The Cross-Border Biotech Blog

Biotechnology, Health and Business in Canada, the United States and Worldwide

Category Archives: Trends in 2009

Biotech Trends Update: ChemGenex and the Importance of Companion Diagnostic Development

Australian cancer drug developer ChemGenex was scolded by the FDA’s oncology panel for “fairly sloppy drug development.” The company’s mistake? It presented its leukemia drug, designed for patients with a particular genetic mutation, without a validated diagnostic test for the mutation.

ChemGenex says it’s a matter of months, not years, before it gets a test validated for Omapro; but the company’s stock took a beating on news of the FDA’s decision, losing 37% of its value on record volume.

Many companies, unwilling to risk such delays, have already shifted to a joint Dx/Rx co-development model.  Until it was bought by Qiagen, DxS was making an entire business out of developing companion diagnostics in partnership with pharmaceutical companies.  Similarly, Dako Denmark is developing companion diagnostics for AstraZeneca’s oncology pipeline.

The FDA’s message here is pretty clear: the quality of companion diagnostics matters.  One day, when everyone’s genome is fully sequenced, many personalized products won’t even need diagnostics.  Until that day, when you’re developing a personalized product, don’t give the diagnostic short shrift.

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Subsequent Entry Biologics (aka Biosimilars) get Final Health Canada Guidance, 6 Years of Data Exclusivity

Health Canada released the finalized version of its Guidance Document for “Subsequent Entry Biologics” (SEBs).  The final version is mostly the same as the draft guidance released last March, and actually comes after the approval of Canada’s first SEB last April.

SEBs are a class of drugs that the EU calls “biosimilars” and the U.S. calls “a class of biologics we may recognize one day if health reform passes”“follow-on biologics,” but the gist is that they are copycat versions of existing large-molecule drugs.  Because of their complexity, different versions of the same biologic cannot be characterized as identical.  Hence “similar”.

Regulators are seeking a balance with respect to how much biosimilars can rely on data from the original (“reference”) drug in applying for their own approval, and with respect to how long to protect the reference drug’s data.  Here are some highlights of Health Canada’s approach:

  • A full New Drug Submission required for SEBs, (not an abbreviated submission, as for small-moelcule generics).
  • The data exclusivity period — the time that must elapse before an SEB can use the data from the reference biologic’s application — is 6 years.
  • Studies comparing the reference biologic to the SEB must be conducted in a side-by-side format.

For more information, check out the Ogilvy Renault Pharma in Brief publication, or read the whole Guidance Document.

Biotech Trends Update — Personalized Medicine: A Big Market, If We Can Just Figure Out How to Get People to Use It

Late last year, a PwC report made the rounds with a big headline number — $232 billion — as the size of the personalized medicine market.  FierceBiotech called it a “tipping point,” for personalized medicine.  George Church called us “the first genomic generation” in Newsweek, and Francis Collins’ new book “offers practical advice on how to utilize these discoveries for you and your family’s current and future health and well-being” (at least according to its publisher).

And this isn’t just idle speculation, it’s being reflected in real investments. Cancer Research UK, the Medical Research Council, University College London, and the Wellcome Trust are developing a £500 million new home for their partnership, called the UK Centre for Medical Research and Innovation (UKCMRI), where “genomic technologies will play a key role in the array of research its partners plan to pursue there.”

However, there are real challenges to realizing the 11% annual growth rate PwC predicts.

  1. Health care providers need to learn a whole new language and a whole new set of tools and approaches.  A new year-long project at Valparaiso University aims to meet the new criteria of the nursing curriculum essentials in genetics that are set by the American Association of Colleges of Nursing (AACN), but this is just the tip of the iceberg. (h/t @mikesgene)
  2. Even when health care providers are educated, it doesn’t mean that the market will grow.  For example, there is high awareness (80-90%) of a new genetic test designed to reveal a breast cancer patient’s sensitivity to tamoxifen.  However, according to research from Duke University Medical Center, “[a] greater awareness of the emerging data for this new test corresponded to less likelihood of ordering the test and lower likelihood of changing practice based on test results.” (emphasis added) (h/t @DukeIGSP)
  3. The Genetic Information Nondiscrimiation Act loopholes are still intimidating.  GINA does not expressly cover long-term care and other types of insurance and is focused to some extent on prohibitions on requiring genetic tests (which will be moot when everyone’s full genome is sequenced).  Some efforts to remedy or mitigate GINA loopholes are underway, including:

    However, many patients (and, anecdotally, everyone in the insurance industry) are vociferously refusing genetic testing and sequencing.

  4. FierceBiotech notes that the PwC report itself identifies another caveat: “Big Pharma will have to bury its blockbuster business model in favor of a more “collaborative model.”

