If you can’t see the wood for the trees the common sense response is to do a little thinning and let the light shine through.

But for the bogged-down-in-bureaucracy European Medicines Agency (EMA), the response last week to the need to increase transparency and streamline its procedures was to set up an expert committee to investigate the activities and operations of its expert committees.

I don’t imagine this is a cynical move by the recently installed head of EMA, Guido Rasi, to kick complaints about a lack of transparency and failure to listen to the needs of patients into the long grass. He knows prevarication won’t magic the issue away.

But a committee to investigate your committees – please!

The immediate reason for the EMA to announce the formation of its scientific coordination board was its embarrassment around the rejection in April – for the third time – of Glybera, a gene therapy treatment for the ultra-rare inherited disorder lipoprotein lipase deficiency developed by Amsterdam Molecular Therapeutics Holding NV.

The EMA’s Committee for Medicinal Products for Human Use (CHMP) ruled that ‑ although it had no safety concerns ‑ the small number of patients who took part in trials meant the data file did not demonstrate that Glybera is effective in limiting the acute attacks of pancreatitis that are the most insidious manifestation of the disorder.

The CHMP exercised its prerogative despite that fact that two other EMA expert bodies, the Committee for Advanced Therapies and the Scientific Advisory Group, recommended Glybera be approved under exceptional circumstances.

Rasi previously told BioWorld it was not surprising that EMA committees had reached differing conclusions, since they are independent of each other. Well maybe not, but there’s little point in preserving the independence of your experts, if the result is to render your organization sclerotic. The EMA needs to function.

What makes the case even worse is that the CHMP members who were at the meeting to consider Glybera voted 16-15 in favor of its approval. Unfortunately, it requires 17 votes for a product to be recommended for approval.

Even without knowing which way the missing 32^nd member of the CHMP might have cast her or his vote, it is clear a majority of expert opinion believes Glybera should be allowed on the market and given an opportunity to build its case.

The many levels at which the shambolic handling of Glybera is reverberating are eloquently expressed in a letter that Alastair Kent, director of the UK charity The Genetic Alliance, wrote to the head of the European Commission’s Directorate for Health and Consumers protesting at the CHMP’s decision.

If there is a single good reason – beyond the procedural – why Glybera should not be approved under exceptional circumstances to allow efficacy data to accumulate, the EMA should say what it is.

Now that would be transparency.

It wasn’t exactly love at first sight back in 1976 when Genentech Inc. became of legal age to go forth and incorporate, but the platonic relationship between biotech and pharma has increasingly mellowed over the ensuing 36 years into an intimate affair. The recent blood transfusion/exit strategy weddings that are rejuvenating the bones of the old molecule money pharmas and fulfilling the dreams of enterprising young biotechs seeking to become the nouveau riche certainly represent something more than just a passing fling. That became patently observable when Roche Inc. finally put a ring on it and took Genentech off the market – literally and metaphorically.

Arguably, the key dynamic in the drug development market is the marriage of convenience between the pharmaceutical and biotechnology industries.

OK, they are shacking up! They’re consummating the deal like rabbits! And that’s about as subtle as I can be without descending into pornographic allusion!

Big Pharma is now Bio Pharma. They’ve been discreetly dating for some time now, but may have thrown many of you off the trail with their entertaining diversions in order to attempt to keep their affair somewhat under wraps. But the fact is that pharma and biotech are intent on producing offspring. In fact, they are planning a brood of May-December biopharma marriages that they hope will translate into a family of drugs that will be delivered into the world by the doctors at the FDA and regulatory agencies worldwide, beginning in 2012.

Canoodling @BIO

You may have seen the two of them canoodling in one-on-ones at the annual Biotechnology Industry Organization’s meetings or overheard them playing cute on boardroom phone lines late into the night. Perhaps you’ve witnessed some of the play-acting they have put on display in all-too public spats over who was going to move in with whom, as they postured for the advantage in the pre-nup merger negotiations.

