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Easter Closure Notice:
The NHS Wales Joint Commissioning Committee will be closed over the Easter period from Friday 3 April to Monday 6 April 2026.
Enquiries will be responded to from Tuesday 7 April 2026. Thank you for your patience.

Rare Diseases

 Asfotase alfa for treating paediatric-onset hypophosphatasia in children aged under 16 years, Policy Position Statement (PP156), November 2023.pdf (PDF, 263Kb)
 Birch bark extract for treating epidermolysis bullosa in people aged 6 months and older, Policy Position Statement (PPS284), December 2024.pdf (PDF, 398Kb)
 Burosumab for Treating X-linked Hypophosphataemia (XLH), Policy Position Statement (PPS177), January 2026.pdf (PDF, 322Kb)
 Canakinumab for treating periodic fever syndromes, TRAPS, HIDS,MKD and FMF (ages 2 and older), Policy Position Statement, (PP228), January 2022.pdf (PDF, 324Kb)
 Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 for children aged under 16 years, Policy Position Statement (PP262), November 2023.pdf (PDF, 233Kb)
 Cystic Fibrosis Adults and Young People, Service Specification (CP193), January 2012.pdf (PDF, 391Kb)
 Cystic Fibrosis Modulator therapies, Policy Position Statement, (PP198), March 2022.pdf (PDF, 354Kb)
 Drug treatment for lysosomal storage disorders (all ages), Commissioning Policy (CP55), February 2026.pdf (PDF, 625Kb)
 Givosiran for Treating Acute Hepatic Porphyria for People Aged 12 Years and Older, Policy Position Statement, (PP252), April 2023.pdf (PDF, 131Kb)
 National Acute Porphyria, Service Specification (CP166), March 2019.pdf (PDF, 274Kb)
 Sapropterin for treating hyperphenylalaninaemia (HPA) in phenylketonuria (PKU) and tetrahyderbiopterin (BH4) disorders, Policy Position Statement (PP223), January 2023 (PDF, 464Kb)
 Treatment Options for Transthyretin Amyloidosis in Adults, Policy Position Statement (PP187), September 2023.pdf (PDF, 307Kb)
 Volanesorsen for treating familial chylomicronaemia syndrome, Policy Position Statement (PPS217), August 2025.pdf (PDF, 390Kb)