HM Insights

Scotland's revised approach to the development and use of ultra-orphan medicines

The development, manufacture and commercialisation of medicines aimed at treating very rare diseases contains certain challenges.

One of those challenges, certainly in the United Kingdom, relates to ensuring doctors are able to access and prescribe such medicines. Prescribing guidelines and requirements are set in Scotland by the Scottish Medicines Consortium (SMC). In Scotland, the Scottish Government's Chief Medical Directorate also has a role.


New guidance on assessment of medicines

The 1st of May 2019 saw the release of guidance by that Directorate relating to the assessment of medicines for rare disease. The guidance is applicable to health boards, clinicians, pharmacists, patient groups and pharmaceutical companies. As such, those developing, manufacturing and/or commercialising such drugs are advised to make themselves aware of the content of the guidance.

To assist in this awareness, we summarise below some of the key points.

  • The guidance addresses Scotland's new approach to the assessment of these medicines, referred to as ultra-orphan medicines, introduced in October 2018, and with applications for new medicines possible from April 2019.
  • If a drug meets the definition of an ultra-orphan medicine and undergoes an in-depth assessment of clinical and cost-effectiveness by the SMC, then it can be made available on the NHS for up to three years while further evidence is generated.
  • Following the end of this period, evidence is further reviewed, and the SMC will make a final decision.
  • To meet the SMC definition of an ultra-orphan medicine, the condition to be treated via the medicine must have a prevalence of 1 in 50,000 or less in Scotland, the medicine must have a European Medicines Agency (EMA) orphan designation for the condition (which is maintained at time of marketing authorisation for the drug), the condition must be chronic and severely disabling, and the condition must require highly specialised management.
  • As regards references to the condition, the SMC uses the description of the condition as set out within the EMA's Orphan Maintenance Assessment Report (OMAR) as a reference, and where the OMAR isn't available the description within the original orphan designation.
    To qualify through this pathway the SMC have to validate the drug as an ultra-orphan, and the pharmaceutical company has to make a full submission for initial assessment meeting SMC requirements.
  • The pharmaceutical company further has to offer a PAS (Patient Access Scheme) in line with standard terms considered acceptable by the PASAG (Patient Access Scheme Assessment Group).
  • The company will also have to engage with data collection arrangements meeting evidence generation requirements.

A copy of the full guidance is available here.

Get in touch

If you would like to find out more about how this guidance might affect your business, please get in touch.