Usually, when we are initially apprised of the news of a hostile takeover and the biotech’s parent board balking at the size of the pharma dowry, we assume it’s just the bluster of the mating dance and not the beginning of the breakup. So now, we just wait for the ultimate invitation to the merger ceremony.

Perfect storms can create frenzied Black Holes or dazzling landscapes, but either end result is usually borne of chaos. Think economic turmoil, patent cliffs, regulatory irregularity, R&D senior moments, failed partnerships, etc. But usually, just like in personal relationships, money issues tend to be at once, the lure that initially attracts opposites, as well as the irreconcilable difference that often renders R&D agendas or companies asunder. No matter how it ends, biopharma’s perfect storm is upon us.

The “P” Word is Becoming Offensive

How long into this decade will it be before we see pharma drop all pretenses and declare its affection for the much younger industry by going all BioPfizer, Merck & BioCo., or Johnson & BioJohnson?

The oft-married GlaxoSmithKline plc has declared its intention to shed its small molecule wardrobe and dress itself up in large molecule hoodie and skinny jeans and hang out more as a “biotech-like” company, while Merck & Co. now sees itself as “a global biopharma leader,” after dropping a few corporate pounds and getting a life sciences facelift.

The former Sanofi-Aventis, which changes its last name more times than P. Diddy or Elizabeth Taylor, has just deleted its post-hyphen once again – for what? Perhaps in preparation of patently betrothing itself to biotech as “Sanofi-Biopharma, Sanofi-Amgen …?”

So, you begin to see that it’s too late to deny the biopharma relationship is passed the “we’re just friends” stage,” as the two smitten markets continue to moving in together on Wall Street.

So, we get a lot of adorable misdirection, designed to keep the markets’ hearts beating in rhythm and to minimize investor arrhythmia, but the fact is that big pharma is shedding its small molecule skin and evolving into the hybrid entity that will characterize the drug company of the 21st century.

It promises to be a decade-long dual bash: big pharma’s retirement get-together is also biotech’s engagement party.

Editor’s note: Since Dr. Breindl first wrote about vaccines and autism in 2008, the paper linking the MMR vaccine to autism has been retracted by the journal that published it, and its author Andrew Wakefield has lost his medical license. But vaccine skepticism is alive and well – and so, during this World Immunization Week, the question remains as pressing as ever: How do you have a productive discussion on policy with people who disagree with you on the facts?

I am not a vaccine skeptic. My children have all their required vaccines and some optional ones as well. We get flu shots – heck, I’m even up to date on my tetanus shots. How many of you can say that for yourself?

The reason I’m not a vaccine skeptic is because of the data. I’m not going to list the studies again because it’s a lot like global warming, or evolution: if you don’t believe it by now, it will take something other than my going over the research one more time to change your mind.

So it’s that “something other” that I want to talk about.

Vaccine Experts: The Role of Listening and Empathy

At the American Association for the Advancement of Science’s (AAAS) annual meeting in 2008, there was a panel on “helping the public understand when scientific information is valid” that included a presentation on vaccines by Anne Schuchat, director of the National Center for Immunization and Respiratory Diseases.

The talk came under the somewhat ominous heading of “Vaccines: Truth or Consequences.” But Schuchat’s message was much more nuanced than that title might lead one to assume.

“Truth calls to mind, in my mind, humility . . . truth evolves,” she told the audience. “There’s a lot more than truth or consequences to communicating risk.”

One of Schuchat’s most interesting points was that what makes people trust expert advice depends on the situation. In low-concern settings, expertise accounts for 85 percent of the credibility that someone weighing in on an issue has. In high-concern settings, that percentage dwindles down to a measly (as it were) 15 percent to 20 percent. The most important factor? Listening skills and empathy, which account for fully half of an expert’s credibility in a high-concern situation.

Such empathy is still not really the strong suit of doctors and researchers; often, their thinly veiled assumption is that parents who don’t vaccinate their kids, or who struggle mightily with what seems like a no-brainer (a term just brimming with empathy and respect) to those in the pro-vaccine camp, are unable to understand the quality of the science supporting the opposing views.

