Header graphic for print
Focus on Regulation

Scottish government to reform approval process for new end-of-life and orphan medicines

The Scottish government has announced plans to implement the 28 recommendations proposed in an independent review of access to new end-of-life, orphan and ultra-orphan medicines by patients in Scotland, published in a report on 14 December 2016. The recommendations aim to further improve the process by which these types of medicines, in particular ultra-orphan products, are currently assessed for recommendation by the Scottish health technology assessment body, the Scottish Medicines Consortium (the “SMC”), and funded. Once medicines receive SMC recommendation, they should be made available by local Health Boards for use by patients through the Scottish National Health Service (“NHS Scotland”).

The recommendations to be implemented include giving the SMC an additional “interim” decision option to “recommend for use subject to ongoing evaluation and future reassessment”, after which if a medicine fails to deliver the anticipated results, the pharmaceutical company would have to refund associated costs and the medicine may be subject to restricted use or withdrawn. This recommendation is coupled with a proposal for a more complex approach for the assessment of the benefits of new medicines using real world data, patient reported outcomes and other qualitative measures. These recommendations are intended to result in the acceptance of new medicines that may not have previously been approved, subject to on-going data collection and evaluation before a final assessment is made.

Other key recommendations put forward in the report include:

  • A revised approval process for ultra-orphan drugs by which the SMC would continue to assess clinical- and cost-effectiveness but a final decision would be taken by a separate panel. This would replace the current system under which there is a trend of negative SMC decisions being systematically overturned by Individual Patient Treatment Requests (“IPTRs”);
  • Increased patient engagement and transparency as to how input from Patient and Clinician Engagement groups are taken into account by the SMC and the reasons for negative outcomes. This recommendation also includes a requirement on pharmaceutical companies to minimise submissions of sensitive commercial data which then needs to be redacted;
  • Further clarity on the future availability of the New Medicines Fund as demand grows;
  • Further review and implementation of Peer Approved Clinical Systems as a clinically-focused alternative assessment to IPTRs; and
  • Prioritising the development of national electronic prescribing and electronic patient records.

The full report is available here.