Haemophilia Scotland plays an active role in the Cross Party Group on Rare Diseases because all bleeding disorders are defined as rare diseases. In fact, even if all the bleeding disorders were counted as a single condition we would still be regarded as rare. Many bleeding disorders affect less than 200 people in Scotland. These conditions are defined as “ultra rare”. The Scottish Government is working to improve the system for securing treatments for all rare conditions and, although factor replacement products often have their own systems, it is important for us to understand the wider situation. On the 18th of November the Cross Party Group looked at what the Scottish Medicines Consortium (SMC) is doing to improve its work on Rare Diseases and patient involvement. We also learnt more about the Scottish Government’s New Medicines Fund.
Ann Lee from the Scottish Medicines Consortium addressed the meeting about changes to make it easier for patients and the public to be involved in their decision making. This is something we have recent experience of as we have been engaging with their work on the new, inteferon-free, treatments for Hepatitis C. She told us that new Patient and Clinical Engagement (PACE) meetings are designed to hear about how a new treatment would impact on the lives of those affected. They are interested in hearing about non-medical impact such as being able to go back to work or care for children and do not only look a the clinical data. She also really encouraged patient groups to use testimony in submission which is certainly something we’ll take on board for our next submission. It was interesting to hear that they have developed “Decision Modifiers” which let them make decisions when there is more uncertainty than usual. This is particularly important for rare conditions where it can be harder to work with enough patients to get strong statistical evidence. The SMC meets in public on the first Tuesday in every month if you every want to learn more about their work by attending.
Kathryn Fergusson from the Scottish Government told us about the Pharmaceutical Price Regulation Scheme (PPRS) which is negotiated by the UK Government and works like a rebate of pharmaceutical company profits. The Scottish Government have calculated that this equates for £40million a year for two years of new money which they are investing totally in the New Medicines Fund. The aim is that local health boards shouldn’t have concerns about funding new drugs for rare conditions approved by the SMC process. Over time it is hoped that more and more treatments for rare conditions will go through the SMC process which will reduce the need to make Individual Patient Treatment Requests (IPTR) on a case by case basis. The next round of the PPRS is due to be negotiated in 2019 and there was a general feeling in the room that there should be more Scottish Government involvement than was the case for negotiations for the current scheme.