Drug Baron

August 19, 2014
by admin

After NICE is it time for NASTY? National Academy for the Study of Things that harm You

The United Kingdom’s National Institute for Clinical Excellence (NICE) has proven its worth as an independent arbiter of value-for-money when it comes to medical interventions.  People may question the details of the methodology they employ to compare effectiveness of drugs and procedures, but few doubt the principle of its existence.

Unfortunately, there is no equivalent framework for assessing public health interventions.  A swathe of unreliable cross0-secvtional studies have highlighted potential harms from a diverse array of sources, but particularly various components of our diet.  A vocal minority is calling for action to limit these perceived “harms”.  But how real is the risk?  Even where the risk is proven, how much of a contributor to our overall health are these factors? There is a very real risk governments will start to intervene (through legislation or tax measures to disincentives certain behaviours) on the basis of public perception rather than a quantitive, rational and transparent assessment of the costs and benefits of such interventions.

The solution, according to DrugBaron, is to adopt the NICE methodology to estimate value-for-money of proposed public health interventions, in exactly the same way we do for medical interventions.  This would be the mission of the National Academy for the Study of Things that harm You (NASTY).

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July 8, 2014
by admin

Twenty six of the best candidate protein biomarkers have no ability to predict Alzheimer’s Disease

DrugBaron woke up this morning to the latest chapter in the “blood test for Alzheimer’s Disease” soap opera, with this article from the BBC trumpeting the latest study to be published as a “major step forward”.

The study behind the headlines was published (unusually for a “major breakthrough”) in the journal Alzheimer’s & Dementia, and described a study based on the measurement of 26 candidate protein biomarkers in blood samples from 1,148 individuals, 476 with Alzheimer’s Disease, 220 with mild cognitive impairment (MCI), and 452 controls of similar age but no dementia.

The authors of the study, and their industrial collaborators at Proteome Sciences plc, were not backward at promoting this work as a significant: Dr Ian Pike, Chief Operating Officer at Proteome Science declared “Having a protein test is really a major step forwards.  [It] will take several years and need many more patients before we can be certain these tests are suitable for routine clinical use, that process can start fairly quickly now.”

Even independent voices were quick to praise the new research: Eric Karran, Director of Research at Alzheimer’s Research UK, described the study as a “technical tour de force”.  Really?

This is, after all, not the first 2014 paper to make such a claim: in March a paper in Nature Medicine made almost identical claims, and BBC article reporting that study even used many of the same stock images!  Even the headline claims of the two studies were similar (90% accuracy for the Nature Medicine paper verus 87% accuracy for the new study).  And both used similar methodology: multivariate signatures, although the earlier study was focused on metabolic biomarkers and the new study on proteins.

So did the new study justify the hype any more than the previous attempts to solve this important problem? DrugBaron reviewed the primary publication with interest.

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June 26, 2014
by admin

“On the way to changing the world, there are no signposts” : the illusory power of precedents

Precedents play a central role in the drug development industry.  Clinical trials are based on earlier studies with ostensibly similar drug candidates.  Business models are judged against the current landscape.  Assets are valued by looking for comparables.  Indeed, big pharma consider the institutional accumulation of such experience to be a significant competitive advantage.

But if you stop and think about it, such an approach makes no sense if you seek true innovation.  The premise of all innovation is that it breaks the mould, changes the goalposts, disrupts the marketplace.  Aiming to change the world, and then judging your progress against historical norms isn’t just meaningless – some precedents can lead you down blind alleys or drive altogether the wrong decisions.

DrugBaron examines a number of recent examples of misapplied precedents to illustrate why, when searching for the breakthrough that changes everything, forwards is the only way to look.

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