Stock Watch: Full Approval And Surrogates Await Clinical Validation

Time Is A Great Leveller Among Outcome Measures

When surrogate endpoints are used for accelerated approvals in advance of more compelling functional outcomes, how long should the duration of the confirmatory studies be?

Stock Watch Image, Andy Smith
ANDY SMITH OFFERS A LIFE SCIENCE INVESTOR'S PERSPECTIVE ON BIOPHARMA BUSINESS

In the 2011 dystopian movie In Time staring Justin Timberlake, people stop aging after 25 and have to buy time to extend their lifespans. The rich already have centuries or even millennia while the poor literally live from one paycheck to another. In this reality, however, a related conundrum for those investing in longevity-extending companies is what would the duration of a clinical trial need to be to demonstrate lifespan extension. The conundrum becomes more acute when elderly billionaire investors who on the one hand are ambivalent to drug reimbursement and perhaps even clinical trial costs, realize on the other that the decades needed to conduct a clinical program will exceed their own lifespans. In more traditional indications like oncology and cardiovascular disease, time – or more often the time between events – is also an archetypal outcome.

Probably for indications like extending longevity, and those where it may be unethical to measure hard outcomes like survival time in ultra orphan diseases, there are surrogate endpoints and the...

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