Access to Eltrombopag

What is eltrombopag and why is it important?  

Eltrombopag is a medication that, when used alongside standard immunosuppressive therapy (ATG + cyclosporine), significantly improves response rates and speeds haematologic recovery in patients with severe aplastic anaemia.  

Use of eltrombopag can be potentially life-saving when a transplant is not a treatment option, and ATG alone may have failed.  

However, not all patients in the UK have access to eltrombopag. Decisions regarding access are often made based on cost rather than what is best for the patient.  There is no national policy or fund in place, meaning access to this life-saving medication more often than not comes down to luck.  

This wasn’t always the case.  

During the Covid-19 pandemic, all patients in the UK with severe or very severe aplastic anaemia had access to eltrombopag. Decision makers recognised how important this medication was to improving outcomes and keeping patients out of hospital during this time of crisis. However, by the end of 2021, access to eltrombopag was removed for patients. 

In 2022, a study further proved the hugely positive outcomes for patients when prescribed eltrombopag. The RACE Trial found that: 

• Standard immunosuppressive treatment, ATG plus cyclosporin with eltrombopag, was more beneficial for participants with severe and very severe aplastic anaemia, compared with standard immunosuppressive treatment.  

• The response in participants with severe aplastic anaemia after the addition of eltrombopag to standard immunosuppressive treatment was faster and of higher quality. 

• Adding eltrombopag to standard immunosuppressive treatment was not associated with more side effects. 

Despite all the evidence, and the NHS previously prescribing eltrombopag as standard, today there is no guarantee that an aplastic anaemia patient in the UK will have access to this vital medication.