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NICE pauses new drug appraisal

CLDF is extremely disappointed and frustrated to learn that NICE, the National Institute for Clinical Excellence, is suspending its appraisal of Maralixibat as a treatment for cholestatic pruritus in patients with Alagille Syndrome.

We’ve been informed that the Appraisal Committee would not be able to recommend its routine use in NHS services due to significant cost-effectiveness issues. Therefore, the appraisal has been suspended to give the manufacturer, Mirum, more time to discuss and agree on a commercial arrangement with NHS England.

CLDF has been fully involved in the appraisal process, which has been ongoing for approximately two years. Our involvement has included working with several parent/patient representatives who agreed to represent the voices of patients and carers in the Appraisal Committee Meeting.

We hope the appraisal will resume soon, and we will continue to be closely involved alongside parent/patient representatives, NICE, NHS England, and the manufacturer.

We will keep you informed of developments as we receive them. In the meantime, if you have questions, queries or concerns, please email or call 0121 212 6009.

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