Drug developers face increasingly risky tightrope walk between top-line results and hard data

Even the biggest pharma companies can hype the results of a big drug study, given the curious dance-of-the-seven-veils choreography provided to late-stage data. And the Wall Street Journal is offering up an example to underscore just how perilous this dance can be for all players.

Case in point: Roche and Perjeta. The Swiss pharma giant first offered a top-line glimpse of its positive results for the APHINITY study involving Perjeta, but had to deal with some bitter reactions when analysts got a chance to examine the marginal benefit that has been provided.

Some of this is the simple result of a system where drug developers toss out top-line outcomes but reserve the results for a scientific conference.

The Journal could have picked from any number of cases, including the big Amgen cardio outcomes study on its PCSK9 drug Repatha. The FOURIER study was built up as a big success, with Amgen executives almost instinctively insisting that statistical significance would translate into market acceptance. But with no mortality benefit in the mix, the whole performance came in looking weak and unconvincing.

For the WSJ writers, the push to tout results is tied to some particular risks for execs who sell shares between the hype and the reality. But a bigger lesson may be that increasingly we’re seeing that success in a clinical trial is no guarantee of a win on market gains. The bar on success can be much higher than clinically statistical success for payers expected to cover these drugs.

One other observation: If you’re going to predict that your drug can clear a specific hurdle, that becomes the bar you have to jump to win applause. Ironwood found that out the hard way recently when its Phase IIb data for a would-be blockbuster failed to hit its own mark – even though the data were statistically significant.

That trend to continually raise the bar – and risk the consequences of falling short of expectations – is only going to get tougher for drug developers.

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VP Oncology Biology
Skyhawk Therapeutics Waltham, MA
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Director Process Development
Elektroki Boston, MA
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Recursion Pharmaceuticals Salt Lake City, UT

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