Asked by: Steve McCabe (Labour - Birmingham, Selly Oak)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, if she will take steps to make (a) Orkambi, (b) Symkevi and (c) Kaftrio available for everyone with cystic fibrosis.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
Since 2019, patients in the National Health Service have been able to access Orkambi, Symkevi and Kaftrio through an interim access agreement between NHS England, the company (Vertex), the National Institute for Health and Care Excellence (NICE) and the UK Cystic Fibrosis Trust.
The agreement makes the medicines available for a limited time at a reduced price, while further information has been collected to inform a NICE appraisal. On 15 November 2023, the Medicines and Healthcare products Regulatory Agency (MHRA) approved a new licence extension and children as young as two years old with cystic fibrosis are now eligible to receive Kaftrio through the interim access agreement.
NICE is currently consulting on its draft recommendations on the use of Orkambi, Symkevi and Kaftrio and has not yet published its final recommendations. NICE continues to work with stakeholders to address the issues highlighted by the committee in the draft guidance. Under the terms of the interim access agreement for the cystic fibrosis medicines, Orkambi, Symkevi and Kaftrio, eligible children and adults with cystic fibrosis can continue to receive ongoing treatment and be initiated onto treatment with these medicines, as clinically appropriate, while NICE concludes its evaluation.
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 whether new or existing patients with Cystic Fibrosis will continue to have access to modular drugs through the NHS to treat that condition.
Answered by Lord Markham - Parliamentary Under-Secretary (Department of Health and Social Care)
Since 2019, National Health Service patients have been able to access Orkambi, Symkevi and Kaftrio through an interim access agreement between NHS England, the company Vertex, the National Institute for Health and Care Excellence (NICE) and the UK Cystic Fibrosis Trust. The agreement makes the medicines available for a limited time at a reduced price, while further information has been collected to inform a NICE appraisal. On 15 November 2023, the Medicines and Healthcare products Regulatory Agency approved a new licence extension and children as young as two years old with cystic fibrosis are now eligible to receive Kaftrio through the interim access agreement.
NICE is currently consulting on its draft recommendations on the use of Orkambi, Symkevi and Kaftrio and has not yet published its final recommendations. NICE continues to work with stakeholders to address the issues highlighted by the committee in the draft guidance. Under the terms of the interim access agreement for the cystic fibrosis medicines, Orkambi, Symkevi and Kaftrio, eligible children and adults with cystic fibrosis can continue to receive ongoing treatment and be initiated onto treatment with these medicines, as clinically appropriate, while NICE concludes its evaluation.
Asked by: Ian Byrne (Labour - Liverpool, West Derby)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, pursuant to the Answer of 9 November 2023 to Question 346 on Cystic Fibrosis: Drugs, whether her Department plans to measure the long-term health outcomes of the cohort of people with cystic fibrosis who have been treated with Orkambi, Symkevi or Kaftrio through the NHS.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
Long-term health outcomes of the cohort of people with cystic fibrosis who have been treated with Orkambi, Symkevi or Kaftrio through the National Health Service are collected by the UK Cystic Fibrosis Registry.
Almost all people with cystic fibrosis in the United Kingdom consent to clinical teams submitting data on an annual basis about their medications including cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy and their clinical outcomes to the UK Cystic Fibrosis Registry, which publishes an annual report. Under the commercial arrangement with Vertex for CFTR modulator therapies real-world evidence is also collected.
Asked by: Catherine West (Labour - Hornsey and Wood Green)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, whether it is his Department's policy that people with cystic fibrosis will have access to (a) Orkambi, (b) Symkevi and (c) Kaftrio through the NHS.
Answered by Will Quince
Orkambi, Symkevi and Kaftrio are currently available as treatment options for eligible National Health Service patients with cystic fibrosis, under the terms of a commercial agreement reached between NHS England and the manufacturer. This agreement has enabled the collection of data to inform a full appraisal of these medicines by the National Institute for Health and Care Excellence (NICE), which is currently underway.
NICE published draft guidance on 3 November 2023 which does not recommend Orkambi, Symkevi and Kaftrio for the treatment of cystic fibrosis. The draft guidance is now subject to a public consultation and NICE’s committee will carefully consider all evidence as well as comments received during the consultation in developing its final guidance, which is expected to be published on 20 March 2024. Patients already receiving these treatments will have continued access following the publication of NICE’s final guidance, regardless of the outcome.
Asked by: Burnett, Alexander (Scottish Conservative and Unionist Party - Aberdeenshire West)
Question
To ask the Scottish Government what discussions it has had regarding modulator therapies to treat cystic fibrosis, such as Orkambi, Symkevi and Kaftrio, being funded on the NHS.
Answered by Minto, Jenni - Minister for Public Health and Women's Health
I refer the member to the answer to question S6W-22774 on 21 November 2023. All answers to written Parliamentary Questions are available on the Parliament's website, the search facility for which can be found at https://www.parliament.scot/chamber-and-committees/written-questions-and-answers.
The other two medicines alluded to in that answer are Orkambi ® , and Symkevi ® .
If any cystic fibrosis medicine is not recommended in the final multiple technology appraisal (MTA) guidance, clinicians in Scotland will be able to request the use of that medicine on a case-by-case basis for individual patients using the Peer Approved Clinical System (PACS) Tier Two process. National guidance explicitly states that Health Boards should not take account of the cost of medicines when considering applications using the PACS Tier Two system.
