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The two broad categories of medical discovery that command the most attention are insights resulting from rare, informative genetic conditions (see here) and advances resulting from fortuitous observations.   A canonical example of the value of extreme genetic phenotypes is the patient with familial hypercholesterolemia who inspired Brown and Goldstein’s scientific pursuit of cholesterol metabolism and led to the statins. Similarly,... Read More

GLP-1 medicines are obviously having a real moment – medically and culturally.  These once-weekly injectables, which reduce appetite and result in significant, long-lasting (so long as you’re taking them…) weight loss, have been demonstrated to have a number of important health benefits beyond simply shedding pounds.  In March, Novo Nordisk’s semaglutide (Wegovy) was approved by the FDA for its ability... Read More

A main theme of this column – perhaps even the dominant theme – is how to operate in a power-law domain like drug development where there are few, outsized successes, where most efforts end in failure, and where it’s incredibly difficult to predict in advance how you are likely to fare. Earlier this week, I discussed the parallels between film... Read More

Film and pharma, like many creative endeavors, exist in a world of power law economics, where a handful of exceptionally successful products account for a massively disproportionate share of the total revenue.  Consequently, in both domains, there’s a powerful incentive to “pick winners.” Every studio head and every R&D leader tries desperately to do this.  But there’s a problem: as... Read More