EU pitches new pharma bonus to cure rare diseases. But will it work?  – POLITICO

“So, depending on the final definition and requirements to be demonstrated (high unmet medical need), we risk that some advanced therapies, although transformative, may not be on the label,” Natz added.

Government agencies that decide whether to fund the purchase of new drugs tend to resist efforts to dilute standards of evidence. They argue that they need as much clinical trial data as possible to judge whether a new drug is worth it.

This is particularly true for gene therapies, which often come with an exorbitant price tag. In the United States, Lenmeldy, treatment for metachromatic leukodystrophy, a rare disease, has a list price of $4.25 million, making it THE most expensive drug in the world.

Speaking before the same working group, a Commission official, who asked to remain anonymous, defended the proposal, stressing that the bonus would be on top. of the existing orphan drug framework, and that a drug that does not meet the criteria of high unmet medical need would still be encouraged.

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

Additionally, for drugs that do not address significant unmet medical needs, other incentives are available, such as PRIME designation, which grants regulatory support and more rapid review. Orphan drugs that do not qualify to address high, unmet medical needs may nevertheless benefit from scientific advice on the conduct of studies and are eligible for national and European incentives to support research into rare diseases.

“We continue to have a very competitive incentive system in Europe,” the official said.

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