One small study showing a possible link between a vaccine and autism, the pro-vaccine thinking goes, gets the same weight as five larger ones showing none; long-debunked associations keep getting trumpeted, and skepticism comes full circle and turns into extreme gullibility for self-proclaimed vaccine authorities.

I’m sure that happens. But by and large, it hasn’t been what I’ve heard about.

Getting to the Point of Trust

The vaccine-skeptic parents I know understand and believe the epidemiology results fine, as far as they go. But here’s what an acquaintance of mine who has an autistic daughter had to say about vaccines and autism:

“I go to a special program for autistic kids every single day. It takes me one hour to get there and one hour back. I am there for 4 hours. In addition, I go to a clinic where my daughter gets more therapy. I have heard (and live) a million ‘autism’ stories. It is more than just a 10-minute Oprah news bite to me. . . . Do I think vaccines are evil? No. But I have seen the videotapes of real life people . . . kids who spoke 50 words clearly and then suddenly lost that ability days after getting a vaccination. Can I really fall in line with the Centers for Disease Control and Prevention and call that coincidence? No. I just can’t.”

These personal observations combine with an awareness of the fact that genome-wide association studies are uncovering genetic risk factors for diseases literally by the dozen. My acquaintance says that “there is a yet-to-be-discovered link – maybe a genetic predisposition triggered by vaccines . . . I can’t put 100 percent of my trust in an agency to say there is no link when I am not convinced that they have exhausted the possibilities.”

Vaccines and Autism: New Research

Up until last year, my only question about objections like these was how to get people to understand the difference between anecdotes and data. But, to quote Schuchat again, “It is daunting to try to be right in a way that will pass the test of time.” Recent research into the immune system of autistic individuals has led me to truly believe for the first time that these people might be seeing things that I am missing.

When I was in college and graduate school, the dominant view was that the nervous and immune systems are separated by a Chinese Wall. But newer research suggests that wall is as leaky as the one that supposedly separated analysts from bankers in the heyday of the dotcom boom.

At the 2007 annual meeting of the Society for Neuroscience, one of the press conferences was dedicated to “The New Neuroimmunology: Immune Proteins in Synapse Formation, Plasticity and Repair.” It highlighted the many ways in which proteins do double duty in the immune and nervous systems.

And a recent review paper, titled “The immune response in autism: a new frontier for autism research,” lists a veritable who’s who of the immune system cells and molecules that may develop or function differently in autistic individuals, including T cells, monocytes/macrophages, natural killer cells, cytokines, neurokines and immunoglobulin.

At the neuroscience press conference, I asked a researcher whose lab is on the forefront of research whether these newly discovered links between the nervous and immune systems could mean that parents who swear their autistic children had a bad reaction to a vaccine were seeing something other than temporal coincidence, and if so, why such a link was not showing up in the epidemiology data.

She made it very clear that her data don’t address this question directly; for now, in fact, there’s no data on whether autistics are any different from neurotypical individuals in the area she studies.

But, she said, it would be consistent with what we know that autistic children could have underlying immune system abnormalities, and odd responses to vaccines due to those abnormalities. No cause and effect, but two different symptoms of the same molecular problem.

What We Know and Don’t Know

So, do I believe that vaccines are more dangerous than we think? No. Most autistic individuals have perfectly normal immune systems. Even for those who don’t, none of the changes I’ve listed above provides an obvious mechanism for how autistic children might be especially sensitive to vaccines, and the epidemiology data are strong. And so I believe that there is no association between vaccines and autism, or vaccine preservatives and autism. And I believe that any additional data we might have a decade from now will continue to support my current opinion.

But I also believe something else: that we don’t know everything.

“We don’t know everything” is one of those things that’s easy to pay lip service to and harder to keep in mind when faced with actual opinions that are different from our own, which are of course, as the saying goes on National Public Radio, always insightful and well-reasoned. But if we want vaccine-skeptic parents to listen to our evidence, we’re going to have to say it like we mean it. And we’re going to have to admit – most importantly, to ourselves – that people may disagree with us for other reasons than a pigheaded refusal to understand the data.

