Review of Access to New Medicines

7 Conclusions

7.1 Discussion, comment and recommendations run through the various sections of this report. This section deals in the main with the core questions highlighted in Paragraph 3.13.

7.2 Access to end-of-life, orphan and ultra-orphan medicines has increased. If this situation is to be maintained there are two significant issues that need to be considered. Firstly, the assessment and decision making process for ultra-orphan and true ultra-orphan medicines and secondly, the funding mechanism and affordability of maintaining the increased level of access.

7.3 It appears that access to end-of-life and orphan medicines has increased as a result of the new approach. SMC is accepting more of these medicines for use in NHSScotland however, SMC acceptance of ultra-orphan medicines remains low. This is even more striking when one looks specifically at ultra-orphan medicines used to treat very rare conditions. In this report these are referred to as true ultra-orphans. Access to ultra-orphan medicines has increased as a result of more IPTR for these medicines being supported by Health Boards.

7.4 One of the main issues that will have to be considered in light of this Review is whether Scotland has achieved the level of access it set out to and whether the intention is now to maintain this level of access across all three definitions of end-of-life, orphan and ultra-orphan medicine. The T&FG was not set a target for access but it appears that the rates of acceptance for end-of-life and orphan medicines are on a par with the other medicines considered by SMC. Similarly, IPTR data would suggest that access to ultra-orphan medicines is at a rate similar to SMC acceptance for other medicines.

7.5 If the increased level of access being delivered by the current arrangements is felt to be satisfactory then maintaining it will require aspects of the assessment and decision-making system to be revised. The system should deliver its assessments and decisions in a manner that achieves this desired level of access and is consistent, timely and transparent.

7.6 One size no longer fits all and since the introduction of the new approach SMC decisions have delivered increased access to end-of-life and orphan medicines while IPTR has become the default route for ultra-orphan medicines. However, a system that relies on individual applications to access medicines and in effect overturns SMC decisions is not efficient or effective and an alternative pathway as discussed in Section 6.3 is recommended. This would create better alignment of processes and SMC would continue to assess and comment on the clinical effectiveness and cost effectiveness of the medicine but the final decision on availability would sit elsewhere. This approach preserves the integrity of SMC and its processes across the full range of medicines it considers yet achieves the intended level of access to ultra-orphan medicines.

7.7 Getting to this point will require further focussed discussion involving stakeholders through a taskforce or similar.