3.1 Medicines play a significant part in the delivery of healthcare, offering prevention, control, palliation or cure of many diseases. Recent years have seen a growth in the number and complexity of treatments available for a number of conditions and in some instances medicines now offer treatment options where none existed previously. All expectations and indications are that this situation will increase further with the advent of new technologies such as genomics and precision medicine.
3.2 The increase in treatment options has come, in some cases, at a significant financial cost and has challenged conventional methods of assessing cost effectiveness and value for money of new treatments.
3.3 In Scotland, since 2002, all newly licensed medicines have been assessed for suitability for use in NHSScotland by the SMC which has an international reputation as a health technology assessment ( HTA) body. Its assessment process concentrates on clinical and cost effectiveness; affordability is not a specific consideration.
3.4 In recent years a higher proportion of medicines for end-of-life and rare conditions when compared with other medicines, have been given "not recommended" status by SMC. Growing concern was expressed by patients, patient groups and the pharmaceutical industry that this appeared to be solely on the basis of cost and consequently patients with certain rare conditions were being denied access to medicines that are clinically effective.
3.5 The conditions treated by these medicines were either end-of-life, where the medicine offered the potential to extend life, or rare conditions where the medicine offered the potential to prevent or slow deterioration of the condition and enhance quality of life. In some instances, the new medicine offered the only treatment option.
3.6 The then Cabinet Secretary for Health and Wellbeing, Alex Neil MSP, asked SMC to review its processes in relation to these circumstances and a Task and Finish Group ( T&FG) was established in 2013 under the chairmanship of Professor David Webb. The group reported in December of that year, making nine recommendations all of which were accepted ( Appendix 1).
3.7 The key recommendations were the introduction of
- New definitions for "end-of-life" and "ultra-orphan" medicines with retention of the European Medicines Agency ( EMA) definition for orphan medicines. ( Appendix 2);
- Revised processes for the assessment of medicines falling under these three definitions. (It was recognised that some medicines could be classed as both end-of-life and orphan or ultra-orphan);
- Patient And Clinician Engagement ( PACE) as an optional addition to the process for the assessment of all three categories and
- A new decision making framework for the assessment of ultra-orphan medicines that was not based on the Quality Adjusted Life Year ( QALY).
Overall the intention was to create a more flexible and enabling process for the assessment of these medicines. This is referred to as "the new approach."
3.8 Two other important related developments took place around the same time. In November 2013, a letter was issued to Health Boards from the Chief Medical Officer SGHD/CMO(2013)20: Access To New Medicines - Transitional Arrangements For Processing Individual Patient Treatment Requests 2 . This required Boards to exclude exceptionality as a consideration for IPTRs and to exercise greater flexibility. This appears to have had the effect of Boards supporting a greater proportion of requests, thus further increasing access.
3.9 The second development was the creation by the Cabinet Secretary of the Rare Conditions Medicines Fund ( RCMF) which was replaced in October 2014 by the New Medicines Fund ( NMF). This fund was derived from Scotland's share of the rebate paid to the UK Government by the pharmaceutical industry via the Pharmaceutical Price Regulation Scheme ( PPRS). Health Boards can access this fund to cover the acquisition costs and appropriate supporting costs to enable SMC recommended medicines to be made available, recognising that making medicines available may also require investment in diagnostics or other infrastructure and staffing. The fund is available to support the cost of end-of-life, orphan and ultra-orphan medicines whether recommended by SMC or accessed via IPTR/ PACS. The stated intent is to ensure that,
"Availability of funding is not a barrier to NHS Board implementation of policy intentions on increased patient access to licensed orphan, ultra-orphan and end of life medicines and that no NHS Board is better or worse off financially on the basis of clinical decisions on prescribing these medicines."
The NMF is discussed in detail in Section 6.6.
3.10 These factors have combined to create the situation where the majority of patients now access end-of-life, orphan and ultra-orphan medicines either through SMC approval or applications through IPTR or PACS. There is also a perception that media campaigns and political lobbying of councillors, MPs and MSPs positively influences access for individuals or groups of patients.
3.11 There is now a need to clarify or restate the policy intention. All stakeholders consulted as part of the Review acknowledged that universal unchallenged access to new medicines was not desirable.
3.12 The eighth of the T&FG's recommendations was that there should be an independent review of the experience with the new approach.
3.13 This independent Review has been undertaken at the request of the current Cabinet Secretary for Health & Sport and was tasked with answering two core questions:
1. Has the new approach adopted by SMC increased access to end-of-life, orphan and ultra-orphan medicines?
2. How might systems and processes be further improved?
3.14 Underneath these questions sit a series of more detailed issues that fall within the scope of the Review. The full scope and remit of the Review is laid out in Appendix 3. This report follows the order and structure of the defined scope. Much of the discussion that took place with stakeholders was not easily accommodated within the scope as specified and so additional sections on engagement, data and general discussion have been added.
3.15 The medicines covered by the definitions of end-of-life, orphan and ultra-orphan constitute only a part of the total workload of SMC. No other aspects of SMC or its process fell within the scope of this Review. In the course of the Review the overwhelming sense encountered was that there was a high level of satisfaction with SMC and that its processes remain robust.
3.16 The Review was not intended to be exhaustive or to provide definitive answers; instead the Review was expected to engage widely with stakeholders and having addressed the core question in relation to the increase in access to new medicines, it was expected to go on to make recommendations that would result in further improvements in processes going forward. By design it concentrated on aspects of the current arrangements for end-of-life, orphan and ultra-orphan medicines that had the potential for improvement and as such the tone and content may appear critical. It should be stressed however that SMC continues to function effectively and enjoys the confidence of stakeholders. Its processes remain appropriate and work well for the majority of medicines it considers. The comments in this report refer to changes and improvements in SMC's processes as they relate to the small number of ultra-orphan medicines under consideration.
3.17 This report is the distillation of the perspectives of stakeholders holding a wide range of views. However, all stakeholders have in common the aspiration that patients get prompt access to medicines likely to be of benefit to them in a way that is uncomplicated, timely and transparent. All stakeholders agreed that the aim was not to achieve universal access to all licensed medicines and that the system has to have the ability to give medicines not approved status if considered appropriate.
3.18 The first of the core questions on increase in access has been relatively easy to address in quantitative terms. What has been more difficult has been to link increased access to improved outcomes. All agreed it will be important to develop systems and processes which report on outcomes. Currently it is possible to look at the impact the new approach has had in terms of the number of medicines accepted by SMC but not in relation to patients treated or their outcomes. This is discussed further in the section on Data, Section 5.