My bottom line:  Those who are counting on seeing the growth predicted by PwC will have to make an unprecedented investment in educational and regulatory changes to sychronize with the unquestionably giant strides in product innovation that are occuring daily.

For more on personalized medicine, check out the Biotech Trends in 2010 page.

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Biotech Trends of 2009: Three Biggest Losers

When we started the blog almost a year ago, we identified what we thought would be key trends for biotech investors and companies to watch.  Most panned out, but a few turned out to be… not so trendy.  You can call them premature (if you’re feeling generous) or call them dumb (if you’re feeling mean); but you can definitely call them losers, since they clearly failed their appointed purpose (making us look smart informing you, the reader).

Without further ado, 2009’s three biotech trends that weren’t:

  • Genetically engineered animals.  Way back in February, the FDA released final guidance on GE animal approvals and approved ATryn (a drug produced in the milk of GE goats).  We thought 2009 would be a year of approvals and other successes in this area, but we’ve hardly herd (sic, sorry) from anyone since.  We’ll keep an eye out for ewe, but this is one trend that probably won’t be baaaaack.
  • Commercialization by Foundations.  Despite a late-breaking story on this front about creative approaches in ALS and Diabetes communities, this “trend” has been another loser.  When we kicked things off, we identified two driving forces behind this trend, and each has taught us a lesson: (1) never use the words “availability heuristic” in a blog post about biotech; and (2) a sinking tide sinks all boats.  The crappy economic environment in 2009 was really no better for foundations than for anyone else, but we look forward to more creativity and private enterprise in this area next year.
  • Patent Reform.  It huffed, and it puffed, but with S.610 still in committee, patent reform carries over to another year.  Maybe now that health care reform is almost done using up all the reform oxygen, the patent system will get another turn. Don’t hold your breath, though.

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Top Four Biotech Trends of 2009

These may not all be consensus picks (and don’t miss the IVB’s year-end deal-centric fun) but I’m sticking with these four trends as the ones that have really shaped the year that was:

  1. Follow-on Biologics. Call them what you want (we like “biosimilars”, but we’re internationalist like that), there’s no denying that biosimilars were a major force in the industry and in politics this year.  Some in the press are calling the 12-year exclusivity period a done deal, but I say don’t count your chickens ’til the fat lady sings.
  2. Comparative Effectiveness and Personalized Medicine (not a two-fer, a trend of convergence). How much of comparative effectiveness variation will turn out to be an artifact of genetic sub-populations (each with binary responses to the drugs in question)?  Nobody knows, but as money pours into both fields, the truth will set us (and drug pricing) free.
  3. Shifting IP Constituencies. What do you get when you cross generics-hungry pharma companies with innovation-hungry Asian countries?  A whole new world of collaboration that will ultimately change the face of TRIPS and pharma R&D.
  4. Electronic Medical Records. Not really biotech (if you want to be picky), but it will have a massive impact on the way physicians, patients and payors interact with each other and with drug companies over the coming years.  Plus, with billions allocated to electronic medical records in Canada and the U.S., the pace of innovation and implementation really took off.

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Trends Update — Electronic Medical Records: Importance of Telemedicine, Implementation and Data Security

Since the Canadian and U.S. stimuli directed fuding towards electronic medical records (EMR), we’ve been following developments in the area as part of our Biotech Trends series here on the blog and have noted successes and failures.  A few recent stories highlight risks and benefits:

A recent Scientific American story (H/T @mikesgene) turned an analytical eye on the University of Pittsburgh Medical Center‘s implementation, the current iteration of which started in 2004.  Case studies have been an important part of the EMR narrative, and many so far have focused on Kaiser Permanente’s implementation, which is the world’s largest civilian system, so it’s nice to see an in-depth analysis of a different experience.  The article closes with a quote from  National Institute of Standards and Technology (NIST) Director Patrick Gallagher, who says the stimulus effort

“is about using technology to bring health care information together to reduce medical error, reduce the need for testing, put information in front of patients, and put information in front of researchers.”

A FierceHealthIT story reported on an initiative by the American Telemedicine Association, which is running a demonstration program with DocTalker Family Medicine.  DocTalker, founded by Dr. Alan Dappen (partnered with @drval) is providing remote health services to Association members and employees.  It’s being pitched as an employee benefit that can promote worker health and productivity by reducing the need for office visits and providing round-the-clock responsiveness. 

Telemedicine’s role in EMR also features in this story about a pacemaker developed by St. Jude Medical that allows patients and doctors at the Montreal Heart Institute to get data and alerts from the device, which also transmits cumulative data to the doctors in advance of patients’ follow-up visits.

With all of these electronic data floating around, security is key, but it remains an elusive target.  Dan Vorhaus tweeted about a ModernHealthCare.com article that highlights numerous security breaches this Fall.  Microsoft’s purchase this week of Sentillion, which focuses on EMR security, was for an undisclosed sum but you can bet it’s key to Microsoft’s EMR strategy.

