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Blue Note leukaemia DTx gets FDA breakthrough tag

pharmaphorum

Breakthrough designations can speed up the development of new therapies, making them available to patients sooner. It is well-established for conventional drugs and medical devices but has only recently started to be used for DTx. The post Blue Note leukaemia DTx gets FDA breakthrough tag appeared first on.

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Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years

Pharmaceutical Technology

This year has already been eventful when it comes to the development of therapies for rare diseases. Additionally, pricing and access for rare disease therapies continue to be scrutinized closely. Most, if not all, of these therapies used the FDA’s Orphan Drug Designation to aid their development plans.