Media release: SMC decisions May 2025

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The Scottish Medicines Consortium (SMC), which advises on newly licensed medicines for use by NHSScotland, has today (Monday, May 12) published advice on six medicines.

An ultra-orphan initial assessment report has been published for exagamglogene autotemcel (Casgevy®) a treatment for transfusion-dependent beta-thalassemia, a rare genetic condition that affects the production of haemoglobin. It is for patients aged 12 and over, where a stem cell transplant is appropriate but there is no suitable donor available. The Scottish Government will confirm when it is available for prescribing and further evidence will be gathered. Following this SMC will review it and decide on routine availability in NHSScotland.

Sodium thiosulfate (Pedmarqsi®) was accepted to reduce the risk of hearing loss, caused by cisplatin-based chemotherapy, when it is used to treat solid tumours that have not spread in children aged one month to 18 years.

Erdafitinib (Balversa®) was accepted to treat adults with urothelial carcinoma, a type of bladder cancer, that has spread to other parts of the body or can’t be removed by surgery, in patients who have received previous treatment.

Mepolizumab (Nucala®) was accepted, for use together with other medicines, to treat a type of asthma called eosinophilic asthma, in adults and children aged six and over. It is used where the asthma is severe and hasn’t been well controlled with previous treatments.

Donanemab (Kisunla®) was not recommended to treat the early stages of Alzheimer’s disease in adults who carry one or no copies of a gene called ApoE4.

Fruquintinib (Fruzaqla®) was not recommended as a treatment for adults with advanced colorectal cancer who have already received at least two lines of treatment.

SMC Chair Dr Scott Muir said:

Ends

Notes to editor

1. The Scottish Medicines Consortium (SMC) is the national source of advice on the clinical and cost-effectiveness of all new medicines for NHSScotland. Our aim is to ensure that people in Scotland have timely access to beneficial new medicines.
2. We are part of Healthcare Improvement Scotland. Our committee is made up of clinicians, pharmacists, NHS board representatives, the pharmaceutical industry and the public. Most of the clinicians have a direct role in patient care, while our three volunteer public partners ensure the views of the Scottish public are taken into account during decision making. This wide mixture of backgrounds ensures decisions are made from a broad perspective.
3. Members of the SMC Committee make their decisions based on a broad range of evidence in order to help health professionals deliver the best possible care within the finite resources available. They consider detailed evidence presented by pharmaceutical companies, patient groups and clinicians in order to decide which medicines provide value for money for NHSScotland.
4. When we talk about value for money or cost effectiveness, this does not just mean how much a medicine costs to buy, but the wider costs and benefits associated with it being regularly used to treat those with a particular condition. The NHS does not have unlimited resources and if we accept a medicine where the benefits are not clear it may mean other patients lose out.
5. When considering a new medicine we look at:

• how well the medicine works
• which patients with the particular condition would benefit from it
• whether it is as good as or better than the medicines already used to treat the particular condition, and
• whether it is good value for money.

For more information on how we make our decisions, go to the SMC website, https://www.scottishmedicines.org.uk/how-we-decide/

1. Changes to the way we assess medicines for end of life and rare conditions (https://www.scottishmedicines.org.uk/how-we-decide/pace/) have allowed our committee additional flexibility in decision making. These changes include the option of a Patient and Clinician Engagement (PACE) meeting which gives patient groups and clinicians the opportunity to provide additional evidence to the committee.
2. A new approach to the assessment of medicines which treat extremely rare conditions has been introduced (https://www.scottishmedicines.org.uk/how-we-decide/ultra-orphan-medicines-for-extremely-rare-conditions/) in line with the Scottish Government announcement in June 2018 on a new pathway (https://news.gov.scot/news/treatments-for-rare-conditions)
3. SMC can make the following decisions on a medicine:

• accepted
• accepted with a restriction(s) (for example, the medicine can only be accepted in a particular group of patients with the condition. This typically occurs because the company has requested this explicitly in the submission)
• accepted on an interim basis, or
• not recommended

SMC can accept some medicines on an interim basis subject to ongoing evaluation and reassessment. You can find out more about this on our website https://www.scottishmedicines.org.uk/how-we-decide/interim-acceptance-decision-option/

SMC may be unable to accept a medicine for use in Scotland if the committee is not satisfied that the benefits of the medicine offer value for money for patients and the NHS or where there is a high degree of uncertainty about the clinical benefits of the medicine. You can find out more about the reasons we may not recommend medicines on our website: https://www.scottishmedicines.org.uk/how-we-decide/

1. For medicines that have not been recommended, SMC welcomes a resubmission from the company at any time with new clinical and/or economic evidence.
2. When a medicine has not been recommended by SMC, all NHS boards have procedures in place to consider individual requests when a doctor feels the medicine would be right for a particular patient.
3. For further information and to view the complete advice for the medicines detailed above, visit our website at: www.scottishmedicines.org.uk.
4. For further information on ILAP, please visit the MHRA website https://www.gov.uk/guidance/innovative-licensing-and-access-pathway