Asked by: Freddie van Mierlo (Liberal Democrat - Henley and Thame)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what recent discussions he has had with NHS England on Innovative Medicines funding for givinostat.
Answered by Zubir Ahmed - Parliamentary Under-Secretary (Department of Health and Social Care)
Department officials have regular discussions with NHS England on a number of topics, including funding for innovative medicines.
The National Institute for Health and Care Excellence (NICE) makes recommendations for the National Health Service on whether new licensed medicines should be routinely funded by the NHS independently, based on an assessment of their costs and benefits. The NHS in England is legally required to fund medicines recommended by NICE, normally within three months of the publication of final guidance.
NICE is currently evaluating givinostat for the treatment of Duchenne muscular dystrophy and, following a call for evidence, its Appraisal Committee will meet to consider its recommendations on 23 October 2025. NICE currently expects to publish final guidance in January 2026.
If recommended by NICE in draft guidance, my Rt Hon. Friend, the Secretary of State for Health and Social Care, has been clear that NHS England should aim to work with the pharmaceutical company, Italfarmaco, to provide early interim funding for givinostat through the Innovative Medicines Fund, which has made available £340 million of ringfenced funding for the NHS to fund early access to medicines. This could potentially speed up access to givinostat by up to five months.
To avoid the possibility of creating a way to circumvent the appraisal process, NHS England is unable to fund givinostat prior to the publication of draft NICE guidance that recommends the treatment.
Asked by: Freddie van Mierlo (Liberal Democrat - Henley and Thame)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what steps his Department is taking to ensure that NICE appraisals for (a) Duchenne muscular dystrophy and (b) other rare, progressive conditions take into account urgency of access.
Answered by Zubir Ahmed - Parliamentary Under-Secretary (Department of Health and Social Care)
The National Institute for Health and Care Excellence (NICE) aims wherever possible to issue guidance on new medicines close to the time of licensing to ensure that patients can benefit from rapid access to clinically and cost-effective new medicines. NICE’s ability to do so is contingent on the company notifying NICE of its launch plans at an early stage and providing a timely evidence submission.
The Life Sciences Sector Plan sets out the measures we are taking that will mean that patients are able to access medicines three to six months faster, including improved alignment between decisions from the Medicines and Healthcare products Regulatory Agency and NICE guidance publication.
The Life Sciences Sector Plan is available at the following link:
Asked by: Helen Morgan (Liberal Democrat - North Shropshire)
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 increase access to Givinostat for patients in North Shropshire constituency.
Answered by Karin Smyth - Minister of State (Department of Health and Social Care)
The Department understands the impact that Duchenne muscular dystrophy has on those living with it and their families, and the urgent need for new treatment options.
The National Institute for Health and Care Excellence (NICE) makes recommendations for the National Health Service on whether new licensed medicines should be routinely funded by the NHS independently, based on an assessment of their costs and benefits. The NHS in England is legally required to fund medicines recommended by NICE, normally within three months of the publication of final guidance. NICE is currently evaluating givinostat for the treatment of Duchenne muscular dystrophy, and its independent Appraisal Committee met to consider the evidence in July 2025.
The committee identified specific areas where additional evidence is required to support a fully informed decision prior to draft guidance for consultation being published. As a result, NICE has launched a four-week targeted call for evidence from stakeholders, ahead of a further committee meeting in October.
Asked by: Iqbal Mohamed (Independent - Dewsbury and Batley)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, whether the review of the early access to medicines system will include Early Access Programmes of the type used for Givinostat; and what steps he is taking to (a) help tackle geographic variations in uptake and (b) improve equitable access to innovative treatments across the NHS.
Answered by Ashley Dalton - Parliamentary Under-Secretary (Department of Health and Social Care)
The UK Rare Diseases Framework sets out improving access to specialist care, treatments, and drugs as a priority, and health equity as a cross-cutting theme. The Department understands the importance of rapid patient access to new medicines for patients with Duchenne muscular dystrophy and has worked with NHS England to consider the issues raised by the Givinostat early access programme.
We are also working to review the effectiveness of access schemes for rare disease therapies. This will focus on the Early Access to Medicines Scheme, the Innovative Licensing and Access Pathway, and the Innovative Medicines Fund, but will broadly consider access to rare disease therapies.
Asked by: Iqbal Mohamed (Independent - Dewsbury and Batley)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, whether his Department has considered providing (a) funding and (b) other support to NHS Trusts to participate in early access programmes for (i) Givinostat for Duchenne Muscular Dystrophy and (ii) other medicines.
