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The National Institute for Health and Care Excellence (NICE) published draft guidance for consultation on 23 March 2016 which did not recommend Orkambi for treating cystic fibrosis on cost grounds.

Ms Qureshi, said: “I am concerned that the NICE medical technologies evaluation programme could limit access to medicines for people with rare diseases, such as cystic fibrosis, and I know that there are widely-held concerns that the process for appraising medicines for rare diseases is opaque and in some regards out of date.

“I also fear that the current system of encouraging investment in technology but not allowing patient access to it is unsustainable and wrong and, without reform, could result in a reduction in funding for research into medicines for rare diseases.”

Ms Qureshi, added: “I have written to the Minister of Life Sciences, George Freeman MP, urging him to facilitate discussions between NHS England and NICE to discuss these proposals.”

Orkambi is a precision medicine for tackling the underlying cause of cystic fibrosis, and I appreciate that most current treatments generally target the complications, rather than the cause of the condition.

Cystic Fibrosis Trust has expressed concern that the NICE decision highlights “a system unable to cope with the challenges of assessing rare-disease medicines”, and is urging the Government to consider a proposal it has put forward which would enable more people to access Orkambi.

It is hoped the Government will listen to the concerns that have been expressed on this issue, and continue to engage with relevant charities such as the Cystic Fibrosis Trust on matters such as this.

The consultation on the draft guidance by NICE closes on 15 April 2016 and this is an opportunity for all interested parties to put forward their comments on these preliminary recommendations.

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