following a full submission for an orphan medicine:
ex vivo expanded autologous human corneal epithelial cells containing stem cells (Holoclar®) is accepted for use within NHSScotland on an interim basis subject to ongoing evaluation and future reassessment.
Indication under review: Treatment of adult patients with moderate to severe limbal stem cell deficiency (defined by the presence of superficial corneal neovascularisation in at least two corneal quadrants, with central corneal involvement, and severely impaired visual acuity), unilateral or bilateral, due to physical or chemical ocular burns.
In a retrospective uncontrolled case series study, Holoclar was associated with transplant success in the majority of patients with limbal stem cell deficiency due to chemical or physical ocular burns.
This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.
Medicine details
- Medicine name:
- ex vivo expanded autologous human corneal epithelial cells containing stem cells (Holoclar)
- SMC ID:
- SMC2261
- Indication:
For the treatment of adult patients with moderate to severe limbal stem cell deficiency (defined by the presence of superficial corneal neovascularisation in at least two corneal quadrants, with central corneal involvement, and severely impaired visual acuity) due to physical or chemical ocular burns. A minimum of 1-2 mm2 of undamaged limbus is required for biopsy.
- Pharmaceutical company
- Holostem Terapie Avanzate srl
- BNF chapter
- Eye
- Submission type
- Full
- Status
- Interim acceptance
- Date advice published
- 07 September 2020