NHS organisations should take into account the reasons why the company did not make an evidence submission when considering whether or not to recommend local use of eltrombopag for severe aplastic anaemia refractory to immunosuppressive therapy. If, after doing this, organisations still wish to consider eltrombopag for severe aplastic anaemia refractory to immunosuppressive therapy, they should follow the advice on rational local decision-making in the NHS Constitution for England and the NHS Commissioning Board and Clinical Commissioning Groups (Responsibilities and Standing Rules) Regulations 2012, which outlines the approach that should be adopted in circumstances in which NICE guidance is unavailable.
NICE will review the position at any point if the company indicates that it wishes to make a full submission.
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