(serene music)
-: We'd love to hear your thoughts
on how you see innovation on the clinical development side.
-: It's very hard to see how we're going to handle hundreds
of gene therapies for rare disorders.
There are hundreds of rare disorders
waiting to be addressed,
and trying to get there towards hard clinical endpoints
that meet the statutory standard
of substantial evidence of effectiveness
for the final clinical endpoint
is going to be very challenging for every one of them.
Whereas getting there for substantial evidence
of effectiveness based on a surrogate
to meet the accelerated approval standard,
that's more possible, I think.
-: So do you see, do you believe then,
that to address the durability issues, we'll need to,
I guess, have a sort of more rigorous approach
to the subsequent trials that are needed
to demonstrate effect over the long term?
Or how do you see the balance between what's done
in the clinical trial setting
versus the real world observations?
-: Yeah, so I mean, the balance here
is dealing with what the underlying disorder is,
because for a disorder that kills a child
within five to seven years of life,
a durability of something that gets you five to ten years
is what parents will take because they'll say, look,
if I can get five to ten years out of this gene therapy,
something will come along next
at generation two that I'll take.
On the other hand, if you have shorter durability,
that could be an issue.
The other issue is that,
when you start to think about this
for more common disorders
for which there might be an alternative therapy,
then the analysis gets complicated by the economics
of what you're doing as well.
In other words, what can you actually charge for something
that only gives you five years of correction on average
when you can give another drug
for that same five years, and it will cost less?
So what is the incremental benefit of not needing
to infuse yourself once a week, or once every two weeks?
And that gets to be more complicated.
I'm not sure I have all the answers to this,
but there's a lot of calculus that goes into here
that I don't think we've worked all of it out yet.