belumosudil (Rezurock)

The Scottish Medicines Consortium (SMC) has completed its initial assessment of the evidence for the above product using the ultra-orphan framework:

Indication under review: treatment of patients aged 12 years and older with chronic graft-versus-host disease (chronic GvHD) who have received at least two prior lines of systemic therapy.

Key points:

• Chronic GvHD is a severely debilitating condition affecting multiple organs that is associated with pain, and difficulties with mobility, sight, eating, self-care and activities of daily living. There are limited effective treatment options.
  
  
• In pooled data from two open-label, phase II studies belumosudil was associated with clinically relevant overall response rate, 73%, in patients with chronic GvHD who had received two prior lines of therapy.
  
  
• The efficacy and safety of belumosudil relative to relevant comparators is unknown.
  
  
• As belumosudil is administered orally at home, it may have advantages compared with alternative treatments administered in hospital or specialist centres. Improvements in quality of life from baseline, assessed using the 7-day Lee Symptom Scale summary score, were identified in some patients treated with belumosudil.
  
  
• The company presented a three state partitioned survival model to estimate the economic outcomes of belumosudil relative to a basket comparator the company believed representative of Scottish practice. Some of the modelling assumptions were conservative, however uncertainty on the long term health benefits had the potential to reduce the modelled cost-effectiveness of belumosudil.
  
  
• The costs of belumosudil relative to the expected health outcomes are high, and there were outstanding uncertainties in the economic case, some of which may contribute to worse results than predicted in the base case.

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From 10 August 2023 belumosudil (Rezurock) can be prescribed within the ultra-orphan pathway while further evidence on its effectiveness is generated. At the end of the data collection period the company will provide an updated submission for reassessment to allow a decision on its routine use in NHSScotland.