Scotland is set to ease access to medicines for patients suffering from an ultra-rare condition. From the beginning of October this year, all medicines that target ultra-rare diseases (fewer than 1 case in 50,000 people) and that the Scottish Medicines Consortium (SMC) decides are clinically effective will be reimbursed through NHS Scotland for a minimum of 3 years. This will include treatments that are ultra-orphan medicines but have recently been rejected for reimbursement by the SMC.

During this 3 year period, the companies will gather additional evidence to assess the effectiveness of the treatment in the real world. This extra data will allow the SMC to issue a final recommendation on the use of the medicine within NHS Scotland.

To make the process simpler and clearer, the Scottish government is also currently working on a new definition for ultra-rare conditions, which should be published before the launch of the new system.

Implications for patients and manufacturers

The new pathway should help the SMC to narrow the access gap between ultra-orphan medicines and orphan medicines while avoiding the impact of overuse of individual applications. The Scottish Government’s pathway update represents a tremendous opportunity to improve the access to medicines for patients affected by ultra-rare conditions.

This is also positive news for manufacturers. The clinical trial designs and reimbursement pathways used to evaluate the effectiveness of non-orphan medicines are not well-suited for the evaluation of orphan or ultra-orphan medicines. The real-world data collected through this new pathway will help manufacturers to create more robust HTA submissions for the SMC, and better substantiated clinical efficacy claims in the final reimbursement application.

However, the question of affordability remains open. Although the number of patients affected by ultra-rare conditions is limited – less than 100 patients in Scotland – the Scottish payer will most probably request a substantial discount to manufacturers to compensate for the clinical value uncertainties and the budget impact that an early access to these drugs will have.

The history of the policy change

The ultra-rare drug policy change follows on from the Review of Access to New Medicines carried out by Dr Brian Montgomery and published in December 2016. This review assessed changes introduced by the SMC in 2014 to improve patient access to end-of-life, orphan and ultra-orphan treatments. Montgomery concluded that while access had improved, acceptance of ultra-orphan medicines had not increased at the same rate as access to end-of-life and orphan medicines. The greatest change to access to ultra-orphan medicines was thanks to the use of Individual Patient Treatment Requests (IPTR), which were replaced by the Peer Approved Clinical System (PACS) Tier 2 in February 2018.

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