Asked by: Margaret Greenwood (Labour - Wirral West)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what recent discussions she has had with people with (a) cystic fibrosis and (b) representative organisations on the draft guidance on the future availability of (i) Orkambi, (ii) Symkevi and (iii) Kaftrio on the NHS.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
The National Institute for Health and Care Excellence (NICE) is an independent body and is responsible for leading engagement with interested parties in the development of its recommendations on medicines. NICE has recently consulted on its draft guidance on the use of Orkambi, Symkevi and Kaftrio for the treatment of cystic fibrosis and continues to work with stakeholders to address the issues highlighted by the committee in the draft guidance. The Department encourages stakeholders such as patients and representative organisations to continue to engage in the NICE appraisal process.
Asked by: Neil O'Brien (Conservative - Harborough)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, with reference to the recommendation from NICE that modulator therapies used to treat cystic fibrosis should no longer be funded on the NHS, when she plans to make a decision on this matter: and what steps her Department is taking to ensure that life-saving treatment is made available for children with cystic fibrosis.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
The National Institute for Health and Care Excellence (NICE) is the independent body that makes evidence-based recommendations for the National Health Service on whether medicines should be routinely funded by the NHS based on an assessment of their costs and benefits. NICE ensures that the price that the NHS pays for medicines represents value and it recommends the vast majority of medicines for use on the NHS, often as a result of commercial arrangements proposed during the NICE evaluation process. The NHS is legally required to fund medicines recommended by NICE.
Since 2019, NHS patients have been able to access Orkambi, Symkevi and Kaftrio through an interim access agreement between NHS England, the company (Vertex), NICE, and the UK Cystic Fibrosis Trust. The agreement makes the medicines available for a limited time at a reduced price, while further information has been collected to inform a NICE appraisal. On 15 November 2023, the Medicines and Healthcare products Regulatory Agency approved a new licence extension and children as young as two years old with cystic fibrosis are now eligible to receive Kaftrio through the interim access agreement.
NICE is currently consulting on its draft recommendations on the use of Orkambi, Symkevi and Kaftrio and has not yet published its final recommendations. NICE continues to work with stakeholders to address the issues highlighted by the committee in the draft guidance. Under the terms of the interim access agreement for the cystic fibrosis medicines, Orkambi, Symkevi and Kaftrio, eligible children and adults with cystic fibrosis can continue to receive ongoing treatment and be initiated onto treatment with these medicines, as clinically appropriate, while NICE concludes its evaluation.
Asked by: Margaret Greenwood (Labour - Wirral West)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, with reference to the draft guidance published by NICE entitled Ivacaftor–tezacaftor–elexacaftor, tezacaftor–ivacaftor and lumacaftor–ivacaftor for treating cystic fibrosis, published on 3 November 2023, whether she has made an assessment of the potential impact of the recommendation that modular therapies should not be provided on the NHS on the health of people with cystic fibrosis.
Answered by Andrew Stephenson - Minister of State (Department of Health and Social Care)
The National Institute for Health and Care Excellence (NICE) has recently consulted on its draft recommendations on the use of lumacaftor–ivacaftor (Orkambi), tezacaftor–ivacaftor (Symkevi) and ivacaftor–tezacaftor–elexacaftor (Kaftrio) and has not yet published its final recommendations. NICE continues to work with stakeholders to address the issues highlighted by the committee in the draft guidance. Under the terms of the interim access agreement for the cystic fibrosis medicines, Orkambi, Symkevi and Kaftrio, eligible children and adults with cystic fibrosis can continue to receive ongoing treatment and be initiated onto treatment with these medicines, as clinically appropriate, while NICE concludes its evaluation.
Asked by: Gregory Campbell (Democratic Unionist Party - East Londonderry)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, if he will take steps to ensure the continued NHS funding of modulator therapies that are used to treat (a) children, (b) new patients and (c) other patients with cystic fibrosis.
Answered by Will Quince
Orkambi, Symkevi and Kaftrio are currently available as treatment options for eligible National Health Service patients with cystic fibrosis, under the terms of a commercial agreement reached between NHS England and the manufacturer. This agreement has enabled the collection of data to inform a full appraisal of these medicines by the National Institute for Health and Care Excellence (NICE), which is currently underway.
NICE published draft guidance on 3 November 2023 which does not recommend Orkambi, Symkevi and Kaftrio for the treatment of cystic fibrosis. The draft guidance is now subject to a public consultation and NICE’s committee will carefully consider all evidence as well as comments received during the consultation in developing its final guidance, which is expected to be published on 20 March 2024. Patients already receiving these treatments will have continued access following the publication of NICE’s final guidance, regardless of the outcome.
Asked by: Gulhane, Sandesh (Scottish Conservative and Unionist Party - Glasgow)
Question
To ask the Scottish Government how many people are currently receiving cystic fibrosis transmembrane conductance regulator (CFTR) modulator treatments, broken down by the number that are receiving (a) Kaftrio, (b) Symkevi and (c) Orkambi.
Answered by Minto, Jenni - Minister for Public Health and Women's Health
The information requested is not held centrally by the Scottish Government.