Only 0.3 percent of American children receive no vaccinations at all, which suggests that the great majority of vaccine-skeptic parents put careful thought and evaluation into which vaccines to give their children; they are open to our best evidence. But as Schuchat put it at the AAAS meeting, “You cannot hear statistics until you have trust.”

Amylin Pharmaceuticals Inc.’s board dodged a dissident shareholder bullet three years ago, but the San Diego-based biotech is back in the crosshairs of billionaire investor and activist shareholder Carl Icahn, who again is seeking to nominate a new slate of board members to pull the trigger on a company sale.

Given Icahn’s personal track record in the sector over the past few years – ImClone Systems Inc., MedImmune Inc. and Genzyme Corp. all landed acquisitions, though he was unsuccessful in facilitating a sale of Biogen Idec Inc. – Amylin’s days as an independent firm could be numbered.

Certainly there are arguments in favor of Amylin’s sale, most notably last year’s exit of exenatide partner Eli Lilly and Co., which left the company pitting its modest sales force against larger and more established teams from competing firms Novo Nordisk A/S and Sanofi SA. A big pharma acquirer also could help grow the markets for Byetta and recently approved Bydureon faster than Amylin on its own, and even get metreleptin, under review for lipodystrophy, off to a strong launch.

I can’t help but wonder, though, whether selling a biotech like Amylin – one which has a growing revenue stream from marketed products, with more to come – is really the best thing for the industry. Sure, investors such as Icahn will pocket substantial profits, some of which might actually go back into earlier-stage drug development ventures. But are those near-term profits worth eliminating the chance to build long-term shareholder value?

And, on a perhaps admittedly idealistic note, shouldn’t it be acceptable to ask investors for a little more patience, given the time frames in biotech – where it takes more than a decade to get from discovery to market – as well as the industry’s spirit of risk, innovation and entrepreneurship? After all, biotech is tackling complex biological discoveries, not building the proverbial better mouse traps.

There’s already talk of the U.S. life sciences industry facing a decline – government funding and venture capital have been trending downward – compared to other regions and emerging markets. It seems a no-brainer that the sector would be in a better position to thrive if more firms are allowed the time to truly build shareholder value. But that won’t happen if Icahn and his ilk keep trading that long-term value for cash to pad their already immense bank accounts.

Will Icahn’s involvement in Amylin end up in the company’s sale? Check out BioWorld’s informal poll at www.bioworld.com

I’m seeing lots of references to BioWorld’s recent analysis by Cynthia Robbins-Roth showing that U.S.-based, VC-backed biotechs raised $391 million in the first quarter, a 34 percent increase compared to the same period last year.

That’s good news, especially given the recent debates about the health of biotech venture and funding for innovation. But it’s interesting to note that if you broaden your view to include not just U.S., but global biotech fundraising, and not just venture-backed deals but all non-partnering money raised by private companies – you see that the $774 million raised in Q1 2012 represents a slight dip from the $845 million raised in Q1 2011 and the $974 million raised in Q4 2011.

What does that mean? My guess is these minor quarter-to-quarter fluctuations don’t mean much. But one interesting point: in 2011 we saw some really big non-venture deals like Pro Bono Bio‘s $600 million from government-owned Rusnano, H3 Biomedicine‘s $200 million round from Eisai Co. Ltd., and Ascletis Inc.’s $100 million mainly from Chinese entrepreneurs. We didn’t see any big deals like that in Q1 2012 – the closest was Supernus Pharmaceuticals Inc.’s $42 million raised through a combination of royalty monetization and venture debt. Most of the private company fundraising so far this year has indeed come from VCs. It will be interesting to see if the non-VC private fundraising component picks up later this year or if the new creative VC partnerships (with folks like Rusnano or big pharma) are the way of the future.

