Cystic Fibrosis: Medical Treatments

(asked on 17th January 2024) - View Source

Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, whether NICE’s assessment of the costs and benefits of funding a medication takes into account the size of the community affected; and what discussions NICE has had with patients on the use of Kaftrio for the treatment of cystic fibrosis.


Answered by
Andrew Stephenson Portrait
Andrew Stephenson
Minister of State (Department of Health and Social Care)
This question was answered on 23rd January 2024

The National Institute for Health and Care Excellence (NICE) does not take the size of the affected community into account when making recommendation on the clinical and cost effectiveness of medicines.

In 2017, a Budget Impact Test was introduced to mitigate the affordability challenge that higher cost medicines for large patient populations can present to the National Health Service, even at a cost-effective price. If the estimated budget impact of a medicine exceeds £20 million in any of the first three years of its use, NHS England may engage in commercial discussions with the company to mitigate the impact that funding the medicine would have on the rest of the NHS.

NICE is currently developing guidance for the NHS on the clinical and cost effectiveness of Kaftrio and other cystic fibrosis medicines and is engaging with patient groups as part of the appraisal process. NICE recently consulted on its draft recommendations and is now engaging with key stakeholders to determine the most appropriate next steps. This includes exploring potential commercial solutions.

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