Answered by Karin Smyth - Minister of State (Department of Health and Social Care)
The Department understands the importance of rapid patient access to new medicines for patients with Duchenne muscular dystrophy, and has worked with NHS England to consider the issues raised by the givinostat early access programme. The first National Institute for Health and Care Excellence (NICE) committee meeting for givinostat took place on 10 July 2025, with a provisional date for a decision in September. If the manufacturer, Italfarmaco, can offer a cost-effective price to enable a positive NICE recommendation, NHS England is ready to work with the company to explore immediate funding options for the treatment.
Although medicines administered via company-sponsored early access programmes (EAPs) are licensed, EAPs do not share common clinical, data, or regulatory standards, meaning each one demands a new protocol to be devised and delivered by each participating trust, and that can create significant pressures on clinical and financial resources.
It would not be appropriate for the Government or NHS England to issue any form of national direction around the participation in these early access programmes as it would both pre-empt and undermine the role of NICE, whose very purpose is to advise the National Health Service on whether or not particular treatments should be made routinely available on the NHS.
The Government does, however, support patients getting early access to new, innovative medicines through established routes such as the Early Access to Medicines Scheme.
Asked by: Freddie van Mierlo (Liberal Democrat - Henley and Thame)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, how many patients have accessed Vamorolone since it was given NICE approval for Duchenne Muscular Dystrophy; what assessment he has made of the adequacy of availability of Vamorolone to patients with DMD in that period; and whether the NHS had fully implemented the NICE recommendation by the April 2025 deadline.
Answered by Karin Smyth - Minister of State (Department of Health and Social Care)
The National Institute for Health and Care Excellence (NICE) published its technology appraisal, titled Vamorolone for treating Duchenne muscular dystrophy in people 4 years and over, on 16 January 2025. NHS England routinely commissioned vamorolone in line with the guidance, from 16 April 2025.
When a patient is started on vamorolone, the prescribing clinician submits an electronic prior approval form, confirming that NICE’s recommendation criteria are met. NICE’s costing report, included in the technology appraisal, suggested that 1,390 people expected to receive vamorolone in 2025/26. The NICE’s technology appraisal is available at the following link:
There is only 10 full weeks of data available since routine commissioning commenced in mid-April, and over this period there have been 32 prior approval forms submitted. Whilst submission of a form is not confirmation that treatment has begun, this is used as a proxy indication for the number of patients starting treatment.
Asked by: Luke Taylor (Liberal Democrat - Sutton and Cheam)
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 help ensure that Givinostat is made available in for people with Duchenne muscular dystrophy across all NHS trusts areas.
Answered by Karin Smyth - Minister of State (Department of Health and Social Care)
The Department understands the impact that Duchenne muscular dystrophy has on those living with it and their families, and the urgent need for new treatment options.
The National Institute for Health and Care Excellence (NICE) makes recommendations for the National Health Service on whether new licensed medicines should be routinely funded by the NHS independently based on an assessment of their costs and benefits. The NHS in England is legally required to fund medicines recommended by NICE, normally within three months of the publication of final guidance. NICE is currently evaluating givinostat for the treatment of Duchenne muscular dystrophy and its independent Appraisal Committee will meet to consider the evidence in July 2025.
Ahead of NICE’s evaluation, ITF Pharma UK, the United Kingdom’s marketing authorisation holder for givinostat, is providing access to givinostat through a type of compassionate use scheme called an Early Access Programme (EAP). The delivery of timely and equitable access to new treatments for Duchenne muscular dystrophy under company-sponsored EAPs is not the responsibility of NHS England. Participation in company-led schemes is decided at an individual NHS trust level and under these programmes, the cost of the drug is free to both the patients taking part in it, and to the NHS, although NHS trusts must still cover administration costs and provide clinical resources to deliver the EAP.
NHS England has published guidance for integrated care systems (ICS) on free of charge medicines schemes, providing advice on potential financial, resourcing, and clinical risks.
ICSs should use the guidance to help determine whether to implement any free of charge scheme, including assessing suitability and any risks in the short, medium, and long term. The guidance is available at the following link:
Asked by: Alison Bennett (Liberal Democrat - Mid Sussex)
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 ensure the availability of givinostat to patients eligible through early access programmes.
Answered by Karin Smyth - Minister of State (Department of Health and Social Care)
The Department understands the impact that Duchenne muscular dystrophy has on those living with it, and their families, and the urgent need for new treatment options. If new therapies for Duchenne muscular dystrophy are approved by the National Institute for Health and Care Excellence (NICE), then appropriate commissioning plans will be put in place to enable equitable access to treatment through Specialised Neurology Services.
