Highly specialised technology evaluation block scoping reports

Content under review

We're updating this page following the publication of our new methods, processes and topic selection manuals. Our previous manuals still apply to evaluations that started before 1 February 2022. The new manuals apply to evaluations that began after this date.

Listed below are the highly specialised technology evaluation block scoping reports which have been submitted to the Department of Health and Social Care.

These reports summarise the results of consultation and scoping workshop discussions. This information helps ministers to decide if a technology should be formally referred to us for evaluation.

If ministers decide to refer a technology, the technology is formally referred to NICE for appraisal along with the final remit.

Please note that any 'commercial in confidence' information has been removed.

Contact Michelle Adhemar at michelle.adhemar@nice.org.uk if you have any questions about these reports.

Batch 63 block scoping report


  • Onasemnogene abeparvovec for treating spinal muscular atrophy type 1.

Batch 58 block scoping report


  • Inotersen for treating hereditary transthyretin-related amyloidosis.
  • Patisiran for treating hereditary transthyretin-related amyloidosis.
  • Volanesorsen for treating familial chylomicronaemia syndrome.

Batch 57 block scoping report


  • Human alpha1-proteinase inhibitor for treating emphysema.

Batch 56 block scoping report


  • Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations.

Batch 55 block scoping report


  • Burosumab for treating X-linked hypophosphataemia.

Batch 52 block scoping report


  • Metreleptin for treating lipodystrophy
  • Afamelanotide for erythropoietic protoporphyria

Batch 51 block scoping report


  • Velmanase alfa for treating alpha-mannosidosis

Batch 50 block scoping report


  • Eteplirsen for treating Duchenne muscular dystrophy
  • Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2

Batch 48 block scoping report


  • Alipogene tiparvovec for treating familial lipoprotein lipase deficiency

Batch 47 block scoping report


  • GSK2696273 for treating severe combined immunodeficiency caused by adenosine deaminase deficiency

Batch 44b block scoping report


  • Migalastat for treating Fabry disease

Batch 41 block scoping report


  • Sebelipase alfa for treating lysosomal acid lipase deficiency

Batch 40 block scoping report


  • Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene

Batch 37b block scoping report


  • Asfotase alfa for treating paediatric-onset hypophosphatasia

Batch 36 block scoping report


  • Elosulfase alfa for treating mucopolysaccharidosis type IVA

Batch 34 block scoping report


  • Eliglustat for treating type 1 Gaucher disease