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Biotech Trends Update — Commercialization by Foundations: New Initiatives from ALS Organizations, Juvenile Diabetes Research Foundation

As part of our Biotech Trends series, we’ve been following the increasing commercialization activity shown by non-profits (although they’ve been having as hard a time succeeding as everyone else).  Two recent stories highlight the important role foundations are playing in this market environment.

  1. JDRF Canada – FedDev Ontario Clinical Research CollaborationThe Juvenile Diabetes Research Foundation (JDRF) Canada is partnering with the Federal Economic Development Agency of Southern Ontario (FedDev Ontario) to fund a clinical trial network for diabetes research. FedDev Ontario is committing $20 million and JDRF is committing $10 million. JDRF will collaborate with Southern Ontario universities and research institutions to work on:
    • Speeding advances in cures and therapies for diabetes and its complications;
    • Positioning Southern Ontario as an international hub for translational research; and
    • Attracting the best international scientists and institutions to Ontario.
  2. ALS Foundations-Academia-Industry Project. Three philanthropic organizations (The Angel Fund, The ALS Therapy Alliance and Project ALS) are financing a new collaboration between Dr. Robert Brown and RXi Pharmaceuticals Corporation (NASDAQ: RXII). Dr. Brown will study the use of RXi’s self-delivering rxRNA™ (sd-rxRNA™) compounds as a potential treatment for ALS in a SOD1-overexpressing mouse model.

Each takes a novel approach:

  • JDRF Canada, by collaborating directly with the government and by focusing on clinical activity; and
  • the ALS project by allowing each party to perform in its specialty — academics on research, corporations on commercialization and the philanthropies on fundraising.

My bottom line:

In a stubbornly difficult financing environment, funding sources other than VCs step up because they derive non-financial (or at least indirect financial) benefits from their investments: corporate VCs get access to future partnership prospects; governments stimulate job growth; and charitable foundations are committed to finding cures and treatments.  These two projects are perfect examples of the work-arounds that committed participants can produce.

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Personalized Medicine Conference Highlights a Busy Month

Last week, Harvard Medical School held a conference entitled “Personalized Medicine: The Time is Now.”  Is the time now?  Looking around, it seems like personalized medicine has had a pretty good month:

PBMs Drive Demand

CVS Caremark, the country’s largest pharmacy services provider, partnered with Generation Health to expand pharmacogenomic testing for cancer, cardiovascular diseases, and HIV.  According the GenomeWeb story, CVS Caremark joins Medco‘s 60 million people, meaning the top two PBMs in the U.S. are investing heavily in personalized medicine.

Though note that PBMs’ interest in personalized medicine isn’t wholly neutral, as this post at IVB by Michael McCaughan points out.

Corporate Deals

Three pairs of corporations found the economics sufficiently attractive to strike new partnership deals:

NCI Investment To Advance Research

Finally, helping ensure that there is sufficient research output to advance the field, the National Cancer Institute put out a program announcement entitled “Development, Application, and Evaluation of Prediction Models for Cancer Risk and Prognosis,” which NCI says will be “essential for tailoring therapy to appropriate groups of patients.”

Counterpoint

Still, as the Washington Post notes in its article on the new “Ignite Institute” in Fairfax County: “[y]ou’d be right, of course, to be a bit skeptical,” citing decades of promise and so far few commercial successes in the region.

Bottom Line

Given this month’s developments, our Magic 8-Ball says “Outlook good.”  Stay tuned to see what develops, particularly as personalized medicine and comparative effectiveness grow in prominence at the same time.

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Trends Update — IP Constituencies: Novartis CEO Vasella Calls Upcoming Gleevec Decision a “Turning Point” for R&D in India

B&W_BlankMap-World-nobordersIndia and China both ranked in the third quintile of countries in the 2009 IPRI Report, with India ranking 46/115 and China ranking 68/115 for protection of IP rights.  In an earlier post, we predicted that this ranking would change rapidly, with both countries strengthening their IP regimes as their domestic R&D capacity ramped up.

Both countries have continued to win R&D collaborations and make investments in research, most recently including an Indian R&D park project by Alexandria (a U.S. developer) and Novartis’ $1 billion investment in R&D in China

However, China and India may not be moving at the same pace.  Novartis is facing its final appeal of lower court rulings in India denying it patent protection for Gleevec, and CEO Daniel Vasella calls the pending decision “the turning point” for Indian R&D in a recent article in The Economic Times (picked up by FierceBiotech).  He also reportedly says the decision to invest in R&D in China is “not driven by its bad experience with Gleevec in India.” 

Vasella has a dog in the fight, of course, but that’s the point.  The correlation between R&D spending and IP enforcement couldn’t be clearer.