The delivery of timely and equitable access to new treatments for Duchenne muscular dystrophy under company-sponsored early access schemes (EAPs) is not the responsibility of NHS England. Participation in company-led schemes is decided at an individual National Health Service trust level and under these programmes, the cost of the drug is free to both the patients taking part in it, and to the NHS, although NHS trusts must still cover the administration costs and provide the clinical resources to deliver the EAP.
NHS England has published guidance for integrated care systems (ICS) on free of charge medicines schemes, providing advice on potential financial, resourcing, and clinical risks.
ICSs should use the guidance to help determine whether to implement any free of charge scheme, including assessing suitability and any risks in the short, medium, and long term. The guidance is available at the following link:
Asked by: Jim Shannon (Democratic Unionist Party - Strangford)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what steps NHS England is taking to ensure that NHS trusts are prepared to deliver (a) timely and (b) equitable access to emerging treatments for Duchenne muscular dystrophy, in the context of (i) challenges highlighted through recent early access programmes and (ii) the likelihood of these therapies entering routine commissioning.
Answered by Karin Smyth - Minister of State (Department of Health and Social Care)
The Department understands the impact that Duchenne muscular dystrophy has on those living with it and their families, and the urgent need for new treatment options. If new therapies for Duchenne muscular dystrophy are approved by the National Institute for Health and Care Excellence (NICE), then appropriate commissioning plans will be put in place to enable equitable access to treatment through Specialised Neurology Services.
The delivery of timely and equitable access to new treatments for Duchenne muscular dystrophy under company-sponsored early access schemes is not the responsibility of NHS England. Individual pharmaceutical companies may put in place Early Access Programmes (EAPs) to allow early access to new medicines that do not yet have a marketing authorisation. Participation in EAPs is decided at an individual National Health Service trust level, and under these programmes, the cost of the drug is free to both patients taking part in it, and to the National Health Service, although NHS trusts must still cover administration costs and provide clinical resources to deliver the EAP.
There are no common clinical, data, or regulatory standards for company-sponsored EAPs, meaning each one demands a new protocol to be devised and delivered by each participating trust, which can create significant pressures on clinical and financial resources. Companies providing a sponsored EAP also reserve the right to limit or to close registration of new patients at any time, meaning that any financial and clinical investment made by trusts to establish an EAP could be undermined by a commercial decision that would most likely happen in the event of the treatment not being recommended following an appraisal by NICE.
NHS England has published guidance for integrated care systems (ICS) on free of charge (FoC) medicine schemes, including EAPs, providing advice on potential financial, resourcing, and clinical risks.
ICSs should use the guidance to help determine whether to implement any FoC scheme, including assessing suitability and any risks in the short, medium, and long term. The guidance is available at the following link:
Asked by: Jim Shannon (Democratic Unionist Party - Strangford)
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, whether NHS England has made an assessment of the adequacy of the preparedness of NHS trusts to implement new treatments for Duchenne muscular dystrophy, in the context of lessons learned from recent early access programmes.
Answered by Karin Smyth - Minister of State (Department of Health and Social Care)
The Department understands the impact that Duchenne muscular dystrophy has on those living with it and their families, and the urgent need for new treatment options. If new therapies for Duchenne muscular dystrophy are approved by the National Institute for Health and Care Excellence (NICE), then appropriate commissioning plans will be put in place to enable equitable access to treatment through Specialised Neurology Services.
The delivery of timely and equitable access to new treatments for Duchenne muscular dystrophy under company-sponsored early access schemes is not the responsibility of NHS England. Individual pharmaceutical companies may put in place Early Access Programmes (EAPs) to allow early access to new medicines that do not yet have a marketing authorisation. Participation in EAPs is decided at an individual National Health Service trust level, and under these programmes, the cost of the drug is free to both patients taking part in it, and to the National Health Service, although NHS trusts must still cover administration costs and provide clinical resources to deliver the EAP.
There are no common clinical, data, or regulatory standards for company-sponsored EAPs, meaning each one demands a new protocol to be devised and delivered by each participating trust, which can create significant pressures on clinical and financial resources. Companies providing a sponsored EAP also reserve the right to limit or to close registration of new patients at any time, meaning that any financial and clinical investment made by trusts to establish an EAP could be undermined by a commercial decision that would most likely happen in the event of the treatment not being recommended following an appraisal by NICE.
NHS England has published guidance for integrated care systems (ICS) on free of charge (FoC) medicine schemes, including EAPs, providing advice on potential financial, resourcing, and clinical risks.
ICSs should use the guidance to help determine whether to implement any FoC scheme, including assessing suitability and any risks in the short, medium, and long term. The guidance is available at the following link: