Rare disease action plan: progress report 2026

6. Priority 4: Improved Access to Specialist Care, Treatments and Medicines

Ultra-Orphan Pathway

The Ultra-Orphan Pathway was established in 2018 to accelerate access to new and innovative medicines for very rare conditions. The Pathway enables medicines for very rare conditions to be made available in the NHS in Scotland for up to three years, while additional data on their effectiveness is collected. Once this data collection period ends, pharmaceutical companies submit the medicine to the Scottish Medicines Consortium (SMC) for re-assessment. The outcome of this re-assessment process will determine whether the SMC accepts the medicine for permanent, routine use in the NHS in Scotland.

An up to date list of medicines included in the Ultra-Orphan Pathway was published in June 2025. As a number of medicines have now completed the full process, the Cabinet Secretary for Health and Social Care has commissioned a review to identify ways to improve and streamline the Pathway. The review will draw on lessons from the first medicines to go through the Pathway, and exploring equivalent approaches used elsewhere in the UK that could be adapted for future use. The Scottish Government is working closely with key partners through the Ultra-Orphan Pathway sponsorship group and will be seeking wider stakeholder engagement in due course.

New Innovative Licensing and Access Pathway (ILAP)

The new ILAP is a UK-wide partnership between the Medicines and Healthcare products Regulatory Agency (MHRA), the NHS, and the Health Technology Assessment (HTA) bodies: the National Institute for Health and Care Excellence (NICE), the SMC and the All Wales Therapeutics and Toxicology Centre (AWTTC).

It is the only example globally of an end-to-end access pathway where a medicine developer can work collaboratively with the NHS, the regulator, and HTA bodies from the early stages of clinical development.

Developing medicines for rare conditions is particularly challenging, as very small patient populations make clinical trials complex and expensive. The new ILAP offers focused support that will be particularly valuable in this area, with the aim of helping to overcome these barriers and accelerate the development of promising new treatments. The SMC will prioritise ILAP medicines for scheduling as part of their HTA process.

Access to New Medicines Horizon Scanning Advisory Board

The Scottish Government’s Access to New Medicines Horizon Scanning Advisory Board (HSAB) has recently been established to identify and consider new medicines or new indications which will potentially have a disruptive impact in Scotland on service delivery and/or Health Board finances (cost greater than £500,000 per year nationally). Its purpose is to support advance planning for the managed introduction of such medicines or indications across Scotland.

The HSAB will draw on the SMC’s horizon scanning publication, Forward Look, and relevant supplementary reports, including the Advanced Therapy Medicinal Product report. These sources will help identify medicines with potential disruptive impact that are currently in licensing and HTA pathways or pipelines and could come before the SMC for routine access within the next 18 to 24 months, if a submission to the SMC is made.

The HSAB will complement the SMC’s existing horizon scanning function by ensuring that decision makers receive early intelligence and analysis on the potential service and financial implications. This will support the necessary forward planning required, should disruptive medicines be accepted for use within the NHS in Scotland.