Review of Access to New Medicines

6.8 Whether there are further opportunities to take a 'once for Scotland' approach in any aspect of access to newly licensed medicines

6.8.1 Since the inception of SMC there has been a "Once for Scotland" approach to the managed introduction of new medicines in NHSScotland. This has worked well and there are several areas where this approach could be further developed.

6.8.2 Arguably in circumstances such as those encountered when dealing with end-of-life, orphan and, in particular, ultra-orphan medicines, consideration should be given to a "Once for the United Kingdom" or even a "Once for Europe" approach. Despite the undoubted strength of its health technology assessment capability, Scotland lacks the critical mass to effectively negotiate and procure high-cost, low volume medicines such as ultra-orphans.

6.8.3 A single National Formulary for NHSScotland has been suggested and key to the success of such an initiative will be how the formulary is developed and implemented. There would be undoubted gains in NHSScotland adopting a national approach to formulary development and use. A possible process and benefits are discussed in Paragraph 6.8.6.

6.8.4 Formularies perform two primary functions. They can be a comprehensive compendium of all medicines available for use or they can be decision-support tools which promote and support the safe and effective use of medicines in the management of medical conditions. The former is the British National Formulary ( BNF) which needs no replication. The latter can take a number of forms but there is demonstrable value in having locally-developed and clinically-owned formularies.

6.8.5 Most Boards have their own formularies which have been developed to meet local need largely in relation to higher volume, lower cost activity. These formularies provide clinicians with advice on the management of specific conditions and detail therapeutic options based on clinical and cost effectiveness. Usually this is presented as a number of ranked choices. Not every medicine for ever condition will be or needs to be included in a formulary but as every medicine approved by SMC is by definition available to clinicians in every Scottish Health Board, there are local mechanisms for clinicians and patients to access non-formulary SMC approved medicines. See Section 6.6.

6.8.7 One area where the apparent lack of a "Once for Scotland" approach was commented on repeatedly is the approach to IPTRs and PACS. It is important to remember though that these are processes dealing with the specific circumstances of individual patients in contrast to SMC processes which take a population view. They are also processes dealing with requests for medicines which SMC has already concluded are not recommended for use in NHSScotland. As a result, the deliberations of IPTR panels on individual circumstances will legitimately reach differing conclusions despite apparently similar circumstances. As the accountability for the decision lies with Health Boards these differences are generally interpreted as inconsistencies between Health Board processes rather than differences between the circumstances of individual patients.

6.8.8 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.

6.8.9 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.

6.8.10 This already happens in some instances. Where the medicine being requested is being used to treat cancer or a rare condition the clinical consideration usually takes place within the cancer centre or equivalent expert centre. The expert centre's IPTR panel or equivalent makes a recommendation which is subject to confirmation of funding and as funding is dealt with at the level of the patient's Board of residence the recommendation is passed to the Board of residence for final decision. The introduction of the NMF has removed affordability as a consideration. It is unlikely that the Board of residence would not accept the clinical recommendation of the expert centre therefore access would be granted in the majority of cases.

6.8.11 As long as the focus of requests is on the individual patient, this would not result in the same decision for every request for access to a medicine given not recommended status by SMC. If the aim though is to achieve the same decision for every request then the process actually becomes one which is about reversing SMC decisions rather than considering whether there are specific circumstances in which an SMC decision should not apply to an individual patient.

6.8.12 PACS has now been introduced as the alternative to IPTR with the expectation that it will help address the perceived inconsistencies and weaknesses of IPTR. Initially PACS was piloted in NHS Greater Glasgow and Clyde and more recently the pilot has been extended to all Boards but a comprehensive evaluation has not yet been undertaken or published. Consequently, the information available to the Review is based on informal feedback from people involved in the pilot.

6.8.13 PACS is seen as a more consistent process driven by clinical opinion which helps address some of the apparent variability seen with Board-level IPTR processes. Concern has been expressed though that the PACS process does not allow a not supported decision. In other words, a vigorously pursued PACS application will inevitably result in access being granted. This concern is the origin of the "not if; but when" concern highlighted in Paragraph 6.7.9.

6.8.14 The discussions with stakeholders in relation to IPTR and PACS highlighted yet again the pleas for consistency and transparency in relation to processes and decision making. As noted elsewhere all stakeholders accepted that the system had to have the ability to reach not recommended decisions but the discussions stopped short of providing a consensus on the circumstances under which these would apply.

6.8.15 Patient Access Schemes ( PAS) and the associated Patient Access Scheme Assessment Group ( PASAG) already represent a "Once for Scotland" approach to NHSScotland's relationship with the pharmaceutical industry. There is the potential for a different and more effective mechanism regarding the pricing and procurement of medicines and this is discussed in Sections 6.9 and 8.

Recommendations

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.

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

20 Review and evaluate the experience of PACS to date with a view to deciding on any required modifications and thereafter agree the final model and timescales for implementation in NHSScotland.