[tptacek]

I worried about the word "clinical" here, which might not be what I meant. It is not, however, inaccurate to say that concerns about efficacy were behind EU health system refusal to pay, and behind the therapy being taken off the market.

[ethbro]

True. The question seems to be correlating the long-term decrease in serious episodes and gene expression (observed, albeit in a low sample size) with a marker (plasma TG did not change, but centrifuged TG composition by weight did).

The injustice here, if it exists, seems to be that the system is poorly configured to treat rare diseases.

Afaik, the FDA (regulatory side, for non-US readers) began granting more permissive experimental waivers for this sort of thing.

But the insurance side seems well within their rights to not pay by saying "This treatment has not been demonstrated effective to our standards." Something which is likely impossible given the cost of running normal trials and target population (small).

Hopefully, in the future, this will be addressed as more gene therapies become available and individualized medicine becomes the norm (similar to the revolution oncology has gone through in the past 30 years).