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Trends Update — Electronic Medical Records: Ontario’s New EMR Adoption Program

floppy-disk1Ontario is providing up to $29,800 per physician over 3 years for new adopters of electronic medical records.  In the few weeks since the program has been implemented, the OMA has gotten over 650 inquiries and over 150 applicants.  There’s a local option and a cloud option, which runs off the eHealth Ontario servers.  Interestingly, up to $14,400 is available for early EMR adopters who complete upgrades to the latest spec.

Some interesting features at OntarioMD try to increase the effectiveness of the implementation:

  • EMR Advisor, an Ontario-based blog that includes information, tips and case studies;
  • A transition support program;
  • A support guide; and
  • Practice management consultants (on the government’s dime).

Meanwhile, Canada Health Infoway got its own clean bill of health from the Auditor General.

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Trends Update — IP Constituencies: China On the Rise as an IP Enforcer

B&W_BlankMap-World-nobordersWe have been tracking increased innovative activity in India and China as part of this blog’s Trends in 2009 series, because it has the potential to impact the constituencies that negotiate the IP aspects of global trade agreements.  Generally, with this blog’s focus on pharma and biotech, posts have mainly considered commercial collaborations to develop novel products.

Two recent stories focussed on different areas highlight just how far China has come from its perceived role as a country completely neglectful of innovators’ IP:

  1. A Thomson Reuters study released yesterday shows “explosive growth in research output from China,” with output doubling since 2004.  There is no way the developments China is making in physical, biological and chemical sciences will fail to translate into innovation and new demands for IP protection.
  2. The first salvo wasn’t in biotech, as it turns out, but in copyright.  According to a recent Forbes article (H/T @TechLaw_Elman):

    A Chinese writers’ society accused Google of infringing on the copyrights of at least 570 Chinese authors by scanning and uploading their books into Google’s digital library without seeking consent.” and is “soliciting all Chinese writers to voice their opposition before a U.S. court finalizes a settlement at a hearing scheduled for Nov 9.”

Still, these developments move China much farther toward being an enforcer of intellectual property rights both domestically and on a global scale.

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Preventing Bias in Comparative Effectiveness Research

Comparative effectiveness research has the potential to avoid wasteful spending and create net benefits for patients if approached properly, but it’s expensive.  Many of the large-scale comparative effectiveness studies include industry funding, and benefits managers are no strangers to the game, but giving those partners a say in study design risks introducing bias. 

An interesting example comes from today’s report that pharmacy benefits giant Medco is planning a head-to-head study of nearly-off-patent Plavix versus brand-new Effient.  The interesting tweak here is that the study will exclude people with a genetic variant (of the CYP2C19 polymorphism) who can’t metabolize Plavix.

This seems like another great example of personalized medicine informing a comparative effectiveness decision.  But, as the In Vivo Blog pointed out in an August post about Plavix and Effient, the effect of the CYP2C19 polymorphism on Effient efficacy is unknown.

So the PBM, with cost-saving incentives, is setting up a study to make payment decisions in which the efficacy of the (cheap) generic is boosted by excluding patients with the CYP2C19 polymorphism, with the validity of the comparison based on the untested assumption that there is no systematic bias to the branded product’s efficacy in the excluded population.  Am I missing something here?

The moral of the story: fund comparative effectiveness research through neutral parties and keep a careful eye on genetic and phenotypic subgroups to maximize the value of these important studies.

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Trends Update — Biosimilars: The State of Play of U.S. Follow-on Biologics Legislation

With the Senate Finance Committee voting this week in favour of its health reform bill, the legislative process will now move on to an attempt to reconcile the House bill and the two Senate bills in conference.

What does this mean for a biosimilars pathway?  Will there be one?  What will the exclusivity period be?  The Senate Finance bill is silent on the topic, and the two other bills both include a biosimilars pathway with a 12-year exclusivity period.

Twelve years makes the Biotechnology Industry Association (BIO) and the National Venture Capital Association pretty happy, but the Obama administration and  the FTC argue in favo(u)r of a much shorter period.

Today, a new opinion piece in the New England Journal of Medicine generated a lot of buzz, mostly because it argues for a 5-year exclusivity period (but also because it was an odd roll-out for NEJM’s new conflicts disclosure policy).

When the In Vivo Blog polled the question earlier this year, the majority vote was for 10-12 years; but me and some peeps on twitter (hi @InVivoBlogChris and Maureen @FierceBiotech) and in real life (anonymous) thought at the time the number would land under 10 years.  I’m sticking to that bet.

