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New drug for patients with myelofibrosis

written by

Leukaemia Care, Charity

  • Conference

Ruxolitinib to be recommended for intermediate and high-risk myelofibrosis patients in England and Wales following a joint response by Leukaemia CARE and MPN Voice.

The National Institute for Health and Care Excellence (NICE) have today published the Final Appraisal Determination for the clinical use of ruxolitinib (marketed by Novartis as Jakavi®), recommending the drug “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” for patients “with intermediate-2 or high-risk disease.”

Myelofibrosis (MF) is a type of myeloproliferative neoplasm (MPN), where there are too many fibroblasts in the bone marrow. Fibroblasts are cells which produce connective tissue, scar and fibrous tissue. The excess fibroblasts scar the bone marrow and prevent it from making regular blood cells. As a result of this, the spleen may become enlarged. Other symptoms may include repeated infections, shortness of breath, fevers and night sweats, weight loss and tiredness.

The positive recommendation of this recent appraisal follows an earlier appraisal in April 2013 where ruxolitinib was not recommended for use within NHS England and Wales. This was largely because the impact of the drug on patients was unclear, due to the overall survival data not yet being available. Patient organisations, including Leukaemia CARE, highlighted its benefit to patients in terms of quality of life (by reducing the significant and debilitating symptom burden associated with myelofibrosis). Subsequently, the Appraisal Committee concluded that ruxolitinib was clinically effective, but could not be considered cost-effective.

Following the emergence of more clinical trial data demonstrating the survival benefit of ruxolitinib, a second appraisal was initiated last year. After reviewing the data submitted by the pharmaceutical company and patient organisations, the new preliminary decision in the appraisal consultation document (ACD) was to recommend the drug for high-risk myelofibrosis patients.

Whilst pleased that ruxolitinib could soon be available to high-risk myelofibrosis patients, Leukaemia CARE (in collaboration with MPN Voice), responded to the ACD, questioning why the effective drug had not been recommended for intermediate risk myelofibrosis patients as well. In our joint response to NICE, we prompted the appraisal committee to consider the inequality of their recommendation. As one of the key benefits of ruxolitinib is its impact on patient’s symptom burden and intermediate-risk myelofibrosis patients have a similar symptom burden to high-risk patients, we considered it unfair that they would be unable to access the treatment. It was further emphasised that early diagnosis (and consequently earlier treatment) significantly improves outcomes for patients - as ruxolitinib appears to have a greater efficacy the earlier a patient is able to start on treatment, it seemed illogical to allow a patient’s disease to worsen before they could access it.  

Following our joint response to the ACD, in addition to the patient access scheme (discount in the cost) offered by the pharmaceutical company, the final appraisal determination (FAD) for the clinical treatment of myelofibrosis with ruxolitinib has been expanded to include intermediate as well as high-risk patients. The decision is subject to an appeal period, but if unchallenged, this means that ruxolitinib will be available to a larger group of patients than initially thought, offering more myelofibrosis patients more treatment options.  

Zack Pemberton-Whiteley, Head of Campaigns and Advocacy, has commented:

“We were very pleased to see that ruxolitinib had a preliminary recommendation for the treatment of high-risk patients with myelofibrosis but the exclusion of the recommendation for those patients who were considered an intermediate-risk was a concern to us.

"That the appraisal committee has taken on board our comments regarding the efficacy of the drug and the inequity of the preliminary recommendation, and has expanded its recommendation to include patients at intermediate-risk (as well as high-risk patients) is a very positive outcome.

"We understand that this decision is subject to an appeal period but we hope that it will be upheld in order to ensure increased access to additional treatment options for myelofibrosis patients in England and Wales.”