2 Executive Summary
2.1 The Review of Access to New Medicines was undertaken to assess the impact of the new approach introduced in 2014 by SMC. The new approach aimed to increase access to end-of-life, orphan and ultra-orphan medicines.
2.2 This Review concludes that access to end-of-life, orphan and ultra-orphan medicines has increased. Not only that, the stakeholders who engaged with the Review indicated a high level of satisfaction with the current situation and engaged enthusiastically in discussion about how the system for assessment and decision making in relation to access to new medicines could be further improved.
2.3 The new approach has had the hoped for effect in increasing access to end-of-life and orphan medicines with SMC accepting a greater proportion of these medicines for use. However, SMC's rate of acceptance of ultra-orphan medicines has not increased to the same extent. Access to ultra-orphan medicines has increased through the use of Individual Patient Treatment Requests ( IPTR) and its successor the Peer Approved Clinical System ( PACS).
2.4 When considering ultra-orphan medicines, one size no longer fits all. If NHSScotland now wishes to maintain this increased level of access, then it would be preferable if this was not through continuing reliance on IPTR and PACS. An alternative assessment pathway should be developed for ultra-orphan medicines that preserves the integrity of SMC and its processes yet achieves the intended level of access to these medicines.
2.5 It is important to emphasise that the aim is not to establish a system that enables direct access to all newly licensed medicines. All stakeholders confirmed that Scotland needs to have a system capable of saying no.
2.6 While many stakeholders were pleased with the current increase in access, some highlighted a concern about the sustainability and affordability of the current arrangements going forward. To date the New Medicines Fund ( NMF) has effectively met the costs of giving patients access to end-of-life, orphan and ultra-orphan medicines but it is not clear if the NMF will be sustained and if so whether it will be increased to meet the anticipated growth in demand.
2.7 The affordability challenge as it relates to access to new medicines is the challenge of managing the interface between NHSScotland and the pharmaceutical industry. Difficult choices will have to be made as NHSScotland seeks to optimise its use of a finite resource subject to ever-increasing demands while at the same time the pharmaceutical industry operates in a competitive commercial environment.
2.8 Many spoken to in the course of the Review highlighted the need to develop a more sophisticated approach to the measurement of outcomes. All saw a need to move beyond reliance on traditional quantitative metrics. The aspiration is for a basket of measures that includes real world data, patient reported outcomes and an assessment of wider societal benefit. This will undoubtedly give greater confidence when assessing the impact of new medicines but developing such datasets will be a major task. It will be vital to ensure that the measures are consistent and allow meaningful comparisons which can in turn inform decision making.
2.9 Some of the discussions undertaken to inform the Review sought to link access to new medicines to the approaches being set out in Realistic Medicine 1 , the annual report of the Chief Medical Officer ( CMO). Those stakeholders involved in the planning and delivery of service were supportive of the general vision articulated in Realistic Medicine but they found difficulty reconciling the principles laid out in the document with the impact of the new approach and IPTR/ PACS. They also expressed a concern that medicines were not being treated equitably when compared with other healthcare treatments and technologies
2.10 The Review has benefitted enormously from the knowledge, expertise and enthusiasm of all who contributed to the process. It will be important as the recommendations of the Review are considered and taken forward that there is continuing engagement with the broad stakeholder community already mobilised by the SMC and given specific focus by the Review.
2.11 If the recommendations of the Review are accepted, a number of specific actions will have to be progressed. Some may be able to be taken through existing groups or processes but others will require the setting up of short life working groups, taskforces or other mechanisms and all stakeholders have expressed their willingness to be involved in these processes.
2.12 There would be merit in considering holding a Government-sponsored stakeholder summit meeting to discuss the Review, the issues it has highlighted and the required actions going forward.