US lawmakers take FDA to task over rare diseases record
Lawmakers and patient advocates in the US are concerned that the FDA's regulation of rare disease therapies has been inconsistent and overly cautious.
Newsletters and Deep Dive digital magazine
Lawmakers and patient advocates in the US are concerned that the FDA's regulation of rare disease therapies has been inconsistent and overly cautious.
Eisai has launched a 'digital destination', called Kompass, that aims to support patients after they have been given a diagnosis of kidney cancer.
NHS patients in England with vitiligo will soon be able to access Incyte's Opzelura, the first drug shown to reverse the skin-lightening disorder.
The FDA has sketched out its 'plausible mechanism' regulatory pathway for individualised therapies that treat ultra-rare diseases.
For patients and GPs, accessing NHS services can feel like a "maze of dead ends and detours," according to a new poll.
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