|Assessment Status||Rapid Review complete|
|Indication||For the treatment of inborn errors in primary bile acid synthesis due to 3β-Hydroxy-Δ5-C27-steroid oxidoreductase deficiency or Δ4-3-Oxosteroid-5β-reductase deficiency in infants, children and adolescents aged one month to 18 years, and adults.|
|Rapid review commissioned||13/12/2021|
|Rapid review completed||12/01/2022|
|Rapid review outcome||A full HTA is not recommended. The NCPE recommends that cholic acid (Orphacol®) not be considered for reimbursement at the submitted price*.|
*This recommendation should be considered while also having regard to the criteria specified in the Health (Pricing and Supply of Medical Goods) Act 2013.
The HSE has approved reimbursement following confidential price negotiations, December 2022.