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Biotech Trends in 2010: Get An Early Start at OGI’s Synthetic Biology Conference

OGI syntheticbiologyIn updating this blog’s Trends in 2009 series for 2010, I noted that synthetic biology has garnered recent attention in The New Yorker and The Economist and it may be poised to attract more commercial attention.   Here’s a great chance to get a jump on the topic…

Ontario Genomics Institute (OGI) and Integrated DNA Technologies (IDT) are hosting a free one-day symposium on synthetic biology on October 27th at MaRS.  It’s invitation only, but you can contact Helen Petropoulos <hpetropoulos at ontariogenomics dot ca> if you want to register.

The symposium will be chaired by Elizabeth Edwards, a Professor in the Department of Chemical Engineering and Applied Chemistry at the University of Toronto, and presenters include leading researchers from across North America:

Here’s the blurb from the organizers:

“This symposium will explore the field of synthetic biology and its many application areas, culminating in a panel discussion focused on challenges and opportunities in one of today’s most promising research fields.  An open public forum on synthetic biology and its impact on society will follow the symposium program.”

Hope to see you there!

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Trends Update — IP Constituencies: India’s Glenmark Pharmaceuticals Sets an Example for Canadian Innovation

This blog has been following the increasing innovative activity taking place in India’s and China’s biopharma industries, and Glenmark Pharmaceuticals is a great example of this trend.

Forbes profiled Glenmark this week (H/T FierceBiotech), noting that it started in 1978 as a generics firm but now has 7 clinical-stage compounds and has partnerships with Forest Labs in the U.S. and Teijin Pharma in Japan.

There are two really interesting points raised in the article:

  1. Glenmark is using its revenues from generics to fund its innovative R&D programs. Biotech business models have long incorporated quick revenue as a funding source for long-term R&D; but the typical focus for the short term is on services revenue (because it typically leverages the platform they’re building anyway).  For long-term survival, though, nothing beats tearing a page out of Big Pharma’s playbook.  In Canada, Bioniche’s tenacity is fueled in no small part by animal health revenues, mimicking the Big Pharma animal health divisions.  Glenmark, similarly, has ridden into R&D on the back of the other Big Pharma cash cow — generics.
  2. Glenmark saw a strong IP regime as an opportunity. I’ve been hypothesizing that more innovative activity will drive more support for IP protection in India and China; but Glenmark is an example of the opposite causal relationship:

    “During his time in the U.S., Saldanha could see that India was likely to join the World Trade Organization and that meant come 2005 it would have to respect patent laws: ‘Generics generate cash, but we needed innovation to take us into the future.'”

My bottom line: Glenmark is a great example of how to look to the future and build for it.  Canada has an opportunity now, on a national scale, to deploy its revenue both within the biopharma industry (from our generics companies) and outside it (from our natural resources) to build for the future Saldanha saw.  We’d better seize that opportunity while we can.

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Biotech Trends in 2010: Crowdsourced Edition

We’ve been running with a number of trends since the blog started early in 2009, and though many of them will continue to be critical stories in 2010, I’ve been turning my thoughts lately to possible additions for next year:

Thoughts? Other suggestions?  Let us know

Update: Timely – see this New Yorker article on synthetic biology and a report on a poll on the topic via ScientificBlogging (both via @gw_dailyscan).

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Trends Update — IP Constituencies: India M&A Spurs Pricing Concerns

world_map_2002We’ve been following the trend of increased innovative activity in developing countries, and have noted its likely effect on IP protection in those jurisdictions. In a variation on that theme, an article last Thursday in DNA suggests that

“Indian companies, which have been actively pursuing pre-grant and post-grant oppositions against the patents of MNCs, would become less aggressive after being acquired by Big Pharma.”

This seems like a plausible outcome, although it is counterbalanced by the increasing stake innovator companies are taking in generics and follow-on biologics companies.  It is also counterbalanced by statistics from Navroz Mahudawala at Ernst & Young (cited at the end of the article) showing that multi-national companies’ share of India’s pharma market has actually dropped by almost half over the last two decades. What is more interesting to me, though, is the nature of those investments, which appear to have been shifting to rely more heavily on India’s R&D capabilities. These domestic R&D capabilities will be the real driver of change in attitudes toward IP protections.

In light of shifting IP attitudes, the pricing of drugs is a legitimate concern, but consider what I’ll dub “the innovation cycle” — India’s increased R&D capabilities will fund higher incomes and help citizens afford higher-priced drugs, while countries that may have been importing India’s generics will be forced to develop their own generics production capabilities, which may eventually (as in India) lead to innovative R&D, etc., etc.

