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(serene music)

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<v ->We'd love to hear your thoughts</v>

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on how you see innovation on the clinical development side.

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<v ->It's very hard to see how we're going to handle hundreds</v>

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of gene therapies for rare disorders.

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There are hundreds of rare disorders

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waiting to be addressed,

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and trying to get there towards hard clinical endpoints

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that meet the statutory standard

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of substantial evidence of effectiveness

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for the final clinical endpoint

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is going to be very challenging for every one of them.

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Whereas getting there for substantial evidence

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of effectiveness based on a surrogate

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to meet the accelerated approval standard,

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that's more possible, I think.

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<v ->So do you see, do you believe then,</v>

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that to address the durability issues, we'll need to,

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I guess, have a sort of more rigorous approach

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to the subsequent trials that are needed

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to demonstrate effect over the long term?

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Or how do you see the balance between what's done

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in the clinical trial setting

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versus the real world observations?

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<v ->Yeah, so I mean, the balance here</v>

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is dealing with what the underlying disorder is,

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because for a disorder that kills a child

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within five to seven years of life,

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a durability of something that gets you five to ten years

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is what parents will take because they'll say, look,

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if I can get five to ten years out of this gene therapy,

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something will come along next

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at generation two that I'll take.

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On the other hand, if you have shorter durability,

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that could be an issue.

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The other issue is that,

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when you start to think about this

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for more common disorders

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for which there might be an alternative therapy,

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then the analysis gets complicated by the economics

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of what you're doing as well.

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In other words, what can you actually charge for something

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that only gives you five years of correction on average

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when you can give another drug

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for that same five years, and it will cost less?

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So what is the incremental benefit of not needing

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to infuse yourself once a week, or once every two weeks?

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And that gets to be more complicated.

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I'm not sure I have all the answers to this,

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but there's a lot of calculus that goes into here

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that I don't think we've worked all of it out yet.