Asked by: Cheryl Gillan (Conservative - Chesham and Amersham)
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 suitability of the Single Technology Appraisal process for new treatments for patients with Duchenne Muscular Dystrophy.
Answered by Steve Brine
The National Institute for Health and Care Excellence (NICE) is the independent body responsible for developing authoritative, evidence-based guidance for the National Health Service on whether drugs and other treatments represent an effective use of NHS resources.
NICE assesses most significant new drugs through its technology appraisal programme and has been able to recommend a number of drugs licenced for the treatment of rare diseases for routine use on the NHS. NICE also operates a separate highly specialised technology (HST) evaluation programme for the assessment of very high cost drugs for the treatment of very small numbers of patients. Decisions on the most appropriate programme for individual drugs and other treatments are made through an established topic selection process that includes a public consultation and thorough consideration against a set of published criteria.
NICE published HST guidance recommending ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in July 2016 and is currently developing guidance, through its technology appraisal and HST programmes, on other drugs for the treatment of Duchenne muscular dystrophy.
Asked by: Cheryl Gillan (Conservative - Chesham and Amersham)
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 potential merits of increasing flexibility within NICE's Single Technology Appraisal process for medicines for rare diseases.
Answered by Steve Brine
The Department has made no such assessment. The National Institute for Health and Care Excellence (NICE) is the independent body responsible for developing authoritative, evidence-based guidance for the NHS on whether drugs and other treatments represent an effective use of National Health Service resources.
NICE assesses most significant new drugs through its technology appraisal programme and has been able to recommend a number of drugs licenced for the treatment of rare diseases for routine use on the NHS. NICE also operates a separate highly specialised technology evaluation programme for the assessment of very high cost drugs for the treatment of very small numbers of patients.
NICE’s methods and processes for the assessment of drugs have been carefully developed over time and are internationally respected. NICE continues to keep its procedures under periodic review to ensure that they remain effective.
Asked by: Cheryl Gillan (Conservative - Chesham and Amersham)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what representations his Department and its agencies have received on the resources necessary to improve patient safety and engage with health professionals to raise awareness of the potential health risks for women of child-bearing age taking Sodium Valproate; and if he will make a statement.
Answered by Jackie Doyle-Price
The Medicines and Healthcare products Regulatory Agency (MHRA) is working in partnership with the Department, professional bodies and the healthcare system to deliver a package of measures to raise awareness of the significant risks associated with sodium valproate in pregnancy and to support healthcare professionals in reducing the harms from valproate in women of child-bearing age and improving patient safety in line with the strengthened regulatory position. Educational materials for healthcare professionals and patients are being sent to general practitioners (GP) and specialist prescribers. The National Institute for Health and Care Excellence has updated its guidelines which mention valproate to reflect the new regulatory measures. GP electronic system providers have provided a search and audit function to facilitate the identification of women of childbearing age on valproate and are updating the alerts for valproate.
All parties involved are fully committed to this important work using the resources available from each relevant organisation. In addition, since 2016 the MHRA has been working with a network of over 40 stakeholder organisations (termed the Valproate Stakeholders’ Network) to foster cross-sector support for the new measures and leverage their collective communications reach.
The effectiveness of the new measures in reducing prescribing of valproate in women of childbearing potential will be closely monitored. Relevant data will be published and there will be ongoing follow-up to ensure that the harms to the child from valproate in pregnancy are minimised.
I would particularly like to thank the families involved in the Valproate Stakeholder Network who have shared their experiences and expertise. Their dedication, support and altruism will help to keep future generations of children safe.