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Dr Aimee Tharaldson Discusses the Orphan Drug Approval Trend

Video

About 40% of new specialty drugs in 2017 are expected to be for orphan drug indications, which is keeping with the trend; however, an upcoming investigation into potential abuses of the Orphan Drug Act could have an unknown impact on development and prices, said Aimee Tharaldson, PharmD, senior clinical consultant for emerging therapeutics at Express Scripts.

About 40% of new specialty drugs in 2017 are expected to be for orphan drug indications, which is keeping with the trend; however, an upcoming investigation into potential abuses of the Orphan Drug Act could have an unknown impact on development and prices, said Aimee Tharaldson, PharmD, senior clinical consultant for emerging therapeutics at Express Scripts.

Transcript

What was the 2016 trend for specialty drugs approved for orphan indications and what do you expect for 2017?

So last year in 2016 about half of new specialty drug approvals were for orphan conditions. Back in 2015, actually two-thirds were orphan drugs. Looking at the pipeline, the near-term pipeline indicates that about 35% to 40% of the new specialty drugs that are going to be approved this year will be for orphan indications as well. So this is kind of a range of somewhere between 40% to even 60%, I think look forward in the long term will be for orphan indications.

Now the Government Accountability Office is investigating potential abuses to the Orphan Drug Act by certain manufacturers. That's going to occur in about 9 months from now. Really don't know the impact that that's going to have in the long term. Possibly, we'll see more moderate prices for these orphan medications over the long term. But that's still yet to be seen. So it's definitely an interesting area to keep an eye on.

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