Scotland’s systems for approving and accessing new medicines for use in NHS Scotland for treatment of ultra-rare diseases such as aHUS are set to change soon and for the better following an announcement by Alex Neil , Cabinet Secretary for Health and Wellbeing today 31 Jan 2014.
The much disliked Individual Patient Treatment Request system will go and be replaced from May 2014 by a Peer Approved Clinical System which the Scottish Government say will give patients and clinicians a greater say on which new medicines are approved by the Scottish Medicines Consortium (SMC) for use in NHS Scotland in treating very rare conditions.
The SMC will introduce a new Patient and Clinician Engagement (PACE) meeting to give patient groups and clinicians a stronger voice in SMC decisions for rare conditions and for medicines such as eculizumab SMC will consider more than just the direct health impact on the patient beyond its conventional assessments. Pharmaceutical companies will also have an additional opportunity in the new process to put forward a Patient Access Scheme, which makes their medicines more affordable.
This decision follows an excellent report by the Sport and Health Committee of the Scottish Parliament last year which highlighted poor access to medicines for those with rare disease in Scotland. It made several wide sweeping recommendations which were accepted by Mr Neil in the Scottish Parliament in October. He asked SMC to set out how the improvements could be implemented and to report to him by Christmas 2013. He has now accepted its Report and asked SMC to deliver the approach it recommended.
The Cabinet Secretary’s letter to the Chair of the Health and Sports Committee can be seen at
The SMC Report on Assessment of medicines for end of life care and very rare conditions in Scotland can be seen at. http://www.scottish.parliament.uk/S4_HealthandSportCommittee/Inquiries/Assessment_of_medicines_for_end_of_life_care_and_very_rare_conditions.pdf
The Scottish Government’s press release is as follows. http://news.scotland.gov.uk/News/Enhancing-access-to-new-medicines-8e1.aspx