Orkambi

(asked on 5th February 2018) - View Source

Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what assessment he has made of the effectiveness of Kalydeco as a treatment for people with cystic fibrosis.


Answered by
Steve Brine Portrait
Steve Brine
This question was answered on 8th February 2018

During the last five years, NHS England commissioned a health technology assessment to review the clinical evidence for Kalydeco (Ivacaftor) and decided at the time that it would support its use to treat one gene mutation. Subsequently it has broadened use for other gene mutations following further evidence reviews.

For commissioned drugs such as Kalydeco (Ivacaftor) for named mutations, prescribing decisions are made by multidisciplinary teams in hospitals commissioned by NHS England to provide specialised services for adults and children with cystic fibrosis.

NHS England is investing significant resources into the provision of Kalydeco which works directly on the genes causing cystic fibrosis and in medicines that reduce the impact of the disease.

NHS England and the Cystic Fibrosis Trust jointly collect outcomes data through the Cystic Fibrosis Registry to inform better management of the disease.

Information about specialised National Health Service commissioning is available at the following link:

https://www.england.nhs.uk/commissioning/spec-services/npc-crg/group-a/a01/

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