6.11 How the new approach will accommodate advances in new medicines and a developing regulatory framework
6.11.1 The Review was involved in much interesting discussion about what lay ahead and how we might collectively address the anticipated challenges to best effect.
6.11.2 There was a shared view of an exciting emerging situation described as precision medicine and associated with highly-targeted therapies informed by genomics. This will potentially bring many benefits but also challenges around using the technologies to achieve optimum benefit. Treatments are likely to be used with a greater degree of confidence but many more could be classed as orphans and ultra-orphans under the current definitions. Highly-specialised, targeted medicines for smaller patient populations are anticipated to become the norm.
6.11.3 Given the relatively small number of patients suitable for treatment the medicines have the potential to be extremely expensive and the costs would not be confined to the medicines. Many will bring with them a requirement for diagnostics or other supporting services to enable their use.
6.11.4 Internationally there is a move to give patients earlier access to medicines which have shown potential benefit and this is being seen in a number of ways in other health systems. Novel approaches to so-called "breakthrough" medicines and Early Access Schemes ( EAS) are already in place elsewhere.
6.11.5 There is a move on the part of regulatory agencies to grant licenses at an earlier stage when compared with the processes for medicines that do not meet the definitions for end-of-life, orphan and ultra-orphan. Because these medicines are used to treat such small populations it means that the evidence bases normally expected of new medicines will never be established. Consequently, licenses are being granted with the requirement for ongoing evaluation of the medicine and even linkage to completion of clinical trials.
6.11.6 This means that in the future the level of evidence required for licensing will be less than that required by SMC for its health technology assessment. The risk then becomes that none of these medicines will be accepted for use by SMC and the IPTR/ PACS route becomes the norm. It will be important as discussed in paragraph 6.3.12 to ensure that SMC's contribution to the assessment process is revised and remains relevant.
6.11.7 There are a number of opportunities. If Scotland is to avoid creating a system that makes it difficult for patients to access new medicines or, worse still, denies them access, then the existing system needs to evolve. The introduction of a conditional yes (as discussed in Section 6.9 above) accompanied by the on-going collection and evaluation of data in collaboration with the treating clinicians, pharmaceutical companies and other agencies such as the Farr Institute would better align SMC's processes with the direction of those of the regulator and work to the advantage of patients.
6.11.8 A different approach will be required that takes account of different and novel types of data including so-called "real world" data, patient reported outcomes and other quantitative measures. There is also likely to be an increasing need to develop and use metrics linked to prevention and preservation of function rather than more traditional outcomes. This will best be achieved by working with a broad set of stakeholders to agree datasets and data definitions to support the ongoing evaluation. Much can be learned from the databases and patient registers held by some patient support groups and in some instances these could provide solid starting points.
6.11.9 These revised processes should be encompassed within a MAS that ensures that access to new medicines is informed and driven by meaningful data.
27 Consider through wide stakeholder engagement the best way for NHSScotland to take advantage of the opportunities afforded by anticipated developments in the way that new medicines will be introduced in the future. This is likely to be through the establishment of a multi-agency taskforce or equivalent group.