№ lp_2_3_20064
File format: docx Character count: 21997 File size: 52 KB
Official summary of an appeal panel hearing concerning NICE’s final draft guidance to the NHS on the use of olipudase alfa for ASMD types AB and B, outlining the grounds of appeal, representations made by stakeholders, and the panel’s consideration of procedural fairness and evidentiary issues.
Year: 2024
Date of hearing: 24 May 2024
Country: England
Document type: Appeal hearing decision summary
Subject: Olipudase alfa for treating Niemann-Pick disease types AB and B (ASMD)
Institution: National Institute for Health and Care Excellence
Appeal body: Highly Specialised Technologies Appeal Panel
Appellant: Niemann-Pick UK
Legal adviser: DAC Beachcroft LLP
Grounds of appeal: Ground 1(a) – failure to act fairly; Ground 2 – unreasonableness in light of the evidence
Technology appraisal identifier: ID3913
Therapeutic area: Acid sphingomyelinase deficiency (ASMD)
Target population: People with ASMD types AB and B in England
Price: 8 / 10 USD
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Appeal Against Final Draft Guidance on Olipudase Alfa for Treating Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type AB and Type B [ID3913], 19 March 2024
Date: 19 March 2024
Organisation: Niemann-Pick UK (NPUK)
Address: Suite 2, Vermont House, Concord, Washington NE37 2SQ
Addressee: Dr Mark Chakravarty, Lead Non-executive Director for Appeals
Institution: National Institute for Health and Care Excellence
Subject: Appeal of Final Draft Guidance decision
Technology Appraised: Olipudase alfa
Indication: Acid sphingomyelinase deficiency (Niemann-Pick disease) type AB and type B
Reference ID: ID3913
Grounds for Appeal: Failure to act fairly; Unreasonable recommendation in light of evidence
Document Type: Formal appeal letter
Geographical Scope: United Kingdom
Cited Studies: Raebel et al., Orphanet Journal of Rare Diseases (2024); Fraser et al.
Appeal Against Final Draft Guidance on Olipudase Alfa for Treating Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type AB and Type B [ID3913], 19 March 2024
Date: 19 March 2024
Organisation: Niemann-Pick UK (NPUK)
Address: Suite 2, Vermont House, Concord, Washington NE37 2SQ
Addressee: Dr Mark Chakravarty, Lead Non-executive Director for Appeals
Institution: National Institute for Health and Care Excellence
Subject: Appeal of Final Draft Guidance decision
Technology Appraised: Olipudase alfa
Indication: Acid sphingomyelinase deficiency (Niemann-Pick disease) type AB and type B
Reference ID: ID3913
Grounds for Appeal: Failure to act fairly; Unreasonable recommendation in light of evidence
Document Type: Formal appeal letter
Geographical Scope: United Kingdom
Cited Studies: Raebel et al., Orphanet Journal of Rare Diseases (2024); Fraser et al.
Appeal Against Final Draft Guidance on Olipudase Alfa for Treating Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type AB and Type B [ID3913], 19 March 2024
Date: 19 March 2024
Organisation: Niemann-Pick UK (NPUK)
Address: Suite 2, Vermont House, Concord, Washington NE37 2SQ
Addressee: Dr Mark Chakravarty, Lead Non-executive Director for Appeals
Institution: National Institute for Health and Care Excellence
Subject: Appeal of Final Draft Guidance decision
Technology Appraised: Olipudase alfa
Indication: Acid sphingomyelinase deficiency (Niemann-Pick disease) type AB and type B
Reference ID: ID3913
Grounds for Appeal: Failure to act fairly; Unreasonable recommendation in light of evidence
Document Type: Formal appeal letter
Geographical Scope: United Kingdom
Cited Studies: Raebel et al., Orphanet Journal of Rare Diseases (2024); Fraser et al.
Topics: Olipudase Niemann-Pick ID3913