I got an invitation from ….

[jon-nyc]

From his lips to gods ears.

Of course there’s such a thing as ‘too good’ - for example, they could say that they’ll give conditional approval for rare disease treatments based on safety alone and let efficacy be proven in a P4 (post marketing) study. That sounds awesome, but the reality is insurance companies wouldn’t approve it considering it ‘experimental’.

What I’d like to see is them allow more flexibility with approvals based on biomarkers rather than clinical outcomes which can take much more time to demonstrate in some diseases.

[Tom-K]

Jon, our very own "Colleague" of RFK, Jr!

[Mik]

I can’t….bear…it.

[jon-nyc]

So my flights are canceled. I guess I’m going to dial in.

When I’m not shoveling.

[89th]

Make sure to wear your jeans

[LuFins Dad]

@jon-nyc said in I got an invitation from ….:

So my flights are canceled. I guess I’m going to dial in.

When I’m not shoveling.

Snorting Coke of your own toilet just isn’t the same.

[jon-nyc]

I’m watching this online. It’s just starting. I’ll livestream anything interesting.

[jon-nyc]

11 minutes in and it’s still the banner screen and hold music.

[jon-nyc]

My friend who’s there said there was a fire alarm and they evacuated the building and brought everyone back in. “And it was very cold outside FYI”. lol

[jon-nyc]

It’s starting for real now.

[jon-nyc]

Kennedy on stage.

[jon-nyc]

It seems to be about individualized gene therapies so far (like single patient diseases - ultra rare). Like that kid in Philadelphia Children’s. They have that kid doctors on stage too.

[jon-nyc]

The plausible mechanism pathway. That would be awesome for us but it’s only for ultra-rare, which has no formal definition but probably would exclude us.

https://www.fda.gov/news-events/press-announcements/fda-launches-framework-accelerating-development-individualized-therapies-ultra-rare-diseases