Review of Access to New Medicines

9 Summary of Recommendations

9.1 Develop, agree and implement national datasets and data definitions for end-of-life, orphan and ultra-orphan medicines and for IPTR/ PACS processes. This will ensure that data from Boards is consistent and can be collated. This in turn will allow Boards' data to be used to support functions such as planning and resource allocation.

9.2 Develop, agree and implement a national chemotherapy dataset and equivalent datasets for medicines used to treat rare conditions.

9.3 Develop, agree and implement sets of outcome measures for classes of medicines or, in the case of very rare conditions, specific medicines.

9.4 Ensure that national systems being developed for electronic prescribing and electronic patient records are prioritised and support the above requirements.

9.5 Establish a multi-agency taskforce or equivalent to report on data requirements to support the assessment and introduction of new medicines going forwards.

9.6 Review the definitions for end-of-life, orphan and ultra-orphan medicines to ensure that the definitions used remain suitable to deal with the assessment of anticipated new treatments such as targeted medicines, increasing use of combination therapies and the impact of genomics.

9.7 Develop, agree and implement a new definition of "true ultra-orphan medicine" to take account of low-volume, high-cost medicines for very rare conditions.

9.8 Review communications of SMC's decisions to patients, patient groups and the pharmaceutical industry with a view to achieving greater transparency.

9.9 Review and clarify the role of the SMC Public Partner.

9.10 Consider key participants at PACE meetings being actively involved in the relevant parts of SMC meetings to enhance the quality of discussion and decision making.

9.11 Develop and implement a new assessment and approval pathway for true ultra-orphan medicines that restricts the role of SMC to health technology assessment and places the responsibility for the final decision on availability elsewhere.

9.12 Refine data collection systems to enable meaningful year-by-year comparisons and the monitoring of emergent trends.

9.13 Review SMC's processes in relation to decision making by secret ballot.

9.14 Minimise the inclusion of commercial in confidence information in SMC submissions.

9.15 Standardise data collection at Board level in relation to systems and process for requests to access non-formulary medicines.

9.16 Clarify the future arrangements for the funding of end-of-life, orphan and ultra-orphan medicines.

9.17 Review the data set and definitions for data relating to IPTRs collected by Boards with the aim of achieving consistency and comparability and also extending the dataset to include data on outcomes.

9.18 Explore opportunities to learn from and collaborate with other health economies in relation to the assessment and managed introduction of new medicines and other health technologies.

9.19 Standardise NHSScotland's approach to formulary development and use.

9.20 Review and evaluate the experience of PACS to date with a view to deciding on any required modifications and thereafter agree a process and timescales for full roll out and implementation.

9.21 Explore MAS with a view to early adoption in NHSScotland. These should build on the experience of complex PAS within NHSScotland and payment-by-results schemes in operation in other health systems.

9.22 Review the proposal to introduce a "pause" in light of some of the wider changes and actions recommended in this report.

9.23 Give SMC the additional decision option of "recommend for use subject to ongoing evaluation and future reassessment."

9.24 Make greater use of National Procurement in NSS to lead negotiations on behalf of NHSScotland on the cost of new medicines.

9.25 Undertake a comparative review of the arrangements in place in the healthcare systems of other countries for the introduction of new medicines and specifically end-of-life orphan and ultra-orphans, seeking to learn from their experiences.

9.26 Monitor the demands made on SMC and its associated processes and ensure that the available capacity and capability and support mechanisms are adequate for SMC's needs.

9.27 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.

9.28 Consider how the experience of NHSScotland's systems for the assessment and managed introduction of new medicines can inform the development of a Scottish Model of Value. This is likely to be through the establishment of a multi-agency taskforce or equivalent group.