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Trends Update — Commercialization by Nonprofit Foundations: iCo-CPDD Deal, plus MJFF Comes to Canada

One of the trends we’ve been keeping an eye on this year is the increasing willingness of nonprofit foundations to fund and support commercial product development.  Two updates today:

  1. iCo Therapeutics Inc. (TSX-V: ICO) is collaborating with the Consortium for Parasitic Drug Development (CPDD) to optimize one of iCo’s products for tropical conditions.  (That’s CPDD as in development of drugs to combat parasitic diseases, by the way, not a new and selfish approach to drug development.)  CPDD, a Bill & Melinda Gates Foundation grantee, is making an initial contribution of USD $182,930 to iCo’s work.
  2. The Michael J. Fox Foundation, which has been very active in providing funding to companies working on Parkinson’s treatments, has registered as a tax-exempt charity in Canada.  Hopefully this will open the door for more Canadian collaborations.

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Trends Update — Personalized Medicine: Montreal CRO ethica Licenses Artificial Intelligence Data Analysis Product for Stratification

B&W_DNA_sequenceethica Clinical Research acquired a worldwide exclusive license to Matrix Pharma’s  artificial intelligence (AI) data analysis platform.  Neither the form of consideration nor payment structure (up-front vs royalty etc.) was disclosed, but the deal is “valued at CAD1.25 Million.”  The companies say the AI can:

“extract interdependencies, correlations, and predictive models from complex data sets that conventional statistical tools are unable to detect.”

In other words, it’s an approach designed for stratification of clinical trial data to identify personalized subgroups for whom a drug may be particularly effective.

ethica says the product, which will be marketed as “eidyia™” (pronounced “idea”)

“has already yielded successful results in a series of clinical applications such as identifying biomarkers, optimizing the predictive power of biomarkers, identifying responder/non-responder indices, genotype-phenotype linkages, elaborating models for early diagnosis, and for classifying participants in Phase II clinical trials for the optimization of Phase III clinical trials.”

I don’t know if  “eidyia” is (or isn’t) the best platform for these tasks; but I am convinced that in many cases, the ability to stratify patient populations will be key to demonstrating the effectiveness of new drugs and winning approval and reimbursement.

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Trends Update — Personalized Medicine: Merck Strategy Head Skeptical

As I’ve been following personalized medicine on this blog, I have become almost convinced that recent advances in genomics technology put us at the brink of an era of personalized diagnosis and treatment.  Not everyone agrees.

Chris Morrison, reporting from the Pharmaceutical Strategic Alliances meeting, quotes Merv Turner (the head of strategy at Merck) as follows:

“‘You can reduce cardiovascular mortality by 50%’ by using statins, he said. ‘That means 50% cardiovascular disease is unsatisfied. Is that 50 different small diseases or one large one? Personalized medicine is like soccer in the US: it’s the game of the future and always will be.'” (emphasis added)

Maybe I’m missing some context here, but I think the answer to Turner’s question (50 different small diseases or one large one) is “we don’t know.”  It is an interesting question and I think it would be prudent to find out the answer.  Preferably before we try treating the 48th disease with a drug developed for the 35th.

Trends Update — Comparative Effectiveness and Personalized Medicine: Is Canada Ahead of the U.S. In the Use of HER2 Testing for Personalized Breast Cancer Treatment?

B&W_DNA_sequenceFor the 20%-30% of breast cancer patients with tumors that overexpress HER2, treatment with Herceptin (an antibody drug from GenetechRoche) is highly effective.  That’s why this article in the journal Cancer is so shocking.  The authors gathered data from a variety of published sources and estimate that:

“up to 66% of eligible patients had no documentation of testing in claims records, up to 20% of patients receiving trastuzumab were not tested or had no documentation of a positive test, and 20% of HER2 results may be incorrect.”

I asked a friend, who is a genetic counsellor in Toronto, if she thought the gaps here were as bad.  She said no, and that she would be extremely surprised not to see a HER2 result in a patient’s file.

Of course it’s not valid to draw conclusions about national health care from a comparison of the Cancer study to my anecdote.  Among other reasons, the authors admit their data is flawed and dated, and my friend works at one of Canada’s top teaching hospitals.  Nevertheless, the possibility that many U.S. patients could be falling through the cracks is extremely disturbing.

If clinicians can’t adopt and maintain near-universal use of a personalized medicine approach like HER2-Herceptin with recognized and measurable benefits, the future of pharmacogenomics is in some big trouble and we will never generate truly useful comparative effectiveness data.

H/T @FiercePharma.

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Trends Update — IP Constituencies: Amylin Partners with Biocon, PerkinElmer Buys Access and Capacity in India and China

world_map_2002Following up on Sunday’s post noting the new survey of Canadian biotech collaborations with companies in the developing world, it’s worth paying attention to two U.S. deals from last week that emphasize the growing role of India and China in the drug development process:

  • Amylin Pharmaceuticals, Inc. (Nasdaq: AMLN) and Biocon Limited (NSE: BIOCONagreed to jointly develop, commercialize and manufacture a novel peptide therapeutic for the potential treatment of diabetes, and will share development costs; and
  • PerkinElmer did one deal each in China and India:
    • In China, they paid over $60 million for SYM-BIO Lifescience, a Shanghai, China-based diagnostics firm that will double its access to hospitals in China and provide it with “substantial” manufacturing plant capacity
    • In India, they picked up the genetic screening business of Surendra Genetic Labs, a lab in Chennai, India, that provides fetal, maternal, and newborn screening.

