On Wednesday 23 March 2016, we launched our new Patient Charter 'Patient perspectives and priorities…
In December 2016 the Scottish Government published Dr Brian Montgomery’s Review of Access to Medicines (The Montgomery Review). The review considered how changes made to the Scottish Medicines Consortium (SMC) process in 2014 have improved patient access to medicines for rare and end-of-life conditions. It also looked more broadly at how the whole system for getting patients access to newly licensed drugs is working.
We welcome the publication of the Review, which concludes that access to end-of-life and rare disease medicines has increased and that there is a high level of satisfaction with the current situation. However, the acceptance rate for ultra-orphan medicines has not increased to the same extent.
In March 2016, Genetic Alliance UK published a Patient Charter 'Patient perspectives and priorities on access to medicines for rare conditions in Scotland’. The Charter produced a number of recommendations on how to further improve the transparency, accountability and appropriateness of the various routes by which rare disease patients in Scotland access the medicines they need to treat their condition. We were pleased to be able to take advantage of a number of opportunities to engage with Dr Montgomery and share the findings of our Charter. It is encouraging to see a number of our recommendations reflected within his report.
Dr Montgomery’s review has demonstrated that the new approach has successfully increased access to end-of-life and orphan medicines, but the SMC’s rate of acceptance of ultra-orphan medicines has not increased to the same extent. Evidence is provided that only one out of seven medicines (14%) used in the treatment of very rare conditions was approved for use. This compares with 11 out of 12 (92%) approved amongst medicines being used at the end-of-life and 2 out of 3 (67%) approved in the treatment of cancer.
Instead, access to ultra-orphan medicines has increased through the use of the Individual Patient Treatment Requests (IPTR) and, where it is being piloted, the Peer Approved Clinical System (PACS). However, we agree with Dr Montgomery’s conclusion that it would be preferable for these patients not to be reliant on this process.
Dr Montgomery recommends that an alternative assessment pathway be introduced for the purposes of assessing ultra-orphan medicines used in the treatment of very rare conditions. It may be that this pathway will exist separately from the SMC and a number of possible options for an alternative pathway are indicated in the report. For example, Managed Access Schemes or a single PACS Panel in NHS Scotland for ultra-orphan medicines.
Genetic Alliance UK welcomes the recommendation to consider how best to achieve improved access to ultra-orphan medicines used in the treatment of very rare conditions and we look forward to engaging in discussion with the Scottish Government on alternative arrangements.
We were pleased to see that the Montgomery Review reflects the assertion made within our Patient Charter that greater clarity of the role of patient responses in Scottish Medicines Consortium decision-making would be beneficial. Whilst it is clear that the Patient and Clinician Engagement (PACE) has been a welcome addition to the SMC appraisal process for orphan, ultra-orphan and end-of-life conditions, it is unclear how PACE impacts SMC decision making. We welcome Dr Montgomery’s recommendation that there is a need for the PACE contribution to decision making to be quantified and better understood. We look forward to engaging with the SMC to input patient group perspectives on how this can be achieved.
Both the Montgomery Review and our Patient Charter note that the role of the Public Partner would benefit from review and clarification. It has been encouraging to see that the SMC has already taken steps to address this, following the publication of our Patient Charter. Videos have been uploaded to the Scottish Medicines Consortium website to describe the role of the Public Partner.
Furthermore, our recommendation that there is a need for an increase in representation and type of patient voice on all decision-making panels is reflected in the Montgomery Review, with recognition that this will enhance the quality of discussion and decision making. We look forward to further discussion with the SMC’s Patient and Public Involvement Team around increasing the role of patients and patient groups at the SMC.
We also asserted in our Patient Charter the need to have the appropriate clinical experts taking part in the SMC PACE process and that this may mean looking outside of Scotland and, in some cases, the UK. We will continue to support our member patient groups to ensure the most appropriate clinician is consulted during this process.
Our Patient Charter raised concerns with the reliance on the Individual Patient Treatment Process (IPTR) process for ultra-orphan medicines and also the lack of transparency with regards to the introduction of the Peer Approved Clinical System (PACS).
Dr Montgomery notes that clinicians and patients are increasing their use of the IPTR systems to successfully access medicines not recommended for use by SMC.
Dr Montgomery also suggests a single National Formulary for NHS Scotland may improve equitable access to new medicines across Scotland. While Boards have complied with the guidance in relation to IPTRs they have undoubtedly put in place different processes which reflect local circumstances. The variations largely reflect the capacity and expertise available to individual Boards to address IPTRs. A “Once for Scotland” approach or process would improve transparency and consistency and build greater confidence in the IPTR system and its successor PACS. This could be achieved by elevating the processing of IPTR/PACS requests to a single national panel or perhaps regional IPTR/PACS panels following consistent methodology. This is in line with recommendations of our Patient Charter and we look forward to exploring this further through engagement with the Scottish Government.
The Review report also noted that there are difficulties in collecting data for orphan, ultra-orphan and end-of-life medicines. Within Boards data collection systems related to medicines largely support medicines ordering and prescribing but do not record outcomes. The systems do not allow tracking of the use of medicines in relation to patients.
Dr Montgomery recommends a review of datasets and definitions and processes for collection and analysis.
The Montgomery Review has produced some positive recommendations, and we are glad that so many of the recommendations that we made last year have been incorporated into this process. We look forward to seeing how this report is taken forward by the Scottish Government. If you have any questions or comments, or if you would like to be included in our future work on this topic, please contact our Policy and Engagement Manager in Scotland, Natalie.