Access to therapy for paroxysmal nocturnal haemoglobinuria

• The PNH Alliance wrote to the Cabinet Secretary for Health and Wellbeing on 28 February 2011 requesting a meeting to discuss inequality in access to therapy for paroxysmal nocturnal haemoglobinuria (PNH) between health boards. The meeting was declined on the basis that the Cabinet Secretary’s diary was “very pressured”.
• The PNH Alliance wrote to the Scottish Medicines Consortium (SMC) on 15 March 2011 requesting a meeting to discuss the appraisal of Eculizumab for the treatment of PNH. The meeting was declined but the SMC has since published updated guidance on the therapy. However, it is still not recommended for use in Scotland.
• The PNH Alliance wrote to the Chief Executive of NHS Greater Glasgow and Clyde, where a patient was being denied access to therapy for PNH, on 11 February 2011 and 11 April 2011 requesting that the health board reassess its policy on the use of Eculizumab. The Chief Executive declined to reassess its policy.
• The PNH Alliance and PNH Scotland have advised MSPs on barriers to access to therapy for PNH which has led to a number of written questions being tabled and correspondence with the Cabinet Secretary (February 2011 onwards).
• The PNH Alliance and PNH Scotland have secured significant media coverage on access to therapy PNH, including in the Herald, the Sunday Post, and on the BBC and STV.
• The PNH Alliance wrote to the Cabinet Secretary for Health and Wellbeing again on 1 July 2011 requesting a meeting following the death of a patient who had been declined access to therapy in NHS Greater Glasgow and Clyde. The meeting was declined on the basis that “decisions relating to IPTRs are a matter for each NHS Board”. This misses the point as we believe that political intervention is required to improve the way local boards assess Individual Patient Treatment Requests.