The McLaughlin-Rotman survey distinguishes (usefully) between R&D collaborations and marketing and distribution activities.  Although Ellen Licking wonders whether “cheaper off-shore manufacturing [is] a reason for the deal,” Amylin is clearly focused on Biocon’s R&D capabilities.  The press release quotes Amylin CEO Daniel M. Bradbury praising Biocon as a “biologics innovator.”

Even if not all transactions in India and China are innovation-centric (cf the PerkinElmer deals), all involvement by innovative companies shifts the incentives of China, India and other participating countries towards a more innovation-friendly IP regime.

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Trends Update — IP Constituencies: Rotman Article Explores Canadian Biotech Collaborations with Developing Countries

A very interesting article in Nature Biotechnology from a group at the McLaughlin-Rotman Centre for Global Health provides some empirical support for a trend we’ve been following of increased innovative activity in developing countries

According to the article, over 25% of Canadian biotechs collaborate with developing countries.  Of these, however, the vast majority of companies do so alongside collaborations with other developed country partners — only 4% collaborate exclusively with developing countries.  Also, gaining access to developing countries’ markets is the most frequent (66%) reason cited for collaboration.

Still, some of the data reflects the growing importance of developing country collaboration (China and India in particular):

  • Canadian firms’ collaborations with India (17) and China (22) nearly equal the number of collaborations with Japan (18) and Germany (23); and
  • Accessing knowledge from developing countries’ partners (24%) is approaching providing knowledge to developing countries’ partners (37%) as a reason for collaboration.

How do these collaborations look overall?

Collaborations article - nbt0909-806-F4

The figure from the paper on the left shows the geography of, and rationale for, the collaborations. Part “a” shows marketing and distribution collaborations, and part “b” shows those involving an R&D component.

 

What is the effect of all this activity?

Well, it’s hard to quantify, but the authors review revenue data from public company respondents and find that:

“average total revenues of firms that have North–South collaborations are nearly four times higher than firms that do not have such partnerships.” 

My bottom line: causal or not, that’s a correlation that should cause all biotech companies to take note.

New York Times’ Brody Counsels DTC Genomics Caution

An article in yesterday’s New York Times calls direct-to-consumer genetic testing, a trend we are following on this blog, “fraught with potential dangers.”  Although our original post on the subject discussed many of the regulatory and ethical issues around DTC genomics, Brody’s article raises some interesting additional points:

  • The risk of false reassurance: “a man told he lacks genes linked to an elevated risk of heart disease might decide to smoke, eat lots of salt and saturated fats, avoid exercise or develop a large paunch.”
  • The article also notes privacy risks, citing Lori Andrews’ comments that “[s]ome companies are just a front end for biotech companies that use it for research.”

Brody is reluctant to confer even a label of “relatively harmless” on the current technology, but the article will probably drive further awareness and additional interest.

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Trends Update — Comparative Effectiveness: Where Data Shows No Difference, Tie Should Go To the Patient

A post by Scott Hensley on the NPR Health Blog yesterday has some good food for thought in the comparative effectiveness debate: what to do when comparative effectiveness studies show no statistically significant difference between treatments.

The post notes that insurance coverage will be a factor in these decisions, but that:

“in the end, it might be you and your gut feeling.”

In one of this blog’s prior posts, I noted that it will be hard to distinguish between treatments that show different effectiveness because of personal differences between patients and those that would show different results even if the patients were identical. 

Hensley’s post illustrates that no matter how much data we gather, there will be gray areas where doctors and patients have to make subjective calls.  I hope payors will be extremely cautious about second-guessing these decisions.  Tie goes to the patient.

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Trends Update — DTC Genetic Tests: NOVA ScienceNOW Program Takes a Look

B&W_DNA_sequenceYesterday’s NOVA ScienceNOW program included a segment on direct-to-consumer genomics (H/T to GenomeWeb’s Daily Scan Blog).  The program was bullish on George Church’s Personal Genome Project; but it took a pretty dim view of the predictive value of current consumer technology.

The program was accessible and interesting, but it went overboard in making a cautionary point about current DTC genomics offerings.  It transitions directly from Neal deGrasse Tyson’s 23andMe results for heart disease and diabetes to Steven Pinker’s genomic scan, which showed that Pinker had “double the risk of baldness,” whereas Steven is anything but bald.  Well, sure, and the weather report yesterday said there was an 80% chance of rain but it didn’t rain.  That doesn’t mean I should stop checking weather reports, or even that I was stupid to pack an umbrella.  It’s just probabilities.  I guess I agree with the program in the sense that anyone who can’t spot that flaw shouldn’t be interpreting their own genomic data, but it seems like an oddly condescending way for them to make the point.

