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Comment by derlvative | Hacker News Reader

Completely false comparison because the drug in question did provw safety though the clinical trial, only effectiveness that could not be determined. People should have options that are not certain death.

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It’s not a false comparison. Effectiveness is what makes a drug not merely dyed water. Hence the “ripped off” part.

The tricky business is defining effectiveness (and the target population). This drug was not effective at the specified clinical end-point for one set of patients. But it did show promise for other end-points (production of a normal dystrophin transcript).

For complex diseases with a range of severities, failing the specified clinical endpoint is not the same as selling dyed water.

Is production of a normal dystrophin transcript a real clinical endpoint, or just a biomarker that's supposed to correlate for one? It's not immediately obvious how normalizing production of a protein involved in muscle function is beneficial, if it doesn't actually improve muscle function.

Absolutely. But DMD has a spectrum of severities, and it seems possible that for some patients, producing some normal transcript would make a real difference, so that until more people are treated, we cannot know when more normal transcripts make a difference.

Ensuring people have options that aren't certain death is exactly why we care about effectiveness.

Opening up a market for "certain death with extra steps" doesn't address this.

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