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Background Info

Paroxysmal nocturnal haemoglobinuria (PNH) is a very rare, chronic, life threatening disease which causes red blood cells to lyse (explode). This can lead to a range of debilitating symptoms including severe fatigue, anaemia, abdominal pain, difficulty with swallowing, erectile dysfunction, kidney dysfunction and thrombosis. Patients are most commonly diagnosed in their 30s and median survival from diagnosis is in the range of 10-22 years.

The only licensed and effective treatment for PNH is Eculizumab, which is nationally commissioned for patients in England and Wales and fully funded in Northern Ireland.

Eculizumab is a highly effective treatment for PNH with successive studies demonstrating excellent outcomes for patients and improved quality of life.  Recent data shows that patients being treated with Eculizumab have a normal life expectancy. 

Eculizumab was not recommended for the treatment of PNH by the Scottish Medicines Consortium (SMC) in 2010 on the basis that “cost-effectiveness was not demonstrated in an independent economic analysis”. 

However, the SMC’s appraisal process is not appropriate for the appraisal of medicines for very rare diseases and it regularly rejects these medicines.  For example, as of May 2010, the SMC had reviewed 46 orphan medicines and of these 37 % (17) had been rejected and a further 24% (11) granted only highly restricted use. 

We are therefore calling for the Scottish Government to review the mechanism and methodology used by the Scottish Medicines Consortium to appraise medicines for rare diseases to ensure that more are recommended for use in Scotland.  

As Eculizumab is not recommended by the SMC, patients access the medicine via Individual Patient Treatment Requests (IPTRs).  This has resulted in a very serious inequality of access to the medicine for patients with PNH across Scotland.   

The refusal of NHS Greater Glasgow & Clyde to fund Eculizumab for one patient led to his death in late April 2011. Another patient has recently been declined access to the medicine in the same health board.  The board has stated that it acts in accordance with the guidance that is provided.   

We are therefore also calling for the Scottish Government to instruct the Chief Medical Officer to revise the criteria by which health boards assess Individual Patient Treatment Requests.