NICE Scoping Workshop Report

Date: May 7th, 2025

By Rocana Bray

Workshop Overview

Myself and fellow F.E.S.T Trustee Ellie Thomas attended the NICE Scoping Workshop in May, which was conducted as part of the approval process for Vosoritide in treating achondroplasia in children aged 4 months and over. The primary objective was to evaluate the clinical and cost-effectiveness of vosoritide within its proposed marketing authorization for treatment.

Workshop Leadership and Attendance

The workshop was chaired by Dr. Paul Arundel and included representatives from:

• BioMarin (the pharmaceutical company)

• Parent/carer groups

• Healthcare professional organizations

• Charities and advocacy groups

• Other relevant stakeholders

Evaluation Criteria

The workshop identified several key outcome measures for consideration in the evaluation:

Primary Health Outcomes:

• Changes in height and weight

• Changes in annual growth velocity

• Changes in body proportions

• Reduction in non-orthopaedic complications

• Mortality rates

• Adverse effects of treatment

• Health-related quality of life improvements

Key Consultation Questions

The workshop addressed several critical questions regarding treatment implementation:

• Identification and Diagnosis: How is achondroplasia currently identified and diagnosed in infants and young children in the UK?

• Current Treatment Pathways: Among identified cases, how many children are currently seen by the NHS for treatment of the condition?

• Practical Implementation: How is vosoritide expected to be used by children and young people in clinical practice?

• Growth Expectations: What is the expected age of growth plate closure in the general population compared to people with achondroplasia?

• Integration with Existing Care: Where does vosoritide fit within the existing treatment pathway for achondroplasia?

• Outcome Appropriateness: Are the proposed outcome measures suitable and comprehensive?

General Discussions

The workshop also facilitated broader discussions about vosoritide availability and stakeholder perspectives on making this treatment accessible within the UK healthcare system.

Personal Reflection

This scoping workshop provided a valuable opportunity for Ellie and myself to contribute to the evaluation process by sharing insights about vosoritide's potential impact on our children and the wider achondroplasia community. We were particularly able to highlight the significant improvements we had observed and the positive effects the treatment has had on day-to-day living and overall quality of life.

Next Steps

As we await the next phase of the NICE appraisal process, we want to thank all families, clinicians, and stakeholders who continue to contribute their voices, experiences, and expertise. The scoping workshop offered an important opportunity for diverse groups, including parent-led advocacy organisations like FEST, to share real-world insights into the impact of vosoritide on children with achondroplasia.

We were encouraged to see that NICE is considering not just clinical growth outcomes like height and proportions, but also broader quality of life measures, health complications, and how the treatment fits into existing NHS care pathways. These are critical to ensuring that decisions reflect what truly matters to children and families.

We understand that for many families, this process feels long and that the uncertainty can be difficult. We will continue to represent the views of families who would like the option to have access to treatments at every stage of the process and share updates as they become available.

Please subscribe below for updates.