It is for the marketing authorisation holder to propose a complex patient access scheme for a medicine being evaluated by the National Institute for Health and Care Excellence (NICE), which would then be considered by the NICE and NHS England through the established process. The marketing authorisation holder has proposed a commercial agreement for ruxolitinib but the NICE was unable to recommend it as a clinically and cost effective use of National Health Service resources at the price proposed in its final draft guidance, published in July 2024. A number of organisations appealed against the draft guidance. The NICE has not yet published final guidance, and its Appraisal Committee will consider the upheld appeal points at a future meeting, scheduled for 14 May 2025. The NICE is open to working with NHS England to ensure that commercial arrangements that are operationally manageable for the NHS can be taken into account.

Where the NICE does not recommend a treatment as a clinically and cost-effective use of NHS resources, it will not usually be routinely funded by the NHS. NHS commissioners are, however, required to consider exceptional funding requests for individual patients.

The Government wants National Health Service patients to be able to benefit from access to clinically and cost-effective new treatments, and the National Institute for Health and Care Excellence (NICE) makes recommendations for the NHS on whether new medicines should be routinely funded by the NHS based on an assessment of their costs and benefits. The NICE is currently developing guidance for the NHS on whether ruxolitinib should be routinely funded by the NHS, based on an assessment of its costs and benefits.

The NICE was unfortunately unable to recommend ruxolitinib as a clinically and cost-effective use of NHS resources in its final draft guidance published on 18 July. The NICE received four appeals against its draft recommendation which were heard by an independent panel on 11 October 2024. The appeal panel remitted the evaluation of ruxolitinib to the appraisal committee with the instruction to take all reasonable steps to address the issues raised in the upheld appeal points. The committee will consider the upheld appeal points at a future meeting scheduled for 14 May 2025.

It is right that the NICE makes its decisions at arm’s length of the Government and in line with its carefully developed methods and processes. As such, it would not be appropriate for me to intervene in the NICE’s decision-making.

The National Institute for Health and Care Excellence (NICE) recommends ruxolitinib as an option for treating disease-related splenomegaly or symptoms in adults with primary myelofibrosis, also known as chronic idiopathic myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis, in people with intermediate-2 or high-risk disease. Ruxolitinib is available to National Health Service patients in line with NICE’s recommendations.

Information on the number of patients who self-fund ruxolitinib for myelofibrosis is not held centrally.

The National Institute for Health and Care Excellence (NICE) is currently developing guidance for the National Health Service on whether ruxolitinib should be routinely funded by the NHS, based on an assessment of its costs and benefits.

The NICE was unfortunately unable to recommend ruxolitinib as a clinically and cost-effective use of NHS resources in its final draft guidance published on 18 July. The NICE received four appeals against its draft recommendation which were heard by an independent panel on 11 October 2024. If any of the appeals are upheld, the NICE’s appraisal committee will reconsider its recommendations in light of the appeal panel’s decision.

It is right that the NICE makes its decisions at arm’s length of the Government and in line with its carefully developed methods and processes. As such, it would not be appropriate for ministers to intervene in the NICE’s decision-making.

Opzelura (ruxolitinib), is currently not approved by the Medicines and Healthcare products Regulatory Agency (MHRA). The MHRA has noted that the European Medicines Agency’s Committee for Medicinal Products for Human Use issued a positive opinion for this on the 23 of February 2023 and is currently pending European Commission (EC) approval.

Should an application for Opzelura be received by the MHRA, we have processes in place to review this and, if appropriate, issue a licence in line with an EC decision.

Reports of suspected adverse drug reactions (ADRs) are collected by the Medicines and Healthcare products Regulatory Agency (MHRA) and Commission for Human Medicines through the spontaneous reporting scheme, the Yellow Card Scheme. The Yellow Card Scheme collects information relating to suspected adverse drug reactions, which include both serious and non-serious effects which a health care professional or patient suspect may have been due to a medicine.

The MHRA has received a total of 74 United Kingdom spontaneous suspected ADR reports in association with ruxolitinib up to and including 10 February 2015. This number includes reports received directly from healthcare professionals, patients and indirectly through pharmaceutical companies which have a legal obligation to report suspected ADRs.

The inclusion of a particular ADR in a report does not necessarily mean it has been caused by the drug, only that the reporter had a suspicion it may have, or it had a close temporal relationship to the administration of the suspect drug. The fact that symptoms occur after a treatment does not necessarily mean that they have been caused by the drug itself, as underlying illnesses and other conditions may be responsible.

In 2013, the product information for ruxolitinib was updated to warn healthcare professionals that cases of progressive multifocal leukoencephalopathy (PML) have been reported with ruxolitinib and to recommend that patients are monitored at regular intervals for any new or worsening neurological symptoms or signs. The Market Authorisation Holder is currently conducting studies to investigate the risk of PML further. The benefits of ruxolitinib to adult patients with myelofibrosis who have an enlarged spleen or symptoms related to the disease are considered to outweigh the risks.