases. They simply do not make enough money to pay for their development….How many potentially good drugs are dropped in silence every year?

Finding treatments for rare disease should concern us all. And as we look closely at genetic signatures of important diseases, we find that each common disease is composed of several rare diseases that only appear the same on the outside.

Nowhere is this truer than with cancer. Every patient’s tumor is genetically unique. That means most cancer patients have in effect a rare disease that may benefit from a drug that works for only a small number of other patients.

…We can reduce the cost of the drug companies’ bet by returning the FDA to its earlier mission of ensuring safety and leaving proof of efficacy for post-approval studies and surveillance.

Harvard Neurologist Peter Lansbury made a similar argument several years ago:

There are also scientific reasons to replace Phase 3. The reasoning behind the Phase 3 requirement — that the average efficacy of a drug is relevant to an individual patient — flies in the face of what we now know about drug responsiveness. Very few drugs are effective in all individuals. In fact, most are not effective in large portions of the population, for reasons that we are just beginning to understand.

It’s much easier to get approval for drugs that are marginally effective in, say, half the population than drugs that are very effective in a small fraction of patients. This statistical barrier discourages the pharmaceutical industry from even beginning to attack diseases, such as Parkinson’s, that are likely to have several subtypes, each of which may respond to a different drug. These drugs are the underappreciated casualties of the Phase 3 requirement; they will never be developed because the risk of failure at Phase 3 is simply too great.

Boldrin and Swamidass offer another suggestion:

In exchange for this simplification, companies would sell medications at a regulated price equal to total economic cost until proven effective, after which the FDA would allow the medications to be sold at market prices. In this way, companies would face strong incentives to conduct or fund appropriate efficacy studies. A “progressive” approval system like this would give cures for rare diseases a fighting chance and substantially reduce the risks and cost of developing safe new drugs.

Instead of price regulations I have argued for more publicly paid for efficacy studies, to be produced by the NIH and other similar institutions. Third party efficacy studies would have the added benefit of being less subject to bias.

Importantly, we already have good information on what a safety-only system would look like: the off-label market. Drugs prescribed off-label have been through FDA required safety trials but not through FDA-approved efficacy trials for the prescribed use. The off-label market has its problems but it is vital to modern medicine because the cutting edge of treatment advances at a far faster rate than does the FDA (hence, a majority of cancer and AIDS prescriptions are often off-label, see my original study and this summary with Dan Klein). In the off-label market, firms are not allowed to advertise the off-label use which also gives them an incentive, above and beyond the sales and reputation incentives, to conduct further efficacy studies. A similar approach might be adopted in a safety-only system.

Addendum: Kevin Outterson at The Incidental Economist and Bill Gardner at Something Not Unlike Research offer useful comments.

[From FDA: Moving to a Safety-Only System]