Also interesting to note that public company financing dipped more than private company financing in the first quarter of the year (vs. the same period last year). Biotechs raised $2.5 billion via public offerings and $1.88 billion via public alternatives (PIPEs, registered directs, loans, etc) in Q12012 vs. $3.2 billion and $2.07 billion, respectively, in Q12011.

Annette Grimaldi, managing director at BMO Capital Markets, said there’s no reason to panic about these dips either. She noted that as public offering terms have improved, companies have been less interested in the alternatives, and as a slew of biotechs covered their financing overhangs in late 2011, there’s now less need in 2012. For all the details, check out this week’s BioWorld Insight . . .

The contenders for becoming the April Fool in Life Sciences for 2012 came down to three candidates, but two of them have persisted past the April 1 deadline, thereby removing themselves from consideration.

President Barack Obama still has a glimmer of hope for preserving his Affordable Health Care for Americans Act and even if that is struck down, he can still overcome his supreme pranking with a wily November trick-up-his-sleeve snappy comeback. And Illumina Inc.’s stockholders continue to resist drinking Roche’s undervalued Kool-Aid unless they add more sugar to sweeten to Illumina’s taste. So, to the would-be biosimilars drugmakers – with no prospect of launching a biosimilars market for yet another year – April Fools’ greetings: you are the winners!

U.S.-based drugmakers hoping to produce biosimilar therapeutics were fooled into thinking they could be in the bio-follow-on business in 2012, but lo and behold, they are only one year deeper into a potentially years-long wait for a plan to actually be implemented that would allow for sales to commence. The joke’s on them – again!

After more than a decade and counting, the brand makers are still the masters of pranking.

It’s Not Rocket Science – It’s Even More Complex:

It’s Biosimilars!

Biotech has finally met its match. After revolutionizing drug development; after staring down unmet and underserved diseases; after outlasting the raging economy; after overtaking pharma in the clinic; after towing the FDA into the 21st century, the superbio-market has met its Kryptonite. Evidently, biosimilar production is just too complicated for the innovative industry in the U.S.

Doctors want them, patients demand them, the Obama administration mandates them, pharmacies fancy them and insurers covet them.

But the market pathway is apparently unsolvable, at least in the U.S. It’s War and Peace mixed with quantum physics, written in Yiddish and with the audio book narrated by the Fran Capo, the world’s fastest talking woman!

Don’t mess with the brand lobby. They may not yet be as adept as the oil lobby is in keeping the biofuels market down to fumes and fuming for a century, but they’re off to a good start. It has been more than a decade since this endeavor began in bright-eyed earnest and the brand-makers are still enjoying unchallenged market access to their exclusive reigns by perpetuating the work-in-progress status of biosimilars in its largest market country.

Biosimilar legislation was passed in the European Union (EU) in 2005 and since 2006 there have been 18 biosimilars approvals by that agency. Why, just a year between legislation and production – can you believe such impetuousness? Why, millions must be dying from recklessly putting such sketchily investigated drugs on the market!

For Puck’s sake, there is a template! By 2009, at least 15 countries, the EU and the World Health Organization had drafted final biosimilar guidelines ahead of the U.S. Canada, not exactly known for being cocksure, approved its first biosimilar (Omnitorpe) in 2009 – wait for it – in advance of final guidance for the regulatory pathway. That decision was based on its regulators’ assessment that they had existing authority, under Canada’s food and drug regulations, to approve biosimilars established on an abridged set of data than was submitted for the brand product, without the need for new regulations. What a bold concept!

I checked with BioWorld’s Washington editor, Mari Serebrov, who has been following the regulatory hoopla closely. She pointed out that “In many ways, the world is not waiting for the FDA to lead the way.”

Following such examples doesn’t guarantee safe and effective biosimilars in the U.S., but it does offer evidence that transitioning from biosimilar bureaucracy to actual commercialization isn’t going to result in an apocalypse of therapeutic proportions, either.

