Asked by: Kerry McCarthy (Labour - Bristol East)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, with reference to section 6 of the National Institute for Health and Care Excellence's (NICE) health technology evaluations: the manual, published on 31 January 2022, whether she has had discussions with NICE on the potential impact of the severity modifier on technology appraisals for (a) cystic fibrosis and (b) other rare diseases.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
Departmental officials regularly discuss a range of issues with the National Institute for Health and Care Excellence (NICE), including in relation to the application of the severity modifier that was introduced in 2022. NICE is responsible for determining whether a severity weighting should apply to the evaluation of individual medicines in line with its published methods and processes. NICE has recently published draft guidance for consultation on the use of Orkambi, Symkevi and Kaftrio for the treatment of cystic fibrosis and stakeholders now have an opportunity to comment on NICE’s recommendations. NICE continues to work with stakeholders to address the issues highlighted by the committee in the draft guidance.
Asked by: Lord Weir of Ballyholme (Democratic Unionist Party - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government what new treatments or strategies are planned to support sufferers of Cystic Fibrosis through the NHS.
Answered by Lord Markham - Parliamentary Under-Secretary (Department of Health and Social Care)
NHS England commissions 47 specialised cystic fibrosis centres for adults and children across England, in addition to supporting the optimal monitoring of patients with cystic fibrosis at home, and best practice in remote consultations.
Through these centres, NHS England provides a range of innovative treatments including inhaled therapies such as nebulised antibiotics. Since 2019, thousands of people with cystic fibrosis have been able to benefit from licensed treatments, firstly Orkambi and Symkevi and then Kaftrio, following its marketing authorisation in 2020. Access to these treatments is provided under the terms of a commercial agreement reached between the manufacturer, Vertex and NHS England, with the full support of the National Institute for Health and Care Excellence (NICE).
Within the agreement between NHS England and Vertex, there is a flexible commercial mechanism to ensure continued access for patients already receiving any of the licensed treatments following the conclusion of a full NICE evaluation.
On 3 November 2023, NICE published a consultation on draft guidance that did not recommend these treatments as a cost-effective use of National Health Service resources. This draft is an initial step in the review of these medicines and does not affect patients’ continued access to these medicines on the NHS in any way. Eligible children and adults with cystic fibrosis can continue to receive ongoing treatment and be initiated onto treatment with these medicines, as clinically appropriate.
On 15 November 2023, the Medicines and Healthcare products Regulatory Agency approved a new licence extension that will mean children as young as two years old with cystic fibrosis are eligible to receive Kaftrio.
NHS England anticipate that as many as 600 children could benefit from this latest approval under the terms of the existing commercial agreement negotiated with Vertex in 2019. NHS cystic fibrosis centres across the country have plans in place to ensure that all children eligible today can be provided long term access to this life-changing treatment.
Asked by: John Hayes (Conservative - South Holland and The Deepings)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what steps he is taking to improve care for patients with cystic fibrosis.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
NHS England commissions 47 specialised cystic fibrosis centres for adults and children across England, in addition to supporting the optimal monitoring of patients with cystic fibrosis at home, and supporting best practice in remote consultations.
Through these centres, NHS England provides a range of innovative treatments including inhaled therapies such as nebulised antibiotics. Since 2019, thousands of people with cystic fibrosis have been able to benefit from licensed treatments – firstly Orkambi and Symkeviand then Kaftrio, following its marketing authorisation in 2020. Access to these treatments is provided under the terms of a commercial agreement reached between the manufacturer, Vertex and NHS England, with the full support of the National Institute for Health and Care Excellence (NICE).
Within the agreement between NHS England and Vertex, there is a flexible commercial mechanism to ensure continued access for patients already receiving any of the licensed treatments following the conclusion of a full NICE evaluation.
On 3 November 2023, NICE published a consultation on draft guidance that did not recommend these treatments as a cost-effective use of NHS resources. This draft is an initial step in the review of these medicines and does not affect patients’ continued access to these drugs on the NHS in any way. Eligible children and adults with cystic fibrosis can continue to receive ongoing treatment and be initiated onto treatment with these drugs, as clinically appropriate.
NHS England remains committed to ensuring that these life-changing cystic fibrosis medicines are available to everyone who may benefit, now and in the future, and will continue to work with Vertex and NICE through the next stages of the appraisal process to make this possible in a way that is fair to patients and taxpayers.
Mentions:
1: Baillie, Jackie (Lab - Dumbarton) It is important to say, though, that those already receiving Orkambi, Symkevi and Kaftrio will continue - Speech Link
Mentions:
1: Ian Paisley (DUP - North Antrim) According to National Institute for Health and Care Excellence guidance, while Orkambi, Symkevi and Kaftrio - Speech Link
Mentions:
1: Baillie, Jackie (Lab - Dumbarton) People across Scotland are waiting to find out whether life-changing drugs such as Orkambi, Symkevi and - Speech Link
Mentions:
1: Kerry McCarthy (Lab - Bristol East) A new generation of drugs, Orkambi, Symkevi and Kaftrio, is transforming the lives of patients with cystic - Speech Link
Mentions:
1: Lee Anderson (Con - Ashfield) Last month, the National Institute for Health and Care Excellence said that Kaftrio, Orkambi and Symkevi - Speech Link
Mentions:
1: Kerry McCarthy (LAB - Bristol East) great news and I congratulate all those who have been involved in developing these drugs—Kalydeco, Orkambi - Speech Link
Mentions:
1: Crispin Blunt (CON - Reigate) reputation ahead of the evidence and what we ought to enable for our citizens, shame on us.In the case of Orkambi - Speech Link
2: Maria Caulfield (CON - Lewes) Orkambi has been mentioned; the issue with that was not licensing—it had a licence—but NHS funding. - Speech Link