Block scoping reports

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 whether or not a technology should be formally referred to NICE for evaluation. If ministers decide to refer a technology, the technology is formally referred to NICE for evaluation along with the final remit.

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

If you have any questions about this page please email Michelle Adhemar - michelle.adhemar@nice.org.uk

 

Batch 63 block scoping report

Batch 63 topic

  • Onasemnogene abeparvovec for treating spinal muscular atrophy type 1.
 

Batch 58 block scoping report

Batch 58 topic

  • 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

Batch 57 topic

  • Human alpha1-proteinase inhibitor for treating emphysema.
 

Batch 56 block scoping report

Batch 56 topic

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

Batch 55 block scoping report

Batch 55 topic

  • Burosumab for treating X-linked hypophosphataemia.
 

Batch 52 block scoping report

Batch 52 topics

  • Metreleptin for treating lipodystrophy
  • Afamelanotide for erythropoietic protoporphyria
 

Batch 51 block scoping report

Batch 51 topic

  • Velmanase alfa for treating alpha-mannosidosis
 

Batch 50 block scoping report

Batch 50 topic

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

Batch 48 block scoping report

Batch 48 topic

  • Alipogene tiparvovec for treating familial lipoprotein lipase deficiency
 

Batch 47 block scoping report

Batch 47 topic

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

Batch 44b block scoping report

Batch 44b topic

  • Migalastat for treating Fabry disease

Batch 41 block scoping report

Batch 41 topic

  • Sebelipase alfa for treating lysosomal acid lipase deficiency
 

Batch 40 block scoping report

Batch 40 topic

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

Batch 37b block scoping report

Batch 37b topic

  • Asfotase alfa for treating paediatric-onset hypophosphatasia
 

Batch 36 block scoping report

Batch 36 topic

  • Elosulfase alfa for treating mucopolysaccharidosis type IVA
 

Batch 34 block scoping report

Batch 34 topic

  • Eliglustat for treating type 1 Gaucher disease