EU pitches new pharma bonus to cure rare diseases. But will it work?  – POLITICO – 24 Global News | Latest International Breaking News Today

EU pitches new pharma bonus to cure rare diseases. But will it work?  – POLITICO – Latest International Breaking News Today

“Thus, depending on the ultimate definition and requirements to demonstrate [high unmet medical need], we risk that some advanced therapies, despite being transformative, miss out on the label,” Natz added.

Government agencies that decide whether to pay for new drugs tend to resist efforts to dilute evidence standards. They argue they need as much clinical trial data as possible to judge whether a new drug is worth their money.

That’s especially true of gene therapies, which often come with an eye-watering price tag. In the U.S., Lenmeldy, a treatment for the rare disorder metachromatic leukodystrophy, has a list price of $4.25 million, making it the most expensive drug in the world.

 Speaking at the same working group, a Commission official who asked to remain anonymous defended the proposal, noting the bonus would be on top of the existing orphan drugs framework, and that a medicine that didn’t qualify for high unmet medical need would still be incentivized.

The official also pointed out that the orphan drug designation is also confirmed at the time of approval, and that it has been successful in stimulating orphan drug development.

Moreover, for medicines that don’t qualify for high unmet medical need, other incentives are available such as the PRIME designation, which grants regulatory support and a quicker assessment. Orphan medicines that don’t qualify for high unmet medical need can still benefit from scientific advice on the conduct of studies and are eligible for national and EU incentives to support research in rare diseases.

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“We continue to have [a] very competitive system of incentives in Europe,” the official said.

This article is the product of a POLITICO Working Group presented by Sanofi and was produced with full editorial independence by POLITICO reporters and editors. Learn more about editorial content presented by outside advertisers.

-24 Global News | Latest International Breaking News Today
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