Even after establishment of a biosimilars market in the U.S. becomes a reality, the joke will still likely be on applicable drugmakers, inasmuch as the U.S. is leaning toward the market version that would require a full schedule of preclinical and clinical trials, which could make biosimilar drug development as expensive and laborious as the brand R&D schedule. That would make it difficult to offer deeply discounted biosimilars.

Legislation has passed and now guidelines have been issued, but U.S. biosimilar regulations are still an unfinished symphony and production is still a pipeline dream. The FDA is creating such a fazing series of clinical hoops for biosimilar producers to jump through, that it stands to constrain the number of companies that are moneyed enough to play the game and also raises the R&D bar for clinicians, while increasing the cost that patients will have to pay to compensate for the pricey clinical trials.

One of the elements of the Act was to facilitate lower-cost versions of expensive biotech drugs, but the eventual savings may not be as deep or come as quickly as hoped. Healthcare providers and other industry authorities say regulatory delays and the possibility of significant patent litigation could thwart the takeoff of the biosimilars market through most of this decade and wind up saving patients as little as10 percent to 20 percent off the cost of brand biotech drugs, compared to savings of as much a 90 percent on generic pharmaceutical drugs.

The big finale prank-in-waiting, according to Mari’s insight, could send the biosimilars movement backpedaling to square one, inasmuch as if the Supreme Court throws out the entire healthcare bill, the biosimilars snail express would be discarded in the mix, since the AHCA granted the FDA the authority to create the biosimilar pathway in the first place. Healthcare reform bill repeal would render the biosimilars guidance nonexistent as well as rendering the biosimilars developers fooled twice in the same year!

One small step for biosimilars; one giant extension for brandmakers.

Though we have only biosimilar path guidance in 2012, hope abounds for 2013 – but no promises. When biosimilars are eventually approved, they’d better be under a perfect system, as that will be the only way to justify its long and winding road. Meanwhile, biosimilar lobbyists and drugmakers, you are the 2012 April Bio-Fools – again – for the 10^th time! And your names are already on the list for the 2013 competition.

This week, researchers are presenting their latest and greatest at the annual meeting of the American Association of Cancer Research. The excitement of all those novel findings, though, received something of an advance puncturing last week by a commentary in Nature that reported a successful replication rate of just over 10 percent for roughly 50 landmark studies in cancer research.

From my perspective, the study makes it even more difficult to figure out which of the many research findings that (briefly) come to my attention every day to write about, and which to ignore. Is this study more interesting, or that one? Well, uh . . . can either of them be replicated?

Authors Glenn Begley and Lee Ellis stressed that they were not out to pillory individual scientists. Indeed, the sheer magnitude of the problem indicates that this is a systemic issue that needs a systemic fix – because journals and funders are valuing perfect stories over accurate data.

In the meantime, though, one of the implications of the study is that far from being riddled with conflicts of interest, preclinical results reported by industry, may be more reliable than those coming out of academia.

This has nothing to do with who’s the better person. Or the better scientist. It has to do with timing. For an academic scientist, a paper in a high-impact journal is a happy ending, the culmination of a lot of effort. For an industry scientist, that paper is also a success – but it is the successful beginning of what could turn into a billion-dollar development effort.

This is not a new view among industry scientists. Several years ago, at a meeting, a friend from a large biotech company and I sat and listened to a speaker hold forth on recent findings that the rate of positive results was higher in papers that come out of industry.

The speaker’s point was that if industry’s rate of positive findings was higher, that was indicative of data massaging. My friend wasn’t buying, though. Instead, she rolled her eyes and snapped, “Well, maybe we run better studies!”

Before industry starts patting itself on the back too hard, let me point out that I am talking specifically about preclinical data. In industry, the problem of selective reporting gets shifted to later in the process – where you can find plenty of stories of whole arms of clinical trials suddenly disappearing from clinical trial reporting and the like.

