Drug Baron

October 16, 2013
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Clinical trial transparency: time for some carrot as well as more stick?

Today, the majority of clinical trials that are run never report their results in a form that is accessible to the public.  To many people that is unacceptable for several reasons: it makes it possible for drug companies to selectively marshal the evidence base surrounding their drugs (if not to subvert the regulatory process, then to spin the story to support adoption in the clinic); it is also morally questionable given that the public are essential participants in trials, and in return deserve to see the results.

High profile commentators, such as Ben Goldacre and his AllTrials movement, have brought this issue to the centre stage, and leverage public opinion effectively to shame pharmaceutical companies in particular into adopting the principles of comprehensive transparency.

Pharmaceutical companies are much less popular than they should be.  Sometimes it feels like these corporations rank only a little above politicians and bankers in the affections of the wider public.  The huge gains in public health they have brought about gain less attention than the profits that they make in the process.  And the most powerful legacy of a decade of socialist government in Britain is disdain for success.

That makes pharmaceutical companies easy targets to subject to the power of public opprobrium, coordinated through the glue of modern social networks.

But there are genuine concerns about transparency.  Data costs money to generate, and controlling the output of expensive research efforts is an essential part of having commercial operations investing in research and development.  Forcing complete disclosure may increase comfort levels in the customers, but risks making pharmaceutical R&D economically unsustainable.

It would not be to the benefit of the public at large to hobble commercial investment in clinical research and development – putting at risk future health gains of the magnitude delivered by medicines such as statins, which have reduced deaths from heart attacks and strokes by almost half in just fifteen years.

If we accept that comprehensive transparency of clinical trials is a goal worth fighting for (and DrugBaron believes that it is), is there a way of achieving it other than by force? Is there a way of encouraging the wider pharmaceutical industry to embrace full public disclosure of clinical trial data, rather than offering the minimum possible standards of transparency necessary to placate their enemies?

DrugBaron believes there is: offer a carrot, whose value is at least as great as the penalty associated with loss of proprietary knowledge.  A carrot that makes disclosure preferable.  A carrot that eliminates the open-ended liability associated with launching a new drug and keeping it on the market.

A new pact between the public and drugmakers could deliver these benefits.  In return for complete disclosure, the state should take on the liability for harm the drug may do in the future which could not be predicted from the data used to support the approval.

Provided the drugmaker complied with these new requirements for complete disclosure of all information (whether deemed relevant or not), they no longer need fear mass tort litigation of the kind that followed the realization that cox2 inhibitors increase the risk of heart attacks, or that certain powerful anti-inflammatory biologics cause PML.

Pharmaceutical companies stand to gain substantially from the certainty that, once approved, they are no longer responsible for events that they could not predict.  That such protection is invalidated by failing to disclose data provides a powerful carrot for full disclosure.  A carrot that, together with the stick of public opinion, could usher in a utopian new world of complete transparency of clinical trial data.

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October 2, 2013
by admin
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PheWAS – the tool that’s revolutionizing drug development that you’ve likely never heard of

Late-stage attrition kills returns on pharma R&D investment.  One way to reduce attrition is to take less risk – incremental innovation reduces the risk of failing in development, but increases the risk of failing in the marketplace with even bigger (though delayed and less sudden) consequences.

The other solution is to address the principle causes of late stage failure: inadequate target validation and unpredictable safety liabilities.

Easier said than done? Not any longer – there is a single tool that can address both problems in one experiment.  That tool is the Phenome-wide Association Study, or PheWAS, and it’s already delivering a quiet revolution across the industry.

Chances are, though, unless you work for one of the handful of global pharma that have been able to embrace this technology, you have haven’t even heard of PheWAS, or certainly appreciated what it can do for drug discovery and development in the 21st Century.

The reason for its stealth is simple enough: constructing a PheWAS platform not only consumes millions of dollars, but takes a decade or more to deliver – so only those with the right combination of cash and foresight are now sitting on the one tool that can deliver a step-change in Phase 3 success rates.

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September 23, 2013
by admin
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On the origins of hypochondria

DrugBaron suffers from a condition for which there are no approved drugs.  At times it can be a debilitating condition, with obscure causes.  Like many psychological illnesses, sufferers are often dismissed as slightly crazy, suffering from something that (to a non-sufferer) appears to have no basis in reality.  DrugBaron suffers from a (relatively mild) case of hypochondria.

Where does this notion that every mild ache or pain might be the harbinger of something particularly nasty, or even fatal, come from?

A little introspection revealed three plausible risk factors.  And given that a fair proportion of DrugBaron’s readership is likely to share more than the average number of these putative predispositions, it will be interesting to see if you, gentle reader, recognize any of these feelings.

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