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(serene music)
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We'd love to hear your thoughts
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on how you see innovation on the clinical development side.
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It's very hard to see how we're going to handle hundreds
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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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So do you see, do you believe then,
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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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Yeah, so I mean, the balance here
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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.