What’s the news?
Today, the SMC has decided not to approve the use of ropeginterferon alpha 2b treatment on the NHS. We are disappointed with this decision, as patients with polycythaemia vera have few other options. The treatment is not available elsewhere in the UK, and there are no plans at present to appraise the drug for use elsewhere.
How was Leukaemia Care involved?
Leukaemia Care worked to gather data, patient experience and put together a written response to the SMC before they made a decision. MPN Voice, a charity specialising in supporting those with MPNs, a group of cancers that include PV, also provided a submission with patient experience. We focused on why PV patients need another treatment option. Most patients with PV are forced to keep taking treatments that have stopped working optimally for them or have few other treatment options if they have to stop their current one because the treatment is making them ill. Therefore there is a large gap in the treatment of these patients.
The SMC also hold a meeting where clinicians and patients can create a joint statement of support for the treatment, called a PACE meeting. The resulting PACE statement was supportive of this treatment as meeting this unmet need for PV patients.
Why was the decision made not to approve the treatment?
The SMC committee felt that, even though the PACE meeting statement highlighted strong support from patients and clinicians for another treatment option, there was not enough evidence from the clinical trials for the committee to be confident the treatment would be effective.
Which patients were being considered for this treatment?
At present, PV patients in Scotland can access ruxolitinib treatment. This is effective for many but can become less so over time or side effects can become severe so patients have to stop. These are the patients that were being considered for treatment with ropeginterferon alpha 2b.
What happens next?
This decision resulted from a lack of evidence about the treatment’s effectiveness, so we will continue to push for further trials or other evidence gathering activities in patients to ensure more treatments become available. The SMC does not have a formal appeals process for challenging its decisions. Leukaemia Care will explore options for next steps alongside other charities and clinical advisors, and we will report any action we take and any changes to this decision if they happen.
If you have any questions about this blog, please contact our advocacy team on firstname.lastname@example.org.
Published date: 10th May 2022