And so, my point is not that it’s academia’s turn to wear the dunce cap. Companies want to develop good medicines, but they also want to – and need to – be economically viable. Academics want to do good research, but they also want to – and need to – be professionally viable by publishing in good journals and getting grants. Everybody has multiple goals, and the better our system of research manages to align those goals, the better the science that our drugs are based on will be – no matter where that science is coming from.

The article I wrote last week for FierceBiotech on the relationship between Series A funding and innovation led to an interesting discussion with Bruce Booth, partner at Atlas Venture and biotech blogger extraordinaire.

The crux of my article was this: We (as an industry) obsess over funding for innovative start-ups because we worry that one day, big pharma will look around and realize they’ve run out of assets to in-license. So we need to look not only at the amount of Series A funding biotech raise, but at which biotechs are raising it, and whether they are really doing innovative work.

BioWorld columnist Cynthia Robbins-Roth addressed this in an analysis showing that while the percentage of Series A money going into so-called specialty pharma firms rose from 29 percent in 2010 to 42 percent in 2011, it is still down significantly from 70 percent in 2006. That would seem to indicate that, on the whole, VCs are investing in more innovative start-ups now than they were five years ago.

But as I point out in the article, the definition of specialty pharma is nebulous, at best. Robbins-Roth generally included firms that are reformulating an existing compound and those that licensed an existing compound from someone else. Her examples included Civitas Therapeutics Inc., PanOptica Inc., Ultragenyx Pharmaceutical Inc. and others. But Booth argued startups that in-license assets targeting novel, unprecedented mechanisms shouldn’t be bucketed as “low risk” specialty pharma. For instance, he said Atlas’ Arteaus Therapeutics LLC shouldn’t be classified as specialty pharma, even though it in-licensed its lead antibody, because that antibody is first-in-class.

I can see his point. If a biotech is developing an asset likely to attract a big pharma deal and have a significant impact on patients, does the fact that they did not discover it in house make them specialty pharma? What is specialty pharma, anyway?

Booth posed the question to the twitterverse, and got a wide range of opinions on the line between biotech and specialty pharma:

• @JasonCRG I believe it’s the specialty disease that defines
• @adamfeuerstein Think of “biotech” as a state of mind… as in “Pharma? … We don’t need no stinking Pharma!”
• @matthewherper “Biotech” should refer to all biological technology, really. Broad definition=good.
• @AkikoaCom It’s like the Emperor’s clothes − in the end it’s the products that count and not the ‘gown’
• @dbsable no consensus definition of “spec pharma” vs “biotech” Spec pharma: SG&A > R&D. Biotech: R&D > SG&A.
• @natesadeghi Biotech often means some sort of patent estate (real or not), spec pharma = Para 4s inbound, pharma = dividend.
• @Bobmorevc All part of a spectrum. Match great teams with products at stage appropriate for them. Biotech and Spec Pharm are loose terms.
• @InVivoBlogChris ‘spec pharm’ has lost any real meaning. Too many different definitions to be at all useful or descriptive any more.

Booth concluded that while back in 2004 to 2006, specialty pharma was often used to refer to companies that were looking to decrease molecule risk by reformulating an existing compound, the definition has “worn out its use in the world.”

But whatever you call it, when we debate whether or not we are funding enough innovation to support the future of the drug industry, is it useful to separate companies working with a known, already-approved chemical entity from those that aren’t? Or those that are doing research from those that are doing development?

I’m interested in your thoughts . . .

*Special thanks to @LifeSciVC for helping gather tweets and for starting the debate!

What a difference two weeks makes. When I was researching last week’s BioWorld Insight cover – about how emerging biotech Verastem Inc., mature biotech Ironwood Pharmaceuticals Inc. and specialty pharma firm Clovis Oncology Inc. shared some surprising similarities in how they approached their initial public offerings – I put together a chart that showed aftermarket performance for the entire 2010-2012 biotech IPO class was about flat.

I was all ready to blog about how that’s better than I would have guessed, given the negative press we tend to hear about IPOs and the market in general. Then Monday morning brought implosions of two biotechs on my list: Anthera Pharmaceuticals Inc., which halted a Phase III trial of acute coronary syndrome drug varespladib for futility, and Tranzyme Pharma Inc., which saw ulimorelin fail a Phase III gastrointestinal recovery trial.