Kudos, though, for pointing out:

  1. the gaps in the Genetic Information Nondiscrimination Act; and 
  2. the low risk to tenured Harvard profs of revealing their sequence data, as they are likely shielded from many of the risks to other participants.

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Trends Update — Personalized Medicine and Comparative Effectiveness: HepC Treatment Gap, Leukemia Genetics and Beckman Coulter Genomics

B&W_DNA_sequenceA few interesting items hit the news this morning that continue the trend of explaining comparative effectiveness data by examining underlying genetic variation.

  1. Genetics explains why white patients respond better than black patients to standard Hepatitis C treatment. Bloomberg reported on a Nature paper showing that Hepatitis C patients with a genetic polymorphism near the IL28B gene show a 2-fold better response to treatment than patients without the variant. Because the variant is more common in patients with European ancestry than in those with African ancestry, it accounts for about half the observed difference in treatment response between the two populations.
  2. Genetic variants were identified that are associated with increased risk of childhood leukemia, which could lead to a better understanding of disease etiology and could lead to personalized treatments down the road.
  3. In a sign that genomic technology will be an increasingly important part of healthcare, Beckman Coulter formed a new company, Beckman Coulter Genomics, that will provide gene sequencing, sample preparation and other genomic services.

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Trends Update — DTC Genetic Testing: Survey of State Laws on False Advertising

B&W_DNA_sequenceOne aspect of direct-to-consumer genetic testing that requires particular vigilance is the “consumer” aspect.  We should expect that as the underlying technology becomes cheaper and testing companies proliferate, there will be more who prey on insecurity and health fears to make a quick buck while providing little value (or worse, missing genuine concerns).

GenomeWeb Daily News today notes a survey of state “false advertising” laws (pdf) conducted by Anya Prince, a student with the Georgetown University Law Center’s Harrison Institute for Public Law.  At the moment, the survey reports, there are no state laws specific to genetic testing.  However, the survey does identify various generic false advertising laws that could apply if DTC providers make false or misleading claims.  As GenomeWeb notes, the Federal Trade Commission has already shown an interest in policing the area.  Together with the CDC, they put out a flyer in July 2006 on DTC genetic tests for consumers, advising that the tests are only truly valuable if interpreted by a doctor or trained counselor.

Some skeptics note that the value of tests for genetic predispositions is minimal.  Even without a genetic test, we know that if we want to avoid heart disease we should eat well and exercise.

Similarly, even without specific laws aimed at genetic shenanigans, we already know that providers who want to avoid liability should, in their literature and in their contracts, be honest with their customers about what the results do and do not mean.

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Trends Update — “Personalized Effectiveness”: Amgen Gets Prospective Data to Back KRAS-Vectibix Plan

B&W_DNA_sequenceA few weeks ago, when the FDA changed the labeling on anti-EGFR drugs, Amgen was pretty enthusiastic about “avoiding unnecessary treatments in patients [with a specific genetic marker] who are unlikely to benefit” from Vectibix.  Avoiding these patients leaves more reimbursement available for patients who would benefit from Amgen’s product.

Now Amgen has even better data to support its personalized approach to colorectal cancer treatment: their study of Vectibix as a first-line treatment tracked the KRAS genetic status of participants and showed “significantly prolonged progression-free survival” for the wt-KRAS group.

In patients with mutated KRAS, Vectibix wasn’t just “unnecessary,” it actually showed worse outcomes than the control group, meaning genetic testing of all colorectal cancer patients will be a top priority.

A second important note for companies thinking about companion diagnostics and personalized effectiveness is that the Amgen study was a prospective study that will support much more robust conclusions.  H/T @ldtimmerman.

In contrast, recall that the CMS decision not to reimburse genetic testing for Warfarin dosing specifically cited the lack of prospective data on which to base a decision.

This new data from Amgen will:

  1. Drive tumor genotyping as a standard of care; and
  2. Help make the economic case for companion diagnostics.

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Trends Update — Electronic Medical Records: Salesforce.com Clouds the EMR Field

floppy-disk1The WSJ Health Blog notes today that Salesforce.com’s investment in Practice Fusion, though not a large financial investment, follows an appealing trend in the EMR space.  Both Salesforce.com and Practice Fusion are cloud computing plays (aka hosted services / ASP) where software and data live on company servers rather than on local PCs in doctors’ offices. This will be particularly attractive for smaller practices without their own IT capabilities.

We’ve been following the world of electronic medical records, which has attracted billions of dollars in U.S. stimulus money and over $400 million in Canada, and have been keeping track of notable implementation successes and privacy challenges.

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