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 (a) enable and (b) fund urgent access to the drug Luspatercept for extremely vulnerable transfusion-dependent RARS Myelodysplastic Synrdomes patients in the absence of a NICE decision on the appraisal of that drug.

The National Institute of Health and Care Excellence’s (NICE) appraisal of luspatercept for the treatment of anaemia caused by myelodysplastic syndromes has been suspended because the company that manufactures the drug was unable to provide an evidence submission. NICE is working with the company to reschedule the appraisal of luspatercept once the company is in a position to provide an evidence submission.

In the absence of NICE guidance on the use of a drug, clinicians are still able to prescribe luspatercept if they consider it appropriate for their patients, subject to the treatment being funded by the relevant commissioner. National Health Service commissioners are expected to take funding decisions based on an assessment of the available evidence and are required to have arrangements in place to consider individual funding requests.