Commissioners have a statutory responsibility to make funding available for a medicine or treatment recommended by a National Institute of Health and Care Excellence (NICE) technology appraisal (TA) or highly specialised technology evaluation (HST) within the timeframe recommended in that guidance, usually within three months of the TA or HST being published.

Under the National Health Service Constitution, patients have a right to receive all medicines and treatments recommended by NICE if they and their healthcare professional think that the medicine is right for them. In practical terms, the effect of this legal obligation and the NHS constitution is that all NICE-approved treatments must be included in local formularies for use in line with the TA or HST recommendations and with no additional funding or formulary restrictions.

The only exception is if the technology is not relevant to the care provided by the organisation; for example, cancer treatments would not need to be included in the formulary of a mental health trust, and treatments for dementia would not need to be included in the formulary of a specialist children’s hospital. There is no provision to take affordability into account when adding NICE-approved medicines to local formularies.

NHS enforcement guidance relating to its statutory accountability and oversight role, under the NHS Act 2006, the 2012 Act and the 2022 Act is available at the following link:

https://www.england.nhs.uk/long-read/nhs-enforcement-guidance/

The evolving NHS operating model will strengthen capabilities and clarify roles in overseeing performance. As set out in the 10-Year Health Plan for England, we will move to a Single National Formulary (SNF) for medicines within the next two years. The SNF will play a role in driving rapid and equitable adoption of clinically and cost-effective innovations.

The National Health Service trusts which have submitted prior approval forms are as follows:

• Alder Hey Children's NHS Foundation Trust;
• Cambridge University Hospitals NHS Foundation Trust;
• Great Ormond Street Hospital for Children NHS Foundation Trust;
• Guy's and St Thomas' NHS Foundation Trust;
• Nottingham University Hospitals NHS Trust;
• The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust;
• University Hospital Southampton NHS Foundation Trust;
• University Hospitals Birmingham NHS Foundation Trust; and
• University Hospitals Bristol and Weston NHS Foundation Trust.

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:

https://www.nice.org.uk/guidance/ta1031/resources/resource-impact-summary-report-15239352925/chapter/Resource-impact-summary-report

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.