The Cross-Border Biotech Blog

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

Tag Archives: clinical trials

Abbott’s ABT-888 Guinea Pig: First Phase 0 Clinical Trial Completed

A press release from the National Cancer Institute yesterday (picked up today by BIO SmartBrief and FierceBiotech) touts a successful first:

The first phase 0 clinical trial of a drug in cancer treatment, involving 13 patients with advanced cancers, showed that the drug, ABT-888, affected its target and was well tolerated. Most importantly, this trial showed that it is possible to enroll a small number of patients, treat them with a low dose of a new drug, identify whether the desired target of the drug was affected, and obtain all of this critical information relatively quickly.

The point of a “phase 0″ (phase zero) trial is to focus “primarily on tolerance and the ability of the drug to hit a target.”  The advantage of phase 0 trials is that they:

involve nontoxic drug doses that are administered for short periods of time to small numbers of patients, [so] the preclinical toxicology data required … are less than those required to support a phase I trial; thus, these first-in-human trials can be initiated earlier in the drug development process than traditional phase I studies.

Phase 0 trials come with their own statistical evaluation scheme and endpoints, and although more long-term data is needed on the impact of this approach on timetables and success rates, “phase 0 trials will be a key part of a new approach” in the NCI Experimental Therapeutics (NExT) program.

Here’s NCI’s FAQ from the 2007 introduction of the program, with more info on NExT and phase 0 trials.

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Existential Question for 2009: Is a Virtual Company Virtually as Good?

A thought-provoking piece over at Capital Rants (and the BlackBerry Partners Fund Blog) from Pierre Donaldson says, basically:

virtual-schmirtual, there’s no substitute for the interpersonal benefits of a physical headquarters where the whole team is regularly under the same roof and interacting face to face.

On the legal side, we have a tendency to view virtual/physical, outsource/in-house issues from a straightforward economic perspective, basically:

is the price of the virtual/outsourced service, plus the cost of managing the service, plus the transaction cost of setting up the service, less than the cost of providing it in-house?

Nominally, the issues Pierre is talking about fall into the service management costs bucket, but his post draws attention to some opportunity costs of virtualization, something generally not given enough attention in the  accounting cost version of these calculations.

Overall,  I agree that lack of physical interaction is an impediment to team spirit — the post’s central “Locker Room Theory”.  

The point I want to quibble with is:

“We can e-mail, IM, SMS, blog, tweet… you name it.  It is exactly as if we are constantly sitting right next to each other, right? … I’m sorry, but I don’t buy that at all…

“It’s about the water cooler conversations and finding out that your sysadmin’s favourite uncle has a golf buddy who is CIO at that large corporation you’ve been trying [to] partner with.” 

Lumping e-mail with Twitter, and leaving out LinkedIn and Facebook from his list is obviously leaving out a lot of nuance.  All three are useful virtual tools with far greater reach (though maybe less (normalized) utility) than sysadmins and uncles and golf buddies.

But, can we really virtualize networking the way we’ve virtualized companies?  When the BlackBerry Partners Fund shackles their portfolio company teams to an office and sends them on corporate trust-building retreats, are they building the next … BlackBerry … or are they taking valuable time away from meetups and status updates and the next great tweet

The current economic climate will likely result in a large-scale experiment as companies in a variety of industries either hunker down and go back to the water cooler or attempt to tweet their way out of their individual crises.  Hindsight, no doubt, will be 20/20.

Pharma and biotech companies are constantly faced with these virtual/physical outsource/in-source questions; and I expect we’ll continue to see a lot of comepeting approaches.  Fully virtual companies are possible and successful, with R&D, formulation, CMC, regulatory and clinical trial capabilities fully available on a contract basis from a bevy of worldwide competitors.

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Disclosure, Disclosure, Disclosure

Disclosure issues have permeated the news lately.  Pharmaceutical companies need to do a better job of disclosing adverse clinical trial results and side effects; companies and doctors need to do a better job of disclosing payments; and journals need to do a better job of disclosing author conflicts.

You could view the question of whether to disclose from a lot of different perspectives, but I’m hard pressed to find one that argues in favor of secrecy: economic (efficient markets), legal (Exchange Act, FDAAA, FTC), and corporate (reputational harm) considerations all seem to point to disclosure.

While there will always be some level of outright fraud, and there is risk to individuals who do disclose (risk to future work, inability to publish, etc.), institutions should be moving toward increased disclosure. 

Some have:

  • GlaxoSmithKline is heading in the right direction.  Last year they promised to publish payments to U.S. doctors for consulting and other services starting in 2010, and to cap those payments at $150,000 per doctor a year. Now, the company is planning to expand its disclosure to include money paid to doctors and their institutions to carry out clinical trials, and fees it pays European doctors for advice on developing new drugs.
  • The American Psychiatric Association (pdf) Board of Trustees voted this month to phase out industry-supported symposia along with industry-supplied meals at its annual meetings.

I vote for more.

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Friday Science Review: March 20, 2009

I had a hard time finding news-making Canadian science stories this week; but in the meantime, here’s a  “numbers” edition of the Friday Science Review:

  • Number of Canadians with cancer: Statistics Canada released its latest figures, showing that of all persons living in Canada on January 1, 2005, 695,000 had been diagnosed with an invasive cancer at some point in the previous 10 years.  That includes the 1 in every 111 women diagnosed with breast cancer, and the 1 in every 118 men diagnosed with prostate cancer.  The headline number shows an increase in the number of Canadians living with cancer; but that’s due to the earlier detection of cancer and improving survival.
  • Number of Canadians who could be helped by nanotech packaging: 11 million Canadians suffer from food-borne illnesses, some of which could be prevented by rapid nanoparticle-based testing.  Nanoparticles could also be used in packaging to signal when food has passed its best-before date.
  • Number of centimeters long you have to be to qualify as the “T-Rex of the Cambrian Period”: twenty.  At the time, most creatures were no bigger than a fingernail … although how could you tell, since there were no fingernails at the time?!? (har.)
  • Number of mussels needed to make a tube of a new medical adhesive: mussels don’t make adhesive, people make adhesive.  Duh.  Here’s a cool story, though, about a new adhesive based on a protein that marine mussels use to stick to rocks.
  • Number of volunteers needed for a 3-way crossover study to show bioequivalence: twenty-three, at least for SemBioSys Genetics Inc.’s trial of its plant-produced human insulin.

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Wendesday Brain Dump: January 28

Things that caught my eye this week:

The FDA made Geron very happy; Sarkozy made French scientists very unhappy.

Here at home, some regulatory milestones for Oncolytics, Welichem and Pfizer Canada.

Europe’s R&D intensity (spending as a percentage of GDP) was stagnant overall from 2000 to 2006, but at least a few detractors are out of commission.

And last, since we don’t want to dwell on the negative, some advice from PwC on transfer pricing and other more general approaches to weathering the downturn.

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