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
Topic
- Onasemnogene abeparvovec for treating spinal muscular atrophy type 1.
Batch 58 block scoping report
Topics
- Inotersen for treating hereditary transthyretin-related amyloidosis.
- Patisiran for treating hereditary transthyretin-related amyloidosis.
- Volanesorsen for treating familial chylomicronaemia syndrome.
Documents
Batch 57 block scoping report
Topics
- Human alpha1-proteinase inhibitor for treating emphysema.
Documents
Batch 56 block scoping report
Topics
- Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations.
Documents
Batch 55 block scoping report
Topics
- Burosumab for treating X-linked hypophosphataemia.
Documents
Batch 52 block scoping report
Topics
- Metreleptin for treating lipodystrophy
- Afamelanotide for erythropoietic protoporphyria
Documents
Batch 51 block scoping report
Topics
- Velmanase alfa for treating alpha-mannosidosis
Documents
Batch 50 block scoping report
Topics
- Eteplirsen for treating Duchenne muscular dystrophy
- Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2
Documents
Batch 48 block scoping report
Topics
- Alipogene tiparvovec for treating familial lipoprotein lipase deficiency
Documents
Batch 47 block scoping report
Topics
- GSK2696273 for treating severe combined immunodeficiency caused by adenosine deaminase deficiency
Documents
Batch 44b block scoping report
Topics
- Migalastat for treating Fabry disease
Documents
Batch 41 block scoping report
Topics
- Sebelipase alfa for treating lysosomal acid lipase deficiency
Documents
Batch 40 block scoping report
Topics
- Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene
Documents
Batch 37b block scoping report
Topics
- Asfotase alfa for treating paediatric-onset hypophosphatasia
Documents
Batch 36 block scoping report
Topics
- Elosulfase alfa for treating mucopolysaccharidosis type IVA
Documents
Batch 34 block scoping report
Topics
- Eliglustat for treating type 1 Gaucher disease