I guess those are the breaks in biotech; wait a day to post your blog and everything changes. So I reanalyzed the chart, and guess what? Now the 2010-2012 biotech IPO class is trading down about 5 percent, and honestly, that’s still not as bad as I expected it would be.

Some other interesting findings from the chart:

• The average (mean) share price at IPO was $7.30, and the average (mean) gross was $59.94. The median share price was $7 and the median gross was $50 million (@LifeSciVc, correct me if I screwed this up). Again, not as bad as I had expected.
• There are 12 firms trading up by an average of 37 percent. The 11 firms trading down are down by an average of 51 percent. So the ones that have fallen have fallen harder. Does this indicate that buysiders who choose wisely could actually be doing quite well on biotech IPOs?
• The last five biotechs to price are all trading up. I would wonder if that means they have learned their lesson and set pricing expectations lower to allow them to trade up, but in fact three of them priced above the average. I think that’s a good sign.

Anyone else see anything interesting in this chart? Make of it what you will. . .

Reading last weekend’s Wall Street Journal review of “The Forever Fix: Gene Therapy and the Boy Who Saved It” I was struck by an anecdote. It’s about an interview the reviewer did with a scientist who works in the field of neuroprostheses, and that scientist’s refusal to talk about the possible practical applications of his work, because, he said, “false hope is a sinful thing.”

Really? To me, it seems like an inevitable part of hope is that it might be false.

To illustrate, I don’t hope that my neighbors will be nice to me, because it’s a sure thing. They are always nice. They are the best neighbors anyone could have. They have been unfailingly nice for years now. If I were still “hoping” that they will be nice the next time I see them, it would be as good a sign as any that it’s time to think about anti-anxiety meds.

Likewise, I don’t hope that my children will be well nourished. I have the money to buy them good food, the time and inclination to cook it, and the mean-mommy genes to make them eat it. Most nights, there’s going to be a healthy dinner, and hope has nothing to do with it.

Instead, what I hope is that my neighbors will live long and healthy lives, right next door. I hope that in feeding my kids well, I will lay a foundation for them to do the same thing for themselves once they are responsible for their own meals. Because those things might not come to pass.

Now, I understand the concept of a long shot. I understand it particularly well for neuroprostheses, which were once the subject of my own dissertation research.

That research may or may not end up ever benefiting a patient. But that was certainly what I hoped back then, and still do.

I mean, really. What else would I be hoping for as the long-term result of my work? That I might one day be able to remote control the family hamster? Of course it’s about hope for patients. I still feel that hope every time I read that another neuroprosthetic advance has been made.

Reviewer Carl Zimmer puts the quote, and the book he is reviewing, into context by using it to illustrate what analyst Jackie Fenn has termed the Hype Cycle. That cycle consists, basically, of inflated expectations of new technologies, followed by a crash when they have their inevitable setbacks, followed by a long slow slog to get the technology to do what it realistically can.

I agree with Zimmer and his unnamed neuroprosthesis researcher that there has got to be a better way of reporting about new technologies that are in the “gee whiz” part of the hype cycle, before they crash down into the “oh crap” stage. Rather than hyperbole followed by angry denunciations, those technologies need a balanced look, right from the beginning.

When writing about spectacular, but preliminary findings, it’s worth remembering, and conveying to readers, the spirit of a slide I saw at last year’s American Society of Clinical Oncology meeting: “If you’re a mouse with cancer, it’s been nothing but good news since 1965!”

But it’s also worth remembering another quote: “Hope springs eternal in the human breast.” That’s true – and as I’ve written about before, it means that if science refuses to give patients hope, they will go looking elsewhere. And leaving those who need hope to the ministrations of the quacks who are only too happy to recommend their own “cures,” with none of the reservations that most scientists have, seems like – well, like the greater sin.